Tamoxifen in Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Dystrophy
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne
Study type: Interventional
Phase: Phase 3

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 stable standard treatment of care with glucocorticoids. Furthermore, the
      investigators plan to include 6-20 non-ambulant patients who do not receive glucocorticoids
      (as parallel group B), 10 to 16 years old, to obtain efficacy and safety data in a broader
      DMD population. All patients will receive 20 mg of tamoxifen (TAM) or placebo once daily
      during 48 weeks.

      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: 
      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 treatment allocation of 1:1.

      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
    

Intervention name(s): Tamoxifen; Matching placebo
Intervention type(s): Drug; Drug
Intervention description(s): 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.; Patients randomised to placebo will be administered matching placebo. Treatment will be given for the total period of 48 weeks.
Intervention MeSH terms: Tamoxifen
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: DMD