Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult... | NCT06738017

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

NCT06738017

Sponsor

BioMarin Pharmaceutical

Status

Active, not recruiting

Last Update Posted

May 6, 2026Actual

Enrollment

6Actual

Phase

Phase 1

Conditions

Alpha 1-Antitrypsin Deficiency

Interventions

BMN 349
Placebo

Countries

United States
United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

May 2026

Overall Recruitment Status or Expanded Access Status

Active, not recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Feb 21, 2025Actual

Primary Completion Date

Aug 19, 2026Estimated

Completion Date

Aug 19, 2026Estimated

First Submitted Date

Nov 27, 2024

First Submission Date that Met QC Criteria

Dec 12, 2024

First Posted Date

Dec 17, 2024Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

May 4, 2026

Last Update Posted Date

May 6, 2026Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Not provided

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

6Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Group A (PiZZ)	Other	5:1 (349:Placebo)	Drug: BMN 349 Drug: Placebo
Group B (PiMZ)	Other	5:1 (349:Placebo)	Drug: BMN 349 Drug: Placebo

Interventions

Name	Type	Description	Arm Group Labels	Other Names
BMN 349	Drug	250mg oral tablet	Group A (PiZZ) Group B (PiMZ)

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Measure	Description	Time Frame
C max	Maximum observed plasma concentration	78 days
AUC 0-t	Area under the concentration-time curve from time 0 to the last measurable concentration

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

75 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Medical Director, MD	BioMarin Pharmaceutical	Study Director

Locations

Facility	Status	City	State	ZIP	Country	Contacts
University of California, San Diego		San Diego	California	92037	United States
Saint Louis University

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D019896	alpha 1-Antitrypsin Deficiency

Ancestor Terms

ID	Term
D008107	Liver Diseases
D004066	Digestive System Diseases
D008171	Lung Diseases
D012140	Respiratory Tract Diseases

Browse Leaves

Not provided

Browse Branches

Not provided