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Cyclophosphamide and rATG With Hematopoietic Stem Cell Support in Systemic Scleroderma

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ClinicalTrials.gov Identifier: NCT00278525
Recruitment Status : Completed
First Posted : January 18, 2006
Results First Posted : April 30, 2014
Last Update Posted : April 30, 2014
Sponsor:
Information provided by (Responsible Party):
Richard Burt, MD, Northwestern University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition SYSTEMIC SCLERODERMA
Interventions Drug: standard of care
Procedure: stem cell transplantation
Enrollment 19
Recruitment Details

Eligible (n=19) Randomized (n=19)

  1. Allocated to Hematopoietic Stem Cell Transplantation (HSCT (n=10) + crossed over to HSCT(n=7)

    • Received HSCT (n=17); Lost to follow-up (n=0); Analysed (n=17)

  2. Allocated to cyclophosphamide (n=9) • Received allocated intervention (n=9) Lost to follow-up (n=0); Crossed over (n=7); Analysed (n=9)
Pre-assignment Details  
Arm/Group Title Standard of Care Stem Cell Trasplantation
Hide Arm/Group Description Cyclophosphamide will be given as approved immunosuppressive therapy intervention as stem cell transplantation after conditioning regimen
Period Title: Overall Study
Started 9 10
Completed 9 [1] 10 [2]
Not Completed 0 0
[1]
7 participants crossed to HSCT arm in accordance with the protocol
[2]
7 participant crossed over to the HSCT arm and underwent HSCT, in accordance with the protocol.
Arm/Group Title Stem Cell Trasplantation Standard of Care Total
Hide Arm/Group Description intervention as stem cell transplantation after conditioning regimen medication as standard of care will be given Total of all reporting groups
Overall Number of Baseline Participants 10 9 19
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 9 participants 19 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
10
 100.0%
9
 100.0%
19
 100.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 10 participants 9 participants 19 participants
45  (13) 44  (10) 44  (10)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 9 participants 19 participants
Female
10
 100.0%
8
  88.9%
18
  94.7%
Male
0
   0.0%
1
  11.1%
1
   5.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 10 participants 9 participants 19 participants
10 9 19
1.Primary Outcome
Title Time to Treatment Failure
Hide Description

-Data are reporting number of participants that were classified as treatment failures

Time to Treatment Failure Definition-Treatment failure will not occur until a minimum of 12 months after enrollment at which time failure is defined as:

  1. Failure of skin score (if > 14 on enrollment) to improve or increase in skin score by a 25% above lowest post treatment value and must be documented on 2 occasion 6 months apart
  2. Deterioration in diffusing capacity of the lung for carbon monoxide (DLCO), diffusing capacity divided by the alveolar volume (DLCO/VA) or forced vital capacity (FVC) by 10% below enrollment level or 10% below best post treatment value, due to systemic sclerosis, and documented on 2 occasion 6 months apart
  3. Renal failure due to systemic sclerosis and defined as chronic dialysis for more than 12 months
  4. Gastrointestinal failure due to systemic sclerosis and defined as initiation of total parenteral nutrition(TPN) for more than 12 months
Time Frame 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All participants were included
Arm/Group Title Stem Cell Trasplantation Standard of Care
Hide Arm/Group Description:
intervention as stem cell transplantation after conditioning regimen
Intravenous (IV) will be given 1000 mg/m2 cyclophosphamide monthly for 6 months.
Overall Number of Participants Analyzed 10 9
Measure Type: Number
Unit of Measure: participants
0 8
2.Primary Outcome
Title Disease Improvement
Hide Description

Data are reporting number of participants that were classified as disease improvement.

Definition of disease improvement:

Disease improvement defined by at least 25% improvement in skin score (Rodnan), or 10% improvement in pulmonary function tests [diffusing capacity of the lung for carbon monoxide (DLCO), diffusing capacity divided by the alveolar volume (DLCO/VA), or forced vital capacity (FVC)], or in cardiac tests [pulmonary artery (PA) systolic pressure by right heart cath] that persists > 6 months or ability to wean off total parenteral nutrition (TPN)

Time Frame 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Stem Cell Trasplantation Standard of Care
Hide Arm/Group Description:
intervention as stem cell transplantation after conditioning regimen
Intravenous (IV) will be given 1000 mg/m2 cyclophosphamide monthly for 6 months.
Overall Number of Participants Analyzed 10 9
Measure Type: Number
Unit of Measure: participants
10 0
Time Frame 100 days after the transplant
Adverse Event Reporting Description Common Toxicity Criteria Scale used to grade all non-hematologic toxicities
 
Arm/Group Title Stem Cell Trasplantation Standard of Care
Hide Arm/Group Description intervention as stem cell transplantation after conditioning regimen medication as standard of care will be given
All-Cause Mortality
Stem Cell Trasplantation Standard of Care
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Stem Cell Trasplantation Standard of Care
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/17 (0.00%)      0/9 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Stem Cell Trasplantation Standard of Care
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   7/17 (41.18%)      3/9 (33.33%)    
Cardiac disorders     
Supraventricular tachycardia  1 [1]  1/17 (5.88%)  1 0/9 (0.00%)  0
Atrial fibrillation  1 [2]  1/17 (5.88%)  1 0/9 (0.00%)  0
Volume overload * 1 [3]  2/17 (11.76%)  2 0/9 (0.00%)  0
Gastrointestinal disorders     
Gastroenteritis  1 [4]  0/17 (0.00%)  0 2/9 (22.22%)  2
Infections and infestations     
Positive stool culture for Clostridium difficile  1 [5]  1/17 (5.88%)  1 0/9 (0.00%)  0
Positive blood culture for micrococcus  1 [6]  1/17 (5.88%)  1 0/9 (0.00%)  0
Cytomegalovirus reactivation on surveillance blood test  1 [7]  1/17 (5.88%)  1 0/9 (0.00%)  0
Cellulitis * 1 [8]  0/17 (0.00%)  0 1/9 (11.11%)  1
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, Other
[1]
During hospitalisation for transplantation, one patient developed Supraventricular tachycardia that was controlled with oral medication
[2]
During hospitalisation for transplantation, 1 patient developed Atrial fibrillation that was controlled with oral medication
[3]
During the hospitalization for transplantation, two patients developed volume overload that were controlled with diuretics
[4]
Two patients from the control group needed protracted outpatient treatment for gastroenteritis with nausea, vomiting and diarrhoea
[5]
During hospitalisation for transplantation, 1 patient had a positive stool culture for Clostridium difficile
[6]
During the hospitalisation for transplantation, 1 patient had a positive blood culture for micrococcus
[7]
The only one infectious event after hospitalisation for transplantation was a cytomegalovirus reactivation on surveillance blood test in one patent, which was treated with oral valganciclovir.
[8]
One patient in the control group developed cellulitis that was treated with oral antibiotics.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr. Richard Burt
Organization: Northwestern University
Phone: 312-908-0059
Responsible Party: Richard Burt, MD, Northwestern University
ClinicalTrials.gov Identifier: NCT00278525     History of Changes
Other Study ID Numbers: DI Scl.Randomized2004
First Submitted: January 15, 2006
First Posted: January 18, 2006
Results First Submitted: April 9, 2013
Results First Posted: April 30, 2014
Last Update Posted: April 30, 2014