Trial Outcomes & Findings for A Study to Evaluate the Efficacy of an Oral Medication in the Treatment and Prevention of Digital Ulcers in Patients With Systemic Sclerosis (Scleroderma). (NCT NCT00725361)

NCT ID: NCT00725361

Last Updated: 2017-03-31

Results Overview

The number of new digital ulcers (DU) that have developed in the preceding 4 weeks assessed at week 24 from baseline.

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 20 participants
• Primary outcome timeframe: 4 weeks prior to week 24
• Results posted on: 2017-03-31

Participant Flow

Participant milestones

Measure	Ambrisentan 5 mg orally daily X 4 weeks then 10 mg daily as tolerated
Overall Study STARTED	20
Overall Study COMPLETED	16
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study to Evaluate the Efficacy of an Oral Medication in the Treatment and Prevention of Digital Ulcers in Patients With Systemic Sclerosis (Scleroderma).

Baseline characteristics by cohort

Measure	Active n=20 Participants Ambrisentan Ambrisentan
Age, Continuous	49.3 years STANDARD_DEVIATION 13.8 • n=5 Participants
Sex: Female, Male Female	16 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Region of Enrollment United States	20 participants n=5 Participants
Mean number of total baseline digital ulcers	3.1 digital ulcers STANDARD_DEVIATION 2.1 • n=5 Participants
Mean diameter of baseline digital ulcers	3.3 mm STANDARD_DEVIATION 1.6 • n=5 Participants

PRIMARY outcome

Timeframe: 4 weeks prior to week 24

The number of new digital ulcers (DU) that have developed in the preceding 4 weeks assessed at week 24 from baseline.

Outcome measures

Measure	Active n=16 Participants Ambrisentan Ambrisentan
New Digital Ulcers (DU) 4 Weeks Prior to Week 24	0.06 new digital ulcers Standard Deviation 0.68

SECONDARY outcome

Timeframe: 4 weeks prior to week 12

Population: At 12 weeks only 1 patient had withdrawn from the study and 19 remained in the study.

The number of new DU that have developed in the preceding 4 weeks assessed at week 12 from baseline.

Outcome measures

Measure	Active n=19 Participants Ambrisentan Ambrisentan
New DU 4 Weeks Prior to Week 12	-0.26 new digital ulcers Standard Deviation 1.1

SECONDARY outcome

Timeframe: 12 weeks

Population: At 12 weeks only 1 patient had withdrawn from the study and 19 remained in the study.

Outcome measures

Measure	Active n=19 Participants Ambrisentan Ambrisentan
Subjects Experiencing Complete (Total Reepithelialization) Healing of All Baseline DU at Week 12.	7 Participants

SECONDARY outcome

Timeframe: 12 weeks

Population: At 12 weeks only 1 patient had withdrawn from the study and 19 remained in the study.

Outcome measures

Measure	Active n=19 Participants Ambrisentan Ambrisentan
Subjects Experiencing Complete Healing of > 50% of the Number of Baseline DU at Week 12.	12 Participants

SECONDARY outcome

Timeframe: 24 weeks

Range is 0-3 with 0 meaning the least amount of disability and 3 the greatest of disability.

Outcome measures

Measure	Active n=16 Participants Ambrisentan Ambrisentan
Change in the Scleroderma Health Assessment Questionnaire (SHAQ) at Week 24 Compared With Baseline.	-0.06 units on a scale Standard Deviation 0.62

Adverse Events

Active

Serious events: 3 serious events

Other events: 15 other events

Deaths: 0 deaths

Serious adverse events

Measure	Active n=20 participants at risk Ambrisentan Ambrisentan
Cardiac disorders Acute myocardial infarction	5.0% 1/20 • Number of events 1 • Screening through week 28 for a total of 32 weeks of data collection.
Respiratory, thoracic and mediastinal disorders Shortness of breath	5.0% 1/20 • Number of events 1 • Screening through week 28 for a total of 32 weeks of data collection.
Vascular disorders Raynaud attack	5.0% 1/20 • Number of events 1 • Screening through week 28 for a total of 32 weeks of data collection.

Other adverse events

Measure	Active n=20 participants at risk Ambrisentan Ambrisentan
Blood and lymphatic system disorders Edema	75.0% 15/20 • Screening through week 28 for a total of 32 weeks of data collection.
Blood and lymphatic system disorders Anemia	50.0% 10/20 • Screening through week 28 for a total of 32 weeks of data collection.
Infections and infestations Infected digital ulcer	40.0% 8/20 • Screening through week 28 for a total of 32 weeks of data collection.

Additional Information

Lorinda Chung

StanfordU

Phone: 650-736-0727

Email: shauwei@stanford.edu

Results disclosure agreements

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