PTC518 for Huntington's Disease

Recruiting at 25 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

250 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

The primary goal of this study is to evaluate the long-term safety and pharmacodynamic effects of PTC518 in participants with HD.

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.

How does the drug PTC518 differ from other treatments for Huntington's disease?

PTC518 is unique because it targets the underlying genetic cause of Huntington's disease by potentially lowering the levels of the mutant huntingtin protein, which is responsible for the disease's progression. This approach is different from other treatments that may focus on managing symptoms rather than addressing the genetic root of the condition. ^{1 2 3 4 5}

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: PTC518 5 mg Experimental Treatment 1 Intervention

Participants will receive PTC518 5 mg tablets once daily orally for 24 months.

Group II: PTC518 20 mg Experimental Treatment 1 Intervention

Participants will receive PTC518 20 mg tablets once daily orally for 24 months.

Group III: PTC518 10 mg Experimental Treatment 1 Intervention

Participants will receive PTC518 10 mg tablets once daily orally for 24 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

Citations

1. Netherlands pubmed.ncbi.nlm.nih.gov

Cortical neurodevelopment in pre-manifest Huntington's disease. [2020]

2. England pubmed.ncbi.nlm.nih.gov

FTY720 (fingolimod) is a neuroprotective and disease-modifying agent in cellular and mouse models of Huntington disease. [2022]

3. Ireland pubmed.ncbi.nlm.nih.gov

Modulation of age at onset in Huntington's disease and spinocerebellar ataxia type 2 patients originated from eastern India. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides. [2021]

5. United States pubmed.ncbi.nlm.nih.gov

Chromosome substitution strain assessment of a Huntington's disease modifier locus. [2018]