Usher Syndrome, Type 1BNCT03814499
Natural History Study in Subjects With Usher Syndrome
An observational study of Usher Syndrome, Type 1B, run by Fondazione Telethon at 3 sites in 3 countries.
Trial summary
Natural History Study in Subjects With Usher Syndrome ((USH1B) is a multi-centre, longitudinal, observational study designed to evaluate disease progression in subjects with USH1B by several vision-related assessments. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Usher Syndrome, Type 1B
- How many people
- 56 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 1 Jun 2018to 30 Dec 2022
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Subjects affected by Usher Syndrome 1B disease fulfilled eligibility criteria
Who may be able to join
- Must be willing to adhere to protocol for long-term follow-up as evidenced by written informed consent or parental permission and subject assent.
- Subjects diagnosed with USH1.
- Molecular diagnosis of USH1B due to MYO7A mutations (homozygotes or compound heterozygotes).
- Age eight years old or older at the time of baseline.
- Visual acuity ≥ 20/640 in at least one eye
Who cannot join
- Unable or unwilling to meet requirements of the study.
- Unable to communicate with suitable verbal/auditory and/or tactile sign language (in the opinion of the investigator)
- Participation in a clinical study with an investigational drug in the past six months.
- Pre-existing eye conditions that would interfere with the interpretation of study endpoints (for example, glaucoma, corneal or significant lenticular opacities, cystoid macular oedema, macular hole).
- Complicating systemic diseases in which the disease itself, or the treatment for the disease, can alter ocular function. Examples are malignancies whose treatment could affect central nervous system function (for example, radiation treatment of the orbit; leukemia with CNS/optic nerve involvement). Also excluded would be subjects with immuno- compromising diseases, as there could be susceptibility to opportunistic infection [such as cytomegalovirus (CMV) retinitis].
- Subjects with diabetes or sickle cell disease would be excluded if they had any manifestation of advanced retinopathy (e.g. macular edema or proliferative changes).
- Prior ocular surgery within three months.
- Any other condition that would not allow the potential subject to complete follow-up examinations during the course of the study and, in the opinion of the investigator, makes the potential subject unsuitable for the study.
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
Spain1 site
Instituto de Investigacion Sanitaria de la Fundacion Jimenez Diaz
Madrid
About this study
Official title: A Multicentre Longitudinal, Observational Natural History Study to Evaluate Disease Progression in Subjects With Usher Syndrome Type 1B (USH1B)
Natural History Study in Subjects With Usher Syndrome ((USH1B) is a multi-centre, longitudinal, observational study designed to evaluate disease progression in subjects with USH1B by several vision-related assessments.
Read the detailed description
Natural History Study in Subjects With Usher Syndrome (USH1B) is being conducted to understand the progression of disease in USH1B patients as measured by visual acuity and visual field testing and a number of other vision-related assessments. Disease progression will be evaluated as change over time in these measures and associations between the endpoints will be examined.
The primary objective of this study is to evaluate the natural progression of disease over time in USHIB patients using visual field testing and best corrected visual acuity.
The secondary objectives of this study are to evaluate the progression of disease over time in USHIB patients through additional assessments:
- Microperimetry (only in selected centres)
- Fundus autofluorescence (FAF)
- Optical Coherence Tomography (OCT)
- Full-field Electroretinogram (ERG),
- Multifocal Electroretinogram,
- Vision-related function and quality of life, as measured by the 25-Item National Eye Institute Visual Function Questionnaire (NEI VFQ-25)
- Conditions
- Usher Syndrome, Type 1B
- Medical subject headings
- Usher syndrome, type 1B
What is being measured
The primary outcome is the main question the study is designed to answer.
BCVA - Better Seeing Eyes
Change (from Baseline) in Best Corrected Visual acuity (BCVA) in the better-seeing eyes.
BCVA in the better seeing eyes was assessed using the ETRS score (minimum value 0 and maximum value 100, with 0 representing the worst, and 100 the best visual functioning).
The results are reported as the change in ETDRS score collected at the 1 year follow-up visit compared with the score at the baseline visit.
Measured: 1 year follow up visit
BCVA - Better Seeing Eyes
Change (from Baseline) in Best Corrected Visual acuity (BCVA) in the better-seeing eyes.
BCVA in the better seeing eyes was assessed using the ETRS score (minimum value 0 and maximum value 100, with 0 representing the worst, and 100 the best visual functioning).
The results are reported as the change in ETDRS score collected at the 2 year follow-up visit compared with the score at the baseline visit.
Measured: 2 year follow up visit
III4e Area - Better Seeing Eyes
Change (from Baseline) in visual field area using III4e stimulus size in the better seeing eyes.
Visual field area using III4e stimulus size in the better seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000).
The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Measured: 1 year follow up visit
III4e Area - Better Seeing Eyes
Change (from Baseline) in visual field area using III4e stimulus size in the better seeing eyes.
Visual field area using III4e stimulus size in the better seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000).
The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Measured: 2 year follow up visit
V4e Area - Better Seeing Eyes
Change (from Baseline) in visual field area using V4e stimulus size in the better seeing eyes.
Visual field area using V4e stimulus size in the better seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000).
The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Measured: 1 year follow up visit
V4e Area - Better Seeing Eyes
Change (from Baseline) in visual field area using V4e stimulus size in the better seeing eyes.
Visual field area using V4e stimulus size in the better seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000).
The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Measured: 2 year follow up visit
BCVA - Worse Seeing Eyes
Change (from Baseline) in Best Corrected Visual acuity (BCVA) in the worse seeing eyes.
BCVA in the worse seeing eyes was assessed using the ETRS score (minimum value 0 and maximum value 100, with 0 representing the worst, and 100 the best visual functioning).
The results are reported as the change in the ETDRS score collected at the 1 year follow-up visit compared with the score at the baseline visit.
Measured: 1 year follow up visit
BCVA - Worse Seeing Eyes
Change (from Baseline) in Best Corrected Visual acuity in the worse seeing eyes.
Best corrected visual acuity in the worse seeing eyes was assessed using the ETRS score (minimum value 0 and maximum value 100, with 0 representing the worst, and 100 the best visual functioning).
The results are reported as the change in the ETDRS score collected at the 2 year follow-up visit compared with the score at the baseline visit.
Measured: 2 years follow up visit
III4e Area - Worse Seeing Eyes
Change (from Baseline) in visual field area using III4e stimulus size in the worse-seeing eyes.
Visual field area using III4e stimulus size in the worse-seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000).
The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Measured: 1 year follow up visit
III4e Area - Worse Seeing Eyes
Change (from Baseline) in visual field area using III4e stimulus size in the worse-seeing eyes.
Visual field area using III4e stimulus size in the worse-seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000).
The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Measured: 2 years follow up visit
V4e Area - Worse Seeing Eyes
Change (from Baseline) in visual field area using V4e stimulus size in the worse-seeing eyes.
Visual field area using V4e stimulus size in the worse-seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000).
The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Measured: 1 year follow up visit
V4e Area - Worse Seeing Eyes
Change (from Baseline) in visual field area using V4e stimulus size in the worse-seeing eyes.
Visual field area using V4e stimulus size in the worse-seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000).
The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Measured: 2 years follow up visit
Secondary outcomes (16)
MS - Better Seeing Eyes
Change (from Baseline) in macular sensitivity, assessed by microperimetry, in the better-seeing eyes.
Macular sensitivity, assessed by microperimetry is expressed in decibels (minimum value 0, maximum value 30).
The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Measured: 1 year follow up visit
MS - Better Seeing Eyes
Change (from Baseline) in macular sensitivity, assessed by microperimetry, in the better-seeing eyes.
Macular sensitivity, assessed by microperimetry is expressed in decibels (minimum value 0, maximum value 30).
The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Measured: 2 years follow up visit
CMT - Better Seeing Eyes
Change (from Baseline) in Central Macular Thickness (CMT), evaluated with Optical Coherence tomography, in the better-seeing eyes.
CMT was reported in micrometers (minimum value 0, maximum value 1000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Measured: 1 year follow up visit
CMT - Better Seeing Eyes
Change (from Baseline) in Central Macular Thickness (CMT), evaluated with Optical Coherence tomography, in the better-seeing eyes.
CMT was reported in micrometers (minimum value 0, maximum value 1000). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Measured: 2 years follow up visit
EZ Band Horizontal - Better Seeing Eyes
Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (horizontal scan), in the better-seeing eyes.
EZ Band Horizontal is reported in micrometers (minimum value 0, maximum value 4000).
The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Measured: 1 year follow up visit
EZ Band Horizontal - Better Seeing Eyes
Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (horizontal scan), in the better-seeing eyes.
EZ Band Horizontal is reported in micrometers (minimum value 0, maximum value 4000).
The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Measured: 2 years follow up visit
EZ Band Vertical - Better Seeing Eyes
Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (Vertical scan), in the better-seeing eyes.
EZ Band Vertical is reported in micrometers (minimum value 0, maximum value 4000).
The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Measured: 1 year follow up visit
EZ Band Vertical - Better Seeing Eyes
Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (Vertical scan), in the better-seeing eyes.
EZ Band Vertical is reported in micrometers (minimum value 0, maximum value 4000).
The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Measured: 2 years follow up visit
MS - Worse Seeing Eyes
Change (from Baseline) in macular sensitivity, assessed by microperimetry, in the worse-seeing eyes.
Macular sensitivity, assessed by microperimetry is expressed in decibels (minimum value 0, maximum value 30).
The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Measured: 1 year follow up visit
MS - Worse Seeing Eyes
Change (from Baseline) in macular sensitivity, assessed by microperimetry, in the worse-seeing eyes.
Macular sensitivity, assessed by microperimetry is expressed in decibels (minimum value 0, maximum value 30).
The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Measured: 2 years follow up visit
CMT - Worse Seeing Eyes
Change (from Baseline) in Central Macular Thickness (CMT), evaluated with Optical Coherence tomography, in the worse-seeing eyes.
CMT is expressed in micrometers (minimum value 0, maximum value 1000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Measured: 1 year follow up visit
CMT - Worse Seeing Eyes
Change (from Baseline) in Central Macular Thickness, evaluated with Optical Coherence tomography, in the worse-seeing eyes.
CMT is expressed in micrometers (minimum value 0, maximum value 1000). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Measured: 2 years follow up visit
EZ Band Horizontal - Worse Seeing Eyes
Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (horizontal scan), in the worse-seeing eyes.
EZ Band Horizontal is expressed in micrometers (minimum value 0, maximum value 4000).
The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Measured: 1 year follow up visit
EZ Band Horizontal - Worse Seeing Eyes
Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (horizontal scan), in the worse-seeing eyes.
EZ Band Horizontal is expressed in micrometers (minimum value 0, maximum value 4000).
The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Measured: 2 years follow up visit
EZ Band Vertical - Worse Seeing Eyes
Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (Vertical scan), in the worse-seeing eyes.
EZ Band Vertical is expressed in micrometers (minimum value 0, maximum value 4000).
The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Measured: 1 year follow up visit
EZ Band Vertical - Worse Seeing Eyes
Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (Vertical scan), in the worse-seeing eyes.
EZ Band Vertical is expressed in micrometers (minimum value 0, maximum value 4000).
The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Measured: 2 years follow up visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jan 2019
- Study start1 Jun 2018
- Primary completion30 Dec 2020
- Study completion30 Dec 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Usher Syndrome, Type 1BStudies 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
- Other
- Time perspective
- Prospective
- Enrolment
- 56 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fondazione Telethon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- FRANCESCA SIMONELLI, Principal investigator, Eye Clinic of the University of Campania Luigi Vanvitelli, Naples, ItalyIngeborgh van den Born, Principal investigator, Stichting Oogziekenhuis Rotterdam, Rotterdam, The Netherland.Carmen Ayuso, Principal investigator, Instituto de Investigacion Sanitaria de la Fundacion Jimenez Diaz, Madrid, Spain
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03814499
- Sponsor's protocol ID
- TIGEM3-UshTher-NHS
- Status last verified
- Oct 2024
- First submitted
- 18 Jan 2019
- Last updated
- 9 Dec 2024
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 9 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicentre Longitudinal, Observational Natural History Study to Evaluate Disease Progression in Subjects With Usher Syndrome Type 1B (USH1B)
- ClinicalTrials.gov ID
- NCT03814499
- Acronym / sponsor ID
- TIGEM3-UshTher-NHS
- Conditions
- Usher Syndrome, Type 1B
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 56 participants (actual)
- Lead sponsor
- Fondazione TelethonUniversity, hospital or other
- Study start
- 1 Jun 2018 (actual)
- Primary completion
- 30 Dec 2020 (actual)
- Study completion
- 30 Dec 2022 (actual)
- First posted
- 24 Jan 2019 (actual)
- Results
- Posted 9 Dec 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Natural History Study in Subjects With Usher Syndrome ((USH1B) is a multi-centre, longitudinal, observational study designed to evaluate disease progression in subjects with USH1B by several vision-related assessments.
Detailed description
Natural History Study in Subjects With Usher Syndrome (USH1B) is being conducted to understand the progression of disease in USH1B patients as measured by visual acuity and visual field testing and a number of other vision-related assessments. Disease progression will be evaluated as change over time in these measures and associations between the endpoints will be examined.
The primary objective of this study is to evaluate the natural progression of disease over time in USHIB patients using visual field testing and best corrected visual acuity.
The secondary objectives of this study are to evaluate the progression of disease over time in USHIB patients through additional assessments:
- Microperimetry (only in selected centres)
- Fundus autofluorescence (FAF)
- Optical Coherence Tomography (OCT)
- Full-field Electroretinogram (ERG),
- Multifocal Electroretinogram,
- Vision-related function and quality of life, as measured by the 25-Item National Eye Institute Visual Function Questionnaire (NEI VFQ-25)
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Subjects affected by Usher Syndrome 1B disease fulfilled eligibility criteria
Key inclusion criteria (5 listed)
- Must be willing to adhere to protocol for long-term follow-up as evidenced by written informed consent or parental permission and subject assent.
- Subjects diagnosed with USH1.
- Molecular diagnosis of USH1B due to MYO7A mutations (homozygotes or compound heterozygotes).
- Age eight years old or older at the time of baseline.
- Visual acuity ≥ 20/640 in at least one eye
Key exclusion criteria (8 listed)
- Unable or unwilling to meet requirements of the study.
- Unable to communicate with suitable verbal/auditory and/or tactile sign language (in the opinion of the investigator)
- Participation in a clinical study with an investigational drug in the past six months.
- Pre-existing eye conditions that would interfere with the interpretation of study endpoints (for example, glaucoma, corneal or significant lenticular opacities, cystoid macular oedema, macular hole).
- Complicating systemic diseases in which the disease itself, or the treatment for the disease, can alter ocular function. Examples are malignancies whose treatment could affect central nervous system function (for example, radiation treatment of the orbit; leukemia with CNS/optic nerve involvement). Also excluded would be subjects with immuno- compromising diseases, as there could be susceptibility to opportunistic infection [such as cytomegalovirus (CMV) retinitis].
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
BCVA - Better Seeing Eyes
Time frame: 1 year follow up visit
Change (from Baseline) in Best Corrected Visual acuity (BCVA) in the better-seeing eyes. BCVA in the better seeing eyes was assessed using the ETRS score (minimum value 0 and maximum value 100, with 0 representing the worst, and 100 the best visual functioning). The results are reported as the change in ETDRS score collected at the 1 year follow-up visit compared with the score at the baseline visit.
BCVA - Better Seeing Eyes
Time frame: 2 year follow up visit
Change (from Baseline) in Best Corrected Visual acuity (BCVA) in the better-seeing eyes. BCVA in the better seeing eyes was assessed using the ETRS score (minimum value 0 and maximum value 100, with 0 representing the worst, and 100 the best visual functioning). The results are reported as the change in ETDRS score collected at the 2 year follow-up visit compared with the score at the baseline visit.
III4e Area - Better Seeing Eyes
Time frame: 1 year follow up visit
Change (from Baseline) in visual field area using III4e stimulus size in the better seeing eyes. Visual field area using III4e stimulus size in the better seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
III4e Area - Better Seeing Eyes
Time frame: 2 year follow up visit
Change (from Baseline) in visual field area using III4e stimulus size in the better seeing eyes. Visual field area using III4e stimulus size in the better seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
V4e Area - Better Seeing Eyes
Time frame: 1 year follow up visit
Change (from Baseline) in visual field area using V4e stimulus size in the better seeing eyes. Visual field area using V4e stimulus size in the better seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
V4e Area - Better Seeing Eyes
Time frame: 2 year follow up visit
Change (from Baseline) in visual field area using V4e stimulus size in the better seeing eyes. Visual field area using V4e stimulus size in the better seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
BCVA - Worse Seeing Eyes
Time frame: 1 year follow up visit
Change (from Baseline) in Best Corrected Visual acuity (BCVA) in the worse seeing eyes. BCVA in the worse seeing eyes was assessed using the ETRS score (minimum value 0 and maximum value 100, with 0 representing the worst, and 100 the best visual functioning). The results are reported as the change in the ETDRS score collected at the 1 year follow-up visit compared with the score at the baseline visit.
BCVA - Worse Seeing Eyes
Time frame: 2 years follow up visit
Change (from Baseline) in Best Corrected Visual acuity in the worse seeing eyes. Best corrected visual acuity in the worse seeing eyes was assessed using the ETRS score (minimum value 0 and maximum value 100, with 0 representing the worst, and 100 the best visual functioning). The results are reported as the change in the ETDRS score collected at the 2 year follow-up visit compared with the score at the baseline visit.
III4e Area - Worse Seeing Eyes
Time frame: 1 year follow up visit
Change (from Baseline) in visual field area using III4e stimulus size in the worse-seeing eyes. Visual field area using III4e stimulus size in the worse-seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
III4e Area - Worse Seeing Eyes
Time frame: 2 years follow up visit
Change (from Baseline) in visual field area using III4e stimulus size in the worse-seeing eyes. Visual field area using III4e stimulus size in the worse-seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
V4e Area - Worse Seeing Eyes
Time frame: 1 year follow up visit
Change (from Baseline) in visual field area using V4e stimulus size in the worse-seeing eyes. Visual field area using V4e stimulus size in the worse-seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
V4e Area - Worse Seeing Eyes
Time frame: 2 years follow up visit
Change (from Baseline) in visual field area using V4e stimulus size in the worse-seeing eyes. Visual field area using V4e stimulus size in the worse-seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
16 secondary outcomes
MS - Better Seeing Eyes
Time frame: 1 year follow up visit
MS - Better Seeing Eyes
Time frame: 2 years follow up visit
CMT - Better Seeing Eyes
Time frame: 1 year follow up visit
CMT - Better Seeing Eyes
Time frame: 2 years follow up visit
EZ Band Horizontal - Better Seeing Eyes
Time frame: 1 year follow up visit
EZ Band Horizontal - Better Seeing Eyes
Time frame: 2 years follow up visit
EZ Band Vertical - Better Seeing Eyes
Time frame: 1 year follow up visit
EZ Band Vertical - Better Seeing Eyes
Time frame: 2 years follow up visit
MS - Worse Seeing Eyes
Time frame: 1 year follow up visit
MS - Worse Seeing Eyes
Time frame: 2 years follow up visit
CMT - Worse Seeing Eyes
Time frame: 1 year follow up visit
CMT - Worse Seeing Eyes
Time frame: 2 years follow up visit
EZ Band Horizontal - Worse Seeing Eyes
Time frame: 1 year follow up visit
EZ Band Horizontal - Worse Seeing Eyes
Time frame: 2 years follow up visit
EZ Band Vertical - Worse Seeing Eyes
Time frame: 1 year follow up visit
EZ Band Vertical - Worse Seeing Eyes
Time frame: 2 years follow up visit
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Dec 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
FRANCESCA SIMONELLI principal investigator
Eye Clinic of the University of Campania Luigi Vanvitelli, Naples, Italy
Ingeborgh van den Born principal investigator
Stichting Oogziekenhuis Rotterdam, Rotterdam, The Netherland.
Carmen Ayuso principal investigator
Instituto de Investigacion Sanitaria de la Fundacion Jimenez Diaz, Madrid, Spain
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Usher Syndrome, Type 1B, run by Fondazione Telethon at 3 sites in 3 countries.
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 5 inclusion and 8 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: finished, as of the sponsor's last update on 9 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Italy, Netherlands, Spain. 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?
Yes. Results are posted in the registry (first posted 9 Dec 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
BCVA - Better Seeing Eyes (time frame: 1 year follow up visit). The study lists 12 primary outcomes in all. 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 9 Dec 2024.