Hematopoietic Stem Cell Support in Patients With Refractory Sarcoidosis - Full Text View

Sponsor:	Richard Burt
Information provided by (Responsible Party):	Richard Burt, Northwestern University
ClinicalTrials.gov Identifier:	NCT00282438

Purpose

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	Intervention	Phase
Sarcoidosis	Procedure: Hematopoietic stem cell transplantation	Phase I

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	High Dose Cyclophosphamide & ATG With Hematopoietic Stem Cell Support in Patients With Refractory Sarcoidosis: A Phase I Trial

Resource links provided by NLM:

MedlinePlus related topics: Sarcoidosis

U.S. FDA Resources

Further study details as provided by Northwestern University:

Primary Outcome Measures:

Toxicity;Survival;Time to disease progression [ Time Frame: 5 years after transplant ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	10
Study Start Date:	December 2003
Estimated Study Completion Date:	December 2014
Estimated Primary Completion Date:	December 2013 (Final data collection date for primary outcome measure)

Intervention Details:

Autologous hematopoietic stem cell transplantation

Eligibility

Ages Eligible for Study:	16 Years to 72 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Age > or = 16 years and < or = 72 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):
  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.
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:

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:
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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00282438

Contacts

Contact: Dzemila Spahovic, MD

312-908-0059

d-spahovic@northwestern.edu

Locations

United States, Illinois
Northwestern University, Feinberg School of Medicine	Recruiting
Chicago, Illinois, United States, 60611
Principal Investigator: Richard Burt, MD
Sub-Investigator: Thomas Corbridge, MD
Sub-Investigator: Mihai Gheorghiade, MD
Sub-Investigator: Robert Sufit, MD
Sub-Investigator: Kathleen Quigley, R.N
Sub-Investigator: Kimberly Yaung, R.N

Sponsors and Collaborators

Richard Burt

Investigators

Principal Investigator:

Richard Burt, MD

Northwestern University

More Information

No publications provided

Responsible Party:	Richard Burt, MD, Northwestern University
ClinicalTrials.gov Identifier:	NCT00282438 History of Changes
Other Study ID Numbers:	NU FDA SARC.AUTO2003
Study First Received:	January 24, 2006
Last Updated:	August 29, 2011
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:

Sarcoidosis
Lymphoproliferative Disorders
Lymphatic Diseases

ClinicalTrials.gov processed this record on February 09, 2012