Non-Segmental Vitiligo
Conditions
Brief summary
The purpose of this clinical trial is to learn how well ACT-777991 works, how safe it is and how well it is tolerated by adults with non-segmental vitiligo. The main question this clinical trial aims to answer is: • Can ACT-777991 help return color to the skin of the face of adults with non-segmental vitiligo? Researchers will compare ACT-777991 to placebo (a look-alike inactive treatment that contains no medicine) to see if ACT-777991 works to treat non-segmental vitiligo. Trial participants will: * Take the trial intervention (either ACT-777991 or placebo) daily for 24 weeks. * Visit the clinic 7 times for check-up and tests.
Detailed description
The trial includes three trial periods: Following a Screening period, during which it will be checked if participants are eligible to take part, eligible participants will be randomized in a 2:1 ratio to receive either ACT-777991 or placebo for 24 weeks (Trial intervention period). On completion of treatment, participants will be followed for 30 (+7) days (Follow-up period). Trial participation will end with a Follow-up visit (Participant Last Visit) at the end of the Follow-up period. The maximum trial duration for an individual participant is approximately 33 weeks including a screening period of up to 28 days, a treatment period of 24 weeks, and a follow-up period of up to 37 days.
Interventions
DRUGACT-777991
ACT-777991 tablets
DRUGPlacebo
ACT-777991-matching placebo tablets
Sponsors
Idorsia Pharmaceuticals Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* Clinical diagnosis of either active or stable non segmental vitiligo for at least 3 months prior to Screening and meet all the following criteria: * F-VASI score ≥ 0.3 based on BICR at Screening. * T-VASI score ≥ 5 based on investigator assessment at Screening and Randomization. * Total body surface area (BSA) involvement, including the face, ≤ 50% based on investigator assessment at Screening and Randomization. * Participants must agree not to use therapeutic agents and procedures to treat vitiligo from Screening until Participant Last Visit.
Exclusion criteria
* Clinical diagnosis of other forms of vitiligo (e.g., segmental) or other hypo- or depigmentation disorders (e.g., piebaldism, leukoderma, Vogt-Koyanagi-Harada disease, malignancy-induced hypopigmentation). * Any autoimmune disease, except adequately treated thyroid disease. * History of systemic immunotherapy treatment, including JAK inhibitors, for any inflammatory disease in the 12 months prior to Randomization. * History of topical JAK inhibitors for any inflammatory disease in the 6 weeks prior to Screening. * Use of laser or light-based treatment (phototherapy), including tanning beds, in the 8 weeks prior to Screening. * eGFR \< 90 mL/min/1.73 m2, defined by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation, at Screening.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Main primary outcome measure: Percentage change from baseline in Facial Vitiligo Area Scoring Index (F-VASI) based on Blinded Independent Central Reading (BICR) at Week 24 | Baseline; Week 24 | The vitiligo area scoring index (VASI) is a validated clinician-reported outcome measure that scores both the extent (surface area) and degree (level of depigmentation) of vitiligo lesions over time. The F-VASI describes involvement of the face, with higher scores indicating more severe disease. Negative changes from baseline indicate improvement. |
| Supplementary primary outcome measure: Percentage change from baseline in F-VASI based on investigator assessment at Week 24 | Baseline; Week 24 | — |
| Supplementary primary outcome measure: Percentage change from baseline in F-VASI at Week 4, 8 and 16 | Baseline; Week 4, Week 8; Week 16 | F-VASI will be assessed by the investigator and by BICR. |
| Supplementary primary outcome measure: Achievement of F-VASI50 at Week 4, 8, 16 and 24 | Baseline; Week 4; Week 8; Week 16; Week 24 | Proportion of patients achieving at least a 50% improvement from baseline in F-VASI. |
| Supplementary primary outcome measure: Achievement of F-VASI75 at Week 4, 8, 16 and 24 | Baseline; Week 4; Week 8; Week 16; Week 24 | Proportion of patients achieving at least a 75% improvement from baseline in F-VASI. |
| Supplementary primary outcome measure: Achievement of F-VASI90 at Week 4, 8, 16 and 24 | Baseline; Week 4; Week 8; Week 16; Week 24 | Proportion of patients achieving at least a 90% improvement from baseline in F-VASI. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage change from baseline in Total Body Vitiligo Area Scoring Index (T-VASI) at Week 4, 8, 16 and 24 | Baseline; Week 24 | The T-VASI is calculated using a formula that includes contributions from all body regions, with higher scores indicating more severe disease. The F-VASI is used as the score for the 'face' component , i.e., the face is not be scored again. Negative changes from baseline indicate improvement. T-VASI will be assessed by the investigator. |
| Achievement of T-VASI50 at Week 4, 8, 16 and 24 | Baseline; Week 4; Week 8; Week 16; Week 24 | Proportion of patients achieving at least a 50% improvement from baseline in T-VASI. |
| Adverse events (AEs) leading to premature discontinuation of trial intervention | From start of trial intervention to last dose of trial intervention, assessed up to Week 24 | — |
| Treatment-emergent AEs and serious AEs (SAEs) | From start of trial intervention up to 37 days after last dose of trial intervention (Follow-up visit) | Treatment-emergent events are AEs and SAEs reported for the first time or as worsening of a pre-existing event after first dose of trial intervention up to 37 days after last dose of trial intervention (Follow-up visit). |
| Treatment-emergent AEs of special interest (AESI) | From start of trial intervention up to 37 days after last dose of trial intervention (Follow-up visit) | — |
| Change from baseline in vital signs: systolic and diastolic blood pressure | Baseline to all pre-defined time points, up to 37 days after last dose of trial intervention (Follow-up visit) | — |
| Change from baseline in vital signs: pulse rate | Baseline to all pre-defined time points, up to 37 days after last dose of trial intervention (Follow-up visit) | — |
| Change from baseline in hematology variables | Baseline to all pre-defined time points, up to 37 days after last dose of trial intervention (Follow-up visit) | The concentration of hematology variables will be measured and the change from baseline summarized. |
| Change from baseline in blood chemistry variables | Baseline to all pre-defined time points, up to 37 days after last dose of trial intervention (Follow-up visit) | The concentration of blood chemistry variables will be measured and the change from baseline summarized. |
| Change from baseline in ECG parameters: PR interval, QRS duration, QTcF Value | Baseline to all pre-defined time points, up to 37 days after last dose of trial intervention (Follow-up visit) | — |
| Change from baseline in ECG parameters: Heart rate | Baseline to all pre-defined time points, up to 37 days after last dose of trial intervention (Follow-up visit) | — |
| Number of Participants with treatment-emergent marked abnormalities in vital signs: systolic and diastolic blood pressure, pulse rate | From start of trial intervention up to 37 days after last dose of trial intervention (Follow-up visit) | — |
| Number of Participants with treatment-emergent marked abnormalities in clinical laboratory variables: hematology and chemistry | From start of trial intervention up to 37 days after last dose of trial intervention (Follow-up visit) | — |
| Number of Participants with treatment-emergent marked abnormalities in ECG parameters: PR interval, QRS duration, QTcF Value, Heart rate | From start of trial intervention up to 37 days after last dose of trial intervention (Follow-up visit) | — |
Contacts
CONTACTClinical Trial Information USA
CONTACTClinical Trial Information Europe
STUDY_DIRECTORClinical Trials
Idorsia Pharmaceuticals Ltd.
Outcome results
None listed