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Systemic Lupus ErythematosusPhase 2NCT05029336

Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

A Phase 2 study testing Depletion of CD3/CD19 in an autologous stem cell transplant in people with Systemic Lupus Erythematosus and Systemic Sclerosis, run by Stephan Grupp MD PhD at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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… Read more about this study

Who is it for?
Ages 8 to 25 years
What is being tested?
Depletion of CD3/CD19 in an autologous stem cell transplant
Could I receive a placebo?
No, everyone receives an active treatment
How many people
20 participants
Where
1 siteUnited States
Study dates
Mar 2026to May 2031
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 8 to 25 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Age 8 ≤ 25 years at time of enrollment.
  • Severe systemic sclerosis or systemic lupus erythematosus based on specific criteria
  • Adequate organ function status
  • No active, untreated infections.

Who cannot join

  • Previous hematopoietic stem cell transplant (HSCT) or solid organ transplant
  • Pregnancy
  • Ongoing participation in a clinical trial testing an investigational drug or ongoing receipt of disallowed disease modifying anti-rheumatic drugs (DMARD)
  • Severe comorbidity that jeopardizes the ability of the subject to tolerate therapy

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Patricia M Hankins contact

(215) 590-5168

HANKINSP@chop.edu

Caitlin Elgarten, MD contact

2158079038

elgartenc@chop.edu

United States1 site, 1 recruiting

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

    Study Coordinator, (215) 590-5168, HANKINSP@chop.edu

    Principal investigator, 2158079038, elgartenc@chop.edu

    Recruiting

Other trials in United States

About this study

Official title: Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases

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.

Being tested
Depletion of CD3/CD19 in an autologous stem cell transplant
Medical subject headings
Lupus Erythematosus, Systemic, Scleroderma, Systemic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Depletion of CD3/CD19 in an autologous stem cell transplantBiological

    Also called CD3/CD19 depletion using cliniMACs device

    The purpose of this study is to determine the safety and feasibility of CD3/CD19 depleted autologous stem cell transplant for the treatment of life threatening autoimmune disease. We will perform CD3/CD19 depletion using the CliniMACs device as a means of purging autoreactive T and B cells from the transfused autologous stem cell product, while retaining some immune function, namely natural killer cells and monocytes in the product.

Study groups in NCT05029336
Study groupRoleWhat they receive
CD3/CD19 depleted ASCTExperimentalThe test article is autologous stem cell transplant with a CD3/CD19-depleted stem cell product.
Receives: Biological: Depletion of CD3/CD19 in an autologous stem cell transplant

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Two-year progression free survival

    Survival without evidence of relapse or disease progression

    Measured: 2 years

Secondary outcomes (11)

  1. Disease-specific response/progression endpoints: SSc cohort

    • Pulmonary function: Change in forced vital capacity (FVC), total lung capacity (TLC) or diffusing capacity of the lung for carbon monoxide (DLCO) > 10%

    Measured: 24 months following transplant

  2. Disease-specific response/progression endpoints: SSc cohort

    • Skin condition: An improvement is indicated by a decrease on modified Rodan Skin Score (mRSS) of > 5 points

    Measured: 24 months following transplant

  3. Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort

    • Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) < 4

    Measured: 24 months following transplant

  4. Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort

    • Complete remission off therapy (BILAG D/E only or SLEDAI=0 and no SLE treatment except hydroxychloroquine)

    Measured: 24 months following transplant

  5. Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort

    • Serologic response: presence of positive ANA, anti-dsDNA and anticardiolpin antibody titers

    Measured: 24 months following transplant

  6. Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort

    • Serologic response: abnormal complement C3 and C4 levels

    Measured: 24 months following transplant

  7. Overall survival (OS)

    Overall survival will be considered as time from transplant to death from any cause

    Measured: 2 and 5 years following transplant

  8. Event free survival (EFS)

    Events include death, and significant persistent organ damage

    • 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)

    Measured: 2 and 5 years following transplant

  9. 100 day treatment-related mortality

    Defined as death from non-disease related causes in the 100 days from stem cell infusion

    Measured: 100 days from stem cell infusion

  10. Time to engraftment

    • Achieving an absolute neutrophil count (ANC) > 500 cells/uL and an unsupported platelet count of > 20,000 cells/uL for three consecutive days

    Measured: 3 days

  11. Change in quality of life

    • 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.

    Measured: prior to autologous stem cell transplant (ASCT) until 5 years post-transplant

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 Aug 2021
  2. Study startMar 2026 (estimated)
  3. Primary completionDec 2027 (estimated)
  4. Study completionMay 2031 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
open label single arm pilot study
Masking
None (open label)
Enrolment
20 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05029336
Sponsor's protocol ID
19-016604
Status last verified
Sep 2025
First submitted
24 Aug 2021
Last updated
14 Oct 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Depletion of CD3/CD19 in an autologous stem cell transplant in people with Systemic Lupus Erythematosus and Systemic Sclerosis, run by Stephan Grupp MD PhD at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 14 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Patricia M Hankins ((215) 590-5168, HANKINSP@chop.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Two-year progression free survival (time frame: 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05029336.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2025.

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