Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Active, not recruitingOBSERVATIONAL
Enrollment

316

Participants

Timeline

Start Date

April 30, 2015

Primary Completion Date

May 30, 2025

Study Completion Date

May 30, 2025

Conditions
Muscular Dystrophy, Duchenne
Trial Locations (71)

1000

University Children's Hospital University Medical Centre Ljubljana, Ljubljana

1100

Sozialmedizinisches Zentrum Süd Kaiser-Franz-Josef Spital mit Gottfried von Preyer´schen Kinderspital, Vienna

4010

Krankenhaus der Barmherzigen Schwestern Linz, Linz

10126

Ospedale Molinette SCDU Neuropsichiatria Infantile AO Città della Salute e della Scienza di Torino, Torino

11527

"Children's Hospital Aghia Sophia", Athens

13353

Sozialpädiatrisches Zentrum (SPZ) Charité Berlin, Berlin

13385

CHU de Marseille Hôpital de la Timone, Marseille

15006

Motol University Hospital, Praha 5 - Motol

16147

Istituto Giannina Gaslini, Genova

17176

Karolinska Institutet, Stockholm

20122

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan

20133

IRCCS Istituto Neurologico Carlo Besta, Milan

20162

ASST Grande Ospedale Metropolitano Niguarda, Milan

20246

Klinik und Poliklinik f. Kinder- und Jugendmedizin, Hamburg

22113

"Spitalul Clinic de Copii Dr. Victor Gomoiu Sectia Clinica Neurologie Pediatrica", Bucharest

29200

CHRU de Brest Hôpital Morvan, Brest

31059

CHU de Toulouse Hôpital Pierre Paul Riquet, Toulouse

33076

CHU de Bordeaux Hôpital Pellegrin Enfant, Bordeaux

34125

Muskelzentrum, Schwerpunkt Neuropädiatrie, Kassel

35128

AO Padova Clinica Neurologica, Padua

35385

Universitätskinderklinik Gießen, Giessen

37044

CHRU de Tours Hôpital Bretonneau, Tours

40133

IRCSS Istituto delle Scienze Neurologiche, Bologna

41685

Göteborgs Universitet Avdelningen för pediatrik vid institutionen för kliniska vetenskaper, Gothenburg

42055

CHU de Saint-Etienne Hôpital Bellevue, Saint-Etienne

44093

CHU de Nantes, Nantes

45122

Universitaetsklinikum Essen (AoR), Essen

49202

Schneider Medical Center Tel Aviv University, Petah Tikva

51092

CHU de Reims American Memorial Hospital, Reims

52621

Sheba Medical Center The Joseph Sagol EMG, Neuroscience Center, Ramat Gan

54400

CHU de Nancy Hôpital de Brabois-Enfants Rue du Morvan, Vandœuvre-lès-Nancy

54621

Ahepa University Hospital, Thessaloniki

59037

CHRU de Lille, Lille

60316

SPZ Frankfurt Mitte, Frankfurt am Main

62500

The University Hospital Brno, Brno

63003

CHU de Clermont-Ferrand, hôpital Estaing, Clermont-Ferrand

67098

CHU de Strasbourg, Strasbourg

69677

"Hospices Civils de Lyon Groupement Hospitalier Est - Bâtiment Les Tilleuls", Bron

75012

Hôpital Armand Trousseau, Paris

75015

Hôpital Necker - Enfants malades, Paris

79106

Universitaetsklinikum Freiburg, Freiburg im Breisgau

80131

II Policlinico Neurofisiopatologia (Edificio 11 G), Napoli

80138

Cardiomiologia e Genetica Medica Primo Policlinico, Napoli

90762

MVZ Kinder- und Jugendärzte Fürth GmbH, Fürth

91054

Universitätsklinikum Erlangen, Erlangen

91240

Hadassah Mount Scopus Medical Center, Jerusalem

95445

Klinikum Bayreuth GmbH, Bayreuth

97261

CHU de Martinique - Hôpital P. Zobda-Quitman, Fort de France Cedex

97448

CHU de la Réunion - GHSR - GH Sud-Réunion, Saint-Pierre

98125

"AOU Policlinico G. Martino", Messina

05403-000

Hospital das Clínicas da Universidade de São Paulo, São Paulo

01094

Semmelweis Egyetem II (Semmelweis University II), Budapest

Unknown

Szent László Kórház Gyermekneurológiai szakrendelés, Budapest

Bērnu klīniskā universitātes slimnīca, Riga

Regina Maria Policlinica Opera Center, Bucharest

00168

Fondazione Policlinico Universitario Agostino Gemelli, Roma

00146

Bambino Gesù Children's Hospital, Rome

NO-7006

St. Olavs hospital University children's clinical hospital, Trondheim

3000-075

Centro Hospitalar e Universitário de coimbra EPE - Hospital Pediátrico de Coimbra, Coimbra

1649-035

Centro Hospitalar Lisboa Norte EPE - Hospital de Santa Maria, Lisbon

4099-001

Centro Hospitalar do Porto EPE - Hospital Geral de Santo António, Porto

B9 5SS

Birmingham Heartlands Hospital, Birmingham

BS2 8BJ

Bristol Children's Hospital, Bristol

CB2 0QQ

Addenbrooke's Hospital, Cambridge

LS1 3EX

Children Hospital - Children's Neurosciences Leeds TEACHING HOSPITALS NHS Trust, Leeds

L12 2AP

Children's Hospital Alder Hey Children's Hospital NHS Trust, Liverpool

SE1 7EH

St Thomas' Hospital Children Services, London

WC1N 1EH

UCL Great Ormond Street Institute of Child Health, London

M13 9WL

Royal Manchester Children's Hospital, Manchester

NE1 3BZ

The John Walton Muscular Dystrophy Research Centre, Newcastle

PR2 9HT

NIHR Lancashire Clinical Research Facility Royal Preston Hospital, Preston

Sponsors

Lead Sponsor

All Listed Sponsors
collaborator

The John Walton Muscular Dystrophy Research Centre (TREAT-NMD)

UNKNOWN

collaborator

Cooperative International Neuromuscular Research Group

NETWORK

lead

PTC Therapeutics

INDUSTRY

NCT02369731 - Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) | Biotech Hunter | Biotech Hunter