Trial · NCT04687072
A Study to Evaluate the Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia
NCT04687072, Phase 3, Completed, argenx; results first posted 2024-10-31
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. A Study to Evaluate the Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia. Trials, record NCT04687072. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04687072
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate the Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia
- NCT number
- NCT04687072
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 207 participants, actual
- Lead sponsor
- argenx
- Conditions
- Primary Immune Thrombocytopenia
- Interventions
- Efgartigimod PH20 SC, Placebo PH20 SC
- First posted
- 2020-12-29
- Last updated
- 2024-10-31
- Other registries' IDs
- eudract:2020-004032-21
- Results first posted
- 2024-10-31
Source ClinicalTrials.gov
Registered outcomes24
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Chronic Immune Thrombocytopenia (ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24 | Up to 6 weeks (between Weeks 19 and 24) | A participant was considered a responder for this endpoint (i.e., had a sustained platelet count response) if the participant had platelet counts of ≥50 × 10\^9/L for ≥4 of the 6 analysis visits between Weeks 19 and 24. |
| Secondary | Extent of Disease Control Over the 24-Week Treatment Period in the Chronic ITP Population | Up to 24 weeks | Extent of disease control was 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. |
| Secondary | Percentage of Participants in the Overall Population (Chronic and Persistent ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24 | Up to 6 weeks (between Weeks 19 and 24) | A participant was considered a responder for this endpoint (i.e., had a sustained platelet count response) if the participant had platelet counts of ≥50 × 10\^9/L for ≥4 of the 6 analysis visits between Weeks 19 and 24. |
| Secondary | Percentage of Participants in the Overall Population With Sustained Platelet Count Response Between Weeks 17 and 24 | Up to 8 weeks (between Weeks 17 and 24) | A participant was considered a responder for this endpoint (i.e., had a sustained platelet count response) if the participant had platelet counts of ≥50 × 10\^9/L for ≥6 of the 8 analysis visits between weeks 17 and 24. |
| Secondary | Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time During the 24-week Treatment Period | Up to 24 weeks | A participant was considered a responder for this endpoint (i.e., had an overall platelet count response) if the participant had platelet counts of ≥50 × 10\^9/L for ≥4 analysis visits at any time during the 24-week treatment period. |
| Secondary | Extent of Disease Control Until Week 12 in the Overall Population | Up to 12 weeks | Extent of disease control was defined as the number of cumulative weeks until Week 12 with platelet counts of ≥50×10\^9/L in the overall population. |
| Secondary | Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time Until Week 12 | Up to 12 weeks | A participant was considered a responder for this endpoint (i.e., had an overall platelet count response) if the participant had platelet counts of ≥50 × 10\^9/L for ≥4 analysis visits at any time until Week 12. |
| Secondary | Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population | Baseline and Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, Safety and Efficacy Follow-up Visit 1 (SEFU1) (up to Week 29), and SEFU2 (up to Week 33) | Change from Baseline at time point t = value at time point t - Baseline value. Baseline was defined as the last available value prior to first administration of the investigational medicinal product (IMP). |
| Secondary | Time to Platelet Count Response in the Overall Population | Up to 24 weeks | Time to platelet count response, defined as the time to have 2 consecutive platelet counts of ≥50 × 10\^9/L via Kaplan-Meier estimates. |
| Secondary | Extent of Disease Control Over the 24-Week Treatment Period in the Overall Population | Up to 24 weeks | Extent of disease control was defined as the number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥30×10\^9/L with at least ≥20×10\^9/L above Baseline in the overall population. |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 registered outcomesOutcomes24
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 Immune Thrombocytopenia (ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24 | Up to 6 weeks (between Weeks 19 and 24) | Full Analysis Set (FAS)-Chronic: Participants from the FAS (all randomized participants) including only participants with chronic ITP. | percentage of participants | NUMBER |
| SECONDARY | Extent of Disease Control Over the 24-Week Treatment Period in the Chronic ITP Population | Up to 24 weeks | FAS-Chronic: Participants from the FAS (all randomized participants) including only participants with chronic ITP. | weeks | MEDIAN |
| SECONDARY | Percentage of Participants in the Overall Population (Chronic and Persistent ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24 | Up to 6 weeks (between Weeks 19 and 24) | FAS: All randomized participants. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants in the Overall Population With Sustained Platelet Count Response Between Weeks 17 and 24 | Up to 8 weeks (between Weeks 17 and 24) | FAS: All randomized participants. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time During the 24-week Treatment Period | Up to 24 weeks | FAS: All randomized participants. | percentage of participants | NUMBER |
| SECONDARY | Extent of Disease Control Until Week 12 in the Overall Population | Up to 12 weeks | FAS: All randomized participants. | weeks | MEDIAN |
| SECONDARY | Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time Until Week 12 | Up to 12 weeks | FAS: All randomized participants. | percentage of participants | NUMBER |
| SECONDARY | Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population | Baseline and Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, Safety and Efficacy Follow-up Visit 1 (SEFU1) (up to Week 29), and SEFU2 (up to Week 33) | FAS: All randomized participants with available data. | platelets x10^9/L | MEAN |
| SECONDARY | Time to Platelet Count Response in the Overall Population | Up to 24 weeks | FAS: All randomized participants. Participants with no occurrence of platelet count response, early discontinuation of treatment, or dose and/or frequency of concurrent ITP therapy increased or a new ITP therapy were censored. If multiple censoring conditions apply, the earliest censoring date is considered. | days | MEDIAN |
| SECONDARY | Extent of Disease Control Over the 24-Week Treatment Period in the Overall Population | Up to 24 weeks | FAS: All randomized participants. | weeks | MEDIAN |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 outcomesResults by group166
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 Immune Thrombocytopenia (ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24 | Efgartigimod PH20 SC | 13.7 | percentage of participants | ||
| Percentage of Participants With Chronic Immune Thrombocytopenia (ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24 | Placebo PH20 SC | 16.2 | percentage of participants | ||
| Extent of Disease Control Over the 24-Week Treatment Period in the Chronic ITP Population | Efgartigimod PH20 SC | 0.5 | Full Range 0 to 24 | weeks | |
| Extent of Disease Control Over the 24-Week Treatment Period in the Chronic ITP Population | Placebo PH20 SC | 0.0 | Full Range 0 to 23 | weeks | |
| Percentage of Participants in the Overall Population (Chronic and Persistent ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24 | Efgartigimod PH20 SC | 16.1 | percentage of participants | ||
| Percentage of Participants in the Overall Population (Chronic and Persistent ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24 | Placebo PH20 SC | 15.7 | percentage of participants | ||
| Percentage of Participants in the Overall Population With Sustained Platelet Count Response Between Weeks 17 and 24 | Efgartigimod PH20 SC | 12.4 | percentage of participants | ||
| Percentage of Participants in the Overall Population With Sustained Platelet Count Response Between Weeks 17 and 24 | Placebo PH20 SC | 14.3 | percentage of participants | ||
| Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time During the 24-week Treatment Period | Efgartigimod PH20 SC | 29.9 | percentage of participants | ||
| Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time During the 24-week Treatment Period | Placebo PH20 SC | 25.7 | percentage of participants |
1 to 10 of 166Source ClinicalTrials.gov
See all 166 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Efgartigimod PH20 SC | 137 | |
| Overall Study | STARTED | Placebo PH20 SC | 70 | |
| Overall Study | COMPLETED | Efgartigimod PH20 SC | 113 | |
| Overall Study | COMPLETED | Placebo PH20 SC | 62 | |
| Overall Study | NOT COMPLETED | Efgartigimod PH20 SC | 24 | |
| Overall Study | NOT COMPLETED | Placebo PH20 SC | 8 |
Source ClinicalTrials.gov
Adverse events112
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 | Anaemia | Blood and lymphatic system disorders | Efgartigimod PH20 SC | 10 | 137 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo PH20 SC | 6 | 70 | |
| other | Diarrhoea | Gastrointestinal disorders | Efgartigimod PH20 SC | 7 | 137 | |
| other | Diarrhoea | Gastrointestinal disorders | Placebo PH20 SC | 1 | 70 | |
| other | Dyspepsia | Gastrointestinal disorders | Efgartigimod PH20 SC | 3 | 137 | |
| other | Dyspepsia | Gastrointestinal disorders | Placebo PH20 SC | 5 | 70 | |
| other | Gingival bleeding | Gastrointestinal disorders | Efgartigimod PH20 SC | 14 | 137 | |
| other | Gingival bleeding | Gastrointestinal disorders | Placebo PH20 SC | 10 | 70 | |
| other | Mouth haemorrhage | Gastrointestinal disorders | Efgartigimod PH20 SC | 19 | 137 | |
| other | Mouth haemorrhage | Gastrointestinal disorders | Placebo PH20 SC | 6 | 70 |
1 to 10 of 112Source ClinicalTrials.gov
See all 112 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Efgartigimod PH20 SC | 1 | 137 |
| Total, all-cause mortality | Placebo PH20 SC | 0 | 70 |
| Total, other adverse events | Efgartigimod PH20 SC | 130 | 137 |
| Total, other adverse events | Placebo PH20 SC | 65 | 70 |
| Total, serious adverse events | Efgartigimod PH20 SC | 14 | 137 |
| Total, serious adverse events | Placebo PH20 SC | 10 | 70 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 41943179 | 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 PH20 SC vs Placebo PH20 SC: deaths from any cause | Trial comparisons |
| Efgartigimod PH20 SC vs Placebo PH20 SC: 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/NCT04687072
GET https://conductscience.org/api/trials/v1/NCT04687072/registered?page=2