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Clinical trials for rare diseases

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   43974   clinical trials with a EudraCT protocol, of which   7311   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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    224 result(s) found for: rare diseases. Displaying page 1 of 12.
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    EudraCT Number: 2020-002821-28 Sponsor Protocol Number: ET20-128 Start Date*: 2020-12-24
    Sponsor Name:Centre Léon Bérard
    Full Title: RAR-Immune: A randomised, comparative, prospective, multicentre study of the efficacy of nivolumab + ipilimumab versus pazopanib alone in patients with metastatic or unresectable advanced sarcoma o...
    Medical condition: Metastatic or unresectable advanced rare sarcomas
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-002607-34 Sponsor Protocol Number: GOIRC-02-2018CHANCETrial Start Date*: 2019-02-20
    Sponsor Name:GRUPPO ONCOLOGICO ITALIANO DI RICERCA CLINICA (GOIRC)
    Full Title: Phase II, open-label study of atezolizumab in a CoHort of pretreated, Advanced Non-small cell lung cancer (NSCLC) patients with rare histologiCal subtypEs – CHANCE trial
    Medical condition: Advanced Non-small cell lung cancer (NSCLC) with rare histological subtypes
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004864 10064049 Lung adenocarcinoma metastatic LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2013-005269-37 Sponsor Protocol Number: 836021009 Start Date*: 2014-10-02
    Sponsor Name:St. Antonius Hospital
    Full Title: RITUXIMAB IN LIFE THREATENING THERAPY RESISTANT PROGRESSIVE INTERSTITIAL PNEUMONITIS
    Medical condition: Patients with rare Immune mediated inflammatory diseases (IMIDs) involving the lungs causing interstitial pneumonitis (IP).
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-000793-27 Sponsor Protocol Number: IRST100.42 Start Date*: 2019-10-23
    Sponsor Name:ISTITUTO SCIENTIFICO ROMAGNOLO PER LO STUDIO E LA CURA DEI TUMORI (IRST) S.R.L. IRCCS
    Full Title: A phase II study on adjuvant Vaccination with dendritic cells loaded with autologous tumor homogenate in resected stage IV rare cancers: Head&Neck (H&N), neuroendocrine Tumors (NET) and soft tissue...
    Medical condition: Resected stage IV rare cancers: Head&Neck (H&N), neuroendocrine tumors (NET) and soft tissue sarcoma (STS).
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10071536 Head and neck cancer stage IV PT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10062476 Neuroendocrine tumor LLT
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10068771 Soft tissue neoplasm PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-002475-34 Sponsor Protocol Number: REC0559-B-001 Start Date*: 2020-03-03
    Sponsor Name:Recordati Rare Diseases
    Full Title: Efficacy, Safety and Pharmacokinetics of 3 Doses of REC 0/0559 Eye Drops for the Treatment of Stage 2 (Moderate) and 3 (Severe) Neurotrophic Keratitis in Adult Patients
    Medical condition: Neurotrophic Keratitis
    Disease: Version SOC Term Classification Code Term Level
    20.0 10015919 - Eye disorders 10069732 Neurotrophic keratopathy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: HU (Ongoing) GB (GB - no longer in EU/EEA) DE (Ongoing) ES (Ongoing) IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2022-003853-70 Sponsor Protocol Number: PNRR-MR1-2022-12375914 Start Date*: 2023-05-02
    Sponsor Name:OSPEDALE SAN RAFFAELE
    Full Title: Towards a personalized precision medicine in rare disease: tirzepatide (a dual glucose­ dependent insulinotropic polypeptide and glucagon-like peptide-I receptor agonist) monotherapy in patients wi...
    Medical condition: Wolfram syndrome type 1
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10078338 Wolfram syndrome PT
    20.0 10010331 - Congenital, familial and genetic disorders 10078338 Wolfram syndrome PT
    20.0 10010331 - Congenital, familial and genetic disorders 10078338 Wolfram syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-004281-19 Sponsor Protocol Number: 1.5 Start Date*: 2022-06-08
    Sponsor Name:Region Stockholm Karolinska University Hospital (Dep. of Dermatology)
    Full Title: Investigator initiated clinical trial of dantrolene as a treatment for Darier disease
    Medical condition: Darier disease is a rare and severe autosomal dominant skin disorder.
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2022-001478-78 Sponsor Protocol Number: MCT8-2021-3 Start Date*: 2023-03-31
    Sponsor Name:Rare Thyroid Therapeutics International AB
    Full Title: Withdrawal of Tiratricol Treatment in Males with Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency): A Double blind, Randomized, Placebo controlled Study
    Medical condition: Monocarboxylate Transporter 8 (MCT8) deficiency
    Disease:
    Population Age: Children, Under 18, Adults Gender: Male
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2010-024026-38 Sponsor Protocol Number: mex201011 Start Date*: 2012-01-10
    Sponsor Name:Radboud University Nijmegen Medical Center
    Full Title: Combining N-of-1 trials to estimate population clinical effectiveness of drugs using Bayesian hierarchical modeling. The case of Mexilitin for patients with Non-Dystrophic Myotonia.
    Medical condition: Patients with non-dystrophic myotonic syndromes (NDMs). NDMs are caused by mutations in the gene encoding for the skeletal muscle sodium (SCN4A) or chloride (CLCN1) channel. In this study patient...
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2006-003957-25 Sponsor Protocol Number: NAC2006 Start Date*: 2006-10-13
    Sponsor Name:AZIENDA OSPEDALIERO UNIVERSITARIA OSPEDALI RIUNITI UMBERTO I - G.M.LANCISI - G.SALESI
    Full Title: Rare Diseases with microvascular involvement. High Dose Intravenous N-acetylcysteine versus Iloprost for early, rapidly progressive diffuse Systemic Sclerosis (Scleroderma)
    Medical condition: Systemic Scleroderma
    Disease: Version SOC Term Classification Code Term Level
    9.1 10055953 Scleroedema LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-004682-39 Sponsor Protocol Number: KFJ-2020-02_NORMOSANG Start Date*: 2020-11-16
    Sponsor Name:Thomayerova nemocnice
    Full Title: Pilot, open-label, monocentric study evaluating safety and clinical effect of application of NORMOSANG® in patients with COVID-19 disease
    Medical condition: Patients with COVID -19 disease
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: CZ (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-005856-12 Sponsor Protocol Number: 20221 Start Date*: 2022-01-31
    Sponsor Name:Helsinki University Central Hospital
    Full Title: Monoclonal gammopathies of renal significance (MGRS) in Finland
    Medical condition: Rare renal diseases linked to monoclonal gammopathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10038359 - Renal and urinary disorders 10038359 Renal and urinary disorders SOC
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FI (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2022-000513-14 Sponsor Protocol Number: NBK154/2/2021 Start Date*: 2023-03-08
    Sponsor Name:Medical University of Gdańsk
    Full Title: Evaluation of the Efficacy of Valsartan in Slowing Down Aortic Root Dilatation in Children and Young Adults with Marfan-type Heritable Thoracic Aortic Diseases – Valsar-TAD, a randomised, double-b...
    Medical condition: Marfan Syndrome Diseases related to Marfan syndrome (rare diseases with aneurysms of the thoracic aorta and dissection of the aorta), among others: Loeys-Dietz syndrome, Vascular type of Ehlers-Da...
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: PL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-002222-23 Sponsor Protocol Number: P170932J Start Date*: 2019-02-05
    Sponsor Name:APHP
    Full Title: Cobimetinib for BRAF-wild-type histiocytoses : a randomized, placebo-controlled, double blind study
    Medical condition: Histiocytoses are rare multisystemic disorders characterized by accumulation of histiocytes in various organs. patients with BRAF-wild type.
    Disease:
    Population Age: Gender:
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2014-000383-18 Sponsor Protocol Number: LAL1913 Start Date*: 2014-05-20
    Sponsor Name:G.I.M.EM.A. Gruppo Italiano Malattie EMatologiche dell'Adulto
    Full Title: National Treatment Program of Philadelphia Chromosome-negative Adult Acute Lymphoblastic Leukemia with Pegylated Asparaginase Added to a Lineage-Targeted Risk- and Minimal Residual Disease-Oriented...
    Medical condition: National Treatment Program of Philadelphia Chromosome-negative Adult Acute Lymphoblastic Leukemia with Pegylated Asparaginase added to a Lineage-Targeted Risk- and Minimal Residual Disease-Oriented...
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-001719-65 Sponsor Protocol Number: CALC-SSc Start Date*: 2018-12-10
    Sponsor Name:University Medial Center Groningen
    Full Title: CALCification as an early marker of vasculopathy and organ involvement in Systemic Sclerosis CALC-SSc study
    Medical condition: Systemic sclerosis (SSc) is a rare progressive autoimmune disease hallmarked by severe vasculopathy. Patients are prone to enhanced calcification of skin and the vasculature. Active calcifications ...
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2011-002516-85 Sponsor Protocol Number: 01-2011 Start Date*: 2011-10-20
    Sponsor Name:A.O. UNIVERSITARIA INTEGRATA DI VERONA
    Full Title: Open-label study to evaluate the efficacy and tolerability of orally administered rosiglitazone in patients with progressive bone Fibrodysplasia (FOP)
    Medical condition: Fibrodysplasia Ossificans Progressive (FOP) is a rare genetically based disability, characterized by progressive heterotopic ossification, usually associated with congenital malformation of the toes.
    Disease: Version SOC Term Classification Code Term Level
    14.1 10028395 - Musculoskeletal and connective tissue disorders 10028393 Musculoskeletal and connective tissue disorders NEC HLGT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-001382-10 Sponsor Protocol Number: CS/2015/4775 Start Date*: 2019-01-21
    Sponsor Name:University Hospitals Bristol NHS Foundation Trust
    Full Title: Treatment of Barth Syndrome by CARDIOlipin MANipulation (CARDIOMAN): A randomised placebo controlled pilot trial conducted by the nationally commissioned Barth Syndrome Service
    Medical condition: Barth Syndrome is a rare, life threatening, genetic disease which affects young males. It is caused by abnormal fats (lipids) in the powerhouses of cells (mitochondria) and those who suffer with it...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2019-003192-18 Sponsor Protocol Number: NCT-PMO-1602 Start Date*: 2021-07-08
    Sponsor Name:German Cancer Research Center
    Full Title: Continuous ReAssessment with Flexible ExTension in Rare Malignancies – CRAFT: The NCT PMO-1602 Phase II Trial
    Medical condition: Metastatic or locally advanced malignancies
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10065147 Malignant solid tumor LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-001181-10 Sponsor Protocol Number: P003077 Start Date*: 2023-04-13
    Sponsor Name:Medical Center - University of Freiburg
    Full Title: Age-adjusted high-dose chemotherapy followed by autologous stem cell transplantation or conventional chemotherapy with R-MP as first-line treatment in elderly primary CNS lymphoma patients – a rand...
    Medical condition: Primary diffuse large B-cell lymphoma of the central nervous system (PCNSL) is a rare lymphoma affecting only the central nervous system compartment. PCNSL patients are typically 60 years or older ...
    Disease:
    Population Age: Elderly Gender: Male, Female
    Trial protocol: AT (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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