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

• Register: ClinicalTrials.gov
• Last refreshed on: 3 October 2023
• Main ID: NCT03518203
• Date of registration: 05/03/2018
• Prospective Registration: Yes
• Primary sponsor: Children's Hospital Medical Center, Cincinnati
• Public title: Eculizumab to Treat Thrombotic Microangiopathy/Atypical Hemolytic Uremic Syndrome -Associated Multiple Organ Dysfunction Syndrome in Hematopoietic Stem Cell Transplant Recipients
• Scientific 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
• Date of first enrolment: August 3, 2018
• Target sample size: 23
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03518203
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Sonata Jodele, MD
• Affiliation: Children's Hospital Medical Center, Cincinnati

Key inclusion & exclusion 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.

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.



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Thrombotic Microangiopathies

Atypical Hemolytic Uremic Syndrome

Multiple Organ Dysfunction Syndrome

Intervention(s)

Drug: Eculizumab

Primary Outcome(s)

Survival [Time Frame: 6 months]

Secondary Outcome(s)

Number of Participants With Organ Dysfunction [Time Frame: 6 months]

Non-relapse Mortality [Time Frame: 1 year]

Number of Participants With Organ Dysfunction [Time Frame: 1 year]

Secondary ID(s)

2018-7119C

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 21/09/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03518203