Study to collect data to further monitor whether the treatment triggers an immune reaction (i.e. development of antibodies, so called “inhibitors” directed against factor 8), to further investigate how well Human-cl rhFVIII prevents and stops bleeding episodes, and to monitor if it is safe and well tolerated in the treatment on a long-term basis.

Extension Study for Patients who completed GENA-05 (NuProtect) – to Investigate Immunogenicity, Efficacy and Safety of Treatment with Human-cl rhFVIII

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2013-003997-28-GB

Enrollment

100

Registered

2013-11-20

Start date

2014-01-15

Completion date

Unknown

Last updated

2020-07-20

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: 16.1 Level: LLT Classification code 10018938 Term: Haemophilia A (Factor VIII) System Organ Class: 100000004850

Interventions

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

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: In order to qualify for study enrolment, the following criteria must be fulfilled before study entry: 1. patients who completed GENA-05 in accordance with the study protocol 2. Voluntarily given, fully informed written and signed consent obtained before any study-related procedures are conducted (obtained from the patient’s parent/legal guardian) Are the trial subjects under 18? yes Number of subjects for this age range: 100 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range 0 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0

Exclusion criteria

Exclusion criteria: Patients will not be included if any of the following exclusion criteria are met: 1. Severe liver or kidney disease (alanine amino transferase (ALT) or aspartate transaminase (AST) levels >5 times of upper limit of normal, creatinine >120 µmol/L); 2. Concomitant treatment with any systemic immunosuppressive drug; 3. Other FVIII concentrate than Human-cl rhFVIII was received between completion visit of GENA-05 and start of GENA-15 (except emergency cases).

Design outcomes

Primary

Measure	Time frame
Main Objective: • To investigate the immunogenicity of Human-cl rhFVIII in patients who completed GENA-05 in accordance with the study protocol • To assess the efficacy of Human-cl rhFVIII during prophylactic treatment (based on the frequency of spontaneous break-through bleeds) • To assess the efficacy of Human-cl rhFVIII during treatment of bleeds • To assess the efficacy of Human-cl rhFVIII in surgical prophylaxis • To assess the safety and tolerability of Human-cl rhFVIII;Secondary Objective: not applicable;Primary end point(s): Immunogenicity of Human-cl rhFVIII is the primary endpoint. Inhibitor activity will be determined by the modified Bethesda assay (Nijmegen modification), using congenital FVIII-deficient human plasma, spiked with Human-cl rhFVIII. In case of a positive inhibitor result, an inhibitor retesting, using a second separately drawn sample, should be performed. A FVIII inhibitor is defined as “positive”, if the retesting confirms the positive result, otherwise the result is considered as “negative”.;Timepoint(s) of evaluation of this end point: • At Screening Visit, which will most likely be the same sampling time-point as the completion visit of GENA-05 • Once every 6 months in the course of the follow-up visits • At study completion • Any time in the case of a suspicion of inhibitor development.	—

Measure

Time frame

Main Objective: • To investigate the immunogenicity of Human-cl rhFVIII in patients who completed GENA-05 in accordance with the study protocol • To assess the efficacy of Human-cl rhFVIII during prophylactic treatment (based on the frequency of spontaneous break-through bleeds) • To assess the efficacy of Human-cl rhFVIII during treatment of bleeds • To assess the efficacy of Human-cl rhFVIII in surgical prophylaxis • To assess the safety and tolerability of Human-cl rhFVIII;Secondary Objective: not applicable;Primary end point(s): Immunogenicity of Human-cl rhFVIII is the primary endpoint. Inhibitor activity will be determined by the modified Bethesda assay (Nijmegen modification), using congenital FVIII-deficient human plasma, spiked with Human-cl rhFVIII. In case of a positive inhibitor result, an inhibitor retesting, using a second separately drawn sample, should be performed. A FVIII inhibitor is defined as “positive”, if the retesting confirms the positive result, otherwise the result is considered as “negative”.;Timepoint(s) of evaluation of this end point: • At Screening Visit, which will most likely be the same sampling time-point as the completion visit of GENA-05 • Once every 6 months in the course of the follow-up visits • At study completion • Any time in the case of a suspicion of inhibitor development.

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Secondary

Measure	Time frame
Secondary end point(s): 1. Efficacy: a.Efficacy of prophylactic treatment The efficacy of Human-cl rhFVIII in the prophylactic treatment will be investigated by calculating the frequency of spontaneous break-through bleeds under prophylactic treatment. Study drug consumption data (FVIII IU/kg per month, per year) per patient and in total will be evaluated. The dates and times of study drug infusions, the details of dose(s), and the product batch numbers used for the prophylactic treatment will be documented. b. Efficacy of treatment of bleeds The efficacy of Human-cl rhFVIII in the treatment of bleeds will be investigated by using a 4-point ordinal haemostatic efficacy scale. Details of the bleed, the amount of Human-cl rhFVIII needed and the number of injections necessary to stop the bleed will be documented. c. Efficacy of surgical prophylaxis In surgical procedures, the following parameters will be documented: • One overall efficacy assessment (taking into account the intra- and post-operative assessment) after the end of surgical prophylactic treatment phase, agreed upon between the surgeon and the haematologist. • Average and maximum expected estimated blood loss, compared to the actual estimated blood loss • Details on surgical procedure: location, severity, type, expected and actual duration • Pre-, intra-, and post-operative FVIII plasma levels, if appropriate • Details of administered dose(s) of Human-cl rhFVIII given pre-, intra- and/or postoperatively including dates, times and batch numbers • Details on concomitantly administered drugs, including all blood and blood product transfusions, excluding standard anaesthetic drugs • Details on all wound haematomas in terms of capturing, analysing, and reporting these, including any need for surgical evacuation • Outcome of the intervention, described by means of a brief narrative. 2.Safety Safety and tolerability will be assessed by monitoring vital signs, standard laboratory parameters, and b	—

Measure

Time frame

Secondary end point(s): 1. Efficacy: a.Efficacy of prophylactic treatment The efficacy of Human-cl rhFVIII in the prophylactic treatment will be investigated by calculating the frequency of spontaneous break-through bleeds under prophylactic treatment. Study drug consumption data (FVIII IU/kg per month, per year) per patient and in total will be evaluated. The dates and times of study drug infusions, the details of dose(s), and the product batch numbers used for the prophylactic treatment will be documented. b. Efficacy of treatment of bleeds The efficacy of Human-cl rhFVIII in the treatment of bleeds will be investigated by using a 4-point ordinal haemostatic efficacy scale. Details of the bleed, the amount of Human-cl rhFVIII needed and the number of injections necessary to stop the bleed will be documented. c. Efficacy of surgical prophylaxis In surgical procedures, the following parameters will be documented: • One overall efficacy assessment (taking into account the intra- and post-operative assessment) after the end of surgical prophylactic treatment phase, agreed upon between the surgeon and the haematologist. • Average and maximum expected estimated blood loss, compared to the actual estimated blood loss • Details on surgical procedure: location, severity, type, expected and actual duration • Pre-, intra-, and post-operative FVIII plasma levels, if appropriate • Details of administered dose(s) of Human-cl rhFVIII given pre-, intra- and/or postoperatively including dates, times and batch numbers • Details on concomitantly administered drugs, including all blood and blood product transfusions, excluding standard anaesthetic drugs • Details on all wound haematomas in terms of capturing, analysing, and reporting these, including any need for surgical evacuation • Outcome of the intervention, described by means of a brief narrative. 2.Safety Safety and tolerability will be assessed by monitoring vital signs, standard laboratory parameters, and b

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Countries

Brazil, Canada, Colombia, France, Georgia, Germany, India, Moldova, Republic of, Morocco, Poland, Russian Federation, Spain, Ukraine, United Kingdom, United States, Venezuela, Bolivarian Republic of

Contacts

Public ContactRegulatory Affairs

Inventiv Health Clinical UK Ltd

Regopseurope@inventivhealth.com00441628 408401

Outcome results

None listed

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