Duchenne muscular dystrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: WVE-210201
Product Code: WVE-210201
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: WVE-210201
Current Sponsor code: WVE-210201
Concentration unit: mg milligram(s)
Concen
Sponsors
Wave Life Sciences Ltd.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Patient and/or parent or legal guardian must have the ability and be willing to provide written informed consent/minor assent prior to any study-related procedures. 2. Patient successfully completed the Phase 1 study with WVE-210201, WVE-DMDX51-001. 3. Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, study restrictions, and all study procedures. 4. Stable pulmonary and cardiac function, as measured by: a. Reproducible percent predicted forced vital capacity (FVC) =50% b. Left ventricular ejection fraction (LVEF) >55% in patients 45% in patients =10 years of age, as measured (and documented) by echocardiogram. 5. Sexually mature males must be willing to use contraception for the duration of the study, if the patient is sexually active. 6. Patient and caregivers must agree not to post any study-related information on social media Are the trial subjects under 18? yes Number of subjects for this age range: 40 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
Exclusion criteria
Exclusion criteria: 1. Clinically significant medical finding on the physical examination other than DMD that, in the judgment of the Investigator will make the patient unsuitable for participation in, and/or unable to complete the study procedures. 2. Other prior or ongoing medical conditions including: a. Acute illness within Screening period; b. Abnormal physical findings, other than those associated with musculoskeletal findings attributable to DMD. 3. Laboratory abnormality, that, in the Investigator's opinion, could adversely affect the safety of the patient, make it unlikely that the course of treatment or follow up would be completed, or impair the assessment of study results. These include, but are not limited to: a. Renal insufficiency; b. Impaired hepatic function as measured by glutamate dehydrogenase (GLDH) = 2.5x upper limit of normal (ULN) and Bilirubin = 2x ULN (or INR = 1.5x ULN; c. Activated partial thromboplastin time [aPTT] values above the ULN; d. Platelet count less than lower limit of normal (LLN). e. Any evidence of clinically significant structural or functional heart abnormality would prohibit participation in this study. f. Troponin I value above 2x ULN 4. Parent or legal guardian is directly or indirectly involved in the conduct and administration of this study as an Investigator, sub-investigator, study coordinator, or other study staff member, or the patient is a first-degree family member, significant other, or relative residing with one of the above persons involved directly or indirectly in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of WVE-210201;Secondary Objective: To evaluate the effect of WVE-210201 treatment on dystrophin production To evaluate the concentration of WVE-210201 in plasma following treatment with WVE-210201 To evaluate the concentration of WVE-210201 in urine following treatment with WVE-210201;Primary end point(s): Safety and tolerability of WVE-210201 as assessed by the number of patients with adverse events (AEs), severity of AEs, number of patients with serious adverse events (SAEs), and the number of patients who withdraw due to AEs.;Timepoint(s) of evaluation of this end point: During study treatment - week 1 to week 13 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Dystrophin level assessed by Western blot of muscle biopsy 2. Concentration of WVE-210201 in plasma at predefined time points 3. Concentration of WVE-210201 in urine at predefined time points;Timepoint(s) of evaluation of this end point: 1. Dystrophin level assessed by Western blot of muscle biopsy - wk1, wk14 2. Concentration of WVE-210201 in plasma at predefined time points - wk1, wk5 3. Concentration of WVE-210201 in urine at predefined time points - wk1, wk2, wk5, wk6-13 | — |
Countries
Belgium, Canada, France, Italy, Netherlands, United Kingdom, United States
Contacts
Public ContactBruno Rocton
PPD
Outcome results
None listed