NCT04898309

Efficacy and Safety of GNR-038 vs Berinert® in Patients With Hereditary Angioedema

A Placebo-controlled Randomized Trial to Evaluate the Efficacy and Safety of GNR-038 in Comparison With Berinert® for Acute Attacks Relief in Patients With Hereditary Angioedema

• Status: Withdrawn
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: AO GENERIUM · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

It is a placebo-controlled randomized trial to evaluate the efficacy and safety of GNR-038 in comparison with Berinert® in patients with hereditary angioedema

Detailed description

Hereditary angioedema is a rare, potentially life-threatening genetically determined disease associated with a deficiency or impairment of the functional activity of the C1-esterase inhibitor (C1-inhibitor). The main clinical manifestation of hereditary angioedema is recurrent subcutaneous or submucosal swelling of various localization. Most often, the development of the disease is based on a mutation in the SERPING1 gene. The prevalence of the disease in the world ranges from 1:10 000 to 1:150 000. GNR-038 is a recombinant C1 inhibitor (rhC1INH), which is a complete structural and functional analog of the plasma C1 inhibitor. Phase I study results showed convincing safety and tolerability evidence of GNR-038.

Conditions

• Hereditary Angioedema

Interventions

Type	Name	Description
DRUG	GNR-038, 50 МЕ/ kg	A single intravenous infusion of GNR-038, 50 МЕ/ kg less than 5 hours after the onset of edema.
DRUG	GNR-038, 100 МЕ/ kg	A single intravenous infusion of GNR-038, 100 МЕ/ kg less than 5 hours after the onset of edema.
DRUG	Berinert®, 20 МЕ/ kg	A single intravenous infusion of Berinert®, 20 МЕ/ kg less than 5 hours after the onset of edema.
DRUG	Placebo	A single intravenous infusion of Placebo less than 5 hours after the onset of edema.
DRUG	GNR-038. The dose will be selected according to results of stage 1 clinical trial.	A single intravenous infusion of GNR-038 less than 5 hours after the onset of edema.
DRUG	Berinert®, 20 МЕ/ kg	A single intravenous infusion of Berinert®, 20 МЕ/ kg less than 5 hours after the onset of edema.

Timeline

• Start date: 2021-12-01
• Primary completion: 2023-05-31
• Completion: 2023-05-31
• First posted: 2021-05-24
• Last updated: 2022-02-04

Locations

5 sites across 1 country: Russia