Hematopoietic Stem Cell Support in Patients With Refractory Sarcoidosis

Stopped early · Phase 1/Phase 2

Conditions studied: Sarcoidosis

In brief

Sarcoidosis is a disease believed to be due to immune cells, cells which normally protect the body, but are now attacking lungs, heart, nerves, or other organs or systems within the body. As a result, the affected organs or systems fail to work properly causing difficulty breathing; heart failure; inability of the nerves to respond properly causing numbing, tingling, pain, and progressive muscle weakness; or other symptoms depending on the organ or body system involved. The likelihood of progression of this disease is high. This study is designed to examine whether treating patients with high dose cyclophosphamide (a drug which reduces the function of the immune system) and ATG (a protein that kills the immune cells that are thought to be causing this disease), followed by return of the previously collected blood stem cells will stop the progression of sarcoidosis. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the high dose cyclophosphamide and ATG is to destroy the cells in the immune system. The purpose of the stem cell infusion is to evaluate whether this treatment will produce a normal immune system that will no longer attack the body.

Key facts

Study ID

NCT00282438

Run by

Northwestern University

People needed

2

Starts

2003-12-01

Expected to finish

2015-08-01

Last updated by the study team

2018-08-31

Who can join

Age: 18 and older, up to 60. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age ≥ 18years and ≤ 60 years at the time of pretransplant evaluation.
• Definitive diagnosis of sarcoidosis in pathologic specimen.
• Patients who failed to respond to conventional treatment of at least 3 months duration with corticosteroids (equivalent dosage of prednisone 1.0mg/kg/day to start). Patients must also have failed two or more of the followings: TNF inhibitors (etanercept, infliximab), methotrexate, azathioprine, 6-MP, cyclosporin, tacrolimus, mycophenolate mofetil, gold, dapsone, colchicine, chloroquine/hydroxychloroquine or any other immunosuppressive or modulating drugs.
• Failure of therapy defined by (not caused by unrelated conditions) any one of following:
• Progressive pulmonary disease (stage II or III) defined by decline in pulmonary function (DLCo, VC or FEV1) of 15% or more over 12 months.
• Progressive CNS disease (worsening symptoms such as paraparesis or medically refractory seizure).
• Persistent peripheral neuropathy (one of following):
• Persistent muscle weakness Grade 3/5 or worse (MRC) in at least one movement (e.g. ankle dorsiflexion) in two limbs.
• Persistent cranial nerve involvement such as persistent facial diplegia.
• Persistent incapacitating sensory loss (e.g. gait ataxia, falls > 1/month).
• Progressive loss of vision.
• Persistent hypercalcemia.
• Cardiac sarcoidosis that is proven by cardiac biopsy or cardiac MRI.

You may not qualify if…

• Alternative diagnosis.
• Noncompliance to medical care.
• > 10 pack-year history of cigarette smoking if lung disease is the major problem.
• Poor performance (PS) status (ECOG >2) at the time of entry, unless decline of PS is due to the disease itself.
• Significant end organ damage such as:
• Overt congestive heart failure (NYHA Class III or IV).
• Active ischemic heart disease, s/p myocardial infarction within 6 months, s/p unstable angina within 3 months, s/p CVA within 6 months, s/p hospitalization for CHF within 3 months.
• Untreated life-threatening arrhythmia.
• Pulmonary hypertension > 40 mmHg.
• End-stage lung disease (TLC < 55%, FVC < 55%, or DLCO < 40% of predicted value).
• Serum creatinine > 2.5 or creatinine clearance < 30 ml/min.
• Liver cirrhosis, transaminases > 3x normal or bilirubin > 2.0 unless due to Gilbert's disease.
• HIV positive.
• Uncontrolled diabetes mellitus, or any other illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive treatment.
• Prior history of malignancy except localized basal cell or squamous skin cancer. Other malignancies for which the patient is judged to be cured by local surgical therapy, such as (but not limited to) head and neck cancer, or stage I or II breast cancer will be considered on an individual basis.
• Positive pregnancy test, inability or unable to pursue effective means of birth control, failure to willingly accept or comprehend irreversible sterility as a side effect of therapy.
• Significant psychological issues, social issues, psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible.
• Inability to give informed consent.
• Major hematological abnormalities such as platelet count less than 100,000/ul, ANC less than 1000/ul.

Where it is running

• Northwestern University, Feinberg School of Medicine — Chicago, Illinois, United States

Full record on ClinicalTrials.gov

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