Who may be eligible for the CAR T Cells to Target GD2 for DMG trial?

Inclusion Criteria: 1. Age ≥ 2 and ≤ 16 years 2. Tissue diagnosis of H3K27M mutant Diffuse Midline Glioma. 3. Radiographically evident tumour restricted to the brain stem or spinal cord. 4. At least 6 weeks following completion of radiation therapy. 5. At least 3 weeks or 5 half-lives, whichever is shorter, after treatment with agents on other early phase clinical trial 6. Performance status: Karnofsky (age ≥ 10 years) or Lansky (age \< 10) score ≥ 40% allowing for stable neurological deficit due to DMG 7. Absolute neutrophil count ≥1.5 x109/L and platelet count ≥ 100 x109/L 8. Total bilirubin \< 1.5 ULN and ALT \< 2.5 ULN 9. Serum creatine \< 1.5 ULN for age. 10. For post-pubertal subjects agreement to have a pregnancy test, use adequate contraception (if applicable) 11. Written informed consent Exclusion Criteria: 1. Systemic corticosteroid therapy ≥ 0.05 mg/kg dexamethasone daily (or equivalent) at time of RQR8/huK28Z CAR T cell infusion 2. Tumour involvement of the thalamus or supratentorial lesions, cerebellar vermis or hemispheres (pontocerebellar peduncle involvement is allowed) 3. Clinical or radiological evidence of true tumour progression 4. Active hepatitis B, C or HIV infection 5. Inability to tolerate leukapheresis 6. Pre-existing significant neurological disorder not related to DMG 7. Clinically significant systemic illness or medical condition (e.g., significant cardiac, pulmonary, hepatic or other organ dysfunction), that in the judgement of the investigator is likely to interfere with assessment of safety or efficacy of the investigational regimen and its requirements. 8. Any contraindication to lymphodepletion or to the use of Cyclophosphamide or Fludarabine as per the local SmPC 9. Any contraindication to the use of Anticoagulant Citrate Dextrose Solution 10. Any contraindication to Ommaya reservoir insertion (or similar catheter) 11. Known allergy to albumin, DMSO or EDTA 12. Primary immunodeficiency or history of autoimmune disease (e.g., Crohn's, rheumatoid arthritis, systemic lupus) requiring systemic immunosuppression /systemic disease modifying agents within the last 2 years 13. Prior treatment with investigational or approved gene therapy or cell therapy products 14. Life expectancy \<3 months 15. Use of rituximab (or rituximab biosimilar) within the last 3 months prior to RQR8/huK28Z CAR T cell infusion 16. Post-pubertal subjects who are pregnant or breastfeeding

ClinicalTrials.gov record NCT05544526

CAR T Cells to Target GD2 for DMG

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

Chimeric Antigen Receptor (CAR)-T Cells to Target GD2 for Diffuse Midline Glioma

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.

Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.

PhasePhase 1An early-stage study, usually small, mainly to check safety, side effects and how much of a treatment can be given. Whether it works is not the main question yet.

AgeUp to 16 years old

SexAll

LocationUnited Kingdom

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is a Phase I, open-label study testing autologous GD2 CAR T-cells in children and young people (ages 2–16) with H3 K27M diffuse midline glioma. Participants give blood for manufacturing, have an intraventricular catheter placed, receive short-course lymphodepletion chemotherapy, then an intravenous GD2 CAR T-cell infusion at one of three dose levels. Patients stay in hospital for 2–4 weeks after infusion and are followed for one year, with longer-term follow-up thereafter.

What it is studying. The study is testing how feasible it is to make the patient’s own GD2 CAR T-cell product and whether giving those cells is safe and tolerable in children and young adults with H3 K27M diffuse midline glioma. It will measure side effects (including severe cytokine release syndrome and neurotoxicity), and how well the infused CAR T-cells engraft, expand and persist in the body. The protocol includes three intravenous dose levels; patients without a sufficient response by Day 28 may be eligible for a later intraventricular CD19 CAR T-cell dose per the study plan.

What the researchers hope to learn. Study goals are to evaluate feasibility of manufacturing the GD2 CAR T-cell product, to measure safety and tolerability (including serious immune and neurological toxicities), and to assess CAR T-cell engraftment, expansion and persistence. The study will also collect early measures of disease outcomes (overall survival, progression-free survival, time to progression, and objective response) up to one year.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

The CARMIGO Trial is a single-centre, non-randomised, open label Phase I clinical trial of an Advanced Therapy Investigational Medicinal Product (ATIMP) in children and young adults aged 2-16 years with Diffuse Midline Glioma (DMG). The study will evaluate the feasibility of generating the ATIMP, the safety and tolerability of the GD2CAR T-cell therapy and how effectively GD2CAR T-cells engraft, expand and persist following administration in patients with DMG.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Biological: GD2 CAR T cells

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

Age ≥ 2 and ≤ 16 years
Tissue diagnosis of H3K27M mutant Diffuse Midline Glioma.
Radiographically evident tumour restricted to the brain stem or spinal cord.
At least 6 weeks following completion of radiation therapy.
At least 3 weeks or 5 half-lives, whichever is shorter, after treatment with agents on other early phase clinical trial
Performance status: Karnofsky (age ≥ 10 years) or Lansky (age < 10) score ≥ 40% allowing for stable neurological deficit due to DMG
Absolute neutrophil count ≥1.5 x109/L and platelet count ≥ 100 x109/L
Total bilirubin < 1.5 ULN and ALT < 2.5 ULN
Serum creatine < 1.5 ULN for age.
For post-pubertal subjects agreement to have a pregnancy test, use adequate contraception (if applicable)
Written informed consent

Exclusion criteria

Systemic corticosteroid therapy ≥ 0.05 mg/kg dexamethasone daily (or equivalent) at time of RQR8/huK28Z CAR T cell infusion
Tumour involvement of the thalamus or supratentorial lesions, cerebellar vermis or hemispheres (pontocerebellar peduncle involvement is allowed)
Clinical or radiological evidence of true tumour progression
Active hepatitis B, C or HIV infection
Inability to tolerate leukapheresis
Pre-existing significant neurological disorder not related to DMG
Clinically significant systemic illness or medical condition (e.g., significant cardiac, pulmonary, hepatic or other organ dysfunction), that in the judgement of the investigator is likely to interfere with assessment of safety or efficacy of the investigational regimen and its requirements.
Any contraindication to lymphodepletion or to the use of Cyclophosphamide or Fludarabine as per the local SmPC
Any contraindication to the use of Anticoagulant Citrate Dextrose Solution
Any contraindication to Ommaya reservoir insertion (or similar catheter)
Known allergy to albumin, DMSO or EDTA
Primary immunodeficiency or history of autoimmune disease (e.g., Crohn's, rheumatoid arthritis, systemic lupus) requiring systemic immunosuppression /systemic disease modifying agents within the last 2 years
Prior treatment with investigational or approved gene therapy or cell therapy products
Life expectancy <3 months
Use of rituximab (or rituximab biosimilar) within the last 3 months prior to RQR8/huK28Z CAR T cell infusion
Post-pubertal subjects who are pregnant or breastfeeding

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry

The CARMIGO Trial is a single-centre, non-randomised, open label Phase I clinical trial of an Advanced Therapy Investigational Medicinal Product (ATIMP) in children and young adults aged 2-16 years with Diffuse Midline Glioma (DMG). The ATIMP for this study is cryopreserved autologous patient-derived T-cells transduced with GD2CAR vector to generate GD2CAR T-cells. Patients will undergo an unstimulated leucapheresis for the generation of the ATIMP which will take approximately 15 days to generate. Patients will have an intraventricular catheter (Ommaya catheter) placed following enrolment and prior to GD2 CAR T cell infusion to allow monitoring, and treatment if necessary, of increased intracranial pressure (ICP). Patients will receive lymphodepleting (LD) chemotherapy with fludarabine 30mg/m2 administered over 4 days (Day -6 to Day -3) and cyclophosphamide 60mg/kg administered over 2 days (Day -4 and Day-3). All patients will be treated on Theme 1 of the study (intravenous CAR T administration) at one of the three dose levels (Dose Level 1: 3 x10\^7 GD2 CAR T cells/m2; Dose Level 2: 10 x10\^7 GD2 CAR T cells/m2; Dose Level 3: 30 x10\^7 GD2 CAR T cells/m2) following LD chemotherapy as described above. Patients with no/partial response at Day 28 (or disease progression after initial CR beyond Day 28) and in the absence of severe/persisting toxicity related to the ATIMP, will be potentially eligible for Theme 2 of the study where they can receive Dose 2, a single dose of 30 x 10\^6 CD19CAR T-cells intraventricularly via an Ommaya reservoir following LD chemotherapy as described above. The study will evaluate the feasibility of generating the ATIMP, the safety of administering GD2CAR T-cell therapy, the tolerability of the GD2CAR T cell in patients and how effectively GD2CAR T-cells engraft, expand and persist following administration in patients with DMG. Following infusion of GD2CAR T-cell therapy patients will be monitored for between 2-4 weeks as an inpatient. Following discharge, patients will enter the interventional follow up phase and be followed up for 1 year. Patients will be seen at 6 weeks post infusion then 3 monthly until 1 year post GD2CAR T-cell infusion. If patients relapse within the first year post last GD2CAR T-cell infusion they will come off the interventional follow up and will be followed up annually until the end of trial is declared. After completing the 1 year interventional phase of the study all patients, irrespective of whether they progressed or responded to treatment, will enter long term follow up until the end of trial is declared.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participation includes an unstimulated leukapheresis (blood cell collection) so your own T cells can be manufactured into GD2 CAR T cells (manufacture takes ~15 days). An intraventricular catheter (Ommaya reservoir) will be placed, you will receive short lymphodepleting chemotherapy, then an intravenous infusion of GD2 CAR T cells; some patients who meet criteria later may receive a single intraventricular CD19 CAR T dose via the Ommaya. You will be monitored in hospital for about 2–4 weeks after infusion, then seen at 6 weeks and every 3 months up to 1 year, with longer-term follow-up thereafter. The study involves close safety monitoring for serious immune or neurological side effects, routine blood tests and scans as part of follow-up.

How the study is structured. Single-centre, non-randomised, open-label Phase I interventional trial. All participants receive leukapheresis to make a cryopreserved, autologous GD2 CAR T-cell product, short lymphodepletion chemotherapy, then an intravenous CAR T infusion at one of three escalating dose levels. Patients are monitored in hospital for 2–4 weeks after infusion, then seen at 6 weeks and every 3 months up to 1 year, with long-term follow-up thereafter.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

The registry lists this study under Diffuse Midline Glioma, H3 K27M-Mutant. Does it apply to my diagnosis?
This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
The listed sites are in United Kingdom. Is any of them reachable for me, and does the study help with travel?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on Gaucher disease

Clinical trials64 trials listed for Gaucher diseaseEvery trial the registry lists for Gaucher disease, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Gaucher disease researchLatest monthly update: September 2026. Includes: Platelet release. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: Aug 15, 2023
Primary completion: August 2027 (estimated)
Study completion: August 2042 (estimated)
Target enrollment: 12 participants (estimated)
Last updated on registry: Aug 11, 2026
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: University College, London

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

CARMIGO Trial Coordinator 📞 0207 679 9599 ✉ ctc.CARMIGO@ucl.ac.uk
Karin Straathof

Participating sites (1)

Great Ormond Street HospitalRecruiting
London · United Kingdom
- Darren Hargrave

Terms used on this page · explanations by MedicalUpdates.ai

randomised: Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
open label: No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
Investigational: A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
5 half-lives: The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
Performance status: A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
Karnofsky: A scale from 100 (no complaints) down to 0 that describes how well a person can carry out daily activities. Studies often require a minimum score.
Absolute neutrophil count: The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
ULN: The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
Written informed consent: The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
corticosteroid: Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
CAR T: A treatment in which a person's own immune cells (T cells) are collected, modified in a laboratory to recognise a target, and returned to the body.
contraindication: A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
Prior treatment: Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
Life expectancy: Some studies require that, in the investigator's judgement, a participant is likely to live long enough to complete the study period. It is a study requirement, not a prediction about any individual.
Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
intraventricular: Given directly into the fluid-filled spaces (ventricles) inside the brain, usually through an implanted catheter or reservoir.
enrolment: The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
follow up: The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
relapse: A condition that improved with treatment and has since returned or worsened.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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