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Novartis Pharmaceuticals.

VIBRANT-HD was discontinued on December 8, 2022. For more information please click here.

This study is a randomized, double-blind, placebo-controlled study which will be conducted in approximately 75 early manifest HD participants.

After screening and baseline assessments are completed, participants will enter the Core Treatment Period which is double-blinded and placebo-controlled. The Core Treatment Period includes a Dose Range Finding (DRF) period of 17 weeks, followed by a Blinded Extension period of up to 53 weeks. Three cohorts will be enrolled in the Core Treatment Period, and data from each cohort will be evaluated for efficacy and safety before proceeding to a new cohort with a higher dose. The Core Treatment Period will serve to evaluate the safety, tolerability, pharmacokinetic(s) (PK) and pharmacodynamic(s) (PD) and to find the best dose(s) to use in future studies.

After the dose(s) is determined at the end of the Core Treatment Period, participants will roll over into the Open Label Extension (OLE) and will be given branaplam for approximately 1 year and attend clinic visits every 4 weeks. At the end of the OLE, the study may be amended to provide open-label branaplam beyond 1 year or a separate extension study may be initiated.

Study Closed – Not Recruiting

Formal Name

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.

Study Type
Interventional (Clinical Trial)


Canadian Locations:


Location: Novartis Investigative Site
Montréal, Quebec, Canada, H2W 1T8
Status: Study Closed – Not Recruiting

Contact: Novartis Pharmaceuticals 1-888-669-6682

Ages Eligible for Study: 25 Years to 75 Years (Adult, Older Adult)

Sexes Eligible for Study: All

Accepts Healthy Volunteers: No

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  (eligibility that would exclude you from this study)

  • 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.


For more detailed information on this study and others and for the most recent updates, please go to is a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world.

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