Diffuse Systemic Sclerosis
Conditions
Brief summary
The purpose of this study is to determine if fresolimumab is safe in treating people with systemic sclerosis (scleroderma) and to investigate the effect of fresolimumab in the skin of these individuals.
Interventions
DRUGFresolimumab
intravenous fresolimumab 1mg/kg, first 8 patients; 5 mg/kg following 8 patients
Sponsors
Boston University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Meet ACR criteria for diffuse systemic sclerosis * \< 24 months since onset of first SSc manifestation other than Raynaud's phenomenon * Modified Rodnan Skin Score ≥ 15 * Male or female adult ( ≥ 18 years of age) * Able and willing to give written informed consent and comply with study protocol
Exclusion criteria
* Moderate or severe pulmonary disease w/ FVC \< 80% or DLCO \< 70% or ground glass and fibrosis \> 20% of lung fields by HRCT * Treatment with investigational drug within 4 weeks of screening * Ongoing use of high dose steroids (\> 10mg/day) or unstable steroid dose in past 4 weeks * Treatment with immunosuppressive, cytotoxic, or antifibrotic drug within 4 weeks of screening * Positive for HIV, HBV, and/or HCV * Known active infection (bacterial, viral, fungal, mycobacterial, or other); not including fungal infection of nail beds or any major infection requiring hospitalization or treatment with IV antibiotics within 4 weeks of screening * Patients w/ history of malignancy or premalignant lesion * History of keratoacanthoma or squamous cell carcinoma * Moderate to severe hepatic impairment * SSc renal crisis within 6 months or creatinine \> 2.0 * Lack of IV access for medication administration * Moderate or severe cardiac disease with significant arrhythmia, heart failure, or unstable angina * Anemia (Hb \< 8.5 gm/dL) * Thrombocytopenia or blood clotting disorder * Patients with organ transplant (including allogeneic bone marrow transplant)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To evaluate safety of fresolimumab in patients with scleroderma | 24 weeks | — |
| To investigate the effect of fresolimumab on TGF-beta responsive gene expression in skin after treatment with fresolimumab compared to pre-treatment TGF-beta responsive gene expression. | 7 weeks | TGF-beta regulated skin gene expression |
Countries
United States
Outcome results
None listed