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Clinical trials for Hemoglobin variants

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   44357   clinical trials with a EudraCT protocol, of which   7383   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 ).  
     
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    69 result(s) found for: Hemoglobin variants. Displaying page 1 of 4.
    1  2  3  4  Next»
    EudraCT Number: 2021-004167-27 Sponsor Protocol Number: RM-493-033 Start Date*: 2022-03-02
    Sponsor Name:Rhythm Pharmaceuticals Limited
    Full Title: A Phase 3 Multi-Center, One-Year, Open-Label study of Setmelanotide in Pediatric Patients Aged 2 to <6 years of age with Rare Genetic Causes of Obesity
    Medical condition: POMC deficiency obesity due to mutations in the POMC gene PCSK1 deficiency due to mutations in the PCSK1 gene LEPR deficiency obesity due to mutations in the LEPR gene Bardet-Biedl syndrome
    Disease: Version SOC Term Classification Code Term Level
    23.0 10010331 - Congenital, familial and genetic disorders 10084105 Leptin receptor deficiency PT
    23.0 10010331 - Congenital, familial and genetic disorders 10083937 Pro-opiomelanocortin deficiency PT
    20.1 10010331 - Congenital, familial and genetic disorders 10048680 Bardet-Biedl syndrome LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: NL (Prematurely Ended) ES (Completed)
    Trial results: View results
    EudraCT Number: 2019-003766-41 Sponsor Protocol Number: COVERAGE Start Date*: 2020-09-16
    Sponsor Name:Amsterdam UMC-AMC
    Full Title: The effect of Voxelotor on Cerebral Perfusion and Oxygenation (Coverage Study)
    Medical condition: Sickle Cell Disease
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-000178-31 Sponsor Protocol Number: TREATMENT-patients Start Date*: 2018-05-23
    Sponsor Name:Fundación para la Investigación e Innovación Biosanitaria de Asturias (FINBA)
    Full Title: Metabolic Dysfunctions Associated with Pharmacological Treatment of Schizophrenia
    Medical condition: Schizophrenia
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: ES (Completed)
    Trial results: (No results available)
    EudraCT Number: 2021-006224-40 Sponsor Protocol Number: AxGD Start Date*: Information not available in EudraCT
    Sponsor Name:Instytut ,,Pomnik-Centrum Zdrowia Dziecka"
    Full Title: Evaluation of the safety and efficacy of ambroxol (ABX) use in Polish patients with Gaucher disease, presenting neuronopathic type (GD type III, GD3) resulting from homozygous c.1448T>C mutation (p...
    Medical condition: Gaucher disease
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: PL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2021-005267-48 Sponsor Protocol Number: 4202-HEM-201 Start Date*: 2022-10-17
    Sponsor Name:FORMA THERAPEUTICS, INC.
    Full Title: A Phase 2 Open-Label Study to Evaluate Safety and Clinical Activity of FT-4202 in Patients with Thalassemia or Sickle Cell Disease
    Medical condition: Sickle cell disease (SCD) or thalassemia
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004850 10040646 Sickle cell disorders LLT
    20.1 100000004850 10040649 Sickle cell thalassaemia LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2020-005736-30 Sponsor Protocol Number: CDAII-LUSPA_FA02-2020 Start Date*: 2023-07-17
    Sponsor Name:FOndazione per la Ricerca sulle ANemie ed EMoglobinopatie in ItaliA - For Anemia
    Full Title: A phase II, multicenter, open label study to evaluate the efficacy and safety of Luspatercept (ACE-536) in adult patients with Congenital Dyserythropoietic Anemia type Il (CDA ll).
    Medical condition: Congenital Dyserythropoietic Anemia type II (CDAII)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10010331 - Congenital, familial and genetic disorders 10081457 Congenital dyserythropoietic anaemia PT
    21.1 10010331 - Congenital, familial and genetic disorders 10081457 Congenital dyserythropoietic anaemia PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2018-001771-21 Sponsor Protocol Number: XL184–311 Start Date*: Information not available in EudraCT
    Sponsor Name:Exelixis, Inc.
    Full Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Cabozantinib (XL184) in Subjects with Radioiodine-Refractory Differentiated Thyroid Cancer Who Have Progressed after Prior VEGFR-...
    Medical condition: Radioiodine-Refractory Differentiated Thyroid Cancer which has progressed after prior VEGFR-targeted therapy
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10066474 Thyroid cancer PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) BE (Completed) FR (Completed) AT (Prematurely Ended) HU (Completed) ES (Ongoing) PL (Trial now transitioned) CZ (Completed) HR (Completed) GB (GB - no longer in EU/EEA) IT (Completed) RO (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-001674-34 Sponsor Protocol Number: AG348-C-020 Start Date*: 2022-02-17
    Sponsor Name:AGIOS PHARMACEUTICALS, INC.
    Full Title: A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease.
    Medical condition: Sickle Cell Disease
    Disease: Version SOC Term Classification Code Term Level
    21.0 10010331 - Congenital, familial and genetic disorders 10040644 Sickle cell disease PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned) FR (Trial now transitioned) BE (Trial now transitioned) DE (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
    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: 2010-021336-33 Sponsor Protocol Number: A8081014 Start Date*: 2010-12-30
    Sponsor Name:Pfizer Inc 235 East 42nd Street, New York, NY10017 US
    Full Title: PHASE 3, RANDOMIZED, OPEN-LABEL STUDY OF THE EFFICACY AND SAFETY OF CRIZOTINIB VERSUS PEMETREXED/CISPLATIN OR PEMETREXED/CARBOPLATIN IN PREVIOUSLY UNTREATED PATIENTS WITH NON-SQUAMOUS CARCINOMA OF ...
    Medical condition: non-small cell lung cancer (NSCLC)
    Disease: Version SOC Term Classification Code Term Level
    19.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10029519 Non-small cell lung cancer stage III PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) ES (Completed) FI (Completed) IE (Completed) AT (Completed) IT (Completed) GB (Completed) BE (Completed) NL (Completed) DK (Prematurely Ended) PT (Completed) NO (Completed)
    Trial results: View results
    EudraCT Number: 2007-005949-38 Sponsor Protocol Number: M10-265 Start Date*: 2008-09-29
    Sponsor Name:Abbott GmbH & Co. KG
    Full Title: A Phase 2 Randomized, Placebo-Controlled, Double-Blind Study of ABT-869 in Combination With Paclitaxel Versus Paclitaxel Alone as First-line Treatment in Subjects With Locally Recurrent or Metastat...
    Medical condition: Metastatic breast cancer
    Disease: Version SOC Term Classification Code Term Level
    9.1 10027475 Metastatic breast cancer LLT
    Population Age: Adults, Elderly Gender: Female
    Trial protocol: GB (Prematurely Ended) CZ (Completed) BE (Completed) NL (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2019-003765-18 Sponsor Protocol Number: AIFA-TRS-2018-00001238 Start Date*: 2020-03-20
    Sponsor Name:DIPARTIMENTO AD ATTIVITà INTEGRATA CHIRURGICO, MEDICO, ODONTOIATRICO E DI SCIENZE MORFOLOGICHE
    Full Title: Gender-related response to Tyrosine Kinase-Inhibitor drugs in hepatocellular carcinoma
    Medical condition: Patients with advanced hepatocellular carcinoma only eligible for systemic treatment
    Disease: Version SOC Term Classification Code Term Level
    20.0 10019805 - Hepatobiliary disorders 10019805 Hepatobiliary disorders SOC
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2010-023783-41 Sponsor Protocol Number: E7080-G000-303 Start Date*: 2011-07-05
    Sponsor Name:Eisai Limited
    Full Title: A Multicenter, Randomized, Double- Blind, Placebo-Controlled, Phase 3 Trial of Lenvatinib (E7080) in 131I- Refractory Differentiated Thyroid Cancer
    Medical condition: 131I-refractory differentiated thyroid cancer (DTC)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10055107 Thyroid cancer metastatic PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) CZ (Completed) PT (Completed) GB (Completed) DE (Completed) ES (Completed) PL (Completed) DK (Completed) AT (Completed) IT (Completed) SE (Completed)
    Trial results: View results
    EudraCT Number: 2016-003370-40 Sponsor Protocol Number: GBT440-031 Start Date*: 2017-04-05
    Sponsor Name:Global Blood Therapeutics, Inc.
    Full Title: A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of GBT440 Administered Orally to Patients With Sickle Cell Disease
    Medical condition: Sickle Cell Disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10040644 Sickle cell disease LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) NL (Completed) FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2006-003786-13 Sponsor Protocol Number: 20050251 Start Date*: 2007-03-30
    Sponsor Name:Amgen Inc
    Full Title: A Phase 3 Randomized Trial of Chemotherapy With or Without Panitumumab in Patients with Metastatic and/or Recurrent Squamous Cell Carcinoma of the Head and Neck
    Medical condition: Metastatic and/or Recurrent Squamous Cell Carcinoma of the Head and Neck
    Disease: Version SOC Term Classification Code Term Level
    9.1 10060121 Squamous cell carcinoma of head and neck LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IE (Completed) GB (Completed) DE (Completed) HU (Completed) BE (Completed) FR (Completed) SE (Completed) PT (Completed) IT (Completed) BG (Completed)
    Trial results: View results
    EudraCT Number: 2014-001549-26 Sponsor Protocol Number: TRASTS Start Date*: 2014-11-03
    Sponsor Name:GEIS
    Full Title: Phase I-II prospective trial, multicenter, open label, exploring the combination of Trabectedin plus Radiotherapy in Soft Tissue Sarcoma patients.
    Medical condition: Soft Tissue Sarcoma
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: ES (Ongoing) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2015-004158-17 Sponsor Protocol Number: GELTAMO-IMCL-2015 Start Date*: 2016-02-15
    Sponsor Name:GELTAMO (Grupo Cooperativo Español de Llinfoma/Trasplante Autólogo de Médula Ósea)
    Full Title: Multicentric phase II trial to evaluate the efficacy and safety of Ibrutinib in combination with rituximab in patients with indolent clinical forms of Mantle Cell Lymphoma.
    Medical condition: Mantle Cell Lyphoma
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004851 10026798 Mantle cell lymphomas HLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2017-000903-26 Sponsor Protocol Number: GBT440-032 Start Date*: 2022-02-02
    Sponsor Name:Global Blood Therapeutics, Inc.
    Full Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Voxelotor (GBT440) in Pediatric Participants with Sickle Cell Disease (HOPE Kids 2)
    Medical condition: Sickle Cell Disease
    Disease: Version SOC Term Classification Code Term Level
    21.0 10010331 - Congenital, familial and genetic disorders 10040644 Sickle cell disease PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed) GB (GB - no longer in EU/EEA) IT (Completed)
    Trial results: View results
    EudraCT Number: 2021-004931-10 Sponsor Protocol Number: R3918-PNH-2050 Start Date*: 2022-12-27
    Sponsor Name:REGENERON PHARMACEUTICALS, INC.
    Full Title: An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria
    Medical condition: Paroxysmal nocturnal hemoglobinuria (PNH)
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004857 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned) PL (Trial now transitioned) GR (Trial now transitioned) HU (Trial now transitioned) ES (Ongoing) RO (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2006-002766-20 Sponsor Protocol Number: CAUY922A2101 Start Date*: 2009-02-25
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A phase I dose escalation, multi-center, open-label study of AUY922 administered IV on a once-weekly schedule in adult patients with advanced solid malignancies including phase II expansion arms in...
    Medical condition: Dose-expansion study to assess safety, tolerability & efficacy: -One arm (MTD expansion) will enroll advanced cancer patients (other than breast carcinoma). -Two arms (phase II) to assess response ...
    Disease:
    Population Age: Adults, Elderly Gender: Female
    Trial protocol: GB (Completed) DE (Prematurely Ended) NL (Completed)
    Trial results: View results
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