Gene Therapy for RPGR Gene Mutation-associated X-linked R... | NCT06492850

Studies
Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa

NCT06492850

Sponsor

Frontera Therapeutics

Status

Recruiting

Last Update Posted

Jul 9, 2024Actual

Enrollment

32Estimated

Phase

Phase 1Phase 2

Conditions

X-Linked Retinitis Pigmentosa (XLRP)

Interventions

FT-002

Countries

China

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jul 2024

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Apr 1, 2024Actual

Primary Completion Date

Aug 1, 2025Estimated

Completion Date

Feb 1, 2026Estimated

First Submitted Date

Jul 1, 2024

First Submission Date that Met QC Criteria

Jul 1, 2024

First Posted Date

Jul 9, 2024Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jul 1, 2024

Last Update Posted Date

Jul 9, 2024Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

The aim of this study was to evaluate the safety, tolerability, and efficacy of one-time subretinal injection of FT-002 in male subjects (8-45 years of age) with RPGR (Retinitis Pigmentosa GTPase Regulator) gene mutation-associated X-linked retinitis pigmentosa, of XLRP. This study includes Phase I (dose escalation phase) and Phase II (dose expansion phase).

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

32Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
FT-002 dose 1	Experimental	Low dose FT-002	Genetic: FT-002
High dose FT-002	Experimental	High dose FT-002	Genetic: FT-002

Interventions

Name	Type	Description	Arm Group Labels	Other Names
FT-002	Genetic	Intraocular injection of a single dose	FT-002 dose 1 High dose FT-002

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
To evaluate the safety and tolerance of FT-002	Incidence and severity of AEs	from FT-002 administration through up to 1 years

Secondary Outcomes

Measure	Description	Time Frame
To evaluate the efficacy of FT-002	Changes in visual sensitivity /FST/BCVAfrom baseline	from FT-002 administration through up to 1 years

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Subjects that are willing and able to follow study procedures including scheduled visits, treatment plan, and laboratory tests, and sign a written informed consent form;
Age: Phase I dose escalation stage, 18-45 years old male (including boundary value) at the time of signing the ICF; Phase II dose extension stage, males 8-45 years old (including boundary values) at the time of signing the ICF;
Clinically diagnosed XLRP, the main symptoms include but are not limited to night blindness, visual field loss, vision loss, etc.;

Exclusion Criteria:

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.

Accepts Healthy Volunteers

Sex

Male

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

8 Years

Maximum Age

45 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Xinyan Li	Contact	+86-021-58206061		xinyan.li@fronteratherapeutics.com
Minghui Xue	Contact	+86-021-58206061		minghui.xue@fronteratherapeutics.com

Overall Officials

Name	Affiliation	Role
Ruifang Sui	Peking Union Medical College Hospital	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Peking Union Medical College Hospital	Recruiting	Beijing	Beijing Municipality	100142	China

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided