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Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants

NCT06738017 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2026-05-06

No results posted yet for this 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

Interventions

DRUG

BMN 349

250mg oral tablet

DRUG

Placebo

250mg oral tablet

Sponsors & Collaborators

Principal Investigators

  • Medical Director, MD · BioMarin Pharmaceutical

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-02-21
Primary Completion
2026-08-19
Completion
2026-08-19
FDA Drug
Yes

Countries

  • United States
  • United Kingdom

Study Locations

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Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06738017 on ClinicalTrials.gov