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

AML, Adult Clinical Trial

— RAPID-01  

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06138990
• Other study ID #: RAPID-01
• Status: Not yet recruiting
• Phase: N/A
• Start date: February 1, 2024
• Est. completion date: December 31, 2025

Study information

• Verified date: November 2023
• Source: ETH Zurich
• Contact: Berend Snijder, PhD
• Phone: +41 44 633 71 49
• Email: bsnijder[@]ethz.ch
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 82
• Est. completion date: December 31, 2025
• Est. primary completion date: October 31, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

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.

Related Conditions & MeSH terms

• AML, Adult

Intervention

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.

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

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
ETH Zurich	University of Zurich

References & Publications (3)

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. — View Citation

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. 2023 Jul 13. doi: 10.3324/haematol.2023.283224. Online ahead of print. — View Citation

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Complete response (CR) rate at day 28		day 28
Secondary	Composite response rate (CR+CRh+CRi) at day 28		day 28
Secondary	Rate of patients bridged to allogeneic hematopoietic stem cell transplantation within 3 months post-treatment initiation		3 months
Secondary	Treatment-related mortality within 3 months post-treatment initiation		3 months

External Links

•   Snijder Lab website