Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

Stephan Grupp MD PhD

Status and phase: Enrolling; Phase 2

Conditions: Systemic Sclerosis; Systemic Lupus Erythematosus

Treatments: Biological: Depletion of CD3/CD19 in an autologous stem cell transplant

Study type: Interventional

Funder types: Other

Identifiers

NCT05029336

19-016604

Details and patient eligibility

About

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.

Enrollment: 20 estimated patients

Sex: All

Ages: 8 to 25 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

CD3/CD19 depleted ASCT

Experimental group

Description:

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

Treatment:

Biological: Depletion of CD3/CD19 in an autologous stem cell transplant