Phase 3
Completed N=66
Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIII
Source: ClinicalTrials.gov NCT00543439 ↗Enrolled (actual)
66
Serious AEs
25.5%
Results posted
Jan 2019
Primary outcomePrimary: Mean Annualized Bleed Rate (ABR) by Treatment: On Demand Cohort — 47.0; 1.5 Bleeds per year — p=0.0020
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
Summary
The purpose of this research study is to determine the effectiveness, safety, and pharmacokinetics (PK) of moroctocog alfa (AF-CC) in previously treated subjects, who are younger than 6 years of age, with severe or moderately severe hemophilia A.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Annualized Bleed Rate (ABR) by Treatment: On Demand Cohort |
47.0; 1.5 | 0.0020 sig |
| SECONDARY Mean Annualized Bleed Rate (ABR) by Treatment: Routine Prophylaxis Cohort |
3.3; 2.2 | — |
| SECONDARY Mean of Moroctocog Alfa (AF-CC) Infusions Administered To Treat Bleeding Episode: All Participants |
1.5 | — |
| SECONDARY Number of Treated Bleeds Classified on Basis of Response to First Infusion of Moroctocog Alfa (AF-CC) as On-Demand Treatment: OD Therapy (OD and RP Cohort) |
376; 150; 27; 2; 4 | — |
| SECONDARY Number of Treated Spontaneous Bleeds by Time Interval Between Bleed Onset and Prior Moroctocog Alfa (AF-CC) Prophylaxis Dose: Routine Prophylaxis Therapy |
1; 3; 4; 6; 10; 2 | — |
| SECONDARY Number of Participants Requiring Prophylaxis Regimen Escalation: Routine Prophylaxis Therapy |
2; 1 | — |
| SECONDARY Mean Routine Prophylaxis Dose (IU/kg) of Moroctocog Alfa (AF-CC) Received: Routine Prophylaxis Therapy |
25; 46; 26; 26 | — |
| SECONDARY Mean of Total Number Moroctocog Alfa (AF-CC) Infusions Received: Routine Prophylaxis Therapy |
170; 91; 150; 154 | — |
| SECONDARY Mean of Total Number of Days Participants Exposed to Moroctocog Alfa (AF-CC): Routine Prophylaxis Therapy |
170; 91; 150; 153 | — |
| SECONDARY Mean of Total Number of Infusions of Moroctocog Alfa (AF-CC) Received Per Week to Assess Compliance: Routine Prophylaxis Therapy |
2.1; 3.3 | — |
| SECONDARY Terminal Phase Half Life (t1/2) of Factor VIII (FVIII) Activity |
8.86 | — |
| SECONDARY Clearance (CL) of Factor VIII Activity |
5.822 | — |
| SECONDARY Incremental Recovery of Factor VIII Activity |
1.4438; 1.4148 | — |
| SECONDARY Maximum Concentration of Factor VIII Activity |
0.7005 | — |
| SECONDARY Area Under the Curve From Time Zero to Extrapolated Infinite Time (AUCinf) of Factor VIII Activity |
9.02 | — |
| SECONDARY Area Under the Curve From Time Zero to Last Measurable Concentration (AUClast) of Factor VIII Activity |
8.04 | — |
| SECONDARY Steady-State Volume of Distribution (Vss) of Factor VIII Activity |
78.38 | — |
| SECONDARY Mean Residence Time (MRT) of Factor VIII Activity |
13.46 | — |
| SECONDARY Number of Participants With Treatment Emergent Adverse Events (AEs) According to Severity |
12; 29; 8; 0 | — |
| SECONDARY Number of Participants With Treatment-Related Adverse Events |
49 | — |
| SECONDARY Number of Participants With Confirmed FVIII Inhibitor Development |
3 | — |
| SECONDARY Number of Participants With Incidence of Less Than Expected Therapeutic Effect (LETE): On Demand Therapy |
0; 0; 0 | — |
| SECONDARY Number of Participants With Incidence of Less Than Expected Therapeutic Effect (LETE): Routine Prophylaxis Therapy |
3; 5 | — |
Eligibility Criteria
Inclusion Criteria
- Male subjects, aged less than 6 years, with moderately severe to severe hemophilia A.
- A negative FVIII inhibitor titer at screening, and a medical history negative for a past FVIII inhibitor.
- At least 20 exposure days to any FVIII replacement product.
- Adequate hepatic and renal function
- CD4 count > 400 cells/uL, and if receiving antiviral therapy must be on a stable regimen
Additional criteria for subjects participating in the PK assessment:
- Male subjects as described immediately above except they must have a FVIII Activity of less than or equal to 1% confirmed by the central laboratory screening test
- Age < 6 years at time of PK assessment.
- The subject's size is sufficient to permit PK-related phlebotomy.
- The subject is able to comply with the procedures conducted during the PK assessment, including a mandatory 72-hour washout period preceding the PK assessment.
Exclusion Criteria
- A history of FVIII inhibitor.
- Presence of a bleeding disorder in addition to hemophilia A.
- Treatment with any investigational drug or device within 30 days before the time of signing the informed consent form.
- Major or orthopedic surgery planned to occur during the course of the study.
- Regular (e.g., daily, every other day) use of antifibrinolytic agents or medications known to influence platelet function such as aspirin or certain nonsteroidal anti-inflammatory drugs (NSAIDs), or regular, concomitant therapy with immunomodulating drugs (e.g., intravenous immunoglobulin [IVIG], routine systemic corticosteroids).
- Known hypersensitivity to hamster protein.
Data sourced from ClinicalTrials.gov (NCT00543439). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.