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Tests treatment safety and results for Thrombotic Microangiopathies
Official title Efficacy and Safety Study of Narsoplimab in Pediatric Patients With High-Risk Hematopoietic Stem Cell Transplant TMA
ClinicalTrials.gov ID: NCT05855083
What this study is testing
What is Biological: narsoplimab?
Biological: narsoplimab is an investigational medicine, being studied as a potential treatment for thrombotic microangiopathies.
Also referred to as Narsoplimab.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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).
- Phase 2: a mid-size study of how well it works
- Time commitment: about 52 weeks
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 28 to 17
You may be able to join if
- 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...
- 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.
- Have a diagnosis of HSCT-TMA defined as meeting both of the following criteria:
You likely can't join if
- All treatments for HSCT-TMA are allowed except eculizumab, ravulizumab, and defibrotide within 3 months prior to informed consent, unless failure of...
- Have Shiga toxin-producing Escherichia coli haemolytic uraemic syndrome (STEC-HUS). Test results obtained within 28 days prior to informed consent...
- 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...
- Have malignant hypertension (blood pressure [BP] \> 99th percentile plus 5 mmHg with bilateral hemorrhages or "cotton-wool" exudates on fundoscopic...
- 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.
See the full eligibility criteria
- 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.
- Have a diagnosis of HSCT-TMA defined as meeting both of the following criteria:
- 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])
- 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:
- Spot protein/creatinine ratio \> 2 mg/mg
- Serum creatinine \> 1.5 x the creatinine level prior to TMA development
- Biopsy-proven gastrointestinal TMA
- TMA-related neurological abnormality
- Pericardial or pleural effusion without alternative explanation
- Pulmonary hypertension without alternative explanation
- Have Grade III or Grade IV graft-versus-host disease (GVHD) or, in the opinion of the Investigator, risk for development of Grade III or Grade IV GVHD if immunosuppression were to be modified
- Have elevated serum C5b-9 (\> 244 ng/mL)
- If sexually active and of childbearing potential (for female pediatric patients, defined as starting at onset of menses), must agree to practice a highly effective method of birth control throughout study drug treatment...
- Male patients must be willing to avoid fathering children for at least 12 weeks following the last dose of study medication.
- 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...
- 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...
- Have malignant hypertension (blood pressure [BP] \> 99th percentile plus 5 mmHg with bilateral hemorrhages or "cotton-wool" exudates on fundoscopic examination).
- 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.
- If pregnant or lactating.
- Have received treatment with an investigational drug or device within 4 weeks of entering study.
- Have abnormal liver function tests defined as alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> 5 times ULN within 28 days prior to informed consent.
- Have a positive test by antigen or polymerase chain reaction (PCR) for human immunodeficiency virus (HIV), if negative within 28 days prior to informed consent, the test does not need to be repeated.
- Patient or one or more of the patient's parents or legal guardians are is an employee or an immediate family member of Omeros, the Clinical Research Organization (CRO), an Investigator, or a study staff member.
- Have a known hypersensitivity to any constituent of the product.
- Presence of any condition that the Investigator believes would put the patient at risk.
The study team makes the final eligibility decision.
Where it's taking place
- San Diego, California, United States
- Gainesville, Florida, United States
- Boston, Massachusetts, United States
- St Louis, Missouri, United States
- New York, New York, United States
- Valhalla, New York, United States
- Houston, Texas, United States
- Seattle, Washington, United States
- Halle, Germany
- Hanover, Germany
- Haifa, Israel
- Jerusalem, Israel
- Ramat Gan, Israel
- Tel Aviv, Israel
- Utrecht, Netherlands
- Pamplona, Spain
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 52 weeks per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 28 days to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include San Diego, California, United States; Gainesville, Florida, United States; Boston, Massachusetts, United States; St Louis, Missouri, United States; New York, New York, United States; Valhalla, New York, United States and 10 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.