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Diffuse Cutaneous Systemic SclerosisPhase 1/2FREEDOM-3NCT05098145

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

A Phase 1/2 study testing FCR001 in people with Diffuse Cutaneous Systemic Sclerosis, run by Talaris Therapeutics Inc. at 1 site in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 70 years
Conditions the study is for
Diffuse Cutaneous Systemic Sclerosis
What is being tested?
FCR001
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
1 siteUnited States
Study dates
24 Nov 2021to Feb 2027
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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:
  • 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.

Who cannot join

  • 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
Show all 19Show fewer
  • 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
  • Previous history of bone marrow transplant, total lymphoid irradiation, solid organ transplant, autologous or allogeneic hematopoietic progenitor or mesenchymal stem cell transplant
  • Presence of donor-specific antibodies
  • Body mass index < 18 or > 35 kg/m^2
  • Key Exclusion Criteria (Donor): Biologically unrelated female donor to male recipient

Trial locations and contacts

1 site in 1 country.

United States1 site

  • University of Michigan

    Ann Arbor, Michigan, 48109

Other trials in United States

About this study

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

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.

Read the 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.

Being tested
FCR001
Keywords
Stem cell therapy, Scleroderma, Severe scleroderma, Allogeneic, Transplant
Medical subject headings
Scleroderma, Diffuse

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • FCR001Biological

    Enriched hematopoietic stem cell infusion

Study groups in NCT05098145
Study groupRoleWhat they receive
FCR001ExperimentalFCR001 is a cryopreserved allogeneic stem cell therapy derived from mobilized peripheral blood cells and delivered as a single infusion with a nonmyeloablative conditioning regimen.
Receives: Biological: FCR001

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of recipient adverse events (AEs)

    Measured: From day before infusion to 60 months

  2. Incidence of recipient serious adverse events (SAEs)

    Measured: From day before infusion to 60 months

  3. Occurrence of Graft versus Host Disease (GvHD)

    Measured: From infusion to 60 months

  4. Time to neutrophil recovery

    Measured: From infusion to 28 days

  5. Time to platelet recovery

    Measured: From infusion to 28 days

Secondary outcomes (4)

  1. Percent donor whole blood chimerism

    Measured: From infusion to 60 months

  2. Percentage of donor T-cell chimerism

    Measured: From infusion to 60 months

  3. Incidence of donor AEs

    Measured: From donation to 12 months

  4. Incidence of donor SAEs

    Measured: From donation to 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Oct 2021
  2. Study start24 Nov 2021
  3. Primary completionNov 2026 (estimated)
  4. Study completionFeb 2027 (estimated)

Why it stopped: all clinical development programs terminated by sponsor

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05098145
Sponsor's protocol ID
FCR001C2201 (FREEDOM-3)
Acronym
FREEDOM-3
Status last verified
Oct 2023
First submitted
20 Oct 2021
Last updated
12 Oct 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing FCR001 in people with Diffuse Cutaneous Systemic Sclerosis, run by Talaris Therapeutics Inc. at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 12 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of recipient adverse events (AEs) (time frame: From day before infusion to 60 months). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05098145.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Oct 2023.

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