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Nonsense Mutation Read-Through Agent

Ataluren for Duchenne Muscular Dystrophy

Phase 2
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
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up predose up to 12 hours postdose at week 24
Awards & highlights

Study Summary

This trial is testing if a drug is safe and effective for boys with a certain kind of muscular dystrophy aged 6 months to 2 years.

Eligible Conditions
  • Duchenne Muscular Dystrophy

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~predose up to 12 hours postdose at week 24
This trial's timeline: 3 weeks for screening, Varies for treatment, and predose up to 12 hours postdose at week 24 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 Treatment-Emergent Adverse Events (TEAEs)
Secondary outcome measures
Area Under the Concentration-Time Curve Between Dosing Interval (AUC0-τ) of Ataluren
Area Under the Concentration-Time Curve From Time 0 to 24 Hours (AUC0-24) of Ataluren
Maximum Concentration (Cmax) of Ataluren
+2 more

Side effects data

From 2018 Phase 4 trial • 1 Patients • NCT03256968
100%
increased cough
100%
80%
60%
40%
20%
0%
Study treatment Arm
Ataluren Administration

Trial Design

1Treatment groups
Experimental Treatment
Group I: AtalurenExperimental Treatment1 Intervention
Participants will receive ataluren oral suspension 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for 24 weeks.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ataluren
2018
Completed Phase 4
~1450

Find a Location

Who is running the clinical trial?

PTC TherapeuticsLead Sponsor
74 Previous Clinical Trials
6,224 Total Patients Enrolled
Vinay PenematsaStudy DirectorPTC Therapeutics, Inc.

Frequently Asked Questions

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

Are there any current vacancies for this research project?

"The listed clinical trial on clinicaltrials.gov is currently looking for enrollees. This specific study was originally made public on December 29th, 2021 and the last update was November 1st, 2022."

Answered by AI

Is this trial only for seniors, or will younger people be able to participate as well?

"Per the inclusion requirements for this experiment, babies that are a minimum of 6 months old and no older than 2 years of age are eligible to participate."

Answered by AI

Are there any other similar studies that have been done with Ataluren?

"There are 4 clinical trials underway for Ataluren, 2 of which have progressed to Phase 3. Although the majority of research is being conducted in Amsterdam, Noord-Holland, there are 108 total locations running these studies."

Answered by AI

Could I possibly be chosen to help with this research?

"10 children suffering from muscular dystrophy, aged 6 months to 2 years old, are being recruited for this study. Most importantly, potential participants should meet the following weight requirement: Body weight ≥7.5 kilograms (kg)."

Answered by AI

Are there any harmful side effects associated with taking Ataluren?

"While there is some data indicating that Ataluren is safe, it only received a score of 2 because there is no evidence yet demonstrating the efficacy of this medication."

Answered by AI

How many people are signing up to participate in this clinical trial?

"That is correct. The information available on clinicaltrials.gov reveals that this experiment, which was first advertised on December 29th 2021, has not yet reached its full quota of 10 subjects from 1 location."

Answered by AI

What are the other similar clinical trials to this one that have been conducted in the past?

"Since 2010, Ataluren has been under regular clinical study. PTC Therapeutics sponsored the first trial in 2010 which recruited 270 patients. After this initial study, Phase 3 drug approval was given to Ataluren. As of now, there are 4 ongoing trials involving 49 cities and 19 nations."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
2021. "A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04336826.
~2 spots leftby Apr 2025
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