13 completed, 7 recruiting, 2 unknown, 1 withdrawn. 6 Not applicable, 3 Phase 2, 2 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 6 | Completed 5, Recruiting 1 |
| Phase 2 | 3 | Completed 2, Withdrawn 1 |
| Phase 1 | 2 | Completed 2 |
Follow Aniridia: new trials by email Literature Review of Aniridia trials Research Report on Aniridia
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Physical Activity and Community EmPOWERment Project PACE Program - Intellectual Disability, Neurodevelopmental Disorders | Not applicable | Recruiting | University of North Carolina, Chapel Hill | 376 | 2025-01-10 | 2026-02-23 |
| Modeling Ocular Developmental Diseases From 3D Cultures of Optic Vesicle Organoids Derived From hiPSCs of Patients With Ocular Malformations Blood sampling - Eye Abnormalities | Recruiting | University Hospital, Toulouse | 20 | 2024-11-05 | 2026-03-18 | |
| National Ophthalmic Genotyping and Phenotyping Network (eyeGENE (Registered Trademark)), Stage 3 - Expansion of DNA and Data Repositories for Rare Inherited Ophthalmic Diseases Inherited Ophthalmic Diseases, Hypopigmentation Disorder | Recruiting | National Eye Institute (NEI) | 1000 | 2024-07-12 | 2026-06-09 | |
| Validation of Human Drugs Target of Repurposed Drugs and Novel Therapies Rare Diseases, Aniridia | Unknown | IRCCS Ospedale San Raffaele | 274 | 2024-05-01 | 2024-05-14 | |
| Treatment of LSCD With DM transplantation of a Descemet's Membrane corneal onlay, partial LSCD, transplantation of a Descemet's Membrane corneal onlay, total/near-total LSCD - Limbal Stem-cell Deficiency, Congenital Aniridia | Phase 1 | Completed | University of Minnesota | 19 | 2023-11-30 | 2026-01-08 |
| Congenital Aniridia Patient Questionnaire Survey - Congenital Aniridia | Recruiting | Assistance Publique - Hôpitaux de Paris | 100 | 2023-06-08 | 2026-06-02 | |
| Comparison of the Healing Properties on Corneal Cells of Groth Factor-enriched Plasma and Autologous Serum From Aniridia Patients Peripheral venous blood sample, Conjunctival impression - Aniridia | Recruiting | Fondation Ophtalmologique Adolphe de Rothschild | 24 | 2023-05-15 | 2025-12-08 | |
| Proteomic Study of Tears From Patients With a PAX6 Mutation Tear collection - Aniridia | Not applicable | Completed | University Hospital, Montpellier | 5 | 2023-02-09 | 2024-08-09 |
| RAFT - Clinical Trial of RAFT for Aniridia Related Keratopathy RAFT-OS - Aniridia | Phase 1 / Phase 2 | Completed | University College, London | 9 | 2021-07-28 | 2025-04-30 |
| A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia Ataluren - Aniridia | Phase 2 | Withdrawn | PTC Therapeutics | 2018-12-31 | 2019-10-07 | |
| Ultrahigh-resolution Optical Coherence Tomography Imaging of the Anterior Eye Segment Structures Ultrahigh resolution Spectral Domain OCT - Meibomian Gland Dysfunction, Cataract | Not applicable | Completed | Medical University of Vienna | 14 | 2017-07-12 | 2025-05-23 |
| National Cohort on Congenital Defects of the Eye Anophthalmia, Microphthalmia | Recruiting | Institut National de la Santé Et de la Recherche Médicale, France | 800 | 2017-07-11 | 2026-07-07 | |
| Study of Ataluren in Participants With Nonsense Mutation Aniridia Ataluren, Placebo - Aniridia | Phase 2 | Completed | PTC Therapeutics | 39 | 2016-01-31 | 2022-05-27 |
| Safety and Performance Study of the ARGOS-IO System in Patients Undergoing Boston Keratoprosthesis Implantation ARGOS-IO system - Graft vs Host Disease, Congenital Aniridia | Not applicable | Completed | Implandata Ophthalmic Products GmbH | 13 | 2015-03-01 | 2025-02-27 |
| Positive Angle Kappa Aniridia | Completed | University of Minnesota | 16 | 2010-08-01 | 2019-11-01 | |
| Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford Rare Disorders, Undiagnosed Disorders | Recruiting | Sanford Health | 20000 | 2010-07-01 | 2026-10-28 | |
| Characterization of WAGR Syndrome and Other Chromosome 11 Gene Deletions WAGR Syndrome, Wilm's Tumor | Completed | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 197 | 2008-09-11 | 2019-12-12 | |
| Cultivated Stem Cell Transplantation for the Treatment of Limbal Stem Cell Deficiency Cultivated limbal stem cell graft transplantation - Limbal Stem Cell Deficiency | Not applicable | Completed | Veerle Van Gerwen | 21 | 2008-07-01 | 2026-06-18 |
| Clinical Outcomes of Implantationof Black Diaphragm Intraocular Lens in Complete Aniridia and Aphakia Due to Posttraumatic Eye Rupture Aphakia Due to Trauma, Eye; Rupture, Traumatic, With Loss of Intraocular Tissue | Completed | Medical University of Lublin | 14 | 2006-01-01 | 2018-07-10 | |
| Correlation of Gene Abnormalities and Clinical Manifestations of Aniridia Aniridia | Completed | National Eye Institute (NEI) | 100 | 2005-12-07 | 2017-07-02 |
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Limbal Stem Cell Deficiency (4) · Axenfeld-Rieger syndrome (3) · Peters anomaly (3) · WAGR Syndrome (3) · Anophthalmos (2) · Congenital Abnormalities (2) · De Lange Syndrome (2) · Eye Abnormalities (2) · Glaucoma (2) · Intellectual Disability (2) · Microphthalmos (2) · Rare Diseases (2) · Stevens-Johnson Syndrome (2) · Urogenital Abnormalities (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (23) · Eye Diseases (23) · Eye Diseases, Hereditary (23) · Genetic Diseases, Inborn (23) · Iris Diseases (23) · Uveal Diseases (23) · Congenital Abnormalities (21) · Eye Abnormalities (21) · Corneal Diseases (7) · Pathological Conditions, Signs and Symptoms (5) · Skin Diseases (5) · Skin and Connective Tissue Diseases (5) · Female Urogenital Diseases (4) · Female Urogenital Diseases and Pregnancy Complications (4) · Kidney Diseases (4)
Blood Specimen Collection (2) · Surveys and Questionnaires (2) · ataluren (2)
National Eye Institute (NEI) (3) · PTC Therapeutics (2) · University of Minnesota (2) · Assistance Publique - Hôpitaux de Paris (1) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (1) · Fondation Ophtalmologique Adolphe de Rothschild (1) · IRCCS Ospedale San Raffaele (1) · Implandata Ophthalmic Products GmbH (1) · Institut National de la Santé Et de la Recherche Médicale, France (1) · M.D. Anderson Cancer Center (1) · Medical University of Lublin (1) · Medical University of Vienna (1) · Sanford Health (1) · University College, London (1) · University Hospital, Montpellier (1)
United States (11) · France (5) · Austria (1) · Belgium (1) · Canada (1) · Germany (1) · Poland (1) · United Kingdom (1)
Alejandra Daruich, MD, PhD (1) · Brian P Brooks, M.D. (1) · Brianne R Tomaszewski, PhD., MPH (1) · Carina Koppen, MD, PhD (1) · Dominique Bremond-Gignac, MD, PhD (1) · Jack A Yanovski, M.D. (1) · Julie Daniels (1) · Julie Plaisancie, MD, PhD (1) · Kevin M Miller, MD (1) · Louise C. Strong, MD (1) · Marie J Tassignon, MD, PhD (1) · Nadia Zakaria, MBBS, PhD (1)
PMID 18812117, PMID 26603402, PMID 27373394, PMID 29515428, PMID 29718281, PMID 30041739, PMID 30855519, PMID 42204496, PMID 42490097
P01CA034936 (1), R01AG076678 (1)