E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10047642 |
E.1.2 | Term | Vitiligo |
E.1.2 | System Organ Class | 10040785 - Skin and subcutaneous tissue disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the duration of clinical response of ruxolitinib cream in participants with vitiligo. |
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E.2.2 | Secondary objectives of the trial |
Key secondary:
To evaluate the duration of clinical response of ruxolitinib cream in participants with vitiligo.
Secondary:
To further evaluate the efficacy of ruxolitinib cream.
To determine the participants' quality of life.
To evaluate the safety and tolerability of ruxolitinib cream.
To evaluate the ruxolitinib PK in plasma after treatment with ruxolitinib cream. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Currently enrolled and receiving treatment in INCB 18424-306 or INCB 18424-307 studies evaluating ruxolitinib cream in participants with vitiligo.
2. Currently tolerating ruxolitinib cream in the parent study and no safety concerns per investigators judgment.
3. Has demonstrated compliance, as assessed by the investigator, with the parent study protocol requirements.
4. Willingness and ability to comply with scheduled visits, treatment plans, and any other study procedures indicated in this protocol.
5. Male and female participants must be willing to take appropriate contraceptive measures to avoid pregnancy or fathering a child for the duration of study participation with the exception of the following:
a. Females of non-childbearing potential (ie, or surgically sterile with a hysterectomy and/or bilateral oophorectomy OR postmenopausal, ≥ 12 months of amenorrhea without an alternative medical cause).
b. Prepubescent adolescents (age 12-18 years old at the time enrolled in parent studies).
6. For adult participant, ability to comprehend and willingness to sign an ICF; for adolescent participant, written informed consent of the parent(s) or legal guardian and written assent from the adolescent participant when possible. |
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E.4 | Principal exclusion criteria |
1. Has been permanently discontinued from study treatment in the parent study for any reason.
2. Participants with an uncontrolled intercurrent illness or any concurrent condition that, in the investigator's opinion, would jeopardize the safety of the participant or compliance with the Protocol.
3. Pregnant or breastfeeding woman.
4. Participants who live with anyone participating in any current Incyte-sponsored ruxolitinib cream study. |
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E.5 End points |
E.5.1 | Primary end point(s) |
For participants who are randomized in Cohort A:
• Time to relapse (defined as < F-VASI75). |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
over 52 week treatment period |
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E.5.2 | Secondary end point(s) |
Key secondary:
For participants who are randomized in Cohort A:
• Time to maintain ≥ F-VASI90 response.
Secondary:
• Proportion of participants who achieve F-VASI50/75/90 during the extension treatment period.
• Actual measurements, change, and percentage change from baseline in F-VASI.
• Proportion of participants who achieve T-VASI50/75/90 during the extension treatment period.
• Actual measurements, change, and percentage change from baseline in T-VASI.
• Actual measurements, change, and percentage change from baseline in F-BSA.
• Actual measurements, change, and percentage change from baseline in T-BSA.
• Proportion of participants achieving a VNS of “4 – A lot less noticeable” or “5 – No longer noticeable” during the extension treatment period.
•Change from Week 52 in DLQI (or CDLQI) during the extension treatment period.
• The frequency and severity of AEs; includes performing physical examinations and collecting vital signs and laboratory data for hematology and serum chemistry.
•Trough plasma concentrations of ruxolitinib at Week 80 and Week 104. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
over 52 week treatment period |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | Yes |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Cohort A is double-blind vehicle controlled; Cohort B is open-label |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 50 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Canada |
United States |
Bulgaria |
France |
Germany |
Italy |
Netherlands |
Poland |
Spain |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |