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CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12

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ClinicalTrials.gov Identifier: NCT01095510
Recruitment Status : Completed
First Posted : March 30, 2010
Results First Posted : July 25, 2014
Last Update Posted : December 12, 2018
Sponsor:
Information provided by (Responsible Party):
Shire

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hereditary Angioedema (HAE)
Intervention Biological: CINRYZE
Enrollment 9
Recruitment Details Participants were not enrolled in the lower body weight category (10-25 kilograms [kg]) 1000 Units (U) dose group despite substantial recruitment efforts.
Pre-assignment Details Of 12 participants screened, 9 participants were enrolled and treated. The reason for 3 participants were screen failures as they did not meet the inclusion criteria.
Arm/Group Title 500 U CINRYZE (10-25 kg Body Weight) 1000 U CINRYZE (10-25 kg Body Weight) 1000 U CINRYZE (>25 kg Body Weight) 1500 U CINRYZE (>25 kg Body Weight)
Hide Arm/Group Description Single intravenous (IV) dose of 500 U CINRYZE Single IV dose of 1000 U CINRYZE Single IV dose of 1000 U CINRYZE Single IV dose of 1500 U CINRYZE
Period Title: Overall Study
Started 3 0 3 3
Completed 3 0 3 3
Not Completed 0 0 0 0
Arm/Group Title 500 U CINRYZE (10-25 kg Body Weight) 1000 U CINRYZE (>25 kg Body Weight) 1500 U CINRYZE (>25 kg Body Weight) Total
Hide Arm/Group Description Single IV dose of 500 U CINRYZE Single IV dose of 1000 U CINRYZE Single IV dose of 1500 U CINRYZE Total of all reporting groups
Overall Number of Baseline Participants 3 3 3 9
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 3 participants 3 participants 3 participants 9 participants
7
(6 to 9)
9
(7 to 9)
10
(8 to 11)
9
(6 to 11)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 3 participants 9 participants
Female
3
 100.0%
3
 100.0%
2
  66.7%
8
  88.9%
Male
0
   0.0%
0
   0.0%
1
  33.3%
1
  11.1%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 3 participants 3 participants 3 participants 9 participants
3 3 3 9
1.Primary Outcome
Title Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom
Hide Description [Not Specified]
Time Frame Within 4 hours following treatment
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat efficacy (ITT-E) population included all participants with baseline and at least one post-infusion investigator assessment of the hereditary angioedema (HAE) attack.
Arm/Group Title 500 U CINRYZE (10-25 kg Body Weight) 1000 U CINRYZE (>25 kg Body Weight) 1500 U CINRYZE (>25 kg Body Weight)
Hide Arm/Group Description:
Single IV dose of 500 U CINRYZE
Single IV dose of 1000 U CINRYZE
Single IV dose of 1500 U CINRYZE
Overall Number of Participants Analyzed 3 3 3
Measure Type: Number
Unit of Measure: participants
Yes 3 3 3
No 0 0 0
2.Secondary Outcome
Title Time to Unequivocal Beginning of Relief of the Defining Attack Symptom
Hide Description [Not Specified]
Time Frame Within 4 hours following treatment
Hide Outcome Measure Data
Hide Analysis Population Description
ITT-E population
Arm/Group Title 500 U CINRYZE (10-25 kg Body Weight) 1000 U CINRYZE (>25 kg Body Weight) 1500 U CINRYZE (>25 kg Body Weight)
Hide Arm/Group Description:
Single IV dose of 500 U CINRYZE
Single IV dose of 1000 U CINRYZE
Single IV dose of 1500 U CINRYZE
Overall Number of Participants Analyzed 3 3 3
Median (Full Range)
Unit of Measure: hours
1.25
(0.25 to 1.75)
0.25
(0.25 to 0.50)
0.50
(0.25 to 2.50)
3.Secondary Outcome
Title Time to Complete Resolution of the Attack
Hide Description [Not Specified]
Time Frame Within 1 week following treatment
Hide Outcome Measure Data
Hide Analysis Population Description
ITT-E population.
Arm/Group Title 500 U CINRYZE (10-25 kg Body Weight) 1000 U CINRYZE (>25 kg Body Weight) 1500 U CINRYZE (>25 kg Body Weight)
Hide Arm/Group Description:
Single IV dose of 500 U CINRYZE
Single IV dose of 1000 U CINRYZE
Single IV dose of 1500 U CINRYZE
Overall Number of Participants Analyzed 3 3 3
Median (Full Range)
Unit of Measure: hours
13.58
(11.48 to 37.35)
10.00
(1.57 to 22.33)
29.07
(1.58 to 102.33)
4.Secondary Outcome
Title Change in C1 Inhibitor (C1 INH) Antigen and Functional C1 INH Concentrations
Hide Description Data was not reported due to change in planned analysis.
Time Frame Pre-dose, 2, 4, 8 hours post dose on Day 1; Day 2, 3, 5, 8
Hide Outcome Measure Data
Hide Analysis Population Description
No participant agreed to obtain pharmacokinetic (PK) blood sampling for antigenic and functional C1 INH levels. Hence, it was planned not to be analyzed.
Arm/Group Title 500 U CINRYZE (10-25 kg Body Weight) 1000 U CINRYZE (>25 kg Body Weight) 1500 U CINRYZE (>25 kg Body Weight)
Hide Arm/Group Description:
Single IV dose of 500 U CINRYZE
Single IV dose of 1000 U CINRYZE
Single IV dose of 1500 U CINRYZE
Overall Number of Participants Analyzed 0 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame Adverse events were collected through 1 week following the dose of study drug.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title 500 U CINRYZE (10-25 kg Body Weight) 1000 U CINRYZE (>25 kg Body Weight) 1500 U CINRYZE (>25 kg Body Weight)
Hide Arm/Group Description Single IV dose of 500 U CINRYZE Single IV dose of 1000 U CINRYZE Single IV dose of 1500 U CINRYZE
All-Cause Mortality
500 U CINRYZE (10-25 kg Body Weight) 1000 U CINRYZE (>25 kg Body Weight) 1500 U CINRYZE (>25 kg Body Weight)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
500 U CINRYZE (10-25 kg Body Weight) 1000 U CINRYZE (>25 kg Body Weight) 1500 U CINRYZE (>25 kg Body Weight)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/3 (0.00%)   0/3 (0.00%)   0/3 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
500 U CINRYZE (10-25 kg Body Weight) 1000 U CINRYZE (>25 kg Body Weight) 1500 U CINRYZE (>25 kg Body Weight)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   1/3 (33.33%)   0/3 (0.00%)   0/3 (0.00%) 
Gastrointestinal disorders       
Diarrhea  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%) 
Nausea  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%) 
1
Term from vocabulary, MedDRA (16.0)
Change in C1 INH antigen and functional C1 INH concentrations endpoint was not analyzed as no participants agreed to additional and optional blood sampling. As a result, no PK parameters were calculated for this study.
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.
Clinical Trial Agreement. Most restrictive provision - PI will not publish results until after first of: multicenter publication is published or 24 months from study end. Thereafter, PI may publish his results. PI must provide copy of proposed publication to Sponsor for pre-review. If Sponsor requests, PI must delete Sponsor confidential information before publication and/or delay publication for 60 days so Sponsor can file for patents or take other action to protect its patent rights.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Jennifer Schranz, MD
Organization: Shire
Phone: 484-595-8940
EMail: jschranz@shire.com
Layout table for additonal information
Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01095510     History of Changes
Other Study ID Numbers: 0624-203
2011-000369-11 ( EudraCT Number )
First Submitted: March 24, 2010
First Posted: March 30, 2010
Results First Submitted: June 26, 2014
Results First Posted: July 25, 2014
Last Update Posted: December 12, 2018