Ataluren
- Generic Name
- Ataluren
- Brand Names
- Translarna
- Drug Type
- Small Molecule
- Chemical Formula
- C15H9FN2O3
- CAS Number
- 775304-57-9
- Unique Ingredient Identifier
- K16AME9I3V
- Background
Ataluren is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. More specifically, ataluren is used in the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.
This drug does not yet have approval by the US Food and Drug Administration or by Health Canada for any indications.
- Indication
Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. More specifically, ataluren is used in the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.
- Associated Conditions
- Duchenne Muscular Dystrophy (DMD)
- Associated Therapies
- -
Effects of Rifampin on the Pharmacokinetics of Ataluren
- First Posted Date
- 2015-04-06
- Last Posted Date
- 2017-12-22
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 15
- Registration Number
- NCT02409004
- Locations
- 🇨🇦
Inventiv, Quebec, Canada
Study of Ataluren (PTC124) in Cystic Fibrosis
- First Posted Date
- 2014-04-08
- Last Posted Date
- 2020-03-25
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 61
- Registration Number
- NCT02107859
- Locations
- 🇺🇸
University of Alabama-Birmingham, Birmingham, Alabama, United States
🇺🇸Miller Children's Hospital Long Beach, Long Beach, California, United States
🇺🇸Denver Children's Hospital, Aurora, Colorado, United States
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
- First Posted Date
- 2014-03-19
- Last Posted Date
- 2020-08-11
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 219
- Registration Number
- NCT02090959
- Locations
- 🇨🇦
British Columbia Children's Hospital, Vancouver, British Columbia, Canada
🇺🇸University of Iowa, Iowa City, Iowa, United States
🇺🇸Oregon Health & Science University, Portland, Oregon, United States
Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
- First Posted Date
- 2013-04-08
- Last Posted Date
- 2020-08-04
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 230
- Registration Number
- NCT01826487
- Locations
- 🇨🇦
Alberta Children's Hospital, Calgary, Alberta, Canada
🇫🇷Hopital Necker - Enfants Malades, Paris, France
🇮🇹Bambino Gesu Hospital, Rome, Italy
Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
- Conditions
- Interventions
- First Posted Date
- 2012-03-19
- Last Posted Date
- 2020-11-25
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 94
- Registration Number
- NCT01557400
- Locations
- 🇦🇺
Royal Children's Hospital, Parkville, Melbourne, Australia
🇸🇪Astrid Lindgren Pediatric Hospital, Stockholm, Sweden
🇮🇹Ospedale Maggiore Policlinico in Milan, Milan, Italy
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
- First Posted Date
- 2010-11-24
- Last Posted Date
- 2024-11-22
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 270
- Registration Number
- NCT01247207
- Locations
- 🇺🇸
Rare Disease Research, LLC, Atlanta, Georgia, United States
🇺🇸Rush Univ Medical Center, Chicago, Illinois, United States
🇺🇸University of Iowa Children's Hospital, Iowa City, Iowa, United States
Ataluren for Nonsense Mutation Methylmalonic Acidemia
- First Posted Date
- 2010-06-10
- Last Posted Date
- 2020-07-07
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 11
- Registration Number
- NCT01141075
- Locations
- 🇮🇹
Istituti Clinici di Perfezionamento, Milano, Milan, Italy
🇧🇪ZNA Queen Paola Child Hospital and Provincial Centre for Metabolic Disorders, Antwerp, Belgium
🇫🇷Hôpital Edouard Herriot, Lyon, France
Extension Study of Ataluren (PTC124) in Cystic Fibrosis
- First Posted Date
- 2010-06-09
- Last Posted Date
- 2020-10-19
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 191
- Registration Number
- NCT01140451
- Locations
- 🇺🇸
Emory University Cystic Fibrosis Center, Atlanta, Georgia, United States
🇺🇸Children's Memorial Hospital, Chicago, Illinois, United States
🇺🇸Rainbow Babies & Children's Hospital, Cleveland, Ohio, United States
Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
- Conditions
- Interventions
- First Posted Date
- 2009-11-06
- Last Posted Date
- 2020-07-29
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 6
- Registration Number
- NCT01009294
- Locations
- 🇬🇧
University of Newcastle, Newcastle upon Tyne, United Kingdom
🇺🇸University of Minnesota, Minneapolis, Minnesota, United States
🇺🇸Nationwide Children's Hospital, Columbus, Ohio, United States
Study of Ataluren (PTC124) in Hemophilia A and B
- First Posted Date
- 2009-07-27
- Last Posted Date
- 2020-06-16
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 13
- Registration Number
- NCT00947193
- Locations
- 🇺🇸
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
🇺🇸The Bleeding and Clotting Disorders Institute, Peoria, Illinois, United States
🇮🇹Azienda Ospedaliero-Universitaria Careggi Viale G.B. Morgagni, Firenze, Italy
Drug Development Updates
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