A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

NCT07127978 | Recruiting

• 3 other identifiers: ITF/2357/62126598217865
• Study Type: observational
• Target: 300
• Locations: 1 country
• Sites: 7

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

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

Outcome Measures

Primary Outcomes (6)

• Characterize the incidence of thrombocytopenia / decreased platelet counts in DMD patients treated with oral givinostat

  Incidence rate of thrombocytopenia (per 100 patient years of observation time) post index date

  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

  Frequency and incidence proportion (overall and by maximum severity) of thrombocytopenia

  [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 to first thrombocytopenia event from index date

  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

  Incidence rate of serious bleeding events (per 100-patient years of observation time) post index date

  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

  Frequency and incidence proportion (overall and by maximum severity) of serious bleeding events

  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 to first serious bleeding event from index date

  From follow up (after first date of givinostat treatment) through end of study (up to 5 years)

Secondary Outcomes (10)

• Characterize the incidence of other adverse events of special interest (AESIs) in DMD patients treated with oral givinostat

  From follow up (after first date of givinostat treatment) through end of study (up to 5 years)

• Characterize the frequency other adverse events of special interest (AESIs) in DMD patients treated with oral givinostat

  From follow up (after first date of givinostat treatment) through end of study (up to 5 years)

• Characterize the severity of other adverse events of special interest (AESIs) in DMD patients treated with oral givinostat

  From follow up (after first date of givinostat treatment) through end of study (up to 5 years)

• Characterize the incidence of all serious adverse events (SAEs) in DMD patients treated with oral givinostat

  From follow up (after first date of givinostat treatment) through end of study (up to 5 years)

• Characterize the frequency of all serious adverse events (SAEs) in DMD patients treated with oral givinostat

  From follow up (after first date of givinostat treatment) through end of study (up to 5 years)

Study Arms (2)

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.

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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.

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• ITF Therapeutics LLC: lead

Study Sites (7)

Arkansas Children's Research Institute

Little Rock, Arkansas, 72202, United States

RECRUITING

Children's National Hospital

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

RECRUITING

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

Chicago, Illinois, 60611, United States

RECRUITING

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

University of Massachusetts Chan Medical School

Worcester, Massachusetts, 01655, United States

RECRUITING

Columbia University Medical Center

New York, New York, 10032, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98105, United States

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Rare Diseases; Muscular Dystrophies

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 10, 2025
• First Posted: August 17, 2025
• Study Start: October 23, 2025
• Primary Completion (Estimated): June 30, 2030
• Study Completion (Estimated): July 30, 2030
• Last Updated: April 15, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (7)