Actively Recruiting
Safety and Efficacy of Ponatinib for Treatment of Pediatric Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors
Led by Incyte Biosciences International Sàrl · Updated on 2026-04-16
70
Participants Needed
23
Research Sites
417 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and efficacy of ponatinib in children aged 1 to \< 18 years with advanced leukemias, lymphomas, and solid tumors.
CONDITIONS
Official Title
Safety and Efficacy of Ponatinib for Treatment of Pediatric Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis confirmed by tissue or cell examination of leukemia, lymphoma, or other tumors including CNS tumors
- For Phase 1: Includes chronic phase, blast phase, accelerated phase CML, ALL, AML, other leukemias, lymphoma, or other tumors without standard therapy options
- For Phase 2 Group A: Chronic phase CML resistant or intolerant to at least one prior BCR-ABL-targeted therapy or with specific mutations
- For Phase 2 Group B: Leukemias or solid tumors with mutations where ponatinib may be active
- Solid tumors or lymphoma must have measurable disease by CT or MRI
- Prior therapies with specified treatment resistance or progression depending on cancer type
- Karnofsky performance status ≥ 40% for ages 16 and older or Lansky Play Scale ≥ 40% for younger participants
- Recovery to less than Grade 2 or baseline from prior non-blood toxicities except hair loss
- Willingness to avoid pregnancy or fathering children
- Specific timing requirements for prior corticosteroids, chemotherapy, radiation, stem cell transplant, surgery, and immunosuppressive therapies
- No recent investigational anticancer agents or monoclonal antibody therapies within specified timeframes
- Availability of bone marrow aspirate showing BCR-ABL translocation within 42 days before treatment
You will not qualify if you...
- Pancreatitis or evidence of pancreatic disease
- Low heart function or abnormal heart rhythm as measured by tests
- Uncontrolled cardiovascular disease or hypertension
- Use of drugs that prolong QT interval unless safely discontinued or substituted
- Intracranial metastasis or active central nervous system leukemia
- History of significant brain injury, dementia, psychosis, or autoimmune CNS disease
- History of stroke or brain hemorrhage with unresolved deficits
- Uncontrolled seizure disorders
- Significant bleeding disorders not related to the cancer
- Gastrointestinal disorders affecting drug absorption
- Genetic syndromes such as Fanconi anemia or Bloom syndrome
- Down syndrome
- Active graft versus host disease grade 2 or higher
- Active infections requiring systemic treatment
- Active hepatitis B or C infection requiring treatment or risk of reactivation
- Known HIV infection
- Use of prohibited medications
- Known allergy or severe reaction to ponatinib or its ingredients
- Pregnancy or breastfeeding
- Inability or unlikely ability to comply with treatment schedule or study evaluations
- Other exclusions as determined by the study team
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 23 locations
1
Ghent University Hospital
Ghent, Belgium, 09000
Actively Recruiting
2
Hopital Robert Debre
Paris, France, 75019
Actively Recruiting
3
Armand Trousseau Hospital
Paris, France, 75571
Actively Recruiting
4
Centre Hospitalier Universitaire de Poitiers
Poitiers, France, 86021
Actively Recruiting
5
Chu de Rennes - Hospital Sud
Rennes, France, 35700
Actively Recruiting
6
Aou Policlinico S. Orsola-Malpighi
Bologna, Italy, 40138
Actively Recruiting
7
Asst Degli Spedali Civili Di Brescia
Brescia, Italy, 25123
Actively Recruiting
8
Ospedale Pediatrico G. Gaslini
Genova, Italy, 16147
Actively Recruiting
9
Comitato Etico Fondazione Irccs Istituto Nazionale Dei Tumori Milano
Milan, Italy, 20133
Actively Recruiting
10
University of Milano Bicocca
Monza, Italy, 20900
Actively Recruiting
11
Aorn Santobono Pausilipon
Naples, Italy, 80122
Not Yet Recruiting
12
Comitato Di Bioetica Della Fondazione Irccs Policlinico San Matteo
Pavia, Italy, 27100
Actively Recruiting
13
Ospedale Pediatrico Bambino Gesu Irccs
Rome, Italy, 00165
Actively Recruiting
14
A.O.U Citta Della Salute E Della Scienza Di Torino Presidio Ospedaliero Infantile Regina Margherita
Torino, Italy, 10126
Actively Recruiting
15
Princess Maxima Center For Pediatric Oncology
Utrecht, Netherlands, 03584
Actively Recruiting
16
Hospital General Universitario Vall D Hebron
Barcelona, Spain, 08035
Actively Recruiting
17
Hospital Sant Joan de Deu de Manresa
Barcelona, Spain, 08035
Actively Recruiting
18
Hospital Infantil Unversitario Nino Jesus
Madrid, Spain, 28009
Actively Recruiting
19
Hospital Universitari I Politecnic La Fe
Valencia, Spain, 46026
Actively Recruiting
20
Karolinska University Hospital Solna
Stockholm, Sweden, 14141
Actively Recruiting
21
Royal Hospital For Sick Children Yorkhill Glasgow
Glasgow, United Kingdom, G514TF
Actively Recruiting
22
Alder Hey Childrens Nhs Foundation Trust
Liverpool, United Kingdom, L12 2AP
Actively Recruiting
23
The Royal Marsden Nhs Foundation Trust - Sutton
Sutton, United Kingdom, SM2 5PT
Actively Recruiting
Research Team
I
Incyte Corporation Call Center (ex-US)
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here