REVUMENIB for Acute Myeloid Leukemia

Inclusion criteria

• {"criterion_text":"- Patients must be > 30 days and ≤ 18 years of age at time of enrollment.\n- Patients must have a performance status ≥50% Lansky or Karnofsky score.\n- Patients may have status of CNS1, CNS2, CNS3 disease without clinical signs or neurologic symptoms suggestive of CNS leukemia, such as facial nerve palsy, brain/eye involvement or hypothalamic syndrome.\n- Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy.\n- Patients must have adequate renal, liver and cardiac function\n- Patients must have R/R KMT2A-translocation (KMT2A-r), NUP98-rearrangement (NUP98-r) or NPM1-mutation (NPM1-mut AML). History of known KMT2A-r, NUP98-r or NPM1-mut is sufficient. Definition of refractory/relapsed disease (patient must have one of the following): primary refractory disease (≥ 5% leukemic blasts) after 2 cycles of induction, or first recurrent disease (≥ 5% leukemic blasts) after having achieved remission (ped-morph-CR).\n- ≥21 days must have elapsed since the patient’s last prior anti-cancer therapy (e.g., chemotherapy), except for protocol-defined pre-phase treatment, which is permitted regardless of the timing.\n- ≥ 84 days since allogeneic (non-autologous) bone marrow or stem cell transplant (with or without TBI) or boost infusion (any stem cell product; not including DLI); No evidence of graft versus host disease (GVHD) with the exception of Grade 1 skin GVHD being treated by topical treatment.\n- Cellular Therapy: ≥ 28 days after the completion of DLI (donor lymphocyte infusion) or any type of cellular therapy (e.g. modified T cells, NK cells, dendritic cells, etc.).\n- Understand and voluntarily provide written permission of parental/legal representative(s) to the ICF prior to conducting any trial related assessments/procedures, also concerning data and biomaterial transfer according to ICH/GCP and national/local regulations.\n- Able to adhere to the trial visit schedule and other protocol requirements.\n- Negative serum pregnancy tests for females of child-bearing potential within 10 days prior to treatment.\n- White Blood Cell (WBC): Count must be < 25.000/µL prior to enrolment. Patients may receive cytoreduction with hydroxyurea or low-dose cytarabine (max. 100mg/m² per day) prior to enrollment."}

Exclusion criteria

• {"criterion_text":"- Patients with isolated extramedullary disease.\n- Patients who are currently receiving another investigational drug.\n- Patients who are receiving cyclosporine, tacrolimus or other agents to prevent either graft-versus-host disease post bone marrow transplant or organ rejection post-transplant. Patients with active GVHD (other than Grade 1 skin GVHD).\n- Patients who are receiving any strong CYP3A4 inhibitors other than itraconazole, ketoconazole, posaconazole, or voriconazole.\n- Patients who are receiving any strong or moderate CYP3A4 inducers while on study or within 14 days of starting revumenib.\n- Patients who are receiving medications known to prolong the QT/QTc interval (exception of drugs with low risk of QT/QTc prolongation that are used as standard supportive therapies, e.g, diphenhydramine, famotidine, granisetron).\n- Patients known to have one of the following concomitant genetic syndromes: Bloom syndrome, ataxia-telangiectasia, Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known bone marrow failure syndrome.\n- Female patients who are pregnant or breastfeeding.\n- Patients who have ever used revumenib or any other menin inhibitor.\n- Female and male subjects with child bearing potential who avoid using highly effective anticonceptive measure(ment)s.\n- Patients whose baseline QTcF >450ms.\n- Hypersensitivity or allergy to the active substance or other excipients contained in the investigational medical product listed in the Summary of Product Characteristics (SmPC) or Investigators Brochure (IB).\n- Patients with documented active, uncontrolled infection at the time of study entry.\n- Patients with Down Syndrome.\n- Patients with a secondary KMT2A-R, NPM1 or NUP98-r leukemia that developed after treatment of prior malignancy with cytotoxic chemotherapy.\n- Patients with known partial duplication of KMT2A (MLL-PTD).\n- Patients with known Mixed Lineage Leukemia.\n- Patients with a history of congenital prolonged QT syndrome, congestive heart failure or uncontrolled arrhythmia in the past 6 months prior to study enrolment.\n- Patients with gastrointestinal issues of the upper gastrointestinal tract that might affect oral drug absorption."}

Primary endpoints

• {"endpoint_text":"- The primary efficacy endpoint of Part I in this study is to estimate the ORR. We will compare the ORR to a historically informed null ORR rate of 37% after up to 2 cycles with revumenib in combination with FLA in R/R KMT2A-r, NUP98-r or NPM1- mut AML.","definition_or_measurement_approach":"Estimate overall response rate (ORR) after up to 2 cycles with revumenib + FLA and compare the observed ORR to a historically informed null ORR rate of 37%; responses assessed according to pediatric specific response criteria (Ped- morph-CR/CRi/CRp) as described in the main objective."}

Austria

Earliest CTIS Part Ii Submission Date

14-01-2026

Latest Decision Or Authorization Date

20-02-2026

Processing Time Days

37

Number Of Sites

1

Number Of Participants

2

Czechia

Earliest CTIS Part Ii Submission Date

04-02-2026

Latest Decision Or Authorization Date

19-02-2026

Processing Time Days

15

Number Of Sites

1

Number Of Participants

2

Denmark

Earliest CTIS Part Ii Submission Date

10-02-2026

Latest Decision Or Authorization Date

16-02-2026

Processing Time Days

6

Number Of Sites

2

Number Of Participants

2

Germany

Earliest CTIS Part Ii Submission Date

20-01-2026

Latest Decision Or Authorization Date

19-02-2026

Processing Time Days

30

Number Of Sites

7

Number Of Participants

10

Italy

Earliest CTIS Part Ii Submission Date

09-02-2026

Latest Decision Or Authorization Date

20-02-2026

Processing Time Days

11

Number Of Sites

3

Number Of Participants

5

Sweden

Earliest CTIS Part Ii Submission Date

18-12-2025

Latest Decision Or Authorization Date

20-02-2026

Processing Time Days

64

Number Of Sites

1

Number Of Participants

2

Netherlands

Earliest CTIS Part Ii Submission Date

04-02-2026

Latest Decision Or Authorization Date

19-02-2026

Processing Time Days

15

Number Of Sites

1

Number Of Participants

3

Poland

Earliest CTIS Part Ii Submission Date

13-01-2026

Latest Decision Or Authorization Date

22-02-2026

Processing Time Days

40

Number Of Sites

4

Number Of Participants

5

Primary sponsor

Full Name

Pediatric Research International GmbH

Organisation Type

Patient organisation/association

Country Of Registered Address

Germany

Third parties

• {"country":"Germany","full_name":"Zentrum fuer Forschungsfoerderung in der Paediatrie GmbH","duties_or_roles":"codes: 1,12,5,6,7,8","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Germany","full_name":"Paediatrisches Forschungsnetzwerk gGmbH","duties_or_roles":"codes: 1,12,5,6,7,8","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Switzerland","full_name":"Novustat GmbH","duties_or_roles":"codes: 10","organisation_type":"SME"}