Phase 1 Gaucher Disease Trial, Recruiting

Written by an AI model from the ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• A parent or legal guardian has signed the required privacy and authorization forms
• The patient is well enough to carry out some daily activities, based on standard health scoring scales used for children or adults
• The patient is expected to live for at least 12 weeks
• The patient was confirmed to have neuroblastoma or ganglioneuroblastoma (a related tumor type) through a tissue or bone marrow test at the time of their original diagnosis, or was confirmed to have osteosarcoma (a type of bone cancer) at diagnosis
• The patient has high-risk neuroblastoma that has come back, is still present, or did not respond to standard treatment — including cases where the disease returned after completing intensive chemotherapy, kept growing during chemotherapy, or did not fully respond after at least 4 cycles of intensive treatment; or the patient has osteosarcoma that has come back or did not respond to standard treatment
• The patient still has signs of disease that can be detected or measured through scans or other tests
• The patient has no known brain or spinal cord involvement from their disease, and does not have a seizure condition requiring medication

Who may not be able to join:

• The patient is pregnant or currently breastfeeding
• The patient has another active cancer that is growing and requires treatment
• The patient has had a severe allergic reaction to products containing mouse-derived proteins in the past
• The patient has had a severe allergic reaction to the chemotherapy drugs cyclophosphamide or fludarabine in the past

Important: Always verify eligibility with the trial site directly before applying.