Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients

Octapharma110 enrolled

Overview

Investigate the inhibitor development rate of Human cl rhFVIII in previously untreated patients with severe Hemophilia A.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

110

Conditions

Severe Hemophilia A

Interventions

Human cl rhFVIIIBIOLOGICAL

Eligibility

Sex: MALE

Medical Language ↔ Plain English

Inclusion Criteria: * Male patients * Severe Hemophilia A (FVIII:C \<1%) * No previous treatment with FVIII concentrates or other blood products containing FVIII Exclusion Criteria: * Diagnosis with a coagulation disorder other than Hemophilia A * Severe liver or kidney disease * Concomitant treatment with any systemic immunosuppressive drug

Locations (37)

Outcomes

Primary Outcomes

Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors

The number of patients developing FVIII inhibitors was observed during the observation period by assessing inhibitor development using the modified Bethesda assay (Nijmegen modification). The definitions for thresholds were ≥0.6 to \<5 BU/mL for a "low titre" inhibitor and ≥5 BU/mL for a "high-titre" inhibitor.

Time frame: maximum 5 years (100 exposure days)

Secondary Outcomes

Frequency of Spontaneous Break-through Bleeds

The annualized bleeding rate (ABR) was calculated during inhibitor-free periods for spontaneous bleeding events (BEs) during prophylactic treatment with Human cl rhFVIII

Time frame: Maximum 5 years (100 exposure days)

Efficacy of Human-cl rhFVIII for the Treatment of Bleeds

A personal efficacy assessment to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes. Efficacy was assessed using a four-point scale (excellent, good, moderate, none).

Time frame: Maximum 5 years (100 exposure days)

Efficacy of Human-cl rhFVIII for Surgical Prophylaxis

An overall efficacy assessment to assess the efficacy of human-cl rhFVIII in surgical prophylaxis of minor and major surgeries. The efficacy assessment was analyzed using a four-point scale (excellent, good, moderate, none).

Time frame: Maximum 5 years (100 exposure days)

Data from ClinicalTrials.gov

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...and 27 more locations