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Multi-modular Chimeric Antigen Receptor T Cells tarGeting B7-H3 in Children, Teenage & Young Adult Sarcoma (MIGHTY)

3. august 2026 opdateret af: University College, London

MIGHTY is a multicentre, open label, phase 1 clinical trial of an Advanced Therapy Investigational Medicinal Product (ATIMP) in children, teenagers and young adults aged 1-24 with relapsed or refractory (r/r) rhabdomyosarcoma (RMS), Ewing sarcoma (ES) or desmoplastic small round cell tumour (DSRCT).

The study will assess the feasibility of generating the ATIMP and administering hBRCA84D CAR T cells to patients with r/r RMS, ES or DSRCT.

Studieoversigt

Status

Rekruttering

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

MIGHTY is a multicentre, open label, phase 1 clinical trial of an Advanced Therapy Investigational Medicinal Product (ATIMP) in children, teenagers and young adults aged 1-24 with relapsed or refractory (r/r) rhabdomyosarcoma (RMS), Ewing sarcoma (ES) or desmoplastic small round cell tumour (DSRCT).

The ATIMP for this study (hBRCA84D CAR T cells) are autologous T cells targeting B7-H3 in patients with r/r RMS, ES, or DSRCT.

Patients will undergo an unstimulated leucapheresis for the generation of the ATIMP which will take approximately 15 days to generate.

Patients will receive lymphodepleting (LD) chemotherapy with fludarabine administered over 4 days (Day -6 to Day -3) and cyclophosphamide administered over 2 days (Day -4 and Day-3).

Patients will be treated at one of three dose levels following LD chemotherapy as described above.

The study will evaluate the feasibility of generating the ATIMP, the safety of administering ATIMP, the tolerability of the ATIMP and how effectively the CAR T cells engraft, expand and persist following administration in patients with r/r RMS, ES or DSRCT.

Following infusion of the CAR T cells, 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 CAR T cells infusion.

If patients relapse within the first-year post CAR T cell infusion, they will come off the interventional follow up and will be followed up annually until 15 years after the CAR T infusion.

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 15 years post CAR T infusion.

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

12

Fase

  • Fase 1

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

  • Navn: Karin Straathof

Undersøgelse Kontakt Backup

Studiesteder

      • London, Det Forenede Kongerige
        • Rekruttering
        • Great Ormond Street Hospital
        • Kontakt:
          • Olga Slater
      • London, Det Forenede Kongerige
        • Rekruttering
        • University College London Hospital
        • Kontakt:
          • Sandra Strauss

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Barn
  • Voksen

Tager imod sunde frivillige

Ingen

Beskrivelse

Inclusion Criteria:

  1. Age ≥ 1 and ≤ 24 years.
  2. Tissue diagnosis of RMS, ES or DSRCT
  3. Expression of B7-H3 in the tumour
  4. Relapsed or refractory disease after one or multiple lines of previous treatment.
  5. Measurable disease by cross sectional imaging. Patients with only bone marrow detectable disease (bone marrow aspirate or trephine) are NOT eligible for the study.
  6. At least 3 weeks or 5 half-lives, whichever is shorter, after treatment with agents on other early phase clinical trial.
  7. Performance status: Karnofsky (age ≥ 10 years) or Lansky (age < 10) score ≥ 50%.
  8. Creatinine ≤1.5 x Upper Limit of Normal (ULN) for age, if higher, an estimated (calculated) creatinine clearance must be ≥ 60 ml/min/1.73 m2.
  9. Left ventricular ejection fraction (LVEF) ≥ 50%
  10. Absolute lymphocyte count ≥ 0.25 x 109/L.
  11. Women of childbearing potential (WOCBP) must have a negative pregnancy test and agree to comply with the pregnancy reporting requirements of the protocol (if applicable).
  12. Written informed consent.

Exclusion Criteria:

  1. Patients with only bone marrow detectable disease in the absence of measurable disease by cross sectional imaging.
  2. Patients with active, inoperative central nervous system (CNS) disease including leptomeningeal disease.
  3. Active hepatitis B, C or HIV infection.
  4. Inability to tolerate leukapheresis.
  5. 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.
  6. Any contraindication to lymphodepletion or to the use of Cyclophosphamide or Fludarabine as per the local SmPC.
  7. Any contraindication to the use of Anticoagulant Citrate Dextrose Solution.
  8. Known allergy to albumin, EDTA or DMSO.
  9. 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.
  10. Prior treatment with investigational or approved gene therapy or cell therapy products.
  11. Life expectancy <3 months.
  12. Systemic corticosteroid therapy ≥ 0.05 mg/kg dexamethasone daily (or equivalent) at time of hBRCA84D CAR T cells infusion.
  13. Women who are pregnant or breastfeeding.
  14. Patients who have received any live vaccine in the six weeks prior to planned lymphodepletion

Exclusion criteria for the ATIMP infusion:

  1. Uncontrolled fungal, bacterial, viral, or other infection. Previously diagnosed infection for which the patient continues to receive antimicrobial therapy is permitted if responding to treatment and clinically stable at the time of scheduled hBRCA84D CAR T cell infusion.
  2. Systemic corticosteroid therapy ≥ 0.05 mg/kg dexamethasone daily (or equivalent) at time of hBRCA84D CAR T cell infusion.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Interventionel model: Enkelt gruppeopgave
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: hBRCA84D CAR T cells
Treatment with hBRCA84D CAR T cells
The hBRCA84D CAR T cells target B7-H3 positive cells.

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Sikkerhed ved administration af ATIMP
Tidsramme: 28 dage
Forekomst af grad 3-5 toksicitet kausalt relateret til ATIMP, især alvorligt cytokinfrigivelsessyndrom og svær neurotoksicitet.
28 dage
Antal genererede terapeutiske produkter og antallet af ATIMP'er infunderet efter vellykket fremstilling
Tidsramme: 28 dage
Gennemførligheden af ​​generering af ATIMP som evalueret ved antallet af genererede terapeutiske produkter og antallet af ATIMP'er infunderet efter vellykket fremstilling.
28 dage

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Progressionsfri overlevelse (PFS)
Tidsramme: 1 år
Progressionsfri overlevelse (PFS) efter ATIMP intravenøs administration
1 år
Tid til fremskridt (TTP)
Tidsramme: 1 år
Time to Progression (TTP) efter ATIMP intravenøs administration
1 år
Samlet overlevelse
Tidsramme: 1 år
Samlet overlevelse efter ATIMP intravenøs administration
1 år
Objective response rate
Tidsramme: 1 year
Based on cross-sectional imaging after the ATIMP intravenous administration
1 year

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

23. juli 2025

Primær færdiggørelse (Anslået)

1. juli 2028

Studieafslutning (Anslået)

1. juli 2042

Datoer for studieregistrering

Først indsendt

3. august 2026

Først indsendt, der opfyldte QC-kriterier

3. august 2026

Først opslået (Faktiske)

7. august 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

7. august 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

3. august 2026

Sidst verificeret

1. august 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Andre undersøgelses-id-numre

  • UCL/150859

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

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