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CAR T-cell Therapy

Genetically Modified T-Cells for Brain Cancer (PNOC018 Trial)

Phase 1
Recruiting
Led By Sabine Mueller, MD, PhD, MAS
Research Sponsored by University of California, San Francisco
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Participants 3 to 21 years of age inclusive, at the time of signing the informed consent. The first two participants will be 12 years of age or older.
Male participants of impregnate potential must agree to use contraception, during the study and for at least 6 months after the last study intervention and refrain from donating sperm during this period.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 24 months
Awards & highlights
No Placebo-Only Group

PNOC018 Trial Summary

This trial is testing the safety of a new treatment for brain cancer made from modified immune cells. The treatment will be given to patients who have a specific gene mutation and have not responded to other treatments.

Who is the study for?
This trial is for HLA-A*0201-positive patients aged 3-21 with a specific brain tumor (H3.3K27M-mutated diffuse midline glioma) who've finished standard radiation therapy. They must not be pregnant, agree to use contraception, and have no immune disorders like HIV or hepatitis B/C. No prior treatments for the tumor are allowed.Check my eligibility
What is being tested?
The trial tests genetically modified KIND T cells after chemotherapy drugs cyclophosphamide and fludarabine in young patients with a certain type of brain cancer. It aims to find the safest dose and see how well these engineered T cells can target and fight the tumor.See study design
What are the potential side effects?
Potential side effects include reactions from the infusion of modified T cells, low blood cell counts due to chemotherapy, increased risk of infections, nausea, fatigue, and possible organ inflammation as an immune response.

PNOC018 Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am between 3 and 21 years old and can sign the consent form.
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I am a man who can father a child and agree to use birth control and not donate sperm for 6 months after the study ends.
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I agree to follow the study's birth control advice for at least 6 months after the last treatment.
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I have taken a pregnancy test in the last 14 days and it was negative.
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I have a specific brain tumor with a certain mutation and have finished standard radiation therapy.
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I tested positive for HLA-A*0201 from a certified lab.
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I agree to provide a sample of my tumor for research.
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I am not on steroids, or I am on a stable dose of dexamethasone (up to 4 mg/day).
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I haven't taken any steroids for at least 7 days.
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I have not had chemotherapy, immunotherapy, or a bone marrow transplant for my tumor.
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I started my standard radiation therapy within 6 weeks of my cancer diagnosis.
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My platelet count is at least 100,000/mm^3 without transfusions for 7 days.
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My kidney function is within the normal range for my age and gender.
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My child is between 3 and 6 years old with a creatinine level of <= 0.8 mg/dL.
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My child is between 6 and 10 years old with a creatinine level of <= 1.0 mg/dL.
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My creatinine level is at or below 1.2 mg/dL and I am aged 10 to less than 13 years.
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My kidney function, based on my age and gender, is within the required range.
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My age is 16 or older and my kidney function tests are within normal range.
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My bilirubin levels are within the normal range for my age.
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I do not have trouble breathing when I am resting.
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My seizures are well-managed with medication.
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I am mostly active and can care for myself.
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I can sign the consent form and follow the study rules.

PNOC018 Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 24 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Maximum tolerated dose (MTD)
Number of participants with treatment-emergent adverse events
Secondary outcome measures
Duration of KIND T cells in-vivo persistence
Percentage of participants who receive KIND T cells

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

PNOC018 Trial Design

1Treatment groups
Experimental Treatment
Group I: Treatment (KIND T cells, cyclophosphamide, fludarabine)Experimental Treatment3 Interventions
Patients receive fludarabine IV on days -4, -3, and -2 and cyclophosphamide IV on day -2 in the absence of disease progression or unacceptable toxicity for the conditioning regimen. Patients also receive KIND T cells IV at dose level 1 (2 x 106 dextramer®+ CD8+ cells/kg) on day 0. If no DLTs are reported, newly enrolling participants may receive dose level 2 of KIND T cells on day 0.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cyclophosphamide
1995
Completed Phase 3
~3780
Fludarabine
2012
Completed Phase 3
~1090

Find a Location

Who is running the clinical trial?

Alliance for Cancer Gene TherapyUNKNOWN
2 Previous Clinical Trials
27 Total Patients Enrolled
University of California, San FranciscoLead Sponsor
2,482 Previous Clinical Trials
12,164,312 Total Patients Enrolled
The V FoundationOTHER
6 Previous Clinical Trials
157 Total Patients Enrolled

Media Library

Autologous Anti-H3.3K27M TCR-expressing T-cells (CAR T-cell Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05478837 — Phase 1
Diffuse Midline Glioma Research Study Groups: Treatment (KIND T cells, cyclophosphamide, fludarabine)
Diffuse Midline Glioma Clinical Trial 2023: Autologous Anti-H3.3K27M TCR-expressing T-cells Highlights & Side Effects. Trial Name: NCT05478837 — Phase 1
Autologous Anti-H3.3K27M TCR-expressing T-cells (CAR T-cell Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05478837 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the eligibility requirements to partake in this research project?

"This clinical trial is seeking 12 individuals aged 3 to 21 with diffuse midline glioma and the h3 k27m mutation. Furthermore, candidates must meet certain criteria including testing positive for HLA-A*0201 (verified by CLIA or equivalent lab), consenting to tumor tissue analysis, being off systemic steroids or on a stable dose of dexamethasone at enrollment, etc. The first two participants will be over 12 years old; male patients of reproductive potential are expected to use contraception during treatment and refrain from donating sperm while female childbearing prospects should have had negative pregnancy tests prior to receiving study interventions."

Answered by AI

Are Autologous Anti-H3.3K27M TCR-expressing T-cells a viable option for patients with regards to safety?

"The safety of Autologous Anti-H3.3K27M TCR-expressing T-cells is estimated to be a 1 due to its Phase 1 status, indicating that there is only preliminary evidence regarding efficacy and scant data on safety."

Answered by AI

Could elderly individuals aged 60 and up qualify for this research?

"The conditions of this research program stipulate that the minimum age for enrollment is 3 and the maximum age must not exceed 21."

Answered by AI

Are there still opportunities to partake in this clinical research?

"According to clinicaltrials.gov, this trial is not currently enrolling patients. The experiment was first published on January 1st 2023 and the most recent update took place November 7th 2022. There are 357 other trials actively recruiting at present, however this particular study is inactive in terms of recruitment."

Answered by AI
~8 spots leftby Aug 2029