IPA Board –
We wanted to provide some more information with regards to the recent change to the Late-Onset EAP posting on clinicaltrials.gov.
Recruitment for this program is now indicated as “suspended”. According to NIH definitions, suspended is defined as: recruiting or enrolling participants has halted but potentially will resume.
This change is in reference to “new” patients, those that have not already requested consideration for the Late-Onset Expanded Access Program. This has no impact on those late-onset patients who have already submitted a request for Expanded Access. As you may know, in all three regions (US, EU, and International) we received more qualified applications than we had enzyme available, so there was a random selection of 4 patients from each region (this process is still pending in the US). Those patients who were not selected will continue to be considered if more enzyme becomes available to this program, and we will be re-evaluating our ability to do that periodically.
The “suspended” status refers “new” Late-Onset EAP patients. We will continue to take the names of patients that would likely qualify for this program, but we will not be collecting information from physicians to determine medical eligibility for new patients at this time. Patients and physicians should call Genzyme Medical Information in the US and Europe with any questions.
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