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

NCT05098145 | Withdrawn Phase 1 DMC FDA Drug

• 1 other identifier: FCR001C2201 (FREEDOM-3)
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Diffuse Cutaneous Systemic Sclerosis

Keywords

Stem cell therapy; Scleroderma; Severe scleroderma; Allogeneic; Transplant

Outcome Measures

Primary Outcomes (5)

• 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

• Time to platelet recovery

  From infusion to 28 days

Secondary Outcomes (4)

• Percent donor whole blood chimerism

  From infusion to 60 months

• 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

Study Arms (1)

FCR001

EXPERIMENTAL

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.

Biological: FCR001

Interventions

FCR001 BIOLOGICAL

Enriched hematopoietic stem cell infusion

FCR001

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 18 and \< 70 years
• Diagnosis of diffuse cutaneous systemic sclerosis
• Disease duration \< 5 years from first non-Raynaud's phenomenon symptom
• Received at least one immunosuppressant in the past to treat the systemic sclerosis (SSc) or currently on an immunosuppressive therapy
• Modified Rodnan Skin Score \> 15 and \< 40
• 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.

You may not qualify if:

• Use of investigational drugs within 30 days (or within 5 drug half-lives) of signing informed consent
• Pregnant or nursing (lactating) woman
• 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
• 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
• Known bone marrow aplasia
• Rheumatic disease, other than systemic sclerosis
• FVC \< 45% of predicted or hemoglobin-adjusted DLco \< 45% of predicted
• Pulmonary arterial hypertension (PAH)
• An LVEF \< 50% by echocardiogram or clinical evidence of significant CHF (New York Heart Association Class III or IV) or symptomatic cardiac disease or uncontrolled clinically significant arrhythmias
• Estimated GFR \< 40 mL/min
• Previous treatment with cyclophosphamide, as defined by combination of prior oral and intravenous cyclophosphamide \> 9 months, independent of dose
• Corticosteroid therapy at prednisone equivalent doses of greater than 10 mg/day, or more than two pulses for concurrent illnesses within prior 12 months
• Uncontrolled hypertension
• Active gastric antral vascular ectasia, also known as "watermelon stomach"
• Use of scleroderma specific therapies beyond protocol specified washout period, except for PDE-5 inhibitors for Raynaud's phenomenon and digital ulcers

Sponsors & Collaborators

• Talaris Therapeutics Inc.: lead

Study Sites (1)

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

MeSH Terms

Conditions

Scleroderma, Diffuse

Condition Hierarchy (Ancestors)

Scleroderma, Systemic; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Study Officials

• Joel Weinthal, MD

  Talaris Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 20, 2021
• First Posted: October 28, 2021
• Study Start: November 24, 2021
• Primary Completion (Estimated): November 1, 2026
• Study Completion (Estimated): February 1, 2027
• Last Updated: October 12, 2023

Record last verified: 2023-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)