hematopoietic stem cell transplant-associated thrombotic microangiopathy MedDRA version: 20.0 Level: PT Classification code 10043645 Term: Thrombotic microangiopathy System Organ Class: 10005329 - Blood and lymphatic system disorders
Conditions
Interventions
Trade Name: Ultomiris
Product Name: Ultomiris
Product Code: ALXN1210
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: RAVULIZUMAB
CAS Number: 1803171-55-2
Current Sponso
Sponsors
Alexion Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. 1 month of age up to 1.5 × ULN or, - Presence of schistocytes = 2 high power field (HPF) • Proteinuria on spot urinalysis • Presence of hypertension 4. Participants must have HSCT-TMA that persists for at least 72 hours after initial management of any triggering agent/condition 5.Body weight = 5 kg at Screening 6. Male or female Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies 7. Vaccinated against meningococcal infections if clinically feasible, according to institutional guidelines for immune reconstitution after HSCT. Participants must be re-vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae if clinically feasible, according to institutional guidelines for immune reconstitution after HSCT. All participants should be administered coverage with prophylactic antibiotics according to institutional posttransplant infection prophylaxis guidances including coverage against N. meningiditis for at least 2 weeks after meningococcal vaccination. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis coverage against N. meningiditis the entire Treatment Period and for 8 months following the final dose of ravulizumab. 8. Participants or their legally authorized representative must be capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent or assent form and in this protocol Are the trial subjects under 18? yes Number of subjects for this age range: 40 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range 0 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: 1. Known familial or acquired ‘a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13’ (ADAMTS13) deficiency (activity < 5%) 2. Known Shiga toxin-related hemolytic uremic syndrome (ST-HUS) 3. Positive direct Coombs test 4. Diagnosis or suspicion of disseminated intravascular coagulation (DIC) 5. Known bone marrow/graft failure 6. Diagnosis of veno-occlusive disease (VOD) 7. Human immunodeficiency virus (HIV) infection (evidenced by HIV-1 or HIV-2 antibody titer, 8. Unresolved meningococcal disease 9. Presence or suspicion of sepsis (treated or untreated) within 7 days prior to Screening 10. Pregnancy or breastfeeding 11. Hypersensitivity to murine proteins or to 1 of the excipients of ravulizumab 12. Previously or currently treated with a complement inhibitor
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the efficacy of ravulizumab plus BSC in the treatment of HSCT-TMA;Secondary Objective: Safety and tolerability of ALXN1210 and additional efficacy measures ;Primary end point(s): TMA response ;Timepoint(s) of evaluation of this end point: Throughout 26 Weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Time to TMA response 2. Change from baseline in TMA-associated organ dysfunctiond in renal system, cardiovascular system, pulmonary system, CNS, and GI system at 6 months and 1 year 3. TMA relapse during the study 4. Overall survival at 6 months and 1 year 5. Non-relapse mortality ( 6. Platelet response 7. Hematologic response ;Timepoint(s) of evaluation of this end point: Week 26 and Week 52 | — |
Countries
Canada, France, Germany, Italy, Japan, Korea, Republic of, Spain, United Kingdom, United States
Contacts
Public ContactEuropean Clinical Trial Information
Alexion Europe SAS
Outcome results
None listed