The main purpose of this study is to investigate the efficacy and safety of human-cl rhFVIII during prophylaxis but also if the patient needs treatment of spontaneous or traumatic bleeding episodes, and prior to and during or after surgery.

Clinical Study to Investigate the Efficacy, Safety, and Immunogenicity of human-cl rhFVIII in Previously Treated Patients With Severe Haemophilia A

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2009-011055-43-DE

Enrollment

Registered

2009-06-25

Start date

2009-10-22

Completion date

Unknown

Last updated

2013-02-11

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

Conditions

Severe haemophilia A (FVIII:C <=1%) MedDRA version: 13.1 Level: LLT Classification code 10018937 Term: Haemophilia A System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: human-cl rhFVIII Product Code: human-cl rhFVIII Pharmaceutical Form: Powder and solvent for solution for infusion INN or Proposed INN: simoctocog alfa Other descriptive name: Human Cell

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: Severe haemophilia A (FVIII:C less/equal 1%).· Male subjects greater/equal 12 years of age.· Previously treated with FVIII concentrate, at least 150 EDs.· Immunocompetent (CD4+ count >200/µL).· Negative for anti-HIV; if positive, viral load =65 years) yes F.1.3.1 Number of subjects for this age range 2

Exclusion criteria

Exclusion criteria: Other coagulation disorder than haemophilia A.· Present or past FVIII inhibitor activity (>0.6 BU).· Severe liver or kidney disease (ALAT and ASAT levels >5 times of upper limit of normal, creatinine >120 µmol/L).· Receiving or scheduled to receive immuno-modulating drugs (other than anti-retroviral chemotherapy) such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs.· Participation in another interventional clinical study currently or during the past month. · Participation in any other study with human-cl rhFVIII.

Design outcomes

Primary

Measure	Time frame
Main Objective: To determine in previously treated subjects suffering from severe haemophilia A the efficacy of human-cl rhFVIII during prophylactic treatment, in the treatment of bleeding episodes and in surgical prophylaxis.;Secondary Objective: To calculate the incremental recovery of FVIII:C for human-cl rhFVIII.· To investigate the immunogenic potential of human-cl rhFVIII.· To assess the safety of human-cl rhFVIII.;Primary end point(s): Prophylactic treatments: •Overall efficacy assessment after a total of at least 50 EDs per subject at the end of the study at 6 months. •The frequency of bleeds under prophylactic treatment will be calculated. •Study drug consumption data (FVIII IU/kg per month, per year) per subject and in total will be evaluated. Treatment of bleeding episodes: The proportion of bleeding episodes successfully treated with human-cl rhFVIII will be evaluated for all BEs as well as for BEs of different severity. “Successfully treated” are all “excellent” and “good” efficacy ratings of treated BEs. Surgical prophylaxis: Overall efficacy assessment (taking the intra- and post operative assessment into account) after the end of the surgical prophylactic treatment phase by the surgeon and haematologist.;Timepoint(s) of evaluation of this end point: end of the study at 6 months	—

Measure

Time frame

Main Objective: To determine in previously treated subjects suffering from severe haemophilia A the efficacy of human-cl rhFVIII during prophylactic treatment, in the treatment of bleeding episodes and in surgical prophylaxis.;Secondary Objective: To calculate the incremental recovery of FVIII:C for human-cl rhFVIII.· To investigate the immunogenic potential of human-cl rhFVIII.· To assess the safety of human-cl rhFVIII.;Primary end point(s): Prophylactic treatments: •Overall efficacy assessment after a total of at least 50 EDs per subject at the end of the study at 6 months. •The frequency of bleeds under prophylactic treatment will be calculated. •Study drug consumption data (FVIII IU/kg per month, per year) per subject and in total will be evaluated. Treatment of bleeding episodes: The proportion of bleeding episodes successfully treated with human-cl rhFVIII will be evaluated for all BEs as well as for BEs of different severity. “Successfully treated” are all “excellent” and “good” efficacy ratings of treated BEs. Surgical prophylaxis: Overall efficacy assessment (taking the intra- and post operative assessment into account) after the end of the surgical prophylactic treatment phase by the surgeon and haematologist.;Timepoint(s) of evaluation of this end point: end of the study at 6 months

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Secondary

Measure	Time frame
Secondary end point(s): Calculate the incremental recorvery of FVIII:C for human-cl rhFVIII. Investigate the immunogenic potential of human-cl rhFVIII. Assess the safety of human-cl rhFVIII.;Timepoint(s) of evaluation of this end point: Recovery: Will be determined for each patient at the beginning of the study, after 3 months and at study completion after 6 months. Immunogenic potential: Inhibitor activity and anti-rhFVIII antibodies will be determined at screening, prior to the first and second infusion of human-cl rhFVIII, after 10 to 15 EDs, and prior to infusion human-cl rhFVIII at the 3-month and 6-month visit. Safety: Will be assessed by monitoring vital signs, routine laboratory parameters at screening, on the day of first infusion, and after 3 and 6 months (completion visit), and by monitoring adverse events throughout the study.	—

Measure

Time frame

Secondary end point(s): Calculate the incremental recorvery of FVIII:C for human-cl rhFVIII. Investigate the immunogenic potential of human-cl rhFVIII. Assess the safety of human-cl rhFVIII.;Timepoint(s) of evaluation of this end point: Recovery: Will be determined for each patient at the beginning of the study, after 3 months and at study completion after 6 months. Immunogenic potential: Inhibitor activity and anti-rhFVIII antibodies will be determined at screening, prior to the first and second infusion of human-cl rhFVIII, after 10 to 15 EDs, and prior to infusion human-cl rhFVIII at the 3-month and 6-month visit. Safety: Will be assessed by monitoring vital signs, routine laboratory parameters at screening, on the day of first infusion, and after 3 and 6 months (completion visit), and by monitoring adverse events throughout the study.

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Countries

Austria, Germany, United Kingdom

Contacts

Public ContactJohann Bichler

Octapharma AG

johann.bichler@octapharma.ch+41(0)554512177

Outcome results

None listed

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