Phase 2a Study of Tafasitamab in Adults with Primary Immune Thrombocytopenia and Warm Autoimmune Hemolytic Anemia
- Trial ID
- 2025-521286-27-00
- Protocol
- INCA000585-201
- Sponsor
- Incyte Corp.
Trial statistics
Diseases & Conditions
Objectives
The primary objectives of this study are to evaluate the safety and tolerability of tafasitamab in adults with primary immune thrombocytopenia or primary warm autoimmune hemolytic anemia. Additionally, the study aims to assess the efficacy of tafasitamab in these populations. Secondary objectives include:
- Further determination of efficacy in participants with primary immune thrombocytopenia and primary warm autoimmune hemolytic anemia.
- Characterization of the pharmacokinetics of tafasitamab.
- Characterization of the immunogenicity of the study drug.
Participants
This study includes 36 participants diagnosed with immune-mediated thrombocytopenia or autoimmune hemolytic anemia. The study population consists of adults who weigh at least 50 kg and possess an ECOG performance status of 0–2. Eligible individuals must have a confirmed history of primary ITP characterized by isolated thrombocytopenia, or primary wAIHA involving isolated anemia with a positive direct antiglobulin test. Participants must have a history of transient response to at least one prior early-line treatment and have received at least one standard course of rituximab at least six months prior to the start of the study. The study objectives are:
- To determine the safety and tolerability of tafasitamab in participants with primary ITP or primary wAIHA.
- To determine the efficacy of tafasitamab in participants with primary ITP.
- To determine the efficacy of tafasitamab in participants with primary wAIHA.
Plans and Procedures
This Phase 2a, open-label, multicenter study is designed to evaluate the safety, tolerability, and efficacy of tafasitamab in adults with primary immune thrombocytopenia or primary warm autoimmune hemolytic anemia. The research methodology focuses on assessing adverse events, stable platelet response, and stable hemoglobin response. Following an initial screening visit to confirm eligibility, participants will receive intravenous infusion of the study medication. The study involves periodic assessments of vital signs, clinical laboratory blood samples, and electrocardiograms to monitor clinical status. Participant involvement includes follow-up evaluations to measure response rates, such as complete remission and partial response, through at least 48 weeks. Early termination of study participation may occur based on clinical necessity or safety requirements. The total duration of the trial is estimated to conclude by July 2027.
Treatment
The experimental medication consists of tafasitamab, provided as MINJUVI 200 mg powder for concentrate for solution for infusion. The administered dose is 1000 mg via intravenous infusion.
Efficacy
Efficacy assessment in this study focuses on specific hematological responses in participants with immune thrombocytopenia or autoimmune hemolytic anemia. The primary efficacy endpoints include a stable platelet response, characterized by a platelet count ≥ 50 × 10⁹/L without clinically significant bleeding or rescue therapy at two consecutive assessments between Day 56 and Week 48. Additionally, a stable hemoglobin response is evaluated, defined as hemoglobin ≥ 10 g/dL and an increase of ≥ 2 g/dL from baseline without rescue therapy during the same period.
Secondary endpoints include the following:
- Complete remission (CR) at Week 24 and Week 48, based on platelet counts ≥ 100 × 10⁹/L or hemoglobin levels ≥ 12 g/dL with normalization of hemolytic markers, including unconjugated bilirubin, LDH, haptoglobin, and reticulocytes.
- Partial response (PR) at Week 24, assessed via platelet count or hemoglobin levels.
- The duration of response, including the duration of stable platelet response, duration of CR, and duration of stable hemoglobin response, measured from the onset of response until the loss of response, rescue therapy, clinically significant bleeding, or death.
- Changes in serum antidrug antibody levels from baseline.
Inclusion and Exclusion Criteria
Inclusion Criteria
- Participant has signed informed consent, is ≥18 years old, and weighs ≥50 kg.
- Confirmed historical diagnosis of one of the following autoimmune blood disorders: a. Primary ITP: isolated thrombocytopenia (peripheral blood count < 100 × 10⁹/L) in the absence of other causes or disorders associated with isolated thrombocytopenia. Participants must have persistent (3- to 12-month duration) or chronic (> 12-month duration) ITP. b. Primary wAIHA: isolated anemia and DAT result positive for IgG, with or without C3d, not due to another cause.
- No history of splenectomy.
- Confirmed transient response to at least 1 prior early-line treatment (eg, corticosteroids, IVIG, rituximab): a. Primary ITP: Increase in platelet count to ≥ 30 × 10⁹/L with at least a 2-fold increase of baseline platelet count b. 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. a. 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. b. 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. ITP: Platelet count <30 × 10⁹/L within 15 days before Day 1. a. Primary ITP: platelet count < 30 × 10⁹/L within the 15 days before treatment is scheduled to begin (Day 1). b. 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 0–2.
- Willingness to avoid pregnancy or fathering children based on the criteria below. a. Male participants with reproductive potential must agree to take appropriate precautions to avoid fathering children, including refraining from donating sperm, from screening through 90 days (a spermatogenesis cycle) after the last dose of tafasitamab. b. Female participants who are WOCBP must have a negative serum pregnancy test at screening and a negative urine pregnancy test before the first dose on Day 1 and must agree to take appropriate precautions to avoid pregnancy and refrain from donating oocytes from screening through 90 days after the last dose of study tafasitamab. c. Women not of childbearing potential: Eligible.
Exclusion Criteria
- Clinical manifestations typical for cold agglutinin disease (eg, cold-induced symptoms, including acrocyanosis; DAT positive for C3d and generally negative for Ig; and/or cold agglutinin titer > 64).
- 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
- Receipt of medications or investigational drugs for primary ITP/wAIHA within the following interval before the first administration of study drug: a. < 2 weeks for immunosuppressant drugs other than corticosteroids b. < 4 weeks or 5 half-lives for any investigational agent
- Changes in doses (> 10%) of permitted disease-related therapies (see Section 6.6.1), including oral corticosteroids, TPO-RA (primary ITP participants), ESA (primary wAIHA participants) 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.
- Pregnant or breastfeeding women.
- History of malignancy except for the following: a. Malignancy treated with curative intent with no evidence of active disease for more than 2 years before screening. b. Adequately treated lentigo maligna melanoma without current evidence of disease or adequately controlled nonmelanoma skin cancer. c. 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).
- Active infections: a. HCV RNA positive. b. Chronic HBV (HBsAg or HBV DNA positive). c. HIV positive.
- Active systemic infection (including COVID-19).
- Severely immunocompromised (per investigator).
- Live-attenuated vaccine within 4 weeks before first tafasitamab dose.
- Coagulation or platelet function disorders; need for anticoagulation (e.g., recent stent).
- Any condition interfering with study participation or data interpretation.
- Unresolved toxicities from prior therapies (must be ≤ Grade 1, or ≤ Grade 2 if chronic).
Trial Status by Country
| Country | Status | Start of Recruitment | Planned Patients |
|---|---|---|---|
 France | Recruiting | 17 Feb 2026 | 5 |
 Italy | Recruiting | 17 Feb 2026 | 5 |
 The Netherlands | Recruiting | 17 Feb 2026 | — |
 Spain | Recruiting | 17 Feb 2026 | 5 |
| Netherlands | — | — | 5 |
Sites & Investigators
Investigational Products
Details about the medicinal products being studied in this clinical trial.
| Product Name | Role in Trial | Formulation | Administration | Max Daily Dose | Treatment Duration | EU MP Number |
|---|---|---|---|---|---|---|
MINJUVI 200 mg powder for concentrate for solution for infusion | Test | POWDER FOR CONCENTRATE FOR SOLUTION FOR INFUSION | INTRAVENIOUS INFUSION | 1000 | 8 | PRD11607801 |