BPX-501 + Rimiducid for Leukemia

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

10 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial tests a treatment for adults with certain blood cancers after a transplant. The treatment aims to fight cancer and infections while controlling side effects with another drug.

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.

What makes the BPX-501 + Rimiducid treatment unique for leukemia?

BPX-501 + Rimiducid is a novel treatment approach that involves using genetically modified T-cells (a type of immune cell) to target leukemia cells, with Rimiducid acting as a safety switch to control the activity of these T-cells. This approach is different from traditional chemotherapy or other immunotherapies because it allows for precise control over the immune response, potentially reducing side effects and improving safety. ^{1 2 3 4 5}

Research Team

Bellicum Pharmaceuticals

Principal Investigator

Bellicum Pharmaceuticals, Inc.

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Mini-hyper CVD + CRIB (condensed rituximab, inotuzumab ozogamicin, and blinatumomab) for refractory pediatric B-acute lymphoblastic leukemia. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

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

3. England pubmed.ncbi.nlm.nih.gov

VANDA regimen followed by blinatumomab leads to favourable outcome in patients with Philadelphia chromosome-negative B-precursor acute lymphoblastic leukaemia in first relapse. [2022]

4. United States 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. [2020]

5. England pubmed.ncbi.nlm.nih.gov

FDA Approval Summary: Brexucabtagene Autoleucel for Treatment of Adults With Relapsed or Refractory B-Cell Precursor Acute Lymphoblastic Leukemia. [2022]