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
Qualifiers
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
Treatments tested in this trial
- Depletion of CD3/CD19 in an autologous stem cell transplant
Treatment groups
20 Participants
are divided into 1 treatment groupSponsors and collaborators
Stephan Grupp MD PhD
Lead sponsor
Children's Hospital of Philadelphia
Sponsor institution
Source ClinicalTrials.gov