An interventional study of genetic study and Pharmacokinetic study in Myelodysplastic Syndromes (MDS) and Leukemia Acute Myeloid - AML, sponsored by Assistance Publique Hopitaux De Marseille. Recruiting at 1 site in France. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-28.
Sponsored by Assistance Publique Hopitaux De Marseille · Not applicable, Interventional, and Other
Among 198 studies in Myelodysplastic Syndromes with a site in France — 36 open to participants now, 54 under way, 13 of them phase 3. Myelodysplastic Syndromes · France.
Among the 198 studies in Myelodysplastic Syndromes with a site in France, this one is the 99th largest by planned enrollment of the 172 interventional ones, is one of 11 that started in 2021, is one of 19 with no phase, reaches primary completion later than 20 of the 53 other live ones, and was the 194th registered.
1,997 studies on the registry are indexed under Myelodysplastic Syndromes; 324 are open to participants now.
This study's planned enrollment of 70 is above the median of 40 across 1,611 interventional studies indexed under Myelodysplastic Syndromes.
Browse Myelodysplastic Syndromes studies →36 of the 198 studies in Myelodysplastic Syndromes with a site in France are open now, in 12 countries. Each count lists the studies.
Of the 262 Myelodysplastic Syndromes studies recruiting now, 165 (63%) set no upper age limit and 70 (27%) stop at 75 or younger; 57 take children under 18; 17 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →198 studies; the count open now beside each. This study's row is marked.
First in 1993; this study’s year is marked. 11 started in 2021.
Median enrollment 93 across the 172 interventional studies, 137 across the 25 observational. Of the 117 that completed or were terminated, 46 (39%) have results posted.
97 of 198 have an industry lead sponsor. The 10 most frequent:
Assistance Publique Hopitaux De Marseille is the lead sponsor of 702 studies on the registry; 139 are open to participants now.
Assistance Publique Hopitaux De Marseille has 2 studies filed under Myelodysplastic Syndromes — fewer than ten, so there is no comparison to draw.
14 of the studies in Myelodysplastic Syndromes with a site in France that are under way reach primary completion within a year, 3 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT04401748Phase 3AbbVieStudy Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome | 2027-07 |
| NCT04256317Phase 3Taiho Oncology, Inc.A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study) | 2027-11-01 |
| NCT06465953Phase 3Institut de Recherches Internationales ServierIvosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an IDH1 Mutation | 2027-11-01 |
| NCT06987058Ryvu Therapeutics SARVU120 Rollover Study | 2026-12 |
| NCT06782373Swedish Orphan BiovitrumA Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS) | 2026-12-30 |
Among the 198 studies in Myelodysplastic Syndromes with a site in France, from their registry updates:
Until now, the development of personalized medicine in oncology has relied on the use of somatic biomarkers to help therapists choose the right molecule(s) to administer, based on the genetic and molecular profile of each hematological disease. In this project, investigators propose to extend the strategy of therapeutic individualization to the field of dosage targeting. Today, azacytidine is a standard treatment for patients with acute myeloid leukemia (AML) and/or myelodysplastic syndromes (MDS), usually as monotherapy. According to the treatment regimen, azacytidine is prescribed at a standard dose (DS=75mg/m²/d), administered subcutaneously every day for 7 days. The treatment cycle is repeated every 28 days.
No study has evaluated the relevance of "a priori" dose adjustment on an individual basis, according to each patient's pharmacogenetic data. In current practice, doses are adapted a posteriori, and reduced empirically, following the occurrence of observed toxicity (6 to 71% of patients) (Schuck A et al. 2017). This ex-post adjustment in the face of grade 3-4 toxicity is a loss of chance for the patient. Similarly, under-dosing patients for fear of toxicity is another loss of chance. Investigator's hypothesis is that the optimal dose of azacytidine depends not only on the characteristics of the patient's pathology (risk groups including cytogenetic and molecular biology data), but also on the patient's individual characteristics (genetic status of metabolic enzymes and transporters). A mathematical model of the PK/PD type could, on the basis of early observations of circulating levels, be capable of rapidly predicting the pharmacodynamic repercussions in each patient, thus enabling rapid individualization of dosages. In the future, such a tool could make it possible to propose dosage adjustments rapidly after treatment initiation, before toxicity occurs, by predicting azacytidine exposure levels, themselves correlated with the patient's clinical condition.
Study design: In this open-label, paucicentric, non-randomized study, patients with AML and/or MDS, all of whom are receiving azacytidine-based chemotherapy as part of their standard treatment regimen, will be included. Each patient will be monitored for toxicities (EORTC), treatment response and progression-free survival. In addition to the standard care described above, each patient will undergo a series of constitutional genetic investigations conducted by NGS on markers linked to azacytidine pharmacokinetics (CDA, dCK). Another series of blood samples will be taken to calculate individual azacytidine pharmacokinetic parameters using a Bayesian approach.
Expected results: This study should make it possible to correlate pharmacogenetics with patient plasma exposure, and ultimately improve the molecule's efficacy/toxicity balance by personalizing dosage regimens, which until now have been carried out on an empirical basis.
Prospects: If the data are validated, a pre-therapeutic ADC assay could predict azacytidine pharmacodynamics and enable individual dose and/or dosage adjustment, as is the case with 5-FU and DPD.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Blood sampling through the differents cures of the patients
Other: genetic study · Other: Pharmacokinetic study · Other: Study of phenotypic activity of cytidine deaminase
Blood sampling : Cure 1 before starting treatment Cure 3 before starting treatment Cure 6 after completion of treatment
Cure 1: after completion of subcutaneous administration of azacytidine A total of 5 samples in the induction phase. Cure 3: after completion of subcutaneous administration of azacytidine A total of 5 samples in the induction phase. Cure 6: after completion of subcutaneous administration of azacytidine A total of 5 samples in the induction phase.
Blood sampling : Cure 1 before starting treatment Cure 3 before starting treatment Cure 6 after completion of treatment
Plasma dosage of azacytidine
assessment of the relationship between azacytidine pharmacokinetic profile and cytidine deaminase status
Time frame: Through study completion, an average of 2 years
phenotypic activity of CDA
searched by Sanger on a ThermoFischer 3500 Dx Genetic automaton using standard procedures, after identification of the most informative regions (exons, promoters). Haplotypic recombinations will be searched for using Haploview software.
Time frame: Through study completion, an average of 2 years
Determination of ADC genotype
searched by Sanger on a ThermoFischer 3500 Dx Genetic automaton using standard procedures, after identification of the most informative regions (exons, promoters). Haplotypic recombinations will be searched for using Haploview software.
Time frame: Through study completion, an average of 2 years
Started Jun 2021 (actual). Primary completion is estimated for Jan 2027, 66 months after the start.
Of 57 non-industry interventional studies with no phase under Myelodysplastic Syndromes, half reached primary completion within 66 months of starting (the median), counting those still running; a quarter within 33 and three quarters within 125. This study's planned 66 months is close to that.
Counting every sponsor, half of 58 studies reached it within 65 months; a quarter within 40 and three quarters within 125.
Primary completion moved once since we started tracking on Sep 25, 2026: Jun 2026 → Jan 2027, on Oct 28, 2026.
Earlier changes are on the record history on ClinicalTrials.gov ↗
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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