cTAP’s insights to assist the design and analysis of clinical trials in patients with DMD

cTAP has published a comprehensive analysis of the North Star Ambulatory Assessment (NSAA), a newly adopted primary outcome measure in clinical trials of patients with Duchenne Muscular Dystrophy.

CAMBRIDGE, Mass.–(BUSINESS WIRE)–#Duchenne–The collaborative Trajectory Analysis Project (cTAP), a multi-stakeholder, global coalition in Duchenne muscular dystrophy (DMD), publishes research in the journal PLOS ONE providing quantitative insights to assist the design and analysis of clinical trials in patients with DMD.

The NSAA outcome measures the ability of patients to conduct a variety of activities – such as walking, running, jumping, climbing stairs, hopping, and rising from the floor – that better reflect the experience of patients in everyday life than clinical outcomes used in ongoing and recently completed clinical trials to date. Using the total NSAA score, the publication characterizes the heterogeneity of rate of disease progression in DMD, identifying multiple different groups of boys that share a similar rate of disease progression, and provides a quantitative risk assessment of the likelihood that a patient will gain, remain stable, or lose function on each NSAA item over time.

Professor Francesco Muntoni, Director of the Dubowitz Neuromuscular Centre, University College London, expressed his enthusiasm for the study. “The comprehensive assessment of the NSAA in this study will be invaluable for DMD clinical trial design and interpretation of findings,” said Professor Muntoni. “Although the NSAA is being used as a primary clinical endpoint, disease progression using this endpoint has not previously been fully characterized for use in clinical trials.”

The study involved the analysis of clinical data from 395 patients with DMD curated by the UK’s North Star Network (NSUK), made possible through the sponsorship of Muscular Dystrophy UK (MDUK), a leading UK charity that brings together people affected by more than 60 rare and very rare progressive muscle-weakening and wasting conditions. Professor Muntoni also noted, “MDUK has been forward-thinking in supporting the curation of patient data in Duchenne muscular dystrophy in the last decade, without which these analyses would not have been possible.”

Statistical analyses were conducted by a data science team lead by James Signorovitch, Managing Partner with cTAP’s coalition partner, Analysis Group.

Dr. Stuart Peltz, Chief Executive Officer, PTC Therapeutics, a leading developer of therapeutics for patients with Duchenne and a founding member of cTAP, highlighted the importance of learning from real world patient data. “The NSAA is an important measure of the real-world impact of such a heterogenous progressive neuromuscular disorder. The collaboration of academia, industry, and patient advocacy is an excellent example of stakeholders pulling together to accelerate insights that have advanced the development of treatments for patients with Duchenne.”

The publication, “Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy, is available online at the PLOS ONE website: https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0221097. This publication, together with further publications from cTAP collaborators, can also be found on cTAP’s newly expanded website http://ctap-duchenne.org.

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