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NCT05029336 | RECRUITING | Systemic Lupus Erythematosus

Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases
Sponsor:

Stephan Grupp MD PhD

Information provided by (Responsible Party):

Stephan Grupp MD PhD

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.

Condition or disease

Systemic Lupus Erythematosus

Systemic Sclerosis

Intervention/treatment

Depletion of CD3/CD19 in an autologous stem cell transplant

Phase

PHASE2

Study Type : INTERVENTIONAL
Estimated Enrollment : 20 participants
Masking : NONE
Primary Purpose : TREATMENT
Official Title : Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases
Actual Study Start Date : 2026-03
Estimated Primary Completion Date : 2027-12
Estimated Study Completion Date : 2031-05

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 8 Years to 25 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
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
Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

Location Details

NCT05029336

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How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

RECRUITING

United States, Pennsylvania

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104