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Clinical trials for Miller syndrome

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 ).  
     
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    33 result(s) found for: Miller syndrome. Displaying page 1 of 2.
    1  2  Next»
    EudraCT Number: 2013-004328-12 Sponsor Protocol Number: Europe Start Date*: 2015-01-23
    Sponsor Name:GMIHO Gesellschaft für Medizinische Innovation - Hämatologie und Onkologie mbH
    Full Title: Prospective validation of a predictive model of response to romiplostim in patients with IPSS low or intermediate-1 risk myelodysplastic syndrome (MDS) and thrombocytopenia - the EUROPE-trial
    Medical condition: Patients with IPSS low or intermediate-1 risk myelodysplastic syndrome (MDS) and thrombocytopenia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004864 10068361 MDS LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) FR (Ongoing) CZ (Completed)
    Trial results: View results
    EudraCT Number: 2010-022235-10 Sponsor Protocol Number: ITCC-015/EWOG-MDS-Azacytidine-2010 Start Date*: 2012-03-07
    Sponsor Name:Erasmus MC
    Full Title: A Phase I/II study of Azacitidine (Vidaza®) in pediatric patients with relapsed high-grade pediatric MDS or JMML
    Medical condition: Relapsed advanced Myelodysplastic Syndromes (MDS) and Juvenile Myelomonocytic Leukemia (JMML)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004864 10054439 Juvenile chronic myelomonocytic leukemia LLT
    20.0 100000004864 10068361 MDS LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Completed) CZ (Completed) IT (Prematurely Ended) AT (Prematurely Ended) DE (Prematurely Ended) DK (Prematurely Ended) IE (Prematurely Ended) ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2014-002675-29 Sponsor Protocol Number: CC-486-MDS-006 Start Date*: 2015-06-19
    Sponsor Name:Celgene Corporation
    Full Title: A Phase 2, International, Multicenter, Single-arm, Open-label Study to Evaluate the Efficacy and Safety of CC-486 (oral azacitidine) in Subjects With Myelodysplastic Syndromes Who Fail to Achieve a...
    Medical condition: Myelodysplastic syndrome (MDS)
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004864 10068361 MDS LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Ongoing) GB (GB - no longer in EU/EEA) BE (Completed) ES (Ongoing) DE (Completed) PL (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2019-004839-23 Sponsor Protocol Number: CA-4948-102 Start Date*: 2023-02-09
    Sponsor Name:Curis, Inc
    Full Title: A Phase 1/2a, Open-Label Dose Escalation and Cohort Expansion Study of Orally Administered CA-4948(IRAK4i) as a Monotherapy in patients with Acute Myelogenous Leukemia or Myelodysplastic Syndrome a...
    Medical condition: Acute Myelogenous Leukemia (AML) or high risk Myelodysplastic Syndrome (MDS).
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004864 10001941 AML LLT
    20.0 100000004864 10068361 MDS LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing) PL (Completed) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2014-000477-39 Sponsor Protocol Number: AMLSG20-13 Start Date*: 2016-01-29
    Sponsor Name:University Hospital Ulm
    Full Title: Dose Finding safety run in Phase followed by a randomized Phase II Trial of Intensive Chemotherapy With or Without Volasertib (BI 6727) Administered Prior or After Chemotherapy in Patients With New...
    Medical condition: patients with newly diagnosed high-risk myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML)
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004864 10068361 MDS LLT
    18.1 100000004864 10001941 AML LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2011-004559-38 Sponsor Protocol Number: RG_12-101 Start Date*: 2012-08-08
    Sponsor Name:University of Birmingham
    Full Title: A phase 2 study of the efficacy and safety of Deferasirox administered at early iron loading in patients with transfusion-dependent Myelodysplastic Syndromes.
    Medical condition: Iron overload in patients being treated for Myelodysplastic syndroms (MDS) by regular blood transfusions
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004864 10068361 MDS LLT
    14.1 100000004848 10040310 Serum iron increased LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2009-011513-24 Sponsor Protocol Number: CC-5013-MDS-005 Start Date*: 2009-12-14
    Sponsor Name:Celgene Corporation
    Full Title: A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO COMPARE THE EFFICACY AND SAFETY OF LENALIDOMIDE (REVLIMID®) VERSUS PLACEBO IN SUBJECTS WITH TRANSFUSION...
    Medical condition: TRANSFUSION-DEPENDENT ANEMIA DUE TO IPSS LOW OR INTERMEDIATE-1 RISK MYELODYSPLASTIC SYNDROMES WITHOUT DELETION 5Q [31] AND UNRESPONSIVE OR REFRACTORY TO ERYTHROPOIESIS-STIMULATING AGENT
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004864 10068361 MDS LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) ES (Completed) DE (Completed) FR (Completed) CZ (Completed) AT (Completed) IT (Completed) PT (Completed) GB (Completed) PL (Completed)
    Trial results: View results
    EudraCT Number: 2020-004899-18 Sponsor Protocol Number: LUSPLUS Start Date*: 2021-09-17
    Sponsor Name:GWT-TUD GmbH
    Full Title: A phase IIIb, open-label, single arm study to evaluate the efficacy and safety of luspatercept in patients with lower-risk MDS and ring-sideroblastic phenotype (MDS-RS)
    Medical condition: Lower-risk myelodysplastic syndrome with ring-sideroblastic phenotype (MDS-RS)
    Disease: Version SOC Term Classification Code Term Level
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10028534 Myelodysplastic syndrome NOS LLT
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10068361 MDS LLT
    20.0 10005329 - Blood and lymphatic system disorders 10050910 Bone marrow disorder NOS LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) AT (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-005399-12 Sponsor Protocol Number: DKMS-16-01 Start Date*: 2017-08-02
    Sponsor Name:DKMS gemeinnützige GmbH
    Full Title: A randomized controlled trial comparing outcome after hematopoietic cell transplantation from a partially matched unrelated versus haploidentical donor
    Medical condition: Patients with high-risk AML, ALL or MDS which have to undergo allogenic transplantaion.
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004864 10024349 Leukemia myeloid LLT
    20.0 100000004864 10024337 Leukemia lymphatic LLT
    20.0 100000004864 10068361 MDS LLT
    20.1 100000004864 10024330 Leukemia acute LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2018-000356-18 Sponsor Protocol Number: NL64877.041.18 Start Date*: 2018-11-19
    Sponsor Name:University Medical Center utrecht
    Full Title: Individualized dosing of fludarabine during innate allo SCT: A randomized phase II study (TARGET Study)
    Medical condition: AML, MDS, ALL, CML, CLL, NHL, HL, or a myeloproliferative disease (MPD)
    Disease: Version SOC Term Classification Code Term Level
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10000835 Acute leukemia LLT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10008948 Chronic leukemia LLT
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10068361 MDS LLT
    20.0 10005329 - Blood and lymphatic system disorders 10025319 Lymphomas Hodgkin's disease HLGT
    20.0 10005329 - Blood and lymphatic system disorders 10025321 Lymphomas non-Hodgkin's T-cell HLGT
    20.0 10005329 - Blood and lymphatic system disorders 10025320 Lymphomas non-Hodgkin's B-cell HLGT
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10077465 Myeloproliferative neoplasm PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2016-000502-11 Sponsor Protocol Number: EKOS-12 Start Date*: 2016-07-21
    Sponsor Name:EKOS Corporation
    Full Title: Study of the Optimum Duration of Acoustic Pulse Thrombolysis (APT) Procedure in the Treatment of Acute Submassive Pulmonary Embolism (OPTALYSE PE).
    Medical condition: Submassive pulmonary embolism
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2018-002915-93 Sponsor Protocol Number: ACE-536-LTFU-001 Start Date*: 2019-06-26
    Sponsor Name:Celgene Corporation
    Full Title: A phase 3B, open-label, single-arm, rollover study to evaluate long-term safety in subjects who have participated in other Luspatercept (ACE-536) clinical trials.
    Medical condition: Prior participation on a clinical trial of luspatercept (ACE-536) in protocols eligible for participation in this study ACE-536-LTFU-001 with the following medical conditions: - myelodysplastic syn...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10074356 Non-transfusion dependent thalassemia LLT
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10068361 MDS LLT
    20.1 100000004850 10054658 Thalassemia LLT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10074689 Post polycythemia vera myelofibrosis LLT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10074690 Post essential thrombocythemia myelofibrosis LLT
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10074691 Post polycythaemia vera myelofibrosis LLT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10074692 Post essential thrombocythaemia myelofibrosis LLT
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10028537 Myelofibrosis PT
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10077161 Primary myelofibrosis PT
    20.0 100000004851 10002272 Anemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) SE (Trial now transitioned) NL (Trial now transitioned) GB (GB - no longer in EU/EEA) FR (Trial now transitioned) GR (Trial now transitioned) ES (Ongoing) BE (Completed) BG (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-006161-11 Sponsor Protocol Number: RC31/20/0421 Start Date*: 2021-06-23
    Sponsor Name: Toulouse University Hospital
    Full Title: LONG -TERM INTERVENTIONAL FOLLOW-UP STUDY UP TO 4 YEARS OF AGE OF CHILDREN WITH PRADER-WILLI SYNDROME INCLUDED IN THE OTBB3 CLINICAL TRIAL AND COMPARISON WITH AN UNTREATED COHORT OF CHILDREN WITH P...
    Medical condition: Prader-willi syndrome
    Disease:
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2017-003423-30 Sponsor Protocol Number: 2017-RCT-OX Start Date*: 2017-12-12
    Sponsor Name:Dutch Growth Research Foundation
    Full Title: Randomized, double-blind, placebo-controlled oxytocin and dose-response trial in children with Prader-Willi syndrome. Effects on social behaviour.
    Medical condition: Prader-Willi syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10036476 Prader-Willi syndrome PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-003820-22 Sponsor Protocol Number: 2016-Longterm-OX Start Date*: 2016-12-22
    Sponsor Name:Dutch Growth Research Foundation
    Full Title: Intranasal administration of oxytocin in children with Prader-Willi Syndrome. A randomized, open-label, cross-over trial of different treatment regimens of oxytocin administration. Effects on eatin...
    Medical condition: Prader-Willi syndrome
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10036476 Prader-Willi syndrome PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Not Authorised)
    Trial results: (No results available)
    EudraCT Number: 2020-004011-28 Sponsor Protocol Number: A4250-012 Start Date*: 2021-01-26
    Sponsor Name:Albireo AB
    Full Title: A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT)
    Medical condition: Alagille Syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10053870 Alagille syndrome PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed) PL (Completed) NL (Completed) BE (Completed) DE (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2021-000996-36 Sponsor Protocol Number: A4250-015 Start Date*: 2021-10-14
    Sponsor Name:Albireo AB
    Full Title: An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT)
    Medical condition: Alagille Syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10053870 Alagille syndrome PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) BE (Trial now transitioned) PL (Trial now transitioned) NL (Trial now transitioned) DE (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2013-005373-43 Sponsor Protocol Number: LUM001-304 Start Date*: 2014-08-05
    Sponsor Name:Lumena Pharmaceuticals Inc
    Full Title: Long-Term, Open-Label Study with a Double-Blind, Placebo Controlled, Randomized Drug Withdrawal Period of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients wit...
    Medical condition: Alagille Syndrome
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10053870 Alagille syndrome PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Completed) PL (Completed) BE (Completed) GB (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2012-005346-38 Sponsor Protocol Number: LUM001-302 Start Date*: 2013-06-11
    Sponsor Name:Lumena Pharmaceuticals, Inc.
    Full Title: A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF LUM001, AN APICAL SODIUM-DEPENDENT BILE ACID TRANSPORTER INHIBITOR (ASBTi), IN THE TREATMENT OF CHOLESTAT...
    Medical condition: Alagille syndrome (ALGS). This is an example of cholestatic liver disease in children. In patients with Alagille syndrome, impairment of the egress of bile acids from the liver leads to cholestasi...
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10053870 Alagille syndrome PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2015-000906-20 Sponsor Protocol Number: LUM001-601 Start Date*: 2015-05-13
    Sponsor Name:Shire Human Genetic Therapies Inc
    Full Title: An Open-label, Multicenter Extension Study to Evaluate the Long-term Safety of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients with Alagille Syndrome (ALGS) ...
    Medical condition: Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC)
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    17.1 10010331 - Congenital, familial and genetic disorders 10053870 Alagille syndrome PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    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
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