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NCT01742286 · OUTCOME

Duration of Response (DoR) Per Investigator Assessment

Phase I Study of LDK378 in Pediatric, Malignancies With a Genetic Alteration in Anaplastic Lymphoma Kinase (ALK) · Source last updated 2020-06-09

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure
MEDIAN
Unit
months
Interval / dispersion
95% Confidence Interval
Time frame
30 months

What was measured

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.

Analysis population: 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.

Groups in this outcome

ALK-activated Neuroblastoma

Participants with ALK-activated neuroblastoma enrolled in the expansion part of the study and were given ceritinib once daily, continuously

ALK-activated Inflammatory Myofibroblastic Tumors (IMT)

Participants with ALK-activated IMT enrolled in the expansion part of the study who were given ceritinib once daily, continuously

ALK-activated Anaplastic Large Cell Lymphoma (ALCL)

Participants with ALK-activated ALCL enrolled in the expansion part of the study who were given ceritinib once daily, continuously

ALK-activated Other

Participants with other ALK-activated tumors enrolled in the expansion part of the study who were given ceritinib once daily, continuously

Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.

Analysis denominator · Participants

GroupSource count
ALK-activated Neuroblastoma6
ALK-activated Inflammatory Myofibroblastic Tumors (IMT)7
ALK-activated Anaplastic Large Cell Lymphoma (ALCL)6
ALK-activated Other1

Reported measurements

Source class 1
Values in months · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
ALK-activated Neuroblastoma15.0Not reported5.822.2Not reported
ALK-activated Inflammatory Myofibroblastic Tumors (IMT)NANot reported3.5NAN/A: Median not reached for subjects with ALCL, IMT \& Other tumor types as most of the subjects did not have a DOR event (progression or death).
ALK-activated Anaplastic Large Cell Lymphoma (ALCL)NANot reported2.8NAN/A: Median not reached for subjects with ALCL, IMT \& Other tumor types as most of the subjects did not have a DOR event (progression or death).
ALK-activated OtherNANot reportedNANAN/A: Median not reached for subjects with ALCL, IMT \& Other tumor types as most of the subjects did not have a DOR event (progression or death).
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        5.8
        upper Limit
        22.2
        value
        15.0
      2. comment
        N/A: Median not reached for subjects with ALCL, IMT \& Other tumor types as most of the subjects did not have a DOR event (progression or death).
        group Id
        OG001
        lower Limit
        3.5
        upper Limit
        NA
        value
        NA
      3. comment
        N/A: Median not reached for subjects with ALCL, IMT \& Other tumor types as most of the subjects did not have a DOR event (progression or death).
        group Id
        OG002
        lower Limit
        2.8
        upper Limit
        NA
        value
        NA
      4. comment
        N/A: Median not reached for subjects with ALCL, IMT \& Other tumor types as most of the subjects did not have a DOR event (progression or death).
        group Id
        OG003
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
denoms
  1. counts
    1. group Id
      OG000
      value
      6
    2. group Id
      OG001
      value
      7
    3. group Id
      OG002
      value
      6
    4. group Id
      OG003
      value
      1
    units
    Participants
description
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.
dispersion Type
95% Confidence Interval
groups
  1. description
    Participants with ALK-activated neuroblastoma enrolled in the expansion part of the study and were given ceritinib once daily, continuously
    id
    OG000
    title
    ALK-activated Neuroblastoma
  2. description
    Participants with ALK-activated IMT enrolled in the expansion part of the study who were given ceritinib once daily, continuously
    id
    OG001
    title
    ALK-activated Inflammatory Myofibroblastic Tumors (IMT)
  3. description
    Participants with ALK-activated ALCL enrolled in the expansion part of the study who were given ceritinib once daily, continuously
    id
    OG002
    title
    ALK-activated Anaplastic Large Cell Lymphoma (ALCL)
  4. description
    Participants with other ALK-activated tumors enrolled in the expansion part of the study who were given ceritinib once daily, continuously
    id
    OG003
    title
    ALK-activated Other
param Type
MEDIAN
population Description
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.
reporting Status
POSTED
time Frame
30 months
title
Duration of Response (DoR) Per Investigator Assessment
type
SECONDARY
unit Of Measure
months

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Preserved source evidence · Not medical advice