15 Participants Needed

Stem Cell Transplant for Crohn's Disease

YG
DZ
YG
Overseen ByYvette Gonzales, MBA
Age: < 65
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: Cedars-Sinai Medical Center
Must be taking: Immunosuppressants, Corticosteroids
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Trial Summary

Do I need to stop my current medications for the trial?

The trial does not specify if you need to stop your current medications, but it mentions that participants should have an unsatisfactory response to certain immunosuppressive drugs. It's best to discuss your current medications with the trial team.

What data supports the effectiveness of the treatment Peripheral Blood Stem Cell Infusion for Crohn's Disease?

Peripheral Blood Stem Cell Infusion has shown benefits in other conditions, such as faster recovery of blood cells after high-dose chemotherapy, which reduces hospital stays and the need for blood transfusions. This suggests it might help in conditions like Crohn's Disease by promoting quicker recovery and reducing complications.12345

Is stem cell transplant generally safe for humans?

Stem cell transplants, including peripheral blood stem cell infusions, can cause some adverse reactions like vomiting and transient shock, but these are usually manageable and not life-threatening. Serious adverse events are rare, occurring in less than 1% of donors, and there have been no fatalities reported in the studies reviewed.678910

How is the stem cell transplant treatment different from other treatments for Crohn's disease?

Stem cell transplant for Crohn's disease is unique because it involves using the patient's own stem cells to reset or modify the immune system, potentially leading to remission. Unlike standard treatments that mainly focus on managing symptoms, this approach aims to address the underlying immune dysfunction.1112131415

What is the purpose of this trial?

Unfortunately, some patients with Crohn's disease (CD) fail to respond to the best clinical treatments and some only experience temporary benefit. For severe Crohn's disease, there is an experimental treatment called "high dose immunoablation" followed by autologous hematopoietic stem cell transplantation (HSCT). This study removes over active lymphocytes (immunoablation) and replaces them using blood stem cells that have been taken from the patient's own body. The aim of the study is to reset or reprogram the patient's immune system to its state prior to diagnosis.

Research Team

DZ

David Ziring, MD

Principal Investigator

Cedars-Sinai Medical Center

Eligibility Criteria

This trial is for young people aged 13-28 with severe Crohn's Disease, who haven't responded well to at least three immunosuppressive drugs and steroids. They must have active disease, be informed about the risks, and not suitable for surgery or at risk of short bowel syndrome. Pregnant individuals or those unwilling to use contraception are excluded.

Inclusion Criteria

Prepared to undergo additional study procedures as per trial schedule
Accepted by a majority of the members of the combined IBD Center as an appropriate candidate (see Selection description below)
You have tried three different medications to control your condition, along with corticosteroids, but they haven't worked well enough.
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Exclusion Criteria

Pregnancy or unwillingness to use adequate contraception during the study, in women of childbearing age
Males who are not willing to use birth control methods when needed.
Concomitant severe disease: renal: creatinine clearance < 30 mL/min (measured or estimated), cardiac: clinical evidence of refractory congestive heart failure; left ventricular ejection fraction < 40% by cardiac echo; chronic atrial fibrillation necessitating oral anticoagulation; uncontrolled ventricular arrhythmia; pericardial effusion with hemodynamic consequences as evaluated by an experienced echo cardiographer, pulmonary: diffusion capacity <40%, psychiatric disorders including active drug or alcohol abuse, concurrent or recent history of malignant disease (excluding non-melanoma skin cancer), uncontrolled hypertension, defined as resting systolic blood pressure ≥ 140 and/or resting diastolic pressure ≥ 90 despite at least 2 anti-hypertensive agents, any infection with HIV, HTLV-1 or 2, hepatitis viruses, or any other infection the investigators consider a contraindication to participation, other chronic disease causing significant organ failure
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Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Mobilization and Conditioning

Participants undergo stem cell mobilization and conditioning prior to transplantation

4-6 weeks
Multiple visits for mobilization and conditioning procedures

Stem Cell Transplantation

Participants receive autologous hematopoietic stem cell transplantation

1 week
Inpatient stay for transplantation

Post-Transplant Conditioning

Participants receive post-transplant conditioning to support engraftment

2-4 weeks
Inpatient and outpatient visits for monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 months
Regular visits at 0, 2, 4, 6, 12, and 24 months post HSCT

Treatment Details

Interventions

  • Peripheral Blood Stem Cell Infusion
Trial Overview The study tests 'high dose immunoablation' followed by autologous hematopoietic stem cell transplantation (HSCT) in patients with Crohn's Disease. It aims to reset the immune system by removing overactive lymphocytes and replacing them with the patient's own blood stem cells.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: HSCT after mobilization and conditioningExperimental Treatment13 Interventions
Mobilization and leukopheresis allow for stem cell harvest. Then conditioning is provided prior to stem cell transplantation, followed by post-transplant conditioning. Interventions include: 1. Stem cell mobilization 2. Leukopheresis 3. Preparative regimen 4. Peripheral blood stem cell infusion 5. Post-PBSC infusion conditioning

Find a Clinic Near You

Who Is Running the Clinical Trial?

Cedars-Sinai Medical Center

Lead Sponsor

Trials
523
Recruited
165,000+

Findings from Research

The use of peripheral blood stem cells (PBSC) for allogeneic transplantation has significantly increased over the past 9 years, with over 50% of sibling and 25% of unrelated donor transplants utilizing PBSC, primarily due to faster recovery of blood cell production.
Randomized trials indicate that PBSC transplantation offers a survival advantage for patients with advanced leukemia, mainly by reducing transplant-related mortality, although it is associated with a higher risk of chronic graft-versus-host disease.
Allogeneic transplantation using peripheral blood stem cells.Russell, NH., Byrne, JL.[2005]
In a study of 17 patients undergoing high-dose chemotherapy for various cancers, the use of peripheral blood stem cells led to a significantly faster recovery of blood cell counts compared to traditional bone marrow infusion, indicating enhanced efficacy of this treatment approach.
The accelerated recovery resulted in fewer transfusions needed for platelets and red blood cells, reduced fever periods, and earlier hospital discharge, suggesting that peripheral stem cell transplantation could be a safer and more effective alternative to autologous bone marrow transplantation.
[Favorable effect of hematopoietic stem cells isolated from blood on hematologic recovery following high-dosage chemotherapy].Richel, DJ., Baars, JW., Wijngaarden, MJ., et al.[2006]
Peripheral blood stem cell transplantation (PBSCT) offers significant advantages over traditional bone marrow transplants, including faster recovery of blood cells, reduced complications, and cost-effectiveness, making it a preferred option for patients with blood cancers.
Recent advancements in stem cell research and techniques, such as cytokine manipulation and gene therapy, are expanding the potential applications of PBSCT beyond cancer treatment to include certain nonmalignant diseases.
Peripheral Blood Stem Cells: Transplantation and Beyond.Lie, AK., To, LB.[2019]

References

Allogeneic transplantation using peripheral blood stem cells. [2005]
[Favorable effect of hematopoietic stem cells isolated from blood on hematologic recovery following high-dosage chemotherapy]. [2006]
Peripheral Blood Stem Cells: Transplantation and Beyond. [2019]
Collection and use of circulating hematopoietic progenitor cells. [2004]
[Multiple organ failure and disseminated adenoviral infection]. [2010]
Adverse reactions during stem cell infusion in children treated with autologous and allogeneic stem cell transplantation. [2018]
Limiting the daily total nucleated cell dose of cryopreserved peripheral blood stem cell products for autologous transplantation improves infusion-related safety with no adverse impact on hematopoietic engraftment. [2021]
Serious Adverse Events in Related Donors: A Report from the Related Donor Safe Study. [2023]
[Clinical toxicity at the infusion of cryopreserved and thawed peripheral blood stem cell grafts in children]. [2007]
Adverse reactions on day zero of hematopoietic stem cell transplantation: integrative review. [2022]
Autologous haematopoietic stem cell transplantation without CD34+ cell selection in refractory Crohn's disease. [2022]
Immunomodulatory effects of unselected haematopoietic stem cells autotransplantation in refractory Crohn's disease. [2017]
Stem cell transplantation for induction of remission in medically refractory Crohn's disease. [2023]
Stem cells as treatment in inflammatory bowel disease. [2013]
Stem cell transplantation for Crohn's disease. [2008]
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