A Phase 2 interventional study of Fludarabine and Cyclophosphamide in Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia and Small Lymphocytic Lymphoma, sponsored by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins. Completed at 1 site in United States. Per ClinicalTrials.gov, last updated 2022-11-03.
Sponsored by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Phase 2, Interventional, and Treatment
To see if it is possible to use short-duration tacrolimus after a peripheral blood stem cell transplant in certain malignancies that are considered difficult to engraft.
The main goal is to learn whether a drug called tacrolimus, which is an immune-lowering drug (an immunosuppressant) given after transplant to help prevent certain complications, can be given safely for a shorter period of time than it has been in the past. The experiences with immunosuppression duration with other allogeneic HSCT platforms cannot be directly extrapolated to the high-dose posttransplantation cyclophosphamide platform (another type of immunosuppressant given after transplant to help prevent GVHD). There are presently no published data on the minimum required duration of tacrolimus after nonmyeloablative HSCT that includes high-dose Cy as part of postgrafting immunosuppression. The effectiveness of high-dose posttransplantation Cy in GVHD prevention, however, permits the investigation of this question. At the present time there are few or no cures for diseases studied on this trial outside of a bone marrow or peripheral blood transplant. The peripheral blood for this transplant comes from a relative who is a half-match or "haplo" match to the participant. Possible donors include parents, siblings, and children. In order to help the bone marrow grow, or "take", inside the body, participants will receive chemotherapy and radiation before the transplant. After the transplant participants will receive high doses of cyclophosphamide (Cytoxan®) along with other medications to lower the immune system, such as tacrolimus. These medications may lower the risk of graft versus host disease (GVHD) and of rejection of the peripheral blood graft.
1,997 studies on the registry are indexed under Myelodysplastic Syndromes; 324 are open to participants now.
This study's enrollment of 117 is above the median of 40 across 1,611 interventional studies indexed under Myelodysplastic Syndromes.
Browse Myelodysplastic Syndromes studies →Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins is the lead sponsor of 582 studies on the registry; 71 are open to participants now.
Of its 114 completed or terminated interventional studies of FDA-regulated products, 68 (60%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
FEV1
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Non-myeloablative peripheral blood stem cell transplant (PBSCT) with a fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis. Tacrolimus will be stopped at either Day 90 or Day 180 depending on GVHD status.
Drug: Fludarabine · Drug: Cyclophosphamide · Radiation: Total body irradiation · Drug: Tacrolimus · Drug: Mycophenolate mofetil
Non-myeloablative peripheral blood stem cell transplant (PBSCT) with a fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis. Tacrolimus will be stopped at either Day 60 or Day 180 depending on GVHD status.
Drug: Fludarabine · Drug: Cyclophosphamide · Radiation: Total body irradiation · Drug: Tacrolimus · Drug: Mycophenolate mofetil
Days -6 through -2: 30 mg/m\^2 IV daily
Also known as: Fludara
Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Also known as: Cytoxan, Cy, CTX
Day -1: 200 cGy in a single fraction
Also known as: TBI
Start on Day 5 through either Day 60 or Day 90 depending on cohort assignment. May be continued through Day 180 depending on GVHD status.
Also known as: Prograf, FK506, FK-506
Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
Also known as: MMF, CellCept
Percentage of Participants Who Are Able to Stop Prophylactic Tacrolimus (D90 Cohort)
This outcome measures the feasibility of stopping prophylactic tacrolimus at Day 90.
Time frame: Day 90
Percentage of Participants Who Are Able to Stop Prophylactic Tacrolimus (D60 Cohort)
This outcome measures the feasibility of stopping prophylactic tacrolimus at Day 60.
Time frame: Day 60
Number of Participants With Grades III-IV Acute GVHD, Days 90-180 (D90)
Number of participants who experience grade III or IV acute GVHD between Day 90 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 90 are evaluable.
Time frame: Between Day 90 and Day 180
Number of Participants With Grades III-IV Acute GVHD, Days 60-180 (D60)
Number of participants who experience grade III or IV acute GVHD between Day 60 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 60 are evaluable.
Time frame: Between Day 60 and Day 180
Number of Participants With Chronic GVHD, Days 90-180 (D90)
Number of participants who experience chronic GVHD requiring additional immunosuppressive therapy between Day 90 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 90 are evaluable.
Time frame: Between Day 90 and Day 180
Number of Participants With Chronic GVHD, Days 60-180 (D60)
Number of participants who experience chronic GVHD requiring additional immunosuppressive therapy between Day 60 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 60 are evaluable.
Time frame: Between Day 60 and Day 180
Number of Participants Who Experience Graft Failure, Days 90-180 (D90)
Number of participants who experience graft failure between Day 90 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 90 are evaluable.
Time frame: Between Day 90 and Day 180
Number of Participants Who Experience Graft Failure, Days 60-180 (D60)
Number of participants who experience graft failure between Day 60 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 60 are evaluable.
Time frame: Between Day 60 and Day 180
Number of Participants Who Experience Disease Relapse, Days 90-180 (D90)
Number of participants who experience disease relapse between Day 90 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 90 are evaluable.
Time frame: Between Day 90 and Day 180
Number of Participants Who Experience Disease Relapse, Days 60-180 (D60)
Number of participants who experience disease relapse between Day 60 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 60 are evaluable.
Time frame: Between Day 60 and Day 180
Number of Participants Who Experience Non-relapse Mortality, Days 90-180 (D90)
Number of participants who die for any reason other than disease relapse between Day 90 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 90 are evaluable.
Time frame: Between Day 90 and Day 180
Number of Participants Who Experience Non-relapse Mortality, Days 60-180 (D60)
Number of participants who die for any reason other than disease relapse between Day 60 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 60 are evaluable.
Time frame: Between Day 60 and Day 180
Number of Participants Who Experience Grades III-IV GVHD, Day 360 (D90)
Number of participants who experience grade III or IV GVHD by Day 360. All participants are evaluable.
Time frame: Day 360
Number of Number of Participants Who Experience Grades III-IV GVHD, Day 360 (D60)
Number of participants who experience grade III or IV GVHD by Day 360. All participants are evaluable.
Time frame: Day 360
Number of Number of Participants With Severe Chronic GVHD, Day 360 (D90)
Number of participants who experience severe chronic GVHD requiring additional immunosuppressive therapy by Day 360. All participants are evaluable.
Time frame: Day 360
Number of Number of Participants With Severe Chronic GVHD, Day 360 (D60)
Number of participants who experience severe chronic GVHD requiring additional immunosuppressive therapy by Day 360. All participants are evaluable.
Time frame: Day 360
Number of Number of Participants Who Experience Graft Failure, Day 360 (D90)
Number of participants who experience graft failure by Day 360. All participants are evaluable.
Time frame: Day 360
Number of Number of Participants Who Experience Graft Failure, Day 360 (D60)
Number of participants who experience graft failure by Day 360. All participants are evaluable.
Time frame: Day 360
Number of Participants Who Experience Relapse, Day 360 (D90)
Number of participants who experience disease relapse by Day 360. All participants are evaluable.
Time frame: Day 360
Number of Participants Who Experience Relapse, Day 360 (D60)
Number of participants who experience disease relapse by Day 360. All participants are evaluable.
Time frame: Day 360
Number of Participants Who Experience Non-relapse Mortality, Day 360 (D90)
Number of participants who die for any reason other than disease relapse by Day 360. All participants are evaluable.
Time frame: Day 360
Number of Participants Who Experience Non-relapse Mortality, Day 360 (D60)
Number of participants who die for any reason other than disease relapse by Day 360. All participants are evaluable.
Time frame: Day 360
| Milestone | PBSCT D90 | PBSCT D60 |
|---|---|---|
| Started | 57 | 60 |
| Completed | 57 | 60 |
| Not completed | 0 | 0 |
This outcome measures the feasibility of stopping prophylactic tacrolimus at Day 90.
| Participants | PBSCT D90 |
|---|---|
| Percentage of Participants Who Are Able to Stop Prophylactic Tacrolimus (D90 Cohort) | 33 |
This outcome measures the feasibility of stopping prophylactic tacrolimus at Day 60.
| Participants | PBSCT D60 |
|---|---|
| Percentage of Participants Who Are Able to Stop Prophylactic Tacrolimus (D60 Cohort) | 42 |
Number of participants who experience grade III or IV acute GVHD between Day 90 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 90 are evaluable.
| Participants | PBSCT D90 |
|---|---|
| Number of Participants With Grades III-IV Acute GVHD, Days 90-180 (D90) | 1 |
Number of participants who experience grade III or IV acute GVHD between Day 60 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 60 are evaluable.
| Participants | PBSCT D60 |
|---|---|
| Number of Participants With Grades III-IV Acute GVHD, Days 60-180 (D60) | 3 |
Number of participants who experience chronic GVHD requiring additional immunosuppressive therapy between Day 90 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 90 are evaluable.
| Participants | PBSCT D90 |
|---|---|
| Number of Participants With Chronic GVHD, Days 90-180 (D90) | 2 |
Number of participants who experience chronic GVHD requiring additional immunosuppressive therapy between Day 60 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 60 are evaluable.
| Participants | PBSCT D60 |
|---|---|
| Number of Participants With Chronic GVHD, Days 60-180 (D60) | 1 |
Number of participants who experience graft failure between Day 90 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 90 are evaluable.
| Participants | PBSCT D90 |
|---|---|
| Number of Participants Who Experience Graft Failure, Days 90-180 (D90) | 0 |
Number of participants who experience graft failure between Day 60 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 60 are evaluable.
| Participants | PBSCT D60 |
|---|---|
| Number of Participants Who Experience Graft Failure, Days 60-180 (D60) | 1 |
Number of participants who experience disease relapse between Day 90 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 90 are evaluable.
| Participants | PBSCT D90 |
|---|---|
| Number of Participants Who Experience Disease Relapse, Days 90-180 (D90) | 14 |
Number of participants who experience disease relapse between Day 60 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 60 are evaluable.
| Participants | PBSCT D60 |
|---|---|
| Number of Participants Who Experience Disease Relapse, Days 60-180 (D60) | 10 |
Number of participants who die for any reason other than disease relapse between Day 90 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 90 are evaluable.
| Participants | PBSCT D90 |
|---|---|
| Number of Participants Who Experience Non-relapse Mortality, Days 90-180 (D90) | 1 |
Number of participants who die for any reason other than disease relapse between Day 60 and Day 180. Only participants who are able to stop prophylactic tacrolimus at Day 60 are evaluable.
| Participants | PBSCT D60 |
|---|---|
| Number of Participants Who Experience Non-relapse Mortality, Days 60-180 (D60) | 2 |
Number of participants who experience grade III or IV GVHD by Day 360. All participants are evaluable.
| Participants | PBSCT D90 |
|---|---|
| Number of Participants Who Experience Grades III-IV GVHD, Day 360 (D90) | 16 |
Number of participants who experience grade III or IV GVHD by Day 360. All participants are evaluable.
| Participants | PBSCT D60 |
|---|---|
| Number of Number of Participants Who Experience Grades III-IV GVHD, Day 360 (D60) | 18 |
Number of participants who experience severe chronic GVHD requiring additional immunosuppressive therapy by Day 360. All participants are evaluable.
| Participants | PBSCT D90 |
|---|---|
| Number of Number of Participants With Severe Chronic GVHD, Day 360 (D90) | 7 |
Number of participants who experience severe chronic GVHD requiring additional immunosuppressive therapy by Day 360. All participants are evaluable.
| Participants | PBSCT D60 |
|---|---|
| Number of Number of Participants With Severe Chronic GVHD, Day 360 (D60) | 5 |
Number of participants who experience graft failure by Day 360. All participants are evaluable.
| Participants | PBSCT D90 |
|---|---|
| Number of Number of Participants Who Experience Graft Failure, Day 360 (D90) | 0 |
Number of participants who experience graft failure by Day 360. All participants are evaluable.
| Participants | PBSCT D60 |
|---|---|
| Number of Number of Participants Who Experience Graft Failure, Day 360 (D60) | 2 |
Number of participants who experience disease relapse by Day 360. All participants are evaluable.
| Participants | PBSCT D90 |
|---|---|
| Number of Participants Who Experience Relapse, Day 360 (D90) | 14 |
Number of participants who experience disease relapse by Day 360. All participants are evaluable.
| Participants | PBSCT D60 |
|---|---|
| Number of Participants Who Experience Relapse, Day 360 (D60) | 10 |
Number of participants who die for any reason other than disease relapse by Day 360. All participants are evaluable.
| Participants | PBSCT D90 |
|---|---|
| Number of Participants Who Experience Non-relapse Mortality, Day 360 (D90) | 4 |
Number of participants who die for any reason other than disease relapse by Day 360. All participants are evaluable.
| Participants | PBSCT D60 |
|---|---|
| Number of Participants Who Experience Non-relapse Mortality, Day 360 (D60) | 4 |
Collected over adverse event data were collected for each particpant for at minimum 1 year. Median follow-up of 41 months in the D90 cohort and 27 months in the D60 cohort.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| PBSCT D90 | 12/57 (21.1%) | 9/57 (15.8%) | 35/57 (61.4%) |
| PBSCT D60 | 10/60 (16.7%) | 5/60 (8.3%) | 16/60 (26.7%) |
| Event | PBSCT D90 | PBSCT D60 |
|---|---|---|
| Severe cGVHDSkin and subcutaneous tissue disorders | 9/57 | 5/60 |
| Event | PBSCT D90 | PBSCT D60 |
|---|---|---|
| neutropenic feverInfections and infestations | 35/57 | 16/60 |
| Age, Categorical(Participants) | PBSCT D90 | PBSCT D60 | Total |
|---|---|---|---|
| <=18 years | 0 | 0 | 0 |
| Between 18 and 65 years | 34 | 36 | 70 |
| >=65 years | 23 | 24 | 47 |
| Age, Continuous(years) | PBSCT D90 | PBSCT D60 | Total |
|---|---|---|---|
| Median | 62 (35 to 78) | 63 (22 to 75) | 62.5 (22 to 78) |
| Sex: Female, Male(Participants) | PBSCT D90 | PBSCT D60 | Total |
|---|---|---|---|
| Female | 17 | 19 | 36 |
| Male | 40 | 41 | 81 |
| Race and Ethnicity Not Collected(Participants) | PBSCT D90 | PBSCT D60 | Total |
|---|---|---|---|
| Count of participants | — | — | 0 |
| Region of Enrollment(Participants) | PBSCT D90 | PBSCT D60 | Total |
|---|---|---|---|
| United States | 57 | 60 | 117 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
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Studies of the drugs tested here: Cyclophosphamide · Tacrolimus
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins →