NCT05121376

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

A Phase 1/2 Open-Label, Dose-Escalation Study to Determine the Safety Tolerability & Efficacy of BMN 331 an AAV Vector-Mediated Gene Transfer of Human SERPING1 Gene in Subjects With HAE Due to Human C1-INH Deficiency

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 44 (estimated)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

BMN 331 is an investigational, single administration gene therapy intended to modify the disease course of HAE. Preclinical studies have shown that BMN 331 can transduce hepatocytes resulting in restoration of the deficient circulating levels of hC1-INH that cause HAE. Study 331-201 is a two-part (part A and part B), first-in-human, Phase 1/2 study designed to assess the safety and efficacy of BMN 331 in patients with HAE. Subjects will be followed for 5 years following BMN 331 infusion. Part A of the study is a dose escalation phase designed to assess the preliminary safety of a single IV administration of BMN 331 and to determine whether there is a dose-dependent increase in C1-INH protein expression following administration of BMN 331. Part B is a dose expansion phase designed to demonstrate that up to three safe doses of BMN 331 (as determined in Part A) sustains a clinically meaningful increase in C1-INH levels.

Conditions

• Hereditary Angioedema
• HAE

Interventions

Type	Name	Description
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

Timeline

• Start date: 2022-02-15
• Primary completion: 2028-11-01
• Completion: 2028-11-01
• First posted: 2021-11-16
• Last updated: 2024-05-16

Locations

16 sites across 3 countries: United States, Australia, Spain

Regulatory

• FDA-regulated drug study