Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

Phase 2

Recruiting

• Conditions: Systemic Lupus Erythematosus; Systemic Sclerosis
• Interventions: Biological: Depletion of CD3/CD19 in an autologous stem cell transplant

• Registration Number: NCT05029336
• Lead Sponsor: 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-08-24
• Study First Submitted QC: 2021-08-29
• Study First Posted: 2021-08-31
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-04
• Last Update Posted: 2025-03-05
• Study Start: 2025-09
• Primary Completion: 2026-12
• Study Completion: 2031-05

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 20

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
CD3/CD19 depleted ASCT	Depletion of CD3/CD19 in an autologous stem cell transplant	The test article is autologous stem cell transplant with a CD3/CD19-depleted stem cell product.

Primary Outcome Measures

Name	Time	Method
Two-year progression free survival	2 years	Survival without evidence of relapse or disease progression

Secondary Outcome Measures

Name	Time	Method
Disease-specific response/progression endpoints: SSc cohort	24 months following transplant	o Skin condition: An improvement is indicated by a decrease on modified Rodan Skin Score (mRSS) of \> 5 points
Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort	24 months following transplant	o Serologic response: abnormal complement C3 and C4 levels
Overall survival (OS)	2 and 5 years following transplant	Overall survival will be considered as time from transplant to death from any cause
Event free survival (EFS)	2 and 5 years following transplant	Events include death, and significant persistent organ damage; o An event based on organ dysfunction must be documented on at least two occasions, at least three months apart and include: respiratory failure (resting O2 saturation \< 88%), renal failure (chronic dialysis) and cardiomyopathy (clinical congestive heart failure New York Class III or IV, left ventricular ejection fraction (LVEF) \< 30% by echocardiogram despite therapy)
100 day treatment-related mortality	100 days from stem cell infusion	Defined as death from non-disease related causes in the 100 days from stem cell infusion
Time to engraftment	3 days	• Achieving an absolute neutrophil count (ANC) \> 500 cells/uL and an unsupported platelet count of \> 20,000 cells/uL for three consecutive days
Change in quality of life	prior to autologous stem cell transplant (ASCT) until 5 years post-transplant	* Quality of life will be measured based on the Patient-Reported Outcomes measurement Information System (PROMIS) that evaluates physical, mental and social health in adults and children.; * patient reported outcome measurement information system (PROMIS) will be administered to each patient (or proxy) prior to autologous stem cell transplant (ASCT) and three times/year for the first two years post-transplant and then annually until five years post-transplant.

Trial Locations

Locations (1)

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States