This main study objective was to evaluate the safety and tolerability of intravenous (IV) SYNT001 (ALXN1830) in participants with WAIHA.
Full Title of Study: “A Phase 1B/2, Multicenter, Open-Label, Safety, Tolerability, and Activity Study of SYNT001 in Patients With Warm Autoimmune Hemolytic Anemia (WAIHA)”
- Study Type: Interventional
- Study Design
- Allocation: Non-Randomized
- Intervention Model: Sequential Assignment
- Primary Purpose: Treatment
- Masking: None (Open Label)
- Study Primary Completion Date: April 15, 2019
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.
- Drug: ALXN1830
- Administered via IV infusion.
Arms, Groups and Cohorts
- Experimental: Cohort 1: ALXN1830
- SYNT001 Dose 1
- Experimental: Cohort 2: ALXN1830
- SYNT001 Dose 2
Clinical Trial Outcome Measures
- Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)
- Time Frame: 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.
- 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
- 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).
- Change From Baseline In Reticulocyte Count At Day 33
- Time Frame: 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.
- Change From Baseline In Hemoglobin At Day 33
- Time Frame: 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.
- Immunogenicity Of ALXN1830 At Day 112, As Assessed By Anti-ALXN1830 Antibody Level
- Time Frame: 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.
Participating in This Clinical Trial
Participants had to meet the following criteria to be included:
Participants who met any of the following criteria were excluded:
Gender Eligibility: All
Minimum Age: 18 Years
Maximum Age: N/A
Are Healthy Volunteers Accepted: No
- Lead Sponsor
- Alexion Pharmaceuticals
- Provider of Information About this Clinical Study
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