Clinical trial NCT05029336: Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases
Official title: Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Systemic Lupus Erythematosus
Systemic Sclerosis
Lead sponsor: Children's Hospital of Philadelphia
Sponsor class: OTHER
First posted: 2021-08-31
Enrollment: 20
Sex: ALL
Minimum age (years): 8
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: open label single arm pilot study
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: Two-year progression free survival
timeFrame: 2 years
Secondary outcomes:
measure: Disease-specific response/progression endpoints: SSc cohort
timeFrame: 24 months following transplant
measure: Disease-specific response/progression endpoints: SSc cohort
timeFrame: 24 months following transplant
measure: Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort
timeFrame: 24 months following transplant
measure: Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort
timeFrame: 24 months following transplant
measure: Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort
timeFrame: 24 months following transplant
measure: Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort
timeFrame: 24 months following transplant
measure: Overall survival (OS)
timeFrame: 2 and 5 years following transplant
measure: Event free survival (EFS)
timeFrame: 2 and 5 years following transplant
measure: 100 day treatment-related mortality
timeFrame: 100 days from stem cell infusion
measure: Time to engraftment
timeFrame: 3 days
measure: Change in quality of life
timeFrame: prior to autologous stem cell transplant (ASCT) until 5 years post-transplant
Other outcomes:
Locations:
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
Study officials:
affiliation: Children's Hospital of Philadelphia
name: Caitlin Elgarten, MD
role: PRINCIPAL_INVESTIGATOR
References: