An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III

Exclusion Criteria11

• Unwilling or unable to follow protocol requirements as per principal investigator.
• Any serious or chronic medical illness, including significant cardiac or severe debilitating pulmonary disease.
• Poorly controlled seizures, defined as more than one seizure per day for the past 6 months.
• Medications identified as a strong inducers or inhibitors of CYP3A, and changing to another alternative drug to treat the condition would place the subject at undue risk.
• Any medical condition that, in the opinion of the PI, would make the subject unsuitable to participate in the study.
• Inability to cooperate for clinical and safety data collection.
• Known hypersensitivity to Ambroxol or any of its excipients.
• Use of genistein or Miglustat within one week of starting screening.
• Evidence of hepatitis B or hepatitis C infection upon serological testing at screening.
• Currently participating in another clinical trial or has completed an interventional trial less than 2 weeks prior to screening visit.
• The subject has received strong inducers (Note: eg, herbal supplements) or inhibitors of CYP3A within 15 days or 5 half-lives from screening, whichever is longer, prior to enrollment. This also includes the consumption of grapefruit, grapefruit juice, or grapefruit containing products within 72 hours of starting Ambroxol administration.