Recruiting
Phase 2

ASCT

Sponsor:

Stephan Grupp MD PhD

Code:

NCT05029336

Conditions

Systemic Lupus Erythematosus

Systemic Sclerosis

Eligibility Criteria

Sex: All

Age: 8 - 25

Healthy Volunteers: Not accepted

Interventions

Depletion of CD3/CD19 in an autologous stem cell transplant

Study Details

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 ID

NCT05029336

Start date

Mar, 2026

Status verified date

Sep, 2025

Completion date

May, 2031

Anticipated

Primary completion date

Dec, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 8 - 25

Healthy Volunteers: Not accepted

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 Design

Enrollment

20 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: CD3/CD19 depleted ASCT

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

Primary outcome measure

  • Two-year progression free survival [ Time Frame: 2 years ]

Central Contacts and Locations

Central contacts

Patricia M Hankins

(215) 590-5168HANKINSP@chop.edu

Caitlin Elgarten, MD

2158079038elgartenc@chop.edu

Locations

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Study Coordinator

(215) 590-5168HANKINSP@chop.edu

Principal investigator

2158079038elgartenc@chop.edu

Principal Investigator:

Caitlin Elgarten, MD

More Information

Sponsor

Stephan Grupp MD PhD

Last update posted

Oct 14, 2025

Last verified

Sep, 2025

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Stephan Grupp MD PhD on 2025-10-14.