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Study of BMN 349 Single Dose in (PiZZ) and (PiMZ) Adult Participants

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Trial Status Recruiting

Trial Identifier

NCT06738017

Condition

AATD, Alpha 1-Antitrypsin Deficiency

Study Resources

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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.

Eligibility Criteria

Birth Sex

All

Age

18 Years to 64 Years

Healthy Volunteers

No

Drug/Treatment:

BMN 349, Placebo

Phase:

Phase 1

Study Type:

Interventional

Number of Participants:

12

Study Started:

2025-02-21

Study Updated:

2025-03-25

Trial Locations

  • University of California, San Diego

    San Diego, California, United States

  • Saint Louis University

    Saint Louis, Missouri, United States

  • Medpace Clinical Pharmacology Unit

    Cincinnati, Ohio, United States

  • The Medical University of South Carolina

    Charleston, South Carolina, United States

Inclusion Criteria

  • Participants must have confirmation of PiZZ or PiMZ genotype
  • Females and males, of any race, 18 to 64 years of age
  • Nonsmokers, defined as not using tobacco or nicotine-containing products for at least 6 months prior to Screening

Exclusion Criteria

  • International normalized ratio (INR) > 1.2
  • Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels > 125 U/L
  • Current or recent use of AAT augmentation therapy
  • Participants with recent (last 3 months) diagnosis of pneumoni

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