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Clinical trials for Orphan Drug

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.
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    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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    72 result(s) found for: Orphan Drug. Displaying page 1 of 4.
    1  2  3  4  Next»
    EudraCT Number: 2021-004002-21 Sponsor Protocol Number: Bio-Eq-20 Start Date*: 2021-11-26
    Sponsor Name:AOP Orphan Pharmaceuticals GmbH
    Full Title: A single center, open-label, two-period, non-randomized, paired-design study to compare steady-state plasma levels following switch from a 10 mg/ml treprostinil formulation to a 20 mg/ml treprostin...
    Medical condition: pulmonary arterial hypertension
    Disease: Version SOC Term Classification Code Term Level
    21.1 10038738 - Respiratory, thoracic and mediastinal disorders 10064911 Pulmonary arterial hypertension PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: AT (Completed)
    Trial results: View results
    EudraCT Number: 2017-002806-10 Sponsor Protocol Number: SOBI003-001 Start Date*: Information not available in EudraCT
    Sponsor Name:Swedish Orphan Biovitrum AB (publ)
    Full Title: An open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of SOBI003 in pediatric MPS IIIA patients
    Medical condition: Mucopolysaccharidosis Type IIIA or Sanfilippo Syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10028094 Mucopolysaccharidosis IH LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) NL (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2018-002502-31 Sponsor Protocol Number: RP-L102-0118 Start Date*: 2018-10-31
    Sponsor Name:Rocket Pharmaceuticals, Inc.
    Full Title: A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemi...
    Medical condition: Fanconi anemia (subtype A)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10055206 Fanconi's anemia LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing) GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2021-001577-24 Sponsor Protocol Number: NI-0501-14 Start Date*: 2021-12-15
    Sponsor Name:Swedish Orphan Biovitrum AG (Sobi AG)
    Full Title: A two-cohort, open-label, single arm, multicenter study to evaluate efficacy, safety and tolerability, pharmacokinetics and pharmacodynamics, of emapalumab in children and adults with macrophage ac...
    Medical condition: Macrophage activation syndrome (MAS) in the context of Systemic juvenile inflammatory arthritis (sJIA) and Adult onset Still’s disease (AOSD). MAS in the context of pediatric and adult Systemic lup...
    Disease: Version SOC Term Classification Code Term Level
    21.1 10021428 - Immune system disorders 10071583 Haemophagocytic lymphohistiocytosis PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing) FR (Ongoing) IT (Ongoing) PL (Ongoing) DE (Ongoing) CZ (Ongoing) BE (Ongoing) SE (Ongoing) NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-003725-42 Sponsor Protocol Number: R121178 Start Date*: 2018-03-23
    Sponsor Name:University of Manchester
    Full Title: Does Interleukin-1 Receptor Antagonist Improve Outcome following aneurysmal Subarachnoid Haemorrhage (aSAH)? A Phase III trial
    Medical condition: subarachnoid haemorrhage
    Disease: Version SOC Term Classification Code Term Level
    20.1 10022117 - Injury, poisoning and procedural complications 10042316 Subarachnoid haemorrhage PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2021-003160-27 Sponsor Protocol Number: Sobi.PEGCET-101 Start Date*: 2022-05-31
    Sponsor Name:Swedish Orphan Biovitrum AB
    Full Title: A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients with Cold Agglutinin Disease (CAD)
    Medical condition: Patients with Cold Agglutinin Disease (CAD)
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) NO (Prematurely Ended) HU (Prematurely Ended) AT (Prematurely Ended) IT (Prematurely Ended) BG (Prematurely Ended) ES (Prematurely Ended) FI (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2012-005259-18 Sponsor Protocol Number: PROUD-PV Start Date*: 2013-08-15
    Sponsor Name:AOP Orphan Pharmaceuticals AG
    Full Title: A randomized, open-label, multicenter, controlled, parallel arm, phase III study assessing the efficacy and safety of AOP2014 vs. Hydroxyurea in patients with Polycythemia Vera
    Medical condition: Polycythemia Vera
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004864 10036061 Polycythemia vera LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: HU (Completed) CZ (Completed) IT (Completed) SK (Completed) AT (Completed) BG (Completed) DE (Completed) PL (Completed) ES (Completed) BE (Completed)
    Trial results: View results
    EudraCT Number: 2011-003825-81 Sponsor Protocol Number: HGT-FIR-086 Start Date*: 2012-02-28
    Sponsor Name:Shire Orphan Therapies, Inc
    Full Title: A Multicenter, Open-Label, Non-Randomized Study to Assess the Pharmacokinetics, Tolerability, and Safety of a Single Subcutaneous Administration of Icatibant in Children and Adolescents with Heredi...
    Medical condition: Hereditary Angioedema
    Disease: Version SOC Term Classification Code Term Level
    16.0 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) HU (Prematurely Ended) ES (Prematurely Ended) IT (Completed) AT (Completed)
    Trial results: View results
    EudraCT Number: 2015-000075-27 Sponsor Protocol Number: HPN-100-021 Start Date*: 2017-05-04
    Sponsor Name:Horizon Therapeutics, LLC
    Full Title: A Randomised, Controlled, Open-Label Parallel Arm Study of the Safety, Pharmacokinetics and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate [GPB]) Oral Liquid and Sodium Phenylbutyrate (NaPBA)...
    Medical condition: Urea Cycle Disorders (UCDs)
    Disease: Version SOC Term Classification Code Term Level
    20.1 200000003094 10013373 Disorders of urea cycle metabolism LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: BE (Ongoing) GB (Completed) AT (Completed) IT (Completed) ES (Ongoing)
    Trial results: View results
    EudraCT Number: 2012-002773-64 Sponsor Protocol Number: RP103-07 Start Date*: 2014-01-22
    Sponsor Name:Horizon Pharma USA, Inc.
    Full Title: A Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis
    Medical condition: Cystinosis
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10011777 Cystinosis PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) BE (Completed) NL (Completed) IT (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2016-004974-16 Sponsor Protocol Number: IL1ra03 Start Date*: 2018-11-08
    Sponsor Name:Cambridge University Hospitals NHS Foundation Trust and the University of Cambridge
    Full Title: DOUBLE-BLIND PLACEBO-CONTROLLED RANDOMISED CLINICAL DOSE-RANGING STUDY TREATING MODERATE-SEVERE TRAUMATIC BRAIN INJURY PATIENTS WITH RECOMBINANT HUMAN INTERLEUKIN 1 RECEPTOR ANTAGONIST.
    Medical condition: We wish to study the drug Kineret in patients suffering from moderate-to-severe traumatic brain injury
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004863 10060690 Traumatic brain injury LLT
    20.1 10029205 - Nervous system disorders 10051288 Central nervous system inflammation PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2010-021384-33 Sponsor Protocol Number: NTXMR1 Start Date*: 2010-08-20
    Sponsor Name:Beroendecentrum Stockholm
    Full Title: The Effect of Naltrexone on Amphetamine Cue Reactivity: An fMRI Study
    Medical condition: Amphetamine dependence
    Disease:
    Population Age: Adults Gender: Male
    Trial protocol: SE (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2008-007104-28 Sponsor Protocol Number: IFN-Multiferon08 Start Date*: 2009-04-01
    Sponsor Name:Karolinska Universitetssjukhuset
    Full Title: Multiferon treatment of non-responders to peg-IFN alfa plus ribavirin standard of care treatment - a study of the early viral kinetics during the initial 12 weeks treatment.
    Medical condition: HCV RNA response to a multivalent IFN (Multiferon 6 MU q.d. + ribavirin standard dosed) in prior non-responders to SOC (peg-IFN + ribavirin) during treatment the initial 12 weeks. Results of HCV RN...
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: SE (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2009-017095-24 Sponsor Protocol Number: AOP13007 Start Date*: 2010-02-17
    Sponsor Name:AOP Orphan Pharmaceuticals AG
    Full Title: A phase III, randomized, multicenter, subject- and sponsor-blinded, placebo controlled study to compare the efficacy and safety of “Anagrelide retard” versus placebo in “at risk” subjects with Esse...
    Medical condition: male and female "at risk" subjects with Essential Thrombocythaemia
    Disease: Version SOC Term Classification Code Term Level
    17.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10015493 Essential thrombocythaemia PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: AT (Completed) SK (Completed) SI (Completed) LT (Completed) BG (Completed)
    Trial results: View results
    EudraCT Number: 2014-001356-31 Sponsor Protocol Number: PEN-PV Start Date*: 2015-05-20
    Sponsor Name:AOP Orphan Pharmaceuticals AG
    Full Title: An open-label, single arm, Phase III study to assess the self-administration of AOP2014 using a pre-filled pen, developed for the treatment of Polycythemia Vera patients
    Medical condition: Polycythemia Vera
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004864 10036061 Polycythemia vera LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: HU (Completed) SK (Completed) AT (Completed) CZ (Completed) BG (Completed) PL (Completed)
    Trial results: View results
    EudraCT Number: 2013-003410-41 Sponsor Protocol Number: AOP18007 Start Date*: 2014-01-13
    Sponsor Name:AOP Orphan Pharmaceuticals AG
    Full Title: A phase III randomized, multicenter, double-blind, active controlled study to compare the efficacy and safety of two different anagrelide formulations in patients with Essential Thrombocythemia (TE...
    Medical condition: male or female patients with confirmed diagnosis of Essential Thrombocythemia
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004864 10015494 Essential thrombocythemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Completed) LT (Completed) BG (Completed) PL (Completed)
    Trial results: View results
    EudraCT Number: 2018-000249-38 Sponsor Protocol Number: R118439 Start Date*: 2019-01-25
    Sponsor Name:University of Manchester
    Full Title: Phase II trial of interleukin-1 receptor antagonist in intracerebral haemorrhage: BLOcking the Cytokine IL-1 in ICH
    Medical condition: intracerebral haemorrhage
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004852 10022753 Intracerebral haemorrhage LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2014-001357-17 Sponsor Protocol Number: CONTINUATION-PV Start Date*: 2014-11-03
    Sponsor Name:AOP Orphan Pharmaceuticals AG
    Full Title: An open-label, multicenter, phase IIIb study assessing the long-term efficacy and safety of AOP2014 and standard first line treatment (BAT) in patients with Polycythemia Vera who previously partici...
    Medical condition: Polycythemia Vera
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004864 10036061 Polycythemia vera LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: HU (Completed) AT (Completed) CZ (Completed) PL (Completed) SK (Completed) BG (Completed) ES (Completed) DE (Completed)
    Trial results: View results
    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: 2011-006100-12 Sponsor Protocol Number: FANCOLEN-1 Start Date*: 2013-04-12
    Sponsor Name:FUNDACION PARA LA INVESTIGACION BIOMEDICA DEL HOSPITAL UNIVERSITARIO NIÑO JESUS
    Full Title: Clinical Trial Phase I / II to evaluate the safety and efficacy of the infusion of autologous CD34+ cells transduced with a lentiviral vector carrying the FANCA gene (orphan drug) for patients with...
    Medical condition: Fanconi anemia (Subtype A)
    Disease: Version SOC Term Classification Code Term Level
    15.1 100000004850 10055206 Fanconi's anemia LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
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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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