Trial · NCT01061177
Nilotinib in Newly Diagnosed Adult Philadelphia Chromosome & /or BCR-ABL Positive Chronic Myeloid Leukaemia in Chronic Phase
NCT01061177, Phase 4, Completed, Novartis Pharmaceuticals; results first posted 2017-02-24
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Nilotinib in Newly Diagnosed Adult Philadelphia Chromosome & /or BCR-ABL Positive Chronic Myeloid Leukaemia in Chronic Phase. Trials, record NCT01061177. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01061177
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Nilotinib in Newly Diagnosed Adult Philadelphia Chromosome & /or BCR-ABL Positive Chronic Myeloid Leukaemia in Chronic Phase
- NCT number
- NCT01061177
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 1,090 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- CML in Chronic Phase
- Interventions
- Nilotinib
- First posted
- 2010-02-02
- Last updated
- 2017-02-24
- Other registries' IDs
- eudract:2009-017775-19
- Results first posted
- 2017-02-24
Source ClinicalTrials.gov
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Molecular Response (MR4^0) at 18 Months | at 18 months | MR4\^0 was defined as either (i) detectable disease ≤ 0.01% BCR-ABL ratio (international scale (IS)) with mean ABL transcripts ≥ 10 000 or (ii) undetectable disease in complementary deoxyribonucleic acid (cDNA) with ≥ 10 000 ABL transcripts. |
| Secondary | Percentage of Participants Free From Progression to Accelerated Phase/Blast Crisis (AP/BC) at 12 and 24 Months | at 12 and 24 months | The following events were considered disease progression to AP/BC: Death due to disease under study; AP, as defined by any of the following: ≥ 15% blasts in the peripheral blood or bone marrow, but \< 30% blasts in both the peripheral blood and bone marrow, ≥ 30% blasts plus promyelocytes in peripheral blood or bone marrow, ≥ 20% basophils in the peripheral blood, Thrombocytopenia (\< 100 × 109/L) that was unrelated to therapy, Evidence of clonal evolution, as determined by medical review with consensus of the SSMC/DMC. BC was defined as: ≥ 30% blasts in peripheral blood or bone marrow, Appearance of extramedullary involvement other than hepatosplenomegaly proven by biopsy. |
| Secondary | Rate of Event Free Survival at 12 and 24 Months | at 12 and 24 months | EFS was defined as the time from the date of Day 1 (first treatment) + 1 day to the first occurrence of any of the following: Loss of complete hematologic response (CHR), Loss of CCyR, Death from any cause, Progression to the AP or BC of CML, Not achieving CHR up to 3 months (ie, 91 + 15 days), Not achieving CCyR up to 18 months (ie, 548 + 15 days), whichever is earlier. |
| Secondary | Percentage of Participants With Major Molecular Response (MMR) at, as Well as by, 12 and 24 Months | 12 months, 24 months | MMR was defined as BCR-ABL ratio (IS) ≤ 0.1% in a peripheral blood sample. BCR-ABL1 is an abnormal gene found in chronic myeloid leukemia (CML) and acute lymphoblastic leukemia (ALL). The chromosomal defect in the Philadelphia chromosome is a translocation, in which parts of two chromosomes, 9 and 22, swap places. The result is that a fusion gene is created by juxtapositioning the Abl1 gene on chromosome 9 to a part of the BCR ("breakpoint cluster region") gene on chromosome 22. Depending upon the breakpoints on the BCR gene, there are several forms of fusion proteins. |
| Secondary | Percentage of Participants With Complete Cytogenetic Response (CCyR) at, as Well as by, 12 and 24 Months | 12 and 24 months | CCyR parameters were defined as 0% Philadelphia positive (Ph+) metaphases. Loss of CCyR was defined as a patient exceeding the CCyR criteria (ie, \> 0% Ph+ metaphases) at a subsequent visit after the patient had achieved CCyR. |
| Secondary | Percentage of Participants With Major Cytogenetic Response (MCyR) at, as Well as by, 12 and 24 Months | 12 and 24 months | Major cytogenetic response (MCyR) parameters were defined as 0 to 35% Philadelphia positive (Ph+) metaphases. |
| Secondary | Percentage of Participants Free From Progression to AP/BC With MR4^0 at 12 Months | at 12 months | The following events were considered disease progression to AP/BC: Death due to disease under study; AP, as defined by any of the following: ≥ 15% blasts in the peripheral blood or bone marrow, but \< 30% blasts in both the peripheral blood and bone marrow, ≥ 30% blasts plus promyelocytes in peripheral blood or bone marrow, ≥ 20% basophils in the peripheral blood, Thrombocytopenia (\< 100 × 109/L) that was unrelated to therapy, Evidence of clonal evolution, as determined by medical review with consensus of the SSMC/DMC. BC was defined as: ≥ 30% blasts in peripheral blood or bone marrow, Appearance of extramedullary involvement other than hepatosplenomegaly proven by biopsy. |
| Secondary | Percentage of Participants With Event Free Survival in Participants Achieving MR4^0 at 12 Months | at 12 months | EFS was defined as the time from the date of Day 1 (first treatment) + 1 day to the first occurrence of any of the following: Loss of complete hematologic response (CHR), Loss of CCyR, Death from any cause, Progression to the AP or BC of CML, Not achieving CHR up to 3 months (i.e. 91 + 15 days). |
| Secondary | Percentage of Participants With Progression Free Survival (PFS) at 12 and 24 Months | 12 months, 24 months | PFS was defined by the study protocol as the time from the date of start of study drug to the date of earliest progression to AP/BC, or the date of death from any cause. |
| Secondary | Rate of Molecular Response (MR4^0) at, as Well as by, 12 and 24 Months | 12 and 24 months | MR4\^0 was defined as either (i) detectable disease ≤ 0.01% BCR-ABL ratio (international scale (IS)) with mean ABL transcripts ≥ 10 000 or (ii) undetectable disease in complementary deoxyribonucleic acid (cDNA) with ≥ 10 000 ABL transcripts. |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 registered outcomesOutcomes16
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Molecular Response (MR4^0) at 18 Months | at 18 months | Intent-to-treat\_Molecular (ITT\_MR) analysis set was a subset of the ITT population including the patients with typical BCR-ABL transcript at screening. | Percentage of Participants | NUMBER |
| SECONDARY | Percentage of Participants Free From Progression to Accelerated Phase/Blast Crisis (AP/BC) at 12 and 24 Months | at 12 and 24 months | The intent-to-treat (ITT) population consisted of all patients who received at least one dose of study drug. | Percentage of participants | NUMBER |
| SECONDARY | Rate of Event Free Survival at 12 and 24 Months | at 12 and 24 months | The intent-to-treat (ITT) population consisted of all patients who received at least one dose of study drug. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Major Molecular Response (MMR) at, as Well as by, 12 and 24 Months | 12 months, 24 months | Intent-to-treat\_Molecular (ITT\_MR) analysis set was a subset of the ITT population including the patients with typical BCR-ABL transcript at screening. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Complete Cytogenetic Response (CCyR) at, as Well as by, 12 and 24 Months | 12 and 24 months | The ITT\_CyR population was a subset of the ITT population including the Ph+ patients at screening was considered. Patients who had either no metaphases recorded at screening bone marrow or only negative metaphases recorded at screening bone marrow but had Ph+ metaphases at any visits after screening were also part of this population. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Major Cytogenetic Response (MCyR) at, as Well as by, 12 and 24 Months | 12 and 24 months | The ITT\_CyR population was a subset of the ITT population including the Ph+ patients at screening was considered. Patients who had either no metaphases recorded at screening bone marrow or only negative metaphases recorded at screening bone marrow but had Ph+ metaphases at any visits after screening were also part of this population. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Free From Progression to AP/BC With MR4^0 at 12 Months | at 12 months | The intent-to-treat (ITT) population consisted of all patients who received at least one dose of study drug. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Event Free Survival in Participants Achieving MR4^0 at 12 Months | at 12 months | The intent-to-treat (ITT) population consisted of all patients who received at least one dose of study drug. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Progression Free Survival (PFS) at 12 and 24 Months | 12 months, 24 months | The intent-to-treat (ITT) population consisted of all patients who received at least one dose of study drug. | Percentage of participants | NUMBER |
| SECONDARY | Rate of Molecular Response (MR4^0) at, as Well as by, 12 and 24 Months | 12 and 24 months | The ITT\_MR population was a subset of the ITT population including the patients with typical BCR-ABL transcript at screening ie, b3a2 and/or b2a2 were considered. This population was referred to as ITT\_MR. | Percentage of participants | NUMBER |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 outcomesResults by group39
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Molecular Response (MR4^0) at 18 Months | Nilotinib | 38.3 | Percentage of Participants | ||
| Percentage of Participants Free From Progression to Accelerated Phase/Blast Crisis (AP/BC) at 12 and 24 Months | Nilotinib | Pts free from progression to AP/BC at 12 months | 99.4 | Percentage of participants | |
| Percentage of Participants Free From Progression to Accelerated Phase/Blast Crisis (AP/BC) at 12 and 24 Months | Nilotinib | Pts free from progression to AP/BC at 24 months | 99.4 | Percentage of participants | |
| Rate of Event Free Survival at 12 and 24 Months | Nilotinib | Percentage of participants with EFS at 12 months | 71.7 | Percentage of participants | |
| Rate of Event Free Survival at 12 and 24 Months | Nilotinib | Percentage of participants with EFS at 24 months | 69.1 | Percentage of participants | |
| Percentage of Participants With Major Molecular Response (MMR) at, as Well as by, 12 and 24 Months | Nilotinib | at 12 months | 56.2 | Percentage of participants | |
| Percentage of Participants With Major Molecular Response (MMR) at, as Well as by, 12 and 24 Months | Nilotinib | at 24 months | 61.1 | Percentage of participants | |
| Percentage of Participants With Major Molecular Response (MMR) at, as Well as by, 12 and 24 Months | Nilotinib | by 12 months | 68.8 | Percentage of participants | |
| Percentage of Participants With Major Molecular Response (MMR) at, as Well as by, 12 and 24 Months | Nilotinib | by 24 months | 80.3 | Percentage of participants | |
| Percentage of Participants With Complete Cytogenetic Response (CCyR) at, as Well as by, 12 and 24 Months | Nilotinib | at 12 months | 72.4 | Percentage of participants |
1 to 10 of 39Source ClinicalTrials.gov
See all 39 results by groupParticipant flow5
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Nilotinib | 1,089 | |
| Overall Study | ITT_MR (b2a2 &/or b3a2 +ve Pts Only) | Nilotinib | 1,056 | |
| Overall Study | ITT_CyR (Ph+ Patients Only) | Nilotinib | 983 | |
| Overall Study | COMPLETED | Nilotinib | 881 | |
| Overall Study | NOT COMPLETED | Nilotinib | 208 |
Source ClinicalTrials.gov
Adverse events250
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Nilotinib | 65 | 1,089 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Nilotinib | 111 | 1,089 | |
| other | Abdominal pain | Gastrointestinal disorders | Nilotinib | 79 | 1,089 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Nilotinib | 88 | 1,089 | |
| other | Constipation | Gastrointestinal disorders | Nilotinib | 65 | 1,089 | |
| other | Diarrhoea | Gastrointestinal disorders | Nilotinib | 93 | 1,089 | |
| other | Nausea | Gastrointestinal disorders | Nilotinib | 122 | 1,089 | |
| other | Vomiting | Gastrointestinal disorders | Nilotinib | 62 | 1,089 | |
| other | Asthenia | General disorders | Nilotinib | 97 | 1,089 | |
| other | Fatigue | General disorders | Nilotinib | 150 | 1,089 |
1 to 10 of 250Source ClinicalTrials.gov
See all 250 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Nilotinib | ||
| Total, other adverse events | Nilotinib | 841 | 1,089 |
| Total, serious adverse events | Nilotinib | 207 | 1,089 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01061177
GET https://conductscience.org/api/trials/v1/NCT01061177/results?page=2