Design of an observational study to assess real-world outcomes of patients with Duchenne muscular dystrophy in the US treated with givinostat

Topic:

Clinical Trials

Poster Number: P84

Author(s):

Erika L Finanger, MD, MS, Oregon Health and Science University, Emma Ciafaloni, MD, University of Rochester, Tina Duong, PT, Ph.D, Department of Neurology and Neurological Sciences, Stanford University, Crystal Proud, MD, Children’s Hospital of the King’s Daughters, Carolina Tesi Rocha, MD, Stanford University, Aravindhan Veerapandiyan, MD, Arkansas Children’s Hospital, University of Arkansas for Medical Sciences, Little Rock, Arkansas, US, Mary Jane Salinas, PharmD, ITF Therapeutics, Sara Cazzaniga, Msc, Italfarmaco SpA., Katiana Gruppioni, MPH, ITF Therapeutics, Tom Crawford, MD, John Hopkins Medicine

Background: Givinostat, an oral histone deacetylase inhibitor, was approved in the US in March 2024 for the treatment of Duchenne muscular dystrophy (DMD) in boys aged ≥6 years.

Objectives: This US-based, prospective, open-label observational study aims to evaluate the real-world safety and effectiveness of givinostat in patients with DMD.

Methods: The study plans to enroll up to 300 patients with DMD (ambulatory and non-ambulatory) after receiving clinically prescribed givinostat; patients will be indexed on their first date of treatment and followed for a minimum of 2 years or until treatment discontinuation. Relevant medical history prior to the index date will be collected retrospectively. Data will be collected through routine clinical care visits (at baseline and approximately every 6 months), with assessments performed according to standard procedures and data captured from medical records. The primary outcomes will assess adverse events including frequency, incidence, and severity of thrombocytopenia and serious bleeding events. Secondary endpoints will assess gastrointestinal issues, hypertriglyceridemia and dehydration, and all serious adverse events. Important exploratory endpoints will evaluate the real-world effectiveness of givinostat using motor function assessments and patient-reported outcomes. For patients who are ambulatory, motor function assessments will include the North Star Ambulatory Assessment, a 10-meter walk/run test, and the time to rise. For patients who are non-ambulatory, motor function will be assessed using the Performance of Upper Limb score, and pulmonary function tests will also be conducted. Quality of life will be assessed for both patients who are ambulatory and non-ambulatory. Analyses will be performed across all cohorts, with subgroup stratifications including ambulatory status, age, and time from diagnosis.

Conclusions: This study will help clarify the role of givinostat in the broader DMD treatment landscape and its potential long-term effects, particularly for different subgroups of patients not included in prior studies. By employing a novel model based on routine visits at supported clinical centers, this study seeks to enhance real-world data collection and the understanding of the safety and effectiveness of givinostat beyond standard registry follow-up.