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Treatment Effect of Tamoxifen on Patients With DMD

Primary Purpose

Duchenne Muscular Dystrophy

Status
Completed
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
Tamoxifen
Sponsored by
Hadassah Medical Organization
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Duchenne Tamoxifen

Eligibility Criteria

5 Years - 16 Years (Child)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  • Ambulatory

Exclusion Criteria:

  • Non Ambulatory

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Other

    Arm Label

    open label study

    Arm Description

    one arm open label study

    Outcomes

    Primary Outcome Measures

    6-minute walk distance (6MWD)
    PT evaluation

    Secondary Outcome Measures

    North Star assesment(NSAA)
    PT evaluation

    Full Information

    First Posted
    June 22, 2016
    Last Updated
    July 21, 2022
    Sponsor
    Hadassah Medical Organization
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    1. Study Identification

    Unique Protocol Identification Number
    NCT02835079
    Brief Title
    Treatment Effect of Tamoxifen on Patients With DMD
    Official Title
    Treatment Effect of Tamoxifen on Patients With DMD
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    July 2022
    Overall Recruitment Status
    Completed
    Study Start Date
    November 2016 (Actual)
    Primary Completion Date
    November 2020 (Actual)
    Study Completion Date
    November 2020 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Principal Investigator
    Name of the Sponsor
    Hadassah Medical Organization

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    No

    5. 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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Duchenne Muscular Dystrophy
    Keywords
    Duchenne Tamoxifen

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 1
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    19 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    open label study
    Arm Type
    Other
    Arm Description
    one arm open label study
    Intervention Type
    Drug
    Intervention Name(s)
    Tamoxifen
    Primary Outcome Measure Information:
    Title
    6-minute walk distance (6MWD)
    Description
    PT evaluation
    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.
    Secondary Outcome Measure Information:
    Title
    North Star assesment(NSAA)
    Description
    PT evaluation
    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.

    10. Eligibility

    Sex
    Male
    Minimum Age & Unit of Time
    5 Years
    Maximum Age & Unit of Time
    16 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Ambulatory Exclusion Criteria: Non Ambulatory

    12. IPD Sharing Statement

    Citations:
    PubMed Identifier
    34304968
    Citation
    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.
    Results Reference
    derived

    Learn more about this trial

    Treatment Effect of Tamoxifen on Patients With DMD

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