Back to results"As Required" Oral Sildenafil in Raynaud's Phenomenon (PROFIL)
Primary Purpose
Raynaud's Phenomenon
Status
Completed
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
Sildenafil 40 mg
Sildenafil 80 mg
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Raynaud's Phenomenon focused on measuring Raynaud's phenomenon, Sildenafil, Systemic Sclerosis, Local cooling, Microcirculation
Eligibility Criteria
Inclusion Criteria:
- Active Raynaud's Phenomenon characterized by a clinical history of primary or secondary Raynaud's Phenomenon
- At least 7 RP attacks per week on 5 or more days per week (assessed over the 2 weeks preceding inclusion)
- Patients who have dated and signed the informed consent form
- Contraception for women
Exclusion Criteria:
- Recently diagnosed RP (less than 2 months).
- Uncontrolled hypertension, diabetes mellitus, angina
- Haemodynamic instability
- Nonarteritic ischemic optic neuropathy
- Pulmonary hypertension
- Subjects currently taking sildenafil, tadalafil or vardenafil
- Subjects currently taking nitrates
- Subjects currently taking strong CYP3A inhibitors
- Pregnancy (or considering pregnancy in the next 4 months)
- Breast feeding
- Participation in another clinical trial
Sites / Locations
- Clinical Investigation Center - Pharmacology Unit - University Hospital of Grenoble
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Placebo Comparator
Arm Label
Sildenafil 80 mg
Sildenafil 40 mg
Placebo
Arm Description
Sildenafil 80 mg
Sildenafil 40 mg
placebo
Outcomes
Primary Outcome Measures
Mean change in the Raynaud's Condition Score (RCS) during treatment, as compared to placebo.
RCS is self-reported every day and averaged over 7-day periods
Secondary Outcome Measures
Frequency of RP: number of RP attacks during treatment, as compared to placebo.
An attack is defined as an episode of pallor or cyanosis (with or without pain,tingling or numbness). The number of attacks is self-reported every day and averaged over 7-day periods
Patients' preference: comparison between the number of patients favoring a treatment to another.
Pain associated with RP: Mean change in the Raynaud's pain score during treatment, as compared to placebo.
Pain associated with each attack is self-reported on a 10-point scale, and averaged over 7-day periods
Frequency and severity of adverse drug events during treatment, as compared to placebo
Microvascular reactivity: index of skin perfusion in response to local/regional cooling after a single dose of sildenafil, as compared to placebo (laboratory experiments)
Full Information
NCT ID
NCT02050360
First Posted
January 28, 2014
Last Updated
January 15, 2016
Sponsor
University Hospital, Grenoble
1. Study Identification
Unique Protocol Identification Number
NCT02050360
Brief Title
"As Required" Oral Sildenafil in Raynaud's Phenomenon
Acronym
PROFIL
Official Title
Efficacy and Safety of "as Required" Sildenafil for Patients With Moderate to Severe Raynaud's Phenomenon (RP)
Study Type
Interventional
2. Study Status
Record Verification Date
January 2016
Overall Recruitment Status
Completed
Study Start Date
November 2013 (undefined)
Primary Completion Date
May 2015 (Actual)
Study Completion Date
May 2015 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Hospital, Grenoble
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of this study is to determine whether "on-demand" sildenafil is effective in the treatment of Raynaud's phenomenon.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Raynaud's Phenomenon
Keywords
Raynaud's phenomenon, Sildenafil, Systemic Sclerosis, Local cooling, Microcirculation
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
41 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Sildenafil 80 mg
Arm Type
Experimental
Arm Description
Sildenafil 80 mg
Arm Title
Sildenafil 40 mg
Arm Type
Experimental
Arm Description
Sildenafil 40 mg
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
placebo
Intervention Type
Drug
Intervention Name(s)
Sildenafil 40 mg
Intervention Type
Drug
Intervention Name(s)
Sildenafil 80 mg
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Mean change in the Raynaud's Condition Score (RCS) during treatment, as compared to placebo.
Description
RCS is self-reported every day and averaged over 7-day periods
Time Frame
7 days
Secondary Outcome Measure Information:
Title
Frequency of RP: number of RP attacks during treatment, as compared to placebo.
Description
An attack is defined as an episode of pallor or cyanosis (with or without pain,tingling or numbness). The number of attacks is self-reported every day and averaged over 7-day periods
Time Frame
7 days
Title
Patients' preference: comparison between the number of patients favoring a treatment to another.
Time Frame
Every 3 weeks of treatment (week 3, 6 and 9)
Title
Pain associated with RP: Mean change in the Raynaud's pain score during treatment, as compared to placebo.
Description
Pain associated with each attack is self-reported on a 10-point scale, and averaged over 7-day periods
Time Frame
7 days
Title
Frequency and severity of adverse drug events during treatment, as compared to placebo
Time Frame
7 days
Title
Microvascular reactivity: index of skin perfusion in response to local/regional cooling after a single dose of sildenafil, as compared to placebo (laboratory experiments)
Time Frame
Week 1, 2 and 3
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Active Raynaud's Phenomenon characterized by a clinical history of primary or secondary Raynaud's Phenomenon
At least 7 RP attacks per week on 5 or more days per week (assessed over the 2 weeks preceding inclusion)
Patients who have dated and signed the informed consent form
Contraception for women
Exclusion Criteria:
Recently diagnosed RP (less than 2 months).
Uncontrolled hypertension, diabetes mellitus, angina
Haemodynamic instability
Nonarteritic ischemic optic neuropathy
Pulmonary hypertension
Subjects currently taking sildenafil, tadalafil or vardenafil
Subjects currently taking nitrates
Subjects currently taking strong CYP3A inhibitors
Pregnancy (or considering pregnancy in the next 4 months)
Breast feeding
Participation in another clinical trial
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jean-Luc CRACOWSKI, MD
Organizational Affiliation
INSERM CIC03 - Unité de Pharmacologie Clinique
Official's Role
Principal Investigator
Facility Information:
Facility Name
Clinical Investigation Center - Pharmacology Unit - University Hospital of Grenoble
City
Grenoble
Country
France
12. IPD Sharing Statement
Citations:
PubMed Identifier
30383134
Citation
Roustit M, Giai J, Gaget O, Khouri C, Mouhib M, Lotito A, Blaise S, Seinturier C, Subtil F, Paris A, Cracowski C, Imbert B, Carpentier P, Vohra S, Cracowski JL. On-Demand Sildenafil as a Treatment for Raynaud Phenomenon: A Series of n-of-1 Trials. Ann Intern Med. 2018 Nov 20;169(10):694-703. doi: 10.7326/M18-0517. Epub 2018 Oct 30.
Results Reference
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"As Required" Oral Sildenafil in Raynaud's Phenomenon
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