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Recruiting NCT05642455

SPEARHEAD-3 Pediatric Study

Phase I / Phase II Interventional Synovial Sarcoma Malignant Peripheral Nerve Sheath Tumor (MPNST) Neuroblastoma (NBL) Osteosarcoma

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Afamitresgene autoleucel.
Who it may be relevant to
Registry conditions: Synovial Sarcoma, Malignant Peripheral Nerve Sheath Tumor (MPNST), Neuroblastoma (NBL), Osteosarcoma. Basic parameters: 2 years — 21 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Interventions

  • Genetic Afamitresgene autoleucel
    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

Primary outcome measures

  • Incidence, duration, and severity of Treatment Emergent Adverse Events as assessed by Investigator Evaluation. [Time frame: 3.5 years]
Secondary outcome measures (8)
  • 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) [Time frame: 3.5 years]
  • Time to response (TTR) [Time frame: 3.5 years]
  • Duration of Response (DoR) [Time frame: 3.5 years]
  • Best overall response (BOR) [Time frame: 3.5 years]
  • Progression Free Survival (PFS) [Time frame: 3.5 years]
  • Overall Survival (OS) [Time frame: 15 years]
  • Characterize the in vivo cellular pharmacokinetics (PK) profile of afamitresgene autoleucel by evaluation of PBMC samples for peak persistence. [Time frame: 3.5 years]
  • Development and validation of an invitro diagnostic (IVD) assay for the screening of tumor antigen expression for regulatory approval. [Time frame: 3.5 years]

Eligibility 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 10 centers
  • Stanford University — Palo Alto
  • National Institutes of Health — Bethesda
  • Dana Farber Cancer Institute — Boston
  • Washington University — St Louis
  • Memorial Sloan Kettering Kids — New York
  • Duke University School of Medicine — Durham
  • Cincinnati Children's Hospital Medical Center — Cincinnati
  • Children's Hospital of Philedephia — Philadelphia
  • … and 2 more centers

Identifiers

NCT: NCT05642455 · ADP-0044-004

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗