Actively Recruiting
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
Led by Ozlem Goker-Alpan · Updated on 2025-08-20
10
Participants Needed
1
Research Sites
82 weeks
Total Duration
On this page
Sponsors
O
Ozlem Goker-Alpan
Lead Sponsor
T
Team Sanfilippo
Collaborating Sponsor
AI-Summary
What this Trial Is About
A dose escalation study to evaluate the safety, tolerability, and pharmacologic properties of Ambroxol in adult participants with Sanfilippo disease(s) (MPS3).
CONDITIONS
Official Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- IRB-approved informed consent or assent signed by participant or legal guardian
- Genetically confirmed diagnosis of MPS III disease
- Genomic DNA showing pathogenic variants in SGSH (type A), NAGLU (type B), HGSNAT (type C), or GNS (type D) genes
- Elevated urinary GAGs and/or serum HS levels, or screening values if no prior data
- Male or female, 18 years or older, able to take Ambroxol orally
- Negative urine pregnancy test for females with child-bearing potential at screening
- Willing to avoid grapefruit products for 72 hours before first dose and during treatment
You will not qualify if you...
- Unable or unwilling to follow study protocol
- Serious or chronic illness including significant cardiac or severe lung disease
- Poorly controlled seizures (more than one per day in past 6 months)
- Use of strong CYP3A inducers or inhibitors that cannot be safely changed
- Any medical condition making participation unsuitable per investigator
- Inability to cooperate with clinical and safety assessments
- Known allergy to Ambroxol or its ingredients
- Use of genistein or Miglustat within one week before screening
- Evidence of hepatitis B or C infection at screening
- Currently in another clinical trial or completed one less than 2 weeks before screening
- Use of strong CYP3A inducers or inhibitors or grapefruit products within specified time before enrollment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Lysosomal & Rare Disorders Research & Treatment Center, Inc.
Fairfax, Virginia, United States, 22030
Actively Recruiting
Research Team
A
Arooj Agha
CONTACT
L
Lauren Noll
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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