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A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema

NCT05121376 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 44

Last updated 2024-05-16

No results posted yet for this study

Summary

This is a Phase 1/2, single-arm, open-label, dose-escalation and dose-expansion study of BMN 331 for the treatment of hereditary angioedema (HAE) due to C1 Esterase Inhibitor (C1-INH) protein deficiency. The study drug BMN 331is identified as AAV5 hSERPING1, an adeno-associated virus (AAV5)-based gene therapy vector that expresses wild-type human C1 Esterase Inhibitor (hC1-INH), under the control of a liver-selective promoter, and is being developed for the treatment of HAE with C1-INH deficiency. The pharmaceutical form of BMN 331 is a solution for intravenous infusion.

Conditions

Interventions

GENETIC

Dose 1 of BMN 331

BMN 331 AAV Gene Therapy

GENETIC

Dose 2 of BMN 331

BMN 331 AAV Gene Therapy

GENETIC

Dose 3 of BMN 331

BMN 331 AAV Gene Therapy

GENETIC

Dose 4 of BMN 331

BMN 331 AAV Gene Therapy

GENETIC

Dose 5 of BMN 331

BMN 331 AAV Gene Therapy

GENETIC

Dose 6 of BMN 331

BMN 331 AAV Gene Therapy

GENETIC

Dose 7 of BMN 331

BMN 331 AAV Gene Therapy

Sponsors & Collaborators

Principal Investigators

  • MD Medical Director · BioMarin Pharmaceutical

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-02-15
Primary Completion
2028-11-30
Completion
2028-11-30
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Spain

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05121376 on ClinicalTrials.gov