Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03116113
Recruitment Status : Completed
First Posted : April 14, 2017
Last Update Posted : May 18, 2021
Sponsor:
Information provided by (Responsible Party):
Biogen ( NightstaRx Ltd, a Biogen Company )

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.

Layout table for study information
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

Resource links provided by the National Library of Medicine


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.



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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03116113


Locations
Layout table for location information
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
Layout table for investigator information
Study Director: Medical Director Biogen
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: NightstaRx Ltd, a Biogen Company
ClinicalTrials.gov Identifier: NCT03116113    
Other Study ID Numbers: 274RP101 (NSR-RPGR-01)
First Posted: April 14, 2017    Key Record Dates
Last Update Posted: May 18, 2021
Last Verified: May 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
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:
Layout table for MeSH terms
Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn