Last updated: March 7, 2024
Sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Overall Status: Active - Recruiting
Phase
N/A
Condition
N/ATreatment
N/AClinical Study ID
NCT05705674
22-LDRTC-01
Ages 5-99 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
- IRB - approved informed consent/assent signed by subject and/or parent(s) or legalguardian(s).
- Genetically confirmed diagnosis of MPS III disease Genomic DNA analysis demonstratinga homozygous or compound heterozygous pathogenic variants in SGSH (type A), NAGLU (type B), HGSNAT (type C), or N- acetylglucosamine-6-sulfatase GNS (type D).
- Male or female; five years of age and older
- Negative urine pregnancy test at screening for female subjects with child-bearingpotential
Exclusion
Exclusion Criteria:
- Unwilling or unable to follow protocol requirements as per principal investigator
- Any serious or chronic medical illness, including significant cardiac or severedebilitating pulmonary disease as determined by the investigator.
- Any medical condition that, in the opinion of the PI, would place a subject at unduerisk
- Inability to cooperate for clinical and safety data collection
- Use of genistein or Miglustat within one week of the study
- Evidence of hepatitis B or hepatitis C infection upon serological testing at screening
- Currently participating in another interventional drug trial or has completed aninterventional trial less than one month prior to the screening visit
Study Design
Total Participants: 6
Study Start date:
May 01, 2023
Estimated Completion Date:
December 31, 2024
Study Description
Connect with a study center
LDRTC
Fairfax, Virginia 22030
United StatesActive - Recruiting
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