International Niemann–Pick Disease Alliance

Sanofi Genzyme: Clinical trial update regarding ASCEND Trial for adults with ASMD

Following an update meeting with Genzyme regarding the Phase 2/3 Olipudase Alfa trial in adult patients (18+ years old) with ASMD (Niemann Pick Disease Type B), we are pleased to provide an update on the status of the Sanofi Genzyme ASCEND clinical trial. It has been confirmed that recruitment has resumed for this clinical trial at multiple sites globally.

The trial aims to enroll 36 patients in total. Currently 10 patients have been recruited for screening or are enrolled.  All interested patients will need to be screened to determine if they meet the inclusion criteria and it is anticipated that a larger number of patients will need to be screened in order to fully enroll the trial with 36 patients.  If you know someone who might be interested in joining the trial, please do not hesitate to share this news with them. They can find out more information regarding the clinical trial including inclusion and exclusion criteria and trial sites on the clinical trials.gov website https://www.clinicaltrials.gov/ct2/show/NCT02004691?cond=Acid+Sphingomyelinase+Deficiency%3B+++Niemann-Pick+Disease&rank=4

The trial currently has 15 sites across the US, Europe, Asia and Pacific. More trial sites are planned and the website will be updated as new trial sites open.  If there is no trial site in the country where the patient resides, he/she can contact Sanofi Genzyme to inquire as to the nearest trial site and to receive details of the trial. There is an email contact link regarding the clinical trial in the section of Contacts and Locations (see the link above).

Please feel free to share this information with those who might be interested.

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