REACT-01: Reversing Autoimmunity Through Cell Therapy

Description

This is a phase 1, open-label, non-randomized study enrolling pediatric and young adult research participants with treatment-refractory Systemic Lupus Erythematosus (SLE), to examine the safety, feasibility, and efficacy of administering T cell products derived from peripheral blood mononuclear cells (PBMC) that have been genetically modified to express CD19 specific chimeric antigen receptor (CAR) A child or young adult meeting all eligibility criteria and meeting none of the exclusion criteria will have their T cells collected. The T cells will then be bioengineered into a CAR T cell that targets circulating and tissue residing B cells.

Conditions

Systemic Lupus Erythematosus

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Study Overview

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Study Details

Study overview

This is a phase 1, open-label, non-randomized study enrolling pediatric and young adult research participants with treatment-refractory Systemic Lupus Erythematosus (SLE), to examine the safety, feasibility, and efficacy of administering T cell products derived from peripheral blood mononuclear cells (PBMC) that have been genetically modified to express CD19 specific chimeric antigen receptor (CAR) A child or young adult meeting all eligibility criteria and meeting none of the exclusion criteria will have their T cells collected. The T cells will then be bioengineered into a CAR T cell that targets circulating and tissue residing B cells.

REACT-01: Reversing Autoimmunity Through Cell Therapy

REACT-01: Reversing Autoimmunity Through Cell Therapy

Condition
Systemic Lupus Erythematosus
Intervention / Treatment

-

Contacts and Locations

Seattle

Seattle Children's Hospital, Seattle, Washington, United States, 98105

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Male and female subjects aged between 2-30 years old. The first 3 subjects will be aged ≥ 17. The FDA will review safety data to determine if the age can be lowered first to ≥ 12 then, following the treatment of 3 further subjects aged 12-17, to ≥ 2
  • * Serologically active Systemic Lupus Erythematosus that is refractory to treatment
  • * Able to tolerate apheresis or already has an apheresis product available for use in manufacturing.
  • * ≥ 24 weeks post last Rituximab or related B cell depleting therapy
  • * ≥ 12 weeks post last Belimumab / Anifrolumab therapy
  • * ≥ 4 weeks post last calcineurin inhibitor treatment
  • * For subjects receiving non-calcineurin immunosuppressive therapy, on a stable dose for ≥ 8 weeks before enrollment
  • * For subjects receiving corticosteroid therapy, on a stable dose for ≥ 2 weeks before enrollment
  • * Adequate organ function
  • * Adequate laboratory values
  • * Subjects of childbearing or child-fathering potential must agree to use highly effective contraception from consent through 12 months following infusion of investigational product on trial
  • * Subjects must be willing to remain within 1 hour's drive of Seattle Children's Hospital for 4 weeks following CAR T cell infusion.
  • * Subject and/or legally authorized representative has signed the informed consent form for this study
  • * History or presence of active CNS lupus or other CNS disease
  • * Kidney dysfunction requiring renal replacement therapy
  • * Pregnant or breastfeeding
  • * Insufficient pulmonary reserve including history of COPD, \>10 pack year smoking history or SLE lung disease with hypoxia at rest with oxygen saturation ≤92% on room air
  • * Unable to tolerate repletion with any formulation of IgG.
  • * Active or prior malignancy, unless the malignancy was treated and there is no evidence of recurrent disease \<5 years from enrollment.
  • * Prior solid organ transplantation.
  • * Presence of an active severe infection
  • * Presence of any condition that, in the opinion of the investigator, would prohibit the subject from undergoing treatment under this protocol

Ages Eligible for Study

2 Years to 30 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Seattle Children's Hospital,

Shaun Jackson, MD, STUDY_CHAIR, Seattle Children's Hospital

Colleen Annesley, MD, STUDY_DIRECTOR, Seattle Children's Hospital

Corinne Summers, MD, STUDY_DIRECTOR, Seattle Children's Hospital

Study Record Dates

2041-10