A Phase 2 Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Narsoplimab in Pediatric Patients (28 Days to ≤ 18 Years of Age.) With High-Risk Hematopoietic Stem Cell Transplant Thrombotic Microangiopathy
Omeros Corporation
Summary
The purpose of this study is to evaluate the safety and efficacy of narsoplimab in pediatric patients with thrombotic microangiopathies (TMA) following hematopoietic stem cell transplant (HSCT).
Description
This is a Phase 2, uncontrolled, single-dosing regimen study in pediatric patients from 28 days to less than 18 years of age with high risk HSCT-TMA. At least 4 patients will be required from each of 3 age cohorts: 28 days to \<2 years of age, 2 years to \<12 years of age, and 12 years to \<18 years of age. Treatment will be for 8 weeks and patients will be followed for up to 52 weeks.
Eligibility
- Age range
- 0–17 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Age at least 28 days and less than 18 years prior to informed consent (Visit 0). 2. Have informed consent from at least one parent or legal guardian as required by local law and regulation. Patient informed consent will be required if the patient has reached the local legal age of majority. 3. Assent from patients as required by local law and regulation. 4. Have received an allogeneic hematopoietic stem cell transplant for the treatment of benign or malignant disease. 5. Have a diagnosis of HSCT-TMA defined as meeting both of the following criteria: * Platelet count…
Interventions
- DrugBiological: narsoplimab
Treatment with narsoplimab 4 mg/kg will be administered
Locations (16)
- Omeros Investigational SiteSan Diego, California
- Omeros Investigational SiteGainesville, Florida
- Omeros Investigational SiteBoston, Massachusetts
- Omeros Investigational SiteSt Louis, Missouri
- Omeros Investigational SiteNew York, New York
- Omeros Investigational SiteValhalla, New York