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Selective Estrogen Receptor Modulator

Tamoxifen Therapy for Myotubular Myopathy (TAM4MTM Trial)

Phase 1 & 2

Recruiting

Led By Jame J Dowling, MD, PhD

Research Sponsored by James Dowling

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

XLMTM resulting from a confirmed mutation in the Myotubularin 1 (MTM1) gene

Male

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to 15 months

Awards & highlights

TAM4MTM Trial Summary

This trial is testing if tamoxifen can improve movement and breathing in boys with XLMTM.

Who is the study for?

This trial is for males with a genetic muscle condition called XLMTM, confirmed by a specific gene mutation. Boys and men aged 2 years and older can join if they agree to follow the study rules. They must not have other diseases that could confuse results or be on certain medications like SSRIs, nor should they have had recent surgery or plan one soon.Check my eligibility

What is being tested?

The study tests Tamoxifen (ApoTamox 10mg) against a placebo to see if it improves muscle and breathing functions in patients with XLMTM. It's randomized, meaning participants are put into groups by chance, double-blinded so neither doctors nor patients know who gets what treatment until after the study ends.See study design

What are the potential side effects?

While not specified here, common side effects of Tamoxifen may include hot flashes, mood swings, blood clots, and vision problems. The exact side effects experienced in this trial will be monitored closely due to the unique patient population.

TAM4MTM Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have a confirmed MTM1 gene mutation causing my condition.

Select...

I am male.

TAM4MTM Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to 15 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to 15 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to 15 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

10 meter walk test

Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders for subjects aged 2-4 years (CHOP INTEND)

Motor Function Measure 32 (MFM32)

Secondary outcome measures

Change in pulmonary function testing scores 1) Forced Expiratory Volume in the first second

Change in pulmonary function testing scores 2) Forced Vital Capacity

Change in pulmonary function testing scores 3) Peak Cough Flow

+5 more

Side effects data

From 2013 Phase 1 trial • 89 Patients • NCT01393990

33%

Leukopenia

33%

Anaemia

33%

Lymphopenia

33%

Hypoxia

33%

Vomiting

33%

Chest discomfort

33%

Insomnia

33%

Neutropenia

33%

Nausea

33%

Chest pain

33%

Chills

33%

Decreased appetite

33%

Dizziness

33%

Cough

33%

Oropharyngeal pain

33%

Dry skin

33%

Pyrexia

33%

Stomatitis

33%

Palpitations

33%

Diarrhoea

33%

Dysphagia

33%

Tremor

33%

Respiratory tract congestion

33%

Bronchitis

33%

Fatigue

33%

Seasonal allergy

100%

80%

60%

40%

20%

Study treatment Arm

Part A: 160 mg LY2228820 Capsules

Part D: 200 mg LY2228820 Tablets

Part D: 300 mg LY2228820 Tablets

Part A: 160 mg LY2228820 Bridge

Part A: 200 mg LY2228820 Tablets

Part A: 120 mg LY2228820 Capsules

Part A: 300 mg LY2228820 Tablets

Part A: 90 mg LY2228820 Capsules

Part A: 420 mg LY2228820 Tablets

Part C: 300 mg LY2228820 Tablets

Part A: 200 mg LY2228820 Capsules

Part A: 65 mg LY2228820 Capsules

Part A: 10 mg LY2228820 Capsules

Part A: 20 mg LY2228820 Capsules

Part A: 160 mg LY2228820 Tablets

Part A: 40 mg LY2228820 Capsules

Part B: 420 mg LY2228820 Tablets

Part A: 560 mg LY2228820 Tablets

TAM4MTM Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Drug: ApoTamox 10mgExperimental Treatment1 Intervention

Drug: Tamoxifen (tamoxifen citrate); ApoTamox 10 mg tablets orally twice daily for 6 months

Group II: PlaceboPlacebo Group1 Intervention

Placebo (no active ingredients) tablets orally twice daily for 6 months

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Mogford Campbell Family Chair FundUNKNOWN

Myotubular TrustUNKNOWN

SparksUNKNOWN

Media Library

Tamoxifen (Selective Estrogen Receptor Modulator) Clinical Trial Eligibility Overview. Trial Name: NCT04915846 — Phase 1 & 2

Myotubular Myopathy Research Study Groups: Drug: ApoTamox 10mg, Placebo

Myotubular Myopathy Clinical Trial 2023: Tamoxifen Highlights & Side Effects. Trial Name: NCT04915846 — Phase 1 & 2

Tamoxifen (Selective Estrogen Receptor Modulator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04915846 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What therapeutic effects can be observed through ApoTamox 10mg?

"ApoTamox 10mg is typically utilized to treat cancerous tumors. It can also be prescribed for high risk patients and those needing ovulation induction therapy or ovarian cancer treatment."

Answered by AI

How many participants are partaking in this experiment?

"To commence the trial, 16 suitable participants are needed. These individuals may come from sites such as National Institutes of Health in Rockville, Maryland or Hospital for Sick Children in Toronto, Ontario so long as they meet all necessary inclusion criteria."

Answered by AI

What benefits are anticipated to result from this medical experiment?

"The purpose of this 15-month medical investigation is to measure the efficacy of CHOP INTEND on participants aged 2-4 years. The primary indicators are Mean change from baseline in subjects not requiring ventilator support, mean variation in time off ventilation for those dependent on it, and incidence/severity of adverse events related to the treatment as judged by CTCAE v4.0."

Answered by AI

Is registration still open for this experiment?

"Confirmed. According to the clinicaltrials.gov website, recruitment for this trial is still in progress. The study was initially made available on December 18th 2020 and it has since been updated most recently on June 17th 2021."

Answered by AI

Are there any additional analyses examining the effects of ApoTamox 10mg?

"Queen Mary University of London was the first to assess ApoTamox 10mg in 1994 and since then, a total 18441 trials have been concluded. Currently, 67 active studies are running across multiple locations with Rockville, Maryland being one of the major sites for research."

Answered by AI

Recent research and studies

Nature CommunicationsJournal

Tamoxifen Therapy in a Murine Model of Myotubular Myopathy

Maani, Nika, Nesrin Sabha, Kamran Rezai, Arun Ramani, Linda Groom, Nadine Eltayeb, Faranak Mavandadnejad, et al.. 2018. “Tamoxifen Therapy in a Murine Model of Myotubular Myopathy”. Nature Communications. Springer Science and Business Media LLC. doi:10.1038/s41467-018-07057-5.

NeurologyJournal

A Natural History Study of X-linked Myotubular Myopathy

Amburgey, Kimberly, Etsuko Tsuchiya, Sabine de Chastonay, Michael Glueck, Rachel Alverez, Cam-Tu Nguyen, Anne Rutkowski, Joseph Hornyak, Alan H. Beggs, and James J. Dowling. 2017. “A Natural History Study of X-linked Myotubular Myopathy”. Neurology. Ovid Technologies (Wolters Kluwer Health). doi:10.1212/wnl.0000000000004415.

Nature CommunicationsJournal

Tamoxifen Prolongs Survival and Alleviates Symptoms in Mice with Fatal X-linked Myotubular Myopathy

Gayi, Elinam, Laurence A. Neff, Xènia Massana Muñoz, Hesham M. Ismail, Marta Sierra, Thomas Mercier, Laurent A. Décosterd, et al.. 2018. “Tamoxifen Prolongs Survival and Alleviates Symptoms in Mice with Fatal X-linked Myotubular Myopathy”. Nature Communications. Springer Science and Business Media LLC. doi:10.1038/s41467-018-07058-4.

clinicaltrials.govStudy