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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.

Register:	ClinicalTrials.gov
Last refreshed on:	17 March 2025
Main ID:	NCT05029336
Date of registration:	24/08/2021
Prospective Registration:	Yes
Primary sponsor:	Stephan Grupp MD PhD
Public title:	Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases
Scientific title:	Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases
Date of first enrolment:	September 2025
Target sample size:	20
Recruitment status:	Recruiting
URL:	https://clinicaltrials.gov/ct2/show/NCT05029336
Study type:	Interventional
Study design:	Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
Phase:	Phase 2

Countries of recruitment
United States

Contacts

Name:	Study Coordinator
Address:
Telephone:	267-425-1935
Email:	leej11@chop.edu
Affiliation:

Name:	Jessica H Lee, BS
Address:
Telephone:	267-425-1935
Email:	leej11@chop.edu
Affiliation:

Name:	Caitlin Elgarten, MD
Address:
Telephone:
Email:
Affiliation:	Children's Hospital of Philadelphia

Key inclusion & exclusion 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

Age minimum: 8 Years
Age maximum: 25 Years
Gender: All

Health Condition(s) or Problem(s) studied

Systemic Lupus Erythematosus

Systemic Sclerosis

Intervention(s)

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

Primary Outcome(s)

Two-year progression free survival [Time Frame: 2 years]

Secondary Outcome(s)

Event free survival (EFS) [Time Frame: 2 and 5 years following transplant]

Disease-specific response/progression endpoints: SSc cohort [Time Frame: 24 months following transplant]

100 day treatment-related mortality [Time Frame: 100 days from stem cell infusion]

Change in quality of life [Time Frame: prior to autologous stem cell transplant (ASCT) until 5 years post-transplant]

Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort [Time Frame: 24 months following transplant]

Time to engraftment [Time Frame: 3 days]

Overall survival (OS) [Time Frame: 2 and 5 years following transplant]

Secondary ID(s)

19-016604

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Ethics review

Results

Results available:
Date Posted:
Date Completed:
URL:

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