AML, AdultPhase 2RAPID-01NCT06138990
Pharmacoscopy-guided Clinical Standard-of-care in r/r AML
A Phase 2 study testing Pharmacoscopy and Clinical standard-of-care (physician's choice) in people with AML, Adult, run by ETH Zurich at 2 sites in Switzerland.
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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- AML, Adult
- What is being tested?
- Pharmacoscopy and Clinical standard-of-care (physician's choice)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 88 participants
- Where
- 2 sitesSwitzerland
- Study dates
- 2 Sep 2024to 30 Jun 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient with refractory or relapsed AML according to ELN2022 criteria.
- Age 18-70 years.
- Considered to be eligible for intensive chemotherapy.
- Written informed consent.
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
Switzerland2 sites, 2 recruiting
- Recruiting
Inselspital Bern
Bern, Canton of Bern, 3010
Thomas Pabst, Prof. Dr. med, +41 31 632 41 14, Thomas.Pabst@insel.ch
- Recruiting
University Hospital Zurich
Zurich, Canton of Zurich, 8091
Alexandre Theocharides, Prof. Dr. med, +41 44 255 37 82, alexandre.theocharides@usz.ch
About this study
Official title: Pharmacoscopy-guided Clinical Standard-of-care in Relapsed/Refractory Acute Myeloid Leukemia, a Randomized Phase-2 Clinical Trial
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
- AML, Adult
- Being tested
- Pharmacoscopy, Clinical standard-of-care (physician's choice)
- Keywords
- relapsed / refractory AML, Pharmacoscopy, Functional precision medicine
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PharmacoscopyDiagnostic test
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.
Clinical standard-of-care (physician's choice)Drug
Clinical standard-of-care therapy for RR AML selected by the physician (physician's choice).
| Study group | Role | What they receive |
|---|---|---|
| Intervention arm: Pharmacoscopy-guided clinical standard-of-care | Experimental | 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. Receives: Diagnostic Test: Pharmacoscopy |
| Control arm | Active comparator | Patients in the control arm will be treated with clinical standard-of-care therapy for RR AML selected by the physician (physician's choice). Receives: Drug: Clinical standard-of-care (physician's choice) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete response (CR) rate at day 28
Measured: day 28
Secondary outcomes (3)
Composite response rate (CR+CRh+CRi) at day 28
Measured: day 28
Rate of patients bridged to allogeneic hematopoietic stem cell transplantation within 3 months post-treatment initiation
Measured: 3 months
Treatment-related mortality within 3 months post-treatment initiation
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2023
- Study start2 Sep 2024
- Primary completion30 Jun 2026 (estimated)
- Study completion30 Jun 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for AML, AdultStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Diagnosis
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This is a randomised, controlled, parallel group, superiority interventional trial.
- Masking
- None (open label)
- Enrolment
- 88 participants (estimated)
Sponsor and investigators
- Lead sponsor
- ETH Zurich
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Alexandre Theocharides, MD PhD, Principal investigator, University of Zurich
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06138990
- Sponsor's protocol ID
- RAPID-01
- Acronym
- RAPID-01
- Status last verified
- Sep 2024
- First submitted
- 14 Nov 2023
- Last updated
- 16 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: De-identified patient data will be made available upon publication of the study results in a peer-reviewed internationally recognized journal.
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 29153976Kornauth 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 34635570Schmid 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
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pharmacoscopy-guided Clinical Standard-of-care in Relapsed/Refractory Acute Myeloid Leukemia, a Randomized Phase-2 Clinical Trial
- ClinicalTrials.gov ID
- NCT06138990
- Acronym / sponsor ID
- RAPID-01 / RAPID-01
- Conditions
- AML, Adult
- Keywords
- relapsed / refractory AML, Pharmacoscopy, Functional precision medicine
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 88 participants (estimated)
- Lead sponsor
- ETH ZurichUniversity, hospital or other
- Collaborators
- University of Zurich, Insel Gruppe AG, University Hospital Bern
- Study start
- 2 Sep 2024 (actual)
- Primary completion
- 30 Jun 2026 (estimated)
- Study completion
- 30 Jun 2026 (estimated)
- First posted
- 18 Nov 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- This is a randomised, controlled, parallel group, superiority interventional trial.
| Arm | Type | Receives |
|---|---|---|
| Intervention arm: Pharmacoscopy-guided clinical standard-of-care | Experimental | Diagnostic Test: PharmacoscopyPatients 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. |
| Control arm | Active comparator | Drug: Clinical standard-of-care (physician's choice)Patients in the control arm will be treated with clinical standard-of-care therapy for RR AML selected by the physician (physician's choice). |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Patient with refractory or relapsed AML according to ELN2022 criteria.
- Age 18-70 years.
- Considered to be eligible for intensive chemotherapy.
- Written informed consent.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PharmacoscopyPharmacoscopy (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. | Diagnostic test | Intervention arm: Pharmacoscopy-guided clinical standard-of-care |
| Clinical standard-of-care (physician's choice)Clinical standard-of-care therapy for RR AML selected by the physician (physician's choice). | Drug | Control arm |
Primary outcome
Complete response (CR) rate at day 28
Time frame: day 28
3 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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 May 2025.
- Background: PubMed 29153976Snijder 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.
- Background: PubMed 34635570Kornauth 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.
- Background: PubMed 37439341Schmid 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.
- Snijder Lab websiteLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. De-identified patient data will be made available upon publication of the study results in a peer-reviewed internationally recognized journal.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alexandre Theocharides, MD PhD principal investigator
University of Zurich
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pharmacoscopy and Clinical standard-of-care (physician's choice) in people with AML, Adult, run by ETH Zurich at 2 sites in Switzerland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 16 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Switzerland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Berend Snijder, PhD (+41 44 633 71 49, bsnijder@ethz.ch), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Complete response (CR) rate at day 28 (time frame: day 28). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06138990, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 May 2025.