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Clinical trials for Dysgenesis

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   44336   clinical trials with a EudraCT protocol, of which   7366   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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    15 result(s) found for: Dysgenesis. Displaying page 1 of 1.
    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: 2004-005054-31 Sponsor Protocol Number: 24531 Start Date*: 2005-07-15
    Sponsor Name:Serono International S.A.
    Full Title: A phase IV open-label study of predictive markers in Growth Hormone Deficient and Turner Syndrome prepubertal children treated with Saizen
    Medical condition: - growth failure in children caused by decreased or absent secretion of endogenous growth hormone. - growth failure in girls with gonadal dysgenesis (Turner Syndrome), confirmed by chromosomal anal...
    Disease: Version SOC Term Classification Code Term Level
    7.1 10056438 PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) FI (Completed) ES (Completed) DE (Completed) AT (Completed) SE (Completed) IT (Completed)
    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: 2020-002924-35 Sponsor Protocol Number: APHP180592 Start Date*: 2021-01-18
    Sponsor Name:ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS
    Full Title: Prenatal treatment of congenital cytomegalovirus infection with letermovir randomized against valaciclovir
    Medical condition: Step 1: Maternal administration of 1 tablet of Letermovir (240 mg or 480 mg /day) during 3 days before TOP Step 2: Maternal daily administration of 240 or 480 milligrams of letermovir (1x240 mg-ta...
    Disease:
    Population Age: Children, Under 18, Adults Gender: Female
    Trial protocol: FR (Trial now transitioned)
    Trial results: (No results available)
    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 (Completed)
    Trial results: View results
    EudraCT Number: 2019-001881-14 Sponsor Protocol Number: 20072650 Start Date*: 2021-03-19
    Sponsor Name:Department of Endocrinology and Internal Medicine, Aarhus University Hospital
    Full Title: Endocrine, cardiovascular, pharmacologic and physiologic aspects of sex hormone treatment in Turner syndrome - project part 1
    Medical condition: Ovarian dysgenesis and related hypogonadism caused by Turner syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10045181 Turner's syndrome PT
    Population Age: Adults Gender: Female
    Trial protocol: DK (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-004934-40 Sponsor Protocol Number: 20072651 Start Date*: 2021-03-19
    Sponsor Name:Department of Endocrinology and Internal Medicine, Aarhus University Hospital
    Full Title: Endocrine, cardiovascular, pharmacologic and physiologic aspects of sex hormone treatment in Turner syndrome - project part B
    Medical condition: Ovarian dysgenesis and related hypogonadism causes by Turner syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10045181 Turner's syndrome PT
    Population Age: Adults Gender: Female
    Trial protocol: DK (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2013-004478-80 Sponsor Protocol Number: Issue1 Start Date*: 2014-01-08
    Sponsor Name:University Hospital Bristol NHS Foundation Trust
    Full Title: XENON AND COOLING THERAPY IN BABIES AT HIGH RISK OF BRAIN INJURY FOLLOWING POOR CONDITION AT BIRTH: A RANDOMISED OUTCOME STUDY (COOLXENON3 STUDY)
    Medical condition: Neonatal hypoxia-ischaemia (HI). There is no exact MedDRA term for this. The closest MedDRA terms matching this are neonatal asphyxia and neonatal hypoxia.
    Disease: Version SOC Term Classification Code Term Level
    16.1 10038738 - Respiratory, thoracic and mediastinal disorders 10028923 Neonatal asphyxia PT
    16.1 100000004868 10028946 Neonatal hypoxia and asphyxia HLT
    16.1 10038738 - Respiratory, thoracic and mediastinal disorders 10050081 Neonatal hypoxia PT
    Population Age: Newborns, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2009-014260-19 Sponsor Protocol Number: CH/2009/3187 Start Date*: 2010-01-29
    Sponsor Name:University Hospital Bristol NHS Foundation Trust
    Full Title: A feasibility study of adding xenon to cooling therapy in babies at high risk of brain injury following poor condition at birth
    Medical condition: Neonatal hypoxia-ischaemia (HI). There is no exact MedDRA term for this. The closest MedDRA terms matching this are neonatal asphyxia and neonatal hypoxia.
    Disease: Version SOC Term Classification Code Term Level
    12 10028923 Neonatal asphyxia LLT
    12 10028923 Neonatal asphyxia PT
    12 10028946 Neonatal hypoxia and asphyxia HLT
    12 10050081 Neonatal hypoxia LLT
    12 10050081 Neonatal hypoxia PT
    Population Age: Newborns, Under 18 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: 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: 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: 2011-005397-34 Sponsor Protocol Number: Issue1 Start Date*: 2012-02-24
    Sponsor Name:University Hospital Bristol NHS Foundation Trust
    Full Title: Xenon and cooling therapy in babies at high risk of brain injury following poor condition at birth: Randomised pilot study. (CoolXenon2 study)
    Medical condition: Neonatal hypoxia-ischaemia (HI). There is no exact MedDRA term for this. The closest MedDRA terms matching this are neonatal asphyxia and neonatal hypoxia.
    Disease: Version SOC Term Classification Code Term Level
    14.1 10038738 - Respiratory, thoracic and mediastinal disorders 10050081 Neonatal hypoxia PT
    14.1 10036585 - Pregnancy, puerperium and perinatal conditions 10028946 Neonatal hypoxia and asphyxia HLT
    14.1 10038738 - Respiratory, thoracic and mediastinal disorders 10028923 Neonatal asphyxia PT
    Population Age: Newborns, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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