Rare Disease Trial, Recruiting (NCT07127978)

Rare Disease Trial, Recruiting NCT07127978 Sponsor: ITF Therapeutics LLC Condition: Rare Disease

Printed from Voxsanity (voxsanity.com.au) — NCT07127978 — Data sourced from ClinicalTrials.gov. Verify directly with the trial site before attending. Not medical advice.

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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 are at least 6 years old, of any gender, and have been diagnosed with Duchenne Muscular Dystrophy (DMD) confirmed by a genetic test, and have been prescribed oral givinostat by your doctor.
You (and/or your parent or guardian, if applicable) have agreed to take part in the study by signing a consent form.
Your first dose of givinostat was taken no more than 6 months before signing the consent form to join this study.
Your medical records from the time you started givinostat are available, including your DMD diagnosis, details about your givinostat doses, and relevant blood test results (blood counts and triglyceride levels).

Who may not be able to join:

You have taken givinostat before (whether as part of another study or as a commercial prescription) and permanently stopped taking it, OR you started taking the commercial version of givinostat more than 6 months before signing the consent form for this study.

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.

GP referral letter

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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: NCT07127978

Sponsor: ITF Therapeutics LLC

Phase: Not Applicable

Status: Recruiting

Condition: Rare Disease

More information: https://clinicaltrials.gov/study/NCT07127978

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:

You are at least 6 years old, of any gender, and have been diagnosed with Duchenne Muscular Dystrophy (DMD) confirmed by a genetic test, and have been prescribed oral givinostat by your doctor.
You (and/or your parent or guardian, if applicable) have agreed to take part in the study by signing a consent form.
Your first dose of givinostat was taken no more than 6 months before signing the consent form to join this study.
Your medical records from the time you started givinostat are available, including your DMD diagnosis, details about your givinostat doses, and relevant blood test results (blood counts and triglyceride levels).

Who may not be able to join:

You have taken givinostat before (whether as part of another study or as a commercial prescription) and permanently stopped taking it, OR you started taking the commercial version of givinostat more than 6 months before signing the consent form for this study.

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

ITF Therapeutics LLC

Registry

ClinicalTrials.gov

Start date

23 October 2025

Est. completion

30 June 2030

Where this trial is recruiting

🇺🇸 United States

Primary endpoints

Characterize the incidence of thrombocytopenia / decreased platelet counts in DMD patients treated with oral givinostat; Characterize the frequency of thrombocytopenia / decreased platelet counts in DMD patients treated with oral givinostat; Characterize severity of serious events thrombocytopenia / decreased platelet counts in DMD patients treated with oral givinostat; Characterize the incidence of serious events of bleeding in DMD patients treated with oral givinostat; Characterize the frequency of serious events of bleeding in DMD patients treated with oral givinostat; Characterize the seve...

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