Clinical trial · Observational
Real World Data Collection Pediatric Neuroblastoma Treated With Lorlatinib
REAL WORLD DATA COLLECTION AMONG PEDIATRIC NEUROBLASTOMA PATIENTS TREATED WITH LORLATINIB THROUGH EXPANDED ACCESS PROGRAM
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): 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.
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Neuroblastoma | Neuroblastoma | ONTOLOGY_EXACT | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| lorlatinib | Drug | Lorlatinib | ALIAS |
Design
Arms and outcomes
Arms (1)
- label
- Pediatric Neuroblastoma Patients Treated with Lorlatinib
- interventionNames
- Drug: lorlatinib
Primary outcomes (14)
- 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)
- description
- 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.
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 0 Years
Show eligibility criteria text
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.
References
Publications (0)
Data not yet available