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Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03508947
Recruitment Status : Completed
First Posted : April 26, 2018
Last Update Posted : April 8, 2019
Sponsor:
Information provided by (Responsible Party):
Wave Life Sciences Ltd.

Study Description
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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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: WVE-210201 Drug: Placebo Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 36 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients With Duchenne Muscular Dystrophy
Actual Study Start Date : January 24, 2018
Actual Primary Completion Date : March 6, 2019
Actual Study Completion Date : March 6, 2019

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: WVE-210201 (Dose A) or placebo Drug: WVE-210201
WVE-210201 is a stereopure antisense oligonucleotide (ASO)

Drug: Placebo
Sodium Chloride
Experimental: WVE-210201 (Dose B) or placebo Drug: WVE-210201
WVE-210201 is a stereopure antisense oligonucleotide (ASO)

Drug: Placebo
Sodium Chloride
Experimental: WVE-210201 (Dose C) or placebo Drug: WVE-210201
WVE-210201 is a stereopure antisense oligonucleotide (ASO)

Drug: Placebo
Sodium Chloride
Experimental: WVE-210201 (Dose D) or placebo Drug: WVE-210201
WVE-210201 is a stereopure antisense oligonucleotide (ASO)

Drug: Placebo
Sodium Chloride
Experimental: WVE-210201 (Dose E) or placebo Drug: WVE-210201
WVE-210201 is a stereopure antisense oligonucleotide (ASO)

Drug: Placebo
Sodium Chloride


Outcome Measures
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Primary Outcome Measures :
  1. Safety: Number of patients with adverse events (AEs) [ Time Frame: Day 1 to Day 85 (end of study) ]
  2. Safety: Severity of AEs [ Time Frame: Day 1 to Day 85 (end of study) ]
  3. Safety: Number of patients with serious AEs (SAEs) [ Time Frame: Day 1 to Day 85 (end of study) ]
  4. Safety and Tolerability: Number of patients who withdraw due to AEs [ Time Frame: Day 1 to Day 85 (end of study) ]

Secondary Outcome Measures :
  1. Pharmacokinetics (PK): Maximum observed concentration (Cmax) [ Time Frame: Day 1, Day 2, and Day 8 ]
  2. PK: Time of occurrence of Cmax (tmax) [ Time Frame: Day 1, Day 2, and Day 8 ]
  3. PK: Area under the plasma concentration-time curve (AUC 0-t) [ Time Frame: Day 1, Day 2, and Day 8 ]

Eligibility Criteria
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Ages Eligible for Study:   5 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
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:

    1. Reproducible percent predicted forced vital capacity (FVC) ≥50%
    2. 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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03508947


Locations
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United States, Georgia
Rare Disease Research, LLC.
Atlanta, Georgia, United States, 30318
Belgium
UZ Gent
Gent, Belgium, 9000
Universitaire Ziekenhuizen Leuven
Leuven, Belgium
CHR de la Citadelle
Liège, Belgium
Canada, Ontario
London Health Sciences Centre - Hospital
London, Ontario, Canada
France
Hôpital Armand Trousseau
Paris, France
Italy
U.O.C di Neurologia e Malattie Neuromuscolari Centro Clinico Nemo Sud
Messina, Italy, 98125
U.O. Immunologia Pediatrica
Milano, Italy, 20132
Netherlands
Radbound University Nijmegen Medical Care
Nijmegen, Netherlands, 6525 GC
United Kingdom
University Hospitals Bristol NHS Foundation Trust
Bristol, United Kingdom
Alder Hey Children's Hospital
Liverpool, United Kingdom, L12 2AP
Evelina London Children's Hospital
London, United Kingdom, SE1 7EH
UCL Institute of Child Health & Great Ormond Street Hospital for Children
London, United Kingdom
Sponsors and Collaborators
Wave Life Sciences Ltd.
Investigators
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Study Director: Michael A Panzara, MD, MPH Wave Life Sciences Ltd.
More Information
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Responsible Party: Wave Life Sciences Ltd.
ClinicalTrials.gov Identifier: NCT03508947    
Other Study ID Numbers: WVE-DMDX51-001
First Posted: April 26, 2018    Key Record Dates
Last Update Posted: April 8, 2019
Last Verified: April 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked