Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants (NCT06738017)
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)
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: University of California, San Diego — San Diego, California
Age range: 18 Years to 75 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (7 Sites)
- University of California, San Diego — San Diego, California
- Saint Louis University — St Louis, Missouri
- Medpace Clinical Pharmacology Unit — Cincinnati, Ohio
- The Medical University of South Carolina — Charleston, South Carolina
- NHS Lothian — Edinburgh, UK
- Royal Free London NHS Foundation Trust — London, UK
- University Hospital Southampton NHS Foundation Trust — Southampton, UK
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Alpha 1-Antitrypsin Deficiency
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- Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease — Birmingham, Alabama
- A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD) — Birmingham, Alabama
- Alpha-1 Research Registry — Coral Gables, Florida
- Gene Therapy for Alpha 1- Antitrypsin Deficiency — New York, New York
Explore by condition: All Alpha 1-Antitrypsin Deficiency clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.