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Trial record 34 of 103 for:    Pompe Disease

Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease

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ClinicalTrials.gov Identifier: NCT00688597
Recruitment Status : Terminated (Per protocol, 3 sequential dose cohorts were planned. Study discontinued by Sponsor based upon serious adverse events in first 2 of 3 participants in Cohort 1.)
First Posted : June 3, 2008
Results First Posted : August 17, 2018
Last Update Posted : August 17, 2018
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Pompe Disease
Intervention Drug: Duvoglustat
Enrollment 3
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Cohort 1 Cohort 2 Cohort 3
Hide Arm/Group Description Regimen 1: Low-dose duvoglustat (2.5 grams [g]) once a day (QD) for 3 days, followed by no drug for 4 days, for 11 weeks. Regimen 1: High-dose duvoglustat (5.0 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks. Regimen 2: High-dose duvoglustat (5.0 g) QD for 7 days, followed by no drug for 7 days, for 11 weeks.
Period Title: Overall Study
Started 3 0 0
Received at Least 1 Dose of Study Drug 3 0 0
Completed 0 0 0
Not Completed 3 0 0
Reason Not Completed
Adverse Event             2             0             0
Physician Decision             1             0             0
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Total
Hide Arm/Group Description Regimen 1: Low-dose duvoglustat (2.5 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks. Regimen 1: High-dose duvoglustat (5.0 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks. Regimen 2: High-dose duvoglustat (5.0 g) QD for 7 days, followed by no drug for 7 days, for 11 weeks. Total of all reporting groups
Overall Number of Baseline Participants 3 0 0 3
Hide Baseline Analysis Population Description
Safety Population: All participants who received at least 1 dose of duvoglustat.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 3 participants 0 participants 0 participants 3 participants
49.07  (11.02) 49.07  (11.02)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 0 participants 0 participants 3 participants
Female
2
  66.7%
2
  66.7%
Male
1
  33.3%
1
  33.3%
1.Primary Outcome
Title Proportion Of Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs)
Hide Description The number of participants experiencing severe TEAEs is presented for participants who received duvoglustat treatment in this open-label study. The duration of duvoglustat exposure for Cohort 1 ranged from 2 to 24 days, and their exposure ranged from a total of 7,500 to 32,500 milligrams of duvoglustat. An adverse event (AE) refers to any unfavorable and unintended sign, symptom, syndrome, or illness that develops or worsens during the period of observation in the clinical study. The following guideline was used to grade the intensity of an AE: mild, the AE is easily tolerated and does not interfere with daily activity; moderate, the AE interferes with the daily activity but the participant is still able to function; severe, the AE is incapacitating and requires medical intervention. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
Time Frame Baseline, Week 11
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All participants who received at least 1 dose of duvoglustat.
Arm/Group Title Cohort 1 Cohort 2 Cohort 3
Hide Arm/Group Description:
Regimen 1: Low-dose duvoglustat (2.5 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks.
Regimen 1: High-dose duvoglustat (5.0 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks.
Regimen 2: High-dose duvoglustat (5.0 g) QD for 7 days, followed by no drug for 7 days, for 11 weeks.
Overall Number of Participants Analyzed 3 0 0
Measure Type: Count of Participants
Unit of Measure: Participants
1
  33.3%
2.Secondary Outcome
Title Change In 6-minute Walk Test (6MWT) From Baseline To End Of Study
Hide Description The 6MWT (American Thoracic Society standards) was evaluated in ambulatory participants at screening, baseline, and to the end of the study. It was a standardized test that measured the distance in meters (m) covered over a 6-minute walk. Reference equations used (for 6MWT distance in healthy adults) included: (height in centimeters [cm], weight in kilograms [kg]) 6MWT distance for men = [7.57 × height (cm)] – [5.02 × age] – [1.76 × weight (kg)] – 309 m; 6MWT distance for women = [2.11 × height (cm)] – [5.78 × age] – [2.29 × weight (kg)] + 667 m
Time Frame Baseline, Week 11
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All participants who received at least 1 dose of duvoglustat.
Arm/Group Title Cohort 1 Cohort 2 Cohort 3
Hide Arm/Group Description:
Regimen 1: Low-dose duvoglustat (2.5 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks.
Regimen 1: High-dose duvoglustat (5.0 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks.
Regimen 2: High-dose duvoglustat (5.0 g) QD for 7 days, followed by no drug for 7 days, for 11 weeks.
Overall Number of Participants Analyzed 3 0 0
Mean (Standard Deviation)
Unit of Measure: m
-19.660  (41.1845)
Time Frame Day 1 (after dosing) through end of follow up.
Adverse Event Reporting Description Due to the discontinuation of the study, AEs were not encoded.
 
Arm/Group Title Cohort 1 Cohort 2 Cohort 3
Hide Arm/Group Description Regimen 1: Low-dose duvoglustat (2.5 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks. Regimen 1: High-dose duvoglustat (5.0 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks. Regimen 2: High-dose duvoglustat (5.0 g) QD for 7 days, followed by no drug for 7 days, for 11 weeks.
All-Cause Mortality
Cohort 1 Cohort 2 Cohort 3
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Cohort 1 Cohort 2 Cohort 3
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/3 (66.67%)   0/0   0/0 
General disorders       
Muscular weakness  1 [1]  2/3 (66.67%)  0/0  0/0 
Indicates events were collected by systematic assessment
1
Term from vocabulary, Uncoded
[1]
[1] The Adverse Event Term was not coded in the database; therefore, "General disorders" was selected as the System Organ Class (SOC) for this AE.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Cohort 1 Cohort 2 Cohort 3
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   3/3 (100.00%)   0/0   0/0 
General disorders       
Muscular weakness  1 [1]  2/3 (66.67%)  0/0  0/0 
Flatulence  1 [1]  1/3 (33.33%)  0/0  0/0 
Abdominal pain upper  1 [1]  1/3 (33.33%)  0/0  0/0 
Alanine aminotransferase increased  1 [1]  3/3 (100.00%)  0/0  0/0 
Aspartate aminotransferase increased  1 [1]  3/3 (100.00%)  0/0  0/0 
Blood creatine phosphokinase increased  1 [1]  3/3 (100.00%)  0/0  0/0 
Fall  1 [1]  2/3 (66.67%)  0/0  0/0 
Dysphagia  1 [1]  1/3 (33.33%)  0/0  0/0 
Joint injury  1 [1]  1/3 (33.33%)  0/0  0/0 
Indicates events were collected by systematic assessment
1
Term from vocabulary, Uncoded
[1]
The Adverse Event Term was not coded in the database; therefore, "General disorders" was selected as the SOC for this AE.
The serious adverse event of muscle weakness was the main contributor to the study not reaching the target number of participants needed to achieve target power and statistically reliable results.
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.
The investigator can only publish the results from this trial provided they supply the sponsor (or authorized entity) a copy of any proposed publication for review. If requested, the investigator will remove information deemed confidential or proprietary by the sponsor and will withhold publication for an additional period of time to allow the sponsor to take appropriate measures to establish and preserve its proprietary rights.
Results Point of Contact
Name/Title: Amicus Therapeutics
Organization: Patient Advocacy
Phone: +1-609-662-2000
Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT00688597     History of Changes
Other Study ID Numbers: POM-CL-201
First Submitted: May 30, 2008
First Posted: June 3, 2008
Results First Submitted: July 23, 2018
Results First Posted: August 17, 2018
Last Update Posted: August 17, 2018