This study is in progress, not accepting new patients

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

a study on Alpha-1 Antitrypsin Deficiency

Summary

Eligibility
for people ages 18-75 (full criteria)
Location
at UCSD
Dates
study started
study ends around

Description

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.

Official Title

A Randomized, Double-Blind, Placebo-Controlled, Single Oral Dose Study Evaluating the Safety and Pharmacokinetics of BMN 349 in Homozygous for the Z Mutation of Alpha 1 Antitrypsin Gene (PiZZ) and Heterozygous for the Z Mutation (PiMZ/MASH)

Keywords

Alpha 1-Antitrypsin Deficiency, 349, 349-102, AATD, MASH, PiZZ, PiMZ, BMN 349

Eligibility

You can join if…

Open to people ages 18-75

  • 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 CAN'T join 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

Locations

  • University of California, San Diego
    San Diego California 92037 United States
  • Saint Louis University
    St Louis Missouri 63110 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
BioMarin Pharmaceutical
ID
NCT06738017
Phase
Phase 1 Alpha-1 Antitrypsin Deficiency Research Study
Study Type
Interventional
Participants
About 6 people participating
Last Updated