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Clinical trials for Orphan

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   44339   clinical trials with a EudraCT protocol, of which   7369   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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    188 result(s) found for: Orphan. Displaying page 1 of 10.
    1  2  3  4  5  6  7  8  9  Next» Last»»
    EudraCT Number: 2020-001167-93 Sponsor Protocol Number: Sobi.IMMUNO-101 Start Date*: 2020-03-27
    Sponsor Name:SWEDISH ORPHAN BIOVITRUM AB (PUBL)
    Full Title: A phase 2/3, randomized, open-label, parallel group, 3-arm, multicenter study investigating the efficacy and safety of intravenous administrations of emapalumab, an anti-interferon gamma (anti-IFN¿...
    Medical condition: SARS-CoV-2 infection
    Disease: Version SOC Term Classification Code Term Level
    20.0 10021881 - Infections and infestations 10021881 Infections and infestations SOC
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-002526-39 Sponsor Protocol Number: 20010133 Start Date*: 2015-07-02
    Sponsor Name:Swedish Orphan Biovitrum AB (publ)
    Full Title: A Phase 1 Dose-escalation Study to Evaluate the Safety and Pharmacokinetics (PK) of Palifermin in Pediatric Subjects with Acute Leukemias Undergoing Myeloblative Therapy and Allogeneic Hematopoieti...
    Medical condition: Oral Mucositis
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2017-000065-73 Sponsor Protocol Number: Sobi.Elocta-003 Start Date*: 2017-08-23
    Sponsor Name:Swedish Orphan Biovitrum AB (publ)
    Full Title: A Non-Controlled, Open-Label, Multicenter, Study of Immune Tolerance Induction Performed with rFVIIIFc within a Timeframe of 60 Weeks in Severe Haemophilia A Patients with Inhibitors who have Faile...
    Medical condition: Severe Haemophilia A Patients with Inhibitors who have Failed Previous Immune Tolerance Induction Therapies
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10018937 Haemophilia A LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male
    Trial protocol: DE (Completed) IE (Completed) GB (Completed) BE (Completed) FR (Completed) SE (Completed) SI (Completed) IT (Completed)
    Trial results: View results
    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: 2013-004132-29 Sponsor Protocol Number: Sobi.NTBC-003 Start Date*: 2014-10-15
    Sponsor Name:Swedish Orphan Biovitrum AB (Publ)
    Full Title: An open-label, non-randomized, sequential, multicenter study to evaluate the pharmacokinetics, efficacy and safety of once daily dosing compared to twice daily dosing of Orfadin in patients diagnos...
    Medical condition: hereditary tyrosinemia type 1
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004850 10069462 Tyrosinemia type I LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: SE (Completed) DK (Completed) BE (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: 2017-000645-48 Sponsor Protocol Number: AGU-001 Start Date*: 2017-09-21
    Sponsor Name:Minna Laine [...]
    1. Minna Laine
    2. Prof. Ritva Tikkanen
    Full Title: Open-label study to evaluate efficacy and safety of Cystadane for the treatment of aspartylglucosaminuria
    Medical condition: Aspartylglucosaminuria
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: FI (Completed)
    Trial results: (No results available)
    EudraCT Number: 2021-000324-36 Sponsor Protocol Number: NL76607.091.21 Start Date*: 2022-03-21
    Sponsor Name:Radboudumc
    Full Title: Anakinra in Cerebral haemorrhage to Target secondary Injury resulting from Neuroinflammation - a phase II clinical trial
    Medical condition: Spontaneous supratentorial intracerebral haemorrhage
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2012-002286-36 Sponsor Protocol Number: Sobi.NTBC-002 Start Date*: 2012-11-19
    Sponsor Name:Swedish Orphan Biovitrum AB
    Full Title: Taste and palatability of Orfadin suspension. An open, non-controlled 3-day study in pediatric patients with hereditary tyrosinemia type 1 treated with Orfadin.
    Medical condition: Hereditary tyrosinemia type 1 (HT-1)
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004850 10069459 Tyrosinaemia type I LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2012-004089-16 Sponsor Protocol Number: 1541-128 Start Date*: 2013-04-03
    Sponsor Name:Universitätsklinik für Innere Medizin III, Klinische Abteilung für Rheumatologie, Medizinische Universität Wien
    Full Title: The effect of the interleukin-1 receptor antagonist Anakinra in patients with osteoarthritis of the hand: The AHOA (Anakinra in hand osteoarthritis) pilot study
    Medical condition: osteoarthritis of the hand
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2013-005402-65 Sponsor Protocol Number: IIBSP-ANA-2013-163 Start Date*: 2015-01-19
    Sponsor Name:Institut de Recerca HSCSP
    Full Title: Efficacy and safety of anakinra (antagonist of the interleukin-1 receptor) in the treatment of periarticular inflammation in patients refractory to NSAID and / or steroids
    Medical condition: Inflammation in periarticular structures in patients refractory to NSAIDs and / or corticosteroids.
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2011-000206-22 Sponsor Protocol Number: 130101 Start Date*: 2011-04-11
    Sponsor Name:Helsingin Yliopisto, Kemian laitos
    Full Title: Elimination kinetics of HCN after inhaled exposure
    Medical condition: Healthy volunteers
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: FI (Completed)
    Trial results: (No results available)
    EudraCT Number: 2004-004280-30 Sponsor Protocol Number: 20031209-2 Start Date*: 2004-12-30
    Sponsor Name:Landstinget Dalarna
    Full Title: Injestion of the steroid betamethasone during operation for inguinal hernia repair for purpose to optimize treatment of postoperative pain, reduction of postoperative discomfort like nausea and pos...
    Medical condition: Betamethasone will be studied for treatment of postoperative pain and discomfort after operations for inguinal hernia.
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2005-004344-30 Sponsor Protocol Number: Moli1901-010A Start Date*: 2005-10-31
    Sponsor Name:AOP Orphan Pharmaceuticals AG
    Full Title: An Evaluation of the Safety and Tolerability of Multiple Dose Regimens of Aerosolized Moli1901 in Adolescents (12 Years of Age or Older) and Adults with Cystic Fibrosis and Stable Lung Disease
    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:
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: AT (Completed) HU (Completed)
    Trial results: View results
    EudraCT Number: 2009-014717-27 Sponsor Protocol Number: 2009/4:7 Start Date*: 2009-12-22
    Sponsor Name:Karolinska Institutet/Södersjukhuset
    Full Title: Betametasone as postoperative pain treatment for knee arthroscopies at a day surgical center
    Medical condition: Unspecified knee pain investigated by an arthroscopy
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (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 (Completed) IT (Trial now transitioned) PL (Completed) DE (Completed) CZ (Trial now transitioned) BE (Completed) SE (Completed) NL (Trial now transitioned)
    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 (Completed) IT (Prematurely Ended) BG (Completed) ES (Prematurely Ended) FI (Prematurely Ended) BE (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2011-003075-11 Sponsor Protocol Number: 9HB01EXT Start Date*: 2011-12-05
    Sponsor Name:Biogen Idec Research Ltd
    Full Title: An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant, Human Coagulation Factor IX Fusion Protein (rFIXFc) in the Prevention and Treatment of Bleeding Episodes i...
    Medical condition: The medical condition to be investigated is Hemophilia B, or Christmas disease. Hemophilia B is a deficiency in the clotting FIX and is a recessively inherited coagulation disorder due to an X-chro...
    Disease: Version SOC Term Classification Code Term Level
    19.1 100000004850 10053754 Hemophilia B without inhibitors LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male
    Trial protocol: GB (Completed) DE (Completed) BE (Completed) SE (Completed) PL (Completed) IT (Completed) IE (Completed) NL (Completed)
    Trial results: View results
    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)
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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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