Phase 2 EI-001 for non-segmental vitiligo

A Phase 2, Randomized, Double-Blind, Placebo-controlled Proof-of-Concept Study to Evaluate the Efficacy and Safety of EI-001 in Patients With Nonsegmental Vitiligo

This trial will test whether the antibody drug EI-001 can help adults (18–65) with non‑segmental vitiligo regain skin pigment more than a placebo.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 45 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Elixiron Immunotherapeutics (Hong Kong) Ltd. (Industry-sponsored)
• Locations: 3 sites (Hollywood, Florida and 2 other locations)
• Trial ID: NCT07223229 on ClinicalTrials.gov

What this trial studies

This Phase 2 interventional trial gives adults with non‑segmental vitiligo either EI-001 or placebo and follows them for skin repigmentation and safety signals. EI-001 is a novel immune‑modulating antibody with preclinical data and a completed Phase 1 in healthy volunteers showing tolerability. Eligible participants must have 5–60% total body surface area affected, meet facial and VASI thresholds, and have had inadequate response or intolerance to standard topical, oral or phototherapy options. Participants must stop other vitiligo treatments during the study and complete zoster vaccination if not previously immunized.

Who should consider this trial

Good fit: Adults 18–65 with clinically diagnosed non‑segmental vitiligo affecting 5–60% total BSA (and meeting facial BSA/VASI thresholds) who have had inadequate response to or cannot use topical/oral therapies or phototherapy, and who can stop other vitiligo treatments and complete zoster vaccination if needed.

Not a fit: Patients with segmental vitiligo, very limited disease (<5% total BSA) or very extensive involvement (>60% BSA), those outside the 18–65 age range, or those who cannot discontinue other vitiligo treatments may not benefit or may not qualify for this trial.

Why it matters

Potential benefit: If successful, EI-001 could offer an additional treatment option that increases repigmentation and lowers disease activity for patients who have not responded to current local or phototherapy treatments.

How similar studies have performed: Immune‑targeting approaches including JAK inhibitors and some biologics have produced repigmentation in other vitiligo studies, but EI-001 itself is a novel antibody with only preclinical and Phase 1 tolerability data so far.

Eligibility criteria

Inclusion Criteria:

* Able to understand and voluntarily sign the informed consent form (ICF).
* Male or female, aged 18-65 years at the time of consent.
* BMI 18-38 kg/m² and weight ≥ 40 kg at consent. Clinically diagnosed non-segmental vitiligo (NSV) for at least 3 months, and meets one of the following:
* Inadequate response to approved treatments:
* Topical therapy (e.g., corticosteroids, calcineurin inhibitors, or JAK inhibitors) ≥ 3 months,
* Phototherapy ≥ 6 months, or Oral therapy (e.g., corticosteroids, calcineurin inhibitors) ≥ 3 months.
* Or unable to use these treatments due to contraindications, intolerance, or unsuitability.
* Depigmentation extent meeting all of the following:

Facial BSA (F-BSA) ≥ 0.5%, Facial VASI (F-VASI) ≥ 0.5, Total BSA (T-BSA) between 5% and 60%, Total VASI (T-VASI) ≥ 5.

* Agree to discontinue all vitiligo treatments from screening until final follow-up.
* If not previously vaccinated against zoster, agree to complete vaccination before Day 1.
* Contraception
* Not applicable to females of non-childbearing potential (surgically sterile or postmenopausal ≥12 months, confirmed by FSH at screening).

Exclusion Criteria:

* Non-eligible skin conditions: Other types of vitiligo (e.g., segmental) or other depigmentation disorders (e.g., piebaldism, leprosy, post-inflammatory hypopigmentation, tinea versicolor, etc.), or ≥30% leukotrichia on face or body.
* Psychiatric risk
* Recent vitiligo treatments
* Surgical treatments or depigmenting agents (e.g., monobenzone)
* High-dose steroids
* Pregnancy or lactation
* Abnormal Medical conditions
* Prohibited prior therapies
* Cardiac abnormalities
* Abnormal chest X-ray
* Renal impairment
* Clinically significant abnormal laboratory results at screening, per investigator judgment.
* Viral infections:
* Hypersensitivity: Known allergy or severe reaction to EI-001 or its excipients.
* Compliance concerns: Any condition that would make it difficult for the subject to follow the study schedule, receive treatment, attend visits, or could interfere with study objectives, data interpretation, or participant safety.

Where this trial is running

Hollywood, Florida and 2 other locations

• Skin Care Research, LLC — Hollywood, Florida, United States (Not_yet_recruiting)
• U Mass Chan Medical School — Worcester, Massachusetts, United States (Withdrawn)
• Linkou Chang Gung Memorial Hospital — Taoyuan, Taiwan (Recruiting)

Conditions Non-segmental Vitiligo