The Collabora+ve Trajectory Analysis Project (cTAP)
Explaining varia,on in Duchenne disease progression to accelerate drug development
Years of age
Meters walked
Individual trajectories of six-‐minute walk distance
Data from Imaging DMD
Years of age
Meters walked
Individual trajectories of six-‐minute walk distance
Data from Imaging DMD
Years of age
Meters walked
Individual trajectories of six-‐minute walk distance
Data from Imaging DMD
Two kinds of phenotypic varia+on – and why we care
• Larger, longer trials • Greater risk of not proving
efficacy
• Disconnect between individual experiences and research on average outcomes
• Smaller, shorter trials
• Greater confidence in trial design
• Pathway for conAnually improving individualized care
Unexplained varia+on (we don’t know what causes it)
Explained varia+on (we do know what causes it)
“Ideal” number of boys in a hypothe+cal efficacy trial
Unexplained variaAon in six minute walk distance (meters)
350
200
89 24 6
0
100
200
300
400
100 75 50 25 10
Unexplained varia+on (we don’t know what causes it)
Explained varia+on (we do know what causes it)
How can we explain more varia+on?
Analyze natural history data
Discover biomarkers
Learn from trials / registries
cTAP
How can we explain more varia+on?
Analyze natural history data
Discover biomarkers
Learn from trials / registries
The Collabora+ve Trajectory Analysis Project (cTAP)
Mission
• Explain phenotypic variaAon in DMD
• Translate into tools for trial design/analysis
• Make insights available to everyone in drug development
• Deliver near-‐term impact
The benefit: accelerate effec,ve treatments to pa,ents
cTAP at year 1 who we are and what we’ve done
Clinical experts and registries
• Mercuri; Telethon -‐ 96 boys • Goemans; UZ Leuven -‐ 65 boys • Muntoni; Northstar UK -‐ > 500 boys … Wong (CincinnaA) … Vandenborne (Imaging DMD)
Drug developers • BioMarin (Prosensa) • Pfizer • PTC TherapeuAcs • Sarepta • Shire … Others in process
Pa+ent advocates • Cure Duchenne • PPMD • Parents
Analy+cs, collabora+on leadership
• Analysis Group • Susan Ward
Collabora+on: central to cTAP success
Drug Developers
Pa+ent advocates
Analy+cs & collabora+on leadership
Clinical experts
Shared funding The right ques6ons
Clinical insight Pa6ent data
Seed, engage, establish, connect,
promote, encourage, advise Data protec6on Analy6cal exper6se Dedicated execu6on team
The Collabora+ve Trajectory Analysis Project (cTAP)
Mission
• Explain phenotypic variaAon in DMD
• Translate into tools for trial design/analysis
• Make insights available to everyone in drug development
• Deliver near-‐term impact
The benefit: accelerate effec,ve treatments to pa,ents
