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PTC518 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
Eligibility for HD-ISS Mild Stage 3 Group (Parts D, E, and F): A UHDRS Total Functional Capacity (TFC) score of 11 or 12, or a UHDRS TFC score of 13 with an UHDRS IS score of <100
Eligibility for HD-ISS Stage 2 Group (Parts A, B, and C): A UHDRS-Independence Scale (IS) score of 100, A UHDRS Total Functional Capacity (TFC) score of 13, A score between 0.18 and 4.93 inclusive on the normed version of the HD prognostic index (PINHD)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline up to month 18
Awards & highlights

Study Summary

This trial is testing a new drug to see if it is safe and effective in people with Huntington's disease.

Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
While specific side effects of PTC518 aren't listed here, common clinical trial risks may include reactions at injection sites, potential allergic responses, general discomforts like headaches or nausea, and possibly unforeseen complications related to the experimental nature of the treatment.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My functional abilities are slightly reduced due to my condition.
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I am in Stage 2 of Huntington's disease based on specific health scores.
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My Huntington's disease is confirmed with a specific genetic test result.
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It seems like there might be a typo or incomplete information for this criterion. Can you please provide more details or clarify?
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My Huntington's disease is confirmed with a specific genetic test result.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to month 18
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to month 18 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Huntingtin Protein
Number of Participants With Adverse Events (AEs)
Secondary outcome measures
Change From Baseline in Blood mHTT Protein at Month 12
Change From Baseline in Blood tHTT Protein at Month 12
Change From Baseline in Caudate Volume as Assessed Via Volumetric Magnetic Resonance Imaging (vMRI) at Month 12
+2 more

Trial Design

4Treatment groups
Experimental Treatment
Placebo Group
Group I: PTC518 5 mgExperimental Treatment1 Intervention
Participants will receive PTC518 5 milligrams (mg) tablets once daily orally for 12 months.
Group II: PTC518 20 mgExperimental Treatment1 Intervention
Participants will receive PTC518 20 mg tablets once daily orally for 12 months.
Group III: PTC518 10 mgExperimental Treatment1 Intervention
Participants will receive PTC518 10 mg tablets once daily orally for 12 months.
Group IV: PlaceboPlacebo Group1 Intervention
Participants will receive placebo matching to PTC518 tablets once daily orally for 12 months.

Find a Location

Who is running the clinical trial?

PTC TherapeuticsLead Sponsor
74 Previous Clinical Trials
5,978 Total Patients Enrolled

Media Library

Placebo Clinical Trial Eligibility Overview. Trial Name: NCT05358717 — Phase 2
Huntington's Disease Research Study Groups: PTC518 5 mg, PTC518 10 mg, Placebo, PTC518 20 mg
Huntington's Disease Clinical Trial 2023: Placebo Highlights & Side Effects. Trial Name: NCT05358717 — Phase 2
Placebo 2023 Treatment Timeline for Medical Study. Trial Name: NCT05358717 — Phase 2
Huntington's Disease Patient Testimony for trial: Trial Name: NCT05358717 — Phase 2

Frequently Asked Questions

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

What are the potential risks associated with PTC518?

"PTC518 safety is rated a 2 by our team because it is currently in Phase 2 clinical trials. While there is some evidence supporting its safety, there are no studies yet testing its efficacy."

Answered by AI

Are there any patients still needed for this clinical trial?

"That is correct. According to clinicaltrials.gov, this trial was initially posted on 4/25/2022 and updated as recently as 11/9/2022. The study is looking for 162 participants from 4 different medical centers."

Answered by AI

How many research facilities are administering this clinical trial?

"Patients can receive treatment at the Rocky Mountain Movement Disorders Center in Englewood, Colorado; University of Alabama at Birmingham in Birmingham, Alabama; Columbia University, G.H. Sergievsky Center in New york, New York; and 4 other locations across the country."

Answered by AI

How many people fit the eligibility requirements for this clinical trial?

"A total of one hundred and sixty-two individuals that fit the study's requirements are necessary to move forward with planned research. PTC Therapeutics is sponsoring the trial, which will take place in several locations including Rocky Mountain Movement Disorders Center (Englewood, Colorado) and University of Alabama at Birmingham (Birmingham, Alabama)."

Answered by AI

Who else is applying?

What state do they live in?
Georgia
What portion of applicants met pre-screening criteria?
Did not meet criteria
How many prior treatments have patients received?
0

Why did patients apply to this trial?

My son in his 20s hasn't been tested yet and now that I have HD, I want to help as much as possible to find a cure. I saw a presentation on this trial.
PatientReceived no prior treatments
~0 spots leftby Mar 2024