Pharmacoscopy-guided Clinical Standard-of-care in r/r AML

Study Overview
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Brief Summary
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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.

Official Title
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Pharmacoscopy-guided Clinical Standard-of-care in Relapsed/Refractory Acute Myeloid Leukemia, a Randomized Phase-2 Clinical Trial

Conditions
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AML, Adult

Intervention / Treatment
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* Diagnostic Test: Pharmacoscopy
* Drug: Clinical standard-of-care (physician's choice)


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Diagnostic
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: This is a randomised, controlled, parallel group, superiority interventional trial.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Intervention arm: Pharmacoscopy-guided clinical standard-of-care<br>Patients in the PCY-guided treatment arm will receive one of the clinical standard-of-care treatments suggested by their own PCY results, and confirmed by the treating physician. | Diagnostic Test: Pharmacoscopy<br>* 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.<br>|
| Active Comparator: Control arm<br>Patients in the control arm will be treated with clinical standard-of-care therapy for RR AML selected by the physician (physician's choice). | Drug: Clinical standard-of-care (physician's choice)<br>* Clinical standard-of-care therapy for RR AML selected by the physician (physician's choice).<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Complete response (CR) rate at day 28 |  | day 28 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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relapsed / refractory AML, Pharmacoscopy, Functional precision medicine