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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

No Placebo-Only Group

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 below

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I have systemic sclerosis with skin involvement, not in Group 5, at risk of getting worse.

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My condition did not improve after 4+ months on MMF/Myfortic or cyclophosphamide.

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My gastrointestinal condition is getting worse.

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I have scleroderma affecting my skin and lungs, with reduced lung function.

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I have diffuse scleroderma, diagnosed within the last 2 years, with a severe skin condition.

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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:

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I have lung, heart, liver, or kidney problems that could affect my treatment.

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I cannot or will not stop taking certain medications for my scleroderma.

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I have had a stem cell transplant using my own cells.

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I haven't had cancer, except for certain skin cancers or early-stage cancers, in the last 2 years.

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I do not have any severe infections that cannot be controlled.

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I have an active or significant case of 'watermelon stomach'.

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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

This trial's timeline: 3 weeks for screening, Varies for treatment, and year 5 for reporting.

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 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial 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