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The purpose of the study is to collect long-term data on the inhibitor development rate of Human-cl rhFVIII in previously untreated patients with severe Hemophilia A.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Human-cl rhFVIII | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Human-cl rhFVIII | Biological |
|
| Measure | Description | Time Frame |
|---|---|---|
| Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors | The number of patients developing FVIII inhibitors was observed during the observation period by assessing inhibitor development by the modified Bethesda assay (Nijmegen modification) using congenital FVIII-deficient human plasma spiked with Human-cl rhFVIII. The definition threshold for a "positive" inhibitor was if the modified Bethesda assay resulted in a titre ≥0.6 BU/mL at any time point during the observation period. | Maximum two years |
| Measure | Description | Time Frame |
|---|---|---|
| Frequency of Spontaneous Break-through Bleeds | The annualized bleeding rate (ABR) was calculated during the time of prophylactic treatment with Human-cl rhFVIII for spontaneous bleeding events (BEs). | Maximum 2 years |
| Efficacy of Human-cl rhFVIII for the Treatment of Bleeds |
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Inclusion Criteria:
1. Patients who completed GENA-05 in accordance with the study protocol
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Sigurd Knaub, PhD | Octapharma | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UC Davis Medical Center | Sacramento | California | 95817 | United States | ||
| University of Alberta |
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| ID | Title | Description |
|---|---|---|
| FG000 | Human-cl rhFVIII | Of the total number of patients that started in the study, all those in the safety (SAF) population received at least 1 infusion of Human-cl rhFVIII. Patients who had data collected post-treatment were included in the intent-to-treat (ITT) population. Prophylactic treatment dose given to all patients in the PROPH population (all patients who received at least 1 administration of Human-cl rhFVIII with prophylaxis documented as the reason for treatment): >20 IU FVIII/kg body weight (BW). On-demand treatment of bleeding episodes (BEs) dose given to the BLEED population (all patients with bleeding episodes treated with Human-cl rhFVIII): 20-30 IU FVIII/kg BW (minor haemorrhage), 30-40 IU FVIII/kg BW (moderate to major haemorrhage) or 50-80 IU FVIII/kg BW (major to life-threatening haemorrhage). Surgical prophylaxis dose was given to the SURG population (all patients with surgeries treated with Human-cl rhFVIII): 25-30 IU FVIII/kg BW (minor surgeries); >50 IU FVIII/kg BW (major surgeries). |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
The safety (SAF) population consist of all patients who received at least one infusion of Human-cl rhFVIII (n=48).
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| ID | Title | Description |
|---|---|---|
| BG000 | Human-cl rhFVIII | The safety (SAF) population consist of all patients who received at least one infusion of Human-cl rhFVIII (n=48). |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors | The number of patients developing FVIII inhibitors was observed during the observation period by assessing inhibitor development by the modified Bethesda assay (Nijmegen modification) using congenital FVIII-deficient human plasma spiked with Human-cl rhFVIII. The definition threshold for a "positive" inhibitor was if the modified Bethesda assay resulted in a titre ≥0.6 BU/mL at any time point during the observation period. | The analysis was performed for the safety (SAF) population which includes all patients who received at least 1 infusion of Human-cl rhFVIII (N=48) | Posted | Number | 95% Confidence Interval | participants | Maximum two years |
|
Maximum 2 years
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | SAF Population | The safety (SAF) population consist of all patients who received at least one infusion of Human-cl rhFVIII (n=48). |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pneumonia | Infections and infestations | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Sylvia Werner | Octapharma | 415 260-9577 | sylvia.werner@octapharma.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 18, 2013 | Dec 21, 2020 | Prot_002.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 30, 2019 | Dec 21, 2020 | SAP_003.pdf |
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| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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A personal efficacy assessment (final outcome) to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes (BEs) at the end of a BE. Efficacy was assessed using a four-point scale (excellent, good, moderate, none) by the patient's parent(s)/legal guardian(s) together with the investigator in case of on site treatment. |
| Maximum 2 years |
| Efficacy of Human-cl rhFVIII for Surgical Prophylaxis | An overall efficacy assessment to assess the efficacy of human-cl rhFVIII in surgical prophylaxis of minor and major surgeries. The efficacy assessment was analyzed using a four-point scale (excellent, good, moderate, none). If surgeries could not be assessed due to limited data available or having taken place outside the study site, the results were classified as "not done". | Maximum 2 years |
| The Occurrence of Any Adverse Event (AE) | The frequency of AEs, as monitored throughout the whole study by the number of patients with at least one adverse event occurrence. | Maximum 2 years |
| Edmonton |
| Alberta |
| Canada |
| BC Children's Hospital | Vancouver | British Columbia | V6H 3V4 | Canada |
| McMaster Children's Hospital | Hamilton | Ontario | L8S4K1 | Canada |
| Hospital for Sick Children | Toronto | Canada |
| Hopital de la Timone | Marseille | France |
| Hôpital Kremlin Bicètre | Paris | France |
| Institute of Hematology and Transfusiology | Tbilisi | Georgia |
| Sahyadri Speciality Hospital | Pune | 411004 | India |
| Christian Medical College | Vellore | 632004 | India |
| IMSP Mother and Child Institute | Chisinau | Moldova |
| University Medical School | Warsaw | Poland |
| The National Children Specialized Hospital "OHMATDET" | Kiev | Ukraine |
| Danylo Halytsky Lviv National Medical University | Lviv | Ukraine |
| Great Ormond Street Hospital for Children | London | WC1N 3JH | United Kingdom |
| Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Body Mass Index (BMI) at screening | Mean | Full Range | kg/m^2 |
|
| Height at screening | Mean | Full Range | Centimeters (cm) |
|
| Weight at screening | Mean | Full Range | Kilograms (kg) |
|
| Family history of inhibitors | Count of Participants | Participants |
|
|
|
| Secondary | Frequency of Spontaneous Break-through Bleeds | The annualized bleeding rate (ABR) was calculated during the time of prophylactic treatment with Human-cl rhFVIII for spontaneous bleeding events (BEs). | The analysis population includes all patients in PROPH population who received at least one prophylactic treatment with Human-cl rhFVIII (n=47). | Posted | Mean | 95% Confidence Interval | Events/year (ABR) | Maximum 2 years |
|
|
|
| Secondary | Efficacy of Human-cl rhFVIII for the Treatment of Bleeds | A personal efficacy assessment (final outcome) to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes (BEs) at the end of a BE. Efficacy was assessed using a four-point scale (excellent, good, moderate, none) by the patient's parent(s)/legal guardian(s) together with the investigator in case of on site treatment. | The analysis population included all patients who received on-demand treatment with Human-cl rhFVIII for bleeding episodes (BLEED population; n=29). | Posted | Count of Units | Bleeding episodes | Maximum 2 years | Bleeding episodes | Bleeding episodes |
|
|
|
| Secondary | Efficacy of Human-cl rhFVIII for Surgical Prophylaxis | An overall efficacy assessment to assess the efficacy of human-cl rhFVIII in surgical prophylaxis of minor and major surgeries. The efficacy assessment was analyzed using a four-point scale (excellent, good, moderate, none). If surgeries could not be assessed due to limited data available or having taken place outside the study site, the results were classified as "not done". | The analysis population includes 3 patients that received Human-cl rhFVIII for surgical prophylaxis during a total of 4 surgeries (SURG population). Of these, 1 patient had two minor surgeries and 2 patients had one major surgery each. | Posted | Count of Units | Surgeries | Maximum 2 years | Surgeries | Surgeries |
|
|
|
| Secondary | The Occurrence of Any Adverse Event (AE) | The frequency of AEs, as monitored throughout the whole study by the number of patients with at least one adverse event occurrence. | The analysis was performed for the safety (SAF) population which includes all patients who received at least 1 infusion of Human-cl rhFVIII (n=48). | Posted | Count of Participants | Participants | Maximum 2 years |
|
|
|
| 0 |
| 48 |
| 5 |
| 48 |
| 29 |
| 48 |
| Varicella | Infections and infestations | Systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Haemorrhage | Vascular disorders | Systematic Assessment |
|
| Gastritis | Gastrointestinal disorders | Systematic Assessment |
|
| Cellulitis | Infections and infestations | Systematic Assessment |
|
| Neuroblastoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment |
|
| Varicella | Infections and infestations | Systematic Assessment |
|
| Bronchitis | Infections and infestations | Systematic Assessment |
|
| Ear infection | Infections and infestations | Systematic Assessment |
|
| Pharyngitis | Infections and infestations | Systematic Assessment |
|
| Tonsillitis | Infections and infestations | Systematic Assessment |
|
| Otitis media | Infections and infestations | Systematic Assessment |
|
| Pneumonia | Infections and infestations | Systematic Assessment |
|
| Pulpitis dental | Infections and infestations | Systematic Assessment |
|
| Respiratory tract infection viral | Infections and infestations | Systematic Assessment |
|
| Tracheitis | Infections and infestations | Systematic Assessment |
|
| Pyrexia | General disorders | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
|
| Anaemia | Blood and lymphatic system disorders | Systematic Assessment |
|
Octapharma agreements may vary with individual investigators, but will not prohibit any investigator from publishing. Octapharma supports the publication of results from all centers of a multi-center trial but requests that reports based on single-site data not precede the primary publication of the entire clinical trial. Octapharma also reserves the right to review data prior to publishing and provide comments/changes within a certain time period.
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| None |
|
| Not done |
|
| Moderate |
|
| None |
|
| Not done |
|
| Title | Measurements |
|---|---|
|
| Temporally related adverse event |
|
| Death |
|
| AE leading to permanent discontinuation |
|