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Efficacy and Safety Study of Narsoplimab in Pediatric Patients With High-Risk Hematopoietic Stem Cell Transplant TMA | Clinical Trials | Clareo Health

RECRUITINGPHASE2

Efficacy and Safety Study of Narsoplimab in Pediatric Patients With High-Risk Hematopoietic Stem Cell Transplant TMA

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

NCT05855083•Omeros Corporation

View on ClinicalTrials.gov

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).

Detailed 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

0 - 17 years

Sex

ALL

Healthy Volunteers

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 \< 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 weeks following modification of calcineurin inhibitors or sirolimus OR
•* Have evidence of high-risk HSCT-TMA defined as at least one of the following:
+10 more criteria

Exclusion Criteria

•1. All treatments for HSCT-TMA are allowed except eculizumab, ravulizumab, and defibrotide within 3 months prior to informed consent, unless failure of therapy can be documented.
•a. Patients may not be on eculizumab, ravulizumab, or defibrotide for any indication at screening.
•2. Have Shiga toxin-producing Escherichia coli haemolytic uraemic syndrome (STEC-HUS). Test results obtained within 28 days prior to informed consent may be used.
•3. Have ADAMTS13 activity \< 10%. Test results obtained within 28 days prior to informed consent may be used.
•4. Have a severe, uncontrolled systemic bacterial or fungal infection requiring antimicrobial therapy, or a severe uncontrolled viral infection (as determined by the investigator); prophylactic antimicrobial therapy administered as standard of care is allowed.
•5. Have malignant hypertension (blood pressure \[BP\] \> 99th percentile plus 5 mmHg with bilateral hemorrhages or "cotton-wool" exudates on fundoscopic examination).
•6. Due to conditions other than HSCT-TMA, have a poor prognosis with a life expectancy of less than 3 months in the opinion of the Investigator.
•7. If pregnant or lactating.
•8. Have received treatment with an investigational drug or device within 4 weeks of entering study.
•9. Have abnormal liver function tests defined as alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> 5 times ULN within 28 days prior to informed consent.
+4 more criteria

Locations (16)

Omeros Investigational Site

San Diego, California, United States

Omeros Investigational Site

Gainesville, Florida, United States

Omeros Investigational Site

Boston, Massachusetts, United States

Omeros Investigational Site

St Louis, Missouri, United States

Omeros Investigational Site

New York, New York, United States

Omeros Investigational Site

Valhalla, New York, United States

Omeros Investigational Site

Houston, Texas, United States

Omeros Investigational Site

Seattle, Washington, United States

Omeros Investigational Site

Halle, Germany

Omeros Investigational Site

Hanover, Germany

Key Dates

Start Date

May 1, 2023

Primary Completion Date

December 1, 2025

Completion Date

December 1, 2025

Last Updated

March 25, 2025

Enrollment

ESTIMATED participants

Conditions

Thrombotic MicroangiopathiesHematopoietic Stem Cell Transplantation

Interventions

Biological: narsoplimab

DRUG

Contacts

Omeros Clinical Trial Information

CONTACT

206-676-5000 ctinfo@omeros.com

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 25, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

Efficacy and Safety Study of Narsoplimab in Pediatric Patients With High-Risk Hematopoietic Stem Cell Transplant TMA

Inclusion Criteria

Exclusion Criteria

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