Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

Part of paid clinical trials in Philadelphia, Pennsylvania.

Sponsor
Stephan Grupp MD PhD
Study ID
NCT05029336
Phase
PHASE2
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
8 Years - 25 Years
Healthy Volunteers
Not accepted

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.

Study Details

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.

Key Dates

Start date
Mar 31, 2026
Status verified
Sep 2025
Primary completion
Dec 31, 2027
Completion
May 31, 2031

Study Design

Enrollment
20 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

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

Primary Outcome Measure

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

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
Study Coordinator
[email protected]
(215) 590-5168
Principal investigator
[email protected]
2158079038
Caitlin Elgarten, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Philadelphia, PA

By condition
Lupus (SLE)
By specialty
RheumatologyAutoimmune disease
By research site
Children's Hospital of Philadelphia· Philadelphia, PA

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