Trial · NCT04188379
A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).
NCT04188379, Phase 3, Completed, argenx; results first posted 2025-03-13
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP). Trials, record NCT04188379. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04188379
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).
- NCT number
- NCT04188379
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 131 participants, actual
- Lead sponsor
- argenx
- Conditions
- Primary Immune Thrombocytopenia
- Interventions
- efgartigimod, Placebo
- First posted
- 2019-12-05
- Last updated
- 2025-03-13
- Other registries' IDs
- eudract:2019-002100-41
- Results first posted
- 2025-03-13
- Limitations and caveats
- No limitations reported for the study.
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Chronic ITP With a Sustained Platelet Count Response Defined as Achieving Platelet Counts of at Least 50×10^9/L for at Least 4 of the 6 Visits Between Week 19 and 24 of the Trial. | From Week 19 up to Week 24 | Percentage of participants with chronic ITP with a sustained platelet count response was defined as achieving platelet counts of at least 50 × 10\^9/L for at least 4 of the 6 visits between Week 19 and 24 of the study. |
| Secondary | Extent of Disease Control Defined as the Number of Cumulative Weeks Over the Planned 24-week Treatment Period With Platelet Counts of ≥50×10^9/L in the Chronic ITP Population | From Week 1 up to Week 24 | Extent of disease control, defined as the cumulative number of weeks over the planned 24-week treatment period with platelet counts of ≥50 × 10\^9/L in the chronic ITP population. |
| Secondary | Percentage of Participants With a Sustained Platelet Count Response for at Least 4 of the 6 Visits Between Week 19 and 24 of the Study | From Week 19 up to Week 24 | Percentage of participants in the overall population (chronic and persistent ITP) with a sustained platelet count response, defined as achieving platelet counts of at least 50 × 10\^9/L for at least 4 of the 6 visits between weeks 19 and 24 of the study. |
| Secondary | Incidence and Severity of the WHO-classified Bleeding Events | From Week 1 to Week 24 | Incidence of the World Health Organization (WHO)-classified bleeding events in the overall population. This secondary endpoint used the WHO-classified bleeding scale. Bleeding was the predominant clinical manifestation of ITP and was typically related to platelet count. Accordingly, measuring bleeding was important for monitoring this participant population. |
| Secondary | Percentage of Participants in the Overall Population Achieving Platelet Counts of at Least 50×10^9/L for at Least 6 of the 8 Visits Between Week 17 and Week 24 | From Week 17 up to Week 24 | Percentage of participants in the overall population achieving platelet counts of at least 50 × 10\^9/L for at least 6 of the 8 visits between Week 17 and 24 of the study. |
Source ClinicalTrials.gov
Outcomes5
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 Chronic ITP With a Sustained Platelet Count Response Defined as Achieving Platelet Counts of at Least 50×10^9/L for at Least 4 of the 6 Visits Between Week 19 and 24 of the Trial. | From Week 19 up to Week 24 | Full Analysis Set-Chronic population: all randomized participants with chronic ITP. | Percentage of participants | NUMBER |
| SECONDARY | Extent of Disease Control Defined as the Number of Cumulative Weeks Over the Planned 24-week Treatment Period With Platelet Counts of ≥50×10^9/L in the Chronic ITP Population | From Week 1 up to Week 24 | Full Analysis Set-Chronic population: all randomized participants with chronic ITP. | Weeks | MEDIAN |
| SECONDARY | Percentage of Participants With a Sustained Platelet Count Response for at Least 4 of the 6 Visits Between Week 19 and 24 of the Study | From Week 19 up to Week 24 | Full Analysis Set: all randomized participants. | Percentage of participants | NUMBER |
| SECONDARY | Incidence and Severity of the WHO-classified Bleeding Events | From Week 1 to Week 24 | Full Analysis Set: all randomized participants. | bleeding events | MEDIAN |
| SECONDARY | Percentage of Participants in the Overall Population Achieving Platelet Counts of at Least 50×10^9/L for at Least 6 of the 8 Visits Between Week 17 and Week 24 | From Week 17 up to Week 24 | Full Analysis Set: all randomized participants. | Percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group10
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 Chronic ITP With a Sustained Platelet Count Response Defined as Achieving Platelet Counts of at Least 50×10^9/L for at Least 4 of the 6 Visits Between Week 19 and 24 of the Trial. | Efgartigimod | 21.8 | Percentage of participants | ||
Percentage of Participants With Chronic ITP With a Sustained Platelet Count Response Defined as Achieving Platelet Counts of at Least 50×10^9/L for at Least 4 of the 6 Visits Between Week 19 and 24 of the Trial. | Placebo | 5.0 | Percentage of participants | ||
| Extent of Disease Control Defined as the Number of Cumulative Weeks Over the Planned 24-week Treatment Period With Platelet Counts of ≥50×10^9/L in the Chronic ITP Population | Efgartigimod | 2.00 | Inter-Quartile Range 0.00 to 11.00 | Weeks | |
| Extent of Disease Control Defined as the Number of Cumulative Weeks Over the Planned 24-week Treatment Period With Platelet Counts of ≥50×10^9/L in the Chronic ITP Population | Placebo | 0.00 | Inter-Quartile Range 0.00 to 1.00 | Weeks | |
| Percentage of Participants With a Sustained Platelet Count Response for at Least 4 of the 6 Visits Between Week 19 and 24 of the Study | Efgartigimod | 25.6 | Percentage of participants | ||
| Percentage of Participants With a Sustained Platelet Count Response for at Least 4 of the 6 Visits Between Week 19 and 24 of the Study | Placebo | 6.7 | Percentage of participants | ||
| Incidence and Severity of the WHO-classified Bleeding Events | Efgartigimod | 4.0 | Inter-Quartile Range 1.0 to 10.0 | bleeding events | |
| Incidence and Severity of the WHO-classified Bleeding Events | Placebo | 5.0 | Inter-Quartile Range 2.0 to 14.0 | bleeding events | |
| Percentage of Participants in the Overall Population Achieving Platelet Counts of at Least 50×10^9/L for at Least 6 of the 8 Visits Between Week 17 and Week 24 | Efgartigimod | 22.1 | Percentage of participants | ||
| Percentage of Participants in the Overall Population Achieving Platelet Counts of at Least 50×10^9/L for at Least 6 of the 8 Visits Between Week 17 and Week 24 | Placebo | 6.7 | Percentage of participants |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Efgartigimod | 86 | |
| Overall Study | STARTED | Placebo | 45 | |
| Overall Study | COMPLETED | Efgartigimod | 64 | |
| Overall Study | COMPLETED | Placebo | 32 | |
| Overall Study | NOT COMPLETED | Efgartigimod | 22 | |
| Overall Study | NOT COMPLETED | Placebo | 13 |
Source ClinicalTrials.gov
Adverse events76
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 | Haematoma | Vascular disorders | Efgartigimod | 8 | 86 | 17 |
| other | Haematoma | Vascular disorders | Placebo | 11 | 45 | 34 |
| other | Hypertension | Vascular disorders | Efgartigimod | 5 | 86 | 6 |
| other | Hypertension | Vascular disorders | Placebo | 0 | 45 | 0 |
| other | Asthenia | General disorders | Efgartigimod | 6 | 86 | 8 |
| other | Asthenia | General disorders | Placebo | 0 | 45 | 0 |
| other | Heavy menstrual bleeding | Reproductive system and breast disorders | Efgartigimod | 5 | 86 | 9 |
| other | Heavy menstrual bleeding | Reproductive system and breast disorders | Placebo | 3 | 45 | 4 |
| other | Epistaxis | Respiratory, thoracic and mediastinal disorders | Efgartigimod | 8 | 86 | 24 |
| other | Epistaxis | Respiratory, thoracic and mediastinal disorders | Placebo | 8 | 45 | 12 |
1 to 10 of 76Source ClinicalTrials.gov
See all 76 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Efgartigimod | 0 | 86 |
| Total, all-cause mortality | Placebo | 0 | 45 |
| Total, other adverse events | Efgartigimod | 80 | 86 |
| Total, other adverse events | Placebo | 43 | 45 |
| Total, serious adverse events | Efgartigimod | 7 | 86 |
| Total, serious adverse events | Placebo | 7 | 45 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 37778358 | 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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Efgartigimod vs Placebo: deaths from any cause | Trial comparisons |
| Efgartigimod vs Placebo: 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.
Source
- Release
- trials-2026-11-12-0.4.0
- Release manifest SHA-256
- 0f4a8d8c48aee2a7573ae0fa94ecbeab70c1adbe449bbc95c424bc7d3e97b9d4
- Registry capture
- 2026-11-12
- Results copy, AACT
- 2026-03-23
- NIH clinical studies file
- 2026-11-13
Source ClinicalTrials.gov
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNone 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/NCT04188379
GET https://conductscience.org/api/trials/v1/NCT04188379/events?page=2