Pharmacokinetic, Efficacy, and Safety Study of Octafibrin Compared to Haemocomplettan/Riastap

Octapharma

Status and phase: Completed; Phase 2

Conditions: Congenital Fibrinogen Deficiency; Afibrinogenemia

Treatments: Biological: Haemocomplettan® P or RiaSTAPTM; Biological: Octafibrin

Study type: Interventional

Funder types: Industry

Identifiers

NCT01575756

FORMA-01

Details and patient eligibility

About

The purpose of this study is to investigate pharmacokinetic properties, surrogate efficacy and safety of Octafibrin compared to Haemocomplettan® P/RiaSTAPTM in patients with congenital fibrinogen deficiency

Enrollment: 22 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Age ≥ 12 years.
• Documented congenital fibrinogen deficiency (afibrinogenemia).

Exclusion criteria

• Life expectancy > 6 month.
• Bleeding disorder other than congenital fibrinogen deficiency.
• Presence or history of hypersensitivity to study medication.
• Presence or history of deep vein thrombosis or pulmonary embolism within 1 year prior to enrollment.
• Presence or history of arterial thrombosis with 1 year prior to enrollment.
• Hypersensitivity to human plasma products.
• Acute bleeding.
• Pregnant or currently breast-feeding women.
• Suspicion of an anti-fibrinogen inhibitor as indicated by previous in vivo recovery (if available).
• Blood or plasma donation in the 3 months prior to enrollment.
• Human immunodeficiency virus (HIV) positive with a viral load > 200 particles/µl or > 400000 copies/mL.
• End-stage liver disease.
• History of oesophageal varicose bleeding.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

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22 participants in 2 patient groups

Octafibrin followed by Haemocomplettan® P or RiaSTAPTM

Experimental group

Description:

Participants received Octafibrin 70 mg/kg intravenously once followed by Haemocomplettan® P or RiaSTAPTM 70 mg/kg intravenously once 45 days later.

Treatment:

Biological: Octafibrin

Biological: Haemocomplettan® P or RiaSTAPTM

Haemocomplettan® P or RiaSTAPTM followed by Octafibrin

Experimental group

Description:

Participants received Haemocomplettan® P or RiaSTAPTM 70 mg/kg intravenously once followed by Octafibrin 70 mg/kg intravenously once 45 days later.

Treatment:

Biological: Octafibrin

Biological: Haemocomplettan® P or RiaSTAPTM