Duchenne Muscular DystrophyPhase 1NCT03508947
Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
A Phase 1 study testing WVE-210201 in people with Duchenne Muscular Dystrophy, run by Wave Life Sciences USA, Inc. at 13 sites in 7 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 to 18 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- WVE-210201
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 36 participants
- Where
- 13 sitesin 7 countries
- Study dates
- 24 Jan 2018to 6 Mar 2019
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
13 sites in 7 countries.
13 sites in 7 countries
United Kingdom4 sites
University Hospitals Bristol NHS Foundation Trust
Bristol
Alder Hey Children's Hospital
Liverpool, L12 2AP
Evelina London Children's Hospital
London, SE1 7EH
UCL Institute of Child Health & Great Ormond Street Hospital for Children
London
Belgium3 sites
UZ Gent
Ghent, 9000
Universitaire Ziekenhuizen Leuven
Leuven
CHR de la Citadelle
Liège
Italy2 sites
U.O.C di Neurologia e Malattie Neuromuscolari Centro Clinico Nemo Sud
Messina, 98125
U.O. Immunologia Pediatrica
Milan, 20132
Netherlands1 site
Radbound University Nijmegen Medical Care
Nijmegen, 6525 GC
About this study
Official title: A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients With Duchenne Muscular Dystrophy
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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- WVE-210201, Placebo
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
WVE-210201Drug
WVE-210201 is a stereopure antisense oligonucleotide (ASO)
PlaceboDrug
Sodium Chloride
| Study group | Role | What they receive |
|---|---|---|
| WVE-210201 (Dose A) or placebo | Experimental | Not described Receives: Drug: WVE-210201; Drug: Placebo |
| WVE-210201 (Dose B) or placebo | Experimental | Not described Receives: Drug: WVE-210201; Drug: Placebo |
| WVE-210201 (Dose C) or placebo | Experimental | Not described Receives: Drug: WVE-210201; Drug: Placebo |
| WVE-210201 (Dose D) or placebo | Experimental | Not described Receives: Drug: WVE-210201; Drug: Placebo |
| WVE-210201 (Dose E) or placebo | Experimental | Not described Receives: Drug: WVE-210201; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: Number of patients with adverse events (AEs)
Measured: Day 1 to Day 85 (end of study)
Safety: Severity of AEs
Measured: Day 1 to Day 85 (end of study)
Safety: Number of patients with serious AEs (SAEs)
Measured: Day 1 to Day 85 (end of study)
Safety and Tolerability: Number of patients who withdraw due to AEs
Measured: Day 1 to Day 85 (end of study)
Secondary outcomes (3)
Pharmacokinetics (PK): Maximum observed concentration (Cmax)
Measured: Day 1, Day 2, and Day 8
PK: Time of occurrence of Cmax (tmax)
Measured: Day 1, Day 2, and Day 8
PK: Area under the plasma concentration-time curve (AUC 0-t)
Measured: Day 1, Day 2, and Day 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Apr 2018
- Study start24 Jan 2018
- Primary completion6 Mar 2019
- Study completion6 Mar 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Wave Life Sciences USA, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Michael A Panzara, MD, MPH, Study director, Wave Life Sciences USA, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03508947
- Sponsor's protocol ID
- WVE-DMDX51-001
- Status last verified
- Apr 2019
- First submitted
- 16 Apr 2018
- Last updated
- 8 Apr 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT03508947
- Acronym / sponsor ID
- WVE-DMDX51-001
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Wave Life Sciences USA, Inc.Industry (pharma, biotech, devices)
- Study start
- 24 Jan 2018 (actual)
- Primary completion
- 6 Mar 2019 (actual)
- Study completion
- 6 Mar 2019 (actual)
- First posted
- 26 Apr 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| WVE-210201 (Dose A) or placebo | Experimental | Drug: WVE-210201, Drug: Placebo |
| WVE-210201 (Dose B) or placebo | Experimental | Drug: WVE-210201, Drug: Placebo |
| WVE-210201 (Dose C) or placebo | Experimental | Drug: WVE-210201, Drug: Placebo |
| WVE-210201 (Dose D) or placebo | Experimental | Drug: WVE-210201, Drug: Placebo |
| WVE-210201 (Dose E) or placebo | Experimental | Drug: WVE-210201, Drug: Placebo |
Who may qualify
- Ages
- 5 to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 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:
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| WVE-210201WVE-210201 is a stereopure antisense oligonucleotide (ASO) | Drug | 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 |
| PlaceboSodium Chloride | Drug | 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 |
Primary outcome
Safety: Number of patients with adverse events (AEs)
Time frame: Day 1 to Day 85 (end of study)
Safety: Severity of AEs
Time frame: Day 1 to Day 85 (end of study)
Safety: Number of patients with serious AEs (SAEs)
Time frame: Day 1 to Day 85 (end of study)
Safety and Tolerability: Number of patients who withdraw due to AEs
Time frame: Day 1 to Day 85 (end of study)
3 secondary outcomes
Pharmacokinetics (PK): Maximum observed concentration (Cmax)
Time frame: Day 1, Day 2, and Day 8
PK: Time of occurrence of Cmax (tmax)
Time frame: Day 1, Day 2, and Day 8
PK: Area under the plasma concentration-time curve (AUC 0-t)
Time frame: Day 1, Day 2, and Day 8
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2019.
Related trials for Duchenne Muscular Dystrophy
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael A Panzara, MD, MPH study director
Wave Life Sciences USA, Inc.
13 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing WVE-210201 in people with Duchenne Muscular Dystrophy, run by Wave Life Sciences USA, Inc. at 13 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United Kingdom, Belgium, Italy, United States, Canada and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety: Number of patients with adverse events (AEs) (time frame: Day 1 to Day 85 (end of study)). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Apr 2019.