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| ID | Type | Description | Link |
|---|---|---|---|
| 2012-002554-23 | EudraCT Number |
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Investigate 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 using the modified Bethesda assay (Nijmegen modification). The definitions for thresholds were ≥0.6 to <5 BU/mL for a "low titre" inhibitor and ≥5 BU/mL for a "high-titre" inhibitor. | maximum 5 years (100 exposure days) |
| Measure | Description | Time Frame |
|---|---|---|
| Frequency of Spontaneous Break-through Bleeds | The annualized bleeding rate (ABR) was calculated during inhibitor-free periods for spontaneous bleeding events (BEs) during prophylactic treatment with Human cl rhFVIII | Maximum 5 years (100 exposure days) |
| Efficacy of Human-cl rhFVIII for the Treatment of Bleeds |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 5 years |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Sigurd Knaub | Octapharma | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UC Davis | Sacramento | California | 95817 | United States | ||
| All Children's Hospital |
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| ID | Title | Description |
|---|---|---|
| FG000 | Human-cl rhFVIII | Of the total number of patients that started in the study, the safety (SAF) and intent-to-treat (ITT) population received at least one treatment with Human-cl rhFVIII. Prophylactic treatment dose given to all patients in the PROPH population (all patients who received at least one administration of Human cI rhFVIII with prophylaxis documented as the reason for treatment): 20-50 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 cI rhFVIII): 20-30 IU FVIII/kg BW (minor haemorrhage), 30-40 IU FVIII/kg BW (moderate to major haemorrhage) or 40-60 IU FVIII/kg BW (major to life-threatening haemorrhage). Surgical prophylaxis dose given to the SURG population (all patients with surgeries performed under Human cI rhFVIII treatment): 25-30 IU FVIII/kg BW (minor surgeries) or 40-60 IU FVIII/kg BW (major surgeries). |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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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 | Feb 1, 2018 | Dec 21, 2020 |
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A personal efficacy assessment to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes. Efficacy was assessed using a four-point scale (excellent, good, moderate, none). |
| Maximum 5 years (100 exposure days) |
| 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). | Maximum 5 years (100 exposure days) |
| St. Petersburg |
| Florida |
| 33701 |
| United States |
| Harvard Children's Hospital Boston | Boston | Massachusetts | 02115 | United States |
| Republican Scientific Practical Center for Pediatric Oncology and Hematology | Minsk | Belarus |
| University of Alberta | Edmonton | Alberta | T6G2V2 | Canada |
| BC Children's Hospital | Vancouver | British Columbia | V6H 3V4 | Canada |
| Mc Master Children's Hospital | Hamilton | Ontario | L8S4K1 | Canada |
| Hospital for Sick Children | Toronto | Ontario | M5G 1X8 | Canada |
| Hopital Ste-Justine | Montreal | Quebec | H3T 1C5 | Canada |
| L'hôpital Côte de Nacre - CHU de Caen | Caen | France |
| Centre de traitement de l'hemophilie, Hôpital Bicêtre | Le Kremlin-Bicêtre | France |
| Hopital de la Timone | Marseille | France |
| Hôtel-Dieu de Nantes, Centre Regional de Traitement de l'hemophilie | Nantes | France |
| Hôpital Necker | Paris | France |
| CHU de Rennes - Hôpital Pontchaillou | Rennes | France |
| Hopital Trousseau - CHU Tours | Tours | France |
| Institute of Haematology and Transfusiology | Tbilisi | Georgia |
| Institut für Experimentelle Hämatologie und Transfusionsmedizin (IHT) | Bonn | Germany |
| University Hospital Frankfurt/M | Frankfurt | 60590 | Germany |
| Universitätsmedizin der Johannes-Gutenberg-Universität Mainz | Mainz | Germany |
| Kasturba Medical College, Dr. TMA Pai Hospital | Manipal | Karnataka | India |
| Sahyadri Speciality Hospital, Haematology & BMT Unit | Pune | India |
| Christian Medical College & Hospital, Dept of Haematology | Vellore | India |
| Univ. Di Perugia | Perugia | Italy |
| Centro di Referimento per le Malattie Emorragiche e Trombotiche | Torino | Italy |
| Scientific Research Institute of Mother and Child Health Care | Chisinau | Moldova |
| Centre Hospitalier Ibn Sina | Rabat | Morocco |
| University Medical School Warsaw | Warsaw | 00-576 | Poland |
| HSJ - Hospital de São João, EPE | Porto | Portugal |
| Morozovsky Children's Hospital | Moscow | Russia |
| Haemophilia Centre, University Clinical Centre | Ljubljana | Slovenia |
| Unitat d'hemofilia, Hospital Universitari Vall d'Hebron | Barcelona | Spain |
| Hospital Universitario La Paz | Madrid | Spain |
| National Children's Specialized Hospital "OHMATDET" | Kiev | Ukraine |
| Institute of Blood Pathology and Transfusion Medicine | Lviv | Ukraine |
| Cambridge University Hospital | Cambridge | CB2 0QQ | United Kingdom |
| Great Ormond Street Hospital for Children | London | WC1N3JH | United Kingdom |
| SAF Population | SAF population = study population of patients in safety analysis |
|
| ITT Population | ITT population = intent-to-treat population |
|
| PP Population | PP Population = per-protocol population |
|
| PROPH Population | PROPH population = study population of patients receiving prophylaxis |
|
| BLEED Population | BLEED population = study population of Bleeding Events (BEs) |
|
| SURG Population | SURG population = study population of patients undergoing surgery treated with Human-cl rhFVIII |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
The safety (SAF) and intent-to-treat (ITT) population consist of all patients who had data collected post-treatment with Human-cl rhFVIII (n=108).
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| ID | Title | Description |
|---|---|---|
| BG000 | Human-cl rhFVIII | The safety (SAF) and intent-to-treat (ITT) population consists all patients who had data collected post-treatment with Human-cl rhFVIII (n=108). |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Age at screening in months | Mean | Full Range | months |
| ||||||||||||||||
| Age, Continuous | Age at exposure day 1 in months | Mean | Full Range | months |
| ||||||||||||||||
| 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 Exposure Day 1 (ED1) | Mean | Full Range | kg/m2 |
| |||||||||||||||||
| Height at Exposure Day 1 (ED1) | Height at exposure day 1 in centimeters | Mean | Full Range | centimeters (cm) |
| ||||||||||||||||
| Weight at Exposure Day 1 (ED1) | Weight at exposure day 1 in kilograms | Mean | Full Range | kilograms (kg) |
| ||||||||||||||||
| Family history of inhibitors | Count of Participants | Participants |
|
| 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 using the modified Bethesda assay (Nijmegen modification). The definitions for thresholds were ≥0.6 to <5 BU/mL for a "low titre" inhibitor and ≥5 BU/mL for a "high-titre" inhibitor. | The analysis was performed for the SAF/ITT population which includes all patients who had data collected post-treatment with Human-cl rhFVIII (n=108). Of the 108 patients, 105 patients had at least one inhibitor test after exposure day (ED) 1. | Posted | Count of Participants | Participants | maximum 5 years (100 exposure days) |
|
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Frequency of Spontaneous Break-through Bleeds | The annualized bleeding rate (ABR) was calculated during inhibitor-free periods for spontaneous bleeding events (BEs) during prophylactic treatment with Human cl rhFVIII | The analysis population includes all patients who received at least one prophylactic treatment with Human-cl rhFVIII (PROPH population; n=103). Of all patients in the PROPH population, data was available on spontaneous break-through bleeds for 102 patients. | Posted | Mean | 95% Confidence Interval | No. BEs per duration (year) (ABR) | Maximum 5 years (100 exposure days) |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Efficacy of Human-cl rhFVIII for the Treatment of Bleeds | A personal efficacy assessment to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes. Efficacy was assessed using a four-point scale (excellent, good, moderate, none). | The analysis population includes patients (n=94) who received Human-cl rhFVIII for on-demand treatment of BEs (BLEED population). | Posted | Count of Units | Number of Bleeding Events | Maximum 5 years (100 exposure days) | Number of Bleeding Events | Number of Bleeding Events |
|
| |||||||||||||||||||||||||||||||||||
| 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). | The analysis population includes 24 patients that received Human-cl rhFVIII for surgical prophylaxis during a total of 26 surgeries (SURG population). Of these, 13 patients had minor surgeries and 11 patients had major surgeries. Of the total 26 surgeries, 21 had an overall efficacy assessment (an assessment was not performed for 5 surgeries). | Posted | Count of Units | Number of surgeries | Maximum 5 years (100 exposure days) | Number of surgeries | Number of surgeries |
| ||||||||||||||||||||||||||||||||||||
| Other Pre-specified | 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. | Of the 110 patients enrolled in the study, 2 were excluded because they had no treatment with Human-cl rhFVIII leaving 108 patients in the safety (SAF) and intent-to-treat (ITT) population. | Posted | Count of Participants | Participants | 5 years |
|
|
Maximum 5 years (100 exposure days)
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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/ITT Population | All patients who had data collected post-treatment with Human-cl rhFVIII (n=108). | 0 | 108 | 48 | 108 | 101 | 108 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Factor VIII inhibition | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Haematoma | Vascular disorders | Systematic Assessment |
| ||
| Tonsillitis | Infections and infestations | Systematic Assessment |
| ||
| Pyrexia | General disorders | Systematic Assessment |
| ||
| Injury | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Head injury | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Skin injury | Injury, poisoning and procedural complications | Systematic Assessment |
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| Pneumonia | Infections and infestations | Systematic Assessment |
| ||
| Type 1 diabetes mellitus | Metabolism and nutrition disorders | Systematic Assessment |
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| Bronchitis | Infections and infestations | Systematic Assessment |
| ||
| Staphylococcal infection | Infections and infestations | Systematic Assessment |
| ||
| Traumatic haematoma | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Device Issue | Product Issues | Systematic Assessment |
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| Gastroenteritis | Infections and infestations | Systematic Assessment |
| ||
| Nasopharyngitis | Infections and infestations | Systematic Assessment |
| ||
| Factor IX inhibition | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Haemorrhagic anaemia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Device related sepsis | Infections and infestations | Systematic Assessment |
| ||
| Device related infection | Infections and infestations | Systematic Assessment |
| ||
| Ileus | Gastrointestinal disorders | Systematic Assessment |
| ||
| Haemorrhage intracranial | Nervous system disorders | Systematic Assessment |
| ||
| Ear infection | Infections and infestations | Systematic Assessment |
| ||
| Influenza | Infections and infestations | Systematic Assessment |
| ||
| Mouth haemorrhage | Gastrointestinal disorders | Systematic Assessment |
| ||
| Anaemia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Haemarthrosis | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Limb injury | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Septic shock | Infections and infestations | Systematic Assessment |
| ||
| Fungal skin infection | Infections and infestations | Systematic Assessment |
| ||
| Fall | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Apnoea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Contusion | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Joint swelling | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Scrotal haematoma | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Subdural haemorrhage | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Subdural haematoma | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Lower respiratory tract infection | Infections and infestations | Systematic Assessment |
| ||
| Gastrointestinal infection | Infections and infestations | Systematic Assessment |
| ||
| Periodontitis | Infections and infestations | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | Systematic Assessment |
| ||
| Rhinitis | Infections and infestations | Systematic Assessment |
| ||
| Upper respiratory tract infection | Infections and infestations | Systematic Assessment |
| ||
| Ear infection | Infections and infestations | Systematic Assessment |
| ||
| Bronchitis | Infections and infestations | Systematic Assessment |
| ||
| Conjunctivitis | Infections and infestations | Systematic Assessment |
| ||
| Gastroenteritis | Infections and infestations | Systematic Assessment |
| ||
| Varicella | Infections and infestations | Systematic Assessment |
| ||
| Respiratory tract infection | Infections and infestations | Systematic Assessment |
| ||
| Laryngitis | Infections and infestations | Systematic Assessment |
| ||
| Pharyngitis | Infections and infestations | Systematic Assessment |
| ||
| Tonsillitis | Infections and infestations | Systematic Assessment |
| ||
| Lower respiratory tract infection | Infections and infestations | Systematic Assessment |
| ||
| Pneumonia | Infections and infestations | Systematic Assessment |
| ||
| Device related information | Infections and infestations | Systematic Assessment |
| ||
| Erythema infectiosum | Infections and infestations | Systematic Assessment |
| ||
| Influenza | Infections and infestations | Systematic Assessment |
| ||
| Viral infection | Infections and infestations | Systematic Assessment |
| ||
| Pyrexia | General disorders | Systematic Assessment |
| ||
| Factor VIII inhibition | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Anaemia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Haemorrhagic anaemia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Diarrhoea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Teething | Gastrointestinal disorders | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | Systematic Assessment |
| ||
| Constipation | Gastrointestinal disorders | Systematic Assessment |
| ||
| Toothache | Gastrointestinal disorders | Systematic Assessment |
| ||
| Stomatitis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Productive cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Rhinorrhea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Wheezing | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Dermatitis allergic | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Urticaria | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Head injury | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Mouth injury | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Pain in extremity | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Arthralgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Haemoglobin decreased | Investigations | Systematic Assessment |
| ||
| Iron deficiency | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Hypersensitivity | Immune system disorders | Systematic Assessment |
| ||
| Haematoma | Vascular 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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Sylvia Werner | Octapharma | 415 260-9577 | sylvia.werner@octapharma.com |
| Prot_002.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 1, 2019 | Dec 21, 2020 | SAP_003.pdf |
| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Title | Measurements |
|---|---|
|
|
| Number of Bleeding Events |
|
|
| Units | Counts |
|---|---|
| Participants |
|
| Number of surgeries |
|
|
|