Study of EPI-001 in Patients With Androgenetic Alopecia

Official title

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

Overview

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.

Detailed description

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.

Interventions

• Biological EPI-001
  EPI-001 is an autologous dermal papilla cell-based investigational product administered by subcutaneous injection to the scalp for the treatment of androgenetic alopecia.
• Other Placebo
  Placebo control administered by subcutaneous injection to the scalp.

Primary outcome measures

• Incidence of Dose-Limiting Toxicities [Time frame: Up to 4 weeks after administration]

Secondary outcome measures (8)

• Incidence of Adverse Events and Local Adverse Events [Time frame: Phase I: up to 24 weeks; Phase IIa: up to 48 weeks after administration]
• Number of Participants With Clinically Significant Abnormal Vital Signs [Time frame: Phase I: up to 24 weeks; Phase IIa: up to 48 weeks after administration]
• Number of Participants With Clinically Significant Laboratory Abnormalities [Time frame: Phase I: up to 24 weeks; Phase IIa: up to 48 weeks after administration]
• Investigator Assessment of Hair Growth Improvement Based on Clinical Photographs Using a 7-Point Scale [Time frame: Phase I: Weeks 4, 12, and 24; Phase IIa: Weeks 12, 24, 36, and 48]
• Subject Self-Assessment of Hair Growth Improvement Using a 7-Point Questionnaire Scale [Time frame: Phase I: Weeks 4, 12, and 24; Phase IIa: Weeks 12, 24, 36, and 48]
• Change From Baseline in Total Hair Count Assessed by Phototrichogram [Time frame: Phase I: Week 24; Phase IIa: Weeks 12, 24, and 48]
• Change From Baseline in Mean Hair Diameter Assessed by Phototrichogram [Time frame: Phase I: Week 24; Phase IIa: Weeks 12, 24, and 48]
• Expert Panel Assessment of Hair Growth Improvement Based on Clinical Photographs Using a 7-Point Scale [Time frame: Phase IIa: Weeks 12, 24, 36, and 48]

Eligibility criteria

Inclusion criteria

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

Exclusion criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Double blind

Primary purpose

Treatment

Study locations

South Korea · 1 center

• Chung-Ang University Hospital — Seoul

Identifiers

NCT: NCT07618195 · EPI-001-A101

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗