Acute Myeloid Leukemia (AML)EXCYTE-2NCT06648512
Association of Ex Vivo Drug Response ( EVDR) and Clinical Outcome in Acute Myeloid Leukaemia (EXCYTE-2)
An observational study of Acute Myeloid Leukemia (AML) and Acute Myeloid Leukemia (AML) Relapse, run by Exscientia AI Limited at 3 sites in 3 countries.
Trial summary
This is a multicentre study on biobanked bone marrow and blood samples of AML patients, conducted by Exscientia GmbH. The study aims to compare the drug response measured 'ex vivo', this means outside of the body, in the samples and the documented outcome of the respective patient's clinical treatment. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia (AML), Acute Myeloid Leukemia (AML) Relapse
- How many people
- 91 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 13 May 2024to 7 Mar 2025
- Phase
- Observational
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: This study is planning to include biobanked samples of the following patient populations: 1. Newly diagnosed fit AML patients (excl. APL), defined as treated with Cytarabine + Daunorubicin (classical 7+3 or CPX-351) independent or combination with GO or a TKI (e.g., Midostaurin). 2. Newly diagnosed unfit AML patients, defined as treated with Venetoclax and Azacitidine (alone or in combination). Upon favourable results in an interim analysis, R/R AML FLT3 mutant patients, treated with Gilteritinib, for which we expect different covariates exist, might also be included.
Who may be able to join
- Patients aged 18 years or older.
- Signed informed consent form that permits use of sample in the proposed study (including permission for genetic analysis).
- Sample of a newly diagnosed patient (sample at time point diagnosis); after interim analysis the population may include samples from patients with FLT3 mutated relapsed/refractory AML.
- The sample may not be older than 5/ 10 years (depending on location)
- Confirmed diagnosed AML according to WHO or ICC criteria; after interim analysis the population may include FLT3 mutated R/R AML according to ELN 2022 criteria after interim analysis.
- Patient received one of the following therapies after sampling for which response data is available:
Show all 12Show fewer
- 7 + 3 (with or without additional GO or TKIs)
- CPX-351
- Venetoclax and AZA in combination or alone
- If R/R AML, FLT3 mutated: treated with Gilteritinib
- Characteristics of sample taken prior to therapy as specified in the study protocol.
- Availability of complete dataset as specified in the study protocol.
Who cannot join
- Known or suspected HIV or active Hepatitis B and/or C infection or active COVID-19 infection (if information not available, samples can still be included) at time of sample collection.
- Known active infection of bone marrow.
- Known pregnancy.
- Received systemic anticancer treatment or radiotherapy within 4 weeks of sampling (pre-treatment of hydroxyurea and/or low dose cytarabine allowed).
- Patient is diagnosed with Acute Promyelocytic Leukaemia (APL).
- Patients with treatment for any other oncologic neoplasm at time of sample collection.
- Patients for whom CR could not be assessed (e.g. death before re-staging).
- Inclusion of samples from the same patient at both diagnosis and relapse is not permitted. In such cases, only the diagnostic sample is to be used.
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
Finland1 site
FHRB (Finnish Hematology Registry and Clinical Biobank)
Vantaa, 01730
Germany1 site
Charité Universitätsmedizin Berlin, Campus Benjamin Franklin, Klinik für Hämatologie und Onkologie
Berlin, 12203
About this study
Official title: Prospective Analysis of the Association of Drug Activity Measured in Viable Tumour Tissues ex Vivo and Clinical Response in Acute Myeloid Leukaemia (EXCYTE-2)
This is a multicentre study on biobanked bone marrow and blood samples of AML patients, conducted by Exscientia GmbH. The study aims to compare the drug response measured 'ex vivo', this means outside of the body, in the samples and the documented outcome of the respective patient's clinical treatment. To measure this Ex Vivo Drug Response (EVDR), Exscientia will use it's AI-( Artificial Intelligence)-based precision medicine platform. In this platform, the cells of each sample are split and distributed in a number of small vials, to which different approved or experimental AML drugs are added. The cells are left with the drugs for a certain period of time (no culturing or expansion is done). After that, the cells are stained (coloured) by using specific dyes and the rates of dead cancer cells in each of these small vials is determined via automated microscopy. The EVDR shows how well the drugs killed the cancer cells in the sample. Taking clinical data into account, which is information on e.g. the patients health status or genetic markers, the EVDR could reveal which patients might especially benefit from the treatment.
If a reproducible correlation between the EVDR and the patient's clinical treatment outcome is found, the scFDS platform could be used in the future to improve treatment selection for AML patients.
Read more
The study will include biobanked samples from newly diagnosed patients, treated with cytarabine + daunorubicin (classical 7+3 or CPX-351) or venetoclax + azacitidine and after favourable results in an interim analysis, biobanked samples from R/R AML FLT3 mutant patients, treated with Gilteritinib might be included.
Key procedures include:
- Viable tumour tissues (i.e. bone marrow or blood) taken prior to therapy are provided by biobanks to Exscientia's central lab (or delegated central laboratory),
- Ex vivo drug response against commonly given standard of care drugs is evaluated in viable tumour tissues, Exscientia-owned drug candidates might be included in the assay for pre-clinical testing.
- Clinical patient data are collected,
- Relationship of EVDR to clinical response is evaluated.
Primary key hypothesis: Ex vivo drug response (EVDR) is significantly associated with Complete Response (CR).
Secondary key hypothesis: EVDR predicts achieving CR with 80% sensitivity and specificity.
The outcome of this observational clinical study will have broad implications both for the clinical routine, preclinical drug development, and translational cancer research. If a robust correlation between drug response measured ex vivo in tumour samples and clinical outcome can be identified, this will pave the way for:
- the use of functional drug testing as a tool for personalised treatment decision making in the clinical routine, in particular where classical molecular precision medicine approaches fail to prioritise effective therapies, and
- the use of human tumour samples as clinically relevant model systems for preclinical development of new drugs and translational cancer research that can potentially overcome the limited clinical relevance of mouse and other animal models.
- Being tested
- No Interventions
- Keywords
- AML, ex vivo, drug response, biobank
- Medical subject headings
- Leukemia, Myeloid, Acute, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionsOther
No Intervention
| Study group | Role | What they receive |
|---|---|---|
| Newly diagnosed | Biobanked samples from AML patients taken at timepoint of initial diagnosis, before start of initial AML treatment. Receives: Other: No Interventions | |
| R/R AML (FLT3-mutated) | Biobanked samples from AML patients at relaps or refractory diagnosis (only if FLT3-mutated)) Receives: Other: No Interventions |
What is being measured
The primary outcome is the main question the study is designed to answer.
Association between ex vivo drug response (EVDR) and clinical outcome (Complete Remission)
To evaluate the association between EVDR measured using the Sponsor's scFDS platform in primary human AML samples and clinical outcomes, in particular complete remission, taking clinicopathological confounders into account.
Measured: From date of sample receipt until the date of CR or first documented therapy outcome evaluation, if CR is not applicable and scFDS full data set is available, assessed up to 18 months
Secondary outcomes (3)
Biomarker and ex vivo drug response association
Statistical association of established biomarkers (e.g. FLT3 mutations) and interaction between these markers and EVDR outcome.
Measured: From date of sample receipt until the date of CR or first documented therapy outcome evaluation, if CR is not applicable and scFDS full data set is available, assessed up to 18 months
Technical success rate
Percentage of patients meeting In-and Exclusion criteria as per protocol and for which an ex vivo drug response from sponsors single cell Functional drug Screening (scFDS) platform meeting Sponsor QC requirements could be obtained out of the total number of included patients.
Measured: From date of sample receipt until the date of CR or first documented therapy outcome evaluation, if CR is not applicable and scFDS full data set is available, assessed up to 18 months
Prognostic value of EVDR of bone marrow or blood sample in CR prediction
To characterise the accuracy, sensitivity and specificity of EVDR to predict clinical outcome based on optimal cut-offs. Specifically, we intend to evaluate the ability of EVDR to predict complete response with more than 80% sensitivity and specificity.
Measured: From date of sample receipt until the date of CR or first documented therapy outcome evaluation, if CR is not applicable and scFDS full data set is available, assessed up to 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2024
- Study start13 May 2024
- Primary completion7 Mar 2025
- Study completion7 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Acute Myeloid Leukemia (AML)Studies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 91 participants (actual)
Sponsor and investigators
- Lead sponsor
- Exscientia AI Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Nikolaus Krall, Dr., Study director, Exscientia GmbH
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06648512
- Sponsor's protocol ID
- AC2305
- Acronym
- EXCYTE-2
- Status last verified
- Aug 2025
- First submitted
- 14 Oct 2024
- Last updated
- 26 Aug 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
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
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Analysis of the Association of Drug Activity Measured in Viable Tumour Tissues ex Vivo and Clinical Response in Acute Myeloid Leukaemia (EXCYTE-2)
- ClinicalTrials.gov ID
- NCT06648512
- Acronym / sponsor ID
- EXCYTE-2 / AC2305
- Keywords
- AML, ex vivo, drug response, biobank
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 91 participants (actual)
- Lead sponsor
- Exscientia AI LimitedIndustry (pharma, biotech, devices)
- Study start
- 13 May 2024 (actual)
- Primary completion
- 7 Mar 2025 (actual)
- Study completion
- 7 Mar 2025 (actual)
- First posted
- 18 Oct 2024 (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.
This is a multicentre study on biobanked bone marrow and blood samples of AML patients, conducted by Exscientia GmbH. The study aims to compare the drug response measured 'ex vivo', this means outside of the body, in the samples and the documented outcome of the respective patient's clinical treatment. To measure this Ex Vivo Drug Response (EVDR), Exscientia will use it's AI-( Artificial Intelligence)-based precision medicine platform. In this platform, the cells of each sample are split and distributed in a number of small vials, to which different approved or experimental AML drugs are added. The cells are left with the drugs for a certain period of time (no culturing or expansion is done). After that, the cells are stained (coloured) by using specific dyes and the rates of dead cancer cells in each of these small vials is determined via automated microscopy. The EVDR shows how well the drugs killed the cancer cells in the sample. Taking clinical data into account, which is information on e.g. the patients health status or genetic markers, the EVDR could reveal which patients might especially benefit from the treatment.
If a reproducible correlation between the EVDR and the patient's clinical treatment outcome is found, the scFDS platform could be used in the future to improve treatment selection for AML patients.
The study will include biobanked samples from newly diagnosed patients, treated with cytarabine + daunorubicin (classical 7+3 or CPX-351) or venetoclax + azacitidine and after favourable results in an interim analysis, biobanked samples from R/R AML FLT3 mutant patients, treated with Gilteritinib might be included.
Key procedures include:
- Viable tumour tissues (i.e. bone marrow or blood) taken prior to therapy are provided by biobanks to Exscientia's central lab (or delegated central laboratory),
- Ex vivo drug response against commonly given standard of care drugs is evaluated in viable tumour tissues, Exscientia-owned drug candidates might be included in the assay for pre-clinical testing.
- Clinical patient data are collected,
- Relationship of EVDR to clinical response is evaluated.
Primary key hypothesis: Ex vivo drug response (EVDR) is significantly associated with Complete Response (CR).
Secondary key hypothesis: EVDR predicts achieving CR with 80% sensitivity and specificity.
The outcome of this observational clinical study will have broad implications both for the clinical routine, preclinical drug development, and translational cancer research. If a robust correlation between drug response measured ex vivo in tumour samples and clinical outcome can be identified, this will pave the way for:
- the use of functional drug testing as a tool for personalised treatment decision making in the clinical routine, in particular where classical molecular precision medicine approaches fail to prioritise effective therapies, and
- the use of human tumour samples as clinically relevant model systems for preclinical development of new drugs and translational cancer research that can potentially overcome the limited clinical relevance of mouse and other animal models.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Newly diagnosed | – | Other: No InterventionsBiobanked samples from AML patients taken at timepoint of initial diagnosis, before start of initial AML treatment. |
| R/R AML (FLT3-mutated) | – | Other: No InterventionsBiobanked samples from AML patients at relaps or refractory diagnosis (only if FLT3-mutated)) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- This study is planning to include biobanked samples of the following patient populations: 1. Newly diagnosed fit AML patients (excl. APL), defined as treated with Cytarabine + Daunorubicin (classical 7+3 or CPX-351) independent or combination with GO or a TKI (e.g., Midostaurin). 2. Newly diagnosed unfit AML patients, defined as treated with Venetoclax and Azacitidine (alone or in combination). Upon favourable results in an interim analysis, R/R AML FLT3 mutant patients, treated with Gilteritinib, for which we expect different covariates exist, might also be included.
Key inclusion criteria (12 listed)
- Patients aged 18 years or older.
- Signed informed consent form that permits use of sample in the proposed study (including permission for genetic analysis).
- Sample of a newly diagnosed patient (sample at time point diagnosis); after interim analysis the population may include samples from patients with FLT3 mutated relapsed/refractory AML.
- The sample may not be older than 5/ 10 years (depending on location)
- Confirmed diagnosed AML according to WHO or ICC criteria; after interim analysis the population may include FLT3 mutated R/R AML according to ELN 2022 criteria after interim analysis.
Key exclusion criteria (8 listed)
- Known or suspected HIV or active Hepatitis B and/or C infection or active COVID-19 infection (if information not available, samples can still be included) at time of sample collection.
- Known active infection of bone marrow.
- Known pregnancy.
- Received systemic anticancer treatment or radiotherapy within 4 weeks of sampling (pre-treatment of hydroxyurea and/or low dose cytarabine allowed).
- Patient is diagnosed with Acute Promyelocytic Leukaemia (APL).
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 |
|---|---|---|
| No InterventionsNo Intervention | Other | Newly diagnosed, R/R AML (FLT3-mutated) |
Primary outcome
Association between ex vivo drug response (EVDR) and clinical outcome (Complete Remission)
Time frame: From date of sample receipt until the date of CR or first documented therapy outcome evaluation, if CR is not applicable and scFDS full data set is available, assessed up to 18 months
To evaluate the association between EVDR measured using the Sponsor's scFDS platform in primary human AML samples and clinical outcomes, in particular complete remission, taking clinicopathological confounders into account.
3 secondary outcomes
Biomarker and ex vivo drug response association
Time frame: From date of sample receipt until the date of CR or first documented therapy outcome evaluation, if CR is not applicable and scFDS full data set is available, assessed up to 18 months
Technical success rate
Time frame: From date of sample receipt until the date of CR or first documented therapy outcome evaluation, if CR is not applicable and scFDS full data set is available, assessed up to 18 months
Prognostic value of EVDR of bone marrow or blood sample in CR prediction
Time frame: From date of sample receipt until the date of CR or first documented therapy outcome evaluation, if CR is not applicable and scFDS full data set is available, assessed up to 18 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 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.
- Individual participant dataSharing planned: No
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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.
Nikolaus Krall, Dr. study director
Exscientia GmbH
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Acute Myeloid Leukemia (AML) and Acute Myeloid Leukemia (AML) Relapse, run by Exscientia AI Limited at 3 sites in 3 countries.
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 12 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: finished, as of the sponsor's last update on 26 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Austria, Finland, Germany. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Association between ex vivo drug response (EVDR) and clinical outcome (Complete Remission) (time frame: From date of sample receipt until the date of CR or first documented therapy outcome evaluation, if CR is not applicable and scFDS full data set is available, assessed up to 18 months). 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 NCT06648512, which also lists 1 linked publication.
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 26 Aug 2025.