Trial · NCT01743989
A Randomized Phase III Study to Assess the Effect of a Longer Duration of Consolidation Treatment With Nilotinib on TFR in CP CML.
NCT01743989, Phase 3, Completed, Novartis Pharmaceuticals; results first posted 2021-07-15
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Randomized Phase III Study to Assess the Effect of a Longer Duration of Consolidation Treatment With Nilotinib on TFR in CP CML. Trials, record NCT01743989. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01743989
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Randomized Phase III Study to Assess the Effect of a Longer Duration of Consolidation Treatment With Nilotinib on TFR in CP CML.
- NCT number
- NCT01743989
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 620 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Philadelphia Chromosome Positive (PH+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP)
- Interventions
- Nilotinib
- First posted
- 2012-12-06
- Last updated
- 2021-07-15
- Other registries' IDs
- eudract:2012-005124-15
- Results first posted
- 2021-07-15
Source ClinicalTrials.gov
Registered outcomes22
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Who Remained in Treatment Free Remission (TFR) Without Molecular Relapse 12 Months After Entering the TFR Phase | 12 months after entering the TFR phase, which is after 36 months from study treatment start for Nilotinib 24-month treatment arm and after 48 months from study treatment start for Nilotinib 36-month treatment arm | Number of participants who remained in TFR (≥molecular response (MR) 4.0) without molecular relapse 12 months after entering the TFR phase (without re-starting nilotinib therapy) divided by the number of participants who entered the TFR phase and multiplied by 100. Molecular relapse during TFR is defined as the loss of major molecular response (MMR), or the confirmed loss of MR4.0 (defined by 3 consecutive tests less than MR4.0 assessed at 3 consecutive visits during TFR phase). Participants dropping out early from the study during the TFR phase were considered as unsuccessful TFR. Confidence intervals were calculated based on the Exact Clopper-Pearson method. MMR is defined as≥3.0 log reduction in BCR-ABL transcripts compared to the standardized baseline or ≤0.1% BCR-ABL. MR4.0 is defined as either detectable disease≤0.01% BCR-ABL or undetectable disease in cDNA with≥10,000 ABL transcripts |
| Secondary | Cumulative Incidence of MMR During the Pre-randomization Induction/Consolidation Phase Among Participants Without That Response at Study Entry | From baseline up to 24 months after study treatment start | Number of participants who were in MMR during pre-randomization induction/consolidation phase divided by the number of participants without that response at baseline and multiplied by 100. Confidence intervals were calculated based on the Exact Clopper-Pearson method. MMR is defined as ≥3.0 log reduction in BCR-ABL transcripts compared to the standardized baseline or ≤0.1% BCR-ABL. |
| Secondary | Cumulative Incidence of MMR During the Post-randomization Consolidation Phase Among Participants Without That Response at Study Entry | From randomization (month 24 after study treatment start) up to 36 months after study treatment start | Number of participants who were in MMR during post-randomization consolidation phase divided by the number of participants without that response at baseline and multiplied by 100. Post-randomization consolidation phase corresponded to the 12-month additional treatment (after randomization) for Nilotinib 36-month treatment arm. Confidence intervals were calculated based on the Exact Clopper-Pearson method. MMR is defined as ≥3.0 log reduction in BCR-ABL transcripts compared to the standardized baseline or ≤0.1% BCR-ABL. |
| Secondary | Cumulative Incidence of MR4.0 During the Pre-randomization Induction/Consolidation Phase Among Participants Without That Response at Study Entry | From baseline up to 24 months after study treatment start | Number of participants who were in MR4.0 during the pre-randomization induction/consolidation phase divided by the number of participants without that response at baseline and multiplied by 100. Confidence intervals were calculated based on the Exact Clopper-Pearson method. MR4.0 is defined as either detectable disease ≤0.01% BCR-ABL IS or undetectable disease in cDNA with ≥10,000 ABL transcripts (numbers of ABL transcripts in the same volume of cDNA used to test for BCR-ABL) |
| Secondary | Cumulative Incidence of MR4.0 During the Post-randomization Consolidation Phase Among Participants Without That Response at Study Entry | From randomization (month 24 after study treatment start) up to 36 months after study treatment start | Number of participants who were in MR4.0 during the post-randomization consolidation phase divided by the number of participants without that response at baseline and multiplied by 100. Post-randomization consolidation phase corresponded to the 12-month additional treatment (after randomization) for Nilotinib 36-month treatment arm. Confidence intervals were calculated based on the Exact Clopper-Pearson method. MR4.0 is defined as either detectable disease ≤0.01% BCR-ABL IS or undetectable disease in cDNA with ≥10,000 ABL transcripts (numbers of ABL transcripts in the same volume of cDNA used to test for BCR-ABL) |
| Secondary | Cumulative Incidence of MR4.5 During the Pre-randomization Induction/Consolidation Phase Among Participants Without That Response at Study Entry | From baseline up to 24 months after study treatment start | Number of participants who were in MR4.5 during the pre-randomization induction/consolidation phase divided by the number of participants without that response at baseline and multiplied by 100. Confidence intervals were calculated based on the Exact Clopper-Pearson method. MR4.5 is defined as either detectable disease ≤ 0.0032% BCR-ABL IS; or undetectable disease within cDNA with ≥ 32,000 ABL transcripts (numbers of ABL transcripts in the same volume of cDNA used to test for BCR-ABL) |
| Secondary | Cumulative Incidence of MR4.5 During the Post-randomization Consolidation Phase Among Participants Without That Response at Study Entry | From randomization (month 24 after study treatment start) up to 36 months after study treatment start | Number of participants who were in MR4.5 during the post-randomization consolidation phase divided by the number of participants without that response at baseline and multiplied by 100. Post-randomization consolidation phase corresponded to the 12-month additional treatment (after randomization) for Nilotinib 36-month treatment arm. Confidence intervals were calculated based on the Exact Clopper-Pearson method. MR4.5 is defined as either detectable disease ≤ 0.0032% BCR-ABL IS; or undetectable disease within cDNA with ≥ 32,000 ABL transcripts (numbers of ABL transcripts in the same volume of cDNA used to test for BCR-ABL) |
| Secondary | Cumulative Incidence of MMR During the Pre-randomization Induction/Consolidation Phase | From baseline up to 24 months after study treatment start | Number of participants who were in MMR during the pre-randomization induction/consolidation phase divided by the number of enrolled participants and multiplied by 100. Confidence intervals were calculated based on the Exact Clopper-Pearson method. MMR is defined as ≥3.0 log reduction in BCR-ABL transcripts compared to the standardized baseline or ≤0.1% BCR-ABL. |
| Secondary | Cumulative Incidence of MMR During the Post-randomization Consolidation Phase | From randomization (month 24 after study treatment start) up to 36 months after study treatment start | Number of participants who were in MMR during the post-randomization consolidation phase divided by the number of enrolled participants and multiplied by 100. Post-randomization consolidation phase corresponded to the 12-month additional treatment (after randomization) for Nilotinib 36-month treatment arm. Confidence intervals were calculated based on the Exact Clopper-Pearson method. MMR is defined as ≥3.0 log reduction in BCR-ABL transcripts compared to the standardized baseline or ≤0.1% BCR-ABL. |
| Secondary | Cumulative Incidence of MR4.0 During the Pre-randomization Induction/Consolidation Phase | From baseline up to 24 months after study treatment start | Number of participants who were in MR4.0 during the pre-randomization induction/consolidation phase divided by the number of enrolled participants and multiplied by 100. Confidence intervals were calculated based on the Exact Clopper-Pearson method. MR4.0 is defined as either detectable disease ≤0.01% BCR-ABL IS or undetectable disease in cDNA with ≥10,000 ABL transcripts (numbers of ABL transcripts in the same volume of cDNA used to test for BCR-ABL) |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 registered outcomesOutcomes23
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants Who Remained in Treatment Free Remission (TFR) Without Molecular Relapse 12 Months After Entering the TFR Phase | 12 months after entering the TFR phase, which is after 36 months from study treatment start for Nilotinib 24-month treatment arm and after 48 months from study treatment start for Nilotinib 36-month treatment arm | All enrolled subjects who entered the TFR phase | Percentage of participants | NUMBER |
| SECONDARY | Cumulative Incidence of MMR During the Pre-randomization Induction/Consolidation Phase Among Participants Without That Response at Study Entry | From baseline up to 24 months after study treatment start | All enrolled subjects for whom written informed consent was obtained prior to any study specific procedure who did not reach MMR at baseline | Percentage of participants | NUMBER |
| SECONDARY | Cumulative Incidence of MMR During the Post-randomization Consolidation Phase Among Participants Without That Response at Study Entry | From randomization (month 24 after study treatment start) up to 36 months after study treatment start | All enrolled subjects for whom written informed consent was obtained prior to any study specific procedure who did not reach MMR at baseline. Only participants randomized to Nilotinib 36-month treatment arm entered the post-randomization consolidation phase. | Percentage of participants | NUMBER |
| SECONDARY | Cumulative Incidence of MR4.0 During the Pre-randomization Induction/Consolidation Phase Among Participants Without That Response at Study Entry | From baseline up to 24 months after study treatment start | All enrolled subjects for whom written informed consent was obtained prior to any study specific procedure who did not reach MR4.0 at baseline | Percentage of participants | NUMBER |
| SECONDARY | Cumulative Incidence of MR4.0 During the Post-randomization Consolidation Phase Among Participants Without That Response at Study Entry | From randomization (month 24 after study treatment start) up to 36 months after study treatment start | All enrolled subjects for whom written informed consent was obtained prior to any study specific procedure who did not reach MR4.0 at baseline. Only participants randomized to Nilotinib 36-month treatment arm entered the post-randomization consolidation phase | Percentage of participants | NUMBER |
| SECONDARY | Cumulative Incidence of MR4.5 During the Pre-randomization Induction/Consolidation Phase Among Participants Without That Response at Study Entry | From baseline up to 24 months after study treatment start | All enrolled subjects for whom written informed consent was obtained prior to any study specific procedure who did not reach MR4.5 at baseline | Percentage of participants | NUMBER |
| SECONDARY | Cumulative Incidence of MR4.5 During the Post-randomization Consolidation Phase Among Participants Without That Response at Study Entry | From randomization (month 24 after study treatment start) up to 36 months after study treatment start | All enrolled subjects for whom written informed consent was obtained prior to any study specific procedure who did not reach MR4.5 at baseline. Only participants randomized to Nilotinib 36-month treatment arm entered the post-randomization consolidation phase | Percentage of participants | NUMBER |
| SECONDARY | Cumulative Incidence of MMR During the Pre-randomization Induction/Consolidation Phase | From baseline up to 24 months after study treatment start | All enrolled subjects for whom written informed consent was obtained prior to any study specific procedure | Percentage of participants | NUMBER |
| SECONDARY | Cumulative Incidence of MMR During the Post-randomization Consolidation Phase | From randomization (month 24 after study treatment start) up to 36 months after study treatment start | All enrolled subjects for whom written informed consent was obtained prior to any study specific procedure. Only participants randomized to Nilotinib 36-month treatment arm entered the post-randomization consolidation phase | Percentage of participants | NUMBER |
| SECONDARY | Cumulative Incidence of MR4.0 During the Pre-randomization Induction/Consolidation Phase | From baseline up to 24 months after study treatment start | All enrolled subjects for whom written informed consent was obtained prior to any study specific procedure | Percentage of participants | NUMBER |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 outcomesResults by group354
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants Who Remained in Treatment Free Remission (TFR) Without Molecular Relapse 12 Months After Entering the TFR Phase | Nilotinib 24-month Treatment | 31.9 | 95% Confidence Interval 23.7 to 41.1 | Percentage of participants | |
| Percentage of Participants Who Remained in Treatment Free Remission (TFR) Without Molecular Relapse 12 Months After Entering the TFR Phase | Nilotinib 36-month Treatment | 37.5 | 95% Confidence Interval 28.2 to 47.5 | Percentage of participants | |
| Cumulative Incidence of MMR During the Pre-randomization Induction/Consolidation Phase Among Participants Without That Response at Study Entry | Nilotinib 24-month Treatment | Up to 3 months after study treatment start (pre-randomization) | 69.6 | 95% Confidence Interval 47.1 to 86.8 | Percentage of participants |
| Cumulative Incidence of MMR During the Pre-randomization Induction/Consolidation Phase Among Participants Without That Response at Study Entry | Nilotinib 36-month Treatment | Up to 3 months after study treatment start (pre-randomization) | 37.5 | 95% Confidence Interval 15.2 to 64.6 | Percentage of participants |
| Cumulative Incidence of MMR During the Pre-randomization Induction/Consolidation Phase Among Participants Without That Response at Study Entry | Not Randomized | Up to 3 months after study treatment start (pre-randomization) | 29.6 | 95% Confidence Interval 20.8 to 39.7 | Percentage of participants |
| Cumulative Incidence of MMR During the Pre-randomization Induction/Consolidation Phase Among Participants Without That Response at Study Entry | Nilotinib 24-month Treatment | Up to 6 months after study treatment start (pre-randomization) | 100 | 95% Confidence Interval 85.2 to 100.0 | Percentage of participants |
| Cumulative Incidence of MMR During the Pre-randomization Induction/Consolidation Phase Among Participants Without That Response at Study Entry | Nilotinib 36-month Treatment | Up to 6 months after study treatment start (pre-randomization) | 100.0 | 95% Confidence Interval 79.4 to 100.0 | Percentage of participants |
| Cumulative Incidence of MMR During the Pre-randomization Induction/Consolidation Phase Among Participants Without That Response at Study Entry | Not Randomized | Up to 6 months after study treatment start (pre-randomization) | 64.3 | 95% Confidence Interval 54.0 to 73.7 | Percentage of participants |
| Cumulative Incidence of MMR During the Pre-randomization Induction/Consolidation Phase Among Participants Without That Response at Study Entry | Nilotinib 24-month Treatment | Up to 9 months after study treatment start (pre-randomization) | 100 | 95% Confidence Interval 85.2 to 100.0 | Percentage of participants |
| Cumulative Incidence of MMR During the Pre-randomization Induction/Consolidation Phase Among Participants Without That Response at Study Entry | Nilotinib 36-month Treatment | Up to 9 months after study treatment start (pre-randomization) | 100.0 | 95% Confidence Interval 79.4 to 100.0 | Percentage of participants |
1 to 10 of 354Source ClinicalTrials.gov
See all 354 results by groupParticipant flow30
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Phase | STARTED | Nilotinib 24-month Treatment | 120 | |
| Treatment Phase | STARTED | Nilotinib 36-month Treatment | 119 | |
| Treatment Phase | STARTED | Not Randomized | 381 | |
| Treatment Phase | Participants Who Signed Informed Consent | Nilotinib 24-month Treatment | 120 | |
| Treatment Phase | Participants Who Signed Informed Consent | Nilotinib 36-month Treatment | 118 | |
| Treatment Phase | Participants Who Signed Informed Consent | Not Randomized | 381 | |
| Treatment Phase | COMPLETED | Nilotinib 24-month Treatment | 119 | |
| Treatment Phase | COMPLETED | Nilotinib 36-month Treatment | 104 | |
| Treatment Phase | COMPLETED | Not Randomized | 0 | |
| Treatment Phase | NOT COMPLETED | Nilotinib 24-month Treatment | 1 |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 participant flowAdverse events776
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 | Abdominal pain | Gastrointestinal disorders | Nilotinib 24-month Treatment | 7 | 120 | |
| other | Abdominal pain | Gastrointestinal disorders | Nilotinib 36-month Treatment | 6 | 118 | |
| other | Abdominal pain | Gastrointestinal disorders | Not Randomized | 15 | 381 | |
| other | Abdominal pain | Gastrointestinal disorders | Total | 28 | 619 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Nilotinib 24-month Treatment | 12 | 120 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Nilotinib 36-month Treatment | 14 | 118 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Not Randomized | 19 | 381 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Total | 45 | 619 | |
| other | Constipation | Gastrointestinal disorders | Nilotinib 24-month Treatment | 19 | 120 | |
| other | Constipation | Gastrointestinal disorders | Nilotinib 36-month Treatment | 14 | 118 |
1 to 10 of 776Source ClinicalTrials.gov
See all 776 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Nilotinib 24-month Treatment | 1 | 120 |
| Total, all-cause mortality | Nilotinib 36-month Treatment | 1 | 118 |
| Total, all-cause mortality | Not Randomized | 3 | 381 |
| Total, all-cause mortality | Total | 5 | 619 |
| Total, other adverse events | Nilotinib 24-month Treatment | 97 | 120 |
| Total, other adverse events | Nilotinib 36-month Treatment | 104 | 118 |
| Total, other adverse events | Not Randomized | 280 | 381 |
| Total, other adverse events | Total | 481 | 619 |
| Total, serious adverse events | Nilotinib 24-month Treatment | 33 | 120 |
| Total, serious adverse events | Nilotinib 36-month Treatment | 27 | 118 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 34123791 | DERIVED |
Source PubMed
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.
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