About This Study


NOW ENROLLING

The FREEDOM-1 Study is a clinical research study of an investigational cell therapy, called FCR001.

The purpose of this study is to learn more about whether FCR001 can prevent the rejection of living donor kidney transplants without the need for lifelong anti-rejection drugs.

FREEDOM-1 is a Phase 3 clinical trial.  It will compare the efficacy and safety of FCR001 treatment to standard anti-rejection treatment.

This study is now enrolling adults who plan to undergo a living donor kidney transplant.  Find out where the study is being offered here.  To request more information, please fill out our contact form here.

Participation in this Study

Will it cost me anything to participate in this study?

It will not cost the donor or recipient anything to participate in the study.  All reasonable travel and accommodation costs for both the donor and the recipient will be covered for all study visits, regardless of whether they are randomized to the control arm or to receive FCR001.  Moreover, the sponsor will cover the cost of all anti-rejection drugs for all study participants (if needed) for up to five years post-transplant.

What does participation in FREEDOM-1 entail for me and my donor?

Study participants and their living donors who enroll in FREEDOM-1 will be randomly assigned as a pair to receive either FCR001 or standard anti-rejection medicines.  Your donor and you will be twice as likely to be assigned to the FCR001 group than to the group that receives standard anti-rejection medicines.

For more details on the FREEDOM-1 Study design, including what donors and recipients can expect in each treatment group, click here.

Am I eligible to participate in FREEDOM-1?

You may qualify for the FREEDOM-1 Study if…

  • You are at least 18 years old
  • Your doctor has recommended that you should have a kidney transplant
  • This would be your first kidney transplant
  • A suitable living kidney donor between ages 18-60 has been identified
  • The living kidney donor is willing to undergo standard mobilization and apheresis procedures to donate his/her stem cells
  • You have not been diagnosed with or treated for any type of cancer

For more information on your eligibility for FREEDOM-1, click here.

Where is a study location near me?

Approximately 15 clinical Centers of Excellence will participate in the FREEDOM-1 Study.  To find a location near you, its enrollment status, and contact information, click here.

How do I learn more about participation in FREEDOM-1?

Click here to submit your questions to learn more about FREEDOM-1.

Background

What is immune tolerance?

Immune tolerance has long been considered to be the “Holy Grail” of organ transplantation. It refers to the ability to train a recipient’s immune system to naturally recognize the donated organ as “self”. Normally, a transplant recipient’s immune system sees a donated organ as foreign and will attack it. To avoid organ rejection, anti-rejection drugs are used to suppress the transplant recipient’s immune system. These drugs must be taken for life and have significant risks and complications. Inducing immune tolerance in a transplant recipient eliminates the need to chronically suppress their immune system to prevent rejection of the donated organ, and avoids the many undesirable side effects associated with these drugs.

What are the benefits of a kidney transplant?

Kidney transplants save lives. For individuals with end stage kidney disease, a transplant provides the chance to regain many aspects of a normal life, starting with freedom from dialysis. In the months following the transplant, recipients can experience dramatic improvements in clinical outcomes and quality of life.

What are anti-rejection drugs and why seek to eliminate them?

Normally, a kidney transplant recipient’s immune system sees the donated kidney as foreign and will attack it–a process called rejection. To prevent this process, anti-rejection medicines are used to suppress the transplant recipient’s immune system. Kidney transplant recipients require a lifelong, daily regimen of anti-rejection drugs. Although anti-rejection medicines are very effective at preventing rejection of kidneys, particularly in the first several years following the transplant, there are multiple side effects and tolerability issues that affect recipients’ ability or willingness to take these medicines, ultimately adversely affecting long-term outcomes for donated kidneys and recipients.

A standard anti-rejection regimen typically includes a high dose immunosuppressant drug administered in the hospital at the time of transplant called induction therapy, followed by lifelong, daily maintenance treatment typically with tacrolimus (brand name Prograf), mycophenolate mofetil (also known as MMF or by the brand names CellCept or Myfortic), and a corticosteroid (usually prednisone). Many kidney transplant recipients take more than 20 pills per day as part of their lifelong anti-rejection regimen. Because these medications suppress the entire immune system, transplant recipients must be very careful to avoid potential infections for as long as they are taking anti-rejection medicines. Recipients must give up certain types of food entirely. Neurological side effects, such as tremors, depression, and impaired cognition are common. Over time, the toxicity of these drugs causes a decline in kidney function, as well as damage to other organs. As kidney function declines, patients will feel less well. The average life of a transplanted kidney is between 12 and 20 years. Roughly 1/3 of living donor kidney transplants and roughly half of deceased donor kidney transplants fail within 10 years. The leading cause of transplant failure is chronic rejection of the kidney (despite treatment with anti-rejection medicines).

Additionally, within ten years after transplant, many patients on anti-rejection medicines have significantly higher rates of high blood pressure, diabetes, high cholesterol, high triglycerides and weight gain, resulting in a higher risk of cardiovascular events. Many patients have to take multiple medicines to manage these life-threatening cardiovascular and metabolic side effects. Patients who take corticosteroids chronically are susceptible to loss of bone density, which increases risk of fractures. Within 15 years, kidney failure and the need for dialysis or another transplant are common. Chronic treatment with anti-rejection medicines has also been linked to a significantly higher risk of certain types of cancer than for the general population. The ongoing cost and decreased quality of life associated with chronic treatment with anti-rejection medicines also leads some recipients to stop taking them, thus increasing their risk of kidney rejection.

Study Overview

What are the benefits of participation in FREEDOM-1?

By participating in FREEDOM-1 you have the opportunity to contribute to advancing the scientific understanding of whether FCR001 treatment could enable living donor kidney transplant recipients to avoid the need for lifelong anti-rejection drugs.

The study sponsor will cover all incremental costs associated with the donor and recipient’s participation in the study, including reimbursing all reasonable travel and accommodation expenses for both donors and recipients. You or your donor will not be paid for participation in the study.

If you are among the volunteers treated with FCR001 in the study, you could potentially benefit by having your need for anti-rejection drugs eliminated.

The study sponsor will cover the cost of anti-rejection medications, if still needed, for up to five years following the transplant.

What is FCR001 cell therapy?

FCR001 is an investigational cell therapy that is comprised of living stem cells obtained from the kidney donor. Stem cells are a unique kind of cell that can generate many other types of cell in the body. These cells are processed and infused in the kidney recipient. The cells then take up residence in the recipient’s bone marrow and generate new immune cells.

The goal of FCR001 treatment is to create a “dual immune system” (part-donor and part-recipient) in the transplant recipient. These two immune systems coexist, recognizing both the recipient’s own body as well as the donated organ as “self”. This is also referred to as achieving “chimerism” in the recipient’s immune system. Achieving durable chimerism in a recipient’s immune system potentially enables long-term immune tolerance of the organ transplanted from the same donor. Ultimately, this could allow the transplant recipient to live free of the daily pill burden typically required to suppress the immune system and prevent organ rejection, and the many complications associated with chronic treatment with anti-rejection medicines.

How does treatment with FCR001 cell therapy differ from typical post-transplant treatment?

Currently, kidney transplant recipients must take lifelong anti-rejection drugs to prevent rejection of the kidney. These drugs are associated with serious side effects, including damage to the transplanted kidney over time. By potentially eliminating the need for anti-rejection drugs, FCR001 offers the potential benefit of avoiding these side effects.

Treatment with FCR001 requires the collection of stem cells from the organ donor a few weeks prior to the transplant. It also requires “conditioning” of the organ recipient five days before the transplant. Learn more about the FCR001 treatment protocol here.

What is the evidence that FCR001 works and is safe?

In a Phase 2 clinical study conducted between 2009 – 2016 at two leading U.S. transplant centers, 26 of 37 participating kidney transplant recipients (70%) treated with FCR001 were able to discontinue all anti-rejection drugs by twelve months after their transplant.  The degree of HLA mismatch (spanning 0/6 to 6/6) between the donor and the recipient did not affect either the safety or the efficacy of the therapy. Every tolerized patient has subsequently remained off anti-rejection drugs, now with a minimum follow-up of three years for all trial subjects, median follow-up of over five years, and the longest follow-up of almost 11 years. Long-term subjects off anti-rejection drugs have shown no evidence of rejection or immune defect, and can be safely and effectively vaccinated.

Overall, the safety profile of FCR001 in the Phase 2 study was deemed acceptable to advance FCR001 into the FREEDOM-1 Study.  Adverse events have been observed in Phase 2, and there can be significant complications associated with FCR001 cellular therapy which you should discuss with your doctor to help you weigh the potential benefits and risks of this investigational therapy.

Patient Early Access Policy

Who do I contact with questions?

If you have additional questions about the FREEDOM-1 Study, please speak with your physician or fill out the contact form at the following link: https://freedom1study.com/contact/

As more information and clinical data become available, Talaris may revise this policy. If you would like to receive regular updates about Talaris please email info@talaristx.com

What are the key consideration for this policy?

  • Whether based on available safety and efficacy data, sufficient evidence is established that the potential benefits to the patient(s) outweighs the potential risks.
  • Whether there is adequate capacity to collect and process the investigational cell therapy to complete the ongoing and planned clinical trials.
  • Whether it will compromise or delay ongoing clinical trials or future access for the broader community.

What is the Patient Early Access Policy?

Talaris is committed to developing single-dose cell therapies that have the potential to free organ transplant recipients of the burdens and toxicities of lifelong immunosuppression, without rejecting their transplant organs; and to restore self-tolerance in individuals with severe autoimmune diseases. To accomplish this, we collaborate with many researchers, individuals living with kidney or autoimmune disease and their family members and caregivers, patient groups, and physicians, and we work closely with regulatory agencies.

Clinical trials serve a vital role in development of new therapeutic options. They are carefully regulated, monitored and conducted in specific populations to assess safety and efficacy.

Currently, access to Talaris investigational therapies is only possible through participation in clinical trials. Completing the necessary clinical trials and obtaining the data needed for review and approval by regulatory agencies is the fastest way towards making Talaris’ cell therapies potentially available to the greatest number of individuals in the future.