RAPID-01: Pharmacoscopy-guided Clinical Standard-of-care in r/r AML

ETH Zurich (Other)
Overall Status
Not yet recruiting
CT.gov ID
University of Zurich (Other)

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
  • Diagnostic Test: Pharmacoscopy
  • Drug: Clinical standard-of-care (physician's choice)

Study Design

Study Type:
Anticipated Enrollment :
82 participants
Intervention Model:
Parallel Assignment
Intervention Model Description:
This is a randomised, controlled, parallel group, superiority interventional trial.This is a randomised, controlled, parallel group, superiority interventional trial.
None (Open Label)
Primary Purpose:
Official Title:
Pharmacoscopy-guided Clinical Standard-of-care in Relapsed/Refractory Acute Myeloid Leukemia, a Randomized Phase-2 Clinical Trial
Anticipated Study Start Date :
Feb 1, 2024
Anticipated Primary Completion Date :
Oct 31, 2025
Anticipated Study Completion Date :
Dec 31, 2025

Arms and Interventions

Arm Intervention/Treatment
Experimental: Intervention arm: Pharmacoscopy-guided clinical standard-of-care

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
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.

Active Comparator: Control arm

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)
Clinical standard-of-care therapy for RR AML selected by the physician (physician's choice).

Outcome Measures

Primary Outcome Measures

  1. Complete response (CR) rate at day 28 [day 28]

Secondary Outcome Measures

  1. Composite response rate (CR+CRh+CRi) at day 28 [day 28]

  2. Rate of patients bridged to allogeneic hematopoietic stem cell transplantation within 3 months post-treatment initiation [3 months]

  3. Treatment-related mortality within 3 months post-treatment initiation [3 months]

Eligibility Criteria


Ages Eligible for Study:
18 Years to 70 Years
Sexes Eligible for Study:
Accepts Healthy Volunteers:

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.

Contacts and Locations


No locations specified.

Sponsors and Collaborators

  • ETH Zurich
  • University of Zurich


  • Principal Investigator: Alexandre Theocharides, MD PhD, University of Zurich

Study Documents (Full-Text)

None provided.

More Information

Additional Information:


Responsible Party:
ETH Zurich
ClinicalTrials.gov Identifier:
Other Study ID Numbers:
  • RAPID-01
First Posted:
Nov 18, 2023
Last Update Posted:
Nov 22, 2023
Last Verified:
Nov 1, 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:
Studies a U.S. FDA-regulated Drug Product:
Studies a U.S. FDA-regulated Device Product:
Keywords provided by ETH Zurich

Study Results

No Results Posted as of Nov 22, 2023