title: A Prospective Short-term Study to Evaluate Methodologies for the Assessment of Disease Extent, Impact, and Wound Evolution in Patients With Dystrophic Epidermolysis Bullosa (DEB)

summary: The purpose of this study is to better understand disease extent and to identify appropriate methodologies to evaluate (dystrophic epidermolysis bullosa) DEB in a quantitative and qualitative manner.

ReferenceCitation: Paller AS, Pope E, Rudin D, Malyala A, Ramsdell D, Johnson R, Landy H, Murrell DF; DEB Investigators. A prospective short-term study to evaluate methodologies for the assessment of disease extent, impact, and wound evolution in patients with dystrophic epidermolysis bullosa. Orphanet J Rare Dis. 2022 Aug 13;17(1):314. doi: 10.1186/s13023-022-02461-z.

lead_sponsor: Shire

location_country:
Germany
Italy
Australia
Austria
United States
Czechia
France

location_facility: Service de Génétique et INSERM UMR 1163, Laboratoire de Génétique des maladies cutanées - Institut Imagine

location_facility: Cincinnati Childrens Hospital Medical Center

location_facility: Istituto Dermopatico Dell'immacolata IRCCS

location_facility: Ann and Robert H Lurie Childrens Hospital of Chicago


type: EudraCT Number

title: Phase III, Randomized, Double-Blind, Double-Dummy, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy (Induction of Remission) and Safety of Etrolizumab Compared With Adalimumab and Placebo in Patients With Moderate to Severe Ulcerative Colitis Who Are Naive to TNF Inhibitors

acronym: HIBISCUS II

summary: This Phase III, double-blind, placebo and active-comparator controlled, multicenter study will investigate the efficacy and safety of etrolizumab in induction of remission in participants with moderately to severely active ulcerative colitis (UC) who are naIve to tumor necrosis factor (TNF) inhibitors and refractory to or intolerant of prior immunosuppressant and/or corticosteroid treatment. In addition to this study, a second Phase III trial with identical study design (GA28948; NCT02163759) was independently conducted.

study_start_date_type: Actual

status: Completed

study_completion_date_type: Actual

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Actual

ReferenceType: derived

ReferenceCitation: Rubin DT, Dotan I, DuVall A, Bouhnik Y, Radford-Smith G, Higgins PDR, Mishkin DS, Arrisi P, Scalori A, Oh YS, Tole S, Chai A, Chamberlain-James K, Lacey S, McBride J, Panes J; HIBISCUS Study Group. Etrolizumab versus adalimumab or placebo as induction therapy for moderately to severely active ulcerative colitis (HIBISCUS): two phase 3 randomised, controlled trials. Lancet Gastroenterol Hepatol. 2022 Jan;7(1):17-27. doi: 10.1016/S2468-1253(21)00338-1. Epub 2021 Nov 17. Erratum In: Lancet Gastroenterol Hepatol. 2022 Apr;7(4):e8.

oa_host_type: closed

repositories_concat: closed

publisher_dissemination: Elsevier

ReferenceCitation: Sandborn WJ, Vermeire S, Tyrrell H, Hassanali A, Lacey S, Tole S, Tatro AR; Etrolizumab Global Steering Committee. Etrolizumab for the Treatment of Ulcerative Colitis and Crohn's Disease: An Overview of the Phase 3 Clinical Program. Adv Ther. 2020 Jul;37(7):3417-3431. doi: 10.1007/s12325-020-01366-2. Epub 2020 May 22.

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via crossref license)

host_type: publisher

license: cc-by-nc

license_normalized: cc-by-nc

version: publishedVersion

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

host_type: repository

license: cc-by

license_normalized: cc-by

repository_institution: KU Leuven - Lirias

repository_normalized: LIRIAS (KU Leuven)

evidence: oa repository (via pmcid lookup)

license_normalized: no license

repository_normalized: PubMed Central

repositories:
LIRIAS (KU Leuven)
PubMed Central

repositories_concat: LIRIAS (KU Leuven);PubMed Central

unpaywall_oa_status: hybrid

publisher_dissemination: Springer-Nature

lead_sponsor: Hoffmann-La Roche

location_country:
Poland
New Zealand
Argentina
Australia
Brazil
Turkey
Croatia
Czechia
France
Lithuania
Colombia
Hungary
United States
Latvia
Ukraine
Malaysia
Greece
Bulgaria
Russian Federation

location_facility: Hospital Raja Perempuan Zainab II; Department of Medicine

location_facility: Irkutsk State Medical Academy of Continuing Education

location_facility: Gaziantep University Medical Faculty Sahinbey Educational Research Hospital; Medical Oncology

location_facility: A.Novak Transcarpathian Regional Clinical Hospital

location_facility: Centro de Investigaciones Medicas Mar Del Plata

location_facility: CI Kherson Afanasii and Olha Tropiny City Clinical Hospital

location_facility: GI L.T.Malaya Therapy National Institute of the NAMS of Ukraine

location_facility: Hospital Universitario Walter Cantidio - UFC

location_facility: FSBEI HE "Stavropol State Medical University" of Ministry of Healthcare of Russian Federation

location_facility: Medical Center "Nov Rehabilitatsionen Tsentar", EOOD

location_facility: Niepubliczny Zaklad Opieki Zdrowotnej SONOMED

location_facility: Uniwersyteckie Centrum Kliniczne im. prof. K. Gibinskiego SUM

location_facility: Vilnius University Hospital Santariskiu Clinic, Public Institution; Cardiology

location_facility: BHI of Omsk region Clinical Oncology Dispensary

location_facility: Pavlov First Saint Petersburg State Medical University

location_facility: M.V. Sklifosovskyi Poltava RCH Dept of Gastroenterology HSEIU UMSA

location_facility: FSBI "Scientific Research Institute of Physyology and Basic Medicine" under the SB of RAMS

location_facility: Hospital Universitario Prof Edgar Santos-Ufba; Ambulatorio Magalhaes Neto 3Andar- Dermatologia

location_facility: Klaipeda Seamen's Hospital, Public Institution

location_facility: CI of SRC Sumy RCH Dept of Gasroenterology Sumy SU MI

location_facility: Med Center of International Institute of Clinical Trials LLC; Medical Center "OK!Clinic+"

location_facility: Ankara Diskapi Yildirim Beyazit Training and Research Hospital; Gastroenterology

location_facility: Center of Emergency and Radiation Medicine; Pulmonology

location_facility: SBEI HPE Altai State Medical University of MoH and SD; Out-patient Department

location_facility: SI inst. of Gastroenterology of NAMSU Dept of Stomach & Duodenum Diseases, D&ThN SI DMA of MoHU

location_facility: Pauls Stradins Clinical University Hospital

location_facility: Pusat Perubatan Universiti Kebangsaan Malaysia

location_facility: Hospital of Lithuanian University of Health. Sciences Kaunas Clinics

location_facility: University General Hospital of Heraklion

location_facility: CI of Kyiv RC Kyiv Regional Clinical Hospital

location_facility: Allmedica Badania Kliniczne Sp z o.o. Sp K.

location_facility: Great Lakes Gastroenterology Research, LLC

location_facility: Private Small Enterprise Medical Center Pulse

location_facility: Cotton-O'Neil Clinical Research Center, Digestive Health

location_facility: Haydarpasa Numune Training and Research Hospital; Gastroenterology

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Completed


summary: This open-label extension of the JIGSAW studies (WA28117 [NCT01904279] and WA28118 [NCT01904292]) is designed to evaluate the long-term safety and efficacy of subcutaneous (SC) tocilizumab treatment in participants with polyarticular-course and systemic juvenile idiopathic arthritis (pJIA and sJIA). Participants from the 2 JIGSAW studies will continue to receive 162 milligrams (mg) of SC tocilizumab with treatment schedule according to arthritis subtype and body weight. Participants will receive the treatment until commercial availability of the drug or for a maximum of 5 years, whichever is earlier.

location_facility: Cincinnati Children'S Hospital Medical Center; Division of Rheumatology

location_facility: Universidade Federal de Sao Paulo - UNIFES

location_facility: Bristol Royal Hospital for Children; Rheumatology Department

location_facility: Asklepios Klinik; Zentrum für Allgemeine Pädiatrie und Neonatologie

location_facility: Hospital Ramon y Cajal ; Servicio de Reumatologia

location_facility: Hospital Universitario la Fe: Servicio de Reumatologia Pediatrica

location_facility: University of Utah; Immunology/Rheumatology/Allergy

location_facility: Hospital Universitario Dr. Jose Eleuterio Gonzalez; Pediatria

location_facility: Hospital Infantil de México "Federico Gomez"; Rheumatology

location_facility: Arkansas Children's Hospital Research Institute

location_facility: Hospital de Ninos de la Santisima Trinidad; Hematología

location_facility: Irccs Ospedale Pediatrico Bambin Gesu - Dip. Di Medicina

location_facility: Hackensack University Medical Center; Pediatric Rheumatology

location_facility: Alder Hey Children's NHS Foundation Trust

location_facility: Uniklinikum Freiburg Zentrum für Kinder- und Jugendmedizin; Pädiatrische Infektio- u. Rheumatologie

location_facility: Hospital Sant Joan De Deu; Servicio de Reumatologia Pediatrica

location_facility: Hospital de La Paz; Unidad de Reumatologia Pediatrica

location_facility: SI Sceintific children health center RAMS

location_facility: Royal Children'S Hospital; Paediatric Rheumatology

location_facility: Westmead Hospital; Paediatric Rheumatology

title: Long-Term Extension Study to Evaluate the Safety and Efficacy of Subcutaneous Tocilizumab in Patients With Polyarticular-Course and Systemic Juvenile Idiopathic Arthritis

source: euctr

location_country:
Germany
Italy
United Kingdom
Argentina
Australia
Brazil
Mexico
United States
Spain
Canada
Russian Federation
France


title: Phase 3 Efficacy and Safety Study of CT-P6 and Herceptin as Neoadjuvant and Adjuvant Treatment in Patients With Her2-positive Early Breast Cancer

summary: This study will determine whether CT-P6 and Herceptin are equivalent in patients with early-stage breast cancer undergoing neoadjuvant chemotherapy. Our hypothesis is that the pathologic complete response rate will be equivalent in patients treated with neoadjuvant CT-P6 or Herceptin. Patients will receive 8 cycles of neoadjuvant systemic therapy and up to 10 cycles of therapy in the adjuvant setting.

ReferenceCitation: Stebbing J, Baranau YV, Baryash V, Manikhas A, Moiseyenko V, Dzagnidze G, Zhavrid E, Boliukh D, Pikiel J, Eniu AE, Li RK, Tiangco B, Lee SJ, Kim S. Long-term efficacy and safety of CT-P6 versus trastuzumab in patients with HER2-positive early breast cancer: final results from a randomized phase III trial. Breast Cancer Res Treat. 2021 Aug;188(3):631-640. doi: 10.1007/s10549-021-06240-5. Epub 2021 Jun 20.

repository_institution: Imperial College London - Spiral

repositories_concat: PubMed Central;spiral.imperial.ac.uk

ReferenceCitation: Esteva FJ, Baranau YV, Baryash V, Manikhas A, Moiseyenko V, Dzagnidze G, Zhavrid E, Boliukh D, Stroyakovskiy D, Pikiel J, Eniu AE, Li RK, Rusyn AV, Tiangco B, Lee SJ, Lee SY, Yu SY, Stebbing J. Efficacy and safety of CT-P6 versus reference trastuzumab in HER2-positive early breast cancer: updated results of a randomised phase 3 trial. Cancer Chemother Pharmacol. 2019 Oct;84(4):839-847. doi: 10.1007/s00280-019-03920-4. Epub 2019 Aug 19.

repository_institution: PubMed Central - Europe PMC

repositories_institution:
Imperial College London - Spiral
PubMed Central - Europe PMC

ReferenceCitation: Stebbing J, Baranau Y, Baryash V, Manikhas A, Moiseyenko V, Dzagnidze G, Zhavrid E, Boliukh D, Stroyakovskii D, Pikiel J, Eniu A, Komov D, Morar-Bolba G, Li RK, Rusyn A, Lee SJ, Lee SY, Esteva FJ. CT-P6 compared with reference trastuzumab for HER2-positive breast cancer: a randomised, double-blind, active-controlled, phase 3 equivalence trial. Lancet Oncol. 2017 Jul;18(7):917-928. doi: 10.1016/S1470-2045(17)30434-5. Epub 2017 Jun 4. Erratum In: Lancet Oncol. 2017 Aug;18(8):e433. Lancet Oncol. 2017 Sep;18(9):e510.

oa_host_type: repository

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

version: submittedVersion

unpaywall_oa_status: green

lead_sponsor: Celltrion

sponsor_collaborators:
Celltrion
Nippon Kayaku Co., Ltd.

location_country:
Poland
Argentina
Japan
Bosnia and Herzegovina
Chile
France
Belarus
Portugal
Hungary
Latvia
Ukraine
India
Romania
Peru
Philippines
Italy
Mexico
Spain
South Africa
Taiwan
Georgia
Russian Federation


title: Assessment of Patterns of Patient-reported Outcomes in Adults With Congenital Heart Disease - International Study

acronym: APPROACH-IS

summary: The purpose of this study is to examine the differences in perceived health, psychosocial functioning, behavioral outcomes and quality of life of adults with congenital heart disease who are living in different areas of the world, and how these differences can be understood (e.g., differences in sense of coherence or illness perceptions).

study_type: Observational

time_perspective: Cross-Sectional

ReferenceCitation: Moons P, Luyckx K, Thomet C, Budts W, Enomoto J, Sluman MA, Yang HL, Jackson JL, Khairy P, Cook SC, Chidambarathanu S, Alday L, Oechslin E, Eriksen K, Dellborg M, Berghammer M, Johansson B, Mackie AS, Menahem S, Caruana M, Veldtman G, Soufi A, Fernandes SM, White K, Callus E, Kutty S, Kovacs AH; APPROACH-IS consortium. Patient-reported outcomes in the aging population of adults with congenital heart disease: results from APPROACH-IS. Eur J Cardiovasc Nurs. 2023 May 25;22(4):339-344. doi: 10.1093/eurjcn/zvac057.

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

repository_institution: University Library Bern - BORIS - Bern Open Repository and Information System

repository_institution: KTH Royal Institute of Technology - KTH Publication Database DiVA

publisher_dissemination: Oxford University Press

ReferenceCitation: Lu CW, Wang JK, Yang HL, Kovacs AH, Luyckx K, Ruperti-Repilado FJ, Van De Bruaene A, Enomoto J, Sluman MA, Jackson JL, Khairy P, Cook SC, Chidambarathanu S, Alday L, Oechslin E, Eriksen K, Dellborg M, Berghammer M, Johansson B, Mackie AS, Menahem S, Caruana M, Veldtman G, Soufi A, Fernandes SM, White K, Callus E, Kutty S, Apers S, Moons P; APPROACH-IS consortium, the International Society for Adult Congenital Heart Disease (ISACHD) *. Heart Failure and Patient-Reported Outcomes in Adults With Congenital Heart Disease from 15 Countries. J Am Heart Assoc. 2022 May 3;11(9):e024993. doi: 10.1161/JAHA.121.024993. Epub 2022 Apr 26.

oa_colors: green gold

evidence: oa journal (via doaj)

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

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

repository_institution: Umeå University - DiVA at Umeå University

repositories_concat: DiVA.org;LIRIAS (KU Leuven);PubMed Central;air.unimi.it;boris.unibe.ch;digitalcommons.wustl.edu

unpaywall_oa_status: gold

publisher_dissemination: Wolters Kluwer Health

ReferenceCitation: Fox KR, Hardy RY, Moons P, Kovacs AH, Luyckx K, Apers S, Cook SC, Veldtman G, Fernandes SM, White K, Kutty S, Jackson JL; APPROACH-IS Consortium and the International Society for Adult Congenital Heart Disease (ISACHD). Smoking among adult congenital heart disease survivors in the United States: Prevalence and relationship with illness perceptions. J Behav Med. 2021 Dec;44(6):772-783. doi: 10.1007/s10865-021-00239-5. Epub 2021 Jun 29.

version: acceptedVersion

repositories_concat: LIRIAS (KU Leuven)

ReferenceCitation: Moons P, Luyckx K, Thomet C, Budts W, Enomoto J, Sluman MA, Lu CW, Jackson JL, Khairy P, Cook SC, Chidambarathanu S, Alday L, Eriksen K, Dellborg M, Berghammer M, Johansson B, Mackie AS, Menahem S, Caruana M, Veldtman G, Soufi A, Fernandes SM, White K, Callus E, Kutty S, Ombelet F, Apers S, Kovacs AH; APPROACH-IS Consortium and the International Society for Adult Congenital Heart Disease (ISACHD). Physical Functioning, Mental Health, and Quality of Life in Different Congenital Heart Defects: Comparative Analysis in 3538 Patients From 15 Countries. Can J Cardiol. 2021 Feb;37(2):215-223. doi: 10.1016/j.cjca.2020.03.044. Epub 2020 Apr 6.

ReferenceCitation: Moons P, Apers S, Kovacs AH, Thomet C, Budts W, Enomoto J, Sluman MA, Wang JK, Jackson JL, Khairy P, Cook SC, Chidambarathanu S, Alday L, Oechslin E, Eriksen K, Dellborg M, Berghammer M, Johansson B, Mackie AS, Menahem S, Caruana M, Veldtman G, Soufi A, Fernandes SM, White K, Callus E, Kutty S, Luyckx K; APPROACH-IS consortium and the International Society for Adult Congenital Heart Disease (ISACHD). Sense of coherence in adults with congenital heart disease in 15 countries: Patient characteristics, cultural dimensions and quality of life. Eur J Cardiovasc Nurs. 2021 Feb 11;20(1):48-55. doi: 10.1177/1474515120930496.

repository_institution: Washington University School of Medicine - Digital Commons@Becker

repositories_concat: DiVA.org;LIRIAS (KU Leuven);University of Melbourne - Minerva Access;air.unimi.it;digitalcommons.wustl.edu

ReferenceCitation: Van Bulck L, Goossens E, Luyckx K, Apers S, Oechslin E, Thomet C, Budts W, Enomoto J, Sluman MA, Lu CW, Jackson JL, Khairy P, Cook SC, Chidambarathanu S, Alday L, Eriksen K, Dellborg M, Berghammer M, Johansson B, Mackie AS, Menahem S, Caruana M, Veldtman G, Soufi A, Fernandes SM, White K, Callus E, Kutty S, Moons P; APPROACH-IS consortium and the International Society for Adult Congenital Heart Disease (ISACHD). Healthcare system inputs and patient-reported outcomes: a study in adults with congenital heart defect from 15 countries. BMC Health Serv Res. 2020 Jun 3;20(1):496. doi: 10.1186/s12913-020-05361-9.

repository_institution: University of Cape Town - Open University of Cape Town

repository_institution: University of Antwerp - Institutional Repository University of Antwerp

repositories_concat: DiVA.org;LIRIAS (KU Leuven);PubMed Central;University of Melbourne - Minerva Access;air.unimi.it;irua;localhost

ReferenceCitation: Holbein CE, Veldtman GR, Moons P, Kovacs AH, Luyckx K, Apers S, Chidambarathanu S, Soufi A, Eriksen K, Jackson JL, Enomoto J, Fernandes SM, Johansson B, Alday L, Dellborg M, Berghammer M, Menahem S, Caruana M, Kutty S, Mackie AS, Thomet C, Budts W, White K, Sluman MA, Callus E, Cook SC, Khairy P, Cedars A; APPROACH-IS consortium and the International Society for Adult Congenital Heart Disease (ISACHD). Perceived Health Mediates Effects of Physical Activity on Quality of Life in Patients With a Fontan Circulation. Am J Cardiol. 2019 Jul 1;124(1):144-150. doi: 10.1016/j.amjcard.2019.03.039. Epub 2019 Apr 10.

ReferenceCitation: Apers S, Kovacs AH, Luyckx K, Alday L, Berghammer M, Budts W, Callus E, Caruana M, Chidambarathanu S, Cook SC, Dellborg M, Enomoto J, Eriksen K, Fernandes SM, Jackson JL, Johansson B, Khairy P, Kutty S, Menahem S, Rempel G, Sluman MA, Soufi A, Thomet C, Veldtman G, Wang JK, White K, Moons P; APPROACH-IS consortium; International Society for Adult Congenital Heart Disease (ISACHD). Assessment of Patterns of Patient-Reported Outcomes in Adults with Congenital Heart disease - International Study (APPROACH-IS): rationale, design, and methods. Int J Cardiol. 2015 Jan 20;179:334-42. doi: 10.1016/j.ijcard.2014.11.084. Epub 2014 Nov 8.

location_country:
Italy
India
Norway
Argentina
Australia
Japan
Malta
Netherlands
Belgium
Sweden
United States
Switzerland
Taiwan
Canada
France

location_facility: Washington University and Barnes Jewish Heart & Vascular Center

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: Northern Alberta Adult Congenital Heart Clinic

location_facility: Children's Hospital of Pittsburgh of UPMC


acronym: PROBESE

summary:
Postoperative respiratory failure, particularly after surgery under general anesthesia, adds to the morbidity and mortality of surgical patients. Anesthesiologists inconsistently use positive end-expiratory pressure (PEEP) and recruitment maneuvers in the hope that this may improve oxygenation and protect against postoperative pulmonary complications (PPCs), especially in obese patients. While anesthesiologists tend to use PEEP higher than in non-obese patients. While it is uncertain whether a strategy that uses higher levels of PEEP with recruitment maneuvers truly prevents PPCs in these patients, use of higher levels of PEEP with recruitment maneuvers could compromise intra-operative hemodynamics.

The investigators aim to compare a ventilation strategy using higher levels of PEEP with recruitment maneuvers with one using lower levels of PEEP without recruitment maneuvers in obese patients at an intermediate-to-high risk for PPCs.

We hypothesize that an intra-operative ventilation strategy using higher levels of PEEP and recruitment maneuvers, as compared to ventilation with lower levels of PEEP without recruitment maneuvers, prevents PPCs in obese patients at an intermediate-to-high risk for PPC.

primary_purpose: Prevention

ReferenceCitation: Ellenberger C, Pelosi P, de Abreu MG, Wrigge H, Diaper J, Hagerman A, Adam Y, Schultz MJ, Licker M; PROBESE investigators, of the PROtective VEntilation Network (PROVEnet); Clinical Trial Network of the European Society of Anaesthesiology and Intensive Care (ESAIC). Distribution of ventilation and oxygenation in surgical obese patients ventilated with high versus low positive end-expiratory pressure: A substudy of a randomised controlled trial. Eur J Anaesthesiol. 2022 Nov 1;39(11):875-884. doi: 10.1097/EJA.0000000000001741. Epub 2022 Sep 12.

evidence: open (via page says license)

repositories_concat: PubMed Central

ReferenceCitation: Writing Committee for the PROBESE Collaborative Group of the PROtective VEntilation Network (PROVEnet) for the Clinical Trial Network of the European Society of Anaesthesiology; Bluth T, Serpa Neto A, Schultz MJ, Pelosi P, Gama de Abreu M; PROBESE Collaborative Group; Bluth T, Bobek I, Canet JC, Cinnella G, de Baerdemaeker L, Gama de Abreu M, Gregoretti C, Hedenstierna G, Hemmes SNT, Hiesmayr M, Hollmann MW, Jaber S, Laffey J, Licker MJ, Markstaller K, Matot I, Mills GH, Mulier JP, Pelosi P, Putensen C, Rossaint R, Schmitt J, Schultz MJ, Senturk M, Serpa Neto A, Severgnini P, Sprung J, Vidal Melo MF, Wrigge H. Effect of Intraoperative High Positive End-Expiratory Pressure (PEEP) With Recruitment Maneuvers vs Low PEEP on Postoperative Pulmonary Complications in Obese Patients: A Randomized Clinical Trial. JAMA. 2019 Jun 18;321(23):2292-2305. doi: 10.1001/jama.2019.7505. Erratum In: JAMA. 2019 Nov 12;322(18):1829-1830.

oa_colors: green other

evidence: open (via free pdf)

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

repositories_concat: PubMed Central;iris.unife.it

unpaywall_oa_status: bronze

publisher_dissemination: American Medical Association

ReferenceCitation: Bluth T, Teichmann R, Kiss T, Bobek I, Canet J, Cinnella G, De Baerdemaeker L, Gregoretti C, Hedenstierna G, Hemmes SN, Hiesmayr M, Hollmann MW, Jaber S, Laffey JG, Licker MJ, Markstaller K, Matot I, Muller G, Mills GH, Mulier JP, Putensen C, Rossaint R, Schmitt J, Senturk M, Serpa Neto A, Severgnini P, Sprung J, Vidal Melo MF, Wrigge H, Schultz MJ, Pelosi P, Gama de Abreu M; PROBESE investigators; PROtective VEntilation Network (PROVEnet); Clinical Trial Network of the European Society of Anaesthesiology (ESA). Protective intraoperative ventilation with higher versus lower levels of positive end-expiratory pressure in obese patients (PROBESE): study protocol for a randomized controlled trial. Trials. 2017 Apr 28;18(1):202. doi: 10.1186/s13063-017-1929-0. Erratum In: Trials. 2017 Jun 1;18(1):247.

repository_institution: Harvard University - Digital Access to Scholarship at Harvard (DASH)

repository_institution: Ghent University - Ghent University Academic Bibliography

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

repository_normalized: HAL

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université Paris Descartes

repository_institution: Nova Science Publishers, Inc. - Nova Science Publishers

repository_institution: Augsburg University - OPUS - Augsburg University Publication Server

repository_institution: Uppsala University - Uppsala University Publications

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

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

sponsor_collaborators:
Technische Universität Dresden
European Society of Anaesthesiology

location_country:
Germany
Italy
Hungary
Israel
United Kingdom
Brazil
Netherlands
Austria
Belgium
Sweden
United States
Spain
Switzerland
Turkey
Canada
France

location_facility: Hospital Universitari Germans Trias I Pujol

location_facility: Massachusetts General Hospital, Harvard University

location_facility: Saint Michael's Hospital, University of Toronto

location_facility: Academic Medical Center, University of Amsterdam

location_facility: Department of Anesthesiology and Intensive Care, University Hospital Carl Gustav Carus


title: Radium-223 Alpha Emitter Agent in Non-intervention Safety Study in mCRPC popUlation for Long-teRm Evaluation

acronym: REASSURE

summary: Observational study in the routine clinical practice setting to evaluate the short and long term safety profile of Radium-223 in metastatic castration resistant prostate cancer patients and to evaluate the risk of developing second primary cancers.

status: Active, not recruiting

study_completion_date_type: Anticipated

time_perspective: Prospective

ReferenceCitation: Sartor O, Fougere C, Essler M, Ezziddin S, Kramer G, Ellinger J, Nordquist L, Sylvester J, Paganelli G, Peer A, Bogemann M, Meltzer J, Sandstrom P, Verholen F, Song DY. 177Lu-Prostate-Specific Membrane Antigen Ligand After 223Ra Treatment in Men with Bone-Metastatic Castration-Resistant Prostate Cancer: Real-World Clinical Experience. J Nucl Med. 2022 Mar;63(3):410-414. doi: 10.2967/jnumed.121.262240. Epub 2021 Jun 24. Erratum In: J Nucl Med. 2022 Jul;63(7):100-1000.

publisher_dissemination: Society of Nuclear Medicine

lead_sponsor: Bayer

location_country:
Germany
Argentina
Luxembourg
Czechia
Canada
France
Colombia
Portugal
Denmark
United States
Belgium
Italy
United Kingdom
Sweden
Netherlands
Austria
Mexico
Spain
Greece
Israel

status_simplified: Ongoing


lead_sponsor: Sucampo Pharma Americas, LLC

summary:
This study is for children with constipation.

Children who completed 3 months of treatment in the earlier study (NCT02042183):

were invited to participate
will receive lubiprostone for 9 more months
will see if lubiprostone safely relieves their constipation if taken for a whole year

ReferenceCitation: Benninga MA, Hussain SZ, Sood MR, Nurko S, Hyman P, Clifford RA, O'Gorman M, Losch-Beridon T, Mareya S, Lichtlen P, Di Lorenzo C. Lubiprostone for Pediatric Functional Constipation: Randomized, Controlled, Double-Blind Study With Long-term Extension. Clin Gastroenterol Hepatol. 2022 Mar;20(3):602-610.e5. doi: 10.1016/j.cgh.2021.04.005. Epub 2021 Apr 7.

oa_host_type: publisher

sponsor_collaborators:
Sucampo Pharma Americas, LLC
Takeda

location_facility: Arizona Children's Center at Maricopa Medical Center

location_facility: Pediatric Gastroenterology Carilion Medical Center

location_facility: University of Louisville Research Foundation Inc. Kosair Charities Pediatric Clinical Research Unit

location_facility: Coastal Pediatrics Associates - Mount Pleasant

location_facility: MultiCare Health System Institute for Research and Innovation

location_facility: University of South Alabama Women's and Children's Hospital

location_facility: UnityPoint Health Des Moines/ Blank Children's Clinic

location_facility: University of Oklahoma Health Sciences Center

location_facility: Chelsea and Westminster Hospital NHS Foundation Trust

location_facility: Morgan Stanley Children's Hospital of New York Presbyterian

location_facility: St. Christopher's Hospital for Children Drexel University

location_facility: Coastal Pediatric Associates - James Island

location_facility: Pediatric Research of Charlottesville, LLC

location_facility: INOVA Pediatric Digestive Diseases Center

location_facility: Nemours/Alfred I. duPont Hospital for Children

location_facility: Arkansas Childrens Hospital Research Institute

location_facility: Children's Center for Digestive Health Care/GI Care for Kids

location_facility: University of Mississippi Medical Center

title: A Multicentre, Long-term Safety, Efficacy, and Pharmacokinetics Study of Lubiprostone in Paediatric Subjects Aged ≥ 6 Years to < 18 Years With Functional Constipation

location_country:
Poland
United Kingdom
Netherlands
Belgium
United States
Canada
France

ipd_sharing: No


title: A Natural History Study to Observe Disease Progression, Standard of Care and Investigate Biomarkers in Alport Syndrome Patients

summary:
There is limited published clinical data about the natural history of renal disease in Alport syndrome. The RG012-01 study will collect data to characterize the progression of renal dysfunction in Alport syndrome patients.

Patients with a confirmed diagnosis of Alport syndrome who have qualifying GFR will be considered for enrollment. The sequential sampling of subjects' urine and/or blood will allow an assessment of the rate of change of established clinical endpoints, such as GFR and/or the rate of change of other renal biomarkers (proteinuria and β-2 microglobulin) in subjects whose renal function is steadily declining. The identification of surrogate markers that track the decline of renal function and could correlate with time to end-stage renal disease (ESRD) is a key goal of the natural history study.

lead_sponsor: Genzyme, a Sanofi Company

location_country:
Germany
United Kingdom
Australia
United States
Canada
France


title: A Phase 1 Study to Evaluate the Safety and Tolerability of MEDI4736 as Monotherapy or in Combination With Tremelimumab With or Without Azacitidine in Subjects With Myelodysplastic Syndrome After Treatment With Hypomethylating Agents

summary: This is a multicenter, open-label, Phase 1 study to assess the safety and antitumor activity of MEDI4736 as Monotherapy or in Combination with Tremelimumab with or without Azacitidine in Subjects with myelodysplastic syndrome after treatment with hypomethylating agents

design_allocation: Non-Randomized

lead_sponsor: MedImmune LLC

location_country:
United States
United Kingdom
Germany
France


title: Fractional Flow Reserve Versus Angiography for Multivessel Evaluation (FAME) 3 Trial A Comparison of Fractional Flow Reserve-Guided Percutaneous Coronary Intervention and Coronary Artery Bypass Graft Surgery in Patients With Multivessel Coronary Artery Disease

acronym: FAME 3

summary: The purpose of this study is to determine whether Fractional flow reserve (FFR, (coronary pressure wire-based index for assessing the ischemic potential of a coronary lesion)-guided percutaneous coronary intervention (PCI) in patients with multivessel coronary artery disease (CAD) will result in similar outcomes to coronary artery bypass graft surgery (CABG).

ReferenceType: background

ReferenceCitation: Zimmermann FM, De Bruyne B, Pijls NH, Desai M, Oldroyd KG, Park SJ, Reardon MJ, Wendler O, Woo J, Yeung AC, Fearon WF. Rationale and design of the Fractional Flow Reserve versus Angiography for Multivessel Evaluation (FAME) 3 Trial: a comparison of fractional flow reserve-guided percutaneous coronary intervention and coronary artery bypass graft surgery in patients with multivessel coronary artery disease. Am Heart J. 2015 Oct;170(4):619-626.e2. doi: 10.1016/j.ahj.2015.06.024. Epub 2015 Jul 9.

ReferenceCitation: Zimmermann FM, De Bruyne B, Pijls NHJ, Desai M, Oldroyd KG, Reardon MJ, Wendler O, Woo J, Yeung AC, Fearon WF. A protocol update of the Fractional Flow Reserve versus Angiography for Multivessel Evaluation (FAME) 3 trial: A comparison of fractional flow reserve-guided percutaneous coronary intervention and coronary artery bypass graft surgery in patients with multivessel coronary artery disease. Am Heart J. 2019 Aug;214:156-157. doi: 10.1016/j.ahj.2019.04.012. Epub 2019 Apr 29. No abstract available.

ReferenceType: result

ReferenceCitation: Fearon WF, Zimmermann FM, De Bruyne B, Piroth Z, van Straten AHM, Szekely L, Davidavicius G, Kalinauskas G, Mansour S, Kharbanda R, Ostlund-Papadogeorgos N, Aminian A, Oldroyd KG, Al-Attar N, Jagic N, Dambrink JE, Kala P, Angeras O, MacCarthy P, Wendler O, Casselman F, Witt N, Mavromatis K, Miner SES, Sarma J, Engstrom T, Christiansen EH, Tonino PAL, Reardon MJ, Lu D, Ding VY, Kobayashi Y, Hlatky MA, Mahaffey KW, Desai M, Woo YJ, Yeung AC, Pijls NHJ; FAME 3 Investigators. Fractional Flow Reserve-Guided PCI as Compared with Coronary Bypass Surgery. N Engl J Med. 2022 Jan 13;386(2):128-137. doi: 10.1056/NEJMoa2112299. Epub 2021 Nov 4.

evidence: open (via free article)

repository_institution: Elsevier - Scopus

repository_normalized: Pure (Denmark)

repositories_concat: Pure (Denmark)

publisher_dissemination: Massachusetts Medical Society

ReferenceCitation: Piroth Z, Otsuki H, Zimmermann FM, Ferenci T, Keulards DCJ, Yeung AC, Pijls NHJ, De Bruyne B, Fearon WF. Prognostic Value of Measuring Fractional Flow Reserve After Percutaneous Coronary Intervention in Patients With Complex Coronary Artery Disease: Insights From the FAME 3 Trial. Circ Cardiovasc Interv. 2022 Nov;15(11):884-891. doi: 10.1161/CIRCINTERVENTIONS.122.012542. Epub 2022 Sep 19.

ReferenceCitation: Fearon WF, Zimmermann FM, Ding VY, Zelis JM, Piroth Z, Davidavicius G, Mansour S, Kharbanda R, Ostlund-Papadogeorgos N, Oldroyd KG, Wendler O, Reardon MJ, Woo YJ, Yeung AC, Pijls NHJ, De Bruyne B, Desai M, Hlatky MA; FAME 3 Investigators. Quality of Life After Fractional Flow Reserve-Guided PCI Compared With Coronary Bypass Surgery. Circulation. 2022 May 31;145(22):1655-1662. doi: 10.1161/CIRCULATIONAHA.122.060049. Epub 2022 Apr 2.

lead_sponsor: Stanford University

collaborators: VZW Cardiovascular Research Center Aalst

collaborators: Houston Methodist DeBakey Heart and Vascular Center, Houston

location_country:
Lithuania
New Zealand
Hungary
Norway
Denmark
Australia
Sweden
Netherlands
United Kingdom
Belgium
United States
Korea, Republic of
Czechia
Canada
Serbia
France

location_facility: Le'Centre Hospitalier de l'Universite de Montreal

location_facility: University Hospitals Coventry and Warwickshire

location_facility: Masaryk University and University Hospital Brno

location_facility: Southampton University Hospitals NHS Trust

location_facility: Vilnius University Hospital Santariskiu Klinikos

location_facility: Karolinska Institutet, Dep of clinical science and education, Södersjukhuset

intervention_type: Procedure

lead_sponsor_type: academique


title: An Open-Label, Non-randomised, Parallel Group, Multicentre, Phase I Study to Assess the Safety and Effect of Olaparib at Steady State on the Pharmacokinetics of the Anti-hormonal Agents Anastrozole, Letrozole and Tamoxifen at Steady State, and the Effect of the Anti-hormonal Agents on Olaparib, Following Administration in Patients With Advanced Solid Cancer

summary: This is an open-label 2-part Phase I study in patients with advanced solid tumours. Part A of the study (mandatory) will assess the effect of olaparib on the pharmacokinetics (PK) of anastrozole, letrozole and tamoxifen and vice versa; Part B will allow patients (if eligible) continued access to olaparib after the PK phase and will provide additional safety data.

primary_purpose: Basic Science

ReferenceCitation: Plummer R, Verheul HM, De Vos FYFL, Leunen K, Molife LR, Rolfo C, Grundtvig-Sorensen P, De Greve J, Rottey S, Jerusalem G, Italiano A, Spicer J, Dirix L, Goessl C, Birkett J, Spencer S, Learoyd M, Bailey C, Dean E. Pharmacokinetic Effects and Safety of Olaparib Administered with Endocrine Therapy: A Phase I Study in Patients with Advanced Solid Tumours. Adv Ther. 2018 Nov;35(11):1945-1964. doi: 10.1007/s12325-018-0804-z. Epub 2018 Oct 15.

lead_sponsor: AstraZeneca

location_country:
United Kingdom
Denmark
Netherlands
Belgium
France


type: Other Identifier

title: EXPERT, EXPosurE Registry RiociguaT in Patients With Pulmonary Hypertension

acronym: EXPERT

summary: In accordance with the regulatory guidance this registry has been designed to collect information about the long-term safety of Adempas in real clinical practice outside the regulated environment of a controlled clinical study.

location_country:
Finland
Germany
Ireland
Argentina
Australia
Luxembourg
Turkey
Czechia
Canada
France
Colombia
Portugal
Slovakia
Denmark
Switzerland
Belgium
Estonia
Italy
Norway
United Kingdom
Netherlands
Austria
Saudi Arabia
Spain
Greece
Taiwan
Russian Federation
Sweden


title: An Open-Label, Single-Group Clinical Study to Evaluate the Pharmacokinetics, Safety,and Efficacy of Omacetaxine Mepesuccinate Given Subcutaneously as a Fixed Dose inPatients With Chronic Phase or Accelerated Phase Chronic Myeloid Leukemia Who Have Failed 2 or More Tyrosine Kinase Inhibitor Therapies

summary: To satisfy a postmarketing requirement, the sponsor has been requested to conduct a Phase 1/Phase 2 single-group clinical study to investigate the pharmacokinetics and preliminary safety and efficacy of omacetaxine following a fixed-dose administration to patients with CP or AP CML who have failed 2 or more tyrosine kinase inhibitor (TKI) therapies.

status: Terminated

primary_purpose: Other

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

location_country:
United States
Korea, Republic of
Belgium
France

location_facility:
Teva Investigational Site 12550
Teva Investigational Site 12543
Teva Investigational Site 12544
Teva Investigational Site 87026
Teva Investigational Site 12547
Teva Investigational Site 12545
Teva Investigational Site 37048
Teva Investigational Site 35157
Teva Investigational Site 37047
Teva Investigational Site 12546

status_simplified: Unknown


title: A Phase 1, Multicenter, Open-Label, Dose-Escalation and Expansion, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-120 in Subjects With Advanced Solid Tumors, Including Glioma, With an IDH1 Mutation

summary: The purpose of this Phase I, multicenter study is to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-120 in advanced solid tumors, including glioma, that harbor an IDH1 mutation. The first portion of the study is a dose escalation phase where cohorts of patients will receive ascending oral doses of AG-120 to determine maximum tolerated dose (MTD) and/or the recommended Phase II dose. The second portion of the study is a dose expansion phase where four arms of patients will receive AG-120 to further evaluate the safety, tolerability, and clinical activity of the recommended Phase II dose. Anticipated time on study treatment is until disease progression, unacceptable toxicity occurs or at Investigator discretion.

enrollment_type: Anticipated

ReferenceCitation: Ellingson BM, Kim GHJ, Brown M, Lee J, Salamon N, Steelman L, Hassan I, Pandya SS, Chun S, Linetsky M, Yoo B, Wen PY, Mellinghoff IK, Goldin J, Cloughesy TF. Volumetric measurements are preferred in the evaluation of mutant IDH inhibition in non-enhancing diffuse gliomas: Evidence from a phase I trial of ivosidenib. Neuro Oncol. 2022 May 4;24(5):770-778. doi: 10.1093/neuonc/noab256.

repository_institution: California Digital Library - eScholarship

repositories_concat: PubMed Central;escholarship.org

ReferenceCitation: Aguado-Fraile E, Tassinari A, Ishii Y, Sigel C, Lowery MA, Goyal L, Gliser C, Jiang L, Pandya SS, Wu B, Bardeesy N, Choe S, Deshpande V. Molecular and morphological changes induced by ivosidenib correlate with efficacy in mutant-IDH1 cholangiocarcinoma. Future Oncol. 2021 Jun;17(16):2057-2074. doi: 10.2217/fon-2020-1274. Epub 2021 Mar 12.

publisher_dissemination: Future Science Ltd

ReferenceCitation: Mellinghoff IK, Ellingson BM, Touat M, Maher E, De La Fuente MI, Holdhoff M, Cote GM, Burris H, Janku F, Young RJ, Huang R, Jiang L, Choe S, Fan B, Yen K, Lu M, Bowden C, Steelman L, Pandya SS, Cloughesy TF, Wen PY. Ivosidenib in Isocitrate Dehydrogenase 1-Mutated Advanced Glioma. J Clin Oncol. 2020 Oct 10;38(29):3398-3406. doi: 10.1200/JCO.19.03327. Epub 2020 Jun 12.

repository_institution: PubMed Central - Europe PMC

publisher_dissemination: American Society of Clinical Oncology (ASCO)

ReferenceCitation: Tap WD, Villalobos VM, Cote GM, Burris H, Janku F, Mir O, Beeram M, Wagner AJ, Jiang L, Wu B, Choe S, Yen K, Gliser C, Fan B, Agresta S, Pandya SS, Trent JC. Phase I Study of the Mutant IDH1 Inhibitor Ivosidenib: Safety and Clinical Activity in Patients With Advanced Chondrosarcoma. J Clin Oncol. 2020 May 20;38(15):1693-1701. doi: 10.1200/JCO.19.02492. Epub 2020 Mar 24.

ReferenceCitation: Lowery MA, Burris HA 3rd, Janku F, Shroff RT, Cleary JM, Azad NS, Goyal L, Maher EA, Gore L, Hollebecque A, Beeram M, Trent JC, Jiang L, Fan B, Aguado-Fraile E, Choe S, Wu B, Gliser C, Agresta SV, Pandya SS, Zhu AX, Abou-Alfa GK. Safety and activity of ivosidenib in patients with IDH1-mutant advanced cholangiocarcinoma: a phase 1 study. Lancet Gastroenterol Hepatol. 2019 Sep;4(9):711-720. doi: 10.1016/S2468-1253(19)30189-X. Epub 2019 Jul 9.

ReferenceCitation: Fan B, Mellinghoff IK, Wen PY, Lowery MA, Goyal L, Tap WD, Pandya SS, Manyak E, Jiang L, Liu G, Nimkar T, Gliser C, Prahl Judge M, Agresta S, Yang H, Dai D. Clinical pharmacokinetics and pharmacodynamics of ivosidenib, an oral, targeted inhibitor of mutant IDH1, in patients with advanced solid tumors. Invest New Drugs. 2020 Apr;38(2):433-444. doi: 10.1007/s10637-019-00771-x. Epub 2019 Apr 26.

repository_institution: Dr Steevens Hospital Library - Lenus, The Irish Health Repository

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.

lead_sponsor: Institut de Recherches Internationales Servier


title: A Double-blind, Placebo-controlled, Parallel-group, Multicenter Study Of The Efficacy And Safety Of Pregabalin As Adjunctive Therapy In Children 1 Month Through <4 Years Of Age With Partial Onset Seizures

summary: This study is designed to evaluate the effectiveness of 2 doses of pregabalin to reduce seizure frequency as an add on therapy in pediatric subjects 1 month to <4 years of age with refractory partial onset seizures. It is hypothesized that both doses of pregabalin will demonstrate superior efficacy when compared to placebo by reducing the partial onset seizure frequency and that pregabalin will be safe and well tolerated.

ReferenceCitation: Mann D, Antinew J, Knapp L, Almas M, Liu J, Scavone J, Yang R, Modequillo M, Makedonska I, Ortiz M, Kyrychenko A, Nordli D, Farkas V, Farkas MK; A0081042 study group. Pregabalin adjunctive therapy for focal onset seizures in children 1 month to <4 years of age: A double-blind, placebo-controlled, video-electroencephalographic trial. Epilepsia. 2020 Apr;61(4):617-626. doi: 10.1111/epi.16466. Epub 2020 Mar 18.

publisher_dissemination: Wiley

lead_sponsor: Pfizer's Upjohn has merged with Mylan to form Viatris Inc.

location_country:
Germany
Bosnia and Herzegovina
Thailand
Turkey
Serbia
France
Belarus
Hungary
United States
Ukraine
Romania
Lebanon
Belgium
China
Malaysia
Philippines
Korea, Republic of
Spain
Greece
Taiwan
Bulgaria
Russian Federation
Israel

location_facility: Semmelweis Egyetem, I. Sz. Gyermekgyogyaszati Klinika

location_facility: Siriraj Hospital, Faculty of Medicine, Mahidol University

location_facility: Institute for Child and Youth Healthcare of Vojvodina

location_facility: Karadeniz Teknik Universitesi Tip Fakultesi Farabi Hastanesi

location_facility: Komunalnyi zaklad "Dnipropetrovska dytiacha miska klinichna likarnia #5"

location_facility: The University of Texas Health Science Center at Houston

location_facility: Ivano-Frankivska oblasna dytiacha klinichna likarnia

location_facility: American University of Beirut Medical Center

location_facility: Spitalul Clinic de Urgente pentru Copii "Sf. Maria"

location_facility: Chang Gung Memorial Hospital- Kaohsiung branch

location_facility: Komunalnyi zaklad "Dnipropetrovska oblasna dytiacha klinichna likarnia"

location_facility: University Clinical Centre of the Republic of Srpska

location_facility: Dr. Kenessey Albert Korhaz es Rendelointezet

location_facility: The First Bethune Hospital of Jilin University

location_facility: Magyarorszagi Reformatus Egyhaz Bethesda Gyermekkorhaz, Gyermekneurologia

location_facility: Pediatric Epilepsy & Neurology Specialists, PA

location_facility: Saint George Hospital - University Medical Center

location_facility: St. Petersburg State Pediatric Medical University

location_facility: Izmir Tepecik Training and Research Hospital

location_facility: RSBHI Smolensk Regional Clinical Hospital

location_facility: Pharmacy of The E. Wolfson Medical Center

location_facility: Perm State Medical University n. a. acad. E.A. Vagner

location_facility: Pediatric Epilepsy Center of Central Florida

location_facility: Eskisehir Osmangazi Universitesi Tip Fakultesi

location_facility: FSFEI HE N.I. Pirogov RNRMU of Minzdrav of Russia

location_facility: Mother and Child Healthcare Institute Dr Vukan Cupic

location_facility: Oblasnyi klinichnyi tsentr neirokhirurhii ta nevrolohii,viddilennia neirokhirurhii #2

location_facility: Pharmacy of Tel Aviv Sourasky Medical Center

location_facility: General Hospital of Thessaloniki Ippokratio

location_facility: SHI Ulyanovsk Regional Children's Clinical Hospital n. a. Y.F.Goryachev

location_facility: DZ "Ukrainskyi medychnyi tsentr reabilitatsii ditei z orhanichnym urazhenniam

location_facility: Szent János Kórház es Észak Budai Egyesitett Kórházak

location_facility: GU Republican Scientific and Practical Center Mother and Child


title: A Prospective, Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel-group, 12-week Study to Evaluate the Safety and Tolerability of Macitentan in Subjects With Combined Pre- and Post-capillary Pulmonary Hypertension (CpcPH) Due to Left Ventricular Dysfunction

summary: Study to evaluate if macitentan is safe and tolerable enough to be used for treatment of subjects with combined pre- and post-capillary pulmonary hypertension (CpcPH) due to left ventricular dysfunction.

lead_sponsor: Actelion

location_country:
Germany
Italy
Israel
Switzerland
Austria
Belgium
United States
Spain
Czechia
Canada
France

location_facility: Universitätsklinik Schleswig-Holstein Campus Kiel Klinik für Innere Medizin III Kardiologie und Angiologie

location_facility: University Hospital Gasthuisberg / Interne Geneeskunde - I.G. Pneumologie

location_facility: FN Olomouc, 1. Interní klinika - kardiologická

location_facility: Krankenhaus der Elisabethinen Linz, 2. Interne Abteilung

location_facility: A.O. Universitaria Policlinico S. Orsola-Malpighi - Dipartimento di Medicina Specialistica, Diagnostica e Sperimentale - Unità Operativa di Cardiologia

location_facility: IKEM (Institut klinické a experimentální medicíny, Institute for Clinical and Experimental Medicine)

location_facility: Hôpital Charles Nicolle Service de Cardiologie

location_facility: Hôpital Erasme, Cliniques Universitaires de Bruxelles, Cardiologie

location_facility: Hospital Reina Sofia Servicio de Cardiologia

location_facility: The Chaim Sheba Medical Center / The Institute of Pulmonology, Physiology and Exercise

location_facility: DRK Klinken Berlin Kopenick Klinik für Innere Medizin Kardiologie

location_facility: Medical University of Vienna and AKH Cardiology

location_facility: Universitätsklinikum Köln Herzzentrum / Klinik III für Innere Medizin (Kardiologie, Pneumologie, Angiologie und Intensivmedizin)

location_facility: Klinikum der Universität München Medizinische Klinik und Poliklinik 1 - Großhadern Schwerpunkt Pneumologie

location_facility: Institute of Pulmonology Hadassah Medical Centre : Ein Karem

location_facility: Hospital Universitario 12 Octubre Servicio de Cardiología

location_facility: Lékařská fakulta a Všeobecná fakultní nemocnice v Praze, II. Interní klinika kardiologie a angiologie

location_facility: Houston Methodist Hospital - Heart Failure/Pulm Hypertension

location_facility: University of Michigan Internal Medicine Cardiology, Pulmonary Hypertension Program

location_facility: FN Brno-Bohunice, I. interní kardiologická klinika

location_facility: Hospital Vall d´Hebron Servicio de Cardiologia

location_facility: Universitätsklinik für Kardiologie Schweizer Herz- und Gefässzentrum Bern

location_facility: Washington University School of Medicine - Center for Advanced Med

location_facility: Centre Hospitalier Universitaire Vaudois Service de Cardiologie

location_facility: Kaplan Medical Centre / Pulmonary Institute and Department of Medicine


lead_sponsor: Galapagos NV

summary:
The primary objective of the study is to evaluate the long-term safety and tolerability of filgotinib (formerly GLPG0634) for the treatment of rheumatoid arthritis.

Participants will be enrolled in this open-label long-term follow-up study after they have completed one of the two core studies, GLPG0634-CL-203 (DARWIN1) or GLPG0634-CL-204 (DARWIN2), and will be evaluated for any side effects that may occur (long-term safety and tolerability) when taking filgotinib. During the course of the study, participants will also be examined for long-term effects of filgotinib administration on disease activity (efficacy), subjects' disability, fatigue, and quality of life.

ReferenceCitation: Combe B, Besuyen R, Gomez-Centeno A, Matsubara T, Sancho Jimenez JJ, Yin Z, Buch MH. Geographic Analysis of the Safety and Efficacy of Filgotinib in Rheumatoid Arthritis. Rheumatol Ther. 2023 Feb;10(1):35-51. doi: 10.1007/s40744-022-00494-1. Epub 2022 Oct 7.

repository_institution: University of Illinois at Chicago - INDIGO

repositories_concat: PubMed Central;figshare.com

ReferenceCitation: Kavanaugh A, Westhovens RR, Winthrop KL, Lee SJ, Tan Y, An D, Ye L, Sundy JS, Besuyen R, Meuleners L, Stanislavchuk M, Spindler AJ, Greenwald M, Alten R, Genovese MC. Safety and Efficacy of Filgotinib: Up to 4-year Results From an Open-label Extension Study of Phase II Rheumatoid Arthritis Programs. J Rheumatol. 2021 Aug;48(8):1230-1238. doi: 10.3899/jrheum.201183. Epub 2021 Feb 1.

publisher_dissemination: The Journal of Rheumatology

sponsor_collaborators:
Galapagos NV
Gilead Sciences

location_facility: Spitalul Clinic Judetean de Urgenta Sf. Apostol Andrei Galati

location_facility: GBOU VPO Orenburg State Medical University

location_facility: NZOZ "Nasz Lekarz" Pratyka Grupowa Lekarzy Rodzinnychz

location_facility: CIMeL Centro dee Investigacion Medico Lanus

location_facility: First Moscow State Medical University n.a. I.M. Sechenova of the Ministry of Health

location_facility: Municipal Non-Profit Institution Consultative and Diagnostic Centre of Desnyasky District of Kyiv

location_facility: Vinnitsya Regional Clinical Hospital Named after M.I.Pirogov, Rheumatology Department

location_facility: Specjalisty Czne Centrum Medyczne Nowomed

location_facility: L.T. Malaya Therapy Institute of National Academy of Medical Sciences of Ukraine

location_facility: Scientific Research Institute of Rheumatology

location_facility: Powiatowy Zakrad Opieki Zdrowotnej w Starachowicach

location_facility: Complejo Hospitalario Universitario A Coruña (CHUAC)

location_facility: Hospital Regional "Guillermo Grant Benavente"

location_facility: Clinica Medica Especializada en Reumatologia

location_facility: University "Multiprofile Hospital for Active Treatment - Kaspela" LTD

location_facility: Vladimir Regional State Instituion of Healthcare

location_facility: Communal Institution of Healthcare - Kharkiv City Clinical Hospital #13

location_facility: Schlossparkklinik - Akad. Lehrkrankenhaus Charite

location_facility: Kharkiv Medical Academy of Postgraduate Education, Department of Cardiology

location_facility: Municipal Non-profit Enterprise Consultative and Diagnostic Center of Pechersk District of Kiev city

location_facility: Organizacion Medica de Investigaciones (OMI)

location_facility: Kherson City Clinical Hospital N. A. Afanasii and Olga Tropin

location_facility: Centro de Investigacion en Reumatologia y Especialidades Medicas (CIREEM)

location_facility: University Multiprofile Hospital for Active Treatment "SV. Ivan Rilski" EAD, Sofia, Rheumatology Clinic

location_facility: NZOZ "DOBRY LEKARZ" Specjalistyczne Poradnie Lekarskie

location_facility: Centro de Estudios de Investigacion Basica y Clinica, SC

location_facility: MVZ Rheumatologie and Autoimmun Medizin HH GmbH

location_facility: Instituto de Asistencia Reumatologia Integral - IARI

location_facility: Arizona Arthritis & Rheumatology Research PLLC

location_facility: Sanatorio Nuestra Señora de la Esperanza

location_facility: National Multiprofile Transport Hospital "Tsar Boris III," Sofia, Clinical of Internal Diseases

location_facility: Diagnostic Consultative Center "Sveta Anna" LTD

location_facility: Multiprofile Hospital for Active Treatment - Ruse

title: A Multicenter, Open-label, Long-term Follow-up Safety and Efficacy Study of GLPG0634 Treatment in Subjects With Moderately to Severely Active Rheumatoid Arthritis

location_country:
Germany
Poland
New Zealand
Argentina
Australia
Chile
Czechia
France
Colombia
Hungary
United States
Latvia
Ukraine
Romania
Belgium
Moldova, Republic of
Guatemala
Mexico
Spain
Bulgaria
Russian Federation
Israel


title: A Phase 2 Clinical Trial of Nivolumab, or Nivolumab Combinations in Recurrent and Metastatic Microsatellite Instability High (MSI-H) and Non-MSI-H Colon Cancer

summary: The purpose of this study is to examine if Nivolumab by itself, or Nivolumab in combination with other anti-cancer drugs, will result in meaningful tumor size reduction, in participants with colon cancer that has come back or has spread, and who have a specific biomarker in their tumors.

ReferenceCitation: Lenz HJ, Van Cutsem E, Luisa Limon M, Wong KYM, Hendlisz A, Aglietta M, Garcia-Alfonso P, Neyns B, Luppi G, Cardin DB, Dragovich T, Shah U, Abdullaev S, Gricar J, Ledeine JM, Overman MJ, Lonardi S. First-Line Nivolumab Plus Low-Dose Ipilimumab for Microsatellite Instability-High/Mismatch Repair-Deficient Metastatic Colorectal Cancer: The Phase II CheckMate 142 Study. J Clin Oncol. 2022 Jan 10;40(2):161-170. doi: 10.1200/JCO.21.01015. Epub 2021 Oct 12.

ReferenceCitation: Overman MJ, Lonardi S, Wong KYM, Lenz HJ, Gelsomino F, Aglietta M, Morse MA, Van Cutsem E, McDermott R, Hill A, Sawyer MB, Hendlisz A, Neyns B, Svrcek M, Moss RA, Ledeine JM, Cao ZA, Kamble S, Kopetz S, Andre T. Durable Clinical Benefit With Nivolumab Plus Ipilimumab in DNA Mismatch Repair-Deficient/Microsatellite Instability-High Metastatic Colorectal Cancer. J Clin Oncol. 2018 Mar 10;36(8):773-779. doi: 10.1200/JCO.2017.76.9901. Epub 2018 Jan 20.

ReferenceCitation: Overman MJ, McDermott R, Leach JL, Lonardi S, Lenz HJ, Morse MA, Desai J, Hill A, Axelson M, Moss RA, Goldberg MV, Cao ZA, Ledeine JM, Maglinte GA, Kopetz S, Andre T. Nivolumab in patients with metastatic DNA mismatch repair-deficient or microsatellite instability-high colorectal cancer (CheckMate 142): an open-label, multicentre, phase 2 study. Lancet Oncol. 2017 Sep;18(9):1182-1191. doi: 10.1016/S1470-2045(17)30422-9. Epub 2017 Jul 19. Erratum In: Lancet Oncol. 2017 Sep;18(9):e510.

lead_sponsor: Bristol-Myers Squibb

location_country:
Ireland
Italy
Australia
Belgium
United States
Spain
Canada
France

location_facility: Allina Health System dba Virginia PIper Cancer Institute


acronym: ACH

lead_sponsor: BioMarin Pharmaceutical

summary: This is a Phase 2, open-label, sequential cohort dose-escalation study of BMN 111 in children with achondroplasia. The primary objective is to assess the safety and tolerability of daily BMN 111 administered to children with achondroplasia.

ReferenceCitation: Chan ML, Qi Y, Larimore K, Cherukuri A, Seid L, Jayaram K, Jeha G, Fisheleva E, Day J, Huntsman-Labed A, Savarirayan R, Irving M, Bacino CA, Hoover-Fong J, Ozono K, Mohnike K, Wilcox WR, Horton WA, Henshaw J. Pharmacokinetics and Exposure-Response of Vosoritide in Children with Achondroplasia. Clin Pharmacokinet. 2022 Feb;61(2):263-280. doi: 10.1007/s40262-021-01059-1. Epub 2021 Aug 25.

ReferenceCitation: Savarirayan R, Irving M, Bacino CA, Bostwick B, Charrow J, Cormier-Daire V, Le Quan Sang KH, Dickson P, Harmatz P, Phillips J, Owen N, Cherukuri A, Jayaram K, Jeha GS, Larimore K, Chan ML, Huntsman Labed A, Day J, Hoover-Fong J. C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia. N Engl J Med. 2019 Jul 4;381(1):25-35. doi: 10.1056/NEJMoa1813446. Epub 2019 Jun 18.

location_facility: Children's Hospital & Research Center Oakland

location_facility: Guys & St. Thomas NHS Foundation Trust Evelina Hospital

location_facility: Johns Hopkins McKusick - Institute of Genetic Medicine

location_facility: Ann and Robert H. Lurie Childrens Hospital of Chicago

title: A Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children With Achondroplasia

location_country:
United States
United Kingdom
Australia
France


title: A Phase 1 Open-label Study of Safety and Tolerability of MEDI4736 in Subjects With Metastatic or Unresectable Melanoma in Combination With Dabrafenib and Trametinib or With Trametinib Alone

summary: The purpose of this study is to determine the maximum tolerated dose and characterize the safety profile of durvalumab (MEDI4736) in combination with dabrafenib and trametinib or with trametinib alone in participants with metastatic or unresectable melanoma with BRAF-mutation positive or wild-type (WT) BRAF, respectively.

ReferenceCitation: Gordon MS, Lutzky J, Lawrence D, Butler M, Ascierto PA, Hug B, et al. Phase 1 study evaluating safety and tolerability of MEDI4736, an anti-programmed cell death ligand-1 (PD-L1) antibody, in combination with dabrafenib and trametinib or trametinib alone in patients with unresectable or metastatic melanoma. Ann Oncol 2014; 25(suppl_4): iv374-iv393 (abstract 8004).

ReferenceCitation: Ribas A, Butler M, Lutzky J, Lawrence DP, Robert C, Miller W, et al. Phase 1 study combining anti-PD-L1 (MEDI4736) and BRAF (dabrafenib) and/or MEK (trametinib) inhibitors in advanced melanoma. J Clin Oncol 2015; 33 (15_suppl): (abstract 3003).

ReferenceCitation: Ribas A, Algazi A, Ascierto PA, Butler MO, Chandra S, Gordon M, Hernandez-Aya L, Lawrence D, Lutzky J, Miller WH Jr, Campbell KM, Delafont B, Marshall S, Mueller N, Robert C. PD-L1 blockade in combination with inhibition of MAPK oncogenic signaling in patients with advanced melanoma. Nat Commun. 2020 Dec 7;11(1):6262. doi: 10.1038/s41467-020-19810-w. Erratum In: Nat Commun. 2021 Aug 12;12(1):5022.

repository_institution: University of California - eScholarship University of California

location_country:
United States
Italy
Canada
France


title: Multicenter, Open-label Extension Study to Investigate the Long-term Safety and Efficacy of IgPro20 in Maintenance Treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) in Subjects Completing Study IgPro20_3003

summary:
This study is an extension study to the pivotal study IgPro20_3003 (NCT01545076). The purpose of this extension study is to investigate the long-term treatment of CIDP with IgPro20, with regard to safety and efficacy.

Subjects who have completed subcutaneous (SC) Week 25 or were successfully rescued from a CIDP relapse during the SC Treatment Period of pivotal study IgPro20_3003 (NCT01545076) will have the option to receive open-label low-dose IgPro20 (0.2 g/kg bodyweight [bw]) weekly for up to 48 weeks. Subjects relapsing on low-dose IgPro20 will either return to high-dose IgPro20 (0.4 g/kg) immediately or be discontinued, depending on investigator's judgment. Subjects returning to high-dose IgPro20 will continue on high-dose until they have completed a total of 48 weeks of IgPro20 treatment. If subjects do not successfully recover from CIDP relapse within 4 weeks, they will be withdrawn.

The treatment duration will be up to 48 weeks, followed by a completion visit (week 49).

lead_sponsor: CSL Behring

location_country:
Germany
Italy
United Kingdom
Australia
Japan
Netherlands
United States
Spain
Czechia
Canada
France

location_facility:
Site Reference 2760039
Site Reference 3920065
Site Reference 2760052
Site Reference 2760047
Site Reference 3920040
Site Reference 1240007
Site Reference 5280001
Site Reference 7240010
Site Reference 8400182
Site Reference 0360011
Site Reference 8400169
Site Reference 0360017
Site Reference 8400166
Site Reference 3920035
Site Reference 2760094
Site Reference 3920061
Site Reference 2760049
Site Reference 3920037
Site Reference 2030009
Site Reference 2760072
Site Reference 1240009
Site Reference 2030002
Site Reference 2760055
Site Reference 8260019
Site Reference 3800031
Site Reference 8260032
Site Reference 2760054
Site Reference 2760069
Site Reference 8400181
Site Reference 7240011
Site Reference 8400167
Site Reference 3920038
Site Reference 2500022

intervention_type: Biological


title: A Randomized, Placebo-controlled, Double-blind, Parallel Group, Multicenter Study to Investigate the Efficacy and Safety of 5 Fixed Doses of BAY85-3934 Administered Orally in the Correction of Anemia in Pre-dialysis Subjects With Chronic Kidney Disease Not Currently Treated With Erythropoiesis-stimulating Agent in Europe and Asia Pacific

acronym: DIALOGUE 1

summary:
Anaemia is a condition in which blood has a lower than normal number of red blood cells. It can also occur if red blood cells do not contain enough haemoglobin, an oxygen carrying part of blood. Anaemia is common in patients with chronic kidney disease. Healthy kidneys produce a hormone called erythropoietin, which stimulates the bone marrow to produce the proper number of red blood cells needed to carry oxygen to vital organs. Chronic kidney disease is a general term that means that the kidneys are not functioning to their full potential. The study drug, BAY85-3934, is being evaluated as a drug to increase the body's ability to produce erythropoietin.

The purpose of this study is to find out if the study drug, a tablet taken orally, is safe and effective for the treatment of anaemia associated with chronic kidney disease.

The study will enroll 120 patients at multiple locations in Europe, Asia and Australia. Participation will involve a screening visit and between 12 and 14 study visits scheduled over a period of approximately 5 to 7 months. The estimated total duration of study treatment will be 16 weeks. During these scheduled visits patients will undergo a number of procedures to confirm efficacy and safety of the study drug, including measurement of heart rate and blood pressure, physical examination, Electrocardiogram and blood/urine sample collection for laboratory tests.

The study will be conducted at 5 hospitals in the UK. Bayer HealthCare AG is funding this research.

ReferenceCitation: Natale P, Palmer SC, Jaure A, Hodson EM, Ruospo M, Cooper TE, Hahn D, Saglimbene VM, Craig JC, Strippoli GF. Hypoxia-inducible factor stabilisers for the anaemia of chronic kidney disease. Cochrane Database Syst Rev. 2022 Aug 25;8(8):CD013751. doi: 10.1002/14651858.CD013751.pub2.

location_country:
Germany
Italy
Poland
Hungary
Israel
Romania
Australia
Japan
United Kingdom
Korea, Republic of
Spain
Turkey
Bulgaria
France

location_facility:
Baskent University Medical Faculty
Ankara Univ. Medical Faculty
Sifa University Medical Faculty


title: A Phase 1b Study of the Safety and Pharmacology of Atezolizumab (Anti-PD-L1 Antibody) Administered With Erlotinib or Alectinib in Patients With Advanced Non-Small Cell Lung Cancer

summary:
This open-label, multicenter study will assess the safety, tolerability, and pharmacokinetics of intravenous (IV) dosing of atezolizumab in combination with oral erlotinib or alectinib in participants with NSCLC.

This study has two stages. In the erlotinib group, the combination treatment will be given to participants with epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI)-treatment-naive, advanced (nonresectable) NSCLC in a safety-evaluation stage and to participants with previously untreated EGFR mutation-positive, advanced NSCLC in an expansion stage (Stage 2). In the alectinib group, for both the safety-evaluation and expansion stages (Stages 1 and 2), the combination will be given to participants who are treatment-naive with anaplastic lymphoma kinase (ALK)-positive advanced NSCLC.

In Stage 1, erlotinib will be given at a starting dose of 150 milligrams (mg) by mouth (PO) once daily (QD) and the starting dose of alectinib will be 600 mg twice daily (BID), for 28 consecutive days during Cycle 1 and on Days 1 through 21 of each cycle thereafter. The starting dose of atezolizumab will be 1200 mg, administered every 3 weeks (q3W) starting on Day 8 of Cycle 1. If the starting regimen for a combination treatment is not tolerated, alternative doses and/or schedules of erlotinib and atezolizumab or alectinib and atezolizumab may be tested to determine potential recommended Phase 2 dose (RP2D) for that combination treatment. In Stage 2, a potential RP2D and schedule for each combination treatment will be investigated in an expansion cohort.

For both stages, continuation of treatment beyond Cycle 1 will be at the discretion of the treating investigator. Study treatment will be discontinued in participants who experience disease progression or unacceptable toxicity, are not compliant with the study protocol, or, in their opinion or in the opinion of the investigator, are not benefiting from study treatment. However, in the absence of unacceptable toxicity, participants with second-line or greater NSCLC who are still receiving atezolizumab at the time of radiographic disease progression may be permitted to continue study treatment.

location_country:
United Kingdom
Hong Kong
United States
Spain
Korea, Republic of
France

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

location_facility: Hospital Clinico Universitario de Valencia

location_facility: Karmanos Cancer Center; Department of Oncology

location_facility: Massachusetts General Hospital;Hematology/ Oncology

location_facility: Case Western Reserve University; Medicine-Hematology and Oncology

location_facility: Beth Israel Deaconess Med Ctr; Neurology/MS Center

location_facility: Memorial Sloan Kettering - Basking Ridge


title: A Phase 1B Dose Escalation Study to Investigate the Safety, Tolerability and Preliminary Efficacy for the Combination Dasatinib (BMS-354825) Plus Nivolumab (BMS-936558) in Patients Chronic Myeloid Leukemia (CML)

summary: The purpose of this study is to find a dose of Nivolumab that can be safely added to Dasatinib in patients with Chronic Myeloid Leukemia.

location_country:
Germany
Italy
Australia
United States
Spain
Canada
France

location_facility: The University Of Texas MD Anderson Cancer Center

location_facility: Froedtert Hospital & Medical College of Wisconsin

location_facility: Ut Southwestern Medical Center At Dallas


title: Micra Transcatheter Pacing Study

summary: The purpose of this clinical study is to evaluate the safety and efficacy of the Micra Transcatheter Pacing System and to assess long term performance.

ReferenceCitation: Piccini JP, Cunnane R, Steffel J, El-Chami MF, Reynolds D, Roberts PR, Soejima K, Steinwender C, Garweg C, Chinitz L, Ellis CR, Stromberg K, Fagan DH, Mont L. Development and validation of a risk score for predicting pericardial effusion in patients undergoing leadless pacemaker implantation: experience with the Micra transcatheter pacemaker. Europace. 2022 Jul 21;24(7):1119-1126. doi: 10.1093/europace/euab315.

repository_institution: KU Leuven - Lirias

ReferenceCitation: El-Chami MF, Shinn T, Bansal S, Martinez-Sande JL, Clementy N, Augostini R, Ravindran B, Sagi V, Ramanna H, Garweg C, Roberts PR, Soejima K, Stromberg K, Fagan DH, Zuniga N, Piccini JP. Leadless pacemaker implant with concomitant atrioventricular node ablation: Experience with the Micra transcatheter pacemaker. J Cardiovasc Electrophysiol. 2021 Mar;32(3):832-841. doi: 10.1111/jce.14881. Epub 2021 Jan 23.

ReferenceCitation: Garg A, Koneru JN, Fagan DH, Stromberg K, Padala SK, El-Chami MF, Roberts PR, Piccini JP, Cheng A, Ellenbogen KA. Morbidity and mortality in patients precluded for transvenous pacemaker implantation: Experience with a leadless pacemaker. Heart Rhythm. 2020 Dec;17(12):2056-2063. doi: 10.1016/j.hrthm.2020.07.035. Epub 2020 Aug 4.

ReferenceCitation: Tjong FVY, Beurskens NEG, de Groot JR, Waweru C, Liu S, Ritter P, Reynolds D, Wilde AAM, Knops RE; Micra Investigators. Health-related quality of life impact of a transcatheter pacing system. J Cardiovasc Electrophysiol. 2018 Dec;29(12):1697-1704. doi: 10.1111/jce.13726. Epub 2018 Oct 15.

ReferenceCitation: El-Chami MF, Al-Samadi F, Clementy N, Garweg C, Martinez-Sande JL, Piccini JP, Iacopino S, Lloyd M, Vinolas Prat X, Jacobsen MD, Ritter P, Johansen JB, Tondo C, Liu F, Fagan DH, Eakley AK, Roberts PR. Updated performance of the Micra transcatheter pacemaker in the real-world setting: A comparison to the investigational study and a transvenous historical control. Heart Rhythm. 2018 Dec;15(12):1800-1807. doi: 10.1016/j.hrthm.2018.08.005. Epub 2018 Aug 10.

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

repository_institution: University of Copenhagen - Research at the University of Copenhagen

repository_institution: University of Southern Denmark - University of Southern Denmark Research Portal

repositories_concat: LIRIAS (KU Leuven);Pure (Denmark);air.unimi.it;sdu.dk

ReferenceCitation: Okabe T, El-Chami MF, Lloyd MS, Buck B, Gornick CC, Moore JC, Augostini RS, Hummel JD. Leadless pacemaker implantation and concurrent atrioventricular junction ablation in patients with atrial fibrillation. Pacing Clin Electrophysiol. 2018 May;41(5):504-510. doi: 10.1111/pace.13312. Epub 2018 Apr 16.

ReferenceCitation: Soejima K, Asano T, Ishikawa T, Kusano K, Sato T, Okamura H, Matsumoto K, Taguchi W, Stromberg K, Lande J, Kobayashi Y; Micra Transcatheter Pacing Study Group. Performance of Leadless Pacemaker in Japanese Patients vs. Rest of the World - Results From a Global Clinical Trial. Circ J. 2017 Oct 25;81(11):1589-1595. doi: 10.1253/circj.CJ-17-0259. Epub 2017 May 30.

ReferenceCitation: Lloyd M, Reynolds D, Sheldon T, Stromberg K, Hudnall JH, Demmer WM, Omar R, Ritter P, Hummel J, Mont L, Steinwender C, Duray GZ. Rate adaptive pacing in an intracardiac pacemaker. Heart Rhythm. 2017 Feb;14(2):200-205. doi: 10.1016/j.hrthm.2016.11.016. Epub 2016 Nov 15.

ReferenceCitation: Reynolds D, Duray GZ, Omar R, Soejima K, Neuzil P, Zhang S, Narasimhan C, Steinwender C, Brugada J, Lloyd M, Roberts PR, Sagi V, Hummel J, Bongiorni MG, Knops RE, Ellis CR, Gornick CC, Bernabei MA, Laager V, Stromberg K, Williams ER, Hudnall JH, Ritter P; Micra Transcatheter Pacing Study Group. A Leadless Intracardiac Transcatheter Pacing System. N Engl J Med. 2016 Feb 11;374(6):533-41. doi: 10.1056/NEJMoa1511643. Epub 2015 Nov 9.

ReferenceCitation: Ritter P, Duray GZ, Steinwender C, Soejima K, Omar R, Mont L, Boersma LV, Knops RE, Chinitz L, Zhang S, Narasimhan C, Hummel J, Lloyd M, Simmers TA, Voigt A, Laager V, Stromberg K, Bonner MD, Sheldon TJ, Reynolds D; Micra Transcatheter Pacing Study Group. Early performance of a miniaturized leadless cardiac pacemaker: the Micra Transcatheter Pacing Study. Eur Heart J. 2015 Oct 1;36(37):2510-9. doi: 10.1093/eurheartj/ehv214. Epub 2015 Jun 4.

ReferenceCitation: Ritter P, Duray GZ, Zhang S, Narasimhan C, Soejima K, Omar R, Laager V, Stromberg K, Williams E, Reynolds D; Micra Transcatheter Pacing Study Group. The rationale and design of the Micra Transcatheter Pacing Study: safety and efficacy of a novel miniaturized pacemaker. Europace. 2015 May;17(5):807-13. doi: 10.1093/europace/euv026. Epub 2015 Apr 7.

lead_sponsor: Medtronic Cardiac Rhythm and Heart Failure

location_country:
Australia
Japan
Czechia
Canada
Serbia
France
Hungary
Denmark
United States
India
China
Malaysia
Italy
United Kingdom
Netherlands
Austria
Spain
Greece
South Africa

location_facility: Institut Jantung Negara - National Heart Institute

location_facility: Azienda Ospedaliero Universitaria Pisana - Stabilimento Santa Chiara

location_facility: University General Hospital of Heraklion

location_facility: Hospital Universitari Clínic de Barcelona

location_facility: Fuwai Hospital Chinese Academy of Medical Sciences and Peking Union Medical College

location_facility: University of Pittsburgh Medical Center UPMC Presbyterian

location_facility: St. Antonius Ziekenhuis - Locatie Nieuwegein

location_facility: National Cerebral and Cardiovascular Center

location_facility: Southampton General Hospital - University Hospital Southampton NHS Foundation Trust

location_facility: Lancaster Heart & Vascular Research Institute

location_facility: Institut Universitaire de Cardiologie et de Pneumologie de Québec (IUCPQ)

location_facility: University of Oklahoma Health Science Center (OUHSC)


type: Registry Identifier

title: A Phase 2/3, Multicenter, Randomized, Double-blinded, Placebo-controlled, Repeat-dose Study to Evaluate the Efficacy, Safety, Pharmacodynamics, and Pharmacokinetics of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

acronym: ASCEND

summary:
Primary Objective:

The primary objective of this phase 2/3 study is to evaluate the efficacy of olipudase alfa (recombinant human acid sphingomyelinase) administered intravenously once every 2 weeks for 52 weeks in adult participants with acid sphingomyelinase deficiency (ASMD) by assessing changes in: 1) spleen volume as measured by abdominal magnetic resonance imaging (MRI) (and, for the United States [US] only, in association with participant perception related to spleen volume as measured by splenomegaly-related score [SRS]); and 2) infiltrative lung disease as measured by the pulmonary function test, diffusing capacity of the lung for carbon monoxide (DLCO).

Secondary Objectives:

To confirm the safety of olipudase alfa administered intravenously once every 2 weeks for 52 weeks.
To characterize the effect of olipudase alfa on the participant perception related to spleen volume as measured by the SRS after 52 weeks of study drug administration. (For the US, the effect of olipudase alfa on the SRS is part of the primary objective).

To characterize the effect of olipudase alfa after 52 weeks of study drug administration on the following outcome measures assessed sequentially:

The effect of olipudase alfa on liver volume;
The effect of olipudase alfa on platelet count;
The effect of olipudase alfa on fatigue;
The effect of olipudase alfa on pain;
The effect of olipudase alfa on dyspnea.

ipd_sharing: Yes

ipd_sharing_description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org.

location_country:
Germany
Italy
Portugal
United Kingdom
Argentina
Australia
Brazil
Japan
Netherlands
Belgium
Tunisia
United States
Spain
Turkey
Chile
Bulgaria
France

location_facility: UCSF Medical Center Site Number : 840005

location_facility: Hospital De Clinicas De Porto Alegre Site Number : 076001

location_facility: Montefiore Medical Center Site Number : 840006


title: Cochlear-Implanted Recipient Observational Study

acronym: IROS

summary:
The purpose of this study is to collect patient related benefit data following treatment for permanent hearing loss with a hearing implant from the company Cochlear over a period of 2 years post treatment.

Assessment of benefits is based on standard questionnaires of hearing ability and quality of life in general.

ReferenceCitation: Maes IH, Joore MA, Cima RF, Vlaeyen JW, Anteunis LJ. Assessment of health state in patients with tinnitus: a comparison of the EQ-5D and HUI mark III. Ear Hear. 2011 Jul-Aug;32(4):428-35. doi: 10.1097/AUD.0b013e3181fdf09f.

ReferenceCitation: Noble W, Tyler R, Dunn C, Bhullar N. Unilateral and bilateral cochlear implants and the implant-plus-hearing-aid profile: comparing self-assessed and measured abilities. Int J Audiol. 2008 Aug;47(8):505-14. doi: 10.1080/14992020802070770.

ReferenceCitation: Mauch H, Kaur J, Irwin C, Wyss J. Design, implementation, and management of an international medical device registry. Trials. 2021 Nov 25;22(1):845. doi: 10.1186/s13063-021-05821-5.

evidence: oa repository (via free pdf)

repository_normalized: Research Square

repositories:
PubMed Central
Research Square

repositories_concat: PubMed Central;Research Square

lead_sponsor: Cochlear

location_country:
Germany
Slovenia
Poland
Colombia
Portugal
Hungary
Argentina
Brazil
Netherlands
Austria
Belgium
Sweden
Spain
South Africa
Turkey
France

location_facility: Implantes Cocleares Equipo Multicentrico (ICEM)

location_facility: Instytut Centrum Zdrowia Matki Polki w Lodzi

location_facility: Samodzielny Publiczny Szpital Kliniczny nr 1 w Szczecinie

location_facility: Centro de Investigaciones Otoaudiologicas (CIOA)

location_facility: Hospital General Universitario Gregorio Maranon

location_facility: Hospital Clinico Universitario Lozano Blesa

location_facility: Johannesburg Cochlear Implant Center, Wits Donald Gordon Medical Centre

location_facility: Samodzielny Publiczny Szpital Kliniczny nr 4 w Lublinie

location_facility: Wojewodzki Szpital Dzieciecy w Bydgoszczy

location_facility: Universitätsklinik für Hals-, Nasen- und Ohrenheilkunde

location_facility: Hospital Clinico Universitario de Valencia

location_facility: Uniwersytecki Szpital Kliniczny w Bialymstoku

location_facility: Hospital Clinico Universitario de Salamanca

location_facility: Samodzielny Publiczny Szpital Kliniczny nr 2 w Poznaniu

location_facility: Uniwersytecki Szpital Kliniczny nr 1 w Lodzi

location_facility: Durban Cochlear Implant Pogramme, Parklands Hospital

location_facility: Hospital Universitario Clinica San Rafael

location_facility: Instituto Nacional de Otologia Garcia Gomez

intervention_type: Device


title: A PHASE 4, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY OF CONTINUED ENZALUTAMIDE TREATMENT BEYOND PROGRESSION IN PATIENTS WITH CHEMOTHERAPY-NAÏVE METASTATIC CASTRATION-RESISTANT PROSTATE CANCER

acronym: PLATO

summary: The purpose of this study is to determine if continued treatment with Enzalutamide is effective in patients with metastatic prostate cancer.

ReferenceCitation: Attard G, Borre M, Gurney H, Loriot Y, Andresen-Daniil C, Kalleda R, Pham T, Taplin ME; PLATO collaborators. Abiraterone Alone or in Combination With Enzalutamide in Metastatic Castration-Resistant Prostate Cancer With Rising Prostate-Specific Antigen During Enzalutamide Treatment. J Clin Oncol. 2018 Sep 1;36(25):2639-2646. doi: 10.1200/JCO.2018.77.9827. Epub 2018 Jul 20.

repository_institution: University College London - UCL Discovery

repository_normalized: UCL Discovery

repositories:
PubMed Central
Pure (Denmark)
UCL Discovery

repositories_concat: PubMed Central;Pure (Denmark);UCL Discovery

ipd_sharing_description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

lead_sponsor: Pfizer

collaborators: Medivation LLC, a wholly owned subsidiary of Pfizer Inc.

location_country:
Finland
Italy
Slovakia
United Kingdom
Denmark
Australia
Sweden
Belgium
United States
Spain
France

location_facility: Institut nuklearnej a molekularnej mediciny

location_facility: Concord Cancer Centre, Medical Oncology Department

location_facility: UOC Radiologia Piasta, Azienda Ospedaliera S. Camillo Forlanini

location_facility: Fakultna nemocnica s poliklinikou F.D. Roosevelta

location_facility: Epic Pharmacy Port Macquarie base hospital

location_facility: Uroexam, spol. s r.o., Urologicka ambulancia

location_facility: Helsingin yliopistollinen keskussairaala, Meilahden sairaala

location_facility: University College London Hospitals NHS Foundation Trust

location_facility: SCDU Oncologia Medica II Pad, A.O.U. San Luigi Gonzaga

location_facility: Concord Hospital Clinical Trials Pharmacy

location_facility: GAMMALAB, spol.s r.o., Oddelenie nuklearnej mediciny

location_facility: Apoteket AB Kliniska Provningar Molnlycke

location_facility: Azienda Ospedaliera S. Camillo Forlanini, UOC per il governo clinico in Oncologia Medica

location_facility: Hospital Universitario Madrid Sanchinarro

location_facility: Tenpharm Pty Ltd trading as EPIC Pharmacy Tennyson

location_facility: North Shore Radiology and Nuclear Medicine

location_facility: ALTAHIA, Xarxa Assistencial Universitaria de Manresa

location_facility: Struttura Complessa di Oncologia, Azienda Socio Sanitaria Territoriale di Cremona

location_facility: SS Medicina Nucleare, A.O.U. San Luigi Gonzaga

location_facility: SCDU Radiodiagnostica, A.O.U. San Luigi Gonzaga

location_facility: Servizio di Radiologia, Azienda Ospedaliera "Istituti Ospitalieri" di Cremona

location_facility: ICO Badalona-Instituto Germans Trias i Pujol

location_facility: Medicina Nucleare, Azienda Ospedaliera "Istituti Ospitalieri" di Cremona

location_facility: U.O. Oncologia Medica, IRCCS Istituto Scientifico Romagnolo per lo Studio e la Cura

location_facility: Port Macquarie Base Hospital,North Coast Cancer Institute

location_facility: Institut Gustave Roussy - Service d'Urologie


lead_sponsor: Ultragenyx Pharmaceutical Inc

summary: The primary objectives of the study are to evaluate the efficacy of UX007 compared to placebo as measured by the reduction from randomization to Week 8 in frequency of seizures and to evaluate the safety of UX007 via adverse event (AE) rates, laboratory values, and electrocardiogram (ECG).

ReferenceCitation: Striano P, Auvin S, Collins A, Horvath R, Scheffer IE, Tzadok M, Miller I, Kay Koenig M, Lacy A, Davis R, Garcia-Cazorla A, Saneto RP, Brandabur M, Blair S, Koutsoukos T, De Vivo D. A randomized, double-blind trial of triheptanoin for drug-resistant epilepsy in glucose transporter 1 deficiency syndrome. Epilepsia. 2022 Jul;63(7):1748-1760. doi: 10.1111/epi.17263. Epub 2022 May 21.

repository_institution: University of Cambridge - Apollo

repositories_concat: PubMed Central;www.repository.cam.ac.uk

location_facility: Children's Hospital Colorado - University of Colorado, Denver, School of Medicine

location_facility: Unita' Operativa Neurologia Pediatrica e Malattie Muscolari Istituto "Giannina Gaslini"

location_facility: Columbia University - Department of Neurology

location_facility: University of Texas Neurometabolic Clinic

location_facility: Service de Neurologie Pédiatrique et des Maladies Métaboliques - INSERM U1141 Hôpital Robert Debré - APHP

title: A Randomized, Double-blind, Placebo-controlled, Parallel-group, Study to Assess the Safety and Efficacy of UX007 in Subjects With Glucose Transporter Type 1 Deficiency Syndrome

location_country:
France
Italy
United Kingdom
Australia
United States
Spain
Israel


title: An Open-Label, Multicenter Study To Evaluate The Efficacy And Safety Of Sofosbuvir/Ledipasvir Fixed-Dose Combination ± Ribavirin For 12 or 24 Weeks In Chronic Genotype 1 HCV Infected Subjects Who Participated In A Prior Gilead-Sponsored HCV Treatment Study

summary: The primary objectives of this study are to evaluate the efficacy, safety, and tolerability of ledipasvir/sofosbuvir (LDV/SOF) fixed-dose combination (FDC) with or without ribavirin (RBV) in participants with chronic genotype 1 hepatitis C virus (HCV) infection who have participated in a prior Gilead-sponsored HCV treatment study, and who did not achieve sustained virologic response (SVR24), defined as HCV RNA < lower limit of quantification (LLOQ) 24 weeks after last dose of study drug (SVR24).

ReferenceCitation: Lawitz E, Pockros PJ, Yang JC, Pang PS, Zhu Y, Svarovskaia E, et al. Ledipasvir/sofosbuvir regimens for the retreatment of patients who failed sofosbuvir-based regimens [Abstract 10868]. Presented at: The 25th Conference of the Asian Pacific Association for the Study of Liver (APASL); 2016 February 20-24; Tokyo, Japan.

ReferenceCitation: Lawitz E, Flamm S, Yang JC, Pang PS, Zhu Y, Svarovskaia E, et al. Retreatment of patients who failed 8 or 12 weeks of ledipasvir/sofosbuvir-based regimens with ledipasvir/sofosbuvir for 24 weeks [Abstract 1627]. Presented at: The 50th Annual Congress of the European Association for the Study of Liver: The International Liver Congress (EASL); 2015 April 22-26; Vienna, Austria

ReferenceCitation: Wyles D, Pockros P, Morelli G, Younes Z, Svarovskaia E, Yang JC, Pang PS, Zhu Y, McHutchison JG, Flamm S, Lawitz E. Ledipasvir-sofosbuvir plus ribavirin for patients with genotype 1 hepatitis C virus previously treated in clinical trials of sofosbuvir regimens. Hepatology. 2015 Jun;61(6):1793-7. doi: 10.1002/hep.27814. Epub 2015 Apr 27.

ipd_sharing_description: Qualified external researchers may request IPD for this study after study completion. For more information, please visit our website at http://www.gilead.com/research/disclosure-and-transparency.

lead_sponsor: Gilead Sciences

location_country:
Australia
United States
Spain
Puerto Rico
France


title: A Randomized, Double-Blind, Placebo Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alpha1 Proteinase Inhibitor (Human) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency

acronym: SPARTA

summary: This is a multi-center, randomized, placebo-controlled, double blind clinical study to assess the efficacy and safety of two separate dose regimens of Alpha-1 MP versus placebo for 156 weeks (i.e., 3 years) using computed tomography (CT) of the lungs as the main measure of efficacy. The two Alpha-1 MP doses to be tested are 60 mg/kg and 120 mg/kg administered weekly by IV infusion for 156 weeks. The study consists of an optional pre-screening phase, Screening Phase, a 156-week Treatment Phase, and an End of Study Visit at Week 160.

ReferenceCitation: Sorrells S, Camprubi S, Griffin R, Chen J, Ayguasanosa J. SPARTA clinical trial design: exploring the efficacy and safety of two dose regimens of alpha1-proteinase inhibitor augmentation therapy in alpha1-antitrypsin deficiency. Respir Med. 2015 Apr;109(4):490-9. doi: 10.1016/j.rmed.2015.01.022. Epub 2015 Feb 13.

lead_sponsor: Grifols Therapeutics LLC

location_country:
Finland
Poland
New Zealand
Denmark
Australia
Brazil
Sweden
United States
Canada
Estonia
Russian Federation
France

location_facility: New Zealand Respiratory and Sleep institute

location_facility: SBEI HPE Altai State Medical University of MoH and SD

location_facility: University of Texas Health Center at Tyler

location_facility: Hôpital Cardio-Vasculaire et Pneumologique Louis Pradel

location_facility: Turku University Central Hospital, Department of Pulmonary Diseases

location_facility: UNIFESP - Universidade Federal de São Paulo

location_facility: Queen Elizabeth II Health Sciences Centre

location_facility: St. Joseph's Hospital and Medical Center

location_facility: SAIH of Yaroslavl region "Clinical Hospital of Emergency Medical Care n.a. N. V. Solovyev

location_facility: Instytut Gruzlicy i Chorob Pluc w Warszawie

location_facility: University of Miami Miller School of Medicine

intervention_type: Other


lead_sponsor: Hoffmann-La Roche

summary:
RO7490677 is an investigational drug that is being developed for possible use in the treatment of myelofibrosis (MF), a disease in which the bone marrow, which is the organ in the body that makes blood cells, is replaced by fibrosis, or excess scar tissue.

The purpose of this study is to gather information on whether RO7490677 has an effect on the MF disease, whether it is safe in patients with MF, and how well it is tolerated.

location_facility: Johannes Wesling Academic Medical Center

title: A Phase 2, Prospective Study Of PRM-151 In Subjects With Primary Myelofibrosis (PMF), Post-Polycythemia Vera MF (Post-PV MF), Or Post-Essential Thrombocythemia MF (Post-ET MF)

ipd_sharing_description: Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).

location_country:
Germany
France
Italy
United Kingdom
Netherlands
United States
Canada
Israel


title: Natural History of Progression of Atrophy Secondary to Stargardt Disease: Retrospective, and Prospective Longitudinal Observational Study Incl. Ancillary SMART Study- Scotopic Microperimetric Assessment of Rod Function in Stargardt Disease

acronym: ProgSTAR

summary:
Stargardt disease is currently an incurable and untreatable macular dystrophy that causes severe visual loss in children and young adults, thereby causing enormous morbidity with economic, psychological, emotional, and social implications. There are no FDA approved therapeutic treatments for this disease. Therefore, the objective of this study is to collect natural history data from a large population of children and adults in order to evaluate possible efficacy measures for planned clinical trials.

Participants will be recruited from each Investigator's own patient population as the study requires the availability of both multiyear retrospective data, as well as ongoing prospectively collected data. A concurrent ancillary study (SMART study) is also being conducted with a subset of the prospective study patients during their regular ProgSTAR study visits to expand the collection of retinal images to include microperimetry measurements gathered under scotopic (low light) conditions.

time_perspective: Other

ReferenceCitation: Schonbach EM, Ibrahim MA, Strauss RW, Birch DG, Cideciyan AV, Hahn GA, Ho A, Kong X, Nasser F, Sunness JS, Zrenner E, Sadda SR, West SK, Scholl HPN; Progression of Stargardt Disease Study Group. Fixation Location and Stability Using the MP-1 Microperimeter in Stargardt Disease: ProgStar Report No. 3. Ophthalmol Retina. 2017 Jan-Feb;1(1):68-76. doi: 10.1016/j.oret.2016.08.009. Epub 2016 Oct 31.

ReferenceCitation: Kong X, West SK, Strauss RW, Munoz B, Cideciyan AV, Michaelides M, Ho A, Ahmed M, Schonbach EM, Cheetham JK, Ervin AM, Scholl HPN; ProgStar study group. Progression of Visual Acuity and Fundus Autofluorescence in Recent-Onset Stargardt Disease: ProgStar Study Report #4. Ophthalmol Retina. 2017 Nov-Dec;1(6):514-523. doi: 10.1016/j.oret.2017.02.008. Epub 2017 Apr 28.

ReferenceCitation: Strauss RW, Munoz B, Ho A, Jha A, Michaelides M, Mohand-Said S, Cideciyan AV, Birch D, Hariri AH, Nittala MG, Sadda S, Scholl HPN; ProgStar Study Group. Incidence of Atrophic Lesions in Stargardt Disease in the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Study: Report No. 5. JAMA Ophthalmol. 2017 Jul 1;135(7):687-695. doi: 10.1001/jamaophthalmol.2017.1121.

ReferenceCitation: Schonbach EM, Wolfson Y, Strauss RW, Ibrahim MA, Kong X, Munoz B, Birch DG, Cideciyan AV, Hahn GA, Nittala M, Sunness JS, Sadda SR, West SK, Scholl HPN; ProgStar Study Group. Macular Sensitivity Measured With Microperimetry in Stargardt Disease in the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Study: Report No. 7. JAMA Ophthalmol. 2017 Jul 1;135(7):696-703. doi: 10.1001/jamaophthalmol.2017.1162.

ReferenceCitation: Strauss RW, Ho A, Munoz B, Cideciyan AV, Sahel JA, Sunness JS, Birch DG, Bernstein PS, Michaelides M, Traboulsi EI, Zrenner E, Sadda S, Ervin AM, West S, Scholl HP; Progression of Stargardt Disease Study Group. The Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Studies: Design and Baseline Characteristics: ProgStar Report No. 1. Ophthalmology. 2016 Apr;123(4):817-28. doi: 10.1016/j.ophtha.2015.12.009. Epub 2016 Jan 16.

ReferenceCitation: Kong X, Strauss RW, Michaelides M, Cideciyan AV, Sahel JA, Munoz B, West S, Scholl HP; ProgStar Study Group. Visual Acuity Loss and Associated Risk Factors in the Retrospective Progression of Stargardt Disease Study (ProgStar Report No. 2). Ophthalmology. 2016 Sep;123(9):1887-97. doi: 10.1016/j.ophtha.2016.05.027. Epub 2016 Jul 2.

repositories_concat: UCL Discovery

repositories_institution: University College London - UCL Discovery

ReferenceCitation: Kong X, Strauss RW, Cideciyan AV, Michaelides M, Sahel JA, Munoz B, Ahmed M, Ervin AM, West SK, Cheetham JK, Scholl HPN; ProgStar Study Group. Visual Acuity Change over 12 Months in the Prospective Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Study: ProgStar Report Number 6. Ophthalmology. 2017 Nov;124(11):1640-1651. doi: 10.1016/j.ophtha.2017.04.026. Epub 2017 May 23.

ReferenceCitation: Strauss RW, Munoz B, Ho A, Jha A, Michaelides M, Cideciyan AV, Audo I, Birch DG, Hariri AH, Nittala MG, Sadda S, West S, Scholl HPN; ProgStar Study Group. Progression of Stargardt Disease as Determined by Fundus Autofluorescence in the Retrospective Progression of Stargardt Disease Study (ProgStar Report No. 9). JAMA Ophthalmol. 2017 Nov 1;135(11):1232-1241. doi: 10.1001/jamaophthalmol.2017.4152.

ReferenceCitation: Kong X, Fujinami K, Strauss RW, Munoz B, West SK, Cideciyan AV, Michaelides M, Ahmed M, Ervin AM, Schonbach E, Cheetham JK, Scholl HPN; ProgStar Study Group. Visual Acuity Change Over 24 Months and Its Association With Foveal Phenotype and Genotype in Individuals With Stargardt Disease: ProgStar Study Report No. 10. JAMA Ophthalmol. 2018 Aug 1;136(8):920-928. doi: 10.1001/jamaophthalmol.2018.2198.

lead_sponsor: Foundation Fighting Blindness

sponsor_collaborators:
Foundation Fighting Blindness
United States Department of Defense

location_country:
United States
United Kingdom
Germany
France

location_facility: Wilmer Eye Institute, Johns Hopkins University

location_facility: Center for Ophthalmic Research, University of Teubingen

location_facility: Scheie Eye Institute, University of Pennsylvania


title: A Randomized Double-blind, Placebo-controlled, Parallel-group, Multicenter, Phase 3 Study to Evaluate the Long-term Safety of Naldemedine for the Treatment of Opioid-induced Constipation in Subjects With Non-malignant Chronic Pain Receiving Opioid Therapy

summary: The purpose of this study is to evaluate the long-term safety of naldemedine for the treatment of constipation due to opioid therapy.

ReferenceCitation: Camilleri M, Hale M, Morlion B, Tack J, Webster L, Wild J. Naldemedine Improves Patient-Reported Outcomes of Opioid-Induced Constipation in Patients with Chronic Non-Cancer Pain in the COMPOSE Phase 3 Studies. J Pain Res. 2021 Jul 16;14:2179-2189. doi: 10.2147/JPR.S282738. eCollection 2021.

publisher_dissemination: Informa

ReferenceCitation: Webster LR, Hale ME, Yamada T, Wild JE. A Renal Impairment Subgroup Analysis of the Safety and Efficacy of Naldemedine for the Treatment of Opioid-Induced Constipation in Patients with Chronic Non-Cancer Pain Receiving Opioid Therapy. J Pain Res. 2020 Mar 24;13:605-612. doi: 10.2147/JPR.S237833. eCollection 2020.

ReferenceCitation: Wild J, Webster L, Yamada T, Hale M. Safety and Efficacy of Naldemedine for the Treatment of Opioid-Induced Constipation in Patients with Chronic Non-Cancer Pain Receiving Opioid Therapy: A Subgroup Analysis of Patients >/= 65 Years of Age. Drugs Aging. 2020 Apr;37(4):271-279. doi: 10.1007/s40266-020-00753-2.

lead_sponsor: Shionogi

location_country:
Germany
Italy
Poland
Hungary
Israel
Denmark
Australia
Sweden
United Kingdom
Austria
United States
Spain
South Africa
Czechia
Canada
France


title: Natural History Study of Children With Metachromatic Leukodystrophy

summary: The purpose of this study is evaluate the natural course of disease progression related to gross motor function in children with metachromatic leukodystrophy (MLD).

lead_sponsor: Shire

location_country:
Germany
Argentina
Denmark
Japan
Brazil
Belgium
United States
Turkey
Canada
France

location_facility: Hacettepe Universitesi Tip Fakultesi Onkoloji Hastanesi

location_facility: Universitair Ziekenhuis Antwerpen (UZA) (University Hospital Antwerpen)

location_facility: Univesitatsklinikum Tubingen Klinik fur Kinder und Jugendmedizin

location_facility: Copenhagen University Hospital, Rigshospitalet

location_facility: Ann & Robert H. Lurie Children's Hospital of Chicago

location_facility: Faculty Of Medicine, Osaka University Graduate School Of Medicine

location_facility: Hospital de Cllnicas de Porto Alegre (HCPA) / UFRGS

location_facility: The Jikei University School Of Medicine - Institute Of Dna Medicine