The Patient Cohort of the National Center for Precision Medicine in Leukemia

Acute Myeloid Leukemia Clinical Trial

— eTHEMA  

Official title:

The Patient Cohort of the National Center for Precision Medicine in Leukemia

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05326919
• Other study ID #: APHP210850
• Status: Recruiting
• Start date: March 28, 2022
• Est. completion date: March 2042

Study information

• Verified date: May 2024
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: Hervé DOMBRET, Pr
• Phone: 1 57 27 68 47
• Email: herve.dombret[@]aphp.fr
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

If for years the treatment strategy of leukemia and related disorders (LRDs, including acute leukemias and predisposition syndromes) has been based solely on whether the patient could receive or not intensive chemotherapy and transplantation, the advent of new targeted or less targeted drugs has led to the development of a growing number of new therapeutic approaches, very often offered to specific patient/disease subsets, justifying the generic term of 'precision medicine'.

As an international leukemia center of excellence, THEMA, the French National Center for Precision Medicine in Leukemia (selected as IHUB-2 by the French National Agency for Research), is a care, research, transfer and education initiative located at the Saint-Louis Research Institute (IRSL) in Paris and devoted to precision medicine in leukemia in a real-life environment.

The present non-interventional study (eTHEMA) is a pillar of the whole THEMA project. As a prerequisite for precision medicine, this program focuses on individual data collection, aiming to collect high-quality data not only in patients treated into prospective clinical trials, but in every THEMA patient with a special interest in outpatients' care and research.

The primary objective of this non-interventional study is to describe the baseline characteristics planned treatments and outcomes of patients newly diagnosed with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), high-risk myelodysplastic syndrome (MDS), or myeloproliferative neoplasm (MPN)-related myelofibrosis, when managed and treated according to standard diagnosis and care practices.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 3000
• Est. completion date: March 2042
• Est. primary completion date: March 2042
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Patient with newly diagnosed previously untreated de novo, secondary or therapy-related leukemia or related disorders (LRD), including AML, ALL, HR-MDS (according to the international score IPSS), and MNP-related myelofibrosis

• Patient informed and not opposed to participating

• Affiliation to social security or any health insurance

Exclusion Criteria:

• LRD which is not morphologically proven (patients with granulocytic sarcoma may be included)

• Previous treatment for LRD, apart from:

• Hydroxyurea or previous MDS/MPN-CML therapy in AML patients

• Steroids, vincristine, intrathecal prophylactic or curative injection or previous CML therapy in ALL patients

• Erythroid stimulating agents (ESAs), luspatercept, granulocyte colony-stimulating factor (G-CSF), eltrombopag or other TPO agonist, iron chelation therapy, hypomethylating agents (HMAs), lenalidomide or any investigational drug previously used to treat MDS in HR-MDS patients

• Hydroxyurea, standard or pegylated interferon alpha, ruxolitinib or other JAK inhibitors, busulfan, anagrelide, ESAs or any investigational drug previously used to treat MPN in MPN-related myelofibrosis patients

• Patient under guardianship / curatorship

• Patient under AME

• Opposition of the patient to be enrolled in the eTHEMA cohort

Related Conditions & MeSH terms

• Acute Lymphoblastic Leukemia
• Acute Myeloid Leukemia
• High-risk Myelodysplastic Syndrome
• Leukemia
• Myelodysplastic Syndromes
• Myeloproliferative Disorders
• Myeloproliferative Neoplasm, Unclassifiable
• Precursor Cell Lymphoblastic Leukemia-Lymphoma

Intervention

Other:
• Biobanking
For storage,limited volumes of blood or bone marrow aspirate will be added to usual sampling and stored.

Locations

Country	Name	City	State
France	Hôpital Avicenne	Bobigny
France	Hopital Robert Debré	Paris
France	Hôpital Saint Louis	Paris

Sponsors (1)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Event Free Survival		at 5 years
Primary	Relapse Free Survival		at 5 years
Primary	Overall Survival		at 5 years
Secondary	Standardized evaluation of hematological response		After induction cycle which is between between day 25 and day 42 for patients treated intensively and between Month 1 and Month 6 for patients treated treated with low intensity regimen
Secondary	Standardized evaluation of hematological response		After first consolidation cycle which is between 1 and 2 months
Secondary	Standardized evaluation of hematological response		After last consolidation cycle which is between 3 and 8 months
Secondary	Standardized evaluation of hematological response		Before HSCT
Secondary	Standardized evaluation of hematological response		at day 100 after HSCT
Secondary	Standardized evaluation of hematological response		at 5 years
Secondary	Minimal measurable residual disease (MRD) response		After induction which is between day 25 and day 42 for patients treated intensively and between month 1 and month 6 for patients with low intensity regimen
Secondary	Minimal measurable residual disease (MRD) response		After first consolidation cycle which is between 1 and 2 months
Secondary	Minimal measurable residual disease (MRD) response		After last consolidation cycle which is between 3 and 8 months
Secondary	Minimal measurable residual disease (MRD) response		Before HSCT
Secondary	Minimal measurable residual disease (MRD) response		at day 100 after HSCT
Secondary	Minimal measurable residual disease (MRD) response		at 5 years
Secondary	Incidence of allogeneic HSCT		at 5 years
Secondary	Modalities of allogeneic HSCT		at 5 years
Secondary	Incidence of hematological relapses		at 5 years
Secondary	Type of hematological relapses		at 5 years
Secondary	Incidence of hematological progressions		at 5 years
Secondary	Type of hematological progressions		at 5 years
Secondary	Incidence of MRD relapses		at 5 years
Secondary	Incidence of MRD progressions		at 5 years
Secondary	Proportions of patients with treatment-related toxicities	Treatment-related toxicities will be Evaluated by the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0	at 5 years
Secondary	Cumulative incidences of relapse		at 5 years
Secondary	Cumulative incidences of non-relapse mortality		at 5 years
Secondary	Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire	Quality of life evaluated using questionnaire "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" (EORTC QLQ-C30- v3). The QLQ-C30 is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / QoL scale, and six single items. Each of the multi-item scales includes a different set of items - no item occurs in more than one scale. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. A high score for a functional scale represents a high/healthy level of functioning. A high score for the global health status/ QoL represents a high QoL and a high score for a symptom scale/item represents a high level of symptomatology/problems.	at inclusion
Secondary	Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire	Quality-of-life will be assessed using the EORTC-QLQ-C30 v3 questionnaire.Quality of life evaluated using questionnaire "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" (EORTC QLQ-C30- v3). The QLQ-C30 is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / QoL scale, and six single items. Each of the multi-item scales includes a different set of items - no item occurs in more than one scale. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. A high score for a functional scale represents a high/healthy level of functioning. A high score for the global health status/ QoL represents a high QoL and a high score for a symptom scale/item represents a high level of symptomatology/problems.	at the end of induction which is between day 25 and day 42 for patients treated intensively and between month 1 and month 6 for patients with low intensity regimen
Secondary	Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire	Quality-of-life will be assessed using the EORTC-QLQ-C30 v3 questionnaire. Quality of life evaluated using questionnaire "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" (EORTC QLQ-C30- v3). The QLQ-C30 is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / QoL scale, and six single items. Each of the multi-item scales includes a different set of items - no item occurs in more than one scale. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. A high score for a functional scale represents a high/healthy level of functioning. A high score for the global health status/ QoL represents a high QoL and a high score for a symptom scale/item represents a high level of symptomatology/problems.	after 2 consolidations courses which is between 3 months and 8 months
Secondary	Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire	Quality-of-life will be assessed using the EORTC-QLQ-C30 v3 questionnaire. Quality of life evaluated using questionnaire "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" (EORTC QLQ-C30- v3). The QLQ-C30 is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / QoL scale, and six single items. Each of the multi-item scales includes a different set of items - no item occurs in more than one scale. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. A high score for a functional scale represents a high/healthy level of functioning. A high score for the global health status/ QoL represents a high QoL and a high score for a symptom scale/item represents a high level of symptomatology/problems.	at 3 months after the end of treatment
Secondary	Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire	Quality-of-life will be assessed using the EORTC-QLQ-C30 v3 questionnaire. Quality of life evaluated using questionnaire "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" (EORTC QLQ-C30- v3). The QLQ-C30 is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / QoL scale, and six single items. Each of the multi-item scales includes a different set of items - no item occurs in more than one scale. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. A high score for a functional scale represents a high/healthy level of functioning. A high score for the global health status/ QoL represents a high QoL and a high score for a symptom scale/item represents a high level of symptomatology/problems.	at 6 months after the end of treatment
Secondary	Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire	Quality-of-life will be assessed using the EORTC-QLQ-C30 v3 questionnaire. Quality of life evaluated using questionnaire "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" (EORTC QLQ-C30- v3). The QLQ-C30 is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / QoL scale, and six single items. Each of the multi-item scales includes a different set of items - no item occurs in more than one scale. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. A high score for a functional scale represents a high/healthy level of functioning. A high score for the global health status/ QoL represents a high QoL and a high score for a symptom scale/item represents a high level of symptomatology/problems.	at 12 months after the end of treatment
Secondary	Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire	Quality-of-life will be assessed using the EORTC-QLQ-C30 v3 questionnaire. Quality of life evaluated using questionnaire "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" (EORTC QLQ-C30- v3). The QLQ-C30 is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / QoL scale, and six single items. Each of the multi-item scales includes a different set of items - no item occurs in more than one scale. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. A high score for a functional scale represents a high/healthy level of functioning. A high score for the global health status/ QoL represents a high QoL and a high score for a symptom scale/item represents a high level of symptomatology/problems.	at day 100 after hematopoietic stem cell transplant
Secondary	Incidence of secondary cancer		at 5 years
Secondary	Incidence of secondary cancer		up to 15 years