PHOX2B PC-CAR T Cells for Neuroblastoma
(PHOX2B Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to determine if PHOX2B PC-CAR T cells, a type of immunotherapy, are safe for individuals with advanced, high-risk neuroblastoma, a cancer typically affecting children. Researchers will begin by testing various doses to assess patient tolerance. If deemed safe, they will enroll more participants to evaluate the treatment's effectiveness. The trial seeks individuals with high-risk neuroblastoma that has not responded to other treatments and have measurable disease, meaning the cancer is visible on scans or biopsies. As a Phase 1 trial, the research focuses on understanding how the treatment works 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 does not specify if you need to stop taking your current medications. However, you cannot use systemic steroids or immunosuppressants at the time of cell infusion or collection, unless they are for disease treatment at other times or are inhaled or physiologic replacement steroids.
Is there any evidence suggesting that PHOX2B PC-CAR T cells are likely to be safe for humans?
Research has shown that PHOX2B PC-CAR T cells have been promising in lab studies, successfully targeting and destroying neuroblastoma cells. However, as this is an early trial in humans, detailed safety information from patients is not yet available.
This treatment is currently in a phase 1 trial, which primarily focuses on safety. At this stage, researchers aim to assess how well people can tolerate the treatment and identify any possible side effects. Since this is the first time the treatment is tested in humans, safety information remains limited. Participants in this trial will help gather crucial data on how the treatment functions in the human body.12345Why do researchers think this study treatment might be promising for neuroblastoma?
Researchers are excited about PHOX2B PC-CAR T cells for neuroblastoma because this treatment uses a cutting-edge approach that differs from traditional therapies like chemotherapy, radiation, or surgery. Unlike these standard options, which target cancer cells broadly, PHOX2B PC-CAR T cells are engineered to specifically recognize and attack neuroblastoma cells by targeting the PHOX2B protein, which is often present in these cancer cells. This precision targeting could lead to more effective and potentially less harmful treatments, as the immune system is harnessed to fight the cancer directly. Additionally, this approach has the potential to offer new hope for patients, especially those whose cancer hasn't responded well to existing treatments.
What evidence suggests that PHOX2B PC-CAR T Cells might be an effective treatment for neuroblastoma?
Research has shown that PHOX2B PC-CAR T cells may effectively treat neuroblastoma, a type of cancer often found in children. Studies have demonstrated that these cells can specifically target and destroy neuroblastoma cells in lab tests. In some experiments, these cells made tumors disappear completely. This occurs because they are designed to recognize a specific marker on the cancer cells, allowing them to attack the tumor directly. In this trial, participants will enroll in either a dose escalation arm to determine the maximum tolerated dose or a dose expansion arm to evaluate the response rate and further characterize the safety profile of PHOX2B PC-CAR T cells. While these results are promising, more research is needed to confirm these effects in humans.12345
Who Is on the Research Team?
Jacquelyn Crane, MD
Principal Investigator
Children's Hospital of Philadelphia
Stephan A. Grupp, MD, PhD
Principal Investigator
Children's Hospital of Philadelphia
Are You a Good Fit for This Trial?
This trial is for patients with advanced, high-risk neuroblastoma. Specific eligibility criteria are not provided, but typically include factors like age, health status, and previous treatments.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Dose Escalation
Determine the maximum tolerated dose of PHOX2B PC-CAR T cells using a standard 3+3 trial design
Dose Expansion
Evaluate the rate of response to PHOX2B-PC CAR T cells and further characterize the safety profile
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- PHOX2B PC-CAR T Cells
Find a Clinic Near You
Who Is Running the Clinical Trial?
Stephan Grupp MD PhD
Lead Sponsor
Alliance for Cancer Gene Therapy
Collaborator
Children's Cancer Research Fund
Collaborator
Children's Hospital of Philadelphia
Collaborator