Tamoxifen Treatment in Patients With Motor Neuron Disease
Information source: Taipei Medical University Shuang Ho Hospital
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Amyotrophic Lateral Sclerosis; ALS Functional Ration Scale; TAR-DNA-binding Protein-43; Tamoxifen; mTOR
Intervention: tamoxifen 40 mg daily for one year (Drug)
Phase: Phase 1/Phase 2
Status: Recruiting
Sponsored by: Taipei Medical University Shuang Ho Hospital Official(s) and/or principal investigator(s): Chaur-Jong Hu, M.D., Principal Investigator, Affiliation: Shung Ho Hospital, Taipei Meidcal University
Overall contact: Po-Chih Chen, M.D., Phone: 886-2-22490088, Ext: 8112, Email: euphemus2003@msn.com
Summary
The aim of this study is to survey the effect of Tamoxifen in motor neuron disease (MND)
patients, amyotrophic lateral sclerosis (ALS) with regular riluzole usage. TDP-43 is
related to ALS. Increased the ubiquitinated or phosphorylated TDP-43 can cause animal model
of ALS, and TDP43 can be degraded either by proteasome or autophagy pathway system.
Autophagy pathway can be activated by mTOR inhibition, resulting in ameliorating TDP-43
accumulation and rescue in motor function in animal model. Tamoxifen had shown ability of
enhance both proteasome and autophagy pathway, therefore the investigators assume that
Tamoxifen probably can ameliorate TDP-43 accumulation and inclusion body formation in ALS.
Clinical Details
Official title: The Study of Tamoxifen Treatment in Patients With Motor Neuron Disease
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Primary outcome: Change from Baseline in Amyotrophic Lateral Sclerosis Functional Ration Scales (ALSFRS) at 1, 3, 6,12 months
Secondary outcome: Change from Baseline in pulmonary function test at 1, 3, 6,12 months
Detailed description:
The investigators will assess the ALSFR-s in ALS patients at start, 1, 3, 6 and 12 months
and correlate the score to the neurological outcome of the patients with and without
tamoxifen treatment at dose of 40mg daily for one year.
The study will be able to prove the investigators hypothesis: Tamoxifen, a protease and
autophagy enhancer, has synergic effect with riluzole in ALS patients to slowing the
progression of neurological dysfunction, and respiratory insufficiency.
Eligibility
Minimum age: 20 Years.
Maximum age: 80 Years.
Gender(s): Both.
Criteria:
Inclusion Criteria:
1. Clinical diagnosed and confirmed ALS patients, with regular follow up and oral form
riluzole at National Taiwan University or Shuang- Ho Hospital for more than 6 months.
2. Age ≧20 years old
Exclusion Criteria:
1. Patients who had already ventilator dependent, not regular followed up for more than
6 months or against medical advice, refuse to follow up at neurology department will
be excluded in this study.
2. Patients with now or previous usage of Tamoxifen
3. Patients with any contraindications of Tamoxifen usage
4. Patients with other internal medicine illiness
Locations and Contacts
Po-Chih Chen, M.D., Phone: 886-2-22490088, Ext: 8112, Email: euphemus2003@msn.com
Po-Chih Chen, New Taipei City, Taiwan; Recruiting Po-Chih Chen, M.D., Phone: 886-2-22490088, Ext: 8112, Email: euphemus2003@msn.com Chaur-Jong Hu, M.D., Phone: 886-2-22490088, Ext: 8112, Email: chaurjongh@tmu.edu.tw Chaur-Jong Hu, M.D., Principal Investigator
Additional Information
Starting date: April 2014
Last updated: June 16, 2014
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