Trial Outcomes & Findings for A Study to Evaluate RIST4721 in Palmoplantar Pustulosis (PPP) (NCT NCT03988335)
NCT ID: NCT03988335
Last Updated: 2022-04-05
Results Overview
Relative change from baseline in fresh pustule count at Day 28
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
35 participants
Primary outcome timeframe
Baseline to Day 28
Results posted on
2022-04-05
Participant Flow
Subjects must have at least a 6-month history of PPP and have moderate or severe PPP.
Participant milestones
| Measure |
RIST4721
Subjects were randomized to receive RIST4721 300 mg oral solution once daily for 28 days.
|
Placebo
Subjects were randomized to receive Placebo oral solution once daily for 28 days.
|
|---|---|---|
|
Overall Study
STARTED
|
16
|
19
|
|
Overall Study
COMPLETED
|
14
|
18
|
|
Overall Study
NOT COMPLETED
|
2
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study to Evaluate RIST4721 in Palmoplantar Pustulosis (PPP)
Baseline characteristics by cohort
| Measure |
RIST4721
n=16 Participants
This arm was randomized to receive 300 mg of RIST4721 once daily for 28 days.
|
Placebo
n=19 Participants
This arm was randomized to receive placebo once daily for 28 days.
|
Total
n=35 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
15 Participants
n=93 Participants
|
19 Participants
n=4 Participants
|
34 Participants
n=27 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=93 Participants
|
16 Participants
n=4 Participants
|
28 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=93 Participants
|
3 Participants
n=4 Participants
|
7 Participants
n=27 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Race (NIH/OMB)
White
|
16 Participants
n=93 Participants
|
17 Participants
n=4 Participants
|
33 Participants
n=27 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Region of Enrollment
Canada
|
11 participants
n=93 Participants
|
12 participants
n=4 Participants
|
23 participants
n=27 Participants
|
|
Region of Enrollment
Germany
|
5 participants
n=93 Participants
|
7 participants
n=4 Participants
|
12 participants
n=27 Participants
|
PRIMARY outcome
Timeframe: Baseline to Day 28Relative change from baseline in fresh pustule count at Day 28
Outcome measures
| Measure |
RIST4721 300 mg
n=14 Participants
RIST4721 300 mg (mITT Analysis Set)
|
Placebo
n=18 Participants
Placebo (mITT Analysis Set)
|
|---|---|---|
|
Log Transformed Ratio of Postbaseline Fresh Pustule Count at Day 28 to Baseline
|
0.86 Log transformed ratio
Standard Deviation 0.692
|
0.53 Log transformed ratio
Standard Deviation 0.561
|
PRIMARY outcome
Timeframe: Baseline to Day 28Relative change from baseline in total pustule count at Day 28
Outcome measures
| Measure |
RIST4721 300 mg
n=14 Participants
RIST4721 300 mg (mITT Analysis Set)
|
Placebo
n=18 Participants
Placebo (mITT Analysis Set)
|
|---|---|---|
|
Log Transformed Ratio of Postbaseline Total Pustule Count at Day 28 to Baseline
|
0.99 Log transformed ratio
Standard Deviation 0.667
|
0.96 Log transformed ratio
Standard Deviation 0.672
|
Adverse Events
RIST4721 300 mg
Serious events: 0 serious events
Other events: 13 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
RIST4721 300 mg
n=15 participants at risk
All subjects who received at least one dose of RIST4721 300 mg (Safety Analysis Set).
|
Placebo
n=19 participants at risk
All subjects who received at least one dose of Placebo (Safety Analysis Set).
|
|---|---|---|
|
General disorders
Fatigue
|
6.7%
1/15 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Psychiatric disorders
Anxiety
|
6.7%
1/15 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Reproductive system and breast disorders
Vaginal haemorrhage
|
0.00%
0/15 • From ICF signature to end of study participation (approximately 73 days)
|
5.3%
1/19 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
|
Injury, poisoning and procedural complications
Procedural pain
|
13.3%
2/15 • Number of events 2 • From ICF signature to end of study participation (approximately 73 days)
|
5.3%
1/19 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
0.00%
0/15 • From ICF signature to end of study participation (approximately 73 days)
|
5.3%
1/19 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
|
Injury, poisoning and procedural complications
Concussion
|
6.7%
1/15 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Investigations
Bacterial test
|
6.7%
1/15 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Investigations
Blood glucose increased
|
6.7%
1/15 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Investigations
Blood triglycerides increased
|
6.7%
1/15 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Investigations
Hepatic enzyme increased
|
6.7%
1/15 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Blood and lymphatic system disorders
Neutropenia
|
13.3%
2/15 • Number of events 2 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Blood and lymphatic system disorders
Leukopenia
|
6.7%
1/15 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.7%
1/15 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Nervous system disorders
Dizziness
|
0.00%
0/15 • From ICF signature to end of study participation (approximately 73 days)
|
10.5%
2/19 • Number of events 2 • From ICF signature to end of study participation (approximately 73 days)
|
|
Nervous system disorders
Headache
|
6.7%
1/15 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
5.3%
1/19 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
|
Gastrointestinal disorders
Diarrhoea
|
33.3%
5/15 • Number of events 6 • From ICF signature to end of study participation (approximately 73 days)
|
5.3%
1/19 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
|
Gastrointestinal disorders
Abnormal faeces
|
13.3%
2/15 • Number of events 2 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Gastrointestinal disorders
Nausea
|
13.3%
2/15 • Number of events 2 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Gastrointestinal disorders
Abdominal pain
|
6.7%
1/15 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Renal and urinary disorders
Urine odour abnormal
|
20.0%
3/15 • Number of events 3 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Renal and urinary disorders
Micturition disorder
|
0.00%
0/15 • From ICF signature to end of study participation (approximately 73 days)
|
5.3%
1/19 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
|
Skin and subcutaneous tissue disorders
Skin fissures
|
6.7%
1/15 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
13.3%
2/15 • Number of events 2 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
6.7%
1/15 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
5.3%
1/19 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/15 • From ICF signature to end of study participation (approximately 73 days)
|
5.3%
1/19 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
|
Musculoskeletal and connective tissue disorders
Osteoarthritis
|
6.7%
1/15 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/15 • From ICF signature to end of study participation (approximately 73 days)
|
5.3%
1/19 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
|
Metabolism and nutrition disorders
Decreased appetite
|
6.7%
1/15 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Metabolism and nutrition disorders
Increased appetite
|
6.7%
1/15 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Infections and infestations
Folliculitis
|
13.3%
2/15 • Number of events 3 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/15 • From ICF signature to end of study participation (approximately 73 days)
|
5.3%
1/19 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
|
Infections and infestations
Post procedural cellulitis
|
6.7%
1/15 • Number of events 1 • From ICF signature to end of study participation (approximately 73 days)
|
0.00%
0/19 • From ICF signature to end of study participation (approximately 73 days)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place