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NCT06614894 · ClinicalTrials.gov registry record · Phase 2
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
A Phase 2 study of Sanfilippo Syndrome and MPS3, sponsored by Ozlem Goker-Alpan.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 10
- Enrollment target
- 1
- Study location
NCT06614894 is a Phase 2 study of Sanfilippo Syndrome and MPS3 that is actively recruiting participants, run by Ozlem Goker-Alpan. The registered enrollment target is 10 participants. The trial reports 1 study location across 1 state.
The verdict
NCT06614894, a Phase 2 study of Sanfilippo Syndrome and MPS3, is actively recruiting participants, sponsored by Ozlem Goker-Alpan.
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 10 participants
- Enrollment target
- 1
- Study location
Study Summary
A dose escalation study to evaluate the safety, tolerability, and pharmacologic properties of Ambroxol in adult participants with Sanfilippo disease(s) (MPS3).
Conditions Studied
Interventions
- DRUG Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day
- DRUG Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day
- DRUG Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day
Study Locations (1)
Virginia
- Lysosomal & Rare Disorders Research & Treatment Center, Inc. - Fairfax
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2024-12-01 |
| Est. Completion | 2026-06-30 |
| Phase | Phase 2 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06614894
The ClinicalTrials.gov registry entry for NCT06614894 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 10 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Ozlem Goker-Alpan, which has 1 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with Sanfilippo Syndrome appearing as the primary indexed condition, and to 3 interventions - of which Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day is the first listed.
NCT06614894 reports 1 study location spanning 1 distinct geographic area - top geographies include Virginia.
Frequently Asked Questions
What is clinical trial NCT06614894 about?
NCT06614894 is a clinical study titled "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". A dose escalation study to evaluate the safety, tolerability, and pharmacologic properties of Ambroxol in adult participants with Sanfilippo disease(s) (MPS3).
What is the current status of trial NCT06614894?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 10 participants. The study started on 2024-12-01. Estimated completion is 2026-06-30.
What conditions does trial NCT06614894 study?
This clinical trial studies the following conditions: Sanfilippo Syndrome, MPS3.
What interventions are being tested in trial NCT06614894?
The interventions under investigation include: Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day (DRUG), Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day (DRUG), Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day (DRUG).
Who is sponsoring clinical trial NCT06614894?
This trial is sponsored by Ozlem Goker-Alpan, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06614894 being conducted?
This trial has 1 study location across Virginia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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