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Eculizumab to Treat Thrombotic Microangiopathy/Atypical Hemolytic Uremic Syndrome -Associated Multiple Organ Dysfunction Syndrome in Hematopoietic Stem Cell Transplant Recipients

NCT03518203 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 23

Last updated 2023-09-21

Study results available
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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.

Conditions

  • Thrombotic Microangiopathies
  • Atypical Hemolytic Uremic Syndrome
  • Multiple Organ Dysfunction Syndrome

Interventions

DRUG

Eculizumab

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.

Sponsors & Collaborators

  • Children's Hospital Medical Center, Cincinnati

    lead OTHER

Principal Investigators

  • Sonata Jodele, MD · Children's Hospital Medical Center, Cincinnati

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-08-03
Primary Completion
2022-04-04
Completion
2022-06-01
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03518203 on ClinicalTrials.gov