A Phase 3 Randomized, Placebo-controlled, Double-blind Study to Evaluate Efficacy and Safety of Upadacitinib in Pediatric Subjects With Severe Alopecia Areata
Trial Snapshot
- Phase
- Phase 3
- Status
- Recruiting
- Sponsor
- Enrollment
- 300
- Locations
- 34
- Primary Endpoint
- Percentage of Participants with the Achievement of Severity of Alopecia Tool (SALT) Score <= 20, defined as less than or equal to 20% scalp hair loss.
Study Overview
Brief Summary
Alopecia areata (AA) is a disease that happens when the immune system attacks hair follicles and causes hair loss. AA usually affects the scalp and face, but hair loss can happen on any hair-bearing part of the body. Some treatment options are available for adults and adolescents with AA, however there is still high unmet need for systemic treatments (treatment that moves throughout the bloodstream) approved for young patients with AA. Treatments may not work for all patients or may stop working over time. Because of this, researchers are developing new AA treatments, like upadacitinib. Upadacitinib is a type of medicine called a Janus- Kinase (JAK) inhibitor and works with the body to fight the inflammation that can cause AA. In this study, different doses (amounts) of upadacitinib are being compared to treatment with placebo (looks like the study treatment but contains no medicine).
Upadacitinib is an investigational JAK inhibitor being developed for the treatment of severe alopecia areata in pediatric patients. This is a randomized, double-blind, placebo-controlled study. Participants are placed in 3 groups, called treatment arms. Each group receives a different treatment. There is a 1 in 5 chance that participants will be assigned to placebo. Pediatric participants with a diagnosis of severe alopecia areata with SALT score ≥ 50 scalp hair loss will be enrolled. Participants will be at least 6 years old at Screening and less than 18 years old at Baseline. Approximately 300 participants will be enrolled in the study at approximately 120 sites worldwide.
Participants will receive oral doses of upadacitinib or matching placebo daily, or twice daily, for approximately 160 weeks. The study comprises a 35-day Screening Period, a 24-week placebo-controlled double-blinded treatment period (Period A), a 28-week blinded extension treatment period (Period B), a 108-week blinded long-term extension period (Period C), and a 30-day follow-up period.
There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 6 Years to 17 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Participants must have a diagnosis of severe alopecia areata with SALT score >= 50 scalp hair loss at Screening and Baseline
- •No spontaneous scalp hair regrowth over the past 6 months
- •Participants will have current episode of alopecia areata of less than 8 years
Exclusion Criteria
- •Participants must not have a current diagnosis of primarily diffuse type of alopecia areata
- •Participants must not have a diagnosis of other types of alopecia that would interfere with evaluation of alopecia areata, including but not limited to female pattern hair loss, male pattern hair loss (androgenetic alopecia) Stage III or greater, traction alopecia, lichen planopilaris, discoid lupus, frontal fibrosing alopecia, central centrifugal cicatricial alopecia, folliculitis decalvans, trichotillomania, and telogen effluvium
- •Participants must not have a diagnosis of other types of inflammatory scalp, eyebrow, or eyelash disorders that would interfere with evaluation of alopecia areata, including but not limited to seborrheic dermatitis, scalp psoriasis, AD, and tinea capitis
Arms & Interventions
Group 2A: Upadacitinib Dose 2
Participants will receive upadacitinib Dose 2 during Period A (Baseline to Week 24) and continue the same treatment through Period B (Week 24 to Week 52) and Period C (Week 52 to Week 160).
Intervention: Upadacitinib (Drug)
Group 1B: Upadacitinib Dose 1
Participants initially randomized to placebo in Period A with SALT score > 20 at Week 24 will be re-randomized to receive upadacitinib Dose 1 during Period B and continue through Period C.
Intervention: Upadacitinib (Drug)
Group 3A: Matching Placebo
Participants will receive matching placebo during Period A (Baseline to Week 24). Participants achieving SALT score ≤ 20 at Week 24 will remain on placebo until SALT score > 20, then switch to blinded upadacitinib. Participants with SALT score > 20 at Week 24 will be re-randomized to upadacitinib Dose 1 or Dose 2.
Intervention: Placebo (Drug)
Group 1A: Upadacitinib Dose 1
Participants will receive upadacitinib Dose 1 during Period A (Baseline to Week 24) and continue the same treatment through Period B (Week 24 to Week 52) and Period C (Week 52 to Week 160).
Intervention: Upadacitinib (Drug)
Group 2B: Upadacitinib Dose 2
Participants initially randomized to placebo in Period A with SALT score > 20 at Week 24 will be re-randomized to receive upadacitinib Dose 2 during Period B and continue through Period C.
Intervention: Upadacitinib (Drug)
Outcomes
Primary Outcomes
Percentage of Participants with the Achievement of Severity of Alopecia Tool (SALT) Score <= 20, defined as less than or equal to 20% scalp hair loss.
Time Frame: At Week 24
The SALT is a global AA severity score based on the combination of extent and density of scalp hair loss. The score is determined by visually defining the amount of terminal hair loss in each of the 4 views of the scalp (left and right side each accounting for 18% of scalp area, the top for 40%, and the back for 24%) and adding these together with a maximum score of 100%.
Number of Participants with Adverse Events (AEs)
Time Frame: Up to Week 160
An AE is defined as any untoward medical occurrence in a patient or clinical investigation in which a participant is administered a pharmaceutical product which does not necessarily have a causal relationship with this treatment.
Secondary Outcomes
- Percentage of Participants Achieving SALT Score <= 10(At Week 24)
- Percentage of Participants Achieving SALT Score <= 20(At Week 12)
- Percentage of Participants with the Achievement of Clinician-Reported Outcome (ClinRO) Measure for Eyebrow Hair Loss of 0 or 1(At Week 24)
- Percentage of Participants with the Achievement of Clinician-Reported Outcome (ClinRO) Measure for Eyelash Hair Loss of 0 or 1(At Week 24)
- Percentage of Participants Achieving SALT Score 0(AT Week 24)
- Percentage of Participants with the Achievement of Patients' Global Impression of Change of Alopecia Areata (PaGIC-AA) Score of 1 "Much Better" or 2 "Moderately Better" (in participants who are 12 years and older at the time of baseline visit)(At Week 24)
- Percentage of Participants with the Achievement of Patients' Caregiver Global Impression of Change (CaGIC-AA) Score of 1 "Much Better" or 2 "Moderately Better" (In Participants 6-11 Years Old at the time of baseline visit)(At Week 24)
- Percentage of Participants with a Change From Baseline in Hospital Anxiety and Depression Scale (HADS) Score (In Participants 12 Years and Older)(Baseline (Week 0) through Week 24)
- Percentage of Participants with a Change From Baseline in Children's Dermatology Life Quality Index (CDLQI) Score(Baseline (Week 0) through Week 24)
- Percentage of Participants with a Change From Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Parent Proxy Depressive Symptoms Short Form 6a Score (In Participants 6-11 Years Old)(Baseline (Week 0) through Week 24)
- Percentage of Participants with a Change From Baseline in PROMIS Pediatric Depressive Symptoms Short Form 8a Score (In Participants 12 Years and Older)(Baseline (Week 0) through Week 24)
- Percentage of Participants with a Change From Baseline in PROMIS Parent Proxy Anxiety Short Form 8a Score (In Participants 6-11 Years Old)(Baseline (Week 0) through Week 24)
- Percentage of Participants with a Change From Baseline in PROMIS Pediatric Anxiety Short Form 8a Score (In Participants 12 Years and Older)(Baseline (Week 0) through Week 24)