A Safety and Efficacy Study of FCR001 in Adults With Rapidly Progressive Diffuse Cutaneous Systemic Sclerosis

Phase 1

Withdrawn

• Conditions: Diffuse Cutaneous Systemic Sclerosis
• Interventions: Biological: FCR001

• Registration Number: NCT05098145
• Lead Sponsor: Talaris Therapeutics Inc.

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.

Study Timeline

• Study First Submitted: 2021-10-20
• Study First Submitted QC: 2021-10-20
• Study First Posted: 2021-10-28
• Study Start: 2021-11-24
• Status Verified: 2023-10
• Last Update Submitted: 2023-10-10
• Last Update Posted: 2023-10-12
• Primary Completion: 2026-11
• Study Completion: 2027-02

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
FCR001	FCR001	FCR001 is a cryopreserved allogeneic stem cell therapy derived from mobilized peripheral blood cells and delivered as a single infusion with a nonmyeloablative conditioning regimen.

Primary Outcome Measures

Name	Time	Method
Time to neutrophil 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 platelet recovery	From infusion to 28 days

Secondary Outcome Measures

Name	Time	Method
Percent donor whole blood chimerism	From infusion to 60 months
Incidence of donor SAEs	From donation to 12 months
Incidence of donor AEs	From donation to 12 months
Percentage of donor T-cell chimerism	From infusion to 60 months

Trial Locations

Locations (1)

University of Michigan; 🇺🇸 Ann Arbor, Michigan, United States