WithdrawnPhase 2

Subcutaneous ALXN1830 in Adult Participants With Warm Autoimmune Hemolytic Anemia

Stopped: Sponsor decision to terminate program

United States0Started 2022-01-01

Plain-language summary

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.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

What they're measuring

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

Timeframe: Baseline through Week 12

Trial details

NCT IDNCT04956276

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-07-31