Venetoclax as treatment of acute graft-versus-host disease in patients after allogeneic hematopoietic stem cell transplantation in a Phase-II dose escalation pilot study.

Conditions

Acute graft-versus-host disease of the skin

Brief summary

Definition of the Maximum Tolerated Dose (MTD) after 28 and 56 days (Phase 1)

Detailed description

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

Related papers

No related papers found yet.

Interventions

DRUGVenclyxto 10 mg film-coated tablets

DRUGVenclyxto 100 mg film-coated tablets

DRUGVenclyxto 50 mg film-coated tablets

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Definition of the Maximum Tolerated Dose (MTD) after 28 and 56 days (Phase 1)	—

Secondary

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

Measure

Time frame

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

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Countries

Austria

Outcome results

None listed