A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Ravulizumab in Adult and Adolescent Participants Who Have Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplant (HSCT)

Who is this study for? Adult and adolescent patients with thrombotic microangiopathy
What treatments are being studied? Ravulizumab
Status: Recruiting
Location: See all (168) locations...
Intervention Type: Other, Biological
Study Type: Interventional
Study Phase: Phase 3
SUMMARY

This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of ravulizumab in adult and adolescent participants with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). In Stage 1, an open-label, single-arm period, the dosing regimen will be confirmed. In Stage 2, participants will be randomized to receive either blinded ravulizumab plus best supportive care or matching placebo plus best supportive care. The treatment period is 26 weeks (open-label for Stage 1, and randomized, double-blind, and placebo-controlled for Stage 2) followed by a 26-week follow-up period.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Maximum Age: 100
Healthy Volunteers: f
View:

• 12 years of age or older at time of consent/assent.

• Received HSCT within the past 12 months.

• Diagnosis of TMA that persists for at least 72 hours after initial management of any triggering agent/condition.

• A TMA diagnosis based on meeting the laboratory-based criteria during the Screening Period and/or ≤14 days prior to the Screening Period.

• Body weight ≥ 30 kilograms at Screening or ≤7 days prior to the start of the Screening Period (date of consent).

• Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception.

• Participants must be vaccinated against meningococcal infections if clinically feasible. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis. Participants \<18 years of age must be re-vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae if clinically feasible.

• Participants or their legally authorized representative must be capable of giving signed informed consent or assent.

Locations
United States
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Hershey
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Nice
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Toulouse
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Granada
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L'hospitalet De Llobregat
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Madrid
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Nottingham
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Contact Information
Primary
Alexion Pharmaceuticals, Inc. (Sponsor)
clinicaltrials@alexion.com
1-855-752-2356
Time Frame
Start Date: 2020-12-10
Estimated Completion Date: 2026-03-31
Participants
Target number of participants: 144
Treatments
Experimental: Ravulizumab
In Stage 1, all participants will receive open-label ravulizumab plus Best Supportive Care (BSC).~In Stage 2, participants will receive blinded ravulizumab plus Best Supportive Care (BSC).
Placebo_comparator: Placebo
In Stage 2, participants randomized to the placebo arm will receive matching placebo plus BSC.
Sponsors
Leads: Alexion Pharmaceuticals, Inc.

This content was sourced from clinicaltrials.gov