Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age8-25
SponsorStephan Grupp MD PhD
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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 design
Design model
Single group
Treatments tested in this trial
Depletion of CD3/CD19 in an autologous stem cell transplant
Biological/VaccineThe 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.
Treatment groups
20 Participants
are divided into 1 treatment groupGroup A: CD3/CD19 depleted ASCTExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Two-year progression free survival
Survival without evidence of relapse or disease progression
Time frame
2 yearsSecondary outcomes
1
Disease-specific response/progression endpoints: SSc cohort
o Pulmonary function: Change in forced vital capacity (FVC), total lung capacity (TLC) or diffusing capacity of the lung for carbon monoxide (DLCO) \> 10%
Time frame
24 months following transplant2
Disease-specific response/progression endpoints: SSc cohort
o Skin condition: An improvement is indicated by a decrease on modified Rodan Skin Score (mRSS) of \> 5 points
Time frame
24 months following transplant3
Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort
o Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) \< 4
Time frame
24 months following transplant4
Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort
o Complete remission off therapy (BILAG D/E only or SLEDAI=0 and no SLE treatment except hydroxychloroquine)
Time frame
24 months following transplantSponsors and contacts
Click on the lead sponsor to view all of their trials.
Stephan Grupp MD PhD
Lead sponsor
Children's Hospital of Philadelphia
Sponsor institution
Source ClinicalTrials.gov