Trial · NCT02234310
Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B
NCT02234310, Phase 3, Completed, Bioverativ, a Sanofi company; results first posted 2020-07-31
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B. Trials, record NCT02234310. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02234310
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B
- NCT number
- NCT02234310
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 33 participants, actual
- Lead sponsor
- Bioverativ, a Sanofi company
- Conditions
- Hemophilia B
- Interventions
- rFIXFc
- First posted
- 2014-09-09
- Last updated
- 2022-03-25
- Other registries' IDs
- eudract:2013-003629-27
- Results first posted
- 2020-07-31
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay | Up to 3 years | Development of an inhibitor was defined as an inhibitor test result of \>= 0.60 Bethesda units per milliliter (BU/mL) that was confirmed by a second test result of \>=0.60 BU/mL from a separate sample, drawn 2 to 4 weeks after the date when the original sample was drawn, with both tests performed by the central laboratory using Nijmegen-modified Bethesda assay. |
| Secondary | Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) Per Participant (Annualized Bleeding Rate [ABR]) | Up to 3 years | ABR was annualized number of bleeding episodes during efficacy period (EP) per participant normalized to a 1-year interval of time. Bleeding episodes were classified as: spontaneous if parent/caregiver/participant records bleeding event when there is no known contributing factor such as definite trauma or antecedent strenuous activity; and traumatic when there is known reason for bleed. ABR=(Number of bleeding episodes during EP/total number of days during EP)\*365.25. EP reflects the sum of all intervals of time during which participants were treated with rFIXFc per treatment regimens excluding surgical/rehabilitation periods and large injection intervals (greater than \[\>\]28 days). Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. |
| Secondary | Annualized Number of Spontaneous Joint Bleeding Episodes | Up to 3 years | Bleeding episodes were classified as spontaneous if parent/caregiver/participant records a bleeding event when there is no known contributing factor such as a definite trauma or antecedent "strenuous" activity. Annualized spontaneous joint bleeding episodes=(Total number of spontaneous joint bleeding episodes during efficacy period (EP) divided by total number of days during EP)\*365.25. EP reflects the sum of all intervals of time during which participants were treated with rFIXFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals (\>28 days). Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. |
| Secondary | Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale | Up to 3 years | Using e-diary, each participant's parent/caregiver rated treatment response to any bleeding episode (BE) at approximately 8 to 12 hours from time of injection and prior to additional doses of rFIXFc given for same BE using 4-point scale:- 1=Excellent: abrupt pain relief and/or improvement in signs of bleeding within approximately 8 hours after initial injection; 2=Good: definite pain relief and/or improvement in signs of bleeding within approx. 8 hours after injection, but possibly requiring more than 1 injection after 24-48 hours for complete resolution; 3=Moderate: Probable/slight beneficial effect within 8 hours after initial injection and requires more than 1 injection and 4=None: No improvement or condition worsens within approx. 8 hours after initial injection. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. |
| Secondary | Total Number of Exposure Days (EDs) | Up to 3 years | An ED was defined as a 24-hour period in which a participant received one or more doses of rFIXFc injections, with the time of the first injection of rFIXFc defined as the start of the ED. Participant who did not have a particular injection type were counted as having zero injections for that type. |
| Secondary | Total Annualized rFIXFc Consumption Per Participant for the Prevention and Treatment of Bleeding Episodes | Up to 3 years | Total annualized rFIXFc consumption (in IU/kg) was calculated for each participant as: Annualized consumption = (Total IU/kg of rFIXFc during efficacy period (EP) divided by total number of days during EP)\*365.25. EP reflects the sum of all intervals of time during which participants were treated with rFIXFc according to the treatment regimens of the study excluding surgical/rehabilitation periods and large injection intervals (\> 28 days). Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. |
| Secondary | Number of Injections of rFIXFc Required to Resolve a Bleeding Episode | Up to 3 years | Number of injections of rFIXFc required to resolve a bleeding episode during efficacy period (EP) were reported. EP reflects the sum of all intervals of time during which participants were treated with rFIXFc according to the treatment regimens of the study excluding surgical/rehabilitation periods and large injection intervals (\>28 days). All injections given from the initial sign of a bleed, until the last date/time within the bleed window were counted. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. |
| Secondary | Average Dose Per Injection of rFIXFc Required to Resolve a Bleeding Episode | Up to 3 years | The average dose of rFIXFc per injection per bleeding episode was calculated as the average of all doses (IU/kg) administered to treat the bleeding episode during efficacy period (EP). EP begins with the first treatment regimen dose of rFIXFc and ends with the last dose (regardless of the reason for dosing). Surgery/rehabilitation periods are not included in the EP. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study. |
| Secondary | Change From Baseline in rFIXFc Incremental Recovery (IR) | Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, and 144 | Blood samples were taken at trough and Cmax (maximum concentration) for assessment of incremental recovery, measured by the one-stage clotting assay. IR (International Units per deciliter \[IU/dL\] per IU/kg) = (Cmax for FIX activity - Pre-dose FIX activity) (IU/dL)/ Actual dose (IU/kg), where Cmax is 30 minute FIX activity post-dose and FIX activity less than (\<)0.5 IU/dL was set to 0 IU/dL for calculation of IR. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay | Up to 3 years | Analysis performed on Safety Analysis set which included all participants who had received at least 1 dose of study treatment. | percentage of participants | NUMBER |
| SECONDARY | Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) Per Participant (Annualized Bleeding Rate [ABR]) | Up to 3 years | Analysis performed on Full Analysis Set (FAS) which included all enrolled participants with at least 1 dose of study treatment. Here, number analyzed signifies number of FAS participants who were analyzed in each treatment regimen. | episodes per participant per year | MEDIAN |
| SECONDARY | Annualized Number of Spontaneous Joint Bleeding Episodes | Up to 3 years | Analysis performed on FAS. Here, number analyzed signifies number of FAS participants who were analyzed in each treatment regimen. | episodes per participant per year | MEDIAN |
| SECONDARY | Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale | Up to 3 years | Analysis performed on FAS. Analysis was based on all injections. Here, number analyzed signifies number of responses to injections reported for each treatment regimen. | responses to injections | COUNT_OF_UNITS |
| SECONDARY | Total Number of Exposure Days (EDs) | Up to 3 years | Analysis performed on safety analysis set. | days | MEDIAN |
| SECONDARY | Total Annualized rFIXFc Consumption Per Participant for the Prevention and Treatment of Bleeding Episodes | Up to 3 years | Analysis performed on FAS. Here, number analyzed signifies number of FAS participants who were analyzed in each treatment regimen. | IU per kilogram per participant per year | MEDIAN |
| SECONDARY | Number of Injections of rFIXFc Required to Resolve a Bleeding Episode | Up to 3 years | Analysis performed on FAS. Here, number analyzed signifies number of bleeding episodes reported for each treatment regimen. | injections | MEDIAN |
| SECONDARY | Average Dose Per Injection of rFIXFc Required to Resolve a Bleeding Episode | Up to 3 years | Analysis performed on FAS. Here, number analyzed signifies number of bleeding episodes reported for each treatment regimen. | IU/kg | MEDIAN |
| SECONDARY | Change From Baseline in rFIXFc Incremental Recovery (IR) | Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, and 144 | Analysis performed on FAS. Here number analyzed signifies number of FAS participants with available data for each visit. | IU/dL per IU/kg | MEDIAN |
Source ClinicalTrials.gov
Results by group32
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 3.03 | 95% Confidence Interval 0.08 to 15.76 | percentage of participants | |
| Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) Per Participant (Annualized Bleeding Rate [ABR]) | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | Episodic regimen | 0.21 | Full Range 0.0 to 6.8 | episodes per participant per year |
| Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) Per Participant (Annualized Bleeding Rate [ABR]) | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | Prophylaxis regimen | 1.24 | Full Range 0.0 to 5.4 | episodes per participant per year |
| Annualized Number of Spontaneous Joint Bleeding Episodes | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | Episodic regimen | 0.00 | Full Range 0.0 to 5.6 | episodes per participant per year |
| Annualized Number of Spontaneous Joint Bleeding Episodes | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | Prophylaxis regimen | 0.00 | Full Range 0.0 to 2.6 | episodes per participant per year |
| Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | Episodic regimen / Excellent or Good | 22 | responses to injections | |
| Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | Episodic regimen / Moderate | 0 | responses to injections | |
| Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | Episodic regimen / None | 0 | responses to injections | |
| Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | Episodic regimen / Response not provided | 58 | responses to injections | |
| Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | Prophylaxis regimen / Excellent or Good | 50 | responses to injections |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 results by groupParticipant flow5
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 33 | |
| Overall Study | Episodic Treatment Regimen | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 22 | |
| Overall Study | Prophylactic Treatment Regimen | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 28 | |
| Overall Study | COMPLETED | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 27 | |
| Overall Study | NOT COMPLETED | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 6 |
Source ClinicalTrials.gov
Adverse events72
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 | Middle ear effusion | Ear and labyrinth disorders | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 2 | 33 | 2 |
| other | Constipation | Gastrointestinal disorders | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 2 | 33 | 3 |
| other | Diarrhoea | Gastrointestinal disorders | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 5 | 33 | 9 |
| other | Teething | Gastrointestinal disorders | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 6 | 33 | 15 |
| other | Vomiting | Gastrointestinal disorders | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 4 | 33 | 8 |
| other | Pyrexia | General disorders | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 14 | 33 | 33 |
| other | Bronchitis | Infections and infestations | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 2 | 33 | 4 |
| other | Conjunctivitis | Infections and infestations | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 2 | 33 | 2 |
| other | Croup infectious | Infections and infestations | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 2 | 33 | 2 |
| other | Ear infection | Infections and infestations | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 4 | 33 | 4 |
1 to 10 of 72Source ClinicalTrials.gov
See all 72 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 0 | 33 |
| Total, other adverse events | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 27 | 33 |
| Total, serious adverse events | Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | 23 | 33 |
Source ClinicalTrials.gov
Papers2
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/NCT02234310
GET https://conductscience.org/api/trials/v1/NCT02234310/results?page=2