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Stem Cell Transplant for Autoimmune Diseases

Phase 2
Recruiting
Led By Caitlin Elgarten, MD
Research Sponsored by Stephan Grupp
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age 8 ≤ 25 years at time of enrollment
No active, untreated infections
Must not have
Severe comorbidity that jeopardizes the ability of the subject to tolerate therapy
Ongoing participation in a clinical trial testing an investigational drug or ongoing receipt of disallowed disease modifying anti-rheumatic drugs (DMARD)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 and 5 years following transplant
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new treatment strategy for children and young adults with autoimmune diseases that are unresponsive to conventional treatments. The new treatment involves delivering high doses of immunosuppressive therapy followed by an autologous stem cell transplant (ASCT). The goal is to purge the pathogenic, autoreactive immune system and reset the immune system.

Who is the study for?
This trial is for young individuals aged 8-25 with severe systemic sclerosis (SSc) or systemic lupus erythematosus (SLE). They must have specific disease characteristics, good organ function, and no current untreated infections. It's not open to those who've had previous transplants, are pregnant, in other drug trials, or have severe health issues affecting therapy tolerance.
What is being tested?
The study tests a treatment where patients' own stem cells are modified by removing certain immune cells (CD3/CD19) and then returned to the body. This aims to reset their immune system and improve life-threatening autoimmune diseases like SSc and SLE in children and young adults.
What are the potential side effects?
Potential side effects may include reactions related to the immune system reset such as infection risks due to lowered immunity post-transplant, possible infusion-related reactions during stem cell return, and complications from high dose immunosuppressive therapy.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am between 8 and 25 years old.
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I do not have any untreated infections.
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I have been diagnosed with severe systemic sclerosis or lupus.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have a serious health condition that may affect my ability to handle treatment.
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I am not currently in a trial for a new drug or taking certain arthritis medications.
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I have had a stem cell or organ transplant in the past.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 and 5 years following transplant
This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 and 5 years following transplant for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Two-year progression free survival
Secondary study objectives
100 day treatment-related mortality
Change in quality of life
Disease-specific response/progression endpoints: SSc cohort
+4 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: CD3/CD19 depleted ASCTExperimental Treatment1 Intervention
The test article is autologous stem cell transplant with a CD3/CD19-depleted stem cell product.

Find a Location

Who is running the clinical trial?

Stephan GruppLead Sponsor
Stephan Grupp MD PhDLead Sponsor
4 Previous Clinical Trials
293 Total Patients Enrolled
Caitlin Elgarten, MDPrincipal InvestigatorChildren's Hospital of Philadelphia

Media Library

Autologous Stem Cell Transplant (ASCT) (Other) Clinical Trial Eligibility Overview. Trial Name: NCT05029336 — Phase 2
Lupus Research Study Groups: CD3/CD19 depleted ASCT
Lupus Clinical Trial 2023: Autologous Stem Cell Transplant (ASCT) Highlights & Side Effects. Trial Name: NCT05029336 — Phase 2
Autologous Stem Cell Transplant (ASCT) (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05029336 — Phase 2
~13 spots leftby Dec 2026
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