OncoMatch

OncoMatch/Clinical Trials/NCT05745714

HEM-iSMART-C: Ruxolitinib + Venetoclax + Dexamethasone + Cyclophosphamide and Cytarabine in Pediatric Patients With Relapsed or Refractory Hematological Malignancies

Is NCT05745714 recruiting? Yes, currently enrolling (Jun 2026). This Phase 1/2 trial studies multiple treatments for acute lymphoblastic leukemia, in relapse.

Phase 1/2RecruitingPrincess Maxima Center for Pediatric OncologyNCT05745714Data as of Jun 2026Location: International · 14 countries

Treatment: Ruxolitinib · Venetoclax · Dexamethasone · Cyclophosphamide · Cytarabine · intrathecal chemotherapyHEM-iSMART is a master protocol which investigates multiple investigational medicinal products in children, adolescents and young adults (AYA) with relapsed/refractory (R/R) ALL and LBL. Sub-protocol C is a phase I/II trial evaluating the safety and efficacy of ruxolitinib and venetoclax in combination with dexamethasone, cyclophosphamide and cytarabine in children and AYA with R/R ped ALL/LBL whose tumor present with alterations in the IL7R/JAK-STAT pathway.

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Extracted eligibility criteria

Treatments studied

Targeted therapy

RuxolitinibVenetoclax

Chemotherapy

CyclophosphamideCytarabine

Other

Dexamethasoneintrathecal chemotherapy

Cancer type

Acute Lymphoblastic Leukemia

Non-Hodgkin Lymphoma

Biomarker criteria

Allowed: CRLF2 rearrangement

CRLF2: Rearrangements and mutations leading to CRLF2 overexpression (P2RY8-CRLF2, IGH-CRLF2, and CRLF2 F232C), CRFL2 overexpression

Allowed: CRLF2 mutation

CRLF2: Rearrangements and mutations leading to CRLF2 overexpression (P2RY8-CRLF2, IGH-CRLF2, and CRLF2 F232C), CRFL2 overexpression

Allowed: CRLF2 overexpression

CRLF2: Rearrangements and mutations leading to CRLF2 overexpression (P2RY8-CRLF2, IGH-CRLF2, and CRLF2 F232C), CRFL2 overexpression

Allowed: EPOR truncating rearrangement

EPOR: Truncating rearrangements or mutations in exon 8, EPOR fusions

Allowed: EPOR mutation in exon 8

EPOR: Truncating rearrangements or mutations in exon 8, EPOR fusions

Allowed: EPOR fusion

EPOR: Truncating rearrangements or mutations in exon 8, EPOR fusions

Allowed: JAK1 missense mutation

JAK1/2/3: Recurrent or novel missense and in-frame indel mutations in or flanking the pseudokinase and kinase domains, JAK fusion

Allowed: JAK1 in-frame indel mutation

JAK1/2/3: Recurrent or novel missense and in-frame indel mutations in or flanking the pseudokinase and kinase domains, JAK fusion

Allowed: JAK2 missense mutation

JAK1/2/3: Recurrent or novel missense and in-frame indel mutations in or flanking the pseudokinase and kinase domains, JAK fusion

Allowed: JAK2 in-frame indel mutation

JAK1/2/3: Recurrent or novel missense and in-frame indel mutations in or flanking the pseudokinase and kinase domains, JAK fusion

Allowed: JAK2 fusion

JAK1/2/3: Recurrent or novel missense and in-frame indel mutations in or flanking the pseudokinase and kinase domains, JAK fusion

Allowed: JAK3 missense mutation

JAK1/2/3: Recurrent or novel missense and in-frame indel mutations in or flanking the pseudokinase and kinase domains, JAK fusion

Allowed: JAK3 in-frame indel mutation

JAK1/2/3: Recurrent or novel missense and in-frame indel mutations in or flanking the pseudokinase and kinase domains, JAK fusion

Allowed: JAK3 fusion

JAK1/2/3: Recurrent or novel missense and in-frame indel mutations in or flanking the pseudokinase and kinase domains, JAK fusion

Allowed: IL7R missense mutation

IL7R: Recurrent or novel missense or in-frame indel mutations in the transmembrane domain; IL7R mutations

Allowed: IL7R in-frame indel mutation

IL7R: Recurrent or novel missense or in-frame indel mutations in the transmembrane domain; IL7R mutations

Allowed: IL7R mutation

IL7R: Recurrent or novel missense or in-frame indel mutations in the transmembrane domain; IL7R mutations

Allowed: SH2B3 copy number deletion

SH2B3: Copy number deletions, or mutations that result in frameshifts or premature termination

Allowed: SH2B3 frameshift mutation

SH2B3: Copy number deletions, or mutations that result in frameshifts or premature termination

Allowed: SH2B3 premature termination mutation

SH2B3: Copy number deletions, or mutations that result in frameshifts or premature termination

Allowed: USP9X truncating mutation

USP9X truncating mutation or USP9X-DDX3X fusion

Allowed: USP9X USP9X-DDX3X fusion

USP9X truncating mutation or USP9X-DDX3X fusion

Allowed: STAT5B mutation

STAT5B and DNM2 mutations

Allowed: DNM2 mutation

STAT5B and DNM2 mutations

Allowed: PTPN2 deletion

PTPN2 deletion described as involved in IL7R/JAK/STAT pathway activation

Demographics

Ages 1–21

Prior therapy

Min 1 prior line

Cannot have received: ruxolitinib and venetoclax in combination (ruxolitinib, venetoclax)

Exception: Patients who have previously received any of these two drugs separately can be eligible for this sub-protocol

Previous treatment with ruxolitinib and venetoclax in combination

Lab requirements

Kidney function

Serum creatinine ≤ 1.5 x ULN for age or calculated creatinine clearance as per the Schwartz formula or radioisotope GFR ≥ 60 mL/min/1.73 m2

Liver function

Direct bilirubin ≤ 2 x ULN (≤ 3.0 × ULN for patients with Gilbert's syndrome); ALT/SGPT ≤ 5 x ULN; AST/SGOT ≤ 5 x ULN. Patients with hepatic dysfunction related to underlying disease may be eligible after discussion with sponsor.

Cardiac function

Shortening fraction (SF) >29% (>35% for children < 3 years) and/or LVEF ≥50% at baseline; Absence of QTcF prolongation (>450 msec on baseline ECG, Friedericia correction), or other clinically significant ventricular or atrial arrhythmia

Adequate organ function: RENAL AND HEPATIC FUNCTION...CARDIAC FUNCTION...

Structured fields extracted by AI. May contain errors — verify against the official protocol.

Frequently asked questions

Is NCT05745714 currently recruiting?

Yes, this trial is currently recruiting patients.

Are there prior therapy exclusions?

Yes. Prior ruxolitinib and venetoclax in combination disqualifies patients from enrollment.

Is there an age limit?

Yes. Patients must be 21 years or younger and at least 1 years old.

Could you qualify for this trial?

Enter your biomarker results to see how this trial's eligibility criteria match your specific cancer profile.

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