NL-OMON54475尚未招募2 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 - APL2
Apellis0 个研究点目标入组 1 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 1
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
- 12 至 17（—）
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 nai*ve patient or a switch patient, as
- •defined below. a. A nai*ve 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 rest
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.
研究者
相似试验
进行中（未招募）
1 期
A single arm study to evaluate the safety and biological activity of pegcetacoplan in patients 12- 17 years old with paroxysmal nocturnal hemoglobinuriaEUCTR2020-001350-21-NLApellis Pharmaceuticals, Inc.12
进行中（未招募）
1 期
A single arm study to evaluate the safety and biological activity of pegcetacoplan in patients 12- 17 years old with paroxysmal nocturnal hemoglobinuriaParoxysmal Nocturnal HemoglobinuriaMedDRA version: 21.1Level: LLTClassification code 10055629Term: Paroxysmal nocturnal hemoglobinuriaSystem Organ Class: 100000004857EUCTR2020-001350-21-CZApellis Pharmaceuticals, Inc.12
进行中（未招募）
1 期
A Phase 2 Study to Evaluate the Effectivness and Safety of ADCT-301 in Patients with Relapsed or Refractory Hodgkin LymphomaRelapsed or Refractory Hodgkin LymphomaMedDRA version: 20.1Level: LLTClassification code 10080208Term: Classical Hodgkin lymphomaSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)MedDRA version: 20.0Level: LLTClassification code 10020328Term: Hodgkin's lymphomaSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)MedDRA version: 20.0Level: PTClassification code 10020267Term: Hodgkin's disease refractorySystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)MedDRA version: 21.0Level: PTClassification code 10020234Term: Hodgkin's disease mixed cellularity refractorySystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)MedDRA version: 21.0Level: PTClassification code 10020233Term: Hodgkin's disease mixed cellularity recurrentSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)EUCTR2018-002556-32-DEADC Therapeutics SA100
进行中（未招募）
1 期
A Phase 2 Study to Evaluate the Effectiveness and Safety of ADCT-301 in Patients with Relapsed or Refractory Hodgkin LymphomaEUCTR2018-002556-32-ESADC Therapeutics SA100
进行中（未招募）
1 期
A Phase 2 Study to Evaluate the Effectivness and Safety of ADCT-301 in Patients with Relapsed or Refractory Hodgkin LymphomaEUCTR2018-002556-32-PLADC Therapeutics SA100