ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia

Study Overview
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Brief Summary
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The main objective of the study is to evaluate the safety and efficacy of ALXN1830 compared to placebo in adult participants with warm autoimmune hemolytic anemia (WAIHA).

Detailed Description
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This study will consist of a 4-week Screening period, 13-week Primary Treatment period, and optional Extended Treatment period (up to 2 years).

Official Title
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A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Study of ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia

Conditions
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Warm Autoimmune Hemolytic Anemia

Intervention / Treatment
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* Biological: ALXN1830
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Primary or secondary WAIHA, diagnosed at least 6 weeks prior to Screening Failed or not tolerated at least 1 prior WAIHA treatment regimen, for example, corticosteroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, mycophenolate mofetil, danazol, or vincristine Hemoglobin < 10 g/dL and ≥ 6 g/dL Positive direct antiglobulin test (Coombs) (IgG positive, complement C3 [C3] positive or negative) Evidence of active hemolysis including any of the following: a) Lactate dehydrogenase (LDH) > upper limit of normal (ULN), b) Haptoglobin < lower limit of normal (LLN), c) Indirect bilirubin > ULN Total IgG > 500 mg/dL Key Exclusion Criteria: Human immunodeficiency virus (HIV) infection (positive HIV-1 or HIV-2 antibody test) Positive hepatitis B surface antigen (HBsAg) or hepatitis C antibody test

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Participants randomized in 1:1:1:1 ratio to 1 of 4 study arms.
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ALXN1830 Dosing Regimen 1<br> | Biological: ALXN1830<br>* Administered via intravenous (IV) infusion<br>* Other names: SYNT001;|
| Experimental: ALXN1830 Dosing Regimen 2<br> | Biological: ALXN1830<br>* Administered via intravenous (IV) infusion<br>* Other names: SYNT001;|
| Experimental: ALXN1830 Dosing Regimen 3<br> | Biological: ALXN1830<br>* Administered via intravenous (IV) infusion<br>* Other names: SYNT001;|
| Active Comparator: Placebo<br> | Other: Placebo<br>* Matching placebo (sterile liquid diluent) administered via IV infusion<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage Of Participants With ≥ 2 Grams (g)/Deciliter (dL) Increase In Hemoglobin (Hgb) From Baseline |  | Baseline (Day 1) through Day 92 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Total Number Of Units Of Packed Red Blood Cells (pRBCs) Transfused |  | Day 15 through Day 92 | 
| Mean Change From Baseline To Day 92 In Quality Of Life, Assessed Via EuroQoL 5 Dimension 5 Level (EQ-5D-5L) Questionnaire |  | Baseline, Day 92 | 
| Mean Change From Baseline To Day 92 In Quality Of Life, Assessed Via Functional Assessment Of Cancer Therapy Anemia (FACT-AN) Questionnaire |  | Baseline, Day 92 | 
| Number Of Weekly Hgb Measurements With Change From Baseline ≥ 2 g/dL |  | Day 1 through Day 92 | 
| Number Of Participants Needing New WAIHA Rescue Medication Or Increase In Dose Of WAIHA Medication |  | Day 15 through Day 92 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Warm autoimmune hemolytic anemia, WAIHA, Immunoglobulin G (IgG)-mediated autoimmune disorder, Pathogenesis