Graft-versus-Host DiseasePhase 3NCT01002742
Acute Graft-versus-Host Disease Treatment (BMT CTN 0802)
A Phase 3 study testing Mycophenolate Mofetil in people with Graft-versus-Host Disease and Immune System Disorders, run by Medical College of Wisconsin at 36 sites in United States.
Trial summary
The study is a Phase III, randomized double blind, placebo controlled, and trial evaluating the addition of Mycophenolate mofetil (MMF) vs. placebo to systemic corticosteroids as initial therapy for acute Graft Vs Host Disease (GVHD). The primary endpoint will be GVHD free survival at Day 56 post randomization. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Graft-versus-Host Disease, Immune System Disorders
- What is being tested?
- Mycophenolate Mofetil
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 236 participants
- Where
- 36 sitesUnited States
- Study dates
- Jan 2010to Jun 2013
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Acute GVHD developing after allogeneic hematopoietic stem cell transplant using either bone marrow, peripheral blood stem cells or cord blood. Recipients of non-myeloablative and myeloablative transplants are eligible.
- Acute GVHD after planned donor lymphocyte infusion or planned T cell add back are eligible.
- De novo acute GVHD requiring systemic therapy. GVHD is defined as the presence of skin rash and/or persistent nausea, vomiting, and/or diarrhea and/or cholestasis presenting in a context in which acute GVHD is likely to occur and where other etiologies such as drug rash, enteric infection, or hepatotoxic syndromes are unlikely or have been ruled out. Note that patients with stage I and II skin only (overall grade I) or isolated upper gastrointestinal (GI) involvement are eligible if the treating physician deems that systemic high-dose corticosteroid treatment is indicated.
- The patient must have had no previous systemic immune suppressive therapy for treatment of acute GVHD except for a maximum 72 hours of prior corticosteroid therapy at >0.5mg/kg methylprednisolone or equivalent after the onset of acute GVHD.
- Clinical status at enrollment to allow tapering of steroids to not less than 0.25 mg/kg/day prednisone (0.2 mg/kg/day methylprednisolone) at Day 28 of therapy.
- Absolute neutrophil count (ANC) greater than 500/µL.
Show all 10Show fewer
- Written informed consent and/or assent from patient, parent or guardian.
- Documentation that the assent document and education materials have been provided to, and reviewed with, patients between the ages of 7 and 17.
- Patients of all ages are eligible.
- Biopsy confirmation of GVHD is recommended, but not required. Enrollment should not be delayed for biopsy or pathology results unless these are to be used to decide about whether to treat for GVHD.
Who cannot join
- Patients receiving mycophenolate mofetil or mycophenolic acid (Myfortic) within seven days of screening for enrollment.
- Patients with uncontrolled infections will be excluded. If a bacterial or viral infection is present, patients must be receiving definitive therapy and have no signs of progressing infection for 72 hours prior to enrollment. If a fungal infection is present, patients must be receiving definitive systemic anti-fungal therapy and have no signs of progressing infection for 1 week prior to enrollment. Progressing infection is defined as hemodynamic instability attributable to sepsis or new symptoms, worsening physical signs or radiographic findings attributable to infection. Persisting fever without other signs or symptoms will not be interpreted as progressing infection.
- Relapsed/persistent malignancy requiring rapid immune suppression withdrawal.
- Patients with GVHD after an unplanned Donor Lymphocyte Infusion (DLI), i.e., DLI that was not part of their original transplant therapy plan, or DLI given for treatment of persistent or recurrent malignancy after transplantation.
- Patients unlikely to be available at the transplantation center on Day 28 and 56 of therapy.
- A clinical syndrome resembling de novo chronic GVHD developing at any time after allotransplantation.
Show all 13Show fewer
- Patients receiving other drugs for the treatment of GVHD.
- Patients receiving methylprednisolone > 0.5 mg/kg/day (or 0.6 mg/kg/day prednisone) within 7 days before the onset of acute GVHD. If steroid therapy has been administered for treatment of a non-GVHD related condition and tapered to ≤ 0.5 mg/kg/day methylprednisolone (0.6 mg/kg/day prednisone) for seven or more days before the onset of acute GVHD, the patient is eligible.
- Patients who are pregnant, breast feeding, or, if sexually active, unwilling to use effective birth control for the duration of the study. Available evidence and/or expert consensus is inconclusive or is inadequate for determining infant risk when used during breastfeeding, therefore breast feeding patients are not eligible.
- Adults unable to provide informed consent.
- Patients on dialysis.
- Patients with severe hepatic Veno-Occlusive Disease (VOD) or sinusoidal obstruction syndrome who in the judgement of the treating physician are not expected to have normalized bilirubin by Day 56 after enrollment.
- Patients with a history of intolerance/allergy to MMF.
Trial locations and contacts
36 sites in 1 country.
36 sites in 1 country
United States36 sites
University of Michigan Medical Center
Ann Arbor, Michigan, 48109
BMT Program at Northside Hospital
Atlanta, Georgia, 30342
University of Maryland Medical Systems
Baltimore, Maryland, 21201
Johns Hopkins University
Baltimore, Maryland, 21231
Indiana BMT at Beech Grove
Beech Grove, Indiana, 46107
Tufts Medical Center
Boston, Massachusetts, 02111
DFCI, Massachusetts General Hospital
Boston, Massachusetts, 02114
Dana Farber Cancer Institute, Brigham & Women's Hospital
Boston, Massachusetts, 02115
University of North Carolina Hospital at Chapel Hill
Chapel Hill, North Carolina, 27599-7305
Medical University of South Carolina
Charleston, South Carolina, 29425
Levine Children's Hospital, Carolinas Medical Center
Charlotte, North Carolina, 28232
Ann & Robert Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Rush University Medical Center
Chicago, Illinois, 60612
Ohio State University
Columbus, Ohio, 43210
Baylor University Medical Center
Dallas, Texas, 75246
Colorado Blood Cancer Institute
Denver, Colorado, 80218
City of Hope National Medical Center
Duarte, California, 91010
Duke University Medical Center
Durham, North Carolina, 27705
University of Florida College of Medicine
Gainesville, Florida, 32610
Hackensack Univ. Medical Center
Hackensack, New Jersey, 07601
University of Texas, MD Anderson Cancer Research Center
Houston, Texas, 77030
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
University of California San Diego Medical Center
La Jolla, California, 92093
Medical College of Wisconsin
Milwaukee, Wisconsin, 53211
University of Minnesota
Minneapolis, Minnesota, 55455
Memorial Sloan-Kettering Cancer Center
New York, New York, 10021
Weill Cornell Medical College, NY Presbyterian Hospital
New York, New York, 10065
University of Pennsylvania Cancer Center
Philadelphia, Pennsylvania, 19104
Oregon Health and Science University
Portland, Oregon, 97239
Virginia Commonwealth University
Richmond, Virginia, 23298
Mayo Clinic
Rochester, Minnesota, 55905
Texas Transplant Institute
San Antonio, Texas, 78229
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109
Avera Hematology & Transplant Center
Sioux Falls, South Dakota, 57105
Washington University, Barnes Jewish Hospital
St Louis, Missouri, 63110
Stanford Hospital and Clinics
Stanford, California, 94305
About this study
Official title: A Multi-Center, Randomized, Double Blind, Phase III Trial Evaluating Corticosteroids With Mycophenolate Mofetil vs. Corticosteroids With Placebo as Initial Systemic Treatment of Acute Graft-Vs-Host-Disease (BMT CTN #0802)
The study is a Phase III, randomized double blind, placebo controlled, and trial evaluating the addition of Mycophenolate mofetil (MMF) vs. placebo to systemic corticosteroids as initial therapy for acute Graft Vs Host Disease (GVHD). The primary endpoint will be GVHD free survival at Day 56 post randomization.
Read the detailed description
Corticosteroids have been used as primary therapy for acute GVHD for many years. Historical published and unpublished data from Johns Hopkins, M. D. Anderson, University of Michigan and others defined an expected 35%-53% complete response (CR) at Day +28 of corticosteroid therapy for previously untreated patients with acute GVHD.
BMT CTN study 0302 (NCT00224874)was a randomized Phase II study evaluating etanercept, mycophenolate mofetil, denileukin diftitox or pentostatin in addition to corticosteroids. The results of that study suggested that mycophenolate mofetil produced the highest rates of CR at Day 28 and overall survival, supporting its evaluation in a Phase III study. Day 56 GVHD-free survival for the four treatment arms (all combining corticosteroids with one of the four study drugs) ranged from 39-71% across the four study arms.
- Conditions
- Graft-versus-Host Disease, Immune System Disorders
- Being tested
- Mycophenolate Mofetil, Placebo
- Keywords
- GVHD
- Medical subject headings
- Graft vs Host Disease, Immune System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Also called Cellcept®, MMF
Oral dosing should be delivered in 250 mg units. For those < 40 kg, IV dosing should be within ± 10% of the exact dose. Intravenous doses are infused over a two-hour period.
- Patients who weight > 60 kg should receive MMF 1 gm PO/IV every 8 hours.
- Patients who weight between 40-60 kg should receive 750 mg PO/IV every 8 hours.
- Patients who weight <40 kg should receive 20 mg/kg IV or PO every 8 hours.
PlaceboDrug
Also called Inactive drug
Oral dosing should be delivered in 250 mg units blinded placebo. For those < 40 kg, IV dosing should be within ± 10% of the exact dose. Intravenous doses are infused over a two-hour period.
- Patients who weight > 60 kg should receive placebo 1 gm PO/IV every 8 hours.
- Patients who weight between 40-60 kg should receive 750 mg PO/IV every 8 hours.
- Patients who weight <40 kg should receive 20 mg/kg IV or PO every 8 hours.
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Experimental | Corticosteroids with placebo Receives: Drug: Placebo |
| Mycophenolate Mofetil | Experimental | Corticosteroids with Mycophenolate Mofetil Receives: Drug: Mycophenolate Mofetil |
What is being measured
The primary outcome is the main question the study is designed to answer.
GVHD-free Survival
Success is defined as alive and free of GVHD at day 56 after randomization, all others are considered to be a study failure.
Measured: Day 56
Secondary outcomes (14)
Percentage of Surviving Participants With Complete Response (CR)
CR is defined as a score of 0 for the GVHD grading in all evaluable organs.
Measured: Days 14, 28, and 56
Incidence of GVHD Flares Requiring Increased Therapy
Flares are defined as any progression of acute GVHD after an initial response (i.e., earlier CR or PR) that requires re-escalation of steroid dosing, or initiation of additional topical or systemic therapy.
Measured: Day 90
Incidence of Discontinuation of Immune Suppression Without Flare
Measured: Day 56, Day 180 and Day 360 post-treatment
Cumulative Steroid Dose
The cumulative steroid dose for each patient will be calculated by adding the doses (end of each week's dose) for each of the first four weeks of treatment, divided by the number of days of survival during this interval. The cumulative steroid dose was calculated for all patients per treatment arm and compared.
Measured: Days 28 and 56
Incidence of Topical/Non-absorbable Therapy
Measured: Day 56
Overall GVHD-free Survival Post-randomization
Measured: Months 6 and 12
Incidence of Chronic GVHD
Measured: 12 months post-randomization
Incidence of Systemic Infections
Number of participants that experienced at least one infection.
Measured: 6 Months
Incidence of Epstein-Barr Virus (EBV)-Associated Lymphoma
Measured: 12 months
Incidence of Cytomegalovirus (CMV) Reactivation
Measured: Year 1
Cumulative Incidence of a Severe/Life-threatening/Fatal Infections
Measured: Year 1
Disease-Free Survival (DFS) Post-Randomization
DFS includes death or progression/relapse of malignancy
Measured: Year 1
Treatment Related Mortality (TRM)
Measured: Year 1
Change in Patient Reported Outcomes From Enrollment to Day 56
Measured: Day 56
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2009 (estimated)
- Study startJan 2010
- Primary completionJan 2012
- Study completionJun 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Graft-versus-Host DiseaseStudies of the same condition, open and under way.
- More trials run by Medical College of WisconsinThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 236 participants (actual)
Sponsor and investigators
- Lead sponsor
- Medical College of Wisconsin
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Mary Horowitz, MD, Study director, Center for International Blood and Marrow Transplant Research
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01002742
- Sponsor's protocol ID
- BMTCTN0802
- Other IDs
- U01HL069294, BMT CTN 0802, U01HL069294-06, 5U24CA076518
- Status last verified
- Dec 2022
- First submitted
- 23 Oct 2009
- Last updated
- 4 Jan 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: Findings will be published in a manuscript.
Publications
Bolanos-Meade J, Logan BR, Alousi AM, Antin JH, Barowski K, Carter SL, Goldstein SC, Hexner EO, Horowitz MM, Lee SJ, Levine JE, MacMillan ML, Martin PJ, Mendizabal AM, Nakamura R, Pasquini MC, Weisdorf DJ, Westervelt P, Ho VT. Phase 3 clinical trial of steroids/mycophenolate mofetil vs steroids/placebo as therapy for acute GVHD: BMT CTN 0802. Blood. 2014 Nov 20;124(22):3221-7; quiz 3335. doi: 10.1182/blood-2014-06-577023. Epub 2014 Aug 28.
PubMed 25170121Shah O, Tamaresis JS, Kenyon LJ, Xu L, Zheng P, Gupta P, Rangarajan K, Lee S, Spellman S, Nikiforow S, Zehnder J, Meyer EH. Analysis of the Whole CDR3 T Cell Receptor Repertoire after Hematopoietic Stem Cell Transplantation in 2 Clinical Cohorts. Biol Blood Marrow Transplant. 2020 Jun;26(6):1050-1070. doi: 10.1016/j.bbmt.2020.01.020. Epub 2020 Feb 18.
PubMed 32081787
Study at a glance
From the registry record, last updated by the sponsor on 4 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center, Randomized, Double Blind, Phase III Trial Evaluating Corticosteroids With Mycophenolate Mofetil vs. Corticosteroids With Placebo as Initial Systemic Treatment of Acute Graft-Vs-Host-Disease (BMT CTN #0802)
- ClinicalTrials.gov ID
- NCT01002742
- Acronym / sponsor ID
- BMTCTN0802
- Conditions
- Graft-versus-Host Disease, Immune System Disorders
- Keywords
- GVHD
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 236 participants (actual)
- Lead sponsor
- Medical College of WisconsinUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), National Cancer Institute (NCI)
- Study start
- Jan 2010 (actual)
- Primary completion
- Jan 2012 (actual)
- Study completion
- Jun 2013 (actual)
- First posted
- 27 Oct 2009 (estimated)
- Results
- Posted 27 May 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study is a Phase III, randomized double blind, placebo controlled, and trial evaluating the addition of Mycophenolate mofetil (MMF) vs. placebo to systemic corticosteroids as initial therapy for acute Graft Vs Host Disease (GVHD). The primary endpoint will be GVHD free survival at Day 56 post randomization.
Detailed description
Corticosteroids have been used as primary therapy for acute GVHD for many years. Historical published and unpublished data from Johns Hopkins, M. D. Anderson, University of Michigan and others defined an expected 35%-53% complete response (CR) at Day +28 of corticosteroid therapy for previously untreated patients with acute GVHD.
BMT CTN study 0302 (NCT00224874)was a randomized Phase II study evaluating etanercept, mycophenolate mofetil, denileukin diftitox or pentostatin in addition to corticosteroids. The results of that study suggested that mycophenolate mofetil produced the highest rates of CR at Day 28 and overall survival, supporting its evaluation in a Phase III study. Day 56 GVHD-free survival for the four treatment arms (all combining corticosteroids with one of the four study drugs) ranged from 39-71% across the four study arms.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Experimental | Drug: PlaceboCorticosteroids with placebo |
| Mycophenolate Mofetil | Experimental | Drug: Mycophenolate MofetilCorticosteroids with Mycophenolate Mofetil |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Acute GVHD developing after allogeneic hematopoietic stem cell transplant using either bone marrow, peripheral blood stem cells or cord blood. Recipients of non-myeloablative and myeloablative transplants are eligible.
- Acute GVHD after planned donor lymphocyte infusion or planned T cell add back are eligible.
- De novo acute GVHD requiring systemic therapy. GVHD is defined as the presence of skin rash and/or persistent nausea, vomiting, and/or diarrhea and/or cholestasis presenting in a context in which acute GVHD is likely to occur and where other etiologies such as drug rash, enteric infection, or hepatotoxic syndromes are unlikely or have been ruled out. Note that patients with stage I and II skin only (overall grade I) or isolated upper gastrointestinal (GI) involvement are eligible if the treating physician deems that systemic high-dose corticosteroid treatment is indicated.
- The patient must have had no previous systemic immune suppressive therapy for treatment of acute GVHD except for a maximum 72 hours of prior corticosteroid therapy at >0.5mg/kg methylprednisolone or equivalent after the onset of acute GVHD.
- Clinical status at enrollment to allow tapering of steroids to not less than 0.25 mg/kg/day prednisone (0.2 mg/kg/day methylprednisolone) at Day 28 of therapy.
Key exclusion criteria (13 listed)
- Patients receiving mycophenolate mofetil or mycophenolic acid (Myfortic) within seven days of screening for enrollment.
- Patients with uncontrolled infections will be excluded. If a bacterial or viral infection is present, patients must be receiving definitive therapy and have no signs of progressing infection for 72 hours prior to enrollment. If a fungal infection is present, patients must be receiving definitive systemic anti-fungal therapy and have no signs of progressing infection for 1 week prior to enrollment. Progressing infection is defined as hemodynamic instability attributable to sepsis or new symptoms, worsening physical signs or radiographic findings attributable to infection. Persisting fever without other signs or symptoms will not be interpreted as progressing infection.
- Relapsed/persistent malignancy requiring rapid immune suppression withdrawal.
- Patients with GVHD after an unplanned Donor Lymphocyte Infusion (DLI), i.e., DLI that was not part of their original transplant therapy plan, or DLI given for treatment of persistent or recurrent malignancy after transplantation.
- Patients unlikely to be available at the transplantation center on Day 28 and 56 of 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 |
|---|---|---|
| Mycophenolate MofetilAlso: Cellcept®, MMFOral dosing should be delivered in 250 mg units. For those < 40 kg, IV dosing should be within ± 10% of the exact dose. Intravenous doses are infused over a two-hour period. * Patients who weight > 60 kg should receive MMF 1 gm PO/IV every 8 hours. * Patients who weight between 40-60 kg should receive 750 mg PO/IV every 8 hours. * Patients who weight <40 kg should receive 20 mg/kg IV or PO every 8 hours. | Drug | Mycophenolate Mofetil |
| PlaceboAlso: Inactive drugOral dosing should be delivered in 250 mg units blinded placebo. For those < 40 kg, IV dosing should be within ± 10% of the exact dose. Intravenous doses are infused over a two-hour period. * Patients who weight > 60 kg should receive placebo 1 gm PO/IV every 8 hours. * Patients who weight between 40-60 kg should receive 750 mg PO/IV every 8 hours. * Patients who weight <40 kg should receive 20 mg/kg IV or PO every 8 hours. | Drug | Placebo |
Primary outcome
GVHD-free Survival
Time frame: Day 56
Success is defined as alive and free of GVHD at day 56 after randomization, all others are considered to be a study failure.
14 secondary outcomes
Percentage of Surviving Participants With Complete Response (CR)
Time frame: Days 14, 28, and 56
Incidence of GVHD Flares Requiring Increased Therapy
Time frame: Day 90
Incidence of Discontinuation of Immune Suppression Without Flare
Time frame: Day 56, Day 180 and Day 360 post-treatment
Cumulative Steroid Dose
Time frame: Days 28 and 56
Incidence of Topical/Non-absorbable Therapy
Time frame: Day 56
Overall GVHD-free Survival Post-randomization
Time frame: Months 6 and 12
Incidence of Chronic GVHD
Time frame: 12 months post-randomization
Incidence of Systemic Infections
Time frame: 6 Months
Incidence of Epstein-Barr Virus (EBV)-Associated Lymphoma
Time frame: 12 months
Incidence of Cytomegalovirus (CMV) Reactivation
Time frame: Year 1
Cumulative Incidence of a Severe/Life-threatening/Fatal Infections
Time frame: Year 1
Disease-Free Survival (DFS) Post-Randomization
Time frame: Year 1
Treatment Related Mortality (TRM)
Time frame: Year 1
Change in Patient Reported Outcomes From Enrollment to Day 56
Time frame: Day 56
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jan 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 25170121Bolanos-Meade J, Logan BR, Alousi AM, Antin JH, Barowski K, Carter SL, Goldstein SC, Hexner EO, Horowitz MM, Lee SJ, Levine JE, MacMillan ML, Martin PJ, Mendizabal AM, Nakamura R, Pasquini MC, Weisdorf DJ, Westervelt P, Ho VT. Phase 3 clinical trial of steroids/mycophenolate mofetil vs steroids/placebo as therapy for acute GVHD: BMT CTN 0802. Blood. 2014 Nov 20;124(22):3221-7; quiz 3335. doi: 10.1182/blood-2014-06-577023. Epub 2014 Aug 28.
- Derived from this study: PubMed 32081787Shah O, Tamaresis JS, Kenyon LJ, Xu L, Zheng P, Gupta P, Rangarajan K, Lee S, Spellman S, Nikiforow S, Zehnder J, Meyer EH. Analysis of the Whole CDR3 T Cell Receptor Repertoire after Hematopoietic Stem Cell Transplantation in 2 Clinical Cohorts. Biol Blood Marrow Transplant. 2020 Jun;26(6):1050-1070. doi: 10.1016/j.bbmt.2020.01.020. Epub 2020 Feb 18.
- Individual participant dataSharing planned: Yes. Findings will be published in a manuscript.
Related trials for Graft-versus-Host Disease
Active, not recruitingPhase 2
De-escalated Cyclophosphamide (PTCy) and Ruxolitinib for Graft-versus-Host Disease (GVHD) Prophylaxis
Graft-versus-host Disease
RecruitingEarly Phase 1
HMCT/CT2401: Abatacept GVHD Prophylaxis Following Omidubicel HCT
Hematologic Malignancy
RecruitingObservational study
Improved Methods of Cell Selection for Bone Marrow Transplant Alternatives
Graft-Versus-Host Disease, Graft-versus-leukemia
RecruitingPhase 1/2
The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation
Peripheral Blood Stem Cell Transplantation, Hematopoietic Stem Cell Transplantation
RecruitingPhase 1/2
Phase I/II Study of Pacritinib, A JAK2/IRAK1/CSF1R Inhibitor, in Refractory Chronic Graft-Versus-Host Disease (cGVHD) After Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
Graft vs Host Disease
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.
Mary Horowitz, MD study director
Center for International Blood and Marrow Transplant Research
36 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Mycophenolate Mofetil in people with Graft-versus-Host Disease and Immune System Disorders, run by Medical College of Wisconsin at 36 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 10 inclusion and 13 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: finished, as of the sponsor's last update on 4 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 36 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?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 27 May 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
GVHD-free Survival (time frame: Day 56). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01002742, which also lists 2 linked publications.
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 4 Jan 2023.