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This is a pediatric basket study to investigate the safety and efficacy of afamitresgene autoleucel in HLA-A*02 eligible and MAGE-A4 positive subjects aged 2-17 years of age with advanced cancers.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Afamitresgene autoleucel | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Afamitresgene autoleucel | Genetic | Single infusion of afamitresgene autoleucel Dose: For subjects ≥10 kg to <40 kg: starting dose of 0.025 - 0.200 x 10'9 transduced cells/kg. For subjects ≥40 kg 1.0x109 to 10x109 transduced by a single intravenous infusion |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence, duration, and severity of Treatment Emergent Adverse Events as assessed by Investigator Evaluation. | Determination of incidence, severity and duration of adverse events
| 3.5 years |
| Measure | Description | Time Frame |
|---|---|---|
| Efficacy: Objective response rate (ORR) assessed by investigator per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 (or by International Neuroblastoma Response Criteria [INRC] 2017 in Neuroblastoma subjects) | ORR is defined as incidence of complete responses or partial responses as assessed by RECIST v1.1 or INRC, 2017 | 3.5 years |
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Inclusion Criteria:
(A) Synovial Sarcoma: 2 to 17 years (B) MPNST, Neuroblastoma and Osteosarcoma: 2 to 21 years
(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:
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| Name | Affiliation | Role |
|---|---|---|
| Fiorella Iglesias Cardenas, MD | Memorial Sloan Kettering Kids | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford University | Recruiting | Palo Alto | California | 94305 | United States |
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| ID | Term |
|---|---|
| D013584 | Sarcoma, Synovial |
| D018319 | Neurofibrosarcoma |
| D009447 | Neuroblastoma |
| D012516 | Osteosarcoma |
| ID | Term |
|---|---|
| D009372 | Neoplasms, Connective Tissue |
| D018204 | Neoplasms, Connective and Soft Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
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| Time to response (TTR) | For patients who are observed to respond to afamitresgene autoleucel in the time from date of infusion to achieve a partial response or complete response (TTR) is assessed | 3.5 years |
| Duration of Response (DoR) | For patients who are observed to respond to afamitresgene autoleucel the DoR is the date of initial response (including confirmation) from date of infusion up until disease progression | 3.5 years |
| Best overall response (BOR) | BOR is assessed by the investigator per RECIST V1.1 or INCR, 2017 (for Neuroblastoma subjects) | 3.5 years |
| Progression Free Survival (PFS) | PFS is assessed by the investigator from date of infusion of ADP-A2M4 up until the date of disease progression per RECIST v1.1 or death. | 3.5 years |
| Overall Survival (OS) | OS is assessed from date of infusion of ADP-A2M4 up until the date of patient death. | 15 years |
| Characterize the in vivo cellular pharmacokinetics (PK) profile of afamitresgene autoleucel by evaluation of PBMC samples for peak persistence. | Obtain PBMC samples for the evaluation of peak persistence of afamitresgene autoleucel. | 3.5 years |
| Development and validation of an invitro diagnostic (IVD) assay for the screening of tumor antigen expression for regulatory approval. | Retention of additional tumor tissue during Pre-Screening to enable development and validation of a MAGE-A4 antigen expression companion diagnostic (CDx) assay. | 3.5 years |
| National Institutes of Health | Recruiting | Bethesda | Maryland | 20892 | United States |
|
| Dana Farber Cancer Institute | Not yet recruiting | Boston | Massachusetts | 10065 | United States |
|
| Washington University | Recruiting | St Louis | Missouri | 63110 | United States |
|
| Memorial Sloan Kettering Kids | Not yet recruiting | New York | New York | 10065 | United States |
|
| Duke University School of Medicine | Not yet recruiting | Durham | North Carolina | 27710 | United States |
|
| Cincinnati Children's Hospital Medical Center | Recruiting | Cincinnati | Ohio | 45229 | United States |
|
| Children's Hospital of Philedephia | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
|
| Seattle Children's Hospital | Not yet recruiting | Seattle | Washington | 98105 | United States |
|
| University of Wisconsin Cancer Center | Recruiting | Madison | Wisconsin | 53715 | United States |
|
| D012509 | Sarcoma |
| D005354 | Fibrosarcoma |
| D018218 | Neoplasms, Fibrous Tissue |
| D009455 | Neurofibroma |
| D018317 | Nerve Sheath Neoplasms |
| D009380 | Neoplasms, Nerve Tissue |
| D010524 | Peripheral Nervous System Neoplasms |
| D009423 | Nervous System Neoplasms |
| D009422 | Nervous System Diseases |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D018241 | Neuroectodermal Tumors, Primitive, Peripheral |
| D018242 | Neuroectodermal Tumors, Primitive |
| D018302 | Neoplasms, Neuroepithelial |
| D017599 | Neuroectodermal Tumors |
| D009373 | Neoplasms, Germ Cell and Embryonal |
| D009375 | Neoplasms, Glandular and Epithelial |
| D018213 | Neoplasms, Bone Tissue |