BRM421 Ophthalmic Solution in Patients With Limbal Stem Cell Deficiency

NCT07422389 | Unknown

• 2 other identifiers: 859634BRM421-25-EA002
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this program is to provide patients with Limbal Stem Cell Deficiency access to the BRM421 eye drops. Participants will:

• Use BRM421 two times a day for at least 12 weeks
• Visit the clinic monthly for checkups and tests

Conditions

Limbal Stem Cell Deficiency (LSCD)

Interventions

BRM421 Ophthalmic Solution, 0.06% DRUG

Patients eligible to participate will receive 0.06% BRM421 Ophthalmic Solutions to be administered two times daily (BID, morning and bedtime) on the eligible eye(s). The administration will be at least 12 weeks. The investigator may continue the treatment beyond the 12 weeks based on clinical response. For patients scheduled for LSC transplant, treatment with BRM421 should start only after surgery.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adult patients with mild to moderate LSCD who unsatisfied with the standard of care treatments and who seek the treatment with BRM421 Ophthalmic Solution. Patients scheduled for LSC transplant may be included, but treatment with BRM421 should begin only after the surgery.
• Be able to sign the informed consent and follow with investigator instructions.
• If a female is of childbearing potential, they must: use an acceptable means of birth control (acceptable methods of contraception include: hormonal oral, implantable, injectable, or transdermal contraceptives, mechanical spermicide in conjunction with a barrier such as a diaphragm or condom, IUD, or surgical sterilization of partner) and have a negative urine pregnancy test at the time of consent.

You may not qualify if:

• The patients eligible for the BRIM clinical trial(s).
• The patients diagnosed with primary LSCD which is the genetic mutations that lead to LSC dysfunction or destruction.
• Have a condition or a situation in which the investigator feels the patients is not suitable and may put the patient at significant risk.
• Be a female who is pregnant, nursing, or planning a pregnancy at the time of consent. Be a woman of childbearing potential who is not using an acceptable means of birth control; acceptable methods of contraception include: hormonal oral, implantable, injectable, or transdermal contraceptives; mechanical spermicide in conjunction with a barrier such as a diaphragm or condom; IUD; or surgical sterilization of partner. The risks of BRM421 among pregnant patients are not known.

Sponsors & Collaborators

• BRIM Biotechnology Inc.: lead
• University of Pennsylvania: collaborator

Study Sites (1)

Scheie Eye Institute

Philadelphia, Pennsylvania, 19104, United States

AVAILABLE

MeSH Terms

Conditions

Limbal Stem Cell Deficiency

Condition Hierarchy (Ancestors)

Corneal Diseases; Eye Diseases

Central Study Contacts

Emma Iacobucci

CONTACT

215-662-9393; emma.iacobucci@pennmedicine.upenn.edu

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 2, 2025
• First Posted: February 20, 2026
• Last Updated: February 20, 2026

Record last verified: 2026-02

Locations

US (1)