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| Diagnosis | Neuroblastoma | Study Status | Closed to enrollment |
| Phase | I |
| Age | Child, Adult, Older Adult - (1 Year to 90 Years) | Randomisation | NO |
| Line of treatment | Disease relapse or progression |
| Routes of Treatment Administration | Lorlatinib: Oral (tablet)
Cyclophosphamide: IV
Topotecan: IV |
| Last Posted Update | 2023-06-05 |
| ClinicalTrials.gov # | NCT03107988 |
International Sponsor
New Approaches to Neuroblastoma Therapy ConsortiumPrincipal Investigators for Canadian Sites
The Hospital for Sick Children - Dr.Daniel Morgenstern
Centres
Study Description
Some neuroblastoma have a specific genetic change or mutation called an ALK aberration. ALK, or anaplastic lymphoma kinase, has been found in several adult and pediatric cancers. ALK aberrations are present in about 14% of newly diagnosed patients with high-risk neuroblastoma, and can be found more frequently at the time of relapse. Lorlatinib is a drug called an ALK inhibitor. It is expected to slow or stop the growth of cancer cells which have the ALK aberration. The aim of this phase I/II study is to evaluate the dose, safety, and tolerability of lorlatinib, including the effect it has on the cancer. Lorlatinib will be given alone or in combination with chemotherapy in children with refractory, relapsed or progressive neuroblastoma with ALK alterations.
Inclusion Criteria
- Patients must have a diagnosis of neuroblastoma either by histologic verification (looking at a sample of the tumour under a microscope) of neuroblastoma and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines (HVA/VMA).
- Patients are required to have an activating ALK aberration in their tumor, which is identified through genetic testing.
- Patients must have high risk neuroblastoma. Patients who were initially considered low or intermediate risk, but then reclassified as high risk are also eligible.
- Patients must have at least ONE of the following: 1) Recurrent/progressive disease at any time prior to study enrollment, 2) Refractory disease, 3) Persistent disease
Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team.
