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New treatment option for Neuroblastoma
Official title PEACH TRIAL- Precision Medicine and Adoptive Cellular Therapy
ClinicalTrials.gov ID: NCT04837547
What this study is testing
What is Tumor-specific ex vivo expanded autologous lymphocyte transfer (TTRNA-xALT)?
Tumor-specific ex vivo expanded autologous lymphocyte transfer (TTRNA-xALT) is an investigational medicine, being studied as a potential treatment for neuroblastoma.
Also referred to as xALT.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- A Phase I open-label, multicenter study, to evaluate the safety, feasibility, and maximum tolerated dose (MTD) of treating children with newly diagnosed DIPG or recurrent neuroblastoma with molecular targeted therapy in combination with adoptive cell therapy (Total tumor mRNA-pulsed autologous Dendritic Cells (DCs) (TTRNA-DCs), Tumor-specific ex vivo expanded autologous lymphocyte transfer (TTRNA-xALT) and Autologous G-CSF mobilized Hematopoietic Stem Cells (HSCs)).
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 30
You may be able to join if
- people must have proven pediatric cancer with confirmation at diagnosis or at the time of recurrence/progression and clinical determination of...
- Disease Status: High Risk Neuroblastoma-
- Patients that have relapsed following standard of care therapy or having progressed during standard of care therapy and non-responsive/progressive to...
- Neuroblastoma must be age \>12 months at enrollment Diffuse Intrinsic Pontine (or other brain stem) Glioma
- Newly-diagnosed patients willing to undergo biopsy
You likely can't join if
- Absence of tumor on biopsy specimen or a diagnosis other than NBL or glioma on biopsy
- Known autoimmune or immunosuppressive disease or human immunodeficiency virus infection.
- people with significant renal, cardiac, pulmonary, hepatic or other organ dysfunction.
- Prior allergic reaction to GM-CSF or Td.
- people who have received any cytotoxic chemotherapy within the last 7 days prior to biopsy or focal radiotherapy in the case of patients with diffuse...
- people with NBL who have received any radiotherapy to the primary sample site within the last 14 days (radiation may be included in treatment...
See the full eligibility criteria
- people must have proven pediatric cancer with confirmation at diagnosis or at the time of recurrence/progression and clinical determination of disease for which there is no known effective curative therapy or disease...
- Disease Status: High Risk Neuroblastoma-
- Patients that have relapsed following standard of care therapy or having progressed during standard of care therapy and non-responsive/progressive to accepted curative chemotherapy.
- Neuroblastoma must be age \>12 months at enrollment Diffuse Intrinsic Pontine (or other brain stem) Glioma
- Newly-diagnosed patients willing to undergo biopsy
- Must be within 2 months of diagnosis and prior to starting radiation
- DIPG must be ≥ 3 years of age at enrollment
- All people must be age ≤ 30 years at enrollment
- Patient and/or parents/guardian willing to consent to biopsy for obtaining tumor material for confirmatory diagnosis and/or tumor RNA extraction and amplification.
- people must have measurable disease as defined Per section 8 at the time of biopsy and tumor or bone marrow must be accessible for biopsy. Tumor or bone marrow samples submitted for analysis must contain \>20% viable...
- Current disease state must be one for which there is currently no known effective therapy
- Specimens will be obtained only in a non-significant risk manner and not solely for the purpose of investigational testing.
- Lansky or Karnofsky Score must be ≥ 60
- Bone Marrow:
- ANC (Absolute neutrophil count) ≥ 1000/µl (unsupported- \>24 hrs off G-CSF and 7 days off neulasta)
- Platelets ≥ 100,000/µl (can be transfused)
- Hemoglobin \> 8 g/dL (can be transfused)
- Renal: Serum creatinine ≤ upper limit of institutional normal.
- Adequate liver function must be demonstrated, defined as:
- Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND
- ALT (SGPT) ≤ 3 times upper limit of normal (ULN) for age
- AST (SGOT) ≤ 3 times upper limit of normal (ULN) for age.
- people with CNS disease currently taking steroids must have been on a stable dose of steroids for at least one week prior to their biopsy and must not have progressive hydrocephalus at enrollment.
- A negative serum pregnancy test is required for female participants of childbearing potential (≥13 years of age or after onset of menses)
- Both male and female post-pubertal study people need to agree to use one of the more effective birth control methods during treatment and for six months after treatment is stopped. These methods include total abstinence...
- Informed Consent: All people and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines
- Post-Biopsy: Patients with post-biopsy neurological deficits should have deficits that are stable for a minimum of 1 week prior to registration.
- Absence of tumor on biopsy specimen or a diagnosis other than NBL or glioma on biopsy
- Known autoimmune or immunosuppressive disease or human immunodeficiency virus infection.
- people with significant renal, cardiac, pulmonary, hepatic or other organ dysfunction.
- Prior allergic reaction to GM-CSF or Td.
- people who have received any cytotoxic chemotherapy within the last 7 days prior to biopsy or focal radiotherapy in the case of patients with diffuse intrinsic pontine (or other brain stem) gliomas
- people with NBL who have received any radiotherapy to the primary sample site within the last 14 days (radiation may be included in treatment decision after biopsy).
- people receiving any investigational drug concurrently.
- people with uncontrolled serious infections or a life-threatening illness (unrelated to tumor)
- people with any other medical condition, including malabsorption syndromes, mental illness or substance abuse, deemed by the Investigator to be likely to interfere with the interpretation of the results or which would...
The study team makes the final eligibility decision.
Where it's taking place
- Gainesville, Florida, United States
- Charlotte, North Carolina, United States
- Hershey, Pennsylvania, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 year to 30 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Gainesville, Florida, United States; Charlotte, North Carolina, United States; Hershey, Pennsylvania, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.