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Trial record 87 of 530 for:    VANCOMYCIN

A Study to Investigate the Safety and Efficacy of Fidaxomicin (Oral Suspension or Tablets) and Vancomycin (Oral Liquid or Capsules) in Pediatric Subjects With Clostridium Difficile-associated Diarrhea (CDAD) (SUNSHINE)

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ClinicalTrials.gov Identifier: NCT02218372
Recruitment Status : Completed
First Posted : August 18, 2014
Results First Posted : December 7, 2018
Last Update Posted : February 15, 2019
Sponsor:
Collaborator:
Merck Sharp & Dohme Corp.
Information provided by (Responsible Party):
Astellas Pharma Inc ( Astellas Pharma Europe B.V. )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Single (Investigator);   Primary Purpose: Treatment
Condition Clostridium Difficile-associated Diarrhea (CDAD)
Interventions Drug: Fidaxomicin oral suspension
Drug: Fidaxomicin tablets
Drug: Vancomycin oral liquid
Drug: Vancomycin capsules
Enrollment 148
Recruitment Details Pediatric participants with clostridium difficile-associated diarrhea (CDAD) were enrolled in this multicenter study.
Pre-assignment Details Eligible participants who met inclusion criteria and none of the exclusion criteria were enrolled, 159 participants were assessed for eligibility of whom 148 were randomized. Participants were randomized to either fidaxomicin or vancomycin in a 2:1 ratio, stratified by age group.
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days. Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
Period Title: Overall Study
Started 100 48
Treated 98 44
Completed 95 42
Not Completed 5 6
Reason Not Completed
Adverse Event             1             1
Miscellaneous             2             1
Randomized but did not receive treatment             2             3
Withdrawal by Parent/Guardian             0             1
Arm/Group Title Fidaxomicin Vancomycin Total
Hide Arm/Group Description Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days. Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days. Total of all reporting groups
Overall Number of Baseline Participants 100 48 148
Hide Baseline Analysis Population Description
The baseline analysis set consisted of the Intent-to-treat (ITT) population. The ITT analysis set consisted of all randomized participants, irrespective of a participant having received a study drug (fidaxomicin or vancomycin) or not.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Months
Number Analyzed 100 participants 48 participants 148 participants
79.7  (61.8) 77.5  (59.2) 79  (60.8)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 100 participants 48 participants 148 participants
Female
41
  41.0%
21
  43.8%
62
  41.9%
Male
59
  59.0%
27
  56.3%
86
  58.1%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 100 participants 48 participants 148 participants
White
83
  83.0%
36
  75.0%
119
  80.4%
Black or African American
6
   6.0%
5
  10.4%
11
   7.4%
Asian
2
   2.0%
0
   0.0%
2
   1.4%
Other
4
   4.0%
1
   2.1%
5
   3.4%
Missing
5
   5.0%
6
  12.5%
11
   7.4%
Ethnicity  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 100 participants 48 participants 148 participants
Hispanic or Latino
12
  12.0%
5
  10.4%
17
  11.5%
Not Hispanic or Latino
82
  82.0%
37
  77.1%
119
  80.4%
Missing
6
   6.0%
6
  12.5%
12
   8.1%
1.Primary Outcome
Title Percentage of Participants With Confirmed Clinical Response (CCR) at End of Treatment (EOT) +2 Days
Hide Description Initial clinical response (ICR) for ages from birth to < 2 years was defined as absence of watery diarrhea for 2 consecutive treatment days, remaining well until study drug discontinuation. ICR for ages ≥ 2 years to < 18 years was defined as improvement in number and character of bowel movements as determined by < 3 unformed bowel movements (UBMs) per day for 2 consecutive treatment days, remaining well until study drug discontinuation. CCR was defined for both age groups as not requiring further CDAD therapy within 2 days after study drug completion, and was reported with a positive (Yes) or negative (No) outcome. Resolution of diarrhea was assessed during interviews of participant/parent/legal guardian, supplemented by review of personal records (if hospitalized) and checked for presence of watery diarrhea (ages from birth to < 2 years) or number of UBMs (for ages ≥ 2 years to < 18 years).
Time Frame Up to day 12
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the full analysis set (FAS) which consisted of all randomized participants who received at least 1 dose of study drug. In the FAS, participants were allocated to the treatment arm corresponding to the study medication that the participant was randomized to (treatment allocation as randomized).
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description:
Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.
Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
Overall Number of Participants Analyzed 98 44
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
77.6
(68.0 to 85.4)
70.5
(54.8 to 83.2)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Vancomycin
Comments Adjusted difference of CCR at EOT + 2 Days. Adjusted treatment difference of proportions was calculated using a stratified Cochran-Mantel-Haenszel (CMH) method. Newcombe 95% confidence intervals (CIs) presented for adjusted treatment difference.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter adjusted treatment difference
Estimated Value 7.5
Confidence Interval (2-Sided) 95%
-7.4 to 23.9
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Percentage of Participants With Sustained Clinical Response (SCR) at EOT +9 Days
Hide Description SCR at EOT + 9 days was defined as CCR (EOT + 2 days) without CDAD recurrence until assessment at EOT +9 days during the follow-up period. Recurrence for ages from birth to < 2 years was defined as re-establishment of watery diarrhea after CCR to an extent that was greater than that noted on the last day of study drug with positive direct or indirect testing for the presence of toxigenic Clostridium difficile (C. difficile) in stool and that, in the investigator's opinion, required retreatment with CDAD anti-infective therapy. Recurrence for ages ≥ 2 years < 18 years was defined as re-establishment of diarrhea after CCR to an extent (as measured by the frequency of UBMs) that was greater than that noted on the last day of study drug with positive direct or indirect testing for the presence of toxigenic C. difficile in stool and that, in the investigator's opinion, required retreatment with CDAD anti-infective therapy.
Time Frame Up to day 19
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS (participants with CCR at EOT +2 days).
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description:
Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.
Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
Overall Number of Participants Analyzed 76 31
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
94.7
(87.1 to 98.5)
77.4
(58.9 to 90.4)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Vancomycin
Comments Adjusted difference of SCR at EOT +9 days. Adjusted treatment difference of proportions was calculated using a stratified CMH method. Newcombe 95% CIs presented for adjusted treatment difference.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter adjusted treatment difference
Estimated Value 16.3
Confidence Interval (2-Sided) 95%
1.8 to 34.2
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Percentage of Participants With Global Cure (GC) at EOT +9 Days
Hide Description GC was reported as a positive (Yes) or negative (No) outcome and was calculated using SCR and ICR/CCR values according to the following conditions: ● if ICR/CCR=Yes and SCR=Yes, then Global Cure was Yes. ● if ICR/CCR=Yes and SCR =No, then Global Cure was No. ● if ICR/CCR=No (SCR not assessed), then Global Cure was No. ● if ICR/CCR=Missing (SCR not assessed), then Global Cure was set to No. No multiple imputation method (MI) was used for global cure at EOT + 9 days.
Time Frame Up to day 19
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS.
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description:
Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.
Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
Overall Number of Participants Analyzed 98 44
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
75.5
(65.8 to 83.6)
54.5
(38.8 to 69.6)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Vancomycin
Comments Adjusted difference of GC at EOT +9 days. Adjusted treatment difference of rates was calculated using a stratified CMH method. Newcombe 95% CIs presented for adjusted treatment difference.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter adjusted treatment difference
Estimated Value 21.3
Confidence Interval (2-Sided) 95%
4.5 to 37.7
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Percentage of Participants With Recurrence of CDAD at EOT +9 Days
Hide Description Recurrence for ages from birth to < 2 years was defined as re-establishment of watery diarrhea after CCR to an extent that was greater than that noted on the last day of study drug with positive direct or indirect testing for the presence of toxigenic C. difficile in stool and that, in the investigator's opinion, required retreatment with CDAD anti-infective therapy. Recurrence for ages ≥ 2 years < 18 years was defined as re-establishment of diarrhea after CCR to an extent (as measured by the frequency of UBMs) that was greater than that noted on the last day of study drug with positive direct or indirect testing for the presence of toxigenic C. difficile in stool and that, in the investigator's opinion, required retreatment with CDAD anti-infective therapy.
Time Frame Up to day 19
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS (participants with CCR at EOT +2 days).
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description:
Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.
Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
Overall Number of Participants Analyzed 76 31
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
5.3
(1.5 to 12.9)
22.6
(9.6 to 41.1)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Vancomycin
Comments Adjusted difference of CDAD recurrence at EOT +9 days. Adjusted treatment difference of proportions was calculated using a stratified CMH method. Newcombe 95% CIs presented for adjusted treatment difference.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter adjusted treatment difference
Estimated Value -16.3
Confidence Interval (2-Sided) 95%
-34.2 to -1.8
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Percentage of Participants With SCR at EOT +16 Days
Hide Description SCR at EOT + 16 days was defined as CCR (EOT + 2 days) without CDAD recurrence until assessment at EOT + 16 days during the follow-up period. Recurrence for ages from birth to < 2 years was defined as re-establishment of watery diarrhea after CCR to an extent that was greater than that noted on the last day of study drug with positive direct or indirect testing for the presence of toxigenic C. difficile in stool and that, in the investigator's opinion, required retreatment with CDAD anti-infective therapy. Recurrence for ages ≥ 2 years < 18 years was defined as re-establishment of diarrhea after CCR to an extent (as measured by the frequency of UBMs) that was greater than that noted on the last day of study drug with positive direct or indirect testing for the presence of toxigenic C. difficile in stool and that, in the investigator's opinion, required retreatment with CDAD anti-infective therapy.
Time Frame Up to day 26
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS (participants with CCR at EOT +2 days).
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description:
Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.
Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
Overall Number of Participants Analyzed 76 31
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
89.5
(80.3 to 95.3)
71.0
(52.0 to 85.8)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Vancomycin
Comments Adjusted difference of SCR at EOT +16 days. Adjusted treatment difference of proportions was calculated using a stratified CMH method. Newcombe 95% CIs presented for adjusted treatment difference.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter adjusted treatment difference
Estimated Value 17.2
Confidence Interval (2-Sided) 95%
1.9 to 35.6
Estimation Comments [Not Specified]
6.Secondary Outcome
Title Percentage of Participants With GC at EOT +16 Days
Hide Description GC was reported as a positive (Yes) or negative (No) outcome and was calculated using SCR and ICR/CCR values according to the following conditions: ● if ICR/CCR=Yes and SCR=Yes, then Global Cure was Yes. ● if ICR/CCR=Yes and SCR =No, then Global Cure was No. ● if ICR/CCR=No (SCR not assessed), then Global Cure was No. ● if ICR/CCR=Missing (SCR not assessed), then Global Cure was set to No. No multiple imputation method (MI) was used for global cure at EOT + 16 days.
Time Frame Up to day 26
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS.
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description:
Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.
Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
Overall Number of Participants Analyzed 98 44
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
71.4
(61.4 to 80.1)
52.3
(36.7 to 67.5)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Vancomycin
Comments Adjusted difference of GC at EOT +16 days. Adjusted treatment difference of rates was calculated using a stratified CMH method. Newcombe 95% CIs presented for adjusted treatment difference.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter adjusted treatment difference
Estimated Value 19.4
Confidence Interval (2-Sided) 95%
2.3 to 35.9
Estimation Comments [Not Specified]
7.Secondary Outcome
Title Percentage of Participants With Recurrence of CDAD at EOT +16 Days
Hide Description Recurrence for ages from birth to < 2 years was defined as re-establishment of watery diarrhea after CCR to an extent that was greater than that noted on the last day of study drug with positive direct or indirect testing for the presence of toxigenic C. difficile in stool and that, in the investigator's opinion, required retreatment with CDAD anti-infective therapy. Recurrence for ages ≥ 2 years < 18 years was defined as re-establishment of diarrhea after CCR to an extent (as measured by the frequency of UBMs) that was greater than that noted on the last day of study drug with positive direct or indirect testing for the presence of toxigenic C. difficile in stool and that, in the investigator's opinion, required retreatment with CDAD anti-infective therapy.
Time Frame Up to day 26
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS (participants with CCR at EOT +2 days).
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description:
Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.
Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
Overall Number of Participants Analyzed 76 31
Median (95% Confidence Interval)
Unit of Measure: percentage of participants
7.9
(3.0 to 16.4)
25.8
(11.9 to 44.6)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Vancomycin
Comments Adjusted difference of CDAD Recurrence at EOT +16 days. Adjusted treatment difference of proportions was calculated using a stratified CMH method. Newcombe 95% CIs presented for adjusted treatment difference.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter adjusted treatment difference
Estimated Value -17.2
Confidence Interval (2-Sided) 95%
-35.6 to -1.9
Estimation Comments [Not Specified]
8.Secondary Outcome
Title Percentage of Participants With SCR at EOT +23 Days
Hide Description SCR at EOT + 23 days was defined as CCR (EOT + 2 days) without CDAD recurrence until assessment at EOT + 16 days during the follow-up period. Recurrence for ages from birth to < 2 years was defined as re-establishment of watery diarrhea after CCR to an extent that was greater than that noted on the last day of study drug with positive direct or indirect testing for the presence of toxigenic C. difficile in stool and that, in the investigator's opinion, required retreatment with CDAD anti-infective therapy. Recurrence for ages ≥ 2 years < 18 years was defined as re-establishment of diarrhea after CCR to an extent (as measured by the frequency of UBMs) that was greater than that noted on the last day of study drug with positive direct or indirect testing for the presence of toxigenic C. difficile in stool and that, in the investigator's opinion, required retreatment with CDAD anti-infective therapy.
Time Frame Up to day 33
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS (participants with CCR at EOT +2 days).
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description:
Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.
Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
Overall Number of Participants Analyzed 76 31
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
85.5
(75.6 to 92.5)
71.0
(52.0 to 85.8)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Vancomycin
Comments Adjusted difference of SCR at EOT +23 days. Adjusted treatment difference of proportions was calculated using a stratified CMH method. Newcombe 95% CIs presented for adjusted treatment difference.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter adjusted treatment difference
Estimated Value 15.8
Confidence Interval (2-Sided) 95%
-0.5 to 34.5
Estimation Comments [Not Specified]
9.Secondary Outcome
Title Percentage of Participants With GC at EOT +23 Days
Hide Description GC was reported as a positive (Yes) or negative (No) outcome and was calculated using SCR and ICR/CCR values according to the following conditions: ● if ICR/CCR=Yes and SCR=Yes, then Global Cure was Yes. ● if ICR/CCR=Yes and SCR =No, then Global Cure was No. ● if ICR/CCR=No (SCR not assessed), then Global Cure was No. ● if ICR/CCR=Missing (SCR not assessed), then Global Cure was set to No. No multiple imputation method (MI) was used for global cure at EOT + 23 days.
Time Frame Up to day 33
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS.
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description:
Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.
Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
Overall Number of Participants Analyzed 98 44
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
68.4
(58.2 to 77.4)
50.0
(34.6 to 65.4)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Vancomycin
Comments Adjusted difference of GC at EOT +23 days. Adjusted treatment difference of rates was calculated using a stratified CMH method. Newcombe 95% CIs presented for adjusted treatment difference.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter adjusted treatment difference
Estimated Value 18.8
Confidence Interval (2-Sided) 95%
1.5 to 35.3
Estimation Comments [Not Specified]
10.Secondary Outcome
Title Percentage of Participants With Recurrence of CDAD at EOT +23 Days
Hide Description Recurrence for ages from birth to < 2 years was defined as re-establishment of watery diarrhea after CCR to an extent that was greater than that noted on the last day of study drug with positive direct or indirect testing for the presence of toxigenic C. difficile in stool and that, in the investigator's opinion, required retreatment with CDAD anti-infective therapy. Recurrence for ages ≥ 2 years < 18 years was defined as re-establishment of diarrhea after CCR to an extent (as measured by the frequency of UBMs) that was greater than that noted on the last day of study drug with positive direct or indirect testing for the presence of toxigenic C. difficile in stool and that, in the investigator's opinion, required retreatment with CDAD anti-infective therapy.
Time Frame Up to day 33
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS (participants with CCR at EOT +2 days).
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description:
Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.
Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
Overall Number of Participants Analyzed 76 31
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
11.8
(5.6 to 21.3)
29.0
(14.2 to 48.0)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Vancomycin
Comments Adjusted difference of CDAD Recurrence at EOT +23 days. Adjusted treatment difference of proportions was calculated using a stratified CMH method. Newcombe 95% CIs presented for adjusted treatment difference.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter adjusted treatment difference
Estimated Value -15.8
Confidence Interval (2-Sided) 95%
-34.5 to 0.5
Estimation Comments [Not Specified]
11.Secondary Outcome
Title Percentage of Participants With SCR at End of Study (EOS) (EOT +30 Days)
Hide Description SCR at EOS was defined as CCR (EOT + 2 days) without CDAD recurrence until assessment at EOS (EOT + 30 days) during the follow-up period. Recurrence for ages from birth to < 2 years was defined as re-establishment of watery diarrhea after CCR to an extent that was greater than that noted on the last day of study drug with positive direct or indirect testing for the presence of toxigenic C. difficile in stool and that, in the investigator's opinion, required retreatment with CDAD anti-infective therapy. Recurrence for ages ≥ 2 years < 18 years was defined as re-establishment of diarrhea after CCR to an extent (as measured by the frequency of UBMs) that was greater than that noted on the last day of study drug with positive direct or indirect testing for the presence of toxigenic C. difficile in stool and that, in the investigator's opinion, required retreatment with CDAD anti-infective therapy.
Time Frame Up to day 40
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The analysis population consisted of the FAS (participants with CCR at EOT +2 days).
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description:
Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.
Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
Overall Number of Participants Analyzed 76 31
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
85.5
(75.6 to 92.5)
71.0
(52.0 to 85.8)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Vancomycin
Comments Adjusted difference of SCR at EOS (EOT +30 days). Adjusted treatment difference of proportions was calculated using a stratified CMH method. Newcombe 95% CIs presented for adjusted treatment difference.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter adjusted treatment difference
Estimated Value 15.8
Confidence Interval (2-Sided) 95%
-0.5 to 34.5
Estimation Comments [Not Specified]
12.Secondary Outcome
Title Percentage of Participants With GC at EOS (EOT +30 Days)
Hide Description GC was reported as a positive (Yes) or negative (No) outcome and was calculated using SCR and ICR/CCR values according to the following conditions: ● if ICR/CCR=Yes and SCR=Yes, then Global Cure was Yes. ● if ICR/CCR=Yes and SCR =No, then Global Cure was No. ● if ICR/CCR=No (SCR not assessed), then Global Cure was No. ● if ICR/CCR=Missing (SCR not assessed), then Global Cure was set to No. Global Cure at EOT +30 days was derived using MI in case ICR/CCR=Missing (SCR not assessed) following Rubin’s multiple imputation method.
Time Frame Up to day 40
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Hide Analysis Population Description
The analysis population consisted of the FAS.
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description:
Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.
Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
Overall Number of Participants Analyzed 98 44
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
68.4
(58.2 to 77.4)
50.0
(34.6 to 65.4)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Vancomycin
Comments Adjusted difference of GC at EOS (EOT +30 days). Adjusted treatment difference of rates was calculated using a stratified CMH method. Newcombe 95% CIs presented for adjusted treatment difference.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter adjusted treatment difference
Estimated Value 18.8
Confidence Interval (2-Sided) 95%
1.5 to 35.3
Estimation Comments [Not Specified]
13.Secondary Outcome
Title Percentage of Participants With Recurrence of CDAD at EOS (EOT +30 Days)
Hide Description Recurrence for ages from birth to < 2 years was defined as re-establishment of watery diarrhea after CCR to an extent that was greater than that noted on the last day of study drug with positive direct or indirect testing for the presence of toxigenic C. difficile in stool and that, in the investigator's opinion, required retreatment with CDAD anti-infective therapy. Recurrence for ages ≥ 2 years < 18 years was defined as re-establishment of diarrhea after CCR to an extent (as measured by the frequency of UBMs) that was greater than that noted on the last day of study drug with positive direct or indirect testing for the presence of toxigenic C. difficile in stool and that, in the investigator's opinion, required retreatment with CDAD anti-infective therapy.
Time Frame Up to day 40
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Hide Analysis Population Description
The analysis population consisted of the FAS (participants with CCR at EOT +2 days).
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description:
Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.
Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
Overall Number of Participants Analyzed 76 31
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
11.8
(5.6 to 21.3)
29.0
(14.2 to 48.0)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Vancomycin
Comments Adjusted difference of CDAD recurrence at EOS/EOT +30 days. Adjusted treatment difference of proportions was calculated using a stratified CMH method. Newcombe 95% CIs presented for adjusted treatment difference.
Type of Statistical Test Other
Comments Newcombe 95% CIs presented for adjusted treatment difference.
Method of Estimation Estimation Parameter adjusted treatment difference
Estimated Value -15.8
Confidence Interval (2-Sided) 95%
-34.5 to 0.5
Estimation Comments [Not Specified]
14.Secondary Outcome
Title Time to Resolution of Diarrhea (TTROD)
Hide Description TTROD for ages from birth < 2 years was defined as time elapsing (hours rounded up from minutes > 30) from treatment start (time of first study drug dose) to diarrhea resolution (time of last episode of watery diarrhea the day prior to the first of 2 consecutive days without watery diarrhea sustained through EOT). TTROD for ages ≥ 2 years to < 18 years was defined as time elapsing (hours rounded up from minutes > 30) from treatment start (time of first dose) to diarrhea resolution (time of the last UBM the day prior to the first of 2 consecutive days of < 3 UBMs sustained through EOT). TTROD by Kaplan-Meier Method. Those who completed treatment but did not show diarrhea resolution until EOT were censored at Day 10/240 hours. Those who did not complete treatment, discontinued earlier but did not show diarrhea resolution until disc. day were censored at disc. (days converted to hours). Those whose diarrhea did not continue after first dose were included with a TTROD of 1 hour.
Time Frame Up to day 10
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The analysis population consisted of the FAS.
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description:
Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.
Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
Overall Number of Participants Analyzed 98 44
Median (95% Confidence Interval)
Unit of Measure: hours
58
(29.0 to 122.0)
97
(42.0 to 146.0)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Vancomycin
Comments Time to resolution of diarrhea.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.579
Comments [Not Specified]
Method Log Rank
Comments [Not Specified]
15.Secondary Outcome
Title Time to Recurrence of CDAD for Participants With CCR at EOT +2 Days
Hide Description Time to recurrence was defined as the time (days) from CCR until the onset of recurrence. Time to recurrence of CDAD by Kaplan-Meier Method. Data for median was estimated and the 95% CI could not be estimated due to low event rate. Data not estimable denoted as NA. Participants with CCR at EOT+2 days, who completed the follow-up period but did not experience a recurrence of CDAD were censored at EOT+30 days and those who did not complete the follow-up period and discontinued during this period and did not experience a recurrence of CDAD were censored at day of discontinuation.
Time Frame Up to day 40
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Hide Analysis Population Description
The analysis population consisted of the FAS (participants with CCR at EOT +2 days).
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description:
Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.
Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
Overall Number of Participants Analyzed 76 31
Median (95% Confidence Interval)
Unit of Measure: days
25 [1] 
(NA to NA)
26 [1] 
(NA to NA)
[1]
Data for Median was estimated and the 95% CI could not be estimated due to low event rate.
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fidaxomicin, Vancomycin
Comments Time to recurrence of CDAD.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.023
Comments [Not Specified]
Method Log Rank
Comments [Not Specified]
16.Secondary Outcome
Title Number of Participants With Adverse Events (AEs)
Hide Description An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a study drug or who had undergone study procedures which did not necessarily have a causal relationship with this treatment. This included abnormal laboratory tests, vital signs, electrocardiogram data or physical examinations that were defined as AEs if the abnormality induced clinical signs or symptoms, required active intervention, interruption or discontinuation of study drug or was clinically significant in the investigator's opinion. The following standard with 3 grades was used to measure the severity of AEs, including abnormal clinical laboratory values: ● Mild: No disruption of normal daily activities ● Moderate: Affected normal daily activities ● Severe: Inability to perform daily activities. A treatment-emergent adverse event (TEAE) was defined as an AE observed after starting administration of the test drug/comparative drug.
Time Frame From the first dose of study drug administration up to 30 days after EOT (up to day 40)
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The analysis population consisted of the safety analysis set (SAF), which consisted of all randomized participants who received at least 1 study drug dose. In the SAF, participants were allocated to the treatment arm corresponding to study drug first administered (fidaxomicin or vancomycin), even if it differed from the treatment randomized to.
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description:
Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days.
Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
Overall Number of Participants Analyzed 98 44
Measure Type: Count of Participants
Unit of Measure: Participants
TEAE
72
  73.5%
33
  75.0%
Drug-related TEAE
7
   7.1%
5
  11.4%
Serious TEAE
24
  24.5%
12
  27.3%
Drug-related Serious TEAE
0
   0.0%
0
   0.0%
Moderate TEAE
39
  39.8%
14
  31.8%
Drug-related Moderate TEAE
4
   4.1%
1
   2.3%
Mild TEAE
56
  57.1%
30
  68.2%
Drug-related Mild TEAE
3
   3.1%
4
   9.1%
TEAE Leading to Death
3
   3.1%
0
   0.0%
Drug-related TEAE Leading to Death
0
   0.0%
0
   0.0%
TEAE leading to Withdrawal of Treatment (Tx)
1
   1.0%
1
   2.3%
Drug-related TEAE Leading to Withdrawal of Tx
0
   0.0%
0
   0.0%
17.Secondary Outcome
Title Plasma Concentrations of Fidaxomicin
Hide Description Drug concentration was derived from the blood samples collected.
Time Frame Within 30 minutes predose and 1 to 5 hours postdose taken between day 5 and day 10
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Hide Analysis Population Description
The analysis population consisted of the pharmacokinetics analysis set (PKAS). The PKAS consisted of all participants randomized to fidaxomicin, having received at least 1 dose of fidaxomicin and having at least 1 valid measurement of plasma concentration or fecal concentration of fidaxomicin or its main metabolite OP-1118.
Arm/Group Title Fidaxomin All Formulations Fidaxomicin Oral Suspension Fidaxomicin Tablets
Hide Arm/Group Description:
Participants received either fidaxomicin oral suspension or tablet formulation.
Participants received fidaxomicin oral suspension formulation.
Participants received fidaxomicin tablets formulation.
Overall Number of Participants Analyzed 98 67 31
Mean (Standard Deviation)
Unit of Measure: ng/mL
Predose Number Analyzed 82 participants 55 participants 27 participants
20.17  (40.16) 15.26  (20.43) 30.16  (63.27)
Postdose Number Analyzed 81 participants 53 participants 28 participants
39.41  (62.15) 34.60  (57.79) 48.53  (69.85)
18.Secondary Outcome
Title Plasma Concentrations of Metabolite OP-1118
Hide Description Drug concentration was derived from the blood samples collected.
Time Frame Within 30 minutes predose and 1 to 5 hours postdose taken between day 5 and day 10
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Hide Analysis Population Description
The analysis population consisted of the PKAS.
Arm/Group Title Fidaxomin All Formulations Fidaxomicin Oral Suspension Fidaxomicin Tablets
Hide Arm/Group Description:
Participants received either fidaxomicin oral suspension or tablet formulation.
Participants received fidaxomicin oral suspension formulation.
Participants received fidaxomicin tablets formulation.
Overall Number of Participants Analyzed 98 67 31
Mean (Standard Deviation)
Unit of Measure: ng/mL
Predose Number Analyzed 82 participants 55 participants 27 participants
63.04  (171.97) 42.18  (76.76) 105.54  (277.67)
Postdose Number Analyzed 81 participants 53 participants 28 participants
116.64  (259.10) 102.38  (245.19) 143.63  (286.31)
19.Secondary Outcome
Title Metabolite-to-Parent Ratio (MPRconc)
Hide Description Drug concentration was derived from the blood samples collected.
Time Frame Within 30 minutes predose and 1 to 5 hours postdose taken between day 5 and day 10
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Hide Analysis Population Description
The analysis population consisted of the PKAS.
Arm/Group Title Fidaxomin All Formulations Fidaxomicin Oral Suspension Fidaxomicin Tablets
Hide Arm/Group Description:
Participants received either fidaxomicin oral suspension or tablet formulation.
Participants received fidaxomicin oral suspension formulation.
Participants received fidaxomicin tablets formulation.
Overall Number of Participants Analyzed 98 67 31
Mean (Standard Deviation)
Unit of Measure: ratio
Predose Number Analyzed 82 participants 55 participants 27 participants
3.18  (1.42) 3.24  (1.46) 3.05  (1.35)
Postdose Number Analyzed 81 participants 53 participants 28 participants
2.86  (1.18) 2.95  (1.23) 2.69  (1.06)
20.Secondary Outcome
Title Fecal Concentrations of Fidaxomicin
Hide Description Drug concentration was derived from the stool samples collected.
Time Frame Within 24 hours of a dose taken between day 5 and day 10
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Hide Analysis Population Description
The analysis population consisted of the PKAS.
Arm/Group Title Fidaxomin All Formulations Fidaxomicin Oral Suspension Fidaxomicin Tablets
Hide Arm/Group Description:
Participants received either fidaxomicin oral suspension or tablet formulation.
Participants received fidaxomicin oral suspension formulation.
Participants received fidaxomicin tablets formulation.
Overall Number of Participants Analyzed 74 47 27
Mean (Standard Deviation)
Unit of Measure: μg/g
2685.56  (2476.92) 2969.87  (2713.58) 2190.63  (1948.68)
21.Secondary Outcome
Title Fecal Concentrations of Metabolite OP-1118
Hide Description Drug concentration was derived from the stool samples collected.
Time Frame Within 24 hours of a dose taken between day 5 and day 10
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the PKAS.
Arm/Group Title Fidaxomin All Formulations Fidaxomicin Oral Suspension Fidaxomicin Tablets
Hide Arm/Group Description:
Participants received either fidaxomicin oral suspension or tablet formulation.
Participants received fidaxomicin oral suspension formulation.
Participants received fidaxomicin tablets formulation.
Overall Number of Participants Analyzed 73 46 27
Mean (Standard Deviation)
Unit of Measure: μg/g
889.23  (817.83) 789.15  (728.58) 1059.73  (941.04)
22.Secondary Outcome
Title MPRconc Within 24 Hours of a Dose
Hide Description Drug concentration was derived from the stool samples collected.
Time Frame Within 24 hours of a dose taken between day 5 and day 10
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Hide Analysis Population Description
The analysis population consisted of the PKAS.
Arm/Group Title Fidaxomin All Formulations Fidaxomicin Oral Suspension Fidaxomicin Tablets
Hide Arm/Group Description:
Participants received either fidaxomicin oral suspension or tablet formulation.
Participants received fidaxomicin oral suspension formulation.
Participants received fidaxomicin tablets formulation.
Overall Number of Participants Analyzed 72 45 27
Mean (Standard Deviation)
Unit of Measure: ratio
0.43  (0.31) 0.32  (0.19) 0.63  (0.38)
23.Secondary Outcome
Title Acceptance of Formulation (Palatability Assessment) in All Participants at First Administration of Study Drug and at Day 7
Hide Description Acceptance of formulation was evaluated in all participants who received fidaxomicin oral suspension and vancomycin oral liquid (i.e., participants from birth to =< 6 years and participants > 6 years unable to swallow tablets) by means of a five-point rating scale (awful, poor, fair, good, excellent) by unblinded staff if hospitalized, and by the participant/parents/legal guardian when at home.
Time Frame Days 1 and 7
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Hide Analysis Population Description
The analysis population consisted of the FAS.
Arm/Group Title Fidaxomicin Oral Suspension Vancomycin Oral Liquid
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Participants received fidaxomicin oral suspension formulation.
Participants received vancomycin oral liquid formulation.
Overall Number of Participants Analyzed 67 30
Measure Type: Count of Participants
Unit of Measure: Participants
Day 1 Awful
4
   6.0%
5
  16.7%
Day 1 Poor
6
   9.0%
3
  10.0%
Day 1 Fair
13
  19.4%
6
  20.0%
Day 1 Good
19
  28.4%
7
  23.3%
Day 1 Excellent
13
  19.4%
4
  13.3%
Day 1 Missing
12
  17.9%
5
  16.7%
Day 7 Awful
2
   3.0%
3
  10.0%
Day 7 Poor
5
   7.5%
5
  16.7%
Day 7 Fair
8
  11.9%
5
  16.7%
Day 7 Good
21
  31.3%
9
  30.0%
Day 7 Excellent
16
  23.9%
3
  10.0%
Day 7 Missing
15
  22.4%
5
  16.7%
Time Frame From the first dose of study drug administration up to 30 days after EOT (up to day 40)
Adverse Event Reporting Description The total number of deaths (all causes) includes deaths reported after time frame above.
 
Arm/Group Title Fidaxomicin Vancomycin
Hide Arm/Group Description Participants from birth to < 6 years of age received weight based doses of fidaxomicin oral suspension (32 mg/kg/day with a maximum dose of 400 mg/day divided in 2 doses) 2 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 200 mg fidaxomicin tablet 2 times daily for 10 days. Participants from birth to < 6 years of age received weight based doses of vancomycin oral liquid (40 mg/kg/day with a maximum dose of 500 mg/day divided in 4 doses) 4 times daily for 10 days. Participants aged ≥ 6 years to < 18 years of age received a 125 mg vancomycin capsule 4 times daily for 10 days.
All-Cause Mortality
Fidaxomicin Vancomycin
Affected / at Risk (%) Affected / at Risk (%)
Total   3/98 (3.06%)      2/44 (4.55%)    
Show Serious Adverse Events Hide Serious Adverse Events
Fidaxomicin Vancomycin
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   24/98 (24.49%)      12/44 (27.27%)    
Blood and lymphatic system disorders     
Febrile bone marrow aplasia  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Febrile neutropenia  1  3/98 (3.06%)  6 1/44 (2.27%)  1
Thrombocytosis  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Congenital, familial and genetic disorders     
Mitochondrial encephalomyopathy  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Gastrointestinal disorders     
Abdominal pain lower  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Colitis  1  0/98 (0.00%)  0 1/44 (2.27%)  1
Diarrhoea  1  0/98 (0.00%)  0 1/44 (2.27%)  1
Gastrointestinal necrosis  1  0/98 (0.00%)  0 1/44 (2.27%)  1
Ileus paralytic  1  0/98 (0.00%)  0 1/44 (2.27%)  1
Intestinal obstruction  1  0/98 (0.00%)  0 1/44 (2.27%)  1
Pancreatitis  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Rectal haemorrhage  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Small intestinal obstruction  1  1/98 (1.02%)  1 0/44 (0.00%)  0
General disorders     
Mucosal inflammation  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Pain  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Pyrexia  1  2/98 (2.04%)  2 2/44 (4.55%)  2
Immune system disorders     
Anaphylactic reaction  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Food allergy  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Infections and infestations     
Bacterial diarrhoea  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Bacterial sepsis  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Clostridium difficile colitis  1  0/98 (0.00%)  0 1/44 (2.27%)  1
Clostridium difficile infection  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Fungal sepsis  1  0/98 (0.00%)  0 1/44 (2.27%)  1
Herpes simplex meningoencephalitis  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Influenza  1  0/98 (0.00%)  0 1/44 (2.27%)  1
Klebsiella bacteraemia  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Pneumonia  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Respiratory syncytial virus infection  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Respiratory tract infection  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Scedosporium infection  1  0/98 (0.00%)  0 1/44 (2.27%)  1
Sepsis  1  2/98 (2.04%)  2 0/44 (0.00%)  0
Staphylococcal sepsis  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Investigations     
Heart rate irregular  1  0/98 (0.00%)  0 1/44 (2.27%)  1
Metabolism and nutrition disorders     
Acidosis  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Dehydration  1  2/98 (2.04%)  2 0/44 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Acute myeloid leukaemia  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Leukaemia  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Malignant ascites  1  0/98 (0.00%)  0 1/44 (2.27%)  1
Nervous system disorders     
Amnesia  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Convulsion  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Headache  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Psychiatric disorders     
Abnormal behaviour  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Renal and urinary disorders     
Renal failure  1  0/98 (0.00%)  0 1/44 (2.27%)  1
Renal failure acute  1  2/98 (2.04%)  2 0/44 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Respiratory distress  1  1/98 (1.02%)  1 0/44 (0.00%)  0
Respiratory failure  1  0/98 (0.00%)  0 1/44 (2.27%)  1
Surgical and medical procedures     
Radiotherapy  1  1/98 (1.02%)  1 0/44 (0.00%)  0
1
Term from vocabulary, MedDRA 17
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Fidaxomicin Vancomycin
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   36/98 (36.73%)      21/44 (47.73%)    
Gastrointestinal disorders     
Abdominal pain  1  5/98 (5.10%)  6 9/44 (20.45%)  11
Constipation  1  5/98 (5.10%)  6 1/44 (2.27%)  1
Diarrhoea  1  7/98 (7.14%)  7 4/44 (9.09%)  5
Vomiting  1  7/98 (7.14%)  8 6/44 (13.64%)  8
General disorders     
Pyrexia  1  11/98 (11.22%)  18 8/44 (18.18%)  11
Infections and infestations     
Oral candidiasis  1  3/98 (3.06%)  3 3/44 (6.82%)  3
Nervous system disorders     
Headache  1  8/98 (8.16%)  11 0/44 (0.00%)  0
Skin and subcutaneous tissue disorders     
Pruritus  1  3/98 (3.06%)  3 3/44 (6.82%)  3
1
Term from vocabulary, MedDRA 17
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Institute and/or Principal Investigator may publish trial data generated at their specific study site after Sponsor publication of the multi-center data. Sponsor must receive a site's manuscript prior to publication for review and comment as specified in the Investigator Agreement.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Clinical Trial Disclosure
Organization: Astellas Pharma Global Development, Inc.
Phone: 800-888-7704
EMail: astellas.resultsdisclosure@astellas.com
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Astellas Pharma Inc ( Astellas Pharma Europe B.V. )
ClinicalTrials.gov Identifier: NCT02218372     History of Changes
Other Study ID Numbers: 2819-CL-0202
2013-000508-40 ( EudraCT Number )
SUNSHINE ( Other Identifier: Acronym )
First Submitted: August 11, 2014
First Posted: August 18, 2014
Results First Submitted: October 23, 2018
Results First Posted: December 7, 2018
Last Update Posted: February 15, 2019