Diffuse Cutaneous Systemic Sclerosis
Conditions
Keywords
Stem cell therapy, Scleroderma, Severe scleroderma, Allogeneic, Transplant
Brief summary
This is a multicenter, open-label study to evaluate the safety and tolerability and explore the efficacy of FCR001 cell therapy in adults with rapidly progressive Diffuse Cutaneous Systemic Sclerosis (dcSSc) at risk for organ failure.
Detailed description
The purpose of this multicenter, single-arm study is to evaluate the safety and tolerability and explore the efficacy of FCR001 cell therapy in adults with rapidly progressive dcSSc at risk for organ failure. It consists of 2 years of treatment and 3 years of follow-up, with the primary analysis performed at 24 months. FCR001 is a cell therapy product that is administered by intravenous (IV) infusion, following nonmyeloablative (NMA) conditioning. It consists of mobilized peripheral blood cells, facilitating cells, and αβ T cells. This therapy is designed to induce donor-specific tolerance by establishing sustained chimerism and to protect against graft versus host disease (GvHD), the major impediment for advancing allogeneic hematopoietic stem cell therapy (HSCT) as a potential therapy in patients.
Interventions
BIOLOGICALFCR001
Enriched hematopoietic stem cell infusion
Sponsors
Talaris Therapeutics Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria (Recipients): 1. Age ≥ 18 and \< 70 years 2. Diagnosis of diffuse cutaneous systemic sclerosis 3. Disease duration \< 5 years from first non-Raynaud's phenomenon symptom 4. Received at least one immunosuppressant in the past to treat the systemic sclerosis (SSc) or currently on an immunosuppressive therapy 5. Modified Rodnan Skin Score \> 15 and \< 40 6. Documented evidence of pulmonary or renal involvement by having at least one of the following: a) Pulmonary, both required: i. FVC \> 45% and \< 80% predicted or hemoglobin-adjusted DLco \> 45% and \< 80% predicted AND ii. Interstitial lung disease evidenced by chest high-resolution computed tomography b) Renal: history of renal crisis that is not active at time of screening. Stable serum creatinine (\< 20% increase) must be documented for a minimum of 3 months post-renal crisis at the time of the screening visit. Key Inclusion Criteria (Donors): Age ≥ 18 and \< 60 years Key
Exclusion criteria
(Donor and Recipient): 1. Use of investigational drugs within 30 days (or within 5 drug half-lives) of signing informed consent 2. Pregnant or nursing (lactating) woman 3. Human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg) or hepatitis C virus (HCV) positive. Those with history of HCV infection which was successfully treated and cured may participate 4. History of malignancy (other than localized squamous or basal cell carcinoma of the skin or in-situ cervical cancer without recurrence) or premalignant syndrome within the past 5 years 5. Known bone marrow aplasia Key
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Time to platelet recovery | From infusion to 28 days |
| Incidence of recipient adverse events (AEs) | From day before infusion to 60 months |
| Incidence of recipient serious adverse events (SAEs) | From day before infusion to 60 months |
| Occurrence of Graft versus Host Disease (GvHD) | From infusion to 60 months |
| Time to neutrophil recovery | From infusion to 28 days |
Secondary
| Measure | Time frame |
|---|---|
| Percentage of donor T-cell chimerism | From infusion to 60 months |
| Incidence of donor AEs | From donation to 12 months |
| Incidence of donor SAEs | From donation to 12 months |
| Percent donor whole blood chimerism | From infusion to 60 months |
Countries
United States
Outcome results
None listed