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A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02247739
Recruitment Status : Completed
First Posted : September 25, 2014
Results First Posted : December 8, 2017
Last Update Posted : December 8, 2017
Sponsor:
Information provided by (Responsible Party):
Pharming Technologies B.V.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Prevention
Condition Hereditary Angioedema
Interventions Biological: Recombinant human C1 inhibitor
Other: Placebo
Enrollment 32
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Treatment Sequence A Treatments Sequence B Treatment Sequesce C Treatment Sequence D Treatment Sequence E Treatment Sequence F
Hide Arm/Group Description rhC1INH twice weekly / rhC1INH once weekly / saline rhC1INH once weekly / rhC1INH twice weekly / Saline Saline / rhC1INH once weekly / rhC1INH twice weekly Saline / rhC1INH twice weekly / rhC1INH once weekly rhC1INH twice weekly / saline / rhC1INH once weekly rhC1INH once weekly / saline / rhC1INH twice weekly
Period Title: Overall Study
Started 5 6 5 5 6 5
Completed 5 3 4 5 5 4
Not Completed 0 3 1 0 1 1
Arm/Group Title All Study Participants
Hide Arm/Group Description All randomized patients
Overall Number of Baseline Participants 32
Hide Baseline Analysis Population Description
Population is intention to treat (ITT) population
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 32 participants
<=18 years
1
   3.1%
Between 18 and 65 years
28
  87.5%
>=65 years
3
   9.4%
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 32 participants
46
(17 to 74)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 32 participants
Female
26
  81.3%
Male
6
  18.8%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 32 participants
Canada 2
Czech Republic 2
Romania 6
United States 7
Macedonia, The Former Yugoslav Republic of 8
Italy 1
Serbia 2
Israel 4
1.Primary Outcome
Title Number of HAE Attacks
Hide Description Average number of HAE attacks normalized to a 28 day period
Time Frame 28 days
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) Population: All patients who were randomized into one of the treatment sequences. The statistical analyses are based on the treatments to which the patient was randomized to receive during that treatment period.
Arm/Group Title rhC1INH Twice Weekly rhC1INH Once Weekly Placebo (Saline) Twice Weekly
Hide Arm/Group Description:

rhC1INH administered twice weekly

Recombinant human C1 inhibitor

rhC1INH administered once weekly

Recombinant human C1 inhibitor

Placebo (Saline) administered twice weekly

Placebo

Overall Number of Participants Analyzed 32 32 32
Mean (95% Confidence Interval)
Unit of Measure: attacks
2.74
(1.8 to 3.7)
4.36
(3.1 to 5.6)
7.18
(5.8 to 8.6)
2.Secondary Outcome
Title Number of Participants With Adverse Events
Hide Description Number of participants that experienced Treatment Emergent Adverse Events observed in safety population
Time Frame 20 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All patients who received at least a partial injection of study drug. The statistical analyses are based on the actual treatment the patient received.
Arm/Group Title rhC1INH Twice Weekly rhC1INH Once Weekly Placebo (Saline) Twice Weekly
Hide Arm/Group Description:

rhC1INH administered twice weekly

Recombinant human C1 inhibitor

rhC1INH administered once weekly

Recombinant human C1 inhibitor

Placebo (Saline) administered twice weekly

Placebo

Overall Number of Participants Analyzed 29 29 28
Measure Type: Count of Participants
Unit of Measure: Participants
10
  34.5%
13
  44.8%
8
  28.6%
3.Secondary Outcome
Title Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks
Hide Description Percentage of participants achieving at least 50% reduction in the number of attacks normalized to a 28-day period as compared to the placebo treatment period
Time Frame 28 days
Hide Outcome Measure Data
Hide Analysis Population Description
Both the rhC1INH twice weekly treatments and the rhC1INH once weekly treatment periods are compared to the placebo treatment for the safety population. One subject withdrew before receiving any treatment and is excluded from the analysis.
Arm/Group Title rhC1INH Twice Weekly rhC1INH Once Weekly
Hide Arm/Group Description:
rhC1INH administered twice weekly
rhC1INH administered once weekly
Overall Number of Participants Analyzed 31 31
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
74
(57 to 86)
42
(26 to 59)
4.Other Pre-specified Outcome
Title Immunogenicity
Hide Description Number of participants analyzed for neutralizing C1INH-specific antibodies and neutralizing rhC1INH-specific antibodies after confirmed anti-C1INH and anti rhC1INH IgM or IgG antibodies
Time Frame 20 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Participants analyzed for Neutralizing antibodies after confirmed testing of anti-C1INH and anti rhC1INH IgM or IgG antibodies. Count of participants displays the number of positives.
Arm/Group Title rhC1INH Twice Weekly rhC1INH Once Weekly Placebo (Saline) Twice Weekly
Hide Arm/Group Description:

rhC1INH administered twice weekly

Recombinant human C1 inhibitor

rhC1INH administered once weekly

Recombinant human C1 inhibitor

Placebo (Saline) administered twice weekly

Placebo

Overall Number of Participants Analyzed 3 5 2
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
Time Frame 20 weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title rhC1INH Twice Weekly rhC1INH Once Weekly Placebo (Saline) Twice Weekly
Hide Arm/Group Description

rhC1INH administered twice weekly

Recombinant human C1 inhibitor

rhC1INH administered once weekly

Recombinant human C1 inhibitor

Placebo (Saline) administered twice weekly

Placebo

All-Cause Mortality
rhC1INH Twice Weekly rhC1INH Once Weekly Placebo (Saline) Twice Weekly
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Hide Serious Adverse Events
rhC1INH Twice Weekly rhC1INH Once Weekly Placebo (Saline) Twice Weekly
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/29 (3.45%)      0/29 (0.00%)      0/28 (0.00%)    
Congenital, familial and genetic disorders       
Phimosis  1  1/29 (3.45%)  1 0/29 (0.00%)  0 0/28 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 17.1
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
rhC1INH Twice Weekly rhC1INH Once Weekly Placebo (Saline) Twice Weekly
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   5/29 (17.24%)      7/29 (24.14%)      2/28 (7.14%)    
Infections and infestations       
Nasopharyngitis * 1  0/29 (0.00%)  0 3/29 (10.34%)  3 2/28 (7.14%)  2
Nervous system disorders       
Headache * 1  5/29 (17.24%)  5 2/29 (6.90%)  2 0/28 (0.00%)  0
Psychiatric disorders       
Anxiety * 1  0/29 (0.00%)  0 2/29 (6.90%)  2 0/28 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA 17.1
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: VP Clinical Research & Medical Affairs
Organization: Pharming Group NV
EMail: a.relan@pharming.com
Layout table for additonal information
Responsible Party: Pharming Technologies B.V.
ClinicalTrials.gov Identifier: NCT02247739    
Other Study ID Numbers: C1 3201
First Submitted: September 16, 2014
First Posted: September 25, 2014
Results First Submitted: July 26, 2017
Results First Posted: December 8, 2017
Last Update Posted: December 8, 2017