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Clinical trials

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   44381   clinical trials with a EudraCT protocol, of which   7393   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 ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
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    1,225 result(s) found. Displaying page 12 of 62.
    «« First « Previous 8  9  10  11  12  13  14  15  16  Next» Last»»
    EudraCT Number: 2015-003108-22 Sponsor Protocol Number: PAR-C14-007 Start Date*: 2015-12-08
    Sponsor Name:NPS Pharmaceuticals, Inc.
    Full Title: An Open-label, Single-dose Study to Determine the Pharmacokinetic/Pharmacodynamic Profile of Parathyroid Hormone (rDNA) Administered Subcutaneously at a Dose of 50 µg in Subjects with Hypoparathyr...
    Medical condition: Chronic hypoparathyroidism
    Disease:
    Population Age: Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2013-001045-14 Sponsor Protocol Number: SAF001 Start Date*: 2013-04-22
    Sponsor Name:Promethera Biosciences
    Full Title: SAF 001: A long-term safety follow-up study of patients suffering from Urea Cycle disorders (UCD) or Crigler-Najjar Syndrome (CN) having received infusions of HepaStem.
    Medical condition: Crigler-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucuronosyltransferase activity and is apparent during the neonatal period by intense jaundice. The ur...
    Disease: Version SOC Term Classification Code Term Level
    15.1 100000004850 10021601 Inborn error of metabolism NOS LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: BE (Completed) PT (Completed) IT (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2021-001535-99 Sponsor Protocol Number: CCTL019BUS03 Start Date*: 2022-03-14
    Sponsor Name:Novartis Pharmaceuticals Corporation
    Full Title: A phase II, open label, multi-center trial to determine the efficacy and safety of tisagenlecleucel re-infusion in Pediatric and Adolescent Young Adult (AYA) patients with acute lymphoblastic leuke...
    Medical condition: Paediatric patients with B-cell acute lymphoblastic leukaemia
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004864 10000845 Acute lymphoblastic leukemia LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2019-001767-67 Sponsor Protocol Number: PTC124-GD-045-DMD Start Date*: 2020-01-30
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: Phase 2 Clinical Pharmacology Study to Assess Dystrophin Levels in Subjects With nmDMD Before and After Treatment with Ataluren
    Medical condition: Non-Sense Mutation Duchenne Muscular Dystrophy (nmDMD)
    Disease:
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2021-005404-36 Sponsor Protocol Number: SHP633-305 Start Date*: 2021-10-19
    Sponsor Name:Shire
    Full Title: A Prospective, Open-label, Long-term Safety and Efficacy Study of Teduglutide in Japanese Pediatric Subjects with Short Bowel Syndrome Who Completed SHP633-302
    Medical condition: Treatment of short bowel syndrome
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2019-001691-11 Sponsor Protocol Number: PTC124-GD-046-DMD Start Date*: 2020-03-06
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: Phase 2, Non-Interventional, Clinical Study to Assess Dystrophin Levels in Subjects With Nonsense Mutation Duchenne Muscular Dystrophy Who Have Been Treated With Ataluren for ≥9 Months
    Medical condition: Non-Sense Mutation Duchenne Muscular Dystrophy (nmDMD)
    Disease:
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2020-003678-28 Sponsor Protocol Number: AVXS-101-CL-102 Start Date*: 2020-09-15
    Sponsor Name:AveXis, Inc.
    Full Title: Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting but Non- Ambulatory Patients with Spinal Muscular Atrophy
    Medical condition: Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy
    Disease:
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2008-008228-34 Sponsor Protocol Number: DPM-PK-102 Start Date*: 2009-01-28
    Sponsor Name:Pharmaxis Ltd
    Full Title: Determination of the pharmacokinetics of inhaled mannitol after single and multiple dosing in cystic fibrosis patients
    Medical condition: Cystic Fibrosis
    Disease: Version SOC Term Classification Code Term Level
    8.1 10011762 Cystic fibrosis LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2009-012564-13 Sponsor Protocol Number: Cystadrops®/09/choc-study Start Date*: 2013-01-03
    Sponsor Name:Orphan Europe SARL
    Full Title: Cysteamine Hydrochloride for nephropathic Cystinosis, open-label Phase III pivotal study
    Medical condition: Nephropatic cystinosis patients with cystine corneal deposits
    Disease: Version SOC Term Classification Code Term Level
    16.0 100000004850 10071112 Nephropathic cystinosis LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2021-005552-11 Sponsor Protocol Number: LTS12869 Start Date*: 2022-03-07
    Sponsor Name:Genzyme Corporation
    Full Title: A Long-term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Alglucosidase Alfa.
    Medical condition: Pompe disease
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10053185 Glycogen storage disease type II PT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2022-001013-39 Sponsor Protocol Number: PTC124-GD-028-ANI Start Date*: 2022-04-14
    Sponsor Name:PTC Therapeutics, Inc
    Full Title: A Phase 2, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study of the Safety and Efficacy of Ataluren (PTC124) for the Treatment of Nonsense Mutation Aniridia
    Medical condition: Nonsense Mutation Aniridia
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2011-003073-28 Sponsor Protocol Number: 8HA02PED Start Date*: 2012-05-31
    Sponsor Name:Biogen Idec Research Ltd
    Full Title: An Open-Label, Multicenter Evaluation of Safety, Pharmacokinetics, and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein, BIIB031, in the Prevention and Treatment of Bleeding Episod...
    Medical condition: Severe Hemophilia A
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004850 10053753 Hemophilia A without inhibitors LLT
    14.1 100000004850 10060612 Hemophilia A LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: GB (Completed) IE (Completed) NL (Completed) PL (Completed)
    Trial results: View results
    EudraCT Number: 2007-003569-42 Sponsor Protocol Number: 20060195 Start Date*: 2007-09-29
    Sponsor Name:Amgen Inc.
    Full Title: Estudio Aleatorizado, Doble Ciego, Controlado con Placebo, de Fase 1/2 para Determinar la Seguridad y la Eficacia de AMG 531 en Sujetos Pediátricos Trombocitopénicos con Púrpura Trombocitopénica I...
    Medical condition: Púrpura Trombocitopénica Inmune (Idiopática) (PTI)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10021245 Idiopathic thrombocytopenic purpura LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Completed)
    Trial results: View results
    EudraCT Number: 2014-000650-11 Sponsor Protocol Number: HEP002 Start Date*: 2014-09-15
    Sponsor Name:Promethera Biosciences
    Full Title: A prospective, open label, multicountry, efficacy and safety study of several infusions of HepaStem in Urea Cycle Disorders pediatric patients.
    Medical condition: The urea cycle disorders are inborn errors of metabolism that affect the transfer of nitrogen into urea. There are seven disorders to be investigated: carbamoylphosphate synthetase I deficiency [CP...
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004861 10013373 Disorders of urea cycle metabolism LLT
    Population Age: Newborns, Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: BE (Completed) ES (Completed) FR (Completed) PL (Completed)
    Trial results: View results
    EudraCT Number: 2011-004106-16 Sponsor Protocol Number: AC-052-375 Start Date*: Information not available in EudraCT
    Sponsor Name:Actelion pharmaceuticals Ltd
    Full Title: A randomized, prospective, double-blind, placebo-controlled, group sequential multicenter study to assess efficacy, safety, and tolerability of the pediatric formulation of bosentan in children wit...
    Medical condition: Pulmonary arterial hypertension (PAH) in children
    Disease: Version SOC Term Classification Code Term Level
    14.1 10038738 - Respiratory, thoracic and mediastinal disorders 10064908 Associated with (APAH) LLT
    14.1 10038738 - Respiratory, thoracic and mediastinal disorders 10064909 Idiopathic (IPAH) LLT
    14.1 10038738 - Respiratory, thoracic and mediastinal disorders 10064910 Familial (FPAH) LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Ongoing) FR (Ongoing) PT (Completed) BG (Completed)
    Trial results: View results
    EudraCT Number: 2011-003063-30 Sponsor Protocol Number: BRD10/6-O Start Date*: Information not available in EudraCT
    Sponsor Name:CHU Nantes
    Full Title:
    Medical condition:
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10034130 Patent ductus arteriosus PT
    Population Age: Preterm newborn infants, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2019-003032-23 Sponsor Protocol Number: AADC-010 Start Date*: 2019-10-09
    Sponsor Name:National Taiwan University Hospital
    Full Title: A Phase I/II Clinical Trial for Treatment of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC
    Medical condition: Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
    Disease:
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2019-003072-39 Sponsor Protocol Number: NTUH-AADC-011 Start Date*: 2019-10-09
    Sponsor Name:National Taiwan University Hospital
    Full Title: A Clinical Trial for Treatment of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
    Medical condition: Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
    Disease:
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2022-002621-98 Sponsor Protocol Number: SHP643-302 Start Date*: 2022-08-19
    Sponsor Name:Takeda
    Full Title: A Phase 3 Multi-center, Open-label Study to Evaluate the Efficacy and Safety of Lanadelumab (SHP643) in Japanese Subjects with Hereditary Angioedema
    Medical condition: Hereditary Angioedema (HAE)
    Disease: Version SOC Term Classification Code Term Level
    23.1 10010331 - Congenital, familial and genetic disorders 10075280 Hereditary angioedema attack LLT
    23.1 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2018-000504-42 Sponsor Protocol Number: ABT-003 Start Date*: 2019-05-21
    Sponsor Name:Abeona Therapeutics Inc
    Full Title: A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients with Middle and Advanced Phases of MPS IIIA Disease
    Medical condition: MPS IIIA is a devastating lysosomal storage disease, caused by a Nsulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progress...
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Prematurely Ended)
    Trial results: View results
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    Note, where multi-state trials are shown in search results, selecting "Full Trial details" will download full information for each of the member states/countries involved in the trial.
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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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