Clinical Trial

Disease: Relapsed/​Refractory T-Cell Acute Lymphoblastic Leukemia or Lymphoma, (NCT06934382)

Disease info:

T-cell acute lymphoblastic leukaemia (T-ALL) is an aggressive blood and bone marrow cancer that disrupts normal T-cell development in the thymus. It represents 25% of adult ALL cases and 10-15% of childhood cases, with median onset at age 9 in children and age 30 in adults.

In T-ALL, T-cells become cancerous during their normal development process in the thymus, often spreading to the central nervous system. About 75% of cases develop mediastinal masses behind the breastbone. Genetic mutations cause T-ALL, and risk factors include family history, genetic conditions such as Down syndrome and previous radiation exposure.

Symptoms include fatigue, easy bruising, excessive bleeding, breathing problems, lymph node swelling, and recurrent infections. Diagnosis involves blood tests, bone marrow biopsy, imaging, and genetic testing. Treatment options include chemotherapy, radiation, targeted therapy, stem cell transplants, and supportive care. Prognosis varies significantly: approximately 75% of children and 60% of adults remain cancer-free after treatment, though about one-third experience relapse within 1-2 years.

Lymphoblastic lymphoma (LL) is a rare, fast-growing type of non-Hodgkin lymphoma that develops when the body produces abnormal lymphocytes. It can arise from both B-cell and T-cell lymphocytes and is most common in children and teenagers.

LL is very similar to acute lymphoblastic leukemia (ALL) and is typically treated in a similar way. The abnormal lymphocytes usually accumulate in lymph nodes but can affect other body parts. The causes are largely unknown. Common symptoms include painless swelling in the neck, armpit, or groin, and mediastinal masses that can cause shortness of breath, cough, and chest pain. Some patients experience so-called "B symptoms", including night sweats, unexplained fevers, and weight loss. General symptoms may include loss of appetite and tiredness.

Diagnosis involves lymph node biopsy and tissue analysis. LL is classified as high-grade (fast-growing), though staging doesn't significantly affect treatment planning. Treatment options include chemotherapy, steroids, targeted therapy, radiotherapy, and stem cell transplants. A multidisciplinary team develops individualised treatment plans based on lymphoma stage and symptom severity. Regular follow-up appointments monitor recovery and manage potential long-term effects after treatment completion.

Sources: Leukemia Research Foundation and Macmillan Cancer Support

Frequency:
The American Cancer Society estimates ~6,100 new ALL cases and ~1,400 deaths in the US for 2025. For non-Hodgkin lymphoma, ~80,350 new cases and ~19,390 deaths are projected. Lymphoblastic lymphoma comprises ~2% of NHL cases.
Official title:
A Phase 1 Study of Allogeneic Anti-CD7 CAR-T Cells (BEAM-201) in Relapsed/Refractory T-cell Acute Lymphoblastic Leukemia (T-ALL) or T-cell Lymphoblastic Lymphoma (T-LLy)
Who:

Name: Cell Therapy Intake Team

Phone Number: 445-942-5891

Email: cartintake@chop.edu

Sponsor:

Stephan Grupp MD PhD, Children's Hospital of Philadelphia

Locations:

United States

Philadelphia, Pennsylvania, United States, 19104

Recruiting, Children's Hospital of Philadelphia

Contact: Melissa S Varghese, BA

8455535358 Varghesem@chop.edu

Study start:
Apr. 29, 2025
Enrollment:
33
Gene editing method:
Base editing
Type of edit:
Gene disruption
Gene:
CD7, TRAC, PDCD1 and CD52
Delivery method:
Electroporation - Ex-vivo
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

Description

Despite favorable outcomes in newly diagnosed patients, approximately 20% of pediatric and young adult T-ALL patients and 40% of adult patients will have refractory disease or will relapse within 2 years of their initial diagnosis. Survival rates of patients with relapsed disease remain below 35%. For recurrent disease, allogeneic hematopoietic stem cell transplant (HSCT) is the only known potentially curative treatment. However, a prerequisite to HSCT is obtaining a complete remission, which remains a significant challenge as only 40 to 50% of patients achieve a second remission with current reinduction regimens with salvage rates even lower for patients with disease that is refractory to first-line chemotherapy.

BEAM-201 is an allogeneic anti-CD7 CAR T cell product that has shown promising early evidence of efficacy, with 3 out of 4 adult T-ALL patients infused had a CRi/CR ≥28 days after infusion. Safety of BEAM-201 has been favorable and consistent with known adverse events associated with other CAR T cell therapies.

Given the current treatment landscape of T-ALL/T-LLy, promising clinical experience with BEAM-201, and that >98% of T-ALL cases highly and homogenously express CD7 protein on the surfaces of their lymphoblasts, the investigators think there is compelling rationale in further investigating the safety and efficacy of BEAM-201 in pediatric patients.

Last updated: Jun. 15, 2025
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