NCT05855083 · Omeros Corporation
Efficacy and Safety Study of Narsoplimab in Pediatric Patients With High-Risk Hematopoietic Stem Cell Transplant TMA
What this study is about
The purpose of this study is to evaluate the safety and effectiveness of narsoplimab in pediatric patients with thrombotic microangiopathies (TMA) following hematopoietic stem cell transplant (HSCT).
View original scientific description
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).
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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 \< 50,000/mL or a decrease in platelet count \> 50% from the highest value obtained following transplant.
- Evidence of microangiopathic hemolysis (presence of schistocytes, serum lactate dehydrogenase \[LDH\] \> upper limit of normal (\[ULN\], or haptoglobin \< lower limit of normal \[LLN\]) 6. Have at least one of the following HSCT-TMA high-risk criteria:
- HSCT-TMA persistence \> 2 wee
Where
- San Diego, California
- Gainesville, Florida
- Boston, Massachusetts
- St Louis, Missouri
- New York, New York
- Valhalla, New York
- Houston, Texas
- Seattle, Washington
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Mar 25, 2025 · Source of record for eligibility and locations