PTC518 for Huntington's Disease

Not currently recruiting at 49 trial locations

Overseen By Patient Advocacy

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: PTC Therapeutics

Disqualifiers: Non-completion of prior study

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 24 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a medication called PTC518 to determine its safety and effectiveness for people with Huntington's disease. Participants will take varying doses of PTC518 tablets daily for up to 48 months. The goal is to understand the drug's long-term effects on the body and its potential to manage symptoms. This trial is open to those who have completed a previous study involving PTC518. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that PTC518 is being tested for safety in people with Huntington's disease. Earlier studies found that PTC518 is generally safe for both healthy individuals and those with Huntington's disease. These studies also demonstrated that PTC518 successfully reduced levels of the harmful protein associated with the disease.

The safety data from these studies suggest that the treatment does not cause major side effects. Although research is ongoing, results so far indicate that it is safe for use. Participants in earlier trials did not report any serious negative effects, making it a promising option for further study. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatment for Huntington's Disease?

Researchers are excited about PTC518 for Huntington's disease because it offers a novel approach by targeting the production of the huntingtin protein, which is central to the disease's progression. Unlike existing treatments that mainly address symptoms, PTC518 aims to reduce the levels of this protein directly, potentially slowing or halting the disease. Additionally, PTC518 is an oral medication, which is more convenient compared to some current therapies that require injections or infusions. This new mechanism and delivery method have generated optimism for more effective management of Huntington's disease in the future.

What evidence suggests that this trial's treatments could be effective for Huntington's Disease?

Research shows that PTC518 may help treat Huntington's Disease. In earlier studies, PTC518 significantly reduced the harmful huntingtin protein, linked to Huntington's Disease, in both blood and the fluid around the brain and spine. After 12 months, the reduction in this protein depended on the dose: higher doses led to greater decreases. Specifically, a 5 mg dose resulted in a 23% reduction, while a 10 mg dose led to a 43% reduction. This trial will test different doses of PTC518, including 5 mg, 10 mg, and 20 mg, to evaluate its effectiveness in lowering the levels of the protein that causes the disease. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is specifically for individuals who have Huntington's Disease and have already completed the treatment period in a prior study, PTC518-CNS-002-HD. It's not open to those who haven't participated in that initial study.

Inclusion Criteria

Participant who has completed the Treatment Period in Study PTC518-CNS-002-HD.

Exclusion Criteria

Participants who have not previously completed the Treatment Period in Study PTC518-CNS-002-HD.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Baseline Evaluation

Participants undergo baseline evaluations before starting the extension study

1 visit

Treatment

Participants receive PTC518 at their assigned dose level for 24 months

24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

What Are the Treatments Tested in This Trial?

Interventions

• PTC518

Trial Overview The focus of this extension study is on assessing the long-term safety and effectiveness of a drug called PTC518 for people with Huntington's Disease who were previously involved in an earlier phase of research.

How Is the Trial Designed?

Group I: Votoplam 5 mg Experimental Treatment 1 Intervention

Participants will receive Votoplam 5 mg tablets once daily orally for 48 months.

Group II: Votoplam 10 mg Experimental Treatment 1 Intervention

Participants will receive votoplam 10 mg tablets once daily orally for 48 months.

Who Is Running the Clinical Trial?

PTC Therapeutics

Lead Sponsor

Trials

75

Recruited

6,300+

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

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

Citations

1. ir.ptcbio.com ir.ptcbio.com/news-releases/news-release-details/ptc518-pivot-hd-study-achieves-primary-endpoint?mobile=1

PTC518 PIVOT-HD Study Achieves Primary Endpoint The study met its primary endpoint of reduction in blood Huntingtin (HTT) protein levels (p<0.0001) at Week 12 and favorable safety and ...

2. ir.ptcbio.com ir.ptcbio.com/news-releases/news-release-details/interim-pivot-hd-results-demonstrate-evidence-favorable-cns?mobile=1

Interim PIVOT-HD Results Demonstrate Evidence of ... At Month 12, PTC518 treatment resulted in dose-dependent lowering of mutant huntingtin (mHTT) protein in the blood and cerebrospinal fluid (CSF) ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05358717

NCT05358717 | A Study to Evaluate the Safety and ... The primary goal of this study is to evaluate the safety and pharmacodynamic effects of PTC518 compared with placebo in participants with HD.

4. neurologylive.com neurologylive.com/view/huntington-drug-ptc518-meets-primary-end-point-phase-2-study

Huntington Drug PTC518 Meets Primary End Point in ... After 12 months of treatment with the small molecule splicing modifier, investigators observed a 23% reduction in blood HTT levels for the 5 mg ...

5. ir.ptcbio.com ir.ptcbio.com/news-releases/news-release-details/fda-grants-fast-track-designation-ptc518-huntingtons-disease?mobile=1

FDA Grants Fast Track Designation to PTC518 Huntington's ... The 12-month data demonstrated durable dose-dependent lowering of mutant Huntingtin protein in blood cells, reaching 43% at the 10-milligram ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11602954/

Pharmacokinetics and pharmacodynamics of PTC518, an oral ... This first‐in‐human study investigated the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of PTC518 in healthy volunteers.

7. en.hdbuzz.net en.hdbuzz.net/433/

Excitement and Anticipation as PTC's Huntington's Disease ... Along with safety measures and huntingtin lowering, PTC are collecting data throughout the trial related to biomarkers of disease progression ...