title: Quantifying Systemic Immunosuppression to Personalize Cancer Therapy

acronym: SERPENTINE

summary: It is nowadays well established that the immune system can profoundly influence disease outcome in cancer patients. Increasing evidence is indeed showing that patients displaying spontaneous T cell-mediated immune response against their tumor (defined as immune surveillance) have higher chance to respond to therapies and display globally better prognosis. Conversely, patients whose tumor is characterized by immunosuppression, usually involving myeloid cells and chronic inflammation pathways, often undergo rapid progression and rarely benefit from therapy. Hence, capturing the immune features of individual tumors can help to predict disease course and tailor the therapeutic workup in clinical setting.

ReferenceCitation: Ribas A, Wolchok JD. Cancer immunotherapy using checkpoint blockade. Science. 2018 Mar 23;359(6382):1350-1355. doi: 10.1126/science.aar4060. Epub 2018 Mar 22. Review.

ReferenceCitation: Galluzzi L, Buqué A, Kepp O, Zitvogel L, Kroemer G. Immunological Effects of Conventional Chemotherapy and Targeted Anticancer Agents. Cancer Cell. 2015 Dec 14;28(6):690-714. doi: 10.1016/j.ccell.2015.10.012. Review.

ReferenceCitation: Apetoh L, Tesniere A, Ghiringhelli F, Kroemer G, Zitvogel L. Molecular interactions between dying tumor cells and the innate immune system determine the efficacy of conventional anticancer therapies. Cancer Res. 2008 Jun 1;68(11):4026-30. doi: 10.1158/0008-5472.CAN-08-0427.

ReferenceCitation: Peguillet I, Milder M, Louis D, Vincent-Salomon A, Dorval T, Piperno-Neumann S, Scholl SM, Lantz O. High numbers of differentiated effector CD4 T cells are found in patients with cancer and correlate with clinical response after neoadjuvant therapy of breast cancer. Cancer Res. 2014 Apr 15;74(8):2204-16. doi: 10.1158/0008-5472.CAN-13-2269. Epub 2014 Feb 17.

ReferenceCitation: Wilmott JS, Long GV, Howle JR, Haydu LE, Sharma RN, Thompson JF, Kefford RF, Hersey P, Scolyer RA. Selective BRAF inhibitors induce marked T-cell infiltration into human metastatic melanoma. Clin Cancer Res. 2012 Mar 1;18(5):1386-94. doi: 10.1158/1078-0432.CCR-11-2479. Epub 2011 Dec 12.

ReferenceCitation: Thorsson V, Gibbs DL, Brown SD, Wolf D, Bortone DS, Ou Yang TH, Porta-Pardo E, Gao GF, Plaisier CL, Eddy JA, Ziv E, Culhane AC, Paull EO, Sivakumar IKA, Gentles AJ, Malhotra R, Farshidfar F, Colaprico A, Parker JS, Mose LE, Vo NS, Liu J, Liu Y, Rader J, Dhankani V, Reynolds SM, Bowlby R, Califano A, Cherniack AD, Anastassiou D, Bedognetti D, Mokrab Y, Newman AM, Rao A, Chen K, Krasnitz A, Hu H, Malta TM, Noushmehr H, Pedamallu CS, Bullman S, Ojesina AI, Lamb A, Zhou W, Shen H, Choueiri TK, Weinstein JN, Guinney J, Saltz J, Holt RA, Rabkin CS; Cancer Genome Atlas Research Network, Lazar AJ, Serody JS, Demicco EG, Disis ML, Vincent BG, Shmulevich I. The Immune Landscape of Cancer. Immunity. 2018 Apr 17;48(4):812-830.e14. doi: 10.1016/j.immuni.2018.03.023. Epub 2018 Apr 5. Erratum in: Immunity. 2019 Aug 20;51(2):411-412.

ReferenceCitation: Spitzer MH, Carmi Y, Reticker-Flynn NE, Kwek SS, Madhireddy D, Martins MM, Gherardini PF, Prestwood TR, Chabon J, Bendall SC, Fong L, Nolan GP, Engleman EG. Systemic Immunity Is Required for Effective Cancer Immunotherapy. Cell. 2017 Jan 26;168(3):487-502.e15. doi: 10.1016/j.cell.2016.12.022. Epub 2017 Jan 19.

ReferenceCitation: Dumeaux V, Fjukstad B, Fjosne HE, Frantzen JO, Holmen MM, Rodegerdts E, Schlichting E, Borresen-Dale AL, Bongo LA, Lund E, Hallett M. Interactions between the tumor and the blood systemic response of breast cancer patients. PLoS Comput Biol. 2017 Sep 28;13(9):e1005680. doi: 10.1371/journal.pcbi.1005680. eCollection 2017 Sep.

ReferenceCitation: Cortez-Retamozo V, Etzrodt M, Newton A, Rauch PJ, Chudnovskiy A, Berger C, Ryan RJ, Iwamoto Y, Marinelli B, Gorbatov R, Forghani R, Novobrantseva TI, Koteliansky V, Figueiredo JL, Chen JW, Anderson DG, Nahrendorf M, Swirski FK, Weissleder R, Pittet MJ. Origins of tumor-associated macrophages and neutrophils. Proc Natl Acad Sci U S A. 2012 Feb 14;109(7):2491-6. doi: 10.1073/pnas.1113744109. Epub 2012 Jan 30.

ReferenceCitation: Gabrilovich DI. Myeloid-Derived Suppressor Cells. Cancer Immunol Res. 2017 Jan;5(1):3-8. doi: 10.1158/2326-6066.CIR-16-0297.

ReferenceCitation: Groth C, Hu X, Weber R, Fleming V, Altevogt P, Utikal J, Umansky V. Immunosuppression mediated by myeloid-derived suppressor cells (MDSCs) during tumour progression. Br J Cancer. 2019 Jan;120(1):16-25. doi: 10.1038/s41416-018-0333-1. Epub 2018 Nov 9.

ReferenceCitation: Ostrand-Rosenberg S. Myeloid derived-suppressor cells: their role in cancer and obesity. Curr Opin Immunol. 2018 Apr;51:68-75. doi: 10.1016/j.coi.2018.03.007. Epub 2018 Mar 13.

ReferenceCitation: Wesolowski R, Markowitz J, Carson WE 3rd. Myeloid derived suppressor cells - a new therapeutic target in the treatment of cancer. J Immunother Cancer. 2013 Jul 15;1:10. doi: 10.1186/2051-1426-1-10. eCollection 2013.

ReferenceCitation: Fleming V, Hu X, Weber R, Nagibin V, Groth C, Altevogt P, Utikal J, Umansky V. Targeting Myeloid-Derived Suppressor Cells to Bypass Tumor-Induced Immunosuppression. Front Immunol. 2018 Mar 2;9:398. doi: 10.3389/fimmu.2018.00398. eCollection 2018.

ReferenceCitation: Engblom C, Pfirschke C, Pittet MJ. The role of myeloid cells in cancer therapies. Nat Rev Cancer. 2016 Jul;16(7):447-62. doi: 10.1038/nrc.2016.54.

ReferenceCitation: Filipazzi P, Valenti R, Huber V, Pilla L, Canese P, Iero M, Castelli C, Mariani L, Parmiani G, Rivoltini L. Identification of a new subset of myeloid suppressor cells in peripheral blood of melanoma patients with modulation by a granulocyte-macrophage colony-stimulation factor-based antitumor vaccine. J Clin Oncol. 2007 Jun 20;25(18):2546-53. doi: 10.1200/JCO.2006.08.5829.

ReferenceCitation: Filipazzi P, Huber V, Rivoltini L. Phenotype, function and clinical implications of myeloid-derived suppressor cells in cancer patients. Cancer Immunol Immunother. 2012 Feb;61(2):255-263. doi: 10.1007/s00262-011-1161-9. Epub 2011 Nov 27. Review.

ReferenceCitation: Blattner C, Fleming V, Weber R, Himmelhan B, Altevogt P, Gebhardt C, Schulze TJ, Razon H, Hawila E, Wildbaum G, Utikal J, Karin N, Umansky V. CCR5+ Myeloid-Derived Suppressor Cells Are Enriched and Activated in Melanoma Lesions. Cancer Res. 2018 Jan 1;78(1):157-167. doi: 10.1158/0008-5472.CAN-17-0348. Epub 2017 Oct 31.

ReferenceCitation: Huber V, Vallacchi V, Fleming V, Hu X, Cova A, Dugo M, Shahaj E, Sulsenti R, Vergani E, Filipazzi P, De Laurentiis A, Lalli L, Di Guardo L, Patuzzo R, Vergani B, Casiraghi E, Cossa M, Gualeni A, Bollati V, Arienti F, De Braud F, Mariani L, Villa A, Altevogt P, Umansky V, Rodolfo M, Rivoltini L. Tumor-derived microRNAs induce myeloid suppressor cells and predict immunotherapy resistance in melanoma. J Clin Invest. 2018 Dec 3;128(12):5505-5516. doi: 10.1172/JCI98060. Epub 2018 Nov 5.

ReferenceCitation: Bronte V, Brandau S, Chen SH, Colombo MP, Frey AB, Greten TF, Mandruzzato S, Murray PJ, Ochoa A, Ostrand-Rosenberg S, Rodriguez PC, Sica A, Umansky V, Vonderheide RH, Gabrilovich DI. Recommendations for myeloid-derived suppressor cell nomenclature and characterization standards. Nat Commun. 2016 Jul 6;7:12150. doi: 10.1038/ncomms12150.

ReferenceCitation: De Henau O, Rausch M, Winkler D, Campesato LF, Liu C, Cymerman DH, Budhu S, Ghosh A, Pink M, Tchaicha J, Douglas M, Tibbitts T, Sharma S, Proctor J, Kosmider N, White K, Stern H, Soglia J, Adams J, Palombella VJ, McGovern K, Kutok JL, Wolchok JD, Merghoub T. Overcoming resistance to checkpoint blockade therapy by targeting PI3Kgamma in myeloid cells. Nature. 2016 Nov 17;539(7629):443-447. doi: 10.1038/nature20554. Epub 2016 Nov 9.

ReferenceCitation: Welters MJ, van der Sluis TC, van Meir H, Loof NM, van Ham VJ, van Duikeren S, Santegoets SJ, Arens R, de Kam ML, Cohen AF, van Poelgeest MI, Kenter GG, Kroep JR, Burggraaf J, Melief CJ, van der Burg SH. Vaccination during myeloid cell depletion by cancer chemotherapy fosters robust T cell responses. Sci Transl Med. 2016 Apr 13;8(334):334ra52. doi: 10.1126/scitranslmed.aad8307.

ReferenceCitation: Crunkhorn S. Cancer: New path to improving immunotherapy. Nat Rev Drug Discov. 2018 Mar;17(3):164. doi: 10.1038/nrd.2018.22. Epub 2018 Feb 16.

lead_sponsor: Institut du Cancer de Montpellier - Val d'Aurelle

location_country: Italy France Norway Germany Egypt

location_facility: Fondazione IRCCS ISTITUTO NAZIONALE TUMORI

intervention_type: Diagnostic Test


title: Daratumumab as a Treatment for Adult Immune Thrombocytopenia (The DART Study)

summary: A multicenter clinical, open-label total dose-escalating phase II study with safety run-in to explore the clinical activity, total dosage, and safety of daratumumab in adult ITP patients who have not responded adequately or relapsed after corticosteroids and at least one second-line therapy including rituximab and/or TPO-RA.

study_start_date_type: Actual

status: Recruiting

study_completion_date_type: Anticipated

study_type: Interventional

primary_purpose: Treatment

enrollment_type: Anticipated

ipd_sharing: No

lead_sponsor: Ostfold Hospital Trust

location_country: Norway Denmark France

location_facility:
Ostfold Hospital Trust
Akershus University Hospital
Henri Mondor University Hospital
Oslo University Hospital
Odense University Hospital
Haukeland University Hospital

intervention_type: Drug

lead_sponsor_type: academique

status_simplified: Ongoing


title: Open-label, Single-arm, Multicenter Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients From 6 Months to Less Than 2 Years Old

summary:
Cystadrops® is currently indicated in adults and children from 2 years of age diagnosed with cystinosis with corneal crystal accumulation observed.

However administration of Cystadrops® in patients below 2 years old could be of value for these patients and prevent the crystal deposit. It is the reason why as part of the Cystadrops® pediatric investigational plan (PIP), RECORDATI Rare Diseases committed to conduct a clinical study to assess Cystadrops® safety and efficacy in the pediatric population from 6 months to less than 2 years old.

lead_sponsor: Recordati Rare Diseases

location_country:
United Kingdom
Italy
France
Belgium
Germany

location_facility:
Great Ormond Street Hospital
Hôpital Necker-Enfants Malades
Manchester Royal Eye Hospital
UZ Leuven
Bambin Gesù Hospital in Palidoro
Klinik für Pädiatrische Nieren

lead_sponsor_type: industriel


title: Multicenter Validation of 3D-QCA Based FFR vs. Pressure Wire Based FFR: The FAST-II Study

acronym: FASTII

summary: The Multicenter FAST (Fast Assessment of STenosis severity) study is a prospective observational multicenter study designed to evaluate the diagnostic accuracy of offline 3D-QCA based FFR, using CAAS Workstation (Pie Medical Imaging, Maastricht, the Netherlands) in identifying hemodynamically significant coronary artery disease with pressure wire-based FFR (≤0.80) as the reference standard.

status: Completed

study_completion_date_type: Actual

study_type: Observational

time_perspective: Prospective

enrollment_type: Actual

ReferenceType: derived

ReferenceCitation: Masdjedi K, Tanaka N, Van Belle E, Porouchani S, Linke A, Woitek FJ, Bartorelli AL, Ali ZA, den Dekker WK, Wilschut J, Diletti R, Zijlstra F, Boersma E, Van Mieghem NM, Spitzer E, Daemen J. Vessel fractional flow reserve (vFFR) for the assessment of stenosis severity: the FAST II study. EuroIntervention. 2022 Apr 22;17(18):1498-1505. doi: 10.4244/EIJ-D-21-00471.

ipd_sharing_description: Individual participant data are needed for the final analysis which will be done by Erasmus MC.

lead_sponsor: Erasmus Medical Center

location_country:
Italy
Netherlands
United States
France
Japan
Germany

location_facility:
Columbia University Medical Center
CHU
Erasmus Medical Center
Tokyo Medical University
Herzzentrum Dresden
Centro Cardiologico Monzino

status_simplified: Completed


title: Prospective Registry on User Experience With The Mapping System For Ablation Procedures

acronym: TRUE-HD

summary: This is an observational, prospective, non-randomized, multicenter, post approval study being conducted in the United States, Europe and Asia-Pacific Regions.

lead_sponsor: Boston Scientific Corporation

location_country:
Hong Kong
Spain
United Kingdom
Australia
Netherlands
United States
France
Germany

location_facility: Strong Memorial Hospital of the University of Rochester

intervention_type: Device


type: EudraCT Number

title: A Phase 3, Randomized, Active Controlled Study to Evaluate Maintenance of Response, Safety and Patient Reported Outcomes in Acromegaly Patients Treated With Octreotide Capsules vs. Parenteral Somatostatin Receptor Ligands

acronym: MPOWERED

summary:
Octreotide capsule is a novel, orally-administered formulation of the commercially-available injectable drug octreotide. In a recent phase 3 trial (OPTMAL; NCT03252353), oral octreotide capsules demonstrated sustained biochemical response up to 13 months in patients with acromegaly previously managed with somatostatin analog injections (ref).

The objective of this study was to compare the efficacy, safety, and patient reported outcomes (PROs) between oral octreotide capsules and injectable somatostatin receptor ligands (SRLs).

design_allocation: Randomized

ReferenceType: background

ReferenceCitation: Melmed S, Popovic V, Bidlingmaier M, Mercado M, van der Lely AJ, Biermasz N, Bolanowski M, Coculescu M, Schopohl J, Racz K, Glaser B, Goth M, Greenman Y, Trainer P, Mezosi E, Shimon I, Giustina A, Korbonits M, Bronstein MD, Kleinberg D, Teichman S, Gliko-Kabir I, Mamluk R, Haviv A, Strasburger C. Safety and efficacy of oral octreotide in acromegaly: results of a multicenter phase III trial. J Clin Endocrinol Metab. 2015 Apr;100(4):1699-708. doi: 10.1210/jc.2014-4113. Epub 2015 Feb 9. Erratum in: J Clin Endocrinol Metab. 2016 Oct;101(10 ):3863. J Clin Endocrinol Metab. 2020 Dec 1;105(12):.

ReferenceCitation: Melmed S. New therapeutic agents for acromegaly. Nat Rev Endocrinol. 2016 Feb;12(2):90-8. doi: 10.1038/nrendo.2015.196. Epub 2015 Nov 27. Review.

ReferenceCitation: Fleseriu M, Dreval A, Bondar I, Vagapova G, Macut D, Pokramovich YG, Molitch ME, Leonova N, Raverot G, Grineva E, Poteshkin YE, Gilgun-Sherki Y, Ludlam WH, Patou G, Haviv A, Gordon MB, Biermasz NR, Melmed S, Strasburger CJ. Maintenance of response to oral octreotide compared with injectable somatostatin receptor ligands in patients with acromegaly: a phase 3, multicentre, randomised controlled trial. Lancet Diabetes Endocrinol. 2022 Feb;10(2):102-111. doi: 10.1016/S2213-8587(21)00296-5. Epub 2021 Dec 22. Erratum in: Lancet Diabetes Endocrinol. 2022 Mar;10(3):e3.

lead_sponsor: Chiasma, Inc.

location_country:
Poland
Serbia
Spain
Italy
Austria
Russian Federation
United Kingdom
Lithuania
Hungary
United States
France
Romania
Germany

location_facility: Federal State Budgetary Institution "V. A. Almazov Federal North-West Medical Research Centre" of the Ministry of Health of the Russian Federation

location_facility: John H. Stroger, Jr. Hospital of Cook County

location_facility: University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital

location_facility: Washington University School of Medicine

location_facility: Katedra i Klinika Endokrynologii i Chorob Wewnetrznych Gdanski Uniwersytet Medyczny

location_facility: Regional State Budgetary Healthcare Institution Regional State Hospital

location_facility: Universitätsklinik für Innere Medizin Klinische Abteilung für Endokrinologie und Diabetologie

location_facility: Novosibirsk State Regional Clinical Hospital

location_facility: Szegedi Tudományegyetem, I. Belgyógyászati Klinika

location_facility: Vladimirsky Moscow Regional Research Clinical Institute

location_facility: "C.I. Parhon" National Institute of Endocrinology I Clinical Endocrinology Department - Endemic goitier and its complications

location_facility: Hospital Universitari Germans Trias i Pujol

location_facility: Central Manchester University Hospitals NHS Foundation Trust, Manchester Royal Infirmary

location_facility: Fondazione Policlinico Universitario A. Gemelli Università Cattolica del S.Cuore S.C. Endocrinologia e Malattie del Metabolismo

location_facility: Università di Pisa Dipartimento di Medicina Clinica e Sperimentale

location_facility: Campus Del Hospital Universitario Virgen del Rocio

location_facility: The Ohio State University Wexner Medical Center

location_facility: Hospital General Universitario Gregorio Maranon

location_facility: Policlinico di Monserrato U.O.C. Endocrinologia e Diabetologia

location_facility: Interregional Clinical Diagnostic Center

location_facility: Complejo Hospitalario Universitario de Santiago de Compostela

location_facility: Keck Medical Center of University of Southern California

location_facility: Antrium Multidisciplinary Medical Clinic

location_facility: Sechenov Moscow First State Medical University

location_facility: Rutgers - Robert Wood Johnson Medical School

location_facility: Samodzielny Publiczny Szpital Kliniczny nr 1 we Wroclawiu, Klinika Endokrynologii, Diabetologii i Leczenia Izotopami

location_facility: Clinical Centre of Serbia, Clinic for Endocrinology Diabetes and Metabolic Diseases

location_facility: Praxis für Endokrinologie und Diabetologie Dr M Droste


title: A Phase 3, Randomized, Double-Blind Study Comparing Upadacitinib (ABT-494) to Placebo in Subjects With Moderately to Severely Active Rheumatoid Arthritis Who Are on a Stable Dose of Conventional Synthetic Disease-Modifying Anti-Rheumatic Drugs (csDMARDs) and Have an Inadequate Response to csDMARDs

acronym: SELECT-NEXT

summary: The primary objectives of this study are to compare the efficacy, safety, and tolerability of upadacitinib 30 mg once daily (QD) and 15 mg QD versus placebo for the treatment of signs and symptoms of adults with moderately to severely active rheumatoid arthritis who were on a stable dose of csDMARDs and had an inadequate response to csDMARDs.

ReferenceCitation: Burmester GR, Kremer JM, Van den Bosch F, Kivitz A, Bessette L, Li Y, Zhou Y, Othman AA, Pangan AL, Camp HS. Safety and efficacy of upadacitinib in patients with rheumatoid arthritis and inadequate response to conventional synthetic disease-modifying anti-rheumatic drugs (SELECT-NEXT): a randomised, double-blind, placebo-controlled phase 3 trial. Lancet. 2018 Jun 23;391(10139):2503-2512. doi: 10.1016/S0140-6736(18)31115-2. Epub 2018 Jun 18.

ReferenceCitation: Bergman M, Buch MH, Tanaka Y, Citera G, Bahlas S, Wong E, Song Y, Zueger P, Ali M, Strand V. Routine Assessment of Patient Index Data 3 (RAPID3) in Patients with Rheumatoid Arthritis Treated with Long-Term Upadacitinib Therapy in Five Randomized Controlled Trials. Rheumatol Ther. 2022 Dec;9(6):1517-1529. doi: 10.1007/s40744-022-00483-4. Epub 2022 Sep 20.

ReferenceCitation: Bergman M, Tundia N, Yang M, Orvis E, Clewell J, Bensimon A. Economic Benefit from Improvements in Quality of Life with Upadacitinib: Comparisons with Tofacitinib and Methotrexate in Patients with Rheumatoid Arthritis. Adv Ther. 2021 Dec;38(12):5649-5661. doi: 10.1007/s12325-021-01930-4. Epub 2021 Oct 12.

ReferenceCitation: Yamaoka K, Tanaka Y, Kameda H, Khan N, Sasaki N, Harigai M, Song Y, Zhang Y, Takeuchi T. The Safety Profile of Upadacitinib in Patients with Rheumatoid Arthritis in Japan. Drug Saf. 2021 Jun;44(6):711-722. doi: 10.1007/s40264-021-01067-x. Epub 2021 May 27.

ReferenceCitation: Cohen SB, van Vollenhoven RF, Winthrop KL, Zerbini CAF, Tanaka Y, Bessette L, Zhang Y, Khan N, Hendrickson B, Enejosa JV, Burmester GR. Safety profile of upadacitinib in rheumatoid arthritis: integrated analysis from the SELECT phase III clinical programme. Ann Rheum Dis. 2021 Mar;80(3):304-311. doi: 10.1136/annrheumdis-2020-218510. Epub 2020 Oct 28. Erratum in: Ann Rheum Dis. 2021 May;80(5):e83.

ReferenceCitation: Strand V, Pope J, Tundia N, Friedman A, Camp HS, Pangan A, Ganguli A, Fuldeore M, Goldschmidt D, Schiff M. Upadacitinib improves patient-reported outcomes in patients with rheumatoid arthritis and inadequate response to conventional synthetic disease-modifying antirheumatic drugs: results from SELECT-NEXT. Arthritis Res Ther. 2019 Dec 9;21(1):272. doi: 10.1186/s13075-019-2037-1. Erratum in: Arthritis Res Ther. 2020 Jun 9;22(1):137.

ReferenceCitation: Nader A, Mohamed MF, Winzenborg I, Doelger E, Noertersheuser P, Pangan AL, Othman AA. Exposure-Response Analyses of Upadacitinib Efficacy and Safety in Phase II and III Studies to Support Benefit-Risk Assessment in Rheumatoid Arthritis. Clin Pharmacol Ther. 2020 Apr;107(4):994-1003. doi: 10.1002/cpt.1671. Epub 2019 Nov 30.

ipd_sharing: Yes

ipd_sharing_description: AbbVie is committed to responsible data sharing regarding the clinical trials we sponsor. This includes access to anonymized, individual and trial-level data (analysis data sets), as well as other information (e.g., protocols and clinical study reports), as long as the trials are not part of an ongoing or planned regulatory submission. This includes requests for clinical trial data for unlicensed products and indications.

lead_sponsor: AbbVie

location_country:
Croatia
Spain
Italy
United Kingdom
Lithuania
Ukraine
Kazakhstan
United States
Belgium
Romania
South Africa
Czechia
Poland
Hong Kong
Portugal
Austria
Australia
Russian Federation
Argentina
Puerto Rico
Turkey
Mexico
Slovakia
Bosnia and Herzegovina
Canada
Switzerland
Hungary
Germany
Bulgaria
Taiwan
Greece
Ireland
Estonia
Latvia
Finland
New Zealand
Korea, Republic of
France

location_facility: Inland Rheum Clin Trials Inc. /ID# 138853

location_facility: National Cheng Kung University Hospital /ID# 140868

location_facility: Universita di Catanzaro Magna Graecia /ID# 139316

location_facility: Hanyang University Seoul Hospi /ID# 138655

location_facility: University General Hospital of Heraklion "PA.G.N.I" /ID# 139115

location_facility: Pacific Arthritis Ctr Med Grp /ID# 138744

location_facility: University of Arizona Cancer Center - North Campus /ID# 140451

location_facility: AZ Arthritis & Rheuma Research /ID# 138598

location_facility: Spitalul Clinic Judetean de Ur /ID# 138393

location_facility: Kaohsiung Medical University Chung-Ho Memorial Hospital /ID# 140869

location_facility: University Clinical Centre of the Republic of Srpska /ID# 140372

location_facility: Comprehensive Arthritis Care, a division of Comprehensive Rheumatology Care PLLC /ID# 141021

location_facility: Taipei Veterans General Hosp /ID# 139234

location_facility: SunValley Arthritis Center, Lt /ID# 140452

location_facility: Unidad de Investigacion de las Enfermedades Reumatologicas SA de CV /ID# 138841

location_facility: Warrington + Halton Hosp NHS /ID# 139195

location_facility: Ctr Arthritis & Rheumatic Dise /ID# 141696

location_facility: Slovak research center Team Member, Thermium s.r.o. /ID# 139924

location_facility: Houston Institute for Clin Res /ID# 138716

location_facility: Revita Reumatologiai Rendelo /ID# 140761

location_facility: China Medical University Hosp /ID# 139232

location_facility: Helsinki Univ Central Hospital /ID# 140381

location_facility: South Karelia Central Hospital /ID# 139973

location_facility: Diag Consult Ctr 17 Sofia EOOD /ID# 141006

location_facility: Centro Peninsular de Investigación Clínica SCP /ID# 148160

location_facility: Rheuma Zentrum Favoriten GmbH /ID# 138787

location_facility: Inst. Rheumatologic Strusberg /ID# 145648

location_facility: Aurora Rheumatology and Immunotherapy Center /ID# 139306

location_facility: Chungnam National University Hospital /ID# 138653

location_facility: University Clinical Centre of the Republic of Srpska /ID# 138819

location_facility: Republican Clin Hos n.a. Baran /ID# 139273

location_facility: East Tallinn Central Hospital /ID# 140618

location_facility: Physician Res. Collaboration /ID# 138533

location_facility: Spitalul Clinic Judetean de Urgenta /ID# 138407

location_facility: Optimus Clinical Research Pty. /ID# 138769

location_facility: Center of Clinical and Basic Research /ID# 141116

location_facility: MNCE "Lviv City Clinical Hospital #4" /ID# 139873

location_facility: JSC Nat Scientific Med Res Ctr /ID# 140575

location_facility: AZ Arthritis and Rheum Researc /ID# 139286

location_facility: The Center for Rheumatology & /ID# 139203

location_facility: Articularis Healthcare Group, Inc d/b/a Low Country Rheumatology /ID# 138689

location_facility: Arth and Osteo Clin Brazo Valley /ID# 147809

location_facility: Diagnostic Consultative Center /ID# 138882

location_facility: Narodny ustav reumatickych chorob Piestany /ID# 138427

location_facility: Chonnam National University Hospital /ID# 138651

location_facility: Odessa National Medical Univ /ID# 139179

location_facility: Four Rivers Clinical Research /ID# 141134

location_facility: Cincinnati Rheumatic Disease Study Group, Inc. /ID# 138868

location_facility: Clinical Investigation Special /ID# 139696

location_facility: Instituto Portugues De Reumatologia /ID# 148315

location_facility: School of Medicine University of Puerto Rico-Medical Sciences Campus /ID# 139328

location_facility: Arthritis Clinical Research Tr /ID# 138945

location_facility: Deerbrook Medical Associates /ID# 139694

location_facility: Hospital General Univ de Elche /ID# 138991

location_facility: Ankara Numune Training and Res /ID# 139237

location_facility: Bluegrass Community Research /ID# 138295

location_facility: Kazan State Medical University /ID# 138413

location_facility: Fejer Megyei Szent Gyorgy Korh /ID# 138554

location_facility: Charité Universitätsmedizin Campus Mitte /ID# 139052

location_facility: AZ Arthritis and Rheum Researc /ID# 138500

location_facility: St Vincent's University Hosp /ID# 138562

location_facility: Klinicki bolnicki centar Rijeka /ID# 138649

location_facility: Voronezh State Medical Univers /ID# 148431

location_facility: Mautalen Salud e Investigacion /ID# 141419

location_facility: Winelands Medical Research Ctr /ID# 138944

location_facility: Klaipeda University Hospital /ID# 141416

location_facility: Accurate Clinical Management /ID# 139338

location_facility: Centro Hospitalar Lisboa Ocidental, EPE /ID# 140594

location_facility: Southampton General Hospital /ID# 139169

location_facility: Medallion Clinical Research Institute, LLC /ID# 140074

location_facility: Far Eastern Memorial Hospital /ID# 140871

location_facility: Trinity Universal Research Association /ID# 148649

location_facility: HFR Fribourg - Hopital Canton /ID# 139155

location_facility: PMG Research of Wilmington LLC /ID# 140951

location_facility: Ulyanovsk Regional Clin Hosp /ID# 139279

location_facility: Wilhelminenspital der Stadt Wien /ID# 138788

location_facility: Arthritis & Osteoporosis Clinic /ID# 138703

location_facility: AZ Arthritis and Rheum Assoc /ID# 148651

location_facility: Groupe de Recherche en Maladies Osseuses /ID# 138906

location_facility: Daegu Catholic University Med /ID# 139249

location_facility: Hosp Nuestra Senora Esperanza /ID# 138997

location_facility: Western Washington Arthritis C /ID# 138728

location_facility: Seoul National University Hospital /ID# 138659

location_facility: Samara Regional Clinical Hosp /ID# 148642

location_facility: Yaroslavi State Medical Univer /ID# 139908

location_facility: NZOZ Centrum Reumatologiczne /ID# 138353

location_facility: CHRU Tours - Hopital Trousseau /ID# 138969

location_facility: Cath Univ Seoul St Mary's Hosp /ID# 138652

location_facility: St. Augustine's Medical Centre /ID# 141352

location_facility: Hospital Clin Univ San Carlos /ID# 138993

location_facility: Clinical Res of West FL, Inc. /ID# 138854

location_facility: Arizona Research Center, Inc. /ID# 140448


title: EUS-guided Fine Needle Tissue Acquisition Using a Newly Developed Nitinol Ultra Flex 19 Gauge Needle for Transduodenal Lesions: a Multicenter Prospective Feasibility Study

acronym: NIFLEX

summary: This study evaluates the feasibility, safety and accuracy of a 19 gauge (19G) needle in nitinol in the performance of endoscopic ultrasound (EUS) guided transduodenal biopsy for the acquisition of samples for histologic analysis. Patients with lesions that can be approached only from the duodenum will be prospectively enrolled.

primary_purpose: Diagnostic

ReferenceType: result

ReferenceCitation: Varadarajulu S, Bang JY, Hebert-Magee S. Assessment of the technical performance of the flexible 19-gauge EUS-FNA needle. Gastrointest Endosc. 2012 Aug;76(2):336-43. doi: 10.1016/j.gie.2012.04.455.

lead_sponsor: Catholic University of the Sacred Heart

location_country:
Japan
United States
Italy
France

location_facility: Teikyo University Mizonokuchi Hospital, Departement of Gastroenterology

location_facility: Digestive Endoscopy Unit, Universita' Cattolica del Sacro Cuore

location_facility: Indiana Univerisity Health Medical Center


title: A Phase 1/1b, Open Label, Multiple Dose, Dose Escalation and Expansion Study to Investigate the Safety, Pharmacokinetics and Antitumor Activity of the Anti-PD-1 Monoclonal Antibody BGB-A317 in Combination With the PARP Inhibitor BGB-290 in Subjects With Advanced Solid Tumors

summary: This trial studied the safety, pharmacokinetics, and antitumor activity of the anti-programmed cell death 1 (PD-1) monoclonal antibody (mAb) BGB-A317 (tislelizumab) in combination with the poly(adenosine diphosphate ribose) polymerase (PARP) inhibitor BGB-290 (pamiparib) in participants with advanced solid tumors.

design_allocation: Non-Randomized

ReferenceCitation: Friedlander M, Meniawy T, Markman B, Mileshkin L, Harnett P, Millward M, Lundy J, Freimund A, Norris C, Mu S, Wu J, Paton V, Gao B. Pamiparib in combination with tislelizumab in patients with advanced solid tumours: results from the dose-escalation stage of a multicentre, open-label, phase 1a/b trial. Lancet Oncol. 2019 Sep;20(9):1306-1315. doi: 10.1016/S1470-2045(19)30396-1. Epub 2019 Aug 1.

lead_sponsor: BeiGene

location_country:
Spain
United Kingdom
New Zealand
Australia
United States
France

location_facility: Hospital Clínico Universitario de Valencia

intervention_type: Biological


type: Other Identifier

title: An Open-Label, Multicenter, Dose Escalation and Expansion Phase Ib Study to Evaluate the Safety, Pharmacokinetics, and Therapeutic Activity of RO6958688 in Combination With Atezolizumab in Patients With Locally Advanced and/or Metastatic CEA-Positive Solid Tumors

summary: This is an open-label, multicenter, dose-escalation and expansion Phase Ib clinical study of RO6958688 in combination with atezolizumab. Part I of the study is subdivided into parts IA and IB. Part IA is dose escalation with a starting dose of 5 mg of RO6958688 given QW (once a week) and a fixed, flat dose of 1200 mg given Q3W (every 3 weeks) of atezolizumab, to evaluate the safety and determine the MTD of RO6958688 in combination with atezolizumab. Part IB is a dose/schedule finding part that will explore different administration schedules of RO6958688 in combination with atezolizumab (1200 mg Q3W) to establish the appropriate dose/schedule of RO6958688 in combination with atezolizumab.

ReferenceCitation: Martinez-Sabadell A, Morancho B, Rius Ruiz I, Roman Alonso M, Ovejero Romero P, Escorihuela M, Chicote I, Palmer HG, Nonell L, Alemany-Chavarria M, Klein C, Bacac M, Arribas J, Arenas EJ. The target antigen determines the mechanism of acquired resistance to T cell-based therapies. Cell Rep. 2022 Oct 18;41(3):111430. doi: 10.1016/j.celrep.2022.111430.

lead_sponsor: Hoffmann-La Roche

location_country:
Denmark
Spain
Italy
Canada
United States
France
Netherlands

location_facility: Antoni Van Leeuwenhoek Ziekenhuis; Gastro-Enterologie

location_facility: Smilow Cancer Hospital at Yale- New Haven Oncology Investigational Drug Pharmacy

location_facility: Hospital Univ Vall d'Hebron; Servicio de Oncologia

location_facility: Clinica Universitaria de Navarra; Servicio de Oncologia

location_facility: IRCCS IST. Tumori Fondaz. Pascale; S.C. Oncologia Medica,Melanoma,Immunoterapia E Terapie Innovative

location_facility: Centre Leon Berard; Departement Oncologie Medicale

location_facility: Azienda Ospedaliera Universitaria Senese, U.O.C. Immunoterapia Oncologica

location_facility: Hospital Universitario 12 de Octubre; Servicio de Oncologia

location_facility: START Madrid. Centro Integral Oncologico Clara Campal; CIOCC

location_facility: START Madrid-FJD, Hospital Fundacion Jimenez Diaz


ipd_sharing: Undecided

status: Active, not recruiting

acronym: RainbowExt

title: RAINBOW Extension Study: an Extension Study to Evaluate the Long Term Efficacy and Safety of RAnibizumab Compared With Laser Therapy for the Treatment of INfants BOrn Prematurely With Retinopathy of Prematurity

lead_sponsor: Novartis Pharmaceuticals

source: euctr

ReferenceCitation: Marlow N, Stahl A, Lepore D, Fielder A, Reynolds JD, Zhu Q, Weisberger A, Stiehl DP, Fleck B; RAINBOW investigators group. 2-year outcomes of ranibizumab versus laser therapy for the treatment of very low birthweight infants with retinopathy of prematurity (RAINBOW extension study): prospective follow-up of an open label, randomised controlled trial. Lancet Child Adolesc Health. 2021 Oct;5(10):698-707. doi: 10.1016/S2352-4642(21)00195-4. Epub 2021 Aug 13.

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

location_country:
Croatia
Saudi Arabia
Italy
United Kingdom
Lithuania
United States
Romania
Belgium
Japan
Czechia
Austria
Russian Federation
India
Turkey
Malaysia
Slovakia
Egypt
Hungary
Germany
Denmark
Taiwan
Greece
Estonia
France

summary: The purpose of this study is to evaluate the long term efficacy and safety of intravitreal ranibizumab compared with laser ablation therapy in patients who were treated for retinopathy of prematurity (ROP) in the core study CRFB002H2301


acronym: SCOUT

title: A Phase 1/2 Study of the Oral TRK Inhibitor Larotrectinib in Pediatric Patients With Advanced Solid or Primary Central Nervous System Tumors

lead_sponsor: Bayer

ReferenceCitation: Doz F, van Tilburg CM, Geoerger B, Højgaard M, Øra I, Boni V, Capra M, Chisholm J, Chung HC, DuBois SG, Gallego-Melcon S, Gerber NU, Goto H, Grilley-Olson JE, Hansford JR, Hong DS, Italiano A, Kang HJ, Nysom K, Thorwarth A, Stefanowicz J, Tahara M, Ziegler DS, Gavrilovic IT, Norenberg R, Dima L, De La Cuesta E, Laetsch TW, Drilon A, Perreault S. Efficacy and safety of larotrectinib in TRK fusion-positive primary central nervous system tumors. Neuro Oncol. 2022 Jun 1;24(6):997-1007. doi: 10.1093/neuonc/noab274.

ReferenceCitation: Bebb DG, Banerji S, Blais N, Desmeules P, Gill S, Grin A, Feilotter H, Hansen AR, Hyrcza M, Krzyzanowska M, Melosky B, Noujaim J, Purgina B, Ruether D, Simmons CE, Soulieres D, Torlakovic EE, Tsao MS. Canadian Consensus for Biomarker Testing and Treatment of TRK Fusion Cancer in Adults. Curr Oncol. 2021 Jan 15;28(1):523-548. doi: 10.3390/curroncol28010053.

ReferenceCitation: Perreault S, Chami R, Deyell RJ, El Demellawy D, Ellezam B, Jabado N, Morgenstern DA, Narendran A, Sorensen PHB, Wasserman JD, Yip S. Canadian Consensus for Biomarker Testing and Treatment of TRK Fusion Cancer in Pediatric Patients. Curr Oncol. 2021 Jan 9;28(1):346-366. doi: 10.3390/curroncol28010038.

ReferenceCitation: Bielack SS, Cox MC, Nathrath M, Apel K, Blattmann C, Holl T, Jenewein R, Klenk U, Klothaki P, Müller-Abt P, Ortega-Lawerenz S, Reynolds M, Scheer M, Simon-Klingenstein K, Stegmaier S, Tupper R, Vokuhl C, von Kalle T. Rapid, complete and sustained tumour response to the TRK inhibitor larotrectinib in an infant with recurrent, chemotherapy-refractory infantile fibrosarcoma carrying the characteristic ETV6-NTRK3 gene fusion. Ann Oncol. 2019 Nov;30 Suppl 8:viii31-viii35. doi: 10.1093/annonc/mdz382. Epub 2019 Dec 24.

ReferenceCitation: Hong DS, DuBois SG, Kummar S, Farago AF, Albert CM, Rohrberg KS, van Tilburg CM, Nagasubramanian R, Berlin JD, Federman N, Mascarenhas L, Geoerger B, Dowlati A, Pappo AS, Bielack S, Doz F, McDermott R, Patel JD, Schilder RJ, Tahara M, Pfister SM, Witt O, Ladanyi M, Rudzinski ER, Nanda S, Childs BH, Laetsch TW, Hyman DM, Drilon A. Larotrectinib in patients with TRK fusion-positive solid tumours: a pooled analysis of three phase 1/2 clinical trials. Lancet Oncol. 2020 Apr;21(4):531-540. doi: 10.1016/S1470-2045(19)30856-3. Epub 2020 Feb 24.

ReferenceCitation: Bielack SS, Cox MC, Nathrath M, Apel K, Blattmann C, Holl T, Jenewein R, Klenk U, Klothaki P, Müller-Abt P, Ortega-Lawerenz S, Reynolds M, Scheer M, Simon-Klingenstein K, Stegmaier S, Tupper R, Vokuhl C, von Kalle T. Rapid, complete and sustained tumour response to the TRK inhibitor larotrectinib in an infant with recurrent, chemotherapy-refractory infantile fibrosarcoma carrying the characteristic ETV6-NTRK3 gene fusion. Ann Oncol. 2019 Nov 1;30(Suppl_8):viii31-viii35. doi: 10.1093/annonc/mdz382.

ReferenceCitation: DuBois SG, Laetsch TW, Federman N, Turpin BK, Albert CM, Nagasubramanian R, Anderson ME, Davis JL, Qamoos HE, Reynolds ME, Cruickshank S, Cox MC, Hawkins DS, Mascarenhas L, Pappo AS. The use of neoadjuvant larotrectinib in the management of children with locally advanced TRK fusion sarcomas. Cancer. 2018 Nov 1;124(21):4241-4247. doi: 10.1002/cncr.31701. Epub 2018 Sep 11.

ReferenceCitation: Laetsch TW, DuBois SG, Mascarenhas L, Turpin B, Federman N, Albert CM, Nagasubramanian R, Davis JL, Rudzinski E, Feraco AM, Tuch BB, Ebata KT, Reynolds M, Smith S, Cruickshank S, Cox MC, Pappo AS, Hawkins DS. Larotrectinib for paediatric solid tumours harbouring NTRK gene fusions: phase 1 results from a multicentre, open-label, phase 1/2 study. Lancet Oncol. 2018 May;19(5):705-714. doi: 10.1016/S1470-2045(18)30119-0. Epub 2018 Mar 29. Erratum in: Lancet Oncol. 2018 May;19(5):e229.

ReferenceCitation: Drilon A, Laetsch TW, Kummar S, DuBois SG, Lassen UN, Demetri GD, Nathenson M, Doebele RC, Farago AF, Pappo AS, Turpin B, Dowlati A, Brose MS, Mascarenhas L, Federman N, Berlin J, El-Deiry WS, Baik C, Deeken J, Boni V, Nagasubramanian R, Taylor M, Rudzinski ER, Meric-Bernstam F, Sohal DPS, Ma PC, Raez LE, Hechtman JF, Benayed R, Ladanyi M, Tuch BB, Ebata K, Cruickshank S, Ku NC, Cox MC, Hawkins DS, Hong DS, Hyman DM. Efficacy of Larotrectinib in TRK Fusion-Positive Cancers in Adults and Children. N Engl J Med. 2018 Feb 22;378(8):731-739. doi: 10.1056/NEJMoa1714448.

ipd_sharing_description:
Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.

Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.

location_country:
Spain
Italy
United Kingdom
Ukraine
United States
Japan
Czechia
Poland
Russian Federation
Australia
Israel
Turkey
Sweden
Canada
Switzerland
China
Germany
Denmark
Ireland
Korea, Republic of
France
Netherlands

location_facility: The Hospital for Sick Children (SickKids)

location_facility: Karolinska Universitetssjukhuset i Solna

location_facility: Rigshospitalet, Dept Pediatrics & Adelescent Med.

location_facility: Morozov Children's City Clinical Hospital

location_facility: Lucille Salter Packard Children's Hospital at Stanford

location_facility: Severance Hospital, Yonsei University Health System

location_facility: UCLA Jonsson Comprehensive Cancer Center

location_facility: Center of Pediatric Hematology, Oncology and Immunology

location_facility: Ciutat Sanitària i Universitaria de la Vall d'Hebron

location_facility: Beijing Children's Hospital, Capital Medical University

location_facility: Osaka City Hospital Organization Osaka City General Hospital

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori

location_facility: Western Ukrainian Specialized Pediatric Medical Centre

location_facility: Cincinnati Children's Hospital and Medical Center

location_facility: Clalit Health Services Schneider Children's Medical Center

location_facility: Istanbul Universitesi Istanbul Tip Fakultesi

location_facility: University of Texas Southwestern Medical Center

location_facility: Tianjin Medical University Cancer Institiute & Hospital

summary:
The study is being done to test the safety of a cancer drug called larotrectinib in children. The cancer must have a change in a particular gene (NTRK1, NTRK2 or NTRK3). Larotrectinib blocks the actions of these NTRK genes in cancer cells and can therefore be used to treat cancer.

The first study part (Phase 1) is done to determine what dose level of larotrectinib is safe for children, how the drug is absorbed and changed by their bodies and how well the cancer responds to the drug. The main purpose of the second study part (Phase 2) is to investigate how well and how long different cancer types respond to the treatment with larotrectininb.


title: Multinational, Observational, Retrospective Study to Evaluate Coagulation Changes and the Influence of Antithrombin III Treatment in Patients With Severe COVID-19 Infection

summary: Multicentre, multinational, non-interventional, observational, retrospective, patient record study to evaluate changes in coagulation parameters in patients with severe COVID-19 infection receiving/not treatment with antithrombin (AT) III

time_perspective: Retrospective

lead_sponsor: Octapharma

location_country: Austria Germany France Sweden

location_facility:
ECMO Centre Karolinska
Octapharma Research Site


title: Study of CNGB1 Retinitis Pigmentosa and Allied Hereditary Disorders

summary: Mutations in the rod-expressed gene, cyclic nucleotide-gated channel beta subunit (CNGB1) and associated inborn errors in metabolism are causes of retinal disease that causes progressive loss of vision. Retinitis pigmentosa (RP) is a major cause of untreatable blindness associated with CNGB1 (CNGB1-RP). RP involves the death of photoreceptor cells that can be caused by mutations in a number of different genes. Treatment by gene therapy could prevent blindness in cases of inherited retinal dystrophies including RP. In the future RP due to mutations in CNGB1 may be treatable by gene therapy since this form of photoreceptor degeneration involves a slow loss of rod photoreceptor cells. This provides a wide window of opportunity for the identification of patients and initiation of treatment. Our efforts are directed toward developing gene therapy as a treatment. To this end, our objective is to better understand the disease process of CNGB1-RP and other allied inherited disorders so that we can develop clinical tests to measure the outcomes of treatment.

lead_sponsor: Columbia University

location_country:
Germany
United States
United Kingdom
France

location_facility: Klinikum der Universität München University Eye Hospital, Ludwig-Maximilians-University (LMU) Munich

location_facility: Institut de la Vision/Centre de maladies rares du Centre Hospitalier National Ophtalmologique des Quinze-Vingts

location_facility: Moorfields Eye Hospital NHS Foundation Trust

intervention_type: Other


title: The CorNeat EverPatch - a First-In-Man Clinical Study for Demonstrating the Safety of a Synthetic Tissue Substitute for Concealment of Artificial Implants and Glaucoma Tube Shunts

summary:
Study Name: The CorNeat EverPatch - a First-In-Man Clinical Study for demonstrating the Safety of a Synthetic Tissue Substitute for concealment of artificial implants and glaucoma tube shunts

Objective: The objective of this clinical trial is to demonstrate the Safety of the Corneat Everpatch for concealment of artificial implants and glaucoma tube shunts

The study will consist 10 subjects requiring concealment of a glaucoma shunt or other ophthalmic implant. Eligible subjects who signs an ICF will be enrolled to the study. Subjects will be implanted with the Corneart EverPatch as part of a glaucoma shunt surgery or during a corrective surgery to repair a breached conjunctiva over an implanted device. Subjects will be monitored for a period of 12 months post-op during which follow up visits will occur at 1 week, 1, 2, 3, 6, 9 & 12 months following surgery including clinical examination of the operated eye using slit-lamp biomicroscopy and imaging using OCT or UBM (will be performed only at the 6 & 12 months follow up visits).

lead_sponsor: CorNeat Vision Ltd.

location_country: Kenya Canada France

location_facility: University Health Network - Toronto Western Hospital

location_facility: Hospital Foundation Adolphe De Rothschild


title: A Prospective, Multicenter Evaluation of the CD HORIZON® Fenestrated Screw Spinal System With Fenestrated Screw Cement When Used in the Treatment of Spinal Conditions in Subjects With Compromised Bone Quality

acronym: FNS

summary: The primary objective of this post-market study is to demonstrate that Oswestry disability index (ODI) score improved significantly at 12 months post-operatively as compared to baseline for each indication (degenerative spinal disease and deformity) in subjects with compromised bone quality, who will receive a surgical procedure requiring posterior stabilization and/or immobilization of one or more spinal segments using CD HORIZON® Fenestrated Screw Spinal System with Fenestrated Screw Cement.

status: Terminated

lead_sponsor: Medtronic Spinal and Biologics

location_country: Italy Greece Finland France Belgium

location_facility: IRCCS Istituto Clinico Humanitas di Milano

status_simplified: Unknown


title: A Randomized, Actively Controlled, Open-label, Multicenter Study of Efficacy and Safety of Evolocumab Compared With Low Density Lipoprotein Cholesterol (LDL-C) Apheresis, Followed by Single-Arm Evolocumab Administration in Subjects Receiving LDL-C Apheresis Prior to Study Enrollment

summary: To evaluate the efficacy of subcutaneous (SC) evolocumab, compared to regularly scheduled low-density lipoprotein cholesterol (LDL-C) apheresis, on reducing the need for future apheresis.

lead_sponsor: Amgen

location_country:
Spain
Italy
United Kingdom
Australia
United States
France
Germany
Czechia


title: An Interventional, Multicenter, Single Arm, Phase I/IIa Clinical Trial to Investigate the Efficacy and Safety of Allo-APZ2-EB on Epidermolysis Bullosa (EB)

lead_sponsor: RHEACELL GmbH & Co. KG

ReferenceCitation: Kiritsi D, Dieter K, Niebergall-Roth E, Fluhr S, Daniele C, Esterlechner J, Sadeghi S, Ballikaya S, Erdinger L, Schauer F, Gewert S, Laimer M, Bauer JW, Hovnanian A, Zambruno G, El Hachem M, Bourrat E, Papanikolaou M, Petrof G, Kitzmüller S, Ebens CL, Frank MH, Frank NY, Ganss C, Martinez AE, McGrath JA, Tolar J, Kluth MA. Clinical trial of ABCB5+ mesenchymal stem cells for recessive dystrophic epidermolysis bullosa. JCI Insight. 2021 Nov 22;6(22). pii: e151922. doi: 10.1172/jci.insight.151922.

ReferenceCitation: Kerstan A, Niebergall-Roth E, Esterlechner J, Schroder HM, Gasser M, Waaga-Gasser AM, Goebeler M, Rak K, Schrufer P, Endres S, Hagenbusch P, Kraft K, Dieter K, Ballikaya S, Stemler N, Sadeghi S, Tappenbeck N, Murphy GF, Orgill DP, Frank NY, Ganss C, Scharffetter-Kochanek K, Frank MH, Kluth MA. Ex vivo-expanded highly pure ABCB5+ mesenchymal stromal cells as Good Manufacturing Practice-compliant autologous advanced therapy medicinal product for clinical use: process validation and first in-human data. Cytotherapy. 2021 Feb;23(2):165-175. doi: 10.1016/j.jcyt.2020.08.012. Epub 2020 Oct 1.

location_country:
United Kingdom
Austria
United States
France
Germany

location_facility: King's College London; St John's Institute of Dermatology;

location_facility: EB-Haus Austria; Salzburger Landeskliniken (SALK); Paracelsus Medizinische Privatuniversität Salzburg (PMU)

location_facility: University of Minnesota, Masonic Cancer Center and Medical Center

location_facility: Department of Dermatology, Medical Center-University of Freiburg

location_facility: Hôpital Saint-Louis; Département de dermatologie

location_facility: Great Ormond Street Hospital; Dermatology Department

summary: The aim of this clinical trial is to investigate the efficacy (by monitoring overall improvement of EB symptoms) and safety (by monitoring adverse events) of three doses of allo-APZ2-EB administered intravenously to patients with recessive dystrophic epidermolysis bullosa (RDEB).


acronym: ARIES

title: Managing Neovascular Age-related Macular Degeneration (nAMD) Over 2 Years With a Treat and Extend (T&E) Regimen of 2 mg Intravitreal Aflibercept - a Randomized, Open-label, Active-controlled, Parallel-group Phase IV/IIIb Study (ARIES)

ReferenceCitation: Wolf S, Holz FG, Midena E, Souied EH, Lambrou G, Machewitz T, Allmeier H, Mitchell P; ARIES Study Investigators. Patients with Neovascular Age-Related Macular Degeneration Requiring Intensive Intravitreal Aflibercept Treatment: An ARIES Post Hoc Analysis. Ophthalmol Ther. 2022 Oct;11(5):1793-1803. doi: 10.1007/s40123-022-00541-8. Epub 2022 Jul 12.

ReferenceCitation: Chaudhary V, Holz FG, Wolf S, Midena E, Souied EH, Allmeier H, Lambrou G, Machewitz T, Mitchell P; ARIES study investigators. Association Between Visual Acuity and Fluid Compartments with Treat-and-Extend Intravitreal Aflibercept in Neovascular Age-Related Macular Degeneration: An ARIES Post Hoc Analysis. Ophthalmol Ther. 2022 Jun;11(3):1119-1130. doi: 10.1007/s40123-022-00491-1. Epub 2022 Mar 18.

ReferenceCitation: Tuerksever C, Somfai GM, Oesch S, Machewitz T, Hasler PW, Zweifel S. Hypothetical Switch of Anti-Vascular Endothelial Growth Factor in Neovascular Age-Related Macular Degeneration: An ARIES Post Hoc Analysis. Ophthalmol Ther. 2022 Apr;11(2):613-627. doi: 10.1007/s40123-021-00448-w. Epub 2022 Jan 23.

ReferenceCitation: Mitchell P, Holz FG, Hykin P, Midena E, Souied E, Allmeier H, Lambrou G, Schmelter T, Wolf S; ARIES study investigators. EFFICACY AND SAFETY OF INTRAVITREAL AFLIBERCEPT USING A TREAT-AND-EXTEND REGIMEN FOR NEOVASCULAR AGE-RELATED MACULAR DEGENERATION: The ARIES Study: A Randomized Clinical Trial. Retina. 2021 Sep 1;41(9):1911-1920. doi: 10.1097/IAE.0000000000003128. Erratum in: Retina. 2022 Sep 1;42(9):e43.

location_country:
Spain
Italy
United Kingdom
Australia
Canada
Hungary
France
Germany

summary: This study aims to evaluate the optimal use, efficacy, and safety of a Treat-and-Extend regimen with aflibercept in subjects with nAMD.


title: Phase I/II Trial of S 81694 Administered Intravenously in Combination With Paclitaxel to Evaluate the Safety, Pharmacokinetic and Efficacy in Metastatic Breast Cancer

lead_sponsor: Institut de Recherches Internationales Servier

ipd_sharing_description:
Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data.

Access can be requested for all interventional clinical studies:

used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US).
where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope.

In addition, access can be requested for all interventional clinical studies in patients:

sponsored by Servier
with a first patient enrolled as of 1 January 2004 onwards
for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.

location_country: Japan Netherlands France Belgium

location_facility: Erasmus MC Section Clinical Pharmacology

location_facility: UZ Leuven Campus Gasthuisberg Dept. of General Medical

location_facility: Institut Jules Bordet Clinique Oncologie Médicale

location_facility: Institut de Cancérologie de l'Ouest site Saint Herblain

summary: The purpose of this study is to determine the safety profile, the maximum tolerated dose (MTD) and the associated dose-limiting toxicities (DLTs) of S 81694 in combination with paclitaxel in metastatic breast cancer (mBC) patients, and to investigate the antitumour activity of the combination in metastatic triple negative breast cancer (mTNBC) patients.


acronym: SPRING

title: A Proof of Concept Study to Explore Safety and Efficacy of Tri-therapy Approach in Advanced/Metastatic NSCLC and Retrospectively Assess the Ability of Integrated Genomics and Transcriptomics to Match Patients to the Combination

lead_sponsor: Worldwide Innovative Networking Association

ReferenceCitation: Solomon B, Callejo A, Bar J, Berchem G, Bazhenova L, Saintigny P, Wunder F, Raynaud J, Girard N, Lee JJ, Sulaiman R, Prouse B, Bresson C, Ventura H, Magidi S, Rubin E, Young B, Onn A, Leyland-Jones B, Schilsky RL, Lazar V, Felip E, Kurzrock R. A WIN Consortium phase I study exploring avelumab, palbociclib, and axitinib in advanced non-small cell lung cancer. Cancer Med. 2022 Jul;11(14):2790-2800. doi: 10.1002/cam4.4635. Epub 2022 Mar 20.

location_country:
Luxembourg
Spain
United States
Israel
France

location_facility:
UCSD Moores Cancer Center
Vall Hebron Institute of Oncology
Chiam Sheba Medical Center
Centre Léon Bérard
Centre Hospitalier Luxembourg
Avera Cancer Center

summary: Patients with advanced/metastatic non-small cell lung cancer (NSCLC) with no documented targetable alterations (Epidermal Growth Factor Receptor (EGFR) mutation, Anaplastic Lymphoma Kinase (ALK) translocation, ROS1 mutation if available or MET exon 14 skipping mutation if available) will receive a tri-therapy associating avelumab, axitinib and palbociclib.


title: A Phase I, Multi-center, Open-label, Study of LXS196, an Oral Protein Kinase C Inhibitor, in Patients With Metastatic Uveal Melanoma

summary: This study was to characterize the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary anti-tumor activity of LXS196 as a single agent and in combination with HDM201 in patients with metastatic uveal melanoma.

location_country:
Spain
Australia
United States
France
Norway
Netherlands

location_facility:
Columbia University Medical Center
Novartis Investigative Site


title: An Open-label, Non-randomized, Multicenter Phase I Dose Escalation Study to Characterize Safety, Tolerability, Pharmacokinetics and Maximum Tolerated Dose of BAY 1238097 in Subjects With Advanced Malignancies

summary:
This is the first study where BAY1238097 is given to humans. Impact of the study is to evaluate if patients with advanced cancer show clinical benefit under the treatment with BET(Bromodomain and extraterminal domain family ) inhibitor.Patients with solid tumors (all comers) and lymphoma will receive the study drug treatment in an escalation scheme (no placebo group) to determine the safety, tolerability and maximum tolerated dose (MTD) of BAY1238097. the relative bioavailability of Liquid Service Formulation and tablets will be determined

After MTD is defined, patients with solid tumors (all comer, hepato cellular carcinoma, lung cancer, NUT(nuclear protein in testis)-midline carcinoma), melanoma and lymphoma will be enrolled A separate escalation scheme will be applied to patients with leucemias, and at the maximal tolerated dose, patients with AML amd multiple myeloma will be enrolled.

the study will also assess the pharmacokinetics, biomarker status, pharmacodynamic parameters of BAY1238097 and tumor response to the treatment.

BAY1238097 will be given twice weekly as oral application. Treatment will be stopped if the tumor continues to grow, if side effects occur, wich the patient cannot tolerate or if the patient decides to withdraw from the treatment.

ReferenceCitation: Postel-Vinay S, Herbschleb K, Massard C, Woodcock V, Soria JC, Walter AO, Ewerton F, Poelman M, Benson N, Ocker M, Wilkinson G, Middleton M. First-in-human phase I study of the bromodomain and extraterminal motif inhibitor BAY 1238097: emerging pharmacokinetic/pharmacodynamic relationship and early termination due to unexpected toxicity. Eur J Cancer. 2019 Mar;109:103-110. doi: 10.1016/j.ejca.2018.12.020. Epub 2019 Jan 31.

location_country:
United Kingdom
Finland
Switzerland
United States
France
Germany


title: An Open-Label, Single-Arm, Phase 3 Study to Evaluate the Effects of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)

lead_sponsor: Ultragenyx Pharmaceutical Inc

ReferenceCitation: Fratzl-Zelman N, Hartmann MA, Gamsjaeger S, Rokidi S, Paschalis EP, Blouin S, Zwerina J. Bone Matrix Mineralization and Response to Burosumab in Adult Patients With X-Linked Hypophosphatemia: Results From the Phase 3, Single-Arm International Trial. J Bone Miner Res. 2022 Sep;37(9):1665-1678. doi: 10.1002/jbmr.4641. Epub 2022 Aug 10.

location_country:
Denmark
Canada
Korea, Republic of
United States
France
Japan

location_facility: CHU de Bicêtre, Service d'Endocrinologie et des Maladies de la Reproduction

location_facility: Indiana University Department of Medicine University Hospital

location_facility: Aarhus University Hospital-Dept of Endocrinology and Internal Medicine

location_facility: Yale University School of Medicine - Yale New-Haven Hospital/Yale Center for Clinical Investigation

summary: The primary objective of this study is to establish the effect of KRN23 treatment on improvement in XLH-associated osteomalacia as determined by osteoid volume (osteoid volume/bone volume, OV/BV).


title: Phase 1b, Randomized, Double-blind, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of QR-010 in Subjects With Homozygous ΔF508 Cystic Fibrosis

summary: A randomized, double-blind, placebo-controlled study of single and multiple ascending doses of QR-010 in adults homozygous for ΔF508 Cystic Fibrosis.

ReferenceCitation: Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27. Review.

lead_sponsor: ProQR Therapeutics

location_country:
Denmark
Spain
Italy
United Kingdom
Canada
United States
France
Belgium
Germany
Czechia

location_facility: University of Kansas Medical Center Research Institute

location_facility: Munich U. Hospital, Cystic Fibrosis Center for Adults

location_facility: Washington University School of Medicine

location_facility: Penn State Milton S. Hershey Medical Center

location_facility: University of Calgary (Health Sciences Centre)

location_facility: Azienda Ospedaliera Universitaria Integrata di Verona

location_facility: University of Southern California USC - Keck School of Medicine

location_facility: University of Texas Southwestern Medical Center


title: A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicenter Study to Evaluate the Net Clinical Benefit of Sotagliflozin as Adjunct to Insulin Therapy in Type 1 Diabetes

summary: This Phase 3 study was designed to demonstrate the net benefit of sotagliflozin versus placebo in patients with Type 1 Diabetes (T1D).

ReferenceCitation: Garg SK, Henry RR, Banks P, Buse JB, Davies MJ, Fulcher GR, Pozzilli P, Gesty-Palmer D, Lapuerta P, Simó R, Danne T, McGuire DK, Kushner JA, Peters A, Strumph P. Effects of Sotagliflozin Added to Insulin in Patients with Type 1 Diabetes. N Engl J Med. 2017 Dec 14;377(24):2337-2348. doi: 10.1056/NEJMoa1708337. Epub 2017 Sep 13.

lead_sponsor: Lexicon Pharmaceuticals

location_country:
Poland
Spain
Colombia
Italy
Australia
New Zealand
Argentina
Canada
United Kingdom
Hungary
United States
France
Israel
South Africa
Belgium
Germany
Bulgaria
Slovakia
Czechia


title: Short to Mid Term Follow-up of Patient Implanted With the HINTEGRA® Total Ankle Prosthesis

summary:
The Hintegra® Total Ankle Prosthesis is a non constrained, three-component prosthesis that consists of a flat tibial component, an anatomically-shaped talar component, and a high-density polyethylene inlay. The Hintegra ankle was specifically developed as an attempt to address the needs of minimal bone resection, extended bone support, proper ligament balancing, and minimal contact stressed within and around the prosthesis.

The objective of this study is to evaluate the safety and efficacy of the HINTEGRA® Total Ankle prosthesis at short and mid term follow up.

lead_sponsor: Integra LifeSciences Services

location_country:
Spain
Austria
Canada
Korea, Republic of
France
Germany

location_facility:
Medical University of Innsbruck
Severance Hospital - Yonsei University
CHU d'Amiens - Hopital Nord
AukammKlinik
Hopital San Rafael
St. Michael's Hospital


title: A Phase I/II Pharmacokinetic Study of Intravenous and Oral Forodesine in Children With Relapsed or Refractory T-cell or B-cell Precursor Acute Lymphoblastic Leukaemia or T-cell Non-Hodgkin's Lymphoma.

summary: The purpose of this study is to evaluate the pharmacokinetics, pharmacodynamics and safety of different doses of intravenous and oral Forodesine in children with relapsed or refractory T-cell or B-cell precursor Acute Lymphoblastic Leukaemia or T-cell Non-Hodgkin's Lymphoma. Preliminary efficacy will also be assessed.

lead_sponsor: Mundipharma Research Limited

location_country:
United Kingdom
Italy
Austria
Czech Republic
France
Germany

location_facility:
Sally Kinsey
Prof Gerard Michel
Charite Universitymedicine
Dr Giovanna Gioriani


title: A Phase Ib Study Investigating the Combination of Everolimus With Trastuzumab and Vinorelbine in Patients With HER2-overexpressing Metastatic Breast Cancer

summary: This study will look at different dose levels and regimens of everolimus combined with weekly trastuzumab and vinorelbine therapy in patients with HER-2 overexpressing metastatic breast cancer.

ReferenceCitation: Jerusalem G, Fasolo A, Dieras V, Cardoso F, Bergh J, Vittori L, Zhang Y, Massacesi C, Sahmoud T, Gianni L. Phase I trial of oral mTOR inhibitor everolimus in combination with trastuzumab and vinorelbine in pre-treated patients with HER2-overexpressing metastatic breast cancer. Breast Cancer Res Treat. 2011 Jan;125(2):447-55. doi: 10.1007/s10549-010-1260-x. Epub 2010 Nov 25.

location_country: Poland Italy France Belgium Sweden


title: Prospective, Controlled, Randomized Study to Evaluate Pain and Further Disabling Complications in Patients Undergoing Lichtenstein Technique for Primary Inguinal Hernia Repair by Fixing the Mesh With Fibrin Sealant Versus Sutures

summary: The purpose of the study is to evaluate mid and long term postoperative pain and further disabling complications in open inguinal hernia repair by Lichtenstein technique after mesh fixation with fibrin sealant (FS), compared to mesh fixation with sutures.

ReferenceCitation: Campanelli G, Pascual MH, Hoeferlin A, Rosenberg J, Champault G, Kingsnorth A, Miserez M. Randomized, controlled, blinded trial of Tisseel/Tissucol for mesh fixation in patients undergoing Lichtenstein technique for primary inguinal hernia repair: results of the TIMELI trial. Ann Surg. 2012 Apr;255(4):650-7. doi: 10.1097/SLA.0b013e31824b32bf.

lead_sponsor: University of Milan

location_country:
Denmark
Spain
United Kingdom
France
Belgium
Germany

location_facility: Copenhagen university hospital, Gentofte

intervention_type: Procedure


title: A Prospective Multicentre Phase II Trial of Zoledronic Acid in Patients With Myelofibrosis With Myeloid Metaplasia (MMM)

summary: In this trial, the question is addressed if zoledronic acid (Zometa, Novartis Pharma)could be of clinical benefit for patients with myelofibrosis and myeloid metaplasia (MMM).

lead_sponsor: Universitaire Ziekenhuizen KU Leuven

location_country: Spain Australia France Israel Belgium Germany

location_facility: Hopital Avicenne and Paris 13 University


title: A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of BLU-554 in Patients With Hepatocellular Carcinoma

summary: This is a Phase 1, open-label, first-in-human (FIH) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antineoplastic activity of fisogatinib (formerly known as BLU- 554) administered orally in patients with FGF19 IHC+ hepatocellular carcinoma (HCC). The study consists of 3 parts, a dose-escalation part (Part 1), an expansion part (Part 2) exploring a once daily (qd) dosing schedule at the recommended Phase 2 dose (RP2D), and a Part 3 expansion of the qd dosing schedule at the RP2D in TKI naive patients.

lead_sponsor: Blueprint Medicines Corporation

location_country:
Taiwan
Hong Kong
Spain
Italy
United Kingdom
Switzerland
Korea, Republic of
United States
France
China
Singapore
Germany

location_facility: Tianjin Medical University Cancer Institute & Hospital, Hepatobiliary Oncology Department

location_facility: IRCCS Foundation - National Institute of Tumors

location_facility: Jilin University the First Affiliated Hospital

location_facility: University of Miami - Sylvester Comprehensive Cancer Center

location_facility: Johannes Gutenberg University Mainz - University Medical Center

location_facility: University of Liverpool - Clatterbridge Cancer Centre

location_facility: The First Affiliated Hospital, College of Medicine, Zhejiang University

location_facility: Hunan Cancer Hospital, Radioactive Interventional Department

location_facility: The Chinese People's Liberation Army 81 Hospital

location_facility: Harbin Medical University Cancer Hospital


title: A Multi-center, Randomized, Double-blind, Placebo-controlled Phase IIa Trial to Compare the Safety of ABX464 Given at a Fixed Dose to Placebo in Fully Controlled HIV Infected Patients Treated With Boosted Protease Inhibitor Treatment (Darunavir/Ritonavir or Darunavir/Cobicistat).

lead_sponsor: Abivax S.A.

ReferenceCitation: Rutsaert S, Steens JM, Gineste P, Cole B, Kint S, Barrett PN, Tazi J, Scherrer D, Ehrlich HJ, Vandekerckhove L. Safety, tolerability and impact on viral reservoirs of the addition to antiretroviral therapy of ABX464, an investigational antiviral drug, in individuals living with HIV-1: a Phase IIa randomised controlled study. J Virus Erad. 2019 Jan 1;5(1):10-22.

location_country: France Spain Belgium

location_facility: Hospital Universitari Germans Trias i Pujo

location_facility: CHU de Montpellier - Hôpital Gui de Chauliac

summary: This study is a placebo-controlled study aimed at assessing the safety of ABX464 administered at 50 mg and 150 mg o.d. versus placebo in HIV infected patients who are treated with darunavir + ritonavir (DRV/RTV) or darunavir + cobicistat (DRV/COBI).


title: A Randomised, Double-blind, Double-dummy, Parallel-group, Multicenter, Phase IIb Study to Evaluate the Effect of Ticagrelor Versus Placebo in Reducing the Number of Days With Pain in Young Adults With Sickle Cell Disease

summary: The purpose of this study is to determine whether ticagrelor is effective in reducing the number of days of pain, intensity of pain, and reducing the use of analgesics due to sickle cell disease

primary_purpose: Prevention

lead_sponsor: AstraZeneca

location_country:
Kenya
United Kingdom
Italy
Lebanon
United States
France
Turkey
Egypt


title: Assessment of the Efficacy of a Sensorimotor Approach After Maximum Three Sessions in Subjects With Symptomatic Temporomandibular Dysfunction (TMD) With Hypertonia: A Prospective, Multicenter Case Study.

summary:
The study aims to demonstrate the efficacy of the sensorimotor approach developed by A. Piron (taught to the patient in three sessions) regarding three parameters related to TMD: facial pain, functional incapacities and hypertonia.

The subjects in the study are 18 to 77 years old and present the following conditions: a TMD disorder caused or aggravated by hypertonia and responsible for cervico-maxillo-facial pain and/or a functional incapacity in the last 30 days.

status: Unknown status

lead_sponsor: Osteovox

location_country: France Italy Belgium

location_facility:
Cabinet d'Ostéopathie Garcion
ANTONIO BIANCO Ortodontista e Osteopata
Osteopata MARCO SBARBARO
Cabinet de groupe LHR
Cabinet d'Ostéopathie PIRON
Cabinet de Logopédie Liesens-Denis

intervention_type: Behavioral


title: A Phase Ib, Open-Label, Dose-Escalation Study of the Safety and Pharmacology of GDC-0919 Administered With Atezolizumab in Patients With Locally Advanced or Metastatic Solid Tumors

summary: This study will evaluate the safety, tolerability, and pharmacokinetics of the combination of GDC-0919 and atezolizumab in participants with locally advanced, recurrent, or metastatic incurable solid malignancy that has progressed after available standard therapy or for which standard therapy is ineffective, intolerable, or inappropriate. Participants will be enrolled in two stages, including a dose-escalation stage and an expansion stage.

ReferenceCitation: Jung KH, LoRusso P, Burris H, Gordon M, Bang YJ, Hellmann MD, Cervantes A, Ochoa de Olza M, Marabelle A, Hodi FS, Ahn MJ, Emens LA, Barlesi F, Hamid O, Calvo E, McDermott D, Soliman H, Rhee I, Lin R, Pourmohamad T, Suchomel J, Tsuhako A, Morrissey K, Mahrus S, Morley R, Pirzkall A, Davis SL. Phase I Study of the Indoleamine 2,3-Dioxygenase 1 (IDO1) Inhibitor Navoximod (GDC-0919) Administered with PD-L1 Inhibitor (Atezolizumab) in Advanced Solid Tumors. Clin Cancer Res. 2019 Jun 1;25(11):3220-3228. doi: 10.1158/1078-0432.CCR-18-2740. Epub 2019 Feb 15.

lead_sponsor: Genentech, Inc.

location_country:
Korea, Republic of
United States
Spain
France

location_facility: HonorHealth Research Institute - Bisgrove

location_facility: The Angeles Clinic and Research Institute, Santa Monica Office

location_facility: Johns Hopkins Sidney Kimmel Comprehensive Cancer Center

location_facility: Hospital Universitario HM Sanchinarro-CIOCC

location_facility: H. Lee Moffitt Cancer Center and Research Inst.

location_facility: Hospital Clinico Universitario de Valencia


title: CardioRoot Post-Marketing Surveillance Registry

acronym: CardioRoot

summary: Post-marketing surveillance registry of the CardioRoot graft.

ReferenceCitation: Tasca G, Lindner J, Barandon L, Santavy P, Antona C, Burkert J, Gamba A. Aortic root surgery with the CARDIOROOT vascular graft: results of a prospective multicenter post-market surveillance study. J Cardiothorac Surg. 2019 May 21;14(1):94. doi: 10.1186/s13019-019-0914-y.

lead_sponsor: Maquet Cardiovascular

location_country: France Italy Czechia

location_facility: Centre Hospitalier Universitaire (CHU) de Bordeaux


title: Evaluation of the Safety and Effectiveness of Sepraspray™ in Limiting the Formation of Postoperative Adhesions to Serosal Tissue (Abdominal Surgery)

summary: This study will examine the safety and efficacy of Sepraspray in the following model of abdominal surgery: total proctocolectomy and pelvic pouch with diverting ileostomy via laparotomy to treat ulcerative colitis or familial polyposis. Adhesion formation will be evaluated laparoscopically at ileostomy take down. NOTE: A decision was made to terminate this study in June 2008 due to low enrollment.

lead_sponsor: Genzyme, a Sanofi Company

location_country:
Denmark
United Kingdom
Czech Republic
France
Germany


title: An Eight-week, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Saredutant 100 mg Once Daily in Combination With Escitalopram 10 mg Once Daily in Patients With Major Depressive Disorder

summary: The main purpose of this study is to evaluate the efficacy of saredutant 100mg once daily in combination with escitalopram 10mg once daily compared to saredutant placebo in combination with escitalopram 10mg once daily in patients with major depressive disorder. This study also includes a double-placebo group (saredutant placebo in combination with escitalopram placebo).

lead_sponsor: Sanofi

location_country:
Finland
Argentina
United States
France
Mexico
Sweden


title: A Phase I/II, Open Label, Multicenter Study of the Safety and Efficacy of LAG525 Single Agent and in Combination With PDR001 Administered to Patients With Advanced Malignancies

lead_sponsor: Novartis Pharmaceuticals

ReferenceCitation: Schöffski P, Tan DSW, Martín M, Ochoa-de-Olza M, Sarantopoulos J, Carvajal RD, Kyi C, Esaki T, Prawira A, Akerley W, De Braud F, Hui R, Zhang T, Soo RA, Maur M, Weickhardt A, Krauss J, Deschler-Baier B, Lau A, Samant TS, Longmire T, Chowdhury NR, Sabatos-Peyton CA, Patel N, Ramesh R, Hu T, Carion A, Gusenleitner D, Yerramilli-Rao P, Askoxylakis V, Kwak EL, Hong DS. Phase I/II study of the LAG-3 inhibitor ieramilimab (LAG525) ± anti-PD-1 spartalizumab (PDR001) in patients with advanced malignancies. J Immunother Cancer. 2022 Feb;10(2). pii: e003776. doi: 10.1136/jitc-2021-003776.

location_country:
Taiwan
Hong Kong
Spain
Italy
Australia
Canada
United States
France
Singapore
Belgium
Japan
Germany

location_facility: Huntsman Cancer Institute Huntsman Cancer Institute

location_facility: Cancer Therapy and Research Center UT Health Science Center CTRC 2

location_facility: University of Texas MD Anderson Cancer Center

location_facility: Columbia University Medical Center SC LAG X2101C

location_facility: Memorial Sloan Kettering Cancer Center SC

summary: This study was to characterize the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and anti-tumor activity of LAG525 as a single agent and in combination with PDR001 to adult patients with solid tumors. The study consists of a dose escalation (phase 1) to determine the maximum tolerated dose (MTD) or recommended Phase 2 dose (RP2D) for LAG525 as a single agent and in combination with PDR001, and a dose expansion (phase 2) which characterized treatment of LAG525 in combination with PDR001 at the MTD or RP2D.


title: A 52-Week Double-Blind, Placebo-Controlled, Parallel-Group Efficacy and Safety Study of Reslizumab 110 mg Fixed, Subcutaneous Dosing in Patients With Uncontrolled Asthma and Elevated Blood Eosinophils

summary: The primary objective of this study is to determine the effect of reslizumab (110 mg) administered subcutaneously every 4 weeks on clinical asthma exacerbations in adults and adolescents with asthma and elevated blood eosinophils who are inadequately controlled on standard-of-care asthma therapy.

ReferenceCitation: Bernstein JA, Virchow JC, Murphy K, Maspero JF, Jacobs J, Adir Y, Humbert M, Castro M, Marsteller DA, McElhattan J, Hickey L, Garin M, Vanlandingham R, Brusselle G. Effect of fixed-dose subcutaneous reslizumab on asthma exacerbations in patients with severe uncontrolled asthma and corticosteroid sparing in patients with oral corticosteroid-dependent asthma: results from two phase 3, randomised, double-blind, placebo-controlled trials. Lancet Respir Med. 2020 May;8(5):461-474. doi: 10.1016/S2213-2600(19)30372-8. Epub 2020 Feb 14.

lead_sponsor: Teva Branded Pharmaceutical Products R&D, Inc.

location_country:
Spain
Ukraine
United States
Romania
South Africa
Belgium
Japan
Czechia
Poland
Russian Federation
Australia
Argentina
Israel
Turkey
Mexico
Canada
Hungary
Germany
New Zealand
Korea, Republic of
France

location_facility:
Teva Investigational Site 80076
Teva Investigational Site 58219
Teva Investigational Site 13763
Teva Investigational Site 13253
Teva Investigational Site 84035
Teva Investigational Site 90027
Teva Investigational Site 13209
Teva Investigational Site 31152
Teva Investigational Site 51216
Teva Investigational Site 13767
Teva Investigational Site 13247
Teva Investigational Site 78087
Teva Investigational Site 37054
Teva Investigational Site 54129
Teva Investigational Site 20045
Teva Investigational Site 13212
Teva Investigational Site 13257
Teva Investigational Site 84031
Teva Investigational Site 13223
Teva Investigational Site 84044
Teva Investigational Site 51222
Teva Investigational Site 32567
Teva Investigational Site 51223
Teva Investigational Site 84048
Teva Investigational Site 13764
Teva Investigational Site 13230
Teva Investigational Site 13239
Teva Investigational Site 51221
Teva Investigational Site 21088
Teva Investigational Site 53313
Teva Investigational Site 50347
Teva Investigational Site 35182
Teva Investigational Site 51228
Teva Investigational Site 20053
Teva Investigational Site 13210
Teva Investigational Site 13197
Teva Investigational Site 20046
Teva Investigational Site 32557
Teva Investigational Site 20032
Teva Investigational Site 13204
Teva Investigational Site 13215
Teva Investigational Site 52106
Teva Investigational Site 79046
Teva Investigational Site 87016
Teva Investigational Site 87014
Teva Investigational Site 82042
Teva Investigational Site 58228
Teva Investigational Site 13238
Teva Investigational Site 32561
Teva Investigational Site 13242
Teva Investigational Site 13241
Teva Investigational Site 13259
Teva Investigational Site 13260
Teva Investigational Site 51255
Teva Investigational Site 51229
Teva Investigational Site 53310
Teva Investigational Site 13199
Teva Investigational Site 51231
Teva Investigational Site 84037
Teva Investigational Site 84032
Teva Investigational Site 78088
Teva Investigational Site 13186
Teva Investigational Site 58221
Teva Investigational Site 13181
Teva Investigational Site 84047
Teva Investigational Site 13202
Teva Investigational Site 13218
Teva Investigational Site 90028
Teva Investigational Site 80079
Teva Investigational Site 32563
Teva Investigational Site 13211
Teva Investigational Site 50349
Teva Investigational Site 58233
Teva Investigational Site 53309
Teva Investigational Site 13243
Teva Investigational Site 13185
Teva Investigational Site 13240
Teva Investigational Site 51224
Teva Investigational Site 90031
Teva Investigational Site 21087
Teva Investigational Site 13229
Teva Investigational Site 20054
Teva Investigational Site 13190
Teva Investigational Site 52107
Teva Investigational Site 80078
Teva Investigational Site 13189
Teva Investigational Site 13217
Teva Investigational Site 84036
Teva Investigational Site 84038
Teva Investigational Site 32562
Teva Investigational Site 79047
Teva Investigational Site 53311
Teva Investigational Site 58229
Teva Investigational Site 13252
Teva Investigational Site 37055
Teva Investigational Site 90029
Teva Investigational Site 13187
Teva Investigational Site 20040
Teva Investigational Site 78085
Teva Investigational Site 51217
Teva Investigational Site 13251
Teva Investigational Site 84043
Teva Investigational Site 13195
Teva Investigational Site 21089
Teva Investigational Site 58213
Teva Investigational Site 58218
Teva Investigational Site 52105
Teva Investigational Site 21086
Teva Investigational Site 58220
Teva Investigational Site 58216
Teva Investigational Site 13232
Teva Investigational Site 53312
Teva Investigational Site 13246
Teva Investigational Site 32570
Teva Investigational Site 53314
Teva Investigational Site 13258
Teva Investigational Site 13205
Teva Investigational Site 84049
Teva Investigational Site 21084
Teva Investigational Site 32568
Teva Investigational Site 82039
Teva Investigational Site 20033
Teva Investigational Site 13224
Teva Investigational Site 31149
Teva Investigational Site 58231
Teva Investigational Site 51220
Teva Investigational Site 13188
Teva Investigational Site 80073
Teva Investigational Site 13226
Teva Investigational Site 21085
Teva Investigational Site 20035
Teva Investigational Site 87015
Teva Investigational Site 35183
Teva Investigational Site 32559
Teva Investigational Site 84040
Teva Investigational Site 13208
Teva Investigational Site 50354
Teva Investigational Site 13184
Teva Investigational Site 54128
Teva Investigational Site 58224
Teva Investigational Site 13249
Teva Investigational Site 80077
Teva Investigational Site 13200
Teva Investigational Site 80075
Teva Investigational Site 13207
Teva Investigational Site 58225
Teva Investigational Site 58234
Teva Investigational Site 84053
Teva Investigational Site 13220
Teva Investigational Site 53315
Teva Investigational Site 58226
Teva Investigational Site 51226
Teva Investigational Site 90030
Teva Investigational Site 58222
Teva Investigational Site 58214
Teva Investigational Site 13222
Teva Investigational Site 13254
Teva Investigational Site 13194
Teva Investigational Site 20041
Teva Investigational Site 78084
Teva Investigational Site 51218
Teva Investigational Site 13196
Teva Investigational Site 31147
Teva Investigational Site 58223
Teva Investigational Site 51225
Teva Investigational Site 58227
Teva Investigational Site 11106
Teva Investigational Site 32565
Teva Investigational Site 58230
Teva Investigational Site 50352
Teva Investigational Site 82043
Teva Investigational Site 52110
Teva Investigational Site 90032
Teva Investigational Site 13203
Teva Investigational Site 84041
Teva Investigational Site 13250
Teva Investigational Site 84046
Teva Investigational Site 13255
Teva Investigational Site 13765
Teva Investigational Site 37053
Teva Investigational Site 82041
Teva Investigational Site 78083
Teva Investigational Site 13766
Teva Investigational Site 11105
Teva Investigational Site 51219
Teva Investigational Site 20037
Teva Investigational Site 13235
Teva Investigational Site 50348
Teva Investigational Site 32566
Teva Investigational Site 82040
Teva Investigational Site 51227
Teva Investigational Site 32564
Teva Investigational Site 32556
Teva Investigational Site 13225
Teva Investigational Site 50351
Teva Investigational Site 84039
Teva Investigational Site 90026
Teva Investigational Site 13769
Teva Investigational Site 50350
Teva Investigational Site 80081
Teva Investigational Site 32560
Teva Investigational Site 84034
Teva Investigational Site 20036
Teva Investigational Site 52104
Teva Investigational Site 13216
Teva Investigational Site 52109
Teva Investigational Site 13191
Teva Investigational Site 32555
Teva Investigational Site 53308
Teva Investigational Site 13227
Teva Investigational Site 58253
Teva Investigational Site 13201
Teva Investigational Site 31154
Teva Investigational Site 13183
Teva Investigational Site 32551
Teva Investigational Site 80094
Teva Investigational Site 80080
Teva Investigational Site 50355
Teva Investigational Site 52108
Teva Investigational Site 54130
Teva Investigational Site 13256
Teva Investigational Site 52111
Teva Investigational Site 32569
Teva Investigational Site 84045
Teva Investigational Site 13233
Teva Investigational Site 11109


title: A Phase II, Single Arm, Multicenter Trial to Determine the Efficacy and Safety of CTL019 in Pediatric Patients With Relapsed and Refractory B-cell Acute Lymphoblastic Leukemia

acronym: ELIANA

summary: This is a single arm, open-label, multi-center, phase II study to determine the efficacy and safety of CTL019 in pediatric patients with r/r B-cell ALL.

ReferenceCitation: Laetsch TW, Maude SL, Rives S, Hiramatsu H, Bittencourt H, Bader P, Baruchel A, Boyer M, De Moerloose B, Qayed M, Buechner J, Pulsipher MA, Myers GD, Stefanski HE, Martin PL, Nemecek E, Peters C, Yanik G, Khaw SL, Davis KL, Krueger J, Balduzzi A, Boissel N, Tiwari R, O'Donovan D, Grupp SA. Three-Year Update of Tisagenlecleucel in Pediatric and Young Adult Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia in the ELIANA Trial. J Clin Oncol. 2022 Nov 18:JCO2200642. doi: 10.1200/JCO.22.00642. [Epub ahead of print]

ReferenceCitation: Newman H, Li Y, Liu H, Myers RM, Tam V, DiNofia A, Wray L, Rheingold SR, Callahan C, White C, Baniewicz D, Winestone LE, Kadauke S, Diorio C, June CH, Getz K, Aplenc R, Teachey DT, Maude SL, Grupp SA, Bona K, Barz Leahy A. Impact of poverty and neighborhood opportunity on outcomes for children treated with CD19-directed CAR T-cell therapy. Blood. 2022 Nov 9. pii: blood.2022017866. doi: 10.1182/blood.2022017866. [Epub ahead of print]

ReferenceCitation: Laetsch TW, Maude SL, Balduzzi A, Rives S, Bittencourt H, Boyer MW, Buechner J, De Moerloose B, Qayed M, Phillips CL, Pulsipher MA, Hiramatsu H, Tiwari R, Grupp SA. Tisagenlecleucel in pediatric and young adult patients with Down syndrome-associated relapsed/refractory acute lymphoblastic leukemia. Leukemia. 2022 Jun;36(6):1508-1515. doi: 10.1038/s41375-022-01550-z. Epub 2022 Apr 14.

ReferenceCitation: Thudium Mueller K, Grupp SA, Maude SL, Levine JE, Pulsipher MA, Boyer MW, August KJ, Myers GD, Tam CS, Jaeger U, Foley SR, Borchmann P, Schuster SJ, Waller EK, Awasthi R, Potthoff B, Warren A, Waldron ER, McBlane F, Chassot-Agostinho A, Laetsch TW. Tisagenlecleucel immunogenicity in relapsed/refractory acute lymphoblastic leukemia and diffuse large B-cell lymphoma. Blood Adv. 2021 Dec 14;5(23):4980-4991. doi: 10.1182/bloodadvances.2020003844.

ReferenceCitation: Levine JE, Grupp SA, Pulsipher MA, Dietz AC, Rives S, Myers GD, August KJ, Verneris MR, Buechner J, Laetsch TW, Bittencourt H, Baruchel A, Boyer MW, De Moerloose B, Qayed M, Davies SM, Phillips CL, Driscoll TA, Bader P, Schlis K, Wood PA, Mody R, Yi L, Leung M, Eldjerou LK, June CH, Maude SL. Pooled safety analysis of tisagenlecleucel in children and young adults with B cell acute lymphoblastic leukemia. J Immunother Cancer. 2021 Aug;9(8). pii: e002287. doi: 10.1136/jitc-2020-002287.

ReferenceCitation: Buechner J, Grupp SA, Hiramatsu H, Teachey DT, Rives S, Laetsch TW, Yanik GA, Wood P, Awasthi R, Yi L, Chassot-Agostinho A, Eldjerou LK, De Moerloose B. Practical guidelines for monitoring and management of coagulopathy following tisagenlecleucel CAR T-cell therapy. Blood Adv. 2021 Jan 26;5(2):593-601. doi: 10.1182/bloodadvances.2020002757.

ReferenceCitation: Laetsch TW, Myers GD, Baruchel A, Dietz AC, Pulsipher MA, Bittencourt H, Buechner J, De Moerloose B, Davis KL, Nemecek E, Driscoll T, Mechinaud F, Boissel N, Rives S, Bader P, Peters C, Sabnis HS, Grupp SA, Yanik GA, Hiramatsu H, Stefanski HE, Rasouliyan L, Yi L, Shah S, Zhang J, Harris AC. Patient-reported quality of life after tisagenlecleucel infusion in children and young adults with relapsed or refractory B-cell acute lymphoblastic leukaemia: a global, single-arm, phase 2 trial. Lancet Oncol. 2019 Dec;20(12):1710-1718. doi: 10.1016/S1470-2045(19)30493-0. Epub 2019 Oct 9.

ReferenceCitation: Maude SL, Laetsch TW, Buechner J, Rives S, Boyer M, Bittencourt H, Bader P, Verneris MR, Stefanski HE, Myers GD, Qayed M, De Moerloose B, Hiramatsu H, Schlis K, Davis KL, Martin PL, Nemecek ER, Yanik GA, Peters C, Baruchel A, Boissel N, Mechinaud F, Balduzzi A, Krueger J, June CH, Levine BL, Wood P, Taran T, Leung M, Mueller KT, Zhang Y, Sen K, Lebwohl D, Pulsipher MA, Grupp SA. Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia. N Engl J Med. 2018 Feb 1;378(5):439-448. doi: 10.1056/NEJMoa1709866.

location_country:
Spain
Italy
Austria
Australia
Canada
United States
France
Norway
Belgium
Japan
Germany

location_facility: Oregon Health & Science University Doernbecher Children's Hosp.

location_facility: The Childrens Hospital of Philadelphia CHOP

location_facility: University of Wisconsin Hospital and Clinics

location_facility: Children's Healthcare of Atlanta SC CTL019

location_facility: Childrens Hospital Los Angeles SC CTL019

location_facility: Cincinnati Children's Hospital Medical Center


acronym: TNBC

title: A Randomized Phase II Study of MCS110 Combined With Carboplatin and Gemcitabine in Advanced Triple Negative Breast Cancer (TNBC)

location_country:
Taiwan
Hong Kong
Spain
Italy
Austria
Australia
Korea, Republic of
United States
France
Turkey
Belgium
Germany

location_facility: Massachusetts General Hospital Cancer Center SC

summary: To determine whether MCS110 antibody therapy improves the efficacy of carboplatin and gemcitabine (carbo/gem) in advanced TNBC patients


title: A Phase 1b Open Label, Dose Escalation Study to Assess Safety, Pharmacokinetics, Pharmacodynamics, and Antitumor Activity of PLX3397 in Combination With Vemurafenib in V600-mutated BRAF Unresectable or Metastatic Melanoma

summary: The purpose of this research study is to test the safety of an investigational new drug called PLX3397 when used in combination with Vemurafenib (Zelboraf™) at different dose levels. Vemurafenib has been approved by the United States Food and Drug Administration (FDA)/European Medicines Agency (EMA) for the treatment of a specific category of unresectable or metastatic melanoma.

ipd_sharing_description: De-identified individual participant data (IPD) and applicable supporting clinical trial documents may be available upon request at https://vivli.org/. In cases where clinical trial data and supporting documents are provided pursuant to our company policies and procedures, Daiichi Sankyo will continue to protect the privacy of our clinical trial participants. Details on data sharing criteria and the procedure for requesting access can be found at this web address: https://vivli.org/ourmember/daiichi-sankyo/

lead_sponsor: Daiichi Sankyo, Inc.

location_country:
Germany
United States
France

location_facility:
University Hospital Essen
Institute Gustave Roussy
Seattle Cancer Care Alliance
Vanderbilt University
University of Colorado, Denver
UCLA


title: pCLE For the Diagnosis Of Cancer in Unknown Bile Duct Stricture

acronym: FOCUS

summary: This study will collect data from patients undergoing an ERCP procedure with probe-based Confocal Laser Endomicroscopy for suspicion of bile duct cancer. the objective is to evaluate the diagnostic performance of pCLE for the diagnosis of indeterminate biliary stricture when associated with other diagnostic information (standard ERCP and tissue sampling).

ReferenceCitation: Slivka A, Gan I, Jamidar P, Costamagna G, Cesaro P, Giovannini M, Caillol F, Kahaleh M. Validation of the diagnostic accuracy of probe-based confocal laser endomicroscopy for the characterization of indeterminate biliary strictures: results of a prospective multicenter international study. Gastrointest Endosc. 2015 Feb;81(2):282-90. doi: 10.1016/j.gie.2014.10.009.

lead_sponsor: Mauna Kea Technologies

location_country:
United States
Italy
France

location_facility: New York presbyterian Weill Cornell Medical center

location_facility: Policlinico Univertitario Agostino Gemelli


title: An Open-label, Randomized, Controlled, Multicenter, Phase II Study Evaluating Safety and Efficacy of Intratumorally Administered Intuvax Pre-nephrectomy Followed by Sunitinib Post-nephrectomy, Compared to Sunitinib Post-nephrectomy in Metastatic Renal Cell Carcinoma Patients

acronym: MERECA

summary: The purpose of this study is to compare tumor response, progression free survival (PFS) and overall survival (OS) in newly diagnosed mRCC patients treated with Intuvax (INN: ilixadencel) pre-nephrectomy followed by Sunitinib post-nephrectomy vs Sunitinib post-nephrectomy patients.

lead_sponsor: Mendus

location_country:
Poland
Spain
Latvia
United Kingdom
Hungary
United States
France
Sweden
Czechia

location_facility: Hospital Universitario Puerta de Hierro Majadahonda

location_facility: Centre Hospitalier Universitaire d'Angers

location_facility: Centre Hospitalier Universitaire de Toulouse-Hôpital Rangueil

location_facility: Niepubliczny Zakład Opieki Zdrowotnej Vesalius Sp. z o.o.

location_facility: Hospital Universitari Germans Trias i Pujol

location_facility: Pauls Stradins Clinical University Hospital


title: A Phase 1b Multiple Ascending Dose Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of BMS-833923 (XL139) in Combination With Carboplatin and Etoposide Followed by BMS-833923 Alone in Subjects With Extensive-Stage Small Cell Lung Cancer

summary: The purpose of this study is to determine the maximum tolerated dose (MTD) of BMS-833923 administered in combination with carboplatin and etoposide followed by BMS-833923 alone in subjects with extensive-stage Small Cell Lung Cancer (SCLC).

lead_sponsor: Bristol-Myers Squibb

location_country:
Ireland
Australia
Canada
United States
France

location_facility:
Karmanos Cancer Institute
Local Institution


title: Endoscopic Retrograde CholangioPancreatography Endomicroscopy Registry Outcomes Database

summary: This registry will collect data from patients routinely undergoing an ERCP and Cellvizio endomicroscopy procedure (and optionally an additional cholangiopancreatoscopy procedure) due to suspected pancreatic or bile duct cancer. The objective is to determine if endomicroscopy images collected using the marketed Cellvizio device may help endoscopists more accurately diagnose, in conjunction with traditional tissue sampling techniques, whether a suspected lesion is malignant or benign.

location_facility: New York Presbyterian Hospital/Columbia University Medical Center


title: A Phase 2, Open-Label, Multiple Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Female Patients With Fabry Disease

summary: Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.

ReferenceCitation: Benjamin ER, Della Valle MC, Wu X, Katz E, Pruthi F, Bond S, Bronfin B, Williams H, Yu J, Bichet DG, Germain DP, Giugliani R, Hughes D, Schiffmann R, Wilcox WR, Desnick RJ, Kirk J, Barth J, Barlow C, Valenzano KJ, Castelli J, Lockhart DJ. The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat. Genet Med. 2017 Apr;19(4):430-438. doi: 10.1038/gim.2016.122. Epub 2016 Sep 22.

ReferenceCitation: Giugliani R, Waldek S, Germain DP, Nicholls K, Bichet DG, Simosky JK, Bragat AC, Castelli JP, Benjamin ER, Boudes PF. A Phase 2 study of migalastat hydrochloride in females with Fabry disease: selection of population, safety and pharmacodynamic effects. Mol Genet Metab. 2013 May;109(1):86-92. doi: 10.1016/j.ymgme.2013.01.009. Epub 2013 Jan 26.

lead_sponsor: Amicus Therapeutics

location_country:
Brazil
United Kingdom
Australia
Canada
United States
France


title: A 12-Week, Multicenter, Randomized, Parallel-Group Study to Assess the Safety, Tolerability, Pharmacokinetics, Biomarker Effects, Efficacy, and Effect on Microglia Activation, as Measured by Positron Emission Tomography, of AZD3241 in Subjects With Multiple System Atrophy

summary: AZD3241 myeloperoxidase (MPO) inhibitor trial is assessing safety and tolerability, randomized trial, in patients with Multiple System Atrophy.

location_country:
United Kingdom
Italy
Austria
Finland
United States
France
Sweden


title: Clinical Performance Evaluation of T-TAS 01 PL Chip

summary: This study will measure primary hemostatic ability using the T-TAS 01 System with PL chip, with a comparison to clinical truth.

lead_sponsor: Hikari Dx, Inc.

location_country:
United States
France

location_facility: Centre Hospitalier Universitaire de Bordeaux


title: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

acronym: GNEM

summary: The primary objective of this study is to evaluate the effect of 6 g/day aceneuramic acid extended-release (Ace-ER) treatment of participants with GNEM on upper extremity muscle strength (upper extremity composite [UEC] score) as measured by dynamometry.

ReferenceCitation: Lochmüller H, Behin A, Caraco Y, Lau H, Mirabella M, Tournev I, Tarnopolsky M, Pogoryelova O, Woods C, Lai A, Shah J, Koutsoukos T, Skrinar A, Mansbach H, Kakkis E, Mozaffar T. A phase 3 randomized study evaluating sialic acid extended-release for GNE myopathy. Neurology. 2019 Apr 30;92(18):e2109-e2117. doi: 10.1212/WNL.0000000000006932. Epub 2019 Jan 25.

ReferenceCitation: Tandon PK, Kakkis ED. The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases. Orphanet J Rare Dis. 2021 Apr 19;16(1):183. doi: 10.1186/s13023-021-01805-5. Review.

lead_sponsor: Ultragenyx Pharmaceutical Inc

location_country:
United Kingdom
Italy
Canada
United States
Israel
France
Bulgaria

location_facility: Washington University School of Medicine

location_facility: Institut de Myologie GH Pitié-Salpêtrière

location_facility: Hadassah-Hebrew University Medical Center


title: Etude Prospective Multicentrique de l'aménorrhée Chimio-induite et de sa réversibilité Dans le Cancer du Sein Chez Des Patientes préménopausées Recevant Une chimiothérapie Adjuvante ou néoadjuvante. Etude Améno-Chimio.

acronym: chemo-ameno

summary: Relation between chemotherapy and amennorhea

lead_sponsor: Cliniques universitaires Saint-Luc- Université Catholique de Louvain

location_country: France Belgium

location_facility:
Institut Claudius Renaud
Centre Hospitalier Université de Liège
Clinique Saint Vincent
Cliniques Universitaires Saint-Luc
CHR Citadelle
Centre hospitalier Peltzer La Tourelle