Trial · NCT01747876
Study of Safety and Efficacy in Patients With Malignant Rhabdoid Tumors (MRT) and Neuroblastoma
NCT01747876, Phase 1, Terminated, Novartis Pharmaceuticals; results first posted 2019-02-28
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Safety and Efficacy in Patients With Malignant Rhabdoid Tumors (MRT) and Neuroblastoma. Trials, record NCT01747876. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01747876
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Safety and Efficacy in Patients With Malignant Rhabdoid Tumors (MRT) and Neuroblastoma
- NCT number
- NCT01747876
- Status
- Terminated
- Phases
- Phase 1
- Enrollment
- 32 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Malignant Rhabdoid Tumors (MRT), Neuroblastoma
- Interventions
- LEE011
- First posted
- 2012-12-12
- Last updated
- 2019-11-22
- Other registries' IDs
- eudract:2012-004228-40
- Results first posted
- 2019-02-28
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Incidence Rate of Dose Limiting Toxicities (DLTs) by Primary System Organ Class, Preferred Term and Treatment | cycle 1 = 28 days (from the time of first dose) | A DLT was defined as an AE or clinically significant abnormal laboratory value assessed as unrelated to disease, disease progression, inter-current illness, or concomitant medications that occurred within the first 28 days of treatment with LEE011 and met any of the predefined criteria. For the purpose of dose-escalation decisions, DLTs were considered and included in the Bayesian Logistic Regression Model (BLRM). Patients who did not experience DLT during the first cycle were considered to have had sufficient safety evaluations if they were observed for ≥ 28 days following the first dose and were considered to have had enough safety data to conclude that a DLT did not occur. Patients who did not meet these minimum safety evaluation requirements were regarded as ineligible for the DDS. A patient with multiple DLTs within a primary system organ class is counted only once in the total row. |
| Secondary | Overall Response Rate | Every 2 cycles (cycle = 28 days) up to end of treatment, the maximum time a patient was on study was 1311 days | This analysis was not done as there were no responders. |
| Secondary | Time to Disease Progression (TTP) Per RECIST 1.1 | Every 2 cycles (cycle = 28 days) up to end of treatment, the maximum time a patient was on study was 1311 days | TTP was assessed per Investigator, for the malignant rhabdoid tumor (MRT) \& neuroblastoma patients for the pooled maximum tolerated dose (MTD) \& recommended dose for expansion (RDE) according to RECIST 1.1 criteria using Kaplan-Meier method. Time to progression (TTP) is the time from date of randomization/start of treatment to the date of event defined as the first documented progression or death due to underlying cancer. If a patient had not had an event, time to progression was censored at the date of last adequate tumor assessment. At least a 20% increase in the sum of diameter of all measured target lesions, taking as reference the smallest sum of diameter of all target lesions recorded at or after baseline. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. |
| Secondary | Duration of Response (DOR) | Every 2 cycles (cycle = 28 days) up to end of treatment, the maximum time a patient was on study was 1311 days | Assess the anti-tumor activity of LEE011 by RECIST 1.1. DOR was not assessed. |
| Secondary | Pharmacokinetics (PK) Parameter: AUC0-24 | 0,1, 2, 4, 8 hours post dose Cycle 1 Day 1 (C1D1) and Cycle 1 Day 15 (C1D15) | The AUC calculated to the end of a dosing interval (tau) following single dose or at steady-state (amount x time x volume-1). PK parameters were estimated from individual plasma concentration-time profiles using noncompartmental methods in Phoenix WinNonlin. |
| Secondary | Pharmacokinetics (PK) Parameter: Cmax | C1D1, C1D15 | Cmax is the maximum (peak) observed plasma, blood, serum, or other body fluid drug concentration after single dose administration or at steady-state (mass x volume-1). PK parameters were estimated from individual plasma concentration-time profiles using noncompartmental methods in Phoenix WinNonlin |
| Secondary | Pharmacokinetics (PK) Parameter: Tmax | C1D1, C1D15 | Tmax is the time to reach maximum (peak) plasma, blood, serum, or other body fluid drug concentration after single dose administration or at steady-state (time). PK parameters were estimated from individual plasma concentration-time profiles using noncompartmental methods in Phoenix WinNonlin. |
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 | Incidence Rate of Dose Limiting Toxicities (DLTs) by Primary System Organ Class, Preferred Term and Treatment | cycle 1 = 28 days (from the time of first dose) | Dose-determining analysis set consisted of all pts from safety set who either met the following minimum exposure criterion \& had scheduled safety evaluations, or experienced a DLT. A patient was considered to have met the minimum exposure criterion if he/she had received at least 16 of 21 planned daily doses of LEE011 in first 28 days of dosing. | Participants | NUMBER |
| SECONDARY | Overall Response Rate | Every 2 cycles (cycle = 28 days) up to end of treatment, the maximum time a patient was on study was 1311 days | Full analysis set (FAS) included all patients who received at least one dose of LEE011. This analysis was not done as there were no responders. | months | MEDIAN |
| SECONDARY | Time to Disease Progression (TTP) Per RECIST 1.1 | Every 2 cycles (cycle = 28 days) up to end of treatment, the maximum time a patient was on study was 1311 days | Full analysis set (FAS) included all patients who received at least one dose of LEE011. | months | MEDIAN |
| SECONDARY | Duration of Response (DOR) | Every 2 cycles (cycle = 28 days) up to end of treatment, the maximum time a patient was on study was 1311 days | Full analysis set included all patients who received at least 1 dose of LEE011. Due to halted enrollment and/or lack of complete responses (CR) \& partial responses (PR), efficacy analysis was only performed in terms of DOR for the patients treated during the dose-escalation part at the MTD and RDE. Therefore, Duration of response was not assessed. | ||
| SECONDARY | Pharmacokinetics (PK) Parameter: AUC0-24 | 0,1, 2, 4, 8 hours post dose Cycle 1 Day 1 (C1D1) and Cycle 1 Day 15 (C1D15) | The pharmacokinetic analysis set (PAS) consisted of all patients who had at least one blood sample providing evaluable drug concentration data. | h*ng/ml | MEDIAN |
| SECONDARY | Pharmacokinetics (PK) Parameter: Cmax | C1D1, C1D15 | The pharmacokinetic analysis set (PAS) consisted of all patients who had at least one blood sample providing evaluable drug concentration data. | ng/ml | MEDIAN |
| SECONDARY | Pharmacokinetics (PK) Parameter: Tmax | C1D1, C1D15 | The pharmacokinetic analysis set (PAS) consisted of all patients who had at least one blood sample providing evaluable drug concentration data. | hour | MEDIAN |
Source ClinicalTrials.gov
Results by group35
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Incidence Rate of Dose Limiting Toxicities (DLTs) by Primary System Organ Class, Preferred Term and Treatment | LEE011 280 mg/m2 - Dose Escalation Only | Any primary system organ class total | 1 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) by Primary System Organ Class, Preferred Term and Treatment | LEE011 350 mg/m2 - Dose Escalation Only | Any primary system organ class total | 0 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) by Primary System Organ Class, Preferred Term and Treatment | LEE011 470 mg/m2 - Dose Escalation Only | Any primary system organ class total | 2 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) by Primary System Organ Class, Preferred Term and Treatment | LEE011 280 mg/m2 - Dose Escalation Only | Blood & lymphatic sys. disorders(Thrombocytopenia) | 0 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) by Primary System Organ Class, Preferred Term and Treatment | LEE011 350 mg/m2 - Dose Escalation Only | Blood & lymphatic sys. disorders(Thrombocytopenia) | 0 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) by Primary System Organ Class, Preferred Term and Treatment | LEE011 470 mg/m2 - Dose Escalation Only | Blood & lymphatic sys. disorders(Thrombocytopenia) | 1 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) by Primary System Organ Class, Preferred Term and Treatment | LEE011 280 mg/m2 - Dose Escalation Only | Gen. disorders & admin. site conditions (fatigue) | 1 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) by Primary System Organ Class, Preferred Term and Treatment | LEE011 350 mg/m2 - Dose Escalation Only | Gen. disorders & admin. site conditions (fatigue) | 0 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) by Primary System Organ Class, Preferred Term and Treatment | LEE011 470 mg/m2 - Dose Escalation Only | Gen. disorders & admin. site conditions (fatigue) | 0 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) by Primary System Organ Class, Preferred Term and Treatment | LEE011 280 mg/m2 - Dose Escalation Only | Investigations (Platelet count decreased) | 0 | Participants |
1 to 10 of 35Source ClinicalTrials.gov
See all 35 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | LEE011 280 mg/m2 - Dose Escalation Only | 5 | |
| Overall Study | STARTED | LEE011 350 mg/m2 - Dose Escalation Only | 15 | |
| Overall Study | STARTED | LEE011 470 mg/m2 - Dose Escalation Only | 12 | |
| Overall Study | COMPLETED | LEE011 280 mg/m2 - Dose Escalation Only | 0 | |
| Overall Study | COMPLETED | LEE011 350 mg/m2 - Dose Escalation Only | 0 | |
| Overall Study | COMPLETED | LEE011 470 mg/m2 - Dose Escalation Only | 0 | |
| Overall Study | NOT COMPLETED | LEE011 280 mg/m2 - Dose Escalation Only | 5 | |
| Overall Study | NOT COMPLETED | LEE011 350 mg/m2 - Dose Escalation Only | 15 | |
| Overall Study | NOT COMPLETED | LEE011 470 mg/m2 - Dose Escalation Only | 12 |
Source ClinicalTrials.gov
Adverse events820
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 | LEE011 280 mg/m2 - Dose Escalation Only | 1 | 5 | |
| other | Anaemia | Blood and lymphatic system disorders | LEE011 350 mg/m2 - Dose Escalation Only | 8 | 15 | |
| other | Anaemia | Blood and lymphatic system disorders | LEE011 470 mg/m2 - Dose Escalation Only | 6 | 12 | |
| other | Anaemia | Blood and lymphatic system disorders | All Patients | 15 | 32 | |
| other | Haemolysis | Blood and lymphatic system disorders | LEE011 280 mg/m2 - Dose Escalation Only | 0 | 5 | |
| other | Haemolysis | Blood and lymphatic system disorders | LEE011 350 mg/m2 - Dose Escalation Only | 0 | 15 | |
| other | Haemolysis | Blood and lymphatic system disorders | LEE011 470 mg/m2 - Dose Escalation Only | 1 | 12 | |
| other | Haemolysis | Blood and lymphatic system disorders | All Patients | 1 | 32 | |
| other | Leukopenia | Blood and lymphatic system disorders | LEE011 280 mg/m2 - Dose Escalation Only | 0 | 5 | |
| other | Leukopenia | Blood and lymphatic system disorders | LEE011 350 mg/m2 - Dose Escalation Only | 1 | 15 |
1 to 10 of 820Source ClinicalTrials.gov
See all 820 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | LEE011 280 mg/m2 - Dose Escalation Only | 2 | 5 |
| Total, all-cause mortality | LEE011 350 mg/m2 - Dose Escalation Only | 2 | 15 |
| Total, all-cause mortality | LEE011 470 mg/m2 - Dose Escalation Only | 1 | 12 |
| Total, all-cause mortality | All Patients | 5 | 32 |
| Total, other adverse events | LEE011 280 mg/m2 - Dose Escalation Only | 5 | 5 |
| Total, other adverse events | LEE011 350 mg/m2 - Dose Escalation Only | 15 | 15 |
| Total, other adverse events | LEE011 470 mg/m2 - Dose Escalation Only | 12 | 12 |
| Total, other adverse events | All Patients | 32 | 32 |
| Total, serious adverse events | LEE011 280 mg/m2 - Dose Escalation Only | 3 | 5 |
| Total, serious adverse events | LEE011 350 mg/m2 - Dose Escalation Only | 7 | 15 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
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 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/NCT01747876
GET https://conductscience.org/api/trials/v1/NCT01747876/results?page=2