* A study investigating Branaplam as a potential new treatment for Huntington’s disease
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled Dose Range Finding Study with Open-Label Extension to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LMI070/branaplam when Administered as Weekly Oral Doses in Participants with Early Manifest Huntington’s Disease
IRAS ID
1004047
Contact name
Klaas Romanino
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2020-000105-92
Clinicaltrials.gov Identifier
Research summary
This is the first study of branaplam treatment (a medicine) in adult Huntington’s disease patients which aims to explore a range of different doses in order to select a dose that is safe and tolerated by HD patients and reduces the amount of a protein called mutant Huntingtin (mHTT) in the body. It is the build-up of mHTT over time that is thought to gradually damage brain cells in this disease, which leads to death in around 15-20 years after symptoms begin.
Up to 75 adult HD patients at 32 hospital sites around the world will be given the opportunity to join the study to evaluate up to 3 different doses of branaplam, in order to select a dose(s) that is/are considered safe and tolerated. The selected dose(s) will be tested in the second part of this study as well as in future studies.
Part 1
After an initial screening period of up to 6 weeks, eligible patients will be in the study for at least 17 weeks to approx. 1 year, although this may be extended up to a maximum of 2 years. Patients will take liquid branaplam or placebo (a medicine with no active ingredient) by mouth once per week, which will be randomly assigned. Neither the patient nor the study doctor will know what treatment has been assigned.
Part 2
Patients in part 1, will be offered the opportunity to move into the second part of the study which will test the selected dose(s) from part 1. Patients will be in part 2 for 1 year and all patients will know which dose level of branaplam they are taking.
During the study patients will have regular visits to the hospital site weekly for the first 5 weeks, and then every 4 weeks for the rest of the study. Hospital visits will include blood draws (some taken at time points over 2 to 3 days), physical examinations, brain scans and examinations, lumbar punctures, examinations of the heart, eye tests as well as a number of assessments/questionnaires completed by patients. The study is being funded by a company called Novartis Pharma AG.REC name
South Central - Hampshire B Research Ethics Committee
REC reference
22/SC/0320
Date of REC Opinion
26 Oct 2021
REC opinion
Further Information Favourable Opinion