EUCTR2020-001350-21-CZ进行中（未招募）1 期
An Open-Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 12
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Between the ages of 12 and 17, inclusive, at time of study entry.
- •2. A diagnosis of PNH, confirmed by high-sensitivity flow cytometry (granulocyte or monocyte clone >10%).
- •3. Be either a naïve patient or a switch patient, as defined below.
- •a. A naïve patient must:
- •i. Not be currently receiving an approved complement inhibitor, and must not have received a complement inhibitor within at least 5 half-lives of that drug prior to starting pegcetacoplan
- •ii. Have evidence of a hemolytic anemia based on a hemoglobin less than the lower limit of the normal range (LLN), and LDH >1.5 × ULN.
- •b. A switch patient must:
- •i. Be currently receiving treatment with an approved complement inhibitor, and the dose of that inhibitor must have been stable for at least 5 half-lives of that drug
- •ii. Have evidence of anemia based on a hemoglobin less than the LLN.
- •iii. Have ARC > ULN.
- •4. Platelet count >75,000/mm3.
- •5. Absolute neutrophil count >1000/mm3.
- •6. Weigh at least 20 kg.
- •7. Have a body mass index (BMI) that is less than the 95th percentile for their age.
- •8. Either not receiving the following medications, or on a stable regimen for at least the minimum time period indicated below, prior to the first screening visit, with no anticipated changes to the regimen over the course of the study:
- •a. Erythropoietin: 8 weeks
- •b. Systemic corticosteroids: 4 weeks
- •c. Immunosuppressants (other than steroids): 8 weeks
- •d. Vitamin K antagonists (eg, warfarin): 4 weeks, with a stable international normalized ratio (INR) over that period
- •e. Iron supplements, vitamin B12, or folic acid: 4 weeks
- •f. Low-molecular weight heparin or direct oral anticoagulants (DOACs): 4 weeks
- •9. Have received vaccinations against Neisseria meningitidis (types A, C, W, Y, and B), Streptococcus pneumoniae, and Haemophilus influenzae (type B) prior to dosing on Day 1, or agree to receive vaccinations within 14 days after starting treatment with pegcetacoplan. Vaccination is mandatory, unless there is documented evidence of titers within acceptable local limits, or documented evidence of nonresponse to vaccination based on titers. Subjects receiving vaccinations after starting pegcetacoplan must be willing to take prophylactic antibiotics from the first day of treatment with pegcetacoplan until at least 2 weeks after vaccination as described in Section 8.2.1.
- •10. Female subjects of childbearing potential must have a negative blood pregnancy test at screening (and negative urine pregnancy test on Day 1) and must agree to practice abstinence or to use another protocol-defined method of contraception, as described in Section 10.3.5.1, from screening through at least 90 days after receiving the last dose of pegcetacoplan.
- •11. Male subjects who have reached sexual maturity must agree to practice abstinence or to use another protocol-defined method of contraception, as described in Section 10.3.5.1, and agree to refrain from donating semen from screening through at least 90 days after receiving the last dose of pegcetacoplan.
- •12. Willing and able to self-administer pegcetacoplan or has a caregiver who is willing and able to do so.
- •13. The subject or their legally authorized representative must be willing and able to provide written informed consent as described in Section 12.1.2, including compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. Where appropriate, the subject must also give their assent to participation in the study.
排除标准
- •1. Known or suspected hereditary fructose intolerance (HFI).
- •2. Active bacterial infection that has not resolved within at least 1 week before the first dose of pegcetacoplan.
- •3. Hereditary complement deficiency.
- •4. History of bone marrow transplantation.
- •5. History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the formulation or SC administration of pegcetacoplan.
- •6. Participation in another investigational drug trial or exposure to another investigational agent, device, or procedure within 30 days or 5 half-lives (whichever is longer) from the last dose of investigational agent prior to screening period.
- •7. Planning to become pregnant during study participation, or currently breastfeeding.
- •8. History of meningococcal disease.
- •9. Inability to cooperate, or any condition that, in the opinion of the investigator makes the subject inappropriate for the study or could confound the outcome of the study.
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