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

BioMarin Pharmaceutical

Status and phase: Enrolling; Phase 1

Conditions: Alpha 1-Antitrypsin Deficiency

Treatments: Drug: Placebo; Drug: BMN 349

Study type: Interventional

Funder types: Industry

Identifiers

NCT06738017

349-102

Details and patient eligibility

About

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.

Enrollment: 12 estimated patients

Sex: All

Ages: 18 to 64 years old

Volunteers: No Healthy Volunteers

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 pneumonia

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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12 participants in 2 patient groups

Group A (PiZZ)

Other group

Description:

5:1 (349:Placebo)

Treatment:

Drug: BMN 349

Drug: Placebo

Group B (PiMZ)

Other group

Description:

5:1 (349:Placebo)

Treatment:

Drug: BMN 349

Drug: Placebo