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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: Recruiting Phase 2 study of Sanfilippo Syndrome and MPS3, sponsored by Ozlem Goker-Alpan.
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, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (92% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
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).
Primary Outcome
1\. Safety and tolerability as measured by number of Participants with at least one serious and at least one non-serious Treatment Emergent Adverse Events (TEAEs), assessed by CTCAE v4.0.
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 |
What NCT06614894 shows while recruiting
NCT06614894 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (92% lower).
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 a single indexed study location in 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.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT06614894's enrollment target sits among peer trials
10 1968th of 2000 higher than 12 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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