Treatment Effect of Tamoxifen on Patients With DMD
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ClinicalTrials.gov Identifier: NCT02835079 |
Recruitment Status :
Completed
First Posted : July 15, 2016
Last Update Posted : July 25, 2022
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Tracking Information | |||||
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First Submitted Date ICMJE | June 22, 2016 | ||||
First Posted Date ICMJE | July 15, 2016 | ||||
Last Update Posted Date | July 25, 2022 | ||||
Actual Study Start Date ICMJE | November 2016 | ||||
Actual Primary Completion Date | November 2020 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
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. ] PT evaluation
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Original Primary Outcome Measures ICMJE |
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. ] | ||||
Change History | |||||
Current Secondary Outcome Measures ICMJE |
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. ] PT evaluation
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Original Secondary Outcome Measures ICMJE |
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. ] | ||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | Treatment Effect of Tamoxifen on Patients With DMD | ||||
Official Title ICMJE | Treatment Effect of Tamoxifen on Patients With DMD | ||||
Brief Summary | 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. |
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Detailed Description | Not Provided | ||||
Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 1 | ||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Duchenne Muscular Dystrophy | ||||
Intervention ICMJE | Drug: Tamoxifen | ||||
Study Arms ICMJE | open label study
one arm open label study
Intervention: Drug: Tamoxifen
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Publications * | 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. Erratum In: Neuromuscul Disord. 2022 Mar;32(3):e5-e7. | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Completed | ||||
Actual Enrollment ICMJE |
19 | ||||
Original Estimated Enrollment ICMJE |
30 | ||||
Actual Study Completion Date ICMJE | November 2020 | ||||
Actual Primary Completion Date | November 2020 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 5 Years to 16 Years (Child) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | Not Provided | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT02835079 | ||||
Other Study ID Numbers ICMJE | DMDTAM001-HMO-CTIL | ||||
Has Data Monitoring Committee | No | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE | Not Provided | ||||
Current Responsible Party | Talia Dor, Hadassah Medical Organization | ||||
Original Responsible Party | Same as current | ||||
Current Study Sponsor ICMJE | Hadassah Medical Organization | ||||
Original Study Sponsor ICMJE | Same as current | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE | Not Provided | ||||
PRS Account | Hadassah Medical Organization | ||||
Verification Date | July 2022 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |