Study of KYV-101 Anti-CD19 CAR T Therapy in Adult Dermatomyositis

Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age25-72
SponsorStanford University

About this trial

The goal of this clinical trial is to characterize to understand the effects of a type of cell therapy called Chimeric Antigen Receptor T lymphocyte (CAR T) therapy in adult patients with the autoimmune disease dermatomyositis. This study will utilize a technology that modifies a type of white blood cell called the cytotoxic T lymphocyte-this T cell normally functions in the immune system to kill infected or potentially harmful cells in the body. In CAR T therapy, the patients' white blood cells are harvested and the cytotoxic T cells are isolated and modified such that they are programmed to kill any cell that has a protein structure called "CD19" on its outer surface (membrane). Since the CD19 protein is only present on a type of white blood cell called the B lymphocyte, when these "re-engineered" cytotoxic T lymphocytes are then given back to the patient (by an infusion), these cells will seek out and kill essentially all of the patient's B cells. B cells are an important part of a person's immune system and have many functions, including the production of antibodies. It is thought that, in dermatomyositis and other autoimmune diseases, a tiny subset of these B cells plays a large role in making autoantibodies (antibodies directed against the patient's own tissues) and causing disease. The idea is that the therapy will "wipe out" all/most of the B cells in the patient so that they can make an entirely new set of B cells to recreate a functional immune system without the autoimmune disease.

The main questions the study intends to answer are:

* Understanding how well patients tolerate undergoing this therapy in terms of side effects; * Getting an early idea if this therapy can help certain aspects of the autoimmune disease, including inflammation in the skin, muscles, and lungs;

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Diagnosis of probable or definite (>55%) IIM and subgroup classification as dermatomyositis according to the 2017 EULAR/ACR classification criteria for idiopathic inflammatory myopathies.

Age > 25 years and < 72 years at time of signing informed consent Refractory disease: subject with previous failure (or intolerance) to glucocorticoids and at least two non-glucocorticoid immunosuppressive therapies (including mycophenolate mofetil or mycophenolic acid, cyclophosphamide, azathioprine, methotrexate, calcineurin inhibitors, tofacitinib or other JAK inhibitors, rituximab, or IVIG) administered for at least 12 weeks within 24 months prior to screening.

Patient global VAS≥2 cm.

Physician's global VAS ≥2 cm.

Disqualifiers

Myositis Global Damage Index (MDI) ≥5.

Severe proximal muscle atrophy of upper or lower extremity on MRI.

Severe proximal muscle atrophy of upper or lower extremity on clinical examination.

Wheelchair-bound at home.

Trial design

Design model

Sequential

Treatments tested in this trial

  • KYV-101

    Biological/Vaccine

    The investigational product, KYV-101, is an autologous CD19-targeted CAR T-cell immunotherapy.

Treatment groups

21 Participants
are divided into 1 treatment group
Group A: Active Intervention with CAR TExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Number of participants experiencing adverse events

This outcome is to characterize the safety and tolerability of administering KYV-101 CAR T cells that meet the established criteria to patients with DM. Participants experiencing adverse events including laboratory abnormalities will be reported.

Time frame
24 weeks

Secondary outcomes

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.