A Study to Evaluate the Safety and Effectiveness of Upadacitinib in Pediatric Participants With Alopecia Areata

AbbVie

Status and phase: Enrolling; Phase 3

Conditions: Severe Alopecia Areata

Treatments: Drug: Placebo; Drug: Upadacitinib

Study type: Interventional

Funder types: Industry

Identifiers

NCT07772492

2025-525048-16-00 (EU Trial (CTIS) Number)

2025

M24-725

Details and patient eligibility

About

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.

Enrollment: 300 estimated patients

Sex: All

Ages: 6 to 17 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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300 participants in 5 patient groups, including a placebo group

Group 1A: Upadacitinib Dose 1

Experimental group

Description:

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).

Treatment:

Drug: Upadacitinib

Group 2A: Upadacitinib Dose 2

Experimental group

Description:

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).

Treatment:

Drug: Upadacitinib

Group 3A: Matching Placebo

Placebo Comparator group

Description:

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.

Treatment:

Drug: Placebo

Group 1B: Upadacitinib Dose 1

Experimental group

Description:

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.

Treatment:

Drug: Upadacitinib

Group 2B: Upadacitinib Dose 2

Experimental group

Description:

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.

Treatment:

Drug: Upadacitinib