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Clinical trials for Orphan Drugs

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 ).  
     
    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.
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    18 result(s) found for: Orphan Drugs. Displaying page 1 of 1.
    EudraCT Number: 2006-000344-10 Sponsor Protocol Number: 06_ENDO_49 Start Date*: 2006-06-08
    Sponsor Name:Christie Hospital
    Full Title: Multi-centre study of cabergoline alone and in combination with pegvisomant in the management of active acromegaly
    Medical condition: acromegaly
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) IE (Completed)
    Trial results: View results
    EudraCT Number: 2006-006693-24 Sponsor Protocol Number: Moli1901-010B Start Date*: 2007-05-09
    Sponsor Name:AOP Orphan Pharmaceuticals AG
    Full Title: A Randomized, Double-Blind, Placebo-Controlled, Dose-finding Study to Evaluate the Efficacy and Safety of Aerosolized Moli1901 in Adolescents (12 Years of Age or Older) and Adults with Cystic Fibrosis
    Medical condition: Cystic fibrosis is the most common fatal inherited disease in the Caucasian population, affecting about 4 in 10.000 children. In cystic fibrosis chloride transport across the respiratory epithelium...
    Disease: Version SOC Term Classification Code Term Level
    9.1 10011763 Cystic fibrosis lung PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Completed) CZ (Completed) FR (Completed) DE (Completed) HU (Completed) SE (Completed) ES (Completed) PL (Ongoing) IT (Completed)
    Trial results: View results
    EudraCT Number: 2010-023311-34 Sponsor Protocol Number: CPA 368-10 Start Date*: 2011-02-04
    Sponsor Name:AOP Orphan Pharmaceuticals AG
    Full Title: A prospective, randomized, double blinded, crossover, two-treatment, two-sequence, short term pharmacokinetic, pharmacodynamic and tolerability, single centre study to compare AOP200704 vs. Esmolol...
    Medical condition: healthy male and female volunteers, intended for the treatment of dobutamine-induced tachycardia
    Disease: Version SOC Term Classification Code Term Level
    12.1 10040752 Sinus tachycardia LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: CZ (Completed)
    Trial results: View results
    EudraCT Number: 2018-005046-10 Sponsor Protocol Number: HEMSC42 Start Date*: 2019-07-23
    Sponsor Name:Radboud university medical center
    Full Title: Anakinra: Efficacy of interleukin-1 pathway inhibitor anakinra for the management of fever during neutropenia and mucositis in patients with multiple myeloma receiving an autologous hematopoietic s...
    Medical condition: Febrile neutropenia and mucositis
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004867 10028127 Mucositis LLT
    20.0 10005329 - Blood and lymphatic system disorders 10016288 Febrile neutropenia PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-005832-31 Sponsor Protocol Number: ALXN1840-HV-Cu-Absorption Start Date*: 2021-04-13
    Sponsor Name:Alexion Pharmaceuticals Inc.
    Full Title: Efficacy of ALXN1840 on human hepatic copper uptake quantified with 64CuCl2 PET/CT-scan.
    Medical condition: Healthy Volunteers (Wilson's disease)
    Disease: Version SOC Term Classification Code Term Level
    22.0 10022891 - Investigations 10022891 Investigations SOC
    Population Age: Adults Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2016-002974-11 Sponsor Protocol Number: IBU24h-EchoG Start Date*: 2016-11-10
    Sponsor Name:María Carmen Bravo Laguna
    Full Title: Phase III, randomized, multicenter, double-blind clinical trial to evaluate two echo-guided administration regimens of ibuprofen in the treatment of patent ductus arteriosus: impact on intestinal p...
    Medical condition: patent ductus arteriosus
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10034130 Patent ductus arteriosus PT
    Population Age: Preterm newborn infants, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2013-000300-42 Sponsor Protocol Number: 03AR0298 Start Date*: 2013-01-18
    Sponsor Name:National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
    Full Title: A long-term outcome study with the IL-1 receptor antagonist Anakinra/Kineret® in patients with Neonatal onset multisystem inflammatory disease (Nomid/Cinca syndrome)
    Medical condition: neonatal onset multisystem inflammatory disease (NOMID/CINCA)
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2020-001246-18 Sponsor Protocol Number: APHP200375 Start Date*: 2020-03-25
    Sponsor Name:Assistance Publique - Hôpitaux de Paris
    Full Title: Cohort Multiple randomized controlled trials open-label of immune modulatory drugs and other treatments in COVID-19 patients
    Medical condition: COVID-19
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2014-001213-12 Sponsor Protocol Number: HGT-FIR-096 Start Date*: 2014-09-05
    Sponsor Name:Shire Orphan Therapies, Inc.
    Full Title: A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Study Evaluating the Safety and Efficacy of Icatibant as a Treatment for Angiotensin-Converting Enzyme Inhibitor (ACE...
    Medical condition: ACE-I-induced angioedema
    Disease: Version SOC Term Classification Code Term Level
    17.0 10040785 - Skin and subcutaneous tissue disorders 10002424 Angioedema PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2015-004717-25 Sponsor Protocol Number: Low-PV Start Date*: 2016-07-08
    Sponsor Name:FONDAZIONE PER LA RICERCA OSPEDALE MAGGIORE DI BERGAMO - FROM
    Full Title: The benefit/risk profile of pegylated proline-Interferon alpha-2b (AOP2014) added to the best available strategy based on phlebotomies in low-risk patients with Polycythemia Vera (PV). The Low-PV r...
    Medical condition: Polycythemia Vera
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004864 10036061 Polycythemia vera LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2013-003611-21 Sponsor Protocol Number: 2013-003611-21 Start Date*: 2013-12-20
    Sponsor Name:Karolinska Institutet
    Full Title: Safety of fluconazol treatment of premature and full-term newborns - a study on interactions by NSAIDs with fluconazole in respect of pharmacodynamic endpoints with urinary excretion of vas...
    Medical condition: Fungal infection prophylaxis Patent ductus arteriosus
    Disease: Version SOC Term Classification Code Term Level
    16.1 10010331 - Congenital, familial and genetic disorders 10034130 Patent ductus arteriosus PT
    16.1 10021881 - Infections and infestations 10028924 Neonatal candida infection PT
    Population Age: Preterm newborn infants, Newborns, Under 18 Gender: Male, Female
    Trial protocol: SE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2018-001044-54 Sponsor Protocol Number: Bosupeg Start Date*: 2019-06-12
    Sponsor Name:St Olavs Hospital -Trondheim University Hospital
    Full Title: A STUDY OF EFFICACY AND SAFETY OF LONG-ACTING LOW DOSE ROPEGINTERFERON IN PATIENTS WITH CHRONIC MYELOID LEUKEMIA TREATED WITH BOSUTINIB FROM DIAGNOSIS: A RANDOMIZED PROSPECTIVE TRIAL
    Medical condition: Chronic myeloid leukemia at diagnosis-chronich phase
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FI (Ongoing) DK (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-001823-30 Sponsor Protocol Number: 043/SI Start Date*: 2017-06-12
    Sponsor Name:SIFI SpA
    Full Title: Randomized, Assessor-Masked, Active-Controlled, Phase 3 Study to Evaluate Efficacy, Safety and Tolerability of 0.08% Polyhexamethylene Biguanide (PHMB) Ophthalmic Solution in Comparison with 0.02% ...
    Medical condition: Acanthamoeba keratitis
    Disease: Version SOC Term Classification Code Term Level
    20.0 10021881 - Infections and infestations 10069408 Acanthamoeba keratitis PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) PL (Completed) IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2013-004955-19 Sponsor Protocol Number: PARIDA-01/2013 Start Date*: 2014-09-03
    Sponsor Name:Azienda Ospedaliera di Padova
    Full Title: PARACETAMOL VERSUS IBUPROFEN FOR PATENT DUCTUS ARTERIOSUS TREATMENT IN PRETERM INFANTS.
    Medical condition: patent ductus arteriosus hemodynamically important
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004865 10034190 PDA Repair patent ductus arteriosus LLT
    Population Age: Preterm newborn infants, Under 18 Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-004419-11 Sponsor Protocol Number: SC35 Start Date*: 2017-04-20
    Sponsor Name:Radboud university medical center
    Full Title: Safety and efficacy of interleukin-1 inhibitor anakinra for the amelioration of fever during neutropenia and mucositis in patients with multiple myeloma receiving an autologous hematopoietic stem c...
    Medical condition: Mucositis and febrile neutropenia
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004867 10028127 Mucositis LLT
    19.0 10005329 - Blood and lymphatic system disorders 10016288 Febrile neutropenia PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: View results
    EudraCT Number: 2015-004526-33 Sponsor Protocol Number: PDARCT1 Start Date*: Information not available in EudraCT
    Sponsor Name:Royal College of Surgeons in Ireland
    Full Title: A Randomised Controlled Trial of Early Targeted Patent Ductus Arteriosus Treatment Using a Risk Based Severity Score
    Medical condition: Patent Ductus Arteriosus
    Disease: Version SOC Term Classification Code Term Level
    18.1 10010331 - Congenital, familial and genetic disorders 10034130 Patent ductus arteriosus PT
    Population Age: Preterm newborn infants, Newborns, Under 18 Gender: Male, Female
    Trial protocol: IE (Completed)
    Trial results: View results
    EudraCT Number: 2020-001500-41 Sponsor Protocol Number: COV-AID Start Date*: 2020-04-03
    Sponsor Name:University Hospital Ghent
    Full Title: A prospective, randomized, factorial design, interventional study to compare the safety and efficacy of combinations of blockade of interleukin-6 pathway and interleukin-1 pathway to best standard ...
    Medical condition: COVID-19 patients with acute hypoxic respiratory failure and systemic cytokine release syndrome.
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2011-005606-30 Sponsor Protocol Number: CAM-THY Start Date*: 2012-04-25
    Sponsor Name:Cambridge University Hospitals NHS Foundation Trust and University of Cambridge
    Full Title: Keratinocyte Growth Factor - promoting thymic reconstitution and preventing autoimmunity after alemtuzumab (Campath-1H) treatment of multiple sclerosis. CAM-THY
    Medical condition: This trial will test the efficacy of Kepivance in the prevention of new autoimmune diseases in patients who have multiple sclerosis (MS)who are being treated with alemtuzumab.
    Disease: Version SOC Term Classification Code Term Level
    14.1 10038359 - Renal and urinary disorders 10058948 Nephritis autoimmune PT
    14.1 10014698 - Endocrine disorders 10049046 Autoimmune thyroiditis PT
    14.1 10021428 - Immune system disorders 10061664 Autoimmune disorder PT
    14.1 10014698 - Endocrine disorders 10068004 Autoimmune hyperthyroidism LLT
    14.1 10005329 - Blood and lymphatic system disorders 10050245 Autoimmune thrombocytopenia PT
    Population Age: Adults 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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