NCT04592692

A Pharmacokinetic and Clotting Activity Study of FVIII-PEGLip

A Pharmacokinetic and Clotting Activity Study of FVIII-PEGLip Administered Intravenously to Severe Haemophilia A Patients With and Without Inhibitors to FVIII

• Status: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Ascension Healthcare Development Limited · Industry
• Sex: Male
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to demonstrate that PEGylated liposomes (PEGLip) can shield FVIII from the immune system and inhibitors, and therefore provide a prophylactic FVIII replacement therapy for patients with inhibitors to FVIII.

Detailed description

This is an open-label multicenter trial for patients with severe haemophilia A with inhibitors to FVIII and without inhibitors as control. The trial consists of 4 periods: Screening, Stage A, Stage B and Safety Follow-up. After signing informed consent, patients are assessed for eligibility during a Screening period lasting up to 21 days. All eligible patients enter Stage A - Regimen estimation. The non-inhibitor patients receive a single IV injection at a dose of 35 IU/kg FVIII reconstituted with Water For Injection. Following a 4-day wash-out period, these patients as well as patients with inhibitors receive a single IV injection of FVIII-PEGLip at a dose of 35 IU/kg FVIII + PEGLip 22 mg/kg to determine the duration of haemostatic cover and therefore required injection frequency to prevent bleeds. Stage B - multiple dosing: all patients receive injections of FVIII-PEGLip for 6 weeks at a frequency determined in Stage A for each individual patient. Safety follow-up: 15 and 30 days after the last injection of FVIII-PEGLip, patients are contacted for any adverse events or bleeding episodes.

Conditions

• Hemophilia A With Inhibitor

Interventions

Type	Name	Description
DRUG	PEGylated Liposome (PEGLip)	Intravenous co-administration of PEGLip with Simoctocog alfa

Timeline

• Start date: 2019-12-23
• Primary completion: 2022-02-28
• Completion: 2022-05-31
• First posted: 2020-10-19
• Last updated: 2021-10-21

Locations

5 sites across 1 country: Russia