A Phase 2 interventional study of Fludarabine Phosphate and Total-Body Irradiation in Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Previously Treated Myelodysplastic Syndrome and Refractory Chronic Lymphocytic Leukemia, sponsored by Fred Hutchinson Cancer Center. Completed at 11 sites in 3 countries. Per ClinicalTrials.gov, last updated 2019-10-30.
Sponsored by Fred Hutchinson Cancer Center · Phase 2, Interventional, and Treatment
This randomized phase II trial studies how well giving tacrolimus and mycophenolate mofetil (MMF) with or without sirolimus works in preventing acute graft-versus-host disease (GVHD) in patients undergoing donor stem cell transplant for hematologic cancer. Giving low doses of chemotherapy, such as fludarabine phosphate, and total-body-irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune system and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving MMF and tacrolimus with or without sirolimus after transplant may stop this from happening.
PRIMARY OBJECTIVES:
I. To determine which of 3 GVHD prophylaxis regimens results in reduction of acute grades II-IV GVHD to =\< 40%.
SECONDARY OBJECTIVES:
I. Reduce the incidence of non-relapse mortality from infections and GVHD before day 200 to =\< 15%.
II. Reduce the utilization of high-dose corticosteroids compared to protocols 1463, 1641, and 1668.
III. Compare survival and progression-free survival to that achieved under protocols 1463, 1641, and 1668.
OUTLINE:
CONDITIONING: All patients receive fludarabine phosphate intravenously (IV) over 30 minutes on days -4 to -2 and undergo total-body irradiation on day 0.
TRANSPLANTATION: All patients undergo allogeneic peripheral blood stem cell transplantation on day 0.
IMMUNOSUPPRESSION: Patients are randomized to 1 of 3 treatment arms.
ARM I: Patients receive tacrolimus IV or orally (PO) every 12 hours on days -3 to 180 with taper beginning on day 100 in the absence of GVHD. Patients also receive MMF PO every 8 hours on days 0-29 and then every 12 hours on days 30-96 with taper beginning on day 40 in the absence of GVHD.
ARM II: Patients receive tacrolimus IV or PO every 12 hours on days -3 to 150 with taper beginning on day 100 in the absence of GVHD. Patients also receive MMF PO every 8 hours on days 0-29 and then every 12 hours on days 30-180 with taper beginning on day 150 in the absence of GVHD.
ARM III: Patients receive tacrolimus and MMF as in arm II. Patients also receive sirolimus PO once daily on days -3 to 80.
After completion of study treatment, patients are followed up at 6 months and then every year thereafter.
The following diseases will be permitted although other diagnoses can be considered if approved by PCC or the participating institutions' patient review committees and the principal investigators:
CLL must have either
DONOR: FHCRC matching allowed will be Grades 1.0 to 2.1: unrelated donors who are prospectively:
Exclusion Criteria:
Patients receive tacrolimus IV or PO every 12 hours on days -3 to 180 with taper beginning on day 100 in the absence of GVHD. Patients also receive MMF PO every 8 hours on days 0-29 and then every 12 hours on days 30-96 with taper beginning on day 40 in the absence of GVHD.
Drug: Fludarabine Phosphate · Radiation: Total-Body Irradiation · Procedure: Peripheral Blood Stem Cell Transplantation · Procedure: Allogeneic Hematopoietic Stem Cell Transplantation · Drug: Tacrolimus · Drug: Mycophenolate Mofetil
Patients receive tacrolimus IV or PO every 12 hours on days -3 to 150 with taper beginning on day 100 in the absence of GVHD. Patients also receive MMF PO every 8 hours on days 0-29 and then every 12 hours on days 30-180 with taper beginning on day 150 in the absence of GVHD.
Drug: Fludarabine Phosphate · Radiation: Total-Body Irradiation · Procedure: Peripheral Blood Stem Cell Transplantation · Procedure: Allogeneic Hematopoietic Stem Cell Transplantation · Drug: Tacrolimus · Drug: Mycophenolate Mofetil
Patients receive tacrolimus and MMF as in arm II. Patients also receive sirolimus PO once daily on days -3 to 80.
Drug: Fludarabine Phosphate · Radiation: Total-Body Irradiation · Procedure: Peripheral Blood Stem Cell Transplantation · Procedure: Allogeneic Hematopoietic Stem Cell Transplantation · Drug: Tacrolimus · Drug: Mycophenolate Mofetil · Drug: Sirolimus
Given IV
Also known as: 2-F-ara-AMP, Beneflur, SH T 586
Undergo total-body irradiation
Also known as: TBI, Total Body Irradiation, Whole-Body Irradiation
Undergo allogeneic peripheral blood stem cell transplantation
Also known as: PBPC transplantation, Peripheral Blood Progenitor Cell Transplantation, Peripheral Stem Cell Support, Peripheral Stem Cell Transplantation
Undergo allogeneic peripheral blood stem cell transplantation
Also known as: HSC, HSCT
Given IV or PO
Also known as: Advagraf, FK 506
Given PO
Also known as: Cellcept, MMF
Given PO
Also known as: AY 22989, RAPA, SILA 9268A, WY-090217
Number of Participants With Grades II-IV Acute GVHD
Number of patients with grades II-IV acute GVHD aGVHD Stages Skin: 1. a maculopapular eruption involving \< 25% BSA 2. a maculopapular eruption involving 25 - 50% BSA 3. generalized erythroderma 4. generalized erythroderma w/ bullous formation and often w/ desquamation Liver: 1. bilirubin 2.0 - 3.0 mg/100 mL 2. bilirubin 3 - 5.9 mg/100 mL 3. bilirubin 6 - 14.9 mg/100 mL 4. bilirubin \> 15 mg/100 mL Gut: Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients w/ visible bloody diarrhea are at least stage 2 gut and grade 3 overall. aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death
Time frame: 150 days after transplant
Number of Non-Relapse Mortalities
Percentage of NRM as estimated by cumulative incidence methods with competing risks. Cumulative incidence methods are the standard way to estimate incidence of an endpoint in the presence of competing risks and censoring (ref)" Here is the reference. Gooley TA, Leisenring W, Crowley J, Storer BE: Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Statistics in Medicine 18:695-706, 1999. PMID 10204198
Time frame: 200 days after transplant
Number of Participants Utilizing High-Dose Corticosteroids
Number of patients utilizing high-dose corticosteroids (as a surrogate marker for reduction of acute GVHD), estimated by cumulative incidence methods. Cumulative incidence methods are the standard way to estimate incidence of an endpoint in the presence of competing risks and censoring (ref)" Here is the reference. Gooley TA, Leisenring W, Crowley J, Storer BE: Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Statistics in Medicine 18:695-706, 1999. PMID 10204198
Time frame: 150 days after transplant
Number of Participants Surviving Overall
Number of patients surviving, estimated by cumulative incidence methods Cumulative incidence methods are the standard way to estimate incidence of an endpoint in the presence of competing risks and censoring (ref)" Here is the reference. Gooley TA, Leisenring W, Crowley J, Storer BE: Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Statistics in Medicine 18:695-706, 1999. PMID 10204198
Time frame: 1 Year post-transplant
Number of Participants Surviving Without Progression
Number of patients with progression-free survival, estimated by cumulative incidence methods Cumulative incidence methods are the standard way to estimate incidence of an endpoint in the presence of competing risks and censoring (ref)" Here is the reference. Gooley TA, Leisenring W, Crowley J, Storer BE: Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Statistics in Medicine 18:695-706, 1999. PMID 10204198
Time frame: 2 Years post-transplant
| Milestone | Arm I (MMF and Tacrolimus) | Arm II (MMF and Tacrolimus Alternate Schedule) | Arm III (MMF, Tacrolimus, and Sirolimus) |
|---|---|---|---|
| Started | 70 | 71 | 69 |
| Completed | 70 | 71 | 69 |
| Not completed | 0 | 0 | 0 |
Number of patients with grades II-IV acute GVHD aGVHD Stages Skin: 1. a maculopapular eruption involving \< 25% BSA 2. a maculopapular eruption involving 25 - 50% BSA 3. generalized erythroderma 4. generalized erythroderma w/ bullous formation and often w/ desquamation Liver: 1. bilirubin 2.0 - 3.0 mg/100 mL 2. bilirubin 3 - 5.9 mg/100 mL 3. bilirubin 6 - 14.9 mg/100 mL 4. bilirubin \> 15 mg/100 mL Gut: Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients w/ visible bloody diarrhea are at least stage 2 gut and grade 3 overall. aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death
| Participants | Arm I (MMF and Tacrolimus) | Arm II (MMF and Tacrolimus Alternate Schedule) | Arm III (MMF, Tacrolimus, and Sirolimus) |
|---|---|---|---|
| Number of Participants With Grades II-IV Acute GVHD | 44 | 34 | 32 |
Percentage of NRM as estimated by cumulative incidence methods with competing risks. Cumulative incidence methods are the standard way to estimate incidence of an endpoint in the presence of competing risks and censoring (ref)" Here is the reference. Gooley TA, Leisenring W, Crowley J, Storer BE: Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Statistics in Medicine 18:695-706, 1999. PMID 10204198
| Participants | Arm I (MMF and Tacrolimus) | Arm II (MMF and Tacrolimus Alternate Schedule) | Arm III (MMF, Tacrolimus, and Sirolimus) |
|---|---|---|---|
| Number of Non-Relapse Mortalities | 3 | 6 | 2 |
Number of patients utilizing high-dose corticosteroids (as a surrogate marker for reduction of acute GVHD), estimated by cumulative incidence methods. Cumulative incidence methods are the standard way to estimate incidence of an endpoint in the presence of competing risks and censoring (ref)" Here is the reference. Gooley TA, Leisenring W, Crowley J, Storer BE: Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Statistics in Medicine 18:695-706, 1999. PMID 10204198
| Participants | Arm I (MMF and Tacrolimus) | Arm II (MMF and Tacrolimus Alternate Schedule) | Arm III (MMF, Tacrolimus, and Sirolimus) |
|---|---|---|---|
| Number of Participants Utilizing High-Dose Corticosteroids | 38 | 35 | 22 |
Number of patients surviving, estimated by cumulative incidence methods Cumulative incidence methods are the standard way to estimate incidence of an endpoint in the presence of competing risks and censoring (ref)" Here is the reference. Gooley TA, Leisenring W, Crowley J, Storer BE: Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Statistics in Medicine 18:695-706, 1999. PMID 10204198
| Participants | Arm I (MMF and Tacrolimus) | Arm II (MMF and Tacrolimus Alternate Schedule) | Arm III (MMF, Tacrolimus, and Sirolimus) |
|---|---|---|---|
| Number of Participants Surviving Overall | 48 | 47 | 40 |
Number of patients with progression-free survival, estimated by cumulative incidence methods Cumulative incidence methods are the standard way to estimate incidence of an endpoint in the presence of competing risks and censoring (ref)" Here is the reference. Gooley TA, Leisenring W, Crowley J, Storer BE: Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Statistics in Medicine 18:695-706, 1999. PMID 10204198
| Participants | Arm I (MMF and Tacrolimus) | Arm II (MMF and Tacrolimus Alternate Schedule) | Arm III (MMF, Tacrolimus, and Sirolimus) |
|---|---|---|---|
| Number of Participants Surviving Without Progression | 28 | 27 | 26 |
Collected over AEs: From the start of conditioning to 100 Days post-transplant SAEs: From the start of conditioning to 200 Days post-transplant All-Cause Mortality: Conditioning through 1 Year. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Arm I (MMF and Tacrolimus) | 21/69 (30.4%) | 9/69 (13%) | 25/69 (36.2%) |
| Arm II (MMF and Tacrolimus Alternate Schedule) | 24/71 (33.8%) | 8/71 (11.3%) | 22/71 (31%) |
| Arm III (MMF, Tacrolimus, and Sirolimus) | 28/68 (41.2%) | 7/68 (10.3%) | 25/68 (36.8%) |
| Event | Arm I (MMF and Tacrolimus) | Arm II (MMF and Tacrolimus Alternate Schedule) | Arm III (MMF, Tacrolimus, and Sirolimus) |
|---|---|---|---|
| Respiratory infectionInfections and infestations | 1/69 | 3/71 | 0/68 |
| GVHDImmune system disorders | 0/69 | 0/71 | 2/68 |
| ThrombosisVascular disorders | 2/69 | 1/71 | 0/68 |
| SepsisInfections and infestations | 1/69 | 1/71 | 1/68 |
| GvHD w/ infectionImmune system disorders | 1/69 | 1/71 | 1/68 |
| Multi-organ failureGeneral disorders | 0/69 | 1/71 | 1/68 |
| HypertensionCardiac disorders | 0/69 | 0/71 | 1/68 |
| Renal insufficiencyRenal and urinary disorders | 0/69 | 0/71 | 1/68 |
| Severe hemoptysisInjury, poisoning and procedural complications | 1/69 | 0/71 | 0/68 |
| CNS cerebrovascular ischemiaVascular disorders | 1/69 | 0/71 | 0/68 |
| Event | Arm I (MMF and Tacrolimus) | Arm II (MMF and Tacrolimus Alternate Schedule) | Arm III (MMF, Tacrolimus, and Sirolimus) |
|---|---|---|---|
| HypoxiaRespiratory, thoracic and mediastinal disorders | 5/69 | 8/71 | 4/68 |
| DiarrheaGastrointestinal disorders | 5/69 | 1/71 | 4/68 |
| Creatinine increasedInvestigations | 1/69 | 1/71 | 4/68 |
| NauseaGastrointestinal disorders | 1/69 | 1/71 | 3/68 |
| FeverGeneral disorders | 0/69 | 3/71 | 0/68 |
| Neutrophil count decreasedInvestigations | 1/69 | 3/71 | 0/68 |
| HeadacheNervous system disorders | 0/69 | 1/71 | 2/68 |
| Pleural effusionRespiratory, thoracic and mediastinal disorders | 0/69 | 1/71 | 2/68 |
| SyncopeNervous system disorders | 2/69 | 0/71 | 2/68 |
| Thromboembolic eventVascular disorders | 1/69 | 1/71 | 2/68 |
| Age, Categorical(Participants) | Arm I (MMF and Tacrolimus) | Arm II (MMF and Tacrolimus Alternate Schedule) | Arm III (MMF, Tacrolimus, and Sirolimus) | Total |
|---|---|---|---|---|
| <=18 years | 0 | 1 | 1 | 2 |
| Between 18 and 65 years | 57 | 50 | 49 | 156 |
| >=65 years | 13 | 20 | 19 | 52 |
| Age, Continuous(years) | Arm I (MMF and Tacrolimus) | Arm II (MMF and Tacrolimus Alternate Schedule) | Arm III (MMF, Tacrolimus, and Sirolimus) | Total |
|---|---|---|---|---|
| Median | 60 (26 to 74) | 60 (13 to 72) | 60 (15 to 75) | 60 (13 to 75) |
| Sex: Female, Male(Participants) | Arm I (MMF and Tacrolimus) | Arm II (MMF and Tacrolimus Alternate Schedule) | Arm III (MMF, Tacrolimus, and Sirolimus) | Total |
|---|---|---|---|---|
| Female | 29 | 30 | 23 | 82 |
| Male | 41 | 41 | 46 | 128 |
| Region of Enrollment(participants) | Arm I (MMF and Tacrolimus) | Arm II (MMF and Tacrolimus Alternate Schedule) | Arm III (MMF, Tacrolimus, and Sirolimus) | Total |
|---|---|---|---|---|
| United States | 59 | 59 | 58 | 176 |
| Denmark | 5 | 5 | 5 | 15 |
| Germany | 6 | 7 | 6 | 19 |
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