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Tests treatment safety and results for Huntington Disease
Official title A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
ClinicalTrials.gov ID: NCT07326709
What this study is testing
What is Votoplam (blinded)?
Votoplam (blinded) is an investigational medicine, being studied as a potential treatment for huntington disease.
Also referred to as HTT227.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
- This is a blinded study.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 21 to 70
You may be able to join if
- Signed informed consents must be obtained prior to participation in the study
- Ambulatory male or female participants between 21 to 70 years of age, inclusive, on the day of Informed Consent signature
- Genetically confirmed HD diagnosis with a cytosine-adenine-guanine (CAG) repeat length of 40 or above. Participants must have prior genetic...
- Meets all of the following criteria:
- UHDRS IS score ≥90
You likely can't join if
- History of gene therapy or cell transplantation or any other experimental brain surgery for the treatment of HD
- Serologic evidence for active viral hepatitis as indicated by:
- positive anti-HBc IgM
- positive anti-HBc IgG confirmed by positive HBsAg and/or HBV DNA
- positive HCV ab test confirmed by positive HCV RNA
- Immunodeficiency diseases, including a positive human immunodeficiency virus (HIV) test result
See the full eligibility criteria
- Signed informed consents must be obtained prior to participation in the study
- Ambulatory male or female participants between 21 to 70 years of age, inclusive, on the day of Informed Consent signature
- Genetically confirmed HD diagnosis with a cytosine-adenine-guanine (CAG) repeat length of 40 or above. Participants must have prior genetic confirmation and known CAG repeat length obtained prior to screening.
- Meets all of the following criteria:
- UHDRS IS score ≥90
- UHDRS TFC score = 13
- UHDRS TMS score = 7-25, inclusive
- CAP100 ≥ 70 Calculation: CAP = Age at study entry × (CAG length - 30) / 6.49
- History of gene therapy or cell transplantation or any other experimental brain surgery for the treatment of HD
- Serologic evidence for active viral hepatitis as indicated by:
- positive anti-HBc IgM
- positive anti-HBc IgG confirmed by positive HBsAg and/or HBV DNA
- positive HCV ab test confirmed by positive HCV RNA
- Immunodeficiency diseases, including a positive human immunodeficiency virus (HIV) test result
- History or current diagnosis of ECG or cardiac abnormalities indicating significant risk of safety for participants such as:
- Concomitant clinically significant cardiac arrhythmias, e.g., sustained ventricular tachycardia, and clinically significant second- or third-degree AV block without a pacemaker
- History of familial long QT syndrome or known family history of Torsade de Pointes
- Women of childbearing potential, defined as all women physiologically capable of becoming pregnant from menarche until becoming post-menopausal, unless they have had surgical bilateral oophorectomy (with or without...
- Pregnant or nursing (breastfeeding) women Other protocol defined inclusion/exclusion criteria may apply
The study team makes the final eligibility decision.
Where it's taking place
- La Jolla, California, United States
- Englewood, Colorado, United States
- Washington D.C., District of Columbia, United States
- Tampa, Florida, United States
- Chicago, Illinois, United States
- Indianapolis, Indiana, United States
- Iowa City, Iowa, United States
- Fairway, Kansas, United States
- Grand Rapids, Michigan, United States
- Albany, New York, United States
- Buffalo, New York, United States
- New York, New York, United States
- Rochester, New York, United States
- Cleveland, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Memphis, Tennessee, United States
- Nashville, Tennessee, United States
- Georgetown, Texas, United States
- Houston, Texas, United States
- Burlington, Vermont, United States
+ 20 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 21 years to 70 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include La Jolla, California, United States; Englewood, Colorado, United States; Washington D.C., District of Columbia, United States; Tampa, Florida, United States; Chicago, Illinois, United States; Indianapolis, Indiana, United States and 34 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.