NCT01284322

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
18

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Jan 2011

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2011

Completed
24 days until next milestone

First Submitted

Initial submission to the registry

January 25, 2011

Completed
2 days until next milestone

First Posted

Study publicly available on registry

January 27, 2011

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2013

Completed
9 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2014

Completed
Last Updated

July 16, 2014

Status Verified

July 1, 2014

Enrollment Period

2.4 years

First QC Date

January 25, 2011

Last Update Submit

July 15, 2014

Conditions

Diffuse Systemic Sclerosis

Outcome Measures

Primary Outcomes (2)

  • 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.

    TGF-beta regulated skin gene expression

    7 weeks

Study Arms (1)

Fresolimumab

EXPERIMENTAL
Drug: Fresolimumab

Interventions

FresolimumabDRUG

intravenous fresolimumab 1mg/kg, first 8 patients; 5 mg/kg following 8 patients

Also known as: GC1008, anti-TGF-beta
Fresolimumab

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Boston University School of Medicine; Rheumatology/Arthritis Center

Boston, Massachusetts, 02118, United States

Location

Related Publications (1)

  • 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.

    PMID: 26098215DERIVED

MeSH Terms

Conditions

Scleroderma, Diffuse

Interventions

fresolimumab

Condition Hierarchy (Ancestors)

Scleroderma, SystemicConnective Tissue DiseasesSkin and Connective Tissue DiseasesSkin Diseases

Study Officials

  • Robert Lafyatis, MD

    Boston 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
PRINCIPAL INVESTIGATOR
PI Title
MD

Study Record Dates

First Submitted

January 25, 2011

First Posted

January 27, 2011

Study Start

January 1, 2011

Primary Completion

June 1, 2013

Study Completion

March 1, 2014

Last Updated

July 16, 2014

Record last verified: 2014-07

Locations

US(1)