Phase 2
N=23
Eculizumab to Treat Thrombotic Microangiopathy/Atypical Hemolytic Uremic Syndrome -Associated Multiple Organ Dysfunction Syndrome in Hematopoietic Stem Cell Transplant Recipients
Thrombotic Microangiopathies · Atypical Hemolytic Uremic Syndrome · Multiple Organ Dysfunction Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT03518203 ↗Enrolled (actual)
23
Serious AEs
82.6%
Results posted
Sep 2023
Primary outcome: Primary: Survival — 15 Participants — p=<0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Eculizumab (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Children's Hospital Medical Center, Cincinnati
- Primary completion
- Apr 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Survival |
15 | <0.0001 sig |
| SECONDARY Number of Participants With Organ Dysfunction |
2 | — |
| SECONDARY Number of Participants With Organ Dysfunction |
2 | — |
| SECONDARY Non-relapse Mortality |
8 | — |
Summary
Hematopoietic stem cell transplantation (HCT)-associated thrombotic microangiopathy (TMA) is an understudied complication of HCT that significantly affects transplant related morbidity and mortality. The investigators hypothesize that early intervention with complement blocker eculizumab will double survival in HCT recipients with high risk TMA, as compared to historical untreated controls. An optimal eculizumab dosing schedule can be determined for this population through eculizumab pharmacokinetic/pharmacodynamic (PK/PD) testing.
Eligibility Criteria
Inclusion Criteria
- Patients of any age undergoing allogeneic or autologous HCT
- Histologic TMA diagnosis OR clinical TMA diagnosis and presenting with high risk disease features including elevated plasma sC5b-9 above laboratory normal value (≥244ng/ml) and proteinuria measured as ≥30mg/dL of protein on random urinalysis x2 or protein/creatinine ratio ≥1mg/mg or patient receiving renal replacement therapy.
- Minimum weight of ≥ 5kg.
Exclusion Criteria
- Known hypersensitivity to any constituent of the study medication.
- Subjects with unresolved serious Neisseria meningitides infection or progressive severe infection.
- Patients with diagnosis of TTP as defined by ADAMST13 activity test <10%.
- Patients previously treated with eculizumab or other complement blocker for TMA within the 60 days prior to first dose of study treatment.
Data sourced from ClinicalTrials.gov (NCT03518203). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.