Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

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

Study Overview

• Status: Recruiting
• Conditions: Systemic Lupus Erythematosus; Systemic Sclerosis
• Intervention / Treatment: Biological: Depletion of CD3/CD19 in an autologous stem cell transplant

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Caitlin Elgarten, MD; Phone Number: 2158079038; Email: elgartenc@chop.edu
• Study Contact Backup: Name: Patricia M Hankins; Phone Number: (215) 590-5168; Email: HANKINSP@chop.edu

Study Locations

• United States
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Children's Hospital of Philadelphia
      • Contact: Principal investigator; Phone Number: 2158079038; Email: elgartenc@chop.edu
      • Principal Investigator: Caitlin Elgarten, MD
      • Contact: Study Coordinator; Phone Number: (215) 590-5168; Email: HANKINSP@chop.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 8 years to 25 years (Child, Adult)
• Accepts Healthy Volunteers: No

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: CD3/CD19 depleted ASCT; The test article is autologous stem cell transplant with a CD3/CD19-depleted stem cell product.

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.; Other Names: CD3/CD19 depletion using cliniMACs device

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Two-year progression free survival	Survival without evidence of relapse or disease progression	2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Disease-specific response/progression endpoints: SSc cohort	o Pulmonary function: Change in forced vital capacity (FVC), total lung capacity (TLC) or diffusing capacity of the lung for carbon monoxide (DLCO) > 10%	24 months following transplant
Disease-specific response/progression endpoints: SSc cohort	o Skin condition: An improvement is indicated by a decrease on modified Rodan Skin Score (mRSS) of > 5 points	24 months following transplant
Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort	o Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) < 4	24 months following transplant
Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort	o Complete remission off therapy (BILAG D/E only or SLEDAI=0 and no SLE treatment except hydroxychloroquine)	24 months following transplant
Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort	o Serologic response: presence of positive ANA, anti-dsDNA and anticardiolpin antibody titers	24 months following transplant
Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort	o Serologic response: abnormal complement C3 and C4 levels	24 months following transplant
Overall survival (OS)	Overall survival will be considered as time from transplant to death from any cause	2 and 5 years following transplant
Event free survival (EFS)	Events include death, and significant persistent organ damage o An event based on organ dysfunction must be documented on at least two occasions, at least three months apart and include: respiratory failure (resting O2 saturation < 88%), renal failure (chronic dialysis) and cardiomyopathy (clinical congestive heart failure New York Class III or IV, left ventricular ejection fraction (LVEF) < 30% by echocardiogram despite therapy)	2 and 5 years following transplant
100 day treatment-related mortality	Defined as death from non-disease related causes in the 100 days from stem cell infusion	100 days from stem cell infusion
Time to engraftment	• Achieving an absolute neutrophil count (ANC) > 500 cells/uL and an unsupported platelet count of > 20,000 cells/uL for three consecutive days	3 days
Change in quality of life	Quality of life will be measured based on the Patient-Reported Outcomes measurement Information System (PROMIS) that evaluates physical, mental and social health in adults and children.; patient reported outcome measurement information system (PROMIS) will be administered to each patient (or proxy) prior to autologous stem cell transplant (ASCT) and three times/year for the first two years post-transplant and then annually until five years post-transplant.	prior to autologous stem cell transplant (ASCT) until 5 years post-transplant

Collaborators and Investigators

• Sponsor: Stephan Grupp MD PhD
• Investigators: Principal Investigator: Caitlin Elgarten, MD, Children's Hospital of Philadelphia

Study record dates

Study Major Dates

• Study Start (Estimated): March 1, 2026
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): May 1, 2031

Study Registration Dates

• First Submitted: August 24, 2021
• First Submitted That Met QC Criteria: August 29, 2021
• First Posted (Actual): August 31, 2021

Study Record Updates

• Last Update Posted (Estimated): October 14, 2025
• Last Update Submitted That Met QC Criteria: October 9, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Skin Diseases; Skin and Connective Tissue Diseases; Lupus Erythematosus, Systemic; Scleroderma, Systemic
• Other Study ID Numbers: 19-016604

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No