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

NCT ID: NCT05326919

Last Updated: 2024-05-29

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 3000 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2022-03-28
• Study Completion Date: 2042-03-31

Brief 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.

Conditions

Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia; High-risk Myelodysplastic Syndrome; Secondary Myelofibrosis in Myeloproliferative Disease; Myeloproliferative Neoplasm, Unclassifiable

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Acute myeloid Leukemia (AML)

Standard and routine care.

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

Biobanking

Intervention Type: OTHER

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

Acute lymphoblastic leukemia (ALL)

Standard and routine care.

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

Biobanking

Intervention Type: OTHER

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

High-risk myelodysplastic syndrome (MDS)

Standard and routine care.

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

Biobanking

Intervention Type: OTHER

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

Myeloproliferative neoplasm -related myelofibrosis

Standard and routine care.

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

Biobanking

Intervention Type: OTHER

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

Interventions

Biobanking

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

Intervention Type: OTHER

Eligibility Criteria

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

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Assistance Publique - Hôpitaux de Paris

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Hôpital Avicenne

Bobigny, , France

Site Status: RECRUITING

Hopital Robert Debré

Paris, , France

Site Status: RECRUITING

Hôpital Saint Louis

Paris, , France

Site Status: RECRUITING

Countries

France

Central Contacts

Hervé DOMBRET, Pr

Role: CONTACT

herve.dombret@aphp.fr

1 57 27 68 47 ext. +33

Jérôme Lambert, Pr

Role: CONTACT

jerome.lambert@u-paris.fr

142499742 ext. +33

Facility Contacts

Thorsten Braun, Pr

Role: primary

thorsten.braun@aphp.fr

André Baruchel, Pr

Role: primary

andre.baruchel@aphp.fr

Hervé Dombret, Pr

Role: primary

herve.dombret@aphp.fr

1 57 27 68 47 ext. +33

References

Zhao LP, Dumas-Rivero T, Barette L, Aguinaga L, Cheffai A, Chauvel C, Dal Bello R, Raffoux E, Clappier E, Duchmann M, Fenaux P, Lemaire P, Mathis S, Sebert M, Ades L, Itzykson R. Prognostic significance of monocytic-like phenotype in patients with AML treated with venetoclax and azacytidine. Blood Adv. 2025 Jul 22;9(14):3556-3565. doi: 10.1182/bloodadvances.2024015734.

Reference Type: DERIVED

PMID: 40249917 (View on PubMed)

Other Identifiers

APHP210850

Identifier Type: -

Identifier Source: org_study_id