A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor

Hereditary Angioedema Clinical Trial

Official title:

A Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled, 3-Period Crossover Study to Evaluate the Efficacy and Safety of Recombinant Human C1 Inhibitor in the Prophylaxis of Angioedema Attacks in Patients With Hereditary Angioedema (HAE)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02247739
• Other study ID #: C1 3201
• Status: Completed
• Phase: Phase 2
• First received: September 16, 2014
• Last updated: November 7, 2017
• Start date: December 2014
• Est. completion date: September 2016

Study information

• Verified date: November 2017
• Source: Pharming Technologies B.V.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Primary Objective:

To evaluate the efficacy of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE

Secondary Objective:

To evaluate the safety and immunogenicity of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE

Description:

Study Design:

This is a multi-center, randomized, double-blind, placebo-controlled, 3-period crossover study of rhC1INH in prophylaxis of angioedema attacks in patients with HAE.

Medical screening (clinical and laboratory parameters) will be performed and patient medical history specific to HAE attacks will be collected to assess eligibility. Each patient will receive three 4 week periods of treatment twice weekly.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 32
• Est. completion date: September 2016
• Est. primary completion date: May 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 13 Years and older

Eligibility

Inclusion Criteria:

Patients fulfilling the following criteria at Screening are eligible for participation in the study:

1. Age 13 years or older

2. Laboratory confirmed diagnosis of HAE

3. A history of frequent HAE attacks (at least 4 attacks per month across a minimum of 3 consecutive months).

4. Female patients of childbearing potential who are sexually active must be willing to use an acceptable form of contraception.

5. Provided written informed consent (and written assent for minors)

6. Willingness and ability to comply with all protocol procedures

Exclusion Criteria:

Patients who meet any of the following criteria at Screening are to be excluded from study participation:

1. Patients with medical history of allergy to rabbits or rabbit-derived products (including rhC1INH)

2. Diagnosis of acquired angioedema (AAE)

3. Patients who are pregnant, or breastfeeding, or are currently intending to become pregnant

4. Treatment with any investigational drug in the past 30 days

5. Patients with any condition or treatment that, in the opinion of the Investigator, might interfere with the evaluation of study objectives

6. Patients currently treated with angiotensin-converting enzyme (ACE) inhibitors

Related Conditions & MeSH terms

• Angioedema
• Hereditary Angioedema

Intervention

Biological:
• Recombinant human C1 inhibitor

Other:
• Placebo

Locations

Country	Name	City	State
Canada	Ottawa Allergy Research Corp	Ottawa	Ontario
Czechia	Faculty Hospital by St. Anna Brno, Department of clinical Immunology and Allergology	Brno
Italy	Azienda Ospedaliera Universitaria Luigi Sacco Di Milano	Milan
Macedonia, The Former Yugoslav Republic of	PHI University Clinic of Dermatology	Skopje
Romania	SC Centrul Clinic Mediquest SRL	Sângeorgiu de Mures	Mures
Serbia	Clinical Center Serbia	Belgrade
United States	Baker Allergy, Asthma and Dermatology Research Center	Lake Oswego	Oregon
United States	Washington University Division of Allergy and Immunology	Saint Louis	Missouri
United States	University of South Florida Asthma, Allergy and Immunology Clinical Research Unit	Tampa	Florida
United States	University of South Florida, Asthma, Allergy & Immunology Clinical Research Unit	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Pharming Technologies B.V.

Countries where clinical trial is conducted

United States,  Canada,  Czechia,  Italy,  Macedonia, The Former Yugoslav Republic of,  Romania,  Serbia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Immunogenicity	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	20 weeks
Primary	Number of HAE Attacks	Average number of HAE attacks normalized to a 28 day period	28 days
Secondary	Number of Participants With Adverse Events	Number of participants that experienced Treatment Emergent Adverse Events observed in safety population	20 weeks
Secondary	Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks	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	28 days