cTAP Publishes Comprehensive Analysis of the North Star Ambulatory Assessment (NSAA), a Newly Adopted Primary Outcome Measure in Clinical Trials of Patients with Duchenne Muscular Dystrophy

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.

About cTAP

The collaborative Trajectory Analysis Project (cTAP) was established in 2015 to solve critical problems in drug development in Duchenne muscular dystrophy with a sense of urgency for the thousands of patients who are waiting for treatments. cTAP’s dynamic global alliance of academic, industry, and patient stakeholders work collaboratively to identify and address priority questions crucial to the research community, regulators, and health authorities. Partnered with Analysis Group, cTAP’s advanced biostatistical analytics platform unlocks the power of individual patient data, turning complexity into unambiguous insights and tools with near-term, real-world application for more effective clinical trial design and analysis. With its large and continually growing multi-national database of natural history and clinical trial data sources, cTAP enables therapy developers to conduct smarter trials and bring new therapies to patients sooner. The cTAP approach offers potential application to any challenging disease state characterized by heterogeneous progression. To learn more about cTAP’s capabilities, visit http://ctap-duchenne.org.

About Analysis Group’s HEOR Practice

Founded in 1981, Analysis Group is one of the largest international economics consulting firms, with more than 1,000 professionals across 14 offices. Analysis Group’s health care experts apply analytical expertise to health economics and outcomes research, clinical research, market access and commercial strategy, and health care policy engagements, as well as drug safety-related engagements in epidemiology. Analysis Group’s internal experts, together with its network of affiliated experts from academia, industry, and government, provide our clients with exceptional breadth and depth of expertise and end-to-end consulting services globally.

Muscular Dystrophy UK (MDUK)

Muscular Dystrophy UK is the charity bringing individuals, families and professionals together to beat muscle-wasting conditions. The charity offers practical and emotional support for people with a muscle-wasting condition and their families at every stage. MDUK works to ensure everyone with a muscle-wasting condition has the right specialist NHS support and healthcare, whatever their age and wherever they live. It funds world-leading research and helps people with all muscle-wasting conditions to improve quality of life, make the most of their independence and ensure every day counts. For more information or to help support MDUK, visit www.musculardystrophyuk.org or call their free helpline during working hours on 0800 652 6352.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a uniformly fatal, progressive muscle-wasting disease affecting about one in 3,500-6,000 male live births. Patients with Duchenne lack the ability to make dystrophin, a protein crucial to muscle function. As their muscles deteriorate, they progressively lose the ability to walk, feed themselves, turn over in bed, and ultimately to breathe. While there is no cure, the past decade has seen an explosion in research resulting in more than 15 new therapies entering clinical development, with some receiving conditional approval. Learn more about Duchenne at http://cureduchenne.org, http://parentprojectmd.org and http://charleysfund.org.

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Contact information

cTAP
Susan J. Ward, +1 617-448-2617
susanjward@ctap-duchenne.org

Analysis Group
Eric Seymour, +1 617-425-8103
eric.seymour@analysisgroup.com