Home
Search
Study Topics
Glossary
|
|
|
|
|
Sponsors and Collaborators: |
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) University of Pittsburgh Amgen Genzyme |
---|---|
Information provided by: | National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) |
ClinicalTrials.gov Identifier: | NCT00040651 |
Scleroderma, or systemic sclerosis (SSc), is a diffuse connective tissue disease characterized by changes in the skin, blood vessels, skeletal muscles, and internal organs. The purpose of this study is to determine the safety and value of self bone marrow transplants after chemotherapy in patients with severe SSc.
Condition | Intervention | Phase |
---|---|---|
Scleroderma Systemic Sclerosis |
Drug: Fludarabine Drug: Cyclophosphamide Drug: Thymoglobulin Procedure: Leukapheresis Procedure: Self bone marrow transplant |
Phase I |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Dose Comparison, Parallel Assignment, Safety/Efficacy Study |
Official Title: | Transplantation With T-Cell Depleted Autologous Peripheral Stem Cells for Severe Systemic Sclerosis: A Phase I Dose Escalation Study |
Estimated Enrollment: | 15 |
Study Start Date: | July 2002 |
Study Completion Date: | March 2007 |
Primary Completion Date: | March 2007 (Final data collection date for primary outcome measure) |
SSc is a chronic disease involving the abnormal growth of connective tissue, which supports the skin and internal organs. This disease can affect the skin, making it hard and tight; it can also damage the blood vessels and internal organs such as the heart, lungs, and kidneys. Initially, precursor blood cells will be mobilized from the bone marrow into the blood stream after chemotherapy and white blood cell growth factors are administered. These precursors (or autologous stem cells) can be harvested from the bloodstream in a procedure called leukapheresis; it is the precursor blood cells that are transplanted back into the patient's body after high dose chemotherapy. Autologous stem cells are preferred over donor bone marrow because there is no risk of rejection. This study will evaluate the safety and effectiveness of self bone marrow transplants after intravenous chemotherapy in patients with SSc.
Prior to transplantation, patients will undergo diphtheria/tetanus (DT) vaccination and blood collection. Two weeks after vaccination, patients will have a Hickman catheter inserted into their bodies and will be admitted to the hospital to receive mobilization chemotherapy with intravenous (IV) cyclophosphamide.
Patients will be discharged after receiving the cyclophosphamide therapy with the understanding that they must stay locally and must return to the outpatient clinic daily to have blood samples drawn and to receive an injection of a growth factor, G-CSF, in stimulate blood cell production. Patients will undergo leukapheresis at the clinic when their white blood cell (WBC) counts reach 2500 cells/mm3 or more. A machine called the Nexell Isolex 300i will be used to remove T-cells from the cells collected by leukapheresis.
After leukapheresis and other pre-transplant procedures have been completed, patients will be hospitalized for approximately 14 to 21 days. On Days 1 through 5 of hospitalization, patients will receive IV fludarabine and one of several possible dose levels of cyclophosphamide. On Days 3 through 5, patients will receive IV thymoglobulin to kill the T-cells that cause the damage from systemic sclerosis. On Day 8, patients will receive their own stem cells from the previous leukapheresis procedure. While in the hospital, patients will be monitored by daily blood collection and will not be discharged until their white blood cell counts return to a safe, stable level. Prior to discharge from the hospital, patients will undergo a second leukapheresis.
Patients are required to stay locally in Pittsburgh up to 100 days post-transplantation. Study visits will occur at the clinic every week for the first three months after transplant and again at 4, 5, 6, 9, 12, 18, and 24 months post-transplantation. Study visits will include a physical exam and blood collection; patients will be also asked to complete a questionnaire. Patients will undergo an electrocardiogram (EKG) at Month 1, a chest x-ray at Month 6, 24-hour urine collection at Months 6 and 18, and pulmonary tests at Months 6, 12, and 24. Additional leukapheresis will be conducted at 12 and 24 months post-transplant to assess patients' health.
Ages Eligible for Study: | 18 Years to 70 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, Pennsylvania | |
UPMC Hillman Cancer Center | |
Pittsburgh, Pennsylvania, United States, 15232 |
Principal Investigator: | Robert Herberman, MD | UPMC Health System |
Study ID Numbers: | N01 AR92239, NIAMS-047 |
Study First Received: | July 5, 2002 |
Last Updated: | December 19, 2007 |
ClinicalTrials.gov Identifier: | NCT00040651 History of Changes |
Health Authority: | United States: Food and Drug Administration |
Systemic Sclerosis Chemotherapy Cyclophosphamide Fludarabine Leukapheresis Mesna |
Systemic Sclerosis Stem Cell T-Cell Thymoglobulin Scleroderma |
Antimetabolites Skin Diseases Immunologic Factors Sclerosis Cyclophosphamide Fludarabine monophosphate Immunosuppressive Agents Connective Tissue Diseases |
Scleroderma, Diffuse Scleroderma Scleroderma, Systemic Antineoplastic Agents, Alkylating Fludarabine Antirheumatic Agents Alkylating Agents Mesna |
Antimetabolites Antimetabolites, Antineoplastic Molecular Mechanisms of Pharmacological Action Skin Diseases Immunologic Factors Antineoplastic Agents Physiological Effects of Drugs Sclerosis Cyclophosphamide Fludarabine monophosphate Immunosuppressive Agents |
Pharmacologic Actions Pathologic Processes Therapeutic Uses Myeloablative Agonists Connective Tissue Diseases Scleroderma, Diffuse Scleroderma, Systemic Antineoplastic Agents, Alkylating Fludarabine Antirheumatic Agents Alkylating Agents |