Status:
COMPLETED
Tamoxifen in Duchenne Muscular Dystrophy
Lead Sponsor:
University Hospital, Basel, Switzerland
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
78-16 years
Phase:
PHASE3
Brief Summary
A randomised, double blind, placebo controlled, 48-week clinical trial with a core population (group A) of 79 ambulant 6.5 to 12 years old Duchenne's muscular dystrophy (DMD) patients that are under s...
Detailed Description
This is a 48-week multicentre, parallel, randomised, double-blind, placebo controlled phase 3 safety and efficacy trial. There are two treatment arms: Tamoxifen (verum) and placebo (control), with tre...
Eligibility Criteria
Inclusion
- Group A (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
- 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
- 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
- Known individual hypersensitivity or allergy to tamoxifen or other ingredients/excipients of IMP
- 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
Key Trial Info
Start Date :
June 12 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 18 2022
Estimated Enrollment :
93 Patients enrolled
Trial Details
Trial ID
NCT03354039
Start Date
June 12 2018
End Date
October 18 2022
Last Update
December 20 2022
Active Locations (11)
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1
Hôpitaux Raymond Poincaré
Garches, France, 92380
2
Hôpital de Hautepierre
Strasbourg, France, 67098
3
DRK Klinik Berlin Westend
Berlin, Germany, 14050
4
Universitätsklinikum Essen
Essen, Germany, 45147