ALXN1830 in Patients with Warm Autoimmune Hemolytic Anemia

A Phase 2, Multiple Ascending Dose, Randomized, Double-Blind, Placebo-Controlled Study of ALXN1830 Administered Subcutaneously in Patients with Warm Autoimmune Hemolytic Anemia (WAIHA)

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2021-001211-90-ES

Enrollment

Registered

2021-07-02

Start date

2021-08-11

Completion date

Unknown

Last updated

2022-05-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Warm Autoimmune Hemolytic Anemia (WAIHA) MedDRA version: 20.0 Level: LLT Classification code 10003825 Term: Autoimmune hemolytic anemia System Organ Class: 100000004851

Interventions

Product Name: ALXN1830 Pharmaceutical Form: Solution for infusion INN or Proposed INN: ORILANOLIMAB CAS Number: 2066544-85-0 Current Sponsor code: ALXN1830 Other descriptive name: SYNT001 Concentratio

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Participant must be at least 18 years of age inclusive, at the time of signing the informed consent 2. Diagnosed with primary or secondary WAIHA at least 6 weeks prior to Screening 3. Failed or have not tolerated at least one prior WAIHA treatment regimen, for example, corticosteroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, mycophenolate mofetil, danazol, or vincristine 4. Hemoglobin =65 years) yes F.1.3.1 Number of subjects for this age range 15

Exclusion criteria

Exclusion criteria: 1. Packed RBC or whole blood transfusions in the 2 weeks prior to Screening 2. History of cold antibody autoimmune hemolytic anemia (AIHA), cold agglutinin syndrome, mixed type AIHA, or paroxysmal cold hemoglobinuria 3. History of drug-induced or infection-related immune hemolytic anemia 4. Iron, folic acid, or vitamin B12 deficiency 5. Participants with Evan’s syndrome 6. History of leukemia, Hodgkin, or non Hodgkin lymphoma 7. History of solid malignancy other than basal cell or squamous cell carcinoma of the skin, carcinoma in situ of the cervix, or other malignancy for which the participant had not been disease free for at least 5 years 8. Active infection or history of recurrent systemic infections in the 2 years prior to Screening 10. Systemic lupus erythematosus (SLE) or other autoimmune disease that is not stable or not well controlled on current therapy

Design outcomes

Primary

Measure	Time frame
Main Objective: Determine the efficacy of ALXN1830 compared to placebo in the treatment of WAIHA;Secondary Objective: 1. Assess the effect of ALXN1830 on the need for transfusions compared to placebo 2. Assess the effect on ALXN1830 on anemia compared to placebo 3. Evaluate the efficacy of ALXN1830 after 4 weeks compared to placebo 4. Assess the effect on ALXN1830 on hemolysis compared to placebo 5. Assess the effect of ALXN1830 on corticosteroid usage compared to placebo 6. Assess the safety and tolerability of ALXN1830 compared to placebo 7. Assess the immunogenicity of ALXN1830 compared to placebo 8. Assess the pharmacokinetics (PK) of ALXN1830 9. Assess the effect of ALXN1830 on serum total IgG levels compared to placebo 10. Assess the effect of ALXN1830 on other PD biomarkers compared to placebo;Primary end point(s): Proportion of participants achieving a = 2 g/dL increase in Hgb from Baseline (Day 1) to the end of Primary Treatment, without requiring any increase in the dose of an existing WAIHA medication after Day 1 and without pRBC transfusions after Day 14;Timepoint(s) of evaluation of this end point: End of Primary Treatment (Day 57 or Day 85)	—

Measure

Time frame

Main Objective: Determine the efficacy of ALXN1830 compared to placebo in the treatment of WAIHA;Secondary Objective: 1. Assess the effect of ALXN1830 on the need for transfusions compared to placebo 2. Assess the effect on ALXN1830 on anemia compared to placebo 3. Evaluate the efficacy of ALXN1830 after 4 weeks compared to placebo 4. Assess the effect on ALXN1830 on hemolysis compared to placebo 5. Assess the effect of ALXN1830 on corticosteroid usage compared to placebo 6. Assess the safety and tolerability of ALXN1830 compared to placebo 7. Assess the immunogenicity of ALXN1830 compared to placebo 8. Assess the pharmacokinetics (PK) of ALXN1830 9. Assess the effect of ALXN1830 on serum total IgG levels compared to placebo 10. Assess the effect of ALXN1830 on other PD biomarkers compared to placebo;Primary end point(s): Proportion of participants achieving a = 2 g/dL increase in Hgb from Baseline (Day 1) to the end of Primary Treatment, without requiring any increase in the dose of an existing WAIHA medication after Day 1 and without pRBC transfusions after Day 14;Timepoint(s) of evaluation of this end point: End of Primary Treatment (Day 57 or Day 85)

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Secondary

Measure	Time frame
Secondary end point(s): 1. Total number of units of pRBCs transfused: 2. Durability: number of Hgb measurements = 2 g/dL 4. 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 5. Proportion of participants achieving a = 2 g/dL increase in Hgb, without requiring any increase in the dose of an existing WAIHA medication 6. Markers of hemolysis: absolute and percentage change in serum lactate dehydrogenase (LDH) levels, absolute reticulocyte count, serum indirect bilirubin, and serum haptoglobin 7. Total corticosteroid usage from Baseline (Day 1) to the end of Primary Treatment 13. Incidence of AEs, SAEs and AESIs over time 15. Serum concentrations of ALXN1830 over time 16. Serum total IgG levels;Timepoint(s) of evaluation of this end point: Throughout the study	—

Measure

Time frame

Secondary end point(s): 1. Total number of units of pRBCs transfused: 2. Durability: number of Hgb measurements = 2 g/dL 4. 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 5. Proportion of participants achieving a = 2 g/dL increase in Hgb, without requiring any increase in the dose of an existing WAIHA medication 6. Markers of hemolysis: absolute and percentage change in serum lactate dehydrogenase (LDH) levels, absolute reticulocyte count, serum indirect bilirubin, and serum haptoglobin 7. Total corticosteroid usage from Baseline (Day 1) to the end of Primary Treatment 13. Incidence of AEs, SAEs and AESIs over time 15. Serum concentrations of ALXN1830 over time 16. Serum total IgG levels;Timepoint(s) of evaluation of this end point: Throughout the study

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Countries

France, Germany, Italy, Korea, Republic of, Spain, United Kingdom, United States

Contacts

Public ContactEuropean Clinical Trial Information

Alexion Europe SAS

clinicaltrials.eu@alexion.com3493 2723019

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026