Cure Sanfilippo Foundation is pleased to share that Denali Therapeutics has opened additional enrollment spots in its clinical trial of DNL-126 for Sanfilippo Type A (MPS IIIA). Cohort A3 enrollment will be expanded to an additional 6 participants.
For more information, read the statement below from Denali …
Current enrolling cohorts in the Phase 1/2 study are below. Details on the inclusion and exclusion criteria can be found on ClinicalTrials.gov.
Cohort A3:
Eligible participants with a confirmed diagnosis of MPS IIIA. Cohort A3 will now enroll up to an additional 6 participants, for a total of approximately 10 participants. Due to high interest in the study, physician investigators and study teams are reviewing eligible participants from the existing waitlist, and many of the available slots have already been allocated across the four active sites.
Ongoing Cohorts without change to enrollment numbers:
Cohort B1 — Eligible participants less than 28 months old (2 years and 4 months) who have a severe phenotype, as determined by the Physician Investigator at the study site. Cohort B1 will enroll up to four participants.
Optional Cohort B2 — Enrolling eligible older siblings of participants in Cohort B1. Before a Cohort B2 participant may be screened, their younger sibling must be confirmed to be eligible for dosing in Cohort B1. Cohort B2 will enroll up to 4 participants.
Note: B1 participants may be enrolled even if they do not have an older sibling and/or their older sibling does not wish to participate.
Read more about this clinical trial and see the listing on ClinicalTrials.gov.