PTC518 for Huntington's Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new drug called PTC518 for individuals with Huntington's disease (HD). The main goal is to assess the drug's safety and its effects on the body compared to a placebo (a pill with no active drug). Participants will take either 5 mg, 10 mg, or 20 mg of PTC518, or a placebo, for 12 months. The trial seeks participants with a confirmed HD diagnosis who face daily life challenges due to the disease. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group of people.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. However, you cannot have received any experimental treatments for Huntington's Disease within 90 days before the study or during the study.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that PTC518 has been well tolerated in studies. The treatment did not cause serious health problems, even at different dose levels. Early human studies found no harmful effects that would prevent increasing the dose. Participants did not experience issues that stopped the treatment from being administered at higher doses. Overall, these studies suggest that the safety of PTC518 looks promising.12345
Why do researchers think this study treatment might be promising for Huntington's Disease?
Researchers are excited about PTC518 for Huntington's Disease because it offers a novel approach compared to current treatments like tetrabenazine and deutetrabenazine, which primarily manage symptoms. PTC518 works by targeting the underlying cause of the disease, aiming to reduce the production of the mutant huntingtin protein, which is responsible for the disease's progression. This direct targeting could potentially slow down or alter the course of Huntington's Disease, offering hope for a more effective treatment. Additionally, the treatment is available in multiple dosages (5 mg, 10 mg, and 20 mg), allowing for flexibility in addressing different patient needs.
What evidence suggests that PTC518 might be an effective treatment for Huntington's Disease?
Research has shown that PTC518 holds promise for treating Huntington's disease. In this trial, participants will receive different dosages of PTC518—specifically 5 mg, 10 mg, or 20 mg—or a placebo. Studies have found that PTC518 lowers levels of the Huntingtin protein in the blood, which is linked to the disease, with noticeable reductions as early as week 12. Early results also suggest that PTC518 can slow the progression of movement problems, indicating early signs of benefits. These studies reported the treatment to be safe and well-tolerated. These findings offer hope that PTC518 could provide real benefits for people with Huntington's disease.12367
Are You a Good Fit for This Trial?
This trial is for people with Huntington's Disease who have a specific range of scores on the UHDRS scale, confirming their functional capacity and independence. They must have a genetically confirmed diagnosis with certain CAG repeat lengths. Excluded are those who've had gene therapy for HD, recent participation in other studies, or conditions that could interfere with study procedures.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive PTC518 or placebo once daily orally for 12 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Placebo
- PTC518
Find a Clinic Near You
Who Is Running the Clinical Trial?
PTC Therapeutics
Lead Sponsor
Dr. Matthew B. Klein
PTC Therapeutics
Chief Executive Officer since 2023
BA from the University of Pennsylvania, MD from Yale University School of Medicine, MS in Epidemiology from the University of Washington School of Public Health
Dr. Stuart W. Peltz
PTC Therapeutics
Chief Medical Officer since 2023
MD from Harvard Medical School