Open Label, Phase II Study to Evaluate Efficacy and Safety of Oral Nilotinib in Philadelphia Positive (Ph+) Chronic Myelogenous Leukemia (CML) Pediatric Patients. (DIALOG)

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

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).

Study Overview

• Status: Completed
• Conditions: Philadelphia Positive (Ph+) Chronic Myelogenous Leukemia
• Intervention / Treatment: Drug: nilotinib

Detailed Description

The study was designed as a multi-center, open-label, non-controlled phase II study to assess efficacy, safety and PK parameters of 230 mg/m2 twice daily nilotinib in pediatric patients (1 to <18 years old). The study population consisted of three cohorts of Ph+ CML pediatric patients:

• Cohort 1: Ph+ CML-CP patients resistant or intolerant to either imatinib or dasatinib
• Cohort 2: Ph+ CML-AP patients resistant or intolerant to either imatinib or dasatinib
• Cohort 3: Newly-diagnosed Ph+ CML-CP patients in first chronic phase A minimum number of 50 pediatric patients (from 1 to <18 years) were enrolled in the study. Of them, at least 15 patients were Ph+ CML-CP patients resistant or intolerant to either imatinib or dasatinib, and at least 15 were newly-diagnosed Ph+ CML-CP patients in first chronic phase patients. There was no minimum number of patients required for Ph+ CML-AP patients resistant or intolerant to either imatinib or dasatinib.

Based on enrollment forecasts as of Jan 2015, and to reflect the agreements with the US FDA and the PDCO, the study remained open for enrollment until the targeted number of 50 patients with at least 15 newly diagnosed Ph+CML patients was achieved or until 31May2015, whichever was later.

Patients who completed the study were treated with nilotinib for a total of 66 cycles of 28 days unless the patient prematurely discontinued study treatment.

The primary analysis cut-off date was the date when all patients enrolled in the trial either completed their visit for treatment cycle 12 or had discontinued study treatment early (EoT/early discontinuation visit). These analyses were reported in the 12-cycle clinical study report (CSR). A 24-cycle analysis was done when all patients had either completed their 24-cycle treatment visit or had discontinued study treatment early.

At trial end, a final comprehensive CSR of all data collected during the trial was produced.

• Study Type: Interventional
• Enrollment (Actual): 59
• Phase: Phase 2

Expanded Access

Available outside the clinical trial. See expanded access record.

Contacts and Locations

Study Locations

• France
  • Lille, France, 59000
    • Novartis Investigative Site
  • Paris Cedex, France, 75019
    • Novartis Investigative Site
  • Poitiers, France, 86021
    • Novartis Investigative Site
  • Aquitaine
    • Bordeaux, Aquitaine, France, 33076
      • Novartis Investigative Site
• Hungary
  • Budapest, Hungary, 1094
    • Novartis Investigative Site
• Italy
  • GE
    • Genova, GE, Italy, 16147
      • Novartis Investigative Site
  • MB
    • Monza, MB, Italy, 20900
      • Novartis Investigative Site
  • PD
    • Padova, PD, Italy, 35128
      • Novartis Investigative Site
  • TO
    • Torino, TO, Italy, 10126
      • Novartis Investigative Site
• Japan
  • Saitama, Japan, 330 8777
    • Novartis Investigative Site
  • Shizuoka, Japan, 420 8660
    • Novartis Investigative Site
  • Kanagawa
    • Yokohama-city, Kanagawa, Japan, 232-8555
      • Novartis Investigative Site
  • Kyoto
    • Sakyo Ku, Kyoto, Japan, 606 8507
      • Novartis Investigative Site
  • Tokyo
    • Shinjuku-ku, Tokyo, Japan, 160 8582
      • Novartis Investigative Site
• Korea, Republic of
  • Seoul, Korea, Republic of, 03080
    • Novartis Investigative Site
  • Seoul, Korea, Republic of, 06351
    • Novartis Investigative Site
• Malaysia
  • Kuala Lumpur, Malaysia, 50589
    • Novartis Investigative Site
• Netherlands
  • Rotterdam, Netherlands, 3015 CE
    • Novartis Investigative Site
• Russian Federation
  • Moscow, Russian Federation, 117198
    • Novartis Investigative Site
• Spain
  • Madrid, Spain, 28009
    • Novartis Investigative Site
• Thailand
  • Bangkok, Thailand, 10330
    • Novartis Investigative Site
  • Bangkok, Thailand, 10700
    • Novartis Investigative Site
  • Chiangmai
    • Muang, Chiangmai, Thailand, 50200
      • Novartis Investigative Site
• Turkey
  • Istanbul, Turkey, 34093
    • Novartis Investigative Site
• United Kingdom
  • Bristol, United Kingdom, BS2 8BJ
    • Novartis Investigative Site
  • Surrey
    • Sutton, Surrey, United Kingdom, SM2 5PT
      • Novartis Investigative Site
• United States
  • California
    • Loma Linda, California, United States, 92350
      • Loma Linda University Cancer Center
    • Palo Alto, California, United States, 94304
      • Lucile Salter Packard Children's Hospital at Stanford
  • Florida
    • Orlando, Florida, United States, 32827
      • Nemours Childrens Hospital
    • West Palm Beach, Florida, United States, 33407
      • St. Mary's Hospital
  • Maryland
    • Baltimore, Maryland, United States, 21231
      • Johns Hopkins Oncology Center ORA
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • UNC Chapel Hill
  • Ohio
    • Columbus, Ohio, United States, 43205
      • Nationwide Childrens Hospital
  • Texas
    • Dallas, Texas, United States, 75235
      • University of Texas Southwestern Medical Center Oncology
    • Fort Worth, Texas, United States, 76104
      • Cook Children's Medical Center Oncology
  • Washington
    • Seattle, Washington, United States, 98105
      • Seattle Childrens Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 17 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NON_RANDOMIZED
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Newly diagnosed and untreated Ph+ CML in first CP; Diagnosis within 6 months of date of first cytogenetic analysis confirming Philadelphia chromosome with (9;22) translocation by standard conventional cytogenetic analysis.	Drug: nilotinib; Nilotinib supplied in 50mg, 150mg, and 200mg capsules. It was administered orally at 230mg/m2, twice daily for up to 66 cycles (1 cycle = 28 days). Dose administration was rounded to the nearest 50mg dose (to a maximum dose of 400mg).; Other Names: AMN107
EXPERIMENTAL: Resistant/intolerant Ph+ CML in CP; Resistant or Intolerant to either imatinib or dasatinib	Drug: nilotinib; Nilotinib supplied in 50mg, 150mg, and 200mg capsules. It was administered orally at 230mg/m2, twice daily for up to 66 cycles (1 cycle = 28 days). Dose administration was rounded to the nearest 50mg dose (to a maximum dose of 400mg).; Other Names: AMN107
EXPERIMENTAL: Resistant/intolerant Ph+ CML in AP; Resistant or intolerant to either imatinib or dasatinib - at the end no patients were enrolled in this arm.	Drug: nilotinib; Nilotinib supplied in 50mg, 150mg, and 200mg capsules. It was administered orally at 230mg/m2, twice daily for up to 66 cycles (1 cycle = 28 days). Dose administration was rounded to the nearest 50mg dose (to a maximum dose of 400mg).; Other Names: AMN107

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Rate of Major Molecular Response (MMR) at 6 Cycles for Ph+ CML CP Patients Resistant or Intolerant to Imatinib or Dasatinib	MMR is defined as ≤ 0.1% BCR-ABL/control gene (ABL) % by international scale, or equivalent to ≥ 3 log reduction of BCR-ABL transcript from standardized baseline, measured by RQ-PCR (Real time quantitative polymerase chain reaction). BCR-ABL is the fusion gene from breakpoint cluster region and Abelson genes. A patient was counted as having MMR at 6 cycles if the patient met the MMR criteria at the Cycle 6 Visit.	6 cycles
MMR Rate by 12 Cycles in Newly Diagnosed Ph+ CML-CP Patients	MMR is defined as ≤ 0.1% BCR-ABL/control gene (ABL) % by international scale, or equivalent to ≥ 3 log reduction of BCR-ABL transcript from standardized baseline, measured by RQ-PCR (Real time quantitative polymerase chain reaction). BCR-ABL is the fusion gene from breakpoint cluster region and Abelson genes. A patient was counted as having MMR by 12 cycles if the patient met the MMR criteria at least once at any time between first study drug intake and Cycle 12 visit included.	12 cycles
Rate of Complete Cytogenic Response (CCyR) at 12 Cycles in Newly Diagnosed Ph+ CML-CP Patients	Cytogenetic response is assessed as the percentage of Philadelphia positive (Ph+) metaphases in the bone marrow. Complete Cytogenetic Response (CCyR) is defined as 0% of Ph+ metaphases. A patient was counted as CCyR at 12 cycles if the patient met the CCyR criteria at the Cycle 12 Visit.	12 cycles

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
MMR Rate by Time Points in Ph+ CML-CP Patients Resistant or Intolerant to Imatinib or Dasatinib	Major molecular response (MMR) was defined as BCR-ABL/ABL % ≤ 0.1% by IS as measured by RQ-PCR, confirmed by duplicate analysis of the same sample.	By 3, 6, 9 , 12, 24, 36, 48, 66 cycles ( 1 cycle = 28 days)
MMR Rate by Time Points in Newly Diagnosed Ph+ CML-CP Patients	Major molecular response (MMR) was defined as BCR-ABL/ABL % ≤ 0.1% by IS as measured by RQ-PCR, confirmed by duplicate analysis of the same sample.	by 3, 6, 9, 12, 24, 36, 48, 66 cycles (1 cycle = 28 days)
Best BCR-ABL Ratio Categories for Resistant/Intolerant Ph+ CML - Overall	MMR is defined as ≤ 0.1% BCR-ABL/control gene (ABL) % by international scale, or equivalent to ≥ 3 log reduction of BCR-ABL transcript from standardized baseline, measured by RQ-PCR (Real time quantitative polymerase chain reaction). BCR-ABL is the fusion gene from breakpoint cluster region and Abelson genes. BCR-ABL ratio by percentage: > 0.0032 to ≤ 0.01% is equal to a log reduction category of >= 4 to <4.5 -log reduction (MR4); BCR-ABL ratio by percentage: <=0.0032% is equal to a log reduction category of >= 4.5-log reduction (MMR4.5)	up to 66 cycles (1 cycle = 28 days)
Best BCR-ABL Ratio Categories for Newly Diagnosed Ph+ CML-CP - Overall	MMR is defined as ≤ 0.1% BCR-ABL/control gene (ABL) % by international scale, or equivalent to ≥ 3 log reduction of BCR-ABL transcript from standardized baseline, measured by RQ-PCR (Real time quantitative polymerase chain reaction). BCR-ABL is the fusion gene from breakpoint cluster region and Abelson genes. BCR-ABL ratio by percentage: > 0.0032 to ≤ 0.01% is equal to a log reduction category of >= 4 to <4.5 -log reduction (MR4); BCR-ABL ratio by percentage: <=0.0032% is equal to a log reduction category of >= 4.5-log reduction (MMR4.5)	up to 66 cycles (1 cycle = 28 days)
Time to First MMR Among Imatinib or Dasatinib Resistant or Intolerant CML-CP Patients Who Achieved MMR	Time from first study drug intake to first MMR amongst imatinib or dasatinib resistant or intolerant patients with CML-CP computed only for patients who achieved MMR.	From first dosing to the first MMR within 66 cycles period
Time to First MMR Among Newly Diagnosed Ph+ CML-CP Patients Who Achieved MMR	Time to MMR is the time from first study drug intake to first major molecular response computed only for participants who achieved MMR.	From first dosing to the first MMR within 66 cycles period
Duration of First MMR Among Patients Who Were Resistant or Intolerant to Either Imatinib or Dasatinib Who Achieved MMR	Duration of MMR is defined as the time between the date of the first MMR and the date of confirmed loss of MMR (i.e. the earliest of confirmed loss of MMR, CML-related death or progression to AP or BC). Participants without loss of MMR were censored at the last molecular assessment date.	from MMR until confirmed loss of MMR (Assessed up to 66 cycles)
Duration of First MMR Among Newly Diagnosed Patients Who Achieved MMR	Duration of MMR is defined as the time between the date of the first MMR and the date of confirmed loss of MMR (i.e. the earliest of confirmed loss of MMR, CML-related death or progression to AP or BC). Participants without loss of MMR were censored at the last molecular assessment date.	from MMR until confirmed loss of MMR (Assessed up to 66 cycles)es)
Best Complete Cytogenetic Response (CCyR) Categories in Ph+ CML-CP Patients Resistant or Intolerant to Imatinib or Dasatinib - Overall	Complete cytogenetic response (CCyR) - 0% Ph+ metaphases; Partial cytogenetic response (PCyR) - >0 to 35% Ph+ metaphases; Minor cytogenetic response (mCyR) - >35 to 65% Ph+ metaphases; Minimal - >65 to 95% Ph+ metaphases; None - >95 to 100% Ph+ metaphases; Major cytogenetic response (MCyR) - 0 to 35% Ph+ metaphases. A major response combines both complete and partial responses.	up to 66 cycles
Best Complete Cytogenetic Response (CCyR) in Newly Diagnosed Ph+ CML-CP Patients - Overall	Complete cytogenetic response (CCyR) - 0% Ph+ metaphases No response - >95 to 100% Ph+ metaphases	up to 66 cycles
Summary of Time to First Complete Cytogenic Response (CCyR) in Newly Diagnosed Ph+ CML-CP Patients	Cytogenetic response is assessed as the percentage of Philadelphia positive (Ph+) metaphases in the bone marrow. Complete Cytogenetic Response (CCyR) is defined as 0% of Ph+ metaphases. A patient was counted as having CCyR by 6 cycles (respectively 12 cycles) if the patient met the CCyR criteria at least once at any time between first study drug intake and cycle 6 (cycle 12 respectively) visit included.	From first dosing to the first CCyR up to 66 cycles
Kaplan-Meier Estimates of Time to First Complete Cytogenic Response (CCyR) in Newly Diagnosed Ph+ CML-CP Patients	Cytogenetic response is assessed as the percentage of Philadelphia positive (Ph+) metaphases in the bone marrow. Complete Cytogenetic Response (CCyR) is defined as 0% of Ph+ metaphases. A patient was counted as having CCyR by 6 cycles (respectively 12 cycles) if the patient met the CCyR criteria at least once at any time between first study drug intake and cycle 6 (cycle 12 respectively) visit included.	From first dosing to the first CCyR up to 66 cycles
Kaplan-Meier Estimates of Duration of First Complete Cytogenic Response (CCyR) Among Patients Who Achieved CCyR in Newly Diagnosed Ph+ CML-CP Patients	Cytogenetic response is assessed as the percentage of Philadelphia positive (Ph+) metaphases in the bone marrow. Complete Cytogenetic Response (CCyR) is defined as 0% of Ph+ metaphases. A patient was counted as having CCyR by 6 cycles (respectively 12 cycles) if the patient met the CCyR criteria at least once at any time between first study drug intake and cycle 6 (cycle 12 respectively) visit included.	From CCyR to loss of CCyR up to 66 cycles
Best Major Cytogenetic Response (MCyR) Rate by Time Point in Newly Diagnosed Ph+ CML Patients	Major cytogenetic response (MCyR) - 0 to 35% Ph+ metaphases. A major response combines both complete and partial responses.	6, 12, 18, 24, 36, 48, 66 cycles
Summary of Time to First Major Cytogenetic Response (MCyR) Among Patients Who Achieved MCyR in Newly Diagnosed CML-CP Patients	Major cytogenetic response (MCyR) - 0 to 35% Ph+ metaphases. A major response combines both complete and partial responses.	up to 66 cycles
Kaplan-Meier Estimates of Time to First Major Cytogenetic Response (MCyR) in Newly Diagnosed CML-CP Patients	Major cytogenetic response (MCyR) - 0 to 35% Ph+ metaphases. A major response combines both complete and partial responses.	up to 66 cycles
Best Complete Hematological Response (CHR) by Time Point	Complete Hematological Response (CHR) was defined as; WBC count <10×109/L; platelet count <450×109/L; basophils <5%; no blasts and promyelocytes in peripheral blood; myelocytes+metamyelocytes <5% in peripheral blood; no evidence of extramedullary disease, including spleen and liver; Assessment confirmation after at least 4 weeks for newly diagnosed Ph+ CML-CP	cycle 3, 6, 9, 12, 18, 24, 36, 48, 66
Summary of Time to First Complete Hematological Response (CHR) Among Patients Who Achieved Confirmed CHR in Newly Diagnosed CML-CP Patients	Complete Hematological Response (CHR) was defined as; WBC count <10×109/L; platelet count <450×109/L; basophils <5%; no blasts and promyelocytes in peripheral blood; myelocytes+metamyelocytes <5% in peripheral blood; no evidence of extramedullary disease, including spleen and liver; Assessment confirmation after at least 4 weeks for newly diagnosed Ph+ CML-CP	from first dosing to CHR, UP TO 66 CYCLES
Kaplan-Meier Estimates of Time to First Complete Hematological Response (CHR) in Newly Diagnosed CML-CP Patients	Complete Hematological Response (CHR) was defined as; WBC count <10×109/L; platelet count <450×109/L; basophils <5%; no blasts and promyelocytes in peripheral blood; myelocytes+metamyelocytes <5% in peripheral blood; no evidence of extramedullary disease, including spleen and liver; Assessment confirmation after at least 4 weeks for newly diagnosed Ph+ CML-CP	from first dosing to CHR, UP TO 66 CYCLES
Time to Disease Progression for Imatinib or Dasatinib Resistant or Intolerant CML-CP Patients - Kaplan-Meier Estimates	Time to disease progression is the time from the date of first study drug intake to the date of event defined as the first progression to AP or BC (from CP) or to BC (from AP) or the date of CML-related death occurring on treatment, whichever was earlier.	From first dosing to the disease progression within 66 cycles
Event Free Survival in Imatinib/Dasatinib Resistant/Intolerant CML-CP Patients	Event Free Survival is defined as the time from the date of first study drug intake to the first occurrence of any of the following loss of CHR, loss of MCyR ( PCyR + CCyR), progression to AP/BC (from CP) or to BC (from AP), or death from any cause. (Including events only during treatment)	From first dosing to the disease progression or death up to 66 cycles
Event Free Survival in Newly Diagnosed CML-CP Patients	Event Free Survival is defined as the time from the date of first study drug intake to the first occurrence of any of the following loss of CHR, loss of MCyR ( PCyR + CCyR), progression to AP/BC (from CP) or to BC (from AP), or death from any cause. (Including events only during treatment)	From first dosing to the disease progression or death up to 66 cycles
Overall Survival (OS) in Imatinib/Dasatinib Resistant/Intolerant CML-CP - Kaplan-Meier Estimates	Overall survival is defined as the time from the date of first study drug intake to the date of death due to any cause. If a patient is not known to have died, survival will be censored at the date of their last assessment for patients on study and date of last contact for patients in follow-up.	from first dosing to death up to 66 cycles
Overall Survival (OS) in Newly Diagnosed CML-CP Patients	Overall survival is defined as the time from the date of first study drug intake to the date of death due to any cause. If a patient is not known to have died, survival will be censored at the date of their last assessment for patients on study and date of last contact for patients in follow-up.	from first dosing to death up to 66 cycles
Pharmacodynamics (BCR-ABL Transcript Levels Determined With Standard Protocols in Peripheral Blood): Best MMR Status by Cycle	BCR-ABL is the fusion gene from breakpoint cluster region and Abelson genes. BCR-ABL transcript levels were summarized by cohort and time point. MMR is defined as ≤ 0.1% BCR-ABL/control gene (ABL) % by international scale, or equivalent to ≥ 3 log reduction of BCR-ABL transcript from standardized baseline, measured by RQ-PCR.	By 3, 6, 9, 12, 18, 24, 36, 48, 66 cycles
Pharmacokinetics (PK): Steady State Concentration of Nilotinib in Imatinib/Dasatinib Resistant/Intolerant CML-CP Patients	PK was analyzed only when all patients has completed 12 cycles on treatment or discontinued the study treatment early.	Cycle 1 Day 8
Pharmacokinetics: Steady State Concentration of Nilotinib in Newly Diagnosed CML-CP Patients	PK was analyzed only when all patients has completed 12 cycles of treatment or discontinued the study treatment early.	Cycle 1 Day 8
Growth Data: Abnormal Height Standard Deviation Scores (SDS) Changes by Cohort	To assess long term effect on growth, development and maturation of nilotinib treatment in pediatric patients with Ph+ CML in participants with both a baseline and post-baseline value.	from first dosing to 66 cycles
Acceptability (Including Palatability) of Dose Forms Used After First Dose, Cycle 1 and Cycle 12 Study Drug Formulation	Acceptability of the study drug was evaluated from a questionnaire completed by patients, with the help from parents or caregivers at visits. The Questionnaire to capture patient assessment of palatability (very good to very bad) and acceptability of taking the medication (very easy to very hard to administration).	up to Cycle 12
Mutational Assessment of BCR-ABL	Emerging signs of resistance to nilotinib	up to 66 cycles

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Long Term Effect of Nilotinib on Bone Metabolism	The summary of bone age and Dual-energy X-ray absorptiometry (DEXA) by cohort. Alteration of bone biochemical markers of hand and wrist X-Ray evaluation was observed in bone age standard deviation scores (SDS) and for bone mineral density for DEXA before and after treatment with nilotinib.	Cycle 66

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals

Publications and helpful links

General Publications

• Hijiya 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.
• Hijiya 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.

Study record dates

Study Major Dates

• Study Start (ACTUAL): August 20, 2013
• Primary Completion (ACTUAL): June 1, 2016
• Study Completion (ACTUAL): August 28, 2020

Study Registration Dates

• First Submitted: April 29, 2013
• First Submitted That Met QC Criteria: April 29, 2013
• First Posted (ESTIMATE): May 1, 2013

Study Record Updates

• Last Update Posted (ACTUAL): April 22, 2021
• Last Update Submitted That Met QC Criteria: March 25, 2021
• Last Verified: March 1, 2021

More Information

Terms related to this study

• Keywords: pediatric; hematologic diseases; leukemia; dasatinib; imatinib; protein kinase inhibitor; Tasigna; BCR-ABL positive; chronic phase; myeloproliferative disorder; Ph+ CML; enzyme inhibitor; accelerated phase; newly diagnosed Ph+ CML; nilotinib treatment; leukemia, pediatric; leukemia, myleiod; leukemia, mylegenous, chronic; leukemia, mylegenous, accelerated; bone marrow disease; neoplastic processes
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Myeloproliferative Disorders; Leukemia; Leukemia, Myeloid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive
• Other Study ID Numbers: CAMN107A2203; 2013-000200-41 (EUDRACT_NUMBER)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No