Trial Outcomes & Findings for Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders (NCT NCT00358657)
NCT ID: NCT00358657
Last Updated: 2021-01-26
Results Overview
Number of patients with graft rejection (CD3 donor chimerisms \<5%).
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
14 participants
Primary outcome timeframe
Day 84
Results posted on
2021-01-26
Participant Flow
Participant milestones
| Measure |
Treatment (Chemo, Total-body Irradiation, Transplant)
See Detailed Description
Allogeneic Bone Marrow Transplantation: Undergo allogeneic bone marrow transplantation
Cyclophosphamide: Given IV
Fludarabine Phosphate: Given IV
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic bone marrow transplantation
Sirolimus: Given PO
Tacrolimus: Given IV or PO
Total-Body Irradiation: Undergo total-body irradiation
|
|---|---|
|
Overall Study
STARTED
|
14
|
|
Overall Study
COMPLETED
|
14
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders
Baseline characteristics by cohort
| Measure |
Treatment (Chemo, Total-body Irradiation, Transplant)
n=14 Participants
See Detailed Description
Allogeneic Bone Marrow Transplantation: Undergo allogeneic bone marrow transplantation
Cyclophosphamide: Given IV
Fludarabine Phosphate: Given IV
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic bone marrow transplantation
Sirolimus: Given PO
Tacrolimus: Given IV or PO
Total-Body Irradiation: Undergo total-body irradiation
|
|---|---|
|
Age, Categorical
<=18 years
|
13 Participants
n=93 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=93 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=93 Participants
|
|
Age, Continuous
|
10.7 years
n=93 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
14 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Black or African American
|
7 Participants
n=93 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=93 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
|
Region of Enrollment
United States
|
14 participants
n=93 Participants
|
PRIMARY outcome
Timeframe: Day 84Number of patients with graft rejection (CD3 donor chimerisms \<5%).
Outcome measures
| Measure |
Treatment (Chemo, Total-body Irradiation, Transplant)
n=14 Participants
See Detailed Description
Allogeneic Bone Marrow Transplantation: Undergo allogeneic bone marrow transplantation
Cyclophosphamide: Given IV
Fludarabine Phosphate: Given IV
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic bone marrow transplantation
Sirolimus: Given PO
Tacrolimus: Given IV or PO
Total-Body Irradiation: Undergo total-body irradiation
|
|---|---|
|
Graft Rejection
|
1 Participants
|
PRIMARY outcome
Timeframe: Day 84Number of patients with graft failure (grade IV thrombocytopenia and neutropenia after day 21 that lasts \> 2 weeks andn is refractory to growth factor support).
Outcome measures
| Measure |
Treatment (Chemo, Total-body Irradiation, Transplant)
n=14 Participants
See Detailed Description
Allogeneic Bone Marrow Transplantation: Undergo allogeneic bone marrow transplantation
Cyclophosphamide: Given IV
Fludarabine Phosphate: Given IV
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic bone marrow transplantation
Sirolimus: Given PO
Tacrolimus: Given IV or PO
Total-Body Irradiation: Undergo total-body irradiation
|
|---|---|
|
Graft Failure
|
0 Participants
|
SECONDARY outcome
Timeframe: By day 84Number of patients who achieve greater than 5% donor T-cell (CD3+) chimerisms
Outcome measures
| Measure |
Treatment (Chemo, Total-body Irradiation, Transplant)
n=14 Participants
See Detailed Description
Allogeneic Bone Marrow Transplantation: Undergo allogeneic bone marrow transplantation
Cyclophosphamide: Given IV
Fludarabine Phosphate: Given IV
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic bone marrow transplantation
Sirolimus: Given PO
Tacrolimus: Given IV or PO
Total-Body Irradiation: Undergo total-body irradiation
|
|---|---|
|
Proportion of Patients Who Achieve Greater Than 5% Donor T-cell Chimerism
|
13 Participants
|
SECONDARY outcome
Timeframe: Day 100 post transplantThe number of patients with transplant related mortality
Outcome measures
| Measure |
Treatment (Chemo, Total-body Irradiation, Transplant)
n=14 Participants
See Detailed Description
Allogeneic Bone Marrow Transplantation: Undergo allogeneic bone marrow transplantation
Cyclophosphamide: Given IV
Fludarabine Phosphate: Given IV
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic bone marrow transplantation
Sirolimus: Given PO
Tacrolimus: Given IV or PO
Total-Body Irradiation: Undergo total-body irradiation
|
|---|---|
|
Number of Patients With Transplant Related Mortality
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 100 post transplantNumber of patients diagnosed with overall GI/G2 acute GVHD by Day 100
Outcome measures
| Measure |
Treatment (Chemo, Total-body Irradiation, Transplant)
n=14 Participants
See Detailed Description
Allogeneic Bone Marrow Transplantation: Undergo allogeneic bone marrow transplantation
Cyclophosphamide: Given IV
Fludarabine Phosphate: Given IV
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic bone marrow transplantation
Sirolimus: Given PO
Tacrolimus: Given IV or PO
Total-Body Irradiation: Undergo total-body irradiation
|
|---|---|
|
Incidence of Grade I/II Acute Graft Versus Host Disease (GVHD)
|
7 Participants
|
SECONDARY outcome
Timeframe: Day 100 post transplantNumber of patients diagnosed with overall GIII/IV acute GVHD by Day 100
Outcome measures
| Measure |
Treatment (Chemo, Total-body Irradiation, Transplant)
n=14 Participants
See Detailed Description
Allogeneic Bone Marrow Transplantation: Undergo allogeneic bone marrow transplantation
Cyclophosphamide: Given IV
Fludarabine Phosphate: Given IV
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic bone marrow transplantation
Sirolimus: Given PO
Tacrolimus: Given IV or PO
Total-Body Irradiation: Undergo total-body irradiation
|
|---|---|
|
Incidence of Grade III/IV Acute Graft Versus Host Disease (GVHD)
|
5 Participants
|
SECONDARY outcome
Timeframe: 1 year post transplantNumber of patients diagnosed with chronic GVHD by 1 year post transplant
Outcome measures
| Measure |
Treatment (Chemo, Total-body Irradiation, Transplant)
n=14 Participants
See Detailed Description
Allogeneic Bone Marrow Transplantation: Undergo allogeneic bone marrow transplantation
Cyclophosphamide: Given IV
Fludarabine Phosphate: Given IV
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic bone marrow transplantation
Sirolimus: Given PO
Tacrolimus: Given IV or PO
Total-Body Irradiation: Undergo total-body irradiation
|
|---|---|
|
Incidence of Chronic GVHD
|
11 Participants
|
SECONDARY outcome
Timeframe: 1 year post transplantNumber of patients with normal range CD3 @ 1 year post transplant
Outcome measures
| Measure |
Treatment (Chemo, Total-body Irradiation, Transplant)
n=13 Participants
See Detailed Description
Allogeneic Bone Marrow Transplantation: Undergo allogeneic bone marrow transplantation
Cyclophosphamide: Given IV
Fludarabine Phosphate: Given IV
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic bone marrow transplantation
Sirolimus: Given PO
Tacrolimus: Given IV or PO
Total-Body Irradiation: Undergo total-body irradiation
|
|---|---|
|
Immune Reconstitution
|
4 Participants
|
SECONDARY outcome
Timeframe: Through day 200 after HCTNumber of patients with clinically significant infections requiring treatment within 200 days after HCT
Outcome measures
| Measure |
Treatment (Chemo, Total-body Irradiation, Transplant)
n=14 Participants
See Detailed Description
Allogeneic Bone Marrow Transplantation: Undergo allogeneic bone marrow transplantation
Cyclophosphamide: Given IV
Fludarabine Phosphate: Given IV
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic bone marrow transplantation
Sirolimus: Given PO
Tacrolimus: Given IV or PO
Total-Body Irradiation: Undergo total-body irradiation
|
|---|---|
|
Number of Patients With Infections
|
11 Participants
|
Adverse Events
Treatment (Chemo, Total-body Irradiation, Transplant)
Serious events: 1 serious events
Other events: 8 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Treatment (Chemo, Total-body Irradiation, Transplant)
n=14 participants at risk
See Detailed Description
Allogeneic Bone Marrow Transplantation: Undergo allogeneic bone marrow transplantation
Cyclophosphamide: Given IV
Fludarabine Phosphate: Given IV
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic bone marrow transplantation
Sirolimus: Given PO
Tacrolimus: Given IV or PO
Total-Body Irradiation: Undergo total-body irradiation
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia requiring mechanical ventilation
|
7.1%
1/14 • Number of events 1 • Day 200 post initiation of conditioning therapy
|
|
Infections and infestations
Sepsis
|
7.1%
1/14 • Number of events 1 • Day 200 post initiation of conditioning therapy
|
|
Gastrointestinal disorders
Pancreatitis with shock
|
7.1%
1/14 • Number of events 1 • Day 200 post initiation of conditioning therapy
|
Other adverse events
| Measure |
Treatment (Chemo, Total-body Irradiation, Transplant)
n=14 participants at risk
See Detailed Description
Allogeneic Bone Marrow Transplantation: Undergo allogeneic bone marrow transplantation
Cyclophosphamide: Given IV
Fludarabine Phosphate: Given IV
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic bone marrow transplantation
Sirolimus: Given PO
Tacrolimus: Given IV or PO
Total-Body Irradiation: Undergo total-body irradiation
|
|---|---|
|
Vascular disorders
Hypotension
|
7.1%
1/14 • Number of events 1 • Day 200 post initiation of conditioning therapy
|
|
Cardiac disorders
Pericardial effusion
|
7.1%
1/14 • Number of events 1 • Day 200 post initiation of conditioning therapy
|
|
Gastrointestinal disorders
Typhlitis
|
7.1%
1/14 • Number of events 1 • Day 200 post initiation of conditioning therapy
|
|
Gastrointestinal disorders
Pancreatisis
|
7.1%
1/14 • Number of events 1 • Day 200 post initiation of conditioning therapy
|
|
Gastrointestinal disorders
Mucositis oral
|
7.1%
1/14 • Number of events 1 • Day 200 post initiation of conditioning therapy
|
|
Investigations
Blood bilirubin increased
|
7.1%
1/14 • Number of events 1 • Day 200 post initiation of conditioning therapy
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
21.4%
3/14 • Number of events 3 • Day 200 post initiation of conditioning therapy
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
7.1%
1/14 • Number of events 1 • Day 200 post initiation of conditioning therapy
|
|
Nervous system disorders
Seizure
|
7.1%
1/14 • Number of events 1 • Day 200 post initiation of conditioning therapy
|
|
Blood and lymphatic system disorders
Hemolytic uremic syndrome
|
7.1%
1/14 • Number of events 1 • Day 200 post initiation of conditioning therapy
|
|
Renal and urinary disorders
Acute kidney injury
|
7.1%
1/14 • Number of events 1 • Day 200 post initiation of conditioning therapy
|
Additional Information
Dr. Kanwaldeep Mallhi
Fred Hutchinson Cancer Research Center
Phone: 206-667-2014
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place