A PHASE II TRIAL AIMING TO INVESTIGATE THE SAFETY AND CLINICAL ACTIVITY OF BELANTAMAB MAFODOTIN IN ADULT PATIENTS WITH PRIMARY IMMUNE THROMBOCYTOPENIA PREVIOUSLY TREATED WITH A THROMBOPOIETIN RECEPTOR AGONIST AND/OR RITUXIMAB AFTER CORTICOSTEROID FIRST-LINE THERAPY

NCT07019545 | Not Yet Recruiting Phase 2

• 2 other identifiers: EAE1402023-509131-12-00
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

Brief Summary

This is a Phase II open label, prospective, multicenter trial designed to assess the safety and clinical activity of belantamab mafodotin in adult patients with primary immune thrombocytopenia (ITP) previously treated with a thrombopoietin receptor agonist (TPO-RA) and/or rituximab after first-line treatment with corticosteroids. Overall, 14 participants will be enrolled in the trial. Participants' follow-up will continue for up to 12 months after the last participant is enrolled. The accrual period will be approximately 12 months. Trial treatment will be given in 28-day cycles for a total period of one year per patient or until treatment failure, physician decision, unacceptable toxicity, withdrawal of consent, or death (whichever occurs first). Participants without confirmed Response (R) or better after two infusions with belantamab mafodotin will be discontinued from trial treatment and will not be replaced.

Conditions

Primary Immune Thrombocytopenia (ITP)

Outcome Measures

Primary Outcomes (1)

• Clinical efficacy of treatment with belantamab mafodotin in terms of Complete response rate (CRR)/Partial response rate (PRR)

  CRR/PRR is defined as the percentage of participants with a confirmed best response of PR or CR (Response-evaluable population). The denominator will be the total number of participants in the Response-evaluable population.

  At 6 months of treatment

Study Arms (1)

Single Arm

OTHER

Trial treatment is defined as the combination of belantamab mafodotin plus dexamethasone. Trial treatment is given in 28-day cycles for a total period of one year per patient or until treatment failure (loss of response), physician decision, unacceptable toxicity, withdrawal of consent, or death (whichever occurs first). Participants without confirmed response or better after two infusions with belantamab mafodotin, and the interval following the dosing, will be discontinued from trial treatment and will not be replaced.

Drug: Belantamab Mafodotin; Drug: Dexamethasone

Interventions

Belantamab Mafodotin DRUG

Belantamab mafodotin is administered intravenously (IV) at a dose of 1.9 mg/kg on Day 1 of every other 28-day cycle for the first two administrations and then 1.9 mg/kg on Day 1 of every third 28-day cycle.

Single Arm

Dexamethasone DRUG

Dexamethasone is administered IV or per OS (PO) at a dose of 40 mg/day on days 1, 2, 3, 4 and 15, 16, 17, 18 of every 28-day cycle for the first two cycles in participants \<75 years; at 20 mg/day on days 1, 2, 3, 4 and 15, 16, 17, 18 of every 28-day cycle for the first two cycles in participants ≥75 years.

Single Arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participant must be 18 years or older.
• Primary ITP with platelet cell count of less than 30x10\^9/L.
• Prior first-line therapy with corticosteroids.
• Prior second-line therapy with TPO-RA and/or rituximab and failure to achieve or retain response.
• Adequate organ system function as defined by the below laboratory assessments. Hematologic
• Absolute neutrophil count ≥1.5 X 10\^9/L; granulocyte colony stimulating factor use within the past 14 days is NOT permitted.
• Hemoglobin ≥8.0 g/dL; transfusions within the past 14 days are NOT permitted. Erythropoietin use is allowed. Hepatic
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• Total bilirubin ≤1.5xULN (isolated bilirubin ≥1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin \<35%).
• Alanine aminotransferase ≤ 2.5xULN. Renal
• a. Estimate glomerular filtration rate ≥30 mL/min/1.73 m\^2; calculated using the Modified Diet in Renal Disease formula.
• Female participants: contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical trials: A female participant is eligible to participate if she is not pregnant or breastfeeding, and at least one of the following conditions applies:
• Is not a woman of childbearing potential (WOCBP) defined as follows:
• ≥45 years of age and has not had menses for \>1 year.
• Participants who have been amenorrhoeic for \<2 years without history of a hysterectomy and oophorectomy must have a follicle stimulating hormone value in the postmenopausal range upon screening evaluation.

You may not qualify if:

• Secondary ITP including:
• i. Drug induced ITP. ii. ITP associated with any autoimmune disorders (e.g., systemic lupus erythematosus and rheumatoid arthritis). iii. ITP associated with chronic infection (e.g., human immunodeficiency virus, hepatitis C virus and helicobacter pylori). iv. ITP associated with malignancy (e.g., chronic lymphocytic leukemia or large granular T-lymphocyte lymphocytic leukemia). v. ITP associated with chronic infection.
• Chronic liver disease. Cirrhosis or current unstable liver or biliary disease per investigator assessment defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminaemia, oesophageal or gastric varices, persistent jaundice. NOTE: Stable non-cirrhotic chronic liver disease (including Gilbert's syndrome or asymptomatic gallstones) is acceptable if participant otherwise meets entry criteria.
• To be seropositive for hepatitis B (defined by a positive test for hepatitis B surface antigen \[HBsAg\]) at screening or within 3 months prior to first dose of trial treatment.
• NOTE 1: Participants with resolved infection (i.e., participants who are positive for antibodies to hepatitis B core antigen \[anti-HBc\] or antibodies to hepatitis B surface antigen \[anti-HBs\]) must be screened using real-time polymerase chain reaction (PCR). Those who are PCR positive will be excluded.
• To be seropositive for hepatitis C at screening or within 3 months prior to first dose of trial treatment.
• NOTE: Participants with positive hepatitis C antibody due to prior resolved disease can be enrolled, only if a confirmatory negative Hepatitis C RNA test is obtained. Hepatitis RNA testing is optional and participants with negative hepatitis C antibody test are not required to also undergo hepatitis C RNA testing.
• Known HIV infection unless the participant can meet all of the following criteria:
• Established anti-retroviral therapy (ART) for at least 4 weeks and HIV viral load \<400 copies/mL
• CD4+ T-cell (CD4+) counts ≥350 cells/uL
• No history of AIDS-defining opportunistic infections within the last 12 months NOTE: consideration must be given to ART and prophylactic antimicrobials that may have a drug:drug interaction and/or overlapping toxicities with belantamab mafodotin or other combination products as relevant.
• Active infection requiring treatment.
• Presence of active renal condition (infection, requirement for dialysis, or any other significant condition that could affect participant's safety).
• Any serious and/or unstable pre-existing medical or psychiatric disorder, or other conditions (including laboratory abnormalities) that could interfere with participant's safety, obtaining informed consent, or compliance to the trial procedures.
• Participant must not have received a live or live-attenuated vaccine within 30 days prior to first dose of belantamab mafodotin.

Sponsors & Collaborators

• Hellenic Society of Hematology: lead

Study Sites (1)

General Hospital of Athens "Alexandra", Athens, Attica

Athens, 11528, Greece

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

belantamab mafodotin; Dexamethasone

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated

Central Study Contacts

Georgios Vasilopoulos, Professor

CONTACT

+302107211806; infohaema@eae.gr

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 5, 2025
• First Posted: June 13, 2025
• Study Start: June 20, 2025
• Primary Completion (Estimated): June 20, 2026
• Study Completion (Estimated): June 20, 2027
• Last Updated: June 13, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

GR (1)