Trial · NCT00109707
A Study of Oral AMN107 in Adults With Chronic Myelogenous Leukemia (CML) or Other Hematologic Malignancies
NCT00109707, Phase 1, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2021-06-29
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Oral AMN107 in Adults With Chronic Myelogenous Leukemia (CML) or Other Hematologic Malignancies. Trials, record NCT00109707. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00109707
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Oral AMN107 in Adults With Chronic Myelogenous Leukemia (CML) or Other Hematologic Malignancies
- NCT number
- NCT00109707
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 507 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Chronic Myelogenous Leukemia, Acute Lymphoblastic Leukemia (Philadelphia Chromosome Positive), Hypereosinophilic Syndrome, Systemic Mastocytosis
- Interventions
- Nilotinib
- First posted
- 2005-05-03
- Last updated
- 2021-06-29
- Results first posted
- 2021-06-29
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Major Cytogenetic Response (MCyR) | Up to End of the Treatment (Approximately 7.5 years) | Major Cytogenetic Response (MCyR) is defined as Complete Cytogenetic Response (CCyR: 0% Ph-chromosome-positive cells in metaphase in bone marrow) or Partial Cytogenetic Response (PCyR: 1-35% Ph-chromosome-positive cells in metaphase in bone marrow). |
| Primary | Number of Participants Confirmed Overall Hematological Response (Phase II) | Up to End of the Treatment (Approximately 7.5 years) | Hematologic response is defined as the percentage of participants in complete hematologic response (defined as the following present for at least 4 weeks: WBC count \<10 x 109/L, Platelet count \<450 x 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). Hematological response was a primary outcome measure for Arm CML-AP with prior imatinib only. |
| Secondary | Number of Participants With Overall Major Cytogenetic Responses (Phase II) | Up to End of the Treatment (Approximately 7.5 years) | Cytogenetic responses (CyRs) in CML are based on the percentage of Ph+ metaphases in bone marrow; a value of 0% Ph+ metaphases defines a complete cytogenetic response (CCyR) and \>0% to 35% defines a major cytogenetic response (MCyR). Achievement of CCyR is associated with a significant survival advantage in participants with CML-CP. MCyR was categorized as either CCyR or partial cytogenetic response (PCyR). |
| Secondary | Number of Participants With Complete Hematologic Response (Phase II) | Up to End of the Treatment (Approximately 7.5 years) | A Complete Hematologic Response (CHR) is obtained when all the following criteria are met: WBC ≤ institutional ULN; platelets ≤ 450,000/mm3; ≤20% basophils in peripheral blood; no blasts or promyelocytes in PB cells; \< 5% myelocytes plus metamyelocytes in PB cells; no extra-medullary involvement including no hepatomegaly or splenomegaly. |
| Secondary | Participants With (MMR) Major Molecular Response (Phase II) | Up to End of the Treatment (Approximately 7.5 years) | MMR was defined for participants who demonstrated a reduction in BCR-ABL/control gene % transcripts to ≤0.1% based on international scale. The control gene used may have been either BCR or ABL. |
| Secondary | Time to Progression (TTP) (Phase II) | Up to End of the Treatment (Approximately 7.5 years) | Time to Progression was defined as the time from the start of nilotinib to the earliest date of Disease Progression (PD), discontinuation due to PD or death. Disease progression was defined as an increase in the number of circulating leukemic cells via cytogenetic assessment using real-time quantitative reverse transcriptase polymerase chain reaction (PCR). |
| Secondary | Overall Survival (OS) (Phase II) | Up to End of the Treatment (Approximately 7.5 years) | OS was calculated for all participants as the time between the start of nilotinib and death. Participants were followed for survival after discontinuation, every 3 months, and were included in the analysis of OS. Censoring was at the date of the last contact for discontinued participants and followed up for survival. |
| Secondary | Number of Participants With Adverse Events and Serious Adverse Events to Evaluate Long Term Safety | From First Participant First Visit to Last Participant Last Visit (Approximately 7.5 years) | Adverse events (AEs) were defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events (SAEs) were defined as any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalization, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgement of investigators represent significant hazards. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Major Cytogenetic Response (MCyR) | Up to End of the Treatment (Approximately 7.5 years) | Full Analysis Set (FAS): All participants who received at least one dose of study medication | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants Confirmed Overall Hematological Response (Phase II) | Up to End of the Treatment (Approximately 7.5 years) | Full Analysis Set (FAS): All participants who received at least one dose of study medication. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Overall Major Cytogenetic Responses (Phase II) | Up to End of the Treatment (Approximately 7.5 years) | Full Analysis Set (FAS): All participants who received at least one dose of study medication | participants | NUMBER |
| SECONDARY | Number of Participants With Complete Hematologic Response (Phase II) | Up to End of the Treatment (Approximately 7.5 years) | Full Analysis Set (FAS): All participants who received at least one dose of study medication | participants | NUMBER |
| SECONDARY | Participants With (MMR) Major Molecular Response (Phase II) | Up to End of the Treatment (Approximately 7.5 years) | Full Analysis Set (FAS): All participants who received at least one dose of study medication.~Major molecular response was not an efficacy variable for participants who received the CML-CP with prior imatinib and other TKI (Group B E8) | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Time to Progression (TTP) (Phase II) | Up to End of the Treatment (Approximately 7.5 years) | Full Analysis Set (FAS): All participants who received at least one dose of study medication.~Time to progression was not an efficacy variable for participants who received the CML-CP with prior imatinib and other TKI (Group B E8). | Months | MEDIAN |
| SECONDARY | Overall Survival (OS) (Phase II) | Up to End of the Treatment (Approximately 7.5 years) | Full Analysis Set (FAS): All participants who received at least one dose of study medication. | months | MEDIAN |
| SECONDARY | Number of Participants With Adverse Events and Serious Adverse Events to Evaluate Long Term Safety | From First Participant First Visit to Last Participant Last Visit (Approximately 7.5 years) | Safety set: All participants who received at least one dose of study medication and had at least one post baseline safety assessment. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group23
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Major Cytogenetic Response (MCyR) | CML-CP With Prior Imatinib Only | 191 | Participants | ||
| Number of Participants With Major Cytogenetic Response (MCyR) | CML-AP With Prior Imatinib Only | 44 | Participants | ||
| Number of Participants With Major Cytogenetic Response (MCyR) | CML-CP | 22 | Participants | ||
| Number of Participants Confirmed Overall Hematological Response (Phase II) | CML-AP With Prior Imatinib Only | 76 | Participants | ||
| Number of Participants With Overall Major Cytogenetic Responses (Phase II) | CML-CP With Prior Imatinib Only | 191 | participants | ||
| Number of Participants With Overall Major Cytogenetic Responses (Phase II) | CML-AP With Prior Imatinib Only | 44 | participants | ||
| Number of Participants With Overall Major Cytogenetic Responses (Phase II) | CML-CP | 22 | participants | ||
| Number of Participants With Complete Hematologic Response (Phase II) | CML-CP With Prior Imatinib Only | 158 | participants | ||
| Number of Participants With Complete Hematologic Response (Phase II) | CML-AP With Prior Imatinib Only | 76 | participants | ||
| Number of Participants With Complete Hematologic Response (Phase II) | CML-CP | 27 | participants |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | CML-CP With Prior Imatinib Only | 321 | |
| Overall Study | STARTED | CML-AP With Prior Imatinib Only | 137 | |
| Overall Study | STARTED | CML-CP | 49 | |
| Overall Study | Entered Extension Study | CML-CP With Prior Imatinib Only | 106 | |
| Overall Study | Entered Extension Study | CML-AP With Prior Imatinib Only | 15 | |
| Overall Study | Entered Extension Study | CML-CP | 15 | |
| Overall Study | COMPLETED | CML-CP With Prior Imatinib Only | 67 | |
| Overall Study | COMPLETED | CML-AP With Prior Imatinib Only | 8 | |
| Overall Study | COMPLETED | CML-CP | 8 | |
| Overall Study | NOT COMPLETED | CML-CP With Prior Imatinib Only | 254 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events576
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 | Thrombocytopenia | Blood and lymphatic system disorders | CML-CP With Prior Imatinib Only | 107 | 321 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | CML-AP With Prior Imatinib Only | 60 | 137 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | CML-CP | 16 | 49 | |
| other | Anaemia | Blood and lymphatic system disorders | CML-CP With Prior Imatinib Only | 71 | 321 | |
| other | Anaemia | Blood and lymphatic system disorders | CML-AP With Prior Imatinib Only | 61 | 137 | |
| other | Anaemia | Blood and lymphatic system disorders | CML-CP | 9 | 49 | |
| other | Neutropenia | Blood and lymphatic system disorders | CML-CP With Prior Imatinib Only | 57 | 321 | |
| other | Neutropenia | Blood and lymphatic system disorders | CML-AP With Prior Imatinib Only | 39 | 137 | |
| other | Neutropenia | Blood and lymphatic system disorders | CML-CP | 15 | 49 | |
| other | Leukopenia | Blood and lymphatic system disorders | CML-CP With Prior Imatinib Only | 16 | 321 |
1 to 10 of 576Source ClinicalTrials.gov
See all 576 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | CML-CP With Prior Imatinib Only | 13 | 321 |
| Total, all-cause mortality | CML-AP With Prior Imatinib Only | 14 | 137 |
| Total, all-cause mortality | CML-CP | 0 | 49 |
| Total, other adverse events | CML-CP With Prior Imatinib Only | 319 | 321 |
| Total, other adverse events | CML-AP With Prior Imatinib Only | 136 | 137 |
| Total, other adverse events | CML-CP | 48 | 49 |
| Total, serious adverse events | CML-CP With Prior Imatinib Only | 146 | 321 |
| Total, serious adverse events | CML-AP With Prior Imatinib Only | 55 | 137 |
| Total, serious adverse events | CML-CP | 21 | 49 |
Source ClinicalTrials.gov
Papers6
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| CML-CP With Prior Imatinib Only vs CML-AP With Prior Imatinib Only: Participants With (MMR) Major Molecular Response (Phase II) |
Source Trial comparisons
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/NCT00109707
GET https://conductscience.org/api/trials/v1/NCT00109707/results?page=2