- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05029336
Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases
Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Estimated)
Phase
- Phase 2
Contacts and Locations
Study Contact
- Name: Jessica H Lee, BS
- Phone Number: 267-425-1935
- Email: leej11@chop.edu
Study Contact Backup
- Name: Caitlin Elgarten, MD
- Phone Number: 2158079038
- Email: elgartenc@chop.edu
Study Locations
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- Recruiting
- Children's Hospital of Philadelphia
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Contact:
- Study Coordinator
- Phone Number: 267-425-1935
- Email: leej11@chop.edu
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Contact:
- Principal investigator
- Phone Number: 2158079038
- Email: elgartenc@chop.edu
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Principal Investigator:
- Caitlin Elgarten, MD
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: CD3/CD19 depleted ASCT
The test article is autologous stem cell transplant with a CD3/CD19-depleted stem cell product.
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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.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Two-year progression free survival
Time Frame: 2 years
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Survival without evidence of relapse or disease progression
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2 years
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Disease-specific response/progression endpoints: SSc cohort
Time Frame: 24 months following transplant
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o Pulmonary function: Change in forced vital capacity (FVC), total lung capacity (TLC) or diffusing capacity of the lung for carbon monoxide (DLCO) > 10%
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24 months following transplant
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Disease-specific response/progression endpoints: SSc cohort
Time Frame: 24 months following transplant
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o Skin condition: An improvement is indicated by a decrease on modified Rodan Skin Score (mRSS) of > 5 points
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24 months following transplant
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Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort
Time Frame: 24 months following transplant
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o Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) < 4
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24 months following transplant
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Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort
Time Frame: 24 months following transplant
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o Complete remission off therapy (BILAG D/E only or SLEDAI=0 and no SLE treatment except hydroxychloroquine)
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24 months following transplant
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Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort
Time Frame: 24 months following transplant
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o Serologic response: presence of positive ANA, anti-dsDNA and anticardiolpin antibody titers
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24 months following transplant
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Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort
Time Frame: 24 months following transplant
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o Serologic response: abnormal complement C3 and C4 levels
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24 months following transplant
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Overall survival (OS)
Time Frame: 2 and 5 years following transplant
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Overall survival will be considered as time from transplant to death from any cause
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2 and 5 years following transplant
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Event free survival (EFS)
Time Frame: 2 and 5 years following transplant
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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) |
2 and 5 years following transplant
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100 day treatment-related mortality
Time Frame: 100 days from stem cell infusion
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Defined as death from non-disease related causes in the 100 days from stem cell infusion
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100 days from stem cell infusion
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Time to engraftment
Time Frame: 3 days
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• Achieving an absolute neutrophil count (ANC) > 500 cells/uL and an unsupported platelet count of > 20,000 cells/uL for three consecutive days
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3 days
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Change in quality of life
Time Frame: prior to autologous stem cell transplant (ASCT) until 5 years post-transplant
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prior to autologous stem cell transplant (ASCT) until 5 years post-transplant
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Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Caitlin Elgarten, MD, Children's Hospital of Philadelphia
Study record dates
Study Major Dates
Study Start (Estimated)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 19-016604
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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