NCT02166944

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Apr 2014

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 2014

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

June 9, 2014

Completed
9 days until next milestone

First Posted

Study publicly available on registry

June 18, 2014

Completed
5.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 17, 2019

Completed
1 day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 18, 2019

Completed
Last Updated

September 19, 2019

Status Verified

June 1, 2014

Enrollment Period

5.5 years

First QC Date

June 9, 2014

Last Update Submit

September 17, 2019

Conditions

Amyotrophic Lateral SclerosisALS Functional Ration ScaleTAR-DNA-binding Protein-43TamoxifenmTOR

Keywords

amyotrophic lateral sclerosisALS functional ration scaleTAR-DNA-binding protein-43tamoxifenmTOR

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (1)

  • Change from Baseline in pulmonary function test at 1, 3, 6,12 months

    baseline, month 1, 3, 6, 12

Other Outcomes (1)

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

    baseline, month 1, 3, 6, 12

Study Arms (2)

tamoxifen

EXPERIMENTAL

tamoxifen 40 mg daily for one year

Drug: tamoxifen 40 mg daily for one year

placebo

PLACEBO COMPARATOR

placebo drugs

Drug: tamoxifen 40 mg daily for one year

Interventions

tamoxifen 40 mg daily for one yearDRUG

both arms with riluzole daily

Also known as: Nolvadex
placebotamoxifen

Eligibility Criteria

Age20 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Po-Chih Chen

New Taipei City, Taiwan

Location

Related Publications (1)

  • Trist BG, Fifita JA, Hogan A, Grima N, Smith B, Troakes C, Vance C, Shaw C, Al-Sarraj S, Blair IP, Double KL. Co-deposition of SOD1, TDP-43 and p62 proteinopathies in ALS: evidence for multifaceted pathways underlying neurodegeneration. Acta Neuropathol Commun. 2022 Aug 25;10(1):122. doi: 10.1186/s40478-022-01421-9.

    PMID: 36008843DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Tamoxifen

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesTDP-43 ProteinopathiesNeuromuscular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

StilbenesBenzylidene CompoundsBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsOrganic Chemicals

Study Officials

  • Chaur-Jong Hu, M.D.

    Shung Ho Hospital, Taipei Meidcal University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 9, 2014

First Posted

June 18, 2014

Study Start

April 1, 2014

Primary Completion

September 17, 2019

Study Completion

September 18, 2019

Last Updated

September 19, 2019

Record last verified: 2014-06

Locations

TW(1)