Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

Description

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.

Conditions

Systemic Lupus Erythematosus, Systemic Sclerosis

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Study Overview

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Study Details

Study 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.

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

Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

Condition
Systemic Lupus Erythematosus
Intervention / Treatment

-

Contacts and Locations

Philadelphia

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19104

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

  • 1. Age 8 ≤ 25 years at time of enrollment.
  • 2. Severe systemic sclerosis or systemic lupus erythematosus based on specific criteria
  • 3. Adequate organ function status
  • 4. No active, untreated infections.
  • 1. Previous hematopoietic stem cell transplant (HSCT) or solid organ transplant
  • 2. Pregnancy
  • 3. Ongoing participation in a clinical trial testing an investigational drug or ongoing receipt of disallowed disease modifying anti-rheumatic drugs (DMARD)
  • 4. Severe comorbidity that jeopardizes the ability of the subject to tolerate therapy

Ages Eligible for Study

8 Years to 25 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Stephan Grupp MD PhD,

Caitlin Elgarten, MD, PRINCIPAL_INVESTIGATOR, Children's Hospital of Philadelphia

Study Record Dates

2031-05