title: Type 3 Von Willebrand International Registries Inhibitor Prospective Study

summary: International Registries and Prospective Study on Type 3 Von Willebrand's Disease (VWD3), aimed to assess number, types and risk factors for bleeding and the efficacy and safety of plasma-derived and/or recombinant Von Willebrand Factor (VWF) concentrates used to treat VWD patients.

time_perspective: Other

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ReferenceCitation: Federici AB, Mannucci PM, Castaman G, Baronciani L, Bucciarelli P, Canciani MT, Pecci A, Lenting PJ, De Groot PG. Clinical and molecular predictors of thrombocytopenia and risk of bleeding in patients with von Willebrand disease type 2B: a cohort study of 67 patients. Blood. 2009 Jan 15;113(3):526-34. doi: 10.1182/blood-2008-04-152280. Epub 2008 Sep 19.

ReferenceCitation: Gritti G, Cortelezzi A, Bucciarelli P, Rezzonico F, Lonati S, La Marca S, Silvestris I, Federici AB. Circulating and progenitor endothelial cells are abnormal in patients with different types of von Willebrand disease and correlate with markers of angiogenesis. Am J Hematol. 2011 Aug;86(8):650-6. doi: 10.1002/ajh.22070. Epub 2011 May 31.

ReferenceCitation: Abshire TC, Federici AB, Alvárez MT, Bowen J, Carcao MD, Cox Gill J, Key NS, Kouides PA, Kurnik K, Lail AE, Leebeek FW, Makris M, Mannucci PM, Winikoff R, Berntorp E; VWD PN. Prophylaxis in severe forms of von Willebrand's disease: results from the von Willebrand Disease Prophylaxis Network (VWD PN). Haemophilia. 2013 Jan;19(1):76-81. doi: 10.1111/j.1365-2516.2012.02916.x. Epub 2012 Jul 23.

ReferenceCitation: Castaman G, Goodeve A, Eikenboom J; European Group on von Willebrand Disease. Principles of care for the diagnosis and treatment of von Willebrand disease. Haematologica. 2013 May;98(5):667-74. doi: 10.3324/haematol.2012.077263. Review.

ReferenceCitation: Federici AB, Bucciarelli P, Castaman G, Mazzucconi MG, Morfini M, Rocino A, Schiavoni M, Peyvandi F, Rodeghiero F, Mannucci PM. The bleeding score predicts clinical outcomes and replacement therapy in adults with von Willebrand disease. Blood. 2014 Jun 26;123(26):4037-44. doi: 10.1182/blood-2014-02-557264. Epub 2014 Apr 30.

ReferenceCitation: Abdul-Kadir R, McLintock C, Ducloy AS, El-Refaey H, England A, Federici AB, Grotegut CA, Halimeh S, Herman JH, Hofer S, James AH, Kouides PA, Paidas MJ, Peyvandi F, Winikoff R. Evaluation and management of postpartum hemorrhage: consensus from an international expert panel. Transfusion. 2014 Jul;54(7):1756-68. doi: 10.1111/trf.12550. Epub 2014 Mar 12. Erratum in: Transfusion. 2015 Mar;55(3):691.

ReferenceCitation: Makris M, Federici AB, Mannucci PM, Bolton-Maggs PH, Yee TT, Abshire T, Berntorp E. The natural history of occult or angiodysplastic gastrointestinal bleeding in von Willebrand disease. Haemophilia. 2015 May;21(3):338-42. doi: 10.1111/hae.12571. Epub 2014 Nov 7.

ReferenceCitation: Federici AB. Clinical and laboratory diagnosis of VWD. Hematology Am Soc Hematol Educ Program. 2014 Dec 5;2014(1):524-30. doi: 10.1182/asheducation-2014.1.524. Epub 2014 Nov 18. Review.

ReferenceCitation: Holm E, Abshire TC, Bowen J, Álvarez MT, Bolton-Maggs P, Carcao M, Federici AB, Gill JC, Halimeh S, Kempton C, Key NS, Kouides P, Lail A, Landorph A, Leebeek F, Makris M, Mannucci P, Mauser-Bunschoten EP, Nugent D, Valentino LA, Winikoff R, Berntorp E. Changes in bleeding patterns in von Willebrand disease after institution of long-term replacement therapy: results from the von Willebrand Disease Prophylaxis Network. Blood Coagul Fibrinolysis. 2015 Jun;26(4):383-8. doi: 10.1097/MBC.0000000000000257.

ReferenceCitation: Randi AM, Laffan MA. Von Willebrand factor and angiogenesis: basic and applied issues. J Thromb Haemost. 2017 Jan;15(1):13-20. doi: 10.1111/jth.13551. Review.

ReferenceCitation: Federici AB. Current and emerging approaches for assessing von Willebrand disease in 2016. Int J Lab Hematol. 2016 May;38 Suppl 1:41-9. doi: 10.1111/ijlh.12540. Review.

ReferenceCitation: De Jong A, Eikenboom J. Developments in the diagnostic procedures for von Willebrand disease. J Thromb Haemost. 2016 Mar;14(3):449-60. doi: 10.1111/jth.13243. Epub 2016 Feb 12. Review.

ReferenceCitation: Abshire T, Cox-Gill J, Kempton CL, Leebeek FW, Carcao M, Kouides P, Donfield S, Berntorp E. Prophylaxis escalation in severe von Willebrand disease: a prospective study from the von Willebrand Disease Prophylaxis Network. J Thromb Haemost. 2015 Sep;13(9):1585-9. doi: 10.1111/jth.12995. Epub 2015 Jul 14. Review.

ReferenceCitation: Engelen ET, van Galen KP, Schutgens RE. Thalidomide for treatment of gastrointestinal bleedings due to angiodysplasia: a case report in acquired von Willebrand syndrome and review of the literature. Haemophilia. 2015 Jul;21(4):419-29. doi: 10.1111/hae.12701. Epub 2015 Apr 30. Review.

ReferenceCitation: Franchini M, Mannucci PM. Gastrointestinal angiodysplasia and bleeding in von Willebrand disease. Thromb Haemost. 2014 Sep 2;112(3):427-31. doi: 10.1160/TH13-11-0952. Epub 2014 Jun 5. Review.

ReferenceCitation: Randi AM, Laffan MA, Starke RD. Von Willebrand factor, angiodysplasia and angiogenesis. Mediterr J Hematol Infect Dis. 2013 Sep 2;5(1):e2013060. doi: 10.4084/MJHID.2013.060. Review.

ReferenceCitation: Thachil J, Hay CR, Campbell S. Tamoxifen for recurrent bleeds due to angiodysplasia in von Willebrand's disease. Haemophilia. 2013 Sep;19(5):e313-5. doi: 10.1111/hae.12196. Epub 2013 Jun 4.

ReferenceCitation: Castaman G, Federici AB, Tosetto A, La Marca S, Stufano F, Mannucci PM, Rodeghiero F. Different bleeding risk in type 2A and 2M von Willebrand disease: a 2-year prospective study in 107 patients. J Thromb Haemost. 2012 Apr;10(4):632-8. doi: 10.1111/j.1538-7836.2012.04661.x.

ReferenceCitation: Starke RD, Ferraro F, Paschalaki KE, Dryden NH, McKinnon TA, Sutton RE, Payne EM, Haskard DO, Hughes AD, Cutler DF, Laffan MA, Randi AM. Endothelial von Willebrand factor regulates angiogenesis. Blood. 2011 Jan 20;117(3):1071-80. doi: 10.1182/blood-2010-01-264507. Epub 2010 Nov 3.

ReferenceCitation: Veyradier A, Balian A, Wolf M, Giraud V, Montembault S, Obert B, Dagher I, Chaput JC, Meyer D, Naveau S. Abnormal von Willebrand factor in bleeding angiodysplasias of the digestive tract. Gastroenterology. 2001 Feb;120(2):346-53.

ReferenceCitation: Bowers M, McNulty O, Mayne E. Octreotide in the treatment of gastrointestinal bleeding caused by angiodysplasia in two patients with von Willebrand's disease. Br J Haematol. 2000 Mar;108(3):524-7.

ReferenceCitation: Van Belle E, Rauch A, Vincent F, Robin E, Kibler M, Labreuche J, Jeanpierre E, Levade M, Hurt C, Rousse N, Dally JB, Debry N, Dallongeville J, Vincentelli A, Delhaye C, Auffray JL, Juthier F, Schurtz G, Lemesle G, Caspar T, Morel O, Dumonteil N, Duhamel A, Paris C, Dupont-Prado A, Legendre P, Mouquet F, Marchant B, Hermoire S, Corseaux D, Moussa K, Manchuelle A, Bauchart JJ, Loobuyck V, Caron C, Zawadzki C, Leroy F, Bodart JC, Staels B, Goudemand J, Lenting PJ, Susen S. Von Willebrand Factor Multimers during Transcatheter Aortic-Valve Replacement. N Engl J Med. 2016 Jul 28;375(4):335-44. doi: 10.1056/NEJMoa1505643.

title: Von Willebrand Factor Multimers during Transcatheter Aortic-Valve Replacement

ReferenceCitation: Susen S, Rauch A, Van Belle E, Vincentelli A, Lenting PJ. Circulatory support devices: fundamental aspects and clinical management of bleeding and thrombosis. J Thromb Haemost. 2015 Oct;13(10):1757-67. doi: 10.1111/jth.13120. Epub 2015 Sep 25. Review.

ReferenceCitation: Blatchford O, Murray WR, Blatchford M. A risk score to predict need for treatment for upper-gastrointestinal haemorrhage. Lancet. 2000 Oct 14;356(9238):1318-21.

ReferenceCitation: Gerson LB, Fidler JL, Cave DR, Leighton JA. ACG Clinical Guideline: Diagnosis and Management of Small Bowel Bleeding. Am J Gastroenterol. 2015 Sep;110(9):1265-87; quiz 1288. doi: 10.1038/ajg.2015.246. Epub 2015 Aug 25.

ReferenceCitation: Strate LL, Gralnek IM. ACG Clinical Guideline: Management of Patients With Acute Lower Gastrointestinal Bleeding. Am J Gastroenterol. 2016 Apr;111(4):459-74. doi: 10.1038/ajg.2016.41. Epub 2016 Mar 1. Erratum in: Am J Gastroenterol. 2016 May;111(5):755.

ReferenceCitation: Jackson CS, Gerson LB. Management of gastrointestinal angiodysplastic lesions (GIADs): a systematic review and meta-analysis. Am J Gastroenterol. 2014 Apr;109(4):474-83; quiz 484. doi: 10.1038/ajg.2014.19. Epub 2014 Mar 18. Review.

ReferenceCitation: Martin-Grace J, Tamagno G. Somatostatin analogs in the medical management of occult bleeding of the lower digestive tract. Gastroenterol Res Pract. 2015;2015:702921. doi: 10.1155/2015/702921. Epub 2015 Mar 9. Review.

ReferenceCitation: Holleran G, Hall B, Breslin N, McNamara D. Long-acting somatostatin analogues provide significant beneficial effect in patients with refractory small bowel angiodysplasia: Results from a proof of concept open label mono-centre trial. United European Gastroenterol J. 2016 Feb;4(1):70-6. doi: 10.1177/2050640614559121. Epub 2015 Nov 5.

location_country:
Italy
Sweden
Netherlands
Finland
France
Hungary
Spain
Germany
United Kingdom
Iran, Islamic Republic of

location_facility: Department of Haematology, Haemostasis, Oncology and Stem Cell Transplantation - Hannover Medical School - Haemophilia Care Centre

location_facility: Hemophilia- Thalassaemia Center of Mashhad (Sarvar Clinic) - Mashad University of Medical Science

location_facility: Complejo Hospitalario Universitario de A Coruña - Servicio de Hematología y Hemoterapia

location_facility: Central Manchester University Hospital NHS Foundation Trust - Manchester Royal Infirmary - Manchester Royal Eye Hospital

location_facility: Iranian Hemophilia Comprehensive Treatment Centre - Iranian Hemophilia Society

location_facility: Azienda Ospedaliera Universitaria Careggi - Agenzia per l'Emofilia - Centro di Riferimento Coagulopatie Congenite

location_facility: Seid-ol-Shohada Hospital - Hemophilia Center - Esfahan University of Medical Science

location_facility: Centre Régional de Traitement de l'Hémophilie - Laboratoire d'Hématologie

location_facility: University Clinic Bonn - Institute of Experimental Haematology & Transfusion Medicine

location_facility: Dipartimento di Terapie Cellulari ed Ematologia - Centro Malattie Emorragiche e Trombotiche - Ospedale San Bortolo

location_facility: Leiden University Medical Center - Department of Hematology - Hemostasis and Thrombosis Center

location_facility: Dipartimento di Biotecnologie Cellulari ed Ematologia - Università "Sapienza" di Roma - Policlinico Umberto I

location_facility: University Children's Hospital - Department of Pediatric Hematology and Oncology

location_facility: St. Istvan & St. Laszlo Hospital of Budapest - Hematology and Stem Cell Transplantation

location_facility: Institut d'Hématologie - Hôpital Cardiologique - University of Lille - Haematology Department

location_facility: Centro Emofilia e Trombosi - Fondazione Angelo Bianchi Bonomi - IRCCS Ospedale Ca' Granda - Dip. di Medicina Interna - Università degli Studi di Milano

location_facility: Erasmus Medical Center - Department of Hematology

location_facility: Lund University - Centre for Thrombosis and Haemostasis - Skane University Hospital

location_facility: Azienda Ospedaliera Policlinico Consorziale di Bari - Unità Operativa Semplice di Emostasi e Trombosi

location_facility: Nemazee Hospital Hemophilia Center - Shiraz University of Medical Science

location_facility: Hospital Universitari General Vall d'Hebron - Unidad de Hemofilia

location_facility: Mofid Comprehensive Care Centre for Children with Hemophilia - Shahid Beheshti University of Medical Science

location_facility: Helsinki University Central Hospital, Department Internal Medicine, Coagulation Disorders, at Haematology and Laboratory Services

location_facility: Ahvaz Jundishpur University of Medical Sciences - Research Center for Thalassemia & Hemoglobinopathy - Division of Hematology & Oncology

location_facility: Thrombosis Hemostasis Research Center - Vali-Asr Hospital - Emam Khmeini Complex Hospital - Tehran University of Medical Science


type: EudraCT Number

title: Open Label Study to Evaluate the Pharmacokinetics of Fidaxomicin in Inflammatory Bowel Disease (IBD) Subjects With Clostridium Difficile Infection (CDI)

acronym: PROFILE

summary:
The purpose of this study is to investigate the plasma pharmacokinetics (PK) of fidaxomicin (FDX) and primary metabolite OP-1118 in Subjects with Inflammatory Bowel Disease (IBD) and C. difficile Infection (CDI).

This study will also compare CDI clinical response to the microbiological response in terms of magnitude of reduction of C. difficile total viable count and spore count during treatment with FDX and if achieved; the time to microbial eradication; determine time to negative CDI toxin assay in stool specimens during treatment with FDX; assess the stool concentrations of FDX and metabolite OP-1118 throughout therapy; assess the length of hospital stay, readmissions and resource utilization for IBD patients receiving FDX; record the incidence and severity of Adverse Events (AEs) and document the impact of treatment on Quality of Life as measured by the changes in Short Inflammatory Bowel Disease Questionnaire (IBDQ) score.

study_start_date_type: Actual

status: Completed

study_completion_date_type: Actual

study_type: Interventional

primary_purpose: Treatment

enrollment_type: Actual

ReferenceType: derived

ReferenceCitation: Högenauer C, Mahida Y, Stallmach A, Marteau P, Rydzewska G, Ivashkin V, Gargalianos-Kakolyris P, Michon I, Adomakoh N, Georgopali A, Tretter R, Karas A, Reinisch W. Pharmacokinetics and safety of fidaxomicin in patients with inflammatory bowel disease and Clostridium difficile infection: an open-label Phase IIIb/IV study (PROFILE). J Antimicrob Chemother. 2018 Dec 1;73(12):3430-3441. doi: 10.1093/jac/dky368.

name: Medical University of Graz, Graz, Austria

family: Högenauer

full_name: Christoph Högenauer

given: Christoph

sequence: first

name: NIHR Nottingham Biomedical Research Centre, Nottingham University Hospitals NHS Trust and the University of Nottingham, Nottingham, UK

family: Mahida

full_name: Yashwant Mahida

given: Yashwant

sequence: additional

family: Stallmach

full_name: Andreas Stallmach

given: Andreas

name: Sorbonne Université & APHP, Hôpital Saint Antoine, Paris, France

family: Marteau

full_name: Philippe Marteau

given: Philippe

name: Central Clinical Hospital of the Ministry of the Interior, Warsaw and Jan Kochanowski University, Kielce, Poland

family: Rydzewska

full_name: Grazyna Rydzewska

given: Grazyna

name: I. M. Sechenov University, Moscow, Russian Federation

family: Ivashkin

full_name: Vladimir Ivashkin

given: Vladimir

name: General Hospital of Athens ‘G. Gennimatas’, Athens, Greece

family: Gargalianos-Kakolyris

full_name: Panagiotis Gargalianos-Kakolyris

given: Panagiotis

name: Astellas Pharma Europe B.V., Leiden, The Netherlands

family: Michon

full_name: Ingrid Michon

given: Ingrid

family: Adomakoh

full_name: Nicholas Adomakoh

given: Nicholas

family: Georgopali

full_name: Areti Georgopali

given: Areti

family: Tretter

full_name: Reiner Tretter

given: Reiner

family: Karas

full_name: Andreas Karas

name: Medical University of Vienna, Vienna, Austria

family: Reinisch

full_name: Walter Reinisch

given: Walter

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Journal of Antimicrobial Chemotherapy

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via crossref license)

host_type: publisher

license: cc-by-nc

license_normalized: cc-by-nc

version: publishedVersion

evidence: oa repository (via OAI-PMH doi match)

host_type: repository

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Inria

repository_normalized: HAL

repository_institution: University of Nottingham - Repository@Nottingham

publisher: Oxford University Press (OUP)

publisher_dissemination: Oxford University Press

publisher_group: Oxford University Press

publisher_normalized: Oxford University Press

ipd_sharing: No

ipd_sharing_description: Access to anonymized individual participant level data will not be provided for this trial as it meets one or more of the exceptions described on www.clinicalstudydatarequest.com under "Sponsor Specific Details for Astellas."

lead_sponsor: Astellas Pharma Europe Ltd.

location_country:
Italy
Austria
Poland
France
Greece
Russian Federation
United Kingdom

location_facility:
Site FR33001
Site AT43001
Site RU70003
Site PL48002
Site RU70001
Site GB44002
Site RU70002
Site PL48003
Site GR30004
Site IT39003
Site IT39001
Site FR33002

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Completed


title: A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C in Order to Characterise the Individual Patient Disease Profile and Historic Signo-symptomatology Progression Pattern

summary:
This is a prospective non-therapeutic observational study in NP-C patients. The aim is to characterize the individual patient disease progression profile through the historical and 6 months prospective evaluation of clinical, imaging, biological(biomarkers) and quality of life data.

Patients will be offered enrollment into a Phase II/III study on arimoclomol at the end of the study.

study_type: Observational

time_perspective: Prospective

ReferenceCitation: Mengel E, Bembi B, Del Toro M, Deodato F, Gautschi M, Grunewald S, Grønborg S, Héron B, Maier EM, Roubertie A, Santra S, Tylki-Szymanska A, Day S, Symonds T, Hudgens S, Patterson MC, Guldberg C, Ingemann L, Petersen NHT, Kirkegaard T, Í Dali C. Clinical disease progression and biomarkers in Niemann-Pick disease type C: a prospective cohort study. Orphanet J Rare Dis. 2020 Nov 23;15(1):328. doi: 10.1186/s13023-020-01616-0. Erratum in: Orphanet J Rare Dis. 2021 Jun 1;16(1):246.

family: Mengel

full_name: Eugen Mengel

given: Eugen

family: Bembi

full_name: Bruno Bembi

given: Bruno

family: del Toro

full_name: Mireia del Toro

given: Mireia

family: Deodato

full_name: Federica Deodato

given: Federica

family: Gautschi

full_name: Matthias Gautschi

given: Matthias

family: Grunewald

full_name: Stephanie Grunewald

given: Stephanie

family: Grønborg

full_name: Sabine Grønborg

given: Sabine

family: Héron

full_name: Bénédicte Héron

given: Bénédicte

family: Maier

full_name: Esther M. Maier

given: Esther M.

family: Roubertie

full_name: Agathe Roubertie

given: Agathe

family: Santra

full_name: Saikat Santra

given: Saikat

family: Tylki-Szymanska

full_name: Anna Tylki-Szymanska

given: Anna

family: Day

full_name: Simon Day

given: Simon

family: Symonds

full_name: Tara Symonds

given: Tara

family: Hudgens

full_name: Stacie Hudgens

given: Stacie

family: Patterson

full_name: Marc C. Patterson

given: Marc C.

family: Guldberg

full_name: Christina Guldberg

given: Christina

family: Ingemann

full_name: Linda Ingemann

given: Linda

family: Petersen

full_name: Nikolaj H. T. Petersen

given: Nikolaj H. T.

family: Kirkegaard

full_name: Thomas Kirkegaard

given: Thomas

family: í Dali

full_name: Christine í Dali

given: Christine

currency_apc_doaj: GBP

journal_name: Orphanet Journal of Rare Diseases

oa_colors: green gold

evidence: oa journal (via doaj)

repository_institution: University of Bern - Bern Open Repository and Information System

evidence: oa repository (via OAI-PMH title and first author match)

repositories:
PubMed Central
Research Square
boris.unibe.ch

title: Clinical disease progression and biomarkers in Niemann–Pick disease type C: a prospective cohort study

location_country:
Italy
Switzerland
France
Poland
Spain
Denmark
Germany
United Kingdom

location_facility: University Hospital Copenhagen (Rigshospitalet)

location_facility: Azienda Ospedaliero-Universitaria "Santa Maria della Misericordia"


ipd_sharing_description:
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

lead_sponsor: Novartis Pharmaceuticals

location_country: France Singapore Spain Germany Canada Taiwan

ipd_sharing: Undecided

source: euctr

title: A Phase Ib/II, Open-label, Multicenter Study of Oral HDM201 in Combination With Oral LEE011 in Adult Patients With Liposarcoma

summary: To determine the MTD/RP2D of the HDM201 and LEE011 combination and evaluate whether the combination is safe and has beneficial effects in patients with liposarcoma.


type: Other Identifier

title: Assessing the Severity of Metabolic-related Liver Injuries in Aging HIV-monoinfected Patients: a European Multicentre Study

acronym: ANRS ECHAM

summary: This study will allow to assess liver related injuries in HIV patients.

primary_purpose: Diagnostic

lead_sponsor: ANRS, Emerging Infectious Diseases

location_country: Germany Belgium France

location_facility: Center for HIV and Hepato-Gastroenterology

intervention_type: Procedure

lead_sponsor_type: academique


ReferenceCitation: Heinhuis KM, Carlino M, Joerger M, Di Nicola M, Meniawy T, Rottey S, Moreno V, Gazzah A, Delord JP, Paz-Ares L, Britschgi C, Schilder RJ, O'Byrne K, Curigliano G, Romano E, Patah P, Wang R, Liu Y, Bajaj G, Siu LL. Safety, Tolerability, and Potential Clinical Activity of a Glucocorticoid-Induced TNF Receptor-Related Protein Agonist Alone or in Combination With Nivolumab for Patients With Advanced Solid Tumors: A Phase 1/2a Dose-Escalation and Cohort-Expansion Clinical Trial. JAMA Oncol. 2020 Jan 1;6(1):100-107. doi: 10.1001/jamaoncol.2019.3848.

name: Division of Pharmacology, The Netherlands Cancer Institute Antoni van Leeuwenhoek, Amsterdam, the Netherlands

family: Heinhuis

full_name: Kimberley M. Heinhuis

given: Kimberley M.

name: Department of Medical Oncology, Crown Princess Mary Cancer Centre Westmead Hospital, Westmead, Australia

family: Carlino

full_name: Matteo Carlino

given: Matteo

name: Department of Internal Medicine, Clinic for Medical Oncology and Hematology, Cantonal Hospital St Gallen, St Gallen, Switzerland

family: Joerger

full_name: Markus Joerger

given: Markus

name: Istituto di Ricovero e Cura a Carattere Scientifico, Istituto Nazionale dei Tumori Milano, Milano, Italy

family: Di Nicola

full_name: Massimo Di Nicola

given: Massimo

name: Linear Clinical Research Ltd, Nedlands, Australia

family: Meniawy

full_name: Tarek Meniawy

given: Tarek

name: Department of Medical Oncology, Universitair Ziekenhuis Ghent, Ghent, Belgium

family: Rottey

full_name: Sylvie Rottey

given: Sylvie

name: South Texas Accelerated Research Therapeutics Madrid-Fundacion Jimenez Diaz, Fundacion Jimenez Diaz Hospital, Madrid, Spain

family: Moreno

full_name: Victor Moreno

given: Victor

name: Drug Development Department, Gustave Roussy, Villejuif, France

family: Gazzah

full_name: Anas Gazzah

given: Anas

name: Medical Oncology Departement, Institut Claudius Regaud and Institut Universitaire du Cancer de Toulouse-Oncopole, Toulouse, France

family: Delord

full_name: Jean-Pierre Delord

given: Jean-Pierre

name: Medical Oncology Department, Hospital Universitario 12 de Octubre, Madrid, Spain

family: Paz-Ares

full_name: Luis Paz-Ares

given: Luis

name: Department of Medical Oncology and Hematology, University Hospital Zurich, Zurich, Switzerland

family: Britschgi

full_name: Christian Britschgi

given: Christian

name: Sidney Kimmel Cancer Center, Thomas Jefferson University Hospital, Philadelphia, Pennsylvania

family: Schilder

full_name: Russell J. Schilder

given: Russell J.

name: Princess Alexandra Hospital and Queensland University of Technology, Brisbane, Australia

family: O’Byrne

full_name: Kenneth O’Byrne

given: Kenneth

name: New Drugs Development Division for Innovative Therapies, University of Milano and Istituto Europeo Di Oncologia, Istituto di Ricovero e Cura a Carattere Scientifico, Milano, Italy

family: Curigliano

full_name: Giuseppe Curigliano

given: Giuseppe

name: Department of Oncology, Center of Cancer Immunotherapy, U932, Institut Curie, Paris, France

family: Romano

full_name: Emanuela Romano

given: Emanuela

name: Bristol-Myers Squibb, Princeton, New Jersey

family: Patah

full_name: Poliana Patah

given: Poliana

family: Wang

full_name: Rui Wang

given: Rui

family: Liu

full_name: Yali Liu

given: Yali

family: Bajaj

full_name: Gaurav Bajaj

given: Gaurav

name: Bras and Family Drug Development Program, Princess Margaret Cancer Centre, Toronto, Ontario, Canada

family: Siu

full_name: Lillian L. Siu

given: Lillian L.

journal_name: JAMA Oncology

evidence: open (via free pdf)

license_normalized: no license

repository_institution: Universita Degli Studi Di Milano - Archivio Istituzionale della Ricerca

repository_institution: PubMed Central - Europe PMC

repository_institution: University of Zurich - Zurich Open Repository and Archive

title: Safety, Tolerability, and Potential Clinical Activity of a Glucocorticoid-Induced TNF Receptor–Related Protein Agonist Alone or in Combination With Nivolumab for Patients With Advanced Solid Tumors

location_country:
Italy
United States
Switzerland
Netherlands
Germany
France
Belgium
Australia
Spain
Canada

location_facility:
IRCCS Istituto Nazionale Tumori Milano
UCSD Moores Cancer Center
Princess Alexandra Hospital
Linear Clinical Research Ltd
Emory University
Providence Portland Medical Center
Thomas Jefferson University Hospital
Universitair Ziekenhuis Gent
Liverpool Cancer Therapy Center
University Of Alabama At Birmingham
Institut Claudius Regaud
Cantonal Hospital St. Gallen
The Ohio State University
Istituto Europeo Di Oncologia
Local Institution
Institut Gustave Roussy
The West Clinic, P.C.

title: A Phase 1/2a Dose Escalation and Cohort Expansion Study for Safety, Tolerability, and Efficacy of BMS-986156 Administered Alone and in Combination With Nivolumab (BMS-936558, Anti PD-1 Monoclonal Antibody) in Advanced Solid Tumors

summary: The purpose of this study is to evaluate the safety and tumor-shrinking ability of experimental medication BMS-986156, when given by itself or in combination with nivolumab in patients with solid cancers that are advanced or cancers that have spread.


title: An Exploratory Multicenter, Open-Label, Single Arm Study of the Safety and Tolerability of Pirfenidone (Esbriet®) in Combination With Nintedanib (Ofev®) in Patients With Idiopathic Pulmonary Fibrosis

summary: This clinical study will evaluate the safety and tolerability of combination treatment of nintedanib and pirfenidone in participants with IPF. Eligible participants must have received pirfenidone for at least 16 weeks on a stable dose. Nintedanib will be added on Day 1 of the study as a combination treatment for IPF for 24 weeks.

ReferenceCitation: Flaherty KR, Fell CD, Huggins JT, Nunes H, Sussman R, Valenzuela C, Petzinger U, Stauffer JL, Gilberg F, Bengus M, Wijsenbeek M. Safety of nintedanib added to pirfenidone treatment for idiopathic pulmonary fibrosis. Eur Respir J. 2018 Aug 2;52(2). pii: 1800230. doi: 10.1183/13993003.00230-2018. Print 2018 Aug. Erratum in: Eur Respir J. 2018 Oct 4;52(4):.

family: Flaherty

full_name: Kevin R. Flaherty

given: Kevin R.

family: Fell

full_name: Charlene D. Fell

given: Charlene D.

family: Huggins

full_name: J. Terrill Huggins

given: J. Terrill

family: Nunes

full_name: Hilario Nunes

given: Hilario

family: Sussman

full_name: Robert Sussman

given: Robert

family: Valenzuela

full_name: Claudia Valenzuela

given: Claudia

family: Petzinger

full_name: Ute Petzinger

given: Ute

family: Stauffer

full_name: John L. Stauffer

given: John L.

family: Gilberg

full_name: Frank Gilberg

given: Frank

family: Bengus

full_name: Monica Bengus

given: Monica

family: Wijsenbeek

full_name: Marlies Wijsenbeek

given: Marlies

journal_name: European Respiratory Journal

repository_normalized: PubMed Central

repository_institution: Red Federada de Repositorios Institucionales de Publicaciones Científicas - LA Referencia

repository_normalized: dnet

evidence: oa repository (via pmcid lookup)

repositories:
PubMed Central
dnet
repub.eur.nl

publisher: European Respiratory Society (ERS)

publisher_dissemination: European Respiratory Society

publisher_group: European Respiratory Society

publisher_normalized: European Respiratory Society

lead_sponsor: Hoffmann-La Roche

location_country:
Italy
Netherlands
Germany
France
Spain
Denmark
United States
Canada

location_facility: Cardio-Pulmonary Associates of St. Luke's Hospital

location_facility: Ruhrlandklinik Lungenzentrum der UNI Essen Abt.Pneumologie-Allergologie

location_facility: John A. Butler, M.D. - Oregon Pulmonary Associates

location_facility: David Geffen School of Medicine at UCLA;Division of Pulmonary & Critical Care/ Department of Medic

location_facility: Azienda Ospedaliero Universitaria Pisana; U.O. Pneumologia

location_facility: Complejo Asistencial Universitario de Leon; Pneumology

location_facility: Hopital de Pontchaillou; Service de Pneumologie

location_facility: Gentofte Hospital, Lungemedicinsk Afdeling

location_facility: Klinikum Fulda gAG; Universitätsmedizin Marburg, Campus Fulda

location_facility: Hospital General Universitario De Valencia; Servicio de Neumologia

location_facility: Stanford University School of Medicine ; Pulmonary/Critical Care Medicine

location_facility: Hospital Universitario Virgen del Rocio; Servicio de Neumologia

location_facility: South Health Campus/Alberta Health Services/ University of Calgary

location_facility: Hospital Universitario de Canarias; Servicio de Neumologia

location_facility: Antonius Ziekenhuis; Dept of Lung Diseases

location_facility: A.O. Univ. Senese Policlinico S. Maria alle Scotte; UOC Malattie Resepiratorie e Trapianto Polmonare

location_facility: Hospital Universitari de Bellvitge ; Servicio de Neumologia

location_facility: Inova Health Care Services; Advanced Lung Disease Transplant Program

location_facility: A.O. Universitaria San Luigi Gonzaga di Orbassano; Malattie Apparato Respiratorio (MAR2)

location_facility: Hospital Universitario La Princesa; Servicio de Neumologia

location_facility: Fachkrankenhaus Coswig GmbH Zentrum f.Pneumologie Beatmungsmedizin Thorax-u.Gefäßchirurgie

location_facility: Medical University of South Carolina (MUSC); MUSC Pulmonary

location_facility: Hospital del Henares; Medicina Interna. Unidad de Neumología


design_allocation: Randomized

primary_purpose: Other

location_country:
Italy
France
Czechia
Poland
United Kingdom

acronym: Not applicable

location_facility:
Site PL71
Site GB45
Site FR32
Site GB42
Site FR33
Site GB44
Site GB46
Site IT52
Site CZ61
Site GB43

title: A Phase II, Parallel Group, Randomized, Multicentre, Open Label Study to Compare the Pharmacokinetics of Tacrolimus in De Novo Pediatric Allograft Recipients Treated With an Advagraf® or Prograf® Based Immunosuppressive Regimen, Including a Long-Term Follow-Up

summary:
The purpose of this study is to compare how the body absorbs and processes two different formulations of the anti-rejection medication tacrolimus (Advagraf® or Prograf®) in children receiving an organ transplant, and how safe and effective they are over a longer period of time.

This study is for children less than 16 years old. No minimum age has been set, however, to be included in this study participants must able to swallow the medication capsules intact.


title: A Phase 3, Randomized, Vehicle-Controlled, Double-Blind, Multicenter Study to Evaluate the Safety and Efficacy of Once-Daily CLS001 Topical Gel Versus Vehicle Administered for 12 Weeks to Subjects With Papulopustular Rosacea With a 4 Week Follow-up Period

summary: This study evaluates the safety and efficacy of once-daily application of CLS001 topical gel compared to vehicle gel in subjects with severe papulopustular rosacea.

lead_sponsor: Maruho Co., Ltd.

location_country:
United States
Sweden
Netherlands
Germany
France
Australia
New Zealand
Canada
United Kingdom


title: Confirmatory Validation of Oral Macimorelin as a Growth Hormone (GH) Stimulation Test (ST) for the Diagnosis of Adult Growth Hormone Deficiency (AGHD) in Comparison With the Insulin Tolerance Test (ITT)

summary: The Macimorelin Growth Hormone Stimulation Test (GHST) will be compared with the Insulin Tolerance Test (ITT) in an open-label, randomized, 2-way crossover Trial. The trial will include subjects suspected to have adult growth hormone deficiency (AGHD) and a group of healthy control subjects.

ReferenceCitation: Garcia JM, Biller BMK, Korbonits M, Popovic V, Luger A, Strasburger CJ, Chanson P, Medic-Stojanoska M, Schopohl J, Zakrzewska A, Pekic S, Bolanowski M, Swerdloff R, Wang C, Blevins T, Marcelli M, Ammer N, Sachse R, Yuen KCJ. Macimorelin as a Diagnostic Test for Adult GH Deficiency. J Clin Endocrinol Metab. 2018 Aug 1;103(8):3083-3093. doi: 10.1210/jc.2018-00665.

name: Geriatric Research Education and Clinical Center, Veterans Affairs Puget Sound Health Care System, University of Washington and SIBCR, Seattle, Washington

family: Garcia

full_name: Jose M Garcia

given: Jose M

name: Massachusetts General Hospital, Boston, Massachusetts

family: Biller

full_name: Beverly M K Biller

given: Beverly M K

name: Barts and the London School of Medicine, Queen Mary University of London, London, United Kingdom

family: Korbonits

full_name: Márta Korbonits

given: Márta

name: University of Belgrade Faculty of Medicine, Belgrade, Serbia

family: Popovic

full_name: Vera Popovic

given: Vera

name: Vienna General Hospital – Medical University Campus, Vienna, Austria

family: Luger

full_name: Anton Luger

given: Anton

name: Charité – Universitatsmedizin, Berlin, Germany

family: Strasburger

full_name: Christian J Strasburger

given: Christian J

name: Service d’Endocrinologie et des Maladies de la Reproduction, Centre de Référence des Maladies Rares de l’Hypophyse, Assistance Publique-Hôpitaux de Paris, Hôpital de Bicêtre, Le Kremlin Bicêtre, France

name: Unité Mixte de Recherche S-1185, Faculté de Médecine Paris Sud, Université Paris Sud, Le Kremlin-Bicêtre, France

family: Chanson

full_name: Philippe Chanson

given: Philippe

name: University of Novi Sad, Faculty of Medicine, Clinical Center of Vojvodina, Novi Sad, Serbia

family: Medic-Stojanoska

full_name: Milica Medic-Stojanoska

given: Milica

name: Medizinische Klinik IV, Ludwig Maximilian University of Munich, Munich, Germany

family: Schopohl

full_name: Jochen Schopohl

given: Jochen

name: Angelius Provita Medical Center, Katowice, Poland

family: Zakrzewska

full_name: Anna Zakrzewska

given: Anna

name: Clinic for Endocrinology, University Clinical Center, Belgrade, Serbia

family: Pekic

full_name: Sandra Pekic

given: Sandra

name: Medical University Wroclaw, Wrocław, Poland

family: Bolanowski

full_name: Marek Bolanowski

given: Marek

name: Los Angeles Biomedical Research Institute, Harbor-UCLA Medical Center, Torrance, California

family: Swerdloff

full_name: Ronald Swerdloff

given: Ronald

full_name: Christina Wang

given: Christina

name: Texas Diabetes and Endocrinology, Austin, Texas

family: Blevins

full_name: Thomas Blevins

given: Thomas

name: Baylor College of Medicine and Michael E. DeBakey Veterans Affairs Medical Center, Houston, Texas

family: Marcelli

full_name: Marco Marcelli

given: Marco

name: Aeterna Zentaris GmbH, Frankfurt, Germany

family: Ammer

full_name: Nicola Ammer

given: Nicola

family: Sachse

full_name: Richard Sachse

given: Richard

name: Swedish Neuroscience Institute, Seattle, Washington

family: Yuen

full_name: Kevin C J Yuen

given: Kevin C J

journal_name: The Journal of Clinical Endocrinology & Metabolism

evidence: open (via free article)

repository_institution: Queen Mary University of London - Queen Mary Research Online

version: submittedVersion

publisher: The Endocrine Society

publisher_dissemination: The Endocrine Society

location_country:
Italy
Austria
Serbia
Germany
France
Poland
Spain
United States
United Kingdom

location_facility: Klinik für Endokrinologie, Diabetes und Ernährungsmedizin der Charité

location_facility: Baylor College of Medicine-Endocrinology

location_facility: Medical University & General Hospital of Vienna, AKH,

location_facility: Phase I - MTZ Clinical Research Sp. z o.o.


title: Dose Escalation, Safety, Pharmacokinetic and Pharmacodynamic, First in Man Study, of SAR125844 Single Agent Administered as Slow Intravenous Infusion in Adult Patients With Advanced Malignant Solid Tumors

acronym: SARMET

summary:
Primary Objectives:

To determine the maximum tolerated dose (MTD) of SAR125844. To confirm safety profile of SAR125844 when administered as single agent at the MTD.

To evaluate the preliminary anti-tumoral effect of SAR125844 in patients with MET-gene amplified solid tumors (including sub-group of MET-amplified non-small cell lung cancer [NSCLC] patients) and in patients with Phospho-MET positive tumors without MET-gene amplification.

Secondary Objectives:

To characterize the global safety profile including cumulative toxicities. To evaluate the pharmacokinetic profile of SAR125844 in the proposed dosing schedule(s).

To assess preliminary antitumor activity in patients with measurable/evaluable disease, according to RECIST 1.1 criteria.

To explore the pharmacodynamic effects (PD) of SAR125844. To explore MET gene amplification status in Circulating Tumoral Cells (CTCs) and on tumor biopsies collected during the study, in the escalation part only.

To evaluate other pharmacodynamic biomarkers and help selection of patients who could benefit from SAR125844.

To explore MET-gene amplification status in circulating DNA.

design_allocation: Non-Randomized

ReferenceCitation: Angevin E, Spitaleri G, Rodon J, Dotti K, Isambert N, Salvagni S, Moreno V, Assadourian S, Gomez C, Harnois M, Hollebecque A, Azaro A, Hervieu A, Rihawi K, De Marinis F. A first-in-human phase I study of SAR125844, a selective MET tyrosine kinase inhibitor, in patients with advanced solid tumours with MET amplification. Eur J Cancer. 2017 Dec;87:131-139. doi: 10.1016/j.ejca.2017.10.016. Epub 2017 Nov 14.

family: Angevin

full_name: Eric Angevin

given: Eric

family: Spitaleri

full_name: Gianluca Spitaleri

given: Gianluca

family: Rodon

full_name: Jordi Rodon

given: Jordi

family: Dotti

full_name: Katia Dotti

given: Katia

family: Isambert

full_name: Nicolas Isambert

given: Nicolas

family: Salvagni

full_name: Stefania Salvagni

given: Stefania

family: Moreno

full_name: Victor Moreno

given: Victor

family: Assadourian

full_name: Sylvie Assadourian

given: Sylvie

family: Gomez

full_name: Corinne Gomez

given: Corinne

family: Harnois

full_name: Marzia Harnois

given: Marzia

family: Hollebecque

full_name: Antoine Hollebecque

given: Antoine

family: Azaro

full_name: Analia Azaro

given: Analia

family: Hervieu

full_name: Alice Hervieu

given: Alice

family: Rihawi

full_name: Karim Rihawi

given: Karim

family: De Marinis

full_name: Filippo De Marinis

given: Filippo

journal_name: European Journal of Cancer

oa_host_type: publisher

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

lead_sponsor: Sanofi

location_country:
Italy
United States
France
Spain

location_facility:
Investigational Site Number 380004
Investigational Site Number 840001
Investigational Site Number 380002
Investigational Site Number 380001
Investigational Site Number 724001
Investigational Site Number 250002
Investigational Site Number 724003
Investigational Site Number 250001


status: Terminated

primary_purpose: Prevention

location_country:
France
Poland
Belgium
Spain
Germany
United Kingdom

acronym: PROGRESSION

location_facility: Site DE31 Kliniken der Medizinischen Hoc

location_facility: Site GB13 Cent. Manchester Uni. Hospital

title: A Long-term, Open-label, Non-comparative Study to Evaluate the Safety and Efficacy of a Modigraf® Based Immunosuppression Regimen in Paediatric Solid Allograft Recipients

summary: The purpose of this study, a follow up to study FG506-CL-0403, is to see how safe and effective Modigraf® is (Part A) and to see how safe and effective it is to change your child's medication from Modigraf® to Prograf® (Part B).

status_simplified: Unknown


ReferenceType: result

ReferenceCitation: Kong A, Rea D, Ahmed S, Beck JT, López López R, Biganzoli L, Armstrong AC, Aglietta M, Alba E, Campone M, Hsu Schmitz SF, Lefebvre C, Akimov M, Lee SC. Phase 1B/2 study of the HSP90 inhibitor AUY922 plus trastuzumab in metastatic HER2-positive breast cancer patients who have progressed on trastuzumab-based regimen. Oncotarget. 2016 Jun 21;7(25):37680-37692. doi: 10.18632/oncotarget.8974.

name: Previous address: Churchill Hospital, Oxford University Hospitals NHS Trust and University of Oxford, Oxford, United Kingdom

name: School of Cancer Sciences, University of Birmingham, Birmingham, United Kingdom

family: Kong

full_name: Anthony Kong

given: Anthony

family: Rea

full_name: Daniel Rea

given: Daniel

name: Department of Oncology, University Hospitals of Leicester, Leicester Royal Infirmary, Leicester, United Kingdom

family: Ahmed

full_name: Samreen Ahmed

given: Samreen

name: Department of Oncology, Highlands Oncology Group, Fayetteville, Arkansas, USA

family: Beck

full_name: J. Thaddeus Beck

given: J. Thaddeus

name: Department of Oncology, Hospital Clinico Universitario, Santiago de Compostela, Spain

family: López

full_name: Rafael López López

given: Rafael López

name: Department of Medical Oncology, Nuovo Ospedale di Prato, Prato, Italy

family: Biganzoli

full_name: Laura Biganzoli

given: Laura

name: Department of Medical Oncology, The Christie NHS Foundation Trust, Manchester, United Kingdom

family: Armstrong

full_name: Anne C. Armstrong

given: Anne C.

name: Department of Medical Oncology, University of Torino, FPO-IRCCS, Candiolo, Italy

family: Aglietta

full_name: Massimo Aglietta

given: Massimo

name: Department of Medical Oncology, University Hospital, IBIMA, Malaga, Spain

family: Alba

full_name: Emilio Alba

given: Emilio

name: Department of Medical Oncology, Institut de Cancérologie de l'ouest René Gauducheau, Nantes, France

family: Campone

full_name: Mario Campone

given: Mario

name: Early Clinical Biostatistics, Oncology, Novartis Pharma AG, Basel, Switzerland

family: Schmitz

full_name: Shu-Fang Hsu Schmitz

given: Shu-Fang Hsu

name: Translational Clinical Oncology, Novartis Pharma AG, Basel, Switzerland

family: Lefebvre

full_name: Caroline Lefebvre

given: Caroline

name: Oncology Global Development, Novartis Pharma AG, Basel, Switzerland

family: Akimov

full_name: Mikhail Akimov

given: Mikhail

name: Department of Hematology-Oncology, National University Cancer Institute Singapore, Singapore

family: Lee

full_name: Soo-Chin Lee

given: Soo-Chin

journal_name: Oncotarget

evidence: open (via page says license)

license: cc-by

license_normalized: cc-by

repository_institution: University of Turin - Institutional Research Information System University of Turin

repository_institution: University of Leicester - Leicester Research Archive

publisher: Impact Journals, LLC

publisher_dissemination: Impact Journals, LLC

publisher_group: Impact Journals, LLC

publisher_normalized: Impact Journals, LLC

location_country:
Italy
United States
Sweden
France
Singapore
Spain
Germany
United Kingdom

location_facility: Highlands Oncology Group Dept of Highlands Oncology Grp

title: A Phase Ib/II, Multi-center, Open-label Study to Evaluate the Efficacy of AUY922 in Combination With Trastuzumab in Patients With Locally Advanced or Metastatic HER2-positive Breast Cancer, That Has Progressed After or During at Least One Trastuzumab-containing Regimen

summary:
The phase Ib part of the trial will assess the MTD of AUY922 in combination with Trastuzumab in patients with Trastuzumab-refractory locally advanced or metastatic HER2+. The MTD is defined as the highest drug dosage not causing in the first cycle of treatment (28 days) medically unacceptable dose limiting toxicity (DLT).

The phase II part of the trial will assess any potential effect on efficacy of adding AUY922 to Trastuzumab in patients with Trastuzumab-refractory locally advanced or metastatic HER2+ breast cancer.

Both AUY922 and Trastuzumab will be administered as a weekly IV infusion. Treatment should be continued as long as the patient does not have disease progression and tolerates the treatment. The following reasons are examples of acceptable reasons for discontinuing the study; tumor progression (by RECIST, as assessed by the investigator), unacceptable toxicity, death, or discontinuation from the study for any other reason, such as patient refusal, withdrawn consent, lost to follow-up or investigator decision.


title: SPIRIT V: A Clinical Evaluation of the XIENCE V® Everolimus Eluting Coronary Stent System in the Treatment of Patients With de Novo Coronary Artery Lesions

summary: The purpose of this Clinical Evaluation is a continuation in the assessment of the performance of the XIENCE V® Everolimus Eluting Coronary Stent System (XIENCE V® EECSS) in the treatment of patients with de novo coronary artery lesions in patients (Diabetic sub-study).

lead_sponsor: Abbott Medical Devices

location_country:
Italy
Austria
Netherlands
France
Malaysia
Poland
Israel
Thailand
Spain
Germany
United Kingdom

location_facility:
CHU Lille - Hôpital Cardiologique
Hospital Virgen de la Arrixaca
Hospital Santa Creu I Sant Pau
Salzburger Landeskliniken
General De Alicante
Hospital Puerta de Hierro
La Paz
King's College Hospital
C.H.U. - Hopital Michallon
King Chulalongkorn Memorial Hospital
Herzzentrum Siegburg GmbH
Clinico San Carlos
Universitätsklinikum
Sheba Medical Center
Medical University of Bydgoszcz
Herzzentrum
Azienda Ospedaliera Riuniti
Azienda Ospedaliera S. Gdi Dio Salerno
Wessex Cardiac Unit
Institute Jantung Negara
Medisch Centrum Alkmaar
Hospital General de Valencia
Azienda Ospedaliera di Padova
Maasstad Ziekenhuis
Lukas Krankenhaus Neuss
Ospedale Civile
IRCCS Policlinico San Matteo
Hospital Belvigte de Barcelona

intervention_type: Device


ReferenceType: background

ReferenceCitation: Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P, Paushkin S, Patel M, Trotta CR, Hwang S, Wilde RG, Karp G, Takasugi J, Chen G, Jones S, Ren H, Moon YC, Corson D, Turpoff AA, Campbell JA, Conn MM, Khan A, Almstead NG, Hedrick J, Mollin A, Risher N, Weetall M, Yeh S, Branstrom AA, Colacino JM, Babiak J, Ju WD, Hirawat S, Northcutt VJ, Miller LL, Spatrick P, He F, Kawana M, Feng H, Jacobson A, Peltz SW, Sweeney HL. PTC124 targets genetic disorders caused by nonsense mutations. Nature. 2007 May 3;447(7140):87-91. Epub 2007 Apr 22.

ReferenceCitation: Hirawat S, Welch EM, Elfring GL, Northcutt VJ, Paushkin S, Hwang S, Leonard EM, Almstead NG, Ju W, Peltz SW, Miller LL. Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers. J Clin Pharmacol. 2007 Apr;47(4):430-44.

lead_sponsor: PTC Therapeutics

location_country:
Italy
Switzerland
France
Belgium
Germany
United Kingdom

location_facility: Istituti Clinici di Perfezionamento, Milano

location_facility: University Hospital, Department of Pediatrics

location_facility: ZNA Queen Paola Child Hospital and Provincial Centre for Metabolic Disorders

title: A Phase 2 Study of Ataluren (PTC124®) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia

summary: Methylmalonic acidemia (MMA) is a rare genetic disorder caused by mutations in the gene for mitochondrial enzyme methylmalonyl-CoA mutase (MCM) or in one of the genes for adenosylcobalamin (AdoCbl). Lack of these proteins causes toxic elevations of methylmalonic acid (MMacid) in blood, urine, and other tissues. A specific type of mutation, called a nonsense (premature stop codon) mutation, is the cause of the disease in approximately 5% to 20% of participants with mutations in the MCM gene, and approximately 20% to >50% of participants with mutations in one of the AdoCbl genes. Ataluren is an orally delivered, investigational drug that acts to overcome the effects of the premature stop codon, potentially enabling the production of functional MCM/AdoCbl. This study is a Phase 2a trial evaluating the safety and activity of ataluren in participants with MMA due to a nonsense mutation. The main purpose of this study is to understand whether ataluren can safely decrease MMacid levels.


title: Evaluation of PillCam™ Colon 2 in Visualization of the Colon

summary: Evaluate accuracy of PCCE-2 in detecting patients with colonic polyps as compared to conventional colonoscopy.

lead_sponsor: Medtronic - MITG

location_country: Italy Sweden Netherlands France Belgium Spain Germany

location_facility: Chef de Service dHepato-Gastro-Enterologie et dAssistance Nutritionnelle CHU Nantes

location_facility: Academic Medical Center University of Amsterdam


lead_sponsor: European Organisation for Research and Treatment of Cancer - EORTC

location_country:
Italy
Switzerland
Netherlands
France
Belgium
Spain
Germany
United Kingdom

location_facility: Clatterbridge Cancer Centre NHS Foundation Trust

location_facility: ICO Badalona - Hospital Germans Trias i Pujol

location_facility: Erasmus MC - Daniel den Hoed Cancer Center

location_facility: Institut de Cancerologie de l'Ouest (ICO) - Centre Rene Gauducheau

title: Radiation Therapy and Concurrent Plus Adjuvant Temsirolimus (CCI-779) Versus Chemo-Irradiation With Temozolomide in Newly Diagnosed Glioblastoma Without Methylation of the MGMT Gene Promoter - A Randomized Multicenter, Open-Label, Phase II Study.

summary:
RATIONALE: Radiation therapy uses high-energy x-rays to kill tumor cells. Temsirolimus may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as temozolomide, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. It is not yet known whether radiation therapy is more effective when given together with temsirolimus or temozolomide in treating patients with glioblastoma.

PURPOSE: This randomized phase II trial is studying giving radiation therapy together with temsirolimus to see how well it works compared with giving radiation therapy together with temozolomide in treating patients with newly diagnosed glioblastoma.


ReferenceCitation: Beelen DW, Trenschel R, Stelljes M, Groth C, Masszi T, Reményi P, Wagner-Drouet EM, Hauptrock B, Dreger P, Luft T, Bethge W, Vogel W, Ciceri F, Peccatori J, Stölzel F, Schetelig J, Junghanß C, Grosse-Thie C, Michallet M, Labussiere-Wallet H, Schaefer-Eckart K, Dressler S, Grigoleit GU, Mielke S, Scheid C, Holtick U, Patriarca F, Medeot M, Rambaldi A, Micò MC, Niederwieser D, Franke GN, Hilgendorf I, Winkelmann NR, Russo D, Socié G, Peffault de Latour R, Holler E, Wolff D, Glass B, Casper J, Wulf G, Menzel H, Basara N, Bieniaszewska M, Stuhler G, Verbeek M, Grass S, Iori AP, Finke J, Benedetti F, Pichlmeier U, Hemmelmann C, Tribanek M, Klein A, Mylius HA, Baumgart J, Dzierzak-Mietla M, Markiewicz M. Treosulfan or busulfan plus fludarabine as conditioning treatment before allogeneic haemopoietic stem cell transplantation for older patients with acute myeloid leukaemia or myelodysplastic syndrome (MC-FludT.14/L): a randomised, non-inferiority, phase 3 trial. Lancet Haematol. 2020 Jan;7(1):e28-e39. doi: 10.1016/S2352-3026(19)30157-7. Epub 2019 Oct 9.

family: Beelen

full_name: Dietrich Wilhelm Beelen

given: Dietrich Wilhelm

family: Trenschel

full_name: Rudolf Trenschel

given: Rudolf

family: Stelljes

full_name: Matthias Stelljes

given: Matthias

family: Groth

full_name: Christoph Groth

given: Christoph

family: Masszi

full_name: Tamás Masszi

given: Tamás

family: Reményi

full_name: Péter Reményi

given: Péter

family: Wagner-Drouet

full_name: Eva-Maria Wagner-Drouet

given: Eva-Maria

family: Hauptrock

full_name: Beate Hauptrock

given: Beate

family: Dreger

full_name: Peter Dreger

given: Peter

family: Luft

full_name: Thomas Luft

given: Thomas

family: Bethge

full_name: Wolfgang Bethge

given: Wolfgang

family: Vogel

full_name: Wichard Vogel

given: Wichard

family: Ciceri

full_name: Fabio Ciceri

given: Fabio

family: Peccatori

full_name: Jacopo Peccatori

given: Jacopo

family: Stölzel

full_name: Friedrich Stölzel

given: Friedrich

family: Schetelig

full_name: Johannes Schetelig

given: Johannes

family: Junghanß

full_name: Christian Junghanß

given: Christian

family: Grosse-Thie

full_name: Christina Grosse-Thie

given: Christina

family: Michallet

full_name: Mauricette Michallet

given: Mauricette

family: Labussiere-Wallet

full_name: Hélène Labussiere-Wallet

given: Hélène

family: Schaefer-Eckart

full_name: Kerstin Schaefer-Eckart

given: Kerstin

family: Dressler

full_name: Sabine Dressler

given: Sabine

family: Grigoleit

full_name: Goetz Ulrich Grigoleit

given: Goetz Ulrich

family: Mielke

full_name: Stephan Mielke

given: Stephan

family: Scheid

full_name: Christof Scheid

given: Christof

location_country: Italy Finland France Poland Hungary Germany

acronym: Clinical phase III trial of Treosulfan-based conditioning vs. RIC

location_facility: St. Istvan and St. Laszlo Hospital of Budapest

location_facility: Universitätsklinikum Leipzig, Haematologie, internistische Onkologie

location_facility: Universitätsmedizin Goettingen, Haematolgie und Onkologie

location_facility: Klinikum Nürnberg, 5. Medizinische Klinik

location_facility: Helsinki University Central Hospital, Dept. of Medicine

location_facility: Johannes-Gutenberg-Universität Mainz, III. Medizinische Klinik

location_facility: Clinica Ematologica ed Unita di Terapie Cellulari 'Carlo Melzi'

location_facility: Policlinico GB Rossi (Borgo Roma), Div. di Ematologia

location_facility: Universitätsklinikum Carl Gustav Carus Dresden, Med. Klinik I

location_facility: Klinikum Rechts der Isar der TU München, III. Med. Klinik

location_facility: Malteser Krankenhaus St. Franziskus-Hospital

title: Clinical Phase III Trial to Compare Treosulfan-based Conditioning Therapy With Busulfan-based Reduced-intensity Conditioning (RIC) Prior to Allogeneic Haematopoietic Stem Cell Transplantation in Patients With AML or MDS Considered Ineligible to Standard Conditioning Regimens

summary: This randomized allogeneic transplantation protocol compares i.v. Treosulfan-based conditioning therapy with reduced intensity i.v. Busulfan-based conditioning in adult AML and MDS patients at increased risk for standard conditioning therapies. The protocol is based on results of previous phase I/II trials evaluating Treosulfan/Fludarabine conditioning prior to allogeneic haematopoietic stem cell transplantation. The reference arm (reduced intensity i.v. Busulfan/Fludarabine) is considered to be accepted medical practice for the study patient population.


title: Efficacy And Safety Of Eraxis/Ecalta (Anidulafungin) Compared To Cancidas (Caspofungin) In Neutropenic Patients With Invasive Candida Infection

summary: The purpose of this study is to gather information on the use of anidulafungin for the treatment of Candida infection in patients with an abnormal immune system. It is expected that anidulafungin will be at least as safe and as effective as the comparator drug, caspofungin.

ReferenceCitation: De Rosa FG, Busca A, Capparella MR, Yan JL, Aram JA. Invasive Candidiasis in Patients with Solid Tumors Treated with Anidulafungin: A Post Hoc Analysis of Efficacy and Safety of Six Pooled Studies. Clin Drug Investig. 2021 Jun;41(6):539-548. doi: 10.1007/s40261-021-01024-7. Epub 2021 Apr 23.

family: De Rosa

full_name: Francesco Giuseppe De Rosa

given: Francesco Giuseppe

family: Busca

full_name: Alessandro Busca

given: Alessandro

family: Capparella

full_name: Maria Rita Capparella

given: Maria Rita

family: Yan

full_name: Jean Li Yan

given: Jean Li

family: Aram

full_name: Jalal A. Aram

given: Jalal A.

journal_name: Clinical Drug Investigation

repositories_institution: University of Turin - Institutional Research Information System University of Turin

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

ReferenceCitation: Kontoyiannis DP, Bassetti M, Nucci M, Capparella MR, Yan JL, Aram J, Hogan PA. Anidulafungin for the treatment of candidaemia caused by Candida parapsilosis: Analysis of pooled data from six prospective clinical studies. Mycoses. 2017 Oct;60(10):663-667. doi: 10.1111/myc.12641. Epub 2017 Jun 9.

name: The University of Texas MD Anderson Cancer Center; Houston TX USA

family: Kontoyiannis

full_name: Dimitrios P. Kontoyiannis

given: Dimitrios P.

name: Department of Medicine; University of Udine; Udine Italy

family: Bassetti

full_name: Matteo Bassetti

given: Matteo

name: Universidade Federal do Rio de Janeiro; Rio de Janeiro Brazil

family: Nucci

full_name: Marcio Nucci

given: Marcio

full_name: Jean L. Yan

given: Jean L.

full_name: Jalal Aram

given: Jalal

family: Hogan

full_name: Patricia A. Hogan

given: Patricia A.

journal_name: Mycoses

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

title: Anidulafungin for the treatment of candidaemia caused by Candida parapsilosis : Analysis of pooled data from six prospective clinical studies

ReferenceCitation: Kullberg BJ, Vasquez J, Mootsikapun P, Nucci M, Paiva JA, Garbino J, Yan JL, Aram J, Capparella MR, Conte U, Schlamm H, Swanson R, Herbrecht R. Efficacy of anidulafungin in 539 patients with invasive candidiasis: a patient-level pooled analysis of six clinical trials. J Antimicrob Chemother. 2017 Aug 1;72(8):2368-2377. doi: 10.1093/jac/dkx116.

name: Department of Medicine and Radboud Center for Infectious Diseases, Radboud University Medical Center, PO Box 9101, Geert Grooteplein 8, 6525 GA Nijmegen, The Netherlands

family: Kullberg

full_name: Bart Jan Kullberg

given: Bart Jan

name: Division of Infectious Diseases, Medical College of Georgia/Augusta University, 1120 15th Street, Augusta, GA 30912, USA

name: Department of Medicine, Srinagarind Hospital, Mueang Khon Kaen 40000, Thailand

name: Department of Internal Medicine, University Hospital, Universidade Federal do Rio de Janeiro, Avenida Pedro Calmon 550, Rio de Janeiro 21941-901, Brazil

name: Department of Emergency and Intensive Care, Centro Hospitalar São João, Faculty of Medicine, University of Porto, Praça Gomes Teixeira, Porto 4099-002, Portugal

name: University Hospitals of Geneva, 4 rue Gabrille-Perret-Gentil, CH 1211 Geneva, Switzerland

name: Pfizer Inc., 235 E 42nd Street, New York, NY, USA

name: Pfizer PFE, 23-25 avenue du Docteur Lannelongue, 75668 Paris cedex 14, France

name: Department of Oncology and Hematology, Hôpital de Hautepierre and Université de Strasbourg, 1 place de l’Hôpital, BP 426, 67091 Strasbourg cedex, France

repositories_institution:
PubMed Central - Europe PMC
Radboud University - Radboud Repository

location_country:
Italy
Bosnia and Herzegovina
France
Poland
Slovakia
Russian Federation


title: Randomized Phase III Intergroup Trial in Resected Stage 2 (Dukes B) Colon Cancer: 6-Month Infusional 5FU-CPT11 (+/- Folinic Acid) Versus Observation - Determination of Biologic Predictive and Response Factors

summary:
RATIONALE: Drugs used in chemotherapy, such as irinotecan, leucovorin, and fluorouracil, work in different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug and giving them after surgery may kill any remaining tumor cells. It is not yet known whether combination chemotherapy is more effective than observation alone in treating patients who have undergone surgery for colon cancer.

PURPOSE: This randomized phase III trial is studying irinotecan and fluorouracil with or without leucovorin to see how well they work compared to observation alone in treating patients who have undergone surgery for stage II colon cancer.

status: Unknown status

enrollment_type: Anticipated

lead_sponsor: Federation Francophone de Cancerologie Digestive

location_country: Italy Spain France Egypt Portugal Germany

location_facility: Universita Degli Studi di Florence - Policlinico di Careggi

location_facility: Instituto Portugues de Oncologia Centro do Porto, S. A.

location_facility: Centre Hospitalier Universitaire Ambroise Pare - Boulogne


title: An International Field Study of the Reliability and Validity of the EORTC-QLQ-C30 and a Disease Specific Questionnaire Module (the EORTC OES-24) in Assessing the Quality of Life of Patients With Oesophageal Cancer

summary:
RATIONALE: Questionnaires that measure quality of life may improve the ability to plan treatment for patients who have cancer of the esophagus.

PURPOSE: This clinical trial studies quality-of-life assessments of patients with cancer of the esophagus who are receiving treatment.

ReferenceCitation: Conroy T, Etienne PL, Adenis A, Ducreux M, Paillot B, Oliveira J, Seitz JF, Francois E, Van Cutsem E, Wagener DJ, Kohser F, Daamen S, Praet M, Gorlia T, Baron B, Wils J; European Organisation for Research and Treatment of Cancer Gastrointestinal Tract Cancer Cooperative Group. Vinorelbine and cisplatin in metastatic squamous cell carcinoma of the oesophagus: response, toxicity, quality of life and survival. Ann Oncol. 2002 May;13(5):721-9.

location_country:
Sweden
Switzerland
France
Norway
Australia
Spain
Germany
United Kingdom

location_facility: Hopital Cantonal Universitaire de Geneva


title: A Phase 1b, Open-label, Single-arm, Multicenter Study to Assess Efficacy, Safety, and Tolerability of Treatment With JNJ-73763989, JNJ-64300535, and Nucleos(t)Ide Analogs in Virologically Suppressed, HBeAg-negative Participants With Chronic Hepatitis B Virus Infection

acronym: OSPREY

summary: The purpose of this study is to evaluate the efficacy of the study intervention based on hepatitis B surface antigen (HBsAg) levels.

study_start_date_type: Anticipated

status: Not yet recruiting

study_completion_date_type: Anticipated

ipd_sharing: Yes

ipd_sharing_description: The data sharing policy of the Janssen Pharmaceutical Companies of Johnson and Johnson is available at www.janssen.com/clinical trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

lead_sponsor: Janssen Research & Development, LLC

location_country:
Italy
France
Poland
Belgium
New Zealand
Spain
Germany
United Kingdom
Taiwan

location_facility: Chang Gung Memorial Hospital Linkou Branch

location_facility: Azienda Ospedaliero Universitaria Pisana

status_simplified: Ongoing


type: Registry Identifier

title: A Multi-centre, Randomised, Placebo Controlled, Double Blinded, Multiple Dose Trial Investigating Safety, Pharmacokinetics and Pharmacodynamics of Concizumab Administered Subcutaneously to Haemophilia A Subjects (Explorer™3)

summary: This trial is conducted globally. The aim of this trial is to investigate safety, pharmacokinetics (the exposure of the trial drug in the body) and pharmacodynamics (the effect of the investigated drug on the body) of concizumab administered subcutaneously to haemophilia A subjects.

lead_sponsor: Novo Nordisk A/S

location_country:
Austria
Netherlands
Germany
Croatia
France
Malaysia
Israel
Poland
Thailand
Australia
Spain
Turkey
United States
United Kingdom
Ukraine


intervention_type: Other

lead_sponsor: UCB BIOSCIENCES, Inc.

location_country:
Greece
Romania
Russian Federation
Taiwan
Ukraine
Croatia
France
Poland
Slovakia
Thailand
Argentina
Moldova, Republic of
Korea, Republic of
Philippines
Bulgaria
Hungary
China
Portugal
United States
Italy
Brazil
Serbia
Mexico
Czechia
Israel
Georgia

title: A Multicenter, Double-Blind, Randomized, Placebo-Controlled, Parallel-Group Study to Investigate the Efficacy and Safety of Lacosamide as Adjunctive Therapy in Subjects With Epilepsy >=1 Month to <4 Years of Age With Partial-Onset Seizures

summary: The purpose of this trial is to assess the efficacy, safety and tolerability of lacosamide administered as add-on therapy with 1 to 3 anti-seizure medications. This trial is for children aged 1 month to less than 4 years with epilepsy who currently have uncontrolled partial-onset seizures.


title: A PROSPECTIVE, MULTI-CENTER, SINGLE-ARM, POST MARKET OBSERVATIONAL STUDY TO EVALUATE THE SAFETY AND CLINICAL PERFORMANCE OF THE ARCHIMEDES BIODEGRADABLE BILIARY AND PANCREATIC STENT

acronym: AM GI Registry

summary:
Significant experience with biodegradable materials has been reported in the orthopedic literature; in particular; biodegradable stents have been used in the endovascular and urologic epithelium. Stents are typically made from polymeric materials including Polylactic Acid (PLA), Polyglycolide (PGA), Polydioxanone (PDO), Polyethyleneglycol (PEG), Polycaprolactone (PCL), etc., which have been cleared or approved by the FDA and CE-marked as drug carriers, sutures, and bone fixatives. The material is degraded by hydrolysis in, e.g., lactic acid, which is removed from the body by normal metabolic pathways. In the field of gastroenterology, these stents were first introduced in patients with an esophageal stenosis with encouraging results, although esophageal hyperplasia was a frequently encountered complication.

For biliary applications, biodegradable stents have been evaluated in several in-vitro and animal studies that demonstrated that the stents were safe and well tolerated. These stents provided an adequate radial force and resulted in complete stricture resolution within several months. The stents did not show any signs of biliary hyperplasia or integration in the epithelium. Moreover, they seem to have a self-clearing effect on attached biofilm as the outer layer sloughs during the degradation process similarly to the exfoliation of human skin. Also, the stent could be removed from the bile duct, thus offering the possibility of extraction if necessary at various times after implantation.

In 2010, Petryl was the first to use a biodegradable stent in the human bile duct. A stent was successfully placed using percutaneous transhepatic cholangiograpy in two patients with a postsurgical intrahepatic biliary stricture. Transient cholangitis was the only complication encountered and during the two years of follow-up, the bile duct remained patent.

Later, Mauri et al. presented in 2013 and 2016 results of a 107 patient study on a polydioxanone biodegradable biliary stent. The results have been published in the Journal of the European Society of Radiology. The authors concluded that percutaneous placement of a biodegradable biliary stent is a feasible and safe strategy to treat benign biliary strictures refractory to standard bilioplasty, with promising results in the mid-term period.

Prior to this study, the ARCHIMEDES device was assessed in the ARCHIMEDES Investigational study which has completed enrolment with 53 patients included, and the results are not yet published. In this study approximately half the stents were deployed in the bile duct (53%) and half in the pancreatic duct. All 53 patients have either completed, exited, or no longer have the study device. No patient remains in the study with a stent in place during the time of the last patient follow-ups. The study concluded without any device-related SAE. Bilirubin was reduced by 25.6%, which exceeded the >20% clinical success criterion, quality of life score improved from 3.7 to 7.9, procedural success was rated 1.4 (good to excellent), and technical success was achieved in all 53 patients.

Data from the ARCHIMEDES Investigational study was used for CE Mark approval, which was obtained in June 2018.

status: Recruiting

lead_sponsor: QualiMed Innovative Medizinprodukte GmbH

location_country: Italy Finland France Israel Belgium Spain Germany Slovenia

location_facility: AUSL Romagna Ospedale Morgagni - Pierantoni

location_facility: Fondazione Policlinico Universitario Agostino Gemelli

location_facility: Endoscopy Unit, Humanitas Research Hospital


title: A Phase 2, Double-blind, Randomized, Placebo-controlled Multicenter Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of JBT-101 in Cystic Fibrosis

summary: The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and efficacy of JBT-101 in adult subjects with cystic fibrosis (CF).

ReferenceCitation: Chmiel JF, Flume P, Downey DG, Dozor AJ, Colombo C, Mazurek H, Sapiejka E, Rachel M, Constantine S, Conley B, Dgetluck N, Dinh Q, White B, Elborn JS; Lenabasum JBT101-CF-001 Study Group. Safety and efficacy of lenabasum in a phase 2 randomized, placebo-controlled trial in adults with cystic fibrosis. J Cyst Fibros. 2021 Jan;20(1):78-85. doi: 10.1016/j.jcf.2020.09.008. Epub 2020 Oct 1.

family: Chmiel

full_name: James F. Chmiel

given: James F.

family: Flume

full_name: Patrick Flume

given: Patrick

family: Downey

full_name: Damian G. Downey

given: Damian G.

family: Dozor

full_name: Allen J. Dozor

given: Allen J.

family: Colombo

full_name: Carla Colombo

given: Carla

family: Mazurek

full_name: Henryk Mazurek

given: Henryk

family: Sapiejka

full_name: Ewa Sapiejka

given: Ewa

family: Rachel

full_name: Marta Rachel

given: Marta

family: Constantine

full_name: Scott Constantine

given: Scott

family: Conley

full_name: Brian Conley

given: Brian

family: Dgetluck

full_name: Nancy Dgetluck

given: Nancy

family: Dinh

full_name: Quinn Dinh

given: Quinn

family: White

full_name: Barbara White

given: Barbara

family: Elborn

full_name: J. Stuart Elborn

given: J. Stuart

journal_name: Journal of Cystic Fibrosis

lead_sponsor: Corbus Pharmaceuticals Inc.

location_country:
Italy
United States
France
Poland
Belgium
Germany
United Kingdom

location_facility: Podkarpacki Ośrodek Pulmunologii i Alergologii

location_facility: Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

location_facility: University of Arkansas for Medical Sciences

location_facility: Long Beach Memorial Medical Center/Miller Children's and Women's Hospital

location_facility: Christiane Herzog CF-Zentrum Frankfurt am Main

location_facility: Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico

location_facility: AOUI di Verona - Ospedale Borgo Trento UOC Fibrosi Cistica

location_facility: Texas Children's Hospital Clinical Care Center

location_facility: University Hospitals Cleveland Medical Center

location_facility: The University of Texas Southwestern Medical Center

location_facility: Szpital Dziecięcy Polanki im. Macieja Płażyńskiego w Gdańsku Spółka z o.o. Poradnia Leczenia Mukowiscydozy

location_facility: Rutgers Robert-Wood Johnson Medical School

location_facility: Instytut Gruźlicy i Chorób Płuc I Klinika Chorób Płuc


title: An Open-Label, Single-Arm, Phase 1/2 Study Evaluating the Safety and Efficacy of Ponatinib for the Treatment of Recurrent or Refractory Leukemias or Solid Tumors in Pediatric Participants

summary: The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and efficacy of ponatinib in children aged 1 to < 18 years with advanced leukemias, lymphomas, and solid tumors.

lead_sponsor: Incyte Biosciences International Sàrl

location_country:
Italy
Sweden
Netherlands
France
Belgium
Spain
Denmark
United Kingdom

location_facility: Centre Hospitalier Universitaire de Poitiers

location_facility: Azienda Ospedaliero Universitaria Citta Della Salute E Della Scienza

location_facility: The Royal Marsden Nhs Foundation Trust - Sutton

location_facility: Fondazione Irccs Istituto Nazionale Dei Tumori

location_facility: Princess Maxima Center For Pediatric Oncology


status: Active, not recruiting

lead_sponsor: Alnylam Pharmaceuticals

location_country:
United States
France
Israel
Germany
United Kingdom

acronym: ILLUMINATE-B

title: ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1

summary: The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).