FCR001 for Scleroderma

(FREEDOM-3 Trial)

Overseen By Clinical Operations

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Talaris Therapeutics Inc.

Must be taking: Immunosuppressive therapy

Must not be taking: Investigational drugs, Corticosteroids

Disqualifiers: Rheumatic disease, Malignancy, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 2 years

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called FCR001 for individuals with rapidly worsening Diffuse Cutaneous Systemic Sclerosis (dcSSc), a serious skin and organ condition. The goal is to determine if this stem cell therapy is safe and effective in improving symptoms. Those diagnosed with dcSSc for less than five years and who have tried immunosuppressive treatments might be suitable candidates, especially if they have lung or kidney issues related to the condition. Participants will receive a single infusion of FCR001, derived from specially prepared blood cells. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the chance to be among the first to receive this new therapy.

Will I have to stop taking my current medications?

The trial protocol does not specify if you must stop taking your current medications, but it mentions a washout period for scleroderma-specific therapies. You may need to discuss your current medications with the trial team to understand any specific requirements.

Is there any evidence suggesting that FCR001 cell therapy is likely to be safe for humans?

Research has shown that FCR001, a type of stem cell treatment, has been tested in studies involving kidney transplants. These studies explore whether it can help the body accept new organs without long-term anti-rejection drugs. However, some serious side effects have occurred. For instance, in one kidney transplant study, a patient died due to complications from graft-versus-host disease (GvHD), where donor cells attack the recipient's body.

While reducing the need for anti-rejection medication offers potential benefits, these risks are significant. Anyone considering joining a trial should carefully consider these risks and discuss them with a healthcare professional. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for rapidly progressive diffuse cutaneous systemic sclerosis, which typically involve immunosuppressive drugs like methotrexate or mycophenolate mofetil, FCR001 offers a unique approach as a stem cell therapy. It's derived from cryopreserved allogeneic stem cells, meaning it uses stem cells from a donor's mobilized peripheral blood, which are then infused into the patient. This method could potentially reset the immune system, offering a more targeted and possibly longer-lasting effect. Researchers are excited about FCR001 because it represents a new mechanism of action that might bring significant improvements in managing this challenging condition.

What evidence suggests that FCR001 cell therapy might be an effective treatment for rapidly progressive Diffuse Cutaneous Systemic Sclerosis?

Research has shown that FCR001 cell therapy, the treatment under study in this trial, is promising because it helps create a "dual immune system" in patients. This means part of the immune system comes from a donor, while the rest remains from the patient. In an earlier study, 72% of people who received FCR001 developed a stable mix of these immune cells, allowing them to stop taking all immune-suppressing drugs. This is important because many patients accepted donor cells without rejection. Although researchers have mainly studied this treatment for kidney transplants, it might also help treat conditions like diffuse cutaneous systemic sclerosis (dcSSc) by altering the immune system's response. ^{3 5 6 7 8}

Who Is on the Research Team?

Joel Weinthal, MD

Principal Investigator

Talaris Therapeutics

Are You a Good Fit for This Trial?

Inclusion Criteria

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Nonmyeloablative Conditioning

Participants undergo nonmyeloablative conditioning prior to receiving FCR001 cell therapy

2 weeks

Treatment

Participants receive a single infusion of FCR001 cell therapy

2 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 years

What Are the Treatments Tested in This Trial?

Interventions

• FCR001

How Is the Trial Designed?

Group I: FCR001 Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Talaris Therapeutics Inc.

Lead Sponsor

Trials

12

Recruited

100+

Citations

1. kidneyregistry.com kidneyregistry.com/wp-content/uploads/2024/12/Kriger_Paper.php_.pdf

Immune tolerance via FCR001 cell therapy compared with ... Real-world evidence (RWE) methods were employed to compare the safety and efficacy of non-myeloablative conditioning with FCR001 with standard ...

2. ashpublications.org ashpublications.org/blood/article/140/Supplement%201/12675/489403/Hematopoietic-Stem-Cell-Transplantation-with

Hematopoietic Stem Cell Transplantation with FCR001 Can ... Hematopoietic stem cell transplantation with FCR001 can induce durable red blood cell chimerism in a majority of recipients: results of a Phase II trial.

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37534547/

Immune tolerance via FCR001 cell therapy compared with ... Among the FCR001 recipients, 26 (72%) developed persistent chimerism allowing durable elimination of all immunosuppression. There was no significant difference ...

4. ce.mayo.edu ce.mayo.edu/sites/default/files/media/2022-12/Kidney%20Transplant_10Mar2022.pdf

FCR001 in Kidney Transplant The goal of FCR001 treatment is to induce immune tolerance by creating a “dual immune system” (part donor and part recipient) in the transplant recipient. These ...

5. cellandgene.com cellandgene.com/doc/talaris-cell-therapy-for-organ-rejection-0001

Talaris' Cell Therapy for Organ Rejection The goal of the FREEDOM-1 trial is to assess the potential of FCR001 to induce durable immune tolerance to the transplanted kidney without the ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05098145

NCT05098145 | A Safety and Efficacy Study of FCR001 in ... 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 ...

7. tts2022.org tts2022.org/images/sponsor_logos/Talaris/TAL2112663A_1-pager_Overall_Clinical_Trial_Program_updated_21July2022.pdf

Talaris Therapeutics FCR001 Clinical Program Overview The goal of FCR001 treatment is to induce immune tolerance by creating a “dual immune system” (part donor and part recipient) in the transplant recipient.

8. cgtlive.com cgtlive.com/view/gvhd-death-fcr001-living-donor-kidney-transplant-trial

GvHD Leads to Death in FCR001 Living Donor Kidney ... A patient that received a living donor kidney transplant and was treated with FCR001 as part of the phase 3 FREEDOM-1 trial (NCT03995901) has died.