Study of EPI-001 in Patients With Androgenetic Alopecia

Phase I/IIa Study to Evaluate the Safety, Tolerability, and Efficacy of EPI-001 in Patients With Androgenetic Alopecia

This is a Phase I/IIa clinical study to evaluate the safety, tolerability, and preliminary efficacy of EPI-001 in patients with androgenetic alopecia.

In the Phase I portion, a traditional 3+3 dose-escalation design will be used to determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of EPI-001. Subjects will be followed for up to 24 weeks after administration.

In the Phase IIa portion, subjects will be randomized in a 2:1 ratio to receive either EPI-001 or placebo. Safety and efficacy will be evaluated through hair count assessment, hair diameter measurement, clinical photography, investigator assessment, expert panel assessment, and subject self-assessment during a follow-up period of up to 48 weeks.

Androgenetic alopecia (AGA) is one of the most common forms of hair loss in both men and women and is characterized by progressive hair follicle miniaturization associated with androgen sensitivity and genetic predisposition. Current treatment options for AGA are limited and may not provide sufficient therapeutic benefit for all patients.

EPI-001 is an autologous dermal papilla cell therapy intended for the treatment of androgenetic alopecia. This study is designed to evaluate the safety, tolerability, and efficacy of EPI-001 in subjects with androgenetic alopecia.

This study consists of two parts: a Phase I dose-escalation study and a Phase IIa dose-expansion study.

In the Phase I portion, subjects will receive a single administration of EPI-001 using a traditional 3+3 dose-escalation design to evaluate dose-limiting toxicity (DLT), determine the maximum tolerated dose (MTD), and establish the recommended Phase 2 dose (RP2D). Subjects will be followed for up to 24 weeks after administration.

In the Phase IIa portion, eligible subjects will be randomized in a 2:1 ratio to receive either EPI-001 at the RP2D or placebo. The study will evaluate efficacy through changes in total hair count and hair diameter, as well as investigator assessment, expert panel assessment based on clinical photographs, and subject self-assessment questionnaires. Subjects will undergo follow-up assessments for up to 48 weeks after administration.

Inclusion Criteria:

1. Male or female subjects aged 19 years or older
2. Subjects diagnosed with androgenetic alopecia
3. Subjects willing to maintain the same hairstyle, hair length, and hair color during the study period
4. Subjects willing to refrain from prohibited hair-related products or procedures during the study period
5. Subjects willing to undergo scalp tattooing and hair trimming for phototrichogram evaluation
6. Subjects who voluntarily signed written informed consent

Exclusion Criteria:

1. Subjects who used prohibited medications or therapies affecting hair growth within the protocol-defined period
2. Subjects with scalp diseases or hair disorders other than androgenetic alopecia
3. Subjects with autoimmune diseases affecting the scalp or hair
4. Subjects with clinically significant cardiovascular, renal, endocrine, infectious, or systemic diseases
5. Subjects positive for HBV, HCV, HIV, or syphilis screening tests
6. Subjects with a history of hair transplantation, stem cell therapy, or gene therapy
7. Subjects with hypersensitivity related to the investigational product or study procedures
8. Pregnant or breastfeeding women
9. Subjects who participated in another clinical study within the protocol-defined period
10. Subjects judged inappropriate for study participation by the investigator