Actively Recruiting
A Phase 2 Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Narsoplimab in Pediatric Patients With High-Risk Hematopoietic Stem Cell Transplant Thrombotic Microangiopathy
Led by Omeros Corporation · Updated on 2025-03-25
18
Participants Needed
16
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating narsoplimab to study its safety and effectiveness in children with thrombotic microangiopathies (TMA) that happen after a hematopoietic stem cell transplant (HSCT). This Phase 2 study focuses on pediatric patients aged from 28 days up to less than 18 years with high-risk HSCT-TMA. The study includes three age groups: infants to under 2 years, children 2 to under 12 years, and adolescents 12 to under 18 years. Participants will receive a single dosing regimen of narsoplimab at 4 mg/kg for 8 weeks. The treatment is given to all participants without a comparison group. After treatment, patients will be monitored and followed for up to 52 weeks to assess longer-term effects and outcomes. During the study, participants will have survival rates evaluated at 100 days and up to one year after diagnosis of high-risk HSCT-TMA. Researchers will monitor adverse events, clinical response, pharmacokinetics, and immune response to the drug. The study involves regular medical assessments and safety monitoring throughout the treatment and follow-up periods, lasting up to one year in total.
CONDITIONS
Brief Title
Efficacy and Safety Study of Narsoplimab in Pediatric Patients With High-Risk Hematopoietic Stem Cell Transplant TMA
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age at least 28 days and less than 18 years prior to informed consent
- Informed consent from at least one parent or legal guardian; patient consent if of legal age
- Assent from patients as required by local law
- Received allogeneic hematopoietic stem cell transplant for benign or malignant disease
- Diagnosis of HSCT-TMA with platelet count below 50,000/mL or a decrease over 50% from highest post-transplant value
- Evidence of microangiopathic hemolysis (schistocytes, elevated LDH, or low haptoglobin)
- At least one high-risk HSCT-TMA criterion, such as persistence over 2 weeks after modifying calcineurin inhibitors or sirolimus, or evidence of high-risk features including proteinuria, elevated creatinine, biopsy-proven gastrointestinal TMA, neurological abnormalities, effusions, pulmonary hypertension, severe graft-versus-host disease, or elevated serum C5b-9
- If sexually active and of childbearing potential, agreement to use a highly effective birth control method during treatment and 12 weeks after
- Male patients must agree to avoid fathering children for 12 weeks after last dose
You will not qualify if you...
- Use of eculizumab, ravulizumab, or defibrotide within 3 months prior to consent unless documented therapy failure
- Presence of Shiga toxin-producing Escherichia coli hemolytic uremic syndrome (STEC-HUS)
- ADAMTS13 activity below 10%
- Severe uncontrolled bacterial, fungal, or viral infection requiring antimicrobial therapy
- Malignant hypertension with specific ocular findings
- Poor prognosis with life expectancy less than 3 months
- Pregnancy or lactation
- Treatment with investigational drug or device within 4 weeks prior to study
- Abnormal liver function tests with ALT or AST more than 5 times upper limit
- Positive HIV test unless confirmed negative within 28 days
- Employment or immediate family relation to study sponsor, research staff, or investigators
- Known allergy to any study drug components
- Any condition deemed by investigator to pose risk to patient
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 8 weeks
Participants receive narsoplimab treatment to address high-risk HSCT-TMA.
Visits scheduled during the 8-week treatment period
Duration - Up to 44 weeks
Participants are followed for safety and efficacy assessments after treatment ends.
Periodic follow-up visits up to 52 weeks from treatment start
Trial Site Locations
Total: 16 locations
1
Omeros Investigational Site
San Diego, California, United States, 92024
Actively Recruiting
2
Omeros Investigational Site
Gainesville, Florida, United States, 32608
Not Yet Recruiting
3
Omeros Investigational Site
Boston, Massachusetts, United States, 02215
Not Yet Recruiting
4
Omeros Investigational Site
St Louis, Missouri, United States, 63104
Actively Recruiting
5
Omeros Investigational Site
New York, New York, United States, 10065
Not Yet Recruiting
6
Omeros Investigational Site
Valhalla, New York, United States, 10595
Actively Recruiting
7
Omeros Investigational Site
Houston, Texas, United States, 77030
Not Yet Recruiting
8
Omeros Investigational Site
Seattle, Washington, United States, 98105
Not Yet Recruiting
9
Omeros Investigational Site
Halle, Germany
Not Yet Recruiting
10
Omeros Investigational Site
Hanover, Germany
Not Yet Recruiting
11
Omeros Investigational Site
Haifa, Israel
Not Yet Recruiting
12
Omeros Investigational Site
Jerusalem, Israel
Not Yet Recruiting
13
Omeros Investigational Site
Ramat Gan, Israel
Not Yet Recruiting
14
Omeros Investigational Site
Tel Aviv, Israel
Not Yet Recruiting
15
Omeros Investigational Site
Utrecht, Netherlands
Not Yet Recruiting
16
Omeros Investigational Site
Pamplona, Spain
Actively Recruiting
Research Team
O
Omeros Clinical Trial Information
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here