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Real World Data Collection Pediatric Neuroblastoma Treated With Lorlatinib

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ClinicalTrials.gov Identifier: NCT04753658
Recruitment Status : Terminated (The study was prematurely discontinued due to operational implementation challenges and insufficient collection of key data due to varied data accessibility across global study sites.)
First Posted : February 15, 2021
Last Update Posted : March 17, 2023
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Description
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Brief Summary:
The overall goal of this real-world data collection is to assess demographic, clinical characteristics and real-world effectiveness of pediatric neuroblastoma patients treated with lorlatinib through the expanded access program.

Condition or disease Intervention/treatment
Neuroblastoma Drug: lorlatinib

Study Design
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Study Type : Observational
Actual Enrollment : 15 participants
Observational Model: Cohort
Time Perspective: Other
Official Title: REAL WORLD DATA COLLECTION AMONG PEDIATRIC NEUROBLASTOMA PATIENTS TREATED WITH LORLATINIB THROUGH EXPANDED ACCESS PROGRAM
Actual Study Start Date : March 19, 2021
Actual Primary Completion Date : September 30, 2022
Actual Study Completion Date : September 30, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Neuroblastoma
Drug Information available for: Lorlatinib

Groups and Cohorts
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Group/Cohort Intervention/treatment
Pediatric Neuroblastoma Patients Treated with Lorlatinib Drug: lorlatinib
Oral


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

Eligibility Criteria
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Ages Eligible for Study:   0 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Up to 50 pediatric patients with ALK-aberrant neuroblastoma being treated with lorlatinib as part of expanded access program will be included.
Criteria

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.

Exclusion Criteria:

  • Any patient who does not meet any of the inclusion criteria defined in the previous section.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04753658


Locations
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United States, Pennsylvania
University of Pennsylvania School of Medicine
Philadelphia, Pennsylvania, United States, 19104
Australia, New South Wales
Westmead Hospital
Westmead, New South Wales, Australia, 2145
Australia
ST0683AU - Chris O'Brien Lifehouse
Camperdown, New South Wales, 2050 Australia, Australia
Korea, Republic of
Seoul National University Bundang Hospital
Seongnam, Korea, Republic of, 46370
Samsung medical Center
Seoul, Korea, Republic of, 06351
New Zealand
Starship Blood and Cancer Centre
Auckland, New Zealand, 1142
Portugal
Instituto Portugues de Oncologia de Lisboa
Lisboa, Portugal
Sweden
Queen Silvia Children's Hospital
Gothenburg, Sweden, 41650
HRH Crown Princess Victoria's Children and Youth Hospital
Linkoping, Sweden, 58185
Karolinska Institutet
Stockholm, Sweden, S-171 77
Norrland University Hospital
Umea, Sweden, 901 85
Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
More Information
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Additional Information:

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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT04753658    
Other Study ID Numbers: B7461036
First Posted: February 15, 2021    Key Record Dates
Last Update Posted: March 17, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: 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.
Additional relevant MeSH terms:
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Neuroblastoma
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
 Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue