ALXN1830 SC in Patients with WAIHA

• Research type

  Research Study

• Full title

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

• IRAS ID

  1003862

• Contact name

  Cristina Guzman

• Contact email

  Cristina.Guzman@alexion.com

• Sponsor organisation

  Alexion Pharmaceuticals, Inc.

• Eudract number

  2021-001211-90

• Research summary

  Warm Autoimmune Haemolytic Anaemia (WAIHA) is caused by antibodies (a type of proteins that helps protect the body against foreign matter, such as bacteria and viruses) that attach to the red blood cells and cause their destruction. This causes anaemia, which means the red blood cell count in the body becomes low.

  There are several symptoms of anaemia and not everyone will have the same or all symptoms; for example, patients can be easily tired, short of breath, or have headaches or dizziness.

  Current treatment options are associated with significant limitations including toxicities, delayed onset of action, high frequency of relapse, patient inconvenience, and limited access.

  ALXN1830 is an investigational drug being developed by Alexion Pharmaceuticals, Inc. to treat WAIHA in patients who have failed or not tolerated the current standard treatment.

  The purpose of the study is to determine if ALXN1830 will decrease the levels of the antibodies causing the destruction of red blood cells which leads to anaemia as compared with placebo.

  About 30 study sites will be opened around the world to enrol up to 36 participants over the age of 18 with WAIHA.

  Patients will be randomly assigned to receive either the study drug ALXN1830 in one of 3 cohorts, or placebo (a dummy treatment that contains no active ingredients) via an infusion under the skin for approximately 45 to 60 minutes:
  • Cohort 1: ALXN1830 at a weekly dose of 1500mg or placebo for 8 weeks
  • Cohort 2: ALXN1830 at a weekly dose of 2250 mg or placebo for 8 weeks
  • Optional Cohort 3: ALXN1830 at a weekly dose of ≤ 2250 mg for 4 weeks and then ALXN1830 ≤ 2250 mg every other week for 8 weeks

  The minimum duration in the study will be around 20 weeks for Cohorts 1 and 2 and 24 weeks for Cohort 3. There is also an option of continuing into the Open-label Extension period (OLE) of this study which may last for a maximum of 2 years and will consist of monthly visits to observe for relapse.

• REC name

  London - Hampstead Research Ethics Committee

• REC reference

  21/LO/0526

• Date of REC Opinion

  23 Aug 2021

• REC opinion

  Further Information Favourable Opinion