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May 2005 - SMA CARNI-VAL TRIAL
Multi-center Phase II Trial of Valproic Acid and Carnitine
in Patients with Spinal Muscular Atrophy
Salt Lake City, UT, May 3, 2005 — Project Cure SMA is pleased
to announce a phase II clinical trial to further evaluate a combined
regimen of valproic acid and carnitine in children with Spinal Muscular
Atrophy (SMA). This phase II trial has been reviewed and approved by the FDA. This trial is a collaborative multi-center
effort funded by Families of Spinal Muscular Atrophy.
Dr. Kathryn Swoboda, associate professor at the
University of Utah School of Medicine and principal investigator for
Project Cure SMA, will lead the effort. Additional sites include
Children’s Hospital of Michigan, Saint Justine Hospital in Quebec, John
Hopkins University, Ohio State University and University of Wisconsin
Children’s Hospital pending IRB approvals.
The primary objective of this Phase II trial is to
assess the efficacy of oral valproic acid (VPA) and carnitine in SMA
children 2-17 years of age.
“Given the success of the Phase I pilot trial, we are
excited to move ahead with this next phase of testing.” said Dr Kathryn
Swoboda. “This is an important step forward in identifying effective
treatments for SMA.”
Two groups of children will be enrolled in this study.
The first group will include children ages 2-8 years with SMA Type II or
non-ambulatory SMA Type III who can sit independently for at least 3
seconds without support. The second group will include children ages
3-17 years with SMA Types II or III who can stand independently without
braces or other support for up to 2 seconds. There will be specific
exclusions for these studies.
“We are very excited to be moving forward with this
important research,” said Audrey Lewis, Executive Director of Families
of SMA. “We are so grateful to all of the families who have come
together to provide the more than $2 million cost of this study.
Without the combined efforts of families and researchers, we could not
have reached this important milestone.”
The projected start date for enrolling patients in the
SMA CARNI-VAL Study is mid July 2005. The new Project Cure SMA web site,
www.projectcureSMA.org, includes status of enrollment at the various centers, and
direct contact information for each site. Families who are interested in
being contacted with regard to this trial or future clinical trials can
also register with the International SMA patient registry at Indiana
University:
Tel (317) 274-5745, or
www.iupui.edu/~medgen/hereditary/sma.html. A link is also available
via the FSMA website - www.fsma.org/registry2002.shtml.
About Project Cure SMA
Project Cure SMA is a collaborative initiative between
Families of SMA and clinical investigators designed to help facilitate
the rapid translation of promising new therapies to individuals with
SMA. A primary goal of Project Cure SMA is to develop safe and
well-tolerated clinical protocols to help identify truly effective
therapies.
Project Cure SMA team members include: Dr. Sandra
Reyna, Project Cure SMA Clinical Trials Manager; Project Cure SMA Team
Investigators: Drs. Gyula Acsadi, Children’s Hospital of Michigan;
Thomas Crawford, Johns Hopkins Medical Center; Guy D’Anjou, Centre de
Recherche de l’Hopital Sainte-Justine; Richard Finkel, Children’s
Hospital of Philadelphia; John Kissel, Ohio State University; Mary
Schroth, University of Wisconsin; Louise Simard, Centre de Recherche de
l’Hopital Sainte-Justine; Kathryn Swoboda (Principal Investigator),
Primary Children’s Medical Center; and Project Cure SMA Consultants:
Joanne Maczulski; Kristin Krosschell, Northwestern University; Charles
Scott, Children’s Hospital of Philadelphia and Bernie La Salle,
University of Utah.
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