Treatment Effect of Tamoxifen on Patients With DMD

• Sponsor: Hadassah Medical Organization (Other)
• Overall Status: Completed
• CT.gov ID: NCT02835079
• Collaborator: (none)
• Enrollment: 19
• Arm: 1
• Actual Duration (Months): 48

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Duchenne Muscular Dystrophy	Drug: Tamoxifen	Phase 1

Study Design

• Study Type: Interventional
• Actual Enrollment: 19 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Treatment Effect of Tamoxifen on Patients With DMD
• Actual Study Start Date: Nov 1, 2016
• Actual Primary Completion Date: Nov 1, 2020
• Actual Study Completion Date: Nov 1, 2020

Arms and Interventions

Arm	Intervention/Treatment
Other: open label study one arm open label study	Drug: Tamoxifen

Outcome Measures

Primary Outcome Measures

1. 6-minute walk distance (6MWD) [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

Secondary Outcome Measures

1. North Star assesment(NSAA) [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

Eligibility Criteria

Criteria

• Ages Eligible for Study: 5 Years to 16 Years
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Ambulatory

Exclusion Criteria:

• Non Ambulatory

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

• Hadassah Medical Organization

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Talia Dor, MD, Hadassah Medical Organization
• ClinicalTrials.gov Identifier: NCT02835079
• Other Study ID Numbers: DMDTAM001-HMO-CTIL
• First Posted: Jul 15, 2016
• Last Update Posted: Jul 25, 2022
• Last Verified: Jul 1, 2022

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Talia Dor, MD, Hadassah Medical Organization: Duchenne Tamoxifen
• Additional relevant MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne; Tamoxifen

Study Results

No Results Posted as of Jul 25, 2022