Nükseden/Dirençli Yüksek Riskli Nöroblastom Hastalarında hALK.CAR T Hücreleri Çalışması

Sade dilde özet

Bu Faz 1/2 (Phase 1/2) çalışma, nükseden veya tedaviye dirençli nöroblastomu (neuroblastoma) olan çocuklarda, insan Anaplastik Lenfoma Kinaz (ALK) reseptörünü hedefleyen kendi hücrelerinden üretilmiş CAR T hücrelerinin güvenliğini ve uygulanabilirliğini araştırıyor. Faz 1 bölümünde 3+3 doz artırım tasarımıyla maksimum tolere edilen doz (MTD) belirleniyor, Faz 2 bölümünde ise yanıt oranları inceleniyor. Katılım için onam sırasında 12 aylık ile 30 yaş arasında olmak, nöroblastomun patolojik olarak doğrulanmış olması ve yüksek riskli hastalık ölçütlerini karşılamak gerekiyor; her doz düzeyindeki ilk hastanın 6 yaş ve üzerinde olması isteniyor.

Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.

Deney bilgileri

Faz

PHASE1/PHASE2

Durum

Katılımcı alıyor

Sponsor

Roberto Chiarle

Müdahale

Autologous hALK.CAR T cells

Başlangıç

31.01.2025

Tahmini bitiş

31.12.2029

Ülkeler

United States

İlgili genler

Merkezler

• Boston, United States
• Boston, United States

Uygunluk kriterleri

Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.

Inclusion Criteria:

1. Age ≥ 12 months and \< 30 years at the time of consent. The first patient on each dose level will need to be age ≥ 6 years old
2. Disease Status:

   1. Patients must have histologic verification of neuroblastoma at diagnosis or at relapse
   2. Patients must have high-risk neuroblastoma according to Children's Oncology Group (COG) risk classification at time of study enrollment
   3. Patients must have persistent/refractory or relapsed disease for which standard curative measures are no longer effective, as defined in the protocol
   4. Patients must have evaluable or measurable disease per the revised International Neuroblastoma Response Criteria (INRC)
3. Adequate washout from prior treatment regimens
4. Adequate organ function
5. Adequate performance status defined as Lansky or Karnofsky performance score ≥50%
6. Subjects of reproductive potential must agree to use acceptable birth control methods
7. Signed informed consent

Exclusion Criteria:

1. Pregnant or nursing (lactating) women
2. Patients with uncontrolled active infection
3. Patients who are concurrently receiving other investigational agents
4. Patients who have received prior CART-cell or other gene-modified immune-effector cell therapy, are not eligible unless they are \>8 weeks from time of infusion, have fully recovered from any associated toxicities and have documented lack of persistence of the product
5. Patients with a known additional malignancy other than non-melanomatous skin cancer or carcinoma in situ, unless not requiring active treatment and stable or disease-free for at least 3 years
6. Uncontrolled CNS metastasis
7. CNS disorder such as cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease, or autoimmune disease with CNS involvement which may impair the ability to evaluate neurotoxicity
8. History of severe hypersensitivity reaction to compounds used in the study
9. HIV/HBV/HCV infection
10. Patients receiving systemic steroid therapy (physiologic replacement, inhaled steroids and premedication for blood products are allowed)
11. Primary immunodeficiency or history of systemic autoimmune disease requiring systemic immunosuppression/disease modifying agents within the last 2 years
12. Uncontrolled intercurrent illness
13. Inability to comply with the study requirements