RECRUITING

Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2024-12
Study Completion:2031-05
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05029336

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.

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

Contacts and Locations

Study Contact

Jessica H Lee, BS
CONTACT
267-425-1935
leej11@chop.edu
Caitlin Elgarten, MD
CONTACT
2158079038
elgartenc@chop.edu

Principal Investigator

Caitlin Elgarten, MD
PRINCIPAL_INVESTIGATOR
Children's Hospital of Philadelphia

Study Locations (Sites)

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

Collaborators and Investigators

Sponsor: Stephan Grupp MD PhD

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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-12
Study Completion Date2031-05

Study Record Updates

Study Start Date2024-12
Study Completion Date2031-05

Terms related to this study

Additional Relevant MeSH Terms

  • Systemic Lupus Erythematosus
  • Systemic Sclerosis