Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE1
19 participants
INTERVENTIONAL
2016-11-30
2020-11-30
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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open label study
one arm open label study
Tamoxifen
Interventions
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Tamoxifen
Eligibility Criteria
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Inclusion Criteria
Exclusion Criteria
5 Years
16 Years
MALE
No
Sponsors
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Hadassah Medical Organization
OTHER
Responsible Party
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Talia Dor
MD
References
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Tsabari R, Simchovitz E, Lavi E, Eliav O, Avrahami R, Ben-Sasson S, Dor T. Safety and clinical outcome of tamoxifen in Duchenne muscular dystrophy. Neuromuscul Disord. 2021 Sep;31(9):803-813. doi: 10.1016/j.nmd.2021.05.005. Epub 2021 Jun 5.
Other Identifiers
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DMDTAM001-HMO-CTIL
Identifier Type: -
Identifier Source: org_study_id
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