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Immunosuppressant
Stem Cell Transplant for Scleroderma (STAT Trial)
Phase 2
Waitlist Available
Led By Leona Holmberg
Research Sponsored by Fred Hutchinson Cancer Research Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
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-month 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
Must not have
Subjects with pulmonary, cardiac, hepatic, or renal impairment that would limit their ability to receive cytoreductive therapy and compromise their survival
Unwilling or unable to discontinue disallowed disease-modifying antirheumatic drugs (DMARDs) for treatment of SSc prior to mobilization
Timeline
Screening 3 weeks
Treatment Varies
Follow Up year 5
Awards & highlights
Summary
This trial tests a treatment for systemic scleroderma involving stem cell collection, high-dose chemotherapy, and immune suppression, followed by returning the patient's own stem cells and using a maintenance drug to prevent disease recurrence. The treatment has shown a satisfactory risk-benefit ratio in earlier studies.
Who is the study for?
This trial is for patients with systemic scleroderma, specifically those who have had the disease for less than two years with severe skin involvement or lung problems, and those not responding to standard treatments. It's not open to individuals with a life expectancy under five years, prior stem cell transplants, uncontrolled infections, recent cancers (except certain skin cancers), GAVE ('watermelon stomach'), smokers in the last three months, pregnant women, or those unable to consent.
What is being tested?
The STAT study tests whether a combination of high-dose cyclophosphamide and anti-thymocyte globulin followed by autologous hematopoietic stem cell transplantation can treat systemic scleroderma. After transplanting the patient's own stored stem cells back into their body post-chemotherapy and immunosuppression therapy, maintenance therapy with mycophenolate mofetil is used to prevent worsening of SSc.
What are the potential side effects?
Potential side effects include immune system suppression leading to increased infection risk; reactions from infusions; organ inflammation due to high-dose chemotherapy; blood count abnormalities; fatigue; digestive issues from medications like mycophenolate mofetil; and complications related to bone marrow suppression.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have systemic sclerosis with skin involvement, not in Group 5, at risk of getting worse.
Select...
My condition did not improve after 4+ months on MMF/Myfortic or cyclophosphamide.
Select...
My gastrointestinal condition is getting worse.
Select...
I have scleroderma affecting my skin and lungs, with reduced lung function.
Select...
I have diffuse scleroderma, diagnosed within the last 2 years, with a severe skin condition.
Select...
I have diffuse scleroderma, diagnosed within the last 2 years, with severe skin thickening.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I have lung, heart, liver, or kidney problems that could affect my treatment.
Select...
I cannot or will not stop taking certain medications for my scleroderma.
Select...
I have had a stem cell transplant using my own cells.
Select...
I haven't had cancer, except for certain skin cancers or early-stage cancers, in the last 2 years.
Select...
I do not have any severe infections that cannot be controlled.
Select...
I have an active or significant case of 'watermelon stomach'.
Select...
I am unable to understand and give consent for treatment.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ year 5
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~year 5
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
EFS of patients undergoing chemotherapy and transplant
Secondary study objectives
Perinatal death
Change in cardiac function
Change in renal function over time
+6 moreTrial Design
1Treatment groups
Experimental Treatment
Group I: Treatment (HDIT autologous PBSCT)Experimental Treatment10 Interventions
STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7.
HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5.
TRANSPLANTATION: Patients undergo autologous PBSCT on day 0.
MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Plerixafor
2011
Completed Phase 3
~710
Peripheral Blood Stem Cell Transplantation
1997
Completed Phase 3
~1330
Cyclophosphamide
2010
Completed Phase 4
~2320
Mycophenolate Mofetil
1997
Completed Phase 4
~2380
Autologous Hematopoietic Stem Cell Transplantation
2017
Completed Phase 3
~2090
Filgrastim
2000
Completed Phase 3
~3690
Anti-Thymocyte Globulin
2009
Completed Phase 4
~1040
Find a Location
Who is running the clinical trial?
Fred Hutchinson Cancer Research CenterLead Sponsor
443 Previous Clinical Trials
148,122 Total Patients Enrolled
Fred Hutchinson Cancer CenterLead Sponsor
566 Previous Clinical Trials
1,342,481 Total Patients Enrolled
National Cancer Institute (NCI)NIH
13,868 Previous Clinical Trials
41,010,816 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have lung, heart, liver, or kidney problems that could affect my treatment.I cannot or will not stop taking certain medications for my scleroderma.I have had a stem cell transplant using my own cells.I have systemic sclerosis with skin involvement, not in Group 5, at risk of getting worse.My condition did not improve after 4+ months on MMF/Myfortic or cyclophosphamide.My gastrointestinal condition is getting worse.I have scleroderma affecting my skin and lungs, with reduced lung function.I haven't had cancer, except for certain skin cancers or early-stage cancers, in the last 2 years.I have other serious health conditions with a life expectancy of less than 5 years.I do not have any severe infections that cannot be controlled.I have an active or significant case of 'watermelon stomach'.I have diffuse scleroderma, diagnosed within the last 2 years, with a severe skin condition.I am unable to understand and give consent for treatment.You have smoked tobacco or related products in the last 3 months.My lung function has worsened in the last 18 months.I have diffuse scleroderma, diagnosed within the last 2 years, with severe skin thickening.I have an autoimmune disease that doesn't require strong immune-suppressing drugs.I have been tested for MDS due to my cancer treatment history.
Research Study Groups:
This trial has the following groups:- Group 1: Treatment (HDIT autologous PBSCT)
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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