Trial · NCT03075904
A Safety and Dose-Finding Study of SYNT001 in Subjects With Pemphigus (Vulgaris or Foliaceus)
NCT03075904, Phase 1, Phase 2, Terminated, Alexion Pharmaceuticals, Inc.; results first posted 2020-02-05
3 sourcestrials-2026-11-12-0.4.0
Cite this record
ConductScience Library. A Safety and Dose-Finding Study of SYNT001 in Subjects With Pemphigus (Vulgaris or Foliaceus). Trials, record NCT03075904. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03075904
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Safety and Dose-Finding Study of SYNT001 in Subjects With Pemphigus (Vulgaris or Foliaceus)
- NCT number
- NCT03075904
- Status
- Terminated
- Phases
- Phase 1, Phase 2
- Enrollment
- 8 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Pemphigus, Pemphigus Vulgaris, Pemphigus Foliaceus
- Interventions
- ALXN1830
- First posted
- 2017-03-09
- Last updated
- 2020-02-05
- Results first posted
- 2020-02-05
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs) | Day 1 (after first dose) through Day 112 | A TEAE was defined as any adverse event (AE) that starts on or after the first dose of study drug or occurs prior to the first dose and worsens in severity on or after the first dose of study drug, during the Treatment Period and Follow-up Period. A TEAE was considered "serious" (Grade 3) if, in the view of either the investigator or sponsor, it resulted in any of the following outcomes: death, life-threatening adverse drug event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, or an event that may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously listed outcomes. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module. |
| Secondary | Maximum Percent Reduction Of Mean Total Immunoglobulin G (IgG) Levels From Baseline | Baseline through Day 112 | Pharmacodynamic samples were collected for analysis throughout the study. The maximum percent reduction of mean serum total IgG levels from Baseline observed during the study is presented. |
| Secondary | Maximum Percent Reduction In Mean Pemphigus Disease Area Index (PDAI) Total Activity Score From Baseline | Baseline through Day 112 | Pemphigus severity and disease activity was measured using the PDAI in regions where a validated questionnaire was available. The PDAI was administered during Treatment Period and Follow-up Period. PDAI total activity was comprised of scores for the skin, mucous membrane, and scalp subscales. The investigator determined a PDAI score as 0 to 250 points for total activity score (0 to 120 for skin, 0 to 10 for scalp, and 0 to 120 for mucosa). A higher score indicated higher impact on skin disease. The maximum percent reduction in PDAI total activity score from Baseline observed during the study is presented. |
| Secondary | Maximum Percent Reduction Of Mean Circulating Immune Complexes (CIC) Levels From Baseline | Baseline through Day 112 | Pharmacodynamic samples were collected for analysis throughout the study. The maximum percent reduction of mean CIC levels from Baseline observed during the study is presented. |
| Secondary | Maximum Percent Reduction Of Mean Anti-Desmoglein (Dsg) 1 And 3 Antibodies From Baseline | Baseline through Day 112 | Pharmacodynamic samples were collected for analysis throughout the study. The maximum percent reduction of mean anti-Dsg 1 and 3 antibodies from Baseline observed during the study is presented. |
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 | Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs) | Day 1 (after first dose) through Day 112 | All participants who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Maximum Percent Reduction Of Mean Total Immunoglobulin G (IgG) Levels From Baseline | Baseline through Day 112 | All participants who received at least 1 dose of study drug and had postdose pharmacodynamic data available. | percentage of reduction | NUMBER |
| SECONDARY | Maximum Percent Reduction In Mean Pemphigus Disease Area Index (PDAI) Total Activity Score From Baseline | Baseline through Day 112 | All participants who received at least 1 dose of study drug and had postdose pharmacodynamic data available. | percentage of reduction | NUMBER |
| SECONDARY | Maximum Percent Reduction Of Mean Circulating Immune Complexes (CIC) Levels From Baseline | Baseline through Day 112 | All participants who received at least 1 dose of study drug and had postdose pharmacodynamic data available. | percentage of reduction | NUMBER |
| SECONDARY | Maximum Percent Reduction Of Mean Anti-Desmoglein (Dsg) 1 And 3 Antibodies From Baseline | Baseline through Day 112 | All participants who received at least 1 dose of study drug and had postdose pharmacodynamic data available. | percentage of reduction | NUMBER |
Source ClinicalTrials.gov
Results by group9
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs) | Cohort 1: ALXN1830 | TEAEs | 7 | Participants | |
| Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs) | Cohort 1: ALXN1830 | Serious TEAEs | 1 | Participants | |
| Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs) | Cohort 1: ALXN1830 | Discontinuations due to TEAEs | 0 | Participants | |
| Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs) | Cohort 1: ALXN1830 | Deaths | 0 | Participants | |
| Maximum Percent Reduction Of Mean Total Immunoglobulin G (IgG) Levels From Baseline | Cohort 1: ALXN1830 | 57.3 | percentage of reduction | ||
| Maximum Percent Reduction In Mean Pemphigus Disease Area Index (PDAI) Total Activity Score From Baseline | Cohort 1: ALXN1830 | 45.7 | percentage of reduction | ||
| Maximum Percent Reduction Of Mean Circulating Immune Complexes (CIC) Levels From Baseline | Cohort 1: ALXN1830 | 51.4 | percentage of reduction | ||
| Maximum Percent Reduction Of Mean Anti-Desmoglein (Dsg) 1 And 3 Antibodies From Baseline | Cohort 1: ALXN1830 | anti-Dsg 1 | 8.9 | percentage of reduction | |
| Maximum Percent Reduction Of Mean Anti-Desmoglein (Dsg) 1 And 3 Antibodies From Baseline | Cohort 1: ALXN1830 | anti-Dsg 3 | 20.4 | percentage of reduction |
Source ClinicalTrials.gov
Participant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1: ALXN1830 | 8 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Cohort 1: ALXN1830 | 8 | |
| Overall Study | COMPLETED | Cohort 1: ALXN1830 | 4 | |
| Overall Study | NOT COMPLETED | Cohort 1: ALXN1830 | 4 |
Source ClinicalTrials.gov
Adverse events21
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 | Dry eye | Eye disorders | Cohort 1: ALXN1830 | 1 | 8 | |
| other | Diarrhoea | Gastrointestinal disorders | Cohort 1: ALXN1830 | 1 | 8 | |
| other | Nausea | Gastrointestinal disorders | Cohort 1: ALXN1830 | 2 | 8 | |
| other | Vomiting | Gastrointestinal disorders | Cohort 1: ALXN1830 | 1 | 8 | |
| other | Fatigue | General disorders | Cohort 1: ALXN1830 | 2 | 8 | |
| other | Malaise | General disorders | Cohort 1: ALXN1830 | 1 | 8 | |
| other | Pyrexia | General disorders | Cohort 1: ALXN1830 | 1 | 8 | |
| other | Hepatic cyst | Hepatobiliary disorders | Cohort 1: ALXN1830 | 1 | 8 | |
| other | Herpes simplex | Infections and infestations | Cohort 1: ALXN1830 | 1 | 8 | |
| other | Staphylococcal infection | Infections and infestations | Cohort 1: ALXN1830 | 1 | 8 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1: ALXN1830 | 0 | 8 |
| Total, other adverse events | Cohort 1: ALXN1830 | 7 | 8 |
| Total, serious adverse events | Cohort 1: ALXN1830 | 1 | 8 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 34126109 | 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 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.
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/NCT03075904
GET https://conductscience.org/api/trials/v1/NCT03075904/events?page=2