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Recruiting NCT05029336

Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

Phase II Interventional Systemic Lupus Erythematosus Systemic Sclerosis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Depletion of CD3/CD19 in an autologous stem cell transplant.
Who it may be relevant to
Registry conditions: Systemic Lupus Erythematosus, Systemic Sclerosis. Basic parameters: 8 years — 25 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Official title

Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases

Overview

A subset of autoimmune diseases (ADs) in children and young adults are life-threatening and unresponsive to conventional treatments. In these patients, the delivery of high dose immunosuppressive therapy followed by autologous stem cell transplant (ASCT) offers a treatment strategy capable of purging the pathogenic, autoreactive immune system and an opportunity for "immune reset." This strategy has been used in adults across a myriad of indications with evidence for efficacy. This study proposes a pilot study to evaluate this therapeutic strategy in children and young adults with systemic sclerosis (SSc) and systemic lupus erythematosis (SLE), two potentially life threatening autoimmune diseases that may response to this therapeutic approach.

Interventions

  • Biological Depletion of CD3/CD19 in an autologous stem cell transplant
    The purpose of this study is to determine the safety and feasibility of CD3/CD19 depleted autologous stem cell transplant for the treatment of life threatening autoimmune disease. We will perform CD3/CD19 depletion using the CliniMACs device as a means of purging autoreactive T and B cells from the transfused autologous stem cell product, while retaining some immune function, namely natural killer cells and monocytes in the product.

Primary outcome measures

  • Two-year progression free survival [Time frame: 2 years]
Secondary outcome measures (11)
  • Disease-specific response/progression endpoints: SSc cohort [Time frame: 24 months following transplant]
  • Disease-specific response/progression endpoints: SSc cohort [Time frame: 24 months following transplant]
  • Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort [Time frame: 24 months following transplant]
  • Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort [Time frame: 24 months following transplant]
  • Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort [Time frame: 24 months following transplant]
  • Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort [Time frame: 24 months following transplant]
  • Overall survival (OS) [Time frame: 2 and 5 years following transplant]
  • Event free survival (EFS) [Time frame: 2 and 5 years following transplant]
  • 100 day treatment-related mortality [Time frame: 100 days from stem cell infusion]
  • Time to engraftment [Time frame: 3 days]
  • Change in quality of life [Time frame: prior to autologous stem cell transplant (ASCT) until 5 years post-transplant]

Eligibility criteria

Inclusion criteria

  • Age 8 ≤ 25 years at time of enrollment.
  • Severe systemic sclerosis or systemic lupus erythematosus based on specific criteria
  • Adequate organ function status
  • No active, untreated infections.

Exclusion criteria

  • Previous hematopoietic stem cell transplant (HSCT) or solid organ transplant
  • Pregnancy
  • Ongoing participation in a clinical trial testing an investigational drug or ongoing receipt of disallowed disease modifying anti-rheumatic drugs (DMARD)
  • Severe comorbidity that jeopardizes the ability of the subject to tolerate therapy

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 1 center
  • Children's Hospital of Philadelphia — Philadelphia

Identifiers

NCT: NCT05029336 · 19-016604

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗