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- Klinische proef NCT02623582
CD123 Redirected Autologous T Cells for AML
Pilot Study of RNA-Redirected Autologous T Cells Engineered to Contain Anti-CD123 Linked to TCR and 4-1BB Signaling Domains in Patients With Refractory or Relapsed Acute Myeloid Leukemia
Studie Overzicht
Toestand
Interventie / Behandeling
Studietype
Inschrijving (Werkelijk)
Fase
- Vroege fase 1
Contacten en locaties
Studie Locaties
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Pennsylvania
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Philadelphia, Pennsylvania, Verenigde Staten, 19104
- Abramson Cancer Center of the University of Pennsylvania
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Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
Accepteert gezonde vrijwilligers
Geslachten die in aanmerking komen voor studie
Beschrijving
Inclusion Criteria:
- Male or female subjects 18 years of age or older with AML with no available curative treatment options using currently available therapies
- Subjects must have a suitable stem cell donor available who may donate cells if the subject needs to undergo allogeneic HCT. Donor may be matched or mismatched and must be found to be suitable according to the institution's standard criteria.
Subjects with second or subsequent relapse, any relapse refractory to salvage, or with persistent disease after at least two lines of therapy.
a. Subjects with relapsed disease after prior allogeneic HCT (myeloablative or non-myeloablative) will be eligible if they meet all other inclusion criteria and i. Have experienced graft rejection (no evidence of donor cells by STR analysis on 2 occasions separated by at least 1 month), OR: ii. Donor cells are present but there is no active GVHD, subject does not require immunosuppression and is more than 6 months from transplant
- Subjects must have evaluable disease defined as >5% blasts on marrow aspirate or biopsy, extramedullary disease (CNS involvement is prohibited), or at least 20% blasts in the peripheral blood within 2 weeks prior to enrollment. Note: subjects with second or subsequent relapse are considered to have evaluable disease even without meeting the above morphologic criteria if they are found to have persistent recurrent disease-associated molecular or cytogenetic abnormalities.
- Creatinine < 1.6 mg/dl
- ALT/AST must be < 5 x upper limit of normal unless related to disease
- Bilirubin < 2.0 mg/dl, unless subject has Gilbert's syndrome (≤3.0 mg/dL);
- ECOG Performance status 0-2.
- Left ventricular ejection fraction > 40% as confirmed by ECHO/MUGA
- Written informed consent is given.
- Subjects of reproductive potential must agree to use acceptable birth control methods.
Exclusion criteria:
- Pregnant or lactating women. The safety of this therapy on unborn children is not known. Female study participants of reproductive potential must have a negative serum pregnancy test at enrollment. A urine pregnancy test will be performed within 48 hours before infusion.
- HIV infection.
- Active hepatitis B or hepatitis C infection.
- Concurrent use of systemic steroids or immunosuppressant medications. Recent or current use of inhaled steroids or physiologic replacement with hydrocortisone is not exclusionary.
- Absolute lymphocyte count <500/uL
- Any uncontrolled active medical disorder that would preclude participation as outlined.
- Subjects with signs or symptoms indicative of CNS involvement. A CNS evaluation should be performed as clinically appropriate to rule out CNS involvement.
- Known history of allergy or hypersensitivity to study product excipients (human serum albumin, DMSO, and Dextran 40).
- Class III/IV cardiovascular disability according to the New York Heart Association Classification.
- Patients with a known history or prior diagnosis of optic neuritis or other immunologic or inflammatory disease affecting the central nervous system
- Subjects with clinically apparent arrhythmia, or arrhythmias that are not stable on medical management, within 2 weeks of the Screening/Enrollment visit.
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: Niet-gerandomiseerd
- Interventioneel model: Parallelle opdracht
- Masker: Geen (open label)
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
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Experimenteel: Cohort 1
The first 3 subjects to receive RNA CART123 cells will receive up to 3 doses of RNA CART123 cells, with no lymphodepleting chemotherapy prior to infusion.
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Given IV
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Experimenteel: Cohort 2
The remaining 12 subjects of the study will receive up to six IV doses of RNA CART123 cells. Subjects in Cohort 2 may be given lymphodepleting chemotherapy 4 days (+/- 1 day) prior to the first CART123 cell infusion (if ALC> 500/uL). Lymphodepleting chemotherapy may be repeated before the fourth dose of RNA CART123 cells (if ALC> 500/uL). Lymphodepleting chemotherapy includes a single dose of cyclophosphamide (1g/m2) Weight used for dosing will be the weight obtained prior to the apheresis procedure Cell numbers are based on CAR+ cells with CAR expression determined by flow cytometry Based on the product release criteria, at least 20% of the total cells will be RNA CART123 cells. Dosing will not be changed for changes in subject weight The indicated doses are +/- 20% to account for manufacturing variability. |
IV gegeven
Given IV
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Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Tijdsspanne |
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Aantal bijwerkingen
Tijdsspanne: 2 jaar
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2 jaar
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Medewerkers en onderzoekers
Sponsor
Onderzoekers
- Hoofdonderzoeker: Saar Gill, MD, PhD, Abramson Cancer Center of the University of Pennsylvania
Studie record data
Bestudeer belangrijke data
Studie start
Primaire voltooiing (Werkelijk)
Studie voltooiing (Werkelijk)
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
Eerst geplaatst (Schatting)
Updates van studierecords
Laatste update geplaatst (Werkelijk)
Laatste update ingediend die voldeed aan QC-criteria
Laatst geverifieerd
Meer informatie
Termen gerelateerd aan deze studie
Aanvullende relevante MeSH-voorwaarden
- Neoplasmata per histologisch type
- Neoplasmata
- Leukemie
- Leukemie, myeloïde
- Leukemie, myeloïde, acuut
- Fysiologische effecten van medicijnen
- Moleculaire mechanismen van farmacologische werking
- Antireumatische middelen
- Antineoplastische middelen
- Immunosuppressieve middelen
- Immunologische factoren
- Antineoplastische middelen, alkylering
- Alkyleringsmiddelen
- Myeloablatieve agonisten
- Cyclofosfamide
Andere studie-ID-nummers
- 823175 - UPCC 04415
Plan Individuele Deelnemersgegevens (IPD)
Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?
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