Trial · NCT02615691
A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A
NCT02615691, Phase 3, Completed, Baxalta now part of Shire; results first posted 2025-07-28
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A. Trials, record NCT02615691. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02615691
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A
- NCT number
- NCT02615691
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 120 participants, actual
- Lead sponsor
- Baxalta now part of Shire
- Conditions
- Hemophilia A
- Interventions
- PEGylated Recombinant Factor VIII, ITI
- First posted
- 2015-11-26
- Last updated
- 2025-07-28
- Other registries' IDs
- eudract:2015-002136-40
- Results first posted
- 2025-07-28
Source ClinicalTrials.gov
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With FVIII Inhibitor Development | Throughout Part A of the study, approximately 5 years | 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. |
| Primary | Number of Participants With Success of Immune Tolerance Induction (ITI) | Up to 33 months in Part B of the study | 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. |
| Secondary | Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies | Throughout Part A of the study, approximately 5 years | 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). |
| Secondary | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Throughout Part A and Part B of the study, approximately 9 years | 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. |
| Secondary | Number of Participants With At Least One Clinically Significant Changes in Vital Signs | Throughout Part A and Part B of the study, approximately 9 years | Vital signs were assessed based on body temperature, respiratory rate, blood pressure, and heart rate. |
| Secondary | Number of Participants With At Least One Clinically Significant Changes in Clinical Laboratory Parameters | Throughout Part A and Part B of the study, approximately 9 years | 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. |
| Secondary | Annualized Bleeding Rate (ABR) for Prophylactic and On-demand Treatment and Immune Tolerance Induction (ITI) | Throughout Part A and Part B of the study, approximately 9 years | 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. |
| Secondary | Bleeding Episodes Categorized by Number of BAX 855 Infusions Required for Treatment | Throughout Part A of the study, approximately 5 years | 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. |
| Secondary | Number of Bleeds by Overall Hemostatic Efficacy Rating at 24 Hours After Initiation of Treatment | At 24 hours after study drug administration during Part A of the study | 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. |
| Secondary | Number of Bleeds by Overall Hemostatic Efficacy Rating at Bleed Resolution | From start of study treatment up to bleed resolution throughout Part A of the study (up to approximately 5 years) | 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. |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 registered outcomesOutcomes20
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With FVIII Inhibitor Development | Throughout Part A of the study, approximately 5 years | 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. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Success of Immune Tolerance Induction (ITI) | Up to 33 months in Part B of the study | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies | Throughout Part A of the study, approximately 5 years | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Throughout Part A and Part B of the study, approximately 9 years | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With At Least One Clinically Significant Changes in Vital Signs | Throughout Part A and Part B of the study, approximately 9 years | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With At Least One Clinically Significant Changes in Clinical Laboratory Parameters | Throughout Part A and Part B of the study, approximately 9 years | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Annualized Bleeding Rate (ABR) for Prophylactic and On-demand Treatment and Immune Tolerance Induction (ITI) | Throughout Part A and Part B of the study, approximately 9 years | 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. | unique bleeds per year | MEAN |
| SECONDARY | Bleeding Episodes Categorized by Number of BAX 855 Infusions Required for Treatment | Throughout Part A of the study, approximately 5 years | The SAS included all participants in the enrolled population with at least one BAX 855 infusion. | bleeding episodes | NUMBER |
| SECONDARY | Number of Bleeds by Overall Hemostatic Efficacy Rating at 24 Hours After Initiation of Treatment | At 24 hours after study drug administration during Part A of the study | 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. | bleeds | NUMBER |
| SECONDARY | Number of Bleeds by Overall Hemostatic Efficacy Rating at Bleed Resolution | From start of study treatment up to bleed resolution throughout Part A of the study (up to approximately 5 years) | 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. | bleeds | NUMBER |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 outcomesResults by group508
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With FVIII Inhibitor Development | Part A: Main Study | 11 | Participants | ||
| Number of Participants With Success of Immune Tolerance Induction (ITI) | Part B: ITI Portion | 5 | Participants | ||
| Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies | Part A: Main Study: On-demand | Screening- Binding IgG Antibodies to FVIII | 1 | Participants | |
| Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies | Part A: Main Study: Prophylaxis | Screening- Binding IgG Antibodies to FVIII | 0 | Participants | |
| Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies | Part A: Main Study: On-demand | Screening- Binding IgM Antibodies to FVIII | 0 | Participants | |
| Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies | Part A: Main Study: Prophylaxis | Screening- Binding IgM Antibodies to FVIII | 0 | Participants | |
| Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies | Part A: Main Study: On-demand | Screening- Binding IgG Antibodies to PEG-FVIII | 3 | Participants | |
| Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies | Part A: Main Study: Prophylaxis | Screening- Binding IgG Antibodies to PEG-FVIII | 2 | Participants | |
| Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies | Part A: Main Study: On-demand | Screening- Binding IgM Antibodies to PEG-FVIII | 4 | Participants | |
| Number of Participants With Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies | Part A: Main Study: Prophylaxis | Screening- Binding IgM Antibodies to PEG-FVIII | 1 | Participants |
1 to 10 of 508Source ClinicalTrials.gov
See all 508 results by groupParticipant flow10
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part A: Main Study (5 Years) | STARTED | All Participants | 120 | |
| Part A: Main Study (5 Years) | Part A: On-demand Treatment | All Participants | 80 | |
| Part A: Main Study (5 Years) | Part A: Prophylaxis Treatment | All Participants | 112 | |
| Part A: Main Study (5 Years) | COMPLETED | All Participants | 106 | |
| Part A: Main Study (5 Years) | NOT COMPLETED | All Participants | 14 | |
| Part B: ITI Portion (3.5 Years) | STARTED | All Participants | 7 | |
| Part B: ITI Portion (3.5 Years) | Part B:FVII Inhibitor Treatment(50IU/kg 3 Times Weekly Regimen) | All Participants | 4 | |
| Part B: ITI Portion (3.5 Years) | Part B:FVII Inhibitor Treatment (100-200IU/kg Daily Regimen) | All Participants | 3 | |
| Part B: ITI Portion (3.5 Years) | COMPLETED | All Participants | 7 | |
| Part B: ITI Portion (3.5 Years) | NOT COMPLETED | All Participants | 0 |
Source ClinicalTrials.gov
Adverse events312
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Part A: Main Study: On-demand | 0 | 80 | |
| other | Anaemia | Blood and lymphatic system disorders | Part A: Main Study: Prophylaxis | 2 | 112 | |
| other | Anaemia | Blood and lymphatic system disorders | Part B: ITI Portion (50 IU/kg Three Times Weekly Regimen) | 0 | 4 | |
| other | Anaemia | Blood and lymphatic system disorders | Part B: ITI Portion (100-200 IU/kg Daily Regimen) | 1 | 3 | |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Part A: Main Study: On-demand | 0 | 80 | |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Part A: Main Study: Prophylaxis | 0 | 112 | |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Part B: ITI Portion (50 IU/kg Three Times Weekly Regimen) | 1 | 4 | |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Part B: ITI Portion (100-200 IU/kg Daily Regimen) | 0 | 3 | |
| other | Bacillus test positive | Investigations | Part A: Main Study: On-demand | 0 | 80 | |
| other | Bacillus test positive | Investigations | Part A: Main Study: Prophylaxis | 0 | 112 |
1 to 10 of 312Source ClinicalTrials.gov
See all 312 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part A: Main Study: On-demand | 0 | 80 |
| Total, all-cause mortality | Part A: Main Study: Prophylaxis | 0 | 112 |
| Total, all-cause mortality | Part B: ITI Portion (50 IU/kg Three Times Weekly Regimen) | 0 | 4 |
| Total, all-cause mortality | Part B: ITI Portion (100-200 IU/kg Daily Regimen) | 0 | 3 |
| Total, other adverse events | Part A: Main Study: On-demand | 47 | 80 |
| Total, other adverse events | Part A: Main Study: Prophylaxis | 77 | 112 |
| Total, other adverse events | Part B: ITI Portion (50 IU/kg Three Times Weekly Regimen) | 4 | 4 |
| Total, other adverse events | Part B: ITI Portion (100-200 IU/kg Daily Regimen) | 2 | 3 |
| Total, serious adverse events | Part A: Main Study: On-demand | 18 | 80 |
| Total, serious adverse events | Part A: Main Study: Prophylaxis | 35 | 112 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 37646148 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Part A: Main Study vs Part B: ITI Portion: Number of Participants With At Least One Clinically Significant Changes in Vital Signs | Trial comparisons |
Part B: ITI Portion (50 IU/kg Three Times Weekly Regimen) vs Part B: ITI Portion (100-200 IU/kg Daily Regimen): Immune Tolerance Induction (ITI) - Number of Participants With At Least One Catheter-related Complication | Trial comparisons |
| Part A: Main Study vs Part B: ITI Portion: Number of Participants With At Least One Clinically Significant Changes in Clinical Laboratory Parameters | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
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