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

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ClinicalTrials.gov Identifier: NCT03508947
Recruitment Status : Recruiting
First Posted : April 26, 2018
Last Update Posted : April 27, 2018
Sponsor:
Information provided by (Responsible Party):
Wave Life Sciences Ltd.

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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 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
Estimated Primary Completion Date : September 2018
Estimated Study Completion Date : September 2018


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




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 ]


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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.

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


Contacts
Contact: Clinical Operations, Wave Life Sciences Ltd. 855-215-4687 clinicaltrials@wavelifesci.com

Locations
United States, California
Stanford Neuroscience Health Center Recruiting
Palo Alto, California, United States, 94304
Principal Investigator: John Day, MD, PhD         
United States, Georgia
Rare Disease Research, LLC. Recruiting
Atlanta, Georgia, United States, 30318
Principal Investigator: Han C Phan, MD         
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Principal Investigator: Jeffrey Statland, MD         
France
Hôpital Armand Trousseau Recruiting
Paris, France
Principal Investigator: Teresa Gidaro, MD         
United Kingdom
University Hospitals Bristol NHS Foundation Trust Recruiting
Bristol, United Kingdom
Principal Investigator: Anirban Majumdar, MD         
UCL Institute of Child Health & Great Ormond Street Hospital for Children Recruiting
London, United Kingdom
Principal Investigator: Francesco Muntoni, MD, PhD         
Sponsors and Collaborators
Wave Life Sciences Ltd.
Investigators
Study Director: Michael A Panzara, MD, MPH Wave Life Sciences Ltd.

Responsible Party: Wave Life Sciences Ltd.
ClinicalTrials.gov Identifier: NCT03508947     History of Changes
Other Study ID Numbers: WVE-DMDX51-001
First Posted: April 26, 2018    Key Record Dates
Last Update Posted: April 27, 2018
Last Verified: April 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked