Phase 4
Completed N=37
Study Evaluating Safety And Efficacy Of Moroctocog Alfa (AF-CC) In Previously Treated Hemophilia A Patients
Source: ClinicalTrials.gov NCT00914459 ↗Enrolled (actual)
37
Serious AEs
16.2%
Results posted
Dec 2016
Primary outcomePrimary: Percentage of Participants With Clinically Significant Factor VIII Inhibitor Development — 0.00; 0.00 percentage of participants
◆ 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 This can indicate results are unpublished — a known publication-bias signal.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 study will be investigating pharmacokinetics, safety and efficacy in patients less than 12 years of age with severe hemophilia A that have been previously treated with Factor VIII products ( including blood products).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Clinically Significant Factor VIII Inhibitor Development |
0.00; 0.00 | — |
| PRIMARY Incremental Recovery |
1.67; 1.97; 1.23; 1.91; 1.66; 1.96 | — |
| PRIMARY Terminal Elimination Half Life of ReFacto AF (t1/2) |
9.12 | — |
| PRIMARY Clearance (CL) |
4.406 | — |
| SECONDARY Mean Annualized Bleeding Rates (ABRs): All Participants |
27.51; NA; 4.18 | — |
| SECONDARY Response to First On-Demand Treatment for New Bleeds: All Participants |
713; 73; 16; 2 | — |
| SECONDARY Number of On-Demand ReFacto AF Infusions to Treat a New Bleed: All Participants |
1.1 | — |
| SECONDARY Number of Breakthrough Bleeds Within 48 Hours of a Prophylaxis Dose of ReFacto AF: All Participants |
2.0 | — |
| SECONDARY Average Infusion Dose of ReFacto AF: All Participants |
37.0; 29.5 | — |
| SECONDARY Total Factor VIII Consumption: All Participants |
97959.4; 84051.7 | — |
| SECONDARY Number of Less-Than-Expected-Therapeutic Effect (LETE) Bleeds in the On-Demand Setting: All Participants |
— | — |
| SECONDARY Number of Less-Than-Expected-Therapeutic Effect (LETE) Bleeds in the Prophylaxis Setting: All Participants |
2 | — |
| SECONDARY Number of Occurrences of Less-Than-Expected-Therapeutic Effect (LETE) in the Low Recovery Setting: All Participants |
9 | — |
| SECONDARY Number of Participants Requiring Escalated Dose of Prescribed Regimen During the Treatment Period: All Participants |
5 | — |
| SECONDARY Plasma Concentration of Factor VIII at 0.5 Hour Post-dose (C0.5) |
0.752; 0.903 | — |
| SECONDARY Area Under the Plasma Time Curve From Time 0 Extrapolated to Infinite Time (AUCinf) |
9.89 | — |
| SECONDARY Area Under the Plasma Time Curve From Time Zero to Time of Last Measurable Concentration (AUClast) |
9.49 | — |
| SECONDARY Volume of Distribution at Steady State (Vss) |
56.42 | — |
| SECONDARY Mean Residence Time (MRT) of ReFacto AF |
13.91 | — |
| SECONDARY Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs): All Participants |
28; 6 | — |
Eligibility Criteria
Inclusion Criteria
- Male subjects less than 12 years of age with a documented history of severe hemophilia A (FVIII:C less than 1%).
- Subjects who are less than 6 years of age must have had at least 50 Exposure Days (EDs) to prior FVIII products (including blood products).
- Subjects who are equal to or greater than 6 years of age must have had greater than 150 EDs to prior FVIII products (including blood products).
Exclusion Criteria
- For laboratory assessments, any measured Bethesda inhibitor titer equal to or greater than 0.6 BU, regardless of the laboratory normal range, or any Bethesda inhibitor titer greater than ULN for the testing laboratory at the time of screening.
- Any other bleeding disorder in addition to hemophilia A.
- Treatment with any investigational drug or device within 30 days before the time of signing the parental informed consent/assent form.
- Major surgery planned to occur during the course of the study.
- Regular (e.g., daily; every other day) use of agents or medications known to influence platelet function such as aspirin or certain nonsteroidal anti-inflammatory drugs (NSAIDS).
- Regular, concomitant therapy with immunomodulating drugs (e.g., intravenous immunoglobulin [IVIG], routine systemic corticosteroids), or currently receiving immune tolerance induction (ITI) for inhibitor treatment.
- The subject is receiving treatment for HIV or hepatitis infection (unless the subject is on a stable antiviral regimen [i.e., consistent treatment regimen for at least 3 months before the parental informed consent/assent form is signed]).
- Platelet count less than 100,000/µL.
- Prothrombin time (PT) equal to or greater than 1.25 x ULN, or international normalized ratio (INR) equal to or greater than 1.5.
- Known hypersensitivity to hamster protein.
Data sourced from ClinicalTrials.gov (NCT00914459). 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.