Trial Outcomes & Findings for Study to Evaluate Nilotinib in Chronic Myelogenous Leukemia (CML) Patients With SubOptimal Response (NCT NCT01043874)

NCT ID: NCT01043874

Last Updated: 2016-04-08

Results Overview

MMR is defined as BCR-ABL ratio (%) on IS ≤ 0.1% (corresponds to ≥ 3 log reduction of BCR-ABL transcripts from standardized baseline value

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 45 participants
• Primary outcome timeframe: 12 months after treatment
• Results posted on: 2016-04-08

Participant Flow

Participant milestones

Measure	Nilotinib Nilotinib 400 mg BID
Overall Study STARTED	45
Overall Study COMPLETED	39
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Measure	Nilotinib Nilotinib 400 mg BID
Overall Study Adverse Event	3
Overall Study Withdrawal by Subject	1
Overall Study Lack of Efficacy	1
Overall Study Administrative Problems	1

Baseline Characteristics

Study to Evaluate Nilotinib in Chronic Myelogenous Leukemia (CML) Patients With SubOptimal Response

Baseline characteristics by cohort

Measure	Nilotinib n=45 Participants Nilotinib 400 mg BID
Age, Continuous	49.5 years STANDARD_DEVIATION 14.89 • n=5 Participants
Sex: Female, Male Female	16 Participants n=5 Participants
Sex: Female, Male Male	29 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months after treatment

Population: Full Analysis Set

MMR is defined as BCR-ABL ratio (%) on IS ≤ 0.1% (corresponds to ≥ 3 log reduction of BCR-ABL transcripts from standardized baseline value

Outcome measures

Measure	Nilotinib n=45 Participants Nilotinib 400 mg BID
MMR Rate at 12 Mos. of Nilotinib Treatment on Study in Patients With Philadelphia Chromosome Positive (Ph+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP) Who Have a Suboptimal Molecular Response to Imatinib at 18 Months or Later.	51.1 % participants achieving MMR Interval 35.8 to 66.3

SECONDARY outcome

Timeframe: 24 months after treatment

Population: Full Analysis Set

MMR is defined as BCR-ABL ratio (%) on IS ≤ 0.1% (corresponds to ≥ 3 log reduction of BCR-ABL transcripts from standardized baseline value

Outcome measures

Measure	Nilotinib n=45 Participants Nilotinib 400 mg BID
MMR Rate at 24 Months of Nilotinib Treatment on Study in Patients With Philadelphia Chromosome Positive (Ph+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP)	66.7 % participants achieving MMR Interval 51.0 to 80.0

SECONDARY outcome

Timeframe: month 24

Population: Full Analysis Set

MMR is defined as BCR-ABL ratio (%) on IS ≤ 0.1% (corresponds to ≥ 3 log reduction of BCR-ABL transcripts from standardized baseline value

Outcome measures

Measure	Nilotinib n=45 Participants Nilotinib 400 mg BID
Time to First MMR of Nilotinib in Patients With Philadelphia Chromosome Positive (Ph+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP) .	5.19 months Standard Deviation 5.494

SECONDARY outcome

Timeframe: month 24

Population: Full Analysis Set

MMR is defined as BCR-ABL ratio (%) on IS ≤ 0.1% (corresponds to ≥ 3 log reduction of BCR-ABL transcripts from standardized baseline value

Outcome measures

Measure	Nilotinib n=45 Participants Nilotinib 400 mg BID
Duration of MMR of Nilotinib in Patients With Philadelphia Chromosome Positive (Ph+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP) .	51.1 % participants w/ durable MMR at 24 mos Interval 35.8 to 66.3

Adverse Events

Nilotinib

Serious events: 7 serious events

Other events: 45 other events

Deaths: 0 deaths

Serious adverse events

Measure	Nilotinib n=45 participants at risk Nilotinib 400 mg BID
Blood and lymphatic system disorders Anaemia	2.2% 1/45
Hepatobiliary disorders Bile duct stone	2.2% 1/45
Infections and infestations Pneumonia	2.2% 1/45
Metabolism and nutrition disorders Dehydration	2.2% 1/45
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Chronic myeloid leukaemia transformation	2.2% 1/45
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Malignant neoplasm of renal pelvis	2.2% 1/45
Skin and subcutaneous tissue disorders Erythema multiforme	2.2% 1/45

Other adverse events

Measure	Nilotinib n=45 participants at risk Nilotinib 400 mg BID
Blood and lymphatic system disorders Anaemia	8.9% 4/45
Blood and lymphatic system disorders Eosinophilia	8.9% 4/45
Eye disorders Conjunctivitis	11.1% 5/45
Gastrointestinal disorders Abdominal pain upper	11.1% 5/45
Gastrointestinal disorders Constipation	22.2% 10/45
Gastrointestinal disorders Diarrhoea	11.1% 5/45
Gastrointestinal disorders Haemorrhoids	6.7% 3/45
Gastrointestinal disorders Nausea	20.0% 9/45
Gastrointestinal disorders Vomiting	13.3% 6/45
General disorders Face oedema	6.7% 3/45
General disorders Fatigue	8.9% 4/45
General disorders Oedema	6.7% 3/45
General disorders Pyrexia	11.1% 5/45
Hepatobiliary disorders Hepatic function abnormal	11.1% 5/45
Hepatobiliary disorders Hyperbilirubinaemia	53.3% 24/45
Infections and infestations Gastroenteritis	6.7% 3/45
Infections and infestations Influenza	6.7% 3/45
Infections and infestations Nasopharyngitis	46.7% 21/45
Infections and infestations Periodontitis	6.7% 3/45
Investigations Alanine aminotransferase increased	31.1% 14/45
Investigations Amylase increased	11.1% 5/45
Investigations Aspartate aminotransferase increased	17.8% 8/45
Investigations Blood bilirubin increased	15.6% 7/45
Investigations Gamma-glutamyltransferase increased	8.9% 4/45
Investigations Lipase increased	24.4% 11/45
Metabolism and nutrition disorders Decreased appetite	6.7% 3/45
Metabolism and nutrition disorders Diabetes mellitus	8.9% 4/45
Metabolism and nutrition disorders Hypercholesterolaemia	15.6% 7/45
Metabolism and nutrition disorders Hyperglycaemia	13.3% 6/45
Metabolism and nutrition disorders Hypophosphataemia	28.9% 13/45
Musculoskeletal and connective tissue disorders Arthralgia	6.7% 3/45
Musculoskeletal and connective tissue disorders Back pain	6.7% 3/45
Musculoskeletal and connective tissue disorders Muscle spasms	8.9% 4/45
Musculoskeletal and connective tissue disorders Myalgia	20.0% 9/45
Musculoskeletal and connective tissue disorders Pain in extremity	6.7% 3/45
Nervous system disorders Headache	37.8% 17/45
Respiratory, thoracic and mediastinal disorders Rhinitis allergic	6.7% 3/45
Skin and subcutaneous tissue disorders Alopecia	11.1% 5/45
Skin and subcutaneous tissue disorders Eczema	8.9% 4/45
Skin and subcutaneous tissue disorders Pruritus	20.0% 9/45
Skin and subcutaneous tissue disorders Rash	33.3% 15/45

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: 862-778-8300

Results disclosure agreements

• Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial
• Publication restrictions are in place

Restriction type: OTHER