Health TrialsbyHealthPanel

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

ConditionsThrombotic MicroangiopathiesHematopoietic Stem Cell Transplantation
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age28-17
SponsorOmeros Corporation

About this trial

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).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Age at least 28 days and less than 18 years prior to informed consent (Visit 0).

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.

Assent from patients as required by local law and regulation.

Have received an allogeneic hematopoietic stem cell transplant for the treatment of benign or malignant disease.

Disqualifiers

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.

Have Shiga toxin-producing Escherichia coli haemolytic uraemic syndrome (STEC-HUS). Test results obtained within 28 days prior to informed consent may be used.

Have ADAMTS13 activity < 10%. Test results obtained within 28 days prior to informed consent may be used.

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.

Trial design

Design model

Single group

Treatments tested in this trial

  • Biological: narsoplimab

    Drug

    Treatment with narsoplimab 4 mg/kg will be administered

Treatment groups

18 Participants
are divided into 1 treatment group
Group A: Narsoplimab single arm-treatmentExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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

Secondary outcomes

1

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
2

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
3

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
4

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Omeros Corporation

Lead sponsor

Source ClinicalTrials.gov
Apply now