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
Verified date | August 2023 |
Source | Children's Hospital Medical Center, Cincinnati |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 23 |
Est. completion date | June 1, 2022 |
Est. primary completion date | April 4, 2022 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
United States | Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio |
United States | Children's Hospital Los Angeles (CHLA) | Los Angeles | California |
United States | Children's Hospital of Philadelphia (CHOP) | Philadelphia | Pennsylvania |
Lead Sponsor | Collaborator |
---|---|
Children's Hospital Medical Center, Cincinnati |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Survival | Survival at 6 months after the date of TMA diagnosis | 6 months | |
Secondary | Number of Participants With Organ Dysfunction | Number of participants with organ dysfunction at 6 months after TMA diagnosis. Organ dysfunction definitions are listed in the protocol Appendix II that is uploaded to ClinicalTrials.gov site. | 6 months | |
Secondary | Number of Participants With Organ Dysfunction | Number of participants with organ dysfunction at 1 year after TMA diagnosis. Organ dysfunction definitions are listed in the protocol Appendix II that is uploaded to ClinicalTrials.gov site. | 1 year | |
Secondary | Non-relapse Mortality | Non-relapse mortality descriptively compared with historical controls at 1 year | 1 year |
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