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NCT06738017 · ClinicalTrials.gov registry record · Phase 1
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
A Phase 1 study of Alpha-1 Antitrypsin Deficiency, sponsored by BioMarin Pharmaceutical.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
- 8
- Study locations
NCT06738017: Recruiting Phase 1 study of Alpha-1 Antitrypsin Deficiency, sponsored by BioMarin Pharmaceutical.
NCT06738017 is a Phase 1 study of Alpha-1 Antitrypsin Deficiency that is actively recruiting participants, run by BioMarin Pharmaceutical. The registered enrollment target is 12 participants, below the 2,341-participant average among 24 other Alpha-1 Antitrypsin Deficiency trials with a reported enrollment target (99% lower). The trial reports 8 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06738017, a Phase 1 study of Alpha-1 Antitrypsin Deficiency, is actively recruiting participants, sponsored by BioMarin Pharmaceutical.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
- 8
- Study locations
Study Summary
The goal of this clinical trial is to assess the safety and tolerability of a single oral dose of BMN 349 in participants with PiZZ or PiMZ/MASH. Primary outcome measures include incidence of any adverse events (including serious adverse events, dose limit toxicities, and adverse events of special interest), incidence of any laboratory test abnormalities, incidence of lung function test abnormalities and 12-lead ECG parameters. Participants will receive a single dose of either BMN 349 or placebo and then monitored for safety and tolerability.
Primary Outcome
Number of participant AEs, SAEs, DLTs, AESIs per physician's assessment, abnormal laboratory tests through whole blood samples, abnormal pulmonary function spirometry tests, and 12-lead ECG parameters changes from baseline following a single oral dose of BMN 349
Conditions Studied
Interventions
- DRUG Placebo
- DRUG BMN 349
Study Locations (8)
UK
- NHS Lothian - Edinburgh
- Royal Free London NHS Foundation Trust - London
- Nottingham University Hospitals - Nottingham
- University Hospital Southampton NHS Foundation Trust - Southampton
California
- University of California, San Diego - San Diego
Missouri
- Saint Louis University - St Louis
Ohio
- Medpace Clinical Pharmacology Unit - Cincinnati
South Carolina
- The Medical University of South Carolina - Charleston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2025-02-21 |
| Est. Completion | 2025-09 |
| Phase | Phase 1 |
What NCT06738017 shows while recruiting
NCT06738017 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 2,341-participant average among 24 other Alpha-1 Antitrypsin Deficiency trials with a reported enrollment target (99% lower).
The record links to 1 condition, with Alpha-1 Antitrypsin Deficiency appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT06738017 lists 8 locations in 5 states (UK, California, Missouri).
Frequently Asked Questions
What is clinical trial NCT06738017 about?
NCT06738017 is a clinical study titled "Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants". The goal of this clinical trial is to assess the safety and tolerability of a single oral dose of BMN 349 in participants with PiZZ or PiMZ/MASH. Primary outcome measures include incidence of any adverse events (including serious adverse events, dose limit toxicities, and adverse events of special ...
What is the current status of trial NCT06738017?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2025-02-21. Estimated completion is 2025-09.
What conditions does trial NCT06738017 study?
This clinical trial studies the following conditions: Alpha-1 Antitrypsin Deficiency.
What interventions are being tested in trial NCT06738017?
The interventions under investigation include: Placebo (DRUG), BMN 349 (DRUG).
Who is sponsoring clinical trial NCT06738017?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06738017 being conducted?
This trial has 8 study locations across California, Missouri, Ohio, South Carolina, UK. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT06738017's enrollment target sits among peer trials
12 22nd of 24 higher than 3 of 24 other Alpha-1 Antitrypsin Deficiency trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Alpha-1 Antitrypsin Deficiency 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.
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