Transcript FOR-DMD Steroid Trial

FOR-DMD:
Recruitment and Retention Challenges
Addressing the Challenges
1. Benefits vs risks of corticosteroids?
2. Why invest so much to study a treatment of established
(short term) benefit?
3. Corticosteroids vs curative new treatment?
4. Does FOR-DMD preclude participation in another study?
5. Can subjects leave FOR-DMD to enter another study?
Benefits vs risks of corticosteroids?
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No longer equipoise concerning
corticosteroids vs placebo (study sections
X3; FOR-DMD investigators; CINRG
Network; Cochrane, AAN reviews)
Long-term data, albeit uncontrolled
New trials are “permitting” corticosteroids
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Why invest so much to study a treatment
of established (short term) benefit?
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No standard treatment (31 regimens)
No standards of side effect
prevention/long-term treatment of DMD
Heterogeneity of regimens may
defeat and unquestionably
complicates new treatment
evaluations (e.g., PTC 124)
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Heterogeneous Steroid Regimens: A
Challenge for New Therapeutic Approaches
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Different benefits from different agents?
Varying time courses of benefit
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Improved in 10-90 days
Plateau in 3-12 mos
Decline in 18-? mos
Worsen off treatment in ?
Effect of variation on study power
Adverse effects differ
Different drug interactions.
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Corticosteroids vs curative new treatment?
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No proposed new treatment likely curative
Few new treatments applicable to all DMD
People underestimate the benefit of
corticosteroids
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30% longer ambulation
30% longer off ventilator
Survive?
(Better than pacemakers, defibrillators, all cancer
chemotherapy
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Does FOR-DMD preclude participation
in another study? Yes
Can subjects leave FOR-DMD to enter
another study? Yes
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This trial will also establish
standards of care in 41 centers
and 11 countries.
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