Trial · NCT02093897
Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A
NCT02093897, Phase 3, Completed, CSL Behring; results first posted 2017-01-27
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A. Trials, record NCT02093897. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02093897
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A
- NCT number
- NCT02093897
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 84 participants, actual
- Lead sponsor
- CSL Behring
- Conditions
- Congenital Hemophilia A
- Interventions
- rVIII-SingleChain
- First posted
- 2014-03-21
- Last updated
- 2017-01-27
- Other registries' IDs
- eudract:2012-001336-65
- Results first posted
- 2017-01-27
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Treatment Success | Up to 1 year | Rate of treatment success where treatment success of a bleeding episode is defined as a rating of "excellent" or "good" based on the investigator's overall clinical assessment of hemostatic efficacy (using a 4-point scale of excellent, good, moderate or poor/no response) on the on-demand and prophylaxis regimens combined. The rate of success was based on the number of treated bleeding events; there were 347 treated bleeding events in the Efficacy Population. |
| Secondary | Annualized Bleeding Rate | Up to 1 year | The annualized bleeding rate was defined as the number of bleeding episodes requiring treatment divided by the efficacy evaluation period in days, x 365.25, and is presented separately for the on-demand regimen and the prophylaxis regimens. |
| Secondary | Percentage of Bleeding Episodes Requiring 1, 2, 3, or More Than 3 Infusions of rVIII-SingleChain to Achieve Hemostasis. | Up to 1 year | |
| Secondary | Consumption of rVIII-SingleChain - IU/kg Per Subject Per Month | Up to 1 year | |
| Secondary | Consumption of rVIII-SingleChain - IU/kg Per Subject Per Year | Up to 1 year | |
| Secondary | Consumption of rVIII-SingleChain - IU/kg Per Bleeding Event | Up to 1 year | |
| Secondary | Consumption of rVIII-SingleChain (On-demand Regimen) - Number of Infusions Per Subject Per Month | Up to 1 year | |
| Secondary | Consumption of rVIII-SingleChain (On-demand Regimen) - Number of Infusions Per Subject Per Year | Up to 1 year | |
| Secondary | Incremental Recovery | At 1 hour after the start of infusion | Incremental recovery expressed as (IU/dL)/(IU/kg) corrected for subject's predose plasma FVIII activity measured using the chromogenic substrate assay. |
| Secondary | Half-life (t1/2) of rVIII-SingleChain | Immediately before dosing, and at approximately 1, 5, 10, 24, and 48 hours after dosing. | Half-life (t1/2) of rVIII-SingleChain, baseline uncorrected; plasma FVIII activity measured using the chromogenic substrate assay. |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Treatment Success | Up to 1 year | Efficacy Population | Percentage of treated bleeding events | NUMBER |
| SECONDARY | Annualized Bleeding Rate | Up to 1 year | The Efficacy Population comprised all subjects who received at least 1 rVIII-SingleChain dose for prophylaxis or on-demand treatment. One subject was excluded from the efficacy population because of a pre-existing inhibitor to FVIII (confirmed by reexamination of a screening sample initially reported as negative due to laboratory error). | Treated bleeding episodes per year | MEDIAN |
| SECONDARY | Percentage of Bleeding Episodes Requiring 1, 2, 3, or More Than 3 Infusions of rVIII-SingleChain to Achieve Hemostasis. | Up to 1 year | Efficacy Population | Percentage (%) of bleeding episodes | NUMBER |
| SECONDARY | Consumption of rVIII-SingleChain - IU/kg Per Subject Per Month | Up to 1 year | The Efficacy Population comprised all subjects who received at least 1 rVIII-SingleChain dose for prophylaxis or on-demand treatment. One subject was excluded from the efficacy population because of a pre-existing inhibitor to FVIII (confirmed by reexamination of a screening sample initially reported as negative due to laboratory error). | IU/kg per subject per month | MEDIAN |
| SECONDARY | Consumption of rVIII-SingleChain - IU/kg Per Subject Per Year | Up to 1 year | The Efficacy Population comprised all subjects who received at least 1 rVIII-SingleChain dose for prophylaxis or on-demand treatment. One subject was excluded from the efficacy population because of a pre-existing inhibitor to FVIII (confirmed by reexamination of a screening sample initially reported as negative due to laboratory error). | IU/kg per subject per year | MEDIAN |
| SECONDARY | Consumption of rVIII-SingleChain - IU/kg Per Bleeding Event | Up to 1 year | The Efficacy Population comprised all subjects who received at least 1 rVIII-SingleChain dose for prophylaxis or on-demand treatment. One subject was excluded from the efficacy population because of a pre-existing inhibitor to FVIII (confirmed by reexamination of a screening sample initially reported as negative due to laboratory error). | IU/kg per event | MEDIAN |
| SECONDARY | Consumption of rVIII-SingleChain (On-demand Regimen) - Number of Infusions Per Subject Per Month | Up to 1 year | Subjects assigned to the on-demand treatment regimen. | number of infusion per subject per month | MEDIAN |
| SECONDARY | Consumption of rVIII-SingleChain (On-demand Regimen) - Number of Infusions Per Subject Per Year | Up to 1 year | Subjects assigned to the on-demand treatment regimen. | number of infusions per subject per year | MEDIAN |
| SECONDARY | Incremental Recovery | At 1 hour after the start of infusion | PK Population | (IU/dL)/(IU/kg) | MEAN |
| SECONDARY | Half-life (t1/2) of rVIII-SingleChain | Immediately before dosing, and at approximately 1, 5, 10, 24, and 48 hours after dosing. | PK Population | hour | MEAN |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group20
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Treatment Success | Efficacy Population | 96.3 | 95% Confidence Interval 91.3 to 98.4 | Percentage of treated bleeding events | |
| Annualized Bleeding Rate | On-demand | 78.56 | Inter-Quartile Range 35.12 to 86.62 | Treated bleeding episodes per year | |
| Annualized Bleeding Rate | Prophylaxis | 3.69 | Inter-Quartile Range 0.00 to 7.20 | Treated bleeding episodes per year | |
| Percentage of Bleeding Episodes Requiring 1, 2, 3, or More Than 3 Infusions of rVIII-SingleChain to Achieve Hemostasis. | Efficacy Population | Requiring 1 infusion | 85.9 | Percentage (%) of bleeding episodes | |
| Percentage of Bleeding Episodes Requiring 1, 2, 3, or More Than 3 Infusions of rVIII-SingleChain to Achieve Hemostasis. | Efficacy Population | Requiring 2 infusions | 9.8 | Percentage (%) of bleeding episodes | |
| Percentage of Bleeding Episodes Requiring 1, 2, 3, or More Than 3 Infusions of rVIII-SingleChain to Achieve Hemostasis. | Efficacy Population | Requiring 3 infusions | 2.3 | Percentage (%) of bleeding episodes | |
| Percentage of Bleeding Episodes Requiring 1, 2, 3, or More Than 3 Infusions of rVIII-SingleChain to Achieve Hemostasis. | Efficacy Population | Requiring > 3 infusions | 2.0 | Percentage (%) of bleeding episodes | |
| Consumption of rVIII-SingleChain - IU/kg Per Subject Per Month | On-demand | 202 | Full Range 126 to 231 | IU/kg per subject per month | |
| Consumption of rVIII-SingleChain - IU/kg Per Subject Per Month | Prophylaxis | 378 | Full Range 153 to 1394 | IU/kg per subject per month | |
| Consumption of rVIII-SingleChain - IU/kg Per Subject Per Year | On-demand | 2429 | Full Range 1508 to 2771 | IU/kg per subject per year |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | rVIII-SingleChain | 84 | |
| Overall Study | COMPLETED | rVIII-SingleChain | 65 | |
| Overall Study | NOT COMPLETED | rVIII-SingleChain | 19 |
Source ClinicalTrials.gov
Adverse events16
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 | Head injury | Injury, poisoning and procedural complications | rVIII-SingleChain | 5 | 84 | 6 |
| other | Cough | Respiratory, thoracic and mediastinal disorders | rVIII-SingleChain | 7 | 84 | 10 |
| other | Headache | Nervous system disorders | rVIII-SingleChain | 7 | 84 | 9 |
| other | Pyrexia | General disorders | rVIII-SingleChain | 5 | 84 | 5 |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | rVIII-SingleChain | 8 | 84 | 8 |
| other | Nasopharyngitis | Infections and infestations | rVIII-SingleChain | 14 | 84 | 15 |
| serious | Hand fracture | Injury, poisoning and procedural complications | rVIII-SingleChain | 1 | 84 | 1 |
| serious | Laceration | Injury, poisoning and procedural complications | rVIII-SingleChain | 1 | 84 | 1 |
| serious | Splenic rupture | Injury, poisoning and procedural complications | rVIII-SingleChain | 1 | 84 | 1 |
| serious | Inhibiting antibodies positive | Investigations | rVIII-SingleChain | 1 | 84 | 1 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | rVIII-SingleChain | ||
| Total, other adverse events | rVIII-SingleChain | 31 | 84 |
| Total, serious adverse events | rVIII-SingleChain | 9 | 84 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT02093897
GET https://conductscience.org/api/trials/v1/NCT02093897/registered