Trial · NCT03277313
Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric PIDD Subjects
NCT03277313, Phase 3, Completed, Baxalta now part of Shire; results first posted 2023-10-23
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric PIDD Subjects. Trials, record NCT03277313. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03277313
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric PIDD Subjects
- NCT number
- NCT03277313
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 44 participants, actual
- Lead sponsor
- Baxalta now part of Shire
- Conditions
- Primary Immunodeficiency Diseases (PID)
- Interventions
- HYQVIA
- First posted
- 2017-09-11
- Last updated
- 2023-10-23
- Results first posted
- 2023-10-23
Source ClinicalTrials.gov
Registered outcomes47
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Rate Represented as Mean Number of Acute Serious Bacterial Infections (ASBI) Per Participant-year | From first dose of study drug up to end of Study Epoch 2 (up to approximately 37.2 months) | The rate of ASBI was defined as the mean number of ASBI per participant-year based on the United States (U.S.) Food and drugs Administration (FDA) guidance for industry to support marketing of human immune globulin intravenous (IGIV) as replacement therapy for primary humoral immunodeficiency and the European Medicines Agency (EMA) guideline on the clinical investigation of human normal immunoglobulin for SC and /or intramuscular administration. ASBI included bacteremia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess, diagnosed according to the Diagnostic Criteria for Acute Serious Bacterial Infections. |
| Secondary | Rate Represented as Mean Number of All Infections Per Participant-year | From first dose of study drug up to end of Study Epoch 2 (up to approximately 37.2 months) | The rate of all infections was defined as the mean number of all infections per participant-year. Number of all infections was calculated as number of infections per participant-year. |
| Secondary | Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses | Study Epoch 2, Year 1: Months 0, 6, and 12; Year 2: Months 18, 24 and 36 | |
| Secondary | Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogens Categorized as Clostridium Tetani Toxoid and Hepatitis B Virus | Study Epoch 2, Year 2: Months 6, 24, and 36 | |
| Secondary | Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogen Haemophilus Influenzae B | Study Epoch 2, Year 2: Months 6, 24, and 36 | |
| Secondary | Epoch 2: Area Under the Curve Normalized for Week (AUCweek) | Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion | |
| Secondary | Epoch 2: Area Under the Curve Over the Infusion Interval (AUCTau) | Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion | |
| Secondary | Epoch 2: Apparent Clearance (CL/F) | Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion | |
| Secondary | Epoch 2: Maximum Concentration (Cmax) | Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion | |
| Secondary | Epoch 2: Minimum Concentration (Cmin) | Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion |
1 to 10 of 47Source ClinicalTrials.gov
See all 47 registered outcomesOutcomes47
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Rate Represented as Mean Number of Acute Serious Bacterial Infections (ASBI) Per Participant-year | From first dose of study drug up to end of Study Epoch 2 (up to approximately 37.2 months) | FAS included all participants who provided informed consent, and met enrollment eligibility. | ASBI per participant-year | MEAN |
| SECONDARY | Rate Represented as Mean Number of All Infections Per Participant-year | From first dose of study drug up to end of Study Epoch 2 (up to approximately 37.2 months) | FAS included all participants who provided informed consent, and met enrollment eligibility. | infections per participant-year | MEAN |
| SECONDARY | Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses | Study Epoch 2, Year 1: Months 0, 6, and 12; Year 2: Months 18, 24 and 36 | FAS=all participants who provided informed consent, and met enrollment eligibility. Only FAS participants in Epoch 2 were analyzed for this outcome measure. Overall number analyzed=number of participants with data available for analyses. Number analyzed=number of participants with data available for analysis at specified timepoint. | grams per liter (g/L) | MEAN |
| SECONDARY | Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogens Categorized as Clostridium Tetani Toxoid and Hepatitis B Virus | Study Epoch 2, Year 2: Months 6, 24, and 36 | FAS=all participants who provided informed consent, and met enrollment eligibility. Only FAS participants in Epoch 2 were analyzed for this outcome measure. Overall number analyzed=number of participants with data available for analyses. Number analyzed=number of participants with data available for analysis at specified timepoint. | international unit per milliliter(IU/mL) | MEAN |
| SECONDARY | Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogen Haemophilus Influenzae B | Study Epoch 2, Year 2: Months 6, 24, and 36 | FAS=all participants who provided informed consent, and met enrollment eligibility. Only FAS participants in Epoch 2 were analyzed for this outcome measure. Overall number analyzed=number of participants with data available for analyses. Number analyzed=number of participants with data available for analysis at specified timepoint. | milligrams per liter (mg/L) | MEAN |
| SECONDARY | Epoch 2: Area Under the Curve Normalized for Week (AUCweek) | Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion | Pharmacokinetic analysis set (PKAS) included all participants in the Safety Analysis Set who have at least 1 evaluable post-dose concentration data for PK assessments without major protocol deviations or events affecting the PK results. Overall number analyzed is the number of participants with data available for analyses. As pre-specified in statistical analysis plan (SAP), this outcome measure was planned only for Epoch 2. | grams*day per liter (g*day/L) | GEOMETRIC_MEAN |
| SECONDARY | Epoch 2: Area Under the Curve Over the Infusion Interval (AUCTau) | Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion | PKAS included all participants in the Safety Analysis Set who have at least 1 evaluable post-dose concentration data for PK assessments without major protocol deviations or events affecting the PK results. Overall number analyzed is the number of participants with data available for analyses. As pre-specified in SAP, this outcome measure was planned only for Epoch 2. | g*day/L | GEOMETRIC_MEAN |
| SECONDARY | Epoch 2: Apparent Clearance (CL/F) | Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion | PKAS included all participants in the Safety Analysis Set who have at least 1 evaluable post-dose concentration data for PK assessments without major protocol deviations or events affecting the PK results. Overall number analyzed is the number of participants with data available for analyses. As pre-specified in SAP, this outcome measure was planned only for Epoch 2. | milliliters per day (mL/day) | GEOMETRIC_MEAN |
| SECONDARY | Epoch 2: Maximum Concentration (Cmax) | Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion | PKAS included all participants in the Safety Analysis Set who have at least 1 evaluable post-dose concentration data for PK assessments without major protocol deviations or events affecting the PK results. Overall number analyzed is the number of participants with data available for analyses. As pre-specified in SAP, this outcome measure was planned only for Epoch 2. | g/L | GEOMETRIC_MEAN |
| SECONDARY | Epoch 2: Minimum Concentration (Cmin) | Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion | PKAS included all participants in the Safety Analysis Set who have at least 1 evaluable post-dose concentration data for PK assessments without major protocol deviations or events affecting the PK results. Overall number analyzed is the number of participants with data available for analyses. As pre-specified in SAP, this outcome measure was planned only for Epoch 2. | g/L | GEOMETRIC_MEAN |
1 to 10 of 47Source ClinicalTrials.gov
See all 47 outcomesResults by group179
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Rate Represented as Mean Number of Acute Serious Bacterial Infections (ASBI) Per Participant-year | Epoch 1 + Epoch 2 | 0.04 | Standard Error 0.027 | ASBI per participant-year | |
| Rate Represented as Mean Number of All Infections Per Participant-year | Epoch 1 + Epoch 2 | 3.12 | Standard Error 0.450 | infections per participant-year | |
| Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses | Epoch 2 | IgG Total: Month 0 | 10.381 | Standard Deviation 2.9191 | grams per liter (g/L) |
| Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses | Epoch 2 | IgG Total: Month 6 | 9.199 | Standard Deviation 1.9577 | grams per liter (g/L) |
| Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses | Epoch 2 | IgG Total: Month 12 | 9.214 | Standard Deviation 1.9800 | grams per liter (g/L) |
| Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses | Epoch 2 | IgG Total: Month 18 | 13.80 | Standard Deviation NA | grams per liter (g/L) |
| Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses | Epoch 2 | IgG Total: Month 24 | 10.80 | Standard Deviation NA | grams per liter (g/L) |
| Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses | Epoch 2 | IgG Total: Month 36 | 13.50 | Standard Deviation NA | grams per liter (g/L) |
| Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses | Epoch 2 | IgG Subclass 1: Month 0 | 5.888 | Standard Deviation 2.7193 | grams per liter (g/L) |
| Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses | Epoch 2 | IgG Subclass 1: Month 6 | 5.317 | Standard Deviation 1.4887 | grams per liter (g/L) |
1 to 10 of 179Source ClinicalTrials.gov
See all 179 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Epoch 1 (Six-week Ramp-up Period) | STARTED | Epoch 1 | 44 | |
| Epoch 1 (Six-week Ramp-up Period) | STARTED | Epoch 2 | 0 | |
| Epoch 1 (Six-week Ramp-up Period) | COMPLETED | Epoch 1 | 43 | |
| Epoch 1 (Six-week Ramp-up Period) | COMPLETED | Epoch 2 | 0 | |
| Epoch 1 (Six-week Ramp-up Period) | NOT COMPLETED | Epoch 1 | 1 | |
| Epoch 1 (Six-week Ramp-up Period) | NOT COMPLETED | Epoch 2 | 0 | |
| Epoch 2 (36 Months After Epoch 1) | STARTED | Epoch 1 | 0 | |
| Epoch 2 (36 Months After Epoch 1) | STARTED | Epoch 2 | 43 | |
| Epoch 2 (36 Months After Epoch 1) | COMPLETED | Epoch 1 | 0 | |
| Epoch 2 (36 Months After Epoch 1) | COMPLETED | Epoch 2 | 34 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events82
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 | Abdominal pain | Gastrointestinal disorders | Epoch 1 | 2 | 44 | |
| other | Abdominal pain | Gastrointestinal disorders | Epoch 2 | 3 | 43 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Epoch 1 | 1 | 44 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Epoch 2 | 3 | 43 | |
| other | Acute sinusitis | Infections and infestations | Epoch 1 | 0 | 44 | |
| other | Acute sinusitis | Infections and infestations | Epoch 2 | 5 | 43 | |
| other | Asthma | Respiratory, thoracic and mediastinal disorders | Epoch 1 | 1 | 44 | |
| other | Asthma | Respiratory, thoracic and mediastinal disorders | Epoch 2 | 5 | 43 | |
| other | Back pain | Musculoskeletal and connective tissue disorders | Epoch 1 | 0 | 44 | |
| other | Back pain | Musculoskeletal and connective tissue disorders | Epoch 2 | 3 | 43 |
1 to 10 of 82Source ClinicalTrials.gov
See all 82 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Epoch 1 | 0 | 44 |
| Total, all-cause mortality | Epoch 2 | 0 | 43 |
| Total, other adverse events | Epoch 1 | 28 | 44 |
| Total, other adverse events | Epoch 2 | 40 | 43 |
| Total, serious adverse events | Epoch 1 | 1 | 44 |
| Total, serious adverse events | Epoch 2 | 3 | 43 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 41937917 | 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 comparisons4
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Epoch 1 vs Epoch 2: Number of Participants With All Temporally Associated TEAEs Excluding Infections | Trial comparisons |
| Epoch 1 vs Epoch 2: Number of Participants With All Related (Causally) and/or Temporally Associated TEAEs Excluding Infections | Trial comparisons |
| Epoch 1 vs Epoch 2: deaths from any cause | Trial comparisons |
| Epoch 1 vs Epoch 2: 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.
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/NCT03277313
GET https://conductscience.org/api/trials/v1/NCT03277313/registered?page=2