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

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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:

Study Locations

United States
Novartis Investigative Site
Recruiting
Baltimore, 21287
Maryland
United States
Novartis Investigative Site
Recruiting
New York, 10033
New York
United States
Canada
Novartis Investigative Site
Recruiting
Montreal, H2W 1T8
Quebec
Canada
France
Novartis Investigative Site
Recruiting
Angers Cedex 1, 49033
-
France
Novartis Investigative Site
Recruiting
Creteil, 94010
-
France
Novartis Investigative Site
Recruiting
Lille Cedex, 59037
-
France
Germany
Novartis Investigative Site
Recruiting
Erlangen, 91054
-
Germany
Novartis Investigative Site
Recruiting
Muenster, 48149
-
Germany
Novartis Investigative Site
Recruiting
Ulm, 89081
-
Germany
Hungary
Novartis Investigative Site
Recruiting
Budapest, 1085
-
Hungary
Novartis Investigative Site
Recruiting
Szeged, 6725
-
Hungary
Spain
Novartis Investigative Site
Recruiting
Barcelona, 08036
Catalunya
Spain
Novartis Investigative Site
Recruiting
Barcelona, 08041
-
Spain
Novartis Investigative Site
Recruiting
Madrid, 28034
-
Spain

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
Name: 
Novartis Pharmaceuticals
Phone: 

Have a question?

Call 1-888-669-6682 or email [email protected]