A study of the efficacy and safety of individually tailored prophylaxis with Human-cl rhFVIII in previously treated adult patients with severe haemophilia A

Prospective, open-label, multicentre phase 3b study to assess the efficacy and safety of individually tailored prophylaxis with Human-cl rhFVIII in previously treated adult patients with severe haemophilia A

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2013-001556-35-GB

Enrollment

Registered

2013-05-30

Start date

2013-07-26

Completion date

Unknown

Last updated

2015-07-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Severe haemophilia A MedDRA version: 16.0 Level: LLT Classification code 10018938 Term: Haemophilia A (Factor VIII) System Organ Class: 100000004850

Interventions

Product Name: Human cell-line derived recombinant human factor VIII Product Code: Human-cl rhFVIII Pharmaceutical Form: Powder and solvent for solution for injection INN or Proposed INN: simoctocog al

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: (a) Severe haemophilia A (FVIII:C 200/µL) (f) HIV-negative according to medical history; if positive, viral load =65 years) yes F.1.3.1 Number of subjects for this age range 5

Exclusion criteria

Exclusion criteria: (a) Any coagulation disorder other than haemophilia A (b) Present or past FVIII inhibitor activity (= 0.6 BU) according to medical history (c) Severe liver or kidney disease (ALT and AST levels > 5 times of upper limit of normal, creatinine > 120 µmol/L) (d) Treatment with any investigational medicinal product (IMP) except FVIII IMP within 14 days prior to the screening visit

Design outcomes

Primary

Measure	Time frame
Main Objective: To compare the annualized total bleeding rate of individually tailored prophylaxis with the historical bleeding rate observed in patients having received on-demand treatment with Human-cl rhFVIII from study GENA-01;Secondary Objective: 1. To compare the annualized spontaneous bleeding rate of individually tailored prophylaxis with the historical bleeding rate observed in patients having received on-demand treatment with Human-cl rhFVIII 2. To compare the annualized total bleeding rate in patients with 2x/week (or less) prophylaxis with the historical bleeding rate observed in patients having received on-demand treatment with Human-cl rhFVIII To assess: 3. Median prophylactic dosing interval 4. Human-cl rhFVIII consumption data 5. PK of Human-cl rhFVIII in terms of FVIII:C 6. Safety of Human-cl rhFVIII Additional objectives: To assess: 1. Thrombin generation assay (TGA) in terms of its usefulness in individualizing therapy for patients on prophylaxis 2. Clinical efficacy of Human-cl rhFVIII in the treatment of breakthrough bleeding episodes (BEs) 3. Clinical efficacy of Human-cl rhFVIII in surgical prophylaxis 4. Correlation of vWF antigen concentration and half-life of Human-cl rhFVIII;Primary end point(s): Reduction of the annualized total bleeding rate observed in the GENA-01 study (58.1 total bleeding episodes per patient per year) by 50% during individually tailored prophylaxis;Timepoint(s) of evaluation of this end point: At the end of the study	—

Measure

Time frame

Main Objective: To compare the annualized total bleeding rate of individually tailored prophylaxis with the historical bleeding rate observed in patients having received on-demand treatment with Human-cl rhFVIII from study GENA-01;Secondary Objective: 1. To compare the annualized spontaneous bleeding rate of individually tailored prophylaxis with the historical bleeding rate observed in patients having received on-demand treatment with Human-cl rhFVIII 2. To compare the annualized total bleeding rate in patients with 2x/week (or less) prophylaxis with the historical bleeding rate observed in patients having received on-demand treatment with Human-cl rhFVIII To assess: 3. Median prophylactic dosing interval 4. Human-cl rhFVIII consumption data 5. PK of Human-cl rhFVIII in terms of FVIII:C 6. Safety of Human-cl rhFVIII Additional objectives: To assess: 1. Thrombin generation assay (TGA) in terms of its usefulness in individualizing therapy for patients on prophylaxis 2. Clinical efficacy of Human-cl rhFVIII in the treatment of breakthrough bleeding episodes (BEs) 3. Clinical efficacy of Human-cl rhFVIII in surgical prophylaxis 4. Correlation of vWF antigen concentration and half-life of Human-cl rhFVIII;Primary end point(s): Reduction of the annualized total bleeding rate observed in the GENA-01 study (58.1 total bleeding episodes per patient per year) by 50% during individually tailored prophylaxis;Timepoint(s) of evaluation of this end point: At the end of the study

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Secondary

Measure	Time frame
Secondary end point(s): 1. Reduction of the annualized spontaneous bleeding rate observed in the GENA- 01study (38.5 spontaneous bleeding episodes per patient per year) by 50% during individually tailored prophylaxis 2. Reduction of the annualized bleeding rate observed in GENA-01 by 50% in patients with 2x/week prophylaxis or less 3. Median prophylactic dosing interval during individually tailored prophylaxis 4. Human-cl rhFVIII consumption data (FVIII IU/kg per month per patient) during individually tailored prophylaxis 5. Safety and tolerability of Human-cl rhFVIII by monitoring adverse events (AEs) throughout the study;Timepoint(s) of evaluation of this end point: All secondary endpoints will be evaluated at the end of the study with the exception of the 5th secondary endpoint, Safety and tolerability of Human-cl rhFVIII by monitoring adverse events (AEs), which will take place throughout the study	—

Measure

Time frame

Secondary end point(s): 1. Reduction of the annualized spontaneous bleeding rate observed in the GENA- 01study (38.5 spontaneous bleeding episodes per patient per year) by 50% during individually tailored prophylaxis 2. Reduction of the annualized bleeding rate observed in GENA-01 by 50% in patients with 2x/week prophylaxis or less 3. Median prophylactic dosing interval during individually tailored prophylaxis 4. Human-cl rhFVIII consumption data (FVIII IU/kg per month per patient) during individually tailored prophylaxis 5. Safety and tolerability of Human-cl rhFVIII by monitoring adverse events (AEs) throughout the study;Timepoint(s) of evaluation of this end point: All secondary endpoints will be evaluated at the end of the study with the exception of the 5th secondary endpoint, Safety and tolerability of Human-cl rhFVIII by monitoring adverse events (AEs), which will take place throughout the study

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Countries

Austria, Bulgaria, Germany, Hungary, Poland, Slovakia, Spain, United Kingdom

Contacts

Public ContactJohann Bichler

Octapharma AG

johann.bichler@octapharma.ch+4155451 21 77

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026