BPX-501 + Rimiducid for Leukemia

Not currently recruiting at 4 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Bellicum Pharmaceuticals

Disqualifiers: Acute GVHD, CNS involvement, Infections, HIV, Pregnancy, others

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

Compensation: Varies

Number of Visits: 3

Duration: 2 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment involving BPX-501 T cell infusions for adults with recurring blood cancers like leukemia, lymphoma, or multiple myeloma after a stem cell transplant. The trial seeks to determine the optimal dose of BPX-501 to aid patients while also evaluating Rimiducid, a drug used to manage potential side effects such as graft-versus-host disease, where donor cells attack the patient's body. Individuals with recurring blood cancer or minimal residual disease after a stem cell transplant might be suitable candidates for this study. As a Phase 1 trial, this research focuses on understanding the treatment's effects in people, offering participants the chance to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that BPX-501 and Rimiducid are likely to be safe?

Research has shown that BPX-501 T cell infusions are generally safe and well-tolerated. In earlier studies, most patients experienced only mild skin issues, such as acute graft-versus-host disease (aGvHD), where donor cells attack the recipient’s body. This condition was manageable and did not lead to serious problems.

For Rimiducid, studies have demonstrated its effectiveness in controlling side effects. It acts as a "safety switch" by reducing problematic cells, such as T cells, when necessary. In one study, Rimiducid removed 85% to 95% of certain T cells within 30 minutes, helping to manage conditions like aGvHD without recurrence.

Overall, both treatments have shown promising safety in earlier research, suggesting they are well-tolerated with manageable side effects in previous patients. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about BPX-501 for leukemia because it uses a novel approach by infusing modified T cells to enhance the immune response after a stem cell transplant. Unlike traditional chemotherapy or radiation, which target cancer cells directly, BPX-501 aims to boost the body's own immune system to fight the disease. Additionally, Rimiducid is explored as a safety switch, potentially allowing for better control of graft-versus-host disease, a common complication after transplants. This combination could offer a more targeted and potentially safer treatment option for leukemia patients.

What evidence suggests that BPX-501 and Rimiducid could be effective for leukemia?

Research has shown that BPX-501 T cell infusions may benefit patients with blood cancers such as leukemia. In one study, 90% of patients achieved complete remission, and 92.7% experienced no major issues afterward. Another study found that patients' immune systems recovered quickly within six months of treatment. In this trial, participants will receive BPX-501 along with Rimiducid, which manages side effects by activating a "safety switch" to reduce graft-versus-host disease (GvHD), a common issue. Together, these treatments aim to fight cancer while controlling side effects. ^{2 3 4 6 7}

Who Is on the Research Team?

Bellicum Pharmaceuticals

Principal Investigator

Bellicum Pharmaceuticals, Inc.

Are You a Good Fit for This Trial?

Adults aged 18-65 with certain blood cancers like leukemia, lymphoma, or myeloma who've had a stem cell transplant but are seeing their cancer return. They need to be relatively healthy otherwise, with good heart, liver, kidney and lung function and no severe complications from the transplant.

Inclusion Criteria

You are expected to live for more than 10 weeks.

My cancer came back more than 100 days after a stem cell transplant or I have minimal residual disease more than 30 days after.

I have a blood cancer that may require a stem cell transplant.

Exclusion Criteria

You have tested positive for HIV.

I do not have an ongoing infection that is getting worse despite treatment.

My cancer has spread to my brain or spinal cord.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 3 cycles of BPX-501 T cell infusions at escalating dose levels, with Rimiducid investigated for aGvHD treatment

2 months

3 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessment of GvHD and response rates

24 months

What Are the Treatments Tested in This Trial?

Interventions

• BPX-501
• Rimiducid

Trial Overview The trial is testing BPX-501 T cells given in increasing doses to see if they can fight the cancer after a stem cell transplant. It's also looking at Rimiducid's ability to control graft versus host disease without hurting the anti-cancer effects of the T cells.

How Is the Trial Designed?

Group I: BPX-501 and Rimiducid Experimental Treatment 2 Interventions

All subjects will receive 3 cycles of BPX-501 T cell infusions at escalating dose levels (DL). DL1 on Day 0, DL2 on Days 30 and 60. The first dose of BPX-501 T cells will occur ≥30 days after hematopoietic stem cell transplant (HSCT). Two doses of Rimiducid ( 0.1 mg/kg and 0.4 mg/kg) will be investigated for the treatment of aGvHD after BPX-501 T cell infusion.

Who Is Running the Clinical Trial?

Bellicum Pharmaceuticals

Lead Sponsor

Trials

28

Recruited

1,400+

Published Research Related to This Trial

In a study of 48 patients with relapsed or refractory acute lymphoblastic leukemia (ALL), the combination of inotuzumab and low-intensity mini-HCVD chemotherapy resulted in a high response rate of 92%, with 73% achieving complete remission.

The treatment showed promising long-term outcomes, with a median overall survival of 25 months and a 2-year overall survival rate of 54%, indicating that this combination therapy may be more effective than traditional intensive chemotherapy options.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Chemoimmunotherapy with inotuzumab ozogamicin combined with mini-hyper-CVD, with or without blinatumomab, is highly effective in patients with Philadelphia chromosome-negative acute lymphoblastic leukemia in first salvage. Jabbour, E., Sasaki, K., Ravandi, F., et al.[2020]

Brexucabtagene autoleucel (brexu-cel) is an FDA-approved CAR T-cell therapy for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (B-ALL), showing a complete remission (CR) rate of 52% within 3 months after treatment in a study of 54 patients.

While brexu-cel demonstrates significant efficacy, it is associated with serious adverse reactions in 79% of patients, including severe cytokine release syndrome (92%) and neurological toxicities (87%), necessitating a risk evaluation and mitigation strategy (REMS) for patient safety.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

FDA Approval Summary: Brexucabtagene Autoleucel for Treatment of Adults With Relapsed or Refractory B-Cell Precursor Acute Lymphoblastic Leukemia. Bouchkouj, N., Lin, X., Wang, X., et al.[2022]

A 5-year-old boy with high-risk B-acute lymphoblastic leukemia (B-ALL) achieved minimal residual disease-negative remission after receiving a novel treatment regimen combining chemotherapy and immunotherapy, which included rituximab, inotuzumab ozogamicin, and blinatumomab.

The treatment was well tolerated, leading to a successful transplant without significant infections or toxicities, marking the first reported use of this condensed sequential therapy in a pediatric leukemia patient.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mini-hyper CVD + CRIB (condensed rituximab, inotuzumab ozogamicin, and blinatumomab) for refractory pediatric B-acute lymphoblastic leukemia. McCall, D., Jabbour, E., Roth, M., et al.[2022]

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10336773/

BPX-501 T-cells interfere with minimal residual disease ... CD19 chimeric antigen receptor T cell therapies are highly effective against B-cell acute lymphoblastic leukemia. Of the few patients who ...

2. ir.bellicum.com ir.bellicum.com/news-releases/news-release-details/bellicum-announces-interim-results-showing-low-rates-cancer

Press Release Details - Bellicum Pharmaceuticals Of 59 pediatric PID patients undergoing a haplo-HSCT and treatment with BPX-501, disease-free survival was reported at 88.1% and overall ...

3. astctjournal.org astctjournal.org/article/S1083-8791(18)30902-9/fulltext

Administration of BPX-501 Cells Following Αβ T and B- ... The best overall response rate (CR/PR) was 100% with 9 pts (90%) achieving CR. EFS was 92.7%. At a median f/u of 17.6 mos, 5 pts (3.3%) experienced TRM. DFS and ...

4. ashpublications.org ashpublications.org/blood/article/126/23/1931/105323/Clinical-Outcome-after-Adoptive-Infusion-of-BPX

Clinical Outcome after Adoptive Infusion of BPX-501 Cells ... Results: BPX-501 cells were infused at a median time of 16 days (range 13-18); median cell viability post-thaw was 91% (range 65-97). Treatment ...

5. firstwordpharma.com firstwordpharma.com/story/4315686

Bellicum Presents Updated Clinical Results at 2017 BMT ... Patients receiving BPX-501 following a haplo-HSCT demonstrated rapid immune reconstitution by month six, including full recovery and normalization of T-cells, B ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT01744223

Safety Study of Gene Modified Donor T-cells Following ... This is a Phase1/2 dose escalation study evaluating the safety and feasibility of BPX-501 infused after partially mismatched, related (haploidentical), T cell- ...

7. sciencedirect.com sciencedirect.com/science/article/pii/S0006497118489100

Clinical Outcome after Adoptive Infusion of BPX-501 Cells ... Conclusions: Overall, these data indicate that the infusion of BPX-501 cells is safe and well tolerated. The 100-day CI of skin-only grade I-II acute GvHD ...