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).
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
18
Treatment with narsoplimab 4 mg/kg will be administered
Omeros Investigational Site
San Diego, California, United States
RECRUITINGOmeros Investigational Site
Gainesville, Florida, United States
100-day survival rate following high-risk HSCT-TMA diagnosis.
Percentage of patients alive at 100 days following the diagnosis of high-risk HSCT-TMA
Time frame: 100 days
Number of participants with treatment-emergent adverse events assessed by CTCAE v5.0
Number and percentage of patients with treatment-emergent adverse events will be summarized by MedDRA system organ class and preferred term
Time frame: 52 weeks
Percentage of patients meeting protocol definition of clinical response
A responder is defined as a patient with HSCT-TMA who demonstrates improvement in laboratory TMA markers (platelet count and LDH) and clinical benefit (either improvement in organ function or reduction in transfusion burden)
Time frame: 52 weeks
52 week survival rate following high-risk HSCT-TMA diagnosis
Percentage of patients alive at 52 weeks following the diagnosis of high-risk HSCT-TMA
Time frame: 52 weeks
Overall survival following the diagnosis of high-risk HSCT-TMA
Median overall survival (days) following the diagnosis of high-risk HSCT-TMA by Kaplan-Meier estimate
Time frame: 52 weeks
Pharmacokinetics (PK) of multiple-dose administration of OMS721
PK parameters including maximum concentration and minimum (trough) concentration
Time frame: 52 weeks
Presence of anti-drug antibody (ADA)
Number and percentage of patient with at least one ADA positive sample
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Omeros Investigational Site
Boston, Massachusetts, United States
NOT_YET_RECRUITINGOmeros Investigational Site
St Louis, Missouri, United States
RECRUITINGOmeros Investigational Site
New York, New York, United States
NOT_YET_RECRUITINGOmeros Investigational Site
Valhalla, New York, United States
RECRUITINGOmeros Investigational Site
Houston, Texas, United States
NOT_YET_RECRUITINGOmeros Investigational Site
Seattle, Washington, United States
NOT_YET_RECRUITINGOmeros Investigational Site
Halle, Germany
NOT_YET_RECRUITINGOmeros Investigational Site
Hanover, Germany
NOT_YET_RECRUITING...and 6 more locations
Time frame: 52 weeks