EUCTR2018-000358-23-IT进行中（未招募）1 期
A phase 3, multicenter, double-blind, placebo-controlled, randomized, outpatient two-period two-treatment crossover study to evaluate the efficacy and safety of amifampridine phosphate (3,4 diaminopyridine phosphate) in patients with Congenital Myasthenic Syndromes (CMS) - CMS-001
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
- 23
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
- •- Patient or parent willing and able to provide written informed consent after the nature of the study has been explained and before the start of any research-related procedures, or the patient’s legal guardian or caregiver with durable power of attorney can provide written informed consent. An assent form must also be signed if in the judgement of the IRB/IEC/REB the children are capable of providing assent
- •- Male or female age 2 years and above
- •- Body weight =10 kg
- •- Genetically-confirmed CMS involving acetylcholine receptor defect, Rapsyn deficiency, MuSK deficiency, Dok-7 deficiency, SYT2 mutations, SNAP25B deficiency, and fast channel syndrome
- •- In patients who are naïve to 3,4-DAP or amifampridine phosphate, MFM 20 or 32 score equal or less than 48 or 76, respectively, at Screening
- •- In patients who are naïve to 3,4-DAP or amifampridine phosphate, improvement of >20% in MFM 20 or MFM 32 scores after open label period of uptitration of dose
- •- In patients who are previously stabilized on 3,4-DAP or amifampridine phosphate, a history of meaningful improvement in motor function (in the opinion of the investigator)
- •- Willingness of patients receiving pyridostigmine, albuterol, ephedrine, or fluoxetine to remain on a stable dose of these medications throughout the study interval
- •- Female patients of childbearing potential must have a negative pregnancy test (serum human chorionic gonadotropin [HCG] at Screening); and must practice effective, reliable contraceptive regimen during the study. Acceptable methods of contraception include hormonal contraception (i.e., birth control pills, injected hormones, dermal patch or vaginal ring), intrauterine device, barrier methods (diaphragm, condom) with spermicide, or surgical sterilization (tubal ligation)
- •- Ability to participate in the study based on overall health of the patient and disease prognosis, as applicable, in the opinion of the investigator; and able to comply with all requirements of the protocol
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 7
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 14
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •- CMS subtype diagnosis of acetylcholinesterase deficiency, slow-channel syndrome, LRP4 deficiency, and plectin deficiency
- •- Cardiac conduction defects on Screening electrocardiogram (ECG)
- •- Seizure disorder
- •- Clinically significant abnormal laboratory values at Screening, in the opinion of the investigator
- •- Pregnancy or breastfeeding at Screening or planning to become pregnant at any time during the study
- •- Any systemic bacterial or other infection, which is clinically significant in the opinion of the investigator and has not been treated with appropriate antibiotics
- •- Treatment with an investigational drug (other than 3,4-DAP or amifampridine phosphate), device, or biological agent within 30 days before Screening or while participating in this study
- •- Any other medical condition that, in the opinion of the investigator, might interfere with the patient’s participation in the study, poses an added risk for the patient, or confounds the assessment of the patient
- •- History of drug allergy to any pyridine-containing substances or any amifampridine phosphate excipient(s)
研究者
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