A Phase 2, Randomized, Double-Blind, Multicenter, Dose-Ranging, Crossover Study to Evaluate the Safety and Efficacy of Subcutaneous Administration of CINRYZE® (C1 Esterase Inhibitor [Human]) With Recombinant Human Hyaluronidase (rHuPH20) for the Prevention of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
Overview
- Phase
- Phase 2
- Intervention
- Not specified
- Conditions
- Hereditary Angioedema (HAE)
- Sponsor
- Shire
- Enrollment
- 47
- Locations
- 1
- Primary Endpoint
- Normalized Number of Angioedema Attacks During the Treatment Period
- Status
- Completed
- Last Updated
- 4 years ago
Overview
Brief Summary
The primary objectives of the study are to evaluate the safety, tolerability, and efficacy of two doses of CINRYZE with recombinant human hyaluronidase (rHuPH20) administered by subcutaneous (SC) injection to prevent angioedema attacks.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Be ≥12 years of age.
- •Have a confirmed diagnosis of Hereditary Angioedema.
Exclusion Criteria
- •Receipt of any C1 inhibitor (C1 INH) therapy or any blood products for treatment or prevention of an angioedema attack within 7 days before the first dose of study drug.
- •Be receiving prophylactic intravenous CINRYZE that exceeds 1000 units every 3 or 4 days (maximum weekly dose 2000 units).
- •Have received any androgen therapy (e.g., danazol, oxandrolone, stanozolol, testosterone) within 7 days prior to the first dose of study drug.
- •If female, have started taking or changed the dose of any hormonal contraceptive regimen or hormone replacement therapy (i.e., estrogen/progestin containing products) within 3 months prior to the first dose of study drug.
- •History of allergic reaction to C1 INH products, including CINRYZE or other blood products.
- •History of abnormal blood clotting.
- •Have a known allergy to hyaluronidase or any other ingredient in the study formulation.
Outcomes
Primary Outcomes
Normalized Number of Angioedema Attacks During the Treatment Period
Time Frame: From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period
Angioedema attack was defined as the participant-reported indication of symptoms or signs such as swelling or pain at any location following a report of no swelling or pain on the previous day. Manifestations of an attack that progress from one site to another, prior to complete resolution, was considered a single attack. Attacks that began to regress and then worsened before complete resolution was also considered one attack. Participants who were dosed but did not have any attacks in the period were assigned a value of zero. The number of attacks was normalized for the number of days participants participated in a given period and expressed as the monthly frequency.
Secondary Outcomes
- Cumulative Daily-severity During the Treatment Period(From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period)
- Cumulative Attack-severity During the Treatment Period(From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period)
- Cumulative Symptomatic Days During the Treatment Period(From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period)
- Number of Angioedema Attacks Requiring Acute Treatment During the Treatment Period(From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period)