VENETOCLAX for Acute graft-versus-host disease (aGVHD) | Acute graft-versus-host disease of the skin

Inclusion criteria

• {"criterion_text":"- Written informed consent, according to local guidelines, signed by the patients prior to any study related screening procedures are performed.\n- Male or female patients\n- ≥ 18 years old at the time of informed consent\n- Able to swallow tablets\n- Have undergone allo-HSCT from any donor source (matched unrelated, sibling, or haplo-identical donor) using bone marrow, peripheral blood stem cells, or cord blood. Recipients of non- myeloablative, myeloablative, and reduced intensity conditioning are eligible\n- Evident myeloid and platelet engraftment: Absolute neutrophil count (ANC) > 1000/mm AND Platelet count > 20,000 /mm3 (7)\n- Patients with newly clinically diagnosed aGVHD: aGVHD clinically diagnosed Grades II to IV as per standard criteria (13, 35, 36) occurring after allo HSCT requiring systemic immune suppressive therapy with prednisone 1-2 mg/kg bodyweight. Biopsy of involved organs with aGVHD is encouraged but not required for study screening. Evident myeloid and platelet engraftment (confirmed within 48h prior to study treatment start)."}

Exclusion criteria

• {"criterion_text":"- Persons incapable of giving consent (permanently or temporarily)\n- Patients with relapsed primary malignancy, or who have been treated for relapse after allo HSCT\n- Known human immunodeficiency virus (HIV) infection\n- Active tuberculosis infection that developed after allo HSCT\n- Evidence of active viral disease including CMV, EBV, HHV -6, HBV, HCV, or BK virus.\n- Prior treatment with venetoclax"}

Primary endpoints

• {"endpoint_text":"- Definition of the Maximum Tolerated Dose (MTD) after 28 and 56 days (Phase 1)","definition_or_measurement_approach":""}

Secondary endpoints

• {"endpoint_text":"- Safety of Venetoclax in aGVHD after 28 and 56 days","definition_or_measurement_approach":""}
• {"endpoint_text":"- Remission rate of aGVHD and rate of progression to SR-aGVHD upon Venetoclax","definition_or_measurement_approach":""}
• {"endpoint_text":"- Non-relapse mortality and overall survival after Venetoclax in aGVHD","definition_or_measurement_approach":""}
• {"endpoint_text":"- Effects of Venetoclax on aGVHD-causing effector cells in peripheral blood and affected tissue","definition_or_measurement_approach":""}
• {"endpoint_text":"- Hematologic grade IV toxicities and Grade III/IV non-hematologic toxicities graded according to the NCI CTCAE criteria CTCAE version 4.03.","definition_or_measurement_approach":"Toxicities graded according to the NCI CTCAE criteria version 4.03."}
• {"endpoint_text":"- Efficacy of venetoclax","definition_or_measurement_approach":""}
• {"endpoint_text":"- Inhibition of BCL2 pathway by venetoclax in peripheral blood and the skin on a single cell level","definition_or_measurement_approach":""}
• {"endpoint_text":"- Immunological signature(s) by genome, epigenome and transcriptome sequencing","definition_or_measurement_approach":""}
• {"endpoint_text":"- Baseline BCL2 activity identifying patients as responders or enabling monitoring of response to venetoclax by flow cytometry and immunofluorescence","definition_or_measurement_approach":"Baseline BCL2 activity assessed by flow cytometry and immunofluorescence as stated."}
• {"endpoint_text":"- Quality of life questionnaire, fatigue scale","definition_or_measurement_approach":""}

Austria

Earliest CTIS Part Ii Submission Date

17-10-2025

Latest Decision Or Authorization Date

10-11-2025

Processing Time Days

24

Number Of Sites

1

Number Of Participants

24

Primary sponsor

Full Name

Medical University Of Vienna

Organisation Type

Educational Institution

Country Of Registered Address

Austria