NCT06738017

Brief 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.

Trial Health

78
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
6

participants targeted

Target at below P25 for phase_1

Timeline
3mo left

Started Feb 2025

Geographic Reach
2 countries

7 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress81%
Feb 2025Aug 2026

First Submitted

Initial submission to the registry

November 27, 2024

Completed
20 days until next milestone

First Posted

Study publicly available on registry

December 17, 2024

Completed
2 months until next milestone

Study Start

First participant enrolled

February 21, 2025

Completed
1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 19, 2026

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 19, 2026

Last Updated

May 6, 2026

Status Verified

May 1, 2026

Enrollment Period

1.5 years

First QC Date

November 27, 2024

Last Update Submit

May 4, 2026

Conditions

Alpha 1-Antitrypsin Deficiency

Keywords

349349-102AATDMASHPiZZPiMZ

Outcome Measures

Primary Outcomes (1)

  • Participant Adverse Events, Serious Adverse Events, Dose Limit Toxicities, Adverse Event of Special Interests, abnormal laboratory tests, abnormal pulmonary function tests, and 12-lead ECG parameters

    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

    78 days

Secondary Outcomes (8)

  • C max

    78 days

  • AUC 0-t

    78 days

  • AUC 0-inf

    78 days

  • CL/F

    78 days

  • T max

    78 days

  • +3 more secondary outcomes

Study Arms (2)

Group A (PiZZ)

OTHER

5:1 (349:Placebo)

Drug: BMN 349Drug: Placebo

Group B (PiMZ)

OTHER

5:1 (349:Placebo)

Drug: BMN 349Drug: Placebo

Interventions

BMN 349DRUG

250mg oral tablet

Group A (PiZZ)Group B (PiMZ)
PlaceboDRUG

250mg oral tablet

Group A (PiZZ)Group B (PiMZ)

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Participants must have confirmation of PiZZ or PiMZ genotype
  • Females and males, of any race, 18 to 75 years of age
  • Nonsmokers, defined as not using tobacco or nicotine-containing products for at least 6 months prior to Screening

You may not qualify if:

  • 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 pneumonia

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

University of California, San Diego

San Diego, California, 92037, United States

Location

Saint Louis University

St Louis, Missouri, 63110, United States

Location

Medpace Clinical Pharmacology Unit

Cincinnati, Ohio, 45227, United States

Location

The Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

NHS Lothian

Edinburgh, UK, United Kingdom

Location

Royal Free London NHS Foundation Trust

London, UK, United Kingdom

Location

University Hospital Southampton NHS Foundation Trust

Southampton, UK, United Kingdom

Location

MeSH Terms

Conditions

alpha 1-Antitrypsin Deficiency

Condition Hierarchy (Ancestors)

Liver DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSubcutaneous EmphysemaEmphysemaPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Medical Director, MD

    BioMarin Pharmaceutical

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 27, 2024

First Posted

December 17, 2024

Study Start

February 21, 2025

Primary Completion (Estimated)

August 19, 2026

Study Completion (Estimated)

August 19, 2026

Last Updated

May 6, 2026

Record last verified: 2026-05

Data Sharing

IPD Sharing
Will not share

Locations

US(4)GB(3)