Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

NCT ID: NCT05029336

Last Updated: 2025-10-14

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2026-03-31
• Study Completion Date: 2031-05-31

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

CD3/CD19 depleted ASCT

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

Group Type: EXPERIMENTAL

Depletion of CD3/CD19 in an autologous stem cell transplant

Intervention Type: 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.

Interventions

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.

Intervention Type: BIOLOGICAL

Other Intervention Names

CD3/CD19 depletion using cliniMACs device

Eligibility Criteria

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

• Minimum Eligible Age: 8 Years
• Maximum Eligible Age: 25 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Stephan Grupp MD PhD

OTHER

Sponsor Role: lead

Responsible Party

Stephan Grupp MD PhD

Director of Cancer Immunotherapy Program

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Caitlin Elgarten, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Philadelphia

Locations

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Patricia M Hankins

Role: CONTACT

HANKINSP@chop.edu

(215) 590-5168

Caitlin Elgarten, MD

Role: CONTACT

elgartenc@chop.edu

2158079038

Facility Contacts

Study Coordinator

Role: primary

HANKINSP@chop.edu

(215) 590-5168

Principal investigator

Role: backup

elgartenc@chop.edu

2158079038

Other Identifiers

19-016604

Identifier Type: -

Identifier Source: org_study_id