Tamoxifen Treatment in Patients With Motor Neuron Disease

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

The Study of Tamoxifen Treatment in Patients With Motor Neuron Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02166944
• Other study ID #: 201307022
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: April 2014
• Est. completion date: September 18, 2019

Study information

• Verified date: June 2014
• Source: Taipei Medical University Shuang Ho Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: September 18, 2019
• Est. primary completion date: September 17, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 20 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• ALS Functional Ration Scale
• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• mTOR
• Sclerosis
• Tamoxifen
• TAR-DNA-binding Protein-43

Intervention

Drug:
• tamoxifen 40 mg daily for one year
both arms with riluzole daily

Locations

Country	Name	City	State
Taiwan	Po-Chih Chen	New Taipei City

Sponsors (1)

Lead Sponsor	Collaborator
Taipei Medical University Shuang Ho Hospital

Country where clinical trial is conducted

Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Change from Baseline in blood TDP43 related biomarkers at 1, 3, 6,12 months		baseline, month 1, 3, 6, 12
Primary	Change from Baseline in Amyotrophic Lateral Sclerosis Functional Ration Scales (ALSFRS) at 1, 3, 6,12 months	Amyotrophic Lateral Sclerosis Functional Ration Scales (ALSFRS) measured by a neurologist	Baseline, month 1, 3, 6, 12
Secondary	Change from Baseline in pulmonary function test at 1, 3, 6,12 months	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)	baseline, month 1, 3, 6, 12