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

Phase 1Active Not RecruitingNCT06738017

BioMarin Pharmaceutical6 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Alpha 1-Antitrypsin Deficiency

Interventions

BMN 349DRUG

250mg oral tablet

PlaceboDRUG

250mg oral tablet

Eligibility

Sex: ALLMin age: 18 YearsMax age: 75 Years

Medical Language ↔ Plain English

Inclusion Criteria: * 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 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 pneumonia

Locations (7)

University of California, San Diego

San Diego, California, United States

Saint Louis University

St Louis, Missouri, United States

Medpace Clinical Pharmacology Unit

Cincinnati, Ohio, United States

The Medical University of South Carolina

Charleston, South Carolina, United States

Outcomes

Primary Outcomes

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

Time frame: 78 days

Secondary Outcomes

C max

Maximum observed plasma concentration

Time frame: 78 days

AUC 0-t

Area under the concentration-time curve from time 0 to the last measurable concentration

Time frame: 78 days

AUC 0-inf

Area under the concentration-time curve from time 0 to infinity

Time frame: 78 days

CL/F

Apparent total body clearance after oral dosing

Time frame: 78 days

T max

Time to reach maximum concentration

Time frame: 78 days

t 1/2

Terminal half-life in plasma

Time frame: 78 days

Vz/F

Apparent volume of distribution during terminal phase

Time frame: 78 days

Data from ClinicalTrials.gov

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