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Tamoxifen in Duchenne Muscular Dystrophy

NCT03354039 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 93

Last updated 2022-12-20

No results posted yet for this study

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.

Conditions

Interventions

DRUG

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.

DRUG

Matching placebo

Patients randomised to placebo will be administered matching placebo. Treatment will be given for the total period of 48 weeks.

Sponsors & Collaborators

  • University Hospital, Basel, Switzerland

    lead OTHER

Principal Investigators

  • Dirk Fischer, MD · University Children's Hospital Basel

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
78 Months
Max Age
16 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-06-12
Primary Completion
2022-07-25
Completion
2022-10-18

Countries

  • France
  • Germany
  • Netherlands
  • Spain
  • Switzerland
  • United Kingdom

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03354039 on ClinicalTrials.gov