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A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor

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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.

Brief 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


Condition or disease Intervention/treatment Phase
Hereditary Angioedema Biological: Recombinant human C1 inhibitor Other: Placebo Phase 2

Detailed 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.


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 32 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Prevention
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)
Study Start Date : December 2014
Actual Primary Completion Date : May 2016
Actual Study Completion Date : September 2016

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: rhC1INH twice weekly
rhC1INH administered twice weekly
Biological: Recombinant human C1 inhibitor
Other Names:
  • rhC1INH
  • Ruconest
  • Conestat alfa

Experimental: rhC1INH once weekly
rhC1INH administered once weekly
Biological: Recombinant human C1 inhibitor
Other Names:
  • rhC1INH
  • Ruconest
  • Conestat alfa

Placebo Comparator: Placebo (Saline) twice weekly
Placebo (Saline) administered twice weekly
Other: Placebo
Other Name: Saline




Primary Outcome Measures :
  1. Number of HAE Attacks [ Time Frame: 28 days ]
    Average number of HAE attacks normalized to a 28 day period


Secondary Outcome Measures :
  1. Number of Participants With Adverse Events [ Time Frame: 20 weeks ]
    Number of participants that experienced Treatment Emergent Adverse Events observed in safety population

  2. Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks [ Time Frame: 28 days ]
    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


Other Outcome Measures:
  1. Immunogenicity [ Time Frame: 20 weeks ]
    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



Information from the National Library of Medicine

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Ages Eligible for Study:   13 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02247739


Locations
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United States, Florida
University of South Florida Asthma, Allergy and Immunology Clinical Research Unit
Tampa, Florida, United States, 33613
University of South Florida, Asthma, Allergy & Immunology Clinical Research Unit
Tampa, Florida, United States, 33613
United States, Missouri
Washington University Division of Allergy and Immunology
Saint Louis, Missouri, United States, 63141
United States, Oregon
Baker Allergy, Asthma and Dermatology Research Center
Lake Oswego, Oregon, United States, 97035
Canada, Ontario
Ottawa Allergy Research Corp
Ottawa, Ontario, Canada, K1G6C6
Czechia
Faculty Hospital by St. Anna Brno, Department of clinical Immunology and Allergology
Brno, Czechia, 65691
Italy
Azienda Ospedaliera Universitaria Luigi Sacco Di Milano
Milan, Italy, 20157
Macedonia, The Former Yugoslav Republic of
PHI University Clinic of Dermatology
Skopje, Macedonia, The Former Yugoslav Republic of, 1000
Romania
SC Centrul Clinic Mediquest SRL
Sângeorgiu de Mureş, Mures, Romania, 547530
Serbia
Clinical Center Serbia
Belgrade, Serbia
Sponsors and Collaborators
Pharming Technologies B.V.

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Pharming Technologies B.V.
ClinicalTrials.gov Identifier: NCT02247739     History of Changes
Other Study ID Numbers: C1 3201
First Posted: September 25, 2014    Key Record Dates
Results First Posted: December 8, 2017
Last Update Posted: December 8, 2017
Last Verified: November 2017

Keywords provided by Pharming Technologies B.V.:
HAE
Hereditary Angioedema
Prophylaxis

Additional relevant MeSH terms:
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Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn
Complement C1 Inhibitor Protein
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs