Clinical trial NCT06138990: Pharmacoscopy-guided Clinical Standard-of-care in r/r AML
Official title: Pharmacoscopy-guided Clinical Standard-of-care in Relapsed/Refractory Acute Myeloid Leukemia, a Randomized Phase-2 Clinical Trial
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: AML, Adult
Keywords: relapsed / refractory AML
Pharmacoscopy
Functional precision medicine
Lead sponsor: ETH Zurich
Sponsor class: OTHER
Collaborators: name: University of Zurich
name: Insel Gruppe AG, University Hospital Bern
First posted: 2023-11-18
Enrollment: 88
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: This is a randomised, controlled, parallel group, superiority interventional trial.
Primary purpose: DIAGNOSTIC

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Complete response (CR) rate at day 28
timeFrame: day 28
Secondary outcomes:
measure: Composite response rate (CR+CRh+CRi) at day 28
timeFrame: day 28
measure: Rate of patients bridged to allogeneic hematopoietic stem cell transplantation within 3 months post-treatment initiation
timeFrame: 3 months
measure: Treatment-related mortality within 3 months post-treatment initiation
timeFrame: 3 months
Other outcomes:
Locations:
city: Bern
country: Switzerland
facility: Inselspital Bern
city: Zurich
country: Switzerland
facility: University Hospital Zurich
Study officials:
affiliation: University of Zurich
name: Alexandre Theocharides, MD PhD
role: PRINCIPAL_INVESTIGATOR
References:
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.
citation: 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.
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. 2024 Feb 1;109(2):617-621. doi: 10.3324/haematol.2023.283224. No abstract available.