Clinical trial NCT01284322: Fresolimumab In Systemic Sclerosis
Official title: OPEN LABEL TRIAL OF ANTI-TGF-BETA MAB, FRESOLIMUMAB, IN SYSTEMIC SCLEROSIS - A PHASE ONE BIOMARKER TRIAL
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Diffuse Systemic Sclerosis
Lead sponsor: Boston University
Sponsor class: OTHER
First posted: 2011-01-27
Enrollment: 18
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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)

Primary outcomes:
measure: To evaluate safety of fresolimumab in patients with scleroderma
timeFrame: 24 weeks
measure: 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.
timeFrame: 7 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Boston
country: United States
facility: Boston University School of Medicine; Rheumatology/Arthritis Center
state: Massachusetts
Study officials:
affiliation: Boston University
name: Robert Lafyatis, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Rice LM, Padilla CM, McLaughlin SR, Mathes A, Ziemek J, Goummih S, Nakerakanti S, York M, Farina G, Whitfield ML, Spiera RF, Christmann RB, Gordon JK, Weinberg J, Simms RW, Lafyatis R. Fresolimumab treatment decreases biomarkers and improves clinical symptoms in systemic sclerosis patients. J Clin Invest. 2015 Jul 1;125(7):2795-807. doi: 10.1172/JCI77958. Epub 2015 Jun 22.