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IKKb-matured, RNA-loaded Dendritic Cells for Metastasised Uveal Melanoma

Phase 1
Conditions
Melanoma, Uveal Metastatic
Interventions
Biological: Vaccination with IKKb matured Dendritic Cells
Registration Number
NCT04335890
Lead Sponsor
Hasumi International Research Foundation
Brief Summary

A Phase I vaccination trial in patients suffering from recently diagnosed metastatic uveal melanoma not cureable with local therapy and needing systemic therapy. IKKb-matured Dendritic Cells loaded with autologous tumor-RNA + RNA coding for defined antigens and driver mutations will be added to a standard therapy chosen by the tumor board (either checkpoint blockade or chemotherapy).

Detailed Description

Intravenous infusion of 7.5 to 30 mio DCIKKb at 9 vaccination time points (week 1, 3, 7, 13, 19, 25, 31, 37 and 42) and in intervals of 2, 4, and 6 intervals of 6 weeks) is scheduled; the first 4 patients will receive reduced doses for the first 4 vaccinations, namely 7.5 mio (1st and 2nd vaccination) and 15 mio (3rd and 4th vaccination) DC followed by the full dose of 30 mio for subsequent vaccinations. Patients number 5 to 8 will receive initially reduced doses of 15 mio (1st and 2nd vaccination) DC for the first 2 vaccinations, and the full dose of 30 mio for subsequent vaccinations. Patients number 8 to 12 will receive the full dose of 30 mio cells from vaccination 1 onwards provided that no major side effects occurred. Patients will be vaccinated in a staggered approach by selectively decelerating release of the vaccine.

DCIKKb = autologous, monocyte-derived DC that are matured with the standard cocktail (TNF-alpha, IL-1 beta, IL-6 and PGE2) and IKKb-RNA loaded by electroporation with 1) autologous PCR-amplified total tumor mRNA, 2) RNA coding for defined tumor associated antigens (TAA) namely gp100, tyrosinase, PRAME, MAGE-A3, IDO) and 3) RNA coding for driver mutations (GNAQ/GNA11Q209 or R183, or the less frequently occurring SF3B1R625, CYSLTR2L129Q or PLCB4D630) by electroporation; RNAs for selected TAAs are in stock and will be transfected into the DCs only if expressed in the individual tumor of a patient (shown by RNA sequencing of the tumor); RNAs for selected driver mutations are in stock and will be loaded into the DCs only if the respective mutation is found (proven by exome and RNA sequencing) in the individual tumor.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
12
Inclusion Criteria
  • Histologically confirmed unresectable stage IV metastatic uveal melanoma as per AJCC staging system 2014, 7th edition (updated 2018) not curable with local therapy modalities
  • WHO performance status of 0, 1 or 2
  • age from 18 and ≤ 75 years
  • negative pregnancy test
  • signed informed consent
Exclusion Criteria
  • Major serious illness
  • evidence for HIV-1, HIV-2, HTLV-1, HBV or HCV infection
  • active autoimmune disease requiring immunosuppressive therapy
  • splenectomy or radiation therapy of the spleen
  • organ allografts
  • pregnancy
  • lactation
  • psychiatric disorders
  • severe organic brain syndrome

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
DC IKKbVaccination with IKKb matured Dendritic CellsVaccination with IKKb matured RNA loaded Dendritic Cells
Primary Outcome Measures
NameTimeMethod
Safety of DCIKKb1 year

Assesment of side effects using the Common Toxicity Criteria (CTC v4.0)

Tolerability of DCIKKb1 year

Assesment of Quality of life using Quality of life EORTC QLQ-C30, Version 2

Dose-limiting toxicities (DLTs) of DCIKKb1 year

Assesment of side effects using the Common Toxicity Criteria (CTC v4.0)

Maximum tolerated dose (MTD) of DCIKKb1 year

Assesment of side effects using the Common Toxicity Criteria (CTC v4.0)

Secondary Outcome Measures
NameTimeMethod
Prolongation of overall survival (OS) after 1 and 2 years2 years

Assesment of survival

Prolongation of median overall survival2 years

Assesment of survival

Induction of antigen specific CD8+ T cells and / or CD4+ T cells against TAA and mutated drivers2 years

Assesment of immune responses

Trial Locations

Locations (1)

University Hospital Erlangen Dept. of Dermatology

🇩🇪

Erlangen, Bavaria, Germany

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