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Experimental drug tested for stubborn blood disorder – but trial cut short
NCT ID NCT05676697
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase study tested the drug Linperlisib in just 4 adults with autoimmune hemolytic anemia (AIHA) whose disease did not improve after at least two prior treatments. The goal was to see if the drug could help control the disease by targeting a key immune pathway. However, the trial was terminated early, so we have very limited information on how well it worked or its safety.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Linperlisib (a PI3K delta inhibitor)
- What this could lead to
- If it works, this could point toward a new treatment option for people with hard-to-treat autoimmune hemolytic anemia.
- What could go wrong
- This was a very small, early-phase trial (only 4 participants) that was terminated early, so results are limited and may not be reliable. The drug may not prove effective or safe in larger studies.
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
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
-
4 people
The number who actually took part.
- Started
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Jan 2023
- Finished
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Feb 2024
- 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
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18 years and older
- Sex
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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: * Male or female age ≥ 18 years * Diagnosis of primary warm antibody hemolytic anemia (AIHA). * Hemoglobin \< 100g/L * Refractory to or relapsed after at least 2 prior treatment line. * ECOG performance status ≤ 2 * Willing and able to comply with the requirements for this study and written informed consent. Exclusion Criteria: * Neutrophils counts \< 0.5×10\^9/L or platelet counts \< 50 x 10\^9/L * Diagnosis of any of the following diseases: Cold agglutinin disease, cold agglutinin syndrome, mixed AIHA, paroxysmal cold hemoglobinuria (PCH). * Diagnosis of the active stage of the connective tissue or systemic autoimmune rheumatic diseases (SARDs) * History of lymphoproliferative neoplasms * Had other inherited or acquired hemolytic diseases. * Secondary AIHA caused by drugs or infection * Previously received organ or stem cell transplantation. * Had malignant tumor within 5 years before enrollment, exclusive of cured basal or squamous cell skin cancer, superficial bladder cancer, prostate intraepithelial tumor, cervical carcinoma in situ or other indolent tumors * Patients with HBV, HCV, HIV or other infections that require treatment. * Abnormal liver function: two consecutive examinations with an interval of ≥1 week suggest that ALT and AST are 2.5 times higher than the upper limit of normal values * Renal impairment: creatinine clearance \<60ml/min * Any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study, including clinically significant cardiac diseases, refractory hypertension, metabolic disorders and other diseases that seriously affect the function of the gastrointestinal tract. * Had a history of any psychiatric diseases, cerebrovascular disease or cognitive sequelae of head injury. * Received rituximab in 6 weeks before enrollment. * Received attenuated vaccine 4 in weeks before enrollment * Participation in another clinical trial within 4 weeks before the start of this trial * Have an allergy to Linperlisib or any other part of this medicine. * Previously treated with other PI3Kδ inhibitor. * Pregnant or breast-feeding patients * Patients considered to be ineligible for the study by the investigator for reasons other than the above
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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.
Contacts and locations
Locations
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Regenerative Medicine Center
Tianjin, Tianjin Municipality, China
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Zhoukou Central Hospital
Zhoukou, Henan, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Experimental drug F182112 tested against Hard-to-Treat blood disorder
- Could a simple blood test predict clot risk in antiphospholipid syndrome?
- Experimental mRNA CAR-T therapy targets autoimmune diseases in first human trial
- Engineered immune cells take on rare blood disorders
- Brain scans reveal hidden thinking problems in anemia patients
- Experimental immune therapy for stubborn anemia shows early promise but trial stalls