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Clinical trials for Mutation accumulation

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    The EU Clinical Trials Register currently displays   44334   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).

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    33 result(s) found for: Mutation accumulation. Displaying page 1 of 2.
    1  2  Next»
    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: 2008-003059-56 Sponsor Protocol Number: ICAND Start Date*: 2008-07-29
    Sponsor Name:ISTITUTO NEUROLOGICO "CARLO BESTA"
    Full Title: Phase II trial to assess safety and efficacy of Iron chelating agent Deferiprone in patients with Pantothenate Kinase-Associated Neurodegeneration – ICAND – Trial
    Medical condition: PKAN
    Disease: Version SOC Term Classification Code Term Level
    9.1 10042259 Structural brain disorders NEC HLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2006-002099-16 Sponsor Protocol Number: 06/Q0703/56 Start Date*: 2006-07-13
    Sponsor Name:King's College London
    Full Title: The effect of milk thistle extract (Legalon) versus tea on serum iron increase after a meal containing non-haem iron in hereditary haemochromatosis
    Medical condition: Type 1 hereditary haemochromatosis is associated with homozygous inheritance of the C282Y mutation in the HFE gene. The phenotype is characterised by increased serum iron levels, tissue iron accumu...
    Disease: Version SOC Term Classification Code Term Level
    8.1 10057873 Hereditary haemochromatosis LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2022-001596-14 Sponsor Protocol Number: TYR300-101 Start Date*: 2023-06-26
    Sponsor Name:Tyra Biosciences, Inc.
    Full Title: A Multicenter, Open-label Phase 1/2 Study of TYRA 300 in Advanced Urothelial Carcinoma and Other Solid Tumors with Activating FGFR3 Gene Alterations (SURF 301)
    Medical condition: Advanced Urothelial Carcinoma and Other Solid Tumors with Activating FGFR3 Gene Alterations
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004864 10049280 Solid tumour LLT
    21.1 100000004864 10065143 Malignant solid tumour LLT
    21.1 100000004864 10065147 Malignant solid tumor LLT
    21.1 100000004864 10065252 Solid tumor LLT
    20.0 100000004864 10064467 Urothelial carcinoma LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2013-001265-16 Sponsor Protocol Number: COEB071X2103 Start Date*: 2013-09-30
    Sponsor Name:Novartis Farma S.p.A
    Full Title: An Open-Label, Single-arm, Phase Ib/II study of AEB071 (a Protein Kinase C Inhibitor) and Everolimus (mTOR inhibitor) in Patients with CD79-mutant or ABC subtype Diffuse Large B-Cell Lymphoma
    Medical condition: Diffuse Large B-cell Lymphoma (DLBCL)
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Prematurely Ended) DE (Prematurely Ended) NL (Prematurely Ended) FR (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-000359-26 Sponsor Protocol Number: ESTEVE-SANF-201 Start Date*: 2016-04-29
    Sponsor Name:Laboratorios del Dr. Esteve, S.A.
    Full Title: Phase I/II safety, tolerability and initial efficacy study of adeno-associated viral vector serotype 9 containing human sulfamidase gene after intracerebroventricular administration to patients wit...
    Medical condition: Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome) is an inherited lysosomal storage disease caused by a specific lysosomal enzyme deficiency that leads to intracellular accumulation of the ...
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-002941-49 Sponsor Protocol Number: ERRITI Start Date*: 2017-01-31
    Sponsor Name:University Hospital Essen (Anstalt des öffentlichen Rechts)
    Full Title: Enhancing Radioiodine Incorporation into Radio Iodine Refractory Thyroid Cancers with MAPK Inhibition: A single center pilot study
    Medical condition: Radio Iodine Refractory Thyroid Cancer
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-001955-29 Sponsor Protocol Number: 024PKAN15004 Start Date*: Information not available in EudraCT
    Sponsor Name:Retrophin, Inc.
    Full Title: Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement therapy, in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN): A Randomized, ...
    Medical condition: Pantothenate kinase associated neurodegeneration (PKAN), an autosomal recessive genetic disorder, the most common form of Neurodegeneration with Brain Iron Accumulation (NBIA). It is a progressive,...
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10053643 Neurodegenerative disorder PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) ES (Prematurely Ended) GB (Prematurely Ended) CZ (Prematurely Ended) NO (Completed) FR (Completed) PL (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2020-002924-35 Sponsor Protocol Number: APHP180592 Start Date*: 2021-01-18
    Sponsor Name:ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS
    Full Title: Prenatal treatment of congenital cytomegalovirus infection with letermovir randomized against valaciclovir
    Medical condition: Step 1: Maternal administration of 1 tablet of Letermovir (240 mg or 480 mg /day) during 3 days before TOP Step 2: Maternal daily administration of 240 or 480 milligrams of letermovir (1x240 mg-ta...
    Disease:
    Population Age: Children, Under 18, Adults Gender: Female
    Trial protocol: FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-005733-16 Sponsor Protocol Number: DNLI-H-0001 Start Date*: 2022-07-19
    Sponsor Name:Denali Therapeutics Inc.
    Full Title: A Phase 1/2, Multicenter, Randomized, Placebo-Controlled, Double-Blind Single Dose and Multiple Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL593 in ...
    Medical condition: Frontotemporal Dementia (FTD)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10068968 Frontotemporal dementia PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Temporarily Halted) PT (Trial now transitioned) CZ (Completed) FR (Trial now transitioned) NL (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2017-003369-85 Sponsor Protocol Number: ID-069A301 Start Date*: 2018-05-14
    Sponsor Name:Idorsia Pharmaceuticals Ltd
    Full Title: A multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.
    Medical condition: Fabry disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) AT (Completed) NL (Completed) BE (Completed) IE (Completed) ES (Ongoing) NO (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2015-000508-24 Sponsor Protocol Number: 7406 Start Date*: 2015-06-12
    Sponsor Name:Newcastle Upon Tyne Hospitals NHS Trust
    Full Title: A Feasibility Study of Bezafibrate in Mitochondrial Myopathy
    Medical condition: Mitochondrial myopathy due to mt.3243A>G mutation
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10027710 Mitochondrial myopathy PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2022-001535-87 Sponsor Protocol Number: BLU-263-2101 Start Date*: 2023-02-03
    Sponsor Name:Blueprint Medicines Corporation
    Full Title: A Phase 1/2, open-label, 2-arm study evaluating BLU-263 as monotherapy and in combination with azacitidine, in patients with KIT altered hematologic malignancies
    Medical condition: Advanced Systemic Mastocytosis (AdvSM)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10042949 Systemic mastocytosis PT
    26.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10056453 Aggressive systemic mastocytosis LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NO (Trial now transitioned) BE (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-004390-41 Sponsor Protocol Number: BLU-667-1101 Start Date*: 2018-03-13
    Sponsor Name:Blueprint Medicines
    Full Title: A Phase 1/2 Study of the Highly-selective RET Inhibitor, BLU-667, in Patients with Thyroid Cancer, Non-Small Cell Lung Cancer (NSCLC) and Other Advanced Solid Tumors
    Medical condition: Thyroid Cancer, Non-Small Cell Lung Cancer (NSCLC) and Other Advanced Solid Tumors
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004864 10066914 Thyroid tumor LLT
    20.0 100000004864 10025044 Lung cancer LLT
    21.1 100000004864 10065252 Solid tumor LLT
    21.1 100000004864 10029514 Non-small cell lung cancer NOS LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed) NL (Completed) BE (Completed)
    Trial results: View results
    EudraCT Number: 2016-001057-40 Sponsor Protocol Number: 301OTC01 Start Date*: 2017-03-14
    Sponsor Name:Ultragenyx Pharmaceutical, Inc.
    Full Title: A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OT...
    Medical condition: Ornithine transcarbamylase deficiency
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10071107 Ornithine transcarbamylase deficiency LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) ES (Completed)
    Trial results: View results
    EudraCT Number: 2018-004050-16 Sponsor Protocol Number: HEAT-AML Start Date*: 2019-08-01
    Sponsor Name:University Hospital Karolinska
    Full Title: HEAT-AML (Hydroxyurea-Enhanced Ara-C Treatment of Adult Acute Myeloid Leukaemia): A phase I/II multicenter study to assess the tolerability and efficacy of the addition of hydroxyurea to standard a...
    Medical condition: Newly diagnosed acute myeloid leukaemia (AML) in patients ≥ 18 years of age
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004851 10024291 Leukaemias acute myeloid HLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2022-002314-17 Sponsor Protocol Number: JR-441-101 Start Date*: 2023-07-03
    Sponsor Name:JCR Pharmaceuticals Co., Ltd.
    Full Title: Phase I/II study of weekly infusions of JR-441 in patients with mucopolysaccharidosis type IIIA
    Medical condition: Mucopolysaccharidosis type IIIA (MPS IIIA) MPS III is an autosomal recessive disease characterized by the accumulation of HS in the tissues throughout the body due to a deficiency in enzymes involv...
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-004909-27 Sponsor Protocol Number: DNLI-E-0002 Start Date*: 2020-07-22
    Sponsor Name:Denali Therapeutics Inc.
    Full Title: A PHASE 1/2, MULTICENTER, OPEN-LABEL STUDY TO DETERMINE THE SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF DNL310 IN PEDIATRIC SUBJECTS WITH HUNTER SYNDROME
    Medical condition: Hunter Syndrome (Mucopolysaccharidosis Type II [MPS II])
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10056917 Hunter's syndrome LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-006048-15 Sponsor Protocol Number: ALL2820 Start Date*: 2021-05-03
    Sponsor Name:FONDAZIONE GIMEMA (GRUPPO ITALIANO MALATTIE EMATOLOGICHE DELL' ADULTO) FRANCO MANDELLI ONLUS
    Full Title: Newly Diagnosed Adult Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ ALL). Sequential Treatment with Ponatinib and the Bispecific Monoclonal Antibody Blinatumomab vs Chemotherap...
    Medical condition: Adult Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia.
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004864 10000845 Acute lymphoblastic leukemia LLT
    21.0 100000004864 10000845 Acute lymphoblastic leukemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2011-002569-39 Sponsor Protocol Number: CINC280X2202 Start Date*: 2013-03-14
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A phase IB/II, open label, multicenter study of INC280 administered orally in combination with gefitinib in adult patients with EGFR mutated, c-MET-amplified non-small cell lung cancer who have pro...
    Medical condition: Non-Small Cell Lung Cancer
    Disease: Version SOC Term Classification Code Term Level
    17.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10061873 Non-small cell lung cancer PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) IT (Completed) ES (Completed) DE (Completed) NL (Completed) FR (Completed)
    Trial results: View results
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