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Stem Cell Transplant to Treat Patients With Systemic Sclerosis

15 gennaio 2020 aggiornato da: Malcolm Brenner, Baylor College of Medicine

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.

Panoramica dello studio

Stato

Completato

Condizioni

Intervento / Trattamento

Descrizione dettagliata

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.

Tipo di studio

Interventistico

Iscrizione

24

Fase

  • Fase 1

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Luoghi di studio

  • Stati Uniti
    • Texas
      • Houston, Texas, Stati Uniti, 77030
        • The Methodist Hospital

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

Da 18 anni a 65 anni (Adulto, Adulto più anziano)

Accetta volontari sani

No

Sessi ammissibili allo studio

Tutto

Descrizione

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

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Trattamento
  • Assegnazione: Non randomizzato
  • Modello interventistico: Assegnazione di gruppo singolo
  • Mascheramento: Nessuno (etichetta aperta)

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Baylor College of Medicine

Collaboratori

The Methodist Hospital Research Institute
Center for Cell and Gene Therapy, Baylor College of Medicine

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio

1 giugno 1999

Completamento dello studio

1 giugno 2004

Date di iscrizione allo studio

Primo inviato

8 aprile 2003

Primo inviato che soddisfa i criteri di controllo qualità

8 aprile 2003

Primo Inserito (Stima)

9 aprile 2003

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

18 gennaio 2020

Ultimo aggiornamento inviato che soddisfa i criteri QC

15 gennaio 2020

Ultimo verificato

1 gennaio 2020

Maggiori informazioni

Termini relativi a questo studio

Termini MeSH pertinenti aggiuntivi

Altri numeri di identificazione dello studio

  • H7157
  • Systemic Sclerosis

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

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