NCT05683860

Brief Summary

This is an OLE study conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of WVE-004 in adult patients with ALS, FTD, or mixed ALS/FTD phenotype with a documented mutation in the C9orf72 gene. To participate in the study, patients must have successfully completed Phase 1b/2a WVE-004-001 study.

Trial Health

60
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
8

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Dec 2022

Shorter than P25 for phase_1

Geographic Reach
2 countries

3 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 14, 2022

Completed
21 days until next milestone

First Submitted

Initial submission to the registry

January 4, 2023

Completed
9 days until next milestone

First Posted

Study publicly available on registry

January 13, 2023

Completed
6 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 30, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 30, 2023

Completed
Last Updated

October 23, 2023

Status Verified

January 1, 2023

Enrollment Period

7 months

First QC Date

January 4, 2023

Last Update Submit

October 18, 2023

Conditions

ALSFTD

Outcome Measures

Primary Outcomes (4)

  • Safety: Number of patients with adverse events (AEs)

    Day 1 to Week 120 (end of study)

  • Safety: Number of patients with a severe AE

    Day 1 to Week 120 (end of study)

  • Safety: Number of patients with serious AEs (SAEs)

    Day 1 to Week 120 (end of study)

  • Safety: Number of patients who withdraw due to AEs

    Day 1 to Week 120 (end of study)

Study Arms (1)

Experimental: WVE-004 (Dose A)

EXPERIMENTAL
Drug: WVE-004

Interventions

WVE-004DRUG

WVE-004 is a stereopure antisense oligonucleotide. It is administered via intrathecal injection

Experimental: WVE-004 (Dose A)

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Patient successfully completed the Phase 1b/2a study with WVE-004, WVE-004-001.

You may not qualify if:

  • Patient has a clinically significant medical finding on the physical examination other than C9orf72-associated ALS or FTD that, in the judgment of the Investigator or Sponsor, will make the patient unsuitable for participation in and/or completion of the trial procedures.
  • Patient received any other investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Patient received an investigational oligonucleotide within the past 6 months or 5 half-lives of the drug, whichever is longer.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Erasmus MC

Rotterdam, 3015, Netherlands

Location

Universitair Medisch Centrum Utrecht

Utrecht, Netherlands

Location

University of Oxford - Nuffield Department of Clinical Neurosciences

Oxford, OX3 7LF, United Kingdom

Location

Study Officials

  • Medical Director, MD

    Wave Life Sciences

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 4, 2023

First Posted

January 13, 2023

Study Start

December 14, 2022

Primary Completion

June 30, 2023

Study Completion

June 30, 2023

Last Updated

October 23, 2023

Record last verified: 2023-01

Locations

NL(2)GB(1)