Muscular Dystrophy Trial, Recruiting (NCT06564974)

Muscular Dystrophy Trial, Recruiting NCT06564974 Sponsor: Catalyst Pharmaceuticals, Inc. Condition: Muscular Dystrophy

Printed from Voxsanity (voxsanity.com.au) — NCT06564974 — Data sourced from ClinicalTrials.gov. Verify directly with the trial site before attending. Not medical advice.

Back to Muscular Dystrophy

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:

The patient (or their parent/legal guardian) is willing and able to sign a consent form after being told what the registry involves, before anything registry-related begins
The patient and/or their parent or guardian is willing and able to fill out quality-of-life questionnaires
The patient is male and is at least 2 years old
The patient has a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD), proven through genetic testing or a muscle biopsy
The patient currently has an active prescription for, or is already taking, AGAMREE® (vamorolone)

Who may not be able to join:

The patient has a medical reason why they should not take AGAMREE®, or has a health condition that, in the doctor's opinion, would interfere with their participation in or the results of the registry (confirm with trial site)

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 28 July 2026

This study is not part of the standard drug-approval phase pathway (for example an observational, device, behavioural, or registry study), so a phase success rate does not apply.

Contact this trial

Principal Investigator: William Andrews, MD, Catalyst Pharmaceuticals

Phone: (305) 420-3200

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.

Voxsanity

Clinical trials and treatments, explained plainly · voxsanity.com.au

Date:

To: My Doctor

Re: Clinical trial enquiry

I am writing to enquire about the following clinical trial which may be relevant to my care:

Trial: NCT06564974

Sponsor: Catalyst Pharmaceuticals, Inc.

Phase: Not Applicable

Status: Recruiting

Condition: Muscular Dystrophy

Trial contact: (305) 420-3200

More information: https://clinicaltrials.gov/study/NCT06564974

Eligibility summary (AI generated)

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

The patient (or their parent/legal guardian) is willing and able to sign a consent form after being told what the registry involves, before anything registry-related begins
The patient and/or their parent or guardian is willing and able to fill out quality-of-life questionnaires
The patient is male and is at least 2 years old
The patient has a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD), proven through genetic testing or a muscle biopsy
The patient currently has an active prescription for, or is already taking, AGAMREE® (vamorolone)

Who may not be able to join:

The patient has a medical reason why they should not take AGAMREE®, or has a health condition that, in the doctor's opinion, would interfere with their participation in or the results of the registry (confirm with trial site)

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

Access in Australia: A treatment being studied in a clinical trial is generally not yet listed on the PBS. Where a medicine is not approved or not available through a trial, the TGA Special Access Scheme may allow a doctor to access unapproved therapeutic goods for individual patients. See voxsanity.com.au/sas-navigator/ for a plain English explainer.

This information was sourced from ClinicalTrials.gov via Voxsanity (data last synced 28 July 2026). It is not medical advice. Please verify the trial's current status directly with the trial site before acting.

Trial details

Status

Recruiting

Phase

Not Applicable

Sponsor

Catalyst Pharmaceuticals, Inc.

Registry

ClinicalTrials.gov

Start date

25 September 2024

Est. completion

1 February 2032

Where this trial is recruiting

Puerto Rico 🇺🇸 United States

Primary endpoints

Change in Height z-score; Change in BMI z-score; Change in North Star Ambulatory Assessment from baseline; Change in Performance of Upper Limb from baseline; Tanner stage documentation; Percentage of patients with cataract and/or glaucoma; Findings from lateral spine x-ray; Findings from anterior-posterior/posterior-anterior X-ray; Findings from hand-wrist X-ray; Summary statistics of bone mineral density measures assessed by dual x-ray absorptiometry; Body Composition Mass Measures; Summary statistics of body fat percentage and regional fat distribution ratios assessed by DXA; Findings from s...

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: 28 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