NCT01141075 - Ataluren for Nonsense Mutation Methylmalonic Acidemia | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

July 19, 2010

Primary Completion Date

November 3, 2011

Study Completion Date

November 3, 2011

Conditions

Amino Acid Metabolism, Inborn Errors

Interventions

DRUG

Ataluren

Ataluren will be provided as a vanilla-flavored powder to be mixed with water.

Trial Locations (9)

ZNA Queen Paola Child Hospital and Provincial Centre for Metabolic Disorders, Antwerp

Hôpital Edouard Herriot, Lyon

Necker-Enfants Malades Hospital, Paris

University Children's Hospital, Düsseldorf

Istituti Clinici di Perfezionamento, Milano, Milan

Federico II University, Naples

University Hospital, Department of Pediatrics, Padua

University Children's Hospital, Zurich

Great Ormand Street Hospital, London