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

A Randomized, Double-Blind, Placebo-Controlled Dose Range Finding Study With Open-Label Extension to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of LMI070/Branaplam Administered as Weekly Oral Doses in Participants With Early Manifest Huntington's Disease

ClinicalTrials.gov Identifier: NCT05111249

Novartis Reference Number: CLMI070C12203

Last Update: Apr 25, 2022

All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

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.

Condition

Early Manifest Huntington Disease

Phase

Phase 2

Overall status

Recruiting

Start date

Dec 08, 2021

Completion date

Feb 17, 2025

Gender

All

Age(s)

25 Years - 75 Years (Adult, Older Adult)

Interventions

Drug

Branaplam

messenger ribonucleic acid (RNA) splicing modifier

Drug

Placebo

Matching placebo oral solution once weekly

Eligibility Criteria

Inclusion Criteria

Signed informed consent must be obtained prior to participation in the study.
Clinically diagnosed Stage 1 or 2 Huntington's disease with a diagnostic confidence level (DCL) = 4 and a United Huntington's Disease Rating Scale (UHDRS) Total Functional Capacity (TFC) >8 at screening.
Genetically confirmed Huntington's disease, with presence of ≥40 cytosine-adenineguanine (CAG) repeats in the huntingtin gene.
Male and female participants between 25 to 75 years of age, inclusive, on the day of Informed Consent signature.

Exclusion Criteria

Prior participation in clinical trial investigating a huntingtin-lowering therapy (unless participant received only placebo).
Participants taking medications prohibited by the protocol.
Any medical history, lumbar surgery or condition that would interfere with the ability to complete the protocol specified assessments,
Participant has other severe, acute or chronic medical conditions including unstable psychiatric conditions, or laboratory abnormalities that in the opinion of the Investigator may increase the risk associated with study participation, or that may interfere with the interpretation of the study results.
Any surgical or medical condition which might put the participant at risk in case of participation in the study. The Investigator should make this determination in consideration of the participant's medical history and/or clinical or laboratory evidence at the Screening visit: