Trial · NCT01742286
Phase I Study of LDK378 in Pediatric, Malignancies With a Genetic Alteration in Anaplastic Lymphoma Kinase (ALK)
NCT01742286, Phase 1, Completed, Novartis Pharmaceuticals; results first posted 2020-06-09
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase I Study of LDK378 in Pediatric, Malignancies With a Genetic Alteration in Anaplastic Lymphoma Kinase (ALK). Trials, record NCT01742286. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01742286
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase I Study of LDK378 in Pediatric, Malignancies With a Genetic Alteration in Anaplastic Lymphoma Kinase (ALK)
- NCT number
- NCT01742286
- Status
- Completed
- Phases
- Phase 1
- Enrollment
- 83 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- ALK-activated Tumors
- Interventions
- Ceritinib
- First posted
- 2012-12-05
- Last updated
- 2020-06-09
- Other registries' IDs
- eudract:2012-002074-31
- Results first posted
- 2020-06-09
Source ClinicalTrials.gov
Registered outcomes16
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) Occurring During First Cycle of Treatment | up to day 21 after the patient's first dose; cycle = within the first 21 days of patient's first dose | A DLT is defined as an adverse event or abnormal laboratory value assessed as unrelated to disease, disease progression, inter-current illness, or concomitant therapies that occurs within the first 21 days of treatment with LDK378 and meets a specified defined criteria. A participant with multiple occurrences of DLTs under one treatment is counted only once in the Adverse Event category for that treatment. A participant with multiple DLTs within a primary system organ class is counted only once in the total row. |
| Secondary | Summary of Best Overall Response by Overall Response Rate (ORR) Per Investigator Assessment | 30 months | ORR is the percentage of participants with a best overall response of complete response (CR) or partial response (PR). ORR was assessed per Investigator as per RECIST 1.1 in participants with neuroblastoma and other solid tumors, and by International Working Group (IWG) criteria in patients with lymphoma. Per RECIST 1.1 (for neuroblastoma \& other solid tumors): CR: disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to \< 10 mm. PR: at least a 30% decrease in the sum of diameter of all target lesion, taking as reference the baseline sum diameters. Per IWG criteria (for patients with lymphoma): CR: normalization of all index nodal lesions or complete disappearance of all index extranodal lesions. PR: at least 50% decrease from baseline in the sum of diameters of all index lesions. |
| Secondary | Duration of Response (DoR) Per Investigator Assessment | 30 months | DOR is defined as the time from first documented response (PR or CR) to the date of first documented disease progression (PD) or death due to any cause. DOR was assessed per Investigator as per RECIST 1.1 in participants with neuroblastoma and other solid tumors, and by International Working Group (IWG) criteria in patients with lymphoma. Per RECIST 1.1 (for neuroblastoma \& other solid tumors): CR: disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to \< 10 mm. PR: at least a 30% decrease in the sum of diameter of all target lesion, taking as reference the baseline sum diameters. Per IWG criteria (for patients with lymphoma): CR: normalization of all index nodal lesions or complete disappearance of all index extranodal lesions. PR: at least 50% decrease from baseline in the sum of diameters of all index lesions. |
| Secondary | Progression Free Survival (PFS) Based on Investigator Assessment | 30 months | PFS 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 any cause. PFS was assessed per Investigator as per RECIST 1.1 in participants with neuroblastoma and other solid tumors, and by International Working Group (IWG) criteria in patients with lymphoma. Per RECIST 1.1 (for neuroblastoma \& other solid tumors): CR: disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to \< 10 mm. PR: at least a 30% decrease in the sum of diameter of all target lesion, taking as reference the baseline sum diameters. Per IWG criteria (for patients with lymphoma): CR: normalization of all index nodal lesions or complete disappearance of all index extranodal lesions. PR: at least 50% decrease from baseline in the sum of diameters of all index lesions. |
| Secondary | Plasma Concentration Time Profiles by Treatment Group in Escalation Phase | 0hr pre-dose, 2hrs post-dose, 4hrs post-dose, 6hrs post-dose & 24hrs post-dose in Cycle1 Day1 & Cycle 2 day 1; 0hr pre-dose in Cycle 1 Day 15, Cycle 2 Day1, Cycle 2 Day 2, Cycle 3 day 1 & Cycle 4 Day 1 | Characterize single and multiple-dose PK of LDK378 in pediatric patients. Only PK plasma concentrations with non-missing sampling date and time, and for which the last dose date and time prior to the PK sample draw are non-missing, were included in the PK analysis. |
| Secondary | Plasma Concentration Time Profiles by Treatment Group in Expansion Phase | 0hr pre-dose Cycle 1 Day 1, cycle 1 Day 15; 0hr pre-dose, 2hrs post-dose, 4hrs post-dose, 6hrs post-dose & 24hrs post-dose in Cycle2 Day1; 0hr pre-dose in Cycle2 Day2, Cycle 3 Day 1 & Cycle 4 Day 1 | Characterize single and multiple-dose PK of LDK378 in pediatric patients. Only PK plasma concentrations with non-missing sampling date and time, and for which the last dose date and time prior to the PK sample draw are non-missing, were included in the PK analysis. |
| Secondary | Pharmacokinetics (PK) Parameters: AUC0 - 24h & AUClast in Cycle 1 Day 1 - Dose Escalation Phase (Single Dose) | 0hr pre-dose, 2, 4, 6 & 24hrs post-dose | Characterize single and multiple-dose PK of LDK378 in pediatric patients. AUC: Area under the plasma (serum, or blood) concentration versus time curve AUClast: Area under the concentration-time curve from time zero to the last measureable concentration time AUC0-24h: Area under the plasma concentration-time curve t=0-24 h |
| Secondary | Pharmacokinetics (PK) Parameters: AUC0 - 24h & AUClast in Cycle 2 Day 1 - Dose Escalation Phase (Single Dose) | 0hr pre-dose, 2, 4, 6 & 24hrs post-dose | Characterize single and multiple-dose PK of LDK378 in pediatric patients. AUC: Area under the plasma (serum, or blood) concentration versus time curve AUClast: Area under the concentration-time curve from time zero to the last measureable concentration time; AUC0-24h: Area under the plasma concentration-time curve t=0-24 h |
| Secondary | Pharmacokinetics (PK) Parameters: AUC0 - 24h & AUClast in Cycle 2 Day 1 - Dose Expansion Phase (Multiple Dose) | 0hr pre-dose, 2, 4, 6 & 24hrs post-dose | Characterize single and multiple-dose PK of LDK378 in pediatric patients. In this phase ceritinib was expanded at 500mg/m2 fed and 510mg/m2 fasted administered orally once daily and was assessed only at steady state, Cycle 2 Day 1. AUC: Area under the plasma (serum, or blood) concentration versus time curve AUClast: Area under the plasma (serum, or blood) concentration versus time curverea under the concentration-time curve from time zero to the last measureable concentration time AUC0-24h: Area under the plasma concentration-time curve t=0-24 h |
| Secondary | PK Parameter: Cmax in Cycle 1 Day 1 - Dose Escalation Phase (Single Dose) | 0hr pre-dose, 2, 4, 6 & 24hrs post-dose | Characterize single and multiple-dose PK of LDK378 in pediatric patients. Cmax: Maximum (peak) concentration of drug |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 registered outcomesOutcomes16
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) Occurring During First Cycle of Treatment | up to day 21 after the patient's first dose; cycle = within the first 21 days of patient's first dose | The dose determining analysis set (DDS) consisted of all patients from the Safety set who either met the minimum exposure criterion and had sufficient safety evaluations or discontinued earlier due to DLT in the escalation phase. | Participants | NUMBER |
| SECONDARY | Summary of Best Overall Response by Overall Response Rate (ORR) Per Investigator Assessment | 30 months | Full Analysis Set (FAS)/Maximum Tolerated Dose MTD)/Recommended dose for expansion (RDE): Results are presented only for patients who received at least 1 dose of ceritinib in either of the 2 MTD/RDE groups and were based on combining data from across the dose-escalation \& dose-expansion phases, \& summarized according to primary diagnosis of tumor. | percentage of participants | NUMBER |
| SECONDARY | Duration of Response (DoR) Per Investigator Assessment | 30 months | Full Analysis Set/MTD/RDE patients with confirmed CR or PR): Efficacy results are presented only for patients with confirmed CR or PR who received at least 1 dose of ceritinib in either of the 2 MTD/RDE groups \& were based on combining data from across the dose-escalation \& dose-expansion phases, \& summarized according to primary tumor diagnosis. | months | MEDIAN |
| SECONDARY | Progression Free Survival (PFS) Based on Investigator Assessment | 30 months | Full Analysis Set (FAS)/MTD/RDE: Efficacy results are presented only for patients who received at least 1 dose of ceritinib in either of the two MTD/RDE groups and were based on combining data from across the dose-escalation \& dose-expansion phases, \& summarized according to primary diagnosis of tumor. | months | MEDIAN |
| SECONDARY | Plasma Concentration Time Profiles by Treatment Group in Escalation Phase | 0hr pre-dose, 2hrs post-dose, 4hrs post-dose, 6hrs post-dose & 24hrs post-dose in Cycle1 Day1 & Cycle 2 day 1; 0hr pre-dose in Cycle 1 Day 15, Cycle 2 Day1, Cycle 2 Day 2, Cycle 3 day 1 & Cycle 4 Day 1 | The PK analysis set (PAS) consisted of all patients who received at least one dose of ceritinib and had at least one evaluable PK sample. | ng/mL | GEOMETRIC_MEAN |
| SECONDARY | Plasma Concentration Time Profiles by Treatment Group in Expansion Phase | 0hr pre-dose Cycle 1 Day 1, cycle 1 Day 15; 0hr pre-dose, 2hrs post-dose, 4hrs post-dose, 6hrs post-dose & 24hrs post-dose in Cycle2 Day1; 0hr pre-dose in Cycle2 Day2, Cycle 3 Day 1 & Cycle 4 Day 1 | The PK analysis set (PAS) consisted of all patients who received at least one dose of ceritinib and had at least one evaluable PK sample. | ng/mL | GEOMETRIC_MEAN |
| SECONDARY | Pharmacokinetics (PK) Parameters: AUC0 - 24h & AUClast in Cycle 1 Day 1 - Dose Escalation Phase (Single Dose) | 0hr pre-dose, 2, 4, 6 & 24hrs post-dose | The PK analysis set (PAS) consisted of all patients who received at least one dose of ceritinib and had at least one evaluable PK sample. | hr*ng/mL | GEOMETRIC_MEAN |
| SECONDARY | Pharmacokinetics (PK) Parameters: AUC0 - 24h & AUClast in Cycle 2 Day 1 - Dose Escalation Phase (Single Dose) | 0hr pre-dose, 2, 4, 6 & 24hrs post-dose | The PK analysis set (PAS) consisted of all patients who received at least one dose of ceritinib and had at least one evaluable PK sample. | hr*ng/mL | GEOMETRIC_MEAN |
| SECONDARY | Pharmacokinetics (PK) Parameters: AUC0 - 24h & AUClast in Cycle 2 Day 1 - Dose Expansion Phase (Multiple Dose) | 0hr pre-dose, 2, 4, 6 & 24hrs post-dose | The PK analysis set (PAS), MTD/RDE consisted of all patients who received at least one dose of ceritinib and had at least one evaluable PK sample. | hr*ng/mL | GEOMETRIC_MEAN |
| SECONDARY | PK Parameter: Cmax in Cycle 1 Day 1 - Dose Escalation Phase (Single Dose) | 0hr pre-dose, 2, 4, 6 & 24hrs post-dose | The PK analysis set (PAS) consisted of all patients who received at least one dose of ceritinib and had at least one evaluable PK sample. | ng/mL | GEOMETRIC_MEAN |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 outcomesResults by group233
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) Occurring During First Cycle of Treatment | Fasted: Ceritinib 300 mg/m2 | Investigations: Alanine aminotransferase incr. | 0 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) Occurring During First Cycle of Treatment | Fasted: Ceritinib 450 mg/m2 | Investigations: Alanine aminotransferase incr. | 0 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) Occurring During First Cycle of Treatment | Fasted: Ceritinib 510 mg/m2 | Investigations: Alanine aminotransferase incr. | 0 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) Occurring During First Cycle of Treatment | Fasted: Ceritinib 560 mg/m2 | Investigations: Alanine aminotransferase incr. | 1 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) Occurring During First Cycle of Treatment | Fed: Ceritinib 320 mg/m2 | Investigations: Alanine aminotransferase incr. | 0 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) Occurring During First Cycle of Treatment | Fed: Ceritinib 400 mg/m2 | Investigations: Alanine aminotransferase incr. | 1 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) Occurring During First Cycle of Treatment | Fed: Ceritinib 500 mg/m2 | Investigations: Alanine aminotransferase incr. | 0 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) Occurring During First Cycle of Treatment | Fasted: Ceritinib 300 mg/m2 | Gastrointestinal disorders: abdominal pain | 0 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) Occurring During First Cycle of Treatment | Fasted: Ceritinib 450 mg/m2 | Gastrointestinal disorders: abdominal pain | 0 | Participants | |
| Incidence Rate of Dose Limiting Toxicities (DLTs) Occurring During First Cycle of Treatment | Fasted: Ceritinib 510 mg/m2 | Gastrointestinal disorders: abdominal pain | 0 | Participants |
1 to 10 of 233Source ClinicalTrials.gov
See all 233 results by groupParticipant flow56
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Escalation Phase | STARTED | Fasted: Ceritinib 300 mg/m2 | 5 | |
| Escalation Phase | STARTED | Fasted: Ceritinib 450 mg/m2 | 12 | |
| Escalation Phase | STARTED | Fasted: Ceritinib 510 mg/m2 | 6 | |
| Escalation Phase | STARTED | Fasted: Ceritinib 560 mg/m2 | 2 | |
| Escalation Phase | STARTED | Fed: Ceritinib 320 mg/m2 | 4 | |
| Escalation Phase | STARTED | Fed: Ceritinib 400 mg/m2 | 5 | |
| Escalation Phase | STARTED | Fed: Ceritinib 500 mg/m2 | 6 | |
| Escalation Phase | Ended Treatment = Not Completed) | Fasted: Ceritinib 300 mg/m2 | 5 | |
| Escalation Phase | Ended Treatment = Not Completed) | Fasted: Ceritinib 450 mg/m2 | 12 | |
| Escalation Phase | Ended Treatment = Not Completed) | Fasted: Ceritinib 510 mg/m2 | 6 |
1 to 10 of 56Source ClinicalTrials.gov
See all 56 participant flowAdverse events1,976
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 | Fasted Ceritinib 300mg/m2 | 1 | 5 | |
| other | Anaemia | Blood and lymphatic system disorders | Fasted Ceritinib 450mg/m2 | 3 | 12 | |
| other | Anaemia | Blood and lymphatic system disorders | Fasted Ceritinib 510mg/m2 | 3 | 13 | |
| other | Anaemia | Blood and lymphatic system disorders | Fasted Ceritinib 560mg/m2 | 1 | 2 | |
| other | Anaemia | Blood and lymphatic system disorders | Fed Ceritinib 320mg/m2 | 3 | 4 | |
| other | Anaemia | Blood and lymphatic system disorders | Fed Ceritinib 400mg/m2 | 0 | 5 | |
| other | Anaemia | Blood and lymphatic system disorders | Fed Ceritinib 500mg/m2 | 11 | 42 | |
| other | Anaemia | Blood and lymphatic system disorders | Fasted+Fed All Patients | 22 | 83 | |
| other | Bone marrow disorder | Blood and lymphatic system disorders | Fasted Ceritinib 300mg/m2 | 0 | 5 | |
| other | Bone marrow disorder | Blood and lymphatic system disorders | Fasted Ceritinib 450mg/m2 | 0 | 12 |
1 to 10 of 1,976Source ClinicalTrials.gov
See all 1,976 adverse eventsAdverse event totals24
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Fasted Ceritinib 300mg/m2 | 1 | 5 |
| Total, all-cause mortality | Fasted Ceritinib 450mg/m2 | 1 | 12 |
| Total, all-cause mortality | Fasted Ceritinib 510mg/m2 | 2 | 13 |
| Total, all-cause mortality | Fasted Ceritinib 560mg/m2 | 0 | 2 |
| Total, all-cause mortality | Fed Ceritinib 320mg/m2 | 2 | 4 |
| Total, all-cause mortality | Fed Ceritinib 400mg/m2 | 1 | 5 |
| Total, all-cause mortality | Fed Ceritinib 500mg/m2 | 5 | 42 |
| Total, all-cause mortality | Fasted+Fed All Patients | 12 | 83 |
| Total, other adverse events | Fasted Ceritinib 300mg/m2 | 5 | 5 |
| Total, other adverse events | Fasted Ceritinib 450mg/m2 | 12 | 12 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 adverse event totalsPapers2
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 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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