Recruiting

Givinostat

Sponsor:

ITF Therapeutics LLC

Code:

NCT07127978

Conditions

Duchene Muscular Dystrophy

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

Study Details

Brief summary:

This is a prospective observational study conducted to evaluate safety, tolerability, and functional outcomes of patients with DMD newly initiating oral givinostat or having started therapy within 6 months as part of routine clinical care in the US. The study has a planned maximum duration of 5 years for the first enrolled patients, including a 24-month enrollment period and a minimum of 2 years of follow-up.

Conditions

Duchene Muscular Dystrophy

Study ID

NCT07127978

Start date

Oct 23, 2025

Status verified date

Apr, 2026

Completion date

Jul 30, 2030

Anticipated

Primary completion date

Jun 30, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

300 participants

Anticipated

Interventions and Outcome Measures

Arms

Ambulatory

Ambulatory is defined as being able to complete the 10-meter Walk-Run (10mWR) test within 30 seconds without an assistive device.

This cohort will include up to 120 patients who are ambulatory prior to starting givinostat treatment.

Within this cohort , 95 patients must have had prior use or a referral for concomitant use of dystrophin-enhancing therapy (DET), such as exon skipping oligonucleotides or gene therapy.

Enrollment is capped at 25 patients without prior or referred DET use.

Non-Ambulatory

Non-ambulatory status is determined by inability to perform the 10mWR test as defined above.

This cohort will include approximately 180 patients who are non-ambulatory prior to starting givinostat.

Primary outcome measure

  • Characterize the incidence of thrombocytopenia / decreased platelet counts in DMD patients treated with oral givinostat [ Time Frame: From follow up (after first date of givinostat treatment) through end of study (up to 5 years) ]
  • Characterize the frequency of thrombocytopenia / decreased platelet counts in DMD patients treated with oral givinostat [ Time Frame: [Time Frame: From follow up (after first date of givinostat treatment) through end of study (up to 5 years)] ]
  • Characterize severity of serious events thrombocytopenia / decreased platelet counts in DMD patients treated with oral givinostat [ Time Frame: From follow up (after first date of givinostat treatment) through end of study (up to 5 years) ]
  • Characterize the incidence of serious events of bleeding in DMD patients treated with oral givinostat [ Time Frame: From follow up (after first date of givinostat treatment) through end of study (up to 5 years) ]
  • Characterize the frequency of serious events of bleeding in DMD patients treated with oral givinostat [ Time Frame: From follow up (after first date of givinostat treatment) through end of study (up to 5 years) ]
  • Characterize the severity of serious events of bleeding in DMD patients treated with oral givinostat [ Time Frame: From follow up (after first date of givinostat treatment) through end of study (up to 5 years) ]

Central Contacts and Locations

Locations

Arkansas Children's Research Institute

Recruiting

Little Rock, Arkansas, United States, 72202

Contacts

Domencia Hunter

973-932-9211hunterdj@archildrens.org

Principal Investigator:

Aravindhan Veerapandiyan

Children's National Hospital

Recruiting

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

Contacts

Margaret Martinez

202-476-2431mbmartin@childrensnational.org

Principal Investigator:

Sarah Wright, DO

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

Recruiting

Chicago, Illinois, United States, 60611

Contacts

Raven Hill

312-227-2937rhill@luriechildrens.org

Principal Investigator:

Bridget McGowan

Boston Children's Hospital

Recruiting

Boston, Massachusetts, United States, 02115

Contacts

Gerald Mastellone

857-218-4677Gerald.mastellone@childrens.harvard.edu

Principal Investigator:

Partha Ghosh, MD

University of Massachusetts Chan Medical School

Recruiting

Worcester, Massachusetts, United States, 01655

Contacts

Sarah Figueira

(508) 856-1604sarah.figueira@umassmed.edu

dmdprogram@umassmemorial.org

Principal Investigator:

Stephen Chrzanowski

Columbia University Medical Center

Recruiting

New York, New York, United States, 10032

Contacts

Kimberly Berry

212-342-3038kb2996@cumc.columbia.edu

Principal Investigator:

Divya Jayaraman

Duke University / Lenox Baker Children's Hospital

Recruiting

Durham, North Carolina, United States, 27705

Contacts

Brittany Nave

919-684-8087brittany.nave@duke.edu

Principal Investigator:

Natalie Katz, MD

Oregon Health and Science University

Recruiting

Portland, Oregon, United States, 97239

Contacts

Nadee Dayananda

503-418-0949dayanand@ohsu.edu

Principal Investigator:

Erika Finanger, MD

Seattle Children's Hospital

Recruiting

Seattle, Washington, United States, 98105

Contacts

Macarena Racciatti

206-995-6847macarena.racciatti@seattlechildrens.org

Principal Investigator:

Alicia Henriquez

More Information

Sponsor

ITF Therapeutics LLC

Last update posted

Jul 2, 2026

Last verified

Apr, 2026

Keywords

  • DMD
  • DUVYZAT
  • U.S study
  • duchenne muscular dystrophy
  • givinostat
  • non-interventional study
  • pediatric neuromuscular disease
  • rare disease
  • quality of life
  • effectiveness
  • post-marketing requirement
  • safety
  • motor function
  • muscular dystrophy
  • standard of care
  • real-world evidence
  • observational study
  • post-marketing study

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by ITF Therapeutics LLC on 2026-07-02.