Clinical Trials Logo
  1. Home
  2. Other
  3. NCT03518203

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

Atypical Hemolytic Uremic Syndrome Clinical Trial

Official title:
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

Clinical Trial 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.

Clinical Trial Description

This clinical trial is a prospective single arm multi-institution study in children and young adults undergoing allogeneic or autologous hematopoietic stem cell transplantation who will receive early therapy with eculizumab to prevent TMA-associated MODS after transplantation. The purpose of this research study is to examine efficacy of complement blocker eculizumab in HCT recipients with high risk TMA and to determine optimal eculizumab dosing regimen for HCT recipients with TMA using PK/PD studies. All patients will receive therapy based on their weight for 24 weeks. Survival will be assessed at 6 months from TMA diagnosis. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03518203
Study type Interventional
Source Children's Hospital Medical Center, Cincinnati
Contact
Status Completed
Phase Phase 2
Start date August 3, 2018
Completion date June 1, 2022
View Details
See also
  Status Clinical Trial Phase
Recruiting NCT03605511 - TTP and aHUS in Complicated Pregnancies
Withdrawn NCT03303313 - A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome Phase 2
Recruiting NCT04861259 - A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Phase 3
Recruiting NCT06065852 - National Registry of Rare Kidney Diseases
Terminated NCT02614898 - Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment
Completed NCT02574403 - Study Assessing an Algorithm-based Strategy of Eculizumab Discontinuation in Children and Adults With aHUS Phase 4
Recruiting NCT04958265 - A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Pediatric Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Phase 3
Recruiting NCT05795140 - Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS Phase 3
Completed NCT00844844 - Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Resistant aHUS Phase 2
Completed NCT00844545 - Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-Resistant aHUS Phase 2
Terminated NCT01522170 - aHUS Observational Long Term Follow-Up N/A
Terminated NCT02464891 - Complement Inhibition in aHUS Dialysis Patients Phase 2
Withdrawn NCT02626663 - The Role of Microparticles as a Biomarker
Recruiting NCT01793168 - Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Completed NCT00844428 - Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Sensitive aHUS Phase 2
Completed NCT00838513 - Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-sensitive Atypical Hemolytic Uremic Syndrome (aHUS) Phase 2
Withdrawn NCT03999840 - Eculizumab to Cemdisiran Switch in aHUS Phase 2
Recruiting NCT04889430 - Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy Phase 3
Recruiting NCT05935215 - Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Phase 3
Recruiting NCT03205995 - Safety and Efficacy Study of OMS721 in Patients With Atypical Hemolytic Uremic Syndrome Phase 3