Trial Outcomes & Findings for TCR Alpha Beta T-cell and CD19 B-cell Depleted Peripheral Blood Stem Cell Transplantation Using the CliniMACS System for Patients With Non-Malignant Hematologic Disorders From Matched or Mismatched, Related or Unrelated Donors (NCT NCT03615144)
NCT ID: NCT03615144
Last Updated: 2021-03-24
Results Overview
Overall survival is defined as time from transplant to death or last follow-up. Rate greater than 0.75 would be considered a success.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
1 participants
Primary outcome timeframe
2 years
Results posted on
2021-03-24
Participant Flow
Participant milestones
| Measure |
Melphalan/Thiotepa/Clofarabine
Melphalan 70 mg/m2/day x 2, Thiotepa 7.5 mg/kg/day x 2 and Clofarabine 20-30 mg/m2/day x 5. Patients will also receive rabbit anti-thymocyte globulin at 2.5 mg/kg/day x 3 doses prior to the start of conditioning.
Melphalan: Melphalan 70 mg/m2/day x 2
Thiotepa: Thiotepa 7.5 mg/kg/day x 2
Clofarabine: Clofarabine 20-30 mg/m2/day x 5
Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®): antithymocyte globulin (ATG) (rabbit ATG 2.5 mg/kg/day x 3 or equine ATG 15 mg/kg/day x 3 (or 40 mg/kg/day x 1 equine ATG if rabbit ATG is not tolerated) during pre-transplant conditioning to deplete host T-cells that could hamper engraftment.
CliniMACS reagents: Products will then undergo TCR-αβ+ and CD-19 depletion using the CliniMACS.
|
Melphalan/Thiotepa/ Fludarabine
Melphalan 70 mg/m2/day x 2, Thiotepa 7.5 mg/kg/day x 2 and Fludarabine 30 mg/m2/day x 5. Patients will also receive rabbit anti-thymocyte globulin at 2.5 mg/kg/day x 3 doses prior to the start of conditioning.
Melphalan: Melphalan 70 mg/m2/day x 2
Thiotepa: Thiotepa 7.5 mg/kg/day x 2
Fludarabine: Fludarabine 30 mg/m2/day x 5
Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®): antithymocyte globulin (ATG) (rabbit ATG 2.5 mg/kg/day x 3 or equine ATG 15 mg/kg/day x 3 (or 40 mg/kg/day x 1 equine ATG if rabbit ATG is not tolerated) during pre-transplant conditioning to deplete host T-cells that could hamper engraftment.
CliniMACS reagents: Products will then undergo TCR-αβ+ and CD-19 depletion using the CliniMACS.
|
|---|---|---|
|
Overall Study
STARTED
|
1
|
0
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
Melphalan/Thiotepa/Clofarabine
Melphalan 70 mg/m2/day x 2, Thiotepa 7.5 mg/kg/day x 2 and Clofarabine 20-30 mg/m2/day x 5. Patients will also receive rabbit anti-thymocyte globulin at 2.5 mg/kg/day x 3 doses prior to the start of conditioning.
Melphalan: Melphalan 70 mg/m2/day x 2
Thiotepa: Thiotepa 7.5 mg/kg/day x 2
Clofarabine: Clofarabine 20-30 mg/m2/day x 5
Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®): antithymocyte globulin (ATG) (rabbit ATG 2.5 mg/kg/day x 3 or equine ATG 15 mg/kg/day x 3 (or 40 mg/kg/day x 1 equine ATG if rabbit ATG is not tolerated) during pre-transplant conditioning to deplete host T-cells that could hamper engraftment.
CliniMACS reagents: Products will then undergo TCR-αβ+ and CD-19 depletion using the CliniMACS.
|
Melphalan/Thiotepa/ Fludarabine
Melphalan 70 mg/m2/day x 2, Thiotepa 7.5 mg/kg/day x 2 and Fludarabine 30 mg/m2/day x 5. Patients will also receive rabbit anti-thymocyte globulin at 2.5 mg/kg/day x 3 doses prior to the start of conditioning.
Melphalan: Melphalan 70 mg/m2/day x 2
Thiotepa: Thiotepa 7.5 mg/kg/day x 2
Fludarabine: Fludarabine 30 mg/m2/day x 5
Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®): antithymocyte globulin (ATG) (rabbit ATG 2.5 mg/kg/day x 3 or equine ATG 15 mg/kg/day x 3 (or 40 mg/kg/day x 1 equine ATG if rabbit ATG is not tolerated) during pre-transplant conditioning to deplete host T-cells that could hamper engraftment.
CliniMACS reagents: Products will then undergo TCR-αβ+ and CD-19 depletion using the CliniMACS.
|
|---|---|---|
|
Overall Study
Participant no longer eligible for this protocol
|
1
|
0
|
Baseline Characteristics
TCR Alpha Beta T-cell and CD19 B-cell Depleted Peripheral Blood Stem Cell Transplantation Using the CliniMACS System for Patients With Non-Malignant Hematologic Disorders From Matched or Mismatched, Related or Unrelated Donors
Baseline characteristics by cohort
| Measure |
Melphalan/Thiotepa/Clofarabine
n=1 Participants
Melphalan 70 mg/m2/day x 2, Thiotepa 7.5 mg/kg/day x 2 and Clofarabine 20-30 mg/m2/day x 5. Patients will also receive rabbit anti-thymocyte globulin at 2.5 mg/kg/day x 3 doses prior to the start of conditioning.
Melphalan: Melphalan 70 mg/m2/day x 2
Thiotepa: Thiotepa 7.5 mg/kg/day x 2
Clofarabine: Clofarabine 20-30 mg/m2/day x 5
Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®): antithymocyte globulin (ATG) (rabbit ATG 2.5 mg/kg/day x 3 or equine ATG 15 mg/kg/day x 3 (or 40 mg/kg/day x 1 equine ATG if rabbit ATG is not tolerated) during pre-transplant conditioning to deplete host T-cells that could hamper engraftment.
CliniMACS reagents: Products will then undergo TCR-αβ+ and CD-19 depletion using the CliniMACS.
|
Melphalan/Thiotepa/ Fludarabine
Melphalan 70 mg/m2/day x 2, Thiotepa 7.5 mg/kg/day x 2 and Fludarabine 30 mg/m2/day x 5. Patients will also receive rabbit anti-thymocyte globulin at 2.5 mg/kg/day x 3 doses prior to the start of conditioning.
Melphalan: Melphalan 70 mg/m2/day x 2
Thiotepa: Thiotepa 7.5 mg/kg/day x 2
Fludarabine: Fludarabine 30 mg/m2/day x 5
Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®): antithymocyte globulin (ATG) (rabbit ATG 2.5 mg/kg/day x 3 or equine ATG 15 mg/kg/day x 3 (or 40 mg/kg/day x 1 equine ATG if rabbit ATG is not tolerated) during pre-transplant conditioning to deplete host T-cells that could hamper engraftment.
CliniMACS reagents: Products will then undergo TCR-αβ+ and CD-19 depletion using the CliniMACS.
|
Total
n=1 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
20 years
n=5 Participants
|
—
|
20 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
—
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=5 Participants
|
—
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
—
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
1 Participants
n=5 Participants
|
—
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 yearsPopulation: Data were not collected
Overall survival is defined as time from transplant to death or last follow-up. Rate greater than 0.75 would be considered a success.
Outcome measures
Outcome data not reported
Adverse Events
Melphalan/Thiotepa/Clofarabine
Serious events: 1 serious events
Other events: 0 other events
Deaths: 0 deaths
Melphalan/Thiotepa/ Fludarabine
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Melphalan/Thiotepa/Clofarabine
n=1 participants at risk
Melphalan 70 mg/m2/day x 2, Thiotepa 7.5 mg/kg/day x 2 and Clofarabine 20-30 mg/m2/day x 5. Patients will also receive rabbit anti-thymocyte globulin at 2.5 mg/kg/day x 3 doses prior to the start of conditioning.
Melphalan: Melphalan 70 mg/m2/day x 2
Thiotepa: Thiotepa 7.5 mg/kg/day x 2
Clofarabine: Clofarabine 20-30 mg/m2/day x 5
Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®): antithymocyte globulin (ATG) (rabbit ATG 2.5 mg/kg/day x 3 or equine ATG 15 mg/kg/day x 3 (or 40 mg/kg/day x 1 equine ATG if rabbit ATG is not tolerated) during pre-transplant conditioning to deplete host T-cells that could hamper engraftment.
CliniMACS reagents: Products will then undergo TCR-αβ+ and CD-19 depletion using the CliniMACS.
|
Melphalan/Thiotepa/ Fludarabine
Melphalan 70 mg/m2/day x 2, Thiotepa 7.5 mg/kg/day x 2 and Fludarabine 30 mg/m2/day x 5. Patients will also receive rabbit anti-thymocyte globulin at 2.5 mg/kg/day x 3 doses prior to the start of conditioning.
Melphalan: Melphalan 70 mg/m2/day x 2
Thiotepa: Thiotepa 7.5 mg/kg/day x 2
Fludarabine: Fludarabine 30 mg/m2/day x 5
Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®): antithymocyte globulin (ATG) (rabbit ATG 2.5 mg/kg/day x 3 or equine ATG 15 mg/kg/day x 3 (or 40 mg/kg/day x 1 equine ATG if rabbit ATG is not tolerated) during pre-transplant conditioning to deplete host T-cells that could hamper engraftment.
CliniMACS reagents: Products will then undergo TCR-αβ+ and CD-19 depletion using the CliniMACS.
|
|---|---|---|
|
Blood and lymphatic system disorders
Blood and lymphatic system disorders - Other, spec
|
100.0%
1/1 • 1 year
Only 1 participants was accrued before the study was closed to accrual due to low accrual
|
—
0/0 • 1 year
Only 1 participants was accrued before the study was closed to accrual due to low accrual
|
|
Cardiac disorders
Heart failure
|
100.0%
1/1 • 1 year
Only 1 participants was accrued before the study was closed to accrual due to low accrual
|
—
0/0 • 1 year
Only 1 participants was accrued before the study was closed to accrual due to low accrual
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hypertension
|
100.0%
1/1 • 1 year
Only 1 participants was accrued before the study was closed to accrual due to low accrual
|
—
0/0 • 1 year
Only 1 participants was accrued before the study was closed to accrual due to low accrual
|
Other adverse events
Adverse event data not reported
Additional Information
Maria Cancio, MD
Memorial Sloan Kettering Cancer Center
Phone: 212-639-7196
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place