Denali Therapeutics is conducting a observational research study, which can also be called a natural history study, into Sanfilippo syndrome Type A (MPS IIIA).
The purpose of the research study is to collect blood and behavioral information from participants with MPS IIIA. The researchers are seeking to gather data to advance the understanding of MPS IIIA to help inform future investigational treatment study in MPS IIIA.
Number of participants: 20 children with MPS IIIA
Duration: 2 years, which includes 7 study visits
Locations: United States
Qualifications to Participate:
- The child is between the ages of 4 months and < 14 years old at time of consent. (At least 10 participants must be between 4 months old and 4 years old at the time of consent.)
- Has a confirmed diagnosed of Sanfilippo syndrome Type A (MPS IIIA) based on at least two out of the three: documented reduced N-sulphoglucosamine sulphohydrolase (SGSH) activity, documented likely pathogenic variant of the SGSH gene, or documented elevated heparan sulfate levels in urine and/or blood.
- For scenarios that disqualify a child, visit this webpage.
Study participants will be compensated up to $1,200.
Learn more: For more information about this study and/or to sign up, click here. You can also read about it on ClinicalTrials.gov.