Amyotrophic Lateral SclerosisPhase 1/2NCT02166944
Tamoxifen Treatment in Patients With Motor Neuron Disease
A Phase 1/2 study testing tamoxifen 40 mg daily for one year in people with Amyotrophic Lateral Sclerosis and ALS Functional Ration Scale, run by Taipei Medical University Shuang Ho Hospital at 1 site in Taiwan.
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. Read more about this study
- Who is it for?
- Ages 20 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, ALS Functional Ration Scale, TAR-DNA-binding Protein-43
- What is being tested?
- tamoxifen 40 mg daily for one year
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteTaiwan
- Study dates
- Apr 2014to 18 Sep 2019
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: The Study of 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.
Read the 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.
- Conditions
- Amyotrophic Lateral Sclerosis, ALS Functional Ration Scale, TAR-DNA-binding Protein-43, Tamoxifen, mTOR
- Being tested
- tamoxifen 40 mg daily for one year
- Keywords
- amyotrophic lateral sclerosis, ALS functional ration scale, TAR-DNA-binding protein-43, tamoxifen, mTOR
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
tamoxifen 40 mg daily for one yearDrug
Also called Nolvadex
both arms with riluzole daily
| Study group | Role | What they receive |
|---|---|---|
| tamoxifen | Experimental | tamoxifen 40 mg daily for one year Receives: Drug: tamoxifen 40 mg daily for one year |
| placebo | Placebo comparator | placebo drugs Receives: Drug: tamoxifen 40 mg daily for one year |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Baseline, month 1, 3, 6, 12
Secondary outcomes (1)
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)
Measured: baseline, month 1, 3, 6, 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jun 2014 (estimated)
- Study startApr 2014
- Primary completion17 Sep 2019
- Study completion18 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Taipei Medical University Shuang Ho Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Chaur-Jong Hu, M.D., Principal investigator, Shung Ho Hospital, Taipei Meidcal University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02166944
- Sponsor's protocol ID
- 201307022
- Status last verified
- Jun 2014
- First submitted
- 9 Jun 2014
- Last updated
- 19 Sep 2019
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 36008843
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Study of Tamoxifen Treatment in Patients With Motor Neuron Disease
- ClinicalTrials.gov ID
- NCT02166944
- Acronym / sponsor ID
- 201307022
- Conditions
- Amyotrophic Lateral Sclerosis, ALS Functional Ration Scale, TAR-DNA-binding Protein-43, Tamoxifen, mTOR
- Keywords
- amyotrophic lateral sclerosis, ALS functional ration scale, TAR-DNA-binding protein-43, tamoxifen, mTOR
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Taipei Medical University Shuang Ho HospitalUniversity, hospital or other
- Study start
- Apr 2014 (actual)
- Primary completion
- 17 Sep 2019 (actual)
- Study completion
- 18 Sep 2019 (actual)
- First posted
- 18 Jun 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| tamoxifen | Experimental | Drug: tamoxifen 40 mg daily for one yeartamoxifen 40 mg daily for one year |
| placebo | Placebo comparator | Drug: tamoxifen 40 mg daily for one yearplacebo drugs |
Who may qualify
- Ages
- 20 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| tamoxifen 40 mg daily for one yearAlso: Nolvadexboth arms with riluzole daily | Drug | placebo, tamoxifen |
Primary outcome
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
1 secondary outcome
Change from Baseline in pulmonary function test at 1, 3, 6,12 months
Time frame: baseline, month 1, 3, 6, 12
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2019.
- Derived from this study: PubMed 36008843Trist 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Chaur-Jong Hu, M.D. principal investigator
Shung Ho Hospital, Taipei Meidcal University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing tamoxifen 40 mg daily for one year in people with Amyotrophic Lateral Sclerosis and ALS Functional Ration Scale, run by Taipei Medical University Shuang Ho Hospital at 1 site in Taiwan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Taiwan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02166944, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2019.