Scleroderma, SystemicPhase 3NCT01347008
Effect of Sildenafil on the Microcirculatory Blood Flow and Endothelial Progenitor Cells in Systemic Sclerosis
A Phase 3 study testing Sildenafil citrate in people with Scleroderma, Systemic and Scleroderma, Diffuse, run by Federal University of São Paulo at 1 site in Brazil.
Trial summary
Early phases of systemic sclerosis is characterized by inflammatory and microvasculature alterations. Sildenafil citrate has been shown to have vasodilatory effects and to enhance vasculogenesis. Read more about this study
- Who is it for?
- Ages 18 years and older, women only
- Conditions the study is for
- Scleroderma, Systemic, Scleroderma, Diffuse, Scleroderma, Limited
- What is being tested?
- Sildenafil citrate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 41 participants
- Where
- 1 siteBrazil
- Study dates
- Apr 2011to Jan 2013
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Women only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Systemic sclerosis according to the classification criteria of the American College of Rheumatology (1980) with no more than 4 years since diagnosis
- Early systemic sclerosis as defined by LeRoy and Medsger (2001)
- 6 or more Raynaud's crisis per week
- Written informed consent provided by the subjects prior to initiating study procedures
Who cannot join
- Smoking
- Peripheral or central vasculopathy other than Systemic sclerosis
- Uncontrolled Diabetes
- Liver disease
- Pregnant or lactating woman
- Current use of cyclosporin, prostanoids, bosentan or any PDE-5 inhibitors (including current Sildenafil use during randomization)
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- History of stroke, myocardial infarction or life threatening cardiac condition within the last 6 months
- History of surgical sympathectomy
- Systolic blood pressure < 85mm Hg
- History of scleroderma renal crisis
- Known hypersensitivity to Sildenafil or any of the excipients
- History of Retinitis Pigmentosa
- Current use of Nitrates
Trial locations and contacts
1 site in 1 country.
Brazil1 site
Systemic Sclerosis Outpatient Clinic, Hospital Sao Paulo, UNIFESP
São Paulo, São Paulo
About this study
Official title: Effect of Sildenafil on the Microcirculatory Blood Flow and on the Endothelial Progenitor Cells in Patients With Systemic Sclerosis: a Randomized, Double-blind, Placebo-controlled Clinical Trial
Early phases of systemic sclerosis is characterized by inflammatory and microvasculature alterations. Sildenafil citrate has been shown to have vasodilatory effects and to enhance vasculogenesis. The purpose of this study is to evaluate the effect of sildenafil citrate on hand blood flow of patients with systemic sclerosis, using Laser Doppler Imaging.
Read the detailed description
Progressive microangiopathy and endothelial dysfunction are identified in early phases of systemic sclerosis. These alterations may result in progressive reduction of vessel lumen, decreased blood flow, and a state of chronic hypoxia resulting in digital ulcers, digital pitting, and, in more severe cases, gangrene and amputation of the extremities. Few studies aimed to evaluate the effects of Sildenafil on the microcirculatory blood flow in patients with Raynaud's phenomenon secondary to systemic sclerosis (SS). Moreover, no study has evaluated the effect of this drug on the number and function of endothelial progenitor cells in SS patients. The relatively new technique of laser Doppler imaging (LDI) allows an objective measurement of superficial cutaneous microvascular blood flow and constitutes a promising approach in the assessment of the digital microvascular vasoreactivity in response to cold stimulus or in response to treatment in SSc patients.
- Being tested
- Sildenafil citrate, Placebo (Sugar pill)
- Keywords
- Sildenafil, Endothelial Progenitor Cells, Laser Doppler Imaging
- Medical subject headings
- Scleroderma, Systemic, Scleroderma, Diffuse, Scleroderma, Limited, Raynaud Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sildenafil citrateDrug
Also called Sildenafil citrate, EMS Sigma Pharma, Ltd, Brazil
Oral sildenafil citratre, 50mg b.i.d., 8 weeks
Placebo (Sugar pill)Drug
Also called Placebo
Placebo pills similar to sildenafil citrate pills, b.i.d for 8 weeks
| Study group | Role | What they receive |
|---|---|---|
| Sildenafil citrate | Active comparator | Oral Sildenafil citrate, 50mg, b.i.d. Receives: Drug: Sildenafil citrate |
| Sugar pill | Placebo comparator | Placebo pill (identical to Sildenafil citrate 50mg), b.i.d. Receives: Drug: Placebo (Sugar pill) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Digital Skin Microvascular Blood Flow Measured by Laser Doppler Imaging (LDI) Before Cold Stimulus
Finger blood flow of the four medial fingers, measured by laser Doppler imaging and expressed in arbitrary perfusion units (p.u.).
Measured: 8 weeks
Digital Skin Microvascular Blood Flow Measured by Laser Doppler Imaging (LDI) After Cold Stimulus.
Measured: 8 weeks
Secondary outcomes (1)
Daily Frequency of Raynaud's Phenomenon Attacks
Daily frequency of RP attacks as self registered in a 1-week diary. Any episode of pallor or cyanosis of the hand/fingers was considered as a RP attack, and patients were supposed to register the daily amount of such episodes on a 1-week diary, previously to the medical visit.
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 May 2011 (estimated)
- Study startApr 2011
- Primary completionJan 2013
- Study completionJan 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Scleroderma, SystemicStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- Federal University of São Paulo
- Sponsor type
- University, hospital or other
- Collaborators
- Fundação de Amparo à Pesquisa do Estado de São Paulo
- Responsible party
- Principal investigator, Fernando Villela Andrigueti, Federal University of São Paulo
- Investigators
- Fernando V Andrigueti, MD, Principal investigator, Federal University of São Paulo
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01347008
- Sponsor's protocol ID
- FAPESP 2011/00012-3
- Status last verified
- Oct 2016
- First submitted
- 2 May 2011
- Last updated
- 5 Dec 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Maltez N, Maxwell LJ, Rirash F, Tanjong Ghogomu E, Harding SE, Tingey PC, Wells GA, Tugwell P, Pope J. Phosphodiesterase 5 inhibitors (PDE5i) for the treatment of Raynaud's phenomenon. Cochrane Database Syst Rev. 2023 Nov 6;11(11):CD014089. doi: 10.1002/14651858.CD014089.
PubMed 37929840
Study at a glance
From the registry record, last updated by the sponsor on 5 Dec 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Sildenafil on the Microcirculatory Blood Flow and on the Endothelial Progenitor Cells in Patients With Systemic Sclerosis: a Randomized, Double-blind, Placebo-controlled Clinical Trial
- ClinicalTrials.gov ID
- NCT01347008
- Acronym / sponsor ID
- FAPESP 2011/00012-3
- Keywords
- Sildenafil, Endothelial Progenitor Cells, Laser Doppler Imaging
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Federal University of São PauloUniversity, hospital or other
- Collaborators
- Fundação de Amparo à Pesquisa do Estado de São Paulo
- Study start
- Apr 2011
- Primary completion
- Jan 2013 (actual)
- Study completion
- Jan 2013 (actual)
- First posted
- 4 May 2011 (estimated)
- Results
- Posted 5 Dec 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Early phases of systemic sclerosis is characterized by inflammatory and microvasculature alterations. Sildenafil citrate has been shown to have vasodilatory effects and to enhance vasculogenesis. The purpose of this study is to evaluate the effect of sildenafil citrate on hand blood flow of patients with systemic sclerosis, using Laser Doppler Imaging.
Detailed description
Progressive microangiopathy and endothelial dysfunction are identified in early phases of systemic sclerosis. These alterations may result in progressive reduction of vessel lumen, decreased blood flow, and a state of chronic hypoxia resulting in digital ulcers, digital pitting, and, in more severe cases, gangrene and amputation of the extremities. Few studies aimed to evaluate the effects of Sildenafil on the microcirculatory blood flow in patients with Raynaud's phenomenon secondary to systemic sclerosis (SS). Moreover, no study has evaluated the effect of this drug on the number and function of endothelial progenitor cells in SS patients. The relatively new technique of laser Doppler imaging (LDI) allows an objective measurement of superficial cutaneous microvascular blood flow and constitutes a promising approach in the assessment of the digital microvascular vasoreactivity in response to cold stimulus or in response to treatment in SSc patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Sildenafil citrate | Active comparator | Drug: Sildenafil citrateOral Sildenafil citrate, 50mg, b.i.d. |
| Sugar pill | Placebo comparator | Drug: Placebo (Sugar pill)Placebo pill (identical to Sildenafil citrate 50mg), b.i.d. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Systemic sclerosis according to the classification criteria of the American College of Rheumatology (1980) with no more than 4 years since diagnosis
- Early systemic sclerosis as defined by LeRoy and Medsger (2001)
- 6 or more Raynaud's crisis per week
- Written informed consent provided by the subjects prior to initiating study procedures
Key exclusion criteria (13 listed)
- Smoking
- Peripheral or central vasculopathy other than Systemic sclerosis
- Uncontrolled Diabetes
- Liver disease
- Pregnant or lactating woman
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 |
|---|---|---|
| Sildenafil citrateAlso: Sildenafil citrate, EMS Sigma Pharma, Ltd, BrazilOral sildenafil citratre, 50mg b.i.d., 8 weeks | Drug | Sildenafil citrate |
| Placebo (Sugar pill)Also: PlaceboPlacebo pills similar to sildenafil citrate pills, b.i.d for 8 weeks | Drug | Sugar pill |
Primary outcome
Digital Skin Microvascular Blood Flow Measured by Laser Doppler Imaging (LDI) Before Cold Stimulus
Time frame: 8 weeks
Finger blood flow of the four medial fingers, measured by laser Doppler imaging and expressed in arbitrary perfusion units (p.u.).
Digital Skin Microvascular Blood Flow Measured by Laser Doppler Imaging (LDI) After Cold Stimulus.
Time frame: 8 weeks
1 secondary outcome
Daily Frequency of Raynaud's Phenomenon Attacks
Time frame: 8 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Dec 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37929840Maltez N, Maxwell LJ, Rirash F, Tanjong Ghogomu E, Harding SE, Tingey PC, Wells GA, Tugwell P, Pope J. Phosphodiesterase 5 inhibitors (PDE5i) for the treatment of Raynaud's phenomenon. Cochrane Database Syst Rev. 2023 Nov 6;11(11):CD014089. doi: 10.1002/14651858.CD014089.
- Individual participant dataSharing planned: No
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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.
Fernando V Andrigueti, MD principal investigator
Federal University of São Paulo
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Sildenafil citrate in people with Scleroderma, Systemic and Scleroderma, Diffuse, run by Federal University of São Paulo at 1 site in Brazil.
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 4 inclusion and 13 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 5 Dec 2016. 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 Brazil. 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Dec 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Digital Skin Microvascular Blood Flow Measured by Laser Doppler Imaging (LDI) Before Cold Stimulus (time frame: 8 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 NCT01347008, 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 5 Dec 2016.