Actively Recruiting
Efficacy and Safety Study of Narsoplimab in Pediatric Patients With High-Risk Hematopoietic Stem Cell Transplant TMA
Led by Omeros Corporation · Updated on 2025-03-25
18
Participants Needed
16
Research Sites
135 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official 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 obtained from at least one parent or legal guardian; patient consent if legally required
- Assent from patients as required by local regulations
- Received an allogeneic hematopoietic stem cell transplant for benign or malignant disease
- Diagnosed with HSCT-TMA with platelet count less than 50,000/mL or decrease in platelet count greater than 50% from highest post-transplant value
- Evidence of microangiopathic hemolysis (presence of schistocytes, elevated LDH, or low haptoglobin)
- At least one high-risk HSCT-TMA criterion such as persistent TMA 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, pericardial or pleural effusion without other cause, pulmonary hypertension without other cause, Grade III or IV graft-versus-host disease (GVHD) or risk of it if immunosuppression changed, or elevated serum C5b-9 levels
- If sexually active and of childbearing potential, agrees to use highly effective birth control during treatment and for 12 weeks after last dose
- Male patients 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 therapy failure documented
- Current use of eculizumab, ravulizumab, or defibrotide at screening
- Diagnosis of Shiga toxin-producing Escherichia coli hemolytic uremic syndrome (STEC-HUS)
- ADAMTS13 activity less than 10%
- Severe uncontrolled bacterial, fungal, or viral infections requiring therapy, except prophylactic antimicrobial treatment
- Malignant hypertension with specific blood pressure criteria and eye changes
- Poor prognosis with life expectancy under 3 months not related to HSCT-TMA
- Pregnancy or lactation
- Treatment with investigational drug or device within 4 weeks prior to study
- Abnormal liver function tests (ALT or AST greater than 5 times upper limit) within 28 days prior
- Positive HIV test without recent negative confirmation
- Employment or immediate family relationship with study sponsor, CRO, investigator, or staff
- Known hypersensitivity to study drug components
- Any condition that investigator believes would put the patient at risk
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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