Leukemia, Myeloid, AcutePERDAMNCT03549351
Evaluation of Measurable Residual Disease in Patients With Acute Myeloid Leukemia as Surrogate Endpoint for Survival
An observational study of Leukemia, Myeloid, Acute, run by University Hospital Heidelberg at 1 site in Germany.
Trial summary
Objectives To demonstrate that measurable residual disease assessed by multiparameter flow cytometry during intensive treatment is a surrogate for overall survival and thus an early read-out for drug efficacy Study design Surrogate endpoint trial to establish that measurable residual disease assessed by multiparameter… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Leukemia, Myeloid, Acute
- What is being tested?
- Measurable residual disease measured by flow cytometry
- How many people
- 51 participants
- Where
- 1 siteGermany
- Study dates
- 1 Jun 2019to 11 Sep 2022
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with AML participating in interventional prospective randomized trials of the Study Alliance Leukemia (SAL) including the Heidelberg Leukemia Network (HeLeNe)
Who may be able to join
- Acute myeloid leukemia according to the WHO classification
- Informed consent in place for a randomized study of the Study Alliance Leukemia (SAL) including the Heidelberg Leukemia Network (HeLeNe) covering assessment of MRD by MPFC in the reference laboratories in Heidelberg and Dresden.
Who cannot join
- No signed informed consent compliant with the requirements of PERDAM
Trial locations and contacts
1 site in 1 country.
Germany1 site
University Hospital Heidelberg
Heidelberg, Baden-Wurttemberg, 69124
About this study
Official title: Prospective Evaluation of Measurable Residual Disease in Intensively Treated Patients With Acute Myeloid Leukemia (AML) as Surrogate Endpoint for Survival
Objectives To demonstrate that measurable residual disease assessed by multiparameter flow cytometry during intensive treatment is a surrogate for overall survival and thus an early read-out for drug efficacy Study design Surrogate endpoint trial to establish that measurable residual disease assessed by multiparameter flow cytometry during intensive treatment is a surrogate for overall survival
Read the detailed description
Acute myeloid leukemia is a genetically and phenotypically heterogeneous disorder with an incidence of 3 to 4 per 100 000 men and women per year and a median age at diagnosis of about 70 years. Prognosis, especially in older patients, has remained very poor. In patients considered suitable for intensive chemotherapy, the combination of an anthracycline and cytarabine remains the standard of care. For patients achieving a complete remission (CR), postremission therapy (PRT) ranging from chemotherapy to allogeneic hematopoietic stem cell transplantation is required; intensive PRT is still under debate in older patients. Beyond pre-treatment genetics-based risk stratification, measurable residual disease (MRD) during treatment and follow up emerges as an important prognostic factor in first CR. Furthermore, MRD may provide a tool for a read-out of therapeutic efficacy. In this diagnostic meta-study the investigators intend to measure MRD using multiparameter flow cytometry across up-front randomized clinical trials which in total will accrue more than 1000 patients. According to the leukemia-associated phenotype at diagnosis or the different-from-normal approach, MRD will be assessed early (after induction) and late (after consolidation) during treatment. The aim of the study is to show that levels of MRD measured early during treatment are closely related to overall survival and thus may serve as an early surrogate. There is a growing public demand that new, promising drugs are approved for therapy as rapidly as possible. Therefore, it is of great interest to obtain these approvals based on early biomarker endpoints such as MRD rather than on long-term survival endpoints.
- Conditions
- Leukemia, Myeloid, Acute
- Being tested
- Measurable residual disease measured by flow cytometry
- Keywords
- Leukemia, Myeloid, Acute, measurable residual disease, surrogate endpoint, multiparameter flow cytometry
- Medical subject headings
- Leukemia, Myeloid, Acute, Neoplasm, Residual
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Measurable residual disease measured by flow cytometryOther
Measurable residual disease measured by flow cytometry
What is being measured
The primary outcome is the main question the study is designed to answer.
Correlation between Measurable Residual Disease (MRD) and Survival with respect to treatment effects
MRD will be assessed using multiparameter flow cytometry early (after induction / salvage).
Survival will be assessed continuously. After 3 and 6 years correlation between MRD and survival will be analysed with respect to treatment effects to assess if MRD may serve as surrogate endpoint.
If levels of MRD measured early during treatment are closely related to overall survival and thus may serve as an early surrogate will be assessed yearly.
Measured: after 3 and 6 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2018
- Study start1 Jun 2019
- Primary completion11 Sep 2022
- Study completion11 Sep 2022
Why it stopped: Study turned out no longer feasible
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leukemia, Myeloid, AcuteStudies of the same condition, open and under way.
- More trials run by University Hospital HeidelbergThe sponsor's other registered studies.
- 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
- 51 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital Heidelberg
- Sponsor type
- University, hospital or other
- Collaborators
- University Hospital Dresden, German Cancer Research Center
- Responsible party
- Principal investigator, Prof. Dr. Richard F Schlenk, University Hospital Heidelberg
- Investigators
- Richard F Schlenk, Prof. Dr., Principal investigator, University Hospital Heidelberg
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03549351
- Sponsor's protocol ID
- NCT-2018-0554
- Acronym
- PERDAM
- Status last verified
- Sep 2022
- First submitted
- 7 May 2018
- Last updated
- 13 Sep 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Evaluation of Measurable Residual Disease in Intensively Treated Patients With Acute Myeloid Leukemia (AML) as Surrogate Endpoint for Survival
- ClinicalTrials.gov ID
- NCT03549351
- Acronym / sponsor ID
- PERDAM / NCT-2018-0554
- Conditions
- Leukemia, Myeloid, Acute
- Keywords
- Leukemia, Myeloid, Acute, measurable residual disease, surrogate endpoint, multiparameter flow cytometry
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Study turned out no longer feasible
- Enrolment
- 51 participants (actual)
- Lead sponsor
- University Hospital HeidelbergUniversity, hospital or other
- Collaborators
- University Hospital Dresden, German Cancer Research Center
- Study start
- 1 Jun 2019 (actual)
- Primary completion
- 11 Sep 2022 (actual)
- Study completion
- 11 Sep 2022 (actual)
- First posted
- 8 Jun 2018 (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.
Objectives To demonstrate that measurable residual disease assessed by multiparameter flow cytometry during intensive treatment is a surrogate for overall survival and thus an early read-out for drug efficacy Study design Surrogate endpoint trial to establish that measurable residual disease assessed by multiparameter flow cytometry during intensive treatment is a surrogate for overall survival
Detailed description
Acute myeloid leukemia is a genetically and phenotypically heterogeneous disorder with an incidence of 3 to 4 per 100 000 men and women per year and a median age at diagnosis of about 70 years. Prognosis, especially in older patients, has remained very poor. In patients considered suitable for intensive chemotherapy, the combination of an anthracycline and cytarabine remains the standard of care. For patients achieving a complete remission (CR), postremission therapy (PRT) ranging from chemotherapy to allogeneic hematopoietic stem cell transplantation is required; intensive PRT is still under debate in older patients. Beyond pre-treatment genetics-based risk stratification, measurable residual disease (MRD) during treatment and follow up emerges as an important prognostic factor in first CR. Furthermore, MRD may provide a tool for a read-out of therapeutic efficacy. In this diagnostic meta-study the investigators intend to measure MRD using multiparameter flow cytometry across up-front randomized clinical trials which in total will accrue more than 1000 patients. According to the leukemia-associated phenotype at diagnosis or the different-from-normal approach, MRD will be assessed early (after induction) and late (after consolidation) during treatment. The aim of the study is to show that levels of MRD measured early during treatment are closely related to overall survival and thus may serve as an early surrogate. There is a growing public demand that new, promising drugs are approved for therapy as rapidly as possible. Therefore, it is of great interest to obtain these approvals based on early biomarker endpoints such as MRD rather than on long-term survival endpoints.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with AML participating in interventional prospective randomized trials of the Study Alliance Leukemia (SAL) including the Heidelberg Leukemia Network (HeLeNe)
Key inclusion criteria (2 listed)
- Acute myeloid leukemia according to the WHO classification
- Informed consent in place for a randomized study of the Study Alliance Leukemia (SAL) including the Heidelberg Leukemia Network (HeLeNe) covering assessment of MRD by MPFC in the reference laboratories in Heidelberg and Dresden.
Key exclusion criteria (1 listed)
- No signed informed consent compliant with the requirements of PERDAM
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 |
|---|---|---|
| Measurable residual disease measured by flow cytometryMeasurable residual disease measured by flow cytometry | Other | – |
Primary outcome
Correlation between Measurable Residual Disease (MRD) and Survival with respect to treatment effects
Time frame: after 3 and 6 years
MRD will be assessed using multiparameter flow cytometry early (after induction / salvage). Survival will be assessed continuously. After 3 and 6 years correlation between MRD and survival will be analysed with respect to treatment effects to assess if MRD may serve as surrogate endpoint. If levels of MRD measured early during treatment are closely related to overall survival and thus may serve as an early surrogate will be assessed yearly.
Results and safety
The study was stopped early (reason given: Study turned out no longer feasible). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Sep 2022.
- 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.
Richard F Schlenk, Prof. Dr. principal investigator
University Hospital Heidelberg
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 Leukemia, Myeloid, Acute, run by University Hospital Heidelberg at 1 site in Germany.
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 2 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: stopped early, as of the sponsor's last update on 13 Sep 2022. 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 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 was stopped early (reason given: Study turned out no longer feasible). Results are not available in the source record.
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?
Correlation between Measurable Residual Disease (MRD) and Survival with respect to treatment effects (time frame: after 3 and 6 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 13 Sep 2022.