Graft Versus Host DiseasePhase 1/2GvHDNCT06304025
Sequential Administration of WJ-MSCs for the Treatment of GvHD Refractory to Second Line Treatment
A Phase 1/2 study testing Wharton's jelly mesenchymal stem cells (WJ-MSCs) in people with Graft Versus Host Disease, run by Fundación Oftalmológica de Santander Clínica Carlos Ardila Lulle.
Trial summary
Hematopoietic stem cell transplantation (HSCT) is the treatment of choice for malignant hemopathies, but highlights the limitations of long-term results due to the high toxicity of the procedure and the development of Graft versus Host Disease (GVHD). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Graft Versus Host Disease
- What is being tested?
- Wharton's jelly mesenchymal stem cells (WJ-MSCs)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- No sites listed yet
- Study dates
- Jun 2024to Aug 2025
- 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Aged between 18 and 65 years.
- With a diagnosis of malignant hemopathies, who have undergone allogeneic HSCT and who are diagnosed with acute GVHD refractory to second-line treatment.
- Patients who have received bone marrow and/or peripheral blood as a source of cells.
- Patients who have received cells from a family or unrelated donor
- Myeloablative or non-myeloablative conditioning method.
- Adequate cardiac function without evidence of uncontrolled hypertension, congestive heart failure, angor pectoris, or acute myocardial infarction in the 6 months prior to the process.
- Adequate lung function without evidence of severe obstructive or restrictive lung disease.
- Informed consent signed by the patient.
Who cannot join
- Patients from the Transplant Unit of the FOSCAL.
- Patients with a diagnosis of hemopathy that has not been controlled by the transplant or is progressing at the time of treatment.
- Bacterial, viral, or fungal infection that is not being controlled with adequate treatment.
- Cardiac and/or pulmonary function in uncontrolled altered conditions.
- According to medical criteria, patients who are not in an adequate situation to tolerate the treatment.
- Pregnant women or women at risk of pregnancy due to inadequate contraceptive measures.
About this study
Official title: Pilot Study: Sequential Administration of Allogeneic Mesenchymal Stem Cells Thawed or Expanded in Vitro for the Treatment of Acute Graft-versus-host Disease Refractory to Second Line Treatment
Hematopoietic stem cell transplantation (HSCT) is the treatment of choice for malignant hemopathies, but highlights the limitations of long-term results due to the high toxicity of the procedure and the development of Graft versus Host Disease (GVHD). Conventional treatments for GVHD have limited success rates, and some patients may be refractory to ruxolitinib, a second-line treatment option. As a result, there is a need to explore alternative immuno-modulatory therapies, such as the use of Wharton's jelly mesenchymal stem cells (WJ-MSCs).
The research question aims to investigate the safety and potential benefits of sequentially infusing thawed or expanding allogeneic WJ-MSCs in the treatment of acute GVHD refractory to second-line treatment in patients from the Colombian population. This pilot clinical study is being conducted to address the unmet need for patients who develop GVHD resistant to ruxolitinib.
Read the detailed description
In recent decades, the number of HSCTs has significantly increased, as it is considered the ideal therapeutic approach for a wide range of hematological diseases. However, GVHD is the most common and severe complication following transplantation, and it remains the major limiting factor for the success of HSCT. It has been reported that the incidence of acute GVHD is approximately 50% in patients who receive HSCT from an HLA-matched donor, and it increases in cases of HLA-mismatched donors, despite having prophylactic treatment. Patients who develop GVHD have a poor prognosis, with a survival rate ranging from 5-25% depending on the severity of the disease. This is strongly related to the lack of effective treatments. In addition to the high morbidity and mortality, GVHD represents a significant financial burden for public healthcare systems worldwide. A recent study in 2018 in the United States reported that the cost of complete remission for a 6-month period with ruxolitinib, one of the most commonly used treatments for both acute and chronic GVHD, was $1,187,657 USD.
In this context, the American Society for Blood and Marrow Transplantation considers MSCs as a prominent therapeutic tool for the treatment of GVHD, as they significantly improve both the symptoms of the most frequently affected organs (skin, liver, and intestines) and the treatment response parameters and overall survival. Colombia is not exempt from this issue; the rise of HSCT in our country is evident. Therefore, the development of a novel and effective therapeutic strategy for GVHD is a priority and demands further scientific research.
Finally, with the development of this pilot study, the aim is to lay the groundwork for conducting medium- and large-scale clinical trials in our Colombian population, where clinical studies with this type of therapeutic approach have not yet been conducted.
- Conditions
- Graft Versus Host Disease
- Being tested
- Wharton's jelly mesenchymal stem cells (WJ-MSCs)
- Keywords
- Graft-versus-host Disease, Ruxolitinib, Mesenchymal Stem Cells
- Medical subject headings
- Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Wharton's jelly mesenchymal stem cells (WJ-MSCs)Biological
Four doses of 1x10 6 of thawed allogeneic WJ-MSCs or expanding allogeneic WJ-MSCs / kg at 1, 4, 11, and 18 days
| Study group | Role | What they receive |
|---|---|---|
| Thawed allogeneic WJ-MSCs | Experimental | Administration intravenously of 1x106 cells/kg weight of thawed allogeneic WJ-MSCs Receives: Biological: Wharton's jelly mesenchymal stem cells (WJ-MSCs) |
| Expanding allogeneic WJ-MSCs | Experimental | Administration intravenously of 1x106 cells/kg weight of expanding allogeneic WJ-MSCs Receives: Biological: Wharton's jelly mesenchymal stem cells (WJ-MSCs) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events: safety and tolerability
The trial's primary endpoint is to determine the safety of WJ-MSCs administration; for that, we will assess the number of treatment-related adverse events (AEs) reported according to the Common Terminology Criteria for AE classification and presentation of infectious complications after administration of WJ-MSCs.
Measured: 12 months
Efficacy clinic profile: response of acute GVDH
- Response of acute GVHD refractory to second-line treatment (yes or no).
Measured: 12 months
Efficacy clinic profile : duration of response
- Duration of response/incidence of relapses( time: days or months).
Measured: 12 months
Efficacy clinic profile: decrease in treatments
- Decrease in corticosteroids or concomitant immunosuppressive treatment (dose).
Measured: 12 months
Efficacy biological profile: secretion pattern of soluble factors
- Evolution of the secretion pattern of the following soluble factors: cytokines CK8, Reg3α, Elafina, ST2, INF-γ, TNF-α, IL-12, IL-10, CXCL-9 and CXCL-10 (concentration: mg or μg or pg or others).
Measured: 12 months
Efficacy biological profile: cell populations
- Determination of the following cell populations: T lymphocytes, Naive, B lymphocytes, dendritic cells, and Natural Killer cells in blood pre and post-treatment with WJ-MSCs (percentage).
Measured: 12 months
Efficacy biological profile: ocrrelation of the biological markers with the clinical response
- Correlation of the profile of biological markers with the clinical response (-1 and 1).
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2024
- Study startJun 2024 (estimated)
- Primary completionJun 2025 (estimated)
- Study completionAug 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Graft Versus Host DiseaseStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Experimental (pilot clinical study), randomized, blind, and parallel.
- Masking
- Double blind
- Who is masked
- Participant, Care provider
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fundación Oftalmológica de Santander Clínica Carlos Ardila Lulle
- Sponsor type
- University, hospital or other
- Collaborators
- Instituto Distrital de Ciencia, Biotecnología e Innovación en Salud - IDCBIS
- Responsible party
- Principal investigator, Martha Ligia Arango Rodríguez, Fundación Oftalmológica de Santander Clínica Carlos Ardila Lulle
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06304025
- Sponsor's protocol ID
- GvHD 01
- Acronym
- GvHD
- Status last verified
- Mar 2024
- First submitted
- 19 Jan 2024
- Last updated
- 12 Mar 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study: Sequential Administration of Allogeneic Mesenchymal Stem Cells Thawed or Expanded in Vitro for the Treatment of Acute Graft-versus-host Disease Refractory to Second Line Treatment
- ClinicalTrials.gov ID
- NCT06304025
- Acronym / sponsor ID
- GvHD / GvHD 01
- Conditions
- Graft Versus Host Disease
- Keywords
- Graft-versus-host Disease, Ruxolitinib, Mesenchymal Stem Cells
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Fundación Oftalmológica de Santander Clínica Carlos Ardila LulleUniversity, hospital or other
- Collaborators
- Instituto Distrital de Ciencia, Biotecnología e Innovación en Salud - IDCBIS
- Study start
- Jun 2024 (estimated)
- Primary completion
- Jun 2025 (estimated)
- Study completion
- Aug 2025 (estimated)
- First posted
- 12 Mar 2024 (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.
Hematopoietic stem cell transplantation (HSCT) is the treatment of choice for malignant hemopathies, but highlights the limitations of long-term results due to the high toxicity of the procedure and the development of Graft versus Host Disease (GVHD). Conventional treatments for GVHD have limited success rates, and some patients may be refractory to ruxolitinib, a second-line treatment option. As a result, there is a need to explore alternative immuno-modulatory therapies, such as the use of Wharton's jelly mesenchymal stem cells (WJ-MSCs).
The research question aims to investigate the safety and potential benefits of sequentially infusing thawed or expanding allogeneic WJ-MSCs in the treatment of acute GVHD refractory to second-line treatment in patients from the Colombian population. This pilot clinical study is being conducted to address the unmet need for patients who develop GVHD resistant to ruxolitinib.
Detailed description
In recent decades, the number of HSCTs has significantly increased, as it is considered the ideal therapeutic approach for a wide range of hematological diseases. However, GVHD is the most common and severe complication following transplantation, and it remains the major limiting factor for the success of HSCT. It has been reported that the incidence of acute GVHD is approximately 50% in patients who receive HSCT from an HLA-matched donor, and it increases in cases of HLA-mismatched donors, despite having prophylactic treatment. Patients who develop GVHD have a poor prognosis, with a survival rate ranging from 5-25% depending on the severity of the disease. This is strongly related to the lack of effective treatments. In addition to the high morbidity and mortality, GVHD represents a significant financial burden for public healthcare systems worldwide. A recent study in 2018 in the United States reported that the cost of complete remission for a 6-month period with ruxolitinib, one of the most commonly used treatments for both acute and chronic GVHD, was $1,187,657 USD.
In this context, the American Society for Blood and Marrow Transplantation considers MSCs as a prominent therapeutic tool for the treatment of GVHD, as they significantly improve both the symptoms of the most frequently affected organs (skin, liver, and intestines) and the treatment response parameters and overall survival. Colombia is not exempt from this issue; the rise of HSCT in our country is evident. Therefore, the development of a novel and effective therapeutic strategy for GVHD is a priority and demands further scientific research.
Finally, with the development of this pilot study, the aim is to lay the groundwork for conducting medium- and large-scale clinical trials in our Colombian population, where clinical studies with this type of therapeutic approach have not yet been conducted.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, care provider)
- Number of arms
- 2
- Model description
- Experimental (pilot clinical study), randomized, blind, and parallel.
| Arm | Type | Receives |
|---|---|---|
| Thawed allogeneic WJ-MSCs | Experimental | Biological: Wharton's jelly mesenchymal stem cells (WJ-MSCs)Administration intravenously of 1x106 cells/kg weight of thawed allogeneic WJ-MSCs |
| Expanding allogeneic WJ-MSCs | Experimental | Biological: Wharton's jelly mesenchymal stem cells (WJ-MSCs)Administration intravenously of 1x106 cells/kg weight of expanding allogeneic WJ-MSCs |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Aged between 18 and 65 years.
- With a diagnosis of malignant hemopathies, who have undergone allogeneic HSCT and who are diagnosed with acute GVHD refractory to second-line treatment.
- Patients who have received bone marrow and/or peripheral blood as a source of cells.
- Patients who have received cells from a family or unrelated donor
- Myeloablative or non-myeloablative conditioning method.
Key exclusion criteria (6 listed)
- Patients from the Transplant Unit of the FOSCAL.
- Patients with a diagnosis of hemopathy that has not been controlled by the transplant or is progressing at the time of treatment.
- Bacterial, viral, or fungal infection that is not being controlled with adequate treatment.
- Cardiac and/or pulmonary function in uncontrolled altered conditions.
- According to medical criteria, patients who are not in an adequate situation to tolerate the treatment.
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 |
|---|---|---|
| Wharton's jelly mesenchymal stem cells (WJ-MSCs)Four doses of 1x10 6 of thawed allogeneic WJ-MSCs or expanding allogeneic WJ-MSCs / kg at 1, 4, 11, and 18 days | Biological | Expanding allogeneic WJ-MSCs, Thawed allogeneic WJ-MSCs |
Primary outcome
Incidence of treatment-emergent adverse events: safety and tolerability
Time frame: 12 months
The trial's primary endpoint is to determine the safety of WJ-MSCs administration; for that, we will assess the number of treatment-related adverse events (AEs) reported according to the Common Terminology Criteria for AE classification and presentation of infectious complications after administration of WJ-MSCs.
Efficacy clinic profile: response of acute GVDH
Time frame: 12 months
• Response of acute GVHD refractory to second-line treatment (yes or no).
Efficacy clinic profile : duration of response
Time frame: 12 months
• Duration of response/incidence of relapses( time: days or months).
Efficacy clinic profile: decrease in treatments
Time frame: 12 months
• Decrease in corticosteroids or concomitant immunosuppressive treatment (dose).
Efficacy biological profile: secretion pattern of soluble factors
Time frame: 12 months
• Evolution of the secretion pattern of the following soluble factors: cytokines CK8, Reg3α, Elafina, ST2, INF-γ, TNF-α, IL-12, IL-10, CXCL-9 and CXCL-10 (concentration: mg or μg or pg or others).
Efficacy biological profile: cell populations
Time frame: 12 months
• Determination of the following cell populations: T lymphocytes, Naive, B lymphocytes, dendritic cells, and Natural Killer cells in blood pre and post-treatment with WJ-MSCs (percentage).
Efficacy biological profile: ocrrelation of the biological markers with the clinical response
Time frame: 12 months
• Correlation of the profile of biological markers with the clinical response (-1 and 1).
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Mar 2024.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Wharton's jelly mesenchymal stem cells (WJ-MSCs) in people with Graft Versus Host Disease, run by Fundación Oftalmológica de Santander Clínica Carlos Ardila Lulle.
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 8 inclusion and 6 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: status not confirmed, as of the sponsor's last update on 12 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
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?
Results are not available in the source record.
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 treatment-emergent adverse events: safety and tolerability (time frame: 12 months). The study lists 7 primary outcomes in all. 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 12 Mar 2024.