Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE3
93 participants
INTERVENTIONAL
2018-06-12
2022-10-18
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
An open label extension (OLE) trial for participants of the TAMDMD main study will be performed. All TAMDMD patients on TAM or placebo are offered to enter this OLE.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
The investigators plan to screen at least 79 and to enroll at least 71 ambulant DMD patients aged between 6.5 and 12 years (group A) and 6 - 20 non-ambulant DMD patients aged between 10 and 16 years (group B). In order to reach statistical power, 60 ambulant patients (group A) need to complete the trial. Treatment with 20 mg Tamoxifen once daily will be given for the total trial duration of 48 weeks.
Only patients with glucocorticoids (standard treatment of care) will be included in group A (ambulant patients) and only non-glucocorticoid users in group B. At baseline as well as at the end of the study clinical, laboratory, and MRI measurements will be performed. These include the Motor Function Measure (MFM) scale, timed function tests, the 6 minute walking distance, quantitative muscle testing (QMT) and quantitative thigh muscle MRI, questionnaires. A physical examination, an ECG, vital signs as well as safety laboratory blood analyses will be performed at every visit. Furthermore, an x-ray of the hand and a dual energy x-ray absorptiometry (DEXA)-scan will be performed at baseline and at the end of the study.
An open label extension (OLE) trial for participants of the TAMDMD main study will be performed. All TAMDMD patients on TAM or placebo are offered to enter this OLE. All OLE patients will receive 20 mg of TAM daily during 48 weeks. The same study specific assessments as in the double-blind randomized phase will be performed during the OLE phase
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Keywords
Explore important study keywords that can help with search, categorization, and topic discovery.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Tamoxifen 20 mg once daily
DMD patients randomised to verum will receive 20 mg (0.6mg/kg) of TAM daily.
Tamoxifen
DMD patients randomised to verum will receive 20 mg (0.6mg/kg) of Tamoxifen daily. Treatment will be given for the total period of 48 weeks.
Matching placebo once daily
Patients randomised to placebo will be administered matching placebo.
Matching placebo
Patients randomised to placebo will be administered matching placebo. Treatment will be given for the total period of 48 weeks.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Tamoxifen
DMD patients randomised to verum will receive 20 mg (0.6mg/kg) of Tamoxifen daily. Treatment will be given for the total period of 48 weeks.
Matching placebo
Patients randomised to placebo will be administered matching placebo. Treatment will be given for the total period of 48 weeks.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Documented diagnosis of DMD by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e. absent or \<5% of normal) on Western blot or immunostaining
* Stable treatment with glucocorticoids \>6 months (no significant change in dosage (\>0.2mg/kg)) at screening; dosing adaptations according to weight change are allowed
* Male gender
* 6.5 to 12 years of age at time of screening
* weight \>20kg
* ambulant patients
* able to walk at least 350 meters in 6 minute walking distance test without assistance at screening
* MFM D1 subdomain of the MFM scale \>40% at screening
* Ability to provide informed consent and to comply with study requirements
* Patients harbouring a nonsense mutation treatable with the approved drug ataluren should be under stable ataluren treatment for at least 3 months or in case of nontolerance being off ataluren treatment for at least 3 months before screening
Group B (non-ambulant patients)
* Documented diagnosis of DMD by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e. absent or \<5% of normal) on Western blot or immunostaining
* Not using glucocorticoids for \>6 months
* Male gender
* Non-ambulant patients (walking distance less than 10 meters)
* 10 to 16 years of age at time of screening
* Ability to provide informed consent and to comply with study requirements
Open label extension
\- Recent participation and completion of TAMDMD study
Exclusion Criteria
* Female gender
* Use of tamoxifen or testosterone within the last 3 months
* Known or suspected malignancy
* Other chronic disease or clinically relevant limitation of renal, liver or heart function
* Known or suspected non-compliance
* Any injury which may impact functional testing, e.g. upper or lower limb fracture
* Planned or expected spinal fusion surgery during the study period (as judged by the Investigator; i.e. due to rapid progressing scoliosis), previous spinal fusion surgery is allowed if it took place more than 6 months prior to screening.
* Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders of the participant/parents (as judged by the investigator)
* Concomitant participation in any other interventional trial (and up to 3 months prior to screening)
* Use of CYP2D6 inhibitors or of CYP3A4 inducers (apart from glucocorticoids), platelet aggregation inhibitors and coumarin-type anti-coagulants
* Use of drugs metabolized by CYP2C9, such as phenprocoumon, phenytoin, warfarin, celecoxib, fluvastatin, ginko biloba, St. John's wort and sulfamethoxazol
* Galactosemia (lack of galactose-1-phosphat-uridylyltransferase or UDP-galactose-4-epimerase or galactokinase; Fanconi-Bickel-syndrome); congenital lack of lactase; glucose-galactose malabsorption
* Presence of one or more of the following eye disorders: cataract, retinopathia, optic neuropathy, alteration of the cornea
* Presence of one or more of the following laboratory abnormalities: anaemia, thrombocytopenia, leukopenia, neutropenia or agranulocytosis
Group A:
* Glucocorticoid naïve patients
* Start of glucocorticoid treatment or change in dosage \<6 month prior to screening (dosing adaptations according to weight change are allowed)
Group B:
* Glucocorticoid treated patients or patients that stopped glucocorticoid treatment \<6 month prior to screening
* Assisted ventilation of any kind necessary
78 Months
16 Years
MALE
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
University Hospital, Basel, Switzerland
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Dirk Fischer, MD
Role: PRINCIPAL_INVESTIGATOR
University Children's Hospital Basel
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Hôpitaux Raymond Poincaré
Garches, , France
Hôpital de Hautepierre
Strasbourg, , France
DRK Klinik Berlin Westend
Berlin, , Germany
Universitätsklinikum Essen
Essen, , Germany
Radboud umc
Nijmegen, , Netherlands
Hospital Sant Joan de Déu. UB
Esplugues de Llobregat, Barcelona, Spain
Hospital Universitario Virgen del Rocío
Seville, , Spain
University Children's Hospital Basel
Basel, , Switzerland
Royal Hospital for Children
Glasgow, , United Kingdom
The Leeds Teaching Hospitals NHS Trust
Leeds, , United Kingdom
Alder Hey Children's Hospital
Liverpool, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
Henzi BC, Putananickal N, Schmidt S, Nagy S, Rubino-Nacht D, Schaedelin S, Amthor H, Childs AM, Deconinck N, Horrocks I, Houwen-van Opstal S, Laugel V, Lobato ML, Osorio AN, Schara-Schmidt U, Spinty S, von Moers A, Lawrence F, Hafner P, Dorchies OM, Fischer D. Safety and efficacy of tamoxifen in non-ambulant patients with Duchenne muscular dystrophy: a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial (TAMDMD Group B). Neuromuscul Disord. 2025 Feb;47:105275. doi: 10.1016/j.nmd.2025.105275. Epub 2025 Jan 16.
Henzi BC, Schmidt S, Nagy S, Rubino-Nacht D, Schaedelin S, Putananickal N, Stimpson G; North Star Consortium; Amthor H, Childs AM, Deconinck N, de Groot I, Horrocks I, Houwen-van Opstal S, Laugel V, Lopez Lobato M, Madruga Garrido M, Nascimento Osorio A, Schara-Schmidt U, Spinty S, von Moers A, Lawrence F, Hafner P, Dorchies OM, Fischer D. Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2023 Oct;22(10):890-899. doi: 10.1016/S1474-4422(23)00285-5.
Nagy S, Hafner P, Schmidt S, Rubino-Nacht D, Schadelin S, Bieri O, Fischer D. Tamoxifen in Duchenne muscular dystrophy (TAMDMD): study protocol for a multicenter, randomized, placebo-controlled, double-blind phase 3 trial. Trials. 2019 Nov 21;20(1):637. doi: 10.1186/s13063-019-3740-6.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
TAMDMD
Identifier Type: -
Identifier Source: org_study_id