Back to resultsSafety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
Primary Purpose
Duchenne Muscular Dystrophy
Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
WVE-210201
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Duchenne Muscular Dystrophy
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of Duchenne muscular dystrophy (DMD) based on clinical phenotype with increased serum creatine kinase
- Documented mutation in the Dystrophin gene associated with DMD that is amenable to exon 51 skipping
- Ambulatory or non-ambulatory male patients aged ≥5 - ≤18 years
Stable pulmonary and cardiac function as measured by:
- Reproducible percent predicted forced vital capacity (FVC) ≥50%
- Left ventricular ejection fraction (LVEF) >55% in patients <10 years of age and >45% in patients ≥10 years of age, as measured (and documented) by echocardiogram within one year prior to enrollment into the study.
Exclusion Criteria:
- Severe cardiomyopathy; cardiomyopathy that is managed by angiotensin-converting enzyme (ACE) inhibitors or beta blockers is acceptable provided the patient meets the LVEF inclusion criteria.
- Need for mechanical or non-invasive ventilation OR anticipated need for mechanical or non-invasive ventilation within the next year, in the opinion of the Investigator.
- Changes in nutritional or herbal supplements or concomitant medications within 1 month prior to Screening visit or plans to modify dose or regimen during the study.
- Currently on anticoagulants or antithrombotics.
- Received treatment with eteplirsen or ataluren within the past 14 weeks.
- Received prior treatment with drisapersen.
- Received any investigational drug within the past 3 months or 5 half-lives, whichever is longer.
Sites / Locations
- Rare Disease Research, LLC.
- UZ Gent
- Universitaire Ziekenhuizen Leuven
- CHR de la Citadelle
- London Health Sciences Centre - Hospital
- Hôpital Armand Trousseau
- U.O.C di Neurologia e Malattie Neuromuscolari Centro Clinico Nemo Sud
- U.O. Immunologia Pediatrica
- Radbound University Nijmegen Medical Care
- University Hospitals Bristol NHS Foundation Trust
- Alder Hey Children's Hospital
- Evelina London Children's Hospital
- UCL Institute of Child Health & Great Ormond Street Hospital for Children
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm Type
Experimental
Experimental
Experimental
Experimental
Experimental
Arm Label
WVE-210201 (Dose A) or placebo
WVE-210201 (Dose B) or placebo
WVE-210201 (Dose C) or placebo
WVE-210201 (Dose D) or placebo
WVE-210201 (Dose E) or placebo
Arm Description
Outcomes
Primary Outcome Measures
Safety: Number of patients with adverse events (AEs)
Safety: Severity of AEs
Safety: Number of patients with serious AEs (SAEs)
Safety and Tolerability: Number of patients who withdraw due to AEs
Secondary Outcome Measures
Pharmacokinetics (PK): Maximum observed concentration (Cmax)
PK: Time of occurrence of Cmax (tmax)
PK: Area under the plasma concentration-time curve (AUC 0-t)
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03508947
Brief Title
Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
Official Title
A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients With Duchenne Muscular Dystrophy
Study Type
Interventional
2. Study Status
Record Verification Date
April 2019
Overall Recruitment Status
Completed
Study Start Date
January 24, 2018 (Actual)
Primary Completion Date
March 6, 2019 (Actual)
Study Completion Date
March 6, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Wave Life Sciences Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This is a Phase 1, double-blind, placebo-controlled, single ascending dose cohort study to evaluate the safety, tolerability, and plasma concentrations of WVE-210201 in ambulatory and non-ambulatory male pediatric patients with DMD amenable to exon 51 skipping intervention.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
36 (Actual)
8. Arms, Groups, and Interventions
Arm Title
WVE-210201 (Dose A) or placebo
Arm Type
Experimental
Arm Title
WVE-210201 (Dose B) or placebo
Arm Type
Experimental
Arm Title
WVE-210201 (Dose C) or placebo
Arm Type
Experimental
Arm Title
WVE-210201 (Dose D) or placebo
Arm Type
Experimental
Arm Title
WVE-210201 (Dose E) or placebo
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
WVE-210201
Intervention Description
WVE-210201 is a stereopure antisense oligonucleotide (ASO)
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Sodium Chloride
Primary Outcome Measure Information:
Title
Safety: Number of patients with adverse events (AEs)
Time Frame
Day 1 to Day 85 (end of study)
Title
Safety: Severity of AEs
Time Frame
Day 1 to Day 85 (end of study)
Title
Safety: Number of patients with serious AEs (SAEs)
Time Frame
Day 1 to Day 85 (end of study)
Title
Safety and Tolerability: Number of patients who withdraw due to AEs
Time Frame
Day 1 to Day 85 (end of study)
Secondary Outcome Measure Information:
Title
Pharmacokinetics (PK): Maximum observed concentration (Cmax)
Time Frame
Day 1, Day 2, and Day 8
Title
PK: Time of occurrence of Cmax (tmax)
Time Frame
Day 1, Day 2, and Day 8
Title
PK: Area under the plasma concentration-time curve (AUC 0-t)
Time Frame
Day 1, Day 2, and Day 8
10. Eligibility
Sex
Male
Gender Based
Yes
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of Duchenne muscular dystrophy (DMD) based on clinical phenotype with increased serum creatine kinase
Documented mutation in the Dystrophin gene associated with DMD that is amenable to exon 51 skipping
Ambulatory or non-ambulatory male patients aged ≥5 - ≤18 years
Stable pulmonary and cardiac function as measured by:
Reproducible percent predicted forced vital capacity (FVC) ≥50%
Left ventricular ejection fraction (LVEF) >55% in patients <10 years of age and >45% in patients ≥10 years of age, as measured (and documented) by echocardiogram within one year prior to enrollment into the study.
Exclusion Criteria:
Severe cardiomyopathy; cardiomyopathy that is managed by angiotensin-converting enzyme (ACE) inhibitors or beta blockers is acceptable provided the patient meets the LVEF inclusion criteria.
Need for mechanical or non-invasive ventilation OR anticipated need for mechanical or non-invasive ventilation within the next year, in the opinion of the Investigator.
Changes in nutritional or herbal supplements or concomitant medications within 1 month prior to Screening visit or plans to modify dose or regimen during the study.
Currently on anticoagulants or antithrombotics.
Received treatment with eteplirsen or ataluren within the past 14 weeks.
Received prior treatment with drisapersen.
Received any investigational drug within the past 3 months or 5 half-lives, whichever is longer.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Michael A Panzara, MD, MPH
Organizational Affiliation
Wave Life Sciences Ltd.
Official's Role
Study Director
Facility Information:
Facility Name
Rare Disease Research, LLC.
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30318
Country
United States
Facility Name
UZ Gent
City
Gent
ZIP/Postal Code
9000
Country
Belgium
Facility Name
Universitaire Ziekenhuizen Leuven
City
Leuven
Country
Belgium
Facility Name
CHR de la Citadelle
City
Liège
Country
Belgium
Facility Name
London Health Sciences Centre - Hospital
City
London
State/Province
Ontario
Country
Canada
Facility Name
Hôpital Armand Trousseau
City
Paris
Country
France
Facility Name
U.O.C di Neurologia e Malattie Neuromuscolari Centro Clinico Nemo Sud
City
Messina
ZIP/Postal Code
98125
Country
Italy
Facility Name
U.O. Immunologia Pediatrica
City
Milano
ZIP/Postal Code
20132
Country
Italy
Facility Name
Radbound University Nijmegen Medical Care
City
Nijmegen
ZIP/Postal Code
6525 GC
Country
Netherlands
Facility Name
University Hospitals Bristol NHS Foundation Trust
City
Bristol
Country
United Kingdom
Facility Name
Alder Hey Children's Hospital
City
Liverpool
ZIP/Postal Code
L12 2AP
Country
United Kingdom
Facility Name
Evelina London Children's Hospital
City
London
ZIP/Postal Code
SE1 7EH
Country
United Kingdom
Facility Name
UCL Institute of Child Health & Great Ormond Street Hospital for Children
City
London
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
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