AML, Adult
Conditions
Keywords
relapsed / refractory AML, Pharmacoscopy, Functional precision medicine
Brief summary
With an overall survival of below 12 months, the outcome of relapsed/refractory AML (RR AML) is poor, making it a critical challenge to identify effective therapies at this stage. The RAPID-01 trial aims to show for the first time in a randomized and controlled clinical trial that Pharmacoscopy (PCY), a functional precision medicine platform, helps improve clinical standard-of-care treatment selection for patients suffering from relapsed/refractory AML.
Interventions
DIAGNOSTIC_TESTPharmacoscopy
Pharmacoscopy (PCY) is an image-based ex vivo drug testing platform developed by the Snijder lab at the ETH Zurich. PCY measures in the drug response of patient cells from small biopsies using automated microscopy and single-cell image analysis. PCY prioritizes treatments based on their specific efficacy against AML cells, while minimizing toxicity to healthy (non-malignant) cells in the patient biopsy.
DRUGClinical standard-of-care (physician's choice)
Clinical standard-of-care therapy for RR AML selected by the physician (physician's choice).
Sponsors
University of Zurich
Insel Gruppe AG, University Hospital Bern
ETH Zurich
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
DIAGNOSTIC
Masking
NONE
Intervention model description
This is a randomised, controlled, parallel group, superiority interventional trial.
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
* Patient with refractory or relapsed AML according to ELN2022 criteria. * Age 18-70 years. * Considered to be eligible for intensive chemotherapy. * Written informed consent.
Exclusion criteria
* Acute promyelocytic leukemia (APL) with PML-RARA or one of the other pathognomonic variant fusion genes/chromosome translocations. * Blast crisis after chronic myeloid leukemia (CML). * Considered not eligible for intensive chemotherapy. * Condition of the patient does not allow to wait for PCY results (patient requires immediate treatment). * PCY not working / patient sample did not pass the QC steps of PCY. * Any other serious underlying medical, psychiatric, psychological, familial or geographical condition, which in the judgment of the principal investigator may interfere with the project or affect patient compliance. * Legal incompetence or Subjects lacking capacity to provide informed consent. * Participation in a clinical trial with an investigational drug within the 30 days preceding and during the present investigation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Complete response (CR) rate at day 28 | day 28 |
Secondary
| Measure | Time frame |
|---|---|
| Composite response rate (CR+CRh+CRi) at day 28 | day 28 |
| Rate of patients bridged to allogeneic hematopoietic stem cell transplantation within 3 months post-treatment initiation | 3 months |
| Treatment-related mortality within 3 months post-treatment initiation | 3 months |
Countries
Switzerland
Contacts
Primary ContactBerend Snijder, PhD
Outcome results
None listed