NCT03549299

Allogeneic ABCB5-positive Limbal Stem Cells for Treatment of LSCD

An Interventional, Open-label, Multicenter Phase I/IIa Clinical Trial to Investigate the Safety and Efficacy of Ascending Doses of Allogeneic ABCB5-positive Limbal Stem Cells (LSC2) for the Treatment of Limbal Stem Cell Deficiency (LSCD)

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 16 (estimated)
• Sponsor: RHEACELL GmbH & Co. KG · Industry
• Sex: All
• Age: 18 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

The aim of this clinical trial is to investigate the efficacy (by monitoring neovascularization and epithelial defects) of up to four doses of the investigational medicinal product (IMP) LSC2 topically administered on the target eye of patients with LSCD. Further, safety of the IMP during and after application will be investigated (by monitoring adverse events \[AEs\]).

Detailed description

This is an interventional, open-label, phase I/IIa clinical trial to investigate the efficacy and safety of up to four doses of the IMP topically administered on the target eye of patients with LSCD. Patients will be treated in up to four ascending dose groups. The allogeneic investigational product LSC2 contains ABCB5-positive limbal stem cells (from corneal rims of cadaveric donors, expanded ex vivo, isolated and stored in a donor cell bank). The IMP will be applied on the target eye. Prior to application, the conjunctival pannus will be removed under general or local anesthesia. Patients will be followed up for efficacy for 1 year. Efficacy of the IMP will be monitored by assessing neovascularization and epithelial defects. To assess long-term safety of LSC2 one follow-up visit at Month 24 post IMP application is included.

Conditions

• Limbal Stem Cell Deficiency

Interventions

Type	Name	Description
BIOLOGICAL	LSC2	Topical application of IMP on target eye

Timeline

• Start date: 2019-07-04
• Primary completion: 2023-09-01
• Completion: 2023-09-01
• First posted: 2018-06-08
• Last updated: 2022-10-07

Locations

5 sites across 2 countries: United States, Germany

Regulatory

• FDA-regulated drug study