Natural History Study
The FDA has approved a Natural History Study of MLD to formally describe how the untreated disease develops to provide the details of an untreated baseline for the progression of MLD to enable a comparison of the outcome of a planned US clinical trial of Shires ERT, HGT-1111 (formerly Zymenex's ERT, Metazym).
Patients are currently being recruited for this Natural History Study. The study will includes 10 patients with late-infantile MLD, and has been approved by the IRB (Internal Review Board) at University of North Carolina, Chapel Hill, North Carolina, USA and is being performed by Dr. Maria Escolar at the Center for the Study of Development and Learning and FPG Child Development Institute, University of North Carolina, Chapel Hill, North Carolina, USA, with support from Shire HGT. Read the full press release here.
The ClinicalTrials.gov information on the Natural History Study can be seen here.
Please see the update below on the Phase II clinical trial now at the 1 year point in Denmark. The US clinical trail has not been announced, however, this Natural History Study is a key precursor to the anticipated trial.
The MLD Foundation met with in Copenhagen with Dr. Fogh, the president of Zymenex, on February 27th, 2008 to discuss the Natural History Study and the anticipated US Clinical Trial. We will continue to work closely with Zymenex and Dr. Escolar at UNC to provide timely information about research developments, including an announcement of the Clinical Trial as soon as such information is made public.