1. FOR-DMD: Recruitment and Retention Challenges
Addressing the Challenges
Benefits vs risks of corticosteroids?
Why invest so much to study a treatment of established (short term) benefit?
Corticosteroids vs curative new treatment?
Does FOR-DMD preclude participation in another study?
Can subjects leave FOR-DMD to enter another study?
presentations/2010/Freiburg 2010

2. Benefits vs risks of corticosteroids?
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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presentations/2010/Freiburg 2010

3. No standard treatment (31 regimens)
Why invest so much to study a treatment of established (short term) benefit?
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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The first level bullet has been set up with a “null” character to make it appear unbulleted and still allow automatic bullets for subsequent levels. The second through fifth levels have bullets: from the left margin, tab once (or click the “increase indent” button) and the second level bullet will appear; further tabbing increases the indent and produces the corresponding bullet.
presentations/2010/Freiburg 2010

4. Heterogeneous Steroid Regimens: A Challenge for New Therapeutic Approaches
Different benefits from different agents?
Varying time courses of benefit
Improved in 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.
This slide has the Slide Design template “content” applied.
The first level bullet has been set up with a “null” character to make it appear unbulleted and still allow automatic bullets for subsequent levels. The second through fifth levels have bullets: from the left margin, tab once (or click the “increase indent” button) and the second level bullet will appear; further tabbing increases the indent and produces the corresponding bullet.
presentations/2010/Freiburg 2010

5. Corticosteroids vs curative new treatment?
No proposed new treatment likely curative
Few new treatments applicable to all DMD
People underestimate the benefit of corticosteroids
30% longer ambulation
30% longer off ventilator
Survive?
(Better than pacemakers, defibrillators, all cancer chemotherapy
This slide has the Slide Design template “content” applied.
The first level bullet has been set up with a “null” character to make it appear unbulleted and still allow automatic bullets for subsequent levels. The second through fifth levels have bullets: from the left margin, tab once (or click the “increase indent” button) and the second level bullet will appear; further tabbing increases the indent and produces the corresponding bullet.
presentations/2010/Freiburg 2010

6. Does FOR-DMD preclude participation in another study? Yes
Can subjects leave FOR-DMD to enter another study? Yes
This slide has the Slide Design template “content” applied.
The first level bullet has been set up with a “null” character to make it appear unbulleted and still allow automatic bullets for subsequent levels. The second through fifth levels have bullets: from the left margin, tab once (or click the “increase indent” button) and the second level bullet will appear; further tabbing increases the indent and produces the corresponding bullet.
presentations/2010/Freiburg 2010

7. This trial will also establish standards of care in 41 centers and 11 countries.