Acute Myeloid LeukemiaNCT03337516
Interest of a Therapeutic Follow-up of the Cytarabine in the Acute Myeloid Leukaemia: Is There Constitutional Biomarkers
A study testing Blood sampling and Genetic analysis in people with Acute Myeloid Leukemia, run by Assistance Publique Hopitaux De Marseille at 1 site in France.
Trial summary
In this open-label, single-center, non-randomized patients with AML (Acute Myeloid Leukemia) and receiving all induction chemotherapy and consolidation consisting of cytarabine under the care usual for this pathology, will be included. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Blood sampling and Genetic analysis
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteFrance
- Study dates
- 21 Jun 2017to 31 Dec 2018
- Phase
- Interventional
- 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
Who may be able to join
- Patient aged over 18 years old
- Patient with acute myeloid leukemia
- Patient treated with cytarabine
- Patient having signed an informed consent form
- Patient having signed an authorization to practice a constitutional genetic analysis
- Need for effective contraception in patients of childbearing age.
- Patient affiliated to a social security scheme
Who cannot join
- Not obtaining free, informed and signed consent
- Patient participating in another biomedical research
- Pregnant patients
Trial locations and contacts
1 site in 1 country.
Régis COSTELLO, MD-PhD contact
Camille DELANNOY contact
France1 site, 1 recruiting
- Recruiting
Assistance Publique - Hopitaux de Marseille
Marseille
Regis Costello, regis.costello@ap-hm.fr
Regis Costello
About this study
In this open-label, single-center, non-randomized patients with AML (Acute Myeloid Leukemia) and receiving all induction chemotherapy and consolidation consisting of cytarabine under the care usual for this pathology, will be included. Each patient will be followed and for the development of toxicities, treatment response and progression-free survival. In addition to the usual care set out above, each patient will undergo a series of constitutional genetic investigations conducted by NGS on markers related to pharmacokinetics cytarabine. Another set of blood samples will also calculate, according to a Bayesian approach, individual pharmacokinetics of cytarabine and its metabolite, arabinosine-uracil.
This study should allow the correlation between pharmacogenetics and patient plasma exposure, that would eventually balance improved efficacy / toxicity of this molecule through a customization regimens, achieved so far on a empirical basis. If validation of our data, a dosage of therapeutic pre CDA could help in predicting pharmacodynamics of cytarabine individual dose adjustment, as is done for the 5-FU and DPD.
Read the detailed description
Background and Rationale: The development of personalized medicine in oncology has so far relied on the use of somatic biomarkers to inform the therapist about the choice of the molecule or molecules to be administered based on the genetic and molecular profile of each blood disease. In this project, we propose to extend the therapeutic individualization strategy targeting dosage domain. Today, cytarabine is one of two pillars of the treatment of leukemia Acute Myeloid Leukemia (AML) in combination with an anthracycline plus cytarabine ("3 + 7" therapeutic scheme) during the induction course and usually during monotherapy consolidation treatments. According to the treatment regimens and protocols, cytarabine is prescribed at a standard dose (SD = 100-200mg / m² / day), intermediate dose (ID = 1 to 1.5 g / 12H for all 3 days) or high dose (DH = 2 to 3 g / m² every 12H during 3 days). The choice between these different dose levels remains highly debated (Lowenberg et al. 2013) with a very narrow risk-benefit balance. Indeed, the various cooperative groups compared two dose regimens with factors between the minimum dose and maximum ranging from 1.7 times to 34 times between groups (Ex Australian arm group 1400 mg / m vs 48 000 mg / m cumulative dose cytarabine ). The magnitude of unmatched dose differentials illustrates the complexity of understanding and apprehension of this pivotal molecule for the treatment of AML. Despite the many studies that focused on the optimal dose of cytarabine ranging from 1400 mg / m² to 90 000 mg / m² (German group) (including currently French Intergroup of leukemia-ALFA FILO adult under the BIG1 protocol), none evaluated the relevance of the a priori individual dose adjustment depending on the pharmacogenetic patient data. In current practice, the doses are adapted a posteriori, and reduced empirically following the observed toxicity of occurrence (20% of patients) (Lowenberg et al. 2013). This adaptation a posteriori is a loss of opportunity for the patient. Similarly, under dosed patients for fear of toxicity is also another lost chance. Our hypothesis is that the optimal cytarabine dose depends not only on the characteristics of the patient's pathology (risk groups including cytogenetic data, biology, molecular), but also the patient's individual characteristics (genetic status of metabolic enzymes and carriers). A mathematical model of PK / PD kind could, based on early observations of circulating levels, be able to quickly predict the pharmacodynamic effect in each patient, allowing a rapid individualization of dosages. Such a tool could enable, in future, to propose dose adjustments early after initiation of treatment before the onset of toxicity, predicting that exposure levels of cytarabine correlate with the patient's clinical evolution.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Blood sampling, Genetic analysis
- 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.
Blood samplingBiological
Blood sampling in order to assess cytarabine pharmacokinetics
Genetic analysisGenetic
Genetic analysis in order to determine genetic polyporphism of Deoxycytidine Kinase and Cytidine Deaminase
| Study group | Role | What they receive |
|---|---|---|
| Patients treated with Cytarabine | Experimental | Not described Receives: Biological: Blood sampling; Genetic: Genetic analysis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Measurements of circulating levels of cytarabine and its metabolites.
Measured: 6 months maximum
Assessment of genetic polymorphism of somatic Cytidine Deaminase (CDA) and Déoxycitidine Kinase (dCK).
Measured: 6 months maximum
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Nov 2017
- Study start21 Jun 2017
- Primary completion21 Dec 2017 (estimated)
- Study completion31 Dec 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by Assistance Publique Hopitaux De MarseilleThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique Hopitaux De Marseille
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jean-Olivier ARNAUD, Study director, Assistance Publique- Hôpitaux de Marseille
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03337516
- Sponsor's protocol ID
- 2017-02
- Other IDs
- 2017-A00070-53
- Status last verified
- Nov 2017
- First submitted
- 6 Nov 2017
- Last updated
- 9 Nov 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Interest of a Therapeutic Follow-up of the Cytarabine in the Acute Myeloid Leukaemia: Is There Constitutional Biomarkers
- ClinicalTrials.gov ID
- NCT03337516
- Acronym / sponsor ID
- 2017-02
- Conditions
- Acute Myeloid Leukemia
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Assistance Publique Hopitaux De MarseilleUniversity, hospital or other
- Study start
- 21 Jun 2017 (actual)
- Primary completion
- 21 Dec 2017 (estimated)
- Study completion
- 31 Dec 2018 (estimated)
- First posted
- 9 Nov 2017 (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.
In this open-label, single-center, non-randomized patients with AML (Acute Myeloid Leukemia) and receiving all induction chemotherapy and consolidation consisting of cytarabine under the care usual for this pathology, will be included. Each patient will be followed and for the development of toxicities, treatment response and progression-free survival. In addition to the usual care set out above, each patient will undergo a series of constitutional genetic investigations conducted by NGS on markers related to pharmacokinetics cytarabine. Another set of blood samples will also calculate, according to a Bayesian approach, individual pharmacokinetics of cytarabine and its metabolite, arabinosine-uracil.
This study should allow the correlation between pharmacogenetics and patient plasma exposure, that would eventually balance improved efficacy / toxicity of this molecule through a customization regimens, achieved so far on a empirical basis. If validation of our data, a dosage of therapeutic pre CDA could help in predicting pharmacodynamics of cytarabine individual dose adjustment, as is done for the 5-FU and DPD.
Detailed description
Background and Rationale: The development of personalized medicine in oncology has so far relied on the use of somatic biomarkers to inform the therapist about the choice of the molecule or molecules to be administered based on the genetic and molecular profile of each blood disease. In this project, we propose to extend the therapeutic individualization strategy targeting dosage domain. Today, cytarabine is one of two pillars of the treatment of leukemia Acute Myeloid Leukemia (AML) in combination with an anthracycline plus cytarabine ("3 + 7" therapeutic scheme) during the induction course and usually during monotherapy consolidation treatments. According to the treatment regimens and protocols, cytarabine is prescribed at a standard dose (SD = 100-200mg / m² / day), intermediate dose (ID = 1 to 1.5 g / 12H for all 3 days) or high dose (DH = 2 to 3 g / m² every 12H during 3 days). The choice between these different dose levels remains highly debated (Lowenberg et al. 2013) with a very narrow risk-benefit balance. Indeed, the various cooperative groups compared two dose regimens with factors between the minimum dose and maximum ranging from 1.7 times to 34 times between groups (Ex Australian arm group 1400 mg / m vs 48 000 mg / m cumulative dose cytarabine ). The magnitude of unmatched dose differentials illustrates the complexity of understanding and apprehension of this pivotal molecule for the treatment of AML. Despite the many studies that focused on the optimal dose of cytarabine ranging from 1400 mg / m² to 90 000 mg / m² (German group) (including currently French Intergroup of leukemia-ALFA FILO adult under the BIG1 protocol), none evaluated the relevance of the a priori individual dose adjustment depending on the pharmacogenetic patient data. In current practice, the doses are adapted a posteriori, and reduced empirically following the observed toxicity of occurrence (20% of patients) (Lowenberg et al. 2013). This adaptation a posteriori is a loss of opportunity for the patient. Similarly, under dosed patients for fear of toxicity is also another lost chance. Our hypothesis is that the optimal cytarabine dose depends not only on the characteristics of the patient's pathology (risk groups including cytogenetic data, biology, molecular), but also the patient's individual characteristics (genetic status of metabolic enzymes and carriers). A mathematical model of PK / PD kind could, based on early observations of circulating levels, be able to quickly predict the pharmacodynamic effect in each patient, allowing a rapid individualization of dosages. Such a tool could enable, in future, to propose dose adjustments early after initiation of treatment before the onset of toxicity, predicting that exposure levels of cytarabine correlate with the patient's clinical evolution.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients treated with Cytarabine | Experimental | Biological: Blood sampling, Genetic: Genetic analysis |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Patient aged over 18 years old
- Patient with acute myeloid leukemia
- Patient treated with cytarabine
- Patient having signed an informed consent form
- Patient having signed an authorization to practice a constitutional genetic analysis
Key exclusion criteria (3 listed)
- Not obtaining free, informed and signed consent
- Patient participating in another biomedical research
- Pregnant patients
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 |
|---|---|---|
| Blood samplingBlood sampling in order to assess cytarabine pharmacokinetics | Biological | Patients treated with Cytarabine |
| Genetic analysisGenetic analysis in order to determine genetic polyporphism of Deoxycytidine Kinase and Cytidine Deaminase | Genetic | Patients treated with Cytarabine |
Primary outcome
Measurements of circulating levels of cytarabine and its metabolites.
Time frame: 6 months maximum
Assessment of genetic polymorphism of somatic Cytidine Deaminase (CDA) and Déoxycitidine Kinase (dCK).
Time frame: 6 months maximum
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Nov 2017.
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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.
Régis COSTELLO, MD-PhD contact
Camille DELANNOY contact
Jean-Olivier ARNAUD study director
Assistance Publique- Hôpitaux de Marseille
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Blood sampling and Genetic analysis in people with Acute Myeloid Leukemia, run by Assistance Publique Hopitaux De Marseille at 1 site in France.
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 7 inclusion and 3 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 9 Nov 2017. 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 France. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Régis COSTELLO, MD-PhD (regis.costello@ap-hm.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Measurements of circulating levels of cytarabine and its metabolites. (time frame: 6 months maximum). The study lists 2 primary outcomes in all. 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 9 Nov 2017.