Intestinal TransplantationPhase 1NCT04804891
Using T-Cell Alloreactivity and Chimerism to Guide Immunosuppression Minimization in Intestinal Transplantation
A Phase 1 study testing Cell Therapy in people with Intestinal Transplantation, run by Columbia University at 1 site in United States.
Trial summary
The purpose of this study is to investigate the safety and feasibility of giving intestinal transplant patients CD34+ stem cells (the cells that make all the types of blood cells) obtained from their organ donor's bone marrow. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Intestinal Transplantation
- What is being tested?
- Cell Therapy
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Oct 2021to Dec 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- All patients actively listed as candidates for intestinal or multi-visceral transplant at the study site; while all patients who are actively listed in United Network for Organ Sharing (UNOS) for intestinal and/or multi-visceral transplantation, including those who have previously received a multi-visceral transplant and are re-listed, are eligible for participation, the following are examples of listing criteria suitable for enrollment in this clinical trial:
- Short Bowel Syndrome (SBS) due to:
- Trauma (multiple resections/explorations and/or vascular abdominal trauma superior mesenteric artery (SMA) / superior mesenteric vein (SMV) injuries)
- Gastroschisis
- Volvulus
- Necrotizing Enterocolitis
Show all 23Show fewer
- Intestinal Atresia
- Crohn's Disease
- Hirschprung's Disease
- Chronic Intestinal Pseudo-Obstruction
- Malabsorption:
- Microvillus Inclusion Disease
- Tufting Enteropathy
- Complete portomesenteric thrombosis with cirrhosis
- Slow-growing, low-malignancy potential tumors infiltrating mesenteric root:
- Gardner's Syndrome
- Familial Adenomatous Polyposis
- Desmoid Tumor with Intra-Abdominal Infiltration
- Endocrine Tumors
- Re-transplant candidates who lost the first graft to rejection or patients who have higher risk of toxicity from chronic long term immunosuppression (i.e., patients with chronic kidney disease)
- Patient commits to planned follow up at a study site for the 48-month duration of study procedures
- Age ≥18 years old and ≤65 years old
- Subjects or capable of signing the informed consent document themselves
Who cannot join
- Active systemic infection with hemodynamic instability and/or sepsis
- Patients with known immunodeficiency syndrome
- Carcinoma with metastasis (except neuro-endocrine tumors, even in the presence of metastasis these patients may undergo multivisceral/cluster transplantation)
- Severe cardiovascular and/or respiratory instability, as defined by requirement of pressors or ventilator
- Severe cerebral edema, with radiologic findings of effaced sulci and/or herniation
- Poorly controlled hypertension (systolic blood pressure > 170 on at least 2 occasions), diabetes mellitus (HbA1c > 8), or uncontrollable seizure disorders
Show all 21Show fewer
- Age > 65 years
- Documented history of non-compliance with medical therapy and follow-up
- Substance addiction in the last six months
- Psychosocial Instability: absence of a consistent reliable social support system
- Significant or active psychiatric disorder associated with the inability to cooperate or comply with medical therapy
- In the judgement of the clinical team, severely limited functional status with poor rehabilitation potential
- Multi-organ failure and preceding CD34+ infusion
- Pre formed panel reactive antibodies (PRA) mean fluorescein intensity (MFI) > 5000 by Luminex
- Patients who are pregnant or breast-feeding or intend to get pregnant during the study period
- Patients who have developed moderate or severe rejection before post-transplant day 11
- Vulnerable populations, such as incarcerated or institutionalized individuals
- Subjects with clinical features suggestive of GVHD
- Subjects who are hemodynamically unstable (i.e., requiring vasopressor support)
- Female subjects of childbearing age and male patients who are not using and/or unwilling to use an effective method of birth control for the duration of the trial activities
- History of previous hematopoietic progenitor cell (HPC) infusion or transplant of any kind. Note: Human leukocyte antigen (HLA) mismatch will not be one of the exclusion criteria
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Columbia University Irving Medical Center/NYP
New York, New York, 10032
Clinical Research Core, 212-305-3839, tk2388@cumc.columbia.edu
About this study
The purpose of this study is to investigate the safety and feasibility of giving intestinal transplant patients CD34+ stem cells (the cells that make all the types of blood cells) obtained from their organ donor's bone marrow. The goal of this is to develop a post-transplant treatment strategy that controls rejection while reducing the high risk of infection and malignant disease associated with the high levels of immunosuppression medication(s) that intestinal and multi-organ transplant patients must take. Infusion of bone marrow cells from the same donor of the transplanted organ(s) could promote a state called "mixed chimerism" in which both donor cells and recipient cells coexist in the body with the ultimate goal of minimizing the amount of immunosuppression medication(s) needed.
Read the detailed description
Abdominal trauma, congenital abnormalities and ischemic injury cause intestinal damage that prevents the digestion and absorption of fluids and nutrients essential for life. Intestinal transplantation is life-saving for patients with complications related to the administration of intravenous nutrients. Approximately 100-160 intestinal transplants (ITx) are performed in the US annually. However, patient survival rates are far from optimal, due to high rejection rates resulting from an immune attack of the recipient against the donor, termed host-vs-graft (HVG) reactivity The high levels of global immunosuppression used to prevent rejection come with a high risk of infections and malignant disease (i.e. lymphoma). Thus, there is an urgent need for a well-tolerated treatment strategy that controls rejection while reducing these risks. Immune tolerance, in which the immune system regards the donor as "self" so that long-term graft acceptance is achieved without life-long immunosuppression, would accomplish this goal . Infusion of bone marrow cells from the same donor of the solid organs could promote a state called "mixed chimerism" in which both donor cells and recipient cells coexist in the body. Mixed chimerism has been shown to induce tolerance to the transplanted organ in animal models and in patients receiving kidney transplants.
The investigators propose studies to promote tolerance induction in intestinal transplant recipients by administering donor bone marrow stem cells to promote lasting mixed chimerism. The investigators' proposal builds on their demonstration that mixed chimerism commonly occurs in intestinal transplant (ITx) recipients without bone marrow transplant, and that its presence correlates with reduced rejection rates. However, this mixed chimerism is not permanent. The investigators have discovered that there are bone marrow stem cells in the donor intestinal grafts and that some of these survive and enter the bone marrow of the recipient. This process is facilitated by a phenomenon called a "lymphohematopoietic graft-vs-host responses (LGVHR)", in which T lymphocytes from the ITx donor attack recipient blood-forming cells to make "space" for their own establishment in the bone marrow, but do not induce GVHD. The investigators have also obtained evidence that this immune response suppresses rejection of the graft.
- Conditions
- Intestinal Transplantation
- Being tested
- Cell Therapy
- Keywords
- CD34+ stem cells, Chimerism, Bone marrow stells
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cell TherapyBiological
Infusion of containing 1x106/kg CD34+ cells from donor bone marrow selected using the CliniMACS® CD34 Reagent System.
| Study group | Role | What they receive |
|---|---|---|
| Cell Therapy | Experimental | Patients will receive an infusion containing 1x106/kg CD34+ cells. No more than 104 CD34+ T cells per kg recipient weight will be included in the infusion. Cadaveric donor CD34 cell infusion will occur at any time between post-operative day 11 to day 13 following transplantation. Receives: Biological: Cell Therapy |
| Control | No intervention | Patients who do not consent to receive donor CD34 cell infusion or whose donor family declines consent for research use of donor bone marrow will receive their usual standard of care. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total number of participants with moderate to severe GVHD
Total number of participants with moderate to severe (at least Grade II) graft-versus-host disease (GVHD) will be monitored.
Measured: Up to 4 years after transplantation
Secondary outcomes (6)
Graft survival rate
Percentage of individuals with graft survival.
Measured: Up to 1 month after transplantation
Retention rate
Percentage of individuals with retention from any cause.
Measured: Up to 1 month after transplantation
Graft survival rate
Percentage of individuals with graft survival.
Measured: Up to 1 year after transplantation
Retention rate
Percentage of individuals with retention from any cause.
Measured: Up to 1 year after transplantation
Graft survival rate
Percentage of individuals with graft survival.
Measured: Up to 3 years after transplantation
Retention rate
Percentage of individuals with retention from any cause.
Measured: Up to 3 years after transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2021
- Study start22 Oct 2021
- Primary completionDec 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Intestinal TransplantationStudies of the same condition, open and under way.
- More trials run by Columbia UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Columbia University
- Sponsor type
- University, hospital or other
- Collaborators
- Ossium Health, Inc.
- Responsible party
- Sponsor
- Investigators
- Tomoaki Kato, MD, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04804891
- Sponsor's protocol ID
- AAAS8908
- Status last verified
- Mar 2026
- First submitted
- 16 Mar 2021
- Last updated
- 1 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 1 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Using T-Cell Alloreactivity and Chimerism to Guide Immunosuppression Minimization in Intestinal Transplantation
- ClinicalTrials.gov ID
- NCT04804891
- Acronym / sponsor ID
- AAAS8908
- Conditions
- Intestinal Transplantation
- Keywords
- CD34+ stem cells, Chimerism, Bone marrow stells
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Columbia UniversityUniversity, hospital or other
- Collaborators
- Ossium Health, Inc.
- Study start
- 22 Oct 2021 (actual)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 18 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to investigate the safety and feasibility of giving intestinal transplant patients CD34+ stem cells (the cells that make all the types of blood cells) obtained from their organ donor's bone marrow. The goal of this is to develop a post-transplant treatment strategy that controls rejection while reducing the high risk of infection and malignant disease associated with the high levels of immunosuppression medication(s) that intestinal and multi-organ transplant patients must take. Infusion of bone marrow cells from the same donor of the transplanted organ(s) could promote a state called "mixed chimerism" in which both donor cells and recipient cells coexist in the body with the ultimate goal of minimizing the amount of immunosuppression medication(s) needed.
Detailed description
Abdominal trauma, congenital abnormalities and ischemic injury cause intestinal damage that prevents the digestion and absorption of fluids and nutrients essential for life. Intestinal transplantation is life-saving for patients with complications related to the administration of intravenous nutrients. Approximately 100-160 intestinal transplants (ITx) are performed in the US annually. However, patient survival rates are far from optimal, due to high rejection rates resulting from an immune attack of the recipient against the donor, termed host-vs-graft (HVG) reactivity The high levels of global immunosuppression used to prevent rejection come with a high risk of infections and malignant disease (i.e. lymphoma). Thus, there is an urgent need for a well-tolerated treatment strategy that controls rejection while reducing these risks. Immune tolerance, in which the immune system regards the donor as "self" so that long-term graft acceptance is achieved without life-long immunosuppression, would accomplish this goal . Infusion of bone marrow cells from the same donor of the solid organs could promote a state called "mixed chimerism" in which both donor cells and recipient cells coexist in the body. Mixed chimerism has been shown to induce tolerance to the transplanted organ in animal models and in patients receiving kidney transplants.
The investigators propose studies to promote tolerance induction in intestinal transplant recipients by administering donor bone marrow stem cells to promote lasting mixed chimerism. The investigators' proposal builds on their demonstration that mixed chimerism commonly occurs in intestinal transplant (ITx) recipients without bone marrow transplant, and that its presence correlates with reduced rejection rates. However, this mixed chimerism is not permanent. The investigators have discovered that there are bone marrow stem cells in the donor intestinal grafts and that some of these survive and enter the bone marrow of the recipient. This process is facilitated by a phenomenon called a "lymphohematopoietic graft-vs-host responses (LGVHR)", in which T lymphocytes from the ITx donor attack recipient blood-forming cells to make "space" for their own establishment in the bone marrow, but do not induce GVHD. The investigators have also obtained evidence that this immune response suppresses rejection of the graft.
Study design
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cell Therapy | Experimental | Biological: Cell TherapyPatients will receive an infusion containing 1x106/kg CD34+ cells. No more than 104 CD34+ T cells per kg recipient weight will be included in the infusion. Cadaveric donor CD34 cell infusion will occur at any time between post-operative day 11 to day 13 following transplantation. |
| Control | No intervention | –Patients who do not consent to receive donor CD34 cell infusion or whose donor family declines consent for research use of donor bone marrow will receive their usual standard of care. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (23 listed)
- All patients actively listed as candidates for intestinal or multi-visceral transplant at the study site; while all patients who are actively listed in United Network for Organ Sharing (UNOS) for intestinal and/or multi-visceral transplantation, including those who have previously received a multi-visceral transplant and are re-listed, are eligible for participation, the following are examples of listing criteria suitable for enrollment in this clinical trial:
- Patient commits to planned follow up at a study site for the 48-month duration of study procedures
- Age ≥18 years old and ≤65 years old
- Subjects or capable of signing the informed consent document themselves
Key exclusion criteria (21 listed)
- Active systemic infection with hemodynamic instability and/or sepsis
- Patients with known immunodeficiency syndrome
- Carcinoma with metastasis (except neuro-endocrine tumors, even in the presence of metastasis these patients may undergo multivisceral/cluster transplantation)
- Severe cardiovascular and/or respiratory instability, as defined by requirement of pressors or ventilator
- Severe cerebral edema, with radiologic findings of effaced sulci and/or herniation
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cell TherapyInfusion of containing 1x106/kg CD34+ cells from donor bone marrow selected using the CliniMACS® CD34 Reagent System. | Biological | Cell Therapy |
Primary outcome
Total number of participants with moderate to severe GVHD
Time frame: Up to 4 years after transplantation
Total number of participants with moderate to severe (at least Grade II) graft-versus-host disease (GVHD) will be monitored.
6 secondary outcomes
Graft survival rate
Time frame: Up to 1 month after transplantation
Retention rate
Time frame: Up to 1 month after transplantation
Graft survival rate
Time frame: Up to 1 year after transplantation
Retention rate
Time frame: Up to 1 year after transplantation
Graft survival rate
Time frame: Up to 3 years after transplantation
Retention rate
Time frame: Up to 3 years after transplantation
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Apr 2026.
- Individual participant dataSharing planned: Undecided
Related trials for Intestinal Transplantation
RecruitingPhase 1/2
Safety, Dosage, and Microbiome Engraftment Study of MGT-006 for Ulcerative Colitis (ROOT Study)
Ulcerative Colitis (UC), Ulcerative Colitis, Active Moderate
Not yet recruiting
Fecal Microbiota Transplantation (FMT) to Slow Biological Aging
Aging, Healthy, Aging and Cognitive Health
Not yet recruiting
Investigation of the Gut-Brain Axis in Alcohol Use Disorder
Alcohol Use Disorder (AUD), Alcohol Abstinence
RecruitingPhase 1/2
Safety and Efficacy of Lyophilized Fecal Microbiota Transplantation Via Capsules for Intestinal Multidrug-resistant Bacteria Decolonization
Colonization, Asymptomatic
RecruitingObservational study
Determine the Prevalence of Exocrine Pancreatic Insufficiency (EPI) in Pediatric and Adult Participants With Alagille Syndrome After Liver Transplantation
Exocrine Pancreatic Insuficiency
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Tomoaki Kato, MD principal investigator
Columbia University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Cell Therapy in people with Intestinal Transplantation, run by Columbia University 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 23 inclusion and 21 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: looking for participants now, as of the sponsor's last update on 1 Apr 2026. 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Research Core (212-305-3839, tk2388@cumc.columbia.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Total number of participants with moderate to severe GVHD (time frame: Up to 4 years after transplantation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 1 Apr 2026.