Trial · NCT02336178
Safety and Efficacy of Benefix in Patients With Hemophilia B in Usual Care Settings in China
NCT02336178, Phase 4, Completed, Pfizer; results first posted 2017-05-04
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety and Efficacy of Benefix in Patients With Hemophilia B in Usual Care Settings in China. Trials, record NCT02336178. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02336178
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy of Benefix in Patients With Hemophilia B in Usual Care Settings in China
- NCT number
- NCT02336178
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 70 participants, actual
- Lead sponsor
- Pfizer
- Conditions
- HEMOPHILIA B
- Interventions
- Benefix
- First posted
- 2015-01-12
- Last updated
- 2017-05-04
- Other registries' IDs
- eudract:2016-000765-22
- Results first posted
- 2017-05-04
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Who Developed Factor IX Inhibitor | Up to 6 months | Factor IX (FIX) inhibitor development was defined as any Bethesda inhibitor titer greater than the laboratory's normal range or Bethesda inhibitor titer \>=0.6 Bethesda Unit (BU)/mL. |
| Primary | Number of Participants With Allergic Reactions | Up to 6 months | FIX product allergy was defined as a hypersensitivity reaction to a FIX product with symptoms such as hives, urticaria, tightness of chest, wheezing, hypotension, and anaphylaxis based on investigator's judgments. |
| Primary | Number of Participants With Thrombotic Events | Up to 6 months | Thrombotic event was defined as any event associated with formation of a blood clot including catheter-associated thrombi and thrombotic complications. |
| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Up to 7 months (28 calendar days after end of 6-month or 50-exposure day study treatment) | An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a product or medical device, regardless of the causality with the treatment or usage. A serious adverse event (SAE) was any untoward occurrence at any dose that resulted in death; was life threatening (immediate risk of death); required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; resulted in congenital anomaly/birth defect. AEs included both serious and non-serious AEs. Treatment-emergent AEs were those with initial onset or increasing in severity after the first dose of study drug. |
| Secondary | Annualized Bleeding Rates (ABRs) in Participants Receiving Prophylaxis Treatment With BeneFIX During Their Prophylaxis Period | Up to 6 months or 50 exposure days whichever occurred first | For prophylaxis period, annualized bleeding rate (ABR) was derived for each participant by the following formula: ABR = number of bleeds in prophylaxis period / (number of days in prophylaxis period/365.25). A prophylaxis period was defined as time from first prophylaxis infusion through 6 calendar days after the day of last prophylaxis infusion, or the day of study conclusion visit, whichever was earlier. A break in the prophylaxis period was any period of 28 days or longer in which no prophylaxis infusions were given. For a prophylaxis regimen to have been qualified to have ABR calculated, the sum of its periods needed to be \>=14 days. |
| Secondary | Number of Spontaneous/Non-traumatic Breakthrough Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX | Up to 6 months or 50 exposure days whichever occurred first | The prophylaxis infusion time, bleed start time and bleed type (etiology) were used to determine the number of spontaneous, non-traumatic breakthrough bleeds that occurred \<=48 hours after a prophylaxis infusion. If there was more than 1 bleed location (eg, ankle and joint) with identical bleed start date and time, it was treated as 1 bleed occurrence. |
| Secondary | Annualized Bleeding Rates (ABRs) in Participants Receiving On-demand Treatment With BeneFIX During Their On-demand Period | Up to 6 months or 50 exposure days whichever occurred first | For on-demand period, the annualized bleeding rate (ABR) was derived for each participant by the following formula: ABR = number of bleeds in on-demand period / (number of days in on-demand period/365.25). The on-demand period was defined as the entire time of enrollment in the study (ie, from the date of the enrollment visit through the day before the Final/Early Termination visit) except time in any prophylaxis period. The breaks in the prophylaxis period were considered on-demand periods. For an on-demand regimen to have been qualified to have ABR calculated, the sum of its periods needed to be \>= 14 days. |
| Secondary | Number of Infusions Resulted in the Following Response to On-demand Treatment of Bleeds: Excellent, Good, Moderate, no Response | Up to 6 months or 50 exposure days whichever occurred first | Response was assessed using 4-point On-Demand Hemostasis Efficacy Rating Scale. Excellent means definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with no additional infusion administered; good means definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with at least one additional infusion administered for complete resolution of the bleeding episode, or definite pain relief and/or improvement in signs of bleeding starting after 8 hours following the infusion, with no additional infusion administered; moderate means probable or slight improvement starting after 8 hours following the infusion, with at least one additional infusion administered for complete resolution of the bleeding episode; no response means no improvement between infusions or during the 24 hour interval following an infusion, or condition worsens. Both first infusions and follow-up infusions were included. |
| Secondary | Number of BeneFIX Infusions to Treat Each New Bleed | Up to 6 months or 50 exposure days whichever occurred first | The number of BeneFIX infusions administered to treat each new bleed was calculated by adding the on-demand initial treatment and any on-demand follow-up treatments for the same bleed. If there was more than one bleed location (eg, ankle and joint) with identical bleed start date and time, it was treated as one bleed occurrence. |
| Secondary | Average Infusion Dose and Total Factor IX Consumption in On-demand Setting | Up to 6 months or 50 exposure days whichever occurred first | The total amount (international units \[IU\]) infused for each BeneFIX infusion was summed to calculate the total factor IX consumption for each participant. The average infusion dose for each participant was calculated as his total factor IX consumption (in IU) divided by the number of infusions administered. |
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 | Percentage of Participants Who Developed Factor IX Inhibitor | Up to 6 months | All participants who received at least 1 dose of BeneFIX during the study. Participants in each arm were not mutually exclusive. | percentage of participants | NUMBER |
| PRIMARY | Number of Participants With Allergic Reactions | Up to 6 months | All participants who received at least 1 dose of BeneFIX during the study. Participants in each arm were not mutually exclusive. | participants | NUMBER |
| PRIMARY | Number of Participants With Thrombotic Events | Up to 6 months | All participants who received at least 1 dose of BeneFIX during the study. Participants in each arm were not mutually exclusive. | participants | NUMBER |
| SECONDARY | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Up to 7 months (28 calendar days after end of 6-month or 50-exposure day study treatment) | All participants who received at least 1 dose of BeneFIX during the study. Participants in each arm were not mutually exclusive. | participants | NUMBER |
| SECONDARY | Annualized Bleeding Rates (ABRs) in Participants Receiving Prophylaxis Treatment With BeneFIX During Their Prophylaxis Period | Up to 6 months or 50 exposure days whichever occurred first | All participants who participated in at least 1 day of a prophylaxis period (ie, had at least 1 prophylaxis dose). Participants in each arm were not mutually exclusive. | episodes/year | MEAN |
| SECONDARY | Number of Spontaneous/Non-traumatic Breakthrough Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX | Up to 6 months or 50 exposure days whichever occurred first | All participants with any spontaneous/non-traumatic breakthrough bleeds within 48 hours of a prophylaxis dose. Participants in each arm were not mutually exclusive. | breakthrough bleeds | MEAN |
| SECONDARY | Annualized Bleeding Rates (ABRs) in Participants Receiving On-demand Treatment With BeneFIX During Their On-demand Period | Up to 6 months or 50 exposure days whichever occurred first | All participants who received on-demand treatment during on-demand period. Participants in each arm were not mutually exclusive. | episodes/year | MEAN |
| SECONDARY | Number of Infusions Resulted in the Following Response to On-demand Treatment of Bleeds: Excellent, Good, Moderate, no Response | Up to 6 months or 50 exposure days whichever occurred first | All participants who had a bleed during the study for which on-demand treatment with BeneFIX was administered. Participants in each arm were not mutually exclusive. | infusions | NUMBER |
| SECONDARY | Number of BeneFIX Infusions to Treat Each New Bleed | Up to 6 months or 50 exposure days whichever occurred first | All participants who had a bleed during the study for which on-demand treatment with BeneFIX was administered. Participants in each arm were not mutually exclusive. | infusions | MEAN |
| SECONDARY | Average Infusion Dose and Total Factor IX Consumption in On-demand Setting | Up to 6 months or 50 exposure days whichever occurred first | All participants who had a bleed during the study for which on-demand treatment with BeneFIX was administered. Participants in each arm were not mutually exclusive. | IU | MEAN |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group124
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants Who Developed Factor IX Inhibitor | Pediatric Participants <6 Years of Age | 3.3 | 95% Confidence Interval 0.1 to 17.2 | percentage of participants | |
| Percentage of Participants Who Developed Factor IX Inhibitor | Pediatric Participants ≥6 to ≤12 Years of Age | 0.0 | 95% Confidence Interval 0.0 to 11.2 | percentage of participants | |
| Percentage of Participants Who Developed Factor IX Inhibitor | Previously Untreated Participants (PUPs) | 0.0 | 95% Confidence Interval 0.0 to 28.5 | percentage of participants | |
| Percentage of Participants Who Developed Factor IX Inhibitor | Participants Receiving Prophylaxis Treatment | 1.8 | 95% Confidence Interval 0.0 to 9.4 | percentage of participants | |
| Percentage of Participants Who Developed Factor IX Inhibitor | Severe Participants (Factor IX Activity <1%) | 0.0 | 95% Confidence Interval 0.0 to 9.7 | percentage of participants | |
| Percentage of Participants Who Developed Factor IX Inhibitor | Overall Participants | 1.4 | 95% Confidence Interval 0.0 to 7.7 | percentage of participants | |
| Number of Participants With Allergic Reactions | Pediatric Participants <6 Years of Age | 0 | participants | ||
| Number of Participants With Allergic Reactions | Pediatric Participants ≥6 to ≤12 Years of Age | 0 | participants | ||
| Number of Participants With Allergic Reactions | Previously Untreated Participants (PUPs) | 0 | participants | ||
| Number of Participants With Allergic Reactions | Participants Receiving Prophylaxis Treatment | 0 | participants |
1 to 10 of 124Source ClinicalTrials.gov
See all 124 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | BeneFIX | 70 | |
| Overall Study | COMPLETED | BeneFIX | 66 | |
| Overall Study | NOT COMPLETED | BeneFIX | 4 |
Source ClinicalTrials.gov
Adverse events14
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 | Diarrhoea | Gastrointestinal disorders | BeneFIX | 5 | 70 | 6 |
| other | Peripheral swelling | General disorders | BeneFIX | 6 | 70 | 9 |
| other | Pyrexia | General disorders | BeneFIX | 11 | 70 | 18 |
| other | Nasopharyngitis | Infections and infestations | BeneFIX | 19 | 70 | 27 |
| other | Upper respiratory tract infection | Infections and infestations | BeneFIX | 5 | 70 | 8 |
| other | Contusion | Injury, poisoning and procedural complications | BeneFIX | 5 | 70 | 7 |
| other | Fall | Injury, poisoning and procedural complications | BeneFIX | 8 | 70 | 10 |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | BeneFIX | 17 | 70 | 76 |
| other | Joint range of motion decreased | Musculoskeletal and connective tissue disorders | BeneFIX | 6 | 70 | 20 |
| other | Joint swelling | Musculoskeletal and connective tissue disorders | BeneFIX | 15 | 70 | 32 |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | BeneFIX | ||
| Total, other adverse events | BeneFIX | 55 | 70 |
| Total, serious adverse events | BeneFIX | 2 | 70 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 34032739 | 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 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/NCT02336178
GET https://conductscience.org/api/trials/v1/NCT02336178/results?page=2