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Small Molecule

PTC518 5 mg for Huntington's Disease

Phase 2

Recruiting

Research Sponsored by PTC Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline up to month 30

Awards & highlights

Study Summary

This trial aims to study the long-term safety and effects of PTC518 in individuals with Huntington's disease.

Who is the study for?

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.Check my eligibility

What is being tested?

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.See study design

What are the potential side effects?

Since this summary doesn't provide specific side effects, it can be assumed that participants will be monitored for any long-term adverse reactions to PTC518 as part of evaluating its safety.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline up to month 30

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline up to month 30

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to month 30 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Blood Total Huntingtin Protein (tHTT) Levels

Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Secondary outcome measures

Blood mHTT Protein Levels

Cerebrospinal Fluid (CSF) Mutant Huntingtin Protein (mHTT) Levels

Change From Baseline in Caudate Volume as Assessed Via Volumetric Magnetic Resonance Imaging (vMRI) at Month 24

+1 more

Trial Design

3Treatment groups

Experimental Treatment

Group I: PTC518 5 mgExperimental Treatment1 Intervention

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

Group II: PTC518 20 mgExperimental Treatment1 Intervention

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

Group III: PTC518 10 mgExperimental Treatment1 Intervention

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

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

PTC TherapeuticsLead Sponsor

74 Previous Clinical Trials

5,980 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any available vacancies for patient participation in this clinical trial?

"As per clinicaltrials.gov, this medical study is actively seeking volunteers. The trial was first listed on August 25th, 2023 and last revised on February 2nd, 2024."

Answered by AI

In how many different geographical areas can patients access this clinical trial?

"At present, this medical research initiative is actively enrolling participants at 26 sites. Notable locations include Westmead, Burgos, and Münster along with various other locales. Opting for the site closest to you can help reduce travel requirements during your involvement in this trial."

Answered by AI

Has the FDA granted approval for PTC518 at a dosage of 10 mg?

"Based on the phase of this trial, our evaluation ranks the safety of PTC518 10 mg at a level 2. This indicates that while there is data supporting its safety profile, efficacy evidence is currently lacking."

Answered by AI

What is the current sample size of patients involved in this clinical investigation?

"Indeed, the details on clinicaltrials.gov highlight that this ongoing research study is actively seeking eligible participants. This trial was initially posted for recruitment on August 25th, 2023 and underwent its latest revision on February 2nd, 2024. The objective is to enroll a total of 250 individuals across an extensive network of 26 designated sites."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

An Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)

2023. "An Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06254482.

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