Trial Outcomes & Findings for Intravenous Iloprost in Subjects With Symptomatic Raynaud's Phenomenon Secondary to Systemic Sclerosis (Phase 2) (NCT NCT03867097)
NCT ID: NCT03867097
Last Updated: 2025-05-25
Results Overview
The primary efficacy parameter is the change in the weekly frequency of symptomatic RP attacks from baseline. The baseline weekly frequency of symptomatic RP attacks was defined as the average number of weekly symptomatic RP attacks that occurred during the 10- to 25-day baseline ePRO diary completion period. The double-blind endpoint weekly frequency of symptomatic RP attacks was defined as the average number of weekly symptomatic RP attacks that occurred during Days 8 to 21, inclusive.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
34 participants
Primary outcome timeframe
Day 8 - Day 21 will be compared to baseline
Results posted on
2025-05-25
Participant Flow
The study was initiated on 07 March 2019 and completed on 06 August 2019. It was conducted in 16 sites located in the United States.
In this study 41 participants with Systemic Sclerosis (SSc) were screened.34 participants who had at least 10 symptomatic Raynaud's phenomenon (RP) attacks on the 5-day eligibility period were randomized on to the study: 17 to the placebo arm and 17 to the Iloprost arm.
Participant milestones
| Measure |
Placebo
Subjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line. Study drug will be initiated at a starting dose 0.5 ng/kg/min up to 2.0 ng/kg/min.
Placebo IV infusion: Study drug will be initiated at a starting dose of 0.5 ng/kg/min up to 2.0 ng/kg/min. Subjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line.
|
Iloprost Injection, for Intravenous Use
Subjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line. Study drug will be initiated at a starting dose 0.5 ng/kg/min up to 2.0 ng/kg/min.
Iloprost Injection, for intravenous use: Study drug will be initiated at a starting dose of 0.5 ng/kg/min up to 2.0 ng/kg/min. Subjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line.
|
|---|---|---|
|
Overall Study
STARTED
|
17
|
17
|
|
Overall Study
COMPLETED
|
17
|
17
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Intravenous Iloprost in Subjects With Symptomatic Raynaud's Phenomenon Secondary to Systemic Sclerosis (Phase 2)
Baseline characteristics by cohort
| Measure |
Placebo
n=17 Participants
Subjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line. Study drug will be initiated at a starting dose 0.5 ng/kg/min up to 2.0 ng/kg/min.
Placebo IV infusion: Study drug will be initiated at a starting dose of 0.5 ng/kg/min up to 2.0 ng/kg/min. Subjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line.
|
Iloprost Injection, for Intravenous Use
n=17 Participants
Subjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line. Study drug will be initiated at a starting dose 0.5 ng/kg/min up to 2.0 ng/kg/min.
Iloprost Injection, for intravenous use: Study drug will be initiated at a starting dose of 0.5 ng/kg/min up to 2.0 ng/kg/min. Subjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line.
|
Total
n=34 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
48.9 years
STANDARD_DEVIATION 11.59 • n=5 Participants
|
50.2 years
STANDARD_DEVIATION 13.48 • n=7 Participants
|
49.6 years
STANDARD_DEVIATION 12.40 • n=5 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
32 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
4 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
13 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
29 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
15 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
28 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Phosphodiesterase inhibitor at baseline
Yes
|
7 n (%)
n=5 Participants
|
5 n (%)
n=7 Participants
|
12 n (%)
n=5 Participants
|
|
Phosphodiesterase inhibitor at baseline
No
|
10 n (%)
n=5 Participants
|
12 n (%)
n=7 Participants
|
22 n (%)
n=5 Participants
|
|
Weekly frequency of symptomatic RP attacks at baseline
|
42.32 no. of attacks
STANDARD_DEVIATION 29.635 • n=5 Participants
|
37.05 no. of attacks
STANDARD_DEVIATION 18.643 • n=7 Participants
|
39.69 no. of attacks
STANDARD_DEVIATION 24.525 • n=5 Participants
|
PRIMARY outcome
Timeframe: Day 8 - Day 21 will be compared to baselinePopulation: The modified Intention-to-Treat (mITT) Population was defined as all randomized subjects who initiated infusion of study drug.
The primary efficacy parameter is the change in the weekly frequency of symptomatic RP attacks from baseline. The baseline weekly frequency of symptomatic RP attacks was defined as the average number of weekly symptomatic RP attacks that occurred during the 10- to 25-day baseline ePRO diary completion period. The double-blind endpoint weekly frequency of symptomatic RP attacks was defined as the average number of weekly symptomatic RP attacks that occurred during Days 8 to 21, inclusive.
Outcome measures
| Measure |
Placebo
n=17 Participants
Subjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line. Study drug will be initiated at a starting dose 0.5 ng/kg/min up to 2.0 ng/kg/min.
Placebo IV infusion: Study drug will be initiated at a starting dose of 0.5 ng/kg/min up to 2.0 ng/kg/min. Subjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line.
|
Iloprost Injection, for Intravenous Use
n=17 Participants
Subjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line. Study drug will be initiated at a starting dose 0.5 ng/kg/min up to 2.0 ng/kg/min.
Iloprost Injection, for intravenous use: Study drug will be initiated at a starting dose of 0.5 ng/kg/min up to 2.0 ng/kg/min. Subjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line.
|
|---|---|---|
|
Change in Frequency of Symptomatic RP Attacks
|
-14.32 Number of RP attacks per week
Interval -20.15 to -8.48
|
-15.09 Number of RP attacks per week
Interval -21.14 to -9.04
|
Adverse Events
Placebo
Serious events: 0 serious events
Other events: 14 other events
Deaths: 0 deaths
Iloprost Injection, for Intravenous Use
Serious events: 0 serious events
Other events: 17 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Placebo
n=17 participants at risk
Subjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line. Study drug will be initiated at a starting dose 0.5 ng/kg/min up to 2.0 ng/kg/min.
Placebo IV infusion: Study drug will be initiated at a starting dose of 0.5 ng/kg/min up to 2.0 ng/kg/min. Subjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line.
|
Iloprost Injection, for Intravenous Use
n=17 participants at risk
Subjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line. Study drug will be initiated at a starting dose 0.5 ng/kg/min up to 2.0 ng/kg/min.
Iloprost Injection, for intravenous use: Study drug will be initiated at a starting dose of 0.5 ng/kg/min up to 2.0 ng/kg/min. Subjects will receive study drug for 5 consecutive days as an IV infusion over 6 hours each day via a peripheral line.
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal distension
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Gastrointestinal disorders
Abdominal pain upper
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
0.00%
0/17 • Day 1 to Day 35
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
General disorders
Fatigue
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
11.8%
2/17 • Number of events 2 • Day 1 to Day 35
|
|
General disorders
Infusion site pain
|
0.00%
0/17 • Day 1 to Day 35
|
17.6%
3/17 • Number of events 3 • Day 1 to Day 35
|
|
General disorders
Malaise
|
0.00%
0/17 • Day 1 to Day 35
|
11.8%
2/17 • Number of events 2 • Day 1 to Day 35
|
|
General disorders
Pain
|
0.00%
0/17 • Day 1 to Day 35
|
11.8%
2/17 • Number of events 2 • Day 1 to Day 35
|
|
General disorders
Chest discomfort
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
General disorders
Feeling abnormal
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
General disorders
Feeling hot
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
0.00%
0/17 • Day 1 to Day 35
|
|
General disorders
Infusion site swelling
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
General disorders
Edema peripheral
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
0.00%
0/17 • Day 1 to Day 35
|
|
General disorders
Vessel puncture site pruritus
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
0.00%
0/17 • Day 1 to Day 35
|
|
Musculoskeletal and connective tissue disorders
Pain in jaw
|
0.00%
0/17 • Day 1 to Day 35
|
23.5%
4/17 • Number of events 4 • Day 1 to Day 35
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
11.8%
2/17 • Number of events 2 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/17 • Day 1 to Day 35
|
17.6%
3/17 • Number of events 3 • Day 1 to Day 35
|
|
Nervous system disorders
Headache
|
29.4%
5/17 • Number of events 5 • Day 1 to Day 35
|
94.1%
16/17 • Number of events 16 • Day 1 to Day 35
|
|
Nervous system disorders
Dizziness
|
11.8%
2/17 • Number of events 2 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Nervous system disorders
Dizziness postural
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Nervous system disorders
Memory impairment
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Nervous system disorders
Presyncope
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
0.00%
0/17 • Day 1 to Day 35
|
|
Nervous system disorders
Tension headache
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
0.00%
0/17 • Day 1 to Day 35
|
|
Gastrointestinal disorders
Nausea
|
17.6%
3/17 • Number of events 3 • Day 1 to Day 35
|
64.7%
11/17 • Number of events 11 • Day 1 to Day 35
|
|
Gastrointestinal disorders
Abdominal pain
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
23.5%
4/17 • Number of events 4 • Day 1 to Day 35
|
|
Gastrointestinal disorders
Diarrhea
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
17.6%
3/17 • Number of events 3 • Day 1 to Day 35
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/17 • Day 1 to Day 35
|
11.8%
2/17 • Number of events 2 • Day 1 to Day 35
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/17 • Day 1 to Day 35
|
11.8%
2/17 • Number of events 2 • Day 1 to Day 35
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Musculoskeletal and connective tissue disorders
Joint stiffness
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
0.00%
0/17 • Day 1 to Day 35
|
|
Musculoskeletal and connective tissue disorders
Limb discomfort
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
0.00%
0/17 • Day 1 to Day 35
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Musculoskeletal and connective tissue disorders
Temporomandibular joint syndrome
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Skin and subcutaneous tissue disorders
Skin ulcer
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
0.00%
0/17 • Day 1 to Day 35
|
|
Skin and subcutaneous tissue disorders
Pruritus generalized
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Skin and subcutaneous tissue disorders
Rash macular
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Skin and subcutaneous tissue disorders
Skin exfoliation
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
0.00%
0/17 • Day 1 to Day 35
|
|
Vascular disorders
Flushing
|
0.00%
0/17 • Day 1 to Day 35
|
29.4%
5/17 • Number of events 5 • Day 1 to Day 35
|
|
Infections and infestations
Infected skin ulcer
|
11.8%
2/17 • Number of events 2 • Day 1 to Day 35
|
0.00%
0/17 • Day 1 to Day 35
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Infections and infestations
Urinary tract infection
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
0.00%
0/17 • Day 1 to Day 35
|
|
Infections and infestations
Vulvovaginal mycotic infection
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
0.00%
0/17 • Day 1 to Day 35
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Investigations
Alanine aminotransferase increased
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Cardiac disorders
Palpitations
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Ear and labyrinth disorders
Ear congestion
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
0.00%
0/17 • Day 1 to Day 35
|
|
Eye disorders
Eye irritation
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Eye disorders
Eye pain
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Injury, poisoning and procedural complications
Thermal burn
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/17 • Day 1 to Day 35
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
|
Reproductive system and breast disorders
Breast discharge
|
5.9%
1/17 • Number of events 1 • Day 1 to Day 35
|
0.00%
0/17 • Day 1 to Day 35
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place