Diffuse Systemic SclerosisPhase 1NCT01284322
Fresolimumab In Systemic Sclerosis
A Phase 1 study testing Fresolimumab in people with Diffuse Systemic Sclerosis, run by Boston University at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Diffuse Systemic Sclerosis
- What is being tested?
- Fresolimumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2011to Mar 2014
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 15Show fewer
- 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)
Trial locations and contacts
1 site in 1 country.
United States1 site
Boston University School of Medicine; Rheumatology/Arthritis Center
Boston, Massachusetts, 02118
About this study
Official title: OPEN LABEL TRIAL OF ANTI-TGF-BETA MAB, FRESOLIMUMAB, IN SYSTEMIC SCLEROSIS - A PHASE ONE BIOMARKER TRIAL
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.
- Conditions
- Diffuse Systemic Sclerosis
- Being tested
- Fresolimumab
- Medical subject headings
- Scleroderma, Diffuse
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FresolimumabDrug
Also called GC1008, anti-TGF-beta
intravenous fresolimumab 1mg/kg, first 8 patients; 5 mg/kg following 8 patients
| Study group | Role | What they receive |
|---|---|---|
| Fresolimumab | Experimental | Not described Receives: Drug: Fresolimumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate safety of fresolimumab in patients with scleroderma
Measured: 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
Measured: 7 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2011 (estimated)
- Study startJan 2011
- Primary completionJun 2013
- Study completionMar 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Diffuse Systemic SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Robert Lafyatis, Boston University
- Investigators
- Robert Lafyatis, MD, Principal investigator, Boston University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01284322
- Sponsor's protocol ID
- H30142
- Status last verified
- Jul 2014
- First submitted
- 25 Jan 2011
- Last updated
- 16 Jul 2014
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 26098215
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- OPEN LABEL TRIAL OF ANTI-TGF-BETA MAB, FRESOLIMUMAB, IN SYSTEMIC SCLEROSIS - A PHASE ONE BIOMARKER TRIAL
- ClinicalTrials.gov ID
- NCT01284322
- Acronym / sponsor ID
- H30142
- Conditions
- Diffuse Systemic Sclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Boston UniversityUniversity, hospital or other
- Study start
- Jan 2011
- Primary completion
- Jun 2013 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 27 Jan 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fresolimumab | Experimental | Drug: Fresolimumab |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (15 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FresolimumabAlso: GC1008, anti-TGF-betaintravenous fresolimumab 1mg/kg, first 8 patients; 5 mg/kg following 8 patients | Drug | Fresolimumab |
Primary outcome
To evaluate safety of fresolimumab in patients with scleroderma
Time frame: 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.
Time frame: 7 weeks
TGF-beta regulated skin gene expression
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2014.
- Derived from this study: PubMed 26098215Rice 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert Lafyatis, MD principal investigator
Boston University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Fresolimumab in people with Diffuse Systemic Sclerosis, run by Boston University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Jul 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate safety of fresolimumab in patients with scleroderma (time frame: 24 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01284322, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2014.