Phase 3 N=58 Prevention

Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A

Severe Haemophilia A

Bottom Line

View on ClinicalTrials.gov: NCT02256917 ↗

Enrolled (actual)

Serious AEs

6.9%

Results posted

Dec 2019

Primary outcome: Primary: Annualized Total Bleeding Rate of Individually Tailored Prophylaxis — 4.67; 58.08 Bleeding events per year (ABR) — p=<0.0001

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Human cl rhFVIII (Biological)
Age: Adult, Older Adult · 18+ yrs
Sex: Male
Sponsor: Octapharma
Primary completion: Sep 2018

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	—

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.

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

View full record on ClinicalTrials.gov →

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.