Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

Phase 3CompletedNCT01992549

Octapharma48 enrolled

Overview

The purpose of the study is to collect long-term data on 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

Conditions

Severe Hemophilia A

Interventions

Human-cl rhFVIIIBIOLOGICAL

Eligibility

Sex: MALE

Medical Language ↔ Plain English

Inclusion Criteria: 1\. Patients who completed GENA-05 in accordance with the study protocol Exclusion Criteria: 1. Severe liver or kidney disease 2. Concomitant treatment with any systemic immunosuppressive drug; 3. Other FVIII concentrate than Human-cl rhFVIII was received between completion visit of GENA-05 and start of GENA-15 (except emergency cases).

Locations (15)

UC Davis Medical Center

Sacramento, California, United States

University of Alberta

Edmonton, Alberta, Canada

BC Children's Hospital

Vancouver, British Columbia, Canada

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 by the modified Bethesda assay (Nijmegen modification) using congenital FVIII-deficient human plasma spiked with Human-cl rhFVIII. The definition threshold for a "positive" inhibitor was if the modified Bethesda assay resulted in a titre ≥0.6 BU/mL at any time point during the observation period.

Time frame: Maximum two years

Secondary Outcomes

Frequency of Spontaneous Break-through Bleeds

The annualized bleeding rate (ABR) was calculated during the time of prophylactic treatment with Human-cl rhFVIII for spontaneous bleeding events (BEs).

Time frame: Maximum 2 years

Efficacy of Human-cl rhFVIII for the Treatment of Bleeds

A personal efficacy assessment (final outcome) to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes (BEs) at the end of a BE. Efficacy was assessed using a four-point scale (excellent, good, moderate, none) by the patient's parent(s)/legal guardian(s) together with the investigator in case of on site treatment.

Time frame: Maximum 2 years

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). If surgeries could not be assessed due to limited data available or having taken place outside the study site, the results were classified as "not done".

Time frame: Maximum 2 years

The Occurrence of Any Adverse Event (AE)

The frequency of AEs, as monitored throughout the whole study by the number of patients with at least one adverse event occurrence.

Data from ClinicalTrials.gov

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