Clinical trial NCT04753658: Real World Data Collection Pediatric Neuroblastoma Treated With Lorlatinib
Official title: REAL WORLD DATA COLLECTION AMONG PEDIATRIC NEUROBLASTOMA PATIENTS TREATED WITH LORLATINIB THROUGH EXPANDED ACCESS PROGRAM
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Neuroblastoma
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2021-02-15
Enrollment: 15
Sex: ALL
Minimum age (years): 0
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
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.

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.


Eligibility 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.

Primary outcomes:
measure: Number of Participants According to Tumor Response of Primary Tumor (Soft Tissue)
timeFrame: From start of lorlatinib treatment until CR, PR, SD or PD, up to maximum of 36.2 months of treatment (data was retrieved and evaluated retrospectively during approximately 18 months of this study)
measure: Number of Participants According to Tumor Response of Soft Tissue Metastasis and Bone Metastasis
timeFrame: From start of lorlatinib treatment until CR, PR, SD or PD, up to maximum of 36.2 months of treatment (data was retrieved and evaluated retrospectively during approximately 18 months of this study)
measure: Number of Participants According to Bone Marrow Response
timeFrame: From start of lorlatinib treatment until CR, PR, MD or SD, up to maximum of 36.2 months of treatment (data was retrieved and evaluated retrospectively during approximately 18 months of this study)
measure: Number of Participants According to Health Care Professional (HCP) Reported Objective Response
timeFrame: From start of lorlatinib treatment until CR, PR, MR, SD or PD, up to maximum of 36.2 months of treatment (data was retrieved and evaluated retrospectively during approximately 18 months of this study)
measure: Number of Participants According to Derived Objective Response
timeFrame: From start of lorlatinib treatment until CR, PR, MR, SD or PD, up to maximum of 36.2 months of treatment (data was retrieved and evaluated retrospectively during approximately 18 months of this study)
measure: Number of Participants With Best Overall Response Based on HCP Reported Objective Response
timeFrame: From start of lorlatinib treatment until CR, PR, MR, SD or PD, Up to maximum of 36.2 months of treatment (data was retrieved and evaluated retrospectively during approximately 18 months of this study)
measure: Number of Participants With Best Overall Response Based on Derived Objective Response
timeFrame: From start of lorlatinib treatment until CR, PR, MR, SD or PD, up to maximum of 36.2 months of treatment (data was retrieved and evaluated retrospectively during approximately 18 months of this study)
measure: Overall Response Rate Based on HCP Reported Response
timeFrame: From start of lorlatinib treatment until CR or PR, up to maximum of 36.2 months of treatment (data was retrieved and evaluated retrospectively during approximately 18 months of this study)
measure: Overall Response Rate Based on Derived Response
timeFrame: From start of lorlatinib treatment until CR or PR, up to maximum of 36.2 months of treatment (data was retrieved and evaluated retrospectively during approximately 18 months of this study)
measure: Duration of HCP Reported Overall Responses
timeFrame: From date of CR or PR until earliest date of PD or death or censoring date, up to maximum of 36.2 months of treatment (data was retrieved and evaluated retrospectively during approximately 18 months of this study)
measure: Progression Free Survival (PFS)
timeFrame: From start of lorlatinib treatment until disease progression or death or censoring date, up to maximum of 36.2 months of treatment (data was retrieved and evaluated retrospectively during approximately 18 months of this study)
measure: Duration of Treatment
timeFrame: From start of lorlatinib treatment until treatment stop date or censoring date, up to maximum of 36.2 months of treatment (data was retrieved and evaluated retrospectively during approximately 18 months of this study)
measure: Overall Survival
timeFrame: From date of first dose of lorlatinib until date of death or censoring date, up to maximum (max) of 36.2 months (M) of treatment (data was retrieved and evaluated retrospectively during approximately (approx.) 18 months of this study)
measure: Number of Participants Reporting Adverse Events, Treatment-Related Adverse Events, Serious Adverse Events and Treatment-Related Serious-Adverse Events
timeFrame: From start of first dose of lorlatinib/date of informed consent(participant treated with lorlatinib) to atleast 28 day after last dose of lorlatinib to max 36.2M of treatment(data was retrieved, evaluated for approx. 18 month)
Secondary outcomes:
Other outcomes:
Locations:
city: Philadelphia
country: United States
facility: University of Pennsylvania School of Medicine
state: Pennsylvania
city: Westmead
country: Australia
facility: Westmead Hospital
state: New South Wales
city: Camperdown, New South Wales, 2050 Australia
country: Australia
facility: ST0683AU - Chris O'Brien Lifehouse
city: Seongnam
country: Korea, Republic of
facility: Seoul National University Bundang Hospital
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Auckland
country: New Zealand
facility: Starship Blood and Cancer Centre
city: Lisboa
country: Portugal
facility: Instituto Portugues de Oncologia de Lisboa
city: Gothenburg
country: Sweden
facility: Queen Silvia Children's Hospital
city: Linkoping
country: Sweden
facility: HRH Crown Princess Victoria's Children and Youth Hospital
city: Stockholm
country: Sweden
facility: Karolinska Institutet
city: Umea
country: Sweden
facility: Norrland University Hospital
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: