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A Safety and Efficacy Study of FCR001 in Adults With Rapidly Progressive Diffuse Cutaneous Systemic Sclerosis (FREEDOM-3)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT05098145
Recruitment Status : Active, not recruiting
First Posted : October 28, 2021
Last Update Posted : May 16, 2023
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Diffuse Cutaneous Systemic Sclerosis Biological: FCR001 Phase 1 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Single-arm, Multi-center, Open-label Proof of Concept Safety and Efficacy Study of FCR001 Cell-based Therapy in Adults With Rapidly Progressive Diffuse Cutaneous Systemic Sclerosis at Risk for Organ Failure
Actual Study Start Date : November 24, 2021
Estimated Primary Completion Date : November 2026
Estimated Study Completion Date : February 2027

Arm Intervention/treatment
Experimental: 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.
Biological: FCR001
Enriched hematopoietic stem cell infusion

Primary Outcome Measures :
  1. Incidence of recipient adverse events (AEs) [ Time Frame: From day before infusion to 60 months ]
  2. Incidence of recipient serious adverse events (SAEs) [ Time Frame: From day before infusion to 60 months ]
  3. Occurrence of Graft versus Host Disease (GvHD) [ Time Frame: From infusion to 60 months ]
  4. Time to neutrophil recovery [ Time Frame: From infusion to 28 days ]
  5. Time to platelet recovery [ Time Frame: From infusion to 28 days ]

Secondary Outcome Measures :
  1. Percent donor whole blood chimerism [ Time Frame: From infusion to 60 months ]
  2. Percentage of donor T-cell chimerism [ Time Frame: From infusion to 60 months ]
  3. Incidence of donor AEs [ Time Frame: From donation to 12 months ]
  4. Incidence of donor SAEs [ Time Frame: From donation to 12 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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 Exclusion Criteria (Recipient):

  1. Rheumatic disease, other than systemic sclerosis
  2. FVC < 45% of predicted or hemoglobin-adjusted DLco < 45% of predicted
  3. Pulmonary arterial hypertension (PAH)
  4. 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
  5. Estimated GFR < 40 mL/min
  6. Previous treatment with cyclophosphamide, as defined by combination of prior oral and intravenous cyclophosphamide > 9 months, independent of dose
  7. Corticosteroid therapy at prednisone equivalent doses of greater than 10 mg/day, or more than two pulses for concurrent illnesses within prior 12 months
  8. Uncontrolled hypertension
  9. Active gastric antral vascular ectasia, also known as "watermelon stomach"
  10. Use of scleroderma specific therapies beyond protocol specified washout period, except for PDE-5 inhibitors for Raynaud's phenomenon and digital ulcers
  11. Previous history of bone marrow transplant, total lymphoid irradiation, solid organ transplant, autologous or allogeneic hematopoietic progenitor or mesenchymal stem cell transplant
  12. Presence of donor-specific antibodies
  13. Body mass index < 18 or > 35 kg/m^2

Key Exclusion Criteria (Donor): Biologically unrelated female donor to male recipient

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05098145

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United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48109
Sponsors and Collaborators
Talaris Therapeutics Inc.
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Study Director: Joel Weinthal, MD Talaris Therapeutics
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Responsible Party: Talaris Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT05098145    
Other Study ID Numbers: FCR001C2201 (FREEDOM-3)
First Posted: October 28, 2021    Key Record Dates
Last Update Posted: May 16, 2023
Last Verified: May 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Talaris Therapeutics Inc.:
Stem cell therapy
Severe scleroderma
Additional relevant MeSH terms:
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Scleroderma, Systemic
Scleroderma, Diffuse
Pathologic Processes
Connective Tissue Diseases
Skin Diseases