Stem Cell Transplant to Treat Patients With Systemic Sclerosis
Autologous T-Cell Depleted Peripheral Blood Stem Cell Transplantation for the Treatment of Selected Patients With Systemic Sclerosis
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.
Studie Overzicht
Toestand
Conditie
Gedetailleerde beschrijving
Before the transplant the research participant will receive daily G-CSF (Neupogen) for 5-6 days. This medication will help to stimulate the production of white blood cells (WBC) that will be used for the stem cell transplant. The G-CSF will be given as an injection into the arm.
If G-CSF does not stimulate the stem cells sufficiently, the patient will receive a single dose of drug called cyclophosphamide (chemotherapy) intravenously (into a vein). This drug will cause the blood cell counts to fall. A drug called MESNA will also be given to help protect the bladder from the Cyclophosphamide. After completing chemotherapy, patients will be started on G-CSF again until blood cell counts reach a certain level, at which time the patient will undergo leukopheresis. Leukopheresis is a procedure where blood is removed from one arm, pumped into a machine where the white blood cells are separated from most of the other cells and then returned through the same needle or through a needle in the other arm. This procedure usually takes 3 to 4 hours a day for up to 4 days in a row, depending on how many cells are collected each time.
After collection of the white blood cells, special agents (called monoclonal antibodies) will be used in the laboratory to select out certain types of white blood cells (CD34+ cells). The blood cells will be separated on a machine which picks out stem cells.
After leukopheresis, patients will receive drugs called cyclophosphamide and Mesna. They will also receive a drug called Atgam and radiation treatment to the entire body. This treatment will kill most of the blood forming cells in the bone marrow. We will then give the CD34+ cells that were collected during leukopheresis.
After the transplant patients will be followed closely, the same as any patient who receives a stem cell transplant. This follow-up will involve blood tests to see how the body is recovering after the chemotherapy and radiation, and a bone marrow aspiration once a year for 2 years.
Studietype
Inschrijving
Fase
- Fase 1
Contacten en locaties
Studie Locaties
-
-
Texas
-
Houston, Texas, Verenigde Staten, 77030
- The Methodist Hospital
-
-
Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
Accepteert gezonde vrijwilligers
Geslachten die in aanmerking komen voor studie
Beschrijving
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
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: Niet-gerandomiseerd
- Interventioneel model: Opdracht voor een enkele groep
- Masker: Geen (open label)
Medewerkers en onderzoekers
Studie record data
Bestudeer belangrijke data
Studie start
Studie voltooiing
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
Eerst geplaatst (Schatting)
Updates van studierecords
Laatste update geplaatst (Werkelijk)
Laatste update ingediend die voldeed aan QC-criteria
Laatst geverifieerd
Meer informatie
Termen gerelateerd aan deze studie
Aanvullende relevante MeSH-voorwaarden
- Pathologische processen
- Huidziektes
- Bindweefselziekten
- Sclerose
- Sclerodermie, systemisch
- Sclerodermie, Diffuus
- Fysiologische effecten van medicijnen
- Moleculaire mechanismen van farmacologische werking
- Antireumatische middelen
- Antineoplastische middelen
- Immunosuppressieve middelen
- Immunologische factoren
- Antineoplastische middelen, alkylering
- Alkyleringsmiddelen
- Myeloablatieve agonisten
- Cyclofosfamide
Andere studie-ID-nummers
- H7157
- Systemic Sclerosis
Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .
Klinische onderzoeken op Systemische sclerose
-
University of ArkansasBeëindigdPediatrische patiënten met SIRS (Systemic Inflammatory Response Syndrome)Verenigde Staten