NCT03814499 is a clinical trial in Usher Syndrome, Type 1B, sponsored by Fondazione Telethon.

Who is sponsoring NCT03814499?

NCT03814499 is sponsored by Fondazione Telethon.

What condition does NCT03814499 study?

NCT03814499 studies Usher Syndrome, Type 1B.

Is NCT03814499 still recruiting?

NCT03814499 is currently completed. Last updated 9 December 2024.

Are results published for NCT03814499?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2024-12-09 · How we verify

NCT03814499

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Natural History Study in Subjects With Usher Syndrome

Completed Results posted Last updated 9 December 2024

What this trial tests

trial in Usher Syndrome, Type 1B in 56 participants. Completed in 30 December 2022.

Timeline

1 June 2018

Primary endpoint
30 December 2020

30 December 2022

Quick facts

Lead sponsor	Fondazione Telethon
Status	Completed
Study type	OBSERVATIONAL
Enrollment	56
Start date	1 June 2018
Primary completion	30 December 2020
Estimated completion	30 December 2022
Sites	3 locations across Italy, Netherlands, Spain

Conditions studied

Usher Syndrome, Type 1B — all drugs for Usher Syndrome, Type 1B →

Sponsor

Fondazione Telethon — full company profile →

Who can join

8 and older, any sex, with Usher Syndrome, Type 1B. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

BCVA - Better Seeing Eyes Primary · 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.

Group	Value	95% CI
Group of Subjects With Usher Syndrome Type 1B	-2.26	± 3.838

BCVA - Better Seeing Eyes Primary · 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.

Group	Value	95% CI
Group of Subjects With Usher Syndrome Type 1B	-1.468	± 4.999

III4e Area - Better Seeing Eyes Primary · 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.

Group	Value	95% CI
Group of Subjects With Usher Syndrome Type 1B	561.966	± 2043.612

III4e Area - Better Seeing Eyes Primary · 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.

Group	Value	95% CI
Group of Subjects With Usher Syndrome Type 1B	70.286	± 1451.128

V4e Area - Better Seeing Eyes Primary · 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.

Group	Value	95% CI
Group of Subjects With Usher Syndrome Type 1B	273.309	± 2432.785

V4e Area - Better Seeing Eyes Primary · 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.

Group	Value	95% CI
Group of Subjects With Usher Syndrome Type 1B	700.245	± 3942.6

BCVA - Worse Seeing Eyes Primary · 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.

Group	Value	95% CI
Group of Subjects With Usher Syndrome Type 1B	-0.625	± 5.35

BCVA - Worse Seeing Eyes Primary · 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.

Group	Value	95% CI
Group of Subjects With Usher Syndrome Type 1B	-0.909	± 7.041

III4e Area - Worse Seeing Eyes Primary · 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.

Group	Value	95% CI
Group of Subjects With Usher Syndrome Type 1B	-445.569	± 2920.491

III4e Area - Worse Seeing Eyes Primary · 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.

Group	Value	95% CI
Group of Subjects With Usher Syndrome Type 1B	-407.928	± 3209.147

V4e Area - Worse Seeing Eyes Primary · 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.

Group	Value	95% CI
Group of Subjects With Usher Syndrome Type 1B	391.212	± 2726.331

V4e Area - Worse Seeing Eyes Primary · 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.

Group	Value	95% CI
Group of Subjects With Usher Syndrome Type 1B	610.017	± 5658.524

Sponsor's own description

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.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

Usher syndrome: clinical features, molecular genetics and advancing therapeutics.
Toms M, Pagarkar W, Moosajee M. · · 2020 · cited 69× · PMID 32995707 · DOI 10.1177/2515841420952194
Multicentric Longitudinal Prospective Study in a European Cohort of MYO7A Patients: Disease Course and Implications for Gene Therapy.
Testa F, Carreño E, van den Born LI, Melillo P, et al · · 2024 · cited 7× · PMID 38884554 · DOI 10.1167/iovs.65.6.25

Verify or expand the search:

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Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT03814499 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Fondazione Telethon
Last refreshed: 9 December 2024

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03814499.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing