Stem Cell Transplant to Treat Patients With Systemic Sclerosis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00058578
• Date of registration: 08/04/2003
• Prospective Registration: No
• Primary sponsor: Baylor College of Medicine
• Public title: Stem Cell Transplant to Treat Patients With Systemic Sclerosis
• Scientific title: Autologous T-Cell Depleted Peripheral Blood Stem Cell Transplantation for the Treatment of Selected Patients With Systemic Sclerosis
• Date of first enrolment: June 1999
• Target sample size: 24
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00058578
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Key inclusion & exclusion criteria

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



Age minimum: 18 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Systemic Sclerosis

Intervention(s)

Procedure: Total Body Irradiation

Drug: G-CSF

Drug: Cyclophosphamide

Drug: Mesna

Procedure: Leukopheresis

Secondary ID(s)

Systemic Sclerosis

H7157

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Center for Cell and Gene Therapy, Baylor College of Medicine

The Methodist Hospital System