New pill could tame rare blood disorder when other treatments fail
NCT ID NCT07453368
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a daily pill called orelabrutinib for people with autoimmune hemolytic anemia (AIHA) that hasn't responded to standard treatments. AIHA is a condition where the immune system mistakenly attacks red blood cells, causing fatigue and anemia. The trial will enroll 50 adults and try different doses to see if the drug can control the disease and reduce symptoms, while monitoring for side effects.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Orelabrutinib
- What this could lead to
- If successful, this could offer a new oral treatment option for people with hard-to-treat autoimmune hemolytic anemia, potentially reducing the need for high-dose steroids or transfusions.
- What could go wrong
- This is an early Phase 2 trial with only 50 participants, so results may not apply to everyone. The drug may cause side effects or not work for some patients, and long-term safety is still being studied.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
About 50 people
The number the study aims to enrol. It can still change while the study runs.
- Started
-
Mar 2026
- Expected to finish
-
Mar 2028
An estimate. End dates often move.
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 to 85 years
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Age ≥ 18 years. * Patients with a definitive diagnosis of AIHA. wAIHA or Evans syndrome, primary or secondary to connective tissue disease (CTD) or lymphoproliferative disorder (LPD). If secondary, CTD should have no indication for treatment for other system involvement, and LPD should be in an asymptomatic, observation phase without treatment indication. cAIHA, primary or secondary to LPD. If secondary, LPD should be in an asymptomatic, observation phase without treatment indication. * For wAIHA: No response (no partial response after 3 weeks) to full-dose glucocorticoid therapy (1-2 mg/kg/day), intolerance, or relapse (after initial response, hemoglobin drop ≥20 g/L or requiring re-initiation of immunosuppressive therapy). And hemoglobin ≤ 100 g/L. * For cAIHA: No response or relapse after anti-CD20 monoclonal antibody therapy (after initial response, hemoglobin drop ≥20 g/L or recurrence of vascular symptoms, or requiring re-initiation of immunosuppressive therapy), or unsuitable for anti-CD20 therapy. And hemoglobin ≤ 100 g/L or presence of vascular symptoms. * Baseline liver and kidney function (ALT, Cr) within 2 times the upper limit of normal (elevated AST, LDH, Bil due to hemolysis are not used as indicators for liver function assessment). * Agreement to sign the informed consent form. Exclusion Criteria: * Active involvement of other major organs due to connective tissue disease. * Uncontrolled infection or bleeding per standard treatment. * Uncontrolled active HIV, HCV, or HBV infection per standard treatment. * Concurrent advanced uncontrolled malignancy, lymphoma. * Presence of other uncorrected types of anemia at screening, such as nutritional anemia, thalassemia, etc. * Currently receiving glucocorticoid therapy at screening and unable to discontinue or taper to ≤ 15 mg/day (prednisone equivalent) within 1 week before enrollment. * Currently receiving treatment with medications such as cyclosporine, tacrolimus, sirolimus, cyclophosphamide, azathioprine, stanozolol, testosterone undecanoate, danazol, etc., at screening, and the treatment duration at a stable dose is \< 12 weeks, or unable to discontinue directly. * Last dose of rituximab \< 3 months prior to enrollment. * Prior treatment with any BTK inhibitor. * Liver cirrhosis or portal hypertension. * Pregnant or breastfeeding women. * Participation in another clinical trial within the past 3 months.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Autoimmune hemolytic anemia (AIHA) are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
-
The places running it
1 site. The list below names each one and where it is.
-
The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
-
A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
-
Peking Union Medical College Hospital
RECRUITINGBeijing, Beijing Municipality, 100730, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can engineered immune cells tame autoimmune anemia?
- Engineered immune cells take aim at stubborn blood disorder
- Experimental mRNA CAR-T therapy targets autoimmune diseases in first human trial
- New drug combo aims to tame stubborn blood disorders
- New combo aims to tame autoimmune anemia without Long-Term steroids
- Thousands with blood diseases to be tracked in massive chinese registry