Ravulizumab in Thrombotic Microangiopathy Associated With a Trigger

Stopped early · Phase 3 · Has a placebo group

Conditions studied: Thrombotic Microangiopathy

In brief

This study will investigate the efficacy and safety of ravulizumab compared to placebo in adult participants with thrombotic microangiopathy (TMA) associated with a trigger. Participants will be randomized to receive either ravulizumab plus best supportive care or placebo plus best supportive care. The treatment period is 26 weeks followed by a 26-week off-treatment follow-up period.

Key facts

Study ID

NCT04743804

Run by

Alexion Pharmaceuticals, Inc.

People needed

16

Starts

2021-06-29

Expected to finish

2022-12-22

Last updated by the study team

2024-10-01

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 18 years of age or older
• Body weight ≥ 30 kilograms
• Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab
• TMA associated with a trigger (autoimmune disease, infection, solid organ transplant, drugs, and malignant hypertension)
• Vaccinated against meningococcal infection (Neisseria meningitidis), within 3 years prior to, or at the time of, randomization. Participants who initiate study drug treatment less than 2 weeks after receiving a meningococcal vaccine must receive appropriate prophylactic antibiotics for at least 2 weeks after the vaccination. If participant cannot receive the meningococcal vaccine, then participant must be willing to receive antibiotic prophylaxis coverage against N. meningitidis during the entire Treatment Period and for 8 months following the final dose of study drug. Additional vaccination (Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae) may be considered based on individual patient condition.

You may not qualify if…

• Any known gene mutation that causes atypical hemolytic uremic syndrome (aHUS)
• Postpartum aHUS
• Known chronic kidney disease
• TMA due to hematopoietic stem cell transplantation ≤ 12 months of Screening
• Primary and secondary glomerular diseases other than lupus
• Diagnosis of primary antiphospholipid antibody syndrome
• Shiga toxin-producing Escherichia coli infections including but not limited to Shiga toxin-related hemolytic uremic syndrome
• Known familial or acquired 'a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13' (ADAMTS13) deficiency (activity < 5%)
• Positive direct Coombs test which in the judgement of the Investigator is indicative of a clinically significant immune-mediated hemolysis not due to TMA
• Clinical diagnosis of disseminated intravascular coagulation (DIC) in the judgement of the Investigator
• Presence of sepsis requiring vasopressors within 7 days prior to or during Screening
• Presence of monoclonal gammopathy including but not limited to multiple myeloma
• Known bone marrow insufficiency or failure evidenced by cytopenias
• Unresolved N. meningitidis infection
• History of malignancy within 5 years of Screening with the exception of nonmelanoma skin cancer or carcinoma in situ of the cervix that has been treated with no evidence of recurrence
• Use of any complement inhibitors within the past 3 years
• Respiratory failure requiring mechanical ventilation

Where it is running

• Research Site — Orange, California, United States
• Research Site — Washington D.C., District of Columbia, United States
• Research Site — Gainesville, Florida, United States
• Research Site — Lexington, Kentucky, United States
• Research Site — Louisville, Kentucky, United States
• Research Site — Boston, Massachusetts, United States
• Research Site — Boston, Massachusetts, United States
• Research Site — Boston, Massachusetts, United States
• Research Site — Rochester, Minnesota, United States
• Research Site — New York, New York, United States
• Research Site — Valhalla, New York, United States
• Research Site — Winston-Salem, North Carolina, United States
• Research Site — Cleveland, Ohio, United States
• Research Site — Cleveland, Ohio, United States
• Research Site — Columbus, Ohio, United States
• Research Site — Philadelphia, Pennsylvania, United States
• Research Site — Pittsburgh, Pennsylvania, United States
• Research Site — Salt Lake City, Utah, United States
• Research Site — Morgantown, West Virginia, United States
• Research Site — Belgium, Belgium
• Research Site — Brussels, Belgium
• Research Site — Edegem, Belgium
• Research Site — Leuven, Belgium
• Research Site — Liège, Belgium
• Research Site — Tucson, Arizona, United States

Full record on ClinicalTrials.gov

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