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Treatment Effect of Tamoxifen on Patients With DMD

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ClinicalTrials.gov Identifier: NCT02835079
Recruitment Status : Not yet recruiting
First Posted : July 15, 2016
Last Update Posted : July 15, 2016
Sponsor:
Information provided by (Responsible Party):
Talia Dor, Hadassah Medical Organization

June 22, 2016
July 15, 2016
July 15, 2016
November 2016
November 2019   (Final data collection date for primary outcome measure)
6-minute walk distance (6MWD) [ Time Frame: The six minute walk distance will be tested during the 36 months of the trial. For the first 12 months, the 6 minute walk test will be tested every 3 months and for the follow up period of 24 months,will be done every 6 months. ]
Same as current
No Changes Posted
North Star assesment(NSAA) [ Time Frame: The NSAA will be tested during the 36 months of the trial. For the first 12 months, the NSAA will be tested every 3 months and for the follow up period of 24 months,will be done every 6 months. ]
Same as current
Not Provided
Not Provided
 
Treatment Effect of Tamoxifen on Patients With DMD
Not Provided

Duchenne muscular dystrophy (DMD) is a progressive devastating disease that affects mainly boys, with an incidence of about 1:3,500 live births. The pathology of DMD is a result of non-repaired muscle damage that leads to muscle-tissue replacement by scar tissue, a process known as fibrosis. Currently, there is no effective treatment for the disease. The only therapy offered to these boys are steroids which slightly delayed the disease progression. The boys lose their ability to walk at around the age of 12, and die in the 4th decade of life from severe heart and lung problems.

In this study investigators will test the efficacy of Tamoxifen treatment in ambulatory DMD boys. Tamoxifen is a drug used for palliative treatment of breast cancer patients and has an outstanding safety profile. In addition, Tamoxifen was tested in the past in boys, for other pediatric indications, and showed an excellent safety with no side effects.

Tamoxifen is being tested in this study, as a therapy for DMD, for the following reasons:

(i) it was shown to have anti-fibrotic effect in multiple in-vivo systems; (ii) it assists in the repair of damaged muscles.

In other words, Tamoxifen is expected to have a synergistic effect on DMD patients, due to its dual mechanism of action. Indeed, Tamoxifen was shown to have significant beneficial effects in the mdx mouse model of DMD. Also, a small compassionate cohort of 3 boys, treated for 6 months with Tamoxifen, yielded very encouraging results.

Not Provided
Interventional
Phase 1
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Duchenne Muscular Dystrophy
Drug: Tamoxifen
open label study
one arm open label study
Intervention: Drug: Tamoxifen
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Not yet recruiting
30
Same as current
Not Provided
November 2019   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Ambulatory

Exclusion Criteria:

  • Non Ambulatory
Sexes Eligible for Study: Male
5 Years to 16 Years   (Child)
No
Not Provided
 
 
NCT02835079
DMDTAM001-HMO-CTIL
Not Provided
Not Provided
Not Provided
Talia Dor, Hadassah Medical Organization
Hadassah Medical Organization
Not Provided
Not Provided
Hadassah Medical Organization
July 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP