← Back to Search

Other

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 and 5 years following transplant

Awards & highlights

Study 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.Check my eligibility

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.See study design

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

Select...

I am between 8 and 25 years old.

Select...

I do not have any untreated infections.

Select...

I have been diagnosed with severe systemic sclerosis or lupus.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 2 and 5 years following transplant

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Two-year progression free survival

Secondary outcome measures

100 day treatment-related mortality

Change in quality of life

Disease-specific response/progression endpoints: SSc cohort

+4 more

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.

0 / 3Eligibility criteria met

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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What hazards can potentially arise from a Depletion of CD3/CD19 autologous stem cell transplant?

"Taking into account the exploratory nature of Phase 2 trials, our team at Power has assigned a score of two for Depletion of CD3/CD19 in an autologous stem cell transplant's safety. To date, there is observational data that demonstrate its security but no evidence confirming efficacy."

Answered by AI

How many participants are engaging in this experiment?

"Affirmative. According to clinicaltrials.gov, this medical trial has been accepting participants since its initial posting on February 1st 2023 and was most recently updated on October 5th 2022. Currently, the research team is recruiting up to 20 patients from a single site."

Answered by AI

Is enrollment available for this clinical trial?

"According to clinicaltrials.gov, this medical trial is currently looking for volunteers; the study was initially posted on February 1st of 2023 and last modified five months later in October 2022."

Answered by AI

Who meets the prerequisites to join this experiment?

"This investigation seeks 20 individuals aged 8 to 25 with Libman-Sacks Disease. The ideal participant must have severe systemic sclerosis or lupus erythematosus, possess sufficient organ functionality, and not be suffering from any untreated infections."

Answered by AI