CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 23 enrolled
Drug / intervention
Eculizumabdrug
Likely dose
Eculizumab 300 mgfrom record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT03518203
NCT03518203Phase 2Completed
CompareCT.gov

Early Intervention With Eculizumab to Treat Thrombotic Microangiopathy/Atypical Hemolytic Uremic Syndrome (TMA/aHUS)-Associated Multiple Organ Dysfunction Syndrome (MODS) in Hematopoietic Stem Cell Transplant (HCT) Recipients

Children's Hospital Medical Center, Cincinnati·interventional·Posted May 8, 2018·Updated Sep 21, 2023

In Brief

A Phase 2 clinical trial evaluating Eculizumab for Thrombotic Microangiopathies and 2 related conditions. Completed, enrolled 23 participants across 3 sites.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsThrombotic Microangiopathies, Atypical Hemolytic Uremic Syndrome, Multiple Organ Dysfunction Syndrome
CountriesUnited States
Collaborators--

Timeline

Phase 2CompletedFinished
20192020202120222023202420252026
First PostedMay 8, 2018
Enrollment StartAug 3, 2018
Primary CompletionApr 4, 2022
Study CompletionJun 1, 2022
TodayJul 2, 2026
Enrollment to primary: 3.7 yearsPosted 8.2 years ago

Interventions

Eculizumabdrug

Eculizumab will be administered as intravenous infusion (IV) over 60 minutes. The dosage form will be 300 mg single-use vials each containing 30 mL of 10 mg/mL sterile, preservative-free solution.