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S243249 Monotherapy in Patients With Relapsed or Refractory Acute Leukemia With a KMT2A or NUP98 Translocation or Relapsed or Refractory AML With NPM1c Mutation

ConditionsRelapsed or Refractory Acute LeukemiaRelapsed or Refractory Acute Myeloid Leukemia
Trial statusNot yet recruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorServier

About this trial

The objective of this study is to establish the efficacy, safety, and tolerability of S243249 and to establish the recommended phase 2 dose (RP2D) of S243249 monotherapy in participants with relapsed/refractory (R/R) acute leukemia with select mutations. Phase 1 dose optimization will determine the RP2D to be used in Phase 2 dose expansion. The study will include a screening period, a treatment period consisting of continuous 28-day cycles of treatment, a safety follow-up period and a long-term follow-up period. Participants may undergo blood tests, electrocardiogram (ECG), bone marrow aspirations, vital sign checks, questionnaires, and physical exams.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Aged ≥ 18 years old.

Negative serum pregnancy (β-hCG) test in women of childbearing potential at screening

Primary refractory disease, defined as non-response to 2 courses of standard induction therapy.

R/R disease, defined as > 5% blasts on bone marrow aspirate (BMA) / bone marrow biopsy (BMB) after completing prior therapy.

Disqualifiers

Active central nervous system (CNS) leukemia (including imaging abnormalities and cerebrospinal fluid (CSF) smear or flow cytometry indicating leukemia cells)).

Active disseminated intravascular coagulation (DIC).

Active uncontrolled infection (prophylaxis because of absolute neutrophil count [ANC] is excepted).

Diagnosis of acute promyelocytic leukemia (APL, M3).

Trial design

Design model

Parallel

Treatments tested in this trial

  • S243249 600mg

    Drug

    Taken twice daily by mouth

  • S243249 400mg

    Drug

    Taken twice daily by mouth

  • S243249 450mg

    Drug

    Taken twice daily by mouth

  • S243249 300mg

    Drug

    Taken twice daily by mouth

Treatment groups

80 Participants
are divided into 4 treatment groups
Group A: Phase 1 Dose Optimization: Cohort 1Experimental treatment 1 intervention
Group B: Phase 1 Dose Optimization: Cohort 2Experimental treatment 1 intervention
Group C: Phase 1 Dose Optimization: Cohort 3Experimental treatment 1 intervention
Group D: Phase 1 Dose Optimization: Cohort 4Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of Adverse Events (AEs)

Time frame
Through Safety Follow-up (Approximately 3 years)
2

Severity of AEs

Time frame
Through Safety Follow-up (Approximately 3 years)
3

Number of changes in laboratory values

Time frame
Through Safety Follow-up (Approximately 3 years)
4

Number of changes in electrocardiogram (ECG)

Time frame
Through Safety Follow-up (Approximately 3 years)

Secondary outcomes

1

Overall response rate (ORR)

CR + CRh + Complete remission with incomplete hematological recovery (CRi) + Morphologically leukemic state (MLFS) + Partial remission (PR)

Time frame
Through Long-term Follow-up (Approximately 5 years)
2

Composite complete remission (CRc) rate

CRc is CR + CRh + CRi

Time frame
Through Long-term Follow-up (Approximately 5 years)
3

CR rate

Time frame
Through Long-term Follow-up (Approximately 5 years)
4

Rate of CR/CRh Minimal residual disease (MRD) negativity

Time frame
Through Long-term Follow-up (Approximately 5 years)

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Servier

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: