NCT00129740

Phase II Nilotinib With Newly Diagnosed Chronic Phase Chronic Myelogenous Leukemia (CML)

Completed Phase 2 Results posted Last updated 24 September 2019

What this trial tests

Phase 2 trial testing Nilotinib in Leukemia, Myelogenous, Chronic in 148 participants. Completed in 11 July 2018.

Timeline

27 June 2005

Primary endpoint
11 July 2018

11 July 2018

Quick facts

Lead sponsor	M.D. Anderson Cancer Center
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	148
Start date	27 June 2005
Primary completion	11 July 2018
Estimated completion	11 July 2018
Sites	1 location across United States

Drugs / interventions tested

• Nilotinib (NILOTINIB) — full drug profile →

Conditions studied

• Leukemia, Myelogenous, Chronic — all drugs for Leukemia, Myelogenous, Chronic →

Sponsor

M.D. Anderson Cancer Center — full company profile →

Who can join

16 and older, any sex, with Leukemia, Myelogenous, Chronic. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Participants With Complete Molecular Response (Molecular CR) Primary · 12 months

Polymerase chain reaction (PCR) Ratio BCR-Abl/Abl of 0% after 12 months of therapy with Nilotinib by international standard.

Group	Value	95% CI
Nilotinib	23

Number of Participants With Complete Cytogenetic Response (CCyR) Secondary · 6 months

Complete hematologic remission classified according to suppression of Philadelphia chromosome (Ph) by cytogenetics or i Fluorescence in situ hybridization (FISH) 1. No cytogenetic response - Ph positive 100% 2. Minor cytogenetic response - Ph positive 35-90% 3. Partial cytogenetic response - Ph positive 1-34% 4. Complete cytogenetic response - Ph positive 0% Major cytogenetic response = complete + partial (Ph positive \<35%)

Group	Value	95% CI
Nilotinib	131

Adverse events — posted to ClinicalTrials.gov

Time frame: Up to 11.5 years. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Nilotinib

Serious: 47/148 (32%)

Deaths: 7/148

Serious adverse events (48 terms)

Reaction	System	Nilotinib
Abdoninal Pain	Gastrointestinal disorders	—
Cardiac Chest Pain	Cardiac disorders	—
Cardiac ischemia/infarction	Cardiac disorders	—
CNS Ischemia	Nervous system disorders	—
Dyspnea	Respiratory, thoracic and mediastinal disorders	—
Pain	General disorders	—
Infection	Infections and infestations	—
Nausea/Vomiting	Gastrointestinal disorders	—
Death	General disorders	—
Fracture	Musculoskeletal and connective tissue disorders	—
GI Pain	Gastrointestinal disorders	—
Gastrointestinal Hemorrhage	Gastrointestinal disorders	—
Hypertension	Vascular disorders	—
Cellulitis	Infections and infestations	—
Hysterectomy	Surgical and medical procedures	—
Pleural Effusion	Respiratory, thoracic and mediastinal disorders	—
Back Pain	General disorders	—
Dehydration	Metabolism and nutrition disorders	—
Pneumonia	Infections and infestations	—
Left Buccal Squamous Cell Carcinoma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—
CNS Hemorrhage	Blood and lymphatic system disorders	—
Colitis	Gastrointestinal disorders	—
Elevated Creatinine	Investigations	—
Revision right hip arthroplasty	Surgical and medical procedures	—
Flu like Syndrome	General disorders	—

Other adverse events (7 terms — click to expand)

Reaction	System	Nilotinib
Elevated bilirubin	Investigations	—
Rash	Skin and subcutaneous tissue disorders	—
Elevated Aminotransferases	Investigations	—
Fatigue	General disorders	—
Hypertension	Vascular disorders	—
Arrhythmia	Cardiac disorders	—
Cardiovascular Ischemia	Cardiac disorders	—

Most-reported serious reactions: Abdoninal Pain, Cardiac Chest Pain, Cardiac ischemia/infarction, CNS Ischemia, Dyspnea, Pain, Infection, Nausea/Vomiting.

Data from ClinicalTrials.gov NCT00129740 adverse events section.

Sponsor's own description

The goal of this clinical research study is to learn if an experimental agent, AMN107 (nilotinib), can help to control CML in chronic phase. The safety of this experimental agent will also be studied.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Relative survival in patients with chronic-phase chronic myeloid leukaemia in the tyrosine-kinase inhibitor era: analysis of patient data from six prospective clinical trials.
   Sasaki K, Strom SS, O'Brien S, Jabbour E, et al · · 2015 · cited 239× · PMID 26688093 · DOI 10.1016/s2352-3026(15)00048-4
2. Nilotinib as front-line treatment for patients with chronic myeloid leukemia in early chronic phase.
   Cortes JE, Jones D, O'Brien S, Jabbour E, et al · · 2010 · cited 169× · PMID 20008621 · DOI 10.1200/jco.2009.25.4896
3. Early responses predict better outcomes in patients with newly diagnosed chronic myeloid leukemia: results with four tyrosine kinase inhibitor modalities.
   Jain P, Kantarjian H, Nazha A, O'Brien S, et al · · 2013 · cited 106× · PMID 23620574 · DOI 10.1182/blood-2013-03-490128
4. Analysis of cardiovascular and arteriothrombotic adverse events in chronic-phase CML patients after frontline TKIs.
   Jain P, Kantarjian H, Boddu PC, Nogueras-González GM, et al · · 2019 · cited 89× · PMID 30885996 · DOI 10.1182/bloodadvances.2018025874
5. Clonal chromosomal abnormalities appearing in Philadelphia chromosome-negative metaphases during CML treatment.
   Issa GC, Kantarjian HM, Gonzalez GN, Borthakur G, et al · · 2017 · cited 47× · PMID 28835440 · DOI 10.1182/blood-2017-07-792143
6. A prospective analysis of symptom burden for patients with chronic myeloid leukemia in chronic phase treated with frontline second- and third-generation tyrosine kinase inhibitors.
   Zulbaran-Rojas A, Lin HK, Shi Q, Williams LA, et al · · 2018 · cited 30× · PMID 30318751 · DOI 10.1002/cam4.1808
7. Conditional survival in patients with chronic myeloid leukemia in chronic phase in the era of tyrosine kinase inhibitors.
   Sasaki K, Kantarjian HM, Jain P, Jabbour EJ, et al · · 2016 · cited 28× · PMID 26479889 · DOI 10.1002/cncr.29745
8. Clinical Safety and Efficacy of Nilotinib or Dasatinib in Patients With Newly Diagnosed Chronic-Phase Chronic Myelogenous Leukemia and Pre-Existing Liver and/or Renal Dysfunction.
   Sasaki K, Lahoti A, Jabbour E, Jain P, et al · · 2016 · cited 24× · PMID 26796981 · DOI 10.1016/j.clml.2015.12.003