Trial · NCT00946647
A Phase Ib/IIb, Open-label, Multi-center, Study of Oral Panobinostat Administered With 5-Azacitidine (in Adult Patients With Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), or Acute Myeloid Leukemia (AML).
NCT00946647, Phase 1, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2020-06-23
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Phase Ib/IIb, Open-label, Multi-center, Study of Oral Panobinostat Administered With 5-Azacitidine (in Adult Patients With Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), or Acute Myeloid Leukemia (AML). Trials, record NCT00946647. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00946647
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
A Phase Ib/IIb, Open-label, Multi-center, Study of Oral Panobinostat Administered With 5-Azacitidine (in Adult Patients With Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), or Acute Myeloid Leukemia (AML).
- NCT number
- NCT00946647
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 113 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, Acute Myeloid Leukemia
- Interventions
- Panobinostat (LBH589), 5-Azacytidine
- First posted
- 2009-07-27
- Last updated
- 2020-08-04
- Other registries' IDs
- eudract:2009-010548-32
- Results first posted
- 2020-06-23
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Dose Limiting Toxicity (DLT) (Phase lb) | within the first 28 days (cycle 1) | Dose limiting toxicity (DLT) was defined as a toxicity requiring treatment withdrawal and included the following: Non-hematologic toxicity qualifying for DLT and Hematologic toxicity qualifying for DLT |
| Primary | Number of Dose Limiting Toxicity (DLT) (Phase lb) | within the first 28 days (cycle 1) | Dose limiting toxicity (DLT) was defined as a toxicity requiring treatment withdrawal and included the following: Non-hematologic toxicity qualifying for DLT and Hematologic toxicity qualifying for DLT |
| Primary | Composite Complete Response (Phase Llb) | 48 months | Composite complete response is defined as complete response (CR), Complete response with incomplete blood count recovery (CRi) or bone marrow complete response (BM-CR) as defined by the International Working Group (IWG) response criteria. |
| Secondary | Clinical Response Other Than Composite Clinical Response for Myeloid Dysplastic Syndromes(MDS)/Chronic Myelomonocytic Leukemia (CMML) Patients Per Investigator (Phase Llb) | 48 months | This is the best overall response as measured by Clinical response. Clinical response is defined as having complete remission (CR), bone marrow complete remission (BM-CR), partial remission or hematologic improvement (HI) as defined by the International Working Group (IWG) response criteria. |
| Secondary | Clinical Response Other Than Composite Clinical Response for Acute Myelogenous Leukemia (AML) Patients Per Investigator (Phase Llb) | 48 months | This is the best overall response as measured by Clinical response. Clinical response is defined as having complete remission (CR), complete remission with incomplete blood count recovery (CRi) or partial remission as defined by the International Working Group (IWG) response criteria. |
| Secondary | Overall Response Rate (ORR) Assessed by Best Overall Response: Participants With MDS/CMML Per Investigator (Phase Llb) | 48 months | Best overall response as measured by complete remission (CR) or bone marrow CR (BM-CR) or partial remission (PR) or hematologic improvement (HI). Overall response patients achieved other than the composite CR by individual response category: CR, CRi, mCR or PR as defined by the International Working Group (IWG) response criteria. |
| Secondary | Overall Response Rate (ORR) Assessed by Best Overall Response: Participants With AML Per Investigator (Phase Llb) | 48 months | Best overall response as measured by complete remission (CR) or complete response with incomplete blood count recovery (CRi) or partial remission (PR). Overall response patients achieved other than the composite CR by individual response category: CR, CRi or PR. |
| Secondary | Hematologic Improvement (HI) for Myeloid Dysplastic Syndromes(MDS)/Chronic Myelomonocytic Leukemia (CMML) Patients Per Investigator (Phase Llb) | 48 months | Hematologic response consists of Erythroid response (HI-E), Platelet response (HI-P) and Neutrophil response (HI-N). HI-E: Hgb increase by ≥ 1.5 g/dL over pretreatment \& relevant reduction of units of RBC transfusions by an absolute number of at least 4 units of PRBCs/8 weeks compared with the pretreatment transfusion number in the previous 8 weeks. Only RBC transfusions given for a Hgb of ≤ 9.0 g/dL pretreatment will count in the RBC transfusion response evaluation. HI-P: Absolute increase of ≥ 30 x 109/L over pretreatment or patients starting with ≥ 20 x 109/L platelets OR increase from \<20 x 109/L at pretreatment to \> 20 x 109/L and by at least 100%. HI-N: At least 100% increase and an absolute increase \> 0.5 x 109/L over pretreatment value. |
| Secondary | 1-year Survival Rate (Phase Llb) | 12 months | Overall survival was defined as the time from date of randomization to date of death due to any cause. If a patient was not known to have died, survival was censored at the date of last contact. Patients not known to have died were censored for 'Lost to follow-up' if the time between their last contact date and the analysis cut-off date was longer than 3 months and 2 weeks (104 days) during the first year after study evaluation completion, and longer than 6 months and 2 weeks (194 days), thereafter. The 1-year survival rate was obtained from the Kaplan-Meier analysis of overall survival, and its variance was estimated by Greenwood's formula. |
| Secondary | Time to Progression (TTP) (Phase Llb) | 48 months | Time to progression (TTP) was defined as the time from the date of randomization to the date of the first documented PD per investigator's assessment or death due to study indication. Time to progression was analyzed by the Kaplan Meier method. Based on the Guidelines for Implementation of international working group (IWG) response criteria in AML, MDS and CMML according to Cheson 2003 and 2006. |
Source ClinicalTrials.gov
Outcomes10
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 Dose Limiting Toxicity (DLT) (Phase lb) | within the first 28 days (cycle 1) | Maximum Tolerated Dose (MTD) determining set: Consisted of all patients of the safety set who either received sufficient study treatment as defined in the minimum exposure criteria in Cycle 1 (patients had to have 100% of the planned dose of each compound), and had sufficient safety evaluations or discontinued due to DLT in Cycle 1. | Participants | NUMBER |
| PRIMARY | Number of Dose Limiting Toxicity (DLT) (Phase lb) | within the first 28 days (cycle 1) | Maximum Tolerated Dose (MTD) determining set: Consisted of all patients of the safety set who either received sufficient study treatment as defined in the minimum exposure criteria in Cycle 1 (patients had to have 100% of the planned dose of each compound), and had sufficient safety evaluations or discontinued due to DLT in Cycle 1. | DLTs | NUMBER |
| PRIMARY | Composite Complete Response (Phase Llb) | 48 months | Full analysis set: Consisted of all patients who were randomized to one of the two treatment arms | Percentage of participants | NUMBER |
| SECONDARY | Clinical Response Other Than Composite Clinical Response for Myeloid Dysplastic Syndromes(MDS)/Chronic Myelomonocytic Leukemia (CMML) Patients Per Investigator (Phase Llb) | 48 months | Full analysis set: Consisted of all patients who were randomized to one of the two treatment arms | Percentage of participants | NUMBER |
| SECONDARY | Clinical Response Other Than Composite Clinical Response for Acute Myelogenous Leukemia (AML) Patients Per Investigator (Phase Llb) | 48 months | Full analysis set: Consisted of all patients who were randomized to one of the two treatment arms | Percentage of participants | NUMBER |
| SECONDARY | Overall Response Rate (ORR) Assessed by Best Overall Response: Participants With MDS/CMML Per Investigator (Phase Llb) | 48 months | Full analysis set (FAS): Consisted of all patients who were randomized to one of the two treatment arms. | Percentage of participants | NUMBER |
| SECONDARY | Overall Response Rate (ORR) Assessed by Best Overall Response: Participants With AML Per Investigator (Phase Llb) | 48 months | Full analysis set (FAS): Consisted of all patients who were randomized to one of the two treatment arms. | Percentage of participants | NUMBER |
| SECONDARY | Hematologic Improvement (HI) for Myeloid Dysplastic Syndromes(MDS)/Chronic Myelomonocytic Leukemia (CMML) Patients Per Investigator (Phase Llb) | 48 months | Full analysis set: Consisted of all patients who were randomized to one of the two treatment arms | Percentage of participants | NUMBER |
| SECONDARY | 1-year Survival Rate (Phase Llb) | 12 months | Full analysis set: Consisted of all patients who were randomized to one of the two treatment arms | months | MEDIAN |
| SECONDARY | Time to Progression (TTP) (Phase Llb) | 48 months | Full analysis set: Consisted of all patients who were randomized to one of the two treatment arms | months | MEDIAN |
Source ClinicalTrials.gov
Results by group38
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 Dose Limiting Toxicity (DLT) (Phase lb) | PAN + 5-Aza 20 mg | 1 | Participants | ||
| Number of Participants With Dose Limiting Toxicity (DLT) (Phase lb) | PAN + 5-Aza 30 mg | 3 | Participants | ||
| Number of Participants With Dose Limiting Toxicity (DLT) (Phase lb) | PAN + 5-Aza 40 mg | 2 | Participants | ||
| Number of Dose Limiting Toxicity (DLT) (Phase lb) | PAN + 5-Aza 20 mg | 2 | DLTs | ||
| Number of Dose Limiting Toxicity (DLT) (Phase lb) | PAN + 5-Aza 30 mg | 6 | DLTs | ||
| Number of Dose Limiting Toxicity (DLT) (Phase lb) | PAN + 5-Aza 40 mg | 4 | DLTs | ||
| Composite Complete Response (Phase Llb) | Panobinostat + 5-Azacytidine | 27.5 | 95% Confidence Interval 14.60 to 43.89 | Percentage of participants | |
| Composite Complete Response (Phase Llb) | 5-Azacytidine | 14.3 | 95% Confidence Interval 5.43 to 28.54 | Percentage of participants | |
| Clinical Response Other Than Composite Clinical Response for Myeloid Dysplastic Syndromes(MDS)/Chronic Myelomonocytic Leukemia (CMML) Patients Per Investigator (Phase Llb) | Panobinostat + 5-Azacytidine | 41.9 | 95% Confidence Interval 24.5 to 60.9 | Percentage of participants | |
| Clinical Response Other Than Composite Clinical Response for Myeloid Dysplastic Syndromes(MDS)/Chronic Myelomonocytic Leukemia (CMML) Patients Per Investigator (Phase Llb) | 5-Azacytidine | 41.4 | 95% Confidence Interval 23.5 to 61.1 | Percentage of participants |
1 to 10 of 38Source ClinicalTrials.gov
See all 38 results by groupParticipant flow35
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Phase 1 Part | STARTED | PAN + 5-Aza 20 mg | 6 | |
| Phase 1 Part | STARTED | PAN + 5-Aza 30 mg | 18 | |
| Phase 1 Part | STARTED | PAN + 5-Aza 40 mg | 7 | |
| Phase 1 Part | STARTED | Panobinostat + 5-Azacytidine | 0 | |
| Phase 1 Part | STARTED | 5-Azacytidine | 0 | |
| Phase 1 Part | COMPLETED | PAN + 5-Aza 20 mg | 0 | |
| Phase 1 Part | COMPLETED | PAN + 5-Aza 30 mg | 0 | |
| Phase 1 Part | COMPLETED | PAN + 5-Aza 40 mg | 0 | |
| Phase 1 Part | COMPLETED | Panobinostat + 5-Azacytidine | 0 | |
| Phase 1 Part | COMPLETED | 5-Azacytidine | 0 |
1 to 10 of 35Source ClinicalTrials.gov
See all 35 participant flowAdverse events3,000
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 | Phase Ib PAN + 5-Aza 20mg | 2 | 6 | |
| other | Anaemia | Blood and lymphatic system disorders | Phase Ib PAN + 5-Aza 30mg | 8 | 18 | |
| other | Anaemia | Blood and lymphatic system disorders | Phase Ib PAN + 5-Aza 40mg | 4 | 7 | |
| other | Anaemia | Blood and lymphatic system disorders | Phase IIb PAN + 5-Aza | 12 | 38 | |
| other | Anaemia | Blood and lymphatic system disorders | Phase IIb 5-Aza | 14 | 42 | |
| other | Anaemia | Blood and lymphatic system disorders | Phase Ib and IIb | 40 | 111 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Phase Ib PAN + 5-Aza 20mg | 0 | 6 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Phase Ib PAN + 5-Aza 30mg | 3 | 18 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Phase Ib PAN + 5-Aza 40mg | 0 | 7 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Phase IIb PAN + 5-Aza | 6 | 38 |
1 to 10 of 3,000Source ClinicalTrials.gov
See all 3,000 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Phase Ib PAN + 5-Aza 20mg | 0 | 6 |
| Total, all-cause mortality | Phase Ib PAN + 5-Aza 30mg | 1 | 18 |
| Total, all-cause mortality | Phase Ib PAN + 5-Aza 40mg | 2 | 7 |
| Total, all-cause mortality | Phase IIb PAN + 5-Aza | 6 | 38 |
| Total, all-cause mortality | Phase IIb 5-Aza | 3 | 42 |
| Total, all-cause mortality | Phase Ib and IIb | 12 | 111 |
| Total, other adverse events | Phase Ib PAN + 5-Aza 20mg | 6 | 6 |
| Total, other adverse events | Phase Ib PAN + 5-Aza 30mg | 18 | 18 |
| Total, other adverse events | Phase Ib PAN + 5-Aza 40mg | 7 | 7 |
| Total, other adverse events | Phase IIb PAN + 5-Aza | 38 | 38 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 28546581 | 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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