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New pill could tame rare blood disorder when other treatments fail

NCT ID NCT07453368

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

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.

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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.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. 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

    RECRUITING

    Beijing, Beijing Municipality, 100730, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.