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Tamoxifen Treatment in Patients With Motor Neuron Disease

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ClinicalTrials.gov Identifier: NCT02166944
Recruitment Status : Unknown
Verified June 2014 by Taipei Medical University Shuang Ho Hospital.
Recruitment status was:  Recruiting
First Posted : June 18, 2014
Last Update Posted : June 18, 2014
Sponsor:
Information provided by (Responsible Party):
Taipei Medical University Shuang Ho Hospital

Brief 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.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis ALS Functional Ration Scale TAR-DNA-binding Protein-43 Tamoxifen mTOR Drug: tamoxifen 40 mg daily for one year Phase 1 Phase 2

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.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: The Study of Tamoxifen Treatment in Patients With Motor Neuron Disease
Study Start Date : April 2014
Estimated Primary Completion Date : May 2016
Estimated Study Completion Date : July 2016


Arm Intervention/treatment
Experimental: tamoxifen
tamoxifen 40 mg daily for one year
Drug: tamoxifen 40 mg daily for one year
both arms with riluzole daily
Other Name: Nolvadex

Placebo Comparator: placebo
placebo drugs



Primary Outcome Measures :
  1. Change from Baseline in Amyotrophic Lateral Sclerosis Functional Ration Scales (ALSFRS) at 1, 3, 6,12 months [ Time Frame: Baseline, month 1, 3, 6, 12 ]
    Amyotrophic Lateral Sclerosis Functional Ration Scales (ALSFRS) measured by a neurologist


Secondary Outcome Measures :
  1. Change from Baseline in pulmonary function test at 1, 3, 6,12 months [ Time Frame: baseline, month 1, 3, 6, 12 ]

    Expiratory reserve volume (ERV) Forced vital capacity (FVC) Forced expiratory volume (FEV) Forced expiratory flow 25% to 75% Functional residual capacity (FRC) Maximum voluntary ventilation (MVV)

    • Residual volume (RV)
    • Peak expiratory flow (PEF).
    • Slow vital capacity (SVC)
    • Total lung capacity (TLC)


Other Outcome Measures:
  1. Change from Baseline in blood TDP43 related biomarkers at 1, 3, 6,12 months [ Time Frame: baseline, month 1, 3, 6, 12 ]


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Ages Eligible for Study:   20 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02166944


Contacts
Contact: Po-Chih Chen, M.D. 886-2-22490088 ext 8112 euphemus2003@msn.com
Contact: Chaur-Jong Hu, M.D. 886-2-22490088 ext 8112 chaurjongh@tmu.edu.tw

Locations
Taiwan
Po-Chih Chen Recruiting
New Taipei City, Taiwan
Contact: Po-Chih Chen, M.D.    886-2-22490088 ext 8112    euphemus2003@msn.com   
Contact: Chaur-Jong Hu, M.D.    886-2-22490088 ext 8112    chaurjongh@tmu.edu.tw   
Principal Investigator: Chaur-Jong Hu, M.D.         
Sponsors and Collaborators
Taipei Medical University Shuang Ho Hospital
Investigators
Principal Investigator: Chaur-Jong Hu, M.D. Shung Ho Hospital, Taipei Meidcal University

Responsible Party: Taipei Medical University Shuang Ho Hospital
ClinicalTrials.gov Identifier: NCT02166944     History of Changes
Other Study ID Numbers: 201307022
First Posted: June 18, 2014    Key Record Dates
Last Update Posted: June 18, 2014
Last Verified: June 2014

Keywords provided by Taipei Medical University Shuang Ho Hospital:
amyotrophic lateral sclerosis
ALS functional ration scale
TAR-DNA-binding protein-43
tamoxifen
mTOR

Additional relevant MeSH terms:
Sclerosis
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Tamoxifen
Estrogen Antagonists
Hormone Antagonists
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Selective Estrogen Receptor Modulators
Estrogen Receptor Modulators
Bone Density Conservation Agents