Trial Outcomes & Findings for Effect of Sildenafil on the Microcirculatory Blood Flow and Endothelial Progenitor Cells in Systemic Sclerosis (NCT NCT01347008)

NCT ID: NCT01347008

Last Updated: 2016-12-05

Results Overview

Finger blood flow of the four medial fingers, measured by laser Doppler imaging and expressed in arbitrary perfusion units (p.u.).

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 41 participants
• Primary outcome timeframe: 8 weeks
• Results posted on: 2016-12-05

Participant Flow

Patients recruited at the Outpatient Clinic of The Federal University of Sao Paulo, between August 2011 and November 2012

Participant milestones

Measure	Sildenafil Citrate Oral Sildenafil citrate, 50mg, b.i.d. Sildenafil citrate: Oral sildenafil citratre, 50mg b.i.d., 8 weeks	Sugar Pill Placebo: Placebo pills similar to sildenafil citrate pills, b.i.d for 8 weeks
Overall Study STARTED	21	20
Overall Study COMPLETED	21	20
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Effect of Sildenafil on the Microcirculatory Blood Flow and Endothelial Progenitor Cells in Systemic Sclerosis

Baseline characteristics by cohort

Measure	Sildenafil Citrate n=21 Participants Oral Sildenafil citrate, 50mg, b.i.d. Sildenafil citrate: Oral sildenafil citratre, 50mg b.i.d., 8 weeks	Sugar Pill n=20 Participants Placebo: Placebo pills similar to sildenafil citrate pills, b.i.d for 8 weeks	Total n=41 Participants Total of all reporting groups
Age, Continuous	47.2 years STANDARD_DEVIATION 10.9 • n=5 Participants	41.6 years STANDARD_DEVIATION 13.3 • n=7 Participants	44.5 years STANDARD_DEVIATION 12.3 • n=5 Participants
Sex: Female, Male Female	21 Participants n=5 Participants	20 Participants n=7 Participants	41 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment Brazil	21 participants n=5 Participants	20 participants n=7 Participants	41 participants n=5 Participants
Digital skin microvascular blood flow measured by Laser Doppler Imaging (LDI) before cold stimulus	213.1 perfusion units STANDARD_DEVIATION 87.9 • n=5 Participants	241.9 perfusion units STANDARD_DEVIATION 104.4 • n=7 Participants	234.6 perfusion units STANDARD_DEVIATION 105.8 • n=5 Participants
Digital skin microvascular blood flow measured by Laser Doppler Imaging (LDI) after cold stimulus.	178.8 perfusion units STANDARD_DEVIATION 92.4 • n=5 Participants	218.6 perfusion units STANDARD_DEVIATION 98.9 • n=7 Participants	200.2 perfusion units STANDARD_DEVIATION 96.1 • n=5 Participants
Daily frequency of Raynuad's Phenomenon attacks	2.3 number of attacks per day STANDARD_DEVIATION 2.5 • n=5 Participants	1.9 number of attacks per day STANDARD_DEVIATION 2.0 • n=7 Participants	2.1 number of attacks per day STANDARD_DEVIATION 2.3 • n=5 Participants

PRIMARY outcome

Timeframe: 8 weeks

Finger blood flow of the four medial fingers, measured by laser Doppler imaging and expressed in arbitrary perfusion units (p.u.).

Outcome measures

Measure	Sildenafil Citrate n=21 Participants Oral Sildenafil citrate, 50mg, b.i.d. Sildenafil citrate: Oral sildenafil citratre, 50mg b.i.d., 8 weeks	Sugar Pill n=20 Participants Placebo: Placebo pills similar to sildenafil citrate pills, b.i.d for 8 weeks
Digital Skin Microvascular Blood Flow Measured by Laser Doppler Imaging (LDI) Before Cold Stimulus	260.0 perfusion units Standard Deviation 108.0	246.3 perfusion units Standard Deviation 122.6

PRIMARY outcome

Timeframe: 8 weeks

Outcome measures

Measure	Sildenafil Citrate n=21 Participants Oral Sildenafil citrate, 50mg, b.i.d. Sildenafil citrate: Oral sildenafil citratre, 50mg b.i.d., 8 weeks	Sugar Pill n=20 Participants Placebo: Placebo pills similar to sildenafil citrate pills, b.i.d for 8 weeks
Digital Skin Microvascular Blood Flow Measured by Laser Doppler Imaging (LDI) After Cold Stimulus.	257.7 perfusion units Standard Deviation 123.3	220.5 perfusion units Standard Deviation 119.9

SECONDARY outcome

Timeframe: 8 weeks

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.

Outcome measures

Measure	Sildenafil Citrate n=21 Participants Oral Sildenafil citrate, 50mg, b.i.d. Sildenafil citrate: Oral sildenafil citratre, 50mg b.i.d., 8 weeks	Sugar Pill n=20 Participants Placebo: Placebo pills similar to sildenafil citrate pills, b.i.d for 8 weeks
Daily Frequency of Raynaud's Phenomenon Attacks	1.9 number of attacks per day Standard Deviation 2.4	1.7 number of attacks per day Standard Deviation 2.2

Adverse Events

Sildenafil Citrate

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Sugar Pill

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Sildenafil Citrate n=21 participants at risk Oral Sildenafil citrate, 50mg, b.i.d. Sildenafil citrate: Oral sildenafil citratre, 50mg b.i.d., 8 weeks	Sugar Pill n=20 participants at risk Placebo: Placebo pills similar to sildenafil citrate pills, b.i.d for 8 weeks
Nervous system disorders Headache	33.3% 7/21 • 10 weeks	5.0% 1/20 • 10 weeks

Additional Information

Fernando Villela Andrigueti, MD

Federal University of Sao Paulo

Phone: +55 11 55764239

Email: fernando.andrigueti@usp.br

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place