Allogeneic Hematopoietic Stem Cell Transplantation (NST) for Patients With Systemic Sclerosis

NCT00282425 | Terminated Phase 1 DMC

• 1 other identifier: DIAD Scl.Allo2005
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

Scleroderma is disease believed to be due to immune cells, cells which normally protect the body but are now causing damage to the body. There has not been any treatment that has been effective in treating this disease. The likelihood of progression of the disease to severe disability and death is high. This study is designed to examine whether treating patients with high dose Cyclophosphamide and Fludarabine (drugs which reduce the function of your immune system) and CAMPATH-1H (a protein that kills the immune cells that are thought to be causing the disease), followed by return of blood stem cells that have been previously collected from patients brother or sister will stop or reverse the disease. The purpose of the Cyclophosphamide, Fludarabine and CAMPATH-1H is to decrease immune system. The purpose of the stem cell infusion is to restore blood production, which will be severely impaired by the Cyclophosphamide, Fludarabine and CAMPATH-1H, and to produce a normal immune system that will no longer attack the body.

Conditions

Scleroderma

Outcome Measures

Primary Outcomes (1)

• Survival; Disease improvement;Time to disease progression

  5 years after transplant

Study Arms (1)

Allogeneic Hematopoietic stem cell transplantation

EXPERIMENTAL

Allogeneic Hematopoietic stem cell transplantation will be performed on eligible patients

Biological: Hematopoietic stem cell transplantation

Interventions

Hematopoietic stem cell transplantation BIOLOGICAL

Allogeneic Hematopoietic stem cell transplantation

Allogeneic Hematopoietic stem cell transplantation

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Age 18 to 55 years old
• An established diagnosis of scleroderma (84)
• Diffuse cutaneous scleroderma with involvement proximal to the elbow or knee and a Rodnan score (see Appendix III) of \> 14 (85)
• And one of the following:
• DLCO \< 80% of predicted or decrease in lung function (TLC, DLCO or FEV1) of 10% or more over 12 months
• Active alveolitis on bronchoalveolar lavage
• Pulmonary fibrosis or alveolitis on CT scan or CXR
• Elevated ESR greater than or equal to 25mm/hour confirmed on a second occasion at least two weeks apart without evidence of active infectious process.
• Abnormal EKG (low QRS voltage, or ventricular hypertrophy) or left ventricle (LV) diastolic dysfunction (expressed by an inverted E/A ratio which represents early and late filling of the LV during atrial contraction) or LV wall thickness
• Since pulmonary disease independent of skin score (NEJM, 2006, 345:25 2655-2709) carries a poor prognosis, patient may be enroled for only lung involvement defined as active alveolitis on BAL or ground-glass opacity on CT, a DLCO \< 80% predicted or decrease in lung function (TLC), DLCO, FVC) of 10% or more in last 12 months.

You may not qualify if:

• Poor performance status (ECOG \> or =2) at the time of entry, unless due to disease.
• Significant end organ damage such as:
• LVEF \<40% or deterioration of LVEF during exercise test on MUGA or echocardiogram
• Untreated life-threatening arrhythmia
• Active ischemic heart disease or heart failure
• DLCO less than 45% of predicted value, unless due to disease.
• Pulmonary hypertension (estimated systolic pulmonary arterial pressure \>40 mmHg by Doppler echocardiography or measurement by pulmonary arterial catheter)
• Serum creatinine \> 2.0 mg/dl
• Liver cirrhosis, transaminases \>3x of normal limits or bilirubin \>2.0 unless due to Gilberts disease
• HIV positive
• Uncontrolled diabetes mellitus or any other illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive treatment
• Prior history of malignancy except localized basal cell or squamous skin cancer. Other malignancies for which the patient is judged to be cured by local surgical therapy, such as (but not limited to) head and neck cancer, or stage I or II breast cancer will be considered on an individual basis.
• Positive pregnancy test, inability or unable to pursue effective means of birth control, failure to willingly accept or comprehend irreversible sterility as a side effect of therapy
• Psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible
• Inability to give informed consent

Sponsors & Collaborators

• Richard Burt, MD: lead

Study Sites (1)

Northwestern University, Feinberg School of Medicine

Chicago, Illinois, 60611, United States

Location

MeSH Terms

Conditions

Scleroderma, Diffuse

Interventions

Hematopoietic Stem Cell Transplantation

Condition Hierarchy (Ancestors)

Scleroderma, Systemic; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Intervention Hierarchy (Ancestors)

Stem Cell Transplantation; Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative

Study Officials

• Richard Burt, MD

  Northwestern University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: January 24, 2006
• First Posted: January 26, 2006
• Study Start: May 1, 2005
• Primary Completion: December 1, 2012
• Study Completion: December 1, 2012
• Last Updated: December 18, 2012

Record last verified: 2012-12

Locations

US (1)