BEAM-201 for T-cell Leukemia/Lymphoma

Not currently recruiting at 10 trial locations

Overseen By Medical Information

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Beam Therapeutics Inc.

Must not be taking: CD7 therapy

Disqualifiers: CNS involvement, Neurological toxicity, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Phase 1/2

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called BEAM-201 for individuals with T-cell leukemia or lymphoma. The focus is on those whose cancer has returned or hasn't responded to treatment. Researchers aim to determine if BEAM-201 is safe and effective. Patients with CD7-positive T-cell leukemia or lymphoma that hasn't improved with past treatments might be suitable for this study. As a Phase 1 trial, this research seeks to understand how the treatment works in people, offering participants the opportunity to be among the first to receive it.

Do I need to stop my current medications to join the trial?

The trial protocol does not specify if you need to stop your current medications. However, you cannot have received systemic antileukemic therapy intended to induce or maintain remission within 14 days before completing screening.

Will I have to stop taking my current medications?

The trial protocol does not specify if you need to stop taking your current medications. However, you cannot have received systemic antileukemic therapy intended to induce or maintain remission within 14 days before completing screening.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that BEAM-201 has a safety profile similar to treatments for related conditions. Early data indicate that the safety of BEAM-201 aligns with the known side effects of CAR-T therapy. CAR-T therapy involves modifying a patient's T-cells (a type of white blood cell) to attack cancer cells.

These side effects might include issues from the disease itself and the preparatory treatment, known as lymphodepletion, which helps make room in the body for new cells. However, the data does not specify the severity or frequency of these side effects.

As this is an early-stage study, it focuses on understanding how well people tolerate the treatment. Researchers are still learning about its safety in humans, so some uncertainty remains. However, the data so far suggest that the treatment behaves as expected compared to similar therapies. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about BEAM-201 for T-cell Leukemia/Lymphoma because it offers a new approach by using genetic engineering to enhance T-cells' ability to target and kill cancer cells. Unlike traditional chemotherapy, which affects both healthy and cancerous cells, BEAM-201 is designed to home in specifically on the malignant cells, potentially reducing side effects. Additionally, the trial investigates two different lymphodepletion regimens, one with alemtuzumab and one without, to optimize the environment for BEAM-201’s effectiveness. This targeted approach and the exploration of optimal pre-treatment regimens could significantly improve outcomes for patients with this challenging condition.

What evidence suggests that BEAM-201 might be an effective treatment for T-cell Leukemia/Lymphoma?

Research shows that BEAM-201, a new treatment, has shown promising early results for treating certain blood cancers, specifically T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LL). In one study, 3 out of 4 adult patients with T-ALL responded well to BEAM-201. Additionally, early results suggest that even with lower doses, 2 out of 3 patients experienced significant cancer reduction or elimination. These findings are encouraging for those considering trials with BEAM-201. In this trial, participants will undergo a lymphodepletion regimen with either fludarabine, cyclophosphamide, and alemtuzumab or fludarabine and cyclophosphamide without alemtuzumab before receiving BEAM-201. ^{1 2 4 5 6}

Are You a Good Fit for This Trial?

This trial is for people aged 1 to 50 with T-Cell Acute Lymphoblastic Leukemia or T-Cell Lymphoblastic Lymphoma that has come back or didn't respond to treatment. They should have a certain amount of cancer cells in their bone marrow or evidence of the disease after a second remission, and be eligible for a type of stem cell transplant.

Inclusion Criteria

I am approved for a stem cell transplant and have a donor.

My T-ALL/T-LL cancer is in its second or later relapse, has come back after a transplant, or hasn't responded to chemotherapy.

I am between 1 and 17 years old.

Exclusion Criteria

I have previously received CD7 targeted therapy.

I have received treatment for leukemia within the last 14 days.

I have brain-related symptoms or permanent nerve damage from past leukemia treatments.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Lymphodepletion

Participants receive a lymphodepletion regimen including fludarabine, cyclophosphamide, and optionally alemtuzumab

1-2 weeks

Treatment

Participants receive BEAM-201 CAR-T cells

Phase 1/2

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• BEAM-201

Trial Overview The study tests BEAM-201's safety and effectiveness on patients with relapsed/refractory T-ALL/T-LL. It includes initial dose-finding, expansion cohorts, a pediatric group (ages 1 to <12), and Phase 2 cohort to determine how well it works at selected doses.

How Is the Trial Designed?

Group I: Fludarabine, cyclophosphamide without alemtuzumab Experimental Treatment 1 Intervention

Lymphodepletion regimen without Alz but consisting of the same dose of Flu/Cy as in the other arm

Group II: Fludarabine, cyclophosphamide and alemtuzumab Experimental Treatment 1 Intervention

Lymphodepletion regimen including fludarabine, cyclophosphamide and alemtuzumab

Who Is Running the Clinical Trial?

Beam Therapeutics Inc.

Lead Sponsor

Trials

5

Recruited

1,200+

Published Research Related to This Trial

A case study of a 50-year-old male with rapidly progressive stage IVB mycosis fungoides showed a remarkable 95% response to low-dose total skin electron beam (TSEB) therapy (24 Gy over 8 weeks) with no apparent side effects, highlighting its potential efficacy even in advanced disease.

The patient has remained in remission for over 4 years after treatment, suggesting that low-dose TSEB can be a viable option for managing advanced-stage mycosis fungoides, which is typically considered palliative.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rapidly progressive stage IVB mycosis fungoides treated with low-dose total skin electron beam therapy. Chowdhary, M., Kabbani, AA., Rimtepathip, P., et al.[2022]

In a phase 2 trial involving 349 patients with newly diagnosed T-cell acute lymphoblastic leukaemia, the ALL-T11 protocol achieved a high 3-year event-free survival rate of 86.4% and an overall survival rate of 91.3%, indicating effective treatment outcomes with reduced reliance on cranial radiotherapy and stem cell transplantation.

The study demonstrated that the addition of nelarabine and intensified L-asparaginase therapy led to a high complete remission rate of 89%, while the treatment was associated with manageable side effects, including a low incidence of severe neuropathy and febrile neutropenia.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Nelarabine, intensive L-asparaginase, and protracted intrathecal therapy for newly diagnosed T-cell acute lymphoblastic leukaemia in children and young adults (ALL-T11): a nationwide, multicenter, phase 2 trial including randomisation in the very high-risk group. Sato, A., Hatta, Y., Imai, C., et al.[2023]

Total skin electron beam therapy (TSEB) demonstrated a high efficacy in treating early-stage mycosis fungoides, with a complete clinical response in 97% of patients three months after treatment, based on a retrospective study of 68 patients.

Despite some acute toxicity, such as localized ulcerations in 13.2% of patients, TSEB showed favorable long-term outcomes with 5-year overall survival rates of 86% and disease-free survival rates of 41%, suggesting it should be considered a first-line treatment option.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Total skin electron beam therapy for early-stage mycosis fungoides: immediate results and long-term follow-up in 68 patients]. Goujon, E., Truc, G., Pétrella, T., et al.[2009]

In patients with advanced-stage cutaneous T-cell lymphoma (CTCL) who achieved a complete response after total skin electron beam therapy (TSEBT), the adjuvant therapy of extracorporeal photochemotherapy (ECP) showed a potential survival benefit, with 100% overall survival at 3 years for T3 and T4 patients, compared to approximately 50% for those who received no adjuvant therapy.

Conversely, the combination of doxorubicin and cyclophosphamide did not significantly improve overall survival for patients who achieved a complete response to TSEBT, and neither adjuvant therapy affected relapse-free survival across all T-stages.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Systemic chemotherapy and extracorporeal photochemotherapy for T3 and T4 cutaneous T-cell lymphoma patients who have achieved a complete response to total skin electron beam therapy. Wilson, LD., Licata, AL., Braverman, IM., et al.[2019]

In a study of 14 patients with primary cutaneous T-cell lymphoma (PCTCL) treated with total skin electron beam therapy (TSEBT), the overall response rate was 85% after 6-8 weeks, indicating that TSEBT is an effective treatment option for this condition.

The study found that low-dose TSEBT (12 Gy) is preferred due to its effectiveness and the possibility of retreatment, with a median progression-free survival of 6 months for the cohort, highlighting its role in managing advanced stages of PCTCL.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Total skin electron beam therapy for primary cutaneous T-cell lymphomas: clinical characteristics and outcomes in a Mexican reference center. Flores-Balcázar, C., Urías-Arce, DM., Charli-Joseph, Y., et al.[2022]

Patients with acute adult T-cell leukemia/lymphoma (ATL) show significant benefits from first-line combined antiviral therapy using zidovudine and interferon alpha, while those with lymphoma-type ATL may respond better to intensive chemotherapy.

Allogeneic stem-cell transplantation offers promising outcomes, with up to 40% of patients achieving long-term survival after remission, highlighting the need for further research into effective treatment strategies for different ATL subsets.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Newer developments in adult T-cell leukemia/lymphoma therapeutics. Dasanu, CA.[2013]

Total skin electron beam radiation treatment for mycosis fungoides resulted in an 82% overall complete response rate, with better outcomes for patients with lower T stages (T1) compared to higher stages (T2-4).

Patients with T1N0 mycosis fungoides who received 35 Gy had a remarkable 10-year mycosis fungoides-specific survival rate of 100%, indicating the effectiveness of this treatment for early-stage disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Prognosis with newly diagnosed mycosis fungoides after total skin electron radiation of 30 or 35 GY. Jones, GW., Tadros, A., Hodson, DI., et al.[2019]

The FDA has approved three innovative therapies for relapsed/refractory B-cell acute lymphoblastic leukemia, including inotuzumab ozogamicin and blinatumomab, which have significantly changed treatment options for this disease.

Understanding the unique toxicities and adverse effects of these therapies is crucial for safe administration, and the article provides recommendations for preventing and managing these side effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Management of adverse effects of new monoclonal antibody treatments in acute lymphoblastic leukemia. Conde-Royo, D., Juárez-Salcedo, LM., Dalia, S.[2020]

CAR T cell therapy, specifically tisagenlecleucel, has transformed the treatment landscape for pediatric patients with relapsed or refractory B-cell acute lymphoblastic leukemia, leading to significant advancements in immunooncology.

Ongoing research is focused on enhancing the efficacy and safety of CAR T cells, as well as addressing challenges related to patient access to this innovative therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The potential of CAR T therapy for relapsed or refractory pediatric and young adult B-cell ALL. Forsberg, MH., Das, A., Saha, K., et al.[2020]

Chimeric antigen receptor (CAR)-T cell therapy, specifically targeting CD19, has shown promising results in treating non-Hodgkin lymphomas (NHLs) resistant to standard therapies, leading to FDA and EMA approvals for tisagenlecleucel and axicabtagene ciloleucel.

While CAR-T cell therapy can cause significant adverse events like cytokine release syndrome and neurological toxicity, these effects are manageable with proper medical support, highlighting the importance of trained teams in administering this novel treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

CAR-T Cell Therapy in Diffuse Large B Cell Lymphoma: Hype and Hope. Hopfinger, G., Jäger, U., Worel, N.[2020]

Brexucabtagene autoleucel is the first FDA-approved CAR T-cell therapy targeting CD19 for adults with relapsed and refractory B-cell acute lymphoblastic leukemia, marking a significant advancement in treatment options.

The approval was based on the outcomes of the Zuma-3 trial, which demonstrated the efficacy of this therapy in improving patient outcomes in a challenging-to-treat population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Approval of brexucabtagene autoleucel for adults with relapsed and refractory acute lymphocytic leukemia. Frey, NV.[2022]

In the phase 3 TOWER study, blinatumomab showed a significant overall survival benefit compared to standard-of-care chemotherapy in adults with relapsed or refractory Philadelphia chromosome-negative B-precursor acute lymphoblastic leukemia, involving 267 patients who received a median of 2 cycles of treatment.

After adjusting for treatment duration, blinatumomab had lower exposure-adjusted event rates for serious adverse events compared to standard chemotherapy, particularly for infections and cytopenias, indicating it is a well-tolerated treatment option despite a higher rate of cytokine-release syndrome.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Exposure-adjusted adverse events comparing blinatumomab with chemotherapy in advanced acute lymphoblastic leukemia. Stein, AS., Larson, RA., Schuh, AC., et al.[2020]

Citations

1. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/4838/533281/BEAM-201-for-the-Treatment-of-Relapsed-and-or

BEAM-201 for the Treatment of Relapsed and/or Refractory (R ... These initial data show a BEAM-201 safety profile consistent with underlying disease, lymphodepletion, and AEs associated with CAR-T therapy. PK ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06934382

Study Details | NCT06934382 | Anti-CD7 CAR-T Cells in ... 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 ...

3. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0006497124075967

BEAM-201 for the Treatment of Relapsed and/or Refractory ... Early evidence of efficacy with CAR-T-cell doses <200 million, as measured by CRi/CR in 2/3 Ps, is very encouraging. Updated data will be presented.

4. stanfordhealthcare.org stanfordhealthcare.org/trials/a/NCT05885464.html

A Study Evaluating the Safety and Efficacy of BEAM-201 in ... This is a Phase 1/2, multicenter, open-label study to evaluate the safety and efficacy of BEAM-201 in patients with relapsed/refractory T-ALL or T-LL. This ...

5. clin.larvol.com clin.larvol.com/trial-detail/NCT05885464

A Study Evaluating the Safety and Efficacy of BEAM-201 ... | "Key Highlights: Initial data as of a June 11, 2024, data cut in 3 patients treated with BEAM-201 show a safety profile consistent with underlying disease, ...

6. beamtx.com beamtx.com/wp-content/uploads/2024/12/BEAM-201-for-the-Treatment-of-Relapsed-andor-Refractory-RR-T-Cell-Acute-Lymphoblastic-Leukemia.pdf

BEAM-201 for the Treatment of Relapsed and/or Refractory ... or T-Cell Lymphoblastic Lymphoma (T-LL): Initial Data from the Phase (Ph) 1 ... • Initial data suggest a BEAM-201 safety profile similar to other.