Trial · NCT03075878
A Safety Study of SYNT001 in Participants With Warm Autoimmune Hemolytic Anemia (WAIHA)
NCT03075878, Phase 1, Phase 2, Terminated, Alexion Pharmaceuticals, Inc.; results first posted 2020-05-13
3 sourcestrials-2026-11-12-0.4.0
Cite this record
ConductScience Library. A Safety Study of SYNT001 in Participants With Warm Autoimmune Hemolytic Anemia (WAIHA). Trials, record NCT03075878. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03075878
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Safety Study of SYNT001 in Participants With Warm Autoimmune Hemolytic Anemia (WAIHA)
- NCT number
- NCT03075878
- Status
- Terminated
- Phases
- Phase 1, Phase 2
- Enrollment
- 8 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Warm Autoimmune Hemolytic Anemia
- Interventions
- ALXN1830
- First posted
- 2017-03-09
- Last updated
- 2020-05-13
- Results first posted
- 2020-05-13
- Limitations and caveats
- The study was terminated after the safety, tolerability, PK, PD, and efficacy were characterized in the Warm Autoimmune Hemolytic Anemia participants in Cohort 1 (SYNT001 Dose 1).
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 0 (after first dose) through Day 112 | A TEAE was defined as any adverse event 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" 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. All serious TEAEs were not considered related to the study drug. |
| Secondary | Maximum Serum Concentration (Cmax) On Day 0 And Day 28 | Predose, 5 minutes, 2, 4, 6, 24, and 48 hours, and 5 days postdose | The Cmax was determined directly from the concentration-time profile. Starting on Days 0 and 28, serum samples were collected just prior to the start of study drug infusion (predose), at 5 minutes, at 2, 4, 6, 24, and 48 hours, and at 5 days postdose after the end-of-study drug infusion. Results are reported in micrograms/milliliter (ug/mL). |
| Secondary | Change From Baseline In Reticulocyte Count At Day 33 | Baseline, Day 33 | Reticulocyte count was measured as a PD biomarker. Pharmacodynamic samples were collected for analyses throughout the study prior to infusion of study drug for Cohort 1. Measurements for PD biomarkers were derived from the laboratory results. Results are reported in cells/liter (cells\*10\^12/L). A decrease in reticulocyte count indicated a potential improvement in condition. |
| Secondary | Change From Baseline In Hemoglobin At Day 33 | Baseline, Day 33 | Hemoglobin was measured as a PD biomarker. Pharmacodynamic samples were collected for analyses throughout the study prior to infusion of study drug for Cohort 1. Measurements for PD biomarkers were derived from the laboratory results. Results are reported in grams/deciliter (g/dL). An increase in hemoglobin indicated a potential improvement in condition. |
| Secondary | Immunogenicity Of ALXN1830 At Day 112, As Assessed By Anti-ALXN1830 Antibody Level | Day 112 | Immunogenicity analyses are reported for Day 112. Testing was carried out to detect binding antidrug antibodies by anti-ALXN1830 antibody level. Results are reported as the reciprocal of the titer where they cross the study cut point. |
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 0 (after first dose) through Day 112 | Safety Population: All participants who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Maximum Serum Concentration (Cmax) On Day 0 And Day 28 | Predose, 5 minutes, 2, 4, 6, 24, and 48 hours, and 5 days postdose | PK Population: All participants who received at least 1 dose of study drug and had postdose PK data available at the specified timepoint. | ug/mL | MEAN |
| SECONDARY | Change From Baseline In Reticulocyte Count At Day 33 | Baseline, Day 33 | PD Population: All participants who received at least 1 dose of study drug and had postdose PD data available at the specified timepoint. | cells*10^12/L | MEAN |
| SECONDARY | Change From Baseline In Hemoglobin At Day 33 | Baseline, Day 33 | PD Population: All participants who received at least 1dose of study drug and had postdose PD data available at the specified timepoint. | g/dL | MEAN |
| SECONDARY | Immunogenicity Of ALXN1830 At Day 112, As Assessed By Anti-ALXN1830 Antibody Level | Day 112 | Safety Population: All participants who received at least 1 dose of study drug. | reciprocal of the titer | MEAN |
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 | 8 | Participants | |
| Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs) | Cohort 1: ALXN1830 | Serious TEAEs | 2 | 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 Serum Concentration (Cmax) On Day 0 And Day 28 | Cohort 1: ALXN1830 | Day 0 | 249.6 | Standard Deviation 35.76 | ug/mL |
| Maximum Serum Concentration (Cmax) On Day 0 And Day 28 | Cohort 1: ALXN1830 | Day 28 | 245.8 | Standard Deviation 41.55 | ug/mL |
| Change From Baseline In Reticulocyte Count At Day 33 | Cohort 1: ALXN1830 | -0.044 | Standard Deviation 0.0948 | cells*10^12/L | |
| Change From Baseline In Hemoglobin At Day 33 | Cohort 1: ALXN1830 | 0.66 | Standard Deviation 1.581 | g/dL | |
| Immunogenicity Of ALXN1830 At Day 112, As Assessed By Anti-ALXN1830 Antibody Level | Cohort 1: ALXN1830 | 99.220 | Standard Deviation 165.8149 | reciprocal of the titer |
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 | 7 | |
| Overall Study | NOT COMPLETED | Cohort 1: ALXN1830 | 1 |
Source ClinicalTrials.gov
Adverse events42
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 | Fatigue | General disorders | Cohort 1: ALXN1830 | 2 | 8 | |
| other | Chills | General disorders | Cohort 1: ALXN1830 | 1 | 8 | |
| other | Face oedema | General disorders | Cohort 1: ALXN1830 | 1 | 8 | |
| other | Influenza like illness | General disorders | Cohort 1: ALXN1830 | 1 | 8 | |
| other | Oedema peripheral | General disorders | Cohort 1: ALXN1830 | 1 | 8 | |
| other | Pyrexia | General disorders | Cohort 1: ALXN1830 | 1 | 8 | |
| other | Headache | Nervous system disorders | Cohort 1: ALXN1830 | 4 | 8 | |
| other | Dizziness | Nervous system disorders | Cohort 1: ALXN1830 | 2 | 8 | |
| other | Dizziness postural | Nervous system disorders | Cohort 1: ALXN1830 | 1 | 8 | |
| other | Hypersomnia | Nervous system disorders | Cohort 1: ALXN1830 | 1 | 8 |
1 to 10 of 42Source ClinicalTrials.gov
See all 42 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 | 8 | 8 |
| Total, serious adverse events | Cohort 1: ALXN1830 | 2 | 8 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT03075878
GET https://conductscience.org/api/trials/v1/NCT03075878/events?page=2