A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa

A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)

Sponsors

Lead Sponsor: NightstaRx Ltd, a Biogen Company

Source NightstaRx Ltd, a Biogen Company
Brief Summary

A clinical trial of AAV8-RPGR retinal gene therapy for patients with X-linked retinitis pigmentosa

Detailed Description

This is a Phase 1/2/3, first-in-human, multi-centre, dose-escalation interventional study of AAV8-RPGR in male subjects with genetically confirmed XLRP. Part I is a dose-selection study; Part II is a dose-expansion study, comparing 2 doses, and a third untreated group to allow for a controlled comparison of efficacy and safety.

Overall Status Recruiting
Start Date March 16, 2017
Completion Date March 2021
Primary Completion Date March 2021
Phase Phase 2/Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Part I: Incidence of dose limiting toxicities (DLTs) 24 months
Part I: Incidence of treatment emergent adverse events (TEAEs) 24 months
Part II: Improvement from Baseline in microperimetry 12 Months
Part II: The safety endpoint is incidence of TEAEs over a 12-month period 12 Months
Secondary Outcome
Measure Time Frame
Best corrected visual acuity (BCVA) 24 Months (Part I),12 Months (Part II)
Microperimetry 24 Months (Part I),12 Months (Part II)
Spectral domain optical coherence tomography (SD-OCT) 24 Months (Part I)
Fundus autofluorescence (AF) 24 Months (Part I)
Visual Fields 12 Months (Part II)
Enrollment 63
Condition
Intervention

Intervention Type: Biological

Intervention Name: AAV8-RPGR

Description: Comparison of different dosages of AAV8-RPGR

Eligibility

Criteria:

Key inclusion Criteria:

- Male

- Part I: >= 18 years

- Part II: >= 10 years

- Documentation of a pathogenic mutation in the RPGR gene

Key exclusion Criteria:

- Participated in a gene therapy trial previously or a clinical trial with an investigational drug in the past 12 weeks

Gender: Male

Minimum Age: 10 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Contact

Last Name: NightstaRx Ltd, a Biogen Company

Phone: +1 866-633-4636

Email: [email protected]

Location
Facility: Status:
Study Site | Gainesville, Florida, 32607, United States Recruiting
Study Site | Miami, Florida, 33136, United States Recruiting
Study Site | Portland, Oregon, 97239, United States Recruiting
Study Site | Philadelphia, Pennsylvania, 19104, United States Recruiting
Study Site | Dallas, Texas, 75231, United States Recruiting
Study Site | Manchester, United Kingdom Active, not recruiting
Study Site | Oxford, United Kingdom Recruiting
Study Site | Southampton, United Kingdom Recruiting
Location Countries

United Kingdom

United States

Verification Date

April 2020

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 9
Arm Group

Label: Part I: Dose 1 AAV8-RPGR

Type: Experimental

Description: Single, subretinal administration of dose 1 as a single dose AAV8-RPGR

Label: Part I: Dose 2 AAV8-RPGR

Type: Experimental

Description: Single, subretinal administration of dose 2 as a single dose AAV8-RPGR

Label: Part I: Dose 3 AAV8-RPGR

Type: Experimental

Description: Single, subretinal administration of dose 3 as a single dose AAV8-RPGR

Label: Part I: Dose 4 AAV8-RPGR

Type: Experimental

Description: Single, subretinal administration of dose 4 as a single dose AAV8-RPGR

Label: Part I: Dose 5 AAV8-RPGR

Type: Experimental

Description: Single, subretinal administration of dose 5 as a single dose AAV8-RPGR

Label: Part I: Dose 6 AAV8-RPGR

Type: Experimental

Description: Single, subretinal administration of dose 6 as a single dose AAV8-RPGR

Label: Part II: High dose AAV8-RPGR

Type: Experimental

Description: Single, subretinal administration of high dose AAV8-RPGR

Label: Part II: Low dose AAV8-RPGR

Type: Experimental

Description: Single, subretinal administration of Low dose AAV8-RPGR

Label: Part II: Untreated Group

Type: No Intervention

Description: Untreated group to allow for a controlled comparison of efficacy and safety

Acronym XIRIUS
Patient Data No
Study Design Info

Allocation: Randomized

Intervention Model: Sequential Assignment

Primary Purpose: Treatment

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Masking Description: Part I unmasked,Part II masked. Outcomes assessor masked to treatment; subject, surgeon, investigator/site team, and sponsor masked to dose.

Source: ClinicalTrials.gov