BridgeMD
Tests treatment safety and results for Acute Myeloid Leukemia
Official title Safety and Efficacy of Ponatinib for Treatment of Pediatric Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors
ClinicalTrials.gov ID: NCT03934372
What this study is testing
What is Ponatinib?
Ponatinib is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for acute myeloid leukemia.
Also referred to as Iclusig, INCB84344.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 17
You may be able to join if
- Histologically or cytologically confirmed diagnosis of the following malignancies: \- Phase 1: CP-CML, BP-CML, AP-CML ALL. AML. Other leukemias...
- Karnofsky performance status ≥ 40% for participants ≥ 16 years old or Lansky Play Scale ≥ 40% for pediatric participants \< 16 years old.
- Participants must have recovered to \< Grade 2 per the NCI CTCAE v5.0 or to baseline from any non-hematologic toxicities (except alopecia) due to...
- Willingness to avoid pregnancy or fathering children.
You likely can't join if
- Prior therapies: \- Participants with BP-CML, ALL, or AML who have received any of the following: Corticosteroids or hydroxyurea within 24 hours...
- Immunosuppressive therapy (including post stem cell transplant regimens) within 14 days before the first dose of ponatinib.
- Any targeted cancer therapy (including TKIs) within 7 days before the first dose of ponatinib.
- Any other investigational anticancer agents within 30 days or 5 half-lives, whichever is longer, before randomization.
- Any monoclonal antibody-directed anticancer therapy within 5 half-lives of the first dose of ponatinib.
- Any chimeric antigen receptor therapy within 28 days before the first dose of ponatinib
See the full eligibility criteria
- Histologically or cytologically confirmed diagnosis of the following malignancies: \- Phase 1: CP-CML, BP-CML, AP-CML ALL. AML. Other leukemias. Lymphoma. Any other tumors, including tumors of the CNS, for which...
- Karnofsky performance status ≥ 40% for participants ≥ 16 years old or Lansky Play Scale ≥ 40% for pediatric participants \< 16 years old.
- Participants must have recovered to \< Grade 2 per the NCI CTCAE v5.0 or to baseline from any non-hematologic toxicities (except alopecia) due to previous therapy.
- Willingness to avoid pregnancy or fathering children.
- Prior therapies: \- Participants with BP-CML, ALL, or AML who have received any of the following: Corticosteroids or hydroxyurea within 24 hours before the first dose of ponatinib. Vincristine within 7 days before the...
- Immunosuppressive therapy (including post stem cell transplant regimens) within 14 days before the first dose of ponatinib.
- Any targeted cancer therapy (including TKIs) within 7 days before the first dose of ponatinib.
- Any other investigational anticancer agents within 30 days or 5 half-lives, whichever is longer, before randomization.
- Any monoclonal antibody-directed anticancer therapy within 5 half-lives of the first dose of ponatinib.
- Any chimeric antigen receptor therapy within 28 days before the first dose of ponatinib
- Ponatinib
- Protocol-defined lab Values
- Significant concurrent, uncontrolled medical condition, including but not limited to the following:
- Pancreatic: clinical, radiological, or laboratory evidence of pancreatitis.
- Cardiac:
- SF \< 27% by ECHO, OR EF \< 50% by MUGA.
- Abnormal QTcF on screening ECG, defined as QTcF of ≥ 450 ms.
- Clinically significant or uncontrolled cardiovascular disease, including unstable angina, acute MI within 6 months from Day 1 of study drug administration, New York Heart Association Class III or IV CHF (see Appendix...
- Uncontrolled hypertension.
- Currently taking drug(s) that are known to have a risk of causing prolonged QTc or TdP unless the drug(s) can be changed to acceptable alternatives (ie, an alternate class of agents that do not affect the cardiac...
- Cerebral:
- Participants with solid tumors with intracranial metastasis OR participants with active CNS leukemia (ie, CNS-2 status [\< 5/μL WBCs and cytospin positive for blasts, or ≥ 5 /μL WBCs but negative by Steinherz/Bleyer...
- Pre-existing significant CNS pathology including history of severe brain injury, dementia, cerebellar disease, organic brain syndrome, psychosis, coordination/movement disorder, or autoimmune disease with CNS...
- History of cerebrovascular ischemia/hemorrhage with residual deficits.
- Note: Participants with a history of cerebrovascular ischemia/hemorrhage remain eligible provided all neurologic deficits have resolved clinically according to Inclusion Criterion 6.
- Uncontrolled seizure disorder.
- Coagulation:
- Significant bleeding disorder or thrombophilia unrelated to the underlying malignancy indication for study participation.
- Gastrointestinal:
- Gastrointestinal disorders, such as malabsorption syndrome or any other illness that could affect oral absorption.
- Genetic:
- Participants with DNA fragility syndromes, such as Fanconi anemia and Bloom syndrome.
- Participants with Down syndrome.
- Participants with any active ≥ Grade 2 graft versus host disease.
- Chronic or current active uncontrolled infectious disease requiring systemic antibiotics, antifungal, or antiviral treatment.
- Active HBV or HCV infection that requires treatment or at risk for HBV reactivation. Hepatitis B virus DNA and HCV RNA must be undetectable upon testing. At risk for HBV reactivation is defined as hepatitis B surface...
- Known HIV infection.
- Current use of prohibited medication (see Section 6.7.2).
- Known hypersensitivity or severe reaction to ponatinib or excipients of ponatinib.
- Receipt of live (including attenuated) vaccines or anticipation of need for such vaccines during the study.
- Inability or unlikeliness to comply with the dose schedule and study evaluations, in the opinion of the investigator.
- Females who are pregnant or lactating.
- Other exclusions may apply.
The study team makes the final eligibility decision.
Where it's taking place
- Ghent, Belgium
- Paris, France
- Poitiers, France
- Rennes, France
- Bologna, Italy
- Brescia, Italy
- Genova, Italy
- Milan, Italy
- Monza, Italy
- Naples, Italy
- Pavia, Italy
- Rome, Italy
- Torino, Italy
- Utrecht, Netherlands
- Barcelona, Spain
- Madrid, Spain
- Valencia, Spain
- Stockholm, Sweden
- Glasgow, United Kingdom
- Liverpool, United Kingdom
+ 1 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 year to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Ghent, Belgium; Paris, France; Poitiers, France; Rennes, France; Bologna, Italy; Brescia, Italy and 15 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.