- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03075878
A Safety Study of SYNT001 in Participants With Warm Autoimmune Hemolytic Anemia (WAIHA)
A Phase 1B/2, Multicenter, Open-Label, Safety, Tolerability, and Activity Study of SYNT001 in Patients With Warm Autoimmune Hemolytic Anemia (WAIHA)
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
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.
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Locations
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Amman, Jordan, 11941
- Alexion Study Site
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California
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Los Angeles, California, United States, 90033
- Alexion Study Site
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San Francisco, California, United States, 94143
- Alexion Study Site
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Massachusetts
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Boston, Massachusetts, United States, 02114
- Alexion Study Site
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Pittsfield, Massachusetts, United States, 01201
- Alexion Study Site
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Minnesota
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Rochester, Minnesota, United States, 55905
- Alexion Study Site
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Ohio
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Cleveland, Ohio, United States, 44106
- Alexion Study Site
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- Alexion Study Site
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Pittsburgh, Pennsylvania, United States, 15232
- Alexion Study Site
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Washington
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Seattle, Washington, United States, 98195
- Alexion Study Site
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
Participants had to meet the following criteria to be included:
- Willing and able to read, understand, and sign an informed consent form
- Confirmed diagnosis of WAIHA by enrolling physician
- Must have used medically acceptable contraception
Exclusion Criteria:
Participants who met any of the following criteria were excluded:
- Participant unable or unwilling to comply with the protocol
- Active non-hematologic malignancy or history of non-hematologic malignancy in the 3 years prior to screening (exclusive of non-melanoma skin cancer and cervical cancer in situ)
- Positive for human immunodeficiency virus or hepatitis C antibody
- Positive for hepatitis B surface antigen
- Any exposure to an investigational drug or device within the 30 days prior to screening
- Intravenous immunoglobulin treatment within 30 days of screening
- Plasmapheresis or immunoadsorption within 30 days of screening
- Participant had any current medical condition that, in the opinion of the Investigator, may have compromised their safety or compliance, precluded successful conduct of the study, or interfered with interpretation of the results
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NON_RANDOMIZED
- Interventional Model: SEQUENTIAL
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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EXPERIMENTAL: Cohort 1: ALXN1830
SYNT001 Dose 1
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Administered via IV infusion.
Other Names:
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EXPERIMENTAL: Cohort 2: ALXN1830
SYNT001 Dose 2
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Administered via IV infusion.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)
Time Frame: Day 0 (after first dose) through Day 112
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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.
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Day 0 (after first dose) through Day 112
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Maximum Serum Concentration (Cmax) On Day 0 And Day 28
Time Frame: Predose, 5 minutes, 2, 4, 6, 24, and 48 hours, and 5 days postdose
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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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Predose, 5 minutes, 2, 4, 6, 24, and 48 hours, and 5 days postdose
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Change From Baseline In Reticulocyte Count At Day 33
Time Frame: Baseline, Day 33
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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.
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Baseline, Day 33
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Change From Baseline In Hemoglobin At Day 33
Time Frame: Baseline, Day 33
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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.
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Baseline, Day 33
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Immunogenicity Of ALXN1830 At Day 112, As Assessed By Anti-ALXN1830 Antibody Level
Time Frame: Day 112
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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.
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Day 112
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Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (ACTUAL)
Primary Completion (ACTUAL)
Study Completion (ACTUAL)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ACTUAL)
Study Record Updates
Last Update Posted (ACTUAL)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- SYNT001-102
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Warm Autoimmune Hemolytic Anemia
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SanofiActive, not recruitingWarm Autoimmune Hemolytic Anemia (wAIHA)United States, Austria, China, Denmark, Germany, Hungary, Italy, Spain, United Kingdom
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SanofiTerminatedWarm Autoimmune Hemolytic Anemia (wAIHA)United Kingdom, Belgium, Netherlands, France, United States, Germany, Hungary, Italy
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Novartis PharmaceuticalsRecruitingWarm Autoimmune Hemolytic Anemia (wAIHA)China, Japan, Spain, Singapore, France, Germany, Taiwan, United States, Italy, India, Malaysia, Argentina, Hungary, Israel, Australia, Thailand, United Kingdom, Romania
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Annexon, Inc.CompletedWarm Autoimmune Hemolytic Anemia (wAIHA)United States
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Eugene NikitinUnknownAIHA - Warm Autoimmune Hemolytic AnemiaRussian Federation
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Incyte CorporationActive, not recruitingWarm Autoimmune Hemolytic Anemia (wAIHA)Spain, United States, Austria, Belgium, Canada, France, Germany, Israel, Italy, Japan, Netherlands, Poland, United Kingdom
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Peking Union Medical College HospitalRecruiting
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Alexion PharmaceuticalsWithdrawnWarm Autoimmune Hemolytic AnemiaUnited States
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Chen MiaoNot yet recruiting
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Immunovant Sciences GmbHTerminatedWarm Autoimmune Hemolytic AnemiaUnited States, Korea, Republic of, Israel, United Kingdom, Spain, Thailand
Clinical Trials on ALXN1830
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Alexion Pharmaceuticals, Inc.TerminatedHealthyUnited Kingdom
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Alexion PharmaceuticalsWithdrawnGeneralized Myasthenia GravisUnited States
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Alexion PharmaceuticalsTerminatedPemphigus | Pemphigus Vulgaris | Pemphigus FoliaceusUnited States
-
AlexionWithdrawnWarm Autoimmune Hemolytic AnemiaUnited States
-
Alexion PharmaceuticalsSyneos HealthTerminated
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Alexion PharmaceuticalsWithdrawnWarm Autoimmune Hemolytic AnemiaUnited States