Investigating afamitresgene autoleucel for pediatric cancers
A Phase 1/2 Open Label, Basket Study to Assess the Safety, Tolerability and Anti-Tumor Activity of Afamitresgene Autoleucel in Pediatric Subjects With MAGE-A4 Positive Tumors
This study is testing a new treatment called afamitresgene autoleucel to see if it can help children and young adults with certain advanced cancers that have specific genetic markers.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 2 Years to 21 Years |
| Sex | All |
| Sponsor | USWM, LLC (dba US WorldMeds) Industry-sponsored |
| Drugs / interventions | chemotherapy, cyclophosphamide, fludarabine |
| Locations | 10 sites (Palo Alto, California and 9 other locations) |
| Trial ID | NCT05642455 on ClinicalTrials.gov |
What this trial studies
This pediatric basket study aims to evaluate the safety and efficacy of afamitresgene autoleucel in children and young adults aged 2-21 years who are HLA-A*02 eligible and MAGE-A4 positive, diagnosed with advanced cancers such as synovial sarcoma, malignant peripheral nerve sheath tumor, neuroblastoma, or osteosarcoma. Participants must have previously received systemic chemotherapy and have measurable disease. The study will assess the treatment's impact on these specific cancer types, focusing on the unique genetic markers present in the tumors.
Who should consider this trial
Good fit: Ideal candidates are children and young adults aged 2-21 years with specific advanced cancers who meet the eligibility criteria.
Not a fit: Patients with HLA-A*02:05 or similar alleles, those with a history of autoimmune diseases, or significant cardiovascular issues may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a novel therapeutic option for pediatric patients with advanced cancers that currently have limited treatment options.
How similar studies have performed: While this approach is innovative, similar studies targeting specific genetic markers in pediatric cancers have shown promise, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Subject has histologically confirmed diagnosis of any one of the following cancers: (A) Synovial Sarcoma (SS), (B) MPNST, (C) Neuroblastoma, or (D) Osteosarcoma (OS). * Age: (A) Synovial Sarcoma: 2 to 17 years (B) MPNST, Neuroblastoma and Osteosarcoma: 2 to 21 years * Body weight ≥ 10 kg * Must have previously received a systemic chemotherapy * Measurable disease prior to lymphodepletion according to RECIST v1.1 (or INCR, 2017 Neuroblastoma only). * HLA-A\*02 positive * Tumor shows MAGE-A4 expression confirmed by central laboratory. * Performance Status: (A) Subjects ≥16: Eastern Cooperative Oncology Group (ECOG) 0 or 1 (B) Subjects 2 to 16: Lansky score ≥ 80 • Subject has anticipated life expectancy of greater than 3 months in the opinion of the investigator. Exclusion Criteria: * Positive for HLA-A\*02:05 in either allele; or any A\*02 having same protein sequence as HLA-A\*02:05 * History of allergic reactions attributed to compounds of similar chemical or biologic composition to fludarabine, cyclophosphamide. * History of autoimmune or immune mediated disease * Known central nervous system (CNS) metastases. * Other prior malignancy that is not considered by the Investigator to be in complete remission * Clinically significant cardiovascular disease * Active infection with human immunodeficiency virus, hepatitis B virus, hepatitis C virus, or human T cell leukemia virus * Pregnant or breastfeeding * Experiencing ongoing rapid disease progression that in the opinion of the Investigator significantly increases the subjects risk associated with treatment.
Where this trial is running
Palo Alto, California and 9 other locations
- Stanford University — Palo Alto, California, United States (Recruiting)
- National Institutes of Health — Bethesda, Maryland, United States (Recruiting)
- Dana Farber Cancer Institute — Boston, Massachusetts, United States (Not_yet_recruiting)
- Washington University — St Louis, Missouri, United States (Recruiting)
- Memorial Sloan Kettering Kids — New York, New York, United States (Not_yet_recruiting)
- Duke University School of Medicine — Durham, North Carolina, United States (Not_yet_recruiting)
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (Recruiting)
- Children's Hospital of Philedephia — Philadelphia, Pennsylvania, United States (Recruiting)
- Seattle Children's Hospital — Seattle, Washington, United States (Not_yet_recruiting)
- University of Wisconsin Cancer Center — Madison, Wisconsin, United States (Recruiting)
Study contacts
- Principal investigator: Fiorella Iglesias Cardenas, MD — Memorial Sloan Kettering Kids
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.