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Clinical trials for Congenital Blindness

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43881   clinical trials with a EudraCT protocol, of which   7295   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
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    30 result(s) found for: Congenital Blindness. Displaying page 1 of 2.
    1  2  Next»
    EudraCT Number: 2018-003500-40 Sponsor Protocol Number: PQ-110-002 Start Date*: 2019-04-08
    Sponsor Name:ProQR Therapeutics IV B.V.
    Full Title: An Open-Label Extension Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of QR-110 in Subjects with Leber Congenital Amaurosis (LCA) due to the C.2991+1655a>G Mutation (P....
    Medical condition: Leber Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10070667 Leber's congenital amaurosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2020-000535-45 Sponsor Protocol Number: PQ-110-005 Start Date*: 2020-11-13
    Sponsor Name:ProQR Therapeutics IV B.V.
    Full Title: An Open-Label, Dose Escalation and Double-Masked, Randomized, Controlled Study to Evaluate the Safety and Tolerability of Sepofarsen in Pediatric Subjects <8 Years of Age with Leber Congenital Amau...
    Medical condition: Leber Congenital Amaurosis 10 (LCA10) due to c.2991+1655A>G mutation (p.Cys998X) in the CEP290 Gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10070667 Leber's congenital amaurosis PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: BE (Completed) DE (Completed) GB (GB - no longer in EU/EEA) NL (Ongoing) IT (Ongoing)
    Trial results: View results
    EudraCT Number: 2016-002109-20 Sponsor Protocol Number: AAV2-hRPE65v2-301 Start Date*: 2016-10-24
    Sponsor Name:Spark Therapeutics, Inc.
    Full Title: A Safety and Efficacy Study in Subjects with Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 to the Retinal Pigment Epithelium (RPE) [AAV2-h...
    Medical condition: Leber Congenital Amaurosis (LCA)
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10070667 Leber's congenital amaurosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2017-000813-22 Sponsor Protocol Number: PQ-110-001 Start Date*: 2017-06-19
    Sponsor Name:ProQR Therapeutics
    Full Title: An Open-Label, Single Arm, Multiple Dose, Dose Escalation Study to Evaluate the Safety and Tolerability of QR-110 in Subjects with Leber’s Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation ...
    Medical condition: Leber’s Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10070667 Leber's congenital amaurosis PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2015-003418-25 Sponsor Protocol Number: CTU/2014/120 Start Date*: 2016-03-29
    Sponsor Name:MeiraGTx UK II Ltd
    Full Title: An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno-Associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults and Children with Retinal Dystrophy associated with D...
    Medical condition: Leber Congenital Amaurosis (LCA) caused by mutations in RPE65
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10070667 Leber's congenital amaurosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2018-003501-25 Sponsor Protocol Number: PQ-110-003 Start Date*: 2019-02-28
    Sponsor Name:ProQR Therapeutics IV B.V.
    Full Title: A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety, Tolerability and Systemic Exposure of QR-110 in Subjects with Leber’s Congenital Amaurosis (LCA) due t...
    Medical condition: Leber’s Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10070667 Leber's congenital amaurosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) NL (Ongoing) BE (Completed) FR (Ongoing) GB (GB - no longer in EU/EEA) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2013-005393-22 Sponsor Protocol Number: RETIRD04 Start Date*: 2016-11-03
    Sponsor Name:QLT Inc.
    Full Title: A Study of the Efficacy and Safety of QLT091001 in Subjects with Inherited Retinal Disease (IRD) Caused by Mutation in Retinal Pigment Epithelium Protein 65 (RPE65) or Lecithin:Retinol Acyltransfer...
    Medical condition: Inherited retinal disease (IRD) phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by mutations in the retinal pigment epithelium protein 65 (RPE65) or...
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa PT
    19.0 10010331 - Congenital, familial and genetic disorders 10070667 Leber's congenital amaurosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DK (Prematurely Ended) NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2011-004214-42 Sponsor Protocol Number: RETIRD02 Start Date*: 2012-09-03
    Sponsor Name:QLT Inc.
    Full Title: An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due ...
    Medical condition: Leber Congenital Amaurosis Retinitis pigmentosa
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10070667 Leber's congenital amaurosis PT
    20.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) NL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2014-003671-30 Sponsor Protocol Number: VHL-HOPE-2014-1 Start Date*: 2014-10-31
    Sponsor Name:ALIANZA ESPAÑOLA DE FAMILIAS DE VON HIPPEL-LINDAU
    Full Title: Therapeutic effect of propranolol in a series of patients with von Hippel-Lindau disease and retinal hemangioblastomas in short, medium and long term treatment.
    Medical condition: VON HIPPEL-LINDAU DISEASE
    Disease:
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Completed)
    Trial results: View results
    EudraCT Number: 2017-002124-24 Sponsor Protocol Number: ROPROP Start Date*: 2017-12-22
    Sponsor Name:Universität Zürich
    Full Title: Oral Propranolol for prevention of threshold retinopathy of prematurity
    Medical condition: Retinopathy of prematurity
    Disease:
    Population Age: Preterm newborn infants, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: DE (Restarted)
    Trial results: View results
    EudraCT Number: 2012-001990-95 Sponsor Protocol Number: LTS13588-(SG1/002/11) Start Date*: Information not available in EudraCT
    Sponsor Name:sanofi-aventis recherche et développement
    Full Title: An Open Label Study to Determine the Long Term Safety, Tolerability and Biological Activity of SAR422459 in Patients with Stargardt’s Macular Degeneration
    Medical condition: Stargardt Macular Dystrophy, also known as fundus flavimaculatus or Stargardt disease.
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10062766 Stargardt's disease PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-005039-94 Sponsor Protocol Number: IC2019-05 Start Date*: 2020-12-08
    Sponsor Name:Institut Curie
    Full Title: RETINO 2018 : Ocular conservative treatment for retinoblastoma: efficacy of the new management strategies and visual outcome.
    Medical condition: Retinoblastoma
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Newborns, Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2011-001953-10 Sponsor Protocol Number: ALD-102 Start Date*: 2013-12-13
    Sponsor Name:bluebird bio, Inc.
    Full Title: A phase 2/3 study of the efficacy and safety of hematopoietic stem cells transduced with Lenti D lentiviral vector for the treatment of cerebral adrenoleukodystrophy (CALD)
    Medical condition: Cerebral Adrenoleukodystrophy (CALD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10051260 Adrenoleukodystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Completed) DE (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2017-004972-74 Sponsor Protocol Number: 20160227 Start Date*: 2020-09-15
    Sponsor Name:Amgen Inc.
    Full Title: An Open-label, Ascending Multiple dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Romosozumab in Children and Adolescents With Osteogenesis Imperfecta
    Medical condition: Osteogenesis imperfecta (OI) is a group of genetic skeletal disorders characterized by increased bone fragility, low bone mass , and increased bone turnover contributing to osteoporosis, fracture...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10031243 Osteogenesis imperfecta PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: HU (Completed) DE (Completed) GR (Completed) IT (Ongoing) AT (Completed)
    Trial results: View results
    EudraCT Number: 2022-001092-14 Sponsor Protocol Number: TIGEM5_USH Start Date*: 2023-10-12
    Sponsor Name:FONDAZIONE TELETHON
    Full Title: A phase I/II open label, dose escalation study of sub-retinal administration of a “mixture of two adeno-associated viral vectors of serotype 8 containing the 5 ´- half sequence of the human MYO7A ...
    Medical condition: Usher syndrome (USH) is characterized by the association of sensorineural hearing loss, Retinitis Pigmentosa (RP), and, in some cases, vestibular dysfunction. It is the most frequent cause of deaf-...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10063396 Usher's syndrome PT
    20.0 10010331 - Congenital, familial and genetic disorders 10063396 Usher's syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-006456-14 Sponsor Protocol Number: K321301 Start Date*: 2023-05-10
    Sponsor Name:Kowa Research Institute, Inc.
    Full Title: A Double-Masked, Randomized, Placebo-Controlled, Parallel-Group, 12-Week Administration With Two-Week Gradual Dose Taper Phase and 38-Week Follow-Up Phase, Phase 3 Study to Investigate the Safety ...
    Medical condition: In Fuchs endothelial corneal dystrophy (FECD), there is an increased rate of loss of endothelial cells, starting in the centre of Descemet’s membrane and spreading to the periphery, resulting in ex...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10011005 Corneal dystrophy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Ongoing) DK (Ongoing) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-003967-21 Sponsor Protocol Number: MGT009 Start Date*: 2017-06-09
    Sponsor Name:MeiraGTx UK II Limited
    Full Title: An open label, multi-centre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/5-hRKp.RPGR) for gene therapy of adults and children with X-linked Retinitis Pigme...
    Medical condition: X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2019-003280-22 Sponsor Protocol Number: K-321-201 Start Date*: 2020-04-29
    Sponsor Name:Kowa Research Institute, Inc.
    Full Title: A Double-Masked, Randomised, Placebo-Controlled, Parallel-Group, 12-Week, Phase 2 Study to Investigate the Safety and Efficacy of Ripasudil (K-321) Eye Drops After Descemetorhexis in Patients wit...
    Medical condition: In Fuchs endothelial corneal dystrophy (FECD), there is an increased rate of loss of endothelial cells, starting in the centre of Descemet’s membrane and spreading to the periphery, resulting in ex...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10011005 Corneal dystrophy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) DK (Completed)
    Trial results: (No results available)
    EudraCT Number: 2018-000425-31 Sponsor Protocol Number: MGT010 Start Date*: 2018-08-30
    Sponsor Name:MeiraGTx UK II Ltd
    Full Title: Long term follow-up study of participants following an open label, multicentre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/5-hRKp.RPGR) for gene therapy o...
    Medical condition: X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) Outside EU/EEA
    Trial results: (No results available)
    EudraCT Number: 2014-000184-40 Sponsor Protocol Number: 20130173 Start Date*: 2014-09-11
    Sponsor Name:Amgen, Inc.
    Full Title: Prospective, Multicenter, Single-arm Study to Evaluate Efficacy, Safety, and Pharmacokinetics of Denosumab in Children With Osteogenesis Imperfecta
    Medical condition: Osteogenesis Imperfecta
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10031243 Osteogenesis imperfecta PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: CZ (Completed) DE (Completed) GB (GB - no longer in EU/EEA) HU (Completed) ES (Completed) Outside EU/EEA BG (Completed) PL (Prematurely Ended) BE (Completed) FR (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
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