Clinical trial • Phase II • Oncology

RUXOLITINIB for Acute lymphoblastic leukemia

Phase II trial of RUXOLITINIB for Acute lymphoblastic leukemia. open-label. 25 participants.

Overview

Trial Therapeutic Area
Oncology
Trial Disease
Acute lymphoblastic leukemia
Trial Stage
Phase II
Drug Modality
Small molecule|Peptide/protein/enzyme
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
29-09-2024
First CTIS Authorization Date
18-11-2024

Trial design

open-label Phase II trial in Poland.

Open Label
Yes
Real World Control
Yes
Biomarker Stratified
True, JAK/STAT pathway activation (CRLF2, JAK2, EPOR, or CRLF2 expression on leukemic cells)
Target Sample Size
25

Eligibility

Recruits 25 paediatric patients.

Vulnerable Population
Children are included (isVulnerablePopulationSelected=true). Informed consent is from parents/legal guardians; age-appropriate subject information/assent documents are provided (documents: L1_IS parents_legal guardians and ICF; L1_SIS 16-18 yr; L1_SIS 11-15 yr; L1_SIS under 11 yr) to handle assent/consent according to participant age.

Inclusion criteria

  • {"criterion_text":"- Newly diagnosed ALL treated according to AIEOP-BFM 2017 Poland or AIEOP-BFM 2017 standard of care protocol.\n- Confirmed genetic lesion causing activation of JAK-STAT pathway (CRLF2, JAK2, EPOR, or CRLF2 expression on leukemic cells`surface).\n- Stratification as early high risk: - no complete remission on day 33 OR - positivity for KMT2A-AFF1 OR - positivity for TCF3-HLF OR o hypodiploidy <45 chromosomes OR - FCM-MRD in bone marrow on day 15 ≥ 10% and not ETV6-RUNX1 positive OR - IKZF1plus and PCR-MRD at TP1 positive or inconclusive and not positive for ETV6-RUNX1, TCF3-PBX1 or KMT2A rearr. other than KMT2A-AFF1 OR - PCR-MRD at TP1 ≥ 5x10-4 OR -age < 1 year and any KMT2A rearrangement"}

Exclusion criteria

  • {"criterion_text":"- ALL classified as a standard or intermediate risk (SR, MR).\n- Early high risk (eHR) ALL without genetic lesions within CRLF2, JAK2, EPOR, or CRLF2 expression on leukemic cells.\n- Participation in another clinical trial except for ad-on trials within the scope of supportive care approved by the sponsor\n- Other condition (either pre-existing or related to leukemia biology as present at diagnosis) or circumstances that significantly conflict with the treatment according to the protocol"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Proportion of patients with MRD(-) at TP2.","definition_or_measurement_approach":"Measured as the proportion of participants who are minimal residual disease negative (MRD(-)) at timepoint TP2."}

Secondary endpoints

  • {"endpoint_text":"- Frequency and grading of adverse events in Consol. IB ext. phase (number, percentage, number per patient-days, number per each grade of significance).","definition_or_measurement_approach":"Recorded and reported as number, percentage, number per patient-days, and counts by grade according to standard AE grading."}
  • {"endpoint_text":"- Incidence of treatment-related adverse and severe adverse events.","definition_or_measurement_approach":"Incidence (counts and rates) of adverse events and serious adverse events judged related to treatment."}

Recruitment

Planned Sample Size
25
Recruitment Window Months
97
Consent Approach
Informed consent from parents/legal guardians; age-specific subject information and assent processes are provided (documents for parents/legal guardians and separate SIS/ICF for ages 16-18, 11-15, and under 11). Languages not specified in available data.

Geography

Total Number Of Sites
15
Total Number Of Participants
25

Poland

Earliest CTIS Part Ii Submission Date
11-10-2024
Latest Decision Or Authorization Date
04-04-2025
Processing Time Days
175
Number Of Sites
15
Number Of Participants
25

Sites

Site Name
Uniwersytecki Dzieciecy Szpital Kliniczny Im. L. Zamenhofa W Bialymstoku
Department Name
Klinika Pediatrii, Onkologii i Hematologii
Contact Person Name
Maryna Krawczuk-Rybak
Site Name
Uniwersytecki Szpital Kliniczny Nr 1 Im. Prof. Tadeusza Sokolowskiego Pum W Szczecinie
Department Name
Klinika Pediatrii, Hemato-Onkologii i Gastroenterologii Dziecięcej
Contact Person Name
Tomasz Urasiński
Contact Person Email
urasin@pum.edu.pl
Site Name
Uniwersyteckie Centrum Kliniczne
Department Name
Klinika Pediatrii, Hematologii i Onkologii
Contact Person Name
Ninela Irga-Jaworska
Contact Person Email
nirga@gumed.edu.pl
Site Name
Wojewodzki Specjalistyczny Szpital Dzieciecy Im. Prof. Dr Stanislawa Popowskiego W Olsztynie Sp. z o.o.
Department Name
Oddział Kliniczny Onkologii i Hematologii Dziecięcej
Contact Person Name
Wanda Badowska
Contact Person Email
hematologia@wssd.olsztyn.pl
Site Name
Swietokrzyskie Centrum Onkologii Samodzielny Publiczny Zaklad Opieki Zdrowotnej W Kielcach
Department Name
II Klinika Pediatrii - Oddział Onkologii i Hematologii Dziecięcej
Contact Person Name
Grażyna Karolczyk
Site Name
Uniwersytecki Szpital Dzieciecy W Lublinie
Department Name
Klinika Hematologii, Onkologii i Transplantologii Dziecięcej
Contact Person Name
Jerzy Kowalczyk
Contact Person Email
jerzy.kowalczyk@uszd.lublin.pl
Site Name
Samodzielny Publiczny Szpital Kliniczny Nr 1 Im.Prof.Stanislawa Szyszko Slaskiego Uniwersytetu Medycznego W Katowicach
Department Name
Katedra i Klinika Pediatrii, Hematologii i Onkologii Dziecięcej
Contact Person Name
Tomasz Szczepański
Contact Person Email
szczep57@poczta.onet.pl
Site Name
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Centralny Szpital Kliniczny Uniwersytetu Medycznego W Lodzi
Department Name
Klinika Pediatrii, Onkologii i Hematologii
Contact Person Name
Wojciech Młynarski
Site Name
Kliniczny Szpital Wojewodzki Nr 2 Im. Sw. Jadwigi Krolowej W Rzeszowie
Department Name
Klinika Onkohematologii Dziecięcej
Contact Person Name
Radosław Chaber
Contact Person Email
rchaber@ur.edu.pl
Site Name
Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu
Department Name
Klinika Transplantacji Szpiku, Onkologii i Hematologii Dziecięcej
Contact Person Name
Krzysztof Kałwak
Contact Person Email
krzysztof.kalwak@gmail.com
Site Name
Szpital Uniwersytecki Nr 1 Im. Dr. A. Jurasza W Bydgoszczy
Department Name
Katedra Pediatrii, Hematologii i Onkologii
Contact Person Name
Jan Styczyński
Contact Person Email
jstyczynski@cm.umk.pl
Site Name
Uniwersytecki Szpital Dzieciecy W Krakowie
Department Name
Klinika Onkologii i Hematologii Dziecięcej
Contact Person Name
Szymon Skoczeń
Contact Person Email
sskoczen@usdk.pl
Site Name
Samodzielny Publiczny Dzieciecy Szpital Kliniczny Im. Jozefa Polikarpa Brudzinskiego W Warszawie
Department Name
Oddział Kliniczny Pediatrii, Hematologii i Onkologii
Contact Person Name
Paweł Łaguna
Contact Person Email
plaguna@wum.edu.pl
Site Name
Gornoslaskie Centrum Zdrowia Dziecka Im. Sw. Jana Pawla II Samodzielny Publiczny Szpital Kliniczny Nr 6 Slaskiego Uniwersytetu Medycznego W Katowicach
Department Name
Oddział Onkologii, Hematologii i Chemioterapii, Klinika Pediatrii
Contact Person Name
Agnieszka Mizia-Malarz
Contact Person Email
a.mizia@wp.pl
Site Name
Szpital Kliniczny Im. Karola Jonschera Uniwersytetu Medycznego Im. Karola Marcinkowskiego W Poznaniu
Department Name
Klinika Onkologii, Hematologii i Transplantologii Pediatrycznej
Contact Person Name
Katarzyna Derwich
Contact Person Email
kderwich123@gmail.com

Sponsor

Primary sponsor

Full Name
Medical University Of Lodz
Organisation Type
Educational Institution
Country Of Registered Address
Poland

Investigational products

Investigational Product Name
RUXOLITINIB
Active Substance
RUXOLITINIB
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Maximum Dose
80 mg/m2 (max daily dose amount provided in record)
Combination Treatment
Yes

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