Drug-screening in AML at Relapse for Targeted Treatment

NCT ID: NCT05732688

Last Updated: 2024-07-08

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-01-01
• Study Completion Date: 2024-07-01

Brief Summary

This is a non-randomised clinical study investigating subsequent patients with specific AML treatment started between January 1, 2022 until December 31, 2022.

Patients with relapsing disease are planned to be analyzed in this study

Detailed Description

The standard treatment for young fit patients with acute myeloid leukemia (AML) is intensive chemotherapy followed by consolidation treatment with curative intent. Usually, two cycles of intensive chemotherapy are given, with subsequent consolidation treatment depending on the genetic risk-assessment of the patients as well as on the response to the induction treatment.

For elderly or unfit patients, such an intensive approach is not feasible, and palliative treatment must be considered. The standard first-line-treatment for such patients since more than a decade comprises repetitive cycles of a hypomethylating agent (either Azacitidine or Decitabine). The median progression free survival following these approaches in this population is between 4 and 8 months, with an overall-survival of up to 12 months. More recently, the addition of the Bcl-2 inhibitor Venetoclax to hypomethylating agents has led to a modest improvement both of progression-free and overall survival. However, overall survival in such patients usually does not exceed 14-16 months.

The laboratory of Prof. Berend Snijder, Institute of Molecular Systems Biology, at the ETH (Eidgenössische Technische Hochschule) Zurich has developed an image-based ex-vivo drug screening platform for patients with aggressive haematological malignancies, also called pharmacoscopy. Using such a technique, leukemic cells from a patient at relapse can be rapidly screened for sensitivity to single compounds. A drug score is calculated for each compound.

Starting in Q2/2021, the investigator at the Department of Medical Oncology, University Hospital Inselspital in Bern, collected experiences using such an approach. Having received information from the laboratory on top sensitivity of leukemic cells of a given patient to a specific drug, a process is initiated to try to obtain access to such off-label drugs.

Conditions

AML

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: SCREENING
• Blinding Strategy: NONE

Study Groups

Pharmacoscopy

Leukemic cells from a patient at relapse can be screened for sensitivity to single compounds

Group Type: EXPERIMENTAL

Image-based ex-vivo drug screening platform (pharmacoscopy)

Intervention Type: DIAGNOSTIC_TEST

Leukemic cells from a patient at relapse can be screened for sensitivity to single compounds. A drug score is calculated for each compound (defined as 1 - (% target cells in drug treated conditions / % target cells under control condition)). If a drug kills all target cells specifically, the best possible score is "1". If the drug is killing all non-target cells, the score goes to negative infinite. If a drug kills both target and non-target cell populations equally, or does nothing, the score is "0".

Interventions

Image-based ex-vivo drug screening platform (pharmacoscopy)

Leukemic cells from a patient at relapse can be screened for sensitivity to single compounds. A drug score is calculated for each compound (defined as 1 - (% target cells in drug treated conditions / % target cells under control condition)). If a drug kills all target cells specifically, the best possible score is "1". If the drug is killing all non-target cells, the score goes to negative infinite. If a drug kills both target and non-target cell populations equally, or does nothing, the score is "0".

Intervention Type: DIAGNOSTIC_TEST

Other Intervention Names

Pharmacoscopy

Eligibility Criteria

Inclusion Criteria

• Included are patients with AML at relapse treated at the Department of Medical Oncology at the University Hospital Inselspital in Bern.
• Patients are not planned to undergo intensive reinduction treatment with subsequent allogeneic hematopoietic transplantation in a curative intent.
• Patients have exhausted all standard therapeutic options and they must have no available licensed standard treatment for relapsed AML.
• Written informed consent

Exclusion Criteria

• Patients able to undergo intensive reinduction treatment with subsequent allogeneic hematopoietic transplantation in a curative intent
• Patients have available standard therapeutic options

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Insel Gruppe AG, University Hospital Bern

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Thomas Pabst, Prof Dr. med

Role: STUDY_CHAIR

Department for Medical Oncology; University Hospital/Inselspital

Locations

Departement of Medical Oncology, University Hospital Berne

Bern, , Switzerland

Countries

Switzerland

Other Identifiers

DARTT-1

Identifier Type: -

Identifier Source: org_study_id