Hematological MalignanciesDRP_ZHNCT06550102
Drug Response Profiling (DRP) Registry Zurich for Hematological Malignancies
An observational study of Hematological Malignancies, run by University Children's Hospital, Zurich at 1 site in Switzerland.
Trial summary
This study is a prospective, non-randomized feasibility study of drug response profiling (DRP) in pediatric blood cancers. Primary cancer cells are isolated from patients and screened ex vivo at single-cell resolution using automated fluorescence microscopy. Read more about this study
- Who is it for?
- Ages Up to 40 years
- Conditions the study is for
- Hematological Malignancies
- How many people
- 1,000 participants
- Where
- 1 siteSwitzerland
- Study dates
- 4 Jan 2022to 31 Dec 2031
- How long will I take part?
- 10 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Up to 40 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Pediatric and adult patients with hematological malignancies who have undergone bone marrow aspiration, peripheral blood or lymph node biopsies as part of routine diagnostics.
Who may be able to join
- Pediatric and adult patients below the age of 40 years
- Diagnosis of hematological malignancy (primary, relapsed or refractory) including leukemia, myeloma or lymphoma
- Tumor material collected as part of routine diagnostics and willingness to donate tumor material for translational research
- Patient and/or guardian has signed the informed consent of the DRP registry or of a clinical trial which includes DRP as add-on research.
Who cannot join
- Missing informed consent for the registry or of a clinical trial which includes DRP as add-on research
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Switzerland1 site, 1 recruiting
- Recruiting
University Children's Hospital Zurich
Zurich, Canton of Zurich, 8032
Beat Bornhauser, PhD, 0041 442497055, Beat.Bornhauser@kispi.uzh.ch
Nastassja Scheidegger, MD
About this study
This study is a prospective, non-randomized feasibility study of drug response profiling (DRP) in pediatric blood cancers. Primary cancer cells are isolated from patients and screened ex vivo at single-cell resolution using automated fluorescence microscopy. Drug sensitivity fingerprints are integrated with genetic annotations to inform the treating physician about personalized treatment options. The study aims to determine the practicability of real-time drug response profiling and its actionability in identifying patient-specific cancer dependencies in refractory disease settings.
Read the detailed description
This observational study offers a platform to assess the drug sensitivity of primary leukemia cells ex vivo. The cancer cells are co-cultured in multi-well plates with supporting mesenchymal stroma cells and exposed to a library of both conventional (e.g. steroids, vincristine, asparaginase) as well as targeted chemotherapeutic agents (e.g. tyrosine kinase inhibitors, proteasome inhibitors, B-cell lymphoma 2 (BCL2) inhibitors). Cells are imaged in parallel by high-content microscopy and subsequently segmented and classified by morphology and surface antigen expression. Cell viability is quantified relative to dimethyl sulfoxide (DMSO) and as a function of drug concentration. From the measured cell counts drug-specific sensitivity parameters (e.g. half-maximal inhibitory concentration IC50, maximal inhibition Imax, area under the curve AUC) and their z-scores across the patient cohorts are calculated. Drug response profiles are correlated to clinical response after a steroid pre-phase at day 8 and multiple minimal residual disease (MRD) timepoints measured by flow cytometry (FCM) or polymerase chain reaction (PCR) as defined by the trial protocol. Data on clinical response to treatment and outcome will be enquired from the treating physician. These include the disease stage (initial diagnosis, 1st relapse, 2nd relapse) and time point of sample collection, the clinical trial and treatment arm the patient is enrolled in and/or any individualized drug treatments. Functional profiling data will be integrated with information about genetic lesions (e.g. tumor protein TP53), subtype-defining translocations such as the Philadelphia chromosome t(9;22)(q34;q11) and surface antigen expression (e.g. clusters of differentiation CD7/19/22/33/117). Cytogenetics and molecular profiling data are collected from the treating clinics in collaboration with the international relapsed acute lymphoblastic leukemia (IntReALL) study group and the international Berlin-Frankfurt-Münster (IBFM) network.
- Conditions
- Hematological Malignancies
- Keywords
- registry, DRP, acute lymphoblastic leukemia, blood cancer
- Medical subject headings
- Hematologic Neoplasms, Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Hematological malignancy | Patients witha diagnosis of hematological malignancy (primary, relapsed or refractory) including leukemia, myeloma or lymphoma |
What is being measured
The primary outcome is the main question the study is designed to answer.
Feasibility of real-time ex vivo drug screening
Rate of successful single-cell, image-based drug response profiling assays which are reported to the treating physician.
Measured: 10 days
Secondary outcomes (2)
Translation to clinic
Percentage of DRP assays translated into the clinic, i.e. the referring physician decides to treat the patient with one or more of the top-ranking drugs identified by DRP
Measured: 10 day
In vitro sensitivity of cancer cells to drug perturbations
Exploring drug sensitivity and resistance patterns by ex vivo DRP and correlations to genetic characteristics and clinical response.
Measured: 1 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Aug 2024
- Study start4 Jan 2022
- Primary completion31 Dec 2031 (estimated)
- Study completion31 Dec 2031 (estimated)
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 Hematological MalignanciesStudies 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
- 1,000 participants (estimated)
- Target duration
- 10 Years
Sponsor and investigators
- Lead sponsor
- University Children's Hospital, Zurich
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jean-Pierre Bourquin, MD, PhD, Study chair, University Children's Hospital Zurich
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06550102
- Sponsor's protocol ID
- 2021-02189
- Acronym
- DRP_ZH
- Status last verified
- Aug 2024
- First submitted
- 8 Aug 2024
- Last updated
- 15 Aug 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Frismantas V, Dobay MP, Rinaldi A, Tchinda J, Dunn SH, Kunz J, Richter-Pechanska P, Marovca B, Pail O, Jenni S, Diaz-Flores E, Chang BH, Brown TJ, Collins RH, Uhrig S, Balasubramanian GP, Bandapalli OR, Higi S, Eugster S, Voegeli P, Delorenzi M, Cario G, Loh ML, Schrappe M, Stanulla M, Kulozik AE, Muckenthaler MU, Saha V, Irving JA, Meisel R, Radimerski T, Von Stackelberg A, Eckert C, Tyner JW, Horvath P, Bornhauser BC, Bourquin JP. Ex vivo drug response profiling detects recurrent sensitivity patterns in drug-resistant acute lymphoblastic leukemia. Blood. 2017 Mar 16;129(11):e26-e37. doi: 10.1182/blood-2016-09-738070. Epub 2017 Jan 25.
PubMed 28122742
Study at a glance
From the registry record, last updated by the sponsor on 15 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Drug Response Profiling (DRP) Registry Zurich for Hematological Malignancies
- ClinicalTrials.gov ID
- NCT06550102
- Acronym / sponsor ID
- DRP_ZH / 2021-02189
- Conditions
- Hematological Malignancies
- Keywords
- registry, DRP, acute lymphoblastic leukemia, blood cancer
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 1,000 participants (estimated)
- Lead sponsor
- University Children's Hospital, ZurichUniversity, hospital or other
- Study start
- 4 Jan 2022 (actual)
- Primary completion
- 31 Dec 2031 (estimated)
- Study completion
- 31 Dec 2031 (estimated)
- First posted
- 12 Aug 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 study is a prospective, non-randomized feasibility study of drug response profiling (DRP) in pediatric blood cancers. Primary cancer cells are isolated from patients and screened ex vivo at single-cell resolution using automated fluorescence microscopy. Drug sensitivity fingerprints are integrated with genetic annotations to inform the treating physician about personalized treatment options. The study aims to determine the practicability of real-time drug response profiling and its actionability in identifying patient-specific cancer dependencies in refractory disease settings.
Detailed description
This observational study offers a platform to assess the drug sensitivity of primary leukemia cells ex vivo. The cancer cells are co-cultured in multi-well plates with supporting mesenchymal stroma cells and exposed to a library of both conventional (e.g. steroids, vincristine, asparaginase) as well as targeted chemotherapeutic agents (e.g. tyrosine kinase inhibitors, proteasome inhibitors, B-cell lymphoma 2 (BCL2) inhibitors). Cells are imaged in parallel by high-content microscopy and subsequently segmented and classified by morphology and surface antigen expression. Cell viability is quantified relative to dimethyl sulfoxide (DMSO) and as a function of drug concentration. From the measured cell counts drug-specific sensitivity parameters (e.g. half-maximal inhibitory concentration IC50, maximal inhibition Imax, area under the curve AUC) and their z-scores across the patient cohorts are calculated. Drug response profiles are correlated to clinical response after a steroid pre-phase at day 8 and multiple minimal residual disease (MRD) timepoints measured by flow cytometry (FCM) or polymerase chain reaction (PCR) as defined by the trial protocol. Data on clinical response to treatment and outcome will be enquired from the treating physician. These include the disease stage (initial diagnosis, 1st relapse, 2nd relapse) and time point of sample collection, the clinical trial and treatment arm the patient is enrolled in and/or any individualized drug treatments. Functional profiling data will be integrated with information about genetic lesions (e.g. tumor protein TP53), subtype-defining translocations such as the Philadelphia chromosome t(9;22)(q34;q11) and surface antigen expression (e.g. clusters of differentiation CD7/19/22/33/117). Cytogenetics and molecular profiling data are collected from the treating clinics in collaboration with the international relapsed acute lymphoblastic leukemia (IntReALL) study group and the international Berlin-Frankfurt-Münster (IBFM) network.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 10 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hematological malignancy | – | –Patients witha diagnosis of hematological malignancy (primary, relapsed or refractory) including leukemia, myeloma or lymphoma |
Who may qualify
- Ages
- Up to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Pediatric and adult patients with hematological malignancies who have undergone bone marrow aspiration, peripheral blood or lymph node biopsies as part of routine diagnostics.
Key inclusion criteria (4 listed)
- Pediatric and adult patients below the age of 40 years
- Diagnosis of hematological malignancy (primary, relapsed or refractory) including leukemia, myeloma or lymphoma
- Tumor material collected as part of routine diagnostics and willingness to donate tumor material for translational research
- Patient and/or guardian has signed the informed consent of the DRP registry or of a clinical trial which includes DRP as add-on research.
Key exclusion criteria (1 listed)
- Missing informed consent for the registry or of a clinical trial which includes DRP as add-on research
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Feasibility of real-time ex vivo drug screening
Time frame: 10 days
Rate of successful single-cell, image-based drug response profiling assays which are reported to the treating physician.
2 secondary outcomes
Translation to clinic
Time frame: 10 day
In vitro sensitivity of cancer cells to drug perturbations
Time frame: 1 month
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Aug 2024.
- Background: PubMed 28122742Frismantas V, Dobay MP, Rinaldi A, Tchinda J, Dunn SH, Kunz J, Richter-Pechanska P, Marovca B, Pail O, Jenni S, Diaz-Flores E, Chang BH, Brown TJ, Collins RH, Uhrig S, Balasubramanian GP, Bandapalli OR, Higi S, Eugster S, Voegeli P, Delorenzi M, Cario G, Loh ML, Schrappe M, Stanulla M, Kulozik AE, Muckenthaler MU, Saha V, Irving JA, Meisel R, Radimerski T, Von Stackelberg A, Eckert C, Tyner JW, Horvath P, Bornhauser BC, Bourquin JP. Ex vivo drug response profiling detects recurrent sensitivity patterns in drug-resistant acute lymphoblastic leukemia. Blood. 2017 Mar 16;129(11):e26-e37. doi: 10.1182/blood-2016-09-738070. Epub 2017 Jan 25.
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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.
Jean-Pierre Bourquin, MD, PhD study chair
University Children's Hospital Zurich
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hematological Malignancies, run by University Children's Hospital, Zurich at 1 site 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 1 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: looking for participants now, as of the sponsor's last update on 15 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jean-Pierre Bourquin, MD, PhD (0041 44 2667304, Jean-Pierre.Bourquin@kispi.uzh.ch), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Feasibility of real-time ex vivo drug screening (time frame: 10 days). 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 NCT06550102, 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 15 Aug 2024; AzTrials last refreshed its copy on 26 Oct 2026.