Duchenne Muscular Dystrophy (DMD)FAIR-DMDNCT07402122
Registry for Duchenne and Becker Muscular Dystrophy
An observational study of Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy, run by Dr. Andreas Ziegler at 30 sites in 2 countries.
Trial summary
Duchenne muscular dystrophy (DMD) is an X-linked, recessive, progressive, and degenerative neuromuscular disorder that affects approximately one in 5,000 newborn boys. The established "standard of care" has improved prognosis; however, a causal therapy is not yet available. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Dystrophinopathy Symptomatic Female Carrier
- How many people
- 1,500 participants
- Where
- 30 sitesin 2 countries
- Study dates
- 13 Jul 2026to Jun 2041
- How long will I take part?
- 15 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: All patients meeting the eligibilty criteria in Germany and Austria.
Who may be able to join
- Genetically confirmed diagnosis of dystrophinopathy
- Female carriers only if they show clinical symptoms of a dystrophinopathy
- Treatment in one of the participating centers
- No age restrictions
- Ability to understand the patient information and sign the informed consent
- Consent capability by the patient themselves and/or by the parents
- Agreement to data exchange between the treating physicians, the telemedicine platform, and the registry
- Possession of a tablet or a smartphone
Who cannot join
- Missing legally valid consent form from the patient and/or legal guardians
- Project content is not understandable to the participant and/or legal guardians
- Not in possession of a tablet or smartphone
Trial locations and contacts
30 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
30 sites in 2 countries
Germany25 sites, 25 recruiting
- Recruiting
Charité - Universitätsmedizin Berlin
Berlin
Claudia Weiß, Dr. med., Claudia.weiss@charite.de
Claudia Weiß, Dr. med.
- Recruiting
DRK Kliniken Berlin Westend
Berlin
Corinna Stoltenburg, Dr. med., c.stoltenburg@drk-kliniken-berlin.de
Corinna Stoltenburg, Dr. med.
- Not yet recruiting
Katholisches Klinikum -Universitätsklinikum der Ruhr-Universität Bochum
Bochum
Cornelia Köhler, Dr. med., cornelia.koehler@kklbo.de
Cornelia Köhler, Dr. med.
- Recruiting
Universitätsklinikum Bonn, Zentrum für Kinderheilkunde
Bonn
Dorothea Holzwarth, Dr. med., Dorothea.Holzwarth@ukbonn.de
Dorothea Holzwarth, Dr. med.
- Not yet recruiting
Gesundheit Nord gGmbH Klinikverbund Bremen
Bremen
Ulf Hustedt, Dr. med., Ulf.Hustedt@gesundheitnord.de
Ulf Hustedt, Dr. med.
- Recruiting
Universitätsklinikum Carl Gustav Carus TU Dresden
Dresden
Maja von der Hagen, Prof. Dr. med., Maja.v.d.Hagen@uniklinikum-dresden.de
Maja von der Hagen, Prof. Dr. med.
- Recruiting
Universitätsklinikum Erlangen
Erlangen
Regina Trollmann, Prof. Dr. med., Regina.Trollmann@uk-erlangen.de
Regina Trollmann, Prof. Dr. med.
- Recruiting
Universitätsklinikum Essen Klinik für Kinderheilkunde I
Essen
Ulrike Schara-Schmidt, Prof. Dr. med., 0049 201 723 2508, Ulrike.Schara-Schmidt@uk-essen.de
Ulrike.Schara-Schmidt@uk-essen.de
- Recruiting
Universitätsklinikum Freiburg
Freiburg im Breisgau
Astrid Pechmann, PD Dr. med., Astrid.pechmann@uniklinik-freiburg.de
Astrid Pechmann, PD Dr. med.
- Recruiting
Universitätsklinikum Gießen und Marburg, Standort Gießen
Giessen
Andreas Hahn, Prof. Dr. med., andreas.hahn@paediat.med.uni-giessen.de
Andreas Hahn, Prof. Dr. med.
- Not yet recruiting
Universitätsmedizin Göttingen
Göttingen
Elke Hobbiebrunken, Dr. med., hobbiebrunken@med.uni-goettingen.de
Elke Hobbiebrunken, Dr. med.
- Recruiting
Universitätsmedizin Greifswald
Greifswald
Astrid Bertsche, Prof. Dr. med., Astrid.bertsche@med.uni-greifswald.de
Astrid Bertsche, Prof. Dr. med.
- Not yet recruiting
Asklepios Klinik Nord-Heidberg
Hamburg
Gert Wiegand, PD Dr. med., g.wiegand@asklepios.com
Gert Wiegand, PD Dr. med.
- Recruiting
Universitätsklinikum Hamburg-Eppendorf
Hamburg
Jessica Johannsen, PD Dr. med., j.johannsen@uke.de
Jessica Johannsen, PD Dr. med.
- Recruiting
Medizinische Hochschule Hannover
Hanover
Sabine Illsinger, PD Dr. med., Illsinger.Sabine@mh-hannover.de
Sabine Illsinger, PD Dr. med.
- Recruiting
Universitätsklinik Heidelberg, Zentrum für Kinder- und Jugendmedizin
Heidelberg
Andreas Ziegler, Dr. med., 004962215632207, Andreas.Ziegler@med.uni-heidelberg.de
Andreas Ziegler, Dr. med.
- Recruiting
Universitätsklinikum des Saarlandes
Homburg
Marina Flotats Bastardas, Dr. med., Marina.flotats-bastardas@uks.eu
Marina Flotats Bastardas, Dr. med.
- Not yet recruiting
Universitätsklinikum Jena
Jena
Ralf Husain, Dr. med., Ralf.Husain@med.uni-jena.de
Ralf Husain, Dr. med.
- Recruiting
Gesundheit Nordhessen Klinikum Kassel
Kassel
Gudrun Schreiber, Dr. med., Gudrun.Schreiber@gnh.net
Gudrun Schreiber, Dr. med.
- Recruiting
Universitätsklinikum Leipzig, Klinik und Poliklinik für Kinder- und Jugendmedizin
Leipzig
Janina Gburek-Augustat, Dr. med., janina.gburek-augustat@medizin.uni-leipzig.de
Janina Gburek-Augustat, Dr. med.
- Recruiting
LMU Klinikum München; Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital
München
Astrid Blaschek, PD Dr. med., Astrid.Blaschek@med.uni-muenchen.de
Astrid Blaschek, PD Dr. med.
- Recruiting
Universitätsklinikum Münster
Münster
Oliver Schwartz, Dr. med., oliver.schwartz@ukmuenster.de
Oliver Schwartz, Dr. med.
- Not yet recruiting
Klinikum Stuttgart, Olgahospital
Stuttgart
Sarah Braun, Dr. med., sa.braun@klinikum-stuttgart.de
Sarah Braun, Dr. med.
- Recruiting
Universitätsklinikum Tübingen, Universitätsklinik für Kinder- und Jugendmedizin
Tübingen
Veronka Horber, Dr. med., Veronka.Horber@med.uni-tuebingen.de
Veronka Horber, Dr. med.
- Not yet recruiting
Universitätsklinikum Ulm
Ulm
Sebahattin Cirak, Prof. Dr. med., Sebahattin.Cirak@uniklinik-ulm.de
Sebahattin Cirak, Prof. Dr. med.
Austria5 sites, 5 recruiting
- Not yet recruiting
LKH Universitätsklinikum Graz
Graz
Anette Schwerin-Nagel, Dr. med., anette.schwerin-nagel@medunigraz.at
Anette Schwerin-Nagel, Dr. med.
- Recruiting
Tirol Kliniken, Landeskrankenhaus Innsbruck
Innsbruck
Matthias Baumann, PD Dr. med., matthias.baumann@tirol-kliniken.at
Matthias Baumann, PD Dr. med.
- Not yet recruiting
Kepler Universitätsklinkum Linz
Linz
Astrid Eisenkölbl, Dr. med., astrid.eisenkoelbl@kepleruniklinikum.at
Astrid Eisenkölbl, Dr. med.
- Not yet recruiting
Landeskrankenhaus Salzburg
Salzburg
Johannes Koch, PD Dr. med., j.koch@salk.at
Johannes Koch, PD Dr. med.
- Not yet recruiting
Wiener Gesundheitsverbund, Klinik Favoriten
Vienna
Simone Mahal, Dr. med., simone.mahal@gesundheitsverbund.at
Simone Mahal, Dr. med.
About this study
Official title: Development and Implementation of a Transparent Registry Based on FAIR Principles for Duchenne and Becker Muscular Dystrophy (DBMD) Under the Auspices of the Society for Neuropediatrics (GNP) - FAIR-DMD
Duchenne muscular dystrophy (DMD) is an X-linked, recessive, progressive, and degenerative neuromuscular disorder that affects approximately one in 5,000 newborn boys. The established "standard of care" has improved prognosis; however, a causal therapy is not yet available. In 2024 and 2025, the first disease-modifying therapies were approved. These include Vamorolone (Agamree®) as a corticosteroid replacement with a more favorable side-effect profile for children aged four and older, and Givinostat (Duvyzat®) as a combination therapy with corticosteroids for ambulatory boys aged six and older. In this context, the FAIR-DMD Registry was initiated. The registry is based on the so-called FAIR principles. The acronym FAIR stands for the data principles Findable, Accessible, Interoperable and Reusable. The international FAIR principles are guidelines for the description, storage, and publication of scientific or administrative data. The FAIR-DMD registry is a disease-specific, academically managed registry for patients with Duchenne and Becker muscular dystrophy (DMD/BMD). Its goal is to systematically collect clinical data, scientifically monitor new disease-modifying therapies in routine care, and create an evidence-based foundation for the further development of diagnostics, therapy, and care structures. Furthermore, the registry collects data on patients' health related quality of live using an app for data entry. The FAIR-DMD Registry is being established under the auspices of the Society for Neuropediatrics (GNP) and operated in close coordination with Swiss Registry for Neuromuscular Disorders (Swiss-Reg-NMD). The GNP is a non-profit professional society that covers the entire spectrum of neuropediatric topics in clinical and cross-sector care. In the planned pilot phase, the GNP will act as trustee for financing. This model creates the opportunity to structurally address central challenges in health services research and establish a high-quality, internationally compatible registry structure. In the long term, the FAIR-DMD Registry aims to significantly improve care for DMD and BMD patients in German-speaking countries, evaluate the effectiveness of new therapies in clinical practice, and establish binding frameworks for quality-assured care.
Read the detailed description
The FAIR-DMD registry is a disease-specific, academically managed registry for patients with Duchenne and Becker muscular dystrophy (DMD/BMD).
Already in early childhood, affected boys show nonspecific developmental abnormalities, such as delays in speech or cognitive development or delayed achievement of motor milestones. Diagnosis is usually made between the ages of three and five through markedly elevated creatine kinase (CK) levels and molecular genetic analyses. The disease course is progressive: after a plateau phase in the preschool years, increasing muscle weakness-despite corticosteroid therapy-typically leads to loss of ambulation in early adolescence. Secondary complications arise from cardiac and respiratory involvement. Life-limiting factors include progressive cardiomyopathy, cardiac arrhythmias, and global respiratory failure, which usually necessitates non-invasive ventilation in early adulthood.
Currently, 26 specialized treatment centers in Germany and 5 in Austria are participating in the project. In Switzerland, a registry for DBMD patients is already being maintained, the Swiss Registry for Neuromuscular Disorders (Swiss-Reg-NMD). The registry is open to all centers that wish to participate in the future. Within the FAIR-DMD project, interoperability between FAIR-DMD and the Swiss registry is being pursued, and additional disease-specific and academically managed European Duchenne registries could be further developed for future data exchange. Data from patients treated with new disease-modifying therapies - which may in the future alter the progression of DMD and BMD - will also be included in the project.
Aims of the study are:
- Harmonization and standardization of care
- Support and evaluation of new disease-modifying therapies
- Improvement of care quality
- Establishment of the registry as an independent research tool
- Ensuring international cooperation and comparability
- Patient-centered approach and collection of subjective indicators
- Aim for sustainability and implementation into routine care To achieve these aims apart of the registry a telemedicine platform will be implemented. The telemedicine platform consists of a web-based dashboard for study personnel and an app for patients. The app will be used for appointment management, communication with the treating doctor and health care professionals, and to fill out Health Related Quality of Life questionnaires. Data from the telemedicine platform will automatically be transferred to the registry and vice versa.
Another measure in the FAIR-DMD project is to simplify the management of adverse drug reactions (ADR). New therapies are often approved despite very small patient populations in clinical trials. As a result, the majority of knowledge regarding their benefits - but especially their potential risks - emerges only after approval. In the future, as soon as new ADRs occur, they must be managed as effectively as possible, drawing on all available expertise and experience, following a "best practice" approach. Alongside the existing legally mandated ADR reporting procedures to the federal authorities and pharmaceutical companies, the process of digitally supported documentation of the clinical course following the occurrence of an ADR is to be optimized.
In FAIR-DMD it is planned that the report of an occurring ADR shall be automatically forwarded to the relevant authorities through the telemedicine platform, without requiring the same forms to be completed multiple times. To achieve this, we work very closely with the federal authority and pharmaceutical companies.
In addition to these measures FAIR-DMD aims to establish a therapy recommendation board, which also uses the telemedicine platform.
Our mission is to harmonize and standardize care for patients with dystrophinopathies, ensuring high-quality treatment and equitable access. We aim to support and evaluate innovative disease-modifying therapies, establish the registry as an independent research instrument, and foster international collaboration for global comparability. By prioritizing patient-centered approaches and integrating subjective indicators, we strive for sustainability and seamless implementation into routine clinical practice.
- Conditions
- Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Dystrophinopathy Symptomatic Female Carrier
- Keywords
- Muscular Dystrophies, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Muscular Dystrophy, Dystrophinopathy, FAIR Principles, Neuromuscular Diseases, Health Related Quality of Life (HRQoL)
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Neuromuscular Diseases
What is being measured
The primary outcome is the main question the study is designed to answer.
EQ-5D-5L
The EQ-5D is a validated generic instrument for measuring health-related quality of life. It assesses five dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) and enables the calculation of a health index from 0 (very poor) to 1 (best possible health).
Measured: * "Baseline" * "every six months" * "through study completion, maximum 15 years"
EQ VAS
A visual analog scale on which patients rate their current state of health on a scale from 0 ("worst possible") to 100 ("best possible").
Measured: * "Baseline" * "every six months" * "through study completion, maximum 15 years"
DMD-QoL
The DMD-QoL is a disease-specific questionnaire designed to assess the health-related quality of life of people with Duchenne muscular dystrophy. It takes into account physical, emotional, and social aspects as well as the impact of the disease on everyday life. In the DMD-QoL, the raw values of the individual scales are usually transformed onto a scale from 0 to 100. 0 represents the worst possible health-related quality of life, while 100 represents the best possible health-related quality of life-higher values therefore indicate a better perceived quality of life.
Measured: * "Baseline" * "every six months" * "through study completion, maximum 15 years"
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2026
- Study start13 Jul 2026
- Primary completionDec 2040 (estimated)
- Study completionJun 2041 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,500 participants (estimated)
- Target duration
- 15 Years
Sponsor and investigators
- Lead sponsor
- Dr. Andreas Ziegler
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Dr. Andreas Ziegler, University Hospital Heidelberg
- Investigators
- Ulrike Schara-Schmidt, Prof. Dr., Principal investigator, Universitätsklinikum Essen, Klinik für KinderheilkundeAndreas Ziegler, Dr., Study director, Universitätsklinikum Heidelberg, Zentrum für Kinder- und Jugendmedizin
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07402122
- Sponsor's protocol ID
- 25-12847-BO
- Acronym
- FAIR-DMD
- Status last verified
- Oct 2026
- First submitted
- 16 Jan 2026
- Last updated
- 8 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Development and Implementation of a Transparent Registry Based on FAIR Principles for Duchenne and Becker Muscular Dystrophy (DBMD) Under the Auspices of the Society for Neuropediatrics (GNP) - FAIR-DMD
- ClinicalTrials.gov ID
- NCT07402122
- Acronym / sponsor ID
- FAIR-DMD / 25-12847-BO
- Conditions
- Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Dystrophinopathy Symptomatic Female Carrier
- Keywords
- Muscular Dystrophies, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Muscular Dystrophy, Dystrophinopathy, FAIR Principles, Neuromuscular Diseases, Health Related Quality of Life (HRQoL)
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 1,500 participants (estimated)
- Lead sponsor
- Dr. Andreas ZieglerUniversity, hospital or other
- Study start
- 13 Jul 2026 (actual)
- Primary completion
- Dec 2040 (estimated)
- Study completion
- Jun 2041 (estimated)
- First posted
- 11 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Duchenne muscular dystrophy (DMD) is an X-linked, recessive, progressive, and degenerative neuromuscular disorder that affects approximately one in 5,000 newborn boys. The established "standard of care" has improved prognosis; however, a causal therapy is not yet available. In 2024 and 2025, the first disease-modifying therapies were approved. These include Vamorolone (Agamree®) as a corticosteroid replacement with a more favorable side-effect profile for children aged four and older, and Givinostat (Duvyzat®) as a combination therapy with corticosteroids for ambulatory boys aged six and older. In this context, the FAIR-DMD Registry was initiated. The registry is based on the so-called FAIR principles. The acronym FAIR stands for the data principles Findable, Accessible, Interoperable and Reusable. The international FAIR principles are guidelines for the description, storage, and publication of scientific or administrative data. The FAIR-DMD registry is a disease-specific, academically managed registry for patients with Duchenne and Becker muscular dystrophy (DMD/BMD). Its goal is to systematically collect clinical data, scientifically monitor new disease-modifying therapies in routine care, and create an evidence-based foundation for the further development of diagnostics, therapy, and care structures. Furthermore, the registry collects data on patients' health related quality of live using an app for data entry. The FAIR-DMD Registry is being established under the auspices of the Society for Neuropediatrics (GNP) and operated in close coordination with Swiss Registry for Neuromuscular Disorders (Swiss-Reg-NMD). The GNP is a non-profit professional society that covers the entire spectrum of neuropediatric topics in clinical and cross-sector care. In the planned pilot phase, the GNP will act as trustee for financing. This model creates the opportunity to structurally address central challenges in health services research and establish a high-quality, internationally compatible registry structure. In the long term, the FAIR-DMD Registry aims to significantly improve care for DMD and BMD patients in German-speaking countries, evaluate the effectiveness of new therapies in clinical practice, and establish binding frameworks for quality-assured care.
Detailed description
The FAIR-DMD registry is a disease-specific, academically managed registry for patients with Duchenne and Becker muscular dystrophy (DMD/BMD).
Already in early childhood, affected boys show nonspecific developmental abnormalities, such as delays in speech or cognitive development or delayed achievement of motor milestones. Diagnosis is usually made between the ages of three and five through markedly elevated creatine kinase (CK) levels and molecular genetic analyses. The disease course is progressive: after a plateau phase in the preschool years, increasing muscle weakness-despite corticosteroid therapy-typically leads to loss of ambulation in early adolescence. Secondary complications arise from cardiac and respiratory involvement. Life-limiting factors include progressive cardiomyopathy, cardiac arrhythmias, and global respiratory failure, which usually necessitates non-invasive ventilation in early adulthood.
Currently, 26 specialized treatment centers in Germany and 5 in Austria are participating in the project. In Switzerland, a registry for DBMD patients is already being maintained, the Swiss Registry for Neuromuscular Disorders (Swiss-Reg-NMD). The registry is open to all centers that wish to participate in the future. Within the FAIR-DMD project, interoperability between FAIR-DMD and the Swiss registry is being pursued, and additional disease-specific and academically managed European Duchenne registries could be further developed for future data exchange. Data from patients treated with new disease-modifying therapies - which may in the future alter the progression of DMD and BMD - will also be included in the project.
Aims of the study are:
- Harmonization and standardization of care
- Support and evaluation of new disease-modifying therapies
- Improvement of care quality
- Establishment of the registry as an independent research tool
- Ensuring international cooperation and comparability
- Patient-centered approach and collection of subjective indicators
- Aim for sustainability and implementation into routine care To achieve these aims apart of the registry a telemedicine platform will be implemented. The telemedicine platform consists of a web-based dashboard for study personnel and an app for patients. The app will be used for appointment management, communication with the treating doctor and health care professionals, and to fill out Health Related Quality of Life questionnaires. Data from the telemedicine platform will automatically be transferred to the registry and vice versa.
Another measure in the FAIR-DMD project is to simplify the management of adverse drug reactions (ADR). New therapies are often approved despite very small patient populations in clinical trials. As a result, the majority of knowledge regarding their benefits - but especially their potential risks - emerges only after approval. In the future, as soon as new ADRs occur, they must be managed as effectively as possible, drawing on all available expertise and experience, following a "best practice" approach. Alongside the existing legally mandated ADR reporting procedures to the federal authorities and pharmaceutical companies, the process of digitally supported documentation of the clinical course following the occurrence of an ADR is to be optimized.
In FAIR-DMD it is planned that the report of an occurring ADR shall be automatically forwarded to the relevant authorities through the telemedicine platform, without requiring the same forms to be completed multiple times. To achieve this, we work very closely with the federal authority and pharmaceutical companies.
In addition to these measures FAIR-DMD aims to establish a therapy recommendation board, which also uses the telemedicine platform.
Our mission is to harmonize and standardize care for patients with dystrophinopathies, ensuring high-quality treatment and equitable access. We aim to support and evaluate innovative disease-modifying therapies, establish the registry as an independent research instrument, and foster international collaboration for global comparability. By prioritizing patient-centered approaches and integrating subjective indicators, we strive for sustainability and seamless implementation into routine clinical practice.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 15 Years
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All patients meeting the eligibilty criteria in Germany and Austria.
Key inclusion criteria (8 listed)
- Genetically confirmed diagnosis of dystrophinopathy
- Female carriers only if they show clinical symptoms of a dystrophinopathy
- Treatment in one of the participating centers
- No age restrictions
- Ability to understand the patient information and sign the informed consent
Key exclusion criteria (3 listed)
- Missing legally valid consent form from the patient and/or legal guardians
- Project content is not understandable to the participant and/or legal guardians
- Not in possession of a tablet or smartphone
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
EQ-5D-5L
Time frame: * "Baseline" * "every six months" * "through study completion, maximum 15 years"
The EQ-5D is a validated generic instrument for measuring health-related quality of life. It assesses five dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) and enables the calculation of a health index from 0 (very poor) to 1 (best possible health).
EQ VAS
Time frame: * "Baseline" * "every six months" * "through study completion, maximum 15 years"
A visual analog scale on which patients rate their current state of health on a scale from 0 ("worst possible") to 100 ("best possible").
DMD-QoL
Time frame: * "Baseline" * "every six months" * "through study completion, maximum 15 years"
The DMD-QoL is a disease-specific questionnaire designed to assess the health-related quality of life of people with Duchenne muscular dystrophy. It takes into account physical, emotional, and social aspects as well as the impact of the disease on everyday life. In the DMD-QoL, the raw values of the individual scales are usually transformed onto a scale from 0 to 100. 0 represents the worst possible health-related quality of life, while 100 represents the best possible health-related quality of life-higher values therefore indicate a better perceived quality of life.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2026.
- App used in the study.Link provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Ulrike Schara-Schmidt, Prof. Dr. principal investigator
Universitätsklinikum Essen, Klinik für Kinderheilkunde
Andreas Ziegler, Dr. study director
Universitätsklinikum Heidelberg, Zentrum für Kinder- und Jugendmedizin
30 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy, run by Dr. Andreas Ziegler at 30 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 8 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in Germany, Austria. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Andreas Ziegler, Dr. (004962215632207, FAIR-DMD@med.uni-heidelberg.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
EQ-5D-5L (time frame: * "Baseline" * "every six months" * "through study completion, maximum 15 years"). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Oct 2026.