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Tafasitamab for adults with primary immune thrombocytopenia or warm autoimmune hemolytic anemia

A Phase 2a, Open-Label, Multicenter Study of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders

Phase 2 Interventional Incyte Corporation · NCT07104565

This trial will try tafasitamab, an anti‑CD19 antibody, in adults with primary immune thrombocytopenia or warm autoimmune hemolytic anemia to see if it is safe and helps blood counts.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment56 (estimated)
Ages18 Years and up
SexAll
SponsorIncyte Corporation Industry-sponsored
Drugs / interventionsrituximab, tafasitamab, CAR T
Locations40 sites (Glendale, Arizona and 39 other locations)
Trial IDNCT07104565 on ClinicalTrials.gov

What this trial studies

This is a phase 2, interventional trial testing tafasitamab (an anti‑CD19 monoclonal antibody) in adults with primary autoimmune blood cell disorders, specifically primary immune thrombocytopenia (ITP) and primary warm autoimmune hemolytic anemia (wAIHA). Eligible participants must be 18 or older, have a confirmed historical diagnosis of primary ITP or wAIHA, have no history of splenectomy, and have shown a prior transient response to at least one early‑line therapy; prior rituximab treatment is required with the last dose given at least six months before study treatment. The trial will monitor safety and tolerability and will track hematologic responses such as platelet and hemoglobin increases to gauge efficacy. The study is sponsored by Incyte and is enrolling at multiple U.S. hematology/oncology centers.

Who should consider this trial

Good fit: Adults (≥18 years) with confirmed primary ITP or primary wAIHA who have not had a splenectomy, who previously had a transient response to at least one early‑line therapy, and who received at least one standard course of rituximab with the last dose ≥6 months before enrollment are the intended candidates.

Not a fit: Patients who were nonresponsive to rituximab when it was their only prior therapy, those with prior splenectomy, or those who do not meet the specified prior‑response criteria may not benefit or be eligible.

Why it matters

Potential benefit: If successful, tafasitamab could offer an additional treatment option that produces meaningful increases in platelet counts or hemoglobin and potentially longer remissions for some patients.

How similar studies have performed: Rituximab (anti‑CD20) has established benefit in many autoimmune cytopenias, while anti‑CD19 agents like tafasitamab have proven activity in B‑cell cancers but have had limited prior testing in primary autoimmune blood cell disorders.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

\- Ability to comprehend and willingness to sign a written ICF for the study.

* Aged ≥ 18 years.
* Confirmed historical diagnosis of one of the following autoimmune blood disorders:

  * Primary ITP.
  * Primary wAIHA.
* No history of splenectomy.
* Confirmed transient response to at least 1 prior early-line treatment (eg, corticosteroids, IVIG, rituximab):

  * Primary ITP: Increase in platelet count to ≥ 30 × 109/L with at least a 2-fold increase of baseline platelet count.
  * Primary wAIHA: Increase in hemoglobin to ≥ 10 g/dL with an increase of at least 2 g/dL from baseline.
* Received ≥ 1 standard course of rituximab (375 mg/kg × 4 weekly doses or 2 doses of 1000 mg flat dose every 2 weeks) with last dose given at least 6 months prior to initiation of study treatment. Note: If rituximab was the only prior therapy, individuals with NR to rituximab will not be eligible.

  * Primary ITP: a PR (platelet count ≥ 30 × 109/L with at least a 2-fold increase of baseline platelet count) within 6 months of the last administered dose followed by relapse OR a CR (platelet count \> 100 × 109/L) lasting \< 48 weeks OR NR (platelet count \< 30 × 109/L or less than 2-fold increase of baseline platelet count or bleeding) within 6 months of the last administered dose.
  * Primary wAIHA: a PR with hemoglobin ≥ 10 g/dL and with an increase of at least 2 g/dL from baseline OR a CR (hemoglobin ≥ 12 g/dL and normalization of hemolytic markers) OR NR (hemoglobin \< 10 g/dL or \< 2 g/dL increase of baseline hemoglobin).
* Persistent or chronic active primary ITP or active primary wAIHA with indication for treatment at the time of inclusion.

  * Primary ITP: platelet count \< 30 × 109/L within the 15 days before treatment is scheduled to begin (Day 1).

Note: Participants treated with a rescue therapy during screening in response to a documented platelet count \< 30 × 109/L are eligible, irrespective of platelet count within 15 days of Day 1.

• Primary wAIHA: hemoglobin \< 10 g/dL documented with DAT result positive for IgG, with or without C3d, and evidence of hemolysis based on low haptoglobin, elevated LDH, and/or indirect bilirubin.

* ECOG performance status of 0 to 2.
* Willingness to avoid pregnancy or fathering children.
* Further inclusion criteria apply.

Exclusion Criteria:

* Clinical manifestations typical for cold agglutinin disease.
* Life-threatening bleeding or urgent need to elevate the platelet count for primary ITP or hemodynamic instability or hemoglobin \< 6 g/dL with urgent need to elevate hemoglobin for primary wAIHA within 2 weeks prior to Day 1.
* Prior treatment with anti-CD19 therapy (eg, mAb, bispecific T-cell engager, or CAR T cell) for any indication.
* Previous severe allergic reaction to a mAb or known allergy to any component/excipient of tafasitamab.
* Changes in doses (\> 10%) of permitted disease-related therapies, including oral corticosteroids and TPO-RA (primary ITP participants) within 2 weeks prior to Day 1, or change in ESA (primary wAIHA participants) dose within 2 weeks prior to Day 1.
* Evidence of hypogammaglobulinemia during screening (IgA \< 70 mg/dL, IgG \< 700 mg/dL, and/or IgM \< 40 mg/dL) and frequent and/or severe infections.
* Women who are pregnant or breastfeeding.
* History of malignancy except for the following:

  * Malignancy treated with curative intent with no evidence of active disease for more than 2 years before screening.
  * Adequately treated lentigo maligna melanoma without current evidence of disease or adequately controlled nonmelanoma skin cancer.
  * Adequately treated carcinoma in situ without current evidence of disease.
* Congestive heart failure (left ventricular ejection fraction of \< 50%, assessed by 2 dimensional echocardiography or a multigated acquisition scan).
* Participants with:

  * Known positive test result for HCV (with HCV antibody serology testing) and a positive test for HCV RNA.

Note: Participants with positive serology must have been tested for HCV RNA and are eligible only in the case of negative HCV RNA test result.

• Known positive test result for chronic HBV infection (defined by HBsAg positivity or positive HBV DNA test result).

Note: Participants with occult or prior HBV infection (defined as negative HBsAg and positive total HBcAb) may be included if HBV DNA was undetectable, provided that they are willing to undergo monthly ongoing DNA testing. Antiviral prophylaxis may be administered as per institutional guidelines.

Note: Participants who have protective titers of HBsAb (HBsAb positive, HBcAb negative, and HBsAg negative) after vaccination or prior HBV infection are eligible.

• Seropositivity for or history of active viral infection with HIV.

* Active systemic infection (including infection with SARS-CoV-2).
* Participants in a severely immunocompromised state, per investigator's clinical assessment.
* Receipt of a live-attenuated vaccine within 4 weeks prior to the first infusion of tafasitamab (inactivated and killed vaccines are acceptable).
* Coagulation or platelet function abnormality.
* An active medical condition with a strong indication for treatment with anticoagulation agents (eg, intracoronary stent within 12 months).
* Any condition that would, in the investigator's judgment, interfere with full participation in the study, including administration of study treatment and attending required study visits; pose a significant risk to the participant; or interfere with interpretation of study data.
* Toxicities related to prior therapies must be CTCAE (v5.0) ≤ Grade 1 at the time of study treatment/enrollment (except for chronic toxicities \[≤ Grade 2\] not expected to resolve).
* Chronic infectious disease requiring systemic antibiotics or antifungal or antiviral medications.
* Unwillingness to undergo transfusion with blood components.
* Current use of prohibited medication as described in the protocol.
* Inadequate recovery from toxicity and/or complications from a major surgery before starting therapy.
* Further exclusion criteria apply.

Where this trial is running

Glendale, Arizona and 39 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Immune Thrombocytopeniaprimary warm autoimmune hemolytic anemiaprimary immune thrombocytopeniaanti-CD19 monoclonal antibody
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.