Phase 3
Completed N=174
An Open-label Safety, Efficacy and Pharmacokinetic Study of a Recombinant FVIII Compared to Recombinant Human Antihemophilic FVIII in Patients With Severe Hemophilia A
Source: ClinicalTrials.gov NCT01486927 ↗Enrolled (actual)
174
Serious AEs
4.6%
Results posted
Aug 2016
Primary outcomePrimary: Treatment Success — 92.4; 92.2; 92.3 % bleeding events successfully treated
◆ Published Evidence
Established
86citations · ~9 / year
Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A.
Summary
This is an open-label, non-randomized, efficacy, safety and pharmacokinetic (PK) study comparing octocog alfa and rVIII-SingleChain. The study consists of three parts, a PK period (Part 1), a continuation of dosing safety and efficacy period (Part 2) and a safety, efficacy, and repeat PK period (Part 3) and also includes a surgical sub-study for subjects enrolled in Parts 2 and 3.
Linked Publications
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Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Treatment Success |
92.4; 92.2; 92.3 | — |
| PRIMARY Inhibitor Formation to FVIII |
— | — |
| PRIMARY Annualized Spontaneous Bleeding Rate |
11.73; 0.00 | < 0.0001 sig |
| PRIMARY Treatment Success During the Peri-operative Surgical Sub-study |
100 | — |
| SECONDARY AUC0-∞ (Part 1) |
1550; 2090 | — |
| SECONDARY Cmax (Part 1) |
116; 113 | — |
| SECONDARY Tmax (Part 1) |
0.583; 0.683 | — |
| SECONDARY Half-life (t1/2) (Part 1) |
13.3; 14.5 | — |
| SECONDARY Mean Residence Time (MRT) (Part 1) |
17.1; 20.4 | — |
| SECONDARY Clearance (Cl) (Part 1) |
3.68; 2.64 | — |
| SECONDARY Volume of Distribution at Steady-state (Vss) (Part 1) |
57.1; 50.0 | — |
| SECONDARY Incremental Recovery (Part 1) |
2.32; 2.24 | — |
| SECONDARY Annualized Bleeding Rate for Total Bleeds and Traumatic Bleeds |
19.64; 1.14; 3.12; 0.00 | — |
| SECONDARY Proportion of Bleeding Episodes Requiring 1, 2, 3 or > 3 Infusions of rVIII-SingleChain to Achieve Hemostasis |
82.7; 76.7; 80.9; 12.0; 14.0; 12.6 | — |
Eligibility Criteria
Inclusion Criteria
- Diagnosis of severe hemophilia A defined as 150 exposure days (EDs) with a FVIII product
- Written informed consent for study participation obtained before undergoing any study specific procedures.
Exclusion Criteria
- Any history of or current FVIII inhibitors
- Any first order family history of FVIII inhibitors
- Use of an Investigational Medicinal Product within 30 days prior to the first rVIII-SingleChain administration.
- Administration of any cryoprecipitate, whole blood or plasma within 30 days prior to administration of rVIII-SingleChain or reference product.
- Known hypersensitivity (allergic reaction or anaphylaxis) to any FVIII product or hamster protein.
- Any known congenital or acquired coagulation disorder other than congenital FVIII deficiency.
- Platelet count 5 times (x) the upper limit of normal (ULN) at Screening.
- Subjects with serum creatinine values > 2 x ULN at Screening.
- Evidence of thrombosis, including deep vein thrombosis, stroke, pulmonary embolism, myocardial infarction and arterial embolus within 3 months prior to Day 1.
- Experienced life-threatening bleeding episode or had major surgery or an orthopedic surgical procedure during the 3 months prior to Day 1.
Data sourced from ClinicalTrials.gov (NCT01486927) and the linked publication. 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.