Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2013-002236-24 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Baxalta Innovations GmbH, now part of Shire | INDUSTRY |
Not provided
Not provided
Not provided
Not provided
To continue the evaluation of the safety and efficacy of BAX 855 for prophylaxis and treatment of bleeding episodes in adult and pediatric previously treated patients (PTPs) aged ≤ 75 years of age with severe hemophilia A.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Fixed BAX855 prophylaxis | Experimental | 45-80 IU/kg twice weekly to once per week. |
|
| Pharmacokinetic (PK)-tailored BAX 855 prophylaxis | Experimental | PK-tailored prophylactic BAX855 regimen based on participant's individual PK profile to maintain a Factor VIII (FVIII) trough level |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BAX855 | Biological | Antihemophilic Factor (Recombinant), PEGylated |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Inhibitory Antibodies to Factor VIII (FVIII) | Inhibitory antibodies to Factor VIII were measured by the Nijmegen modification of the Bethesda assay. Inhibitors had to be confirmed by 2 separate assessments within a 2 to 4 week period from the central laboratory. | Baseline through end of study (53 months) |
| Annualized Bleed Rate (ABR) - Spontaneous Bleeds | The ABR was assessed based upon each individual bleeding episode. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. The ABR of spontaneous bleeds was reported separately for twice weekly, PK-t R, each of the every 5 days and every 7 days treatment regimens at the time of bleed. | Baseline through end of study (53 months) |
| Measure | Description | Time Frame |
|---|---|---|
| Total Annualized Bleed Rate (ABR) | The ABR was assessed based upon each individual bleeding episode. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. Bleeding occurring at multiple locations related to the same injury (e.g., knee and ankle bleed following a fall) was counted as a single bleeding episode. Total annualized bleed rate (spontaneous and traumatic bleeding episodes) was reported. |
Not provided
INCLUSION CRITERIA
Participants Transitioning from Other BAX 855 Studies:
Participants transitioning from other BAX 855 studies can be provided with the continuation study informed consent form (ICF) prior to the end of study visit to review and consider participation in this continuation study. These participants will complete any additional screening assessments within 2 weeks of the previous study's end of study visit and will return to the study site within 6 (± 1) weeks of the previous study end of study visit to confirm eligibility for this continuation study.
Participants transitioning from other BAX 855 studies who meet ALL of the following criteria are eligible for this study:
BAX 855 Naïve Participants:
BAX 855 naïve participants who are ≥ 12 years of age can only be enrolled in this continuation study after enrollment in the phase 2/3 pivotal study is closed. BAX 855 naïve participants who are < 12 years of age can only be enrolled in this continuation study after enrollment in the pediatric previously treated patient (PTP) study is closed.
- Enrolment of BAX 855 naïve participants will only start once the sponsor has notified the study sites accordingly.
BAX 855 naïve participants who meet ALL of the following criteria are eligible for this study:
EXCLUSION CRITERA
- Participants Transitioning from Other BAX 855 Studies:
Participants transitioning from other BAX 855 studies who meet ANY of the following criteria are not eligible for this study:
Participant had detectable factor VIII (FVIII) inhibitory antibodies (≥ 0.6 Bethesda unit (BU) using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.
Participant has developed FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay as determined at central laboratory in a previous BAX 855 study).
Participant has acquired a hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease) in a previous BAX 855 study.
Participant has severe chronic hepatic dysfunction (eg, ≥ 5 times upper limit of normal alanine aminotransferase [ALT], as confirmed by central laboratory at screening).
Participant has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening.
Participant experienced a life-threatening or gastrointestinal bleeding episode within 3 months prior to study entry.
Participant is scheduled to use other PEGylated drugs during study participation.
Participant is planning to take part in any other clinical study during the course of the continuation study, with the exception of any other parallel BAX 855 study.
Participant has medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance.
Participant is a family member or employee of the investigator.
BAX 855 naïve participants who meet ANY of the following criteria are not eligible for this study:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Phoenix Childrens Hospital | Phoenix | Arizona | 85016-7710 | United States | ||
| University of Colorado |
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Not provided
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
A total of 218 subjects were enrolled, of them 216 subjects received treatment.
The study was conducted at 86 centers in 23 countries between 15 October 2013 (first participant first visit) and 02 March 2018 (last participant last visit).
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | BAX 855: Age < 2 Years | Participants of age less than (<) 2 years received an infusion of 50 +/- 10 International Units (IU)/kilogram (kg) of BAX 855 twice weekly; could be increased to 80 IU/kg or a pharmacokinetically tailored (PK-tailored) prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain Factor VIII (FVIII) trough levels of greater than or equal to (>=) 3% until at least 100 exposure days (EDs) were reached. |
| 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: Protocol | Jun 18, 2013 | Feb 27, 2019 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Baseline through end of study (53 months) |
| Overall Hemostatic Efficacy Rating of BAX 855 for Treatment of Breakthrough Bleeding Episodes | The participant or caregiver rated the overall treatment response at 24 (+/- 2) hours after the initiation of treatment using a 4-point efficacy rating scale as Excellent: Full relief of pain and cessation of objective signs of bleeding after a single infusion and no additional infusion is required for the control of bleeding; Good: Definite pain relief and/or improvement in signs of bleeding after a single infusion and possibly requires more than 1 infusion for complete resolution; Fair: Slight relief of pain and slight improvement in signs of bleeding after a single infusion and required more than 1 infusion for complete resolution and None: No improvement or condition worsens. | Baseline through end of study (53 months) |
| BAX 855 Infusions Needed to Treat Bleeding Episodes | The BAX 855 infusions to treat each bleeding episode was determined by the participant, the participant's caregiver, and/or investigator, and was based upon the participant's response to treatment. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. | Baseline through end of study (53 months) |
| Total Time Intervals Between Bleeding Episodes | The time interval between bleeding episodes was calculated based upon the date and time reported for each bleeding episode. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. | Baseline through end of study (53 months) |
| Average Dose of BAX 855 Per Prophylactic Infusion | The average dose of BAX 855 per prophylactic infusion was reported. | Baseline through end of study (53 months) |
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | An AE was any unfavorable and unintended sign (eg, an abnormal laboratory finding), symptom (eg, rash, pain, discomfort, fever, dizziness, etc.), disease (eg, peritonitis, bacteremia, etc.), or outcome of death temporally associated with the use of an investigational product (IP), whether or not considered causally related to the IP. A serious adverse event (SAE) was defined as an untoward medical occurrence that at any dose met one or more of the following criteria: outcome was fatal/resulted in death; was life-threatening; required inpatient hospitalization or resulted in prolongation of an existing hospitalization; resulted in persistent or significant disability/incapacity; was a congenital anomaly/birth defect; was a medically important event. | Baseline through end of study (53 months) |
| Change From Baseline in Body Temperature | Change in body temperature at pre-infusion and post-infusion at end of the study was reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling. | Baseline, end of study (53 months) |
| Change From Baseline in Pulse Rate | Change in pulse rate at pre-infusion and post-infusion at end of the study was reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling. | Baseline, end of study (53 months) |
| Change From Baseline in Respiratory Rate | Change in respiratory rate at pre-infusion and post-infusion at end of the study was reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling. | Baseline, end of study (53 months) |
| Change From Baseline in Blood Pressure | Change in systolic and diastolic blood pressure at pre-infusion and post-infusion at end of the study were reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling, SBP refers to systolic blood pressure, DBP refers to diastolic blood pressure. | Baseline, end of study (53 months) |
| Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments. | The number of participants with clinically significant shifts from "normal" "abnormal clinically significant (CS)" and "abnormal not clinically significant (abnormal NCS)" at baseline to "normal" "abnormal clinically significant (CS) and abnormal clinically significant (NCS)" at completion were reported. In the below table, FDR refers to fixed dose regimen at the time of sampling, AlA refers to alanine aminotransferase, AP refers to alkaline phosphatase, AsA refers to aspartate aminotransferase. | Baseline through end of study (53 months) |
| Number of Participants With Shifts in Hematology Laboratory Assessments | The number of participants with clinically significant shifts from "normal" "abnormal clinically significant (CS)" and "abnormal not clinically significant (abnormal NCS)" at baseline to "normal" "abnormal clinically significant (CS) and abnormal clinically significant (NCS)" at completion were reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling, Leu refers to leukocytes, MCV refers to mean corpuscular volume, Lym/Leu refers to lymphocytes/leukocytes. | Baseline through end of study (53 months) |
| Number of Participants With Shifts in Lipid Panel Assessments | The number of participants with clinically significant shifts from "normal" "abnormal clinically significant (CS)" and "abnormal not clinically significant (abnormal NCS)" at baseline to "normal" "abnormal clinically significant (CS) and abnormal clinically significant (NCS)" at completion were reported.. In the below table, HDL refers to high density lipoprotein, LDL refers to low density lipoprotein, VLDL refers to very low density lipoprotein. | Baseline through end of study (53 months) |
| Number of Participants With Binding Antibodies | Binding antibodies (IgG and IgM) against FVIII, polyethylene glycol (PEG) and PEGylated FVIII (PEG-FVIII) were analyzed using enzyme-linked immunosorbent assay (ELISA). | Baseline through end of study (53 months) |
| Number of Participants With Anti-Chinese Hamster Ovary (CHO) Antibodies | Testing for binding of anti-CHO protein antibodies was performed on citrate-anti-coagulated plasma using an ELISA employing polyclonal antihuman IgG antibodies. | Baseline through end of study (53 months) |
| Change From Baseline in Bleed Severity | Hemophilia symptom (haemo-SYM) questionnaire has two subscales: pain and bleed. It was used to asses the bleed severity for participants >=18 years of age as: severity of spontaneous bleeding in my joints (unrelated to injury or activity), spontaneous bleeding in my muscles (unrelated to injury or activity), prolonged bleeding after injury in spite of treatment, intense pain because of bleeding event, joint pain due to active bleed and bleeding during personal hygiene routine, blood in my urine, nose bleeds and assigned a score of 0=Absent, 1=very mild, 2=mild, 3=moderate, 4=severe and 5=very severe. The score was determined as (mean score/5)*100 where mean score is the mean of the available results in the particular subscale. Higher scores on the Haemo-SYM indicate more severe symptoms. Therefore, negative change scores indicate that symptoms have improved. Here 'n' refers to the number of participants evaluable for this endpoint. | Baseline, end of study (53 months) |
| Change From Baseline in Pain Severity | Hemophilia symptom (haemo-SYM) questionnaire has two subscales: pain and bleed. It was used to asses the pain severity for participants >=18 years of age as: pain because of swelling in my joints, climbing stairs, upon waking in the morning, active arthritis; constant pain, in my muscles, that needs medication; joint sensitivity to weather conditions; reduced range of joint movement, joint deformity, sleep disturbance because of pain or bleeds, blood in my urine, nose bleeds and assigned a score of 0=Absent, 1=very mild, 2=mild, 3=moderate, 4=severe and 5=very severe. The score was determined as (mean score/5)*100 where mean score is the mean of the available results in the particular subscale. Higher scores on the Haemo-SYM indicate more severe symptoms. Therefore, negative change scores indicate that symptoms have improved. Here 'n' refers to the number of participants evaluable for this endpoint. | Baseline, end of study (53 months) |
| Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36) | HRQoL in participants aged >=14 years was measured using the SF-36 questionnaire. The questionnaire was divided into 8 domains and scored as: physical functioning (1=yes, limited a lot to 3=no, not limited at all), role-physical (1=all of the time to 5=none of the time), bodily pain (1=very severe to 6=none), general health (1=poor to 5=excellent), vitality (1=none of the time to 5=all of the time), social functioning (1=all of the time: to 5=none of the time), role emotional (1=all of the time to 5=none of the time) and mental health (1=all of the time to 5=none of the time). The score for each domain is then to be transformed to a 0-100 range as [(actual raw score-lowest possible raw score)/possible raw score range]*100. Positive change scores indicate improved HRQoL. in the below table 'FDR' indicates fixed dose regimen, 'PK-tr' indicates pharmacokinetically tailored regimen and 'n' refers to the number of participants evaluable for this endpoint. | Baseline, end of study (53 months) |
| Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire | HRQoL in participants aged <14 years was measured using the PedsQL. It capture data for the following domains: physical functioning, emotional functioning, social functioning, school functioning, psychosocial functioning, physical health and a total score. Each question of the PedsQL was scored as Never: 100, almost never: 75, sometimes: 50, often: 25, almost always: 0. The mean of the individual question scores was calculated. Lower scores on the PedsQL indicating worse HRQoL. Here, FDR refers to fixed dose regimen, PK-t R refers to PK-tailored regimen. Here 'n' refers to the number of participants evaluable for this endpoint. Here 'n' refers to the number of participants evaluable for this endpoint. | Baseline, end of study (53 months) |
| Aurora |
| Colorado |
| 80045 |
| United States |
| University of Florida College of Medicine | Gainesville | Florida | 32610-0278 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611-2605 | United States |
| Bleeding and Clotting Disorders Institute | Peoria | Illinois | 61614 | United States |
| University of Kentucky Medical Center | Lexington | Kentucky | 40536-0284 | United States |
| University of Louisville | Louisville | Kentucky | 40202 | United States |
| Tulane University School of Medicine | New Orleans | Louisiana | 70112 | United States |
| Children's Mercy Hospitals & Clinics | Kansas City | Missouri | 64108 | United States |
| North Shore-Long Island Jewish Health System | New Hyde Park | New York | 11040 | United States |
| New York - Presbyterian/Weill Cornell Medical Center | New York | New York | 10065 | United States |
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27599-7016 | United States |
| Duke University Medical Center | Durham | North Carolina | 27705 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Cleveland Clinic Foundation | Cleveland | Ohio | 44195 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| University of Oklahoma | Oklahoma City | Oklahoma | 73104 | United States |
| Penn State Hershey Cancer Center | Hershey | Pennsylvania | 17033 | United States |
| Palmetto Health Richland | Columbia | South Carolina | 29203-6863 | United States |
| University of Utah | Salt Lake City | Utah | 84108 | United States |
| Puget Sound Blood Group | Seattle | Washington | 98104 | United States |
| Royal Adelaide Hospital | Adelaide | South Australia | 5000 | Australia |
| The Alfred Hospital | Melbourne | Victoria | 3004 | Australia |
| Fremantle Hospital | Fremantle | Western Australia | 6160 | Australia |
| Landes-Frauen-und Kinderklinik Linz | Linz | 4020 | Austria |
| Universitatsklinik fur Innere Medizin I | Vienna | 1090 | Austria |
| UMHAT "Sv. Georgi", EAD | Plovdiv | 4002 | Bulgaria |
| SHAT of Oncohaematology Diseases | Sofia | 1527 | Bulgaria |
| MHAT 'Sv. Marina', EAD | Varna | 9010 | Bulgaria |
| Fakultni nemocnice Brno | Brno | 625 00 | Czechia |
| Fakultni nemocnice Olomouc | Olomouc | 775 20 | Czechia |
| Fakultni nemocnice v Motole | Prague | 150 06 | Czechia |
| Werlhof-Institut GmbH | Hanover | Lower Saxony | 30159 | Germany |
| Gerinnungszentrum Rhein-Ruhr | Duisburg | North Rhine-Westphalia | 47051 | Germany |
| Vivantes Klinikum im Friedrichshain - Landsberger Allee | Berlin | 10249 | Germany |
| Universitaetsklinikum Hamburg-Eppendorf | Hamburg | 20246 | Germany |
| Prince of Wales Hospital | Shatin | 00000 | Hong Kong |
| Rambam Health Care Campus | Haifa | 3109601 | Israel |
| Chaim Sheba Medical Center | Ramat Gan | 52621 | Israel |
| Nagoya University Hospital | Nagoya | Aichi-ken | 466-8560 | Japan |
| University of Occupational and Environmental Health Hospital | Kitakyushu-shi | Fukuoka | 807-8556 | Japan |
| Hiroshima University Hospital | Hiroshima | Hiroshima | 734-8551 | Japan |
| St. Marianna University School of Medicine Hospital | Kawasaki-shi | Kanagawa | 216-8511 | Japan |
| Nara Medical University Hospital | Kashihara-shi | Nara | 634-8522 | Japan |
| Ogikubo Hospital | Suginami City | Tokyo | 167-8515 | Japan |
| Tokyo Medical University Hospital | Shinjuku-ku | Tokyo-To | 160-0023 | Japan |
| Vilnius University Hospital Santariskiu Clinics, Public Institution | Vilnius | 8661 | Lithuania |
| Children's Hospital, Affiliate of Vilnius University Hospital Santariskiu Klinikos | Vilnius | LT-08406 | Lithuania |
| Penang General Hospital | George Town | Pulau Pinang | 10990 | Malaysia |
| Hospital Umum Sarawak | Kuching | Sarawak | 93586 | Malaysia |
| Hospital Sibu | Sibu | Sarawak | 96000 | Malaysia |
| Hospital Ampang | Ampang | Selangor | 68000 | Malaysia |
| Pusat Darah Negara | Kuala Lumpur | 50400 | Malaysia |
| Hospital Pulau Pinang | Pulau Pinang | 10450 | Malaysia |
| Academisch Medisch Centrum | Amsterdam | 1105 AZ | Netherlands |
| Uniwersyteckie Centrum Kliniczne | Gdansk | 80-952 | Poland |
| Wojewodzki Szpital Specjalistyczny im.M.Kopernika w Lodzi | Lodz | 93-510 | Poland |
| Sanador SRL | Bucharest | 11038 | Romania |
| LLC "Alba Dent" | Kirov | 610000 | Russia |
| Regional Clinical Hospital | Krasnoyarsk | 660022 | Russia |
| Chonnam National University Hwasun Hospital | Hwasun-gun | Jeollanam-do | 519-763 | South Korea |
| Pusan National University Hospital | Busan | 602-739 | South Korea |
| Eulji University Hospital | Daejeon | 302-799 | South Korea |
| Kyung Hee University Hospital at Gangdong | Seoul | 134-727 | South Korea |
| Ulsan University Hospital | Ulsan | 682-714 | South Korea |
| Hospital Universitari Son Espases | Palma de Mallorca | Balearic Islands | 7010 | Spain |
| Complejo Hospitalario Universitario A Coruña | A Coruña | La Coruña | 15006 | Spain |
| Hospital Regional Universitario de Malaga | Málaga | Málaga | 29010 | Spain |
| Hospital Universitario La Paz | Madrid | 28046 | Spain |
| Hospital Universitari i Politecnic La Fe | Valencia | 46026 | Spain |
| Sjukhusapoteket Malmo | Malmö | 21428 | Sweden |
| Karolinska | Stockholm | 17164 | Sweden |
| Universitaetsspital Zuerich | Zurich | 8091 | Switzerland |
| Taichung Veterans General Hospital | Taichung | 40705 | Taiwan |
| Tri-Service General Hospital | Taipei | 11490 | Taiwan |
| Ankara University Medical Faculty | Ankara | 063100 | Turkey (Türkiye) |
| Akdeniz University Faculty of Medicine | Antalya | 07059 | Turkey (Türkiye) |
| Istanbul University Cerrahpasa Medical Faculty | Istanbul | 34098 | Turkey (Türkiye) |
| SI V.K.Gusak Emergency and Reconstructive Surgery Institute of NAMSU | Donetsk | 83045 | Ukraine |
| SI Institute of Blood Pathology and Transfusion Medicine of NAMSU | Lviv | 79044 | Ukraine |
| Bristol Royal Hospital for Children | Bristol | Avon | BS2 8BJ | United Kingdom |
| St Thomas' Hospital | London | Greater London | SE1 7EH | United Kingdom |
| Royal Free Hospital | London | Greater London | WC1E 6AG | United Kingdom |
| Great Ormond Street Hospital for Children | London | Greater London | WC1N 3JH | United Kingdom |
| Royal Manchester Children's Hospital | Manchester | Greater Manchester | M13 9WL | United Kingdom |
| Southampton General Hospital | Southampton | Hampshire | SO16 6YD | United Kingdom |
| Leicester Royal Infirmary | Leicester | Leicestershire | LE1 5WW | United Kingdom |
| Birmingham Children's Hospital | Birmingham | West Midlands | B4 6NH | United Kingdom |
| The Churchill Hospital | Oxford | OX3 7LE | United Kingdom |
| FG001 | BAX 855: Age >= 2 to <12 Years | Participants of age >= 2 to <12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| FG002 | BAX 855: Age >= 12 to <17 Years | Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| FG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Safety analysis set included all participants with at least 1 BAX855 infusion.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | BAX 855: Age < 2 Years | Participants of age < 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| BG001 | BAX 855: Age >= 2 to <12 Years | Participants of age >= 2 to <12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| BG002 | BAX 855: Age >= 12 to <17 Years | Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| BG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | 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 | Number of Participants With Inhibitory Antibodies to Factor VIII (FVIII) | Inhibitory antibodies to Factor VIII were measured by the Nijmegen modification of the Bethesda assay. Inhibitors had to be confirmed by 2 separate assessments within a 2 to 4 week period from the central laboratory. | Safety analysis set (SAS) included all participants with at least 1 BAX 855 infusion. The analysis included participants that developed inhibitory antibodies (IA) to FVIII and participants that did not develop IA to FVIII and had 100 or more exposure days (ED) to BAX 855 across all studies and a FVIII inhibitory test result after the 100th ED. | Posted | Count of Participants | Participants | Baseline through end of study (53 months) |
|
|
| |||||||||||||||||||||||||||||||||||
| Primary | Annualized Bleed Rate (ABR) - Spontaneous Bleeds | The ABR was assessed based upon each individual bleeding episode. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. The ABR of spontaneous bleeds was reported separately for twice weekly, PK-t R, each of the every 5 days and every 7 days treatment regimens at the time of bleed. | Full analysis set (FAS) included all participants with at least 1 BAX 855 infusion. | Posted | Median | 95% Confidence Interval | Bleeds per year | Baseline through end of study (53 months) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Total Annualized Bleed Rate (ABR) | The ABR was assessed based upon each individual bleeding episode. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. Bleeding occurring at multiple locations related to the same injury (e.g., knee and ankle bleed following a fall) was counted as a single bleeding episode. Total annualized bleed rate (spontaneous and traumatic bleeding episodes) was reported. | FAS included all participants with at least 1 BAX 855 infusion. | Posted | Mean | Standard Deviation | Bleeds per year | Baseline through end of study (53 months) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Overall Hemostatic Efficacy Rating of BAX 855 for Treatment of Breakthrough Bleeding Episodes | The participant or caregiver rated the overall treatment response at 24 (+/- 2) hours after the initiation of treatment using a 4-point efficacy rating scale as Excellent: Full relief of pain and cessation of objective signs of bleeding after a single infusion and no additional infusion is required for the control of bleeding; Good: Definite pain relief and/or improvement in signs of bleeding after a single infusion and possibly requires more than 1 infusion for complete resolution; Fair: Slight relief of pain and slight improvement in signs of bleeding after a single infusion and required more than 1 infusion for complete resolution and None: No improvement or condition worsens. | FAS included all participants with at least 1 BAX 855 infusion. Here 'N' refers to the number of participants evaluable for this outcome. | Posted | Number | Treated bleeds | Baseline through end of study (53 months) |
| |||||||||||||||||||||||||||||||||||||
| Secondary | BAX 855 Infusions Needed to Treat Bleeding Episodes | The BAX 855 infusions to treat each bleeding episode was determined by the participant, the participant's caregiver, and/or investigator, and was based upon the participant's response to treatment. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. | FAS included all participants with at least 1 BAX 855 infusion. | Posted | Mean | Standard Deviation | Infusions | Baseline through end of study (53 months) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Total Time Intervals Between Bleeding Episodes | The time interval between bleeding episodes was calculated based upon the date and time reported for each bleeding episode. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. | FAS included all participants with at least 1 BAX 855 infusion. Here number of participants analyzed refers to the number of participants evaluable for this outcome. | Posted | Median | Full Range | Months | Baseline through end of study (53 months) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Average Dose of BAX 855 Per Prophylactic Infusion | The average dose of BAX 855 per prophylactic infusion was reported. | SAS included all participants with at least 1 BAX 855 infusion. | Posted | Mean | Standard Deviation | International units per kilogram (IU/kg) | Baseline through end of study (53 months) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | An AE was any unfavorable and unintended sign (eg, an abnormal laboratory finding), symptom (eg, rash, pain, discomfort, fever, dizziness, etc.), disease (eg, peritonitis, bacteremia, etc.), or outcome of death temporally associated with the use of an investigational product (IP), whether or not considered causally related to the IP. A serious adverse event (SAE) was defined as an untoward medical occurrence that at any dose met one or more of the following criteria: outcome was fatal/resulted in death; was life-threatening; required inpatient hospitalization or resulted in prolongation of an existing hospitalization; resulted in persistent or significant disability/incapacity; was a congenital anomaly/birth defect; was a medically important event. | SAS included all participants with at least 1 BAX 855 infusion. | Posted | Count of Participants | Participants | Baseline through end of study (53 months) |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Body Temperature | Change in body temperature at pre-infusion and post-infusion at end of the study was reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling. | SAS included all participants with at least 1 BAX 855 infusion. Here, number of participants analyzed refer to the number of participants evaluable for this outcome at specified time point. | Posted | Mean | Standard Deviation | Degree celsius | Baseline, end of study (53 months) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Pulse Rate | Change in pulse rate at pre-infusion and post-infusion at end of the study was reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling. | SAS included all participants with at least 1 BAX 855 infusion. Here, number of participants analyzed refer to the number of participants evaluable for this outcome at specified time point. | Posted | Mean | Standard Deviation | Beats per minute (beats/min) | Baseline, end of study (53 months) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Respiratory Rate | Change in respiratory rate at pre-infusion and post-infusion at end of the study was reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling. | SAS included all participants with at least 1 BAX 855 infusion. Here, number of participants analyzed refer to the number of participants evaluable for this outcome at specified time point. | Posted | Mean | Standard Deviation | Breaths per minute (breaths/min) | Baseline, end of study (53 months) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Blood Pressure | Change in systolic and diastolic blood pressure at pre-infusion and post-infusion at end of the study were reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling, SBP refers to systolic blood pressure, DBP refers to diastolic blood pressure. | SAS with evaluable participants for this endpoint were analyzed. Here, number of participants analyzed refer to the number of participants evaluable for this outcome at specified time point. | Posted | Mean | Standard Deviation | Millimeter of mercury (mmHg) | Baseline, end of study (53 months) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments. | The number of participants with clinically significant shifts from "normal" "abnormal clinically significant (CS)" and "abnormal not clinically significant (abnormal NCS)" at baseline to "normal" "abnormal clinically significant (CS) and abnormal clinically significant (NCS)" at completion were reported. In the below table, FDR refers to fixed dose regimen at the time of sampling, AlA refers to alanine aminotransferase, AP refers to alkaline phosphatase, AsA refers to aspartate aminotransferase. | SAS included all participants with at least 1 BAX 855 infusion. Here, number of participants analyzed refer to the number of participants evaluable for this outcome at specified time point. | Posted | Count of Participants | Participants | Baseline through end of study (53 months) |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Shifts in Hematology Laboratory Assessments | The number of participants with clinically significant shifts from "normal" "abnormal clinically significant (CS)" and "abnormal not clinically significant (abnormal NCS)" at baseline to "normal" "abnormal clinically significant (CS) and abnormal clinically significant (NCS)" at completion were reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling, Leu refers to leukocytes, MCV refers to mean corpuscular volume, Lym/Leu refers to lymphocytes/leukocytes. | SAS included all participants with at least 1 BAX 855 infusion. Here, number of participants analyzed refer to the number of participants evaluable for this outcome at specified time point. | Posted | Count of Participants | Participants | Baseline through end of study (53 months) |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Shifts in Lipid Panel Assessments | The number of participants with clinically significant shifts from "normal" "abnormal clinically significant (CS)" and "abnormal not clinically significant (abnormal NCS)" at baseline to "normal" "abnormal clinically significant (CS) and abnormal clinically significant (NCS)" at completion were reported.. In the below table, HDL refers to high density lipoprotein, LDL refers to low density lipoprotein, VLDL refers to very low density lipoprotein. | SAS included all participants with at least 1 BAX 855 infusion.Here, number of participants analyzed refer to the number of participants evaluable for this outcome at specified time point. | Posted | Count of Participants | Participants | Baseline through end of study (53 months) |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Binding Antibodies | Binding antibodies (IgG and IgM) against FVIII, polyethylene glycol (PEG) and PEGylated FVIII (PEG-FVIII) were analyzed using enzyme-linked immunosorbent assay (ELISA). | SAS included all participants with at least 1 BAX 855 infusion. | Posted | Count of Participants | Participants | Baseline through end of study (53 months) |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Anti-Chinese Hamster Ovary (CHO) Antibodies | Testing for binding of anti-CHO protein antibodies was performed on citrate-anti-coagulated plasma using an ELISA employing polyclonal antihuman IgG antibodies. | SAS included all participants with at least 1 BAX 855 infusion. | Posted | Count of Participants | Participants | Baseline through end of study (53 months) |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Bleed Severity | Hemophilia symptom (haemo-SYM) questionnaire has two subscales: pain and bleed. It was used to asses the bleed severity for participants >=18 years of age as: severity of spontaneous bleeding in my joints (unrelated to injury or activity), spontaneous bleeding in my muscles (unrelated to injury or activity), prolonged bleeding after injury in spite of treatment, intense pain because of bleeding event, joint pain due to active bleed and bleeding during personal hygiene routine, blood in my urine, nose bleeds and assigned a score of 0=Absent, 1=very mild, 2=mild, 3=moderate, 4=severe and 5=very severe. The score was determined as (mean score/5)*100 where mean score is the mean of the available results in the particular subscale. Higher scores on the Haemo-SYM indicate more severe symptoms. Therefore, negative change scores indicate that symptoms have improved. Here 'n' refers to the number of participants evaluable for this endpoint. | FAS with population of age greater than or equal to 18 years were analyzed. | Posted | Mean | Standard Deviation | Score on a scale | Baseline, end of study (53 months) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Pain Severity | Hemophilia symptom (haemo-SYM) questionnaire has two subscales: pain and bleed. It was used to asses the pain severity for participants >=18 years of age as: pain because of swelling in my joints, climbing stairs, upon waking in the morning, active arthritis; constant pain, in my muscles, that needs medication; joint sensitivity to weather conditions; reduced range of joint movement, joint deformity, sleep disturbance because of pain or bleeds, blood in my urine, nose bleeds and assigned a score of 0=Absent, 1=very mild, 2=mild, 3=moderate, 4=severe and 5=very severe. The score was determined as (mean score/5)*100 where mean score is the mean of the available results in the particular subscale. Higher scores on the Haemo-SYM indicate more severe symptoms. Therefore, negative change scores indicate that symptoms have improved. Here 'n' refers to the number of participants evaluable for this endpoint. | FAS with population of age greater than or equal to 18 years were analyzed. | Posted | Mean | Standard Deviation | Score on a scale | Baseline, end of study (53 months) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36) | HRQoL in participants aged >=14 years was measured using the SF-36 questionnaire. The questionnaire was divided into 8 domains and scored as: physical functioning (1=yes, limited a lot to 3=no, not limited at all), role-physical (1=all of the time to 5=none of the time), bodily pain (1=very severe to 6=none), general health (1=poor to 5=excellent), vitality (1=none of the time to 5=all of the time), social functioning (1=all of the time: to 5=none of the time), role emotional (1=all of the time to 5=none of the time) and mental health (1=all of the time to 5=none of the time). The score for each domain is then to be transformed to a 0-100 range as [(actual raw score-lowest possible raw score)/possible raw score range]*100. Positive change scores indicate improved HRQoL. in the below table 'FDR' indicates fixed dose regimen, 'PK-tr' indicates pharmacokinetically tailored regimen and 'n' refers to the number of participants evaluable for this endpoint. | FAS with population of age greater than or equal to 14 years were analyzed. | Posted | Mean | Standard Deviation | Score on a scale | Baseline, end of study (53 months) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire | HRQoL in participants aged <14 years was measured using the PedsQL. It capture data for the following domains: physical functioning, emotional functioning, social functioning, school functioning, psychosocial functioning, physical health and a total score. Each question of the PedsQL was scored as Never: 100, almost never: 75, sometimes: 50, often: 25, almost always: 0. The mean of the individual question scores was calculated. Lower scores on the PedsQL indicating worse HRQoL. Here, FDR refers to fixed dose regimen, PK-t R refers to PK-tailored regimen. Here 'n' refers to the number of participants evaluable for this endpoint. Here 'n' refers to the number of participants evaluable for this endpoint. | FAS with population of age less than 14 years were analyzed. | Posted | Mean | Standard Deviation | Score on a scale | Baseline, end of study (53 months) |
|
From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
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 | BAX 855: Age < 2 Years | Participants of age < 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. | 0 | 3 | 0 | 3 | 3 | 3 |
| EG001 | BAX 855: Age >= 2 to <12 Years | Participants of age >= 2 to <12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. | 0 | 62 | 7 | 62 | 40 | 62 |
| EG002 | BAX 855: Age >= 12 to <17 Years | Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. | 1 | 26 | 4 | 26 | 12 | 26 |
| EG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. | 0 | 125 | 22 | 125 | 62 | 125 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Splenic haematoma | Blood and lymphatic system disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Haemarthrosis | Musculoskeletal and connective tissue disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Muscle haemorrhage | Musculoskeletal and connective tissue disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Metastases to lung | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 20.1 | Non-systematic Assessment |
| |
| Renal cancer metastatic | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 20.1 | Non-systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Ileus | Gastrointestinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Abscess oral | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Bacteraemia | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Device related infection | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Device related sepsis | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Incision site abscess | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Laryngitis | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Plasmodium falciparum infection | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Skin infection | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Streptococcal bacteraemia | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Facial bones fracture | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Femoral neck fracture | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Nasal injury | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Scapula fracture | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Splenic rupture | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Traumatic fracture | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Wound haemorrhage | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Wrist fracture | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Ureterolithiasis | Renal and urinary disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Cerebral haemorrhage | Nervous system disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Enterovirus infection | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Otitis media acute | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Skull fracture | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
| |
| Febrile convulsion | Nervous system disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Pharyngeal ulceration | Respiratory, thoracic and mediastinal disorders | MedDRA 20.1 | Non-systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 20.1 | Non-systematic Assessment |
|
If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Shire | +1 866 842 5335 | ClinicalTransparency@shire.com |
| Prot_000.pdf |
| Prot | Yes | No | No | Study Protocol: Protocol Amendment 1 | Oct 2, 2013 | Feb 27, 2019 | Prot_001.pdf |
| Prot | Yes | No | No | Study Protocol: Protocol Amendment 4 | May 23, 2014 | Feb 27, 2019 | Prot_002.pdf |
| Prot | Yes | No | No | Study Protocol: Protocol Amendment 7 | Mar 20, 2015 | Feb 27, 2019 | Prot_003.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 18, 2018 | Feb 27, 2019 | SAP_004.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
| ID | Term |
|---|---|
| C000609799 | BAX 855 |
Not provided
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| OG002 | BAX 855: Age >= 12 to <17 Years | Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| OG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
|
|
| OG002 | BAX 855: Age >= 12 to <17 Years | Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| OG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
|
|
Participants of age >= 2 to <12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached.
| OG002 | BAX 855: Age >= 12 to <17 Years | Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| OG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
|
|
| OG002 | BAX 855: Age >= 12 to <17 Years | Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| OG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
|
|
| OG002 | BAX 855: Age >= 12 to <17 Years | Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| OG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
|
|
Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached.
| OG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
|
|
Participants of age >= 2 to <12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached.
| OG002 | BAX 855: Age >= 12 to <17 Years | Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| OG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
|
|
| OG002 |
| BAX 855: Age >= 12 to <17 Years |
Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| OG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
|
|
| OG002 |
| BAX 855: Age >= 12 to <17 Years |
Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| OG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
|
|
| OG002 | BAX 855: Age >= 12 to <17 Years | Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| OG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
|
|
| OG002 | BAX 855: Age >= 12 to <17 Years | Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| OG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
|
|
| OG002 | BAX 855: Age >= 12 to <17 Years | Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| OG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
|
|
| OG002 | BAX 855: Age >= 12 to <17 Years | Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| OG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
|
|
| OG002 | BAX 855: Age >= 12 to <17 Years | Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| OG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
|
|
Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| OG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
|
|
Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
| OG003 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| OG001 | BAX 855: Age >= 17 Years | Participants of age >= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached. |
|
|
Participants of age >= 12 to <17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of >= 3% until at least 100 EDs were reached.
|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|