Sildenafil for Scleroderma, Systemic

Phase-Based Estimates
1
Effectiveness
2
Safety
Johns Hopkins, Baltimore, MD
Scleroderma, Systemic+5 More
Sildenafil - Drug
Eligibility
18+
All Sexes
Eligible conditions
Scleroderma, Systemic

Study Summary

This study is evaluating whether a drug used to treat erectile dysfunction may help treat pulmonary hypertension in patients with scleroderma.

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Eligible Conditions

  • Scleroderma, Systemic
  • Morphea
  • Scleroderma, Diffuse
  • Vascular Diseases
  • Mildly Elevated Pulmonary Pressures
  • Scleroderma, Localized
  • Scleroderma

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Sildenafil will improve 1 primary outcome and 9 secondary outcomes in patients with Scleroderma, Systemic. Measurement will happen over the course of Baseline and 4 months.

Baseline and 12 months
Difference in change in distance walked in 6MWT at 12 months
Difference in change in right ventricular function as assessed by cardiac MRI
Difference in change in right ventricular function as assessed by echocardiography
Difference in change in right ventricular function as assessed by invasive hemodynamics
Baseline and 4 months
Difference in change in distance walked in 6 minute walk test (6MWT) at 4 months
Month 12
Difference in change in N-terminal pro b-type natriuretic peptide level
Difference in change in health related quality of life as assessed by the 36-Item Short Form Health Survey (SF36) questionnaire
Difference in change in health related quality of life as assessed by the emPHasis-10 health-related quality of life questionnaire (emPHasis-10).
Year 4
Difference in safety profile as assessed by frequency of adverse events
Difference in safety profile as assessed by severity of adverse events

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Side Effects for

Sildenafil
Nasopharyngitis
50%
Bronchitis
50%
Upper respiratory tract infection
50%
Gastroenteritis
33%
Diarrhoea
33%
Epistaxis
33%
Headache
33%
Conjunctivitis allergic
17%
Eczema
17%
Ammonia increased
17%
Flushing
17%
Erection increased
17%
Molluscum contagiosum
17%
Myalgia
17%
Cardiac failure
17%
Alanine aminotransferase increased
17%
Vomiting
17%
Dental caries
17%
Feeling abnormal
17%
Influenza
17%
Dysmenorrhoea
17%
Aspartate aminotransferase increased
17%
Chest pain
17%
Vision blurred
17%
Weight increased
17%
Rhinitis allergic
17%
Acne
17%
Colitis
17%
Dermatitis diaper
17%
Blood urine present
17%
Pulmonary arterial hypertension
17%
Visual acuity reduced transiently
17%
Rash
17%
Dry skin
17%
Streptococcal infection
17%
This histogram enumerates side effects from a completed 2018 Phase 4 trial (NCT01642407) in the Sildenafil ARM group. Side effects include: Nasopharyngitis with 50%, Bronchitis with 50%, Upper respiratory tract infection with 50%, Gastroenteritis with 33%, Diarrhoea with 33%.

Trial Design

2 Treatment Groups

Placebo
Sildenafil
Placebo group

This trial requires 30 total participants across 2 different treatment groups

This trial involves 2 different treatments. Sildenafil is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 and have already been tested with other people.

Sildenafil
Drug
Sildenafil 20 mg by mouth three(3) times each day
Placebo
Other
Placebo by mouth three(3) times each day
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Sildenafil
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: ongoing until study closes, up to 4 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly ongoing until study closes, up to 4 years for reporting.

Closest Location

Johns Hopkins - Baltimore, MD

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 6 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Diagnosis of SSc according to 2013 American College of Rheumatology/ European League Against Rheumatism (ACR/EULAR) classification criteria.
Pulmonary function tests with forced expiratory volume in one second/forced vital capacity (FEV1/FVC) >50% AND either a) total lung capacity (TLC) or forced vital capacity (FVC) > 70% predicted or b) TLC or FVC between 60% and 70% predicted with no more than mild interstitial lung disease on computerized tomography scan of the chest on studies obtained within 6 months of enrollment.
Ventilation perfusion scan or computed tomography with intravenous contrast (CT angiogram) without evidence of chronic thromboembolism at anytime before study entry.
Previous documentation of mean pulmonary artery pressure between 21 and 24 mm Hg with a pulmonary capillary wedge pressure (or left ventricular end-diastolic pressure) ≤ 15 mm Hg within six months before study entry.
Ability to perform six minute walk testing without significant limitations in musculoskeletal function or coordination.
Informed consent.

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is sildenafil?

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In people with SSc-associated vascular disease, sildenafil therapy may reduce the extent of pulmonary arterial disease and RV failure, in part through a VEGF-independent mechanism.

Unverified Answer

What are common treatments for scleroderma, systemic?

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This article documents the use of available medications in the clinic, and presents a review of the evidence for these treatments in the literature of scleroderma. These data may provide some guidance to the practitioner's decision-making.

Unverified Answer

Has sildenafil proven to be more effective than a placebo?

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Sildenafil does demonstrate an improvement in exercise capacity in patients with scleroderma and an improvement in quality of life in patients not using concomitant medications. These benefits are seen in both treatment groups in a clinically meaningful manner.

Unverified Answer

Does sildenafil improve quality of life for those with scleroderma, systemic?

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This pilot study suggests that sildenafil significantly improves quality of life for those with systemic scleroderma, without any significant adverse effects. Sildenafil may prove to be beneficial, particularly in those with concomitant pulmonary hypertension.

Unverified Answer

How many people get scleroderma, systemic a year in the United States?

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The exact figure of scleroderma, systemic a year could not be given due to low incidence. However, SSc seems to be more common in Caucasians than Hispanics and African Americans.

Unverified Answer

What are the signs of scleroderma, systemic?

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Patients with SSc may experience a number of symptoms such as Raynaud's phenomenon, nail involvement, cutaneous vascular abnormalities, joint involvement and arthritis. These symptoms may mimic those of other systemic connective tissue diseases such as SLE. Patients should be aware that the symptoms and the clinical manifestations of these diseases may be similar.

Unverified Answer

What causes scleroderma, systemic?

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There is a clear overlap between systemic sclerosis and the other scleroderma syndromes, particularly polymyositis. Even before scleroderma is diagnosed, nearly half of patients will have polymyositis.

Unverified Answer

What is scleroderma, systemic?

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This article is a systematic review, that looks at the known effects on one of the most common autoimmune disorders, diffuse cutaneous systemic sclerosis. It looks at the main manifestations of scleroderma, including skin involvement, autoimmunity, renal crises and aortic involvement. It also describes therapies that are being investigated to treat the autoimmune symptoms and complications of systemic scleroderma.

Unverified Answer

Can scleroderma, systemic be cured?

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Oral steroids, oral methotrexate or oral cyclophosphamide can alleviate many of the symptoms of systemic sclerosis, and the clinical course of the disease is less severe compared with those who are not treated. Therefore, such treatments should be applied strictly in an effort to promote a better prognosis when necessary.

Unverified Answer

Have there been other clinical trials involving sildenafil?

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Trials involving sildenafil that have not been mentioned in the literature suggest that sildenafil does have good therapeutic effects in patients with pulmonary hypertension. One study suggests sildenafil may also benefit SSc patients by improving endothelial function. Whether these findings replicate those from studies investigating the use of sildenafil in vascular disease is not clear. At present the precise impact sildenafil, or similar agents, may have on SSc patients can only be ascertained by clinical trials.

Unverified Answer

What is the average age someone gets scleroderma, systemic?

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The majority of persons (69.1%) with scleroderma, systemic were women. The average age the disease was diagnosed for women was 47.2 years. The average age the disease was diagnosed for men was 50.5 years. There was a significant difference between the average age of diagnosis of scleroderma, systemic in men in comparison to women.

Unverified Answer

What does sildenafil usually treat?

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Sildenafil might be one of the first line drugs used to treat SSc, particularly in patients with pulmonary or cardiovascular disease. This retrospective study suggests that sildenafil has pleiotropic properties: anti-inflammatory, vasorelaxant and anticancer activities; a unique combination of properties with anti-inflammatory and vasodilatory effects. Results from a recent paper might pave the way for a prospective drug trial to test the hypothesis that sildenafil could be used as a therapeutic agent in SSc patients.

Unverified Answer
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