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Real-World data on duchenne drug translarna collected in large safety study

NCT ID NCT02369731

Knowledge-focused Sponsor: PTC Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study followed 316 people with Duchenne muscular dystrophy who were taking Translarna (ataluren) as part of their normal medical care. The goal was to monitor side effects and see how well the drug works in everyday use. Researchers also checked if doctors and patients followed the official prescribing instructions.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

316 people

The number who actually took part.

Started

Apr 2015

Finished

Mar 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

The target study population will include participants for whom the decision to prescribe Translarna (outside of a clinical trial) has already been made, including participants in named participant early access programs. Participants within each prescriber's practice who are or will be receiving treatment with Translarna, and who meet the eligibility criteria and provide informed consent (either by the participant or through authorisation by a legal guardian) will be invited to enroll into the study and will be followed according to the protocol.

Ages

2 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Receiving or will be receiving usual care treatment with commercial supply of Translarna (or receiving care within a named participant early access program) * Willing to provide written informed consent to allow the study data collection procedures (either by the participant or through authorisation by a legal guardian) Exclusion Criteria: * Participants who are receiving ataluren or placebo in a blinded, randomized clinical trial, or ataluren in any other ataluren clinical trial or cohort early access program that prevents participation in this study

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Muscular dystrophy, Duchenne

Contacts and locations

Locations

  • AO Padova Clinica Neurologica

    Padova, 35128, Italy

  • AOU Policlinico "G. Martino"

    Messina, 98125, Italy

  • ASST Grande Ospedale Metropolitano Niguarda

    Milan, 20162, Italy

  • Addenbrooke's Hospital

    Cambridge, CB2 0QQ, United Kingdom

  • Ahepa University Hospital

    Thessaloniki, 54621, Greece

  • Bambino Gesù Children's Hospital

    Rome, 00146, Italy

  • Birmingham Heartlands Hospital

    Birmingham, B9 5SS, United Kingdom

  • Bristol Children's Hospital

    Bristol, BS2 8BJ, United Kingdom

  • Bērnu klīniskā universitātes slimnīca

    Riga, Latvia

  • CHRU de Brest Hôpital Morvan

    Brest, 29200, France

  • CHRU de Lille

    Lille, 59037, France

  • CHRU de Tours Hôpital Bretonneau

    Tours, 37044, France

  • CHU de Bordeaux Hôpital Pellegrin Enfant

    Bordeaux, 33076, France

  • CHU de Clermont-Ferrand, hôpital Estaing

    Clermont-Ferrand, 63003, France

  • CHU de Marseille Hôpital de la Timone

    Marseille, 13385, France

  • CHU de Martinique - Hôpital P. Zobda-Quitman

    Fort de France Cedex, 97261, France

  • CHU de Nancy Hôpital de Brabois-Enfants Rue du Morvan

    Vandœuvre-lès-Nancy, 54400, France

  • CHU de Nantes

    Nantes, 44093, France

  • CHU de Reims American Memorial Hospital

    Reims, 51092, France

  • CHU de Saint-Etienne Hôpital Bellevue

    Saint-Etienne, 42055, France

  • CHU de Strasbourg

    Strasbourg, 67098, France

  • CHU de Toulouse Hôpital Pierre Paul Riquet

    Toulouse, 31059, France

  • CHU de la Réunion - GHSR - GH Sud-Réunion

    Saint-Pierre, 97448, France

  • Cardiomiologia e Genetica Medica Primo Policlinico

    Naples, 80138, Italy

  • Centro Hospitalar Lisboa Norte EPE - Hospital de Santa Maria

    Lisbon, 1649-035, Portugal

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

    Porto, 4099-001, Portugal

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

    Coimbra, 3000-075, Portugal

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

    Leeds, LS1 3EX, United Kingdom

  • Children's Hospital "Aghia Sophia"

    Athens, 11527, Greece

  • Children's Hospital Alder Hey Children's Hospital NHS Trust

    Liverpool, L12 2AP, United Kingdom

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan, 20122, Italy

  • Fondazione Policlinico Universitario Agostino Gemelli

    Roma, 00168, Italy

  • Göteborgs Universitet Avdelningen för pediatrik vid institutionen för kliniska vetenskaper

    Gothenburg, 41685, Sweden

  • Hadassah Mount Scopus Medical Center

    Jerusalem, 91240, Israel

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

    Bron, 69677, France

  • Hospital das Clínicas da Universidade de São Paulo

    São Paulo, 05403-000, Brazil

  • Hôpital Armand Trousseau

    Paris, 75012, France

  • Hôpital Necker - Enfants malades

    Paris, 75015, France

  • II Policlinico Neurofisiopatologia (Edificio 11 G)

    Naples, 80131, Italy

  • IRCCS Istituto Neurologico Carlo Besta

    Milan, 20133, Italy

  • IRCSS Istituto delle Scienze Neurologiche

    Bologna, 40133, Italy

  • Istituto Giannina Gaslini

    Genova, 16147, Italy

  • Karolinska Institutet

    Stockholm, 17176, Sweden

  • Klinik und Poliklinik f. Kinder- und Jugendmedizin

    Hamburg, 20246, Germany

  • Klinikum Bayreuth GmbH

    Bayreuth, 95445, Germany

  • Krankenhaus der Barmherzigen Schwestern Linz

    Linz, 4010, Austria

  • MVZ Kinder- und Jugendärzte Fürth GmbH

    Fürth, 90762, Germany

  • Motol University Hospital

    Praha 5 - Motol, 15006, Czechia

  • Muskelzentrum, Schwerpunkt Neuropädiatrie

    Kassel, 34125, Germany

  • NIHR Lancashire Clinical Research Facility Royal Preston Hospital

    Preston, PR2 9HT, United Kingdom

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

    Torino, 10126, Italy

  • Regina Maria Policlinica Opera Center

    Bucharest, Romania

  • Royal Manchester Children's Hospital

    Manchester, M13 9WL, United Kingdom

  • SPZ Frankfurt Mitte

    Frankfurt am Main, 60316, Germany

  • Schneider Medical Center Tel Aviv University

    Petah Tikva, 49202, Israel

  • Semmelweis Egyetem II (Semmelweis University II)

    Budapest, 01094, Hungary

  • Sheba Medical Center The Joseph Sagol EMG, Neuroscience Center

    Ramat Gan, 52621, Israel

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

    Vienna, 1100, Austria

  • Sozialpädiatrisches Zentrum (SPZ) Charité Berlin

    Berlin, 13353, Germany

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

    Bucharest, 22113, Romania

  • St Thomas' Hospital Children Services

    London, SE1 7EH, United Kingdom

  • St. Olavs hospital University children's clinical hospital

    Trondheim, NO-7006, Norway

  • Szent László Kórház Gyermekneurológiai szakrendelés

    Budapest, Hungary

  • The John Walton Muscular Dystrophy Research Centre

    Newcastle, NE1 3BZ, United Kingdom

  • The University Hospital Brno

    Brno, 62500, Czechia

  • UCL Great Ormond Street Institute of Child Health

    London, WC1N 1EH, United Kingdom

  • Universitaetsklinikum Essen (AoR)

    Essen, 45122, Germany

  • Universitaetsklinikum Freiburg

    Freiburg im Breisgau, 79106, Germany

  • University Children's Hospital University Medical Centre Ljubljana

    Ljubljana, 1000, Slovenia

  • Universitätskinderklinik Gießen

    Giessen, 35385, Germany

  • Universitätsklinikum Erlangen

    Erlangen, 91054, Germany

More trials for these conditions

Other studies related to the condition(s) this trial covers.