Clinical trial NCT02247739: A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hereditary Angioedema
Keywords: HAE
Hereditary Angioedema
Prophylaxis
Lead sponsor: Pharming Technologies B.V.
Sponsor class: INDUSTRY
First posted: 2014-09-25
Enrollment: 32
Sex: ALL
Minimum age (years): 13
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: PREVENTION

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

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.

Eligibility 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

Primary outcomes:
measure: Number of HAE Attacks
timeFrame: 28 days
Secondary outcomes:
measure: Number of Participants With Adverse Events
timeFrame: 20 weeks
measure: Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks
timeFrame: 28 days
Other outcomes:
measure: Immunogenicity
timeFrame: 20 weeks
Locations:
city: Tampa
country: United States
facility: University of South Florida Asthma, Allergy and Immunology Clinical Research Unit
state: Florida
city: Tampa
country: United States
facility: University of South Florida, Asthma, Allergy & Immunology Clinical Research Unit
state: Florida
city: Saint Louis
country: United States
facility: Washington University Division of Allergy and Immunology
state: Missouri
city: Lake Oswego
country: United States
facility: Baker Allergy, Asthma and Dermatology Research Center
state: Oregon
city: Ottawa
country: Canada
facility: Ottawa Allergy Research Corp
state: Ontario
city: Brno
country: Czechia
facility: Faculty Hospital by St. Anna Brno, Department of clinical Immunology and Allergology
city: Milan
country: Italy
facility: Azienda Ospedaliera Universitaria Luigi Sacco Di Milano
city: Skopje
country: Macedonia, The Former Yugoslav Republic of
facility: PHI University Clinic of Dermatology
city: Sângeorgiu de Mureş
country: Romania
facility: SC Centrul Clinic Mediquest SRL
state: Mures
city: Belgrade
country: Serbia
facility: Clinical Center Serbia
Study officials:
References:
citation: Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.
citation: Riedl MA, Grivcheva-Panovska V, Moldovan D, Baker J, Yang WH, Giannetti BM, Reshef A, Andrejevic S, Lockey RF, Hakl R, Kivity S, Harper JR, Relan A, Cicardi M. Recombinant human C1 esterase inhibitor for prophylaxis of hereditary angio-oedema: a phase 2, multicentre, randomised, double-blind, placebo-controlled crossover trial. Lancet. 2017 Sep 30;390(10102):1595-1602. doi: 10.1016/S0140-6736(17)31963-3. Epub 2017 Jul 25.