A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112 (XIRIUS)

• ClinicalTrials.gov Identifier: NCT03116113
• Recruitment Status: Completed
• First Posted: April 14, 2017
• Last Update Posted: February 13, 2023
• Sponsor: NightstaRx Ltd, a Biogen Company
• Information provided by (Responsible Party): Biogen ( NightstaRx Ltd, a Biogen Company )

Study Description

Brief Summary:

The objective of the study is to evaluate the safety, tolerability and efficacy of a single sub-retinal injection of BIIB112 in participants with X-linked retinitis pigmentosa (XLRP).

Condition or disease	Intervention/treatment	Phase
X-Linked Retinitis Pigmentosa	Biological: BIIB112	Phase 1; Phase 2

Detailed Description:

This study was previously posted by NightstaRx Ltd. In October, 2020, sponsorship of the trial was transferred to Biogen.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 50 participants
• Allocation: Randomized
• Intervention Model: Sequential Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: 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)
• Actual Study Start Date: March 16, 2017
• Actual Primary Completion Date: November 18, 2020
• Actual Study Completion Date: November 18, 2020

Arms and Interventions

Arm	Intervention/treatment
Experimental: Part 1: BIIB112 Dose 1; Participants will receive a single Dose 1 of BIIB112 by sub-retinal injection on Day 0.	Biological: BIIB112; Administered as specified in the treatment arm; Other Name: AAV8-RPGR
Experimental: Part 1: BIIB112 Dose 2; Participants will receive a single Dose 2 of BIIB112 by sub-retinal injection on Day 0.	Biological: BIIB112; Administered as specified in the treatment arm; Other Name: AAV8-RPGR
Experimental: Part 1: BIIB112 Dose 3; Participants will receive a single Dose 3 of BIIB112 by sub-retinal injection on Day 0.	Biological: BIIB112; Administered as specified in the treatment arm; Other Name: AAV8-RPGR
Experimental: Part 1: BIIB112 Dose 4; Participants will receive a single Dose 4 of BIIB112 by sub-retinal injection on Day 0.	Biological: BIIB112; Administered as specified in the treatment arm; Other Name: AAV8-RPGR
Experimental: Part 1: BIIB112 Dose 5; Participants will receive a single Dose 5 of BIIB112 by sub-retinal injection on Day 0.	Biological: BIIB112; Administered as specified in the treatment arm; Other Name: AAV8-RPGR
Experimental: Part 1: BIIB112 Dose 6; Participants will receive a single Dose 6 of BIIB112 by sub-retinal injection on Day 0.	Biological: BIIB112; Administered as specified in the treatment arm; Other Name: AAV8-RPGR
Experimental: Part 2: BIIB112 High Dose; Participants will receive a single high dose of BIIB112 by sub-retinal injection.	Biological: BIIB112; Administered as specified in the treatment arm; Other Name: AAV8-RPGR
Experimental: Part 2: BIIB112 Low Dose; Participants will receive a single low dose of BIIB112 by sub-retinal injection.	Biological: BIIB112; Administered as specified in the treatment arm; Other Name: AAV8-RPGR
No Intervention: Part 2: Untreated Group; Participants will receive no intervention to allow for a controlled comparison.

Outcome Measures

Primary Outcome Measures :

1. Part 1: Number of Participants with Dose-Limiting Toxicities (DLTs) [ Time Frame: Up to Month 24 ]
   DLTs are defined as any of the following events considered to be related to AAV8-RPGR: Sustained decrease in best-corrected visual acuity (BCVA) of ≥30 letters on the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart compared to baseline (sustained is defined as lasting 48 hours or more until recovery, with recovery defined as visual acuity (VA) returning to within 10 letters of baseline VA. An exception is made for surgery-related events occurring in close temporal association {within <24 hours} of the surgery); Vitreous inflammation, vitritis (>Grade 3 using standardised Nussenblatt vitreous inflammation scale grading); Any clinically significant retinal damage observed that is not directly attributed to complications of surgery; Any clinically relevant suspected unexpected serious adverse reaction, with the exception of vision loss or vision threatening.
2. Part 1: Number of Participants with Treatment-Emergent Adverse Events (TEAEs) [ Time Frame: Up to Month 24 ]
   An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
3. Part 2: Percentage of Study Eyes with ≥7 Decibels (dB) Improvement from Baseline at ≥5 of the 16 Central Loci of the 10-2 Grid Assessed by Macular Integrity Assessment (MAIA) Microperimetry [ Time Frame: Months 12 ]
   MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labeled as '< 0', '0', or a positive integer. The point labeled as '< 0' was assigned a value of '-1' by MAIA in the calculation. Improvement in Retinal Sensitivity in center grid was defined as an increase from baseline of 7 or more decibels in any 5 or more points out of the 16 central points.
4. Part 2: Number of Participants with TEAEs [ Time Frame: Up to Month 12 ]
   An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.

Secondary Outcome Measures :

1. Parts 1 and 2 : Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 out of the 16 Central Loci in Microperimetry [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18 and 24; Part 2: Month 1, 2, 3, 6, 9, and 12 ]
   MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labeled as '< 0', '0' or a positive integer. The point labeled as '< 0' was assigned a value of '-1' by MAIA in the calculation. Improvement in Retinal Sensitivity in center grid was defined as an increase from baseline of 7 or more decibels in any 5 or more points out of the 16 central points.
2. Parts 1 and 2: Percentage of Study Eyes with ≥7 dB Improvement from Baseline at ≥5 Out of the 68 Loci in Microperimetry [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18 and 24; Part 2: Month 1, 2, 3, 6, 9, and 12 ]
   MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labeled as '< 0', '0' or a positive integer. The point labeled as '< 0' was assigned a value of '-1' by MAIA in the calculation. Improvement in Retinal Sensitivity in whole grid was defined as an increase from baseline of 7 or more decibels in any 5 or more points of the grid as a whole (68 points).
3. Parts 1 and 2: Change from Baseline in Mean Sensitivity of the 16 Central Loci [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18 and 24; Part 2: Month 1, 2, 3, 6, 9, and 12 ]
   MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labeled as '< 0', '0' or a positive integer. The point labeled as '< 0' is assigned a value of '-1' by MAIA in the calculation. Improvement in Retinal Sensitivity in center grid was defined as an increase from baseline of 7 or more decibels in any 5 or more points out of the 16 central points. Here negative values indicate a decline in retinal sensitivity.
4. Parts 1 and 2: Change from Baseline in Mean Sensitivity of the 68 Central Loci [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18 and 24; Part 2: Month 1, 2, 3, 6, 9, and 12 ]
   MAIA microperimetry assessment was measured in dB using a 10-2 grid of 68 points. Each point was labeled as '< 0', '0' or a positive integer. The point labeled as '< 0' is assigned a value of '-1' by MAIA in the calculation. Improvement in Retinal Sensitivity in whole grid was defined as an increase from baseline of 7 or more decibels in any 5 or more points of the grid as a whole (68 points). Here negative values indicate a decline in retinal sensitivity.
5. Parts 1 and 2: Change from Baseline in Best Corrected Visual Acuity (BCVA) Score [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18 and 24; Part 2: Month 1, 2, 3, 6, 9, and 12 ]
   BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved.
6. Parts 1 and 2: Change from Baseline in Low Luminance Visual Acuity (LLVA) Score [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 3, 6, 9, and 12 ]
   LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject.
7. Parts 1 and 2: Percentage of Eyes with a ≥15 Letters Increase from Baseline for BCVA [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 2, 3, 6, 9, and 12 ]
   BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved.
8. Parts 1 and 2: Percentage of Eyes with a ≥15 Letters Increase from Baseline for LLVA [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 3, 6, 9, and 12 ]
   LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject.
9. Parts 1 and 2: Percentage of Eyes with a ≥10 Letters Increase from Baseline for BCVA [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 2, 3, 6, 9, and 12 ]
   BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved.
10. Parts 1 and 2: Percentage of Eyes with a ≥10 Letters Increase from Baseline for LLVA [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 3, 6, 9, and 12 ]
    LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject.
11. Parts 1 and 2: Percentage of Eyes with a ≥5 Letters Increase from Baseline for BCVA [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 2, 3, 6, 9, and 12 ]
    BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved.
12. Parts 1 and 2: Percentage of Eyes with a ≥5 Letters Increase from Baseline for LLVA [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 3, 6, 9, and 12 ]
    LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject.
13. Parts 1 and 2: Percentage of Eyes with a ≥15 Letters Loss from Baseline for BCVA [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 2, 3, 6, 9, and 12 ]
    BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved.
14. Parts 1 and 2: Percentage of Eyes with a ≥15 Letters Loss from Baseline for LLVA [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 3, 6, 9, and 12 ]
    LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject.
15. Parts 1 and 2: Percentage of Eyes with a ≥10 Letters Loss from Baseline for BCVA [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 2, 3, 6, 9, and 12 ]
    BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved.
16. Parts 1 and 2: Percentage of Eyes with a ≥10 Letters Loss from Baseline for LLVA [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 3, 6, 9, and 12 ]
    LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject.
17. Parts 1 and 2: Percentage of Eyes with a ≥5 Letters Loss from Baseline for BCVA [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 2, 3, 6, 9, and 12 ]
    BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved.
18. Parts 1 and 2: Percentage of Eyes with a ≥5 Letters Loss from Baseline for LLVA [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 3, 6, 9, and 12 ]
    LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject.
19. Parts 1 and 2: Percentage of Eyes with Change from Baseline > -5 Letters for BCVA [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 2, 3, 6, 9, and 12 ]
    BCVA was assessed using the ETDRS VA chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. BCVA was to be reported as number of letters read correctly by the subject. An increase in the number of letters read correctly means that vision has improved.
20. Part 1 and 2: Percentage of Eyes with Change from Baseline > -5 Letters for LLVA [ Time Frame: Part 1: Month 1, 3, 6, 9, 12, 18, and 24; Part 2: Month 1, 3, 6, 9, and 12 ]
    LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. Initially, letters were read at a distance of 4 meters from the chart. If <20 letters were read at 4 meters, testing at 1 meter was performed. LLVA was reported as number of letters read correctly by the subject.
21. Part 1: Change from Baseline in Central Ellipsoid Area [ Time Frame: Month 1, 3, 6, 9, 12, 18 and 24 ]
    Spectral Domain Optical Coherence Tomography (SD-OCT) was used to assess change in central ellipsoid area.
22. Part 1: Change from Baseline in Central Horizontal Ellipsoid Width [ Time Frame: Month 1, 3, 6, 9, 12, 18 and 24 ]
    SD-OCT was used to assess change in central horizontal ellipsoid width. Here negative values indicate decline in central horizontal ellipsoid width.
23. Part 1: Change from Baseline in Fundus Autofluorescence- Total Area of Preserved Autofluoroscence [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ]
    Fundus Autofluorescence was used to assess change in total area of preserved autofluorescence and distance from foveal center (FC) to nearest border of preserved autofluorescence.
24. Part 1: Change from Baseline in Fundus Autofluorescence- Distance from Foveal Center (FC) to Nearest Border of Preserved Autofluorescence [ Time Frame: Month 1, 3, 6, 9, 12, 18 and 24 ]
    Fundus Autofluorescence was used to assess change in distance from foveal center (FC) to nearest border of preserved autofluorescence.
25. Part 1: Change from Baseline in Volume of 30-Degree Hill of Vision [ Time Frame: Month 6, 12 and 24 ]
    Visual field testing was performed to assess change in volume of 30-degree hill vision. Reliability Factor (RF)=number of false positive responses + number of false negative responses/number of false positive presentations + number of false negative presentations*100. If there are 0 responses, then RF value=0. RFpositive=number of false positive responses/number of false positive presentations*100. If RF≤ 20% measurement is considered reliable. If 20% < RF ≤ 25% and RFpositive ≤ 10% measurement is also considered reliable. Otherwise if 20% < RF ≤ 25% and RFpositive > 10%, or RF > 25%, measurement is not reliable.
26. Part 1: Change from Baseline in Volume of Full Field Hill of Vision [ Time Frame: Month 6, 12, and 24 ]
    Visual field testing was performed to assess change in volume of full field hill vision. Reliability Factor (RF)=number of false positive responses + number of false negative responses/number of false positive presentations + number of false negative presentations*100. If there are 0 responses, then RF value=0. RFpositive=number of false positive responses/number of false positive presentations*100. If RF≤ 20% measurement is considered reliable. If 20% < RF ≤ 25% and RFpositive ≤ 10% measurement is also considered reliable. Otherwise if 20% < RF ≤ 25% and RFpositive > 10%, or RF > 25%, measurement is not reliable.
27. Part 1: Change from Baseline in Contrast Sensitivity [ Time Frame: Month 3, 6, 12 and 24 ]
    Change in contrast sensitivity (CS) was assessed by Pelli-Robson chart which uses a single large letter size (20/60 optotype), with contrast varying across groups of letters. Chart uses letters (6 per line), arranged in groups whose contrast varies from high to low. Subjects read the letters, starting with the highest contrast, until they are unable to read two or three letters in a single group. Each group has three letters of the same contrast level, so there are three trials per contrast level. Subject is assigned a score based on the contrast of the last group in which two or three letters were correctly read. Score is a measure of the subject's log contrast sensitivity ranging from 0-2.25, with 0 being no letters read, and 2.25 being all letters read. Total CS score = [(total # letters correct - 3) x 0.05].
28. Part 2: Change from Baseline in Volume of 30-Degree Hill of Vision Assessed by Octopus 900 [ Time Frame: Baseline, Month 3, 6 and 12 ]
    Visual field testing was performed to assess change in volume of 30-degree hill vision. Reliability Factor (RF)=number of false positive responses + number of false negative responses/number of false positive presentations + number of false negative presentations*100. If there are 0 responses, then RF value=0. RFpositive=number of false positive responses/number of false positive presentations*100. If RF≤ 20% measurement is considered reliable. If 20% < RF ≤ 25% and RFpositive ≤ 10% measurement is also considered reliable. Otherwise if 20% < RF ≤ 25% and RFpositive > 10%, or RF > 25%, measurement is not reliable. Here negative values indicate decline in the volume of 30-degree hill vision.
29. Part 2: Change from Baseline in Volume of Full Field Hill of Vision Assessed by Octopus 900 [ Time Frame: Baseline, Month 3, 6 and 12 ]
    Visual field testing was performed to assess change in volume of full field hill vision. Reliability Factor (RF)=number of false positive responses + number of false negative responses/number of false positive presentations + number of false negative presentations*100. If there are 0 responses, then RF value=0. RFpositive=number of false positive responses/number of false positive presentations*100. If RF≤ 20% measurement is considered reliable. If 20% < RF ≤ 25% and RFpositive ≤ 10% measurement is also considered reliable. Otherwise if 20% < RF ≤ 25% and RFpositive > 10%, or RF > 25%, measurement is not reliable. Here negative values indicate decline in volume of full field hill vision.

Eligibility Criteria

• Ages Eligible for Study: 10 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: Male
• Gender Based Eligibility: Yes
• Accepts Healthy Volunteers: No

Criteria

Key Inclusion Criteria:

Part 1:

• Participants with genetically confirmed diagnosis of XLRP (with RPGR mutation).
• Participant with active disease clinically visible within the macular region in both eyes.

Part 2:

- Participant with mean total retinal sensitivity in the study eye as assessed by microperimetry ≥ 0.1 dB and ≤8 dB.

Key exclusion Criteria:

Parts 1 and 2:

• Participant with history of amblyopia in either eye.
• Participated in a gene therapy trial previously or a clinical trial with an investigational drug in the past 12 weeks or received a gene/cell-based therapy at any time previously.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Contacts and Locations

Locations

United States, Florida

Research Site

Gainesville, Florida, United States, 32607

Research Site

Miami, Florida, United States, 33136

United States, Oregon

Research Site

Portland, Oregon, United States, 97239

United States, Pennsylvania

Research Site

Philadelphia, Pennsylvania, United States, 19104

United States, Texas

Research Site

Dallas, Texas, United States, 75231

United Kingdom

Research Site

Manchester, United Kingdom

Research Site

Oxford, United Kingdom

Research Site

Southampton, United Kingdom

Sponsors and Collaborators

NightstaRx Ltd, a Biogen Company

Investigators

• Study Director: Medical Director; Biogen

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Cehajic-Kapetanovic J, Xue K, Martinez-Fernandez de la Camara C, Nanda A, Davies A, Wood LJ, Salvetti AP, Fischer MD, Aylward JW, Barnard AR, Jolly JK, Luo E, Lujan BJ, Ong T, Girach A, Black GCM, Gregori NZ, Davis JL, Rosa PR, Lotery AJ, Lam BL, Stanga PE, MacLaren RE. Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR. Nat Med. 2020 Mar;26(3):354-359. doi: 10.1038/s41591-020-0763-1. Epub 2020 Feb 24.

• Responsible Party: NightstaRx Ltd, a Biogen Company
• ClinicalTrials.gov Identifier: NCT03116113
• Other Study ID Numbers: 274RP101; NSR-RPGR-01 ( Other Identifier: NightstaRx Ltd, a Biogen Company )
• First Posted: April 14, 2017
• Last Update Posted: February 13, 2023
• Last Verified: February 2023

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Plan Description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/
• URL: https://vivli.org/

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Biogen ( NightstaRx Ltd, a Biogen Company ):

XLRP; Ophthalmology; Gene Therapy; Retinitis Pigmentosa GTPase Regulator (RPGR); AAV8

Additional relevant MeSH terms:

Retinitis; Retinitis Pigmentosa; Retinal Diseases; Eye Diseases; Eye Diseases, Hereditary; Retinal Dystrophies; Retinal Degeneration; Genetic Diseases, Inborn