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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 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 3,077-participant average among 18 other Alpha-1 Antitrypsin Deficiency trials with a reported enrollment target (100% lower). The trial reports 8 study locations across 5 states.

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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.

Conditions Studied

Alpha-1 Antitrypsin Deficiency

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

FieldValue
Enrollment Target 12 participants
Start Date 2025-02-21
Est. Completion 2025-09
Phase Phase 1

Sponsor

BioMarin Pharmaceutical

80 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT06738017

The ClinicalTrials.gov registry entry for NCT06738017 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 3,077-participant average among 18 other Alpha-1 Antitrypsin Deficiency trials with a reported enrollment target (100% lower). The listed sponsor is BioMarin Pharmaceutical, which has 80 total studies on file at ClinicalTrials.gov.

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 reports 8 study locations spanning 5 distinct geographic areas - top geographies include 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.

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Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.