Duchenne Muscular Dystrophy regardless the genotype MedDRA version: 20.1 Level: PT Classification code 10052655 Term: Duchenne muscular dystrophy gene carrier System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Code: BIO101
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: BIO101
CAS Number: 5289-74-7
Current Sponsor code: BIO101
Other descriptive name: BIO101
Concentration unit: m
Sponsors
Biophytis
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Part 1 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 =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Part 1 To evaluate the safety, tolerability and PK profile of BIO101 and its main metabolites after a single dose (Day 1) and after multiple doses at Day 7, 14 and 56 Part 2 To evaluate the safety, tolerability, and efficacy on respiratory function, of BIO101 following 48 weeks double blind dosing, in a small population (48 participants) Part 3 To evaluate the safety, tolerability, and efficacy on respiratory function of BIO101 following 48 weeks double blind dosing, in a large population (100-200 participants);Secondary Objective: Part 2 Population PK analysis to characterize the PK of BIO101 following 48 weeks double blind dosing To determine the number of new participants to enroll into confirmatory Part 3 Part 3 To further evaluate efficacy of BIO101 versus placebo and external controls on strength and respiratory function of participants according to their initial disease status To evaluate the safety and tolerability of BIO101 versus placebo ;Primary end point(s): Efficacy: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF % predictive) at Week 48 (assessed by hospital-based spirometry measurements) ;Timepoint(s) of evaluation of this end point: During the trial | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Efficacy: Change From Baseline in Forced Vital Capacity (FVC% predictive) at Week 48 (assessed by hospital-based spirometry measurements) Safety: Type, frequency, intensity, timing, and relationship to study drug of: • Adverse events (AEs), discontinuations due to AEs, serious AEs (SAEs), deaths • 12-lead triplicate electrocardiograms (ECGs) • Local Clinical laboratory tests • Physical examination and vital signs • Anthropometry Pharmacokinetics: • Non-compartmental PK parameters including maximum concentration (Cmax), time to Cmax (tmax), area under the curve (AUC) and other parameters for participants at Part 1: o On Day 1 (pre-treatment) o On Day 7, 14 and 56 • Population PK parameters including apparent clearance and volume of distribution (for participants of parts 2 and Part 3) ;Timepoint(s) of evaluation of this end point: During this trial | — |
Countries
Belgium
Contacts
Public ContactMounia Chabane De Saint Aubin
Biophytis
Outcome results
None listed