Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases
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 assess 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
🗓️ Study Start (Actual) December 2024
🗓️ Primary Completion (Estimated) December 2026
✅ Study Completion (Estimated) May 2031
👥 Enrollment (Estimated) 20
🔬 Study Type INTERVENTIONAL
📊 Phase PHASE2
Locations:
📍 Philadelphia, Pennsylvania, United States

📋 Eligibility Criteria

Description

    Inclusion 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.

    Exclusion Criteria:

    • 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 (CHILD, ADULT)
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No

🗓️ 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

  • First Submitted 24 August 2021
  • First Submitted that Met QC Criteria 29 August 2021
  • First Posted 31 August 2021

Study Record Updates

  • Last Update Submitted that Met QC Criteria 11 June 2024
  • Last Update Posted 12 June 2024
  • Last Verified June 2024