Trial · NCT04281472
A Study to Assess the Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves)
NCT04281472, Phase 2, Completed, argenx; results first posted 2024-08-20
3 sourcestrials-2026-11-12-0.4.0
Download JSON
Cite this record
ConductScience Library. A Study to Assess the Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves). Trials, record NCT04281472. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04281472
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
A Study to Assess the Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves)
- NCT number
- NCT04281472
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 322 participants, actual
- Lead sponsor
- argenx
- Conditions
- Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
- Interventions
- efgartigimod PH20 SC in stage B, placebo in stage B
- First posted
- 2020-02-24
- Last updated
- 2024-08-20
- Other registries' IDs
- eudract:2019-003076-39
- Results first posted
- 2024-08-20
Source ClinicalTrials.gov
Registered outcomes26
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Stage A: Percentage of Participants With Confirmed Evidence of Clinical Improvement(ECI) | Up to 12 weeks during the open-label stage A | |
| Primary | Stage B: Time to First Adjusted INCAT Deterioration Compared to Stage B Baseline | Up to 48 weeks during the randomized placebo-controlled stage B | |
| Secondary | Stage A: Time to Initial Confirmed Evidence of Clinical Improvement (ECI) | Up to 12 weeks during the open-label stage A | |
| Secondary | Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Adjusted INCAT Score | Up to 12 weeks during the open-label stage A | Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) scores range from 0-10 with a score of 10 indicating the greatest degree of disability. |
| Secondary | Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Medical Research Council (MRC) Sum Score | Up to 12 weeks during the open-label stage A | The Medical Research Council (MRC) Sum scores range from 0 to 60 with a lower score indicating greater muscle weakness. |
| Secondary | Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in I-RODS Disability Scores | Up to 12 weeks during the open-label stage A | The Inflammatory Rasch-built Overall Disability Scale (I-RODS) score ranges from 0-100, with lower scores indicating the greatest degree of disability. |
| Secondary | Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in TUG Score | Up to 12 weeks during the open-label stage A | The Timed Up and Go (TUG) score is calculated as the number of seconds needed to complete a series of actions. The longer time needed to complete this test (expressed in seconds) indicates lower mobility. |
| Secondary | Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Mean Grip Strength | Up to 12 weeks during the open-label stage A | This is measured with a handheld device called a vigometer |
| Secondary | Stage A: Exposure Adjusted Occurrence of Treatment-emergent Adverse Events and Serious Adverse Events | Up to 12 weeks during the open-label stage A | Treatment-emergent (serious) AEs expressed in number of events/100 PYFU (participant years of follow-up) |
| Secondary | Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time | Up to 13 weeks during the open-label stage A (12 weeks + optional 1 additional week to confirm evidence of clinical improvement (ECI)) |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 registered outcomesOutcomes26
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Stage A: Percentage of Participants With Confirmed Evidence of Clinical Improvement(ECI) | Up to 12 weeks during the open-label stage A | SAF-A (Stage A safety analysis set): Participants who received at least 1 dose or part of a dose of efgartigimod PH20 SC in Stage A | percentage of participants | NUMBER |
| PRIMARY | Stage B: Time to First Adjusted INCAT Deterioration Compared to Stage B Baseline | Up to 48 weeks during the randomized placebo-controlled stage B | mITT (modified intent-to-treat) analysis set; Participants who were randomized in Stage B and who received at least 1 dose or part of a dose of IMP in Stage B. | Days | MEDIAN |
| SECONDARY | Stage A: Time to Initial Confirmed Evidence of Clinical Improvement (ECI) | Up to 12 weeks during the open-label stage A | SAF-A (Stage A safety analysis set): Participants who received at least 1 dose or part of a dose of efgartigimod PH20 SC in Stage A | days | NUMBER |
| SECONDARY | Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Adjusted INCAT Score | Up to 12 weeks during the open-label stage A | SAF-A (Stage A safety analysis set): Participants who received at least 1 dose or part of a dose of efgartigimod PH20 SC in Stage A | score on a scale | MEAN |
| SECONDARY | Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Medical Research Council (MRC) Sum Score | Up to 12 weeks during the open-label stage A | SAF-A (Stage A safety analysis set): Participants who received at least 1 dose or part of a dose of efgartigimod PH20 SC in Stage A | score on a scale | MEAN |
| SECONDARY | Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in I-RODS Disability Scores | Up to 12 weeks during the open-label stage A | SAF-A (Stage A safety analysis set): Participants who received at least 1 dose or part of a dose of efgartigimod PH20 SC in Stage A | score on a scale | MEAN |
| SECONDARY | Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in TUG Score | Up to 12 weeks during the open-label stage A | SAF-A (Stage A safety analysis set): Participants who received at least 1 dose or part of a dose of efgartigimod PH20 SC in Stage A | seconds | MEAN |
| SECONDARY | Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Mean Grip Strength | Up to 12 weeks during the open-label stage A | SAF-A (Stage A safety analysis set): Participants who received at least 1 dose or part of a dose of efgartigimod PH20 SC in Stage A | Kilopascal (kPa) | MEAN |
| SECONDARY | Stage A: Exposure Adjusted Occurrence of Treatment-emergent Adverse Events and Serious Adverse Events | Up to 12 weeks during the open-label stage A | SAF-A (Stage A safety analysis set): Participants who received at least 1 dose or part of a dose of efgartigimod PH20 SC in Stage A | Events/100 PYFU | NUMBER |
| SECONDARY | Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time | Up to 13 weeks during the open-label stage A (12 weeks + optional 1 additional week to confirm evidence of clinical improvement (ECI)) | The number analyzed in rows decreases due to participants withdrawing from the study and missing participant data. Week 13 was an optional additional week for the confirmation of the ECI. PK-A (Stage A PK analysis set): Participants from the SAF-A for whom at least 1 serum PK concentration during Stage A is available | ug/mL | MEAN |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 outcomesResults by group125
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Stage A: Percentage of Participants With Confirmed Evidence of Clinical Improvement(ECI) | Stage A: Efgartigimod PH20 SC | 66.5 | 95% Confidence Interval 61.0 to 71.6 | percentage of participants | |
| Stage B: Time to First Adjusted INCAT Deterioration Compared to Stage B Baseline | Stage B: Efgartigimod PH20 SC | NA | 95% Confidence Interval NA to NA | Days | |
| Stage B: Time to First Adjusted INCAT Deterioration Compared to Stage B Baseline | Stage B: Placebo PH20 SC | 140.0 | 95% Confidence Interval 75.0 to NA | Days | |
| Stage A: Time to Initial Confirmed Evidence of Clinical Improvement (ECI) | Stage A: Efgartigimod PH20 SC | 25th percentile | 22 | 95% Confidence Interval 22.0 to 23.0 | days |
| Stage A: Time to Initial Confirmed Evidence of Clinical Improvement (ECI) | Stage A: Efgartigimod PH20 SC | 50th percentile (median) | 43.0 | 95% Confidence Interval 31.0 to 51.0 | days |
| Stage A: Time to Initial Confirmed Evidence of Clinical Improvement (ECI) | Stage A: Efgartigimod PH20 SC | 75th percentile | 71.0 | 95% Confidence Interval 70.0 to 78.0 | days |
| Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Adjusted INCAT Score | Stage A: Efgartigimod PH20 SC | -0.9 | Standard Deviation 1.71 | score on a scale | |
| Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Medical Research Council (MRC) Sum Score | Stage A: Efgartigimod PH20 SC | 3.8 | Standard Error 0.41 | score on a scale | |
| Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in I-RODS Disability Scores | Stage A: Efgartigimod PH20 SC | 7.7 | Standard Deviation 15.48 | score on a scale | |
| Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in TUG Score | Stage A: Efgartigimod PH20 SC | -4.3 | Standard Error 0.83 | seconds |
1 to 10 of 125Source ClinicalTrials.gov
See all 125 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Stage A | STARTED | Stage A: Efgartigimod PH20 SC | 322 | |
| Stage A | STARTED | Stage B: Efgartigimod PH20 SC | 0 | |
| Stage A | STARTED | Stage B: Placebo PH20 SC | 0 | |
| Stage A | COMPLETED | Stage A: Efgartigimod PH20 SC | 221 | |
| Stage A | COMPLETED | Stage B: Efgartigimod PH20 SC | 0 | |
| Stage A | COMPLETED | Stage B: Placebo PH20 SC | 0 | |
| Stage A | NOT COMPLETED | Stage A: Efgartigimod PH20 SC | 101 | |
| Stage A | NOT COMPLETED | Stage B: Efgartigimod PH20 SC | 0 | |
| Stage A | NOT COMPLETED | Stage B: Placebo PH20 SC | 0 | |
| Stage B | STARTED | Stage A: Efgartigimod PH20 SC | 0 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events78
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 | INJECTION SITE ERYTHEMA | General disorders | Stage A: Efgartigimod PH20 SC | 33 | 322 | |
| other | INJECTION SITE ERYTHEMA | General disorders | Stage B: Efgartigimod PH20 SC | 6 | 111 | |
| other | INJECTION SITE ERYTHEMA | General disorders | Stage B: Placebo PH20 SC | 0 | 110 | |
| other | HEADACHE | Nervous system disorders | Stage A: Efgartigimod PH20 SC | 16 | 322 | |
| other | HEADACHE | Nervous system disorders | Stage B: Efgartigimod PH20 SC | 4 | 111 | |
| other | HEADACHE | Nervous system disorders | Stage B: Placebo PH20 SC | 2 | 110 | |
| other | INJECTION SITE BRUISING | General disorders | Stage A: Efgartigimod PH20 SC | 4 | 322 | |
| other | INJECTION SITE BRUISING | General disorders | Stage B: Efgartigimod PH20 SC | 6 | 111 | |
| other | INJECTION SITE BRUISING | General disorders | Stage B: Placebo PH20 SC | 1 | 110 | |
| other | COVID-19 | Infections and infestations | Stage A: Efgartigimod PH20 SC | 6 | 322 |
1 to 10 of 78Source ClinicalTrials.gov
See all 78 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Stage A: Efgartigimod PH20 SC | 2 | 322 |
| Total, all-cause mortality | Stage B: Efgartigimod PH20 SC | 0 | 111 |
| Total, all-cause mortality | Stage B: Placebo PH20 SC | 1 | 110 |
| Total, other adverse events | Stage A: Efgartigimod PH20 SC | 60 | 322 |
| Total, other adverse events | Stage B: Efgartigimod PH20 SC | 31 | 111 |
| Total, other adverse events | Stage B: Placebo PH20 SC | 15 | 110 |
| Total, serious adverse events | Stage A: Efgartigimod PH20 SC | 21 | 322 |
| Total, serious adverse events | Stage B: Efgartigimod PH20 SC | 6 | 111 |
| Total, serious adverse events | Stage B: Placebo PH20 SC | 6 | 110 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Stage B: Efgartigimod PH20 SC vs Stage B: Placebo PH20 SC: Stage B: Number of Participants With Improved Functional Level Compared to Stage B Baseline |
Source 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/NCT04281472
GET https://conductscience.org/api/trials/v1/NCT04281472/registered?page=2