Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
For general information about clinical research, read Learn About Studies.
Eligibility Criteria
- 1. IRB - approved informed consent/assent signed by subject and/or parent(s) or legal guardian(s).
- 2. Genetically confirmed diagnosis of MPS III disease Genomic DNA analysis demonstrating a homozygous or compound heterozygous pathogenic variants in SGSH (type A), NAGLU (type B), HGSNAT (type C), or N- acetylglucosamine-6-sulfatase GNS (type D).
- 3. Male or female; five years of age and older
- 4. Negative urine pregnancy test at screening for female subjects with child-bearing potential
- 1. Unwilling or unable to follow protocol requirements as per principal investigator
- 2. Any serious or chronic medical illness, including significant cardiac or severe debilitating pulmonary disease as determined by the investigator.
- 3. Any medical condition that, in the opinion of the PI, would place a subject at undue risk
- 4. Inability to cooperate for clinical and safety data collection
- 5. Use of genistein or Miglustat within one week of the study
- 6. Evidence of hepatitis B or hepatitis C infection upon serological testing at screening
- 7. Currently participating in another interventional drug trial or has completed an interventional trial less than one month prior to the screening visit
Ages Eligible for Study
5 Years to 99 Years
Sexes Eligible for Study
ALL
Accepts Healthy Volunteers
No