NCT00058578

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
24

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Jun 1999

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 1999

Completed
3.9 years until next milestone

First Submitted

Initial submission to the registry

April 8, 2003

Completed
1 day until next milestone

First Posted

Study publicly available on registry

April 9, 2003

Completed
1.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2004

Completed
Last Updated

January 18, 2020

Status Verified

January 1, 2020

First QC Date

April 8, 2003

Last Update Submit

January 15, 2020

Conditions

Interventions

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may not qualify if:

  • 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
  • +7 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

The Methodist Hospital

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Scleroderma, Systemic

Interventions

CyclophosphamideMesnaGranulocyte Colony-Stimulating FactorLeukapheresisWhole-Body Irradiation

Condition Hierarchy (Ancestors)

Connective Tissue DiseasesSkin and Connective Tissue DiseasesSkin Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus CompoundsAlkanesulfonatesAlkanesulfonic AcidsAlkanesHydrocarbons, AcyclicSulfhydryl CompoundsSulfur CompoundsSulfonic AcidsSulfur AcidsColony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological FactorsCytapheresisBiological TherapyTherapeuticsBlood Component RemovalLeukocyte Reduction ProceduresCell SeparationCytological TechniquesClinical Laboratory TechniquesInvestigative TechniquesRadiotherapy

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Dist Serv Prof, Center for Gene Therapy

Study Record Dates

First Submitted

April 8, 2003

First Posted

April 9, 2003

Study Start

June 1, 1999

Study Completion

June 1, 2004

Last Updated

January 18, 2020

Record last verified: 2020-01

Locations