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

Recruiting at 2 trial locations

Overseen By Monica Trapp, BSN, RN

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Children's Hospital Medical Center, Cincinnati

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This trial tests eculizumab, a drug that blocks part of the immune system to prevent blood vessel damage, in children and young adults undergoing stem cell transplants who are at high risk for a serious complication called TMA. The goal is to see if early treatment with eculizumab improves survival rates. Eculizumab has been used in the treatment of transplant-associated thrombotic microangiopathy (TA-TMA) and has shown benefits in several studies, though results remain controversial.

Research Team

Sonata Jodele, MD

Principal Investigator

Children's Hospital Medical Center, Cincinnati

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.

Treatment Details

Interventions

• Eculizumab (Monoclonal Antibodies)

Participant Groups

Group I: Eculizumab Experimental Treatment 1 Intervention

All patients will receive eculizumab based on their weight for 24 weeks.

Who Is Running the Clinical Trial?

Children's Hospital Medical Center, Cincinnati

Lead Sponsor

Trials

844

Recruited

6,566,000+