* Efficacy, safety and PK of rilzabrutinib in patients with wAIHA
Research type
Research Study
Full title
A multicenter, open-label, Phase IIb study to evaluate the efficacy, safety, and pharmacokinetics of rilzabrutinib in patients with warm autoimmune hemolytic anemia
IRAS ID
300118
Contact name
Steven Davis
Contact email
Sponsor organisation
Sanofi-Aventis Research and Development
Eudract number
2021-001671-16
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 10 months, 21 days
Research summary
Warm autoimmune hemolytic anemia (wAIHA) is a rare condition that is characterized by an accelerated rate of red blood cell (RBC) destruction. (Auto)Antibodies within the blood incorrectly bind to healthy RBCs which are then recognised and destroyed by immune cells within the spleen and liver. This increased destruction of RBC’s leads to anemia which can cause fatigue, shortness of breath, dizziness, palpitations, and jaundice. Severe cases can cause chest pain, lethargy and increased risk of blood clots in the veins, with the condition carrying a mortality rate of 11%.
There are currently no approved treatments for wAIHA and the first-line therapy typically consists of steroids, followed by rituximab. Limited options exist for patients who do not have an adequate response to these interventions which include immunosuppressants or splenectomy, both of which can have significant side effects. There is therefore a clear need for the development of safe and effective treatments for patients with wAIHA.
Bruton’s tyrosine kinase (BTK) plays a key role in many of the cell functions related to wAIHA. It has been shown that inhibiting BTK can lead to a decrease in the levels of autoantibodies in the blood and can also block the process by which immune cells incorrectly destroy healthy RBCs. Rilzabrutinib (SAR444671, formerly PRN1008) is a high-affinity inhibitor of BTK. It is hypothesized that treating patients with wAIHA with rilzabrutinib may result in a reduction in RBC destruction and rapid and sustained increase in hemoglobin levels.
The study will aim to determine if rilzabrutinib is a safe and effective treatment for patients with wAIHA. The study will be conducted in 2 parts: Part A to evaluate efficacy and safety and Part B, a long-term extension, for assessment of long-term safety and maintenance of effect. Approximately 22 participants are expected to enroll in this study worldwide.
REC name
North West - Liverpool Central Research Ethics Committee
REC reference
22/NW/0263
Date of REC Opinion
23 Jul 2021
REC opinion
Further Information Favourable Opinion