Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-24 @ 11:56 PM
Ignite Modification Date:
2025-12-24 @ 11:56 PM
NCT ID:
NCT05648851
Eligibility Criteria:
Inclusion Criteria:
To be eligible to participate in the study, patients must meet all of the following criteria:
1. Confirmed diagnosis of Sanfilippo syndrome type D disease by all of the following:
1. Deficiency in alpha-GNS enzyme activity
2. Has presented with signs/symptoms consistent with Sanfilippo syndrome type D, or, for individuals who have not presented with signs/symptoms of disease (eg, siblings of known patients), the determination of eligibility will be at the discretion of the Sponsor in conjunction with the site Investigator
3. Genomic DNA analysis demonstrating homozygous or compound heterozygous, pathogenic and/or potentially pathogenic variants in the GNS gene
4. Accumulated GAG HS in urine
2. Written informed consent from parent or legal guardian and assent from patient, if required
3. Parent/legal guardian willing to accompany the patient to all study visits
4. Ability to comply with protocol requirements, in the opinion of the Investigator
5. Negative urine pregnancy test at screening (nonsterile females of childbearing potential only) -
Exclusion Criteria:
Patients who meet any of the following criteria will not be eligible to participate in the study:
1. Have received an investigational drug within 30 days prior to the Baseline Visit
2. Concomitant illness or medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the patient's ability to comply with protocol requirements, the patient's well-being or safety, or the interpretability of the patient's clinical data
3. The presence of significant non-MPS IIID-related CNS impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study -
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
12 Months
Study:
NCT05648851
Study Brief:
Protocol Section:
NCT05648851