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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04753658




Registration number
NCT04753658
Ethics application status
Date submitted
8/02/2021
Date registered
15/02/2021

Titles & IDs
Public title
Real World Data Collection Pediatric Neuroblastoma Treated With Lorlatinib
Scientific title
REAL WORLD DATA COLLECTION AMONG PEDIATRIC NEUROBLASTOMA PATIENTS TREATED WITH LORLATINIB THROUGH EXPANDED ACCESS PROGRAM
Secondary ID [1] 0 0
B7461036
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Neuroblastoma 0 0
Condition category
Condition code
Cancer 0 0 0 0
Neuroendocrine tumour (NET)
Cancer 0 0 0 0
Children's - Other

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Treatment: Drugs - lorlatinib

Pediatric Neuroblastoma Patients Treated with Lorlatinib -


Treatment: Drugs: lorlatinib
Oral
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Objective tumor response of primary tumor (soft tissue), soft tissue metastasis, and bone metastasis
Timepoint [1] 0 0
March 2017- December 2023
Primary outcome [2] 0 0
Bone marrow response
Timepoint [2] 0 0
March 2017-December 2023
Primary outcome [3] 0 0
Health care professional (HCP) Reported Objective Response
Timepoint [3] 0 0
March 2017-December 2023
Primary outcome [4] 0 0
Derived Objective Response
Timepoint [4] 0 0
March 2017-December 2023
Primary outcome [5] 0 0
Best Overall response
Timepoint [5] 0 0
March 2017-December 2023
Primary outcome [6] 0 0
Overall Response Rate (ORR)
Timepoint [6] 0 0
March 2017-December 2023
Primary outcome [7] 0 0
Duration of response
Timepoint [7] 0 0
March 2017-December 2023
Primary outcome [8] 0 0
Progression free survival (PFS)
Timepoint [8] 0 0
March 2017-December 2023
Primary outcome [9] 0 0
Duration of treatment
Timepoint [9] 0 0
March 2017-December 2023
Primary outcome [10] 0 0
Overall survival (OS)
Timepoint [10] 0 0
March 2017-December 2023
Primary outcome [11] 0 0
The number of patients reporting Adverse Events (AE)
Timepoint [11] 0 0
March 2017-December 2023
Primary outcome [12] 0 0
The percentage of patients reporting Adverse Events (AE)
Timepoint [12] 0 0
March 2017-December 2023
Primary outcome [13] 0 0
The number of patients reporting treatment related Adverse Events (AE)
Timepoint [13] 0 0
March 2017-December 2023
Primary outcome [14] 0 0
The percentage of patients reporting treatment related Adverse Events (AE)
Timepoint [14] 0 0
March 2017-December 2023
Primary outcome [15] 0 0
The number of patients reporting Serious Adverse Events (SAE)
Timepoint [15] 0 0
March 2017-December 2023
Primary outcome [16] 0 0
The percentage of patients reporting treatment related Serious Adverse Events (SAE)
Timepoint [16] 0 0
March 2017-December 2023

Eligibility
Key inclusion criteria
* Patient receives lorlatinib through Pfizer's expanded access program for treatment of ALK+ neuroblastoma.
* HCP documentation of at least one tumor assessment of response after patient has had at least one dose of lorlatinib
* Evidence of a personally signed and dated informed consent document indicating that the patient (or a legally acceptable representative) has been informed of all pertinent aspects of the study.
Minimum age
0 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Any patient who does not meet any of the inclusion criteria defined in the previous section.

Study design
Purpose
Duration
Selection
Timing
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
19/03/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
30/09/2022
Sample size
Target
Accrual to date
Final
15
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Westmead Hospital - Westmead
Recruitment hospital [2] 0 0
ST0683AU - Chris O'Brien Lifehouse - Camperdown, New South Wales, 2050 Australia
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
- Camperdown, New South Wales, 2050 Australia
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Pennsylvania
Country [2] 0 0
Korea, Republic of
State/province [2] 0 0
Seongnam
Country [3] 0 0
Korea, Republic of
State/province [3] 0 0
Seoul
Country [4] 0 0
New Zealand
State/province [4] 0 0
Auckland
Country [5] 0 0
Portugal
State/province [5] 0 0
Lisboa
Country [6] 0 0
Sweden
State/province [6] 0 0
Gothenburg
Country [7] 0 0
Sweden
State/province [7] 0 0
Linkoping
Country [8] 0 0
Sweden
State/province [8] 0 0
Stockholm
Country [9] 0 0
Sweden
State/province [9] 0 0
Umea

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Pfizer
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Pfizer CT.gov Call Center
Address 0 0
Pfizer
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT04753658