Subcutaneous ALXN1830 in Adult Participants With Warm Autoimmune Hemolytic Anemia

Alexion Pharmaceuticals, Inc.

Overview

This is a Phase 2, multiple ascending, dose-finding, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, health-related quality of life, tolerability, pharmacokinetic, pharmacodynamic, and immunogenicity, of up to 3 dose regimens of ALXN1830 administered subcutaneous(ly) (SC) in the treatment of WAIHA. This study will include 2 randomized, double-blind, placebo-controlled cohorts (Cohorts 1 and 2) to evaluate an 8-week treatment regimen, and an optional third open-label cohort (Cohort 3) to evaluate an alternative 12-week dosing regimen. Participants may continue participation in this study at the participant's and investigator's discretion in an open-label extension (OLE) period, consisting of monthly visits to observe participants for relapse, which will require going back on active treatment.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Conditions

Warm Autoimmune Hemolytic Anemia

Interventions

ALXN1830DRUG

Administered as an SC infusion.

PlaceboDRUG

Administered as an SC infusion.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Key Inclusion Criteria: * Diagnosed with primary or secondary WAIHA at least 6 weeks prior to Screening. * Failed or have not tolerated at least one prior WAIHA treatment regimen, for example, corticosteroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, mycophenolate mofetil, danazol, or vincristine. * Hemoglobin \< 10 g/dL and ≥ 6 g/dL at Screening. * Positive direct antiglobulin test (Coombs) (IgG positive who are positive or negative for the presence of complement C3) at Screening. * Evidence of active hemolysis including any one of the below: * LDH \> upper limit of normal (ULN) or * Haptoglobin \< lower limit of normal or * Indirect bilirubin \> ULN * Total IgG \> 500 mg/dL at Screening * Platelet count ≥ 75 x 10\^9/liter (L) * Absolute neutrophil count greater than 1.0 x 10\^9/L Key Exclusion Criteria: * Participants with Evan's syndrome. * Human immunodeficiency virus (HIV) infection (positive HIV 1 or HIV 2 antibody test). * Positive hepatitis B surface antigen or hepatitis C antibody test. * Inability to travel to the clinic for specified visits during the Primary Treatment Period or fulfill the logistical requirements of study intervention administration.

Locations (1)

Clinical Study Site

Riverside, California, United States

Outcomes

Primary Outcomes

Proportion Of Participants Achieving A ≥ 2 Grams/Deciliter (g/dL) Increase In Hemoglobin (Hgb) From Baseline To The End Of Primary Treatment

Participants will have to achieve this increase without requiring any increase in the dose of an existing WAIHA medication after Day 1 (baseline) and without packed red blood cells (pRBC) transfusions after Day 14.

Time frame: Baseline through Week 12

Secondary Outcomes

Total Number Of Units Of pRBCs Transfused

Time frame: Baseline through Week 12

Number Of Hgb Measurements ≥ 2 g/dL From Baseline To The End Of Primary Treatment

Time frame: Baseline, Week 12

Time To Hgb Increase By ≥ 2 g/dL From Baseline

Time frame: Baseline through Week 12

Proportion Of Participants Who Require New WAIHA Rescue Medication Or Any Increase In The Dose Of An Existing WAIHA Medication Or pRBC Transfusions For The Treatment Of Anemia

Time frame: Day 15 through Week 12

Proportion Of Participants Achieving A ≥ 2 g/dL Increase In Hgb From Baseline Through Week 4

Participants need to achieve this increase without requiring any increase in the dose of an existing WAIHA medication after Day 1 and without pRBC transfusions after Day 14.

Time frame: Baseline through Week 4

Change From Baseline To The End Of Primary Treatment In Serum Lactate Dehydrogenase (LDH) Levels

Time frame: Baseline, Week 12

Change From Baseline To The End Of Primary Treatment In Absolute Reticulocyte Count

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.