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Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05029336
Recruitment Status : Recruiting
First Posted : August 31, 2021
Last Update Posted : May 25, 2022
Sponsor:
Information provided by (Responsible Party):
Stephan Grupp MD PhD, Children's Hospital of Philadelphia

Study Description
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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.

Condition or disease Intervention/treatment Phase
Systemic Lupus Erythematosus Systemic Sclerosis Biological: Depletion of CD3/CD19 in an autologous stem cell transplant Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: open label single arm pilot study
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases
Estimated Study Start Date : July 2022
Estimated Primary Completion Date : May 2023
Estimated Study Completion Date : May 2031

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: CD3/CD19 depleted ASCT
The test article is autologous stem cell transplant with a CD3/CD19-depleted stem cell product.
 Biological: Depletion of CD3/CD19 in an autologous stem cell transplant
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 Name: CD3/CD19 depletion using cliniMACs device


Outcome Measures
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Primary Outcome Measures :
  1. Two-year progression free survival [ Time Frame: 2 years ]
    Survival without evidence of relapse or disease progression


Secondary Outcome Measures :
  1. Disease-specific response/progression endpoints: SSc cohort [ Time Frame: 24 months following transplant ]
    o Pulmonary function: Change in forced vital capacity (FVC), total lung capacity (TLC) or diffusing capacity of the lung for carbon monoxide (DLCO) > 10%

  2. Disease-specific response/progression endpoints: SSc cohort [ Time Frame: 24 months following transplant ]
    o Skin condition: An improvement is indicated by a decrease on modified Rodan Skin Score (mRSS) of > 5 points

  3. Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort [ Time Frame: 24 months following transplant ]
    o Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) < 4

  4. Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort [ Time Frame: 24 months following transplant ]
    o Complete remission off therapy (BILAG D/E only or SLEDAI=0 and no SLE treatment except hydroxychloroquine)

  5. Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort [ Time Frame: 24 months following transplant ]
    o Serologic response: presence of positive ANA, anti-dsDNA and anticardiolpin antibody titers

  6. Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort [ Time Frame: 24 months following transplant ]
    o Serologic response: abnormal complement C3 and C4 levels

  7. Overall survival (OS) [ Time Frame: 2 and 5 years following transplant ]
    Overall survival will be considered as time from transplant to death from any cause

  8. Event free survival (EFS) [ Time Frame: 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)


  9. 100 day treatment-related mortality [ Time Frame: 100 days from stem cell infusion ]
    Defined as death from non-disease related causes in the 100 days from stem cell infusion

  10. Time to engraftment [ Time Frame: 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

  11. Change in quality of life [ Time Frame: 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.


Eligibility Criteria
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Ages Eligible for Study:   8 Years to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05029336


Contacts
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Contact: Jessica H Lee, BS 267-425-1935 leej11@chop.edu
Contact: Caitlin Elgarten, MD 2158079038 elgartenc@chop.edu

Locations
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United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Study Coordinator    267-425-1935    leej11@chop.edu   
Contact: Principal investigator    2158079038    elgartenc@chop.edu   
Principal Investigator: Caitlin Elgarten, MD         
Sponsors and Collaborators
Stephan Grupp MD PhD
Investigators
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Principal Investigator: Caitlin Elgarten, MD Children's Hospital of Philadelphia
More Information
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Responsible Party: Stephan Grupp MD PhD, Director of Cancer Immunotherapy Program, Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT05029336    
Other Study ID Numbers: 19-016604
First Posted: August 31, 2021    Key Record Dates
Last Update Posted: May 25, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Lupus Erythematosus, Systemic
Scleroderma, Systemic
Scleroderma, Diffuse
Autoimmune Diseases
 Connective Tissue Diseases
Immune System Diseases
Skin Diseases