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Hematopoietic Stem Cell Support in Patients With Refractory Sarcoidosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00282438
Recruitment Status : Terminated (No plan to continue enrollment)
First Posted : January 26, 2006
Results First Posted : February 24, 2017
Last Update Posted : August 31, 2018
Information provided by (Responsible Party):
Richard Burt, MD, Northwestern University

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Sarcoidosis Biological: Autologous hematopoietic stem cell transplantation Biological: Allogeneic stem cell transplantation Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Hematopoietic Stem Cell Transplant in Patients With Refractory Sarcoidosis: A Phase I/II Trial
Study Start Date : December 2003
Actual Primary Completion Date : August 2015
Actual Study Completion Date : August 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Sarcoidosis

Arm Intervention/treatment
Experimental: Autologous hematopoietic stem cell transplantation
Autologous stem cells will be injected after conditioning
Biological: Autologous hematopoietic stem cell transplantation
Autologous hematopoietic stem cells will be injected after conditioning
Other Name: Autologous stem cell injection

Experimental: Allogeneic stem cell transplantation
Allogeneic stem cells will be injected after conditioning
Biological: Allogeneic stem cell transplantation
Allogeneic stem cells will be injected after conditioning

Primary Outcome Measures :
  1. Presence of Toxicity [ Time Frame: For length of hospital stay (until discharge). ]
    Daily assessment will be made with regards to toxicity by one of the protocol investigators.National Cancer Institute Common Toxicity Criteria will be used to grade all non-hematologic toxicities. Toxicity grades as follows: 1 = Mild; 2 = Moderate; 3 = Severe and undesirable; 4 = life threatening or disabling ; 5 = Death

  2. Survival [ Time Frame: Participants are to be followed at 6 months and then yearly until 5 years ]
    Patient has not died.

  3. Time to Disease Progression [ Time Frame: Participants are to be followed at 6 months and then yearly until 5 years ]
    Worsening symptoms, pulmonary function studies, cardiac function and arrhythmia including EKG assessments, and neurological symptoms.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Age ≥ 18years and ≤ 60 years at the time of pretransplant evaluation.
  2. Definitive diagnosis of sarcoidosis in pathologic specimen.
  3. 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.
  4. 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):

      1. Persistent muscle weakness Grade 3/5 or worse (MRC) in at least one movement (e.g. ankle dorsiflexion) in two limbs.
      2. Persistent cranial nerve involvement such as persistent facial diplegia.
      3. Persistent incapacitating sensory loss (e.g. gait ataxia, falls > 1/month).
    • Progressive loss of vision.
    • Persistent hypercalcemia.
  5. Cardiac sarcoidosis that is proven by cardiac biopsy or cardiac MRI.

Exclusion Criteria:

  1. Alternative diagnosis.
  2. Noncompliance to medical care.
  3. > 10 pack-year history of cigarette smoking if lung disease is the major problem.
  4. Poor performance (PS) status (ECOG >2) at the time of entry, unless decline of PS is due to the disease itself.
  5. Significant end organ damage such as:

    1. Overt congestive heart failure (NYHA Class III or IV).
    2. 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.
    3. Untreated life-threatening arrhythmia.
    4. Pulmonary hypertension > 40 mmHg.
    5. End-stage lung disease (TLC < 55%, FVC < 55%, or DLCO < 40% of predicted value).
    6. Serum creatinine > 2.5 or creatinine clearance < 30 ml/min.
    7. Liver cirrhosis, transaminases > 3x normal or bilirubin > 2.0 unless due to Gilbert's disease.
  6. HIV positive.
  7. 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.
  8. 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.
  9. 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.
  10. Significant psychological issues, social issues, psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible.
  11. Inability to give informed consent.
  12. Major hematological abnormalities such as platelet count less than 100,000/ul, ANC less than 1000/ul.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00282438

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United States, Illinois
Northwestern University, Feinberg School of Medicine
Chicago, Illinois, United States, 60611
Sponsors and Collaborators
Northwestern University
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Principal Investigator: Richard Burt, MD Northwestern University
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Responsible Party: Richard Burt, MD, MD, Northwestern University Identifier: NCT00282438    
Other Study ID Numbers: NU FDA SARC.2003
First Posted: January 26, 2006    Key Record Dates
Results First Posted: February 24, 2017
Last Update Posted: August 31, 2018
Last Verified: August 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Additional relevant MeSH terms:
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Lymphoproliferative Disorders
Lymphatic Diseases