An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc; BIIB031) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia A
Study ID: STU 072015-042
This is an open-label, single-arm, multicenter study evaluating the safety and efficacy of rFViiiFc in previously untreated pediatric subjects with severe hemophilia a when used according to local standard of care. Following the Baseline Visit, the investigator has the option to treat a subject episodically (on-demand) prior to initiating a prophylaxis regimen. The duration of episodic treatment is at the investigator's discretion, in accordance with local standard of care. at least 100 previously untreated subjects are planned to complete the study after reaching at least 100 eDs with rFViiiFc. one eD is defined as a 24-hour period in which a subject receives 1 or more doses of rFViiiFc, with the time of the first injection of rFViiiFc defined as the start of the eD. For the purpose of this study, a PuP is defined as a subject who has not had prior exposure to any FViii replacement product or any blood component prior to enrollment and has had no injection with rFViiiFc prior to confirmation of eligibility. Subjects with a documented plasma FViii activity of [Less Than]1%, who are actively bleeding and requiring emergent treatment, may be enrolled (see Section 9.2) and receive study drug after samples for inhibitor testing at the central laboratory have been obtained, with results pending. However, any such subject must be withdrawn if the central laboratory screening results indicate a positive inhibitor. up to 3 injections of rFViiiFc study drug are allowed following confirmation of eligibility and prior to the Baseline Visit. Surgery is allowed during the study. See Section 10.2.6 for further instructions. iTi with rFViiiFc is allowed during the study for those subjects developing, after exposure to rFViiiFc study drug, a positive high titer inhibitor ([GreaterThanorequalTo]5.00 Bu/mL) or a positive low titer inhibitor ([GreaterThanorequalTo]0.60 and [Less Than]5.00 Bu/mL) with bleeding episodes that cannot be adequately treated with rFViiiFc. See Section 10.2.7 for further instructions. Separate consent/assent is required before starting an iTi regimen. The first injection of rFViiiFc study drug after the Screening Visit must be administered by the investigator or a qualified delegate. Thereafter, study treatment may be administered by a parent/caregiver, a qualified medical professional under the direction of the investigator, self-administered by older children, or given at the clinic. Study treatment may also be injected in the hospital during surgery or during hospitalization due to major bleeding.
1. Ability of the subject or his parent or legal guardian to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations. Subjects may provide assent in addition to the parental/guardian consent, if appropriate. 2. Male, age <6 years at the time of informed consent. 3. Weight >=3.5 kg 4. Severe hemophilia A defined as <1 IU/dL (<1%) endogenous FVIII documented in the medical record or as tested during the Screening Period. Any subject who is enrolled based on results of the local laboratory must be withdrawn if the central laboratory screening results indicate a baseline FVIII activity level >=1% of normal.
- Cancer Related
- Healthy Volunteers
- UT Southwestern Principal Investigator
- Janna Marie Journeycake