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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.
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Pediatric Neuroblastoma Patients Treated with Lorlatinib |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| lorlatinib | Drug | Oral |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants According to Tumor Response of Primary Tumor (Soft Tissue) | Tumor response was collected by the data collection tool (DCT) and responses included complete response (CR), partial response (PR), stable disease (SD) and progressive disease (PD). As per International Neuroblastoma Response Criteria (INRC): CR=<10 millimeter (mm) residual soft tissue at primary site and complete resolution of metaiodobenzylguanidine (MIBG) or [18F] fluorodeoxyglucose (FDG)/positron emission tomography (PET) uptake (for MIBG-nonavid tumors) at primary site; PR: >=30% decrease in longest diameter (LD) of primary site & MIBG or FDG-PET uptake at primary site stable, improved, or resolved; SD: neither sufficient shrinkage for PR nor sufficient increase for PD at the primary site. PD greater than 20% increase in longest diameter taking as reference the smallest sum on study (this included the baseline sum if that was the smallest on study) and minimum absolute increase of 5 mm in longest dimension. One participant may have more than one tumor response. | 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) |
| Number of Participants According to Tumor Response of Soft Tissue Metastasis and Bone Metastasis | Tumor response collected by DCT.Response included CR=resolution of all sites of disease; PR:>=30% decrease in sum of diameter of non-primary (NP) target lesion (TL) compared to baseline, non-target (NT) lesion may be stable/small in size, no new lesion,>=50% reduced MIBG absolute(abs) bone score (BS) (relative MIBG BS>=0.1 to <=0.5)/>=50% reduced number of FDG-PET avid bone lesion; PD:new soft tissue lesion (STL) detected by computed tomography (CT)/magnetic resonance imaging (MRI) that's MIBG avid/FDG-PET avid, new STL on anatomic imaging, biopsied, confirmed as neuroblastoma(NB)/ganglioneuroblastoma(GNB),new bone site:MIBG avid/FDG-PET avid (for MIBG non-avid tumor) with CT/MRI finding consistent with tumor/confirmed histologically as NB/GNB; >20% increase in LD as reference smallest sum on study,minimum abs. increase of 5mm in sum of diameter of target STL; SD:No sufficient (suff) shrinkage for PR/suff increase for PD of NP lesion.One participant may have more than 1 tumor response | 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) |
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Inclusion Criteria:
Exclusion Criteria:
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Up to 50 pediatric patients with ALK-aberrant neuroblastoma being treated with lorlatinib as part of expanded access program will be included.
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Pennsylvania School of Medicine | Philadelphia | Pennsylvania | 19104 | United States | ||
| Westmead Hospital |
Not provided
| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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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.
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Participants with anaplastic lymphoma kinase (ALK)-aberrant neuroblastoma who initiated treatment with lorlatinib as part of expanded access program were included in this non-interventional study. Data was collected using the data collection tool (DCT).
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| ID | Title | Description |
|---|---|---|
| FG000 | Lorlatinib | Participants with ALK-aberrant neuroblastoma who initiated treatment with lorlatinib as part of an expanded access program were included. Data was collected on a continual basis until discontinuation of treatment with lorlatinib, death, loss to follow-up or end of study, whichever occurred first. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
All participants for whom data was collected using the data collection tool and observed in this study were included in the analysis.
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| ID | Title | Description |
|---|---|---|
| BG000 | Lorlatinib | Participants with ALK-aberrant neuroblastoma who initiated treatment with lorlatinib as part of an expanded access program were included. Data was collected on a continual basis until discontinuation of treatment with lorlatinib, death, loss to follow-up or end of study, whichever occurred first. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants According to Tumor Response of Primary Tumor (Soft Tissue) | Tumor response was collected by the data collection tool (DCT) and responses included complete response (CR), partial response (PR), stable disease (SD) and progressive disease (PD). As per International Neuroblastoma Response Criteria (INRC): CR=<10 millimeter (mm) residual soft tissue at primary site and complete resolution of metaiodobenzylguanidine (MIBG) or [18F] fluorodeoxyglucose (FDG)/positron emission tomography (PET) uptake (for MIBG-nonavid tumors) at primary site; PR: >=30% decrease in longest diameter (LD) of primary site & MIBG or FDG-PET uptake at primary site stable, improved, or resolved; SD: neither sufficient shrinkage for PR nor sufficient increase for PD at the primary site. PD greater than 20% increase in longest diameter taking as reference the smallest sum on study (this included the baseline sum if that was the smallest on study) and minimum absolute increase of 5 mm in longest dimension. One participant may have more than one tumor response. | All participants for whom data was collected using the data collection tool and observed in this study were included in the analysis. | Posted | Count of Participants | Participants | 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) |
From start of first dose of lorlatinib or date of informed consent (for participants being treated with lorlatinib at study start) until at least 28 days following last administration of lorlatinib (maximum treatment duration of 36.2 months), data was retrieved and evaluated retrospectively during approx.18 months of this study
An AE term may be reported as both a serious and non-serious AE but are distinct events. An AE may be serious for 1 participant and non-serious for another participant, or a participant may have experienced both a serious and non-serious episode of the same event.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Lorlatinib | Participants with ALK-aberrant neuroblastoma who initiated treatment with lorlatinib as part of an expanded access program were included. Data was collected on a continual basis until discontinuation of treatment with lorlatinib, death, loss to follow-up or end of study, whichever occurred first. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hemiparesis | Nervous system disorders | MedDRA v25.0 | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Blood cholesterol increased | Investigations | MedDRA v25.0 | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Feb 23, 2021 | Aug 22, 2023 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D009447 | Neuroblastoma |
| ID | Term |
|---|---|
| D018241 | Neuroectodermal Tumors, Primitive, Peripheral |
| D018242 | Neuroectodermal Tumors, Primitive |
| D018302 | Neoplasms, Neuroepithelial |
| D017599 | Neuroectodermal Tumors |
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| ID | Term |
|---|---|
| C000590786 | lorlatinib |
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| Number of Participants According to Bone Marrow Response | Bone marrow response was collected by the DCT and responses included CR, PR, minimal disease (MD) and SD. As per INRC, CR= bone marrow with no tumor infiltration upon reassessment, independent of baseline tumor involvement; PD= bone marrow without tumor infiltration that became > 5% tumor infiltration upon reassessment; or bone marrow with tumor infiltration that increased by > 2 fold and had > 20% tumor infiltration upon reassessment; MD= Bone marrow with less than or equal to (<=) 5% tumor infiltration and remained > 0 to <= 5% tumor infiltration upon reassessment; or bone marrow with no tumor infiltration that became <= 5% tumor infiltration upon reassessment; or bone marrow with >20% tumor infiltration that had > 0 to <= 5% tumor infiltration upon reassessment and SD= bone marrow with tumor infiltration that remained positive with > 5% tumor infiltration upon reassessment but did not meet CR, MD or PD criteria. | 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) |
| Number of Participants According to Health Care Professional (HCP) Reported Objective Response | Objective response was collected by the DCT and comprised of responses in 3 components based on INRC: primary tumor, soft tissue and bone metastases and bone marrow, CR=All components met criteria for CR; PR=PR in at least one component and all other components are either CR, MD (bone marrow), PR (soft tissue or bone), or not involved (NI); no component with PD; Minor response (MR) = PR or CR in at least one component but at least one other component; no component with PD; SD= SD in one component with no better than SD or NI in any other component; PD=Any component with PD. One participant may have more than one tumor response. | 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) |
| Number of Participants According to Derived Objective Response | Derived objective response was derived using rules based on responses in 3 components: primary tumor, soft tissue and bone metastases, and bone marrow. CR=All components met criteria for CR. PR=PR in at least one component and all other components are either CR, MD (in bone marrow), PR (soft tissue or bone) or not involved (NI; no component with PD). MR = PR or CR in at least one component but at least one other component with stable disease; no component with PD. SD = Stable disease in one component with no better than SD or NI in any other component; no component with PD. PD = Any component with PD. | 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) |
| Number of Participants With Best Overall Response Based on HCP Reported Objective Response | Best overall response based on HCP reported objective response comprised of responses (CR, PR, MR, SD and PD) in 3 components: primary tumor, soft tissue and bone metastases, and bone marrow. CR=All components met criteria for CR. PR=At least one component and all other components are either CR, MD (in bone marrow), PR (soft tissue or bone) or not involved (NI; no component with PD). MR = PR or CR in at least one component but at least one other component with stable disease; no component with PD. SD = It is in one component with no better than SD or NI in any other component PD = Any component with PD. Best overall response taking the HCP reported response observed up to the data cut-off date for a specific milestone. | 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) |
| Number of Participants With Best Overall Response Based on Derived Objective Response | Best overall response based on derived objective response comprised of responses (CR, PR, MR, SD and PD) in 3 components: primary tumor, soft tissue and bone metastases, and bone marrow. CR=All components met criteria for CR. PR=PR in at least one component and all other components are either CR, MD (in bone marrow), PR (soft tissue or bone) or not involved (NI; no component with PD). MR= PR or CR in at least one component but at least one other component with stable disease; no component with PD. SD= Stable disease in one component with no better than SD or NI in any other component; no component with PD. PD= Any component with PD. | 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) |
| Overall Response Rate Based on HCP Reported Response | Overall response rate was defined as the percentage of participants with a best overall response of CR or PR. CR=All components (primary tumor, soft tissue and bone metastases, and bone marrow) met criteria for CR. PR=PR in at least one component and all other components are either CR, MD (in bone marrow), PR (soft tissue or bone) or not involved (NI; no component with PD). Two sided 95% confidence interval was based on Clopper Pearson method. | 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) |
| Overall Response Rate Based on Derived Response | Overall response rate was defined as the percentage of participants with a best overall response of CR or PR. CR=All components (primary tumor, soft tissue and bone metastases, and bone marrow) met criteria for CR. PR=PR in at least one component and all other components are either CR, MD (in bone marrow), PR (soft tissue or bone) or not involved (NI; no component with PD). | 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) |
| Duration of HCP Reported Overall Responses | Duration of HCP reported overall response was derived as (earliest date of disease progression or death minus earliest date of complete or partial response + 1)/30.4. Participants who had not progressed or died were censored at their last assessment date prior to the data cut-off date. CR=All components (primary tumor, soft tissue and bone metastases, and bone marrow) met criteria for CR. PR=PR in at least one component and all other components are either CR, MD (in bone marrow), PR (soft tissue or bone) or not involved. PD=Any component with PD. | 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) |
| Progression Free Survival (PFS) | PFS was derived as (date of PD or death [by any cause in the absence of PD] minus date of first dose of lorlatinib plus 1) divided by 30.4. Participants who had not progressed or died were censored at the date of last contact prior to the data cut-off date. PD: new soft tissue lesion (STL) detected by CT/MRI that's MIBG avid/FDG-PET avid, new STL on anatomic imaging, biopsied and confirmed as neuroblastoma (NB)/ganglioneuroblastoma (GNB), new bone site: MIBG avid/FDG-PET avid (for MIBG non-avid tumor) with CT/MRI finding consistent with tumor or confirmed histologically as NB/GNB; >20% increase in LD as reference smallest sum on study (included baseline sum if that was the smallest on study), minimum abs increase of 5mm in sum of diameter of target STL. | 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) |
| Duration of Treatment | Duration of treatment was derived as treatment stop date minus treatment start date plus 1. Participants continuing treatment at the data cut-off date were censored based on the last recorded date when the participant was known to be continuing treatment prior to the data cut-off date. | 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) |
| Overall Survival | Overall survival was derived as date of death minus treatment start date plus 1. Participants who had not died were censored at the date of last contact prior to the data cut-off date. | 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) |
| Number of Participants Reporting Adverse Events, Treatment-Related Adverse Events, Serious Adverse Events and Treatment-Related Serious-Adverse Events | An AE is any untoward medical occurrence in participants administered a medicinal product. The event need not necessarily have a causal relationship with the product treatment or usage. A SAE is any untoward medical occurrence in a participant administered a medicinal or nutritional product at any dose that: resulted in death, was life-threatening, required participant hospitalization or prolongation of hospitalization, resulted in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions) and resulted in congenital anomaly/birth defect. Relatedness to treatment was determined by investigator. | 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) |
| Westmead |
| New South Wales |
| 2145 |
| Australia |
| ST0683AU - Chris O'Brien Lifehouse | Camperdown, New South Wales, 2050 Australia | Australia |
| Starship Blood and Cancer Centre | Auckland | 1142 | New Zealand |
| Instituto Portugues de Oncologia de Lisboa | Lisbon | Portugal |
| Seoul National University Bundang Hospital | Seongnam | 46370 | South Korea |
| Samsung Medical Center | Seoul | 06351 | South Korea |
| Queen Silvia Children's Hospital | Gothenburg | 41650 | Sweden |
| HRH Crown Princess Victoria's Children and Youth Hospital | Linköping | 58185 | Sweden |
| Karolinska Institutet | Stockholm | S-171 77 | Sweden |
| Norrland University Hospital | Umeå | 901 85 | Sweden |
| Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
|
|
|
| Primary | Number of Participants According to Tumor Response of Soft Tissue Metastasis and Bone Metastasis | Tumor response collected by DCT.Response included CR=resolution of all sites of disease; PR:>=30% decrease in sum of diameter of non-primary (NP) target lesion (TL) compared to baseline, non-target (NT) lesion may be stable/small in size, no new lesion,>=50% reduced MIBG absolute(abs) bone score (BS) (relative MIBG BS>=0.1 to <=0.5)/>=50% reduced number of FDG-PET avid bone lesion; PD:new soft tissue lesion (STL) detected by computed tomography (CT)/magnetic resonance imaging (MRI) that's MIBG avid/FDG-PET avid, new STL on anatomic imaging, biopsied, confirmed as neuroblastoma(NB)/ganglioneuroblastoma(GNB),new bone site:MIBG avid/FDG-PET avid (for MIBG non-avid tumor) with CT/MRI finding consistent with tumor/confirmed histologically as NB/GNB; >20% increase in LD as reference smallest sum on study,minimum abs. increase of 5mm in sum of diameter of target STL; SD:No sufficient (suff) shrinkage for PR/suff increase for PD of NP lesion.One participant may have more than 1 tumor response | All participants for whom data was collected using the data collection tool and observed in this study were included in the analysis. | Posted | Count of Participants | Participants | 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) |
|
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|
| Primary | Number of Participants According to Bone Marrow Response | Bone marrow response was collected by the DCT and responses included CR, PR, minimal disease (MD) and SD. As per INRC, CR= bone marrow with no tumor infiltration upon reassessment, independent of baseline tumor involvement; PD= bone marrow without tumor infiltration that became > 5% tumor infiltration upon reassessment; or bone marrow with tumor infiltration that increased by > 2 fold and had > 20% tumor infiltration upon reassessment; MD= Bone marrow with less than or equal to (<=) 5% tumor infiltration and remained > 0 to <= 5% tumor infiltration upon reassessment; or bone marrow with no tumor infiltration that became <= 5% tumor infiltration upon reassessment; or bone marrow with >20% tumor infiltration that had > 0 to <= 5% tumor infiltration upon reassessment and SD= bone marrow with tumor infiltration that remained positive with > 5% tumor infiltration upon reassessment but did not meet CR, MD or PD criteria. | All participants for whom data was collected using the data collection tool and observed in this study were included in the analysis. | Posted | Count of Participants | Participants | 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) |
|
|
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| Primary | Number of Participants According to Health Care Professional (HCP) Reported Objective Response | Objective response was collected by the DCT and comprised of responses in 3 components based on INRC: primary tumor, soft tissue and bone metastases and bone marrow, CR=All components met criteria for CR; PR=PR in at least one component and all other components are either CR, MD (bone marrow), PR (soft tissue or bone), or not involved (NI); no component with PD; Minor response (MR) = PR or CR in at least one component but at least one other component; no component with PD; SD= SD in one component with no better than SD or NI in any other component; PD=Any component with PD. One participant may have more than one tumor response. | All participants for whom data was collected using the data collection tool and observed in this study were included in the analysis. | Posted | Count of Participants | Participants | 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) |
|
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| Primary | Number of Participants According to Derived Objective Response | Derived objective response was derived using rules based on responses in 3 components: primary tumor, soft tissue and bone metastases, and bone marrow. CR=All components met criteria for CR. PR=PR in at least one component and all other components are either CR, MD (in bone marrow), PR (soft tissue or bone) or not involved (NI; no component with PD). MR = PR or CR in at least one component but at least one other component with stable disease; no component with PD. SD = Stable disease in one component with no better than SD or NI in any other component; no component with PD. PD = Any component with PD. | All participants for whom data was collected using the data collection tool and observed in this study were included in the analysis. | Posted | Count of Participants | Participants | 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) |
|
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| Primary | Number of Participants With Best Overall Response Based on HCP Reported Objective Response | Best overall response based on HCP reported objective response comprised of responses (CR, PR, MR, SD and PD) in 3 components: primary tumor, soft tissue and bone metastases, and bone marrow. CR=All components met criteria for CR. PR=At least one component and all other components are either CR, MD (in bone marrow), PR (soft tissue or bone) or not involved (NI; no component with PD). MR = PR or CR in at least one component but at least one other component with stable disease; no component with PD. SD = It is in one component with no better than SD or NI in any other component PD = Any component with PD. Best overall response taking the HCP reported response observed up to the data cut-off date for a specific milestone. | All participants for whom data was collected using the data collection tool and observed in this study were included in the analysis. | Posted | Count of Participants | Participants | 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) |
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| Primary | Number of Participants With Best Overall Response Based on Derived Objective Response | Best overall response based on derived objective response comprised of responses (CR, PR, MR, SD and PD) in 3 components: primary tumor, soft tissue and bone metastases, and bone marrow. CR=All components met criteria for CR. PR=PR in at least one component and all other components are either CR, MD (in bone marrow), PR (soft tissue or bone) or not involved (NI; no component with PD). MR= PR or CR in at least one component but at least one other component with stable disease; no component with PD. SD= Stable disease in one component with no better than SD or NI in any other component; no component with PD. PD= Any component with PD. | All participants for whom data was collected using the data collection tool and observed were included in the analysis. | Posted | Count of Participants | Participants | 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) |
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| Primary | Overall Response Rate Based on HCP Reported Response | Overall response rate was defined as the percentage of participants with a best overall response of CR or PR. CR=All components (primary tumor, soft tissue and bone metastases, and bone marrow) met criteria for CR. PR=PR in at least one component and all other components are either CR, MD (in bone marrow), PR (soft tissue or bone) or not involved (NI; no component with PD). Two sided 95% confidence interval was based on Clopper Pearson method. | All participants for whom data was collected using the data collection tool and observed in this study were included in the analysis. | Posted | Number | 95% Confidence Interval | Percentage of participants | 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) |
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| Primary | Overall Response Rate Based on Derived Response | Overall response rate was defined as the percentage of participants with a best overall response of CR or PR. CR=All components (primary tumor, soft tissue and bone metastases, and bone marrow) met criteria for CR. PR=PR in at least one component and all other components are either CR, MD (in bone marrow), PR (soft tissue or bone) or not involved (NI; no component with PD). | All participants for whom data was collected using the data collection tool and observed in this study were included in the analysis. Here, 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure. | Posted | Number | 95% Confidence Interval | Percentage of Participants | 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) |
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| Primary | Duration of HCP Reported Overall Responses | Duration of HCP reported overall response was derived as (earliest date of disease progression or death minus earliest date of complete or partial response + 1)/30.4. Participants who had not progressed or died were censored at their last assessment date prior to the data cut-off date. CR=All components (primary tumor, soft tissue and bone metastases, and bone marrow) met criteria for CR. PR=PR in at least one component and all other components are either CR, MD (in bone marrow), PR (soft tissue or bone) or not involved. PD=Any component with PD. | All participants for whom data was collected using the data collection tool and observed in this study were included in the analysis. | Posted | Median | 95% Confidence Interval | Months | 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) |
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| Primary | Progression Free Survival (PFS) | PFS was derived as (date of PD or death [by any cause in the absence of PD] minus date of first dose of lorlatinib plus 1) divided by 30.4. Participants who had not progressed or died were censored at the date of last contact prior to the data cut-off date. PD: new soft tissue lesion (STL) detected by CT/MRI that's MIBG avid/FDG-PET avid, new STL on anatomic imaging, biopsied and confirmed as neuroblastoma (NB)/ganglioneuroblastoma (GNB), new bone site: MIBG avid/FDG-PET avid (for MIBG non-avid tumor) with CT/MRI finding consistent with tumor or confirmed histologically as NB/GNB; >20% increase in LD as reference smallest sum on study (included baseline sum if that was the smallest on study), minimum abs increase of 5mm in sum of diameter of target STL. | All participants for whom data was collected using the data collection tool and observed in this study were included in the analysis. | Posted | Median | 95% Confidence Interval | Months | 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) |
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| Primary | Duration of Treatment | Duration of treatment was derived as treatment stop date minus treatment start date plus 1. Participants continuing treatment at the data cut-off date were censored based on the last recorded date when the participant was known to be continuing treatment prior to the data cut-off date. | All participants for whom data was collected using the data collection tool and observed in this study were included in the analysis. | Posted | Median | 95% Confidence Interval | Months | 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) |
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| Primary | Overall Survival | Overall survival was derived as date of death minus treatment start date plus 1. Participants who had not died were censored at the date of last contact prior to the data cut-off date. | All participants for whom data was collected using the data collection tool and observed in this study were included in the analysis. | Posted | Median | 95% Confidence Interval | Months | 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) |
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|
|
| Primary | Number of Participants Reporting Adverse Events, Treatment-Related Adverse Events, Serious Adverse Events and Treatment-Related Serious-Adverse Events | An AE is any untoward medical occurrence in participants administered a medicinal product. The event need not necessarily have a causal relationship with the product treatment or usage. A SAE is any untoward medical occurrence in a participant administered a medicinal or nutritional product at any dose that: resulted in death, was life-threatening, required participant hospitalization or prolongation of hospitalization, resulted in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions) and resulted in congenital anomaly/birth defect. Relatedness to treatment was determined by investigator. | All participants for whom data was collected using the data collection tool and observed in this study were included in the analysis. | Posted | Count of Participants | Participants | 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) |
|
|
|
| 8 |
| 15 |
| 6 |
| 15 |
| 12 |
| 15 |
| Peripheral motor neuropathy | Nervous system disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Peripheral sensory neuropathy | Nervous system disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Spinal cord compression | Nervous system disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA v25.0 | Non-systematic Assessment |
|
| Rhinovirus infection | Infections and infestations | MedDRA v25.0 | Non-systematic Assessment |
|
| Sepsis | Infections and infestations | MedDRA v25.0 | Non-systematic Assessment |
|
| Weight increased | Investigations | MedDRA v25.0 | Non-systematic Assessment |
|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Hypothyroidism | Endocrine disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Primary hypogonadism | Endocrine disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Pyrexia | General disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Hyperlipidaemia | Metabolism and nutrition disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Tumour haemorrhage | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v25.0 | Non-systematic Assessment |
|
| Urinary retention | Renal and urinary disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Weight increased | Investigations | MedDRA v25.0 | Non-systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA v25.0 | Non-systematic Assessment |
|
| Blood triglycerides increased | Investigations | MedDRA v25.0 | Non-systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | MedDRA v25.0 | Non-systematic Assessment |
|
| Blood lactate dehydrogenase increased | Investigations | MedDRA v25.0 | Non-systematic Assessment |
|
| Gamma-glutamyltransferase increased | Investigations | MedDRA v25.0 | Non-systematic Assessment |
|
| Lymphocyte count decreased | Investigations | MedDRA v25.0 | Non-systematic Assessment |
|
| Platelet count decreased | Investigations | MedDRA v25.0 | Non-systematic Assessment |
|
| White blood cell count decreased | Investigations | MedDRA v25.0 | Non-systematic Assessment |
|
| Hypercholesterolaemia | Metabolism and nutrition disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Hypertriglyceridaemia | Metabolism and nutrition disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Hypernatraemia | Metabolism and nutrition disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Cognitive disorder | Nervous system disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Fine motor delay | Nervous system disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Paraesthesia | Nervous system disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Peripheral sensory neuropathy | Nervous system disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Speech disorder developmental | Nervous system disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Anaemia | Blood and lymphatic system disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Candida infection | Infections and infestations | MedDRA v25.0 | Non-systematic Assessment |
|
| Pyelonephritis | Infections and infestations | MedDRA v25.0 | Non-systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Oedema peripheral | General disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Ascites | Gastrointestinal disorders | MedDRA v25.0 | Non-systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA v25.0 | Non-systematic Assessment |
|
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| D009373 | Neoplasms, Germ Cell and Embryonal |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D009375 | Neoplasms, Glandular and Epithelial |
| D009380 | Neoplasms, Nerve Tissue |
| Title | Measurements |
|---|
|
| PD |
|
| Missing |
|
| Title | Measurements |
|---|
|
| SD |
|
| Missing |
|
| Title | Measurements |
|---|
|
| SD |
|
| PD |
|
| Title | Measurements |
|---|
|
| PD |
|
| Missing |
|
| Not Evaluable |
|
| SD |
|
| PD |
|
| Title | Measurements |
|---|---|
|
| Serious Treatment related AEs |
|