Skip to main content
OpenTrials
Completed

NCT Number: NCT02166944

Tamoxifen Treatment in Patients With Motor Neuron Disease

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Amyotrophic Lateral Sclerosis ALS Functional Ration Scale Central Nervous System Diseases MTOR Metabolic Diseases Motor Neuron Disease Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Proteostasis Deficiencies Spinal Cord Diseases TAR-DNA-binding Protein-43 TDP-43 Proteinopathies Tamoxifen

Age range

20 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Po-Chih Chen

New Taipei City, Taiwan

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.
  • Age ≧20 years old

Exclusion criteria

  • 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.
  • Patients with now or previous usage of Tamoxifen
  • Patients with any contraindications of Tamoxifen usage
  • Patients with other internal medicine illiness

Treatment and study plan

tamoxifen 40 mg daily for one year

Drug

both arms with riluzole daily

Other names: Nolvadex

Primary outcomes

  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 outcomes

  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 outcomes

  1. Change from Baseline in blood TDP43 related biomarkers at 1, 3, 6,12 months

    Time frame: baseline, month 1, 3, 6, 12

Sponsors and collaborators

Lead sponsor

Taipei Medical University Shuang Ho Hospital

Other

Registry information

Official study title

The Study of Tamoxifen Treatment in Patients With Motor Neuron Disease

Important dates

Study start
2014
Primary completion
2019
Study completion
2019
First posted
Jun 18, 2014
Registry last updated
Sep 19, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.