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Focus resources on a planned phase 2/3 study
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This main study objective was to evaluate the safety and tolerability of intravenous (IV) SYNT001 (ALXN1830) in participants with WAIHA.
This study planned to evaluate 2 cohorts: Cohort 1, up to 8 participants to receive IV doses of ALXN1830 (SYNT001 Dose 1); Cohort 2, up to 12 participants to receive IV doses of ALXN1830 (SYNT001 Dose 2).
This study was terminated after the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy were characterized in participants with WAIHA in Cohort 1 (SYNT001 Dose 1), before any participants were enrolled in Cohort 2.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1: ALXN1830 | Experimental | SYNT001 Dose 1 |
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| Cohort 2: ALXN1830 | Experimental | SYNT001 Dose 2 |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ALXN1830 | Drug | Administered via IV infusion. |
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| Measure | Description | Time Frame |
|---|---|---|
| Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs) | 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. | Day 0 (after first dose) through Day 112 |
| Measure | Description | Time Frame |
|---|---|---|
| Maximum Serum Concentration (Cmax) On Day 0 And Day 28 | 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). |
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Inclusion Criteria:
Participants had to meet the following criteria to be included:
Exclusion Criteria:
Participants who met any of the following criteria were excluded:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Alexion Study Site | Los Angeles | California | 90033 | United States | ||
| Alexion Study Site |
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This study was terminated after the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy were characterized in Cohort 1 (SYNT001 Dose 1), before any participants were enrolled in Cohort 2 (SYNT001 Dose 2). This results disclosure is for Cohort 1 only.
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| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1: ALXN1830 | SYNT001 Dose 1: Participants received ALXN1830. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 9, 2019 | Apr 9, 2020 |
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| Predose, 5 minutes, 2, 4, 6, 24, and 48 hours, and 5 days postdose |
| Change From Baseline In Reticulocyte Count At 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. | Baseline, Day 33 |
| Change From Baseline In Hemoglobin At 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. | Baseline, Day 33 |
| Immunogenicity Of ALXN1830 At Day 112, As Assessed By Anti-ALXN1830 Antibody Level | 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. | Day 112 |
| San Francisco |
| California |
| 94143 |
| United States |
| Alexion Study Site | Boston | Massachusetts | 02114 | United States |
| Alexion Study Site | Pittsfield | Massachusetts | 01201 | United States |
| Alexion Study Site | Rochester | Minnesota | 55905 | United States |
| Alexion Study Site | Cleveland | Ohio | 44106 | United States |
| Alexion Study Site | Philadelphia | Pennsylvania | 19104 | United States |
| Alexion Study Site | Pittsburgh | Pennsylvania | 15232 | United States |
| Alexion Study Site | Seattle | Washington | 98195 | United States |
| Alexion Study Site | Amman | 11941 | Jordan |
| Received at Least 1 Dose of Study Drug | Safety Population |
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| COMPLETED |
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| NOT COMPLETED |
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Safety Population: All participants who received at least 1 dose of study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | Cohort 1: ALXN1830 | SYNT001 Dose 1: Participants received ALXN1830. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Age at Warm Autoimmune Hemolytic Anemia Diagnosis | Mean | Standard Deviation | years |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs) | 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. | Safety Population: All participants who received at least 1 dose of study drug. | Posted | Count of Participants | Participants | Day 0 (after first dose) through Day 112 |
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| Secondary | Maximum Serum Concentration (Cmax) On Day 0 And Day 28 | 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). | PK Population: All participants who received at least 1 dose of study drug and had postdose PK data available at the specified timepoint. | Posted | Mean | Standard Deviation | ug/mL | Predose, 5 minutes, 2, 4, 6, 24, and 48 hours, and 5 days postdose |
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| Secondary | Change From Baseline In Reticulocyte Count At 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. | PD Population: All participants who received at least 1 dose of study drug and had postdose PD data available at the specified timepoint. | Posted | Mean | Standard Deviation | cells*10^12/L | Baseline, Day 33 |
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| Secondary | Change From Baseline In Hemoglobin At 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. | PD Population: All participants who received at least 1dose of study drug and had postdose PD data available at the specified timepoint. | Posted | Mean | Standard Deviation | g/dL | Baseline, Day 33 |
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| Secondary | Immunogenicity Of ALXN1830 At Day 112, As Assessed By Anti-ALXN1830 Antibody Level | 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. | Safety Population: All participants who received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | reciprocal of the titer | Day 112 |
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Baseline through Day 112
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cohort 1: ALXN1830 | SYNT001 Dose 1: Participants received ALXN1830. | 0 | 8 | 2 | 8 | 8 | 8 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 19.1 | Systematic Assessment |
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| Gastritis | Gastrointestinal disorders | MedDRA 19.1 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Fatigue | General disorders | MedDRA 19.1 | Systematic Assessment |
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| Chills | General disorders | MedDRA 19.1 | Systematic Assessment |
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| Face oedema | General disorders | MedDRA 19.1 | Systematic Assessment |
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| Influenza like illness | General disorders | MedDRA 19.1 | Systematic Assessment |
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| Oedema peripheral | General disorders | MedDRA 19.1 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 19.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 19.1 | Systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA 19.1 | Systematic Assessment |
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| Dizziness postural | Nervous system disorders | MedDRA 19.1 | Systematic Assessment |
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| Hypersomnia | Nervous system disorders | MedDRA 19.1 | Systematic Assessment |
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| Syncope | Nervous system disorders | MedDRA 19.1 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 19.1 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 19.1 | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA 19.1 | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA 19.1 | Systematic Assessment |
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| Rhinitis | Infections and infestations | MedDRA 19.1 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 19.1 | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA 19.1 | Systematic Assessment |
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| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 19.1 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 19.1 | Systematic Assessment |
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| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | MedDRA 19.1 | Systematic Assessment |
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| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 19.1 | Systematic Assessment |
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| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 19.1 | Systematic Assessment |
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| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 19.1 | Systematic Assessment |
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| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA 19.1 | Systematic Assessment |
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| Anaemia | Blood and lymphatic system disorders | MedDRA 19.1 | Systematic Assessment |
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| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 19.1 | Systematic Assessment |
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| Sinus tachycardia | Cardiac disorders | MedDRA 19.1 | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | MedDRA 19.1 | Systematic Assessment |
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| Infusion-related reaction | Injury, poisoning and procedural complications | MedDRA 19.1 | Systematic Assessment |
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| Limb injury | Injury, poisoning and procedural complications | MedDRA 19.1 | Systematic Assessment |
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| Post-traumatic pain | Injury, poisoning and procedural complications | MedDRA 19.1 | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 19.1 | Systematic Assessment |
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| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 19.1 | Systematic Assessment |
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| Blepharospasm | Eye disorders | MedDRA 19.1 | Systematic Assessment |
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| Haematocrit decreased | Investigations | MedDRA 19.1 | Systematic Assessment |
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| Haemoglobin decreased | Investigations | MedDRA 19.1 | Systematic Assessment |
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| Increased appetite | Metabolism and nutrition disorders | MedDRA 19.1 | Systematic Assessment |
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| Restlessness | Psychiatric disorders | MedDRA 19.1 | Systematic Assessment |
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| Flushing | Vascular disorders | MedDRA 19.1 | Systematic Assessment |
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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).
Sponsor can review results communications at least 60 days prior to public release. Within 30 days of receipt, Sponsor shall advise of any information which is Confidential Information (other than Study Data) or which may impair the availability of patent protection for Inventions. Sponsor can require removal of specifically identified Confidential Information (other than Study Data) and/or delay the communication an additional 60 days to enable Sponsor to seek patent protection for Inventions.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals, Inc. | Alexion Pharmaceuticals, Inc. | 855-752-2356 | clinicaltrials@alexion.com |
| SAP_000.pdf |
| Prot | Yes | No | No | Study Protocol | Oct 23, 2018 | Apr 9, 2020 | Prot_001.pdf |
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| ID | Term |
|---|---|
| C000708950 | orilanolimab |
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| Unknown or Not Reported |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Title | Measurements |
|---|---|
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| Deaths |
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