ASCEND-WAIHA

• Research type

  Research Study

• Full title

  A Phase 2, Multicenter, Non-Randomized, Open-Label Study of RVT-1401 for the Treatment of Patients with Warm Autoimmune Hemolytic Anemia

• IRAS ID

  274855

• Contact name

  Charlotte Pai

• Contact email

  Charlotte.Pai@parexel.com

• Sponsor organisation

  Immunovant, Inc

• Eudract number

  2019-003924-19

• Clinicaltrials.gov Identifier

  XX, XX

• Duration of Study in the UK

  0 years, 6 months, 30 days

• Research summary

  Summary of Research

  Warm Autoimmune Haemolytic Anaemia (WAIHA) is an autoimmune disease where the immune system makes an antibody (blood protein) that attacks the red blood cells in the body by mistake. Normally, antibodies protect against infections but the abnormal antibodies that are formed in WAIHA cause red blood cells to be destroyed. Symptoms include tiredness, difficulty breathing, pale skin, and enlarged spleen (organ involved in the production and removal of blood cells) as well as some abnormal blood test results.

  Immunovant Sciences GmbH is sponsoring the ASCENDE - WAIHA study to investigate the safety and efficacy of a new, experimental drug RVT-1401, to treat WAIHA.

  The purpose of this study is:
  • To look at the effects, good and bad, that the study drug has on participants
  • To measure the amount of study drug in participants blood at different times
  • To measure how antibodies in participants blood are affected by the study drug
  • To examine how the study drug affects WAIHA symptoms

  Participants will be in the study for up to approximately 24 weeks (6 months). This includes a screening phase of 4 weeks (1 month), a 12-week treatment phase (3 months), and an 8 week follow-up period (2 months). RVT-1401 is injected under the skin. It is a human monoclonal antibody that targets the immune system.

  The study drug is not approved by the UK Medicines and Healthcare products Regulatory Agency (MHRA) or any other regulatory agency but is currently being tested in research studies. The study drug has been administered to 77 healthy participants. There are 3 ongoing studies of the study drug in participants with diseases other than WAIHA.

  This study will involve a total of approximately 16 participants across approximately 22 study hospitals worldwide.

  Summary of Results

  Treatment was initiated in 5 patients and of those, only 2 completed treatment and follow up. The data is too limited to analyze for any specific results, therefore, the CSR outlines the facts of the assessments for the patients. The CSR serves as the Sponsor’s internal report and regulatory authority submissions are in process.

• REC name

  HSC REC B

• REC reference

  20/NI/0006

• Date of REC Opinion

  26 Feb 2020

• REC opinion

  Further Information Favourable Opinion