SHP630-001
Terminated / Withdrawn
Observational Natural History Study of Autosomal Dominant Retinitis Pigmentosa (adRP)
Clinicaltrials.gov
#NCT02926092About this clinical trial
The purpose of this study is to gain an understanding of how adRP progresses over time in patients with misfolded rod opsin mutations.
US
Observational clinical trial.
At a glance
What medical conditions were being studied?
Retinitis Pigmentosa
What was the clinical trial testing?
Observation
How many participants were enrolled?
1
Were placebos part of the clinical trial?
No
When was the clinical trial conducted?
Mar 2017 - Apr 2017
How long was participation in the clinical trial?
The maximum duration of study participation is expected to be approximately 48 months of assessments plus a screening period of up to 60 days. The study will be completed in approximately 5 years. This is an observational study and no treatment for adRP with an investigational medicinal product will be provided.
Key requirements
Sex
All
Age
All
Healthy volunteers?
No
Entry criteria
Have 1 documented pre-specified heterozygous RHO mutation confirmed by genetic testing (mutations will include P23H, T17M, and R135M
Have at least 1 eye that meets all 3 of the following criteria: 1.) a measurable EZ area as determined by an evaluation of EZ limits on sdOCT scan, with a horizontal EZ width of no greater than 3 mm 2.) BCVA of greater than of equal to 35 letters as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS; equivalent to 20/200 on a Snellen chart) 3.) a kinetic VF of greater than 10 degrees diameter in the horizontal meridian with a spot size of III
Cannot be participating in an interventional clinical trial or have participated in an interventional clinical trial within 90 days of screening; participation in non-interventional observational studies is permitted
Cannot have received treatment or have been in the treatment arm of a clinical trial for gene therapy, stem cell therapy, retinal progenitor cell therapy, tissue transplantation, device or drug delivery implantation, or other similar invasive therapy