Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

NCT05029336 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 19-016604
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

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

Outcome Measures

Primary Outcomes (1)

• Two-year progression free survival

  Survival without evidence of relapse or disease progression

  2 years

Secondary Outcomes (11)

• Disease-specific response/progression endpoints: SSc cohort

  24 months following transplant

• Disease-specific response/progression endpoints: SSc cohort

  24 months following transplant

• Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort

  24 months following transplant

• Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort

  24 months following transplant

• Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort

  24 months following transplant

Study Arms (1)

CD3/CD19 depleted ASCT

EXPERIMENTAL

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

Interventions

Depletion of CD3/CD19 in an autologous stem cell transplant BIOLOGICAL

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.

Also known as: CD3/CD19 depletion using cliniMACs device

CD3/CD19 depleted ASCT

Eligibility Criteria

• Age: 8 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Stephan Grupp MD PhD: lead

Study Sites (1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

MeSH Terms

Conditions

Lupus Erythematosus, Systemic; Scleroderma, Systemic

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Skin Diseases

Study Officials

• Caitlin Elgarten, MD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Patricia M Hankins

CONTACT

(215) 590-5168; HANKINSP@chop.edu

Caitlin Elgarten, MD

CONTACT

2158079038; elgartenc@chop.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Director of Cancer Immunotherapy Program

Model Details: open label single arm pilot study

Study Record Dates

• First Submitted: August 24, 2021
• First Posted: August 31, 2021
• Study Start: March 1, 2026
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): May 1, 2031
• Last Updated: October 14, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)