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Clinical trials for Compassionate use

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   44336   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.
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    174 result(s) found for: Compassionate use. Displaying page 1 of 9.
    1  2  3  4  5  6  7  8  9  Next»
    EudraCT Number: 2012-003499-37 Sponsor Protocol Number: DSC/11/2357/44 Start Date*: 2013-01-20
    Sponsor Name:ITALFARMACO
    Full Title: Long-term study evaluating the effect of Givinostat in patients with JAK2V617F positive chronic myeloproliferative neoplasms.
    Medical condition: Chronic myeloproliferative neoplasms (cMPN).
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004851 10028578 Myeloproliferative disorders (excl leukaemias) HLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned) DE (Completed) PL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2012-002692-34 Sponsor Protocol Number: MitoFibrateCT1-2012 Start Date*: 2013-06-26
    Sponsor Name:Univ.-Klinik für Kinder- und Jugendheilkunde
    Full Title: Clinical study for the assessment of safety and efficacy of Bezafibrate as a drug therapy for patients with mitochondrial myopathies
    Medical condition: mitochondrial disorder
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: AT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2005-005263-29 Sponsor Protocol Number: 25-01 Start Date*: 2006-01-31
    Sponsor Name:Gesellschaft fuer klinische Forschung e.V. (Society for Clinical Research) [...]
    1. Gesellschaft fuer klinische Forschung e.V. (Society for Clinical Research)
    2. WELEDA AG
    Full Title: Multiple Sclerosis and Extract of Cannabis (MUSEC): A randomised, double-blind, placebo-controlled phase III trial to determine the efficacy and safety of a standardised oral extract of Cannabis sa...
    Medical condition: Multiple Sclerosis
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: (No results available)
    EudraCT Number: 2005-004827-20 Sponsor Protocol Number: NC6004-001 Start Date*: 2006-03-15
    Sponsor Name:NanoCarrier Co., Ltd.
    Full Title: Phase I Study to Determine the Maximum Tolerated Dose of NC-6004 in Patients with Solid Tumours
    Medical condition: Histologically-confirmed advanced solid tumours for which no standard therapy exists or has failed therapy
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2006-003879-12 Sponsor Protocol Number: MK-0683 042-00 Start Date*: 2007-03-23
    Sponsor Name:Merck & Co., Inc.
    Full Title: Compassionate Use of Vorinostat (MK-0683) for the Treatment of Patients with Advanced Cutaneous T-Cell Lymphoma
    Medical condition: Cutaneous T-cell lymphoma (CTCL)
    Disease: Version SOC Term Classification Code Term Level
    8.1 10011677 Cutaneous T-cell lymphoma LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) IE (Completed) DK (Completed) FI (Completed) SI (Completed) IT (Prematurely Ended) NL (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2013-000930-37 Sponsor Protocol Number: STEMQUIRI/12ES01 Start Date*: 2014-04-29
    Sponsor Name:Laboratorios Salvat, S.A.
    Full Title: A phase II clinical trial to assess the effect of HC-SVT-1001 (autologous fat stem adult mesenchymal cells expanded and combined with a tricalcium phosphate biomaterial) and HC-SVT-1002 (allogeneic...
    Medical condition: Atrophic pseudarthrosis
    Disease: Version SOC Term Classification Code Term Level
    21.1 10028395 - Musculoskeletal and connective tissue disorders 10048617 Pseudarthrosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Completed)
    Trial results: (No results available)
    EudraCT Number: 2005-001843-49 Sponsor Protocol Number: P04099 Start Date*: 2005-09-13
    Sponsor Name:Schering Plough Research Institute
    Full Title: Vicriviroc (SCH 417690) Treatment Protocol in HIV-Infected Subjects: A Substudy of P03802
    Medical condition: HIV infection
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2005-000590-21 Sponsor Protocol Number: FFC33-24 Start Date*: 2005-08-24
    Sponsor Name:Faustus Forschung Compagnie, Translational Cancer Research GmbH
    Full Title: A Phase II Study of 1-(2-chloroethyl)-1-nitroso-3-(2-hydroxyethyl)-urea (HECNU) in pancreatic cancer
    Medical condition: Pancreatic cancer
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2005-000627-42 Sponsor Protocol Number: BCX1777-Bi-04-106 Start Date*: 2006-05-23
    Sponsor Name:BioCryst Pharmaceuticals Inc.
    Full Title: A Phase I/II, Multi-Center, Open-Label, Repeat-Dose Study of Forodesine Hydrochloride Infusion in Patients with B-cell Acute Lymphoblastic Leukemia with an Option of Extended Use of Forodesine Hydr...
    Medical condition: B-cell acute lymphoblastic leukemia (B-ALL), which under WHO Guidelines is now referred to as precursor B-lymphoblastic leukemia/lymphoma
    Disease: Version SOC Term Classification Code Term Level
    8.1 10036523 hlgt
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2018-001321-68 Sponsor Protocol Number: B1761026 Start Date*: 2018-05-22
    Sponsor Name:Pfizer, Inc.
    Full Title: Gemtuzumab Ozogamicin (mylotarg (registered)) Expanded Access Protocol For Treatment Of Patients In The United States With Relapsed/refractory Acute Myelogenous Leukemia Who May Benefit From Treatm...
    Medical condition: Acute Myelogenous Leukemia, including myelodysplastic syndrome and acute promyelocytic leukemia
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10000880 Acute myeloid leukaemia PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-003705-34 Sponsor Protocol Number: RDC-PDE6A-01 Start Date*: 2019-07-02
    Sponsor Name:Universitätsklinikum Tübingen
    Full Title: PIGMENT – PDE6A gene therapy for retinitis pigmentosa
    Medical condition: PDE6A-linked retinitis pigmentosa
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-003151-11 Sponsor Protocol Number: CNTO1275UCO2001 Start Date*: 2020-05-08
    Sponsor Name:UZ Leuven
    Full Title: Stelara fOr ChRonic AntibioTic rEfractory pouchitiS (SOCRATES): A Belgian open label multicenter pilot-study
    Medical condition: Chronic antibiotic refractory and relapsing pouchitis
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2004-004249-16 Sponsor Protocol Number: F02207GE204 Start Date*: 2005-02-02
    Sponsor Name:PIERRE FABRE MEDICAMENT
    Full Title: THE EFFECT OF MILNACIPRAN 100MG BID ON SENSITIVITY TO STIMULUS-EVOKED PAIN IN PATIENTS WITH FIBROMYALGIA: A FMRI NEUROIMAGING STUDY
    Medical condition: Fibromyalgia is a chronic disorder characterized by widespread musculoskeletal pain, widespread tenderness and fatigue. The disorder is well defined in the USA, where the current diagnosis of FMS i...
    Disease:
    Population Age: Adults Gender: Female
    Trial protocol: GB (Completed) SE (Completed) DE (Completed)
    Trial results: View results
    EudraCT Number: 2011-004259-38 Sponsor Protocol Number: CAC-91-10-10 Start Date*: 2011-10-06
    Sponsor Name:Asklepion Pharmaceuticals, LLC
    Full Title: Investigation in the Pathogenesis of Liver Disease in Patients with Inborn Errors of bile Acid Metabolism
    Medical condition: Patients with inborn errors of bile acid synthesis and metabolism
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10070882 Inborn error in primary bile acid synthesis PT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: (No results available)
    EudraCT Number: 2007-006345-40 Sponsor Protocol Number: rhASA-03 Start Date*: 2008-08-26
    Sponsor Name:Shire Pharmaceutical Ireland Limited
    Full Title: A single center, open-label, non-randomized, uncontrolled, multiple-dose study of the efficacy and long-term safety of Metazym (recombinant human arylsulfatase A or rhASA) for the treatment of pati...
    Medical condition: Metachromatic Leukodystrophy (MLD) in late infantile patients
    Disease: Version SOC Term Classification Code Term Level
    9.1 10024381 Leukodystrophy LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2007-007165-20 Sponsor Protocol Number: rhASA-04 Start Date*: 2008-02-26
    Sponsor Name:Shire Pharmaceuticals Ireland Limited
    Full Title: A single center, open-label, non-randomized, uncontrolled, multiple-dose study of the efficacy and safety of Metazym (recombinant human arylsulfatase A or rhASA) for the treatment of MLD patients w...
    Medical condition: Metachromatic Leukodystrophy (MLD) in late infantile patients
    Disease: Version SOC Term Classification Code Term Level
    9.1 10024381 Leukodystrophy LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2004-000482-35 Sponsor Protocol Number: ARA102198 Start Date*: 2004-10-11
    Sponsor Name:GlaxoSmithKline Research & Development Ltd.
    Full Title: A randomised, double-blind, placebo-controlled, parallel group study to investigate the anti-inflammatory and metabolic effects of rosiglitazone XR, 8mg once daily, in subjects with rheumatoid arth...
    Medical condition: Rheumatoid Arthritis
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) LT (Completed)
    Trial results: View results
    EudraCT Number: 2006-000648-15 Sponsor Protocol Number: BH4/III/05/001 Start Date*: 2006-06-08
    Sponsor Name:ORPHANETICS Pharma Entwicklungs- GmbH
    Full Title: A double-blind, placebo-controlled, multicentre study with an open-label extension to evaluate the efficacy and safety of tetrahydrobiopterin (BH4) in children and adolescents with hyperphenylalani...
    Medical condition: Hyperphenylalaninemia due to phenylalanine hydroxylase deficiency. Phenotypes: classic phenylketonuria (PKU), mild PKU (MPK) or mild hyperphenylalaninemia (HPA).
    Disease: Version SOC Term Classification Code Term Level
    81 10034873 LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: AT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2006-003581-34 Sponsor Protocol Number: DASATINIB compassionate use Start Date*: 2006-07-27
    Sponsor Name:OSPEDALE S. RAFFAELE
    Full Title: DASATINIB compassionate use
    Medical condition: CML and Ph
    Disease: Version SOC Term Classification Code Term Level
    6.1 10000880 PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-002158-35 Sponsor Protocol Number: IVA_01_ODI_HMPS_17_002 Start Date*: 2017-11-16
    Sponsor Name:Inventiva S.A.
    Full Title: A phase IIa study to investigate safety, Pharmacokinetics, and efficacy of odiparcil in patients 16 years and above with mucopolysaccharidosis (MPS) type VI.
    Medical condition: Mucopolysaccharidosis (MPS) type VI.
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10028095 Mucopolysaccharidosis IV PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) FR (Completed) PT (Completed)
    Trial results: View results
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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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