NL-OMON56242招募中2 期
A Phase 2 Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Narsoplimab in Paediatric Patients (28 Days to 18 Y.O.) with High Risk Haematopoietic Stem Cell Transplant Thrombotic Microangiopathy - OMS721
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 2
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 至 17（—）
入选标准
- •1. Age at least 28 days and less than 18 years prior to informed consent (Visit
- •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 haematopoietic stem cell transplant for the
- •treatment of non-malignant or malignant disease. All donor cell sources are
- •allowed (i.e., matched, mismatched, and haploidentical; related and unrelated;
- •bone marrow, peripheral blood stem cells, and umbilical cord blood).
- •5. Have a diagnosis of HSCT-TMA defined as having both of the following:
- •-Platelet count < 50,000/µL 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
- •- 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 (e.g., confusion, stroke, transient
- •ischemic attack [TIA] or seizures)
- •- 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)
- •7. If sexually active and of childbearing potential, must agree to practice a
- •highly effective method of birth control throughout the study drug treatment
- •and for at least 12 weeks after the last dose of study drug, such method of
- •birth control defined as one that results in a low failure rate (i.e., less
- •than 1% per year) when used consistently and correctly, such as implants,
- •injectables, combined oral contraceptives, some intrauterine devices, sexual
- •abstinence (abstinence is acceptable when it is in line with the patient*s
- •preferred and usual lifestyle and is defined as complete abstinence of sexual
- •intercourse, not periodic abstinence or withdrawal), or vasectomized partner.
- •8. Male patients must be willing to avoid fathering children for at least 12
- •weeks following the last dose of study medication.
排除标准
- •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
- •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 (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 through prior to the first dose.
- •10. Have a positive test by antigen, antibody, 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.
- •11. Patients or their parents or legal guardians are an employee of Omeros,
- •Clinical Research Organization (CRO), an Investigator, a study staff member, or
- •an immediate family member.
- •12. Have a known hypersensitivity to any constituent of the product.
- •13. Presence of any condition that the Investigator believes would put the
- •patient at risk.
研究者
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