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Trial ยท NCT02795767

A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors

NCT02795767, Phase 3, Completed, Hoffmann-La Roche; results first posted 2019-04-10

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors. Trials, record NCT02795767. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02795767

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title
A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors
NCT number
NCT02795767
Status
Completed
Phases
Phase 3
Enrollment
88 participants, actual
Lead sponsor
Hoffmann-La Roche
Conditions
Hemophilia A
Interventions
Emicizumab
First posted
2016-06-10
Last updated
2021-06-02
Other registries' IDs
eudract:2016-000073-21
Results first posted
2019-04-10

Source ClinicalTrials.gov

Registered outcomes70

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

ListOutcomeTime frameDescription
PrimaryCohort A: Model-Based Annualized Bleed Rate (ABR) for Treated Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.
The number of treated bleeds over the efficacy period is presented here as a model-based ABR that was analyzed using a negative binomial regression model with efficacy period as an offset to account for the difference in follow-up times. A bleed is considered a "treated bleed" if it is directly followed (i.e., no intervening bleed) by a hemophilia medication reported to be a "treatment for bleed", irrespective of time between treatment and the preceding bleed. A bleed and the first treatment thereafter and before a new bleed starts, are considered to be pairs, with the following exception: if multiple bleeds occur on the same calendar day, the subsequent treatment is considered to apply for each of these multiple bleeds. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure are excluded.
PrimaryCohort A: Model-Based Annualized Bleed Rate (ABR) for All Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.
The number of all bleeds over the efficacy period is presented here as a model-based ABR that was analyzed using a negative binomial regression model with efficacy period as an offset to account for the difference in followup times (i.e., the time that each participant stays in the study). In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule was implemented exactly as defined for the "treated bleeds" outcome measure. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
PrimaryCohort A: Model-Based Annualized Bleed Rate (ABR) for Treated Spontaneous Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.
The number of treated spontaneous bleeds over the efficacy period is presented here as a model-based ABR that was analyzed using a negative binomial regression model with efficacy period as an offset to account for the difference in follow-up times (i.e., the time that each participant stays in the study). A bleed is classified as "spontaneous" if there is no other known contributing factor such as trauma or procedure/surgery. A "treated spontaneous bleed" is a spontaneous bleed that also fulfills the conditions of a treated bleed (see ABR for Treated Bleeds for the definition). Treated bleeds that fulfilled the 72-hour rule were included in the analysis of spontaneous bleeds. Bleeds due to surgery/procedure are excluded.
PrimaryCohort A: Model-Based Annualized Bleed Rate (ABR) for Treated Joint Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.
The number of treated joint bleeds over the efficacy period is presented here as a model-based ABR that was analyzed using a negative binomial regression model with efficacy period as an offset to account for the difference in follow-up times (i.e., the time that each participant stays in the study). A "joint bleed" is defined as a bleed with type reported as "joint" and with at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline. A "treated joint bleed" is a joint bleed that also fulfills the conditions of a treated bleed (see ABR for Treated Bleeds for the definition). Only treated bleeds that fulfilled the 72-hour rule were included in the analysis of treated joint bleeds, excluding bleeds due to surgery/procedure.
PrimaryCohort A: Model-Based Annualized Bleed Rate (ABR) for Treated Target Joint Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.
The number of treated target joint bleeds over the efficacy period is presented here as a model-based ABR that was analyzed using a negative binomial regression model with efficacy period as an offset to account for the difference in follow-up times (i.e., the time that each participant stays in the study). A "target joint bleed" is defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry. A "treated target joint bleed" is a target joint bleed that also fulfills the conditions of a treated bleed (see ABR for Treated Bleeds for the definition). Bleeds due to surgery/procedure are excluded.
PrimaryCohort A: Mean Calculated Annualized Bleed Rate (ABR) for Treated Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.
The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A bleed is considered a "treated bleed" if it is directly followed (i.e., no intervening bleed) by a hemophilia medication reported to be a "treatment for bleed", irrespective of time between treatment and the preceding bleed. A bleed and the first treatment thereafter and before a new bleed starts, are considered to be pairs, with the following exception: if multiple bleeds occur on the same calendar day, the subsequent treatment is considered to apply for each of these multiple bleeds. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure are excluded.
PrimaryCohort A: Mean Calculated Annualized Bleed Rate (ABR) for All Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule was implemented exactly as defined for the "treated bleeds" outcome measure. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
PrimaryCohort A: Mean Calculated Annualized Bleed Rate (ABR) for Treated Spontaneous Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.
The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A bleed is classified as "spontaneous" if there is no other known contributing factor such as trauma or procedure/surgery. A "treated spontaneous bleed" is a spontaneous bleed that also fulfills the conditions of a treated bleed (see ABR for Treated Bleeds for the definition). Treated bleeds that fulfilled the 72-hour rule were included in the analysis of spontaneous bleeds. Bleeds due to surgery/procedure are excluded.
PrimaryCohort A: Mean Calculated Annualized Bleed Rate (ABR) for Treated Joint Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A "joint bleed" is defined as a bleed with type reported as "joint" and with at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline. A "treated joint bleed" is a joint bleed that also fulfills the conditions of a treated bleed (see ABR for Treated Bleeds for the definition). Only treated bleeds that fulfilled the 72-hour rule were included in the analysis of treated joint bleeds, excluding bleeds due to surgery/procedure.
PrimaryCohort A: Mean Calculated Annualized Bleed Rate (ABR) for Treated Target Joint Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A "target joint bleed" is defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry. A "treated target joint bleed" is a target joint bleed that also fulfills the conditions of a treated bleed (see ABR for Treated Bleeds for the definition). Bleeds due to surgery/procedure are excluded.

Outcomes70

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

TypeOutcomeTime framePopulationUnitsMeasured as
PRIMARYCohort A: Model-Based Annualized Bleed Rate (ABR) for Treated Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.All treated participants in Cohort A who were on the same dose of emicizumab for at least 12 weeks and were \<12 years of agetreated bleed rate per yearNUMBER
PRIMARYCohort A: Model-Based Annualized Bleed Rate (ABR) for All Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.All treated participants in Cohort A who were on the same dose of emicizumab for at least 12 weeks and were \<12 years of ageall bleed rate per yearNUMBER
PRIMARYCohort A: Model-Based Annualized Bleed Rate (ABR) for Treated Spontaneous Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.All treated participants in Cohort A who were on the same dose of emicizumab for at least 12 weeks and were \<12 years of agetreated spontaneous bleed rate per yearNUMBER
PRIMARYCohort A: Model-Based Annualized Bleed Rate (ABR) for Treated Joint Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.All treated participants in Cohort A who were on the same dose of emicizumab for at least 12 weeks and were \<12 years of agetreated joint bleed rate per yearNUMBER
PRIMARYCohort A: Model-Based Annualized Bleed Rate (ABR) for Treated Target Joint Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.All treated participants in Cohort A who were on the same dose of emicizumab for at least 12 weeks and were \<12 years of agetreated target joint bleed rate per yearNUMBER
PRIMARYCohort A: Mean Calculated Annualized Bleed Rate (ABR) for Treated Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.All treated participants in Cohort A who were on the same dose of emicizumab for at least 12 weeks and were \<12 years of agetreated bleed rate per yearMEAN
PRIMARYCohort A: Mean Calculated Annualized Bleed Rate (ABR) for All Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.All treated participants in Cohort A who were on the same dose of emicizumab for at least 12 weeks and were \<12 years of ageall bleed rate per yearMEAN
PRIMARYCohort A: Mean Calculated Annualized Bleed Rate (ABR) for Treated Spontaneous Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.All treated participants in Cohort A who were on the same dose of emicizumab for at least 12 weeks and were \<12 years of agetreated spontaneous bleed rate per yearMEAN
PRIMARYCohort A: Mean Calculated Annualized Bleed Rate (ABR) for Treated Joint Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.All treated participants in Cohort A who were on the same dose of emicizumab for at least 12 weeks and were \<12 years of agetreated joint bleed rate per yearMEAN
PRIMARYCohort A: Mean Calculated Annualized Bleed Rate (ABR) for Treated Target Joint Bleeds in Treated Participants <12 Years of AgeFrom Baseline to 52 weeks; At the primary completion date, the median (min-max) duration of the efficacy period in Cohort A was 57.57 (17.9-92.6) weeks.All treated participants in Cohort A who were on the same dose of emicizumab for at least 12 weeks and were \<12 years of agetreated target joint bleed rate per yearMEAN

Results by group704

Each measurement the trial posted, group by group, in the words and numbers it posted.

OutcomeGroupCategoryValueSpreadUnits
Cohort A: Model-Based Annualized Bleed Rate (ABR) for Treated Bleeds in Treated Participants <12 Years of AgeCohort A: 1.5 mg/kg Emicizumab QW0.395% Confidence Interval 0.17 to 0.50treated bleed rate per year
Cohort A: Model-Based Annualized Bleed Rate (ABR) for All Bleeds in Treated Participants <12 Years of AgeCohort A: 1.5 mg/kg Emicizumab QW3.295% Confidence Interval 1.94 to 5.22all bleed rate per year
Cohort A: Model-Based Annualized Bleed Rate (ABR) for Treated Spontaneous Bleeds in Treated Participants <12 Years of AgeCohort A: 1.5 mg/kg Emicizumab QW0.095% Confidence Interval 0.0 to 0.1treated spontaneous bleed rate per year
Cohort A: Model-Based Annualized Bleed Rate (ABR) for Treated Joint Bleeds in Treated Participants <12 Years of AgeCohort A: 1.5 mg/kg Emicizumab QW0.295% Confidence Interval 0.08 to 0.29treated joint bleed rate per year
Cohort A: Model-Based Annualized Bleed Rate (ABR) for Treated Target Joint Bleeds in Treated Participants <12 Years of AgeCohort A: 1.5 mg/kg Emicizumab QWNA95% Confidence Interval NA to NAtreated target joint bleed rate per year
Cohort A: Mean Calculated Annualized Bleed Rate (ABR) for Treated Bleeds in Treated Participants <12 Years of AgeCohort A: 1.5 mg/kg Emicizumab QW0.395% Confidence Interval 0.00 to 4.31treated bleed rate per year
Cohort A: Mean Calculated Annualized Bleed Rate (ABR) for All Bleeds in Treated Participants <12 Years of AgeCohort A: 1.5 mg/kg Emicizumab QW3.295% Confidence Interval 0.70 to 9.04all bleed rate per year
Cohort A: Mean Calculated Annualized Bleed Rate (ABR) for Treated Spontaneous Bleeds in Treated Participants <12 Years of AgeCohort A: 1.5 mg/kg Emicizumab QW0.095% Confidence Interval 0.00 to 3.74treated spontaneous bleed rate per year
Cohort A: Mean Calculated Annualized Bleed Rate (ABR) for Treated Joint Bleeds in Treated Participants <12 Years of AgeCohort A: 1.5 mg/kg Emicizumab QW0.295% Confidence Interval 0.00 to 4.01treated joint bleed rate per year
Cohort A: Mean Calculated Annualized Bleed Rate (ABR) for Treated Target Joint Bleeds in Treated Participants <12 Years of AgeCohort A: 1.5 mg/kg Emicizumab QW0.195% Confidence Interval 0.00 to 3.84treated target joint bleed rate per year

Participant flow18

How many started, completed or left each group, period by period.

PeriodMilestoneGroupParticipantsUnits counted
Overall StudySTARTEDCohort A: 1.5 mg/kg Emicizumab QW68
Overall StudySTARTEDCohort B: 3 mg/kg Emicizumab Q2W10
Overall StudySTARTEDCohort C: 6 mg/kg Emicizumab Q4W10
Overall StudyReceived at Least One Dose of EmicizumabCohort A: 1.5 mg/kg Emicizumab QW68
Overall StudyReceived at Least One Dose of EmicizumabCohort B: 3 mg/kg Emicizumab Q2W10
Overall StudyReceived at Least One Dose of EmicizumabCohort C: 6 mg/kg Emicizumab Q4W10
Overall StudyDose Up-Titrated to 3 mg/kg QWCohort A: 1.5 mg/kg Emicizumab QW0
Overall StudyDose Up-Titrated to 3 mg/kg QWCohort B: 3 mg/kg Emicizumab Q2W0
Overall StudyDose Up-Titrated to 3 mg/kg QWCohort C: 6 mg/kg Emicizumab Q4W3
Overall StudyCompleted 52 Weeks in StudyCohort A: 1.5 mg/kg Emicizumab QW67

Adverse events261

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

TypeEventOrgan systemGroupAffectedAt riskEvents
otherAnaemiaBlood and lymphatic system disordersCohort A: 1.5 mg/kg Emicizumab QW2682
otherAnaemiaBlood and lymphatic system disordersCohort B: 3 mg/kg Emicizumab Q2W0100
otherAnaemiaBlood and lymphatic system disordersCohort C: 6 mg/kg Emicizumab Q4W1101
otherAbdominal painGastrointestinal disordersCohort A: 1.5 mg/kg Emicizumab QW4684
otherAbdominal painGastrointestinal disordersCohort B: 3 mg/kg Emicizumab Q2W2102
otherAbdominal painGastrointestinal disordersCohort C: 6 mg/kg Emicizumab Q4W0100
otherAbdominal pain upperGastrointestinal disordersCohort A: 1.5 mg/kg Emicizumab QW4686
otherAbdominal pain upperGastrointestinal disordersCohort B: 3 mg/kg Emicizumab Q2W0100
otherAbdominal pain upperGastrointestinal disordersCohort C: 6 mg/kg Emicizumab Q4W1101
otherConstipationGastrointestinal disordersCohort A: 1.5 mg/kg Emicizumab QW4684

Adverse event totals9

The totals the trial posted for each group.

TotalGroupAffectedAt risk
Total, all-cause mortalityCohort A: 1.5 mg/kg Emicizumab QW068
Total, all-cause mortalityCohort B: 3 mg/kg Emicizumab Q2W010
Total, all-cause mortalityCohort C: 6 mg/kg Emicizumab Q4W010
Total, other adverse eventsCohort A: 1.5 mg/kg Emicizumab QW6468
Total, other adverse eventsCohort B: 3 mg/kg Emicizumab Q2W910
Total, other adverse eventsCohort C: 6 mg/kg Emicizumab Q4W1010
Total, serious adverse eventsCohort A: 1.5 mg/kg Emicizumab QW2368
Total, serious adverse eventsCohort B: 3 mg/kg Emicizumab Q2W110
Total, serious adverse eventsCohort C: 6 mg/kg Emicizumab Q4W310

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMIDListed asSource
31697801DERIVEDPubMed
35939785DERIVEDPubMed

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.

ClinicalTrials.gov
2026-11-12
ClinicalTrials.gov
Results, from AACT
2026-03-23
AACT
NIH grant links
2026-11-13
NIH RePORTER

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/NCT02795767

GET https://conductscience.org/api/trials/v1/NCT02795767/registered?page=2