Ozlem Goker-Alpan
A dose escalation study to evaluate the safety, tolerability, and pharmacologic properties of Ambroxol in adult participants with Sanfilippo disease(s) (MPS3).
This is a dose escalation study in which open label Ambroxol 30mg (study drug) will be administered to adult patients with Sanfilippo Disease (MPS3). Administration route of study drug will be either crushed and mixed with soft foods or as an Ambroxol suspension through a feeding tube, if applicable. Study Timeline - Screening: 4 weeks (28 days) Treatment Period: 52 weeks Post-Treatment (after Week 52): 4 weeks withdrawal/safety follow-up period Patients will be screened at which point a thorough review of the Informed Consent form will be completed, sNFL levels, urinary GAGs, and serum HS r…
Inclusion 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. 3. Genomic DNA analysis demonstrating a homozygous or compound heterozygous pathogenic variants in SGSH (type A), NAGLU (type B), HGSNAT (type C), or GNS (type D) genes. Type E will not be studied. 4. Elevated excretion of urinary GAGs and/or serum HS (if no historical data is available, screening GAGs and serum HS values will be utilized to assess inclusion criteria). 5. Male or female; eighteen years of age and older, w…
Ambroxol Hydrochloride 30 mg oral pill/tablet - 9 mg/kg/day
Ambroxol Hydrochloride 30 mg oral pill/tablet - 18 mg/kg/day
Ambroxol Hydrochloride 30 mg oral pill/tablet - 27 mg/kg/day
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