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WVE-210201

Phase 1

Duchenne Muscular Dystrophy | Small molecule | Neurology |Wave Life Sciences Ltd.|Last Updated: Apr 8, 2019

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials1
Total Enrollment36

FDA Designations

No designations recorded

Clinical trial landscape

WVE-210201 · 1 trial · 1 indication

Phase 1 1
NCT03508947Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular DystrophyDuchenne Muscular Dystrophy
COMPLETED36 Analytics
PHASE1COMPLETED
Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
Duchenne Muscular DystrophyUnlock trial analytics

Study Endpoints

Primary Endpoints

Safety: Number of patients with adverse events (AEs)
Day 1 to Day 85 (end of study)
Safety: Severity of AEs
Day 1 to Day 85 (end of study)
Safety: Number of patients with serious AEs (SAEs)
Day 1 to Day 85 (end of study)
Safety and Tolerability: Number of patients who withdraw due to AEs
Day 1 to Day 85 (end of study)

Secondary Endpoints

Pharmacokinetics (PK): Maximum observed concentration (Cmax)
Day 1, Day 2, and Day 8
PK: Time of occurrence of Cmax (tmax)
Day 1, Day 2, and Day 8
PK: Area under the plasma concentration-time curve (AUC 0-t)
Day 1, Day 2, and Day 8
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
WVE-210201 (Dose A) or placeboEXPERIMENTAL -
WVE-210201 (Dose B) or placeboEXPERIMENTAL -
WVE-210201 (Dose C) or placeboEXPERIMENTAL -
WVE-210201 (Dose D) or placeboEXPERIMENTAL -
WVE-210201 (Dose E) or placeboEXPERIMENTAL -

Interventions

NameTypeDescription
WVE-210201DRUGWVE-210201 is a stereopure antisense oligonucleotide (ASO)
PlaceboDRUGSodium Chloride
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Eligibility Criteria

Age Range5 Years to 18 Years
SexMALE
Healthy VolunteersNo
Study Sites13

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 year...

Countries:United StatesBelgiumCanadaFranceItalyNetherlandsUnited Kingdom
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Frequently asked questions about WVE-210201

What is WVE-210201 used for in Duchenne Muscular Dystrophy?

WVE-210201 is an investigational small molecule being developed for the treatment of Duchenne Muscular Dystrophy, a genetic disorder characterized by progressive muscle degeneration and weakness. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.

Who makes WVE-210201?

WVE-210201 is being developed by Wave Life Sciences Ltd., a biopharmaceutical company traded on the NASDAQ under the ticker symbol WVE. The company is conducting clinical trials to evaluate the safety and tolerability of this investigational drug in patients with Duchenne Muscular Dystrophy.

What phase is WVE-210201 in?

WVE-210201 is in Phase 1 clinical development. A Phase 1 trial has been completed, which assessed the safety and tolerability of the drug in patients with Duchenne Muscular Dystrophy. The drug remains investigational and has not received FDA approval.

What clinical trials is WVE-210201 in?

WVE-210201 has one completed clinical trial, registered as NCT03508947, titled 'Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy.' This Phase 1 study enrolled 36 male participants aged 5 years and older across multiple countries, including the United States, Belgium, Canada, France, Italy, Netherlands, and the United Kingdom.

Is WVE-210201 the same as any other drug?

WVE-210201 is the primary name used for this investigational drug in clinical trials. No alternative names have been reported in the available clinical trial information, and it is distinct from other drugs in Wave Life Sciences' pipeline.