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A Dose Range Finding Study With Open-Label Extension to Evaluate the Safety of Oral LMI070/Branaplam in Early Manifest Huntington's Disease

NCT05111249 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 26

Last updated 2025-05-16

Study results available
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Summary

This is the first study of branaplam in adults with Huntington's Disease (HD) to determine the correct dose required to lower mutant huntingtin protein (mHTT) levels in the cerebrospinal fluid (CSF) to a degree expected to be efficacious over longer periods of time.

Conditions

  • Early Manifest Huntington Disease

Interventions

DRUG

Branaplam

messenger ribonucleic acid (RNA) splicing modifier. Branaplam was administered as an oral solution once weekly.

DRUG

Placebo

Matching placebo oral solution once weekly

Sponsors & Collaborators

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
25 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-12-08
Primary Completion
2023-10-27
Completion
2023-10-27
FDA Drug
Yes

Countries

  • Canada
  • France
  • Germany
  • Hungary
  • Spain

Study Locations

More Related Trials

Entities

Drugs
Companies

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 NCT05111249 on ClinicalTrials.gov