Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A
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
1 other identifier
interventional
58
9 countries
30
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started May 2015
Typical duration for phase_3
30 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 30, 2014
CompletedFirst Posted
Study publicly available on registry
October 6, 2014
CompletedStudy Start
First participant enrolled
May 1, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 1, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
September 1, 2018
CompletedResults Posted
Study results publicly available
December 3, 2019
CompletedJanuary 19, 2021
December 1, 2020
3.3 years
September 30, 2014
August 28, 2019
December 21, 2020
Conditions
Outcome Measures
Primary Outcomes (1)
Annualized Total Bleeding Rate of Individually Tailored Prophylaxis
Total annualized bleeding rate (ABR) of individually tailored prophylaxis (GENA-21b) compared to historical bleeding rate in patients having received on-demand treatment (GENA-01) with Human-cl rhFVIII
6 months
Secondary Outcomes (12)
Annualized Spontaneous Bleeding Rate of Individually Tailored Prophylaxis
6 months
Annualized Total Bleeding Rate in Patients With 2x/Week (or Less) Prophylaxis
6 months
Median Prophylactic Dosing Interval
6 months
Mean Prophylactic Dosing Interval
6 months
AUC Divided by the Dose (AUCnorm) of Human-cl rhFVIII
Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
- +7 more secondary outcomes
Study Arms (1)
Human-cl rhFVIII
EXPERIMENTALInterventions
Eligibility Criteria
You may qualify if:
- 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)
You may not qualify if:
- Any coagulation disorder other than Haemophilia A
- Present of past FVIII inhibitor activity
- Severe liver or kidney disease
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Octapharmalead
Study Sites (30)
Octapharma Research Site
Sacramento, California, 95817, United States
Octapharma Research Site
Aurora, Colorado, 80045, United States
Octapharma Research Site
Washington D.C., District of Columbia, 20057, United States
Octapharma Research Site
Miami, Florida, 33136, United States
Octapharma Research Site
Chicago, Illinois, 60612, United States
Octapharma Research Site
Indianapolis, Indiana, 46260, United States
Octapharma Research Site
Memphis, Tennessee, 38163, United States
Octapharma Research Site
Houston, Texas, 77030, United States
Octapharma Research Site
Salt Lake City, Utah, 84112, United States
Octapharma Research Site
Edmonton, Alberta, T6G1C9, Canada
Octapharma Research Site
St. John's, Newfoundland and Labrador, Canada
McMaster University
Hamilton, Ontario, L8N 4K1, Canada
University Hospital Centre Zagreb
Zagreb, Croatia
Helsinki University Hospital
Helsinki, 00290, Finland
Centre Régional de Traitement de l'Hémophilie
Bron, 69677, France
CHU Estaing
Clermont-Ferrand, France
Centre Hospitalier Universitaire Félix Guyon
La Réunion, 97400, France
Centre Régional de Traitement de l'hémophilie
Nantes, 44093, France
Hôpital Purpan - Centre de Traitment Regional de l'Hemophilie Pole
Toulouse, 31059, France
Nagoya University Hospital
Nagoya, Aichi-ken, Japan
Hospital of the Univ of Occupational and Environmental Health
Kitakyushu, Fukuoka, Japan
St. Marianna Univ School of Medicine Hospital
Kawasaki, Kanagawa, Japan
Nara Medical University Hospital
Kashihara, Nara, Japan
Gunma University Hospital
Maebashi, Japan
Osaka National Hospital
Osaka, Japan
Ogikubo Hospital
Tokyo, Japan
Teikyo University Hospital
Tokyo, Japan
University Medical Center Groningen
Groningen, 9713, Netherlands
PHI Institute of Transfusion Medicine of Republic of Macedonia
Skopje, North Macedonia
University Medical Centre Ljubljana
Ljubljana, Slovenia
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Sylvia Werner
- Organization
- Octapharma
Study Officials
- PRINCIPAL INVESTIGATOR
Craig M Kessler, MD
Georgetown University
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- PREVENTION
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 30, 2014
First Posted
October 6, 2014
Study Start
May 1, 2015
Primary Completion
September 1, 2018
Study Completion
September 1, 2018
Last Updated
January 19, 2021
Results First Posted
December 3, 2019
Record last verified: 2020-12