TerminatedNot Applicable

Observational Natural History Study of Autosomal Dominant Retinitis Pigmentosa (adRP)

Stopped: Development of SHP630 was discontinued based on lack of preclinical efficacy.

United States1 participantsStarted 2017-03-13

Plain-language summary

The purpose of this study is to gain an understanding of how adRP progresses over time in patients with misfolded rod opsin mutations.

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. The subject has 1 documented pre-specified heterozygous rhodopsin gene (RHO) mutation confirmed by genetic testing (mutations will include P23H, T17M, and R135W).
✓. The subject has at least 1 eye that meets all 3 of the following criteria:
✓. A measurable EZ area as determined by an evaluation of EZ limits on sdOCT scan, with a horizontal EZ width of greater than 3 mm
✓. BCVA of greater than or equal to 35 letters as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS; equivalent to 20/200 on a Snellen chart).
✓. A kinetic VF of greater than 10 degrees diameter in the horizontal meridian with a spot size of III
✓. The subject has the ability to comply with the clinical protocol, in the opinion of the investigator.
✓. The subject has a clear ocular media and adequate pupillary dilation in both eyes to permit adequate visual assessments in the opinion of the investigator.
✓. The subject has agreed to abstain from any protocol-prohibited medication(s) during study participation.

✕. The subject is participating in an interventional clinical trial or has participated in an interventional clinical trial within 90 days of screening; participation in non-interventional observational studies is permitted.
✕. The subject has received treatment or has 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.

Progression of disease over time in adRP patients with misfolded rod opsin mutations using ellipsoid zone (EZ) area measurements

Timeframe: Baseline to 4 years

NCT IDNCT02926092

SponsorShire

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2017-04-13

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. The subject has 1 documented pre-specified heterozygous rhodopsin gene (RHO) mutation confirmed by genetic testing (mutations will include P23H, T17M, and R135W).
✓. The subject has at least 1 eye that meets all 3 of the following criteria:
✓. A measurable EZ area as determined by an evaluation of EZ limits on sdOCT scan, with a horizontal EZ width of greater than 3 mm
✓. BCVA of greater than or equal to 35 letters as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS; equivalent to 20/200 on a Snellen chart).
✓. A kinetic VF of greater than 10 degrees diameter in the horizontal meridian with a spot size of III
✓. The subject has the ability to comply with the clinical protocol, in the opinion of the investigator.
✓. The subject has a clear ocular media and adequate pupillary dilation in both eyes to permit adequate visual assessments in the opinion of the investigator.
✓. The subject has agreed to abstain from any protocol-prohibited medication(s) during study participation.

✕. The subject is participating in an interventional clinical trial or has participated in an interventional clinical trial within 90 days of screening; participation in non-interventional observational studies is permitted.
✕. The subject has received treatment or has 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.