Treatment Effect of Tamoxifen on Patients With DMD

• ClinicalTrials.gov Identifier: NCT02835079
• Recruitment Status: Completed
• First Posted: July 15, 2016
• Last Update Posted: July 25, 2022
• Sponsor: Hadassah Medical Organization
• Information provided by (Responsible Party): Talia Dor, Hadassah Medical Organization

Tracking Information

• First Submitted Date: June 22, 2016
• First Posted Date: July 15, 2016
• Last Update Posted Date: July 25, 2022
• Actual Study Start Date: November 2016
• Actual Primary Completion Date: November 2020 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: July 21, 2022)

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

• Original Primary Outcome Measures (submitted: July 13, 2016): 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. ]

Current Secondary Outcome Measures (submitted: July 21, 2022)

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

• Original Secondary Outcome Measures (submitted: July 13, 2016): 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: Treatment Effect of Tamoxifen on Patients With DMD
• Official Title: 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.

• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Duchenne Muscular Dystrophy
• Intervention: Drug: Tamoxifen

Study Arms

open label study

one arm open label study

Intervention: Drug: Tamoxifen

• 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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: August 1, 2018): 19
• Original Estimated Enrollment (submitted: July 13, 2016): 30
• Actual Study Completion Date: November 2020
• Actual Primary Completion Date: November 2020 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Ambulatory

Exclusion Criteria:

• Non Ambulatory

Sex/Gender

• Sexes Eligible for Study: Male

• Ages: 5 Years to 16 Years (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Not Provided

Administrative Information

• NCT Number: NCT02835079
• Other Study ID Numbers: DMDTAM001-HMO-CTIL
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Talia Dor, Hadassah Medical Organization
• Original Responsible Party: Same as current
• Current Study Sponsor: Hadassah Medical Organization
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Hadassah Medical Organization
• Verification Date: July 2022