- ICH GCP
- US Clinical Trials Registry
- Klinisk utprøving NCT07751380
Multi-modular Chimeric Antigen Receptor T Cells tarGeting B7-H3 in Children, Teenage & Young Adult Sarcoma (MIGHTY)
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.
Studieoversikt
Status
Forhold
Intervensjon / Behandling
Detaljert 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.
Studietype
Registrering (Antatt)
Fase
- Fase 1
Kontakter og plasseringer
Studiekontakt
- Navn: Karin Straathof
Studer Kontakt Backup
- Navn: MIGHTY Trial Coordinator
- Telefonnummer: +4420 7679 9852
- E-post: ctc.mighty@ucl.ac.uk
Studiesteder
-
-
-
London, Storbritannia
- Rekruttering
- Great Ormond Street Hospital
-
Ta kontakt med:
- Olga Slater
-
London, Storbritannia
- Rekruttering
- University College London Hospital
-
Ta kontakt med:
- Sandra Strauss
-
-
Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
- Barn
- Voksen
Tar imot friske frivillige
Beskrivelse
Inclusion Criteria:
- Age ≥ 1 and ≤ 24 years.
- Tissue diagnosis of RMS, ES or DSRCT
- Expression of B7-H3 in the tumour
- Relapsed or refractory disease after one or multiple lines of previous treatment.
- Measurable disease by cross sectional imaging. Patients with only bone marrow detectable disease (bone marrow aspirate or trephine) are NOT eligible for the study.
- 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 ≥ 50%.
- 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.
- Left ventricular ejection fraction (LVEF) ≥ 50%
- Absolute lymphocyte count ≥ 0.25 x 109/L.
- 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).
- Written informed consent.
Exclusion Criteria:
- Patients with only bone marrow detectable disease in the absence of measurable disease by cross sectional imaging.
- Patients with active, inoperative central nervous system (CNS) disease including leptomeningeal disease.
- Active hepatitis B, C or HIV infection.
- Inability to tolerate leukapheresis.
- 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.
- Known allergy to albumin, EDTA or DMSO.
- 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.
- Systemic corticosteroid therapy ≥ 0.05 mg/kg dexamethasone daily (or equivalent) at time of hBRCA84D CAR T cells infusion.
- Women who are pregnant or breastfeeding.
- Patients who have received any live vaccine in the six weeks prior to planned lymphodepletion
Exclusion criteria for the ATIMP infusion:
- 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.
- Systemic corticosteroid therapy ≥ 0.05 mg/kg dexamethasone daily (or equivalent) at time of hBRCA84D CAR T cell infusion.
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
|
Eksperimentell: hBRCA84D CAR T cells
Treatment with hBRCA84D CAR T cells
|
The hBRCA84D CAR T cells target B7-H3 positive cells.
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Sikkerhet ved administrering av ATIMP
Tidsramme: 28 dager
|
Forekomst av grad 3-5 toksisitet kausalt relatert til ATIMP, spesielt alvorlig cytokinfrigjøringssyndrom og alvorlig nevrotoksisitet.
|
28 dager
|
|
Antall genererte terapeutiske produkter og antall ATIMP-er infundert etter vellykket produksjon
Tidsramme: 28 dager
|
Gjennomførbarheten av generering av ATIMP som evaluert av antall genererte terapeutiske produkter og antall ATIMPer infundert etter vellykket produksjon.
|
28 dager
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Progresjonsfri overlevelse (PFS)
Tidsramme: 1 år
|
Progresjonsfri overlevelse (PFS) etter ATIMP intravenøs administrering
|
1 år
|
|
Tid til progresjon (TTP)
Tidsramme: 1 år
|
Tid til progresjon (TTP) etter intravenøs administrering av ATIMP
|
1 år
|
|
Samlet overlevelse
Tidsramme: 1 år
|
Total overlevelse etter ATIMP intravenøs administrering
|
1 år
|
|
Objective response rate
Tidsramme: 1 year
|
Based on cross-sectional imaging after the ATIMP intravenous administration
|
1 year
|
Samarbeidspartnere og etterforskere
Sponsor
Studierekorddatoer
Studer hoveddatoer
Studiestart (Faktiske)
Primær fullføring (Antatt)
Studiet fullført (Antatt)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- UCL/150859
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