title: β-SPECIFIC 4 Patients: Study of Pediatric EffiCacy and Safety wIth FIrst-line Use of Canakinumab An Open-label Canakinumab (ACZ885) Dose Reduction or Dose Interval Prolongation Efficacy and Safety Study in Patients With Systemic Juvenile Idiopathic Arthritis (SJIA)

acronym: ß-SPECIFIC 4

summary: The purpose of this study was to evaluate the efficacy observed with canakinumab dose reduction in a subgroup of patients in the extension study CACZ885G2301E1.

ReferenceCitation: Quartier P, Alexeeva E, Constantin T, Chasnyk V, Wulffraat N, Palmblad K, Wouters C, I Brunner H, Marzan K, Schneider R, Horneff G, Martini A, Anton J, Wei X, Slade A, Ruperto N, Abrams K; Paediatric Rheumatology International Trials Organisation and the Pediatric Rheumatology Collaborative Study Group. Tapering Canakinumab Monotherapy in Patients With Systemic Juvenile Idiopathic Arthritis in Clinical Remission: Results From a Phase IIIb/IV Open-Label, Randomized Study. Arthritis Rheumatol. 2021 Feb;73(2):336-346. doi: 10.1002/art.41488. Epub 2020 Dec 11.

repository_institution: KU Leuven - Lirias

repository_normalized: LIRIAS (KU Leuven)

repositories:
LIRIAS (KU Leuven)
PubMed Central

repositories_concat: LIRIAS (KU Leuven);PubMed Central

location_country:
Germany
Italy
Poland
Hungary
Israel
Brazil
Netherlands
Austria
Belgium
Sweden
United States
Spain
Turkey
Canada
Russian Federation
France


title: A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)

acronym: MARS

summary: The objectives of this program are: to characterize and describe the Mucopolysaccharidosis IV type A (MPS IVA) population as a whole, including the heterogeneity, progression, and natural history of MPS IVA; to evaluate the long-term effectiveness and safety of Vimizim®, including, but not limited to, the occurrence of serious hypersensitivity reactions, anaphylaxis, and changes in antibody status; to help the medical community with the development of recommendations for monitoring MPS IVA patients and reports on patient outcomes to optimize patient care; to collect data on other treatment paradigms, and evaluate the prevalences of their use and their effectiveness; to characterize the effects and safety of Vimizim treatment 5 years from enrollment in the Registry for patients under 5 years of age; to monitor pregnancy exposure, including maternal, neonatal, and infant outcomes; and to monitor patients who have completed the MOR-005 and MOR-007 clinical trials. These patients will be encouraged to enroll in the applicable Registry Substudy and will be monitored using the MOR-005 and MOR-007 assessment schedules, respectively.

status: Active, not recruiting

study_completion_date_type: Anticipated

study_type: Observational

time_perspective: Prospective

enrollment_type: Actual

ipd_sharing: No

lead_sponsor: BioMarin Pharmaceutical

sponsor_collaborators:
BioMarin Pharmaceutical
ICON plc

location_country:
Ireland
Italy
Poland
Portugal
United Kingdom
Denmark
Australia
Netherlands
Austria
Belgium
United States
Malaysia
Taiwan
Czechia
Canada
Puerto Rico
France

location_facility: Temple Street Children's University Hospital

location_facility: National Cheng Kung University (NCKU) Hospital

location_facility: Adult Metabolic Diseases Clinic Vancouver General Hospital

location_facility: Chang Gung Memorial Hospital Kaohsiung Branch

location_facility: Great Ormond Street Hospital NHS Foundation Trust

location_facility: Children's Hospital of the King's Daughters

location_facility: NYU-Langone Medical Center School of Medicine

location_facility: Rotterdam University Hospital Medical Centre

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

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

location_facility: Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust

location_facility: Azienda Ospedaliero San Gerardo di Monza

location_facility: San Jorge Children's Hospital, Centro de Investigaciones Clinicas

location_facility: Hospital Pediátrico de Coimbra (Centro Hospitalar de Coimbra)

location_facility: Copenhagen University Hospital, Klinik For Sjaeldne Handicap

location_facility: University Hospital Birmingham - Selly Oaks

location_facility: Children's Hospitals and Clinics of Minnesota

location_facility: Kaohsiung Medical University Chung-Ho Memorial Hospital

location_facility: Centro Hospitalar Lisboa Centro - Hospital de Dona Estefânia

location_facility: Children's Hospital of Pittsburgh of UPMC

location_facility: Azienda Policlinico Vittorio Emanuele, presidio G Rodolico, Università di Catania

location_facility: Kapiolani Medical Center for Women and Children

location_facility: Academic Medical Center - University of Amsterdam

location_facility: Klinika Dětského a Dorostového Lékařstvi

location_facility: National Hospital for Neurology and Neurosurgery

location_facility: Children's Health Queensland Hospital and Health Service

location_facility: Children's & Women's Centre of British Columbia

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Ongoing


study_start_date_type: Actual

lead_sponsor: AbbVie

summary: The study seeks to assess the efficacy of veliparib (ABT-888) in combination with carboplatin and etoposide in participants with extensive disease small cell lung cancer (ED SCLC).

ReferenceType: result

ReferenceCitation: Atrafi F, Groen HJM, Byers LA, Garralda E, Lolkema MP, Sangha RS, Viteri S, Chae YK, Camidge DR, Gabrail NY, Hu B, Tian T, Nuthalapati S, Hoening E, He L, Komarnitsky P, Calles A. A Phase I Dose-Escalation Study of Veliparib Combined with Carboplatin and Etoposide in Patients with Extensive-Stage Small Cell Lung Cancer and Other Solid Tumors. Clin Cancer Res. 2019 Jan 15;25(2):496-505. doi: 10.1158/1078-0432.CCR-18-2014. Epub 2018 Oct 16.

oa_colors: green other

oa_host_type: publisher;repository

evidence: open (via free pdf)

host_type: publisher

license_normalized: no license

version: publishedVersion

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

host_type: repository

repository_institution: University of Groningen / Centre for Information Technology - University of Groningen research database

repository_institution: DANS - Data Archiving and Networked Services - NARCIS - National Academic Research and Collaborations Information System

evidence: oa repository (via page says license)

license: cc-by

license_normalized: cc-by

version: submittedVersion

unpaywall_oa_status: bronze

publisher_dissemination: American Association for Cancer Research

location_facility: Northwestern University Feinberg School of Medicine /ID# 137088

location_facility: Hopital du Sacre Coeur Montreal /ID# 154436

location_facility: Hospital Universitario Gregori /ID# 164982

location_facility: Southern Medical Day Care Ctr /ID# 155498

location_facility: Fejer Megyei Szent Gyorgy Korh /ID# 151352

location_facility: Atrium-Orbis Zuyderland Medisch Centrum /ID# 149830

location_facility: Univ of Colorado Cancer Center /ID# 129220

location_facility: Oncocenter Oncologie Clinica S /ID# 151694

location_facility: Veszprem Megyei Tudogyogyintez /ID# 158807

location_facility: Road Hospital Open Joint Stock Company Russian Railways /ID# 152731

location_facility: Centre Hosp Intercommunal de Creteil /ID# 157970

location_facility: Universitair Medisch Centrum Groningen /ID# 131252

location_facility: Belgorod Oncology Dispensary /ID# 152330

location_facility: Hosp Univ Puerta de Hierro Maj /ID# 151253

location_facility: Debreceni Egyetem Klinikai Központ /ID# 151354

location_facility: Hospital Stanta Creu i Sant Pau /ID# 151254

location_facility: Cliniques Universitaires Saint Luc /ID# 151024

location_facility: Orszagos Koranyi Pulmonologiai Intezet /ID# 151351

location_facility: Gabrail Cancer Center Research /ID# 129216

location_facility: Petz Aladar Megyei Oktato Korh /ID# 155352

location_facility: S.C. Centrul de Oncologie Sf. Nectarie S.R.L. /ID# 161137

location_facility: Chonnam National University Hwasun Hospital /ID# 153188

location_facility: Univercity Headache Clynic,LTD /ID# 161708

location_facility: Markusovszky Egyetemi Oktatókórház /ID# 158806

location_facility: Herbert Herman Cancer Center /ID# 167020

location_facility: University of Texas MD Anderson Cancer Center /ID# 129213

location_facility: S.C. Radiotherapy Center Cluj /ID# 165137

location_facility: Sverdlovsk Regional Oncology Center Dispensary /ID# 152328

location_facility: Hosp Univ Madrid Sanchinarro /ID# 130301

location_facility: Peninsula & South Eastern Haem /ID# 155497

status: Completed

title: A Phase 1 Dose Escalation and Phase 2 Randomized Double-Blind Study of Veliparib in Combination With Carboplatin and Etoposide as a Therapy of Treatment-Naïve Extensive Stage Disease Small Cell Lung Cancer

primary_purpose: Treatment

type: EudraCT Number

source: euctr

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

design_allocation: Randomized

study_completion_date_type: Actual

location_country:
Hungary
Romania
Australia
Netherlands
Belgium
United States
Korea, Republic of
Spain
Czechia
Canada
Russian Federation
France

study_type: Interventional

ipd_sharing: Yes

status_simplified: Completed


acronym: SOLO 2

lead_sponsor: Regeneron Pharmaceuticals

summary: This is a randomized, double-blind, placebo-controlled, parallel-group study to confirm the efficacy and safety of Dupilumab monotherapy in adults with moderate-to-severe atopic dermatitis (AD).

ReferenceCitation: Simpson EL, Bieber T, Guttman-Yassky E, Beck LA, Blauvelt A, Cork MJ, Silverberg JI, Deleuran M, Kataoka Y, Lacour JP, Kingo K, Worm M, Poulin Y, Wollenberg A, Soo Y, Graham NM, Pirozzi G, Akinlade B, Staudinger H, Mastey V, Eckert L, Gadkari A, Stahl N, Yancopoulos GD, Ardeleanu M; SOLO 1 and SOLO 2 Investigators. Two Phase 3 Trials of Dupilumab versus Placebo in Atopic Dermatitis. N Engl J Med. 2016 Dec 15;375(24):2335-2348. doi: 10.1056/NEJMoa1610020. Epub 2016 Sep 30.

evidence: open (via free article)

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

repository_institution: Tor Vergata University - Cineca Institutional Research Information System

repository_institution: University of Leeds, The University of Sheffield, University of York - White Rose Research Online

version: acceptedVersion

publisher_dissemination: Massachusetts Medical Society

ReferenceType: derived

ReferenceCitation: Silverberg JI, Boguniewicz M, Hanifin J, Papp KA, Zhang H, Rossi AB, Levit NA. Dupilumab Treatment in Adults with Moderate-to-Severe Atopic Dermatitis is Efficacious Regardless of Age of Disease Onset: a Post Hoc Analysis of Two Phase 3 Clinical Trials. Dermatol Ther (Heidelb). 2022 Dec;12(12):2731-2746. doi: 10.1007/s13555-022-00822-x. Epub 2022 Oct 21.

oa_colors: green gold

evidence: oa journal (via doaj)

license: cc-by-nc

license_normalized: cc-by-nc

evidence: oa repository (via pmcid lookup)

repository_normalized: PubMed Central

repository_institution: University of Illinois at Chicago - INDIGO

repositories_concat: PubMed Central;figshare.com

unpaywall_oa_status: gold

publisher_dissemination: Springer-Nature

ReferenceCitation: Wechsler ME, Klion AD, Paggiaro P, Nair P, Staumont-Salle D, Radwan A, Johnson RR, Kapoor U, Khokhar FA, Daizadeh N, Chen Z, Laws E, Ortiz B, Jacob-Nara JA, Mannent LP, Rowe PJ, Deniz Y. Effect of Dupilumab on Blood Eosinophil Counts in Patients With Asthma, Chronic Rhinosinusitis With Nasal Polyps, Atopic Dermatitis, or Eosinophilic Esophagitis. J Allergy Clin Immunol Pract. 2022 Oct;10(10):2695-2709. doi: 10.1016/j.jaip.2022.05.019. Epub 2022 May 28.

oa_host_type: publisher

evidence: open (via crossref license)

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

repositories_concat: closed

unpaywall_oa_status: hybrid

publisher_dissemination: Elsevier

ReferenceCitation: Armstrong A, Blauvelt A, Simpson EL, Smith CH, Herranz P, Kataoka Y, Seo SJ, Ferrucci SM, Chao J, Chen Z, Rossi AB, Shumel B, Tomondy P. Continued Treatment with Dupilumab is Associated with Improved Efficacy in Adults with Moderate-to-Severe Atopic Dermatitis Not Achieving Optimal Responses with Short-Term Treatment. Dermatol Ther (Heidelb). 2022 Jan;12(1):195-202. doi: 10.1007/s13555-021-00643-4. Epub 2021 Dec 13.

repositories_concat: PubMed Central

ReferenceCitation: Paller AS, Tan JKL, Bagel J, Rossi AB, Shumel B, Zhang H, Abramova A. IGAxBSA composite for assessing disease severity and response in patients with atopic dermatitis. Br J Dermatol. 2022 Mar;186(3):496-507. doi: 10.1111/bjd.20872. Epub 2022 Feb 25.

oa_host_type: repository

unpaywall_oa_status: green

publisher_dissemination: Oxford University Press

ReferenceCitation: Griffiths C, de Bruin-Weller M, Deleuran M, Fargnoli MC, Staumont-Salle D, Hong CH, Sanchez-Carazo J, Foley P, Seo SJ, Msihid J, Chen Z, Cyr SL, Rossi AB. Dupilumab in Adults with Moderate-to-Severe Atopic Dermatitis and Prior Use of Systemic Non-Steroidal Immunosuppressants: Analysis of Four Phase 3 Trials. Dermatol Ther (Heidelb). 2021 Aug;11(4):1357-1372. doi: 10.1007/s13555-021-00558-0. Epub 2021 Jun 18.

repositories_concat: PubMed Central;University of Melbourne - Minerva Access

ReferenceCitation: Hamilton JD, Harel S, Swanson BN, Brian W, Chen Z, Rice MS, Amin N, Ardeleanu M, Radin A, Shumel B, Ruddy M, Patel N, Pirozzi G, Mannent L, Graham NMH. Dupilumab suppresses type 2 inflammatory biomarkers across multiple atopic, allergic diseases. Clin Exp Allergy. 2021 Jul;51(7):915-931. doi: 10.1111/cea.13954. Epub 2021 Jun 26.

oa_colors: green hybrid

ReferenceCitation: Boguniewicz M, Beck LA, Sher L, Guttman-Yassky E, Thaci D, Blauvelt A, Worm M, Corren J, Soong W, Lio P, Rossi AB, Lu Y, Chao J, Eckert L, Gadkari A, Hultsch T, Ruddy M, Mannent LP, Graham NMH, Pirozzi G, Chen Z, Ardeleanu M. Dupilumab Improves Asthma and Sinonasal Outcomes in Adults with Moderate to Severe Atopic Dermatitis. J Allergy Clin Immunol Pract. 2021 Mar;9(3):1212-1223.e6. doi: 10.1016/j.jaip.2020.12.059. Epub 2021 Jan 13.

ReferenceCitation: Silverberg JI, Simpson EL, Guttman-Yassky E, Cork MJ, de Bruin-Weller M, Yosipovitch G, Eckert L, Chen Z, Ardeleanu M, Shumel B, Hultsch T, Rossi AB, Hamilton JD, Orengo JM, Ruddy M, Graham NMH, Pirozzi G, Gadkari A. Dupilumab Significantly Modulates Pain and Discomfort in Patients With Atopic Dermatitis: A Post Hoc Analysis of 5 Randomized Clinical Trials. Dermatitis. 2021 Oct 1;32(1S):S81-S91. doi: 10.1097/DER.0000000000000698.

ReferenceCitation: Alexis AF, Rendon M, Silverberg JI, Pariser DM, Lockshin B, Griffiths CE, Weisman J, Wollenberg A, Chen Z, Davis JD, Li M, Eckert L, Gadkari A, Shumel B, Rossi AB, Graham NM, Ardeleanu M. Efficacy of Dupilumab in Different Racial Subgroups of Adults With Moderate-to-Severe Atopic Dermatitis in Three Randomized, Placebo-Controlled Phase 3 Trials. J Drugs Dermatol. 2019 Aug 1;18(8):804-813.

ReferenceCitation: Wollenberg A, Beck LA, Blauvelt A, Simpson EL, Chen Z, Chen Q, Shumel B, Khokhar FA, Hultsch T, Rizova E, Rossi AB, Graham NMH, Pirozzi G, Lu Y, Ardeleanu M. Laboratory safety of dupilumab in moderate-to-severe atopic dermatitis: results from three phase III trials (LIBERTY AD SOLO 1, LIBERTY AD SOLO 2, LIBERTY AD CHRONOS). Br J Dermatol. 2020 May;182(5):1120-1135. doi: 10.1111/bjd.18434. Epub 2019 Dec 1.

ReferenceCitation: Silverberg JI, Simpson EL, Ardeleanu M, Thaci D, Barbarot S, Bagel J, Chen Z, Eckert L, Chao J, Korotzer A, Rizova E, Rossi AB, Lu Y, Graham NMH, Hultsch T, Pirozzi G, Akinlade B. Dupilumab provides important clinical benefits to patients with atopic dermatitis who do not achieve clear or almost clear skin according to the Investigator's Global Assessment: a pooled analysis of data from two phase III trials. Br J Dermatol. 2019 Jul;181(1):80-87. doi: 10.1111/bjd.17791. Epub 2019 Apr 11.

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

ReferenceCitation: Simpson EL. Dupilumab Improves General Health-Related Quality-of-Life in Patients with Moderate-to-Severe Atopic Dermatitis: Pooled Results from Two Randomized, Controlled Phase 3 Clinical Trials. Dermatol Ther (Heidelb). 2017 Jun;7(2):243-248. doi: 10.1007/s13555-017-0181-6. Epub 2017 May 13.

title: A Phase 3 Confirmatory Study Investigating the Efficacy and Safety of Dupilumab Monotherapy Administered to Adult Patients With Moderate-to-Severe Atopic Dermatitis

location_country:
Germany
Italy
Lithuania
Poland
United Kingdom
Hong Kong
Sweden
United States
Korea, Republic of
Canada
France


title: A Phase 2, Multicenter, International, Single Arm Study To Assess The Safety And Efficacy Of Single Agent Cc-486 (Oral Azacitidine) In Previously Treated Subjects With Locally Advanced Or Metastatic Nasopharyngeal Carcinoma

summary: The purpose of this study is to evaluate the safety and efficacy of CC-486 in previously treated patients with locally advanced or metastatic nasopharyngeal carcinoma having failed one to two previous regimens, including platinum-based chemotherapy. Participants will be enrolled according to a Simon two-stage design; if the predefined activity is met (>4 responses [complete response; partial response {CR/PR}] out of the first 17 evaluable participants based on independent radiological assessment), then the study will continue to enroll an additional 34 participants. If 4 or less responses out of 17 are observed, then the study enrollment will be stopped.

ReferenceCitation: Von Hoff DD, Rasco DW, Heath EI, Munster PN, Schellens JHM, Isambert N, Le Tourneau C, O'Neil B, Mathijssen RHJ, Lopez-Martin JA, Edenfield WJ, Martin M, LoRusso PM, Bray GL, DiMartino J, Nguyen A, Liu K, Laille E, Bendell JC. Phase I Study of CC-486 Alone and in Combination with Carboplatin or nab-Paclitaxel in Patients with Relapsed or Refractory Solid Tumors. Clin Cancer Res. 2018 Sep 1;24(17):4072-4080. doi: 10.1158/1078-0432.CCR-17-3716. Epub 2018 May 15.

repository_institution: PubMed Central - Europe PMC

repository_institution: California Digital Library - eScholarship

lead_sponsor: Celgene

location_country:
Italy
Singapore
Tunisia
United States
Spain
Greece
Taiwan
Canada
France

location_facility: Hospital Universitario Madrid Sanchinarro

location_facility: University General Hospital of Heraklion

location_facility: Instituto Catalan de Oncologia-Hospital Duran

location_facility: Columbia Comprehensive Cancer Care Clinic

location_facility: Chang Gung Medical Foundation, Kaohsiung Memorial Hospital

location_facility: Hopital Abderrahman Mami de Pneumo-Phtisiologie de l'Ariana

location_facility: Winship Cancer Institute of Emory University

location_facility: Johns Hopkins Singapore International Medical Centre


title: A Phase 2b, Randomized, Double-Blind, Placebo-Controlled Multi-Center Study Evaluating Antiviral Effects, Pharmacokinetics, Safety, and Tolerability of GS-5806 in Hematopoietic Cell Transplant (HCT) Recipients With Respiratory Syncytial Virus (RSV) Infection of the Lower Respiratory Tract

summary: The primary objective of this study is to evaluate the effect of presatovir on respiratory syncytial virus (RSV) viral load in autologous or allogeneic hematopoietic cell transplant (HCT) recipients with an acute RSV lower respiratory tract infection (LRTI).

ReferenceCitation: Marty FM, Chemaly RF, Mullane KM, Lee DG, Hirsch HH, Small CB, Bergeron A, Shoham S, Ljungman P, Waghmare A, Blanchard E, Kim YJ, McKevitt M, Porter DP, Jordan R, Guo Y, German P, Boeckh M, Watkins TR, Chien JW, Dadwal SS. A Phase 2b, Randomized, Double-blind, Placebo-Controlled Multicenter Study Evaluating Antiviral Effects, Pharmacokinetics, Safety, and Tolerability of Presatovir in Hematopoietic Cell Transplant Recipients with Respiratory Syncytial Virus Infection of the Lower Respiratory Tract. Clin Infect Dis. 2020 Dec 31;71(11):2787-2795. doi: 10.1093/cid/ciz1167.

lead_sponsor: Gilead Sciences

location_country:
France
Switzerland
United States
Korea, Republic of
Sweden

location_facility:
Hopital Saint-Louis, APHP
University of Minnesota
Medical College of Wisconsin
John Hopkins
University of Michigan
Dana Farber Cancer Institute
MD Anderson Cancer Center
City of Hope
Duke University
Seoul Saint Mary's Hospital
New York Weill Cornell Medical Center
University Clinical Basel
Karolinska Institutet
University of Chicago
CHU de Bordeaux
Fred Hutchison Cancer Research Center
Hopital Foch


title: DISCOVERY: A Study Examining the Prevalence of TTR Mutations in Subjects Suspected of Having Cardiac Amyloidosis

summary: The purpose of this study is to characterize the frequency of TTR mutations in subjects suspected of having cardiac amyloidosis

ipd_sharing: Undecided

lead_sponsor: Alnylam Pharmaceuticals

location_country:
United Kingdom
Brazil
Belgium
United States
Spain
France


summary: This is a Phase 2, open-label, multicenter study to assess the efficacy and safety of second/third-line treatment with nab-paclitaxel in combination with the epigenetic modifying therapy of CC-486 or immunotherapy of durvalumab, and nab-paclitaxel monotherapy in subjects with advanced non-small cell lung cancer (NSCLC).

ReferenceType: background

ReferenceCitation: Yardley DA, Coleman R, Conte P, Cortes J, Brufsky A, Shtivelband M, Young R, Bengala C, Ali H, Eakel J, Schneeweiss A, de la Cruz-Merino L, Wilks S, O'Shaughnessy J, Gluck S, Li H, Miller J, Barton D, Harbeck N; tnAcity investigators. nab-Paclitaxel plus carboplatin or gemcitabine versus gemcitabine plus carboplatin as first-line treatment of patients with triple-negative metastatic breast cancer: results from the tnAcity trial. Ann Oncol. 2018 Aug 1;29(8):1763-1770. doi: 10.1093/annonc/mdy201.

ReferenceCitation: Metts JL, Alazraki AL, Clark D, Amankwah EK, Wasilewski-Masker KJ, George BA, Olson TA, Cash T. Gemcitabine/nab-paclitaxel for pediatric relapsed/refractory sarcomas. Pediatr Blood Cancer. 2018 Sep;65(9):e27246. doi: 10.1002/pbc.27246. Epub 2018 May 17.

ReferenceCitation: Kim S, Signorovitch JE, Yang H, Patterson-Lomba O, Xiang CQ, Ung B, Parisi M, Marshall JL. Comparative Effectiveness of nab-Paclitaxel Plus Gemcitabine vs FOLFIRINOX in Metastatic Pancreatic Cancer: A Retrospective Nationwide Chart Review in the United States. Adv Ther. 2018 Oct;35(10):1564-1577. doi: 10.1007/s12325-018-0784-z. Epub 2018 Sep 12.

ReferenceCitation: Weiss J, Gilbert J, Deal AM, Weissler M, Hilliard C, Chera B, Murphy B, Hackman T, Liao JJ, Grilley Olson J, Hayes DN. Induction chemotherapy with carboplatin, nab-paclitaxel and cetuximab for at least N2b nodal status or surgically unresectable squamous cell carcinoma of the head and neck. Oral Oncol. 2018 Sep;84:46-51. doi: 10.1016/j.oraloncology.2018.06.028. Epub 2018 Jul 19.

ReferenceCitation: Cartwright TH, Parisi M, Espirito JL, Wilson TW, Pelletier C, Patel M, Babiker HM. Clinical Outcomes with First-Line Chemotherapy in a Large Retrospective Study of Patients with Metastatic Pancreatic Cancer Treated in a US Community Oncology Setting. Drugs Real World Outcomes. 2018 Sep;5(3):149-159. doi: 10.1007/s40801-018-0137-x.

ReferenceCitation: Li F, Zhang H, He M, Liao J, Chen N, Li Y, Zhou S, Palmisano M, Yu A, Pai MP, Yuan H, Sun D. Different Nanoformulations Alter the Tissue Distribution of Paclitaxel, Which Aligns with Reported Distinct Efficacy and Safety Profiles. Mol Pharm. 2018 Oct 1;15(10):4505-4516. doi: 10.1021/acs.molpharmaceut.8b00527. Epub 2018 Sep 21.

ReferenceCitation: Langer CJ, Kim ES, Anderson EC, Jotte RM, Modiano M, Haggstrom DE, Socoteanu MP, Smith DA, Dakhil C, Konduri K, Berry T, Ong TJ, Sanford A, Amiri K, Goldman JW, Weiss J; ABOUND.70+ Investigators. nab-Paclitaxel-Based Therapy in Underserved Patient Populations: The ABOUND.70+ Study in Elderly Patients With Advanced NSCLC. Front Oncol. 2018 Jul 24;8:262. doi: 10.3389/fonc.2018.00262. eCollection 2018.

ReferenceCitation: Gajra A, Karim NA, Mulford DA, Villaruz LC, Matrana MR, Ali HY, Santos ES, Berry T, Ong TJ, Sanford A, Amiri K, Spigel DR. nab-Paclitaxel-Based Therapy in Underserved Patient Populations: The ABOUND.PS2 Study in Patients With NSCLC and a Performance Status of 2. Front Oncol. 2018 Jul 24;8:253. doi: 10.3389/fonc.2018.00253. eCollection 2018.

ReferenceCitation: Pelzer U, Wislocka L, Juhling A, Striefler J, Klein F, Roemmler-Zehrer J, Sinn M, Denecke T, Bahra M, Riess H. Safety and efficacy of Nab-paclitaxel plus gemcitabine in patients with advanced pancreatic cancer suffering from cholestatic hyperbilirubinaemia-A retrospective analysis. Eur J Cancer. 2018 Sep;100:85-93. doi: 10.1016/j.ejca.2018.06.001. Epub 2018 Jul 4.

ReferenceCitation: Moreno L, Casanova M, Chisholm JC, Berlanga P, Chastagner PB, Baruchel S, Amoroso L, Gallego Melcon S, Gerber NU, Bisogno G, Fagioli F, Geoerger B, Glade Bender JL, Aerts I, Bergeron C, Hingorani P, Elias I, Simcock M, Ferrara S, Le Bruchec Y, Slepetis R, Chen N, Vassal G. Phase I results of a phase I/II study of weekly nab-paclitaxel in paediatric patients with recurrent/refractory solid tumours: A collaboration with innovative therapies for children with cancer. Eur J Cancer. 2018 Sep;100:27-34. doi: 10.1016/j.ejca.2018.05.002. Epub 2018 Jun 21.

ReferenceCitation: Topcul M, Ceti N IL, Ozbas Turan S, Kolusayin Ozar MO. In vitro cytotoxic effect of PARP inhibitor alone and in combination with nab-paclitaxel on triple-negative and luminal A breast cancer cells. Oncol Rep. 2018 Jul;40(1):527-535. doi: 10.3892/or.2018.6364. Epub 2018 Apr 12.

ReferenceCitation: Young R, Mainwaring P, Clingan P, Parnis FX, Asghari G, Beale P, Aly A, Botteman M, Romano A, Ferrara S, Margunato-Debay S, Harris M. nab-Paclitaxel plus gemcitabine in metastatic pancreatic adenocarcinoma: Australian subset analyses of the phase III MPACT trial. Asia Pac J Clin Oncol. 2018 Oct;14(5):e325-e331. doi: 10.1111/ajco.12999. Epub 2018 Jun 22.

ReferenceCitation: Neumann CCM, von Horschelmann E, Reutzel-Selke A, Seidel E, Sauer IM, Pratschke J, Bahra M, Schmuck RB. Tumor-stromal cross-talk modulating the therapeutic response in pancreatic cancer. Hepatobiliary Pancreat Dis Int. 2018 Oct;17(5):461-472. doi: 10.1016/j.hbpd.2018.09.004. Epub 2018 Sep 7.

ReferenceCitation: Veenstra VL, Damhofer H, Waasdorp C, van Rijssen LB, van de Vijver MJ, Dijk F, Wilmink HW, Besselink MG, Busch OR, Chang DK, Bailey PJ, Biankin AV, Kocher HM, Medema JP, Li JS, Jiang R, Pierce DW, van Laarhoven HWM, Bijlsma MF. ADAM12 is a circulating marker for stromal activation in pancreatic cancer and predicts response to chemotherapy. Oncogenesis. 2018 Nov 16;7(11):87. doi: 10.1038/s41389-018-0096-9.

ReferenceCitation: Morgensztern D, Cobo M, Ponce Aix S, Postmus PE, Lewanski CR, Bennouna J, Fischer JR, Juan-Vidal O, Stewart DJ, Fasola G, Ardizzoni A, Bhore R, Wolfsteiner M, Talbot DC, Jin Ong T, Govindan R, On Behalf Of The Abound L Investigators. ABOUND.2L+: A randomized phase 2 study of nanoparticle albumin-bound paclitaxel with or without CC-486 as second-line treatment for advanced nonsquamous non-small cell lung cancer (NSCLC). Cancer. 2018 Dec 15;124(24):4667-4675. doi: 10.1002/cncr.31779. Epub 2018 Nov 1.

ReferenceCitation: Marschner N, Salat C, Soling U, Hansen R, Grebhardt S, Harde J, Nusch A, Potthoff K. Final Effectiveness and Safety Results of NABUCCO: Real-World Data From a Noninterventional, Prospective, Multicenter Study in 697 Patients With Metastatic Breast Cancer Treated With nab-Paclitaxel. Clin Breast Cancer. 2018 Dec;18(6):e1323-e1337. doi: 10.1016/j.clbc.2018.07.010. Epub 2018 Aug 10.

ReferenceCitation: Watson S, de la Fouchardiere C, Kim S, Cohen R, Bachet JB, Tournigand C, Ferraz JM, Lefevre M, Colin D, Svrcek M, Meurisse A, Louvet C. Oxaliplatin, 5-Fluorouracil and Nab-paclitaxel as perioperative regimen in patients with resectable gastric adenocarcinoma: A GERCOR phase II study (FOXAGAST). Eur J Cancer. 2019 Jan;107:46-52. doi: 10.1016/j.ejca.2018.11.006. Epub 2018 Dec 7. Erratum In: Eur J Cancer. 2019 Sep;118:190.

ReferenceCitation: Li YF, Zhang C, Zhou S, He M, Zhang H, Chen N, Li F, Luan X, Pai M, Yuan H, Sun D, Li Y. Species difference in paclitaxel disposition correlated with poor pharmacological efficacy translation from mice to humans. Clin Pharmacol. 2018 Nov 8;10:165-174. doi: 10.2147/CPAA.S185449. eCollection 2018.

ReferenceCitation: Hurria A, Soto-Perez-de-Celis E, Blanchard S, Burhenn P, Yeon CH, Yuan Y, Li D, Katheria V, Waisman JR, Luu TH, Somlo G, Noonan AM, Lee T, Sudan N, Chung S, Rotter A, Arsenyan A, Levi A, Choi J, Rubalcava A, Morrison R, Mortimer JE. A Phase II Trial of Older Adults With Metastatic Breast Cancer Receiving nab-Paclitaxel: Melding the Fields of Geriatrics and Oncology. Clin Breast Cancer. 2019 Apr;19(2):89-96. doi: 10.1016/j.clbc.2018.10.002. Epub 2018 Oct 16.

ReferenceCitation: Fernandez A, Salgado M, Garcia A, Buxo E, Vera R, Adeva J, Jimenez-Fonseca P, Quintero G, Llorca C, Canabate M, Lopez LJ, Munoz A, Ramirez P, Gonzalez P, Lopez C, Reboredo M, Gallardo E, Sanchez-Canovas M, Gallego J, Guillen C, Ruiz-Miravet N, Navarro-Perez V, De la Camara J, Ales-Diaz I, Pazo-Cid RA, Carmona-Bayonas A. Prognostic factors for survival with nab-paclitaxel plus gemcitabine in metastatic pancreatic cancer in real-life practice: the ANICE-PaC study. BMC Cancer. 2018 Nov 29;18(1):1185. doi: 10.1186/s12885-018-5101-3.

ReferenceCitation: Awasthi N, Schwarz MA, Zhang C, Schwarz RE. Augmentation of Nab-Paclitaxel Chemotherapy Response by Mechanistically Diverse Antiangiogenic Agents in Preclinical Gastric Cancer Models. Mol Cancer Ther. 2018 Nov;17(11):2353-2364. doi: 10.1158/1535-7163.MCT-18-0489. Epub 2018 Aug 30.

ReferenceCitation: Cristea MC, Frankel P, Synold T, Rivkin S, Lim D, Chung V, Chao J, Wakabayashi M, Paz B, Han E, Lin P, Leong L, Hakim A, Carroll M, Prakash N, Dellinger T, Park M, Morgan RJ. A phase I trial of intraperitoneal nab-paclitaxel in the treatment of advanced malignancies primarily confined to the peritoneal cavity. Cancer Chemother Pharmacol. 2019 Mar;83(3):589-598. doi: 10.1007/s00280-019-03767-9. Epub 2019 Jan 8.

ReferenceCitation: Hegewisch-Becker S, Aldaoud A, Wolf T, Krammer-Steiner B, Linde H, Scheiner-Sparna R, Hamm D, Janicke M, Marschner N; TPK-Group (Tumour Registry Pancreatic Cancer). Results from the prospective German TPK clinical cohort study: Treatment algorithms and survival of 1,174 patients with locally advanced, inoperable, or metastatic pancreatic ductal adenocarcinoma. Int J Cancer. 2019 Mar 1;144(5):981-990. doi: 10.1002/ijc.31751. Epub 2018 Oct 3.

ReferenceCitation: Wang Z, Huang C, Yang JJ, Song Y, Cheng Y, Chen GY, Yan HH, Ben XS, Wang BC, Xu CR, Jiang BY, Zhou Q, Chen HJ, Wu YL. A randomised phase II clinical trial of nab-paclitaxel and carboplatin compared with gemcitabine and carboplatin as first-line therapy in advanced squamous cell lung carcinoma (C-TONG1002). Eur J Cancer. 2019 Mar;109:183-191. doi: 10.1016/j.ejca.2019.01.007. Epub 2019 Feb 7.

ReferenceCitation: Barnes JA, Ellis ML, Hwang S, Emarine J, Merwin P, Salinas GD, Musher BL. Identification of Educational Gaps Among Oncologists Who Manage Patients with Pancreatic Cancer. J Gastrointest Cancer. 2019 Mar;50(1):84-90. doi: 10.1007/s12029-017-0033-8.

ReferenceCitation: Woo W, Carey ET, Choi M. Spotlight on liposomal irinotecan for metastatic pancreatic cancer: patient selection and perspectives. Onco Targets Ther. 2019 Feb 21;12:1455-1463. doi: 10.2147/OTT.S167590. eCollection 2019.

ReferenceCitation: Seiwert TY, Foster CC, Blair EA, Karrison TG, Agrawal N, Melotek JM, Portugal L, Brisson RJ, Dekker A, Kochanny S, Gooi Z, Lingen MW, Villaflor VM, Ginat DT, Haraf DJ, Vokes EE. OPTIMA: a phase II dose and volume de-escalation trial for human papillomavirus-positive oropharyngeal cancer. Ann Oncol. 2019 Feb 1;30(2):297-302. doi: 10.1093/annonc/mdy522. Erratum In: Ann Oncol. 2019 Oct 1;30(10):1673.

ReferenceCitation: De Luca R, Profita G, Cicero G. Nab-paclitaxel in pretreated metastatic breast cancer: evaluation of activity, safety, and quality of life. Onco Targets Ther. 2019 Feb 26;12:1621-1627. doi: 10.2147/OTT.S191519. eCollection 2019.

ReferenceCitation: Morgensztern D, Ko A, O'Brien M, Ong TJ, Waqar SN, Socinski MA, Postmus PE, Bhore R. Association between depth of response and survival in patients with advanced-stage non-small cell lung cancer treated with first-line chemotherapy. Cancer. 2019 Jul 15;125(14):2394-2399. doi: 10.1002/cncr.32114. Epub 2019 Apr 1.

ReferenceCitation: Li F, Yuan H, Zhang H, He M, Liao J, Chen N, Li Y, Zhou S, Palmisano M, Yu A, Pai M, Sun D. Neonatal Fc Receptor (FcRn) Enhances Tissue Distribution and Prevents Excretion of nab-Paclitaxel. Mol Pharm. 2019 Jun 3;16(6):2385-2393. doi: 10.1021/acs.molpharmaceut.8b01314. Epub 2019 May 1.

ReferenceCitation: Sonbol MB, Ahn DH, Goldstein D, Okusaka T, Tabernero J, Macarulla T, Reni M, Li CP, O'Neil B, Van Cutsem E, Bekaii-Saab T. CanStem111P trial: a Phase III study of napabucasin plus nab-paclitaxel with gemcitabine. Future Oncol. 2019 Apr;15(12):1295-1302. doi: 10.2217/fon-2018-0903. Epub 2019 Feb 15.

ReferenceCitation: Morgensztern D, Dols MC, Ponce Aix S, Postmus PE, Bennouna J, Fischer JR, Juan-Vidal O, Stewart DJ, Ardizzoni A, Bhore R, Wolfsteiner M, Reck M, Talbot D, Govindan R, Ong TJ. nab-Paclitaxel Plus Durvalumab in Patients With Previously Treated Advanced Stage Non-small Cell Lung Cancer (ABOUND.2L+). Front Oncol. 2021 Feb 11;10:569715. doi: 10.3389/fonc.2020.569715. eCollection 2020.

endpoint_id: fhisxppmrqpajgzqkvft

repository_institution: Alma Mater Studiorum Università di Bologna - Archivio istituzionale della ricerca - Alma Mater Studiorum Università di Bologna

repository_institution: Leiden University - Leiden Repository

publisher_dissemination: Frontiers

location_facility: UCSF Helen Diller Medical Center at Parnassus Heights

location_facility: Weill Cornell Medical College - New York - Presbyterian Hospital

location_facility: John Theurer Cancer Center at Hackensack University Medical Center

location_facility: Hospital of Central Connecticut Gynecologic Oncology

location_facility: Penn State Milton S Hershey Medical Center

title: A Phase 2, Open-Label, Multi-Center Study to Assess Safety and Efficacy of Second/Third-Line Treatment With NAB®-Paclitaxel (ABI-007) In Combination With Epigenetic Modifying Therapy Of CC-486, Or Immunotherapy of Durvalumab (MEDI4736), Or As Monotherapy In Subjects With Advanced Non-Small Cell Lung Cancer (NSCLC): Abound.2L+

design_allocation: Non-Randomized

location_country:
Germany
Italy
United Kingdom
United States
Spain
Canada
France


title: An Open-Label, Extension Study of the Effects of LMTM in Subjects With Alzheimer's Disease or Behavioral Variant Frontotemporal Dementia (bvFTD)

summary: The purpose of this study is to provide subjects who have completed participation in a Phase 2 or Phase 3 trial of LMTM continued access to therapy and to evaluate the long-term safety of LMTM.

status: Terminated

lead_sponsor: TauRx Therapeutics Ltd

location_country:
Finland
Germany
United Kingdom
Singapore
Romania
Australia
Netherlands
Belgium
United States
Korea, Republic of
Spain
Malaysia
Croatia
Taiwan
Canada
Russian Federation
France

location_facility: University Hospital Ghent Department of Neurology

location_facility: University Kuala Lumpur Royal College of Medicine

location_facility: True North Clinical Research Kentville Inc

location_facility: Belfast Health and Social Care Trust (BHSCT)

location_facility: Research Foundation for Mental Hygiene, Inc.

location_facility: University Hospital Centre Zagreb, Department of Neurology

location_facility: Wessex Neurological Centre, Southampton General Hospital

location_facility: Imperial College Healthcare NHS Trust - Charing Cross Hospital

location_facility: Clinical Neuroscience Solutions CNS Healthcare

location_facility: St Margaret's Hospital Mental Health Unit

location_facility: City Clinical Hospital #34, City Scientific Practical Neurological Center

location_facility: McCusker Alzheimer's Research Foundation Inc

location_facility: Mayo Clinic, Alzheimer's Disease Research Center

location_facility: University of Eastern Finland, Brain Research Unit Mediteknia

location_facility: Nuffield Department of Clinical Neurosciences

location_facility: Neurodegenerative Disorders Research Pty Ltd

location_facility: City Geriatric Medical and Social Center

location_facility: Charité, University Medicine Berlin, CBF, Neurology

location_facility: Non-governmental Healthcare Institution "Central Clinical Hospital #6 of the JSC "Russian Railways"

location_facility: State Budgetary Healthcare Institution of Sverdlovsk region "Sverdlovsk Regional Clinical Psychiatric Hospital"

location_facility: Memory Enhancement Center of America, Inc

location_facility: Memory Assessment and Research Centre (MARC)

location_facility: Neurological Research Center - Hattiesburg Clinic

location_facility: Xenoscience, Inc / 21st Century Neurology

location_facility: Mental Health Research Center of the Russian Academy of Medical Sciences, Gerontopsychiatry department

location_facility: Ruan Neurology Clinic and Research Center

location_facility: Medical and Cognitive Research Unit, Austin Health Heidelberg Repatriation Hospital

location_facility: Hospital of the University of Pennsylvania, Department of Neurology

location_facility: Saint Petersburg Psychoneurological Research Institute n. a. V.M. Bekhterev

location_facility: Institute for Neurodegenerative Disorders

location_facility: Alexian Brothers Neurosciences Institute

location_facility: True North Clinical Research Halifax Inc

location_facility: Advanced Memory Research Institute of NJ PC

location_facility: Royal Adelaide Hospital Memory Trials Centre

location_facility: University Hospitals Case Medical Center, Neurology Clinical Trials Unit

location_facility: Psychiatry Hospital "Dr. Gheorghe Preda", Department of Psychiatry III

location_facility: Geriatric Clinical Trials Group, St. Joseph's Health Care, Parkwood Hospital Site

location_facility: Neurological Associates of Albany, P. C.

location_facility: Kingshill Research Centre, Victoria Hospital

location_facility: Discipline of Psychiatry, University of Queensland

location_facility: University of Virginia Adult Neurology, Primary Care Center

location_facility: Leonard Wolfson Experimental Neurology Centre

location_facility: Seoul National University Boramae Medical Center

location_facility: Sussex Partnership NHS Foundation Trust, Cognitive Treatment and Research Unit

location_facility: RICE - The Research Institute for the Care of Older People

location_facility: McGill Centre for Studies in Aging, Alzheimer Disease Research Unit

location_facility: University of British Columbia Hospital, Clinic for Alzheimer Disease and Related Disorders

location_facility: Division of Rehabilitation and Aged Care

location_facility: UNC Department of Neurology, Physicians Office Building

status_simplified: Unknown


title: A Phase 2A Study of ALXN1007 in Subjects With Newly Diagnosed Acute Graft-Versus-Host Disease Involving the Lower Gastrointestinal Tract

acronym: GIGVHD

summary: The objectives of this trial were to evaluate the safety, tolerability, pharmacokinetics/pharmacodynamics (PK/PD) and efficacy of intravenous (IV) ALXN1007 in participants with acute graft-versus-host disease (GVHD) of the lower gastrointestinal (GI) tract.

lead_sponsor: Alexion Pharmaceuticals, Inc.

location_country:
United States
France

location_facility: The University of Texas MD Anderson Cancer Center

location_facility: Groupe Hospitalier Sud - Hôpital Haut-Lévêque - Centre François

location_facility: Abramson Cancer Perelman Center for Advanced Medicine, University of Pennsylvania

location_facility: University of Minnesota Medicine - Hematology, Oncology and Transplantation Office

intervention_type: Biological


acronym: EINSTEIN Jr

lead_sponsor: Bayer

summary: The purpose of this study is to evaluate comparative efficacy and safety of rivaroxaban to standard of care in children with acute venous thromboembolism.

ReferenceCitation: Palumbo JS, Lensing AWA, Brandao LR, Hooimeijer HL, Kenet G, van Ommen H, Pap AF, Majumder M, Kubitza D, Thelen K, Willmann S, Prins MH, Monagle P, Male C. Anticoagulation in pediatric cancer-associated venous thromboembolism: a subgroup analysis of EINSTEIN-Jr. Blood Adv. 2022 Nov 22;6(22):5821-5828. doi: 10.1182/bloodadvances.2022008160.

evidence: open (via page says license)

repository_institution: Erasmus University Rotterdam - EUR Research Repository

repositories_concat: PubMed Central;pure.eur.nl;pure.rug.nl

repositories_institution: DANS - Data Archiving and Networked Services - NARCIS - National Academic Research and Collaborations Information System

repositories_institution: University of Groningen / Centre for Information Technology - University of Groningen research database

publisher_dissemination: American Society of Hematology

ReferenceCitation: Connor P, Sanchez van Kammen M, Lensing AWA, Chalmers E, Kallay K, Hege K, Simioni P, Biss T, Bajolle F, Bonnet D, Grunt S, Kumar R, Lvova O, Bhat R, Van Damme A, Palumbo J, Santamaria A, Saracco P, Payne J, Baird S, Godder K, Labarque V, Male C, Martinelli I, Morales Soto M, Motwani J, Shah S, Hooimeijer HL, Prins MH, Kubitza D, Smith WT, Berkowitz SD, Pap AF, Majumder M, Monagle P, Coutinho JM. Safety and efficacy of rivaroxaban in pediatric cerebral venous thrombosis (EINSTEIN-Jr CVT). Blood Adv. 2020 Dec 22;4(24):6250-6258. doi: 10.1182/bloodadvances.2020003244.

evidence: oa journal (via observed oa rate)

repository_institution: Ural Federal University - Electronic scientific archive of UrFU

repositories:
LIRIAS (KU Leuven)
PubMed Central
elar.urfu.ru

repositories_concat: LIRIAS (KU Leuven);PubMed Central;elar.urfu.ru

ReferenceCitation: Thom K, Lensing AWA, Nurmeev I, Bajolle F, Bonnet D, Kenet G, Massicotte MP, Karakas Z, Palumbo JS, Saracco P, Amedro P, Chain J, Chan AK, Ikeyama T, Lam JCM, Gauger C, Pap AF, Majumder M, Kubitza D, Smith WT, Berkowitz SD, Prins MH, Monagle P, Young G, Male C. Safety and efficacy of anticoagulant therapy in pediatric catheter-related venous thrombosis (EINSTEIN-Jr CVC-VTE). Blood Adv. 2020 Oct 13;4(19):4632-4639. doi: 10.1182/bloodadvances.2020002637.

repository_normalized: HAL

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université de Nantes

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

repositories_institution: Le Centre pour la Communication Scientifique Directe - HAL - memSIC

ReferenceCitation: Male C, Lensing AWA, Palumbo JS, Kumar R, Nurmeev I, Hege K, Bonnet D, Connor P, Hooimeijer HL, Torres M, Chan AKC, Kenet G, Holzhauer S, Santamaria A, Amedro P, Chalmers E, Simioni P, Bhat RV, Yee DL, Lvova O, Beyer-Westendorf J, Biss TT, Martinelli I, Saracco P, Peters M, Kallay K, Gauger CA, Massicotte MP, Young G, Pap AF, Majumder M, Smith WT, Heubach JF, Berkowitz SD, Thelen K, Kubitza D, Crowther M, Prins MH, Monagle P; EINSTEIN-Jr Phase 3 Investigators. Rivaroxaban compared with standard anticoagulants for the treatment of acute venous thromboembolism in children: a randomised, controlled, phase 3 trial. Lancet Haematol. 2020 Jan;7(1):e18-e27. doi: 10.1016/S2352-3026(19)30219-4. Epub 2019 Nov 5.

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

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université Toulouse - Jean Jaurès

ReferenceCitation: Monagle P, Lensing AWA, Thelen K, Martinelli I, Male C, Santamaria A, Samochatova E, Kumar R, Holzhauer S, Saracco P, Simioni P, Robertson J, Grangl G, Halton J, Connor P, Young G, Molinari AC, Nowak-Gottl U, Kenet G, Kapsa S, Willmann S, Pap AF, Becka M, Twomey T, Beyer-Westendorf J, Prins MH, Kubitza D; EINSTEIN-Jr Phase 2 Investigators. Bodyweight-adjusted rivaroxaban for children with venous thromboembolism (EINSTEIN-Jr): results from three multicentre, single-arm, phase 2 studies. Lancet Haematol. 2019 Oct;6(10):e500-e509. doi: 10.1016/S2352-3026(19)30161-9. Epub 2019 Aug 13.

ReferenceCitation: Lensing AWA, Male C, Young G, Kubitza D, Kenet G, Patricia Massicotte M, Chan A, Molinari AC, Nowak-Goettl U, Pap AF, Adalbo I, Smith WT, Mason A, Thelen K, Berkowitz SD, Crowther M, Schmidt S, Price V, Prins MH, Monagle P. Rivaroxaban versus standard anticoagulation for acute venous thromboembolism in childhood. Design of the EINSTEIN-Jr phase III study. Thromb J. 2018 Dec 21;16:34. doi: 10.1186/s12959-018-0188-y. eCollection 2018.

repository_institution: University of Melbourne - Minerva Access

sponsor_collaborators:
Bayer
Janssen Research & Development, LLC

title: Multicenter, Open-label, Active-controlled, Randomized Study to Evaluate the Efficacy and Safety of an age-and Body Weight-adjusted Rivaroxaban Regimen Compared to Standard of Care in Children With Acute Venous Thromboembolism

location_country:
Finland
Germany
Ireland
Argentina
Australia
Brazil
Japan
Turkey
Canada
France
Portugal
Slovakia
Hungary
Hong Kong
Switzerland
United States
Belgium
China
Italy
United Kingdom
Singapore
Sweden
Netherlands
Austria
Mexico
Spain
Russian Federation
Israel


title: An Open-label Extension Study of DS-5565 for 52 Weeks in Pain Associated With Fibromyalgia

summary: This is an open-label study of DS-5565 in subjects who either completed participation in a preceding Phase 3 study of DS-5565 in fibromyalgia (FM); i.e. DS5565-A-E309 (NCT02146430), DS5565-A-E310 (NCT02187471), or DS5565-A-E311 (NCT02187159) or are de novo subjects. Eligible subjects will be assigned to receive open-label DS-5565 for 52 weeks. All subjects will receive DS-5565 15 mg once daily (QD) for the first three weeks of the treatment period. After three weeks, subjects may be titrated to 15 mg twice daily (BID) based on protocol-specified criteria.

ReferenceCitation: Arnold LM, Whitaker S, Hsu C, Jacobs D, Merante D. Efficacy and safety of mirogabalin for the treatment of fibromyalgia: results from three 13-week randomized, double-blind, placebo- and active-controlled, parallel-group studies and a 52-week open-label extension study. Curr Med Res Opin. 2019 Oct;35(10):1825-1835. doi: 10.1080/03007995.2019.1629757. Epub 2019 Jul 9.

oa_host_type: closed

publisher_dissemination: Informa

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

lead_sponsor: Daiichi Sankyo, Inc.

location_country:
Finland
Germany
Poland
New Zealand
Australia
Chile
Czechia
Canada
Serbia
France
Slovenia
Lithuania
Portugal
Slovakia
Hungary
Denmark
United States
Latvia
Ukraine
Romania
Estonia
Puerto Rico
Norway
United Kingdom
Austria
Spain
South Africa
Bulgaria
Russian Federation


title: A Phase 2, Dose-Ranging, Randomized, Double-Blind, Placebo-Controlled Study of GS-4997 in Subjects With Pulmonary Arterial Hypertension

acronym: ARROW

summary: The primary objective of this study is to evaluate the effect of selonsertib (GS-4997) on pulmonary vascular resistance (PVR), as measured by right heart catheterization (RHC) in adults with pulmonary arterial hypertension (PAH). The study will consist of a 24-week placebo-controlled treatment period and a long-term selonsertib treatment period. Participants completing the 24-week placebo-controlled period will be eligible to receive active treatment with selonsertib in the long-term treatment period.

ReferenceCitation: Rosenkranz S, Feldman J, McLaughlin VV, Rischard F, Lange TJ, White RJ, Peacock AJ, Gerhardt F, Ebrahimi R, Brooks G, Satler C, Frantz RP; ARROW Study Group. Selonsertib in adults with pulmonary arterial hypertension (ARROW): a randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Respir Med. 2022 Jan;10(1):35-46. doi: 10.1016/S2213-2600(21)00032-1. Epub 2021 Aug 20.

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

location_facility: Universita "Sapienza"-Azienda Policlinico Umberto 1

location_facility: Institut Universitaire de caridologie et de pneumologie de Quebee (IUCPQ)

location_facility: CHU de Bicetre, Service de Pneumologie-Reanimation Respiratoire

location_facility: University of Arizona Clinical and Translational Science (CATS) Research Center

location_facility: LA Biomedical Research Institute Harbor-UCLA Medical Center

location_facility: Klinik und Poliklinik fur Innere Medizin II, Universitatsklinikum Regensburg

location_facility: Victoria Hospital - London Health Sciences Centre

location_facility: Universitatsklinikul Giessen und Marburg GmbH

location_facility: Vancouver General Hospital, The Lung Centre // Vancouver Coastal Health, Vancouver General Hospital

location_facility: CHU de Grenoble Clinique Universitaire de Pneumologie

location_facility: University of South Alabama Medical Center

location_facility: Washington University School of Medicine

location_facility: Klinik III fur Innere Medizin, Herzzentrum Uniklinik Koln

location_facility: Hospital Universitario Doce (12) de Octubre

location_facility: Scottish Pulmonary Vascular Unit, Golden Jubilee National Hospital

location_facility: University of Colorado Cardiac and Vascular Center, Anschutz Inpatient Pavillion

location_facility: Clinical Research Facility, Royal Hallamshrie Hospital

location_facility: Mary M. Parkes Center // University of Rochester Medical Center

location_facility: VA Greater Los Angeles Healthcare System

location_facility: Hopital Cardiologique-CHRU Lille, Service de cardiologie


title: Pilot Testing of SAGIT in Patients With Acromegaly in Clinical Practice

acronym: SAGIT Pilot

summary:
The purpose of this pilot study is to test SAGIT (Signs and symptoms - Associated comorbidities - GH concentration level - IGF-1 - Tumour). SAGIT is a Clinician-Reported Outcomes (ClinROs) tool developed to describe patients with acromegaly. This study will determine the potential use of a finalised operational version for patient classification in clinical practice and studies.

In addition, this study intends to carry out a qualitative evaluation of the acceptability of SAGIT by the practicing endocrinologist in terms of relevance, ease of use, applicability and usefulness of the tool in practice.

time_perspective: Cross-Sectional

ReferenceCitation: Giustina A, Bevan JS, Bronstein MD, Casanueva FF, Chanson P, Petersenn S, Thanh XM, Sert C, Houchard A, Guillemin I, Melmed S; SAGIT Investigator Group. SAGIT(R): clinician-reported outcome instrument for managing acromegaly in clinical practice--development and results from a pilot study. Pituitary. 2016 Feb;19(1):39-49. doi: 10.1007/s11102-015-0681-2.

lead_sponsor: Ipsen

location_country:
Germany
Italy
United Kingdom
Brazil
United States
Spain
France

location_facility: Praxisgemeinschaft an der Kaisereiche, Wilhelm-Hauff-Straße 21

location_facility: NW Pituitary Center and Neurological Surgery

location_facility: Azienda Ospedaliera Spedali Civil di Brescia

location_facility: Universitary Hospital Virgen de la Vitoria

location_facility: Azienda Ospedaliera Città della Salute e della Scienza

intervention_type: Other


lead_sponsor: MedImmune LLC

summary: The primary objective of this study is to evaluate the efficacy of moxetumomab pasudotox in pediatric participants with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) or B-cell lymphoblastic lymphoma.

ReferenceCitation: Mussai F, Campana D, Bhojwani D, Stetler-Stevenson M, Steinberg SM, Wayne AS, Pastan I. Cytotoxicity of the anti-CD22 immunotoxin HA22 (CAT-8015) against paediatric acute lymphoblastic leukaemia. Br J Haematol. 2010 Aug;150(3):352-8. doi: 10.1111/j.1365-2141.2010.08251.x. Epub 2010 Jun 7.

title: A Phase 2, Multicenter, Single-arm Study of Moxetumomab Pasudotox in Pediatric Subjects With Relapsed or Refractory Pediatric Acute Lymphoblastic Leukemia (pALL) or Lymphoblastic Lymphoma of B-cell Origin

location_country:
Italy
United Kingdom
Australia
Netherlands
United States
Spain
Canada
France


title: A Combined Phase IIa / IIb Study of the Efficacy, Safety, and Tolerability of Repeated Topical Doses of Regorafenib Eye Drops, in Treatment-naïve Subjects With Neovascular Age Related Macular Degeneration

acronym: DREAM

summary:
Part A (Phase IIa):

Primary objectives:

The study part A is designed to investigate whether the use of regorafenib eye drops can help patients with neovascular (wet) Age-Related Macular Degeneration (wAMD) to see better after 4 weeks and 12 weeks after inclusion into this study.

Secondary objectives:

The study will also evaluate the safety and tolerability of the regorafenib eye drops.

Part B (Phase IIb):

Primary objectives:

The study part B is designed to investigate:

how often the regorafenib eye drops need to be given per day
whether the use of regorafenib eye drops can help patients with neovascular (wet) Age-Related Macular Degeneration (wAMD) to see better after 4 weeks and 12 weeks after inclusion into this study.

Secondary objectives:

The study will also evaluate how the different dosings of regorafenib eye drops affect patients vision, the safety and the tolerability.

location_country:
France
Germany
Italy
Colombia
Slovakia
Hungary
Hong Kong
Israel
Australia
Japan
Switzerland
Austria
United States
Korea, Republic of
Spain
Chile
Canada
Czech Republic


title: An Open-label, Multicenter, Phase IIIb Study Assessing the Long-term Efficacy and Safety of AOP2014 and Standard First Line Treatment (BAT) in Patients With Polycythemia Vera Who Previously Participated in the PROUD-PV Study

acronym: CONTI-PV

summary:
Polycythemia Vera (PV) is a disease of bone marrow stem cells that manifests in a drastic increase of red blood cells and frequently also of white blood cells. The "thickening" of the blood in relation with a modified function of the cells has several consequences like increased blood pressure, pruritus of the skin, fatigue, disturbed blood circulation in the brain as well as fingers and toes and an increased risk of arterial and venous thrombosis (thrombosis is the formation of a blood clot in a vessel); like stroke, cardiac infarction, deep vein thrombosis in the legs. In case of a strong increase of platelets there is an additional risk of bleedings. As the disease progresses the size of spleen and liver increased in most cases and the bone marrow shows signs of fibrosis. In some cases of PV a progression at a later time point to a leukemia (increased formation of white blood cells) can occur.

The aim of this study is to show that the study drug AOP2014 (pegylated proline interferon alpha-2b) has the long term efficacy and safety in controlling the disease. A comparison arm is receiving best available therapy as selected by the investigator. Response to the treatment is measured by several blood parameters as well as size of the spleen.

Interferon-alpha has been shown to be effective in controlling the blood parameters by immunologically influencing the blood building cells. This can lead to a suppression of the disease-causing stem cells and help healthy stem cells to proliferate. Through this mechanism it is possible that Interferon-alpha can avoid long-term damaging effects of the disease.

primary_purpose: Other

lead_sponsor: AOP Orphan Pharmaceuticals AG

collaborators: PharmaEssentia Corporation (for the U.S.)

location_country:
Germany
Poland
Slovakia
Hungary
Romania
Austria
Spain
Czechia
Bulgaria
Ukraine
Russian Federation
France

location_facility: University Multiprofile Hospital for Active Treatment "Sveti Georgi", Plovdiv

location_facility: Independent Public Teaching Hospital No.1 in Lublin

location_facility: Saint Cyril and Metod University Hospital Bratislava

location_facility: Samara Kalinin Regional Clinical Hospital

location_facility: Dnipropetrovsk City Multispecialty Clinical Hospital #4

location_facility: Emergency Clinical County Hospital Brasov

location_facility: St Istvan and St Laszlo Hospital of Budapest

location_facility: Specialized Hospital for Active Treatment of Hematological Diseases

location_facility: University of Szeged, Albert Szent-Gyorgyi Clinical Center, Koranyi fasor 6

location_facility: Multiprofile Hospital for Active Treatment - Hristo Botev, Vratsa, First Department of Internal Medicine

location_facility: National Research Center for Radiation Medicine, Institute of Clinical Radiology

location_facility: Cherkasy Regional Oncology Center, Regional Treatment and Diagnostics Hematology Center

location_facility: University Hospital Carl Gustav Carus, Medical Clinic and Polyclinic I

location_facility: University Hospital with Outpatient Clinic F.D. Roosevelt

location_facility: Nicolaus Copernicus Municipal Specialist Hospital

location_facility: University Hospital Bonn, Center for Internal Medicine, Medical Clinic and Outpatient Clinic III

location_facility: O.F. Herbachevskyi Regional Clinical Hospital

location_facility: Institute of Blood Pathology and Transfusion Medicine

location_facility: Aachen University Hospital, Medical Clinic IV

location_facility: Multiprofile Hospital for Active Treatment "Sveta Marina", Varna

location_facility: Bekes County Pandy Kalman Hospital, 1st Department of Medicine, Hematology

location_facility: Fryderyk Chopin Provincial Specialized Hospital

location_facility: Institute of Hematology and Transfusion Medicine


title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of ISIS 304801 Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)

summary: The purpose of this study is to evaluate the efficacy and safety of volanesorsen given for 52 weeks in participants with Familial Chylomicronemia Syndrome

ReferenceCitation: Witztum JL, Gaudet D, Freedman SD, Alexander VJ, Digenio A, Williams KR, Yang Q, Hughes SG, Geary RS, Arca M, Stroes ESG, Bergeron J, Soran H, Civeira F, Hemphill L, Tsimikas S, Blom DJ, O'Dea L, Bruckert E. Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome. N Engl J Med. 2019 Aug 8;381(6):531-542. doi: 10.1056/NEJMoa1715944.

repository_institution: Universidad de Zaragoza - Zaguan

ReferenceCitation: Hegele RA, Berberich AJ, Ban MR, Wang J, Digenio A, Alexander VJ, D'Erasmo L, Arca M, Jones A, Bruckert E, Stroes ES, Bergeron J, Civeira F, Witztum JL, Gaudet D. Clinical and biochemical features of different molecular etiologies of familial chylomicronemia. J Clin Lipidol. 2018 Jul-Aug;12(4):920-927.e4. doi: 10.1016/j.jacl.2018.03.093. Epub 2018 Apr 4.

ReferenceCitation: Digenio A, Dunbar RL, Alexander VJ, Hompesch M, Morrow L, Lee RG, Graham MJ, Hughes SG, Yu R, Singleton W, Baker BF, Bhanot S, Crooke RM. Antisense-Mediated Lowering of Plasma Apolipoprotein C-III by Volanesorsen Improves Dyslipidemia and Insulin Sensitivity in Type 2 Diabetes. Diabetes Care. 2016 Aug;39(8):1408-15. doi: 10.2337/dc16-0126. Epub 2016 Jun 6.

publisher_dissemination: American Diabetes Association

lead_sponsor: Ionis Pharmaceuticals, Inc.

sponsor_collaborators:
Ionis Pharmaceuticals, Inc.
Akcea Therapeutics

location_country:
Germany
Italy
Hungary
Israel
United Kingdom
Brazil
Netherlands
United States
Spain
South Africa
Canada
France


title: A Phase 1b Study of ABT-199 (GDC-0199) in Combination With Azacitidine or Decitabine in Treatment-Naive Subjects With Acute Myelogenous Leukemia Who Are Greater Than or Equal to 60 Years of Age and Who Are Not Eligible for Standard Induction Therapy

summary: This is a Phase 1b, open-label, non-randomized, multicenter study to evaluate the safety and pharmacokinetics of orally administered venetoclax (ABT-199) combined with decitabine or azacitidine and the preliminary efficacy of these combinations. In addition, there is a drug-drug interaction (DDI) sub-study only at a single site, to assess the pharmacokinetics and safety of venetoclax (ABT-199) in combination with posaconazole.

ReferenceCitation: Badawi M, Chen X, Marroum P, Suleiman AA, Mensing S, Koenigsdorfer A, Schiele JT, Palenski T, Samineni D, Hoffman D, Menon R, Salem AH. Bioavailability Evaluation of Venetoclax Lower-Strength Tablets and Oral Powder Formulations to Establish Interchangeability with the 100 mg Tablet. Clin Drug Investig. 2022 Aug;42(8):657-668. doi: 10.1007/s40261-022-01172-4. Epub 2022 Jul 13.

ReferenceCitation: Konopleva M, Thirman MJ, Pratz KW, Garcia JS, Recher C, Pullarkat V, Kantarjian HM, DiNardo CD, Dail M, Duan Y, Chyla B, Potluri J, Miller CL, Wei AH. Impact of FLT3 Mutation on Outcomes after Venetoclax and Azacitidine for Patients with Treatment-Naive Acute Myeloid Leukemia. Clin Cancer Res. 2022 Jul 1;28(13):2744-2752. doi: 10.1158/1078-0432.CCR-21-3405.

ReferenceCitation: Pollyea DA, DiNardo CD, Arellano ML, Pigneux A, Fiedler W, Konopleva M, Rizzieri DA, Smith BD, Shinagawa A, Lemoli RM, Dail M, Duan Y, Chyla B, Potluri J, Miller CL, Kantarjian HM. Impact of Venetoclax and Azacitidine in Treatment-Naive Patients with Acute Myeloid Leukemia and IDH1/2 Mutations. Clin Cancer Res. 2022 Jul 1;28(13):2753-2761. doi: 10.1158/1078-0432.CCR-21-3467.

ReferenceCitation: DiNardo CD, Pratz K, Pullarkat V, Jonas BA, Arellano M, Becker PS, Frankfurt O, Konopleva M, Wei AH, Kantarjian HM, Xu T, Hong WJ, Chyla B, Potluri J, Pollyea DA, Letai A. Venetoclax combined with decitabine or azacitidine in treatment-naive, elderly patients with acute myeloid leukemia. Blood. 2019 Jan 3;133(1):7-17. doi: 10.1182/blood-2018-08-868752. Epub 2018 Oct 25.

repository_institution: Monash University - Monash University Research Portal

ReferenceCitation: DiNardo CD, Pratz KW, Letai A, Jonas BA, Wei AH, Thirman M, Arellano M, Frattini MG, Kantarjian H, Popovic R, Chyla B, Xu T, Dunbar M, Agarwal SK, Humerickhouse R, Mabry M, Potluri J, Konopleva M, Pollyea DA. Safety and preliminary efficacy of venetoclax with decitabine or azacitidine in elderly patients with previously untreated acute myeloid leukaemia: a non-randomised, open-label, phase 1b study. Lancet Oncol. 2018 Feb;19(2):216-228. doi: 10.1016/S1470-2045(18)30010-X. Epub 2018 Jan 12.

ReferenceCitation: Agarwal SK, DiNardo CD, Potluri J, Dunbar M, Kantarjian HM, Humerickhouse RA, Wong SL, Menon RM, Konopleva MY, Salem AH. Management of Venetoclax-Posaconazole Interaction in Acute Myeloid Leukemia Patients: Evaluation of Dose Adjustments. Clin Ther. 2017 Feb;39(2):359-367. doi: 10.1016/j.clinthera.2017.01.003. Epub 2017 Feb 1.

location_facility: Columbia University Medical Center /ID# 130289

location_facility: Universitaetsklinikum Carl Gustav Carus an der TU Dresden /ID# 130342

location_facility: Duplicate_Hopital Universitaire Purpan /ID# 134389

location_facility: Univ of Colorado Cancer Center /ID# 127859

location_facility: University of California, Davis Comprehensive Cancer Center /ID# 129719

location_facility: Northwestern University Feinberg School of Medicine /ID# 128741

location_facility: University of Texas MD Anderson Cancer Center /ID# 141581

location_facility: University of Texas MD Anderson Cancer Center /ID# 127860

location_facility: Universitaetsklinikum Leipzig /ID# 130346

location_facility: Dana-Farber Cancer Institute /ID# 127857

location_facility: Duplicate_Klinikum Rechts der Isar /ID# 130347

location_facility: The University of Chicago Medical Center /ID# 128742

location_facility: Emory Midtown Infectious Disease Clinic /ID# 129715


lead_sponsor: Novartis Pharmaceuticals

summary: This study is designed to assess the safety, tolerability, pharmacokinetics and preliminary pharmacodynamics (proof of concept) of QBW251 in healthy subjects and cystic fibrosis patients following single and multiple doses. This first-in-human and proof of concept study will consist of 4 parts, with Parts 1 and 2 in healthy volunteers and Parts 3 and 4 in cystic fibrosis patients.

ReferenceCitation: Kazani S, Rowlands DJ, Bottoli I, Milojevic J, Alcantara J, Jones I, Kulmatycki K, Machineni S, Mostovy L, Nicholls I, Nick JA, Rowe SM, Simmonds NJ, Vegesna R, Verheijen J, Danahay H, Gosling M, Ayalavajjala PS, Salman M, Strieter R. Safety and efficacy of the cystic fibrosis transmembrane conductance regulator potentiator icenticaftor (QBW251). J Cyst Fibros. 2021 Mar;20(2):250-256. doi: 10.1016/j.jcf.2020.11.002. Epub 2020 Dec 6.

title: A Randomized, Double Blind Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Preliminary Pharmacodynamics of Single and Multiple Ascending Doses of QBW251 in Healthy Subjects and Multiple Doses in Cystic Fibrosis Patients

location_country:
Ireland
Germany
United Kingdom
Romania
Belgium
United States
France


title: 10-day Decitabine Versus Conventional Chemotherapy ("3+7") Followed by Allografting in AML Patients ≥ 60 Years: a Randomized Phase III Study of the EORTC Leukemia Group, CELG, GIMEMA and German MDS Study Group

summary:
Older patients with acute myeloid leukemia (AML) have a small (< 10%) chance of long-term survival. Despite the treatment of elderly AML patients with intensive chemotherapy, the survival has not been improved during the last decades.

The purpose of this study is to determine whether frontline therapy with a 10-day decitabine schedule provides a better survival than standard intensive combination chemotherapy in elderly AML patients (>= 60 years).

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

collaborators: Gruppo Italiano Malattie EMatologiche dell'Adulto

location_country: Germany Italy Lithuania Portugal Netherlands Belgium Croatia Bulgaria France

location_facility: Azienda Ospedallera Universitaria - Policlinico Tor Vergata

location_facility: Universitätsklinikum Aachen AÖR - Medizinische Fakultät der RWTH

location_facility: Vilnius University Hospital Santariskiu Clinics

location_facility: National Specialized Hospital for Active Treatment of Haematological Diseases

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

location_facility: Helios Kliniken - Helios Klinikum Berlin-Buch

location_facility: AUSL Romagna - Ospedale Infermi di Rimini

location_facility: Universitaet Rostock - Medizinische Fakultaet

location_facility: Universita Degli Studi Di Roma La Sapienza - Ospedale Sant'Andrea

location_facility: Assistance Publique - Hôpitaux de Paris - Hôpital Saint Antoine

location_facility: Universitaetsklinikum Tuebingen-Uni Kliniken Berg

location_facility: Amedeo Avogadro University of Eastern Piedmont-Ospedale Maggiore della Carita

location_facility: Clinica Ematologica dell'Universita di Roma La Sapienza

location_facility: Instituto Regina Elena / Instituti Fisioterapici Ospitalieri

location_facility: AUSL Romagna - Ospedale Santa Maria dell Croci

location_facility: Azienda Ospedaliera Città della Salute e della Scienza di Torino - Ospedale Molinette

location_facility: Universitaetklinikum Halle - Martin Luther Universitaet - Universitaetsklinikum Halle (Saale)

location_facility: Azienda Ospedaliero - Universitaria Policlinico di Modena

location_facility: Radboud University Medical Center Nijmegen

location_facility: Azienda Ospedaliero Universitaria - Ospedali Riuniti

location_facility: Klinikum Der Phillips - Universität Marburg

location_facility: Universita Degli Studi Di Bari - Policlinico


type: Other Identifier

type: Registry Identifier

title: An Open-label Single-arm Multicentre Non-controlled Phase 3 a Trial Investigating Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Patients With Haemophilia B (FIX Activity Below or Equal to 2 Percent)

summary: This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of nonacog beta pegol (N9-GP) in previously untreated patients with Haemophilia B.

ReferenceCitation: Chan AK, Alamelu J, Barnes C, Chuansumrit A, Garly ML, Meldgaard RM, Young G. Nonacog beta pegol (N9-GP) in hemophilia B: First report on safety and efficacy in previously untreated and minimally treated patients. Res Pract Thromb Haemost. 2020 Jul 29;4(7):1101-1113. doi: 10.1002/rth2.12412. eCollection 2020 Oct.

lead_sponsor: Novo Nordisk A/S

location_country:
Italy
Israel
Argentina
Algeria
Australia
Japan
United Kingdom
Austria
United States
Spain
Thailand
Malaysia
Taiwan
Canada
France


summary: This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of turoctocog alfa pegol (N8-GP) in previously untreated patients (PUPs) with haemophilia A.

title: An Open-label Single-arm Multicentre Non-controlled Phase 3a Trial Investigating Safety and Efficacy of N8-GP in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Paediatric Patients With Severe Haemophilia A

enrollment_type: Anticipated

location_country:
Germany
Italy
Israel
Argentina
Algeria
Australia
Japan
Romania
Austria
United States
Spain
Thailand
Greece
Malaysia
Taiwan
Bulgaria
Canada
France


title: LOWER: Lomitapide Observational Worldwide Evaluation Registry

acronym: LOWER

summary: This global product exposure registry is a multicentre, long-term, prospective, observational cohort study (exposure registry), designed to evaluate the long term safety and effectiveness of lomitapide.

status: Recruiting

ReferenceCitation: Giammanco A, Cefalu AB, Noto D, Averna MR. Therapeutic Options for Homozygous Familial Hypercholesterolemia: The Role of Lomitapide. Curr Med Chem. 2020;27(23):3773-3783. doi: 10.2174/0929867326666190121120735.

publisher_dissemination: Bentham Science Publishers Ltd.

lead_sponsor: Amryt Pharma

location_country:
Italy
United Kingdom
Argentina
Netherlands
United States
Greece
Canada
France

location_facility: Azienda Ospedaliero-Universitaria San Luigi Gonzaga

location_facility: The University of Vermont Health Network

location_facility: Cardiology Specialists of the Carolinas PA

location_facility: Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone

location_facility: Centre Hospitalier Régional Universitaire de Lille

location_facility: ASST Grande Ospedale Metropolitano Niguarda

location_facility: St. Luke's University Health Network - Pennsylvania

location_facility: Complete Family Care Cholesterol Treatment Center

location_facility: Thomas Jefferson University Health System

location_facility: Università degli Studi di Roma La Sapienza

location_facility: CNR Regione Toscana - Fondazione Toscana Gabriele Monasterio

location_facility: Azienda Ospedaliera Universita di Padova

location_facility: Lehigh Valley Hospital And Health Network

location_facility: Centre Hospitalier Universitaire Strasbourg

location_facility: Azienda Ospedaliera Specialistica Dei Colli

location_facility: Azienda Ospedaliera Universitaria Federico II

location_facility: Azienda Ospedaliero Universi consorziale policlinico di Bari


title: A Randomized, Double-Blind, Phase 2 Study of Ruxolitinib or Placebo in Combination With Capecitabine in Subjects With Advanced or Metastatic HER2-Negative Breast Cancer

summary: This was a randomized, double-blind, placebo-controlled phase 2 clinical trial comparing the overall survival of women with advanced or metastatic HER2-negative breast cancer who received treatment with capecitabine in combination with ruxolitinib versus those who received treatment with capecitabine alone.

lead_sponsor: Incyte Corporation

location_country:
Italy
Portugal
United Kingdom
United States
Spain
France


lead_sponsor: Hoffmann-La Roche

summary: This Phase II, single-arm study is designed to evaluate the effect of atezolizumab treatment in participants with locally advanced or metastatic urothelial bladder cancer. Participants will be enrolled into 1 of 2 cohorts. Cohort 1 will consist of participants who are treatment-naïve and ineligible for cisplatin-containing chemotherapy. The results of Cohort 1 are reported separately (NCT02951767). Cohort 2 (reported here) will contain participants who have progressed during or following a prior platinum-based chemotherapy regimen. Participants in both cohorts will be given a 1200 milligrams (mg) intravenous (IV) dose of atezolizumab on Day 1 of 21-day cycles. Treatment of participants in Cohort 1 will continue until disease progression per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1) or unmanageable toxicity. Treatment of participants in Cohort 2 will continue until loss of clinical benefit or unmanageable toxicity.

ReferenceCitation: Holm JS, Funt SA, Borch A, Munk KK, Bjerregaard AM, Reading JL, Maher C, Regazzi A, Wong P, Al-Ahmadie H, Iyer G, Tamhane T, Bentzen AK, Herschend NO, De Wolf S, Snyder A, Merghoub T, Wolchok JD, Nielsen M, Rosenberg JE, Bajorin DF, Hadrup SR. Neoantigen-specific CD8 T cell responses in the peripheral blood following PD-L1 blockade might predict therapy outcome in metastatic urothelial carcinoma. Nat Commun. 2022 Apr 11;13(1):1935. doi: 10.1038/s41467-022-29342-0.

repository_institution: Technical University of Denmark, DTU - Technical University of Denmark, DTU Orbit

repository_normalized: Pure (Denmark)

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

ReferenceCitation: Shemesh CS, Chan P, Legrand FA, Shames DS, Das Thakur M, Shi J, Bailey L, Vadhavkar S, He X, Zhang W, Bruno R. Pan-cancer population pharmacokinetics and exposure-safety and -efficacy analyses of atezolizumab in patients with high tumor mutational burden. Pharmacol Res Perspect. 2020 Dec;8(6):e00685. doi: 10.1002/prp2.685.

publisher_dissemination: Wiley

ReferenceCitation: Teo MY, Seier K, Ostrovnaya I, Regazzi AM, Kania BE, Moran MM, Cipolla CK, Bluth MJ, Chaim J, Al-Ahmadie H, Snyder A, Carlo MI, Solit DB, Berger MF, Funt S, Wolchok JD, Iyer G, Bajorin DF, Callahan MK, Rosenberg JE. Alterations in DNA Damage Response and Repair Genes as Potential Marker of Clinical Benefit From PD-1/PD-L1 Blockade in Advanced Urothelial Cancers. J Clin Oncol. 2018 Jun 10;36(17):1685-1694. doi: 10.1200/JCO.2017.75.7740. Epub 2018 Feb 28.

publisher_dissemination: American Society of Clinical Oncology (ASCO)

ReferenceCitation: Perez-Gracia JL, Loriot Y, Rosenberg JE, Powles T, Necchi A, Hussain SA, Morales-Barrera R, Retz MM, Niegisch G, Duran I, Theodore C, Grande E, Shen X, Wang J, Nelson B, Derleth CL, van der Heijden MS. Atezolizumab in Platinum-treated Locally Advanced or Metastatic Urothelial Carcinoma: Outcomes by Prior Number of Regimens. Eur Urol. 2018 Mar;73(3):462-468. doi: 10.1016/j.eururo.2017.11.023. Epub 2017 Dec 20. Erratum In: Eur Urol. 2019 Mar;75(3):e82-e83.

repository_institution: University of Plymouth - PEARL

ReferenceCitation: Necchi A, Joseph RW, Loriot Y, Hoffman-Censits J, Perez-Gracia JL, Petrylak DP, Derleth CL, Tayama D, Zhu Q, Ding B, Kaiser C, Rosenberg JE. Atezolizumab in platinum-treated locally advanced or metastatic urothelial carcinoma: post-progression outcomes from the phase II IMvigor210 study. Ann Oncol. 2017 Dec 1;28(12):3044-3050. doi: 10.1093/annonc/mdx518.

license: publisher-specific-oa

license_normalized: publisher-specific

ReferenceCitation: Snyder A, Nathanson T, Funt SA, Ahuja A, Buros Novik J, Hellmann MD, Chang E, Aksoy BA, Al-Ahmadie H, Yusko E, Vignali M, Benzeno S, Boyd M, Moran M, Iyer G, Robins HS, Mardis ER, Merghoub T, Hammerbacher J, Rosenberg JE, Bajorin DF. Contribution of systemic and somatic factors to clinical response and resistance to PD-L1 blockade in urothelial cancer: An exploratory multi-omic analysis. PLoS Med. 2017 May 26;14(5):e1002309. doi: 10.1371/journal.pmed.1002309. eCollection 2017 May.

repositories:
PubMed Central
bioRxiv
pdfs.semanticscholar.org

ReferenceCitation: Balar AV, Galsky MD, Rosenberg JE, Powles T, Petrylak DP, Bellmunt J, Loriot Y, Necchi A, Hoffman-Censits J, Perez-Gracia JL, Dawson NA, van der Heijden MS, Dreicer R, Srinivas S, Retz MM, Joseph RW, Drakaki A, Vaishampayan UN, Sridhar SS, Quinn DI, Duran I, Shaffer DR, Eigl BJ, Grivas PD, Yu EY, Li S, Kadel EE 3rd, Boyd Z, Bourgon R, Hegde PS, Mariathasan S, Thastrom A, Abidoye OO, Fine GD, Bajorin DF; IMvigor210 Study Group. Atezolizumab as first-line treatment in cisplatin-ineligible patients with locally advanced and metastatic urothelial carcinoma: a single-arm, multicentre, phase 2 trial. Lancet. 2017 Jan 7;389(10064):67-76. doi: 10.1016/S0140-6736(16)32455-2. Epub 2016 Dec 8. Erratum In: Lancet. 2017 Aug 26;390(10097):848.

ReferenceCitation: Rosenberg JE, Hoffman-Censits J, Powles T, van der Heijden MS, Balar AV, Necchi A, Dawson N, O'Donnell PH, Balmanoukian A, Loriot Y, Srinivas S, Retz MM, Grivas P, Joseph RW, Galsky MD, Fleming MT, Petrylak DP, Perez-Gracia JL, Burris HA, Castellano D, Canil C, Bellmunt J, Bajorin D, Nickles D, Bourgon R, Frampton GM, Cui N, Mariathasan S, Abidoye O, Fine GD, Dreicer R. Atezolizumab in patients with locally advanced and metastatic urothelial carcinoma who have progressed following treatment with platinum-based chemotherapy: a single-arm, multicentre, phase 2 trial. Lancet. 2016 May 7;387(10031):1909-20. doi: 10.1016/S0140-6736(16)00561-4. Epub 2016 Mar 4.

location_facility: Kaiser Permanente; Oncology Clinical Trials

location_facility: Institut Catala d Oncologia Hospital Duran i Reynals

location_facility: University Hospital Birmingham The Cancer Centre, Queen Elizabeth Hospital

location_facility: Charité - Universitätsmedizin Berlin; CC 8: Chirurgische Medizin; Klinik für Urologie

location_facility: Klinikum rechts der Isar der TU München; Urologische Klinik und Poliklinik

location_facility: Beth Israel Deaconess Medical Center, Harvard Medical School; Department of Medicine

location_facility: Universitätsklinikum Hamburg-Eppendorf Onkologisches Zentrum Medizinische Klinik II

location_facility: The Netherlands Cancer Institute - Antoni Van Leeuwenhoekziekenhuis

location_facility: Willamette Valley Cancer Ctr - 520 Country Club

location_facility: Universitätsklinikum Freiburg; Chirurgische Klinik; Abteilung Urologie

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

location_facility: Virginia Oncology Associates - Lake Wright Cancer Center

location_facility: University of Michigan Comprehensive Cancer Center

location_facility: Universitätsklinikum Düsseldorf; Urologische Klinik

location_facility: University of Chicago; Hematology/Oncology

location_facility: Azienda USL8 Arezzo-Presidio Ospedaliero 1 San Donato;U.O.C. Oncologia

location_facility: University Health Network; Princess Margaret Hospital; Medical Oncology Dept

location_facility: University of Texas Health Science Center at San Antonio

location_facility: Royal Marsden Hospital; Dept of Medical Oncology

location_facility: Georgetown University Medical Center Lombardi Cancer Center

location_facility: Urology Cancer Center & GU Research Network

location_facility: McGill University; Sir Mortimer B Davis Jewish General Hospital; Oncology

location_facility: Institut Gustave Roussy; Oncologie Medicale

location_facility: Dana Farber Cancer Inst. ; Dept. of Medical Oncology

location_facility: The Angeles Clinic and Research Institute - W LA Office

location_facility: Kimmel Cancer Center Thomas Jefferson University

location_facility: The Ottawa Hospital Cancer Centre; Oncology

location_facility: Hospital General Universitario Gregorio Marañon; Servicio de Oncologia

location_facility: Hospital Ramon y Cajal; Servicio de Oncologia

location_facility: Hospital Universitario 12 de Octubre; Servicio de Oncologia

location_facility: The Clatterbridge Cancer Centre NHS Foundation Trust

location_facility: Clinica Universitaria de Navarra; Servicio de Oncologia

location_facility: Hospital Universitario Virgen del Rocio; Servicio de Oncologia

location_facility: Sarah Cannon Cancer Center - Tennessee Oncology, Pllc

location_facility: Mount Sinai School of Medicine - Tisch Cancer Institute

location_facility: Indiana University Health; Goshen Center for Cancer Care

location_facility: Irccs Istituto Nazionale Dei Tumori (Int);S.C. Medicina Oncologica 2

title: A Phase II, Multicenter, Single-Arm Study of Atezolizumab in Patients With Locally Advanced or Metastatic Urothelial Bladder Cancer


acronym: CheckMate 141: CHECKpoint pathway and nivoluMAb clinical Trial Evaluation 141

lead_sponsor: Bristol-Myers Squibb

summary: The purpose of this study is to find out whether Nivolumab will significantly improve overall survival as compared to therapy of investigator's choice in patients with recurrent or metastatic head and neck carcinoma.

ReferenceCitation: Yen CJ, Kiyota N, Hanai N, Takahashi S, Yokota T, Iwae S, Shimizu Y, Hong RL, Goto M, Kang JH, Li WSK, Ferris RL, Gillison M, Endo T, Jayaprakash V, Tahara M. Two-year follow-up of a randomized phase III clinical trial of nivolumab vs. the investigator's choice of therapy in the Asian population for recurrent or metastatic squamous cell carcinoma of the head and neck (CheckMate 141). Head Neck. 2020 Oct;42(10):2852-2862. doi: 10.1002/hed.26331. Epub 2020 Jun 24.

ReferenceCitation: Saba NF, Blumenschein G Jr, Guigay J, Licitra L, Fayette J, Harrington KJ, Kiyota N, Gillison ML, Ferris RL, Jayaprakash V, Li L, Brossart P. Nivolumab versus investigator's choice in patients with recurrent or metastatic squamous cell carcinoma of the head and neck: Efficacy and safety in CheckMate 141 by age. Oral Oncol. 2019 Sep;96:7-14. doi: 10.1016/j.oraloncology.2019.06.017. Epub 2019 Jul 3.

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

repositories_concat: PubMed Central;air.unimi.it

ReferenceCitation: Cocks K, Contente M, Simpson S, DeRosa M, Taylor FC, Shaw JW. A Q-TWiST Analysis Comparing Nivolumab and Therapy of Investigator's Choice in Patients with Recurrent/Metastatic Platinum-Refractory Squamous Cell Carcinoma of the Head and Neck. Pharmacoeconomics. 2019 Aug;37(8):1041-1047. doi: 10.1007/s40273-019-00798-1.

ReferenceCitation: Pai SI, Faivre S, Licitra L, Machiels JP, Vermorken JB, Bruzzi P, Gruenwald V, Giglio RE, Leemans CR, Seiwert TY, Soulieres D. Comparative analysis of the phase III clinical trials of anti-PD1 monotherapy in head and neck squamous cell carcinoma patients (CheckMate 141 and KEYNOTE 040). J Immunother Cancer. 2019 Apr 3;7(1):96. doi: 10.1186/s40425-019-0578-0.

repository_institution: University of Antwerp - Institutional Repository University of Antwerp

repository_normalized: irua

repositories:
PubMed Central
air.unimi.it
irua

repositories_concat: PubMed Central;air.unimi.it;irua

publisher_dissemination: British Medical Journal

ReferenceCitation: Ferris RL, Blumenschein G Jr, Fayette J, Guigay J, Colevas AD, Licitra L, Harrington KJ, Kasper S, Vokes EE, Even C, Worden F, Saba NF, Docampo LCI, Haddad R, Rordorf T, Kiyota N, Tahara M, Lynch M, Jayaprakash V, Li L, Gillison ML. Nivolumab vs investigator's choice in recurrent or metastatic squamous cell carcinoma of the head and neck: 2-year long-term survival update of CheckMate 141 with analyses by tumor PD-L1 expression. Oral Oncol. 2018 Jun;81:45-51. doi: 10.1016/j.oraloncology.2018.04.008. Epub 2018 Apr 17.

ReferenceCitation: Kiyota N, Hasegawa Y, Takahashi S, Yokota T, Yen CJ, Iwae S, Shimizu Y, Hong RL, Goto M, Kang JH, Sum Kenneth Li W, Ferris RL, Gillison M, Namba Y, Monga M, Lynch M, Tahara M. A randomized, open-label, Phase III clinical trial of nivolumab vs. therapy of investigator's choice in recurrent squamous cell carcinoma of the head and neck: A subanalysis of Asian patients versus the global population in checkmate 141. Oral Oncol. 2017 Oct;73:138-146. doi: 10.1016/j.oraloncology.2017.07.023. Epub 2017 Sep 1.

ReferenceCitation: Harrington KJ, Ferris RL, Blumenschein G Jr, Colevas AD, Fayette J, Licitra L, Kasper S, Even C, Vokes EE, Worden F, Saba NF, Kiyota N, Haddad R, Tahara M, Grunwald V, Shaw JW, Monga M, Lynch M, Taylor F, DeRosa M, Morrissey L, Cocks K, Gillison ML, Guigay J. Nivolumab versus standard, single-agent therapy of investigator's choice in recurrent or metastatic squamous cell carcinoma of the head and neck (CheckMate 141): health-related quality-of-life results from a randomised, phase 3 trial. Lancet Oncol. 2017 Aug;18(8):1104-1115. doi: 10.1016/S1470-2045(17)30421-7. Epub 2017 Jun 23.

ReferenceCitation: Ferris RL, Blumenschein G Jr, Fayette J, Guigay J, Colevas AD, Licitra L, Harrington K, Kasper S, Vokes EE, Even C, Worden F, Saba NF, Iglesias Docampo LC, Haddad R, Rordorf T, Kiyota N, Tahara M, Monga M, Lynch M, Geese WJ, Kopit J, Shaw JW, Gillison ML. Nivolumab for Recurrent Squamous-Cell Carcinoma of the Head and Neck. N Engl J Med. 2016 Nov 10;375(19):1856-1867. doi: 10.1056/NEJMoa1602252. Epub 2016 Oct 8.

sponsor_collaborators:
Bristol-Myers Squibb
Ono Pharmaceutical Co. Ltd

location_facility: Ist.Scient. Romagnolo Per Lo Studio E Cura Tumori

title: An Open Label, Randomized Phase 3 Clinical Trial of Nivolumab vs Therapy of Investigator's Choice in Recurrent or Metastatic Platinum-refractory Squamous Cell Carcinoma of the Head and Neck (SCCHN)

location_country:
Germany
Italy
United Kingdom
Argentina
Hong Kong
Japan
Brazil
Netherlands
Switzerland
United States
Korea, Republic of
Spain
Taiwan
Canada
France


title: A Retrospective, Non-interventional, Epidemiologic Study of the Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)

summary: The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP.

time_perspective: Retrospective

location_country:
United Kingdom
Australia
Netherlands
United States
Turkey
Canada
Russian Federation
France


lead_sponsor: Allergan

summary: This was a multicenter, randomized, double-blind, parallel-group, multiple-dose study to evaluate the efficacy and safety of BOTOX in adolescents with urinary incontinence due to overactive bladder (OAB) with inadequate management with anticholinergic therapy. Participants were randomized in a 1:1:1 ratio to receive a single Tx of 25 U, 50 U, or 100 U BOTOX (not to exceed 6 U/kg) on Day 1, were seen after each treatment at Weeks 2, 6, and 12 post-treatment, and thereafter at alternating telephone and clinic visits every 6 weeks until they qualified for further retreatment/exited the study. Participants could receive multiple treatments dependent upon the number and timing of patient requests/qualification for retreatment. At each retreatment the investigator could keep the dose the same or increase it one dose level in a blinded fashion. Participants exited the study once 96 weeks have elapsed since entry on Day 1 and at least 12 weeks follow-up since their last study treatment had occurred.

location_facility: Medical Concierge Centrum Medyczne /ID# 235200

location_facility: Universitair Ziekenhuis Antwerpen /ID# 237997

location_facility: Hôpital de la Mère et de l'Enfant /ID# 235227

location_facility: Norfolk and Norwich University Hospitals NHS Foundation Trust /ID# 234819

location_facility: Universitair Ziekenhuis Leuven /ID# 237218

location_facility: The Children's Hospital at Westmead /ID# 234337

location_facility: Alder Hey Children's NHS Foundation Trust /ID# 237279

location_facility: Specjalistyczny Gabinet Lekarski /ID# 235257

location_facility: Royal Berkshire NHS Foundation Trust /ID# 236915

location_facility: Universitaetsklinikum Schleswig-Holstein Campus Luebeck /ID# 234288

location_facility: Sheffield Children's NHS Foundation Trust /ID# 237854

location_facility: Orlando Health-Arnold Palmer Hospital for Children Pediatric Urology /ID# 235283

location_facility: Yale New Haven Hospital - Yale School of Medicine /ID# 238222

location_facility: Duplicate_CHU Bordeaux-Hopital Pellegrin /ID# 237392

location_facility: London Health Sciences Center /ID# 234304

location_facility: Urologische Gemeinschaftspraxis /ID# 234978

location_facility: Hôpitaux Pédiatriques de Nice CHU-LENVAL /ID# 235278

location_facility: Associated Urologist of North Carolina /ID# 235437

location_facility: Children's Hospital Wisconsin - Milwaukee Campus /ID# 237544

location_facility: Children's Hospital Colorado /ID# 237621

location_facility: Arkansas Children's Hospital /ID# 237787

location_facility: Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu /ID# 238166

location_facility: Evangelisches Krankenhaus Bielefeld /ID# 235234

location_facility: Manchester University NHS Foundation Trust /ID# 234380

location_facility: AOU Universita degli Studi della Campania Luigi Vanvitelli /ID# 237308

location_facility: Radboud Universitair Medisch Centrum /ID# 237043

location_facility: Maastricht Universitair Medisch Centrum /ID# 237678

location_facility: NHS Greater Glasgow and Clyde /ID# 237430

title: BOTOX® in the Treatment of Urinary Incontinence Due to Overactive Bladder in Patients 12 to 17 Years of Age

location_country:
Germany
Italy
Poland
Norway
United Kingdom
Australia
Netherlands
Belgium
United States
South Africa
Czechia
Canada
France


lead_sponsor: Grünenthal GmbH

summary: The purpose of the study was to evaluate the efficacy of tapentadol oral solution, based on the total amount of supplemental opioid analgesic used over 12 hours or 24 hours after initiation of investigational medicinal product (IMP) in children and adolescents who had undergone surgery that would produce moderate to severe pain during opioid treatment.

ReferenceCitation: Schwartz GJ, Feld LG, Langford DJ. A simple estimate of glomerular filtration rate in full-term infants during the first year of life. J Pediatr. 1984 Jun;104(6):849-54. doi: 10.1016/s0022-3476(84)80479-5.

sponsor_collaborators:
Grünenthal GmbH
Depomed

title: An Evaluation of the Efficacy and Safety of Tapentadol Oral Solution in the Treatment of Post-operative Acute Pain Requiring Opioid Treatment in Pediatric Subjects Aged From Birth to Less Than 18 Years Old

ipd_sharing_description: Information available on the Grünenthal Web Site

location_country:
Germany
Poland
Hungary
United Kingdom
United States
Spain
Croatia
Czechia
Bulgaria
France


lead_sponsor: Actelion

summary: Long-term study to evaluate if macitentan is safe, tolerable and efficient enough to be used for treatment of inoperable chronic thromboembolic pulmonary hypertension (CTEPH)

title: MERIT-2 : Long Term, Multicenter, Single-arm, Open-label Extension Study of the MERIT-1 Study, to Assess the Safety, Tolerabilty and Efficacy of Macitentan in Subjects With Inoperable Chronic Thromboembolic Pulmonary Hypertension (CTEPH)

location_country:
Germany
Lithuania
Poland
Hungary
United Kingdom
Switzerland
Mexico
Belgium
China
Thailand
Turkey
Czechia
Ukraine
Russian Federation
France


acronym: Not Applicable

lead_sponsor: LEO Pharma

summary: An international, multi-centre, prospective, non-controlled, open, single-group, 8-week trial in adolescent subjects (aged 12 to 16 years, 11 months) with scalp and body psoriasis.

location_facility: Spitalul Clinic Judetean de Urgenta Cluj-Napoca

location_facility: Clinical Research Center, Morsani Center for Advanced Healthcare

location_facility: Policlinica de Diagnostic Rapid S.A. Clinic

location_facility: Dermatology and Laser Center of Charleston, PA

location_facility: Spitalul Clinic Municipal de Urgenta Timisoara

location_facility: Cabinet Medical Individual Tatu G. Alin Laurentiu

location_facility: Specjalistyczny Ofrodek Leczniczo Clinic

title: Effect of Calcipotriol Plus Betamethasone Dipropionate Gel on the HPA Axis and Calcium Metabolism in Adolescent Subjects (Aged 12 to 16 Years, 11 Months) With Scalp and Body Psoriasis

location_country:
Germany
Poland
United Kingdom
Romania
United States
Canada
France


title: A Phase 1B, Multi-Center, Open-Label Study of Novel Combinations of CC-122, CC-223, CC-292, and Rituximab in Diffuse Large B-Cell Lymphoma and Follicular Lymphoma

summary: First study, at multiple clinical centers, exploring the effects of different combinations of compounds (CC-122, CC-223 ,CC-292 and rituximab) to treat Diffuse Large B Cell Lymphoma (DLBCL) and Follicular Lymphoma

lead_sponsor: Celgene

location_country:
United States
Italy
Canada
France

location_facility:
Local Institution - 102
Mayo Clinic
Local Institution - 200
Local Institution - 402
Local Institution - 400
MD Anderson Cancer Center
Stanford Cancer Center
Local Institution - 100
Northwestern University
Yale Cancer Center
Local Institution - 101
Local Institution - 007
Local Institution - 005
Local Institution - 001
Local Institution - 202
Local Institution - 201


title: A Multicenter, Long-term Extension Study to Further Evaluate the Safety and Tolerability of Telotristat Etiprate (LX1606)

acronym: TELEPATH

summary: The primary objective of this study is to evaluate the long-term safety and tolerability of orally administered telotristat etiprate.

ReferenceCitation: Horsch D, Anthony L, Gross DJ, Valle JW, Welin S, Benavent M, Caplin M, Pavel M, Bergsland E, Oberg K, Kassler-Taub KB, Binder P, Banks P, Lapuerta P, Kulke MH. Long-Term Treatment with Telotristat Ethyl in Patients with Carcinoid Syndrome Symptoms: Results from the TELEPATH Study. Neuroendocrinology. 2022;112(3):298-310. doi: 10.1159/000516958. Epub 2021 May 3.

repository_institution: Spanish National Research Council (CSIC) - DIGITAL.CSIC

repository_normalized: DIGITAL.CSIC (Spain)

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

publisher_dissemination: S. Karger AG

lead_sponsor: Lexicon Pharmaceuticals

location_country:
Germany
Italy
Israel
United Kingdom
Australia
Sweden
Netherlands
Belgium
United States
Spain
Canada
France

location_facility:
Lexicon Investigational Site
Lexicon Investgational Site


title: A Three Arm Randomized, Open-label Phase II Study of Radium-223 Dichloride 50 kBq/kg (55 kBq/kg After Implementation of NIST Update) Versus 80 kBq/kg (88 kBq/kg After Implementation of NIST Update), and Versus 50 kBq/kg (55 kBq/kg After Implementation of NIST Update) in an Extended Dosing Schedule in Subjects With Castration-resistant Prostate Cancer Metastatic to the Bone

summary: This study will assess different doses and regimens of radium-223 dichloride on the incidence of symptomatic skeletal events. Eligible subjects must have castration resistant prostate cancer with 2 or more skeletal metastases documented within 8 weeks of randomization. Subjects will be randomized to one of 3 treatment arms in a 1:1:1 fashion: a standard regimen of radium-223 dichloride of 50 kBq/kg (55 kBq/kg after implementation of NIST update) injections every month for 6 months, a high dose regimen of 80 kBq/kg (88 kBq/kg after implementation of NIST update)injections every month for 6 months or an extended duration regimen of 50 kBq/kg (55 kBq/kg after implementation of NIST update) injections every month for 12 months. Following the treatment phase, subjects will be followed up every 12 weeks for a minimum of 2 years, at which point they will enter a long term follow-up period during which they are seen every 6 months for up to 7 years after the last dose of radium dichloride. Symptomatic skeletal event and safety endpoints will be assessed at each clinic visit. Pain and analgesic use data will be collected every 4 weeks through Week 48. Additionally, radiological assessments including MRI/CT of the abdomen and pelvis and chest CT, as well as technetium-99 bone scans will be performed at Weeks 8, 16, and 24 and continue every 12 weeks thereafter until disease progression is documented in either the bone or in soft tissue. Radiological imaging will be evaluated by blinded central review.

ReferenceCitation: Sternberg CN, Saad F, Graff JN, Peer A, Vaishampayan UN, Leung E, Rosenbaum E, Gurney H, Epstein RJ, Davis ID, Wu B, Trandafir L, Wagner VJ, Hussain M. A randomised phase II trial of three dosing regimens of radium-223 in patients with bone metastatic castration-resistant prostate cancer. Ann Oncol. 2020 Feb;31(2):257-265. doi: 10.1016/j.annonc.2019.10.025. Epub 2019 Dec 23.

repositories_institution: Monash University - Monash University Research Portal

lead_sponsor: Bayer

location_country:
Germany
Italy
Israel
United Kingdom
Australia
Brazil
Sweden
United States
Korea, Republic of
Spain
Chile
Taiwan
Czechia
Canada
France

location_facility: Irmandade Santa Casa de Misericordia de Porto Alegre

location_facility: IBCC - Instituto Brasileiro de Controle do Cancer

location_facility: Instituto do Câncer do Estado de São Paulo