Trial Outcomes & Findings for Shorter Course Tacro After NMA, Related Donor PBSCT With High-dose Posttransplant Cy for Hard-to-Engraft Malignancies (NCT NCT02556931)
NCT ID: NCT02556931
Last Updated: 2022-11-03
Results Overview
This outcome measures the feasibility of stopping prophylactic tacrolimus at Day 90.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
117 participants
Primary outcome timeframe
Day 90
Results posted on
2022-11-03
Participant Flow
Participant milestones
| Measure |
PBSCT D90
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
PBSCT D60
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|---|
|
Overall Study
STARTED
|
57
|
60
|
|
Overall Study
COMPLETED
|
57
|
60
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
PBSCT D90
n=57 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
PBSCT D60
n=60 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
Total
n=117 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=57 Participants
|
0 Participants
n=60 Participants
|
0 Participants
n=117 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
34 Participants
n=57 Participants
|
36 Participants
n=60 Participants
|
70 Participants
n=117 Participants
|
|
Age, Categorical
>=65 years
|
23 Participants
n=57 Participants
|
24 Participants
n=60 Participants
|
47 Participants
n=117 Participants
|
|
Age, Continuous
|
62 years
n=57 Participants
|
63 years
n=60 Participants
|
62.5 years
n=117 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=57 Participants
|
19 Participants
n=60 Participants
|
36 Participants
n=117 Participants
|
|
Sex: Female, Male
Male
|
40 Participants
n=57 Participants
|
41 Participants
n=60 Participants
|
81 Participants
n=117 Participants
|
|
Race and Ethnicity Not Collected
|
—
|
—
|
0 Participants
Race and Ethnicity were not collected from any participant.
|
|
Region of Enrollment
United States
|
57 Participants
n=57 Participants
|
60 Participants
n=60 Participants
|
117 Participants
n=117 Participants
|
PRIMARY outcome
Timeframe: Day 90Population: Safety stopping criteria were not met after discontinuation of tacrolimus at day 90.
This outcome measures the feasibility of stopping prophylactic tacrolimus at Day 90.
Outcome measures
| Measure |
PBSCT D90
n=57 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Percentage of Participants Who Are Able to Stop Prophylactic Tacrolimus (D90 Cohort)
|
33 Participants
|
PRIMARY outcome
Timeframe: Day 60Population: Of the 42 patients in the D60 cohort who stopped IS early as prespecified, 10 subsequently relapsed.
This outcome measures the feasibility of stopping prophylactic tacrolimus at Day 60.
Outcome measures
| Measure |
PBSCT D90
n=60 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Percentage of Participants Who Are Able to Stop Prophylactic Tacrolimus (D60 Cohort)
|
42 Participants
|
SECONDARY outcome
Timeframe: Between Day 90 and Day 180Population: Of the 57 patients in the D90 cohort, 33 stopped tactolimus early as planned.
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.
Outcome measures
| Measure |
PBSCT D90
n=33 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Participants With Grades III-IV Acute GVHD, Days 90-180 (D90)
|
1 Participants
|
SECONDARY outcome
Timeframe: Between Day 60 and Day 180Number 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.
Outcome measures
| Measure |
PBSCT D90
n=42 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Participants With Grades III-IV Acute GVHD, Days 60-180 (D60)
|
3 Participants
|
SECONDARY outcome
Timeframe: Between Day 90 and Day 180Population: Of the 57 patients in the D90 cohort, 33 stopped tacrolimus early as planned.
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.
Outcome measures
| Measure |
PBSCT D90
n=33 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Participants With Chronic GVHD, Days 90-180 (D90)
|
2 Participants
|
SECONDARY outcome
Timeframe: Between Day 60 and Day 180Population: Of the 60 patients in the D60 cohort, 42 stopped tacrolimus early as planned.
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.
Outcome measures
| Measure |
PBSCT D90
n=42 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Participants With Chronic GVHD, Days 60-180 (D60)
|
1 Participants
|
SECONDARY outcome
Timeframe: Between Day 90 and Day 180Population: Of the 57 patients in the D90 cohort, 33 stopped IS early as planned.
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.
Outcome measures
| Measure |
PBSCT D90
n=33 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Participants Who Experience Graft Failure, Days 90-180 (D90)
|
0 Participants
|
SECONDARY outcome
Timeframe: Between Day 60 and Day 180Population: Of the 60 patients in the D60 cohort, 42 stopped tacrolimus early as planned.
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.
Outcome measures
| Measure |
PBSCT D90
n=42 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Participants Who Experience Graft Failure, Days 60-180 (D60)
|
1 Participants
|
SECONDARY outcome
Timeframe: Between Day 90 and Day 180Population: Of the 57 patients in the D90 cohort, 33 stopped tacrolimus early as planned.
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.
Outcome measures
| Measure |
PBSCT D90
n=33 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Participants Who Experience Disease Relapse, Days 90-180 (D90)
|
14 Participants
|
SECONDARY outcome
Timeframe: Between Day 60 and Day 180Population: Of the 60 patients in the D60 cohort, 42 stopped tacrolimus early as planned.
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.
Outcome measures
| Measure |
PBSCT D90
n=42 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Participants Who Experience Disease Relapse, Days 60-180 (D60)
|
10 Participants
|
SECONDARY outcome
Timeframe: Between Day 90 and Day 180Population: Of the 57 patients in the D90 cohort, 33 stopped tacrolimus early as planned.
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.
Outcome measures
| Measure |
PBSCT D90
n=33 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Participants Who Experience Non-relapse Mortality, Days 90-180 (D90)
|
1 Participants
|
SECONDARY outcome
Timeframe: Between Day 60 and Day 180Population: Of the 60 patients in the D60 cohort, 42 stopped tacrolimus early as planned.
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.
Outcome measures
| Measure |
PBSCT D90
n=42 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Participants Who Experience Non-relapse Mortality, Days 60-180 (D60)
|
2 Participants
|
SECONDARY outcome
Timeframe: Day 360Population: Of the 57 patients in the D90 cohort, 33 stopped tacrolimus early as planned.
Number of participants who experience grade III or IV GVHD by Day 360. All participants are evaluable.
Outcome measures
| Measure |
PBSCT D90
n=33 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Participants Who Experience Grades III-IV GVHD, Day 360 (D90)
|
16 Participants
|
SECONDARY outcome
Timeframe: Day 360Population: Of the 60 patients in the D60 cohort, 42 stopped tacrolimus early as planned.
Number of participants who experience grade III or IV GVHD by Day 360. All participants are evaluable.
Outcome measures
| Measure |
PBSCT D90
n=42 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Number of Participants Who Experience Grades III-IV GVHD, Day 360 (D60)
|
18 Participants
|
SECONDARY outcome
Timeframe: Day 360Population: Of the 57 patients in the D90 cohort, 33 stopped tacrolimus early as planned.
Number of participants who experience severe chronic GVHD requiring additional immunosuppressive therapy by Day 360. All participants are evaluable.
Outcome measures
| Measure |
PBSCT D90
n=33 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Number of Participants With Severe Chronic GVHD, Day 360 (D90)
|
7 Participants
|
SECONDARY outcome
Timeframe: Day 360Population: Of the 60 patients in the D60 cohort, 42 stopped tacrolimus early as planned.
Number of participants who experience severe chronic GVHD requiring additional immunosuppressive therapy by Day 360. All participants are evaluable.
Outcome measures
| Measure |
PBSCT D90
n=42 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Number of Participants With Severe Chronic GVHD, Day 360 (D60)
|
5 Participants
|
SECONDARY outcome
Timeframe: Day 360Population: Of the 57 patients in the D90 cohort, 33 stopped tacrolimus early as planned.
Number of participants who experience graft failure by Day 360. All participants are evaluable.
Outcome measures
| Measure |
PBSCT D90
n=33 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Number of Participants Who Experience Graft Failure, Day 360 (D90)
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 360Population: Of the 60 patients in the D60 cohort, 42 stopped tacrolimus early as planned.
Number of participants who experience graft failure by Day 360. All participants are evaluable.
Outcome measures
| Measure |
PBSCT D90
n=42 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Number of Participants Who Experience Graft Failure, Day 360 (D60)
|
2 Participants
|
SECONDARY outcome
Timeframe: Day 360Population: Of the 57 patients in the D90 cohort, 33 stopped tacrolimus early as planned.
Number of participants who experience disease relapse by Day 360. All participants are evaluable.
Outcome measures
| Measure |
PBSCT D90
n=33 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
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|---|---|
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Number of Participants Who Experience Relapse, Day 360 (D90)
|
14 Participants
|
SECONDARY outcome
Timeframe: Day 360Population: Of the 60 patients in the D60 cohort, 42 stopped tacrolimus early as planned.
Number of participants who experience disease relapse by Day 360. All participants are evaluable.
Outcome measures
| Measure |
PBSCT D90
n=42 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
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|---|---|
|
Number of Participants Who Experience Relapse, Day 360 (D60)
|
10 Participants
|
SECONDARY outcome
Timeframe: Day 360Population: Of the 57 patients in the D90 cohort, 33 stopped tacrolimus early as planned.
Number of participants who die for any reason other than disease relapse by Day 360. All participants are evaluable.
Outcome measures
| Measure |
PBSCT D90
n=33 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Participants Who Experience Non-relapse Mortality, Day 360 (D90)
|
4 Participants
|
SECONDARY outcome
Timeframe: Day 360Population: Of the 60 patients in the D60 cohort, 42 stopped tacrolimus early as planned.
Number of participants who die for any reason other than disease relapse by Day 360. All participants are evaluable.
Outcome measures
| Measure |
PBSCT D90
n=42 Participants
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|
|
Number of Participants Who Experience Non-relapse Mortality, Day 360 (D60)
|
4 Participants
|
Adverse Events
PBSCT D90
Serious events: 9 serious events
Other events: 35 other events
Deaths: 12 deaths
PBSCT D60
Serious events: 5 serious events
Other events: 16 other events
Deaths: 10 deaths
Serious adverse events
| Measure |
PBSCT D90
n=57 participants at risk
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
PBSCT D60
n=60 participants at risk
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|---|
|
Skin and subcutaneous tissue disorders
Severe cGVHD
|
15.8%
9/57 • 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.
|
8.3%
5/60 • 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.
|
Other adverse events
| Measure |
PBSCT D90
n=57 participants at risk
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
PBSCT D60
n=60 participants at risk
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.
Fludarabine: Days -6 through -2: 30 mg/m\^2 IV daily
Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Total body irradiation: Day -1: 200 cGy in a single fraction
Tacrolimus: 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.
Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
|
|---|---|---|
|
Infections and infestations
neutropenic fever
|
61.4%
35/57 • 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.
|
26.7%
16/60 • 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.
|
Additional Information
Dr. Amy Dezern
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Phone: 410-502-7208
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place