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Trial record 11 of 14 for:    mg/m2 | "familial hemophagocytic lymphohistiocytosis"

Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204) (RICHI)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01998633
Recruitment Status : Completed
First Posted : December 2, 2013
Results First Posted : June 1, 2018
Last Update Posted : June 1, 2018
Sponsor:
Collaborators:
National Heart, Lung, and Blood Institute (NHLBI)
Blood and Marrow Transplant Clinical Trials Network
National Cancer Institute (NCI)
National Marrow Donor Program
Information provided by (Responsible Party):
Medical College of Wisconsin

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Hemophagocytic Lymphohistiocytosis
Chronic Active Epstein-Barr Virus Infection
Chronic Granulomatous Disease
HIGM-1
Leukocyte Adhesion Deficiency
IPEX
Intervention Biological: Hematopoietic Stem Cell Transplant
Enrollment 47
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Hematopoietic Stem Cell Transplant
Hide Arm/Group Description

Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.

Hematopoietic Stem Cell Transplant: NOTE: The - sign is the number of days before the transplant and the + sign is the number of days after the transplant.

  • Alemtuzumab 0.2mg/kg Day-14,-13,-12,-11,-10
  • Fludarabine 30 mg/m2 on Day -8,-7,-6,-5,-4
  • Melphalan 140mg/m2 on Day -3

The GVHD prophylaxis will consist of the following:

  • Cyclosporine on Day -3 to Day +100, maintaining a level of 250-500 ng/mL, then taper to Day +180.
  • Methylprednisolone 2 mg/kg/day on Day -2 and -1, 1 mg/kg/day on Day 0 to Day +28, then taper over 1 month. Oral prednisone may be substituted starting on Day 0 (1.2 mg/kg/day)
Period Title: Overall Study
Started 47
Completed 46
Not Completed 1
Reason Not Completed
Death             1
Arm/Group Title Hemophagocytic Lymphohistiocytosis (HLH) Other Primary Immune Deficiencies (PID) Total
Hide Arm/Group Description Participants with Hemophagocytic Lymphohistiocytosis Participants with other primary immune deficiencies, including chronic active EBV disease, chronic granulomatous disease, hyper-immunoglobulin M syndrome, and immune dysregulation, polyendocrinopathy, enteropathy and X-linked (IPEX) syndrome Total of all reporting groups
Overall Number of Baseline Participants 34 12 46
Hide Baseline Analysis Population Description
[Not Specified]
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants 12 participants 46 participants
0-9 Years
26
  76.5%
8
  66.7%
34
  73.9%
10-19 Years
7
  20.6%
2
  16.7%
9
  19.6%
20-29 Years
1
   2.9%
2
  16.7%
3
   6.5%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants 12 participants 46 participants
Female
11
  32.4%
4
  33.3%
15
  32.6%
Male
23
  67.6%
8
  66.7%
31
  67.4%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants 12 participants 46 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
2
   5.9%
0
   0.0%
2
   4.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
4
  11.8%
2
  16.7%
6
  13.0%
White
26
  76.5%
9
  75.0%
35
  76.1%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
2
   5.9%
1
   8.3%
3
   6.5%
Karnofsky Performance Score (KPS)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants 12 participants 46 participants
90-100
24
  70.6%
10
  83.3%
34
  73.9%
80
6
  17.6%
2
  16.7%
8
  17.4%
70
2
   5.9%
0
   0.0%
2
   4.3%
60
1
   2.9%
0
   0.0%
1
   2.2%
50
1
   2.9%
0
   0.0%
1
   2.2%
[1]
Measure Description:

KPS describes patient-perceived global quality of life and functioning on a scale of 0-100.

100: No evidence of disease; 90: Normal activity. Minor signs or symptoms of disease; 80: Normal activity with effort. Some signs or symptoms of disease; 70: Cares for self. Unable to continue normal activity; 60: Needs occasional assistance, but cares for most personal needs; 50: Needs considerable assistance and medical care; 40: Disabled. Needs special care and assistance; 30: Severely disabled. Hospital admission indicated; 20: Very sick. Active supportive therapy needed; 10: Moribund; 0: Dead

Primary Immune Deficiency Type   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants 12 participants 12 participants
Chronic Active Epstein-Barr Virus
3
  25.0%
3
  25.0%
Chronic Granulomatous Disease
5
  41.7%
5
  41.7%
Hyperimmunoglobulin M Syndrome
2
  16.7%
2
  16.7%
IPEX
2
  16.7%
2
  16.7%
[1]
Measure Analysis Population Description: Participants with Primary Immune Deficiencies
Donor Type   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants 12 participants 46 participants
HLA Matched Sibling
7
  20.6%
0
   0.0%
7
  15.2%
1 HLA Locus Mismatched Relative
0
   0.0%
1
   8.3%
1
   2.2%
HLA Matched Unrelated
16
  47.1%
9
  75.0%
25
  54.3%
1 HLA Locus Mismatched Unrelated
11
  32.4%
2
  16.7%
13
  28.3%
[1]
Measure Description: HLA matching and relatedness of donor to recipient
1.Primary Outcome
Title Percentage of Participants With Overall Survival (OS)
Hide Description Overall survival is defined as survival of death from any cause.
Time Frame 1 year and 18 months post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplant
Hide Arm/Group Description:

Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.

Hematopoietic Stem Cell Transplant: NOTE: The - sign is the number of days before the transplant and the + sign is the number of days after the transplant.

  • Alemtuzumab 0.2mg/kg Day-14,-13,-12,-11,-10
  • Fludarabine 30 mg/m2 on Day -8,-7,-6,-5,-4
  • Melphalan 140mg/m2 on Day -3

The GVHD prophylaxis will consist of the following:

  • Cyclosporine on Day -3 to Day +100, maintaining a level of 250-500 ng/mL, then taper to Day +180.
  • Methylprednisolone 2 mg/kg/day on Day -2 and -1, 1 mg/kg/day on Day 0 to Day +28, then taper over 1 month. Oral prednisone may be substituted starting on Day 0 (1.2 mg/kg/day)
Overall Number of Participants Analyzed 46
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
1 year OS
80.4
(68.6 to 88.2)
18 month OS
66.7
(52.9 to 77.3)
2.Secondary Outcome
Title Percentage of Participants With Overall Survival (OS) by Disease Type
Hide Description Overall survival is defined as survival of death from any cause.
Time Frame 1 year and 18 months post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hemophagocytic Lymphohistiocytosis (HLH) Other Primary Immune Deficiencies (PID)
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 34 12
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
1 year OS
82.4
(68.4 to 90.6)
75.0
(47.4 to 89.5)
18 month OS
68.0
(51.8 to 79.7)
62.5
(32.7 to 82.0)
3.Secondary Outcome
Title Percentage of HLH Participants With HLH Reactivation Post-Transplant
Hide Description

Systemic HLH Reactivation: Post-transplant HLH reactivation is defined by clinical and lab evidence of pathologic inflammation (persistent fever, progressive cytopenias, rising ferritin and soluble IL2Rα, decreasing fibrinogen, hepatosplenomegaly, end-organ damage) not attributable to other causes.

Central nervous system (CNS) HLH Reactivation: Reactivation of CNS inflammation in patients with HLH may present with or without altered mental status and is defined by pleocytosis in Cerebrospinal fluid (CSF) or an MRI consistent with CNS inflammation not attributable to other causes.

Time Frame 1 year post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with HLH
Arm/Group Title Hematopoietic Stem Cell Transplant
Hide Arm/Group Description:

Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.

Hematopoietic Stem Cell Transplant: NOTE: The - sign is the number of days before the transplant and the + sign is the number of days after the transplant.

  • Alemtuzumab 0.2mg/kg Day-14,-13,-12,-11,-10
  • Fludarabine 30 mg/m2 on Day -8,-7,-6,-5,-4
  • Melphalan 140mg/m2 on Day -3

The GVHD prophylaxis will consist of the following:

  • Cyclosporine on Day -3 to Day +100, maintaining a level of 250-500 ng/mL, then taper to Day +180.
  • Methylprednisolone 2 mg/kg/day on Day -2 and -1, 1 mg/kg/day on Day 0 to Day +28, then taper over 1 month. Oral prednisone may be substituted starting on Day 0 (1.2 mg/kg/day)
Overall Number of Participants Analyzed 34
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
14.7
(5.2 to 28.7)
4.Secondary Outcome
Title Percentage of Participants With Neutrophil Engraftment
Hide Description Time to absolute neutrophil count (ANC) engraftment is defined as the first of three measurements on different days that the patient has an absolute neutrophil count of ≥ 500x10^6/liter following conditioning regimen induced nadir.
Time Frame Day 42 post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplant
Hide Arm/Group Description:

Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.

Hematopoietic Stem Cell Transplant: NOTE: The - sign is the number of days before the transplant and the + sign is the number of days after the transplant.

  • Alemtuzumab 0.2mg/kg Day-14,-13,-12,-11,-10
  • Fludarabine 30 mg/m2 on Day -8,-7,-6,-5,-4
  • Melphalan 140mg/m2 on Day -3

The GVHD prophylaxis will consist of the following:

  • Cyclosporine on Day -3 to Day +100, maintaining a level of 250-500 ng/mL, then taper to Day +180.
  • Methylprednisolone 2 mg/kg/day on Day -2 and -1, 1 mg/kg/day on Day 0 to Day +28, then taper over 1 month. Oral prednisone may be substituted starting on Day 0 (1.2 mg/kg/day)
Overall Number of Participants Analyzed 46
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
100.0
(90.8 to 100.0)
5.Secondary Outcome
Title Percentage of Participants With Platelet Engraftment
Hide Description Platelet engraftment is defined as the first day of a minimum of three measurements on different days that the patient has achieved a platelet count > 20,000 / microliter AND the patient is platelet transfusion independent for a minimum of seven days following conditioning regimen induced nadir.
Time Frame Day 100 post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplant
Hide Arm/Group Description:

Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.

Hematopoietic Stem Cell Transplant: NOTE: The - sign is the number of days before the transplant and the + sign is the number of days after the transplant.

  • Alemtuzumab 0.2mg/kg Day-14,-13,-12,-11,-10
  • Fludarabine 30 mg/m2 on Day -8,-7,-6,-5,-4
  • Melphalan 140mg/m2 on Day -3

The GVHD prophylaxis will consist of the following:

  • Cyclosporine on Day -3 to Day +100, maintaining a level of 250-500 ng/mL, then taper to Day +180.
  • Methylprednisolone 2 mg/kg/day on Day -2 and -1, 1 mg/kg/day on Day 0 to Day +28, then taper over 1 month. Oral prednisone may be substituted starting on Day 0 (1.2 mg/kg/day)
Overall Number of Participants Analyzed 46
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
88.9
(73.3 to 95.6)
6.Secondary Outcome
Title Percentage of Participants Alive With Sustained Engraftment
Hide Description Sustained engraftment is defined as the occurrence of whole blood donor chimerism > 5% by Day 42 accompanied by the absence of any primary or secondary graft failure. Primary graft failure is defined as < 5% donor chimerism by Day +42, second stem cell infusion, DLI (except in the case of donor CTLs given for infection control), or second HCT following original HCT. Secondary graft failure is defined as < 5% donor chimerism following initial engraftment.
Time Frame 1 year post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplant
Hide Arm/Group Description:

Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.

Hematopoietic Stem Cell Transplant: NOTE: The - sign is the number of days before the transplant and the + sign is the number of days after the transplant.

  • Alemtuzumab 0.2mg/kg Day-14,-13,-12,-11,-10
  • Fludarabine 30 mg/m2 on Day -8,-7,-6,-5,-4
  • Melphalan 140mg/m2 on Day -3

The GVHD prophylaxis will consist of the following:

  • Cyclosporine on Day -3 to Day +100, maintaining a level of 250-500 ng/mL, then taper to Day +180.
  • Methylprednisolone 2 mg/kg/day on Day -2 and -1, 1 mg/kg/day on Day 0 to Day +28, then taper over 1 month. Oral prednisone may be substituted starting on Day 0 (1.2 mg/kg/day)
Overall Number of Participants Analyzed 46
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
39.1
(25.1 to 54.6)
7.Secondary Outcome
Title Percentage of Participants Alive With Sustained Engraftment by Disease Type
Hide Description Sustained engraftment is defined as the occurrence of whole blood donor chimerism > 5% by Day 42 accompanied by the absence of any primary or secondary graft failure. Primary graft failure is defined as < 5% donor chimerism by Day +42, second stem cell infusion, DLI (except in the case of donor CTLs given for infection control), or second HCT following original HCT. Secondary graft failure is defined as < 5% donor chimerism following initial engraftment.
Time Frame 1 year post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hemophagocytic Lymphohistiocytosis (HLH) Other Primary Immune Deficiencies (PID)
Hide Arm/Group Description:
Participants with Hemophagocytic Lymphohistiocytosis
Participants with other primary immune deficiencies, including chronic active EBV disease, chronic granulomatous disease, hyper-immunoglobulin M syndrome, and immune dysregulation, polyendocrinopathy, enteropathy and X-linked (IPEX) syndrome
Overall Number of Participants Analyzed 34 12
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
41.2
(24.7 to 59.3)
33.3
(9.9 to 65.1)
8.Secondary Outcome
Title Number of Participants With Acute Graft-Versus-Host Disease (GVHD)
Hide Description

Acute GVHD is graded according to the scoring system proposed by Przepiorka et al.1995:

Skin stage:

0: No rash

  1. Rash <25% of body surface area
  2. Rash on 25-50% of body surface area
  3. Rash on > 50% of body surface area
  4. Generalized erythroderma with bullous formation

Liver stage (based on bilirubin level)*:

0: <2 mg/dL

  1. 2-3 mg/dL
  2. 3.01-6 mg/dL
  3. 6.01-15.0 mg/dL
  4. >15 mg/dL

GI stage*:

0: No diarrhea or diarrhea <500 mL/day

  1. Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD
  2. Diarrhea 1000-1499 mL/day
  3. Diarrhea >1500 mL/day
  4. Severe abdominal pain with or without ileus * If multiple etiologies are listed for liver or GI, the organ system is downstaged by 1.

GVHD grade:

0: All organ stages 0 or GVHD not listed as an etiology I: Skin stage 1-2 and liver and GI stage 0 II: Skin stage 3 or liver or GI stage 1 III: Liver stage 2-3 or GI stage 2-4 IV: Skin or liver stage 4

Time Frame 1 year post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplant
Hide Arm/Group Description:

Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.

Hematopoietic Stem Cell Transplant: NOTE: The - sign is the number of days before the transplant and the + sign is the number of days after the transplant.

  • Alemtuzumab 0.2mg/kg Day-14,-13,-12,-11,-10
  • Fludarabine 30 mg/m2 on Day -8,-7,-6,-5,-4
  • Melphalan 140mg/m2 on Day -3

The GVHD prophylaxis will consist of the following:

  • Cyclosporine on Day -3 to Day +100, maintaining a level of 250-500 ng/mL, then taper to Day +180.
  • Methylprednisolone 2 mg/kg/day on Day -2 and -1, 1 mg/kg/day on Day 0 to Day +28, then taper over 1 month. Oral prednisone may be substituted starting on Day 0 (1.2 mg/kg/day)
Overall Number of Participants Analyzed 46
Measure Type: Count of Participants
Unit of Measure: Participants
Grade 0 or I
35
  76.1%
Grade II
3
   6.5%
Grade III
5
  10.9%
Grade IV
3
   6.5%
9.Secondary Outcome
Title Percentage of Participants With Grade II-IV and Grade III-IV Acute GVHD
Hide Description

Acute GVHD is graded according to the scoring system proposed by Przepiorka et al.1995:

Skin stage:

0: No rash

  1. Rash <25% of body surface area
  2. Rash on 25-50% of body surface area
  3. Rash on > 50% of body surface area
  4. Generalized erythroderma with bullous formation

Liver stage (based on bilirubin level)*:

0: <2 mg/dL

  1. 2-3 mg/dL
  2. 3.01-6 mg/dL
  3. 6.01-15.0 mg/dL
  4. >15 mg/dL

GI stage*:

0: No diarrhea or diarrhea <500 mL/day

  1. Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD
  2. Diarrhea 1000-1499 mL/day
  3. Diarrhea >1500 mL/day
  4. Severe abdominal pain with or without ileus * If multiple etiologies are listed for liver or GI, the organ system is downstaged by 1.

GVHD grade:

0: All organ stages 0 or GVHD not listed as an etiology I: Skin stage 1-2 and liver and GI stage 0 II: Skin stage 3 or liver or GI stage 1 III: Liver stage 2-3 or GI stage 2-4 IV: Skin or liver stage 4

Time Frame Day 100 and 6 months post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplant
Hide Arm/Group Description:

Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.

Hematopoietic Stem Cell Transplant: NOTE: The - sign is the number of days before the transplant and the + sign is the number of days after the transplant.

  • Alemtuzumab 0.2mg/kg Day-14,-13,-12,-11,-10
  • Fludarabine 30 mg/m2 on Day -8,-7,-6,-5,-4
  • Melphalan 140mg/m2 on Day -3

The GVHD prophylaxis will consist of the following:

  • Cyclosporine on Day -3 to Day +100, maintaining a level of 250-500 ng/mL, then taper to Day +180.
  • Methylprednisolone 2 mg/kg/day on Day -2 and -1, 1 mg/kg/day on Day 0 to Day +28, then taper over 1 month. Oral prednisone may be substituted starting on Day 0 (1.2 mg/kg/day)
Overall Number of Participants Analyzed 46
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
Grade II-IV Acute GVHD at Day 100
17.4
(8.1 to 29.7)
Grade II-IV Acute GVHD at 6 Months
26.1
(14.4 to 39.4)
Grade III-IV Acute GVHD at Day 100
10.9
(4.0 to 21.3)
Grade III-IV Acute GVHD at 6 Months
17.4
(8.1 to 29.7)
10.Secondary Outcome
Title Number of Participants With Chronic GVHD
Hide Description Chronic GVHD is classified per 2005 NIH Consensus Criteria (Filipovich et al. 2005) into categories of severity: none, mild, moderate, and severe.
Time Frame 1 year post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplant
Hide Arm/Group Description:

Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.

Hematopoietic Stem Cell Transplant: NOTE: The - sign is the number of days before the transplant and the + sign is the number of days after the transplant.

  • Alemtuzumab 0.2mg/kg Day-14,-13,-12,-11,-10
  • Fludarabine 30 mg/m2 on Day -8,-7,-6,-5,-4
  • Melphalan 140mg/m2 on Day -3

The GVHD prophylaxis will consist of the following:

  • Cyclosporine on Day -3 to Day +100, maintaining a level of 250-500 ng/mL, then taper to Day +180.
  • Methylprednisolone 2 mg/kg/day on Day -2 and -1, 1 mg/kg/day on Day 0 to Day +28, then taper over 1 month. Oral prednisone may be substituted starting on Day 0 (1.2 mg/kg/day)
Overall Number of Participants Analyzed 46
Measure Type: Count of Participants
Unit of Measure: Participants
None
34
  73.9%
Mild
10
  21.7%
Severe
2
   4.3%
11.Secondary Outcome
Title Percentage of Participants With Chronic GVHD
Hide Description Chronic GVHD is classified per 2005 NIH Consensus Criteria (Filipovich et al. 2005) into categories of severity: none, mild, moderate, and severe. Occurrence of chronic GVHD is defined as the occurrence of mild, moderate, or severe chronic GVHD per this classification.
Time Frame 1 year post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplant
Hide Arm/Group Description:

Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.

Hematopoietic Stem Cell Transplant: NOTE: The - sign is the number of days before the transplant and the + sign is the number of days after the transplant.

  • Alemtuzumab 0.2mg/kg Day-14,-13,-12,-11,-10
  • Fludarabine 30 mg/m2 on Day -8,-7,-6,-5,-4
  • Melphalan 140mg/m2 on Day -3

The GVHD prophylaxis will consist of the following:

  • Cyclosporine on Day -3 to Day +100, maintaining a level of 250-500 ng/mL, then taper to Day +180.
  • Methylprednisolone 2 mg/kg/day on Day -2 and -1, 1 mg/kg/day on Day 0 to Day +28, then taper over 1 month. Oral prednisone may be substituted starting on Day 0 (1.2 mg/kg/day)
Overall Number of Participants Analyzed 46
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
26.7
(14.6 to 40.4)
Time Frame 1 year post-transplant
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Hematopoietic Stem Cell Transplant
Hide Arm/Group Description

Participants will undergo a non-myeloablative allogeneic hematopoietic stem cell transplant.

Hematopoietic Stem Cell Transplant: NOTE: The - sign is the number of days before the transplant and the + sign is the number of days after the transplant.

  • Alemtuzumab 0.2mg/kg Day-14,-13,-12,-11,-10
  • Fludarabine 30 mg/m2 on Day -8,-7,-6,-5,-4
  • Melphalan 140mg/m2 on Day -3

The GVHD prophylaxis will consist of the following:

  • Cyclosporine on Day -3 to Day +100, maintaining a level of 250-500 ng/mL, then taper to Day +180.
  • Methylprednisolone 2 mg/kg/day on Day -2 and -1, 1 mg/kg/day on Day 0 to Day +28, then taper over 1 month. Oral prednisone may be substituted starting on Day 0 (1.2 mg/kg/day)
All-Cause Mortality
Hematopoietic Stem Cell Transplant
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Hematopoietic Stem Cell Transplant
Affected / at Risk (%) # Events
Total   14/46 (30.43%)    
Blood and lymphatic system disorders   
Febrile neutropenia  1/46 (2.17%)  1
Haemolytic anaemia  1/46 (2.17%)  1
Cardiac disorders   
Cardiac arrest  2/46 (4.35%)  2
Ventricular hypertrophy  1/46 (2.17%)  1
General disorders   
Death  1/46 (2.17%)  1
Immune system disorders   
Serum sickness  1/46 (2.17%)  1
Infections and infestations   
Sepsis  2/46 (4.35%)  2
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Post transplant lymphoproliferative disorders  1/46 (2.17%)  1
Nervous system disorders   
Cerebrovascular accident  1/46 (2.17%)  1
Haemorrhagic stroke  1/46 (2.17%)  1
Posterior reversible encephalopathy syndrome  1/46 (2.17%)  1
Renal and urinary disorders   
Proteinuria  1/46 (2.17%)  1
Respiratory, thoracic and mediastinal disorders   
Respiratory failure  2/46 (4.35%)  2
Vascular disorders   
Hypotension  1/46 (2.17%)  1
1
Term from vocabulary, MedDRA 20.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Hematopoietic Stem Cell Transplant
Affected / at Risk (%) # Events
Total   0/46 (0.00%)    
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Adam Mendizabal, PhD
Organization: The Emmes Corporation
Phone: 301-251-1161
Responsible Party: Medical College of Wisconsin
ClinicalTrials.gov Identifier: NCT01998633     History of Changes
Other Study ID Numbers: BMTCTN1204
2U10HL069294-11 ( U.S. NIH Grant/Contract )
5U24CA076518 ( U.S. NIH Grant/Contract )
First Submitted: October 29, 2013
First Posted: December 2, 2013
Results First Submitted: March 29, 2018
Results First Posted: June 1, 2018
Last Update Posted: June 1, 2018