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HER2-specific CAR T Cell Locoregional Immunotherapy for HER2-positive Recurrent/Refractory Pediatric CNS Tumors
Bu Faz 1 (Phase 1) çalışma, nüksetmiş veya tedaviye dirençli (recurrent or refractory) HER2 pozitif merkezi sinir sistemi (central nervous system, CNS) tümörü olan 1–26 yaş arası çocuk ve genç yetişkinlerde, HER2'ye özgü bir kimerik antijen reseptörü (chimeric antigen receptor, CAR) taşıyacak şekilde lentivirüs ile değiştirilmiş, hastanın kendi (otolog, autologous) T hücrelerini inceliyor. CAR T hücreleri, tümörün çıkarıldığı boşluğa veya beyin ventrikül sistemine yerleştirilmiş kalıcı bir kateter (Ommaya veya Rickham gibi) aracılığıyla, her biri 3 hafta süren haftalık dozlardan oluşan iki kür halinde veriliyor; ardından etkileri MR görüntüleme (MRI) gibi incelemelerle değerlendiriliyor. Katılım için standart tedavisi bulunmayan dirençli veya nüksetmiş hastalık ve en az 8 haftalık yaşam beklentisi gerekiyor.
Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
Inclusion Criteria: 1. Age ≥ 1 and ≤ 26 years 2. Histologically diagnosed HER2-positive Central Nervous System (CNS) tumor 3. Evidence of refractory or recurrent CNS disease for which there is no standard therapy 4. Able to tolerate apheresis, or has apheresis product available for use in manufacturing 5. CNS reservoir catheter, such as an Ommaya or Rickham catheter 6. Life expectancy ≥ 8 weeks 7. Lansky or Karnofsky score ≥ 60 8. If patient does not have previously obtained apheresis product, patient must have recovered from acute toxic effects of all prior chemotherapy, immunotherapy, and radiotherapy and discontinue the following prior to enrollment: 1. ≥ 7 days post last chemotherapy/biologic administration 2. 3 half-lives or 30 days, whichever is shorter post last dose of anti-tumor antibody therapy 3. Must be at least 30 days from most recent cell infusion 4. All systemically administered corticosteroid treatment therapy must be stable or decreasing within 1 week prior to enrollment with maximum dexamethasone dose of 2.5 mg/m2/day. Corticosteroid physiologic replacement therapy is allowed. 9. Adequate organ function 10. Adequate laboratory values 11. Patients of childbearing/fathering potential must agree to use highly effective contraception Exclusion Criteria: 1. Diagnosis of classic diffuse intrinsic pontine glioma (DIPG) 2. Presence of Grade ≥ 3 cardiac dysfunction or symptomatic arrhythmia requiring intervention 3. Presence of primary immunodeficiency/bone marrow failure syndrome 4. Presence of clinical and/or radiographic evidence of impending herniation 5. Presence of active malignancy other than the primary CNS tumor under study 6. Presence of active severe infection 7. Pregnant or breastfeeding 8. Subject and/or authorized legal representative unwilling or unable to provide consent/assent for participation in the 15-year follow up period 9. Presence of any condition that, in the opinion of the investigator, would prohibit the patient from undergoing treatment under this protocol