Study of efficacy and safety of nilotinib in pediatric CML patients

Phase 1

• Conditions: 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; MedDRA version: 14.1Level: LLTClassification code 10054352Term: Chronic phase chronic myeloid leukemiaSystem Organ Class: 100000004864; Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2013-000200-41-ES
• Lead Sponsor: ovartis Farmacéutica, S.A.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2013-06-13
• Study Completion: 2020-08-28
• Last Update Posted: 2 August 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 59

Inclusion Criteria

Patients eligible for inclusion in this study have to meet all of the following criteria:
1.Male or female patients from 1 year of age to less than 18 years of age at study entry.
2.Patients must have the diagnosis of newly diagnosed and untreated Ph+ CML CP or Ph+ CML CP or AP resistant or intolerant to either imatinib or dasatinib
3.Performance status: Karnofsky ? 50% for patients > 10 years of age, and Lansky ? 50 for patients ? 10 years of age.
4.Patients must have adequate renal, hepatic and pancreatic function
5.Patients must have potassium, magnesium, phosphorus and total calcium values ? LLN (lower limit of normal) or corrected to within normal limits with supplements prior to the first dose of study medication.
Written informed consent must be obtained prior to any screening procedures.
Are the trial subjects under 18? yes
Number of subjects for this age range: 70
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.Female patients of childbearing potential who do not agree to abstinence or, if sexually active, do not agree to the use of contraception as defined in Section 7.2.2.5.5.
2.Patients actively receiving therapy with strong CYP3A4 inhibitors or inducers and the treatment cannot be either discontinued or switched to a different medication at least 14 days prior to starting study drug.
3.Patients who are currently receiving treatment with any medications that have a known risk or possible risk to prolong the QT interval and the treatment cannot be either discontinued or switched to a different medication prior to starting study drug. A list of QT prolonging compounds can be found at http://www.azcert.org/medical-pros/drug-lists/drug-lists.cfm.
4.Acute or chronic liver, pancreatic or severe renal disease considered unrelated to CML.
5.History of pancreatitis within 12 months of starting study drug or past medical history of chronic pancreatitis.
6.Severe and/or uncontrolled concurrent medical disease that in the opinion of the investigator could cause unacceptable safety risks or compromise compliance with the protocol (e.g. uncontrolled diabetes, uncontrolled infection)
7.Impaired cardiac function
8.Patients with a known T315I mutation in BCR-ABL.
9.Previous treatment with more than one TKI for imatinib or dasatinib resistant/intolerant Ph+ CML patients. Previous treatment with any TKI for newly diagnosed Ph+ CML patients.
10.Patients who have received myelosuppressive chemotherapy less than 3 weeks prior to first dose of study drug.
11.Patients who have not recovered from all acute toxicities from all prior myelosuppressive chemotherapy prior to starting study drug.
12.Patients who have received hematopoietic growth factors within 7 days of starting study drug.
13.Patients who have received Pegfilgrastim (Neulasta®) within 14 days of starting study drug.
14.In case of Stem Cell Transplant (SCT) or Rescue without total body irradiation (TBI): Evidence of active graft vs. host disease and < 3 months since SCT.
15.In case of radiation therapy: less than 2 weeks if local palliative, less than 3 months after total body irradiation (TBI), or craniospinal radiation therapy or if at least 50% radiation of pelvis; less than 6 weeks after other substantial BM radiation.
16.Patients with known Hepatitis B, Hepatitis C, or HIV infection.
17.Patients who, in the opinion of the investigator, are unlikely to comply with the protocol or safety monitoring requirements.
18.Patients who are breast feeding
19.Patients who have a known hypersensitivity to the active ingredient or any of the excipients including lactose.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified