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Study Summary
This trial is testing a new drug to see if it is safe and effective in people with Huntington's disease.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
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Who is running the clinical trial?
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- You have a UHDRS Total Functional Capacity (TFC) score of 13 or less.My functional abilities are slightly reduced due to my condition.I have not been in any experimental studies or treatments in the last 90 days.I am in Stage 2 of Huntington's disease based on specific health scores.I haven't taken any experimental drugs or had experimental brain surgery recently.My Huntington's disease is confirmed with a specific genetic test result.I have no brain or spinal conditions that affect lumbar puncture safety.You have a perfect score on the Independence Scale of the Unified Huntington's Disease Rating Scale.It seems like there might be a typo or incomplete information for this criterion. Can you please provide more details or clarify?I have never received gene therapy for Huntington's disease.My Huntington's disease is confirmed with a specific genetic test result.You have a specific score on a test called the HD prognostic index between 0.18 and 4.93.
- Group 1: PTC518 5 mg
- Group 2: PTC518 10 mg
- Group 3: Placebo
- Group 4: PTC518 20 mg
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
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."
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."
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."
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)."
Who else is applying?
What state do they live in?
What portion of applicants met pre-screening criteria?
How many prior treatments have patients received?
Why did patients apply to this trial?
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