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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   44349   clinical trials with a EudraCT protocol, of which   7376   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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    148 result(s) found for: rare diseases. Displaying page 1 of 8.
    1  2  3  4  5  6  7  8  Next»
    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: 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: 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: 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: 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: 2011-000130-11 Sponsor Protocol Number: EryDexFCTx1.0 Start Date*: 2011-07-25
    Sponsor Name:AZIENDA UNIVERSITARIA POLICLINICO UMBERTO I DI ROMA
    Full Title: Clinical trials in rare diseases: therapeutic alternative to the use of oral corticosteroids in patients with cystic fibrosis underwent double lung transplantation.
    Medical condition: Double lung transplantation in patients with Cystic Fibrosis
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10011763 Cystic fibrosis lung PT
    14.1 10042613 - Surgical and medical procedures 10025127 Lung transplant PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2022-002611-29 Sponsor Protocol Number: 82050 Start Date*: 2023-06-12
    Sponsor Name:Leiden University Medical Center
    Full Title: DEnosumab for the treatment of FIbrous Dysplasia/McCune-Albright Syndrome in adults (DeFiD): a randomized double-blind placebo-controlled trial
    Medical condition: Fibrous Dysplasia/ McCune Albright Syndrome
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-004611-50 Sponsor Protocol Number: CCTU0228 Start Date*: 2019-06-14
    Sponsor Name:Cambridge University Hospitals NHS Foundation Trust
    Full Title: A randomised, two-arm (1:1 ratio), double blind, placebo controlled phase III trial to assess the efficacy, safety, cost and cost-effectiveness of rituximab in treating de novo or relapsing NS in p...
    Medical condition: Minimal Change Disease (MCD) and Focal segmental glomerulosclerosis (FSGS)
    Disease:
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2017-000653-39 Sponsor Protocol Number: IMR-SCD-102 Start Date*: 2017-10-19
    Sponsor Name:Imara, Inc
    Full Title: A Phase 2a, Randomised, Double-Blind, Placebo-Controlled Study of IMR-687 in Adult Patients with Sickle Cell Anaemia (Homozygous HbSS or Sickle-β0 Thalassemia)
    Medical condition: Sickle cell disease (SCD) is a rare inherited red blood cell (RBC) disorder characterized by chronic hemolysis, debilitating pain, progressive multiorgan damage, and premature death. SCD is caused ...
    Disease: Version SOC Term Classification Code Term Level
    21.0 10010331 - Congenital, familial and genetic disorders 10040644 Sickle cell disease PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2019-004628-39 Sponsor Protocol Number: ABACUS-2 Start Date*: 2020-12-16
    Sponsor Name:Queen Mary University of London
    Full Title: A phase II study of neoadjuvant immune checkpoint inhibitors in urothelial cancer
    Medical condition: Tumours of the urothelial tract requiring surgery (T1 high grade-T4a of the bladder, rare histological subtypes) and upper urinary tract (high grade or high risk)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10046384 Ureteral cancer LLT
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10026426 Malignant neoplasm of renal pelvis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2014-002496-28 Sponsor Protocol Number: PVO-1A-202 Start Date*: 2015-11-13
    Sponsor Name:Clementia Pharmaceuticals Inc.
    Full Title: A Phase 2, Open-Label Extension, Efficacy and Safety Study of a RARγ-Specific Agonist (Palovarotene) in the Treatment of Preosseous Flare-ups in Subjects with Fibrodysplasia Ossificans Progressiva ...
    Medical condition: Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) and abnormal heterotopic ossificat...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10068715 Fibrodysplasia ossificans progressiva PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2018-002721-29 Sponsor Protocol Number: 08486 Start Date*: 2018-12-24
    Sponsor Name:Newcastle upon Tyne Hospitals NHS Foundation Trust
    Full Title: Randomised, double-blinded, placebo-controlled, adaptive design trial of the efficacy of acipimox in patients with Mitochondrial Myopathy
    Medical condition: Mitochondrial Myopathy
    Disease:
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2019-004345-32 Sponsor Protocol Number: ANAVEX2-73-RS-002 Start Date*: 2020-05-26
    Sponsor Name:Anavex Germany GmbH
    Full Title: A Double-Blind, Randomised, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients with Rett Syndrome
    Medical condition: Rett Syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10077709 Rett syndrome PT
    Population Age: Adults, Elderly Gender: Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2017-004021-33 Sponsor Protocol Number: ANB019-002 Start Date*: 2018-05-04
    Sponsor Name:AnaptysBio Inc.
    Full Title: A Single Arm Multiple Dose Study to Assess the Efficacy and Safety of ANB019 in Subjects with Generalized Pustular Psoriasis
    Medical condition: Generalized Pustular Psoriasis (GPP)
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004858 10037159 Psoriasis pustular LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2018-002857-31 Sponsor Protocol Number: SB12-3003 Start Date*: 2019-09-09
    Sponsor Name:Samsung Bioepis Co., Ltd.
    Full Title: A Phase III Randomised, Double-blind, Multicentre Study to Compare the Efficacy, Safety, Pharmacokinetics, and Immunogenicity between SB12 (proposed eculizumab biosimilar) and Soliris® in Subjects ...
    Medical condition: Paroxysmal Nocturnal Haemoglobinuria
    Disease: Version SOC Term Classification Code Term Level
    21.1 10038359 - Renal and urinary disorders 10034042 Paroxysmal nocturnal haemoglobinuria PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: RO (Ongoing)
    Trial results: View results
    EudraCT Number: 2017-003916-37 Sponsor Protocol Number: 08448 Start Date*: 2018-04-13
    Sponsor Name:Newcastle Upon Tyne Hospitals NHS Foundation Trust
    Full Title: Multicentre, open label, prospective, single arm study of the safety and impact of eculizumab withdrawal in patients with atypical haemolytic uraemic syndrome
    Medical condition: Atypical Haemolytic Uraemic Syndrome (aHUS)
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2015-003060-37 Sponsor Protocol Number: ANZUP1302 Start Date*: 2017-04-05
    Sponsor Name:Cambridge University Hospitals NHS Foundation Trust
    Full Title: UK P3BEP - A randomised phase 3 trial of accelerated versus standard BEP chemotherapy for patients with intermediate and poor-risk metastatic germ cell tumours
    Medical condition: International germ cell classification catergory (IGCCC) intermediate or poor-risk metastatic germ cell tumours
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10068971 Germ cell cancer metastatic PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2019-003529-36 Sponsor Protocol Number: mRNA-3927-P101 Start Date*: 2020-12-18
    Sponsor Name:ModernaTX, Inc
    Full Title: A Global, Phase 1/2, Open-label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants with Propionic Acidemia
    Medical condition: Propionic Acidemia- Propionic acidemia is an autosomal recessive disorder caused by loss-of-function mutations in PCCA or PCCB (and in rare instances, mutations in both PCCA and PCCB), resulting in...
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004850 10080615 Propionic acidemia LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2017-004761-28 Sponsor Protocol Number: GEIS41 Start Date*: 2019-06-20
    Sponsor Name:Grupo Español de Investigación en Sarcomas
    Full Title: A multicenter, phase Ib/II trial of selinexor in combination with imatinib in patients with metastatic and/or unresectable gastrointestinal stromal tumors (GISTs)
    Medical condition: Gastrointestinal stromal tumor (GIST)
    Disease:
    Population Age: 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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