Phase 3 Muscular Dystrophy Trial, Recruiting NCT05004129 Sponsor: AMO Pharma Limited Condition: Muscular Dystrophy
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Phase 3 Muscular Dystrophy Trial, Recruiting

NCT05004129
Recruiting Phase 3 🇦🇺 Australian site

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

  • You have been diagnosed with Congenital or Childhood Onset Myotonic Dystrophy Type 1 (DM1), confirmed through genetic testing, and are between 6 and 45 years old — OR you are joining directly from completing a specific earlier related study called AMO-02-MD-2-003.
  • Your doctor has rated your condition as moderately severe or worse using a standard medical scale called the CGI-S (score of 3 or higher) — this applies if you are not coming directly from the earlier related study.
  • You (or your parent/legal guardian) are willing and able to give written consent to participate, and if you are a child, you are also willing to agree to take part yourself.
  • Your caregiver is willing and able to support your participation throughout the whole study.
  • You are willing and able to follow specific food and diet rules set out by the study.

Who may not be able to join:

  • Your body weight relative to your height (BMI) is below 13.5 or above 40 (confirm with trial site for exact calculation).
  • You have started a new medication or changed an existing one within the last 4 weeks before joining.
  • You have taken certain strong medications that affect how the body processes drugs (such as clarithromycin, ketoconazole, or ritonavir) within the last 4 weeks.
  • You are currently taking certain other medications that need very precise dosing to be safe, such as warfarin or digitoxin.
  • You are currently in another clinical trial testing an experimental drug, or you were in one within the last 6 months (unless it was the specific earlier related study).
  • You have existing or past serious health conditions affecting your heart, brain, kidneys, liver, stomach, hormones, or breathing that could affect the study results.
  • You have a known allergy to the study drug (tideglusib) or any of its ingredients, including a strawberry allergy.

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 27 July 2026
Phase 3: approximately ~65% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

Contact this trial

Principal Investigator: Harriet Gray-Stephens, BM BCh, MA (Oxon), MFPM, AMO Pharma

Australian sites

The Bright Alliance, Randwick, New South Wales
02 9382 5534

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Trial details

Status
Recruiting
Phase
Phase 3
Sponsor
AMO Pharma Limited
Registry
ClinicalTrials.gov
Start date
23 August 2021
Est. completion
31 December 2026

Where this trial is recruiting

🇦🇺 Australia 🇨🇦 Canada 🇳🇿 New Zealand 🇺🇸 United States

1 site(s) in Australia. Confirm current status and contact details directly with the trial site.

Primary endpoints

Safety (Adverse Events); Safety (Adverse Events) - With Optional Expanded Access; Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 27 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov