Graft-Versus-Host Disease(GVHD)Early Phase 1NCT06598332
5-Azacytidine With Steroids for Gastrointestinal GVHD (5-AZA FOR GVHD)
A Early Phase 1 study testing 5-Azacytidine in people with Graft-Versus-Host Disease(GVHD), run by Baylor College of Medicine at 2 sites in United States.
Trial summary
This study aims to evaluate the safety and feasibility of administering AZA in conjunction with steroids as first line therapy for GI GVHD. A risk for patients who have received a transplant from another donor is graft versus host disease (GVHD). Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Graft-Versus-Host Disease(GVHD)
- What is being tested?
- 5-Azacytidine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 2 sitesUnited States
- Study dates
- Aug 2025to 1 May 2029
- Phase
- Early Phase 1
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients 12 years of age or older at time of enrollment.
- Patients experiencing their initial presentation of stage 1 or greater acute LGI GVHD requiring systemic therapy after allogeneic transplant for any malignant or non-malignant indication using any graft/donor source or conditioning intensity.
- Patients can be enrolled with only a clinically established diagnosis. Biopsy of involved organs with acute GVHD is encouraged but is not required and should not delay study entry.
- Patients should not have received systemic immune suppressive therapy for treatment of active GVHD except for a maximum of 72 hours of steroid therapy prior to enrollment. Topical skin and GI corticosteroids (such as budesonide and oral beclomethasone diproprionate) are allowed.
- Informed Consent explained to, understood by and signed by patient/guardian. Patient/guardian given copy of informed consent.
Who cannot join
- Relapsed, progressing or persistent malignancy or evidence of minimal residual disease (MRD) requiring withdrawal of systemic immune suppression.
- Patients with acute GVHD developing after administration of a donor lymphocyte infusion (DLI) for relapse / progression of disease. Patients with acute GVHD after planned donor lymphocyte infusion or planned T cell or NK cell add back are eligible.
- Patients with uncontrolled infections will be excluded. Infections are considered controlled if appropriate therapy has been instituted and, at the time of enrollment, no signs of progression are present. Progression of infection is defined as hemodynamic instability attributable to sepsis, new symptoms, worsening physical signs or radiographic findings attributable to infection. Persisting fever without other signs or symptoms
- Patients receiving other drugs for the treatment of GVHD. Note, GVHD prophylaxis agents (e.g., calcineurin inhibitors) may be continued at local Investigator's discretion.
- Patients on renal replacement therapy.
- Patients requiring continuous supplemental oxygen (O2 requirement >2L/min to maintain peripheral O2 saturation [SpO2] >90%).
Show all 12Show fewer
- Patients with active hepatic sinusoidal obstructive syndrome (SOS) and/or clinical evidence of impaired hepatic function (ascites or encephalopathy related to liver disease)
- Abnormal coagulation parameters (PT > 15 seconds, PTT > 40 seconds, and/or INR >1.5)
- Significant active cardiac disease within the previous 6 months including:
- NYHA class 4 CHF Unstable angina Myocardial infarction
- Known or suspected hypersensitivity to azacytidine.
- Platelets <20 and or absolute neutrophil count (ANC) < 1000.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Houston Methodist Hospital
Houston, Texas, 77030
Texas Children's Hospital
Houston, Texas, 77030
About this study
Official title: Administration of 5-Azacytidine With Steroids for First Line Therapy of Gastrointestinal GVHD
This study aims to evaluate the safety and feasibility of administering AZA in conjunction with steroids as first line therapy for GI GVHD.
A risk for patients who have received a transplant from another donor is graft versus host disease (GVHD). This happens because of differences between the donated cells (graft) and the patient body's cells (host). The new cells from the donor might see the patients body's cells as different and attack them. GVHD can be very serious and cause death. The standard first treatment for GVHD is corticosteroids but not all patients respond and they then have to receive other treatments. In addition, when GHVD involves the gut it can damage stem cells and can cause long term gut problems such as abdominal pain bowel disturbance. In laboratory studies giving a medicine called 5 -azacytidine (AZA) has been able to protect the gut stem cells and help them recover. In this trial the investigators would like to see if AZA can do the same thing when given with steroids in patients with GVHD.
Read more
Right now, doctors and researchers don't know the best treatment for GVHD. Acute GVHD is usually treated using high-dose corticosteroids, but these don't always work well. Even if the GVHD gets better when it involves the gut there can be long term damage to gut stem cells. In the laboratory 5 azacytidine (AZA) has been able to protect gut stem cells and help them recover and the investigators would like to learn if this happens in people too.
AZA has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of patients with leukemias. It has also been used post transplant to try and risk the chance of leukemia coming back and to try and treat GVHD but AZA has not been approved by the FDA for the treatment of acute GVHD.
Read the detailed description
Patients enrolled in this study will receive one cycle of AZA through the vein or as a shot under the skin daily for 5 days. This will start at the same time or within 3 days of starting standard treatment for gut GVHD with steroids
Medical tests before treatment--
Before being treated, patient will receive a series of standard medical tests:
- History and Physical exam
- Blood tests to measure blood cells, kidney and liver function
- A biopsy of the gut to look for GVHD is possible
- Serum pregnancy test for female patients who are of child bearing potential
- GVHD prophylaxis (prevention) medication
- Corticosteroid treatment
- An optional stool research sample
Medical tests during and after treatment--
Patient will receive standard medical tests when getting the GVHD treatment and afterwards:
- History and Physical exams
- Monitoring for GVHD
- Blood tests to measure blood cells, kidney and liver function
- A follow-up biopsy of the gut to look for GVHD is possible
- GVHD prophylaxis (prevention) medication
- Corticosteroid treatment
- Blood tests to measure blood cells, kidney and liver function
- Measurements of the tumor by routine imaging studies 6 weeks after the infusion.
To learn more about the way that AZA may work on gut stem cells an extra amount of blood will be obtained on the day AZA starts, 1, 2, 4, and 8 weeks after the T-cell infusion(s) and then at 3, 6 and 12 months. The amount of blood taken will be based on patient weight with up to a maximum of 60 mL (12 teaspoons) of blood to be obtained at any one time. This volume is considered safe, but may be decreased if patient is anemic (have a low red blood cell count).
If the patient has a repeat gut biopsy to check on their GVHD, investigators will request a sample to be used for research purposes.
Patients will receive supportive care for any acute or chronic toxicities, including blood components or antibiotics, and other intervention as appropriate.
- Conditions
- Graft-Versus-Host Disease(GVHD)
- Being tested
- 5-Azacytidine
- Keywords
- Graft-Versus-Host-Disease, GVHD, 5-AZACYTIDINE
- 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.
5-AzacytidineDrug
Patients will be treated with 5 azacytidine at a dose of 32 mg/m2 SQ or IV. The investigators aim to start AZA on Day 1 of steroids but it can be started up to 72 hours after steroids are started . Patients will be premedicated with antiemetics.
Patients must receive steroids at a minimum dose of prednisone 2 mg/kg/day PO (or methylprednisolone 1.6 mg/kg/day IV) divided into 1-2 daily doses as therapy for acute GVHD. For patients that weigh over 100 kg, maximal starting dose of prednisone will be 200 mg (or methylprednisolone-equivalent).
Steroid Taper for responding patients (Prednisone/Methylprednisolone) Days 1-5 2 mg/kg/day (taper cannot start until 3 days after enrollment) Days 6-10 1.5 mg/kg/day Day 11-15 1.0 mg/kg/day Days 16-20 0.5 mg/kg/day Days 21-28* 0.25 mg/kg/day Days 29-56 Gradual further taper with a goal of reaching < 0.2 mg/kg/day of prednisone (or < 0.16 mg/kg/day of methylprednisolone) by Day 56.
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Patients will receive one cycle of AZA through the vein or as a shot under the skin daily for 5 days. This will start at the same time or within 3 days of starting standard treatment for gut GVHD with steroids. Receives: Drug: 5-Azacytidine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Feasibility rate
The proportion of patients who can tolerate and complete one cycle of AZA (5-day) in combination with steroid among patients who are eligible and receive at least one dose of AZA.
Measured: 30 days after first dose of AZA
Treatment-related adverse event (tAE) rate
The proportion of patients who develop any Grade 3-5 adverse events (per Common Terminology Criteria for Adverse Events [CTCAE] Version 5.0) that are considered probably, or definitely related to AZA or AZA in combination with steroid that occur in the first cycle of combination therapy until 30 days after first dose of AZA.
Measured: 30 days after first dose of AZA
Secondary outcomes (4)
GVHD response rate
The proportion of patients who have complete and partial response in patients who received steroids in combination with azacytidine.
Measured: Day 28
Overall survival (OS)
Overall survival (OS) will be evaluated at 6 months and 12 months after combination therapy initiation. An event for OS is death from any cause.
Measured: 6 months and 12 months after combination therapy initiation
Progression-free survival (PFS)
Progression-free survival (PFS) will be evaluated at 6 months and 12 months after combination therapy initiation. An event for PFS is death from any cause or relapse/progression of the primary disease.
Measured: 6 months and 12 months after combination therapy initiation
Systemic Infections
The incidence of Grade 2 to 3 systemic infections (per BMT CTN Infection MOP), until 30 days after last dose of AZA will be described.
Measured: 30 days after last dose of AZA
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study startAug 2025 (estimated)
- Primary completion1 May 2028 (estimated)
- Study completion1 May 2029 (estimated)
Why it stopped: There is no funding.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Graft-Versus-Host Disease(GVHD)Studies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- The Methodist Hospital Research Institute, Center for Cell and Gene Therapy, Baylor College of Medicine
- Responsible party
- Principal investigator, LaQuisa Hill, Baylor College of Medicine
- Investigators
- Helen Heslop, MD, Principal investigator, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06598332
- Sponsor's protocol ID
- H-55921 5-AZA for GVHD
- Status last verified
- Aug 2025
- First submitted
- 13 Sep 2024
- Last updated
- 22 Sep 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Administration of 5-Azacytidine With Steroids for First Line Therapy of Gastrointestinal GVHD
- ClinicalTrials.gov ID
- NCT06598332
- Acronym / sponsor ID
- H-55921 5-AZA for GVHD
- Conditions
- Graft-Versus-Host Disease(GVHD)
- Keywords
- Graft-Versus-Host-Disease, GVHD, 5-AZACYTIDINE
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: There is no funding.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Collaborators
- The Methodist Hospital Research Institute, Center for Cell and Gene Therapy, Baylor College of Medicine
- Study start
- Aug 2025 (estimated)
- Primary completion
- 1 May 2028 (estimated)
- Study completion
- 1 May 2029 (estimated)
- First posted
- 19 Sep 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.
This study aims to evaluate the safety and feasibility of administering AZA in conjunction with steroids as first line therapy for GI GVHD.
A risk for patients who have received a transplant from another donor is graft versus host disease (GVHD). This happens because of differences between the donated cells (graft) and the patient body's cells (host). The new cells from the donor might see the patients body's cells as different and attack them. GVHD can be very serious and cause death. The standard first treatment for GVHD is corticosteroids but not all patients respond and they then have to receive other treatments. In addition, when GHVD involves the gut it can damage stem cells and can cause long term gut problems such as abdominal pain bowel disturbance. In laboratory studies giving a medicine called 5 -azacytidine (AZA) has been able to protect the gut stem cells and help them recover. In this trial the investigators would like to see if AZA can do the same thing when given with steroids in patients with GVHD.
Right now, doctors and researchers don't know the best treatment for GVHD. Acute GVHD is usually treated using high-dose corticosteroids, but these don't always work well. Even if the GVHD gets better when it involves the gut there can be long term damage to gut stem cells. In the laboratory 5 azacytidine (AZA) has been able to protect gut stem cells and help them recover and the investigators would like to learn if this happens in people too.
AZA has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of patients with leukemias. It has also been used post transplant to try and risk the chance of leukemia coming back and to try and treat GVHD but AZA has not been approved by the FDA for the treatment of acute GVHD.
Detailed description
Patients enrolled in this study will receive one cycle of AZA through the vein or as a shot under the skin daily for 5 days. This will start at the same time or within 3 days of starting standard treatment for gut GVHD with steroids
Medical tests before treatment--
Before being treated, patient will receive a series of standard medical tests:
- History and Physical exam
- Blood tests to measure blood cells, kidney and liver function
- A biopsy of the gut to look for GVHD is possible
- Serum pregnancy test for female patients who are of child bearing potential
- GVHD prophylaxis (prevention) medication
- Corticosteroid treatment
- An optional stool research sample
Medical tests during and after treatment--
Patient will receive standard medical tests when getting the GVHD treatment and afterwards:
- History and Physical exams
- Monitoring for GVHD
- Blood tests to measure blood cells, kidney and liver function
- A follow-up biopsy of the gut to look for GVHD is possible
- GVHD prophylaxis (prevention) medication
- Corticosteroid treatment
- Blood tests to measure blood cells, kidney and liver function
- Measurements of the tumor by routine imaging studies 6 weeks after the infusion.
To learn more about the way that AZA may work on gut stem cells an extra amount of blood will be obtained on the day AZA starts, 1, 2, 4, and 8 weeks after the T-cell infusion(s) and then at 3, 6 and 12 months. The amount of blood taken will be based on patient weight with up to a maximum of 60 mL (12 teaspoons) of blood to be obtained at any one time. This volume is considered safe, but may be decreased if patient is anemic (have a low red blood cell count).
If the patient has a repeat gut biopsy to check on their GVHD, investigators will request a sample to be used for research purposes.
Patients will receive supportive care for any acute or chronic toxicities, including blood components or antibiotics, and other intervention as appropriate.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Drug: 5-AzacytidinePatients will receive one cycle of AZA through the vein or as a shot under the skin daily for 5 days. This will start at the same time or within 3 days of starting standard treatment for gut GVHD with steroids. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Patients 12 years of age or older at time of enrollment.
- Patients experiencing their initial presentation of stage 1 or greater acute LGI GVHD requiring systemic therapy after allogeneic transplant for any malignant or non-malignant indication using any graft/donor source or conditioning intensity.
- Patients can be enrolled with only a clinically established diagnosis. Biopsy of involved organs with acute GVHD is encouraged but is not required and should not delay study entry.
- Patients should not have received systemic immune suppressive therapy for treatment of active GVHD except for a maximum of 72 hours of steroid therapy prior to enrollment. Topical skin and GI corticosteroids (such as budesonide and oral beclomethasone diproprionate) are allowed.
- Informed Consent explained to, understood by and signed by patient/guardian. Patient/guardian given copy of informed consent.
Key exclusion criteria (12 listed)
- Relapsed, progressing or persistent malignancy or evidence of minimal residual disease (MRD) requiring withdrawal of systemic immune suppression.
- Patients with acute GVHD developing after administration of a donor lymphocyte infusion (DLI) for relapse / progression of disease. Patients with acute GVHD after planned donor lymphocyte infusion or planned T cell or NK cell add back are eligible.
- Patients with uncontrolled infections will be excluded. Infections are considered controlled if appropriate therapy has been instituted and, at the time of enrollment, no signs of progression are present. Progression of infection is defined as hemodynamic instability attributable to sepsis, new symptoms, worsening physical signs or radiographic findings attributable to infection. Persisting fever without other signs or symptoms
- Patients receiving other drugs for the treatment of GVHD. Note, GVHD prophylaxis agents (e.g., calcineurin inhibitors) may be continued at local Investigator's discretion.
- Patients on renal replacement therapy.
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 |
|---|---|---|
| 5-AzacytidinePatients will be treated with 5 azacytidine at a dose of 32 mg/m2 SQ or IV. The investigators aim to start AZA on Day 1 of steroids but it can be started up to 72 hours after steroids are started . Patients will be premedicated with antiemetics. Patients must receive steroids at a minimum dose of prednisone 2 mg/kg/day PO (or methylprednisolone 1.6 mg/kg/day IV) divided into 1-2 daily doses as therapy for acute GVHD. For patients that weigh over 100 kg, maximal starting dose of prednisone will be 200 mg (or methylprednisolone-equivalent). Steroid Taper for responding patients (Prednisone/Methylprednisolone) Days 1-5 2 mg/kg/day (taper cannot start until 3 days after enrollment) Days 6-10 1.5 mg/kg/day Day 11-15 1.0 mg/kg/day Days 16-20 0.5 mg/kg/day Days 21-28* 0.25 mg/kg/day Days 29-56 Gradual further taper with a goal of reaching < 0.2 mg/kg/day of prednisone (or < 0.16 mg/kg/day of methylprednisolone) by Day 56. | Drug | Treatment |
Primary outcome
Feasibility rate
Time frame: 30 days after first dose of AZA
The proportion of patients who can tolerate and complete one cycle of AZA (5-day) in combination with steroid among patients who are eligible and receive at least one dose of AZA.
Treatment-related adverse event (tAE) rate
Time frame: 30 days after first dose of AZA
The proportion of patients who develop any Grade 3-5 adverse events (per Common Terminology Criteria for Adverse Events [CTCAE] Version 5.0) that are considered probably, or definitely related to AZA or AZA in combination with steroid that occur in the first cycle of combination therapy until 30 days after first dose of AZA.
4 secondary outcomes
GVHD response rate
Time frame: Day 28
Overall survival (OS)
Time frame: 6 months and 12 months after combination therapy initiation
Progression-free survival (PFS)
Time frame: 6 months and 12 months after combination therapy initiation
Systemic Infections
Time frame: 30 days after last dose of AZA
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Sep 2025.
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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.
Helen Heslop, MD principal investigator
Baylor College of Medicine
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing 5-Azacytidine in people with Graft-Versus-Host Disease(GVHD), run by Baylor College of Medicine at 2 sites 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 5 inclusion and 12 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: withdrawn, as of the sponsor's last update on 22 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Feasibility rate (time frame: 30 days after first dose of AZA). The study lists 2 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 22 Sep 2025.