Stem Cell Transplant to Treat Patients With Systemic Sclerosis

• Status: Completed
• Trial end date: 2004-06-01
• Target enrollment: 0
• Participant gender: All

Summary

Systemic Sclerosis is a disease that may be caused by the immune system reacting against skin and certain organs. It is possible, that by changing the immune system we can modify the progression of this disease.

Stem cells are created in the bone marrow. They mature into different types of blood cells that are needed including red blood cells, white blood cells, and platelets. In this study, we will stimulate the bone marrow to make extra stem cells. Next we will collect the stem cells, select specific cells, and store them. We will then give high dose chemotherapy that will destroy the patients immune system. We will then give back the selected stem cells we collected. We believe that these selected stem cells may be able to "re-create" the immune system without the portion that causes Systemic Sclerosis.

The purpose of this study is to try to discover if stem cell transplantation can help patients with Systemic Sclerosis. We will also try to learn what the side effects are of this treatment in patients with Systemic Sclerosis. We hope that this treatment will help to relieve the symptoms patients are experiencing, although we do not know if it will.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Baylor College of Medicine
• Collaborators: Center for Cell and Gene Therapy, Baylor College of Medicine; The Methodist Hospital Research Institute; The Methodist Hospital System
• Treatments: Cyclophosphamide

Criteria

Eligibility Criteria:

• Patients aged < 60 years

• Patients must have either one major or 2 minor criteria for systemic sclerosis as per the criteria developed during the Scleroderma Criteria Cooperative Study.

• Rapidly progressive diffuse skin disease without other organ involvement (at least one of the following):

• Scl-70 positive

• Rodnam Skin score 16

• With/without U3RNP antibodies; RNA polymerases 1-111 OR

• Internal Organ Involvement (at least one of the following):

• Renal Proteinuria > 500mg/dl

• Creatinine clearance > 50ml/min.

• Blood pressure controlled to ≤ 160/110

• Interstitial lung disease on high resolution CT

• Hypoxemia (pO2 > 70 mmHg)

• FVC > 50%

• DLCO > 45%

• Cardiac Disease

• Myocarditis

• Pericarditis

• Coronary Artery Ejection Fraction > 30%

• Patients must meet the following hematological parameters:

• Have an ANC > 500/mm3

• Have a platelet count > 120 x lO9/l

• Have a hemoglobin > 10g/dl

Exclusion Criteria:

• Patients > 60 years

• Patients with pulmonary, cardiac, hepatic, or renal impairment which would limit their ability to receive cytoreductive therapy and compromise their survival. This should include patients with any of the following:

• Severe Lung Disease

• Hypoxemia (pO2 £70 mmHg)

• FVC of < 50%

• DLCO of < 45%

• Cardiac Disease

• Ejection fraction < 30%

• Uncontrolled arrhythmias

• Cor. Pulmonale

• Pulmonary hypertension (mPAP >/=60 mmHg)

• Loss of digits or vascular access secondary to Raynaud's ischemia

• History of oliguric renal failure or episode of renal crisiswith Glomerular filtration rate < 50ml/min Creatinine. Weight loss > 20% baseline since first involvement of gastrointestinal tract (midgut); or any patient requiring hyperalimentation prior to transplant because of gut dysfunction related to systemic sclerosis

• SGOT/bilirubin > 2 x UPN on 2 repeated tests

• Has active uncontrolled infection

• Is sero-positive for HIV

• Has demonstrated lack of compliance with prior medical care

• Has active malignancy

• Life expectancy is severely limited by illness other than scleroderma

• Has evidence of myelodysplasia or prior extensive chemotherapy

• Has uncontrolled hypertension

• Positive pregnancy test