Double-blind, randomised, multi-centre study to evaluate the efficacy and safety of LH-8 cutaneous solution versus placebo in children and adolescents with moderate to severe scalp alopecia areata.

Phase 2/3 study performed in France, Germany, Bulgaria and India in 100 patients.

Methods / trial design:

Randomised, double-blind, vehicle-controlled multicentre trial in parallel groups.

At screening (Visit 0), subjects will discontinue their previous treatment for alopecia areata, if any. Screening period will last up to 28 days. The 24-week treatment phase will include assessment Visits 1 to 3, which will take place at 12-week intervals. At assessment Visit 1, eligible subjects will be randomly assigned in a 2:1 ratio to receive LH-8 cutaneous solution or vehicle (placebo) twice daily for a 24 week treatment period. During the treatment phase the subjects will complete daily their drug diaries. The post-treatment safety and efficacy follow-up phase will include Visit 4 and Visit 5, 12 and 24 weeks after end of treatment, respectively.

Subjects (as applicable) and parents will be instructed to contact the investigator, if an event on scalp (intolerance) occurs during the treatment or post-treatment period. They may be asked to come to the site for an unscheduled visit, in order to perform additional examinations.

Male and female children and adolescents aged 2 to less than 18 years with active alopecia areata involving 25% to 95% of the scalp between 6 months and 3 years in duration.

Diagnosis and main criteria for inclusion: Male and female children and adolescents aged 2 to less than 18 years with active alopecia areata involving 25% to 95% of the scalp between 6 months and 3 years in duration.

Inclusion criteria:

1. Male and female children and adolescents aged 2 to less than 18 years.
2. Active scalp alopecia areata, involving 25% to 95% of the scalp (as measured by SALT score at screening).
3. Duration of hair loss between 6 months and 3 years.
4. Female subjects of childbearing potential (postmenarcheal) must have a negative urine pregnancy test at screening. Females of childbearing potential must either not be sexually active or be using an adequate birth control method throughout the duration of the study.
5. All subjects taking thyroid medication or hormonal therapy must be on a stable dose for 6 months and maintain such throughout the study.
6. Subjects must be willing to maintain the same hair style, including hair dye, throughout the study period.
7. Written informed consent signed by parent(s) or legally authorized representative and assent or consent signed by the subjects, if applicable, according to national regulations prior to any protocol specific procedures.

Exclusion criteria:

1. Hypersensitivity or intolerance to any active IMP substances (onion, citrus, caffeine, theobromine) or excipients (glycerine, betaine or ethanol).
2. Any cause of hair loss other than alopecia areata.
3. Active scalp inflammation except alopecia areata.
4. Nevi, cutaneous or non-cutaneous lesions currently undiagnosed but suspicious for malignancy.
5. Female adolescents who are pregnant or who are nursing or plan pregnancy during the trial period.
6. Use of topical medication (listed in protocol Section 10.7.1) within 2 weeks prior to Visit 1.
7. Use of systemic alopecia areata therapies (e.g. prednisone, cyclosporine, methotrexate), including use of these medications for other indications, and intralesional corticosteroids within 1 month prior to Visit 1.
8. Administration of hydroxychloroquine or finasteride within two months prior to Visit 1.
9. Use of phototherapy, laser therapy or excimer laser therapy on the scalp within three months prior to Visit 1.
10. Use of infliximab within two months, adalimumab within three months, and ustekinumab within four months prior to Visit 1 or use of other TNF inhibitors and biologic agents within one month or five half-lives before Visit 1, whichever is longer.
11. Prior treatment with IMP.
12. Evidence or history of alcohol, medication or drug abuse.
13. History of systemic or cutaneous medical, or psychiatric disease which will put subject at risk or interfere with assessments.
14. Participation in any other clinical trial within 30 days prior to Visit 1.
15. Subject is in a dependent relationship (e.g. relative or family member) with the investigator's or sponsor's staff.
16. Any other condition or circumstance that, in the opinion of the investigator, could compromise the subject's ability to comply with the study protocol.