Clinical Trials in Muscular Dystrophy

If you have some information of this trials or for another I have pleasure to include in this site (send e-mail to [email protected] )

1) Research phase I clinical trial of full-length dystrophin plasmid DNA in Duchenne/Becker muscular dystrophies - from Institute de Myologie and Transgene (France):

The most recent information obtained by our friend from Norway, Berit Sofie: 

e-mail from Serge Braun, PharmD, PhD - Vice-President Research Transgene S.A.:

Dear Berit,

We are working on gene therapy of Duchenne dystrophy, but still at a preliminary stage (phase I clinical trial). As you may have heard, we are currently completing a phase I clinical trial with a dystrophin-plasmid non-viral vector injected intra-muscularly. The goal is to demonstrate good tolerance and safety of the vector and of the newly produced dystrophin. The next step is to take the intra-arterial delivery procedure to the clinic (trial expected 2004); at this stage we would like to target a small foot muscle, again for safety reasons. Should it work, we would than move on to a more ambitious treatment, getting to whole limb muscles (expected 2006 ?). Then, we would target respiratory and cardiac muscles. But, there is still a long way to go.
I understand time is running too fast for the Duchenne kids and families. It is however impossible to proceed faster for security reasons. It would be a catastrophy should a severe side effect or any undesired event happend.
Keep in touch.

Serge Braun 

2) Phase I clinical trial to evaluate in humans the efficacy of mioblast therapy for Duchenne Muscular Dystrophy - Dr. Jacques P. Tremblay, Canada

http://www.newswire.ca/releases/October2002/16/c1427.html

The most recent information obtained by our friend Uli Gehrt from Germany:

Dear Mr Gehrt, thank you for your interest in my research work. We have started a Phase I clinical trial! Myoblasts will be transplanted in only 1 cm cube of the Tibialis muscle. A muscle biopsy will be made 1 month later to assess the presence of dystrophin positive fibers. We will have the results of 10 patients. I hope by the end of 2003.
 Have a Merry Christmas!

Jacques P. Tremblay

3) Albuterol in DMD - UCLA, USA

The most recent information obtained by our friend Berit Sofie from Norway:

We have completed a pilot study and found that when the children were on long acting albuterol pills they exhibited an average of an 8% increase in strength compared to a slight loss in strength while on the placebo pills. We are now performing a larger scale study with 25 children and some slight changes in the protocol to further study this treatment. The youngest child in our study so far was 5 years old.

Sincerely
Eileen Fowler, PhD, PT

4) An open-label pilot study of Oxatomide in steroid-naive Duchenne muscular dystrophy

5) An open-label pilot study of Coenzyme Q10 in steroid-treated Duchenne muscular dystrophy

6) A multicenter randomized placebo-controlled double-blind study to assess efficacy and safety of glutamine and creatine monohydrate in Duchenne muscular dystrophy (DMD)

7) Study of Albuterol and Oxandrolone in Patients With Facioscapulohumeral Dystrophy (FSHD)

8) Study of Inherited Neurological Disorders

9) Phase I Clinical Trial Utilizing Gene Therapy for Limb Girdle Muscular Dystrophy: a, b, g, or d-Sarcoglycan Sarcoglycan Gene Delivered with Intramuscular Instillations of Adeno-Associated Vectors   NEW

Principal investigator - Dr. Jerry Mendell
Ohio State University
Columbus, Ohio
USA
http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=10757357&dopt=Abstract

10) Registry of Arrhythmias in Myotonic Muscular Dystrophy (MMD). A Long Term Study To Evaluate Heart Rhythm Disturbance in Subjects With MMD  NEW

http://www.centerwatch.com/patient/studies/stu41452.html

11) Prednisone and Cyclosporin A in Duchenne Muscular Dystrophy - Clinical trial in Germany

More information:
Dr. Janbernd Kirschner - [email protected]
University Children's Hospital
Division of Neuropediatrics and Muscle Disorders
Mathildenstrasse 1
79106 Freiburg
Tel. (+49) 761/270-4300
Fax (+49) 761/270-4344