Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2015-002136-40 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Takeda Development Center Americas, Inc. | INDUSTRY |
Not provided
Not provided
Not provided
Not provided
This study is for young children with severe hemophilia A who have previously not been treated with BAX855 or other FVIII concentrates.
The main aim of the study is to check for side effects from treatment with BAX855. This includes the buildup of antibodies against FVIII which may stop BAX855 from working properly. Another aim is to learn how well BAX855 controls bleeding.
In this study, the children can receive BAX855 either as preventative treatment (prophylaxis), or as needed to treat bleeding (on-demand).
In case a participant develops antibodies, treatment will be provided as part of the study.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| All Participants | Experimental | Previously Untreated Patients (PUPs) < 6 years of age with severe hemophilia A (FVIII < 1%) and < 3 exposure days (EDs) to ADVATE, BAX 855 or plasma transfusion were enrolled in a single arm group. Part A (Main Study): Participants age <3 years - who had not experienced two joint bleeds received on-demand treatment of 10-80 international units per kilogram (IU/kg) intravenously (IV) depending on the severity of the bleeding episode; and - who experienced maximum of two joint bleeds received prophylaxis treatment with dose of 25-80 IU/kg of BAX 855 IV (based on investigator discretion) once weekly for up to 100 EDs. Part B (Immune tolerance induction [ITI] Portion): Participants who met the pre-defined Part B treatment criteria entered Part B of the study for ITI. Participants either received prophylaxis treatment of BAX 855 low dose 50 IU/kg IV, three times a week or high dose 100-200 IU/kg IV, daily at discretion of the investigator according to the institution's standard of care. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PEGylated Recombinant Factor VIII | Biological | Polyethylene glycol (PEG)-ylated full-length recombinant FVIII (rFVIII). |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With FVIII Inhibitor Development | Number of participants who developed an inhibitor (at any time) confirmed by a central laboratory based on a second repeat blood sample draw within 2 weeks of site notification of an inhibitor and all participants who had not developed an inhibitor and had greater than or equal to (>=) 100 EDs when the sample for the last valid inhibitor test was drawn. | Throughout Part A of the study, approximately 5 years |
| Number of Participants With Success of Immune Tolerance Induction (ITI) | Success is defined as 1) a persistently negative inhibitor titer less than (<) 0.6 Bethesda unit (BU), 2) FVIII IR >=66% of the baseline value following a wash-out period of 84-96 hours, and 3) a FVIII half-life of >=6 hours. | Up to 33 months in Part B of the study |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies | Binding IgG and IgM antibodies to FVIII , Factor VIII-Polyethylene glycol (PEG-FVIII) and Polyethylene glycol (PEG) was assessed. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen. Visits and their approximate time in weeks after baseline visit for individual participants: Visit 1(Week 5), Visit 2 (Week 10), Visit 3 (Week 15), Visit 4 (Week 20), Visit 5 (Week 30), Visit 6 (Week 40), Visit 7 (Week 55), Visit 8 (Week 75) and Study Completion Visit (Weeks 100-110). |
Not provided
Inclusion Criteria
Additional inclusion criteria for Part B (immune tolerance induction [ITI]).
Parent or legal representative has/have voluntarily provided signed informed consent for ITI portion.
Participant has a confirmed positive high titer inhibitor (> 5.00 Bethesda unit (BU)) or has a positive confirmed low titer inhibitor (greater than or equal to [>=] 0.6 BU) as determined by the central laboratory based on a second repeat blood sample with
Exclusion Criteria
Additional exclusion criteria for Part B (ITI)
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 | United States | ||
| Kaiser Permanente Oakland M.C. |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37646148 | Derived | Sidonio RF Jr, Thompson AA, Peyvandi F, Stasyshyn O, Yeoh SL, Sosothikul D, Antmen AB, Maggiore C, Engl W, Ewenstein B, Tangada S. Immunogenicity, safety, and efficacy of rurioctocog alfa pegol in previously untreated patients with severe hemophilia A: interim results from a phase 3, prospective, multicenter, open-label study. Expert Rev Hematol. 2023 Jul-Dec;16(10):793-801. doi: 10.1080/17474086.2023.2247160. Epub 2023 Sep 7. |
| Label | URL |
|---|---|
| To obtain more information on the study, click here/on this link | View source |
Not provided
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.
Previously untreated patients (PUPs) <6 years with severe hemophilia A (Factor VIII [FVIII] <1%) were treated with BAX 855 in Part A of this study for at least 100 exposure days (EDs) or until they had developed a confirmed FVIII inhibitor. Then, participants who developed high or low titer FVIII inhibitors entered Part B. In Part B they underwent immune tolerance induction (ITI) with BAX 855.
Participants took part in the study at various investigative sites globally from 12 November 2015 to 29 October 2024.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | All Participants | PUPs < 6 years of age with severe hemophilia A (FVIII < 1%) and < 3 EDs to ADVATE, BAX 855 or plasma transfusion were enrolled in a single arm group. Part A (Main Study): Participants age <3 years - who had not experienced two joint bleeds received on-demand treatment of 10-80 international units per kilogram (IU/kg) intravenously (IV) depending on the severity of the bleeding episode; and - who experienced maximum of two joint bleeds received prophylaxis treatment with dose of 25-80 IU/kg of BAX 855 IV (based on investigator discretion) once weekly for up to 100 EDs. Part B (ITI Portion): Participants who met the pre-defined Part B treatment criteria entered Part B of the study for ITI. Participants either received prophylaxis treatment of BAX 855 low dose 50 IU/kg IV, three times a week or high dose 100-200 IU/kg IV, daily at the discretion of the investigator according to the institution's standard of care. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part A: Main Study (5 Years) |
|
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 | Jun 21, 2021 | Apr 27, 2025 |
Not provided
Not provided
Not provided
Not provided
Not provided
| ITI | Biological | Immune tolerance induction therapy |
|
| Throughout Part A of the study, approximately 5 years |
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | An AE is defined as any untoward medical occurrence in a participant administered an investigational product (IP) that does not necessarily have a causal relationship with the treatment. A SAE is any untoward clinical manifestation of signs, symptoms or outcomes (whether considered related to investigational product or not and at any dose) which results in death, is life-threatening, requires inpatient hospitalization, prolongation of hospitalization, is an important medical event. Number of participants with AEs and SAEs in both Part A and Part B were assessed. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen. | Throughout Part A and Part B of the study, approximately 9 years |
| Number of Participants With At Least One Clinically Significant Changes in Vital Signs | Vital signs were assessed based on body temperature, respiratory rate, blood pressure, and heart rate. | Throughout Part A and Part B of the study, approximately 9 years |
| Number of Participants With At Least One Clinically Significant Changes in Clinical Laboratory Parameters | Clinical laboratory parameters included hematology and clinical chemistry. Changes in laboratory values could be considered as AE if they were judged to be clinically significant. | Throughout Part A and Part B of the study, approximately 9 years |
| Annualized Bleeding Rate (ABR) for Prophylactic and On-demand Treatment and Immune Tolerance Induction (ITI) | 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 (example, knee and ankle bleed following a fall) was counted as a single bleeding episode. Mean total annualized bleed rate is reported. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen. | Throughout Part A and Part B of the study, approximately 9 years |
| Bleeding Episodes Categorized by Number of BAX 855 Infusions Required for Treatment | A bleeding episode is defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. The number of BAX 855 infusions needed for each bleeding episode was determined by the participant, caregiver, clinician treating the participant, and is based upon the participant's response to treatment, using the Efficacy Rating Scale for Treatment of Bleeding Episodes. Number of bleeding episodes are categorized by number of infusions required to treat the bleeding episodes. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen. | Throughout Part A of the study, approximately 5 years |
| Number of Bleeds by Overall Hemostatic Efficacy Rating at 24 Hours After Initiation of Treatment | The participant or caregiver rated overall treatment response using a 4-point efficacy rating scale as Excellent:Full relief of pain & cessation of objective signs of bleeding after single infusion & no additional infusion is required for the control of bleeding; Good:Definite pain relief &/or improvement in signs of bleeding after single infusion & possibly requires more than 1 infusion for complete resolution; Fair:Probable &/or slight relief of pain & slight improvement in signs of bleeding after single infusion & required more than 1 infusion for complete resolution & None:No improvement or condition worsens.Number of bleeds with each efficacy rating are reported.Participant that received on-demand treatment first & then moved to prophylaxis treatment was counted for both on-demand & prophylaxis regimens.Participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on prophylaxis regimen. | At 24 hours after study drug administration during Part A of the study |
| Number of Bleeds by Overall Hemostatic Efficacy Rating at Bleed Resolution | The participant or caregiver rated overall treatment response using a 4-point efficacy rating scale as Excellent:Full relief of pain & cessation of objective signs of bleeding after single infusion & no additional infusion is required for the control of bleeding; Good:Definite pain relief &/or improvement in signs of bleeding after a single infusion & possibly requires more than 1 infusion for complete resolution; Fair:Probable &/or slight relief of pain & slight improvement in signs of bleeding after single infusion & required more than 1 infusion for complete resolution & None:No improvement or condition worse.Number of bleeds with each efficacy rating are reported.Participant that received on-demand treatment first & then moved to prophylaxis treatment was counted for both on-demand & prophylaxis regimens.Participant that started with prophylaxis treatment was counted only for prophylaxis regimen even if the participant received on-demand treatment while on prophylaxis regimen. | From start of study treatment up to bleed resolution throughout Part A of the study (up to approximately 5 years) |
| Weight-adjusted Consumption of BAX 855: Average Prophylactic Dose | Weight-adjusted consumption of BAX 855 was determined based upon the record in participants diaries of the actual amount of BAX 855 infused as measured in the clinic. Average dose per prophylactic infusion, per month and per year are reported as categories. | Throughout Part A of the study, approximately 5 years |
| Weight-adjusted Consumption of BAX 855: Average Number of Prophylactic Infusions | Weight-adjusted consumption of BAX 855 was determined based upon the record in participants diaries of the actual number of BAX 855 infusions as measured in the clinic. Average number of infusions per month and year are reported as categories. | Throughout Part A and Part B of the study, approximately 9 years |
| Weight-adjusted Consumption of BAX 855: Average Dose | Weight-adjusted consumption of BAX 855 was determined based upon the record in participants diaries of the actual amount of BAX 855 infused as measured in the clinic. Average dose to treat bleeding episode and average FVIII inhibitor treatment Dose [IU/kg] per Week, Month and per Year are reported as categories. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen. | Throughout Part A of the study, approximately 5 years |
| Number of Participants by Hemostatic Efficacy Rating in Case of Surgery | Hemostatic efficacy was assessed during & after any surgical or invasive procedures,& overall as a perioperative assessment.Operating surgeon assessed hemostatic efficacy compared to that expected for the type of procedure performed in non-hemophilic population,prior to discharge from recovery room(intraoperative),on postoperative Day 1 & at discharge or 14 days post-surgery(perioperative).Participants rated efficacy using following ratings:1.Excellent:Postoperative blood loss was ≤100% than expected;2.Good:Postoperative blood loss was up to 50% more (101-150%) than expected;3.Fair:Postoperative blood loss was more than 50% (>150%) of that expected;4.None:Significant postoperative bleeding that was result of inadequate therapeutic response despite proper dosing,necessitating rescue therapy.Perioperative ratings also considered amount of blood components required for transfusions compared to expected.Participant-provided ratings for each of the assessments are reported as categories. | Surgery Day 0 up to postoperative Day 14 or discharge (whichever occurs first) |
| Blood Loss Per Participant in Case of Surgery | The intraoperative blood loss was measured by determining the volume of blood and fluid removal through suction into the collection container (waste box and/or cell saver) and the estimated blood loss into swabs and towels during the procedure, per the anesthesiologist's record. Post-operatively, blood loss was determined by the drainage volume collected, which mainly consisted of drainage fluid via vacuum or gravity drain, as applicable. The assessment was done for the intra-operative time period (prior to discharge from recovery room) and for the post-operative time period (from completion of the procedure until approximately 24 hours post-surgery). | Surgery Day 0 up to postoperative Day 14 or discharge (whichever occurs first) |
| Incremental Recovery (IR) of BAX 855 | BAX 855 was administered in participants for the determination of FVIII IR at study site at baseline & every study visit other than study visits at 5 EDs, 15 EDs & 30 EDs. FVIII assays were done using following methods:1-stage clotting FVIII activity & FVIII chromogenic activity. Data is reported for each of these methods as categories per visit. Study Completion assessment was conducted at end of the Main Study & again at end of the ITI portion. Thus, more number of participants were analyzed at study completion visit than those who actually completed the overall study. IR is reported as a ratio of (IU/deciliter [dL])/(IU/kg), calculated as: IR = (Cmax- (C pre-infusion)) / (IU/kg), where C=concentration. Visits and their approximate time in weeks after baseline visit for individual participants: Visit 1 (Week 5), Visit 2 (Week 10), Visit 3 (Week 15), Visit 4 (Week 20), Visit 5 (Week 30), Visit 6 (Week 40), Visit 7 (Week 55), Visit 8 (Week 75) & Study Completion Visit (Weeks 100-110). | Baseline up to Study Completion (Up to 5 years in Part A and up to 3.5 years in Part B) |
| Half-life (T1/2) of BAX 855 | The Half-life to determine FVIII half-life was an optional assessment that was planned to be performed at baseline, Visit 1, or Visit 2. | Pre-infusion, Post-infusion: 15-30 minutes and 24-48 hours at Baseline |
| Immune Tolerance Induction (ITI) - Number of Participants With Partial Success and Failure of ITI | Partial success defined as which meet two of following criteria, 1) inhibitor titer <0.6 BU (confirmed by a central laboratory with a second blood specimen obtained within 2 months), 2) FVIII in vivo recovery >=66% of baseline value (confirmed within a two month period), and 3) FVIII half-life >=6 hours. Failure defined as the failure to meet the criteria for partial success. | Up to 33 months in Part B of the study |
| Immune Tolerance Induction (ITI) - Number of Participants With At Least One Catheter-related Complication | Number of participants with catheter-related complications are reported. | Up to 33 months in Part B of the study |
| Immune Tolerance Induction (ITI) - Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies | Binding IgG and IgM antibodies to Factor VIII (FVIII), Factor VIII-Polyethylene glycol (PEG-FVIII) and Polyethylene glycol (PEG) are reported as categories per visit. | Up to 33 months in Part B of the study |
| Cupertino |
| California |
| 95014 |
| United States |
| Kaiser Permanente Oakland M.C. | Oakland | California | 94611 | United States |
| Kaiser Permanente Oakland M.C. | Roseville | California | 95661 | United States |
| UC Davis Health System | Sacramento | California | 95817 | United States |
| Connecticut Children's Med Ctr | Hartford | Connecticut | 06106 | United States |
| Univ Florida College Medicine | Gainesville | Florida | 17033-0850 | United States |
| Center for Advanced Pediatrics | Atlanta | Georgia | 30322 | United States |
| Ann & Robert H. Lurie Children's H | Chicago | Illinois | 60611-2605 | United States |
| Bleeding and Clotting Dis.Inst. | Peoria | Illinois | 61615 | United States |
| UMHS | Ann Arbor | Michigan | 48109-5008 | United States |
| New York Presbyterian Hospital | New York | New York | 10065 | United States |
| Novant Health Presbyterian Medical Center | Charlotte | North Carolina | 28204 | United States |
| Wake Forest Baptist Medical Center | Winston-Salem | North Carolina | 27157 | United States |
| Cincinnati Children's Hospital | Cincinnati | Ohio | 45229-3039 | United States |
| Rainbow Babies/Childrens Htl | Cleveland | Ohio | 44106 | United States |
| Penn State MS Hershey Med Ctr | Hershey | Pennsylvania | 17033-0850 | United States |
| Texas Tech University Health Sciences Center | El Paso | Texas | 79905 | United States |
| Texas Children's Hospital | Houston | Texas | 77030 | United States |
| Primary Children's Hospital | Salt Lake City | Utah | 84113 | United States |
| Medizinische Universitat Wien | Vienna | 1090 | Austria |
| HUDERF | Brussels | 1020 | Belgium |
| Cliniques Uni Saint-Luc | Brussels | 1200 | Belgium |
| Univ. Ziekenhuis Gent Apotheek | Ghent | 9000 | Belgium |
| Universitair Ziekenhuis Leuven | Leuven | 3000 | Belgium |
| UMHAT Sv. Georgi, EAD | Plovdiv | 4000 | Bulgaria |
| SHAT Oncohaematology Diseases | Sofia | 1527 | Bulgaria |
| MHAT Sv. Marina, EAD | Varna | 9010 | Bulgaria |
| Kaye Edmonton Clinic | Edmonton | Alberta | T6G 1Z1 | Canada |
| McMaster Health Science | Hamilton | Ontario | L8N 3Z5 | Canada |
| Rigshospitalet Copenhagen | Copenhagen | 2100 | Denmark |
| Helsinki Univ Hospital | Helsinki | 00290 | Finland |
| CHU CAEN Hopital Cote de Nacre | Caen | Calvados | 14033 | France |
| Essais cliniques CHU Rennes | Rennes | Ille Et Vilaine | 35033 | France |
| Hopital Necker Enfants Malades | Paris | Paris | 75743 | France |
| Hopital Jeanne de Flandre - CHU Lille | Lille | 59037 | France |
| CHU de Rouen | Rouen | 76031 | France |
| Werlhof-Institut GmbH | Hanover | Lower Saxony | 30159 | Germany |
| Inst. f. Experimentelle | Bonn | 53127 | Germany |
| Klinik F.Haematologie,Onkologie | Düsseldorf | 40225 | Germany |
| Poliklinik PaediaHaematologie | Hamburg | 20246 | Germany |
| The University of Hong Kong Queen Mary Hospital | Hong Kong | Hong Kong |
| Chinese University Of Hong Kong | Shatin | Hong Kong |
| Belgyogyaszat Onkohaematologia | Budapest | 1086 | Hungary |
| Debreceni Egyetem | Debrece | 4032 | Hungary |
| Presidio Ospedaliero F. Alessi | Catania | 95124 | Italy |
| Azienda Ospedaliera Universitaria Careggi | Florence | 50134 | Italy |
| Ospedale Maggiore Policlinico | Milan | 20122 | Italy |
| Umberto I Pol. di Roma-Università di Roma La Sapienza | Rome | 00144 | Italy |
| Hospital Ampang | Ampang | Kuala Lumpur | 68000 | Malaysia |
| Hospital HRPB | Ipoh | Perak | 30990 | Malaysia |
| Hospital Pulau Pinang | George Town | Pulau Pinang | 10990 | Malaysia |
| Hospital Kuala Lumpur | Kuala Lumpur | 50586 | Malaysia |
| Hospital Umum Sarawak | Kuching | 93586 | Malaysia |
| Hospital Sultanah Nur Zahirah | Terengganu | 20400 | Malaysia |
| Universitair Medisch Centrum Groningen (UMCG) | Groningen | 9713 GZ | Netherlands |
| Oslo Universitetssykehus - Rikshospitalet | Oslo | N-0372 | Norway |
| NUS YLL School of Medicine | Singapore | 117599 | Singapore |
| KKH | Singapore | 229899 | Singapore |
| Eulji University Hospital | Daejeon | 35233 | South Korea |
| Severance Hospital, Yonsei | Seoul | 03722 | South Korea |
| Kyung Hee University Hospital | Seoul | 05278 | South Korea |
| Ulsan University Hospital | Ulsan | 44033 | South Korea |
| Hospital Univ. Son Espases | Palma de Mallorca | Balearic Islands | 07120 | Spain |
| HOSPITAL A Coruna | A Coruña | 15006 | Spain |
| Hospital Universitario La Paz | Madrid | 28046 | Spain |
| Hospital Univ del Rio Hortega | Valladolid | 47012 | Spain |
| Kaohsiung Chung- Ho Memorial Hosp | Kaohsiung City | 80756 | Taiwan |
| China Medical University Hospital | Taichung | 40447 | Taiwan |
| Taichung Veterans General | Taichung | 40705 | Taiwan |
| Tri-Service General Hospital | Taipei | 11490 | Taiwan |
| Siriraj Hospital | Bangkoknoi | Bangkok | 10700 | Thailand |
| King Chulalongkorn Memorial | Patumwan | Bangkok | 10330 | Thailand |
| Ramathibodi Hospital | Ratchathewi | Bangkok | 10400 | Thailand |
| Srinagarind Hospital | Muang | Changwat Khon Kaen | 40002 | Thailand |
| Maharaj Nakorn Chiang Mai | Muang | Chiang Mai | 50200 | Thailand |
| Acibadem Adana Hospital | Adana | 1130 | Turkey (Türkiye) |
| Hacettepe Üniversitesi | Ankara | 06100 | Turkey (Türkiye) |
| Akdeniz Universitesi | Antalya | 7058 | Turkey (Türkiye) |
| Uludag Universitesi Tip Fakültesi | Bursa | 16059 | Turkey (Türkiye) |
| Istanbul Üniversitesi Cerrahpaşa | Istanbul | 34098 | Turkey (Türkiye) |
| Ege Universitesi Tip Fakultesi | Izmir | 35040 | Turkey (Türkiye) |
| Erciyes Univers Tip Fakultesi | Kayseri | 38039 | Turkey (Türkiye) |
| 19 Mayis Universitesi | Samsun | 55319 | Turkey (Türkiye) |
| MI Cherkasy Reg Onc Dis of CRC | Cherkasy | 18009 | Ukraine |
| SI Institute of Blood Pathology and Transfusion Medicine of NAMSU | Lviv | 79044 | Ukraine |
| CI Zaporizhzhia Reg CCH of ZRC | Zaporizhzhia | 69063 | Ukraine |
| Royal Manchester Children's Hospital | Manchester | Greater Manchester | M13 9WL | United Kingdom |
| Univ Hospital Southampton | Southampton | Hampshire | SO16 6YD | United Kingdom |
| Bristol Royal H. for Children | Bristol | BS2 8AE | United Kingdom |
| Evelina Children's Hospital - St Thomas' Hospital | London | SE1 7EH | United Kingdom |
| Part A: On-demand Treatment | Participants who received at least one on-demand treatment of BAX 855 in Part A of the study. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen. |
|
| Part A: Prophylaxis Treatment | Participants who received at least one prophylaxis treatment of BAX 855 in Part A of the study. |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Part B: ITI Portion (3.5 Years) |
|
The Safety Analysis Set (SAS) included all participants in the enrolled population with at least one BAX 855 infusion.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | All Participants | PUPs < 6 years of age with severe hemophilia A (FVIII < 1%) and < 3 EDs to ADVATE, BAX 855 or plasma transfusion were enrolled in a single arm group. Part A (Main Study): Participants age <3 years - who had not experienced two joint bleeds received on-demand treatment of 10-80 international units per kilogram (IU/kg) intravenously (IV) depending on the severity of the bleeding episode; and - who experienced maximum of two joint bleeds received prophylaxis treatment with dose of 25-80 IU/kg of BAX 855 IV (based on investigator discretion) once weekly for up to 100 EDs. Part B (ITI Portion): Participants who met the pre-defined Part B treatment criteria entered Part B of the study for ITI. Participants either received prophylaxis treatment of BAX 855 low dose 50 IU/kg IV, three times a week or high dose 100-200 IU/kg IV, daily at the discretion of the investigator according to the institution's standard of care. |
| 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 FVIII Inhibitor Development | Number of participants who developed an inhibitor (at any time) confirmed by a central laboratory based on a second repeat blood sample draw within 2 weeks of site notification of an inhibitor and all participants who had not developed an inhibitor and had greater than or equal to (>=) 100 EDs when the sample for the last valid inhibitor test was drawn. | The SAS included all participants in the enrolled population with at least one BAX 855 infusion. Included in the analysis were participants who had equal or greater than 100 EDs or developed a confirmed FVIII inhibitor. | Posted | Count of Participants | Participants | Throughout Part A of the study, approximately 5 years |
|
|
| ||||||||||||||||||||||||||
| Primary | Number of Participants With Success of Immune Tolerance Induction (ITI) | Success is defined as 1) a persistently negative inhibitor titer less than (<) 0.6 Bethesda unit (BU), 2) FVIII IR >=66% of the baseline value following a wash-out period of 84-96 hours, and 3) a FVIII half-life of >=6 hours. | The FVIII Inhibitor Treatment Analysis Set (IAS) included all participants who received at least one FVIII inhibitor treatment with BAX 855 during the study after the date that participant moved to FVIII inhibitor treatment. | Posted | Count of Participants | Participants | Up to 33 months in Part B of the study |
|
| |||||||||||||||||||||||||||
| Secondary | Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies | Binding IgG and IgM antibodies to FVIII , Factor VIII-Polyethylene glycol (PEG-FVIII) and Polyethylene glycol (PEG) was assessed. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen. Visits and their approximate time in weeks after baseline visit for individual participants: Visit 1(Week 5), Visit 2 (Week 10), Visit 3 (Week 15), Visit 4 (Week 20), Visit 5 (Week 30), Visit 6 (Week 40), Visit 7 (Week 55), Visit 8 (Week 75) and Study Completion Visit (Weeks 100-110). | The SAS included all participants in the enrolled population with at least one BAX 855 infusion. Number analyzed is the number of participants with data available for analyses. | Posted | Count of Participants | Participants | Throughout Part A of the study, approximately 5 years |
| ||||||||||||||||||||||||||||
| Secondary | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | An AE is defined as any untoward medical occurrence in a participant administered an investigational product (IP) that does not necessarily have a causal relationship with the treatment. A SAE is any untoward clinical manifestation of signs, symptoms or outcomes (whether considered related to investigational product or not and at any dose) which results in death, is life-threatening, requires inpatient hospitalization, prolongation of hospitalization, is an important medical event. Number of participants with AEs and SAEs in both Part A and Part B were assessed. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen. | The SAS included all participants in the enrolled population with at least one BAX 855 infusion for Part A of the study and the IAS included all participants who received at least one FVIII inhibitor treatment with BAX 855 during the study after the date that participant moved to FVIII inhibitor treatment for Part B of the study. | Posted | Count of Participants | Participants | Throughout Part A and Part B of the study, approximately 9 years |
| ||||||||||||||||||||||||||||
| Secondary | Number of Participants With At Least One Clinically Significant Changes in Vital Signs | Vital signs were assessed based on body temperature, respiratory rate, blood pressure, and heart rate. | The SAS included all participants in the enrolled population with at least one BAX 855 infusion for Part A of the study and the IAS included all participants who received at least one FVIII inhibitor treatment with BAX 855 during the study after the date that participant moved to FVIII inhibitor treatment for Part B of the study. | Posted | Count of Participants | Participants | Throughout Part A and Part B of the study, approximately 9 years |
| ||||||||||||||||||||||||||||
| Secondary | Number of Participants With At Least One Clinically Significant Changes in Clinical Laboratory Parameters | Clinical laboratory parameters included hematology and clinical chemistry. Changes in laboratory values could be considered as AE if they were judged to be clinically significant. | The SAS included all participants in the enrolled population with at least one BAX 855 infusion for Part A of the study and the IAS included all participants who received at least one FVIII inhibitor treatment with BAX 855 during the study after the date that participant moved to FVIII inhibitor treatment for Part B of the study. | Posted | Count of Participants | Participants | Throughout Part A and Part B of the study, approximately 9 years |
| ||||||||||||||||||||||||||||
| Secondary | Annualized Bleeding Rate (ABR) for Prophylactic and On-demand Treatment and Immune Tolerance Induction (ITI) | 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 (example, knee and ankle bleed following a fall) was counted as a single bleeding episode. Mean total annualized bleed rate is reported. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen. | The SAS used for included all participants in the enrolled population with at least one BAX 855 infusion for Part A of the study and the IAS included all participants who received at least one FVIII inhibitor treatment with BAX 855 during the study after the date that participant moved to FVIII inhibitor treatment for Part B of the study. | Posted | Mean | Standard Deviation | unique bleeds per year | Throughout Part A and Part B of the study, approximately 9 years |
| |||||||||||||||||||||||||||
| Secondary | Bleeding Episodes Categorized by Number of BAX 855 Infusions Required for Treatment | A bleeding episode is defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. The number of BAX 855 infusions needed for each bleeding episode was determined by the participant, caregiver, clinician treating the participant, and is based upon the participant's response to treatment, using the Efficacy Rating Scale for Treatment of Bleeding Episodes. Number of bleeding episodes are categorized by number of infusions required to treat the bleeding episodes. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen. | The SAS included all participants in the enrolled population with at least one BAX 855 infusion. | Posted | Number | bleeding episodes | Throughout Part A of the study, approximately 5 years | Number of treated bleeds | Number of treated bleeds |
| ||||||||||||||||||||||||||
| Secondary | Number of Bleeds by Overall Hemostatic Efficacy Rating at 24 Hours After Initiation of Treatment | The participant or caregiver rated overall treatment response using a 4-point efficacy rating scale as Excellent:Full relief of pain & cessation of objective signs of bleeding after single infusion & no additional infusion is required for the control of bleeding; Good:Definite pain relief &/or improvement in signs of bleeding after single infusion & possibly requires more than 1 infusion for complete resolution; Fair:Probable &/or slight relief of pain & slight improvement in signs of bleeding after single infusion & required more than 1 infusion for complete resolution & None:No improvement or condition worsens.Number of bleeds with each efficacy rating are reported.Participant that received on-demand treatment first & then moved to prophylaxis treatment was counted for both on-demand & prophylaxis regimens.Participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on prophylaxis regimen. | The SAS included all participants in the enrolled population with at least one BAX 855 infusion. The number of units analyzed indicates the number of treated bleeds for which efficacy rating at 24 hours after initiation of treatment was available. | Posted | Number | bleeds | At 24 hours after study drug administration during Part A of the study | Number of treated bleeds | Number of treated bleeds |
| ||||||||||||||||||||||||||
| Secondary | Number of Bleeds by Overall Hemostatic Efficacy Rating at Bleed Resolution | The participant or caregiver rated overall treatment response using a 4-point efficacy rating scale as Excellent:Full relief of pain & cessation of objective signs of bleeding after single infusion & no additional infusion is required for the control of bleeding; Good:Definite pain relief &/or improvement in signs of bleeding after a single infusion & possibly requires more than 1 infusion for complete resolution; Fair:Probable &/or slight relief of pain & slight improvement in signs of bleeding after single infusion & required more than 1 infusion for complete resolution & None:No improvement or condition worse.Number of bleeds with each efficacy rating are reported.Participant that received on-demand treatment first & then moved to prophylaxis treatment was counted for both on-demand & prophylaxis regimens.Participant that started with prophylaxis treatment was counted only for prophylaxis regimen even if the participant received on-demand treatment while on prophylaxis regimen. | The SAS included all participants in the enrolled population with at least one BAX 855 infusion. The number of units analyzed indicates the number of treated bleeds for which overall efficacy rating was available. | Posted | Number | bleeds | From start of study treatment up to bleed resolution throughout Part A of the study (up to approximately 5 years) | Number of treated bleeds | Number of treated bleeds |
| ||||||||||||||||||||||||||
| Secondary | Weight-adjusted Consumption of BAX 855: Average Prophylactic Dose | Weight-adjusted consumption of BAX 855 was determined based upon the record in participants diaries of the actual amount of BAX 855 infused as measured in the clinic. Average dose per prophylactic infusion, per month and per year are reported as categories. | The SAS included all participants in the enrolled population with at least one BAX 855 infusion. Number analyzed for each category is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | IU/kg | Throughout Part A of the study, approximately 5 years |
|
| ||||||||||||||||||||||||||
| Secondary | Weight-adjusted Consumption of BAX 855: Average Number of Prophylactic Infusions | Weight-adjusted consumption of BAX 855 was determined based upon the record in participants diaries of the actual number of BAX 855 infusions as measured in the clinic. Average number of infusions per month and year are reported as categories. | The SAS included all participants in the enrolled population with at least one BAX 855 infusion. Number analyzed for each category is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | infusions | Throughout Part A and Part B of the study, approximately 9 years |
|
| ||||||||||||||||||||||||||
| Secondary | Weight-adjusted Consumption of BAX 855: Average Dose | Weight-adjusted consumption of BAX 855 was determined based upon the record in participants diaries of the actual amount of BAX 855 infused as measured in the clinic. Average dose to treat bleeding episode and average FVIII inhibitor treatment Dose [IU/kg] per Week, Month and per Year are reported as categories. A participant that received on-demand treatment first and then moved to prophylaxis treatment was counted for both on-demand and prophylaxis regimens. A participant that started with prophylaxis treatment was counted only for the prophylaxis regimen even if the participant received on-demand treatment while on the prophylaxis regimen. | The SAS included all participants in the enrolled population with at least one BAX 855 infusion for Part A of the study and the IAS included all participants who received at least one FVIII inhibitor treatment with BAX 855 during the study after the date that participant moved to FVIII inhibitor treatment for Part B of the study. Number analyzed for each category is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | IU/kg | Throughout Part A of the study, approximately 5 years |
| |||||||||||||||||||||||||||
| Secondary | Number of Participants by Hemostatic Efficacy Rating in Case of Surgery | Hemostatic efficacy was assessed during & after any surgical or invasive procedures,& overall as a perioperative assessment.Operating surgeon assessed hemostatic efficacy compared to that expected for the type of procedure performed in non-hemophilic population,prior to discharge from recovery room(intraoperative),on postoperative Day 1 & at discharge or 14 days post-surgery(perioperative).Participants rated efficacy using following ratings:1.Excellent:Postoperative blood loss was ≤100% than expected;2.Good:Postoperative blood loss was up to 50% more (101-150%) than expected;3.Fair:Postoperative blood loss was more than 50% (>150%) of that expected;4.None:Significant postoperative bleeding that was result of inadequate therapeutic response despite proper dosing,necessitating rescue therapy.Perioperative ratings also considered amount of blood components required for transfusions compared to expected.Participant-provided ratings for each of the assessments are reported as categories. | The Invasive Procedure Analysis Set (IPRAS) included all participants who were treated with BAX 855 for one or more surgeries or invasive procedures in the context of the study. Number of participants analyzed is the number of participants with data available for analyses. | Posted | Count of Participants | Participants | Surgery Day 0 up to postoperative Day 14 or discharge (whichever occurs first) |
| ||||||||||||||||||||||||||||
| Secondary | Blood Loss Per Participant in Case of Surgery | The intraoperative blood loss was measured by determining the volume of blood and fluid removal through suction into the collection container (waste box and/or cell saver) and the estimated blood loss into swabs and towels during the procedure, per the anesthesiologist's record. Post-operatively, blood loss was determined by the drainage volume collected, which mainly consisted of drainage fluid via vacuum or gravity drain, as applicable. The assessment was done for the intra-operative time period (prior to discharge from recovery room) and for the post-operative time period (from completion of the procedure until approximately 24 hours post-surgery). | The IPRAS included all participants who were treated with BAX 855 for one or more surgeries or invasive procedures in the context of the study. Number analyzed are the participants who experienced blood loss. | Posted | Mean | Standard Deviation | milliliters (mL) | Surgery Day 0 up to postoperative Day 14 or discharge (whichever occurs first) |
| |||||||||||||||||||||||||||
| Secondary | Incremental Recovery (IR) of BAX 855 | BAX 855 was administered in participants for the determination of FVIII IR at study site at baseline & every study visit other than study visits at 5 EDs, 15 EDs & 30 EDs. FVIII assays were done using following methods:1-stage clotting FVIII activity & FVIII chromogenic activity. Data is reported for each of these methods as categories per visit. Study Completion assessment was conducted at end of the Main Study & again at end of the ITI portion. Thus, more number of participants were analyzed at study completion visit than those who actually completed the overall study. IR is reported as a ratio of (IU/deciliter [dL])/(IU/kg), calculated as: IR = (Cmax- (C pre-infusion)) / (IU/kg), where C=concentration. Visits and their approximate time in weeks after baseline visit for individual participants: Visit 1 (Week 5), Visit 2 (Week 10), Visit 3 (Week 15), Visit 4 (Week 20), Visit 5 (Week 30), Visit 6 (Week 40), Visit 7 (Week 55), Visit 8 (Week 75) & Study Completion Visit (Weeks 100-110). | The Pharmacokinetic (PK) analysis set (PKAS) included all participants in the SAS who had at least one post-dose measurement of FVIII activity without protocol deviations and/or events with potential to affect concentration (FVIII activity levels). Number analyzed is the number of participants with data available for analyses. | Posted | Geometric Mean | Geometric Coefficient of Variation | ratio | Baseline up to Study Completion (Up to 5 years in Part A and up to 3.5 years in Part B) |
| |||||||||||||||||||||||||||
| Secondary | Half-life (T1/2) of BAX 855 | The Half-life to determine FVIII half-life was an optional assessment that was planned to be performed at baseline, Visit 1, or Visit 2. | As pre-specified in the protocol, the determination of FVIII half-life by abbreviated PK at baseline was optional and was not performed. | Posted | Pre-infusion, Post-infusion: 15-30 minutes and 24-48 hours at Baseline |
|
| |||||||||||||||||||||||||||||
| Secondary | Immune Tolerance Induction (ITI) - Number of Participants With Partial Success and Failure of ITI | Partial success defined as which meet two of following criteria, 1) inhibitor titer <0.6 BU (confirmed by a central laboratory with a second blood specimen obtained within 2 months), 2) FVIII in vivo recovery >=66% of baseline value (confirmed within a two month period), and 3) FVIII half-life >=6 hours. Failure defined as the failure to meet the criteria for partial success. | FVIII inhibitor treatment analysis set (IAS) included all participants who received at least one FVIII inhibitor treatment with BAX 855 during the study after the date that participant moved to FVIII inhibitor treatment. | Posted | Count of Participants | Participants | Up to 33 months in Part B of the study |
|
| |||||||||||||||||||||||||||
| Secondary | Immune Tolerance Induction (ITI) - Number of Participants With At Least One Catheter-related Complication | Number of participants with catheter-related complications are reported. | IAS included all participants who received at least one FVIII inhibitor treatment with BAX 855 during the study after the date that participant moved to FVIII inhibitor treatment for Part B of the study. | Posted | Count of Participants | Participants | Up to 33 months in Part B of the study |
|
| |||||||||||||||||||||||||||
| Secondary | Immune Tolerance Induction (ITI) - Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies | Binding IgG and IgM antibodies to Factor VIII (FVIII), Factor VIII-Polyethylene glycol (PEG-FVIII) and Polyethylene glycol (PEG) are reported as categories per visit. | The IAS included all participants who received at least one FVIII inhibitor treatment with BAX 855 during the study after the date that participant moved to FVIII inhibitor treatment. | Posted | Count of Participants | Participants | Up to 33 months in Part B of the study |
|
|
Up to approximately 9 years
SAS:participants in enrolled population with at least 1 BAX 855 infusion.IAS:participants who received at least 1 FVIII inhibitor treatment with BAX 855 during study after participant moved to FVIII inhibitor treatment.Participant that received on-demand treatment 1st and then moved to prophylaxis treatment was counted for both on-demand & prophylaxis regimens and those who started with prophylaxis treatment was counted only for the prophylaxis regimen even if they received on-demand treatment.
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 | Part A: Main Study: On-demand | Participants age <3 years and who had not experienced two joint bleeds received on-demand treatment of 10-80IU/kg of BAX 855 IV depending on the severity of the bleeding episode. | 0 | 80 | 18 | 80 | 47 | 80 |
| EG001 | Part A: Main Study: Prophylaxis | Participants age <3 years or after a maximum of two joint bleeds received prophylaxis treatment with dose of 25-80 IU/kg of BAX 855 IV (based on investigator discretion) once weekly for up to 100 EDs. | 0 | 112 | 35 | 112 | 77 | 112 |
| EG002 | Part B: ITI Portion (50 IU/kg Three Times Weekly Regimen) | Participants received prophylaxis treatment of 50 IU/kg BAX 855 IV three times in a week. | 0 | 4 | 1 | 4 | 4 | 4 |
| EG003 | Part B: ITI Portion (100-200 IU/kg Daily Regimen) | Participants received prophylaxis treatment of 100-200 IU/kg BAX 855 IV daily. | 0 | 3 | 3 | 3 | 2 | 3 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Accidental exposure to product | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Bacterial infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Croup infectious | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Device related sepsis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Extradural haematoma | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Factor VIII inhibition | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Febrile convulsion | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Haemarthrosis | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Haematoma muscle | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Haemorrhage | Vascular disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Haemorrhage intracranial | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Immune thrombocytopenia | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Intussusception | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Irritability | Psychiatric disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Lip injury | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Mouth haemorrhage | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Mouth injury | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Muscle haemorrhage | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Nail bed bleeding | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pharyngeal haemorrhage | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Pneumonia influenzal | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Strabismus | Eye disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Subcutaneous haematoma | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Systemic viral infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Tongue haemorrhage | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Tongue injury | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Tongue ulceration | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Vascular device infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Vascular device occlusion | General disorders | MedDRA 27.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Bacillus test positive | Investigations | MedDRA 27.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Dermatitis allergic | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Oral discomfort | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Scarlet fever | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Skin wound | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Vascular device infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Vessel puncture site cellulitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Takeda | +1-877-825-3327 | TrialDisclosures@takeda.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Dec 5, 2024 | Jun 6, 2025 | SAP_001.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
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
|
|
| OG001 | Part A: Main Study: Prophylaxis | Participants age <3 years or after a maximum of two joint bleeds received prophylaxis treatment with dose of 25-80 IU/kg of BAX 855 IV (based on investigator discretion) once weekly for up to 100 EDs. |
| OG002 | Part B: ITI Portion (50 IU/kg Three Times Weekly Regimen) | Participants received prophylaxis treatment of 50 IU/kg BAX 855 IV three times in a week. |
| OG003 | Part B: ITI Portion (100-200 IU/kg Daily Regimen) | Participants received prophylaxis treatment of 100-200 IU/kg BAX 855 IV daily. |
|
|
|
|
|
|
| Part A: Main Study: Prophylaxis |
Participants age <3 years or after a maximum of two joint bleeds received prophylaxis treatment with dose of 25-80 IU/kg of BAX 855 IV (based on investigator discretion) once weekly for up to 100 EDs. |
| OG002 | Part B: ITI Portion (50 IU/kg Three Times Weekly Regimen) | Participants received prophylaxis treatment of 50 IU/kg BAX 855 IV three times in a week. |
| OG003 | Part B: ITI Portion (100-200 IU/kg Daily Regimen) | Participants received prophylaxis treatment of 100-200 IU/kg BAX 855 IV daily. |
|
|
Participants age <3 years or after a maximum of two joint bleeds received prophylaxis treatment with dose of 25-80 IU/kg of BAX 855 IV (based on investigator discretion) once weekly for up to 100 EDs.
|
|
| OG001 | Part A: Main Study: Prophylaxis | Participants age <3 years or after a maximum of two joint bleeds received prophylaxis treatment with dose of 25-80 IU/kg of BAX 855 IV (based on investigator discretion) once weekly for up to 100 EDs. |
|
|
| OG001 | Part A: Main Study: Prophylaxis | Participants age <3 years or after a maximum of two joint bleeds received prophylaxis treatment with dose of 25-80 IU/kg of BAX 855 IV (based on investigator discretion) once weekly for up to 100 EDs. |
|
|
|
|
Participants age <3 years or after a maximum of two joint bleeds received prophylaxis treatment with dose of 25-80 IU/kg of BAX 855 IV (based on investigator discretion) once weekly for up to 100 EDs.
| OG002 | Part B: ITI Portion (50 IU/kg Three Times Weekly Regimen) | Participants received prophylaxis treatment of 50 IU/kg BAX 855 IV three times in a week. |
| OG003 | Part B: ITI Portion (100-200 IU/kg Daily Regimen) | Participants received prophylaxis treatment of 100-200 IU/kg BAX 855 IV daily. |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|