VAYHIA
Research type
Research Study
Full title
A phase 3, randomized, double-blind, study to assess efficacy and safety of ianalumab (VAY736) versus placebo in warm autoimmune hemolytic anemia (wAIHA) patients who failed at least one line of treatment (VAYHIA)
IRAS ID
1006586
Contact name
Abigail Spiteri
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2022-001773-31
Clinicaltrials.gov Identifier
Research summary
The purpose of the study is to understand if ianalumab is safe and effective and can improve the course of the disease for people who have warm autoimmune hemolytic anemia (wAIHA). Participants will have a 2 in 3 (66.6%) possibility of receiving the study treatment (ianalumab at a lower or higher dose) and a 1 in 3 (33.33%) possibility of receiving placebo. Participants could be in the study between 29 and 73 months (depending on the status of their health during their participation in the study). During that time, they will visit the Study Doctor between 23 to 61 times but will have the option to do some of the visits outside of the study site. During this time, participant will need to visit the Study Doctor:
• at screening
• every other week during the blinded treatment period
• every other week if they enter the open label cross over phase
• on a monthly/quarterly basis during the follow-up period
At the study visits, the following procedures should happen:
• a physical examination
• blood draws for laboratory testing
• analysis of urine samples
• a urine or blood pregnancy test (if participants are female and able to have children)
• answering questionnaires on how they are feeling
Participants will also receive study treatment through a vein every 4 weeks (4 in total) during the blinded treatment period. If they are randomized to placebo, and their doctor thinks is beneficial with their consent, participants may be given the possibility to receive ianalumab at the higher dose in an open label period, every 4 weeks (4 doses in total).
Ianalumab has been studied in other diseases (in almost 500 patients) and demonstrated to be safe. However, it has not been studied in wAIHA so we dont know whether it will work for these participants. Their condition may improve, may get worse, or there may be no change. A study performed in a similar autoimmune disease using drugs that work in the same way as ianalumab has shown promising efficacy results.REC name
London - Harrow Research Ethics Committee
REC reference
22/LO/0815
Date of REC Opinion
26 Jan 2023
REC opinion
Further Information Favourable Opinion