What side effects are associated with this type of intervention?

The most common treatments for Duchenne Muscular Dystrophy, such as eteplirsen, golodirsen, viltolarsen, and ataluren, are associated with several side effects. Eteplirsen and golodirsen can cause balance disorders, vomiting, contact dermatitis, rash, and upper respiratory tract infections. Viltolarsen may lead to upper respiratory tract infections, injection site reactions, cough, and fever. Ataluren's side effects include vomiting, decreased appetite, weight loss, headache, hypertension, cough, and abdominal pain. Monitoring for renal toxicity is also recommended for treatments involving antisense oligonucleotides like golodirsen and viltolarsen.

What prior FDA approvals exist?

The FDA has approved several genetic therapies for the treatment of Duchenne Muscular Dystrophy (DMD), particularly those that increase dystrophin production through exon skipping. These include eteplirsen (ExonDys 51), which skips exon 51 and is suitable for about 13% of DMD patients, golodirsen (Vyondys 53), which skips exon 53 and is suitable for approximately 8% of patients, and viltolarsen, also targeting exon 53. Casimersen, which skips exon 45, was approved in February 2021 for patients with deletions amenable to exon 45 skipping. These treatments aim to produce a shorter but functional dystrophin protein, similar to the approach of Ataluren, which promotes ribosomal readthrough of premature stop codons.

What other types of research exist?

Promising alternatives to Ataluren for Duchenne Muscular Dystrophy patients include antisense oligonucleotides such as viltolarsen, casimersen, eteplirsen, and golodirsen. These therapies promote exon skipping to restore dystrophin production. Each targets specific exons (e.g., viltolarsen for exon 53, casimersen for exon 45, eteplirsen for exon 51, and golodirsen for exon 53) and has shown efficacy in clinical trials.

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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

Must not have

Known hypersensitivity to any of the ingredients or excipients of ataluren (refined polydextrose, polyethylene glycol 3350, poloxamer 407, mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, colloidal silica, magnesium stearate).

Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy.

Timeline

Screening 3 weeks

Treatment Varies

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

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Summary

This trial tests the safety and tolerance of ataluren in patients with a specific type of Duchenne Muscular Dystrophy who have been part of previous studies. Ataluren helps the body make important proteins that are usually missing due to genetic errors. The study includes these patients and their siblings who have completed earlier trial phases. Ataluren is the first approved drug for Duchenne muscular dystrophy (DMD) patients with premature stop codon mutations and has been conditionally approved in Europe.

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.

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.

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.

Eligibility Criteria

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I am not allergic to any components of ataluren medication.

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I am currently receiving IV treatment with aminoglycoside or vancomycin.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of Participants With Abnormal Physical Findings

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

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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

0 / 2Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Duchenne Muscular Dystrophy (DMD) include genetic therapies such as exon skipping drugs (eteplirsen, golodirsen, viltolarsen) and ataluren, which promotes ribosomal readthrough of premature stop codons. These treatments work by increasing the production of dystrophin, a protein that is deficient in DMD patients. Dystrophin is essential for stabilizing muscle cell membranes, and its increased production can improve muscle function and slow disease progression. This is vital for DMD patients as it can enhance their quality of life and delay the onset of severe symptoms.