Session Information
Date: Wednesday, June 22, 2016
Session Title: Huntington's disease
Session Time: 12:00pm-1:30pm
Location: Exhibit Hall located in Hall B, Level 2
Objective: To investigate the trends in Huntington’s disease (HD) therapeutic development by analyzing interventional clinical trials publicly registered for the last 15 years.
Background: Like in other neurodegenerative diseases, drug development in HD has proven to be very difficult. Being a rare serious disease makes drug development even more challenging. There is only one drug FDA-approved for symptom control. Learning from the current drug-development pipeline may improve research for new treatments.
Methods: We analyzed all HD interventional clinical trials registered in the World Health Organization International Clinical Trials Search Portal (ICTRP) from its inception (1999) to September 2015. We abstracted the year of trial registration, start and end trial dates, sponsor, number of sites, number of patients, trial design and rate of success defined as level of progress through the various phases of drug-development.
Results: Ninety-five HD trials were found corresponding to 41 compounds and 7 non-pharmacological interventions. Twenty (21.1%) were phase 1 trials, 45 (48.4%) were phase 2, 19 (20%) were phase 3 and 3 (3.2%) were phase 4. The United States (US) was the country conducting the highest number of trials. Fifty-six trials (59%) were at least partially sponsored by industry. Eighty-seven trials (91.6%) evaluated pharmacological interventions and 41 individual compounds were tested. The most often tested compounds were creatine, latrepirdine and pridopidine. The mean number of participants enrolled in a trial was 103.3 and the mean trial duration was 35.2 weeks, both increasing from phase 1 to phase 3 trials. The intervention’s attrition rate was 77.8% from phase 1 to phase 2 and 80.6% from phase 2 to phase 3, and the overall success rate was 1.8%.
Conclusions: Although HD is a rare condition, 95 trials were so far registered in HD. We found a large attrition rate at an earlier trials phase and a very high trial failure rate in trials at later phases of drug-development. There is huge gap in drug discovery and clinical development methods in HD that warrants improvement.
To cite this abstract in AMA style:
A.M. Travessa, F.B. Rodrigues, T.A. Mestre, C. Sampaio, J.J. Ferreira. Fifteen years of clinical trials in Huntington’s disease: Too many clinical trial failures [abstract]. Mov Disord. 2016; 31 (suppl 2). https://www.mdsabstracts.org/abstract/fifteen-years-of-clinical-trials-in-huntingtons-disease-too-many-clinical-trial-failures/. Accessed May 24, 2025.« Back to 2016 International Congress
MDS Abstracts - https://www.mdsabstracts.org/abstract/fifteen-years-of-clinical-trials-in-huntingtons-disease-too-many-clinical-trial-failures/