Treatment Effect of Tamoxifen on Patients With DMD

Status Completed

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT02835079
Study type	Interventional
Source	Hadassah Medical Organization
Contact
Status	Completed
Phase	Phase 1
Start date	November 2016
Completion date	November 2020

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT05575648` - Dual Task in Duchenne Muscular Dystrophy	N/A
Terminated	`NCT03907072` - Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy	Phase 2/Phase 3
Not yet recruiting	`NCT06450639` - An Open-label Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy	Phase 2
Completed	`NCT04335942` - Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor	N/A
Active, not recruiting	`NCT04906460` - Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)	Phase 1/Phase 2
Active, not recruiting	`NCT02500381` - Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)	Phase 3
Enrolling by invitation	`NCT05967351` - A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study	Phase 3
Recruiting	`NCT03067831` - Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy	Phase 1/Phase 2
Recruiting	`NCT01834040` - Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy	Phase 1/Phase 2
Completed	`NCT02246478` - A Study of TAS-205 for Duchenne Muscular Dystrophy	Phase 1
Active, not recruiting	`NCT01772043` - Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping	N/A
Terminated	`NCT01168908` - Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy	Phase 2
Completed	`NCT00758225` - Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)	Phase 2
Completed	`NCT03680365` - Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
Recruiting	`NCT03513367` - The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM.
Recruiting	`NCT05712447` - Duchenne Muscular Dystrophy Video Assessment Registry
Recruiting	`NCT01484678` - Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
Completed	`NCT03319030` - Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)
Terminated	`NCT01753804` - A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.	N/A
Completed	`NCT02530905` - Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients	Phase 1