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Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)

NCT04931862 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 35

Last updated 2023-10-23

No results posted yet for this study

Summary

This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of intrathecal (IT) WVE-004 in adult patients with C9orf72-associated ALS or FTD. To participate in the study, patients must have a documented mutation (GGGGCC \[G4C2\] repeat expansion) in the first intronic region of the C9orf72 gene and be diagnosed with ALS or FTD.

Conditions

Interventions

DRUG

WVE-004

WVE-004 is a stereopure antisense oligonucleotide (ASO)

DRUG

Placebo

Artificial cerebrospinal fluid (aCSF)

Sponsors & Collaborators

  • Wave Life Sciences Ltd.

    lead INDUSTRY

Principal Investigators

  • Medical Director, MD · Wave Life Sciences

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
80 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-06-28
Primary Completion
2023-06-27
Completion
2023-06-27
FDA Drug
Yes

Countries

  • Australia
  • Belgium
  • Canada
  • Ireland
  • Netherlands
  • New Zealand
  • Sweden
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04931862 on ClinicalTrials.gov