Fresolimumab In Systemic Sclerosis

Diffuse Systemic Sclerosis Clinical Trial

Official title:

OPEN LABEL TRIAL OF ANTI-TGF-BETA MAB, FRESOLIMUMAB, IN SYSTEMIC SCLEROSIS - A PHASE ONE BIOMARKER TRIAL

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01284322
• Other study ID #: H30142
• Status: Completed
• Phase: Phase 1
• First received: January 25, 2011
• Last updated: July 15, 2014
• Start date: January 2011
• Est. completion date: March 2014

Study information

• Verified date: July 2014
• Source: Boston University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 18
• Est. completion date: March 2014
• Est. primary completion date: June 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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)

Study Design

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Diffuse Systemic Sclerosis
• Scleroderma, Diffuse
• Scleroderma, Systemic
• Sclerosis

Intervention

Drug:
• Fresolimumab
intravenous fresolimumab 1mg/kg, first 8 patients; 5 mg/kg following 8 patients

Locations

Country	Name	City	State
United States	Boston University School of Medicine; Rheumatology/Arthritis Center	Boston	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Boston University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To evaluate safety of fresolimumab in patients with scleroderma		24 weeks	Yes
Primary	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.	TGF-beta regulated skin gene expression	7 weeks	No