MDSPhase 2NCT07896928
Study of Allogeneic Stem Cell Transplant in Myelodysplastic Syndrome Following Conditioning With Fludarabine and Total Body Irradiation 800 cGy
A Phase 2 study testing Allogeneic Stem Cell Transplant in people with MDS and Myelodysplastic Syndrome MDS, run by Ronald Paquette at 1 site in United States.
Trial summary
This is a single center phase II study of allogeneic stem cell transplantation in MDS who are unlikely to benefit from conventional therapy. Patients will undergo standard allogeneic stem cell transplant protocol following standard Flu/TBI 800 conditioning, PBSC transplant, and GVHD prophylaxis with PTCy and… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- MDS, Myelodysplastic Syndrome MDS, Myelodysplastic Syndrome (MDS)
- What is being tested?
- Allogeneic Stem Cell Transplant
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2026to Dec 2030
- Phase
- Phase 2
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- MDS diagnosis and:
- Very low, low, or low-intermediate risk by IPSS-M. This can be determined using the online calculator: https://mds-risk-model.com/. Must be relapsed, refractory or ineligible (due to serum erythropoietin level >500 U/L) for erythropoiesis stimulating agent (ESA). Prior luspatercept or imetelstat is permitted. Transfusion-dependent requiring >4 units packed red blood cells (PRBCs) over an 8 week period during the 16 weeks before enrollment. or
- TP53 mutation/deletion with combined VAF < 50%
- Meets standard criteria for allogeneic stem cell transplant
- Patient and related Donor (if applicable) must sign the Informed Consent Form for the study. If donor is unrelated, donor does not sign Informed Consent Form and this will not affect recipient study eligibility
- Patient is deemed suitable to receive Flu/TBI 800 conditioning regimen as standard of care transplant.
Who cannot join
- Prior therapy with hypomethylating agent
- Prior autologous or allogeneic stem cell transplant
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Ronald Paquette, MD contact
United States1 site
Cedars-Sinai Medical Center
Los Angeles, California, 90048
Clinical Trial Recruitment Navigator, 310-423-5842, GroupCancerTrailInformation@cshs.org
Ronald Paquette, MD, ronald.paquette@csmc.edu
About this study
Official title: IIT2026-01-PAQUETTE-MDSBMT: A Phase 2 Study of Allogeneic Stem Cell Transplant in Myelodysplastic Syndrome Following Conditioning With Fludarabine and Total Body Irradiation 800 cGy
This is a single center phase II study of allogeneic stem cell transplantation in MDS who are unlikely to benefit from conventional therapy. Patients will undergo standard allogeneic stem cell transplant protocol following standard Flu/TBI 800 conditioning, PBSC transplant, and GVHD prophylaxis with PTCy and tacrolimus.
- Conditions
- MDS, Myelodysplastic Syndrome MDS, Myelodysplastic Syndrome (MDS), Myelodysplastic Syndrome
- Being tested
- Allogeneic Stem Cell Transplant
- Keywords
- fludarabine, MDS low risk, allogeneic stem cell transplant
- Medical subject headings
- Anemia, Refractory, with Excess of Blasts, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic Stem Cell TransplantProcedure or surgery
Standard RIC with fludarabine TBI-800 cGy. GVHD prophylaxis with cyclophosphamide on days +3 and +4, with dose established by ongoing GeriBMT trial, and tacrolimus/mycophenolate beginning day +5
| Study group | Role | What they receive |
|---|---|---|
| Single Arm | Experimental | Not described Receives: Procedure: Allogeneic Stem Cell Transplant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival (OS)
Evaluate the 1-year OS of two groups of MDS patients following allogeneic stem cell transplantation: transfusion-dependent lower-risk patients and patients with TP53 mutations/deletions with VAF <50%.
Measured: 1 year following transplantation
Secondary outcomes (9)
Time to neutrophil and platelet engraftment
Measure the time to neutrophil and platelet engraftment
Measured: 30 days after PTCy Administration
Rate of Acute Graft-versus-host disease (GVHD)
Evaluate the rate of acute GVHD by 100 days
Measured: 100 days after PTCy Administration
Severity of Acute Graft-versus-host disease (GVHD)
Evaluate the severity of acute GVHD by 100 days
Measured: 100 days after PTCy Administration
Rate of Acute Graft-versus-host disease (GVHD)
Evaluate the rate of acute GVHD by 1 year
Measured: 1 year after PTCy Administration
Severity of Acute Graft-versus-host disease (GVHD)
Evaluate the severity of acute GVHD by 1 year
Measured: 1 year after PTCy Administration
Non-relapse mortality
Assess rate of non-relapse mortality at 30 days and 1 year
Measured: 30 days after PTCy Administration, 1 year after PTCy Administration
Relapse Rate
Determine the relapse rate at 1 year
Measured: 1 year after PTCy Administration
GVHD-free relapse-free survival (GRFS)
Calculate the rate of GVHD-free relapse-free survival (GRFS) at 1 year
Measured: 1 year after PTCy Administration
Transfusion-independence
Determine the frequency of transfusion-independence at 1 year
Measured: 1 year after PTCy Administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2026
- Study startDec 2026 (estimated)
- Primary completionDec 2030 (estimated)
- Study completionDec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MDSStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ronald Paquette
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Ronald Paquette, Cedars-Sinai Medical Center
- Investigators
- Ronald Paquette, MD, Principal investigator, Cedars-Sinai Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07896928
- Sponsor's protocol ID
- IIT2026-01-PAQUETTE-MDSBMT
- Status last verified
- Oct 2026
- First submitted
- 20 Oct 2026
- Last updated
- 28 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- IIT2026-01-PAQUETTE-MDSBMT: A Phase 2 Study of Allogeneic Stem Cell Transplant in Myelodysplastic Syndrome Following Conditioning With Fludarabine and Total Body Irradiation 800 cGy
- ClinicalTrials.gov ID
- NCT07896928
- Acronym / sponsor ID
- IIT2026-01-PAQUETTE-MDSBMT
- Conditions
- MDS, Myelodysplastic Syndrome MDS, Myelodysplastic Syndrome (MDS), Myelodysplastic Syndrome
- Keywords
- fludarabine, MDS low risk, allogeneic stem cell transplant
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Ronald PaquetteUniversity, hospital or other
- Study start
- Dec 2026 (estimated)
- Primary completion
- Dec 2030 (estimated)
- Study completion
- Dec 2030 (estimated)
- First posted
- 27 Oct 2026 (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 is a single center phase II study of allogeneic stem cell transplantation in MDS who are unlikely to benefit from conventional therapy. Patients will undergo standard allogeneic stem cell transplant protocol following standard Flu/TBI 800 conditioning, PBSC transplant, and GVHD prophylaxis with PTCy and tacrolimus.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm | Experimental | Procedure: Allogeneic Stem Cell Transplant |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- MDS diagnosis and:
- Meets standard criteria for allogeneic stem cell transplant
- Patient and related Donor (if applicable) must sign the Informed Consent Form for the study. If donor is unrelated, donor does not sign Informed Consent Form and this will not affect recipient study eligibility
- Patient is deemed suitable to receive Flu/TBI 800 conditioning regimen as standard of care transplant.
Key exclusion criteria (2 listed)
- Prior therapy with hypomethylating agent
- Prior autologous or allogeneic stem cell transplant
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 |
|---|---|---|
| Allogeneic Stem Cell TransplantStandard RIC with fludarabine TBI-800 cGy. GVHD prophylaxis with cyclophosphamide on days +3 and +4, with dose established by ongoing GeriBMT trial, and tacrolimus/mycophenolate beginning day +5 | Procedure or surgery | Single Arm |
Primary outcome
Overall Survival (OS)
Time frame: 1 year following transplantation
Evaluate the 1-year OS of two groups of MDS patients following allogeneic stem cell transplantation: transfusion-dependent lower-risk patients and patients with TP53 mutations/deletions with VAF <50%.
9 secondary outcomes
Time to neutrophil and platelet engraftment
Time frame: 30 days after PTCy Administration
Rate of Acute Graft-versus-host disease (GVHD)
Time frame: 100 days after PTCy Administration
Severity of Acute Graft-versus-host disease (GVHD)
Time frame: 100 days after PTCy Administration
Rate of Acute Graft-versus-host disease (GVHD)
Time frame: 1 year after PTCy Administration
Severity of Acute Graft-versus-host disease (GVHD)
Time frame: 1 year after PTCy Administration
Non-relapse mortality
Time frame: 30 days after PTCy Administration, 1 year after PTCy Administration
Relapse Rate
Time frame: 1 year after PTCy Administration
GVHD-free relapse-free survival (GRFS)
Time frame: 1 year after PTCy Administration
Transfusion-independence
Time frame: 1 year after PTCy Administration
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 28 Oct 2026.
- Individual participant dataSharing planned: Undecided
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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.
Ronald Paquette, MD contact
Ronald Paquette, MD principal investigator
Cedars-Sinai Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Allogeneic Stem Cell Transplant in people with MDS and Myelodysplastic Syndrome MDS, run by Ronald Paquette 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 6 inclusion and 2 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: opening soon, as of the sponsor's last update on 28 Oct 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 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?
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 Trial Navigator (310-423-5842, GroupCancerTrialInformation@cshs.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Survival (OS) (time frame: 1 year following 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 28 Oct 2026.