Clinical trial NCT04312672: Long-term Follow-up Gene Therapy Study for RPGR- XLRP
Official title: Long-term Follow-up Study of Participants Following an Open Label, Multi-Centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV5- hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: X-Linked Retinitis Pigmentosa
Lead sponsor: Janssen Research & Development, LLC
Sponsor class: INDUSTRY
First posted: 2020-03-18
Enrollment: 42
Sex: MALE
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Males receiving AAV5-hRKp.RPGR in the MGT009 study

Brief summary: This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.


Eligibility criteria: Inclusion Criteria:

* Received AAV5-hRKp.RPGR in the MGT009 Study

Exclusion Criteria:

* Unwilling or unable to meet with the requirements of the study

Primary outcomes:
measure: Assess the Longer-term Safety of AAV5-hRKp.RPGR Administered to Participants
timeFrame: 60 Months
Secondary outcomes:
measure: Change in Functional Vision of Walk Time in Vision-guided Mobility Assessment (VMA)
timeFrame: Up to 60 Months
measure: Change in Functional Vision of Low Luminance Questionnaire (LLQ) Domain Scores in Patient Reported Outcome (PRO)
timeFrame: Up to 60 Months
measure: Change in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score
timeFrame: Up to 60 Months
measure: Change in Low Luminance Visual Acuity (LLVA) as Assessed by ETDRS Chart Under Low Luminance Conditions
timeFrame: Up to 60 Months
measure: Change in Mean Retinal Sensitivity Within the Central 10 Degree Visual Field Excluding Scotoma (MRS10) in Static Perimetry
timeFrame: Up to 60 months
measure: Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Full Visual Field
timeFrame: Up to 60 months
measure: Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Central 30 Degrees Visual Field
timeFrame: Up to 60 months
measure: Change in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry
timeFrame: Up to 60 months
Other outcomes:
Locations:
city: Boston
country: United States
facility: Massachusetts Eye and Ear Infirmary
state: Massachusetts
city: Ann Arbor
country: United States
facility: Univ of Michigan Medical Center
state: Michigan
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Leeds
country: United Kingdom
facility: Leeds Teaching Hospitals NHS Trust
city: London
country: United Kingdom
facility: Moorfields Eye Hospital
Study officials:
affiliation: Janssen Research & Development, LLC
name: Janssen Research & Development, LLC Clinical Trial
role: STUDY_DIRECTOR
References: