Trial · NCT01311648
BAY81-8973 Pediatric Safety and Efficacy Trial
NCT01311648, Phase 3, Completed, Bayer; results first posted 2020-11-03
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. BAY81-8973 Pediatric Safety and Efficacy Trial. Trials, record NCT01311648. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01311648
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- BAY81-8973 Pediatric Safety and Efficacy Trial
- NCT number
- NCT01311648
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 94 participants, actual
- Lead sponsor
- Bayer
- Conditions
- Haemophilia A
- Interventions
- Recombinant Factor VIII (Kovaltry, BAY81-8973), Recombinant Factor VIII (Kovaltry, BAY81-8973)
- First posted
- 2011-03-09
- Last updated
- 2023-12-05
- Other registries' IDs
- eudract:2010-021781-29
- Results first posted
- 2020-11-03
- Limitations and caveats
Due to the small number of participants per reporting group, all presented summary measures (e.g. mean and proportion) have to be evaluated with caution. If displayed standard deviation should be taken into account. The participant who had a low titer inhibitor detected after 549 EDs during the extension study was considered to have a false positive result due to cross reactivity with IgG anticardiolipin antibodies.
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Annualized Number of Total Bleeds Within 48 h | Within 48 hours post infusion | Annualized number (mean +/- standard deviation) of total bleeds that occurred within 48 hours after all prophylaxis infusions (Part A: 6 months and at least 50 exposure days \[EDs\]; Part B: at least 50 EDs or until inhibitor development) was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other'). |
| Primary | Annualized Number of Total Bleeds Within 48 h | Within 48 hours post infusion | Annualized number (median \[inter-quartile range (Q1-Q3)\]) of total bleeds that occurred within 48 hours after all prophylaxis infusions (Part A: 6 months and at least 50 exposure days \[EDs\]; Part B: at least 50 EDs or until inhibitor development) was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other'). |
| Secondary | Annualized Number of Total Bleeds During Prophylaxis Treatment | Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months) | Annualized number (mean +/- standard deviation) of total bleeds that occurred during prophylaxis treatment was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other'). |
| Secondary | Annualized Number of Total Bleeds During Prophylaxis Treatment | Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months) | Annualized number (median \[inter-quartile range (Q1-Q3)\]) of total bleeds that occurred during prophylaxis treatment was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other'). |
| Secondary | Hemostatic Control During Major and Minor Surgeries | Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months) | For participants who underwent major or minor surgeries during the study, hemostasis during the surgeries was assessed as excellent, good, moderate or poor. Number of surgeries per assessment was summarized and reported. |
| Secondary | Number of Participants With Inhibitor Development in Main Study | Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months) | Number of participants with confirmed positive FVIII inhibitor titer (≥ 0.6 Bethesda unit \[BU/mL\]) during the main study was summarized and classified as participants developing low titer inhibitor (i.e. ≥ 0.6 to ≤ 5.0 BU/mL) and participants developing high titer inhibitor (i.e. \> 5.0 BU/mL). |
| Secondary | Number of Participants With New Inhibitor Development in Extension Study | From start of extension study to at least 100 cumulative exposure days (EDs) (median 421 EDs; median 3.8 years) | Number of participants who had not developed an inhibitor during the main study but developed an inhibitor (confirmed positive FVIII inhibitor titer \[≥ 0.6 BU/mL\]) during the extension study was summarized and classified as participants developing low titer inhibitor (i.e. ≥ 0.6 to ≤ 5.0 BU/mL) and participants developing high titer inhibitor (i.e. \> 5.0 BU/mL). |
| Secondary | Factor VIII Recovery Values | Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months) | Incremental recovery of Factor VIII (FVIII) at 20-30 min after end of infusions was determined and mean recovery values were reported. |
| Secondary | Consumption of Factor VIII in All Infusions | Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months) | Factor VIII (FVIII) usage/consumption was summarized for all infusions. Consumption per participant's body weight per year was calculated and reported. |
| Secondary | Consumption of FVIII in Infusions for Prophylaxis | Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months) | Factor VIII (FVIII) usage/consumption was summarized for prophylaxis infusions. Consumption per participant's body weight per year was calculated and reported. |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Annualized Number of Total Bleeds Within 48 h | Within 48 hours post infusion | Intent-to-treat (ITT) analysis set - main study: all participants of the SAF in main study who had infusion/bleeding data from the electronic patient diary (EPD) | Bleeds | MEAN |
| PRIMARY | Annualized Number of Total Bleeds Within 48 h | Within 48 hours post infusion | Intent-to-treat (ITT) analysis set - main study: all participants of the SAF in main study who had infusion/bleeding data from the electronic patient diary (EPD) | Bleeds | MEDIAN |
| SECONDARY | Annualized Number of Total Bleeds During Prophylaxis Treatment | Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months) | Intent-to-treat (ITT) analysis set - main study: all participants of the SAF in main study who had infusion/bleeding data from the electronic patient diary (EPD) | Bleeds | MEAN |
| SECONDARY | Annualized Number of Total Bleeds During Prophylaxis Treatment | Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months) | Intent-to-treat (ITT) analysis set - main study: all participants of the SAF in main study who had infusion/bleeding data from the electronic patient diary (EPD) | Bleeds | MEDIAN |
| SECONDARY | Hemostatic Control During Major and Minor Surgeries | Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months) | Participants in SAF in main study who underwent major or minor surgeries during the study | Surgeries | COUNT_OF_UNITS |
| SECONDARY | Number of Participants With Inhibitor Development in Main Study | Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months) | Participants in main study - safety analysis set (SAF, all participants who entered Study Part A or Part B and received at least one infusion of study medication) with inhibitor measurements done | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With New Inhibitor Development in Extension Study | From start of extension study to at least 100 cumulative exposure days (EDs) (median 421 EDs; median 3.8 years) | All participants who entered the extension study and had not developed inhibitors during the main study Part A or Part B | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Factor VIII Recovery Values | Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months) | Participants in intent-to-treat (ITT) analysis set in main study with valid FVIII recovery values | International unit (IU)/dL per IU/kg | MEAN |
| SECONDARY | Consumption of Factor VIII in All Infusions | Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months) | Intent-to-treat (ITT) analysis set - main study: all participants of the SAF in main study who had infusion/bleeding data from the electronic patient diary (EPD) | international units/kilogram/year | MEAN |
| SECONDARY | Consumption of FVIII in Infusions for Prophylaxis | Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months) | Participants in intent-to-treat (ITT) analysis set in main study with at least one dose of prophylaxis treatment with study drug | international units/kilogram/year | MEAN |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group83
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Annualized Number of Total Bleeds Within 48 h | Main Study - Part A: PTPs 0-<6 Years | 2.23 | Standard Deviation 2.77 | Bleeds | |
| Annualized Number of Total Bleeds Within 48 h | Main Study - Part A: PTPs 6-12 Years | 1.86 | Standard Deviation 3.08 | Bleeds | |
| Annualized Number of Total Bleeds Within 48 h | Main Study - Part B: PUPs/MTPs 0-<6 Years | 1.9 | Standard Deviation 3.3 | Bleeds | |
| Annualized Number of Total Bleeds Within 48 h | Main Study - Part A: PTPs 0-<6 Years | 1.88 | Inter-Quartile Range 0.00 | Bleeds | |
| Annualized Number of Total Bleeds Within 48 h | Main Study - Part A: PTPs 6-12 Years | 0.00 | Inter-Quartile Range 0.00 | Bleeds | |
| Annualized Number of Total Bleeds Within 48 h | Main Study - Part B: PUPs/MTPs 0-<6 Years | 0.0 | Inter-Quartile Range 0.0 | Bleeds | |
| Annualized Number of Total Bleeds During Prophylaxis Treatment | Main Study - Part A: PTPs 0-<6 Years | 4.16 | Standard Deviation 5.02 | Bleeds | |
| Annualized Number of Total Bleeds During Prophylaxis Treatment | Main Study - Part A: PTPs 6-12 Years | 3.37 | Standard Deviation 5.01 | Bleeds | |
| Annualized Number of Total Bleeds During Prophylaxis Treatment | Main Study - Part B: PUPs/MTPs 0-<6 Years | 7.1 | Standard Deviation 8.6 | Bleeds | |
| Annualized Number of Total Bleeds During Prophylaxis Treatment | Main Study - Part A: PTPs 0-<6 Years | 2.03 | Inter-Quartile Range 0.00 | Bleeds |
1 to 10 of 83Source ClinicalTrials.gov
See all 83 results by groupParticipant flow16
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Main Study | STARTED | PTPs 0-12 Years | 51 | |
| Main Study | STARTED | PUPs/MTPs 0-<6 Years | 43 | |
| Main Study | Started 0-<6 Years | PTPs 0-12 Years | 25 | |
| Main Study | Started 0-<6 Years | PUPs/MTPs 0-<6 Years | 43 | |
| Main Study | Started 6-12 Years | PTPs 0-12 Years | 26 | |
| Main Study | Started 6-12 Years | PUPs/MTPs 0-<6 Years | 0 | |
| Main Study | COMPLETED | PTPs 0-12 Years | 51 | |
| Main Study | COMPLETED | PUPs/MTPs 0-<6 Years | 22 | |
| Main Study | NOT COMPLETED | PTPs 0-12 Years | 0 | |
| Main Study | NOT COMPLETED | PUPs/MTPs 0-<6 Years | 21 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 participant flowAdverse events960
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 | Main Study - Part A: PTPs 0-<6 Years | 0 | 25 | 0 |
| other | Anaemia | Blood and lymphatic system disorders | Main Study - Part A: PTPs 6-12 Years | 1 | 26 | 3 |
| other | Anaemia | Blood and lymphatic system disorders | Main Study - Part B: PUPs/MTPs 0-<6 Years | 3 | 43 | 3 |
| other | Anaemia | Blood and lymphatic system disorders | Extension Study - Former Part A Participants | 0 | 46 | 0 |
| other | Anaemia | Blood and lymphatic system disorders | Extension Study - Former Part B Participants | 3 | 36 | 10 |
| other | Blood loss anaemia | Blood and lymphatic system disorders | Main Study - Part A: PTPs 0-<6 Years | 0 | 25 | 0 |
| other | Blood loss anaemia | Blood and lymphatic system disorders | Main Study - Part A: PTPs 6-12 Years | 0 | 26 | 0 |
| other | Blood loss anaemia | Blood and lymphatic system disorders | Main Study - Part B: PUPs/MTPs 0-<6 Years | 2 | 43 | 2 |
| other | Blood loss anaemia | Blood and lymphatic system disorders | Extension Study - Former Part A Participants | 1 | 46 | 1 |
| other | Blood loss anaemia | Blood and lymphatic system disorders | Extension Study - Former Part B Participants | 0 | 36 | 0 |
1 to 10 of 960Source ClinicalTrials.gov
See all 960 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Main Study - Part A: PTPs 0-<6 Years | 0 | 25 |
| Total, all-cause mortality | Main Study - Part A: PTPs 6-12 Years | 0 | 26 |
| Total, all-cause mortality | Main Study - Part B: PUPs/MTPs 0-<6 Years | 0 | 43 |
| Total, all-cause mortality | Extension Study - Former Part A Participants | 0 | 46 |
| Total, all-cause mortality | Extension Study - Former Part B Participants | 0 | 36 |
| Total, other adverse events | Main Study - Part A: PTPs 0-<6 Years | 16 | 25 |
| Total, other adverse events | Main Study - Part A: PTPs 6-12 Years | 19 | 26 |
| Total, other adverse events | Main Study - Part B: PUPs/MTPs 0-<6 Years | 28 | 43 |
| Total, other adverse events | Extension Study - Former Part A Participants | 41 | 46 |
| Total, other adverse events | Extension Study - Former Part B Participants | 24 | 36 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers11
The papers the trial lists on ClinicalTrials.gov.
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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.
GET https://conductscience.org/api/trials/v1/NCT01311648
GET https://conductscience.org/api/trials/v1/NCT01311648/results?page=2