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Clinical Trial For Gaucher Disease Type 3

21 February 2017

Sanofi Genzyme is investigating an orally administered substrate reduction therapy in Gaucher disease type 3 as part of the 2-part LEAP clinical research study.

The first part of the LEAP study will measure and compare certain factors in blood and in the fluid that bathes the brain and spinal cord (also known as cerebrospinal fluid or CSF) in patients with Gaucher disease type 1 and type 3. Patients participating in part 1 of the study will not receive study drug. Gaucher disease type 3 patients may in participate in Part 2 if eligible.

The second part of the LEAP study will investigate if the study drug GZ/SAR402671, a glucosylceramide synthase inhibitor, works and is safe in patients with Gaucher disease type 3.  Patients eligible for part 2 will receive study drug for 12 months.  For some study visits patients may be required to stay overnight to complete all of the tests and procedures.

Patients participating in the trial will be evaluated through physical and neurological exams; CSF, blood and urine testing; and lung imaging.

The study is being conducted in multiple countries including the US, UK, and Germany.

Patients may be eligible to participate in LEAP who:

  • Are 18 years or older
  • Have Gaucher disease type 1 (part 1 only) or have Gaucher disease type 3 (must complete part 1 to participate in part 2)
  • Have received treatment with enzyme replacement therapy for at least 3 years and received Cerezyme at a stable dose for the past 6 months.

More information about the LEAP trial (PDY13949) can be obtained at www.clinicaltrials.gov, using study identifier NCT02843035.