Trial · NCT04759131
Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A
NCT04759131, Phase 3, Completed, Bioverativ, a Sanofi company; results first posted 2024-02-13
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A. Trials, record NCT04759131. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04759131
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A
- NCT number
- NCT04759131
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 74 participants, actual
- Lead sponsor
- Bioverativ, a Sanofi company
- Conditions
- Hemophilia A
- Interventions
- efanesoctocog alfa (BIVV001)
- First posted
- 2021-02-18
- Last updated
- 2025-09-11
- Other registries' IDs
- eudract:2020-000769-18
- Results first posted
- 2024-02-13
Source ClinicalTrials.gov
Registered outcomes48
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Neutralising Antibodies (Development of Inhibitors) Directed Against Factor VIII | Baseline up to Week 52 | Inhibitor development was defined as an inhibitor result of greater than or equal to (\>=0.6) Bethesda units (BU/mL) that was confirmed by a second test result from a separate sample, drawn 2 to 4 weeks following the date when the original sample was drawn. Both tests must have been performed by the central laboratory using the Nijmegen modified Bethesda assay. |
| Secondary | Annualized Bleeding Rate (ABR): For Treated Bleeds | Baseline up to Week 52 | ABR: annualized number of treated bleeding episodes (BE) per participant per year. ABR=number of treated BE during efficacy period (EP)/total number of days during EP\*365.25. Treated BE:any occurrence of hemorrhage required administration of BIVV001. It started from 1st sign of bleed and ended no more than 72 hours after last injection to treat BE, any subsequent bleeding at same location/injections administered less than or equal to (\<=) 72 hours apart from previous injection were considered same BE. Any injection after \>72 hours post preceding one=considered 1st injection to treat new BE in same location. Any bleed at different location:considered as separate BE, regardless of time from last injection. EP=sum of all intervals of time during which participants were treated with BIVV001 according to study arms and treatment regimens. ABR:by negative binomial model with total number of treated BE during EP as response variable and log transformed EP duration (years) as offset variable. |
| Secondary | Sensitivity Analysis: Annualized Bleeding Rate: For Treated Bleeds | Baseline up to Week 52 | ABR: annualized number of treated BE per participant per year. ABR = number of treated BE during EP/total number of days during EP\*365.25. Treated BE: any occurrence of hemorrhage that required administration of BIVV001. It started from 1st sign of bleed and ended no more than 72 hours after last injection to treat BE, any subsequent bleeding at same location/injections administered \<=72 hours apart from previous injection were considered same BE. Any injection to treat bleed, taken \>72 hours after preceding one, was considered 1st injection to treat new bleeding episode in same location. Any bleed at different location: considered as separate BE, regardless of time from last injection. EP reflects sum of all intervals of time during which participants were treated with BIVV001 according to study arms and treatment regimens. ABR: using negative binomial (NB) model with total number of treated BE during EP as response variable and log-transformed EP duration (years) as offset variable. |
| Secondary | Annualized Bleeding Rate for All Bleeding Episodes | Baseline up to Week 52 | ABR:annualized number of all BE (treated and untreated)/participant/year. ABR=number of all BE during EP/total number of days during EP\*365.25. BE:any occurrence of hemorrhage required administration of BIVV001. It started from 1st sign of bleed and ended no more than 72 hours after last injection to treat BE, any subsequent bleeding at same location/injections administered \<=72 hours apart from previous injection were considered same BE. Any injection after \>72 hours post preceding one=considered 1st injection to treat new BE in same location. Any bleed at different location: considered as separate BE, regardless of time from last injection. EP:sum of all intervals of time during which participants treated with BIVV001 according to study arms \& treatment regimens. ABR:NB model with total number of treated BE during EP as response variable and log-transformed EP duration (years) as offset variable. Spontaneous:bleeding without contributing factor, Traumatic:bleeding with known reason. |
| Secondary | Sensitivity Analysis: Annualized Bleeding Rate for All Bleeding Episodes | Baseline up to Week 52 | ABR: annualized number of all BE (treated \& untreated)/participant/year. ABR=number of all BE during EP/total number of days during EP\*365.25. BE: any hemorrhage occurrence required administration of BIVV001. It started from 1st sign of bleed \& ended no more than 72 hours after last injection to treat BE, any subsequent bleeding at same location/injections administered \<=72 hours apart from previous injection were considered same BE. Any bleed at different location: considered as separate BE, regardless of time from last injection. EP: sum of all intervals of time during which participants treated with BIVV001 according to study arms and treatment regimens. ABR: estimated by NB model with total number of treated BE during EP as response variable and log-transformed EP duration (years) as offset variable. Spontaneous: bleeding without contributing factor (definite trauma/antecedent strenuous activity). Traumatic: bleeding with known/believed reason. |
| Secondary | Annualized Bleeding Rate by Type of Bleed (Spontaneous, Traumatic and Unknown Type) | Baseline up to Week 52 | ABR: annualized number of treated bleeding episodes per participant per year. ABR = number of all BE during EP/total number of days during EP\*365.25. EP reflects sum of all intervals of time during which participants were treated with BIVV001 according to study arms and treatment regimens. Treated BE: episode that started from 1st sign of bleed and ended no more than 72 hours after last injection to treat bleeding episode, any subsequent bleeding at same location/injections administered \<=72 hours apart from previous injection were considered same bleeding episode. Any injection to treat bleed, taken \>72 hours after preceding one was considered 1st injection to treat new BE in same location. Any bleed at different location was considered as separate bleeding episode, regardless of time from last injection. Spontaneous bleeding: BE without contributing factor (definite trauma/antecedent "strenuous" activity). Traumatic bleeding: BE with known/believed reason for bleed. |
| Secondary | Sensitivity Analysis: Annualized Bleeding Rate by Type of Bleed (Spontaneous, Traumatic and Unknown Type) | Baseline up to Week 52 | ABR: annualized number of treated bleeding episodes per participant per year. ABR = number of all BE during EP/total number of days during EP\*365.25. EP reflects sum of all intervals of time during which participants were treated with BIVV001 according to study arms and treatment regimens. Treated BE: episode that started from 1st sign of bleed and ended no more than 72 hours after last injection to treat bleeding episode, any subsequent bleeding at same location/injections administered \<=72 hours apart from previous injection were considered same bleeding episode. Any injection to treat bleed, taken \>72 hours after preceding one, was considered 1st injection to treat new BE in same location. Any bleed at different location was considered as separate bleeding episode, regardless of time from last injection. Spontaneous bleeding: BE without contributing factor (definite trauma/antecedent "strenuous" activity). Traumatic bleeding: BE with known/believed reason for bleed. |
| Secondary | Annualized Bleeding Rate by Location of Bleed (Joint, Muscle, Internal and Skin/Mucosa) | Baseline up to Week 52 | ABR: annualized number of treated bleeding episodes per participant per year. ABR = number of all BE during EP/total number of days during EP\*365.25. Efficacy period reflects sum of all intervals of time during which participants were treated with BIVV001 according to study arms and treatment regimens. Treated bleeding episode: episode that started from 1st sign of bleed and ended no more than 72 hours after last injection to treat bleeding episode, any subsequent bleeding at same location/injections administered \<=72 hours apart from previous injection were considered same bleeding episode. Any injection \>72 hours after preceding one, was considered 1st injection to treat new BE in same location. Any bleed at different location was considered as separate bleeding episode, regardless of time from last injection. Spontaneous bleeding: BE without contributing factor (definite trauma/antecedent "strenuous" activity). Traumatic bleeding: BE with known/believed reason for bleed. |
| Secondary | Sensitivity Analysis: Annualized Bleeding Rate by Location of Bleed (Joint, Muscle, Internal and Skin/Mucosa) | Baseline up to Week 52 | ABR: annualized number of treated bleeding episodes per participant per year. ABR = number of all BE during EP/total number of days during EP\*365.25. EP reflects sum of all intervals of time during which participants were treated with BIVV001 according to study arms \& treatment regimens. Treated BE: episode that started from 1st sign of bleed and ended no more than 72 hours after last injection to treat bleeding episode any subsequent bleeding at same location/injections administered \<=72 hours apart from previous injection were considered same bleeding episode. Any injection to treat bleed, taken \>72 hours after preceding one, was considered 1st injection to treat new BE in same location. Any bleed at different location was considered as separate bleeding episode, regardless of time from last injection. Spontaneous bleeding: BE without contributing factor (definite trauma/antecedent "strenuous" activity). Traumatic bleeding: BE with known/believed reason for bleed. |
| Secondary | Percentage of Participants Achieving FVIII Activity Levels Above 1%, 3%, 5%, 10%, 15%, and 20% | Baseline up to Week 52 | FVIII activity level was measured using activated partial thromboplastin time (aPTT)-based one stage clotting assay. Percentage of participants who achieved steady-state trough FVIII activity levels above (\>) 1%, 3%, 5%, 10%, 15%, and 20% were reported in this outcome measure. Participants were counted in more than one category, as applicable. |
1 to 10 of 48Source ClinicalTrials.gov
See all 48 registered outcomesOutcomes48
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 Neutralising Antibodies (Development of Inhibitors) Directed Against Factor VIII | Baseline up to Week 52 | Analysis was performed on safety analysis set which included all participants who took at least 1 dose of study intervention. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Annualized Bleeding Rate (ABR): For Treated Bleeds | Baseline up to Week 52 | Analysis was performed on FAS population. | episodes per participant per year | MEAN |
| SECONDARY | Sensitivity Analysis: Annualized Bleeding Rate: For Treated Bleeds | Baseline up to Week 52 | Analysis was performed on FAS population. Sensitivity analysis excluded 1 participant who did not receive weekly prophylaxis treatment as specified in the protocol for an extended period of time. | episodes per participant per year | MEAN |
| SECONDARY | Annualized Bleeding Rate for All Bleeding Episodes | Baseline up to Week 52 | Analysis was performed on FAS population. | episodes per participant per year | MEAN |
| SECONDARY | Sensitivity Analysis: Annualized Bleeding Rate for All Bleeding Episodes | Baseline up to Week 52 | Analysis was performed on FAS population. Sensitivity analysis excluded 1 participant who did not receive weekly prophylaxis treatment as specified in the protocol for an extended period of time. | episodes per participant per year | MEAN |
| SECONDARY | Annualized Bleeding Rate by Type of Bleed (Spontaneous, Traumatic and Unknown Type) | Baseline up to Week 52 | Analysis was performed on FAS population. | episodes per participant per year | MEAN |
| SECONDARY | Sensitivity Analysis: Annualized Bleeding Rate by Type of Bleed (Spontaneous, Traumatic and Unknown Type) | Baseline up to Week 52 | Analysis was performed on FAS population. Sensitivity analysis excluded 1 participant who did not receive weekly prophylaxis treatment as specified in the protocol for an extended period of time. | episodes per participant per year | MEAN |
| SECONDARY | Annualized Bleeding Rate by Location of Bleed (Joint, Muscle, Internal and Skin/Mucosa) | Baseline up to Week 52 | Analysis was performed on FAS population. | episodes per participant per year | MEAN |
| SECONDARY | Sensitivity Analysis: Annualized Bleeding Rate by Location of Bleed (Joint, Muscle, Internal and Skin/Mucosa) | Baseline up to Week 52 | Analysis was performed on FAS population. Sensitivity analysis excluded 1 participant who did not receive weekly prophylaxis treatment as specified in the protocol for an extended period of time. | episodes per participant per year | MEAN |
| SECONDARY | Percentage of Participants Achieving FVIII Activity Levels Above 1%, 3%, 5%, 10%, 15%, and 20% | Baseline up to Week 52 | Analysis was performed on FAS. Here, 'overall number of participants analyzed = participants with available data for this outcome measure. | percentage of participants | NUMBER |
1 to 10 of 48Source ClinicalTrials.gov
See all 48 outcomesResults by group160
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 Neutralising Antibodies (Development of Inhibitors) Directed Against Factor VIII | BIVV001: Participants Aged <6 Years | 0 | Participants | ||
| Number of Participants With Neutralising Antibodies (Development of Inhibitors) Directed Against Factor VIII | BIVV001: Participants Aged 6 to <12 Years | 0 | Participants | ||
| Annualized Bleeding Rate (ABR): For Treated Bleeds | BIVV001: Participants Aged <6 Years | 0.48 | 95% Confidence Interval 0.30 to 0.77 | episodes per participant per year | |
| Annualized Bleeding Rate (ABR): For Treated Bleeds | BIVV001: Participants Aged 6 to <12 Years | 1.33 | 95% Confidence Interval 0.64 to 2.76 | episodes per participant per year | |
| Sensitivity Analysis: Annualized Bleeding Rate: For Treated Bleeds | BIVV001: Participants Aged <6 Years | 0.48 | 95% Confidence Interval 0.30 to 0.77 | episodes per participant per year | |
| Sensitivity Analysis: Annualized Bleeding Rate: For Treated Bleeds | BIVV001: Participants Aged 6 to <12 Years | 0.75 | 95% Confidence Interval 0.41 to 1.40 | episodes per participant per year | |
| Annualized Bleeding Rate for All Bleeding Episodes | BIVV001: Participants Aged <6 Years | 2.78 | 95% Confidence Interval 1.39 to 5.58 | episodes per participant per year | |
| Annualized Bleeding Rate for All Bleeding Episodes | BIVV001: Participants Aged 6 to <12 Years | 2.85 | 95% Confidence Interval 1.59 to 5.12 | episodes per participant per year | |
| Sensitivity Analysis: Annualized Bleeding Rate for All Bleeding Episodes | BIVV001: Participants Aged <6 Years | 2.78 | 95% Confidence Interval 1.39 to 5.58 | episodes per participant per year | |
| Sensitivity Analysis: Annualized Bleeding Rate for All Bleeding Episodes | BIVV001: Participants Aged 6 to <12 Years | 2.32 | 95% Confidence Interval 1.30 to 4.13 | episodes per participant per year |
1 to 10 of 160Source ClinicalTrials.gov
See all 160 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | BIVV001: Participants Aged <6 Years | 38 | |
| Overall Study | STARTED | BIVV001: Participants Aged 6 to <12 Years | 36 | |
| Overall Study | COMPLETED | BIVV001: Participants Aged <6 Years | 36 | |
| Overall Study | COMPLETED | BIVV001: Participants Aged 6 to <12 Years | 36 | |
| Overall Study | NOT COMPLETED | BIVV001: Participants Aged <6 Years | 2 | |
| Overall Study | NOT COMPLETED | BIVV001: Participants Aged 6 to <12 Years | 0 |
Source ClinicalTrials.gov
Adverse events80
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 | Asymptomatic Covid-19 | Infections and infestations | BIVV001: Participants Aged <6 Years | 2 | 38 | 2 |
| other | Asymptomatic Covid-19 | Infections and infestations | BIVV001: Participants Aged 6 to <12 Years | 5 | 36 | 5 |
| other | Covid-19 | Infections and infestations | BIVV001: Participants Aged <6 Years | 1 | 38 | 1 |
| other | Covid-19 | Infections and infestations | BIVV001: Participants Aged 6 to <12 Years | 2 | 36 | 2 |
| other | Ear Infection | Infections and infestations | BIVV001: Participants Aged <6 Years | 1 | 38 | 1 |
| other | Ear Infection | Infections and infestations | BIVV001: Participants Aged 6 to <12 Years | 2 | 36 | 2 |
| other | Gastroenteritis Viral | Infections and infestations | BIVV001: Participants Aged <6 Years | 5 | 38 | 6 |
| other | Gastroenteritis Viral | Infections and infestations | BIVV001: Participants Aged 6 to <12 Years | 1 | 36 | 2 |
| other | Influenza | Infections and infestations | BIVV001: Participants Aged <6 Years | 0 | 38 | 0 |
| other | Influenza | Infections and infestations | BIVV001: Participants Aged 6 to <12 Years | 2 | 36 | 2 |
1 to 10 of 80Source ClinicalTrials.gov
See all 80 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | BIVV001: Participants Aged <6 Years | 0 | 38 |
| Total, all-cause mortality | BIVV001: Participants Aged 6 to <12 Years | 0 | 36 |
| Total, other adverse events | BIVV001: Participants Aged <6 Years | 31 | 38 |
| Total, other adverse events | BIVV001: Participants Aged 6 to <12 Years | 28 | 36 |
| Total, serious adverse events | BIVV001: Participants Aged <6 Years | 5 | 38 |
| Total, serious adverse events | BIVV001: Participants Aged 6 to <12 Years | 4 | 36 |
Source ClinicalTrials.gov
Papers4
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 comparisons4
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| BIVV001: Participants Aged <6 Years vs BIVV001: Participants Aged 6 to <12 Years: Number of Participants With Neutralising Antibodies (Development of Inhibitors) Directed Against Factor VIII | Trial comparisons |
| BIVV001: Participants Aged <6 Years vs BIVV001: Participants Aged 6 to <12 Years: deaths from any cause | Trial comparisons |
| BIVV001: Participants Aged <6 Years vs BIVV001: Participants Aged 6 to <12 Years: Number of Participants With Occurrence of Embolic and Thrombotic Events | Trial comparisons |
| BIVV001: Participants Aged <6 Years vs BIVV001: Participants Aged 6 to <12 Years: serious adverse events | 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.
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