A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
Condition(s) studied
Investigational drug(s) / intervention(s)
Votoplam (blinded): Votoplam (blinded) active treatment
Placebo: Placebo
Study summary
The purpose is to assess safety and tolerability of votoplam and to determine whether votoplam slows disease progression in patients with early symptomatic Huntington's disease (HD) compared to the control arm.
HTT227 - current compound code (former code is PTC518 from PTC Therapeutics), HTT227 is Novartis code under Novartis sponsorship.
Eligibility
Primary outcome measure(s)
- Change from Baseline in cUHDRS score — Baseline, Month 36
The Composite Unified Huntingtons Disease Ratings Scale (cUHDRS) is an improved composite measure to assess multi-domain clinical progression in HD. The cUHDRS is a combined weighted score of measures of motor function (TMS), cognition (SDMT and SWRT) and overall functional capacity (TFC): cUHDRS = \[(TFC-10.4)/1.9 - (TMS-29.7)/14.9 + (SDMT-28.4)/11.3 + (SWRT - 66.1)/20.1\] + 10. In conjunction with one another, these measures provide a comprehensive, sensitive and specific tool for monitoring disease progression, with lower scores indicating more severe disease. cUHDRS score range from -8 to 25.
Trial sites (117)
| Facility | City | Region | Status |
|---|---|---|---|
| University of Alabama Birmingham | Birmingham | Alabama | Recruiting |
| Univ of Arkansas for Med Sciences | Little Rock | Arkansas | Recruiting |
| University of CA San Diego | La Jolla | California | Recruiting |
| UC Davis Health System | Sacramento | California | Recruiting |
| UCSF | San Francisco | California | Recruiting |
| Stanford Healthcare | Stanford | California | Recruiting |
| CenExcel Rocky Mtn Clin Research | Englewood | Colorado | Recruiting |
| U Conn Health | Farmington | Connecticut | Recruiting |
| Georgetown University | Washington D.C. | District of Columbia | Recruiting |
| UM Department Of Neurology | Miami | Florida | Recruiting |
| University Of South Florida | Tampa | Florida | Recruiting |
| Grady Memorial Hospital and Clinics | Atlanta | Georgia | Recruiting |
| Parkinsons-Movement Disorder Ctr | Chicago | Illinois | Recruiting |
| Rush University Medical Center | Chicago | Illinois | Recruiting |
| Indiana University Health | Indianapolis | Indiana | Recruiting |
| University of Iowa Health Care | Iowa City | Iowa | Recruiting |
| University of Kansas Hospital | Fairway | Kansas | Recruiting |
| Hereditary Neurological Disease Centre | Wichita | Kansas | Recruiting |
| Johns Hopkins University | Baltimore | Maryland | Recruiting |
| Beth Israel Deaconess Med Ctr | Boston | Massachusetts | Recruiting |
| Henry Ford Health System | Detroit | Michigan | Recruiting |
| Trinity Health Michigan | Grand Rapids | Michigan | Recruiting |
| Albany Medical College | Albany | New York | Recruiting |
| UBMD Neurology | Buffalo | New York | Recruiting |
| Columbia University Irving Medical | New York | New York | Recruiting |
| Universi of Rochester School of Med | Rochester | New York | Recruiting |
| University Of NC At Chapel Hill | Chapel Hill | North Carolina | Recruiting |
| Duke University Health System | Durham | North Carolina | Recruiting |
| Cleveland Clinic Foundation | Cleveland | Ohio | Recruiting |
| Oregon Health Sciences University | Portland | Oregon | Recruiting |
| University of Pennsylvania | Philadelphia | Pennsylvania | Recruiting |
| University Of Pittsburgh | Pittsburgh | Pennsylvania | Recruiting |
| Veracity Neuroscience LLC | Memphis | Tennessee | Recruiting |
| Vanderbilt University Medical CenterX | Nashville | Tennessee | Recruiting |
| TX Movement Disorder Spec PLLC | Georgetown | Texas | Recruiting |
| U of TX Health Science Ct | Houston | Texas | Recruiting |
| The University of Vermont MC | Burlington | Vermont | Recruiting |
| VCU Health Systems MCV | Richmond | Virginia | Recruiting |
| University of Washington Medical Center | Seattle | Washington | Recruiting |
| Uni Of Wisconsin Hosp And Clinic | Madison | Wisconsin | Recruiting |
+ 77 more sites — see the full list on the official registry below.
More Novartis Pharmaceuticals trials in South Korea
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07326709 on ClinicalTrials.gov ↗ ← All trials in South Korea