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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03518203
Other study ID # 2018-7119C
Secondary ID
Status Completed
Phase Phase 2
First received
Last updated
Start date August 3, 2018
Est. completion date June 1, 2022

Study information

Verified date August 2023
Source Children's Hospital Medical Center, Cincinnati
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

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.


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.


Recruitment information / eligibility

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.

Study Design


Related Conditions & MeSH terms


Intervention

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.

Locations

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

Sponsors (1)

Lead Sponsor Collaborator
Children's Hospital Medical Center, Cincinnati

Country where clinical trial is conducted

United States, 

Outcome

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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