RecruitingNCT06138990RAPID-01Updated May 16, 2025

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

A Phase 2 interventional study of Pharmacoscopy and Clinical standard-of-care (physician's choice) in AML, Adult, sponsored by ETH Zurich. Recruiting at 2 sites in Switzerland. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2025-05-16.

Sponsored by ETH Zurich · Phase 2, Interventional, and Diagnostic

Phase: Phase 2
Study type: Interventional
Enrollment: 88
Allocation: Randomized
Ages: 18 Years to 70 Years
Sex: All

Study 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.

Conditions studied

AML, Adult

Keywords

relapsed / refractory AML
Pharmacoscopy
Functional precision medicine

Who can participate

Ages eligible: 18 Years to 70 Years
Sexes eligible: All
Accepts 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.

Study design

Phase: Phase 2
Primary purpose: Diagnostic
Allocation: Randomized
Intervention model: Parallel assignment
Masking: None (open label)
Enrollment: 88 participants (estimated)

Study arms

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
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)

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).

What researchers measure

Primary outcomes

Complete response (CR) rate at day 28
Time frame: day 28

Secondary outcomes

Composite response rate (CR+CRh+CRi) at day 28
Time frame: day 28
Rate of patients bridged to allogeneic hematopoietic stem cell transplantation within 3 months post-treatment initiation
Time frame: 3 months
Treatment-related mortality within 3 months post-treatment initiation
Time frame: 3 months

Study locations

2 of 2 sites recruiting

Inselspital Bern
Bern, 3010, Switzerland
- Thomas Pabst, Prof. Dr. med · Contact · Thomas.Pabst@insel.ch · +41 31 632 41 14
Recruiting
University Hospital Zurich
Zurich, 8091, Switzerland
- Alexandre Theocharides, Prof. Dr. med · Contact · alexandre.theocharides@usz.ch · +41 44 255 37 82
Recruiting

References and documents

Publications

Snijder B, Vladimer GI, Krall N, Miura K, Schmolke AS, Kornauth C, Lopez de la Fuente O, Choi HS, van der Kouwe E, Gultekin S, Kazianka L, Bigenzahn JW, Hoermann G, Prutsch N, Merkel O, Ringler A, Sabler M, Jeryczynski G, Mayerhoefer ME, Simonitsch-Klupp I, Ocko K, Felberbauer F, Mullauer L, Prager GW, Korkmaz B, Kenner L, Sperr WR, Kralovics R, Gisslinger H, Valent P, Kubicek S, Jager U, Staber PB, Superti-Furga G. Image-based ex-vivo drug screening for patients with aggressive haematological malignancies: interim results from a single-arm, open-label, pilot study. Lancet Haematol. 2017 Dec;4(12):e595-e606. doi: 10.1016/S2352-3026(17)30208-9. Epub 2017 Nov 15. PubMed 29153976 ↗
Kornauth C, Pemovska T, Vladimer GI, Bayer G, Bergmann M, Eder S, Eichner R, Erl M, Esterbauer H, Exner R, Felsleitner-Hauer V, Forte M, Gaiger A, Geissler K, Greinix HT, Gstottner W, Hacker M, Hartmann BL, Hauswirth AW, Heinemann T, Heintel D, Hoda MA, Hopfinger G, Jaeger U, Kazianka L, Kenner L, Kiesewetter B, Krall N, Krajnik G, Kubicek S, Le T, Lubowitzki S, Mayerhoefer ME, Menschel E, Merkel O, Miura K, Mullauer L, Neumeister P, Noesslinger T, Ocko K, Ohler L, Panny M, Pichler A, Porpaczy E, Prager GW, Raderer M, Ristl R, Ruckser R, Salamon J, Schiefer AI, Schmolke AS, Schwarzinger I, Selzer E, Sillaber C, Skrabs C, Sperr WR, Srndic I, Thalhammer R, Valent P, van der Kouwe E, Vanura K, Vogt S, Waldstein C, Wolf D, Zielinski CC, Zojer N, Simonitsch-Klupp I, Superti-Furga G, Snijder B, Staber PB. Functional Precision Medicine Provides Clinical Benefit in Advanced Aggressive Hematologic Cancers and Identifies Exceptional Responders. Cancer Discov. 2022 Feb;12(2):372-387. doi: 10.1158/2159-8290.CD-21-0538. Epub 2021 Oct 11. PubMed 34635570 ↗
Schmid JA, Festl Y, Severin Y, Bacher U, Kronig MN, Snijder B, Pabst T. Efficacy and feasibility of pharmacoscopy-guided treatment for acute myeloid leukemia patients who have exhausted all registered therapeutic options. Haematologica. 2024 Feb 1;109(2):617-621. doi: 10.3324/haematol.2023.283224. No abstract available. PubMed 37439341 ↗

Individual participant data

Plan to share: Yes — De-identified patient data will be made available upon publication of the study results in a peer-reviewed internationally recognized journal.

Supporting information: Csr

Registry details

Key details

Study ID: NCT06138990
Lead sponsor: ETH Zurich
Collaborators: University of Zurich, Insel Gruppe AG, University Hospital Bern
Responsible party: Sponsor
First posted: Nov 18, 2023
Start date: Sep 2, 2024
Primary completion: Jun 30, 2026 (estimated)
Completion: Jun 30, 2026 (estimated)
Last update: May 16, 2025

Study contacts

Berend Snijder, PhD

Contact

bsnijder@ethz.ch

+41 44 633 71 49

Alexandre Theocharides, MD PhD

principal investigator · University of Zurich

Oversight

Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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