EUCTR2019-004602-94-BE进行中（未招募）1 期
A 3-part, Randomized, Double Blind, Adaptive Seamless Phase 1-3 Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of BIO101 in Non-Ambulatory Patients with a Genetically Confirmed Diagnosis of Duchenne Muscular Dystrophy and Evidence of Respiratory Deterioration - Adaptive Seamless Phase 1-3 Study of Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of BIO
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Biophytis
- 入组人数
- 260
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
- Male
入选标准
- •1. Genetically confirmed DMD
- •2. Age 12 and above at screening
- •3. Non-ambulant patients (The non-ambulatory patient is defined as non-ambulant for at least 6 months with inability to walk 10 meters without assistance, by another person or device)
- •4. FVC =80% and >45% of predicted value based on most recent assessment noted in the patient's medical record and subsequently confirmed at Screening and at Baseline and who, in the opinion of the investigator are in the respiratory function decline phase.
- •5. Stable cardiac function, defined as:
- •No clinically important ECG abnormalities.
- •Echocardiography left ventricular ejection fraction (LVEF) at screening >50%.
- •6. If clinically indicated, approved concomitant treatment for DMD is allowed (e.g., Eteplirsen [Exondys 51™], Golodirsen [Vyondys 53™], Ataluren [Translarna™])
- •7. On or off corticosteroid therapy
- •If on steroids, including but not limited to Prednisone/ Prednisolone and Deflazacort [Emflaza®]:
- •o At a minimum dose of 0.3 mg/Kg/day prednisone equivalent
- •o Deflazacort approximately 0.9 mg/Kg/Day
- •o As long as the treatment is stable (i.e., unchanged and with no significant side effects) for at least 3 months.
- •o As long as the treatment is stable (i.e., unchanged and with no significant side effects) for at least 3 months.
- •Participants who were treated with corticosteroids and stopped (for any reason) will wait for 3 months until they can be eligible for inclusion.
- •Steroid treatment is expected to remain stable during study, except for adjustment for weight.
- •8. Participants on antihypertensive, lipid lowering, and/or thyroid replacement therapies, carnitine, creatineare eligible only if they are on a stable dose for at least 1 month prior to screening.
- •9. Stable hepatic function:
- •Gamma-glutamyl transferase (GGT) = upper limit of normal (ULN)
- •Total bilirubin < 2×ULN
- •GLDH < 2×ULN
- •Alkaline phosphatase =ULN
- •Serum albumin = lower limit of normal (LLN)
- •10. Ability to provide reliable and reproducible repeat PEF within 15% of the first assessment (i.e. Baseline vs. Screening)
- •11. Ability to provide reliable and reproducible repeat FVC within 15% of the first assessment (i.e. Baseline vs. Screening)
- •12. Willing and able to participate and comply with all study procedures.
- •For participants aged <18: Parent or legal guardians will sign an informed consent prior to start of screening procedures and patient will sign an informed ascent.
- •For participants aged 18 and about – patient will sign an informed consent.
- •13. Must be accompanied, during the whole study activities, by a parent, guardian or a caregiver, who is 18 years or older.
- •14. Sexually active participants must use double contraception (e.g. the male uses a condom and the female uses an oral contraceptive pills)
- •15. For France only: Being affiliated with a European Social Security.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 36
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Participants will not be eligible to participate in any part of this study (Part 1, Part 2, or Part 3) if one or more of the following is/are met:
- •1. Any serious medical/surgical or psychiatric condition/illness that in the opinion of the Investigator would jeopardize participant’s safety or would interfere with the study assessments/results.
- •2. History or active signs or symptoms of gallbladder/biliary disease (e.g., previous episodes of cholestasis/biliary tract obstruction, cholelithiasis, cholecystitis). Of note, history of cholecystectomy and no active biliary signs or symptoms, is not an exclusion criterion.
- •3. Any known allergies to products likely to be used in the study (e.g., antiseptics, anesthetics), known hypersensitivity to any of the ingredients, or excipients of the study drug.
- •4.Participation in other investigational study within 30 days or 5 half lives (whichever is longer) prior to randomization.
- •5. Patients undergoing Idebenone (Raxone ®)
- •6. Intellectual disability or behavioral problem such that he cannot comply with study procedures.
- •7. Patients who require daytime invasive ventilatory assistance(defined as use of any assisted ventilation while awake).
- •8. Renal disease requiring dialysis, or known renal insufficiency (moderate or severe reduction of eGFR=30 ml/min/1.73 m2, based on Cockroft & Gault formula)
- •9. Asthma, bronchiolitis/COPD, bronchiectasis, emphysema, pneumonia or the presence of any other non-DMD respiratory illness that affects PEF.
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