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.

Study Overview

• Status: Recruiting
• Conditions: Androgenetic Alopecia
• Intervention / Treatment: Biological: EPI-001; Other: Placebo

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.

• Study Type: Interventional
• Enrollment (Estimated): 42
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Epibiotech Clinical Operations; Phone Number: +82-70-4209-0556; Email: info@epibiotech.com

Study Locations

• South Korea
  • Seoul
    • Seoul, Seoul, South Korea, 06973
      • Recruiting
      • Chung-Ang University Hospital
      • Contact: Chung-Ang University Hospital; Phone Number: +82-1800-1114
      • Principal Investigator: Beom Joon Kim, MD, PhD.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Other: Placebo; Placebo control administered by subcutaneous injection to the scalp.
Experimental: EPI-001	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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of Dose-Limiting Toxicities	Up to 4 weeks after administration

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Adverse Events and Local Adverse Events		Phase I: up to 24 weeks; Phase IIa: up to 48 weeks after administration
Number of Participants With Clinically Significant Abnormal Vital Signs	Clinically significant abnormal vital signs include abnormalities in systolic blood pressure, diastolic blood pressure, pulse rate, and body temperature.	Phase I: up to 24 weeks; Phase IIa: up to 48 weeks after administration
Number of Participants With Clinically Significant Laboratory Abnormalities		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	Investigators assess hair growth improvement by comparing clinical photographs with baseline photographs using a 7-point scale ranging from -3 (greatly decreased) to +3 (greatly increased). Higher scores indicate greater improvement.	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	Participants assess overall hair growth improvement using a questionnaire evaluating whether hair became fuller, reduction in hair shedding, increase in hair thickness, improvement in hair gloss and elasticity, improvement in overall scalp appearance, and increase in hair growth rate. Each item is scored on a 7-point scale ranging from -3 (greatly worsened) to +3 (greatly improved). Higher scores indicate greater perceived improvement.	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	Total hair count is assessed as the number of hairs identified within the target assessment area using phototrichogram imaging. Changes from baseline in total hair count within the target assessment area are evaluated.	Phase I: Week 24; Phase IIa: Weeks 12, 24, and 48
Change From Baseline in Mean Hair Diameter Assessed by Phototrichogram	Mean hair diameter is assessed by measuring hair shaft thickness within the target assessment area using phototrichogram imaging. Changes from baseline in mean hair diameter are evaluated.	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	Expert panel members assess hair growth improvement by comparing clinical photographs with baseline photographs using a 7-point scale ranging from -3 (greatly decreased) to +3 (greatly increased). Higher scores indicate greater improvement.	Phase IIa: Weeks 12, 24, 36, and 48

Collaborators and Investigators

• Sponsor: Epibiotech

Study record dates

Study Major Dates

• Study Start (Actual): January 12, 2026
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): June 1, 2029

Study Registration Dates

• First Submitted: May 20, 2026
• First Submitted That Met QC Criteria: May 26, 2026
• First Posted (Actual): June 1, 2026

Study Record Updates

• Last Update Posted (Actual): June 1, 2026
• Last Update Submitted That Met QC Criteria: May 26, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Alopecia; Cell therapy; Hair Loss; Autologous Cell Therapy; Hair Regeneration; Dermal Papilla Cell
• Additional Relevant MeSH Terms: Pathological Conditions, Anatomical; Skin Diseases; Hypotrichosis; Hair Diseases; Pathological Conditions, Signs and Symptoms; Skin and Connective Tissue Diseases; Alopecia; EPI 001
• Other Study ID Numbers: EPI-001-A101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Individual participant data (IPD) collected during the study will not be made publicly available due to the proprietary nature of the investigational product and ongoing clinical development.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No