Real World Data Collection Pediatric Neuroblastoma Treated With Lorlatinib

REAL WORLD DATA COLLECTION AMONG PEDIATRIC NEUROBLASTOMA PATIENTS TREATED WITH LORLATINIB THROUGH EXPANDED ACCESS PROGRAM

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.

Study Overview

• Status: Terminated
• Conditions: Neuroblastoma
• Intervention / Treatment: Drug: lorlatinib

• Study Type: Observational
• Enrollment (Actual): 15

Contacts and Locations

Study Locations

• Australia
  • Camperdown, New South Wales, 2050 Australia, Australia
    • ST0683AU - Chris O'Brien Lifehouse
  • New South Wales
    • Westmead, New South Wales, Australia, 2145
      • Westmead Hospital
• Korea, Republic of
  • Seongnam, Korea, Republic of, 46370
    • Seoul National University Bundang Hospital
  • Seoul, Korea, Republic of, 06351
    • Samsung Medical Center
• New Zealand
  • Auckland, New Zealand, 1142
    • Starship Blood and Cancer Centre
• Portugal
  • Lisboa, Portugal
    • Instituto Português de Oncologia de Lisboa
• Sweden
  • Gothenburg, Sweden, 41650
    • Queen Silvia Children's Hospital
  • Linkoping, Sweden, 58185
    • HRH Crown Princess Victoria's Children and Youth Hospital
  • Stockholm, Sweden, S-171 77
    • Karolinska Institutet
  • Umea, Sweden, 901 85
    • Norrland University Hospital
• United States
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • University of Pennsylvania School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 second and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• 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.

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Other

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Pediatric Neuroblastoma Patients Treated with Lorlatinib	Drug: lorlatinib; Oral

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Objective tumor response of primary tumor (soft tissue), soft tissue metastasis, and bone metastasis	March 2017- December 2023
Bone marrow response	March 2017-December 2023
Health care professional (HCP) Reported Objective Response	March 2017-December 2023
Derived Objective Response	March 2017-December 2023
Best Overall response	March 2017-December 2023
Overall Response Rate (ORR)	March 2017-December 2023
Duration of response	March 2017-December 2023
Progression free survival (PFS)	March 2017-December 2023
Duration of treatment	March 2017-December 2023
Overall survival (OS)	March 2017-December 2023
The number of patients reporting Adverse Events (AE)	March 2017-December 2023
The percentage of patients reporting Adverse Events (AE)	March 2017-December 2023
The number of patients reporting treatment related Adverse Events (AE)	March 2017-December 2023
The percentage of patients reporting treatment related Adverse Events (AE)	March 2017-December 2023
The number of patients reporting Serious Adverse Events (SAE)	March 2017-December 2023
The percentage of patients reporting treatment related Serious Adverse Events (SAE)	March 2017-December 2023

Collaborators and Investigators

• Sponsor: Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): March 19, 2021
• Primary Completion (Actual): September 30, 2022
• Study Completion (Actual): September 30, 2022

Study Registration Dates

• First Submitted: February 8, 2021
• First Submitted That Met QC Criteria: February 11, 2021
• First Posted (Actual): February 15, 2021

Study Record Updates

• Last Update Posted (Actual): March 17, 2023
• Last Update Submitted That Met QC Criteria: March 15, 2023
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neuroectodermal Tumors, Primitive; Neuroectodermal Tumors, Primitive, Peripheral; Neuroblastoma
• Other Study ID Numbers: B7461036

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD 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.