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Introduction <p>Duchenne muscular dystrophy (DMD) is a rare, X-linked neuromuscular disease. The first approved DMD gene therapy, delandistrogene moxeparvovec, was evaluated in the phase&#xa0;3, two-part, randomized, placebo-controlled EMBARK trial (NCT05096221), which did not meet statistical significance for its primary endpoint assessed at the end of Part 1 (Week 52). To gather patient experience data possibly not captured by traditional clinical outcome assessments, a qualitative in-trial interview substudy was conducted among caregivers (parents) (≥ 18&#xa0;years) of male patients (≥ 4 to &lt; 8&#xa0;years with DMD) who completed Part&#xa0;1 (Week&#xa0;52) of EMBARK.</p> Methods <p>Semi-structured interviews, conducted with 23 parents within 14&#xa0;days after the last EMBARK Part&#xa0;1 visit, assessed DMD-related symptoms and impacts prior to EMBARK, perceptions of meaningful outcomes based on hypothetical examples using DMD-specific Caregiver Global Impression measures for Severity (CaGI-S) and Change (CaGI-C), and observed changes during the trial. Initial analyses were blinded to treatment assignment; follow-up analyses examining changes in physical activities were unblinded.</p> Results <p>Debriefing questions demonstrated parents’ understanding and correct&#xa0;interpretation of items and response options. Most parents indicated that “no change” or a 1-point improvement on CaGI-S items and “no change” or “minimal” improvement on CaGI-C items would be meaningful. Most (<i>n</i> = 17/23) reported improvements in ≥ 1 CaGI-C item&#xa0;1&#xa0;year after treatment; for patients receiving delandistrogene moxeparvovec, most parents (<i>n</i> = 7/10) rated ≥ 1 item as “very much” or “much” improved compared with placebo (<i>n</i> = 5/13). These differences were based on descriptive reporting and were not formally tested for statistical significance. Parents identified improvements in physical abilities (i.e.,&#xa0;running, climbing stairs, jumping) as the “most important” types of improvement.</p> Conclusion <p>These findings underscore the importance of maintaining stability or achieving minimal improvements in treatment outcomes for patients with DMD. Parental experiences and perceptions provided additional insight beyond clinical outcome assessment measures, offering a complementary subjective perspective on treatment impact.</p> Trial registration <p>ClinicalTrials.gov identifier, NCT05096221.</p>

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Caregiver-reported Patient Experiences with Duchenne Muscular Dystrophy: Qualitative In-trial Interviews 1 Year After Delandistrogene Moxeparvovec in the Pivotal EMBARK Trial

  • 期刊论文

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  • 发表日期：

    2025年11月3日

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  • Ivana Audhya,
  • Alise B. Nacson,
  • Katherine Gooch,
  • Bonita Basnyat,
  • Christina Slota,
  • Susan Martin,
  • Alex Murphy,
  • Claire J. Lansdall,
  • Teofil Ciobanu,
  • Andres Nascimento,
  • Aravindhan Veerapandiyan

摘要

Introduction

Duchenne muscular dystrophy (DMD) is a rare, X-linked neuromuscular disease. The first approved DMD gene therapy, delandistrogene moxeparvovec, was evaluated in the phase 3, two-part, randomized, placebo-controlled EMBARK trial (NCT05096221), which did not meet statistical significance for its primary endpoint assessed at the end of Part 1 (Week 52). To gather patient experience data possibly not captured by traditional clinical outcome assessments, a qualitative in-trial interview substudy was conducted among caregivers (parents) (≥ 18 years) of male patients (≥ 4 to < 8 years with DMD) who completed Part 1 (Week 52) of EMBARK.

Methods

Semi-structured interviews, conducted with 23 parents within 14 days after the last EMBARK Part 1 visit, assessed DMD-related symptoms and impacts prior to EMBARK, perceptions of meaningful outcomes based on hypothetical examples using DMD-specific Caregiver Global Impression measures for Severity (CaGI-S) and Change (CaGI-C), and observed changes during the trial. Initial analyses were blinded to treatment assignment; follow-up analyses examining changes in physical activities were unblinded.

Results

Debriefing questions demonstrated parents’ understanding and correct interpretation of items and response options. Most parents indicated that “no change” or a 1-point improvement on CaGI-S items and “no change” or “minimal” improvement on CaGI-C items would be meaningful. Most (n = 17/23) reported improvements in ≥ 1 CaGI-C item 1 year after treatment; for patients receiving delandistrogene moxeparvovec, most parents (n = 7/10) rated ≥ 1 item as “very much” or “much” improved compared with placebo (n = 5/13). These differences were based on descriptive reporting and were not formally tested for statistical significance. Parents identified improvements in physical abilities (i.e., running, climbing stairs, jumping) as the “most important” types of improvement.

Conclusion

These findings underscore the importance of maintaining stability or achieving minimal improvements in treatment outcomes for patients with DMD. Parental experiences and perceptions provided additional insight beyond clinical outcome assessment measures, offering a complementary subjective perspective on treatment impact.

Trial registration

ClinicalTrials.gov identifier, NCT05096221.