Flag of the European Union EU Clinical Trials Register Help

Clinical trials for Severe congenital neutropenia

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   44341   clinical trials with a EudraCT protocol, of which   7370   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.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     
    6 result(s) found for: Severe congenital neutropenia. Displaying page 1 of 1.
    EudraCT Number: 2011-001118-32 Sponsor Protocol Number: EP06-401 Start Date*: 2011-05-25
    Sponsor Name:Sandoz GmbH
    Full Title: Twelve-month study on the immunogenicity, safety, and efficacy of Zarzio®/Filgrastim HEXAL® in patients with severe chronic neutropenia
    Medical condition: Severe chronic neutropenia (i.e. congenital neutropenia, cyclic neutropenia and chronic idiopathic neutropenia)
    Disease: Version SOC Term Classification Code Term Level
    14.1 10005329 - Blood and lymphatic system disorders 10051645 Idiopathic neutropenia PT
    14.1 10010331 - Congenital, familial and genetic disorders 10069819 Congenital neutropenia LLT
    14.1 10005329 - Blood and lymphatic system disorders 10053176 Cyclic neutropenia PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) SE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2018-004191-35 Sponsor Protocol Number: GLYCO-1B Start Date*: 2019-04-12
    Sponsor Name:Cliniques universitaires Saint-Luc
    Full Title: Evaluation of the safety and efficacy of administration of Empagliflozin in a new treatment for neutropenia in patients with Glycogen Storage Disease type 1b (GSD1b) and G6PC3 deficiency.
    Medical condition: (1) Severe Congenital Neutropenia type 4 (SNC4) due to a deficiency in G6PC3, a phosphatase of the endoplasmic also known a Ubiquitous glucose-6-phosphatase and (2) the neutropenia in Glycogen Sto...
    Disease:
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2009-016069-27 Sponsor Protocol Number: DORIPED3003 Start Date*: 2010-05-28
    Sponsor Name:Janssen-Cilag International NV, Turnhoutseweg 30, 2340 Beerse, Belgium
    Full Title: A Prospective, Randomized, Double-Blind, Multicenter Study to Establish the Safety and Tolerability of Doripenem Compared With Cefepime in Hospitalized Children With Bacterial Pneumonia
    Medical condition: Bacterial Pneumonia
    Disease: Version SOC Term Classification Code Term Level
    14.1 10021881 - Infections and infestations 10004051 Bacterial pneumonia, unspecified LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: LT (Prematurely Ended) LV (Prematurely Ended) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2019-002267-10 Sponsor Protocol Number: MK-5592-104 Start Date*: 2019-12-17
    Sponsor Name:Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc.
    Full Title: A Phase 2, Open-Label, Non-Comparative Clinical Trial to Study the Safety and Efficacy of Posaconazole (POS, MK-5592) in Pediatric Participants Aged 2 to <18 Years With Invasive Aspergillosis
    Medical condition: Invasive aspergillosis (IA)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004862 10003488 Aspergillosis LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: HU (Completed) Outside EU/EEA GR (Completed) BE (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2018-001309-95 Sponsor Protocol Number: MPH966-2-01 Start Date*: 2018-11-30
    Sponsor Name:Mereo BioPharma 4 Ltd
    Full Title: A Phase 2, proof-of-concept, multicentre, double-blind, randomised, dose-ascending, sequential group, placebo-controlled study to evaluate the mechanistic effect, safety, and tolerability of 12 wee...
    Medical condition: Alpha-1 antitrypsin deficiency
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10001806 Alpha-1 anti-trypsin deficiency PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) SE (Completed) DK (Completed) ES (Ongoing) PL (Completed) BE (Completed)
    Trial results: View results
    EudraCT Number: 2020-004184-12 Sponsor Protocol Number: DTX401-CL301 Start Date*: 2022-08-22
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus Serotype 8-mediated Gene Transfer of Glucose-6-phosphatase in Patients With Glycogen Storage Disease Type Ia....
    Medical condition: Glycogen Storage Disease Type Ia (GSDIa).
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10056911 Glycogen storage disease type IA LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed) ES (Trial now transitioned) IT (Trial now transitioned) DK (Trial now transitioned) PT (Completed) DE (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
    Download Options:
    Number of Trials to download:
    Download Content:
    Download Format:
    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.
    Query did not match any studies.
    For support, Contact us.
    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.

    European Medicines Agency © 1995-Sun Jun 08 00:38:12 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA