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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   44335   clinical trials with a EudraCT protocol, of which   7366   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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    410 result(s) found for: Rare diseases. Displaying page 1 of 21.
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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 (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-002260-14 Sponsor Protocol Number: UC-0105/1612 Start Date*: 2017-04-03
    Sponsor Name:UNICANCER
    Full Title: Secured access to pembrolizumab for adult patients with selected rare cancer types.
    Medical condition: Patients with unresectable locally advanced or metastatic, rare soft tissue sarcoma, visceral sarcoma, or bone sarcoma, rare ovarian cancer, primary central nervous system lymphomas (PCNSL), rare t...
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2021-006702-78 Sponsor Protocol Number: BraimTOR-NEURO Start Date*: Information not available in EudraCT
    Sponsor Name:The Children's Memorial Health Institute
    Full Title: Open, randomized, IInd phase clinical study evaluating the safety and efficacy of rapamycin in the treatment of drug-resistant epilepsy in children with rare and ultra-rare diseases of the central ...
    Medical condition: epilepsy, mTORopathies, focal cortical dysplasia, LEATS
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: PL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-002257-37 Sponsor Protocol Number: UC-0105/1611 Start Date*: 2017-02-10
    Sponsor Name:UNICANCER
    Full Title: Secured access to nivolumab for adult patients with selected rare cancer types
    Medical condition: Unresectable locally advanced or metastatic, non-clear cell renal cell carcinoma, rare head and neck cancer, rare skin cancer, MSI-nonCRC or penile cancer, which is resistant or refractory to stand...
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2013-001705-87 Sponsor Protocol Number: BRF117019 Start Date*: 2014-03-06
    Sponsor Name:GlaxoSmithKline Research & Development Ltd
    Full Title: A Phase II, Open-label, Study in Subjects with BRAF V600E Mutated Rare Cancers with Several Histologies to Investigate the Clinical Efficacy and Safety of the Combination Therapy of Dabrafenib and ...
    Medical condition: Mutations of BRAF V600E have been identified at a high frequency in melanoma, PTC, colorectal and ovarian cancers. Such mutations have also been reported in some rare cancers such as anaplastic t...
    Disease: Version SOC Term Classification Code Term Level
    16.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10029104 Neoplasms benign, malignant and unspecified (incl cysts and polyps) SOC
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed) SE (Completed) BE (Completed) AT (Completed) DK (Completed) NL (Completed) ES (Ongoing) NO (Completed)
    Trial results: View results
    EudraCT Number: 2019-001238-32 Sponsor Protocol Number: ADVL1622 Start Date*: 2019-09-26
    Sponsor Name:National Cancer Institute Cancer Therapy Evaluation
    Full Title: Phase 2 Trial of XL184 (Cabozantinib) an Oral Small-Molecule Inhibitor of Multiple Kinases, in Children and Young Adults with Refractory Sarcomas, Wilms Tumor, and Other Rare Tumors
    Medical condition: Ewing sarcoma, Rhabdomyosarcoma, Non rhabdomyosarcoma, Wilms Tumor, Osteosarcoma and Other Rare Tumors
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2006-005003-33 Sponsor Protocol Number: WSA-CS-003 Start Date*: 2008-06-26
    Sponsor Name:Astellas Global Pharma Development, Inc
    Full Title: Open label study of isavuconazole in the treatment of patients with aspergillosis and renal impairment or of patients with invasive fungal disease caused by rare moulds, yeasts or dimorphic fungi.
    Medical condition: Invasive fungal disease caused by Aspergillus species in patients with renal impairment as well as disease caused by rare moulds, yeasts or dimorphic fungi.
    Disease: Version SOC Term Classification Code Term Level
    15.0 10021881 - Infections and infestations 10003486 Aspergillus infections HLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) BE (Completed) HU (Completed) CZ (Prematurely Ended) DE (Completed) ES (Completed)
    Trial results: View results
    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 (Completed)
    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: 2018-002984-24 Sponsor Protocol Number: CYT-C2-001 Start Date*: 2019-10-25
    Sponsor Name:RECORDATI Rare Diseases SARL
    Full Title: Open-label, Single-arm, Multicenter Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Than 2 Years Old [SCOB2 Study]
    Medical condition: Corneal cystine crystal deposits in pediatric patients with nephropathic cystinosis from 6 months to less than 2 years old
    Disease:
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Ongoing) DE (Ongoing) BE (Completed) IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2014-002392-28 Sponsor Protocol Number: RM-493-Supplementation-Therapy Start Date*: 2014-11-28
    Sponsor Name:Charité Univeritaetsmedizin Berlin
    Full Title: Setmelanotide (RM-493) Treatment Trial in Patients with rare genetic disorders of obesity
    Medical condition: Body weight is regulated within the hypothalamus. In rare cases mutations in genes, which are embedded in the signaling cascades of the hypothalamus lead to early onset severe obesity. POMC is one ...
    Disease:
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2017-000387-14 Sponsor Protocol Number: RM-493-014 Start Date*: 2017-05-24
    Sponsor Name:Rhythm Pharmaceuticals, Inc.
    Full Title: Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients with rare genetic disorders of obesity
    Medical condition: - POMC/PCSK1/LEPR heterozygous - POMC/PCSK1/LEPR compound heterozygous or homozygous deficiency obesity - POMC/PCSK1/LEPR composite heterozygous deficiency obesity - Smith-Magenis Syndrome - SH2B1...
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) ES (Ongoing) DE (Completed) FR (Ongoing) NL (Completed) GR (Completed)
    Trial results: View results
    EudraCT Number: 2013-000975-33 Sponsor Protocol Number: RDCRN_RKSC_6412 Start Date*: 2013-11-19
    Sponsor Name:Landspitali - The National University Hospital of Iceland
    Full Title: Novel Assays for the Determination of Urinary 2,8-Dihydroxyadenine and Other Key Urinary Purine Metabolites - Rare Diseases Clinical Research Network Protocol Version 1
    Medical condition: Adenine phosphoribosyltransferase deficiency
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IS (Completed)
    Trial results: View results
    EudraCT Number: 2021-004548-64 Sponsor Protocol Number: RaRETS Start Date*: Information not available in EudraCT
    Sponsor Name:The Children's Memorial Health Institute
    Full Title: Multicenter, randomized, double-blind, placebo controlled study to assess the efficacy and safety of Rapamycin in drug Resistant Epilepsy associated with TSC (RaRE-TS)
    Medical condition: tuberous sclerosis complex, epilepsy, organ tumors associated with tuberous sclerosis
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: PL (Trial now transitioned)
    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 (Completed) FR (Completed) GB (GB - no longer in EU/EEA) DE (Completed) ES (Ongoing) IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-003311-19 Sponsor Protocol Number: AIH-MAB Start Date*: 2018-01-09
    Sponsor Name:University Medical Centre Hamburg-Eppendorf
    Full Title: Phase IIa proof-of-principle study for the induction treatment of autoimmune hepatitis using infliximab AIH-MAB
    Medical condition: Autoimmune Hepatitis (AIH); rare inflammatory liver disease associated with elevated transaminases, elevated Immunoglobulin G, the presence of autoantibodies and interface hepatitis in liver histol...
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2016-000901-35 Sponsor Protocol Number: 2016-02 Start Date*: 2017-07-10
    Sponsor Name:ASISTANCE PUBLIQUE HOPITAUX DE MARSEILLE
    Full Title: Riluzole for the treatment of spasticity in the traumatic chronic spinal cord injury condition: Adaptive, Multicenter, placebo-controlled, randomised, double blind trial in a Rare Disorder” RILUSCI
    Medical condition: the traumatic chronic spinal cord injury condition
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-001386-33 Sponsor Protocol Number: APX001-202 Start Date*: 2019-08-20
    Sponsor Name:Amplyx Pharmaceuticals, Inc., a wholly owned subsidiary of Pfizer, Inc.
    Full Title: A Phase 2, Open-Label Study to Evaluate the Safety and Efficacy of APX001 in the Treatment of Patients with Invasive Mold Infections Caused by Aspergillus Species or Rare Molds
    Medical condition: Treatment of patients with invasive mold infections (IMIs) caused by Aspergillus species (spp.) or rare molds
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004862 10062642 Invasive mycosis LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2016-002638-54 Sponsor Protocol Number: ALN-AS1-002 Start Date*: 2017-12-20
    Sponsor Name:Alnylam Pharmaceuticals, Inc.
    Full Title: A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients with Acute Intermittent Porphyria who have Comple...
    Medical condition: Acute Intermittent Porphyria (AIP)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000818 Acute intermittent porphyria LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed) GB (GB - no longer in EU/EEA)
    Trial results: View results
    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 (Trial now transitioned)
    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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