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

Part of paid clinical trials in Fairfax, Virginia.

Sponsor
Ozlem Goker-Alpan
Study ID
NCT06614894
Phase
PHASE2/PHASE3
Status
Recruiting
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Conditions

  • MPS3
  • Sanfilippo Syndrome

Eligibility Criteria

Sex
ALL
Age
18 Years - 99 Years
Healthy Volunteers
Not accepted

Interventions

  • Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day — DRUG
    Ambroxol Hydrochloride 30 mg oral pill/tablet - 9 mg/kg/day
  • Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day — DRUG
    Ambroxol Hydrochloride 30 mg oral pill/tablet - 18 mg/kg/day
  • Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day — DRUG
    Ambroxol Hydrochloride 30 mg oral pill/tablet - 27 mg/kg/day

Study Details

A dose escalation study to evaluate the safety, tolerability, and pharmacologic properties of Ambroxol in adult participants with Sanfilippo disease(s) (MPS3).

Key Dates

Start date
Dec 1, 2024
Status verified
Sep 2024
Primary completion
May 1, 2026
Completion
Jun 30, 2026

Study Design

Enrollment
10 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Ambroxol Hydrochloride 30mg - 9mg/kg/day
    Ambroxol Hydrochloride 30mg Dose escalation: Initial dose of 9mg/kg/day (or max dose 150mg TID)
  • Experimental: Ambroxol Hydrochloride 30mg - 18mg/kg/day
    Ambroxol Hydrochloride 30mg Dose escalation: 18mg/kg/day (or max dose 300mg TID)
  • Experimental: Ambroxol Hydrochloride 30mg - 27mg/kg/day
    Ambroxol Hydrochloride 30mg Dose escalation: 27mg/kg/day (or max dose 1350mg QD)

Primary Outcome Measure

Safety and Tolerability [ Time Frame: From baseline to 52 weeks ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Lysosomal & Rare Disorders Research & Treatment Center, Inc.FairfaxVirginia22030
Arooj Agha
[email protected]
571-732-4575
Lauren Noll
[email protected]
571-732-4655
Ozlem Goker-Alpan, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Fairfax, VA

By research site
Lysosomal & Rare Disorders Research & Treatment Center, Inc.· Fairfax, VA

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