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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.
Studie Overzicht
Toestand
Conditie
Interventie / Behandeling
Gedetailleerde beschrijving
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
Inschrijving (Geschat)
Fase
- Fase 1
Contacten en locaties
Studiecontact
- Naam: Karin Straathof
Studie Contact Back-up
- Naam: MIGHTY Trial Coordinator
- Telefoonnummer: +4420 7679 9852
- E-mail: ctc.mighty@ucl.ac.uk
Studie Locaties
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London, Verenigd Koninkrijk
- Werving
- Great Ormond Street Hospital
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Contact:
- Olga Slater
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London, Verenigd Koninkrijk
- Werving
- University College London Hospital
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Contact:
- Sandra Strauss
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Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
- Kind
- Volwassen
Accepteert gezonde vrijwilligers
Beschrijving
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.
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: NVT
- Interventioneel model: Opdracht voor een enkele groep
- Masker: Geen (open label)
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
|---|---|
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Experimenteel: hBRCA84D CAR T cells
Treatment with hBRCA84D CAR T cells
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The hBRCA84D CAR T cells target B7-H3 positive cells.
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Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
|
Veiligheid van het toedienen van de ATIMP
Tijdsspanne: 28 dagen
|
Incidentie van graad 3-5 toxiciteit die oorzakelijk verband houdt met de ATIMP, in het bijzonder ernstig cytokine-afgiftesyndroom en ernstige neurotoxiciteit.
|
28 dagen
|
|
Aantal gegenereerde therapeutische producten en het aantal ATIMP's dat is toegediend na succesvolle productie
Tijdsspanne: 28 dagen
|
Haalbaarheid van het genereren van het ATIMP, zoals beoordeeld aan de hand van het aantal gegenereerde therapeutische producten en het aantal ATIMP's dat na succesvolle productie wordt toegediend.
|
28 dagen
|
Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
|
Progressievrije overleving (PFS)
Tijdsspanne: 1 jaar
|
Progressievrije overleving (PFS) na intraveneuze toediening van ATIMP
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1 jaar
|
|
Tijd tot progressie (TTP)
Tijdsspanne: 1 jaar
|
Tijd tot progressie (TTP) na intraveneuze toediening van ATIMP
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1 jaar
|
|
Algemeen overleven
Tijdsspanne: 1 jaar
|
Algehele overleving na intraveneuze toediening van ATIMP
|
1 jaar
|
|
Objective response rate
Tijdsspanne: 1 year
|
Based on cross-sectional imaging after the ATIMP intravenous administration
|
1 year
|
Medewerkers en onderzoekers
Sponsor
Studie record data
Bestudeer belangrijke data
Studie start (Werkelijk)
Primaire voltooiing (Geschat)
Studie voltooiing (Geschat)
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
Eerst geplaatst (Werkelijk)
Updates van studierecords
Laatste update geplaatst (Werkelijk)
Laatste update ingediend die voldeed aan QC-criteria
Laatst geverifieerd
Meer informatie
Termen gerelateerd aan deze studie
Trefwoorden
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- UCL/150859
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct
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