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New drug under Real-World watch for duchenne patients

NCT ID NCT07127978

Disease control Sponsor: ITF Therapeutics LLC Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 02, 2026 · Updated 1 time

Summary

This study follows about 300 people with Duchenne muscular dystrophy who are taking the drug givinostat as part of their normal care. Researchers will track safety issues like low platelet counts and bleeding, and see how patients' muscle function changes over up to 5 years. The goal is to understand how well the drug works outside of a controlled trial setting.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 300 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2025

Expected to finish

Jul 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

This study will be conducted in the US and plans to enroll 300 patients diagnosed with DMD and treated with givinostat as part of their routine clinical care. Patients will be recruited from expert centers treating DMD patients with specialists in neuromuscular conditions who are routinely involved in the care and treatment of patients with DMD, some of whom would have already gained experience with givinostat.

Ages

6 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Patients of any gender at least 6 years of age, diagnosed with DMD (according to genetic test) and have received a prescription for oral givinostat in accordance with the USPI. * Patient has provided informed consent (and assent when applicable) for participation in the study. * Patient's index date (first date of givinostat treatment) is no more than 6 months prior to signing of informed consent. * Patient has the required data available (DMD diagnosis, givinostat administration \[dose and schedule\], laboratory results \[hematology and triglycerides\]) for addressing the study objectives for the period between index date and study entry (for patients enrolled after the index date). Exclusion Criteria: * Patient previously received givinostat (commercial or investigational product) and permanently discontinued treatment or patient started commercial givinostat for \> 6 months before signing of informed consent.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy muscular dystrophy Rare Diseases

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Duchene muscular dystrophy

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    9 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Ann and Robert H. Lurie Children's Hospital of Chicago

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Arkansas Children's Research Institute

    RECRUITING

    Little Rock, Arkansas, 72202, United States

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • Children's National Hospital

    RECRUITING

    Washington D.C., District of Columbia, 20010, United States

  • Columbia University Medical Center

    RECRUITING

    New York, New York, 10032, United States

  • Duke University / Lenox Baker Children's Hospital

    RECRUITING

    Durham, North Carolina, 27705, United States

  • Oregon Health and Science University

    RECRUITING

    Portland, Oregon, 97239, United States

  • Seattle Children's Hospital

    RECRUITING

    Seattle, Washington, 98105, United States

  • University of Massachusetts Chan Medical School

    RECRUITING

    Worcester, Massachusetts, 01655, United States

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