Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2017-000065-73 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Bioverativ Therapeutics Inc. | INDUSTRY |
Not provided
Not provided
Not provided
The primary purpose of this study is to describe the outcome of Immune Tolerance Induction (ITI) treatment performed with rFVIIIFc within a timeframe of 60 weeks in patients with haemophilia A who have failed previous attempts at tolerization.
This is an open-label, single-arm, interventional multi-center study designed to explore ITI performed with recombinant coagulation factor VIII Fc fusion protein (rFVIIIFc) within a timeframe of 60 weeks in patients with severe haemophilia A, who have failed previous attempts at tolerization including use of immunosuppressants.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Recombinant coagulation factor VIII Fc (rFVIIIFc) | Experimental | Participants will receive rFVIIIFc at a dose of 200 international units (IU)/kilogram (kg) as once daily injections or divided on several injections per day at the discretion of the Investigator, starting at baseline visit up to maximum of 60 Weeks during the ITI Period. Participants who meet the criteria for ITI success will enter a tapering period of 16 weeks where the dose will be tapered down until a prophylactic dose, as judged by the Investigator, is achieved and thereafter a follow-up period of 32 weeks where the patient will continue to receive prophylactic treatment with rFVIIIFc. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Recombinant coagulation factor (rFVIIIFc) | Biological | rFVIIIFc 200 IU/kg/day during ITI Period and thereafter adjusted according to the Investigator's judgement administered intravenously. |
| Measure | Description | Time Frame |
|---|---|---|
| ITI Success | Number of patients who achieve ITI success where ITI success is defined as achieving all 3 of the following criteria:
| up to 60 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Time to ITI Success | Time to the patient reaches ITI success according to the pre-defined criteria For the subset of patients who were classified as partial success at the end of the ITI period, the time to fulfillment of the criteria for partial success was also analyzed descriptively. | up to 60 weeks |
| Occurrence of Relapse During a 48-week Period Following Successful ITI Treatment |
Not provided
Inclusion Criteria:
Signed and dated informed consent provided by the patient, or the patient's legally authorized representative for patients under the legal age. Assent should be obtained from pediatric patients according to local regulations
Male patients of any age diagnosed with severe haemophilia A, as confirmed from the medical record
Previously treated with any plasma-derived or recombinant conventional or extended half-life FVIII
Diagnosed with high titer inhibitors (historical peak ≥5 Bethesda units (BU)/mL according to medical records)
Inhibitor titer >0.6 BU at screening
Failed previous ITI attempt(s) with any plasma-derived or recombinant conventional or extended half-life FVIII including the use of immunosuppressant The attempt should be documented in the medical records and have the following characteristics:
All patients must practice effective contraception during the study and for 3 months after their last dose of study treatment
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Study Physician | Study Medical Director | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Swedish Orphan Biovitrum Research Site | Washington D.C. | District of Columbia | 20010-2970 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39583653 | Derived | Konigs C, Meeks SL, Nolan B, Schmidt A, Lofqvist M, Dumont J, Leickt L, Nayak S, Lethagen S. Rescue immune tolerance induction with a recombinant factor Fc-fused VIII: prospective ReITIrate study of clinical, humoral and cellular immune responses. Ther Adv Hematol. 2024 Nov 23;15:20406207241300809. doi: 10.1177/20406207241300809. eCollection 2024. |
Not provided
Not provided
According to Sobi's data sharing policy Sobi may share anonymized clinical study data with qualified researchers. Sobi commits to sharing clinical study data on participant level and summary data for medicines and indications approved by EMA and/or FDA. Data access will be granted in response to qualified research requests. All requests are evaluated by a cross functional panel of experts within Sobi.
Not provided
Evaluated on a case by case basis
A decision on data sharing will be based on the following:
After informed consent was provided, patients underwent study specific screening procedures. During the 4- to 6-week screening period, patients continued with their usual treatment regimen in accordance with the local standard of care. Patients who met all inclusion and no exclusion criteria specified by the protocol were enrolled into the study. The 2 screening failures did not meet inclusion criteria 1 (signed informed consent) and 5 (inhibitor titer ≥0.6 BU at screening) respectively.
Recruitment was open at 18 sites in 9 countries in Northern America and Europe. 11 sites in 7 countries recruited patients. It was initially planned to enroll 20 patients but due to recruitment challenges and a changing treatment landscape the recruitment was stopped after enrolling 16 patients. Recruitment was open from Aug 2017 to Nov 2019. 18 patients were screened and 16 enrolled into this study.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Recombinant Coagulation Factor VIII Fc (rFVIIIFc) | Participants received rFVIIIFc at a dose of 200 international units (IU)/kilogram (kg) as once daily injections or divided on several injections per day at the discretion of the Investigator, starting at baseline visit up to maximum of 60 Weeks during the ITI Period. Participants who met the criteria for ITI success entered a tapering period of 16 weeks where the dose was tapered down until a prophylactic dose, as judged by the Investigator, was achieved and thereafter a follow-up period of 32 weeks where the patient continued to receive prophylactic treatment with rFVIIIFc. Recombinant coagulation factor (rFVIIIFc): rFVIIIFc 200 IU/kg/day during ITI Period and thereafter adjusted according to the Investigator's judgement administered intravenously. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Feb 26, 2018 | Mar 30, 2021 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
Relapse was defined as a positive inhibitor (≥0.6 BU/mL) on 2 consecutive assessments and incremental recovery ≤66 % of the expected incremental recovery on 2 consecutive assessments |
| Up to 48 weeks |
| Number of Bleedings During ITI Treatment | Only bleeds requiring treatment with rFVIIIFc or bypassing agents should be registered. A bleeding episode starts from the first sign of a bleed and ends no more than 72 hours after the last injection of bypassing agents or rFVIIIFc to treat the bleeding episode. | up to 60 weeks |
| Bleeding Rate During a 48-week Period Following Successful ITI Treatment | Only bleeds requiring treatment with rFVIIIFc or bypassing agents should be registered. A bleeding episode starts from the first sign of a bleed and ends no more than 72 hours after the last injection of bypassing agents or rFVIIIFc to treat the bleeding episode. | up to 48 weeks |
| Adverse Events (AEs) | All observed adverse events as a measure of tolerability. (AE=adverse event, SAE=serious adverse event, TEAE=treatment emergent adverse event) | SAEs - approx 166 weeks AEs - approx 110 weeks |
| Consumption of rFVIIIFc | Consumption will be assessed based on amount of administered study treatment during the ITI period. | Up to 60 weeks |
| Number of Days Missed School or Work During ITI Treatment | Days missed school or work will be registered by the patients in an electronic diary | up to 60 weeks |
| Number of Days Missed School or Work During a 48-week Period Following Successful ITI Treatment | Days missed school or work will be registered by the patients in an electronic diary | up to 48 weeks |
| Number of Hospitalizations During ITI Treatment | Days of hospitalization will be collected by the Investigator at the study visits | up to 60 weeks |
| Number of Hospitalizations During a 48-week Period Following Successful ITI Treatment | Days of hospitalization will be collected by the Investigator at the study visits | Up to 48 weeks |
| Adherence | Defined as percentage of administered doses versus planned doses | up to 108 weeks |
| Swedish Orphan Biovitrum Research Site |
| Hamilton |
| Canada |
| Swedish Orphan Biovitrum Research Site | Vancouver | Canada |
| Swedish Orphan Biovitrum Research Site | Bonn | Germany |
| Swedish Orphan Biovitrum Research Site | Frankfurt am Main | Germany |
| Swedish Orphan Biovitrum Research Site | Mörfelden-Walldorf | Germany |
| Swedish Orphan Biovitrum Research site | Dublin | Ireland |
| Swedish Orphan Biovitrum Research Site | Ljubljana | 1000 | Slovenia |
| Swedish Orphan Biovitrum Research site | Gothenburg | 41345 | Sweden |
| Swedish Orphan Biovitrum Research Site | Birmingham | United Kingdom |
| Swedish Orphan Biovitrum Research Site | Liverpool | United Kingdom |
| Swedish Orphan Biovitrum Research Site | London | WC1N 3JH | United Kingdom |
| COMPLETED |
|
| NOT COMPLETED |
|
|
All enrolled patients
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Recombinant Coagulation Factor VIII Fc (rFVIIIFc) | Participants will receive rFVIIIFc at a dose of 200 international units (IU)/kilogram (kg) as once daily injections or divided on several injections per day at the discretion of the Investigator, starting at baseline visit up to maximum of 60 Weeks during the ITI Period. Participants who meet the criteria for ITI success will enter a tapering period of 16 weeks where the dose will be tapered down until a prophylactic dose, as judged by the Investigator, is achieved and thereafter a follow-up period of 32 weeks where the patient will continue to receive prophylactic treatment with rFVIIIFc. Recombinant coagulation factor (rFVIIIFc): rFVIIIFc 200 IU/kg/day during ITI Period and thereafter adjusted according to the Investigator's judgement administered intravenously. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Age at baseline | Count of Participants | Participants |
| ||||||||||||||||||||||
| Age, Continuous | Median | Full Range | years |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| |||||||||||||||||||||||
| Region of Enrollment | Number | participants |
| |||||||||||||||||||||||
| Hemophilia history - severity | Count of Participants | Participants |
| |||||||||||||||||||||||
| Hemophilia history - Family history of Inhibitors | Count of Participants | Participants |
| |||||||||||||||||||||||
| Number of previous ITI Treatments | Count of Participants | Participants |
| |||||||||||||||||||||||
| Inhibitor History - Historical Peak Titre Level | Median | Full Range | BU/ml |
| ||||||||||||||||||||||
| Inhibitor history - Age at Inhibitor development | Mean | Standard Deviation | years |
|
| 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 | ITI Success | Number of patients who achieve ITI success where ITI success is defined as achieving all 3 of the following criteria:
| Posted | Count of Participants | Participants | up to 60 weeks |
|
|
| |||||||||||||||||||||||||||||||
| Secondary | Time to ITI Success | Time to the patient reaches ITI success according to the pre-defined criteria For the subset of patients who were classified as partial success at the end of the ITI period, the time to fulfillment of the criteria for partial success was also analyzed descriptively. | 1 patient achieved ITI success and 2 patients achieved partial success | Posted | Mean | Standard Deviation | weeks | up to 60 weeks |
|
| ||||||||||||||||||||||||||||||
| Secondary | Occurrence of Relapse During a 48-week Period Following Successful ITI Treatment | Relapse was defined as a positive inhibitor (≥0.6 BU/mL) on 2 consecutive assessments and incremental recovery ≤66 % of the expected incremental recovery on 2 consecutive assessments | Only patients achieving ITI success are included in the analysis for this endpoint. 1 patient achieved ITI success. | Posted | Count of Participants | Participants | Up to 48 weeks |
|
| |||||||||||||||||||||||||||||||
| Secondary | Number of Bleedings During ITI Treatment | Only bleeds requiring treatment with rFVIIIFc or bypassing agents should be registered. A bleeding episode starts from the first sign of a bleed and ends no more than 72 hours after the last injection of bypassing agents or rFVIIIFc to treat the bleeding episode. | Posted | Median | Full Range | Annualized bleeding rate(bleedings/year) | up to 60 weeks |
|
| |||||||||||||||||||||||||||||||
| Secondary | Bleeding Rate During a 48-week Period Following Successful ITI Treatment | Only bleeds requiring treatment with rFVIIIFc or bypassing agents should be registered. A bleeding episode starts from the first sign of a bleed and ends no more than 72 hours after the last injection of bypassing agents or rFVIIIFc to treat the bleeding episode. | Only patients achieving ITI success are included in the analysis for this endpoint. 1 patient achieved ITI success. | Posted | Median | Full Range | Annualized bleeding rate(bleedings/year) | up to 48 weeks |
|
| ||||||||||||||||||||||||||||||
| Secondary | Adverse Events (AEs) | All observed adverse events as a measure of tolerability. (AE=adverse event, SAE=serious adverse event, TEAE=treatment emergent adverse event) | Posted | Number | events | SAEs - approx 166 weeks AEs - approx 110 weeks |
|
| ||||||||||||||||||||||||||||||||
| Secondary | Consumption of rFVIIIFc | Consumption will be assessed based on amount of administered study treatment during the ITI period. | Posted | Median | Full Range | IU | Up to 60 weeks |
|
| |||||||||||||||||||||||||||||||
| Secondary | Consumption of rFVIIIFc | Consumption will be assessed based on amount of administered study treatment during the ITI period. | Posted | Median | Full Range | IU/kg | Up to 60 weeks |
|
| |||||||||||||||||||||||||||||||
| Secondary | Number of Days Missed School or Work During ITI Treatment | Days missed school or work will be registered by the patients in an electronic diary | Posted | Median | Full Range | Days/year | up to 60 weeks |
|
| |||||||||||||||||||||||||||||||
| Secondary | Number of Days Missed School or Work During ITI Treatment | Days missed school or work will be registered by the patients in an electronic diary | Posted | Median | Full Range | Days | up to 60 weeks |
|
| |||||||||||||||||||||||||||||||
| Secondary | Number of Days Missed School or Work During a 48-week Period Following Successful ITI Treatment | Days missed school or work will be registered by the patients in an electronic diary | Only patients achieving ITI success are included in the analysis for this endpoint. 1 patient achieved ITI success. | Posted | Median | Full Range | Days | up to 48 weeks |
|
| ||||||||||||||||||||||||||||||
| Secondary | Number of Hospitalizations During ITI Treatment | Days of hospitalization will be collected by the Investigator at the study visits | Posted | Median | Full Range | Days | up to 60 weeks |
|
| |||||||||||||||||||||||||||||||
| Secondary | Number of Hospitalizations During a 48-week Period Following Successful ITI Treatment | Days of hospitalization will be collected by the Investigator at the study visits | Only patients achieving ITI success are included in the analysis for this endpoint. 1 patient achieved ITI success. | Posted | Number | Days | Up to 48 weeks |
|
| |||||||||||||||||||||||||||||||
| Secondary | Adherence | Defined as percentage of administered doses versus planned doses | 16 patients analyzed for the ITI period. 1 patient analyzed for the tapering and follow-up period (1 patient achieved ITI success and entered tapering and follow-up periods). | Posted | Mean | Standard Deviation | % of administered vs planned doses | up to 108 weeks |
|
|
SAEs: From signature of the informed consent form to the safety follow-up visit (approximately 116 weeks). Non-serious AEs: From the time of first dose of IMP to the safety follow-up visit (approximately 110 weeks).
The investigator were to report all directly observed adverse events, and all adverse events spontaneously reported by the patient. In addition, each patient were to be questioned about AEs at each study visit.
AEs are reported on overall level. Please note the difference in the reporting period of SAEs and non-serious AEs (SAEs also collected during screening period). 18 patients entered screening, 16 patients were eventually enrolled and are included in the analysis.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Recombinant Coagulation Factor VIII Fc (rFVIIIFc) | Participants will receive rFVIIIFc at a dose of 200 international units (IU)/kilogram (kg) as once daily injections or divided on several injections per day at the discretion of the Investigator, starting at baseline visit up to maximum of 60 Weeks during the ITI Period. Participants who meet the criteria for ITI success will enter a tapering period of 16 weeks where the dose will be tapered down until a prophylactic dose, as judged by the Investigator, is achieved and thereafter a follow-up period of 32 weeks where the patient will continue to receive prophylactic treatment with rFVIIIFc. Recombinant coagulation factor (rFVIIIFc): rFVIIIFc 200 IU/kg/day during ITI Period and thereafter adjusted according to the Investigator's judgement administered intravenously. | 0 | 16 | 7 | 16 | 16 | 16 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Brachiocephalic vein thrombosis | Vascular disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Vena cava thrombosis | Vascular disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Thrombosis | Vascular disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Immune thrombocytopenic purpura | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Spontaneous haemorrhage | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Thrombosis in device | Product Issues | MedDRA 22.0 | Systematic Assessment |
| |
| Haemarthrosis | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Vascular device infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Device related infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Device related sepsis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Haemorrhage | Vascular disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Catheter site irritation | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Gait disturbance | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Autism spectrum disorder | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Thermal burn | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Tongue injury | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Traumatic haematoma | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Traumatic haemorrhage | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Activated partial thromboplastin time prolonged | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Blood urine present | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Haematocrit decreased | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Human rhinovirus test positive | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Protein urine present | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Red blood cell count decreased | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Tri-iodothyronine free increased | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Urine ketone body present | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| White blood cell count decreased | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Rhinorrhoea | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Spontaneous haemorrhage | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Jugular vein occlusion | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Visual impairment | Eye disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Abdominal pain lower | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Anal incontinence | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Lip ulceration | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Loose tooth | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Mouth ulceration | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Tooth development disorder | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Onychomadesis | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Red man syndrome | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Thrombosis in device | Product Issues | MedDRA 22.0 | Systematic Assessment |
| |
| Device damage | Product Issues | MedDRA 22.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Arthropathy | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Growing pains | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Haemarthrosis | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Mobility decreased | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Posture abnormal | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Soft tissue swelling | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Adenovirus infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Fungal skin infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Gastrointestinal infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Impetigo | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Tinea infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA 22.0 | Systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Information | Swedish Orphan Biovitrum AB | +468 6972000 | medical.info@sobi.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 22, 2020 | Mar 30, 2021 | SAP_001.pdf |
Not provided
| 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
| ID | Term |
|---|---|
| C587014 | factor VIII-Fc fusion protein |
Not provided
Not provided
Not provided
| Black or African American |
|
| American Indian or Alaska Native |
|
| United States |
|
| Ireland |
|
| United Kingdom |
|
| Slovenia |
|
| Germany |
|
| Severe |
|
| Unknown |
|
| 3 Treatments |
|
| Not determinable due to withdrawal during the ITI |
|
|
|
|
|
| Participants |
|
|
|
|
|
|
|
|
|
|
|