Subcutaneous ALXN1830 in Adult Participants With Warm Autoimmune Hemolytic Anemia

Alexion Pharmaceuticals

Status and phase: Withdrawn; Phase 2

Conditions: Warm Autoimmune Hemolytic Anemia

Treatments: Drug: Placebo; Drug: ALXN1830

Study type: Interventional

Funder types: Industry

Identifiers

NCT04956276

ALXN1830-WAI-202

Details and patient eligibility

About

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.

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Double Blind

---

0 participants in 3 patient groups

Cohort 1: ALXN1830/Placebo

Experimental group

Description:

Participants will be randomized 3:1 to receive ALXN1830 or placebo. Treatment will be received for 8 weeks followed by a follow-up period (no treatment) for 8 weeks. Once complete, participants may continue participation in the study at the participant's and investigator's discretion during the OLE period for up to 2 years inclusive of primary treatment period.

Treatment:

Drug: ALXN1830

Drug: Placebo

Cohort 2: ALXN1830/Placebo

Experimental group

Description:

Participants will be randomized 3:1 to receive ALXN1830 or placebo. Treatment will be received for 8 weeks followed by a follow-up period (no treatment) for 8 weeks. Once complete, participants may continue participation in the study at the participant's and investigator's discretion during the OLE period for up to 2 years inclusive of primary treatment period.

Treatment:

Drug: ALXN1830

Drug: Placebo

Cohort 3: ALXN1830

Experimental group

Description:

If initiated, participants will receive ALXN1830. Treatment will be received for 12 weeks followed by a follow-up period (no treatment) for 8 weeks. Once complete, participants may continue participation in the study at the participant's and investigator's discretion during the OLE period for up to 2 years inclusive of primary treatment period.

Treatment:

Drug: ALXN1830