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Hemophilia A With Inhibitors
+1

A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa

Sponsored by Laboratoire français de Fractionnement et de Biotechnologies

About this trial

Last updated 3 years ago

Study ID

LFB-FVIIa-007-14

Status

Completed

Type

Interventional

Phase

Phase 3

Placebo

No

Accepting

18-75 Years
Up to 11 Years
Male
Male

Not accepting

Not accepting
Healthy Volunteers

Trial Timing

Ended 8 years ago

What is this trial about?

The purpose of the study is to assess the safety, efficacy and pharmacokinetics of 2 separate dose regimens (75µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or IX in 12 patients ( birth to <6 years old), and 12 patients (≥6 years old to <12 years old).

What are the participation requirements?

Yes

Inclusion Criteria

- be male with a diagnosis of congenital hemophilia A or B of any severity

- have one of the following:

- a positive inhibitor test BU ≥5, OR

- a Bethesda Unit (BU) <5 but expected to have a high anamnestic response to FVIII or FIX, as demonstrated from the patient's medical history, precluding the use of factor VIII or IX products to treat bleeding episodes, OR

- a BU <5 but expected to be refractory to increased dosing of FVIII or FIX, as demonstrated from the patient's medical history, precluding the use of factor VIII or IX products to treat bleeding episodes

- be aged from birth to <12 years old

- have experienced at least 3 bleeding episodes of any severity in the past 6 months

- parents or legal guardians must be capable of understanding and be willing to comply with the conditions of the protocol

- parents or legal guardians must have read, understood, and provided written informed consent

No

Exclusion Criteria

- have any coagulation disorder other than hemophilia A or B

- be immunosuppressed (i.e., the patient may not be receiving systemic immunosuppressive medication; cluster of differentiation 4 (CD4) counts at screening must be >200/µL)

- have a known allergy or hypersensitivity to rabbits

- have platelet count <100,000/mL

- have had a major surgical procedure (e.g. orthopedic, abdominal) within 1 month prior to first administration of study drug

- have received an investigational drug within 30 days of first study drug administration, or be expected to receive such drug during participation in this study

Locations

Location

Status