The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease.
- Conditions
- Duchenne muscular dystrophyMedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2017-004554-42-DE
- Lead Sponsor
- niversity of Basel Children's Hospital, Division of Neuropediatrics
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- Not specified
- Target Recruitment
- 100
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
- 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
Are the trial subjects under 18? yes
Number of subjects for this age range: 100
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
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-Bickelsyndrome);
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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method