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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 12 December 2020
Main ID:  NCT01413100
Date of registration: 08/08/2011
Prospective Registration: Yes
Primary sponsor: Fred Hutchinson Cancer Research Center
Public title: Scleroderma Treatment With Autologous Transplant (STAT) Study STAT
Scientific title: A Phase II Multi-center Study of High-Dose Cyclophosphamide and Antithymocyte Globulin Followed by Autologous Hematopoietic Cell Transplantation With Post Transplant Maintenance for the Treatment of Systemic Sclerosis
Date of first enrolment: September 15, 2011
Target sample size: 20
Recruitment status: Active, not recruiting
URL:  https://clinicaltrials.gov/show/NCT01413100
Study type:  Interventional
Study design:  Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 2
Countries of recruitment
Canada United States
Contacts
Name:     George Georges
Address: 
Telephone:
Email:
Affiliation:  Fred Hutch/University of Washington Cancer Consortium
Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients with SSc as defined by the American College of Rheumatology with diffuse
cutaneous disease (except Group 5) at risk of disease progression

- Patients must have failed a prior >= 4-mponth course of either MMF/Myfortic or
cyclophosphamide before being eligible for the study (determined at >= 1 week before
start of mobilization); "failure" is defined as evidence of disease progression or
absence of improvement; the response prior to MMF of cyclophosphamide will be assessed
by the participating site study rheumatologist

- Patients must meet eligibility in at least 1 of the following 6 groups:

- GROUP 1:

- Patients must have 1) both a and b below; and 2) either c, or d

- a) Diffuse cutaneous scleroderma as defined by skin thickening proximal to
the elbows and knees and/or involving the torso in addition to distal
extremity involvement; a skin score will be obtained but not used to
determine eligibility

- b) Duration of systemic sclerosis =< 7 years from the onset of first
non-Raynaud's symptom; for those patients with disease activity between 5-7
years from the onset of first non-Raynaud's symptom, recent progression or
activity of disease must be documented

- c) Presence of SSc-related pulmonary disease with forced vital capacity
(FVC) < 80% or hemoglobin-adjusted diffusing capacity for carbon monoxide
(DLCO) < 70% of predicted AND evidence of alveolitis or SSc-related
interstitial lung disease by high-resolution chest computed tomography (CT)
scan and/or by bronchoalveolar lavage (BAL) (interstitial lung disease may
be nonspecific interstitial pneumonia [NSIP] or usual interstitial pneumonia
[UIP]; a bronchoalveolar lavage [BAL] should be done to confirm the findings
of alveolitis only if the high resolution CT scan [HRCT] fails to show
findings typically associated with systemic sclerosis changes [ground glass
NSIP, UIP, SSc related interstitial lung disease]); alveolitis by BAL cell
count will be defined based on a BAL cell differential count (> 3%
neutrophils and/or > 2% eosinophils) from any lavaged lobe

- d) History of SSc-related renal disease that may not be active at the time
of screening; stable serum creatinine must be documented for a minimum of 3
months post-renal crisis at the time of the baseline visit; history of
scleroderma hypertensive renal crisis is included in this criterion and is
defined as follows:

- History of new-onset hypertension based on any of the following
(measurements must be repeated and confirmed at least 2 hours apart
within 3 days of first event-associated observation, with a change from
baseline):

- Systolic blood pressure (SBP) >= 140 mmHg

- Diastolic blood pressure (DBP) >= 90 mmHg

- Rise in SBP >= 30 mmHg compared to baseline

- Rise in DBP >= 20 mmHg compared to baseline

- AND one of the following 5 laboratory criteria:

- Increase of >= 50 % above baseline in serum creatinine

- Proteinuria: >= 2+ by dipstick confirmed by
protein:creatinine ratio > 2.5

- Hematuria: >= 2+ by dipstick or > 10 red blood cell
(RBC)s/hematopoietic-promoting factor (HPF) (without
menstruation)

- Thrombocytopenia: < 100,000 platelets/mm^3

- Hemolysis: by blood smear or increased reticulocyte count

- The above definition of SSc hypertensive renal crisis is independent of
whether concomitant anti-hypertensive medications are used

- Subjects who present with solely skin and renal disease in the absence
of other organ involvement, except classic SSc renal crisis as
described above and including non-hypertensive renal crisis, must see a
nephrologist to confirm that their renal disease is secondary to only
SSc

- Note: Subjects may be re-screened if they fail to meet inclusion
criteria on initial evaluation

- GROUP 2:

- Progressive pulmonary disease as defined by a decrease in the FVC or
DLCO-adjusted by 10 or 15 percent or greater, respectively, from a prior FVC or
DLCO-adjusted in the previous 18-month period

- Patients will have diffuse cutaneous disease and may have both FVC and DLCOcorr
>= 70% at screening for the study

- Patients must also have evidence of alveolitis as defined by abnormal chest
computed tomography (CT) or bronchoalveolar lavage (BAL)

- GROUP 3: Diffuse scleroderma with disease duration =< 2 years since development of
first sign of skin thickening plus modified Rodnan skin score >= 25 plus either

- Erythrocyte sedimentation rate (ESR) > 25 mm/1st hour and/or hemoglobin (Hb) < 11
g/dL, not explained by causes other than active scleroderma

- Lung involvement (either FVC or DLCO < 80% and evidence of interstitial lung
disease by CT scan or alveolitis by BAL)

- GROUP 4: Diffuse scleroderma with disease duration =< 2 years and skin score >= 30

- GROUP 5:

- Limited cutaneous scleroderma and SSc-related pulmonary disease with FVC < 80% or
hemoglobin-adjusted DLCO < 70% of predicted

- AND evidence of alveolitis/interstitial lung disease by high-resolution chest CT
scan and/or by BAL (interstitial lung disease may be nonspecific interstitial
pneumonia [NSIP] or usual interstitial pneumonia [UIP]; A bronchoalveolar lavage
[BAL] should be done to confirm the findings of alveolitis only if the high
resolution CT scan [HRCT] fails to show findings typically associated with
systemic sclerosis changes [ground glass, NSIP, UIP, SSc related interstitial
lung disease])

- Alveolitis by BAL cell count will be defined based on a BAL cell differential
count (> 3% neutrophils and/or > 2% eosinophils) from any lavaged lobe

- GROUP 6: Progressive gastrointestinal disease as defin



Age minimum: N/A
Age maximum: 70 Years
Gender: All
Health Condition(s) or Problem(s) studied
Systemic Scleroderma
Intervention(s)
Biological: Anti-Thymocyte Globulin
Biological: Filgrastim
Drug: Cyclophosphamide
Drug: Mycophenolate Mofetil
Drug: Plerixafor
Other: Laboratory Biomarker Analysis
Other: Quality-of-Life Assessment
Other: Questionnaire Administration
Procedure: Autologous Hematopoietic Stem Cell Transplantation
Procedure: Peripheral Blood Stem Cell Transplantation
Primary Outcome(s)
EFS of patients undergoing chemotherapy and transplant [Time Frame: At 5 years]
Secondary Outcome(s)
All-cause mortality [Time Frame: Assessed up to 5 years]
Change in cardiac function [Time Frame: Year 1]
Change in cardiac function [Time Frame: Year 2]
Change in cardiac function [Time Frame: Year 3]
Change in cardiac function [Time Frame: Year 4]
Change in cardiac function [Time Frame: Year 5]
Change in renal function over time [Time Frame: Annually for 5 years]
Change in renal function over time [Time Frame: Baseline]
Change in renal function over time [Time Frame: Week 12]
Change in renal function over time [Time Frame: Week 26]
Disease progression [Time Frame: 6 months and then annually for 5 years]
Health care utilization as assessed by UCSD Healthcare Utilization surveys [Time Frame: Assessed up to 5 years]
HRQOL as measured by the PROMIS-29 version 1.0 [Time Frame: Annually for 5 years]
HRQOL as measured by the SF-36 [Time Frame: Annually for 5 years]
HRQOL as measured by the SGRQ [Time Frame: Annually for 5 years]
HRQOL as measured by the SHAQ [Time Frame: Annually for 5 years]
Improvement in pulmonary function [Time Frame: 1 month]
Improvement in pulmonary function [Time Frame: Annually for 5 years]
Improvement in pulmonary function [Time Frame: Baseline]
Improvement in pulmonary function [Time Frame: Week 12]
Improvement in pulmonary function [Time Frame: Week 26]
Infectious complications [Time Frame: Assessed up to 5 years]
Non-progression mortality [Time Frame: Assessed up to 5 years]
Overall survival [Time Frame: Assessed up to 5 years]
Regimen-related toxicities [Time Frame: Up to 1 year post-transplant]
Time of initiation of putative disease-modifying antirheumatic drugs (DMARDS) to modify disease [Time Frame: After transplant, up to 5 years]
Time to treatment failure [Time Frame: The time interval between transplant (day 0) and the initial visit at which death or the qualifying event first occurs, assessed up to 5 years]
Treatment-related mortality [Time Frame: Day 90]
Work productivity Survey (WPS) [Time Frame: Assessed up to 5 years]
Secondary ID(s)
2533
2533.00
NCI-2011-01190
P30CA015704
RG1711052
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
National Cancer Institute (NCI)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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