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Tamoxifen Therapy for Myotubular Myopathy

NCT04915846 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2024-09-19

No results posted yet for this study

Summary

This is a phase 1 / 2, randomized, double-blinded, single cross-over study, with a washout period between treatment regimens, to test the efficacy and safety of tamoxifen therapy to improve motor and respiratory function in males with XLMTM.

Conditions

  • X Linked Myotubular Myopathy

Interventions

DRUG

ApoTamox 10mg

All participants will receive tamoxifen (ApoTamox) for approximately 6 months (6 months + 1 week). Participants and study staff will be blinded as to whether the participants are starting with the placebo or the drug. Depending on randomization, drug or placebo will be dispensed at the end of the t=0 study visit (Phase 1). Dosing will commence the day after the t=0 study visit. At the end of Phase 1, participants will enter a 'washout' period, when they will cease treatment. After approximately 3 months of washout, participants will cross-over to the other treatment regimen and receive the other interventional product (IP) for the final 6 months of their study participation (Phase 2).

DRUG

Placebo

placebo comparator

Sponsors & Collaborators

  • Canadian Institutes of Health Research (CIHR)

    collaborator OTHER_GOV

  • Cures Within Reach

    collaborator OTHER

  • The Joshua Frase Foundation USA

    collaborator UNKNOWN

  • Will Cure USA

    collaborator UNKNOWN

  • Mogford Campbell Family Chair Fund

    collaborator UNKNOWN

  • Myotubular Trust

    collaborator UNKNOWN

  • Great Ormond Street Hospital Charity

    collaborator UNKNOWN

  • Sparks

    collaborator UNKNOWN

  • James Dowling

    lead OTHER

Principal Investigators

  • Jame J Dowling, MD, PhD · The Hospital for Sick Children

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
6 Months
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-12-18
Primary Completion
2024-05-09
Completion
2024-05-09
FDA Drug
Yes

Countries

  • United States
  • Canada
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04915846 on ClinicalTrials.gov