Retinitis PigmentosaNCT02926092
Observational Natural History Study of Autosomal Dominant Retinitis Pigmentosa (adRP)
An observational study of Retinitis Pigmentosa, run by Shire at 1 site in United States.
Trial summary
The purpose of this study is to gain an understanding of how adRP progresses over time in patients with misfolded rod opsin mutations. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- Observation
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 13 Mar 2017to 13 Apr 2017
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Study population will come from a clinical setting where a diagnosis of adRP has been made
Who may be able to join
- 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.
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- 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 medically stable in the opinion of the investigator and able to fulfill the protocol requirements, including the ability to complete the assessments, without placing an undue burden on the subject/subject's family.
- The subject and/or subject's parent(s) or legally authorized guardian(s) has voluntarily signed an Institutional Review Board (IRB)/ ethics committee (EC)-approved informed consent and assent form(s), as applicable, after all relevant aspects of the study have been explained and discussed with the subject and/or the subject's parent(s) or legally authorized guardian(s).
- The subject, subject's parent(s), or legally authorized guardian(s) is able to understand the nature, scope, and possible consequences of the study and agrees to comply with the protocol-defined, scheduled assessments.
Who cannot join
- 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.
- The subject has any of the following medical conditions that will interfere with consistent follow-up over any part of the study:
- Stroke
- Severe or unstable coronary disease
- End-stage or aggressive malignancy
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- General poor health or uncontrolled or severe disease (eg, cardiovascular, neurological, psychological, pulmonary,renal, hepatic, endocrine, or gastrointestinal disorders) that in the opinion of the investigator would interfere with participation in the study
- The subject has any of the following ocular conditions that could interfere with or confound follow-up of disease progression:
- Glaucoma
- Diabetic retinopathy
- Choroidal neovascularization
- Retinal inflammatory disease
- Cataract worse than grade 2 (nuclear, posterior subcapsular [PSC], or cortical)
- High myopia (≥8 diopters)
- Herpes simplex virus of the eye
- Acute infection or inflammation
- Any ocular condition that in the opinion of the investigator would interfere with the ability to assess retinal morphology and functionality
- The subject has had intraocular surgery within 90 days prior to screening.
- The subject currently requires the following protocol prohibited medications or has ingested such medication within 30 days of screening:
- Plaquenil Thioridazine Clofazimine Deferoxamine Phenothiazine Chlorpromazine Cisplatin Valproic acid Any other drugs with known visual side effects
- The subject has 3 first- or second-degree family members already enrolled in the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Prospective, Multicenter, Longitudinal, Observational Natural History Study to Evaluate Disease Progression in Subjects With Autosomal Dominant Retinitis Pigmentosa (adRP) With Misfolded Rod Opsin Mutations
The purpose of this study is to gain an understanding of how adRP progresses over time in patients with misfolded rod opsin mutations.
- Conditions
- Retinitis Pigmentosa
- Being tested
- Observation
- Medical subject headings
- Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ObservationDiagnostic test
Observation of progression of disease over time
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Observation of progression of disease over time. Receives: Diagnostic Test: Observation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Progression of disease over time in adRP patients with misfolded rod opsin mutations using ellipsoid zone (EZ) area measurements
Measured: Baseline to 4 years
Secondary outcomes (6)
Progression of disease over time in adRP patients with misfolded rod opsin mutations as measured by EZ width
Measured: Baseline to 4 years
Progression of disease over time in adRP patients with misfolded rod opsin mutations as measured by visual fields (kinetic and static)
Measured: Baseline to 4 years
Progression of disease over time in adRP patients with misfolded rod opsin mutations as measured by dark-adapted rod visual fields
Measured: Baseline to 4 years
Progression of disease over time in adRP patients with misfolded rod opsin mutations as measured by electroretinography (ERG): dark- and light-adapted
Measured: Baseline to 4 years
Progression of disease over time in adRP patients with misfolded rod opsin mutations as measured by best corrected visual acuity (BCVA)
Measured: Baseline to 4 years
Vision-related function and quality of life as measured by 25-item National Eye Institute Visual Function Questionnaire (NEI VFQ-25) plus its additional items
Measured: Baseline to 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Oct 2016 (estimated)
- Study start13 Mar 2017
- Primary completion13 Apr 2017
- Study completion13 Apr 2017
Why it stopped: Development of SHP630 was discontinued based on lack of preclinical efficacy.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Shire Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02926092
- Sponsor's protocol ID
- SHP630-001
- Status last verified
- Mar 2021
- First submitted
- 5 Oct 2016
- Last updated
- 17 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No: De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants).
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Multicenter, Longitudinal, Observational Natural History Study to Evaluate Disease Progression in Subjects With Autosomal Dominant Retinitis Pigmentosa (adRP) With Misfolded Rod Opsin Mutations
- ClinicalTrials.gov ID
- NCT02926092
- Acronym / sponsor ID
- SHP630-001
- Conditions
- Retinitis Pigmentosa
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Development of SHP630 was discontinued based on lack of preclinical efficacy.
- Enrolment
- 1 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 13 Mar 2017 (actual)
- Primary completion
- 13 Apr 2017 (actual)
- Study completion
- 13 Apr 2017 (actual)
- First posted
- 6 Oct 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to gain an understanding of how adRP progresses over time in patients with misfolded rod opsin mutations.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | – | Diagnostic Test: ObservationObservation of progression of disease over time. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Study population will come from a clinical setting where a diagnosis of adRP has been made
Key inclusion criteria (11 listed)
- 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:
- 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.
Key exclusion criteria (21 listed)
- 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.
- The subject has any of the following medical conditions that will interfere with consistent follow-up over any part of the study:
- The subject has any of the following ocular conditions that could interfere with or confound follow-up of disease progression:
- The subject has had intraocular surgery within 90 days prior to screening.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ObservationObservation of progression of disease over time | Diagnostic test | Arm 1 |
Primary outcome
Progression of disease over time in adRP patients with misfolded rod opsin mutations using ellipsoid zone (EZ) area measurements
Time frame: Baseline to 4 years
6 secondary outcomes
Progression of disease over time in adRP patients with misfolded rod opsin mutations as measured by EZ width
Time frame: Baseline to 4 years
Progression of disease over time in adRP patients with misfolded rod opsin mutations as measured by visual fields (kinetic and static)
Time frame: Baseline to 4 years
Progression of disease over time in adRP patients with misfolded rod opsin mutations as measured by dark-adapted rod visual fields
Time frame: Baseline to 4 years
Progression of disease over time in adRP patients with misfolded rod opsin mutations as measured by electroretinography (ERG): dark- and light-adapted
Time frame: Baseline to 4 years
Progression of disease over time in adRP patients with misfolded rod opsin mutations as measured by best corrected visual acuity (BCVA)
Time frame: Baseline to 4 years
Vision-related function and quality of life as measured by 25-item National Eye Institute Visual Function Questionnaire (NEI VFQ-25) plus its additional items
Time frame: Baseline to 4 years
Results and safety
The study was stopped early (reason given: Development of SHP630 was discontinued based on lack of preclinical efficacy.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 2021.
- To obtain more information on the study, click here/on this link.Link provided by the sponsor.
- Individual participant dataSharing planned: No. De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants).
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Shire Director study director
Takeda
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Retinitis Pigmentosa, run by Shire at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 17 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Development of SHP630 was discontinued based on lack of preclinical efficacy.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Progression of disease over time in adRP patients with misfolded rod opsin mutations using ellipsoid zone (EZ) area measurements (time frame: Baseline to 4 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Mar 2021.