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Fresolimumab In Systemic Sclerosis

Primary Purpose

Diffuse Systemic Sclerosis

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Fresolimumab

About this trial

This is an interventional treatment trial for Diffuse Systemic Sclerosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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)

Sites / Locations

Boston University School of Medicine; Rheumatology/Arthritis Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Fresolimumab

Arm Description

Outcomes

Primary Outcome Measures

To evaluate safety of fresolimumab in patients with scleroderma

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

Secondary Outcome Measures

Full Information

NCT ID

NCT01284322

First Posted

January 25, 2011

Last Updated

July 15, 2014

Sponsor

Boston University

1. Study Identification

Unique Protocol Identification Number

NCT01284322

Brief Title

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

2. Study Status

Record Verification Date

July 2014

Overall Recruitment Status

Completed

Study Start Date

January 2011 (undefined)

Primary Completion Date

June 2013 (Actual)

Study Completion Date

March 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Boston University

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Diffuse Systemic Sclerosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

18 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Fresolimumab

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

Fresolimumab

Other Intervention Name(s)

GC1008, anti-TGF-beta

Intervention Description

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

Primary Outcome Measure Information:

Title

To evaluate safety of fresolimumab in patients with scleroderma

Time Frame

24 weeks

Title

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.

Description

TGF-beta regulated skin gene expression

Time Frame

7 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

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)

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Robert Lafyatis, MD

Organizational Affiliation

Boston University

Official's Role

Principal Investigator

Facility Information:

Facility Name

Boston University School of Medicine; Rheumatology/Arthritis Center

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02118

Country

United States

12. IPD Sharing Statement

Citations:

PubMed Identifier

26098215

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.

Results Reference

derived

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Fresolimumab In Systemic Sclerosis

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