Type B Enzyme Replacement Therapy

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Enzyme Replacement

Therapy - Type B

Genzyme Corporation has announced the open enrollment of two IRB-approved clinical research studies for individuals with Niemann-Pick disease caused by acid sphingomyelinase deficiency (Types A and B).

The first study is a Phase 1 clinical study of recombinant human acid sphingomyelinase administered as a single dose to adults (18-65 years old) with Niemann-Pick disease Type B. This enzyme replacement study will evaluate a range of sequential doses among approximately 15 patients at one center in the U.S. The primary objective of the study is safety, and the secondary objectives are pharmacokinetics and efficacy. Following a 3-day screening visit, eligible patients will make 3 additional visits to the study site over a 28-day period. One of the study visits involves a 4-day inpatient hospitalization during which the treatment will be administered.

For further information, please contact Genzyme Medical Information at 800-745-4447, option 2 and mention SPHINGO00605. The study is also posted on www.clinicaltrials.gov.

The second study is a non-treatment, chart review study to characterize the natural history of Niemann-Pick disease Types A and B. Findings from this study will help to define the natural progression of the disease and will assist in the selection of endpoints for future clinical treatment studies. Both living and non-living patients with Niemann-Pick disease Types A and B from the US and Canada are eligible for enrollment. Following the informed consent of patients or the next of kin, medical records will be requested and sent to one of the three study sites. De-identified information related to patient demographics, disease history, and health care utilization will be entered into a secure database maintained by Abt Associates, Lexington, MA. Enrollment of new patients will be completed in mid-2007.

For further information, please contact: Genzyme Medical Information at 800-745-4447, option 2 and mention SPHINGO00302.

You can also contact NNPDF for more information about these and other studies.