Clinical trial NCT02256917: Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A
Official title: Prospective, Open-label, Multi-centre Phase 3b Study to Assess the Efficacy and Safety of Personalized Prophylaxis With Human-cl rhFVIII in Previously Treated Adult Patients With Severe Haemophilia A
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Severe Haemophilia A
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2014-10-06
Enrollment: 58
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

Brief 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 \< 1%)
* Male patients \>= 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

Primary outcomes:
measure: Annualized Total Bleeding Rate of Individually Tailored Prophylaxis
timeFrame: 6 months
Secondary outcomes:
measure: Annualized Spontaneous Bleeding Rate of Individually Tailored Prophylaxis
timeFrame: 6 months
measure: Annualized Total Bleeding Rate in Patients With 2x/Week (or Less) Prophylaxis
timeFrame: 6 months
measure: Median Prophylactic Dosing Interval
timeFrame: 6 months
measure: Mean Prophylactic Dosing Interval
timeFrame: 6 months
measure: AUC Divided by the Dose (AUCnorm) of Human-cl rhFVIII
timeFrame: Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
measure: In-vivo Recovery (IVR) of Human-cl rhFVIII
timeFrame: Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
measure: Half Life (t1/2) of Human-cl rhFVIII
timeFrame: Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
measure: Mean Residence Time (MRT) of Human-cl rhFVIII
timeFrame: Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
measure: Clearance (CL) of Human-cl rhFVIII
timeFrame: Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
measure: Volume of Distribution at Steady State (Vss) of Human-cl rhFVIII
timeFrame: Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
measure: Usage of Human-cl rhFVIII (FVIII IU/kg BW Per Week Per Patient)
timeFrame: 6 months
measure: Number of Patients With Adverse Events (AEs)
timeFrame: At each study visit over the study duration (7-9 months)
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: Octapharma Research Site
state: California
city: Aurora
country: United States
facility: Octapharma Research Site
state: Colorado
city: Washington
country: United States
facility: Octapharma Research Site
state: District of Columbia
city: Miami
country: United States
facility: Octapharma Research Site
state: Florida
city: Chicago
country: United States
facility: Octapharma Research Site
state: Illinois
city: Indianapolis
country: United States
facility: Octapharma Research Site
state: Indiana
city: Memphis
country: United States
facility: Octapharma Research Site
state: Tennessee
city: Houston
country: United States
facility: Octapharma Research Site
state: Texas
city: Salt Lake City
country: United States
facility: Octapharma Research Site
state: Utah
city: Edmonton
country: Canada
facility: Octapharma Research Site
state: Alberta
city: St. John's
country: Canada
facility: Octapharma Research Site
state: Newfoundland and Labrador
city: Hamilton
country: Canada
facility: McMaster University
state: Ontario
city: Zagreb
country: Croatia
facility: University Hospital Centre Zagreb
city: Helsinki
country: Finland
facility: Helsinki University Hospital
city: Bron
country: France
facility: Centre Régional de Traitement de l'Hémophilie
city: Clermont-Ferrand
country: France
facility: CHU Estaing
city: La Réunion
country: France
facility: Centre Hospitalier Universitaire Félix Guyon
city: Nantes
country: France
facility: Centre Régional de Traitement de l'hémophilie
city: Toulouse
country: France
facility: Hôpital Purpan - Centre de Traitment Regional de l'Hemophilie Pole
city: Nagoya
country: Japan
facility: Nagoya University Hospital
state: Aichi
city: Kitakyushu
country: Japan
facility: Hospital of the Univ of Occupational and Environmental Health
state: Fukuoka
city: Kawasaki
country: Japan
facility: St. Marianna Univ School of Medicine Hospital
state: Kanagawa
city: Kashihara
country: Japan
facility: Nara Medical University Hospital
state: Nara
city: Maebashi
country: Japan
facility: Gunma University Hospital
city: Osaka
country: Japan
facility: Osaka National Hospital
city: Tokyo
country: Japan
facility: Ogikubo Hospital
city: Tokyo
country: Japan
facility: Teikyo University Hospital
city: Groningen
country: Netherlands
facility: University Medical Center Groningen
city: Skopje
country: North Macedonia
facility: PHI Institute of Transfusion Medicine of Republic of Macedonia
city: Ljubljana
country: Slovenia
facility: University Medical Centre Ljubljana
Study officials:
affiliation: Georgetown University
name: Craig M Kessler, MD
role: PRINCIPAL_INVESTIGATOR
References: