A Phase 3 Study of Obexelimab in Patients with Warm Autoimmune Hemolytic Anemia
Research type
Research Study
Full title
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study, with a Safety and Dose Confirmation Run-in Period, to Evaluate the Efficacy and Safety of Obexelimab in Patients with Warm Autoimmune Hemolytic Anemia (SApHiAre)
IRAS ID
1007139
Contact name
Lisa Crockett
Contact email
Sponsor organisation
Zenas BioPharma (USA) LLC
ISRCTN Number
n/a
Clinicaltrials.gov Identifier
n/a
Research summary
Warm autoimmune haemolytic anemia (wAIHA) is an autoimmune disorder where the body’s B cells (a type of immune cell) attack and destroy red blood cells leading to anemia. Symptoms include fatigue, jaundice, dark urine, and an enlarged spleen.
Standard first-line treatment for wAIHA is daily doses of corticosteroids. However, long term treatment can lead to significant toxicity and complications. B cell directed therapies have obtained promising results in clinical trials, for example, rituximab which is becoming the preferred second-line therapy.
Obexelimab (a B cell targeted therapy) is an investigational drug being developed by Zenas BioPharma, which means it has not yet been approved for the treatment of a disease by any regulatory agencies and can only be tested in a study like this one. This study will test obexelimab administered as a subcutaneous injection (under the skin) given once a week.
The purpose of this study is to learn more about how safe and how well obexelimab works to treat wAIHA.
This study is divided into three parts:
• Part A: Safety and Dose Confirmation Run-in Period. All patients will receive obexelimab and this part will evaluate safety and tolerability.
• Part B: Randomised, double-blind, placebo-controlled. Half (50%) of the patients who enroll will receive placebo. This part will evaluate the efficacy and safety of obexelimab.
• Part C: Open-label extension portion. This part will look at how obexelimab works over an extended period of time. If participants complete Part A or B, they will be allowed to enroll in Part C.Approximately 134 patients will participate in this global study. Participants in Parts A and B will be on the study for 40 weeks, and participants in Part C will be on the study for an additional 52-week treatment period and a 12-week follow-up . During this time, they will have several visits to have tests and procedures to check their health and the effects of the study drug.
REC name
London - City & East Research Ethics Committee
REC reference
23/LO/0335
Date of REC Opinion
8 Sep 2023
REC opinion
Further Information Favourable Opinion