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Allogeneic Hematopoietic Cell Transplantation for Severe Systemic Sclerosis

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: NCT00622895
Recruitment Status : Completed
First Posted : February 25, 2008
Results First Posted : June 4, 2018
Last Update Posted : June 4, 2018
Sponsor:
Collaborator:
National Institute of Allergy and Infectious Diseases (NIAID)
Information provided by (Responsible Party):
George Georges, Fred Hutchinson Cancer Research Center

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Systemic Scleroderma
Severe Systemic Sclerosis
Interventions Drug: fludarabine phosphate
Drug: Mycophenolic Acid
Drug: tacrolimus
Radiation: total-body irradiation
Procedure: bone marrow transplantation
Procedure: reduced intensity allogeneic hematopoietic stem cell transplantation
Procedure: quality-of-life assessment
Other: laboratory biomarker analysis
Other: flow cytometry
Procedure: biopsy
Enrollment 3
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Treatment: Allogeneic HCT After Reduced Intensity Conditioning
Hide Arm/Group Description

Conditioning regimen

  • Day -6: cyclophosphamide 50mg/kg. MESNA will be given for bladder prophylaxis .
  • Day -6, -5, -4: Horse ATG 30mg/kg IV x 3 days.
  • Days -6, -5, -4, -3 and –2: Fludarabine 40 mg/m2/day IV over one hour x 5 days.
  • Day -1: TBI 200 cGy at 6-7 cGy/min from a linear accelerator TBI.
  • Day 0: UCB infusion ( 2 units)

Day –3: Commence tacrolimus at 0.03 mg/kg/day continuous IV infusion until the patient is tolerating oral intake then convert to oral PO b.i.d., continue to day +180 and taper to day +365.

Day 0: After UCB transplant on day 0, mycophenolate mofetil will be given 1gm IV t.i.d. until the patient is tolerating oral intake and can convert to 1 gram PO T.I.D. Stop MMF at Day +30, if no acute GVHD, until day +100, and then taper 11% week over 8 weeks.

Period Title: Overall Study
Started 3
Completed 3
Not Completed 0
Arm/Group Title Treatment: Allogeneic HCT After Reduced Intensity Conditioning
Hide Arm/Group Description

Conditioning regimen

  • Day -6: cyclophosphamide 50mg/kg. MESNA will be given for bladder prophylaxis .
  • Day -6, -5, -4: Horse ATG 30mg/kg IV x 3 days.
  • Days -6, -5, -4, -3 and –2: Fludarabine 40 mg/m2/day IV over one hour x 5 days.
  • Day -1: TBI 200 cGy at 6-7 cGy/min from a linear accelerator TBI.
  • Day 0: UCB infusion ( 2 units)

Day –3: Commence tacrolimus at 0.03 mg/kg/day continuous IV infusion until the patient is tolerating oral intake then convert to oral PO b.i.d., continue to day +180 and taper to day +365.

Day 0: After UCB transplant on day 0, mycophenolate mofetil will be given 1gm IV t.i.d. until the patient is tolerating oral intake and can convert to 1 gram PO T.I.D. Stop MMF at Day +30, if no acute GVHD, until day +100, and then taper 11% week over 8 weeks.

Overall Number of Baseline Participants 3
Hide Baseline Analysis Population Description
Patients are evaluated pre-transplant for assessment of eligibility criteria including chest CT, pulmonary function tests, echocardiogram, bone marrow biopsy, and physical examination as well as standard pre-transplant infectious disease markers.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants
<=18 years
0
   0.0%
Between 18 and 65 years
3
 100.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 3 participants
44
(21 to 51)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants
Female
2
  66.7%
Male
1
  33.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 3 participants
3
1.Primary Outcome
Title Event-free Survival (EFS)
Hide Description The events will be defined as any one of the following: death; respiratory failure; renal failure, as defined by chronic dialysis > or = 6 months or kidney transplantation; occurrence of cardiomyopathy, confirmed by clinical CHF (New York Class III or IV) or LVEF < 30% by echocardiogram, sustained for at least 3 months despite therapy; organ dysfunction specific events must be documented on at least two occasions > or = 3 months apart, or sustained for a 3-month period (documented from the first occurrence).
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
UCB transplant recipients
Arm/Group Title Treatment: Allogeneic HCT After Reduced Intensity Conditioning
Hide Arm/Group Description:

Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid.

fludarabine phosphate: Given IV

Mycophenolic Acid: Given PO

tacrolimus: Given PO

total-body irradiation: Undergo TBI

bone marrow transplantation: Undergo transplantation

reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation

quality-of-life assessment: Ancillary studies

laboratory biomarker analysis: Correlative studies

flow cytometry: Correlative studies

biopsy: Punch biopsy of skin involved with scleroderma

cyclophosphamide: Given IV

Overall Number of Participants Analyzed 3
Measure Type: Count of Participants
Unit of Measure: Participants
1
  33.3%
2.Secondary Outcome
Title EFS
Hide Description event-free survival after umbilical cord blood transplant
Time Frame 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment: Allogeneic UCB After Reduced Intensity Conditioning
Hide Arm/Group Description:

Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive hematopoietic cell transplantation on day 0.

fludarabine phosphate: Given IV

Mycophenolic Acid: Given PO

tacrolimus: Given PO

total-body irradiation: Undergo TBI

bone marrow transplantation: Undergo transplantation

reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation

quality-of-life assessment: Ancillary studies

laboratory biomarker analysis: Correlative studies

flow cytometry: Correlative studies

biopsy: Punch biopsy of skin involved with scleroderma

Overall Number of Participants Analyzed 3
Measure Type: Count of Participants
Unit of Measure: Participants
1
  33.3%
3.Secondary Outcome
Title Overall Survival
Hide Description Event is defined as death due to any cause.
Time Frame Up to 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
survival of patients after UCB
Arm/Group Title Treatment: Allogeneic HCT After Reduced Intensity Conditioning
Hide Arm/Group Description:

Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid.

fludarabine phosphate: Given IV

Mycophenolic Acid: Given PO

tacrolimus: Given PO

total-body irradiation: Undergo TBI

bone marrow transplantation: Undergo transplantation

reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation

quality-of-life assessment: Ancillary studies

laboratory biomarker analysis: Correlative studies

flow cytometry: Correlative studies

biopsy: Punch biopsy of skin involved with scleroderma

cyclophosphamide: Given IV

Overall Number of Participants Analyzed 3
Measure Type: Count of Participants
Unit of Measure: Participants
1
  33.3%
4.Secondary Outcome
Title Treatment-related Mortality
Hide Description Defined as death occurring at any time after start of allogeneic HCT and definitely or probably resulting from treatment given in the study and not associated with disease progression.
Time Frame From time of transplant to 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment: Allogeneic HCT After Reduced Intensity Conditioning
Hide Arm/Group Description:

Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid.

fludarabine phosphate: Given IV

Mycophenolic Acid: Given PO

tacrolimus: Given PO

total-body irradiation: Undergo TBI

bone marrow transplantation: Undergo transplantation

reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation

quality-of-life assessment: Ancillary studies

laboratory biomarker analysis: Correlative studies

flow cytometry: Correlative studies

biopsy: Punch biopsy of skin involved with scleroderma

cyclophosphamide: Given IV

Overall Number of Participants Analyzed 3
Measure Type: Count of Participants
Unit of Measure: Participants
2
  66.7%
5.Secondary Outcome
Title Regimen-related Toxicity (Greater Than or Equal to Grade III) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 3.0
Hide Description

Grades 3, 4 and 5 adverse events will be tracked from the start of mobilization or conditioning until day +100 after transplant or until patient departure from the center, whichever occurs first.

Certain adverse events are usual and expected after transplant and will only be reported if they are > Grade 4. Some Grade 4 events that are routinely expected (i.e. pancytopenia) will not be reported.

Time Frame Up to 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
3 patients received umbilical cord blood (UCB) transplant.
Arm/Group Title Treatment: Allogeneic HCT After Reduced Intensity Conditioning
Hide Arm/Group Description:

Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid.

fludarabine phosphate: Given IV

Mycophenolic Acid: Given PO

tacrolimus: Given PO

total-body irradiation: Undergo TBI

bone marrow transplantation: Undergo transplantation

reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation

cyclophosphamide: Given IV

Overall Number of Participants Analyzed 3
Measure Type: Count of Participants
Unit of Measure: Participants
Renal failure
1
  33.3%
Diffuse alveolar hemorrhage
1
  33.3%
Pneumonitis
1
  33.3%
6.Secondary Outcome
Title The Percent of Participants With Definite and Probable Viral, Fungal, and Bacterial Infections
Hide Description The percent of participants with definite and probable viral, fungal, and bacterial infections after transplant
Time Frame Up to 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment: Allogeneic HCT After Reduced Intensity Conditioning
Hide Arm/Group Description:

Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid.

fludarabine phosphate: Given IV

Mycophenolic Acid: Given PO

tacrolimus: Given PO

total-body irradiation: Undergo TBI

bone marrow transplantation: Undergo transplantation

reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation

quality-of-life assessment: Ancillary studies

laboratory biomarker analysis: Correlative studies

flow cytometry: Correlative studies

biopsy: Punch biopsy of skin involved with scleroderma

cyclophosphamide: Given IV

Overall Number of Participants Analyzed 3
Measure Type: Count of Participants
Unit of Measure: Participants
2
  66.7%
7.Secondary Outcome
Title Quality of Life as Assessed by the Modified Scleroderma Health Assessment Questionnaire (SHAQ)
Hide Description The questionnaire includes measure of quality of life and measure of the scale of skin tightness, activity level and function specifically designed for patients with systemic sclerosis
Time Frame Up to 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
One patient completed the pre-transplant and 5 year post transplant SHAQ (scleroderma health assessment questionnaire). Score range from 0 to 3. Minimum score: Score of 0 is excellent health. Maximum score: score of 3 is most severely impaired, poor quality of life.
Arm/Group Title Treatment: Allogeneic HCT After Reduced Intensity Conditioning
Hide Arm/Group Description:

Conditioning regimen

  • Day -6: cyclophosphamide 50mg/kg. MESNA will be given for bladder prophylaxis .
  • Day -6, -5, -4: Horse ATG 30mg/kg IV x 3 days.
  • Days -6, -5, -4, -3 and –2: Fludarabine 40 mg/m2/day IV over one hour x 5 days.
  • Day -1: TBI 200 cGy at 6-7 cGy/min from a linear accelerator TBI.
  • Day 0: UCB infusion ( 2 units)

Day –3: Commence tacrolimus at 0.03 mg/kg/day continuous IV infusion until the patient is tolerating oral intake then convert to oral PO b.i.d., continue to day +180 and taper to day +365.

Day 0: After UCB transplant on day 0, mycophenolate mofetil will be given 1gm IV t.i.d. until the patient is tolerating oral intake and can convert to 1 gram PO T.I.D. Stop MMF at Day +30, if no acute GVHD, until day +100, and then taper 11% week over 8 weeks.

Overall Number of Participants Analyzed 1
Measure Type: Number
Unit of Measure: units on a scale
pre-transplant SHAQ 1.125
5 year post transplant 0.125
pre-transplant Raynaud symptoms 3
5 year post transplant Raynaud symptoms 0.5
pre-transplant Finger ulcer symptoms 3
5 year post-transplant Finger ulcer symptoms 0
pre-transplant Overall health symptoms 2.5
5 year post-transplant overall health symptoms 0.2
8.Secondary Outcome
Title Quality of Life as Assessed by the Medical Outcome Short Form (36) Health Survey Instrument (SF-36)
Hide Description The Medical Outcome Short Form (36) Health Survey instrument (SF-36) is a general assessment of health quality of life with eight components: physical functioning, role limitations due to physical health, pain index, general health perceptions, vitality, social functioning, role limitations due to emotional problems and Mental Health Index. Each domain is positively scored, indicating that higher scores are associated with positive outcome.
Time Frame Up to 5 years
Hide Outcome Measure Data
Hide Analysis Population Description

Pre-transplant (baseline) evaluation of physical functioning. Score from 0 to100 with 0 perfect health and 100 poor health.

The pre-transplant (baseline) evaluation was completed by study participant. The participant did not complete the 5 year post transplant evaluation.

The 5 year post-transplant SF32 form was not completed by the patient.

Arm/Group Title Treatment: Allogeneic HCT After Reduced Intensity Conditioning
Hide Arm/Group Description:

Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid.

fludarabine phosphate: Given IV

Mycophenolic Acid: Given PO

tacrolimus: Given PO

total-body irradiation: Undergo TBI

bone marrow transplantation: Undergo transplantation

reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation

quality-of-life assessment: Ancillary studies

laboratory biomarker analysis: Correlative studies

flow cytometry: Correlative studies

biopsy: Punch biopsy of skin involved with scleroderma

cyclophosphamide: Given IV

Overall Number of Participants Analyzed 1
Measure Type: Number
Unit of Measure: units on a scale
SF-36 pretransplant overall score 49.3
pretransplant limitations due to physical health 50
pretransplant limitations due to emotional health 50
9.Secondary Outcome
Title Skin Score
Hide Description The skin score measure is a scale: the name of the scale is the modified Rodnan skin score (mRSS). Total score of mRSS is from 0 to 51. Higher values represents worse skin score. Highest value is 51, represents very hidebound tight thick skin. Lowest value is 0, represent normal skin, no tightness.
Time Frame Up to 5 years post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
One patient was evaluated at baseline and at 5 years post-transplant. Skin score was evaluated using modified Rodnan skin score (mRSS).
Arm/Group Title Treatment: Allogeneic HCT After Reduced Intensity Conditioning
Hide Arm/Group Description:

Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid.

fludarabine phosphate: Given IV

Mycophenolic Acid: Given PO

tacrolimus: Given PO

total-body irradiation: Undergo TBI

bone marrow transplantation: Undergo transplantation

reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation

quality-of-life assessment: Ancillary studies

laboratory biomarker analysis: Correlative studies

flow cytometry: Correlative studies

biopsy: Punch biopsy of skin involved with scleroderma

cyclophosphamide: Given IV

Overall Number of Participants Analyzed 1
Measure Type: Number
Unit of Measure: units on a scale (mRSS)
Pre-transplant (baseline) skin score 17
5 year post-transplant skin score 4
10.Secondary Outcome
Title Incidence of Graft Rejection
Hide Description Engraftment is defined as achieving > 5% donor peripheral blood T cell chimerism by Day 56 after HCT. Primary graft failure is defined as a donor peripheral blood T cell chimerism peak of < 5% by Day 56 post-HCT. Methodological requirements for chimerism are as defined by institutional standard of practice. Secondary Graft Failure is defined as documented engraftment followed by loss of the graft with donor peripheral blood T cell chimerism < 5% as demonstrated by a chimerism assay
Time Frame Up to day +56
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment: Allogeneic HCT After Reduced Intensity Conditioning
Hide Arm/Group Description:

Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid.

fludarabine phosphate: Given IV

Mycophenolic Acid: Given PO

tacrolimus: Given PO

total-body irradiation: Undergo TBI

bone marrow transplantation: Undergo transplantation

reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation

quality-of-life assessment: Ancillary studies

laboratory biomarker analysis: Correlative studies

flow cytometry: Correlative studies

biopsy: Punch biopsy of skin involved with scleroderma

cyclophosphamide: Given IV

Overall Number of Participants Analyzed 3
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
11.Secondary Outcome
Title Incidence and Severity of Graft-versus-host Disease (GVHD)
Hide Description The grading of acute and chronic GVHD will follow previously published guidelines and according to institutional standard of practice but will also include capture of symptoms and characterization of alternative causes. The highest level of organ abnormalities, the etiologies contributing to the abnormalities and biopsy results pertaining to GVHD will be identified. Since both GVHD and SSc involve the skin and the gastrointestinal tract, all diagnostic biopsies of these organs will be centrally reviewed by a study pathologist.
Time Frame Up to 5 years post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description

1 patient out of the 3 patients enrolled was evaluable for both acute and chronic GVHD. The grade of acute GVHD ranges from 0 to 4. Minimum score of 0 is normal or no GVHD. Maximum score of 4 is fatal GVHD.

Chronic GVHD minimum score is 0 (none), maximum score 3: extensive and severe.

Arm/Group Title Treatment: Allogeneic HCT After Reduced Intensity Conditioning
Hide Arm/Group Description:

Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid.

fludarabine phosphate: Given IV

Mycophenolic Acid: Given PO

tacrolimus: Given PO

total-body irradiation: Undergo TBI

bone marrow transplantation: Undergo transplantation

reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation

quality-of-life assessment: Ancillary studies

laboratory biomarker analysis: Correlative studies

flow cytometry: Correlative studies

biopsy: Punch biopsy of skin involved with scleroderma

cyclophosphamide: Given IV

Overall Number of Participants Analyzed 1
Measure Type: Number
Unit of Measure: units on a scale
acute GVHD severity maximum grade 2
chronic GVHD maximum grade 2
12.Secondary Outcome
Title Incidence of Disease-modifying Antirheumatic Drugs (DMARDs) Initiated Post Transplant to Modify Disease
Hide Description Percent of patients treated with DMARDS after allogeneic transplant in order to treat scleroderma disease signs and symptoms.
Time Frame Up to 5 years post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
3 patients enrolled were evaluable.
Arm/Group Title Treatment: Allogeneic HCT After Reduced Intensity Conditioning
Hide Arm/Group Description:

Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid.

fludarabine phosphate: Given IV

Mycophenolic Acid: Given PO

tacrolimus: Given PO

total-body irradiation: Undergo TBI

bone marrow transplantation: Undergo transplantation

reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation

quality-of-life assessment: Ancillary studies

laboratory biomarker analysis: Correlative studies

flow cytometry: Correlative studies

biopsy: Punch biopsy of skin involved with scleroderma

cyclophosphamide: Given IV

Overall Number of Participants Analyzed 3
Measure Type: Count of Participants
Unit of Measure: Participants
participants requiring DMARDs
0
   0.0%
participants evaluable
3
 100.0%
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Treatment: Allogeneic HCT After Reduced Intensity Conditioning
Hide Arm/Group Description

Patients receive fludarabine phosphate IV on days -4, -3 and -2, cyclophosphamide IV over 1-2 hours on days -6, -5, 3, and 4, and undergo low-dose TBI on day -1. Patients receive bone marrow transplantation on day 0. Patients then receive cyclophosphamide IV on days +3 and +4, and beginning day +5 they start tacrolimus orally (PO) and mycophenolic acid.

fludarabine phosphate: Given IV

Mycophenolic Acid: Given PO

tacrolimus: Given PO

total-body irradiation: Undergo TBI

bone marrow transplantation: Undergo transplantation

reduced intensity allogeneic hematopoietic stem cell transplantation: Undergo transplantation

quality-of-life assessment: Ancillary studies

laboratory biomarker analysis: Correlative studies

flow cytometry: Correlative studies

biopsy: Punch biopsy of skin involved with scleroderma

cyclophosphamide: Given IV

All-Cause Mortality
Treatment: Allogeneic HCT After Reduced Intensity Conditioning
Affected / at Risk (%)
Total   2/3 (66.67%)    
Hide Serious Adverse Events
Treatment: Allogeneic HCT After Reduced Intensity Conditioning
Affected / at Risk (%) # Events
Total   2/3 (66.67%)    
Renal and urinary disorders   
Renal failure   1/3 (33.33%)  1
Respiratory, thoracic and mediastinal disorders   
Diffuse Alveolar Hemorrhage   1/3 (33.33%)  1
Pneumonitis  [1]  1/3 (33.33%)  1
Indicates events were collected by systematic assessment
[1]
Hypoxia
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Treatment: Allogeneic HCT After Reduced Intensity Conditioning
Affected / at Risk (%) # Events
Total   1/3 (33.33%)    
Skin and subcutaneous tissue disorders   
GVHD  [1]  1/3 (33.33%)  3
Indicates events were collected by systematic assessment
[1]
Skin graft versus host disease
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
Layout table for Results Point of Contact information
Name/Title: George E. Georges MD
Organization: Fred Hutchinson Cancer Research Center
Phone: 206-667-6886
EMail: ggeorges@fredhutch.org
Layout table for additonal information
Responsible Party: George Georges, Fred Hutchinson Cancer Research Center
ClinicalTrials.gov Identifier: NCT00622895    
Other Study ID Numbers: 2067.00
NCI-2011-01352 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
R01AI041721 ( U.S. NIH Grant/Contract )
First Submitted: February 22, 2008
First Posted: February 25, 2008
Results First Submitted: August 16, 2017
Results First Posted: June 4, 2018
Last Update Posted: June 4, 2018