NCT05093166

Clinical Trial to Assess the Safety and Efficacy of Investigational Product in Patients Due to Hypospadias Treatment Failure

Prospective, Open-label, Uncontrolled Pilot Clinical Trial to Assess the Safety and Efficacy of Autologous Cultured Oral Mucosa Grafts for Urethral Reconstruction in Patients Due to Hypospadias Treatment Failure

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Holostem s.r.l. · Industry
• Sex: Male
• Age: 5 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this trial is to assess the safety and efficacy of Holour in patients suffering from hypospadias treatment failure. Holour is intended for urethral replacement and is made from ex vivo expanded autologous oral mucosal cells including stem cells.

Detailed description

Holour is a prospective, open-label, uncontrolled pilot clinical trial phase i/ii. The aim of this clinical trial is to perform a tissue engineering therapy to assess the safety (at 3 and 12 months) and efficacy (at 12 months) of the autologous treatment. Patients will be screened according to the study inclusion and exclusion criteria and will be candidate for the treatment if all eligibility criteria are met. Up to 8 patients will be treated by means of autologous cultured oral mucosa grafts containing oral mucosa stem cells. Holour is produced by ex vivo expansion of cells obtained from oral mucosa biopsy collected from patient. The biopsy will be processed in a laboratory of a regenerative medicine manufacturing site where the epithelial cells will be isolated, expanded and prepared as final graft to be implanted. Holour administration will be planned according to the procedures and the need of the patient. The entire procedure envisages the following steps: 1. Oral mucosa biopsy to manufacture autologous grafts of Holour. 2. Penile urethroplasty in two stages: * First stage: application of holour on the wound bed prepared according to standard surgery. * Second stage: penis reconstruction according to standard surgical procedure. The surgical procedures may be followed by a post-implantation treatment (if necessary) with corticosteroids and antibiotics. The treatment includes a single administration of Holour; in case of "failure" of the first procedure eligible patients can undergo to a second implantation. Three months after the transplantation, primary endpoint will be evaluated by the investigator. The study completion will be reached when 1 year (secondary endpoint) of follow-up after the last transplant in the last patient will be accomplished. The end of the trial is defined as the last visit of the last patient after the last treatment if any.

Conditions

• Hypospadias

Interventions

Type	Name	Description
DRUG	"Ex vivo" expanded autologous human oral epithelium containing stem cells	The treatment includes a single administration of investigational product through a dedicated surgical procedure of penis wound bed preparation, epithelial surface adaption and product application followed by urethra tubularization and penis reconstruction.

Timeline

• Start date: 2021-11-26
• Primary completion: 2022-12-12
• Completion: 2022-12-12
• First posted: 2021-10-26
• Last updated: 2024-11-12

Locations

1 site across 1 country: Italy