Phase 3
N=289
Macitentan for the Treatment of Digital Ulcers in Systemic Sclerosis Patients
Systemic Sclerosis · Ulcers
Bottom Line
View on ClinicalTrials.gov: NCT01474109 ↗Enrolled (actual)
289
Serious AEs
15.3%
Results posted
Jan 2015
Primary outcome: Primary: Incidence Rate of New Digital Ulcers (DUs) up to Week 16 — 0.9082; 0.9567; 0.8115 number of new DUs/observation days — p=0.706
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- macitentan 3mg (Drug); macitentan 10mg (Drug); placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Actelion
- Primary completion
- Nov 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incidence Rate of New Digital Ulcers (DUs) up to Week 16 |
0.9082; 0.9567; 0.8115 | 0.706 |
| SECONDARY Percentage of Participants Without a New DU Up To Week 16 |
64.1; 63.0; 67.0 | 0.6670 |
| SECONDARY Percentage of Participants With at Least One DU Complication |
14.1; 19.6; 19.1 | 0.3625 |
| SECONDARY Change in Hand Functionality Health Assessment Questionnaire - Disability Index (HAQ-DI) Hand Component From Baseline to Week 16 |
1.3; 1.4; 1.3; 1.2; 1.2; 1.2 | 0.863 |
| SECONDARY Health Assessment Questionnaire - Disability Index (HAQ-DI) Overall Score From Baseline to Week 16 |
1.1; 1.2; 1.1; 1.1; 1.1; 1.1 | 0.456 |
| SECONDARY Change in Hand Functionality - Hand Disability in Systemic Sclerosis - Digital Ulcers (HDISS-DU) Score From Baseline to Week 16 |
3.0; 3.0; 3.0; 2.7; 2.6; 2.7 | 0.464 |
Summary
The DUAL-1 study is designed as a multicenter, double-blind two-period study with an initial fixed 16-week Period 1, followed by a Period 2 of variable duration. All patients completing Period 1 will continue on their original randomized treatment into Period 2, until the last randomized patient has completed Period 1.
Patients will be randomized in a 1:1:1 ratio (macitentan 3mg: macitentan 10mg: placebo).
The primary objective is to demonstrate the effect of macitentan on the reduction of the number of new digital ulcers in patients with systemic sclerosis and ongoing digital ulcers.
Other objectives include:
* the evaluation of the efficacy of macitentan on hand functionality and DU burden at Week 16 in SSc patients with ongoing DU disease.
* the evaluation of the safety and tolerability of macitentan in these patients.
* the evaluation of the efficacy of macitentan on time to first DU complication during the entire treatment period.
Eligibility Criteria
Inclusion Criteria
- Patients ≥ 18 years of age
- Women of childbearing potential must use two reliable methods of contraception
- Diagnosis of SSc according to the classification criteria of the American College of Rheumatology (ACR)
- At least one visible, active ischemic digital ulcers (DU) at baseline
- History of at least one additional recent active ischemic DU
Exclusion Criteria
- DUs due to condition other than SSc
- Symptomatic Pulmonary arterial hypertension (PAH)
- Body mass index (BMI) 1.5 x upper limit of the normal range (ULN)
- Hemoglobin 10 mg/day of prednisone or equivalent).
- Treatment with ERAs within 3 months.
- Systemic antibiotics to treat infected DU(s) within 4 weeks.
Data sourced from ClinicalTrials.gov (NCT01474109). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.