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Nonsense Mutation Readthrough Agent

Ataluren for Duchenne Muscular Dystrophy

Phase 3

Waitlist Available

Research Sponsored by PTC Therapeutics

Eligibility Criteria Checklist

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

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline up to end of study (up to approximately 8 years)

Awards & highlights

Study Summary

This trial is for people with nmDBMD who have had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants. The purpose is to see if the treatment is safe and tolerated.

Who is the study for?

This trial is for individuals with a type of muscular dystrophy called nmDBMD who have previously taken Ataluren in a PTC sponsored clinical trial or treatment plan. It's also open to siblings of these participants if they've finished the placebo part of another study. Participants must understand and agree to the study requirements, be able to attend all visits, and men must use birth control during the study.Check my eligibility

What is being tested?

The trial is testing the safety and tolerability of Ataluren at doses of 10 mg/kg (twice daily) and 20 mg/kg (once daily). The medication will be given until certain conditions are met such as consent withdrawal, worsening condition after starting treatment, eligibility for another Ataluren trial, or commercial availability.See study design

What are the potential side effects?

While specific side effects aren't listed here, common ones from medications like Ataluren could include gastrointestinal discomforts like nausea or diarrhea, potential liver enzyme changes which would be monitored by blood tests, possible allergic reactions if there's known hypersensitivity to its ingredients.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline up to end of study (up to approximately 8 years)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline up to end of study (up to approximately 8 years)

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to end of study (up to approximately 8 years) for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Number of Participants With Abnormal Physical Findings

Number of Participants With Adverse Events

Number of Participants With Laboratory Parameters Abnormalities

Side effects data

From 2018 Phase 4 trial • 1 Patients • NCT03256968

100%

increased cough

100%

80%

60%

40%

20%

Study treatment Arm

Ataluren Administration

Trial Design

1Treatment groups

Experimental Treatment

Group I: AtalurenExperimental Treatment1 Intervention

Participants will receive 3 doses of ataluren oral suspension per day (10 mg/kg in the morning, 10 mg/kg at mid-day and 20 mg/kg in the evening).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Ataluren

2018

Completed Phase 4

~1450

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

PTC TherapeuticsLead Sponsor

74 Previous Clinical Trials

5,960 Total Patients Enrolled

Vinay Penematsa, MDStudy DirectorPTC Therapeutics, Inc.

3 Previous Clinical Trials

520 Total Patients Enrolled

Media Library

Ataluren (Nonsense Mutation Readthrough Agent) Clinical Trial Eligibility Overview. Trial Name: NCT01247207 — Phase 3

Duchenne Muscular Dystrophy Research Study Groups: Ataluren

Duchenne Muscular Dystrophy Clinical Trial 2023: Ataluren Highlights & Side Effects. Trial Name: NCT01247207 — Phase 3

Ataluren (Nonsense Mutation Readthrough Agent) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01247207 — Phase 3

Duchenne Muscular Dystrophy Patient Testimony for trial: Trial Name: NCT01247207 — Phase 3

Frequently Asked Questions

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

How many participants are in this clinical trial?

"There are no active recruitment drives for this clinical trial at this time. Although, it's worth noting that the study was first posted on 11/30/2010 and was last updated on 11/1/2022. Additionally, if you are interested in other trials, there are 88 trials actively enrolling patients with becker muscular dystrophy and 4 trials for Ataluren that are actively looking for patients."

Answered by AI

Is this an original research project?

"PTC Therapeutics sponsored the first clinical trial for Ataluren in 2010, which involved a pool of 270 patients. The success of this initial study led to Phase 3 drug approval in 2010. As of now, there are 4 active trials being conducted in 49 cities and 19 countries."

Answered by AI

Are researchers actively recruiting participants for this trial?

"No, this particular trial is not open for recruitment at the moment. However, there are other 92 clinical trials that are still looking for patients. Based on the information found on clinicaltrials.gov, this study was first posted on November 30th, 2010 and was most recently edited on November 1st, 2022."

Answered by AI

Where can I find a list of hospitals participating in this research project?

"This study is hosted at 33 different centres, some of which are Southwestern University in Dallas, Duke University Medical Center in Durham, and Stanford University in Palo Alto."

Answered by AI

What is the efficacy of Ataluren in other forms of research?

"University of Texas Health Science Center at San Antonio first researched ataluren in 2010. Since then, there have been a further 17 completed clinical trials. As of now, 4 studies are ongoing with several taking place in Dallas and North carolina."

Answered by AI

Is it safe to take Ataluren if you have no other health conditions?

"On a scale from 1 to 3, our team at Power estimates the safety of Ataluren to be a 3. This is because it is a Phase 3 trial, meaning there is some data supporting efficacy as well as multiple rounds of data supporting safety."

Answered by AI

Who else is applying?

What state do they live in?

California

Puerto Rico

How old are they?

18 - 65

What site did they apply to?

Stanford University

Columbia University Medical School

What portion of applicants met pre-screening criteria?

Met criteria

Did not meet criteria

How many prior treatments have patients received?

Why did patients apply to this trial?

Hi my child is suffering from. Nonsence mutation dmd and attlurin is best medicine available for that can u pls add my child name in it Barleen singh father of samarbir +919653600020.

PatientReceived 2+ prior treatments

I had been trying to apply for clinical trial since last year , but unfortunately it didnot work. since we live in Iran most of centers are refusing to provide me with recommendation letter. I hope your center would help me in this regard to help me to travel overthere for treatment of my only child. I can afford my cost of accommodation during the study. What I all need is your confirmation and recommendation letter.

PatientReceived 2+ prior treatments

Recent research and studies

The Journal of Clinical PharmacologyJournal

Safety, Tolerability, and Pharmacokinetics of PTC124, a Nonaminoglycoside Nonsense Mutation Suppressor, Following Single- and Multiple-dose Administration to Healthy Male and Female Adult Volunteers

Hirawat, Samit, Ellen M. Welch, Gary L. Elfring, Valerie J. Northcutt, Sergey Paushkin, Seongwoo Hwang, Eileen M. Leonard, et al.. 2007. “Safety, Tolerability, and Pharmacokinetics of PTC124, a Nonaminoglycoside Nonsense Mutation Suppressor, Following Single- and Multiple-dose Administration to Healthy Male and Female Adult Volunteers”. The Journal of Clinical Pharmacology. Wiley. doi:10.1177/0091270006297140.

NatureJournal

PTC124 Targets Genetic Disorders Caused by Nonsense Mutations

Welch, Ellen M., Elisabeth R. Barton, Jin Zhuo, Yuki Tomizawa, Westley J. Friesen, Panayiota Trifillis, Sergey Paushkin, et al.. 2007. “PTC124 Targets Genetic Disorders Caused by Nonsense Mutations”. Nature. Springer Science and Business Media LLC. doi:10.1038/nature05756.

clinicaltrials.govStudy

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

2010. "Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT01247207.

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