Informations about the clinical trial
"Prednisone and Cyclosporin A in DMD" from Prof.Dr. Rudolf
Korinthenberg:
Our study is a randomized
placebo controlled trial that started in January 2004. There are 10 study
centers in Germany, Swizzerland and probably Austria who will have to recruit
150 Boys over 5 years. During the first 3 months of treatment we compare
Cyclosporin monotherapy to placebo. We want to see whether strength increases
as was suggested by 2 open studies. During the following 12 months both groups
(CsA and Placebo) will in addition be treated with prednisone in an
intermittent scheme. Here we want to
find out whether the effect of prednisone can be improved by CsA. Additionally,
of course, we monitor side effects.
Previous studies with cyclosporin:
1993 - Neurology,
Vol 43, Issue 3 527-532: Cyclosporine increases
muscular force generation in Duchenne muscular dystrophy KR Sharma, MA Mynhier
and RG Miller
We investigated the effect of cyclosporine (CsA) on force generation in 15
boys with Duchenne muscular dystrophy (DMD) by obtaining monthly measures
of tetanic force and maximum voluntary contraction (MVC) of both anterior
tibial muscles. During 4 months of a natural history phase, both tetanic
force and MVC declined significantly. During 8 weeks of CsA treatment (5
mg/kg/day), significantly increased tetanic force (25.8 +/- 6.6%) and MVC
(13.6 +/- 4.0%) occurred within 2 weeks. The maximum mean increase during
treatment was 35.2 +/- 5.9% (tetanic force) and 19.0 +/- 4.6% (MVC). Side
effects from CsA, gastrointestinal and flu-like symptoms, were transient
and self-limiting. Thus, as previously reported with prednisone, CsA
increases muscular force generation in the anterior tibial muscles of DMD
patients.
1997 - Muscle Nerve, 20, 469–478. Myoblast implantation in
Duchenne Muscular Dystrophy: The San Francisco Study
ABSTRACT: We evaluated myoblast implantation
in 10 boys with Duchenne muscular dystrophy (DMD) and absent dystrophin (age
5–10 years) who were implanted with 100 million myoblasts in the anterior
tibial muscle of one leg and placebo in the other. Cyclosporine (5 mg/kg/day)
was administered for 7 months. Pre- and postimplantation (after 1 and 6 months)
muscle biopsies were analyzed. Force generation (tetanic tension and maximum
voluntary contraction) was measured monthly in a double-blind design. There was
increased force generation in both legs of all boys, probably due to cyclosporine.
Using the polymerase chain reaction, evidence of myoblast survival and
dystrophin mRNA expression was obtained in 3 patients after 1 month and in 1
patient after 6 months. These studies suggest a salutary effect of cyclosporine
upon muscular force generation in Duchenne muscular dystrophy; however,
myoblast implantation was not effective in replacing clinically significant
amounts of dystrophin inDMD muscle.