Hematopoietic Stem Cell Support in Patients With Refractory Sarcoidosis

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 by Northwestern University
Sponsor:
Information provided by (Responsible Party):
Richard Burt, MD, Northwestern University
ClinicalTrials.gov Identifier:
NCT00282438
First received: January 24, 2006
Last updated: February 4, 2014
Last verified: February 2014

January 24, 2006
February 4, 2014
December 2003
December 2015   (final data collection date for primary outcome measure)
Toxicity;Survival;Time to disease progression [ Time Frame: 5 years after transplant ] [ Designated as safety issue: Yes ]
  • 2.4 Time to disease progression
  • 2.1 Toxicity.
  • 2.2 Survival.
  • 2.3 Disease improvement
Complete list of historical versions of study NCT00282438 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
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Hematopoietic Stem Cell Support in Patients With Refractory Sarcoidosis
Hematopoietic Stem Cell Transplant in Patients With Refractory Sarcoidosis: A Phase I/II Trial

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.

Not Provided
Interventional
Phase 1
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Sarcoidosis
Biological: Hematopoietic stem cell transplantation
Hematopoietic stem cell transplantation
Experimental: Hematopoietic stem cell transplantation
Hematopoietic stem cell transplantation will be performed after conditioning
Intervention: Biological: Hematopoietic stem cell transplantation
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
10
December 2016
December 2015   (final data collection date for primary outcome measure)

Inclusion Criteria:

  1. Age > or = 18years and < or = 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 (patients with proven cardiac sarcoidosis do not need to meet the eligibility criteria listed in #'s 3 and 4 above).

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.
Both
18 Years to 60 Years
No
Contact: Dzemila Spahovic, MD 312-695-4960 d-spahovic@northwestern.edu
United States
 
NCT00282438
NU FDA SARC.2003
No
Richard Burt, MD, Northwestern University
Northwestern University
Not Provided
Principal Investigator: Richard Burt, MD Northwestern University
Northwestern University
February 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP