Study of Ravulizumab in Pediatric Participants With HSCT-TMA

Study Identifier:
ALXN1210-TMA-314
ClinicalTrials.gov Identifier:
NCT04557735
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete

Study Details

Medical Condition
  • Thrombotic Microangiopathy
Study Drug
  • Drug: Ravulizumab
Date
Dec 2020 - Nov 2024
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A - 17 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • ≥ 28 days of age up to \< 18 years of age at the time of signing the informed consent.
  • 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 ≥ 5 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.
Exclusion Criteria
  • Thrombotic thrombocytopenic purpura (TTP) evidenced by ADAMTS13 deficiency.
  • Known Shiga toxin-related hemolytic uremic syndrome as demonstrated by positive test.
  • Positive direct Coombs test indicative of a clinically significant immune-mediated hemolysis not due to TMA.
  • Clinical diagnosis of disseminated intravascular coagulation (DIC).
  • Known bone marrow/graft failure for the current HSCT.
  • Diagnosis of veno-occlusive disease (VOD) which is unresolved at the time of Screening.
  • Human immunodeficiency virus (HIV) infection.
  • Unresolved meningococcal disease.
  • Presence of sepsis requiring vasopressor support.
  • Pregnancy or breastfeeding.
  • Hypersensitivity to murine proteins or to 1 of the excipients of Ravulizumab.
  • Any ongoing or history of medical or psychological conditions unrelated to HSCT-TMA that could increase the risk to the participant or confound the outcome of the study.
  • Respiratory failure requiring mechanical ventilation.
  • Previously or currently treated with a complement inhibitor.
  • Participation in an interventional treatment study of any therapy for TMA.
Sex
Female & Male
Age
N/A - 17 Years

Study Details

Medical Condition
  • Thrombotic Microangiopathy
Study Drug
  • Drug: Ravulizumab
Date
Dec 2020 - Nov 2024
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A - 17 Years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • ≥ 28 days of age up to \< 18 years of age at the time of signing the informed consent.
  • 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 ≥ 5 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.
Exclusion Criteria
  • Thrombotic thrombocytopenic purpura (TTP) evidenced by ADAMTS13 deficiency.
  • Known Shiga toxin-related hemolytic uremic syndrome as demonstrated by positive test.
  • Positive direct Coombs test indicative of a clinically significant immune-mediated hemolysis not due to TMA.
  • Clinical diagnosis of disseminated intravascular coagulation (DIC).
  • Known bone marrow/graft failure for the current HSCT.
  • Diagnosis of veno-occlusive disease (VOD) which is unresolved at the time of Screening.
  • Human immunodeficiency virus (HIV) infection.
  • Unresolved meningococcal disease.
  • Presence of sepsis requiring vasopressor support.
  • Pregnancy or breastfeeding.
  • Hypersensitivity to murine proteins or to 1 of the excipients of Ravulizumab.
  • Any ongoing or history of medical or psychological conditions unrelated to HSCT-TMA that could increase the risk to the participant or confound the outcome of the study.
  • Respiratory failure requiring mechanical ventilation.
  • Previously or currently treated with a complement inhibitor.
  • Participation in an interventional treatment study of any therapy for TMA.

Protocol Summary

This study will evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab administered by intravenous infusion to pediatric participants, from 1 month to \< 18 years of age, with HSCT-TMA. The treatment period is 26 weeks, followed by a 26-week off-treatment follow-up period.

Trial Locations

Location
Status
Location
Research Site
Tucson, Arizona, United States, 85724
Status
N/A
Location
Research Site
Aurora, Colorado, United States, 80045
Status
N/A
Location
Research Site
Atlanta, Georgia, United States, 30322
Status
N/A
Location
Research Site
Chicago, Illinois, United States, 60611
Status
N/A
Location
Research Site
Portland, Oregon, United States, 97239
Status
N/A
Location
Research Site
Dallas, Texas, United States, 75235
Status
N/A
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