Trial · NCT01826214
Study of Efficacy and Safety of LDE225 in Adult Patients With Relapsed/Refractory Acute Leukemia
NCT01826214, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2016-05-20
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Efficacy and Safety of LDE225 in Adult Patients With Relapsed/Refractory Acute Leukemia. Trials, record NCT01826214. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01826214
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Efficacy and Safety of LDE225 in Adult Patients With Relapsed/Refractory Acute Leukemia
- NCT number
- NCT01826214
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 70 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Acute Leukemias
- Interventions
- LDE225
- First posted
- 2013-04-08
- Last updated
- 2016-08-30
- Results first posted
- 2016-05-20
- Limitations and caveats
- Only 34 of the 35 patients in the LDE225 800 mg once daily arm received at least dose of study drug were analyzed for safety.
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Rate of Complete Remission (CR) | at screening, every week up to Week 9, every 2 weeks thereafter until CR, every 4 weeks after CR up to 24 months | Complete Response (CR) was based on the International Working Group (IWG) criteria based on weekly peripheral blood count measurements and bone marrow biopsy/aspiration collection. Efficacy assessments were performed to determine CR. A treatment cycle was defined as 4 weeks. The outcome measure for the study is based on standardized response criteria as defined by the International Working Group (IWG) for AML. The IWG was established by a group of investigators interested in the design and conduct of clinical trials in acute myeloid leukemia (AML). The criteria established by this group (a set of recommendations for response assessment) are well established, endorsed by major institutions and Health Authorities, and are widely used in clinical trials. No statistical analysis was planned for this primary outcome. |
| Primary | Complete Remission With Incomplete Blood Count Recovery (CRi) | within 3 days after clearance of blasts from peripheral blood (PB), monthly thereafter until CR or reappearance of blasts in the PB, after CR every other month until discontination up to 24 months | The other primary efficacy endpoint was CRi based on the International Working Group (IWG) criteria based on weekly peripheral blood count measurements and bone marrow biopsy/aspiration collection. Efficacy assessments were performed to determine CRi. A treatment cycle was defined as 4 weeks. No statistical analysis was planned for this primary outcome. |
| Secondary | Overall Response Rate (ORR) | Every 8 weeks for the first 6 months and every 12 weeks until 53 weeks after the last patient is enrolled or until relapse up to 24 months | ORR was the rate of complete remission (CR), complete remission with incomplete blood count recovery (CRi) or partial response (PR) according to IWG criteria. CR, CRi or PR will be assessed through bone marrow biopsy/aspirate and peripheral blood blasts counts. |
| Secondary | Parmacokintics (PK) Parameter: Cmax | Week 1 Day 1, Week 9 Day 1 | Cmax is the maximum observed plasma concentration after drug administration.The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the pharmacokineticist. Cmax was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters. |
| Secondary | Parmacokintics (PK) Parameter: Tmax | Week 1 Day 1,Week 9 Day 1 | Tmax is the time to reach Cmax. The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the Pharmacokineticist. Tmax was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters. |
| Secondary | Parmacokintics (PK) Parameter: AUC0-8h | Week 1 Day 1,Week 9 Day 1 | AUC0-8h is the area under the concentration-time curve from time zero to 8 hours. The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the Pharmacokineticist. AUC0-8h was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters. |
| Secondary | Parmacokintics (PK) Parameter: AUC0-24h | Week 1 Day 1,Week 9 Day 1 | AUC0-24 is the area under the concentration-time curve from time zero to 24 hours done only on 800 mg once a day schedule. The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the Pharmacokineticist. AUC0-24h was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Rate of Complete Remission (CR) | at screening, every week up to Week 9, every 2 weeks thereafter until CR, every 4 weeks after CR up to 24 months | Full analysis set (FAS): comprised of all patients who were randomized to a study treatment.~According to the intent-to-treat principle, patient data were analyzed according to the treatment to which they had been randomized. No statistical analysis were reported in this study. | Participants | NUMBER |
| PRIMARY | Complete Remission With Incomplete Blood Count Recovery (CRi) | within 3 days after clearance of blasts from peripheral blood (PB), monthly thereafter until CR or reappearance of blasts in the PB, after CR every other month until discontination up to 24 months | Full analysis set (FAS): comprised of all patients who were randomized to a study treatment.~According to the intent-to-treat principle, patient data were analyzed according to the treatment to which they had been randomized. | Participants | NUMBER |
| SECONDARY | Overall Response Rate (ORR) | Every 8 weeks for the first 6 months and every 12 weeks until 53 weeks after the last patient is enrolled or until relapse up to 24 months | Full analysis set (FAS): comprised of all patients who were randomized to a study treatment.~According to the intent-to-treat principle, patient data were analyzed according to the treatment to which they had been randomized. | Participants | NUMBER |
| SECONDARY | Parmacokintics (PK) Parameter: Cmax | Week 1 Day 1, Week 9 Day 1 | Pharmacokinetic analysis set (PAS): consisted of all patients who received at least one dose of sonidegib and provided at least one evaluable PK blood sample. | ng/mL | MEAN |
| SECONDARY | Parmacokintics (PK) Parameter: Tmax | Week 1 Day 1,Week 9 Day 1 | Pharmacokinetic analysis set (PAS): consisted of all patients who received at least one dose of sonidegib and provided at least one evaluable PK blood sample. | hr | MEDIAN |
| SECONDARY | Parmacokintics (PK) Parameter: AUC0-8h | Week 1 Day 1,Week 9 Day 1 | Pharmacokinetic analysis set (PAS): consisted of all patients who received at least one dose of sonidegib and provided at least one evaluable PK blood sample. | ng*hr/mL | MEAN |
| SECONDARY | Parmacokintics (PK) Parameter: AUC0-24h | Week 1 Day 1,Week 9 Day 1 | Pharmacokinetic analysis set (PAS): consisted of all patients who received at least one dose of sonidegib and provided at least one evaluable PK blood sample. | ng*hr/mL | MEAN |
Source ClinicalTrials.gov
Results by group22
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Rate of Complete Remission (CR) | LDE225-400 | 0 | Participants | ||
| Rate of Complete Remission (CR) | LDE225-800 | 0 | Participants | ||
| Complete Remission With Incomplete Blood Count Recovery (CRi) | LDE225-400 | 1 | Participants | ||
| Complete Remission With Incomplete Blood Count Recovery (CRi) | LDE225-800 | 0 | Participants | ||
| Overall Response Rate (ORR) | LDE225-400 | 1 | Participants | ||
| Overall Response Rate (ORR) | LDE225-800 | 0 | Participants | ||
| Parmacokintics (PK) Parameter: Cmax | LDE225-400 | Week 1 Day 1 | 237 | Standard Deviation 158 | ng/mL |
| Parmacokintics (PK) Parameter: Cmax | LDE225-800 | Week 1 Day 1 | 343 | Standard Deviation 275 | ng/mL |
| Parmacokintics (PK) Parameter: Cmax | LDE225-400 | Week 9 Day 1(n: 7, 7) | 1640 | Standard Deviation 612 | ng/mL |
| Parmacokintics (PK) Parameter: Cmax | LDE225-800 | Week 9 Day 1(n: 7, 7) | 1500 | Standard Deviation 874 | ng/mL |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 results by groupParticipant flow10
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | LDE225-400 | 35 | |
| Overall Study | STARTED | LDE225-800 | 34 | |
| Overall Study | Untreated | LDE225-400 | 0 | |
| Overall Study | Untreated | LDE225-800 | 1 | |
| Overall Study | Treated | LDE225-400 | 35 | |
| Overall Study | Treated | LDE225-800 | 34 | |
| Overall Study | COMPLETED | LDE225-400 | 0 | |
| Overall Study | COMPLETED | LDE225-800 | 0 | |
| Overall Study | NOT COMPLETED | LDE225-400 | 35 | |
| Overall Study | NOT COMPLETED | LDE225-800 | 34 |
Source ClinicalTrials.gov
Adverse events284
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 | LDE225-400 | 5 | 35 | |
| other | ANAEMIA | Blood and lymphatic system disorders | LDE225-800 | 6 | 34 | |
| other | FEBRILE NEUTROPENIA | Blood and lymphatic system disorders | LDE225-400 | 3 | 35 | |
| other | FEBRILE NEUTROPENIA | Blood and lymphatic system disorders | LDE225-800 | 2 | 34 | |
| other | LEUKOCYTOSIS | Blood and lymphatic system disorders | LDE225-400 | 1 | 35 | |
| other | LEUKOCYTOSIS | Blood and lymphatic system disorders | LDE225-800 | 4 | 34 | |
| other | LEUKOPENIA | Blood and lymphatic system disorders | LDE225-400 | 2 | 35 | |
| other | LEUKOPENIA | Blood and lymphatic system disorders | LDE225-800 | 3 | 34 | |
| other | NEUTROPENIA | Blood and lymphatic system disorders | LDE225-400 | 3 | 35 | |
| other | NEUTROPENIA | Blood and lymphatic system disorders | LDE225-800 | 2 | 34 |
1 to 10 of 284Source ClinicalTrials.gov
See all 284 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | LDE225-400 | ||
| Total, all-cause mortality | LDE225-800 | ||
| Total, other adverse events | LDE225-400 | 35 | 35 |
| Total, other adverse events | LDE225-800 | 34 | 34 |
| Total, serious adverse events | LDE225-400 | 25 | 35 |
| Total, serious adverse events | LDE225-800 | 25 | 34 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons4
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| LDE225-400 vs LDE225-800: Rate of Complete Remission (CR) | Trial comparisons |
| LDE225-400 vs LDE225-800: Overall Response Rate (ORR) | Trial comparisons |
| LDE225-400 vs LDE225-800: Complete Remission With Incomplete Blood Count Recovery (CRi) | Trial comparisons |
| LDE225-400 vs LDE225-800: serious adverse events | 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/NCT01826214
GET https://conductscience.org/api/trials/v1/NCT01826214/results?page=2