Phase 3
Completed N=58
Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A
Severe Haemophilia A
Source: ClinicalTrials.gov NCT02256917 ↗
Enrolled (actual)
58
Serious AEs
6.9%
Results posted
Dec 2019
Primary outcomePrimary: Annualized Total Bleeding Rate of Individually Tailored Prophylaxis — 4.67; 58.08 Bleeding events per year (ABR) — p=<0.0001
◆ 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 This can indicate results are unpublished — a known publication-bias signal.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 rationale of this study is to further fine-tune and individualize prophylactic treatment of patients with severe Haemophilia A with the goal of keeping the trough FVIII level above 1% between doses. Because trough FVIII levels are likely to be important predictors of the efficacy of prophylaxis, the focus of this study is on pharmacokinetic (PK) data.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Annualized Total Bleeding Rate of Individually Tailored Prophylaxis |
4.67; 58.08 | <0.0001 sig |
| SECONDARY Annualized Spontaneous Bleeding Rate of Individually Tailored Prophylaxis |
2.98; 38.46 | — |
| SECONDARY Annualized Total Bleeding Rate in Patients With 2x/Week (or Less) Prophylaxis |
4.82; 58.08 | — |
| SECONDARY Median Prophylactic Dosing Interval |
83.9 | — |
| SECONDARY Mean Prophylactic Dosing Interval |
83.0 | — |
| SECONDARY AUC Divided by the Dose (AUCnorm) of Human-cl rhFVIII |
0.302 | — |
| SECONDARY In-vivo Recovery (IVR) of Human-cl rhFVIII |
1.775 | — |
| SECONDARY Half Life (t1/2) of Human-cl rhFVIII |
15.725 | — |
| SECONDARY Mean Residence Time (MRT) of Human-cl rhFVIII |
20.762 | — |
| SECONDARY Clearance (CL) of Human-cl rhFVIII |
3.859 | — |
| SECONDARY Volume of Distribution at Steady State (Vss) of Human-cl rhFVIII |
72.901 | — |
| SECONDARY Usage of Human-cl rhFVIII (FVIII IU/kg BW Per Week Per Patient) |
83.7 | — |
| SECONDARY Number of Patients With Adverse Events (AEs) |
34; 24 | — |
Eligibility Criteria
Inclusion Criteria
- Severe Haemophilia A (FVIII: C = 18 years of age
- Previous treatment with a FVIII concentrate for at least 150 EDs
- Good documentation regarding dosing and bleeding frequency in the 6 months preceding study start
- Immunocompetence (CD4+ count > 200/uL)
Exclusion Criteria
- Any coagulation disorder other than Haemophilia A
- Present of past FVIII inhibitor activity
- Severe liver or kidney disease
Data sourced from ClinicalTrials.gov (NCT02256917). 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.