Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa

X-Linked Retinitis Pigmentosa Clinical Trial

Official title:

An Open-label, Single-center, Dose-escalation Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Intraocular Administration of FT-002 in Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa.

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05874310
• Other study ID #: FT002RP-1
• Status: Recruiting
• Phase: Early Phase 1
• Start date: February 1, 2023
• Est. completion date: November 1, 2027

Study information

• Verified date: August 2022
• Source: Frontera Therapeutics
• Contact: Xinyan Li
• Phone: +86-021-58206061
• Email: xinyan.li[@]fronteratherapeutics.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

A clinical trial of gene therapy for patients with X-linked retinitis pigmentosa (XLRP).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 18
• Est. completion date: November 1, 2027
• Est. primary completion date: October 31, 2023
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 8 Years to 45 Years

Eligibility

Inclusion Criteria:

1.Subjects that are willing and able to follow study procedures; 2.Males aged 8-45 years old at the time of signing the Informed Consent Form; 4.Subjects who are confirmed with variants of RPGR ; Exclusion Criteria: 1.Have other retinal degenerative diseases, such as retinal degeneration caused by other known Inherited retinal disease gene variants or previously received an gene therapy product.

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa
• X-Linked Retinitis Pigmentosa

Intervention

Genetic:
• FT-002
Comparison of different dosages of FT-002

Locations

Country	Name	City	State
China	Peking Union Medical College Hospital	Beijing	Beijing
China	Eye & ENT hospital of Fudan university	Shanghai	Shanghai

Sponsors (4)

Lead Sponsor	Collaborator
Frontera Therapeutics	Eye & ENT Hospital of Fudan University, Henan Provincial People's Hospital, Peking Union Medical College Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of AEs	Number and proportion of Adverse Events	from FT-002 administration through up to 1 years
Secondary	Change in visual function	Change in retinal function as assessed by mean retinal sensitivity within the 30-degree visual field	from FT-002 administration through up to 1 years
Secondary	Change in retinal structure as assessed by Optical Coherence Tomography	Change in Outer Nuclear Layer Thickness from baseline as assessed by Optical Coherence Tomography	from FT-002 administration through up to 1 years