PTC518 for Huntington's Disease

Recruiting at 31 trial locations

Overseen By Jody Corey-Bloom

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: PTC Therapeutics

Disqualifiers: Gene therapy, Pregnancy, Brain disease, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

252 Participants Needed

Trial Summary

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.

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

PTC518 is unique because it is being tested specifically for Huntington's disease, a condition with no current cure or standard treatment to slow its progression. Unlike other treatments that may focus on symptoms, PTC518 aims to address the underlying disease mechanisms. ^{1 2 3 4 5}

What is the purpose of this trial?

This trial is testing a new drug called PTC518 to see if it is safe and how it affects people with Huntington's Disease. The goal is to find out if PTC518 can help manage symptoms or slow down the disease.

Eligibility Criteria

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

You have a UHDRS Total Functional Capacity (TFC) score of 13 or less.

My functional abilities are slightly reduced due to my condition.

I am in Stage 2 of Huntington's disease based on specific health scores.

Exclusion Criteria

Any medical history or condition that would interfere with the ability to complete the protocol-specified assessments (for example, implanted shunt, conditions precluding magnetic resonance imaging [MRI] scans)

Pregnancy, planning on becoming pregnant during the course of the study or within 6 months of end of treatment, or currently breastfeeding

I have not been in any experimental studies or treatments in the last 90 days.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive PTC518 or placebo once daily orally for 12 months

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Placebo
• PTC518

Trial Overview The study tests PTC518's safety and its effects compared to a placebo in individuals with Huntington’s Disease. Participants will be randomly assigned to receive either the drug or placebo to determine if there are any differences in outcomes between the two groups.

Participant Groups

Group I: PTC518 5 mg Experimental Treatment 1 Intervention

Participants will receive PTC518 5 milligrams (mg) tablets once daily orally for 12 months.

Group II: PTC518 20 mg Experimental Treatment 1 Intervention

Participants will receive PTC518 20 mg tablets once daily orally for 12 months.

Group III: PTC518 10 mg Experimental Treatment 1 Intervention

Participants will receive PTC518 10 mg tablets once daily orally for 12 months.

Group IV: Placebo Placebo Group 1 Intervention

Participants will receive placebo matching to PTC518 tablets once daily orally for 12 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

Findings from Research

In a study involving 227 participants with Huntington's disease, pridopidine at a dosage of 90 mg/day showed a potential improvement in motor function, although the primary analysis did not reach statistical significance (P = .08).

Pridopidine was generally well tolerated by participants, indicating it may be a safe option for further investigation in treating motor symptoms of Huntington's disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A randomized, double-blind, placebo-controlled trial of pridopidine in Huntington's disease. [2022]

References

1. England pubmed.ncbi.nlm.nih.gov

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

2. New Zealand pubmed.ncbi.nlm.nih.gov

Novel targets for Huntington's disease: future prospects. [2020]

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

Motor, cognitive, and functional declines contribute to a single progressive factor in early HD. [2022]

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

A randomized, double-blind, placebo-controlled trial of pridopidine in Huntington's disease. [2022]

5. Netherlands pubmed.ncbi.nlm.nih.gov

Cytotoxicity models of Huntington's disease and relevance of hormetic mechanisms: A critical assessment of experimental approaches and strategies. [2021]