Clinical trial · Interventional
Open Label, Phase II Study to Evaluate Efficacy and Safety of Oral Nilotinib in Philadelphia Positive (Ph+) Chronic Myelogenous Leukemia (CML) Pediatric Patients.
A Multi-center, Open Label, Non-controlled Phase II Study to Evaluate Efficacy and Safety of Oral Nilotinib in Pediatric Patients With Newly Diagnosed Ph+ Chronic Myelogenous Leukemia (CML) in Chronic Phase (CP) or With Ph+ CML in CP or Accelerated Phase (AP) Resistant or Intolerant to Either Imatinib or Dasatinib
NCT01844765CI-TRIAL-00051406DIALOGcompletedPhase 2Results postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
To evaluate the safety, efficacy and pharmacokinetics of nilotinib over time in the Ph+ chronic myelogenous leukemia (CML) in pediatric patients (from 1 to \<18 years).
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Philadelphia Positive (Ph+) Chronic Myelogenous Leukemia | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| nilotinib | Drug | Nilotinib | ALIAS |
Design
Arms and outcomes
Arms (3)
- type
- EXPERIMENTAL
- label
- Newly diagnosed and untreated Ph+ CML in first CP
- description
- Diagnosis within 6 months of date of first cytogenetic analysis confirming Philadelphia chromosome with (9;22) translocation by standard conventional cytogenetic analysis.
- interventionNames
- Drug: nilotinib
- type
- EXPERIMENTAL
- label
- Resistant/intolerant Ph+ CML in CP
- description
- Resistant or Intolerant to either imatinib or dasatinib
- interventionNames
- Drug: nilotinib
- type
- EXPERIMENTAL
- label
- Resistant/intolerant Ph+ CML in AP
- description
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 1 Year
- Maximum age
- 17 Years
Show eligibility criteria text
Key Inclusion Criteria: * Newly diagnosed and untreated Ph+ CML CP or Ph+ CML CP or AP resistant or intolerant to either imatinib or dasatinib * Karnofsky ≥ 50% for patients \> 10 years of age and Lansky ≥ 50 for patients ≤ 10 years of age * Adequate renal, hepatic and pancreatic function * Potassium, magnesium, phosphorus and total calcium values ≥ LLN (lower limit of normal) * Written informed consent Key Exclusion Criteria: * Treatment with strong CYP3A4 inhibitors or inducers * Use or planned use of any medications that have a known risk or possible risk to prolong the QT interval * Acute or chronic liver, pancreatic or severe renal disease * History of pancreatitis or chronic pancreatitis. * Impaired cardiac function * No evidence of active graft vs host and \<3mo since Stem Cell Transplant * Total body irradiation (TBI) or craniospinal radiation therapy \<6months * Hypersensitivity to the active ingredient or any of the excipients including lactose. * the criteria regarding pregnancy and contraception * Active or systemic bacterial, fungal, or viral infection * known Hepatitis B, Hepatitis C, or HIV infection
References
Publications (3)
- DERIVEDHijiya N, Maschan A, Rizzari C, Shimada H, Dufour C, Goto H, Kang HJ, Guinipero T, Karakas Z, Bautista F, Ducassou S, Yoo KH, Zwaan CM, Millot F, Patterson BC, Samis J, Izquierdo M, Titorenko K, Li S, Sosothikul D. The long-term efficacy and safety of nilotinib in pediatric patients with CML: a 5-year update of the DIALOG study. Blood Adv. 2023 Dec 12;7(23):7279-7289. doi: 10.1182/bloodadvances.2023010122. PMID 37738125
- DERIVEDHijiya N, Maschan A, Rizzari C, Shimada H, Dufour C, Goto H, Kang HJ, Guinipero T, Karakas Z, Bautista F, Ducassou S, Yoo KH, Zwaan CM, Millot F, Patterson B, Samis J, Aimone P, Allepuz A, Titorenko K, Sosothikul D. A phase 2 study of nilotinib in pediatric patients with CML: long-term update on growth retardation and safety. Blood Adv. 2021 Jul 27;5(14):2925-2934. doi: 10.1182/bloodadvances.2020003759. PMID 34309636
- DERIVEDHijiya N, Maschan A, Rizzari C, Shimada H, Dufour C, Goto H, Kang HJ, Guinipero T, Karakas Z, Bautista F, Ducassou S, Yoo KH, Zwaan CM, Millot F, Aimone P, Allepuz A, Quenet S, Hourcade-Potelleret F, Hertle S, Sosothikul D. Phase 2 study of nilotinib in pediatric patients with Philadelphia chromosome-positive chronic myeloid leukemia. Blood. 2019 Dec 5;134(23):2036-2045. doi: 10.1182/blood.2019000069. PMID 31511239