title: Abiliti™ Treatment in Obese Subjects: A Randomized Post-Market Surveillance Multicenter Study

summary: The main objective of this post-market surveillance study is to compare the subject's weight loss and quality of life, and device safety performance of the abiliti System and the adjustable gastric banding therapies in obese to morbidly obese subjects.

ReferenceCitation: Morales-Conde S, Alarcón Del Agua I, Busetto L, Favretti F, Anselmino M, Rovera GM, Socas-Macias M, Barranco-Moreno A, Province-Azalde R, Torres AJ. Implanted Closed-Loop Gastric Electrical Stimulation (CLGES) System with Sensor-Based Feedback Safely Limits Weight Regain at 24 Months. Obes Surg. 2018 Jun;28(6):1766-1774. doi: 10.1007/s11695-017-3093-8.

ReferenceCitation: Busetto L, Torres AJ, Morales-Conde S, Alarcón Del Agua I, Moretto C, Fierabracci P, Rovera G, Segato G, Rubio MA, Favretti F. Impact of the feedback provided by a gastric electrical stimulation system on eating behavior and physical activity levels. Obesity (Silver Spring). 2017 Mar;25(3):514-521. doi: 10.1002/oby.21760. Epub 2017 Feb 6.

ReferenceCitation: Alarcón Del Agua I, Socas-Macias M, Busetto L, Torres-Garcia A, Barranco-Moreno A, Garcia de Luna PP, Morales-Conde S. Post-implant Analysis of Epidemiologic and Eating Behavior Data Related to Weight Loss Effectiveness in Obese Patients Treated with Gastric Electrical Stimulation. Obes Surg. 2017 Jun;27(6):1573-1580. doi: 10.1007/s11695-016-2495-3.

lead_sponsor: IntraPace, Inc

location_country:
Spain
Italy
United Kingdom
Switzerland
France
Germany

location_facility: Polyclinique de Rillieux, Clinique Lyon-Nord

location_facility: Complutense University of Madrid Hospital Clinico "San Carlos"


type: EudraCT Number

title: Safety and Efficacy of Brimonidine Posterior Segment Drug Delivery System in Patients With Geographic Atrophy Secondary to Age-related Macular Degeneration

acronym: BEACON

summary: This study will assess the safety and efficacy of the brimonidine intravitreal implant in participants with geographic atrophy due to age-related macular degeneration.

study_start_date_type: Actual

status: Terminated

study_completion_date_type: Actual

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Actual

ReferenceType: derived

ReferenceCitation: Miere A, Capuano V, Kessler A, Zambrowski O, Jung C, Colantuono D, Pallone C, Semoun O, Petit E, Souied E. Deep learning-based classification of retinal atrophy using fundus autofluorescence imaging. Comput Biol Med. 2021 Mar;130:104198. doi: 10.1016/j.compbiomed.2020.104198. Epub 2020 Dec 28.

ReferenceCitation: Kuppermann BD, Patel SS, Boyer DS, Augustin AJ, Freeman WR, Kerr KJ, Guo Q, Schneider S, López FJ; Brimo DDS Gen 1 Study Group. PHASE 2 STUDY OF THE SAFETY AND EFFICACY OF BRIMONIDINE DRUG DELIVERY SYSTEM (BRIMO DDS) GENERATION 1 IN PATIENTS WITH GEOGRAPHIC ATROPHY SECONDARY TO AGE-RELATED MACULAR DEGENERATION. Retina. 2021 Jan 1;41(1):144-155. doi: 10.1097/IAE.0000000000002789.

lead_sponsor: Allergan

location_country:
United Kingdom
Italy
Australia
United States
France
Germany

location_facility: Northern California Retina Vitreous Associates

location_facility: "National Ophthalmic Research Institute Retina Consultants of Southwest Florida"

location_facility: Lions Eye Institute, University of Western Australia

location_facility: Charlotte Eye Ear Nose & Throat Associates, PA

location_facility: Casey Eye Institute, Oregon Health and Science University

location_facility: Retina Consultants of Southern Colorado, PC

location_facility: Retinal Consultants of Arizona, Ltd., Retinal Research Institute

location_facility: Universita degli Studi di Padova, Dipartimento di Neuroscienze

location_facility: Frimley Park Hospital, Eye clinical trials Unit, Department of Opthalmology

location_facility: Mid Atlantic Retina, Wills Eye Retina Surgeons

location_facility: "Centre Hospitalier Intercommunal de Creteil "

location_facility: University of California, San Diego, Jacobs Retina Center, Shiley Eye Center

location_facility: Clinical Research Unit, Level2, Bristol Eye Hospital, Lower Maudlin Street

location_facility: Università di Cagliari (presidio San Giovanni di Dio)

location_facility: "Dir IV Clinica Oculistica di Milano, Ospedale Generale ""Luigi Sacco"" "

location_facility: Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico

location_facility: Royal Hallamshire Hospital, Eye Department

location_facility: "Universitat Bonn, Abteilung fur Augenheilkunde "

intervention_type: Other

lead_sponsor_type: industriel

status_simplified: Unknown


title: Performance of Acoustic Radiation Force Impulse-Imaging in Comparison to Transient Elastography for the Non-invasive Staging of Liver Fibrosis in Patients With Chronic Hepatitis C

summary: This study will evaluate the effectiveness of Acoustic Radiation Force Impulse (ARFI)- Imaging compared to Transient Elastography (FibroScan) in differentiating liver fibrosis in patients with chronic hepatitis C. Patients who are scheduled to have a liver biopsy will also undergo ARFI and FibroScan testing. The liver biopsy will be used as the reference method. The target sample size is 433.

status: Completed

primary_purpose: Diagnostic

lead_sponsor: Johann Wolfgang Goethe University Hospital

location_country: Germany France Netherlands

location_facility: Klinikum der Ludwig-Maximilians-Universität-Campus Großhadern

intervention_type: Device

lead_sponsor_type: academique

status_simplified: Completed


title: A Prospective, Double Blind Randomised Controlled Trial to Evaluate the Immunological Benefits and Clinical Effects of an Elimination Diet Using an Amino Acid Based Formula (AAF)

summary: The purpose of this study is to evaluate whether a new version of an amino acid based formula improves tolerance to milk in cow's milk allergic infants/young children.

primary_purpose: Supportive Care

ReferenceCitation: Amari S, Shahrook S, Namba F, Ota E, Mori R. Branched-chain amino acid supplementation for improving growth and development in term and preterm neonates. Cochrane Database Syst Rev. 2020 Oct 2;10:CD012273. doi: 10.1002/14651858.CD012273.pub2.

ipd_sharing: No

lead_sponsor: Nutricia Research

location_country:
Germany
France
United Kingdom

location_facility: David Hyde Allergy Clinic - St. Mary's hospital

location_facility: Evelina Children's Hospital - St Thomas' Hospital


intervention_type: Drug

title: An International, Randomised, Double Blinded, Multi-centre, Active- and Placebo-controlled Dose Response Trial to Evaluate the Efficacy and Safety of SABER-Bupivacaine for Postoperative Pain Control in Patients Undergoing Primary, Elective, Open, Abdominal Hysterectomy

lead_sponsor: Durect

source: euctr

location_country:
United Kingdom
Latvia
Sweden
Hungary
France
Germany

summary: The objective is to identify the optimal dose of SABER-Bupivacaine for postoperative pain control in patients undergoing hysterectomy on the basis of pharmacokinetics, efficacy and safety evaluations. The study duration consists of a screening period up to 14 days and a treatment period 14 days with a long term follow up visit at 6 months. The study will provide further data on the efficacy and safety of the product.


title: A Substudy of the IBCSG 24-02/ SOFT Trial to Investigate Estrogen Suppression for Patients Participating in Arms B and C of the IBCSG 24-02/ SOFT Trial

acronym: SOFT-EST

summary:
RATIONALE: Studying samples of blood from patients with breast cancer in the laboratory may help doctors learn how well triptorelin given together with tamoxifen citrate or exemestane works in lowering estrogen levels.

PURPOSE: This clinical trial is studying estrogen levels in premenopausal women who have undergone surgery for breast cancer and are receiving triptorelin and tamoxifen citrate or exemestane on clinical trial IBCSG-2402.

lead_sponsor: ETOP IBCSG Partners Foundation

location_country: Portugal Spain Italy Switzerland Hungary Peru France Sweden

location_facility: INEN (Instituto de Enfermedades Neoplasicas)

location_facility: Hospital Clinic i Provincial de Barcelona

location_facility: Sahlgrenska University Hospital Gothenburg

location_facility: Clinica Oncologica, Policlinico Univeritario

location_facility: Hospital Clinico Universitario de Valencia


ipd_sharing: Yes

primary_purpose: Other

intervention_type: Procedure

title: AN OPEN-LABEL STUDY OF THE SAFETY AND EFFICACY OF REFACTO AF IN PREVIOUSLY UNTREATED PATIENTS IN USUAL CARE SETTINGS

lead_sponsor: Pfizer

type: Other Identifier

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

location_country: Spain Italy Netherlands Ukraine France Turkey Germany

location_facility: Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik

location_facility: Istanbul Universitesi Cerrahpasa Tip Fakultesi

location_facility: Derzhavna ustanova "Instytut patolohii krovi ta transfuziinoi medytsyny Natsionalnoi akademii

location_facility: Agenzia per L'emofilia e Centro Regionale Riferimento Coagulopatie Congenite

summary: Study to evaluate the safety and effectiveness of ReFacto AF for the treatment of severe hemophilia A in patients who have not yet received treatment for their hemophilia. Study subjects will be males less than 6 years old who have not taken any clotting factor or other blood products before the study. The safety and effectiveness of ReFacto AF will be determined in this study by tests and procedures done at the doctor's office.


title: Phase II Efficacy and Safety Study of Dasatinib in Patients With Chronic and Accelerated Phase Chronic Myeloid Leukemia Relapsing After Allogeneic Blood or Bone Marrow Transplantation

acronym: Dasatinib

summary:
This is a phase II efficacy (indicates the capacity for beneficial change or therapeutic effect) and safety study of Dasatinib in patients with relapsed Chronic Myeloid Leukemia (CML) following a Stem Cell Transplant (SCT) and who are not benefiting from other treatment, such as imatinib therapy.

A relapse is when an illness that has seemed to be getting better, or to have been cured, comes back or gets worse again.

A total of 50 patients ≥18 years of age will be registered on the trial.

design_allocation: Non-Randomized

enrollment_type: Anticipated

lead_sponsor: European Society for Blood and Marrow Transplantation

location_country:
Germany
France
Switzerland
United Kingdom

location_facility:
Hammersmith Hospital
University Hospital
Stiftung Deutsche Klinik für Diagnostik
Hopitaux Universitaires de Geneve
Uniklinik Leipzig
E Herriot Hospital


title: A Randomized, Open-label, Controlled, Multi-center Phase II Study to Evaluate the Efficacy and Safety of Adecatumumab Alone or Sequentially to FOLFOX Relative to FOLFOX After R0 Resection of Colorectal Liver Metastases

summary: The purpose of this study is to determine the effect of adecatumumab alone or following FOLFOX in patients with R0 resected liver metastases from CRC (colorectal carcinoma) and to compare the effect to FOLFOX alone.

lead_sponsor: Amgen Research (Munich) GmbH

location_country: Germany France

location_facility: Klinikum der Johannes-Gutenberg Universität

location_facility: Universitätsklinikum der LMU Grosshadern

location_facility: Klinikum am Bruderwald, Sozialstiftung Bamberg

location_facility: Klinikum Mannheim GmbH Universitätsklinikum


intervention_type: Biological

title: A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency (IgPro20 EU Extension Study)

lead_sponsor: CSL Behring

ReferenceType: result

ReferenceCitation: Jolles S, Borte M, Nelson RP Jr, Rojavin M, Bexon M, Lawo JP, Wasserman RL. Long-term efficacy, safety, and tolerability of Hizentra® for treatment of primary immunodeficiency disease. Clin Immunol. 2014 Feb;150(2):161-9. doi: 10.1016/j.clim.2013.10.008. Epub 2013 Oct 26.

location_country:
Poland
Spain
United Kingdom
Switzerland
Sweden
France
Romania
Germany

summary: This study is a continuation of the study ZLB06_001CR with the objective of assessing efficacy, tolerability, safety of IgPro, as well as long-term health-related quality of life in patients with PID.


title: A Prospective, Non-comparative Study to Evaluate the Performance of the DePuy P.F.C. Sigma Rotating Platform TC3 Knee Prosthesis in Revision Knee Arthroplasty.

summary: The purpose of this study is to evaluate the long-term clinical and functional performance of the P.F.C. Sigma Rotating Platform TC3 knee system in revision knee surgery and to determine whether the implant design results in a longer-lasting knee replacement.

lead_sponsor: DePuy International

location_facility: Robert Jones & Agnes Hunt Orthopaedic Hospital


title: An Open-Label, Multicenter, Multinational Extension Study Of The Long-Term Safety And Pharmacokinetics Of Repeated Biweekly Infusions Of Avalglucosidase Alfa In Patients With Pompe Disease

acronym: NEO-EXT

summary:
Primary Objective:

Long-term safety and pharmacokinetics (PK) of avalglucosidase alfa

Secondary Objective:

Long-term effect of avalglucosidase alfa on pharmacodynamic and exploratory efficacy variables

status: Active, not recruiting

study_completion_date_type: Anticipated

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

lead_sponsor: Genzyme, a Sanofi Company

location_country:
Denmark
United Kingdom
Netherlands
United States
France
Belgium
Germany

location_facility:
Investigational Site Number 840011
Investigational Site Number 250003
Investigational Site Number 056001
Investigational Site Number 840002
Investigational Site Number 840001
Investigational Site Number 250002
Investigational Site Number 208001
Investigational Site Number 276002
Investigational Site Number 840010
Investigational Site Number 840006
Investigational Site Number 826003
Investigational Site Number 840003
Investigational Site Number 276003
Investigational Site Number 276001
Investigational Site Number 840008
Investigational Site Number 528001
Investigational Site Number 840009

status_simplified: Ongoing


title: HIP Fracture Accelerated Surgical TreaTment And Care tracK (HIP ATTACK) Trial

acronym: HIP ATTACK

summary: HIP ATTACK is an international randomized controlled trial of 3000 patients with a hip fracture that requires a surgical intervention. This trial will determine the effect of accelerated medical clearance and accelerated surgery compared to standard care on the 90-day risk of mortality and major perioperative complication (i.e., a composite of mortality, nonfatal myocardial infarction, nonfatal pulmonary embolism, nonfatal pneumonia, nonfatal sepsis, nonfatal stroke, and nonfatal life-threatening and major bleeding).

ReferenceCitation: HIP ATTACK Investigators. Accelerated surgery versus standard care in hip fracture (HIP ATTACK): an international, randomised, controlled trial. Lancet. 2020 Feb 29;395(10225):698-708. doi: 10.1016/S0140-6736(20)30058-1. Epub 2020 Feb 9. Erratum in: Lancet. 2021 Nov 27;398(10315):1964.

ReferenceCitation: Borges FK, Devereaux PJ, Cuerden M, Bhandari M, Guerra-Farfán E, Patel A, Sigamani A, Umer M, Neary J, Tiboni M, Tandon V, Ramokgopa MT, Sancheti P, John B, Lawendy A, Balaguer-Castro M, Jenkinson R, Ślęczka P, Nabi Nur A, Wood GCA, Feibel R, McMahon JS, Sigamani A, Biccard BM, Landoni G, Szczeklik W, Wang CY, Tomas-Hernandez J, Abraham V, Vincent J, Harvey V, Pettit S, Sontrop J, Garg AX; HIP ATTACK investigators. Effects of accelerated versus standard care surgery on the risk of acute kidney injury in patients with a hip fracture: a substudy protocol of the hip fracture Accelerated surgical TreaTment And Care tracK (HIP ATTACK) international randomised controlled trial. BMJ Open. 2019 Sep 24;9(9):e033150. doi: 10.1136/bmjopen-2019-033150.

ReferenceCitation: Borges FK, Bhandari M, Patel A, Avram V, Guerra-Farfán E, Sigamani A, Umer M, Tiboni M, Adili A, Neary J, Tandon V, Sancheti PK, Lawendy A, Jenkinson R, Ramokgopa M, Biccard BM, Szczeklik W, Wang CY, Landoni G, Forget P, Popova E, Wood G, Nabi Nur A, John B, Ślęczka P, Feibel RJ, Balaguer-Castro M, Deheshi B, Winemaker M, de Beer J, Kolesar R, Teixidor-Serra J, Tomas-Hernandez J, McGillion M, Shanthanna H, Moppett I, Vincent J, Pettit S, Harvey V, Gauthier L, Alvarado K, Devereaux PJ. Rationale and design of the HIP fracture Accelerated surgical TreaTment And Care tracK (HIP ATTACK) Trial: a protocol for an international randomised controlled trial evaluating early surgery for hip fracture patients. BMJ Open. 2019 May 1;9(4):e028537. doi: 10.1136/bmjopen-2018-028537.

lead_sponsor: McMaster University

location_country:
Poland
Belgium
Hong Kong
Spain
Italy
United Kingdom
Netherlands
Canada
Thailand
United States
France
China
India
Colombia
South Africa
Malaysia
Pakistan

location_facility: Sancheti Institute for Orthopaedics and Rehabilitation

location_facility: Hospital Sant Camil (Consorci Sanitari Garraf)

location_facility: SPZOZ Szpital Powiatowy w Bochni im M. Wieckiej

location_facility: Hospital Clinico Universitario de Vallad

location_facility: Victoria Hospital, London Health Sciences

location_facility: Thunder Bay Regional Health Sciences Centre

location_facility: Hamilton Health Sciences - Juravinski Hospital

location_facility: Samodzielny Publiczny Zakład Opieki Zdrowotnej W Myślenicach

location_facility: University Hospital & Educational Center of Medical University in Lodz


title: MPACT Extension Study: Multicenter, Survival Data Collection in Subjects Previously Enrolled in Protocol CA046

summary: A study to collect survival data on patients previously enrolled in Abraxane pancreatic cancer study CA046.

study_type: Observational

time_perspective: Prospective

ReferenceType: background

ReferenceCitation: Ramanathan RK, Goldstein D, Korn RL, Arena F, Moore M, Siena S, Teixeira L, Tabernero J, Van Laethem JL, Liu H, McGovern D, Lu B, Von Hoff DD. Positron emission tomography response evaluation from a randomized phase III trial of weekly nab-paclitaxel plus gemcitabine versus gemcitabine alone for patients with metastatic adenocarcinoma of the pancreas. Ann Oncol. 2016 Apr;27(4):648-53. doi: 10.1093/annonc/mdw020. Epub 2016 Jan 22.

ReferenceCitation: Chiorean EG, Von Hoff DD, Reni M, Arena FP, Infante JR, Bathini VG, Wood TE, Mainwaring PN, Muldoon RT, Clingan PR, Kunzmann V, Ramanathan RK, Tabernero J, Goldstein D, McGovern D, Lu B, Ko A. CA19-9 decrease at 8 weeks as a predictor of overall survival in a randomized phase III trial (MPACT) of weekly nab-paclitaxel plus gemcitabine versus gemcitabine alone in patients with metastatic pancreatic cancer. Ann Oncol. 2016 Apr;27(4):654-60. doi: 10.1093/annonc/mdw006. Epub 2016 Jan 22.

ReferenceCitation: Goldstein D, Von Hoff DD, Moore M, Greeno E, Tortora G, Ramanathan RK, Macarulla T, Liu H, Pilot R, Ferrara S, Lu B. Development of peripheral neuropathy and its association with survival during treatment with nab-paclitaxel plus gemcitabine for patients with metastatic adenocarcinoma of the pancreas: A subset analysis from a randomised phase III trial (MPACT). Eur J Cancer. 2016 Jan;52:85-91. doi: 10.1016/j.ejca.2015.10.017. Epub 2015 Dec 1.

ReferenceCitation: Portal A, Pernot S, Tougeron D, Arbaud C, Bidault AT, de la Fouchardière C, Hammel P, Lecomte T, Dréanic J, Coriat R, Bachet JB, Dubreuil O, Marthey L, Dahan L, Tchoundjeu B, Locher C, Lepère C, Bonnetain F, Taieb J. Nab-paclitaxel plus gemcitabine for metastatic pancreatic adenocarcinoma after Folfirinox failure: an AGEO prospective multicentre cohort. Br J Cancer. 2015 Sep 29;113(7):989-95. doi: 10.1038/bjc.2015.328. Epub 2015 Sep 15.

ReferenceCitation: Goldstein D, El-Maraghi RH, Hammel P, Heinemann V, Kunzmann V, Sastre J, Scheithauer W, Siena S, Tabernero J, Teixeira L, Tortora G, Van Laethem JL, Young R, Penenberg DN, Lu B, Romano A, Von Hoff DD. nab-Paclitaxel plus gemcitabine for metastatic pancreatic cancer: long-term survival from a phase III trial. J Natl Cancer Inst. 2015 Jan 31;107(2). pii: dju413. doi: 10.1093/jnci/dju413. Print 2015 Feb.

ReferenceCitation: Vogel A, Pelzer U, Salah-Eddin AB, Köster W. First-line nab-paclitaxel and gemcitabine in patients with metastatic pancreatic cancer from routine clinical practice. In Vivo. 2014 Nov-Dec;28(6):1135-40.

ReferenceCitation: Von Hoff DD, Ervin T, Arena FP, Chiorean EG, Infante J, Moore M, Seay T, Tjulandin SA, Ma WW, Saleh MN, Harris M, Reni M, Dowden S, Laheru D, Bahary N, Ramanathan RK, Tabernero J, Hidalgo M, Goldstein D, Van Cutsem E, Wei X, Iglesias J, Renschler MF. Increased survival in pancreatic cancer with nab-paclitaxel plus gemcitabine. N Engl J Med. 2013 Oct 31;369(18):1691-703. doi: 10.1056/NEJMoa1304369. Epub 2013 Oct 16.

ReferenceCitation: Al-Hajeili M, Azmi AS, Choi M. Nab-paclitaxel: potential for the treatment of advanced pancreatic cancer. Onco Targets Ther. 2014 Feb 4;7:187-92. doi: 10.2147/OTT.S40705. eCollection 2014. Review.

ReferenceCitation: Tabernero J, Chiorean EG, Infante JR, Hingorani SR, Ganju V, Weekes C, Scheithauer W, Ramanathan RK, Goldstein D, Penenberg DN, Romano A, Ferrara S, Von Hoff DD. Prognostic factors of survival in a randomized phase III trial (MPACT) of weekly nab-paclitaxel plus gemcitabine versus gemcitabine alone in patients with metastatic pancreatic cancer. Oncologist. 2015 Feb;20(2):143-50. doi: 10.1634/theoncologist.2014-0394. Epub 2015 Jan 12.

ReferenceCitation: Chiorean EG, Von Hoff DD, Tabernero J, El-Maraghi R, Ma WW, Reni M, Harris M, Whorf R, Liu H, Li JS, Manax V, Romano A, Lu B, Goldstein D. Second-line therapy after nab-paclitaxel plus gemcitabine or after gemcitabine for patients with metastatic pancreatic cancer. Br J Cancer. 2016 Jul 12;115(2):188-94. doi: 10.1038/bjc.2016.185. Epub 2016 Jun 28. Erratum in: Br J Cancer. 2016 Oct 25;115(9):e13.

ReferenceCitation: Tehfe M, Dowden S, Kennecke H, El-Maraghi R, Lesperance B, Couture F, Letourneau R, Liu H, Romano A. nab-Paclitaxel Plus Gemcitabine Versus Gemcitabine in Patients with Metastatic Pancreatic Adenocarcinoma: Canadian Subgroup Analysis of the Phase 3 MPACT Trial. Adv Ther. 2016 May;33(5):747-59. doi: 10.1007/s12325-016-0327-4. Epub 2016 Apr 16. Erratum in: Adv Ther. 2016 Nov 24;:.

ReferenceCitation: Scheithauer W, Ramanathan RK, Moore M, Macarulla T, Goldstein D, Hammel P, Kunzmann V, Liu H, McGovern D, Romano A, Von Hoff DD. Dose modification and efficacy of nab-paclitaxel plus gemcitabine vs. gemcitabine for patients with metastatic pancreatic cancer: phase III MPACT trial. J Gastrointest Oncol. 2016 Jun;7(3):469-78. doi: 10.21037/jgo.2016.01.03.

ReferenceCitation: Vogel A, Römmler-Zehrer J, Li JS, McGovern D, Romano A, Stahl M. Efficacy and safety profile of nab-paclitaxel plus gemcitabine in patients with metastatic pancreatic cancer treated to disease progression: a subanalysis from a phase 3 trial (MPACT). BMC Cancer. 2016 Oct 21;16(1):817.

ReferenceCitation: Tabernero J, Kunzmann V, Scheithauer W, Reni M, Shiansong Li J, Ferrara S, Djazouli K. nab-Paclitaxel plus gemcitabine for metastatic pancreatic cancer: a subgroup analysis of the Western European cohort of the MPACT trial. Onco Targets Ther. 2017 Feb 2;10:591-596. doi: 10.2147/OTT.S124097. eCollection 2017.

ReferenceCitation: Kunzmann V, Ramanathan RK, Goldstein D, Liu H, Ferrara S, Lu B, Renschler MF, Von Hoff DD. Tumor Reduction in Primary and Metastatic Pancreatic Cancer Lesions With nab-Paclitaxel and Gemcitabine: An Exploratory Analysis From a Phase 3 Study. Pancreas. 2017 Feb;46(2):203-208. doi: 10.1097/MPA.0000000000000742.

lead_sponsor: Celgene

location_country:
Spain
Italy
Austria
Australia
Canada
Ukraine
United States
France
Germany

location_facility: HOCA - the Wesley Clinic for Heamatology and Oncology

location_facility: South Texas Oncology and Hematology (STOH)

location_facility: Municipal institution Multifield City Clinical Hospital #4 of Dnipropetrovsk Regional Council

location_facility: Ospedale Casa Sollievo Della Sofferenza IRCCS

location_facility: Centre Hospsitalier Universitaire de Montreal - Notre Dame

location_facility: University of Massachusetts Memorial Medical Center

location_facility: Azienda Ospedaliera Universitaria Integrata Di Verona


title: Intensified Pre-Operative Chemotherapy And Radical Surgery For High Risk Hepatoblastoma

summary:
RATIONALE: Drugs used in chemotherapy work in different ways to stop tumor cells from dividing so they stop growing or die. Giving chemotherapy drugs before surgery may shrink the tumor so that it can be removed.

PURPOSE: This phase II trial is studying how well neoadjuvant chemotherapy works in treating young patients who are undergoing surgical resection for high-risk hepatoblastoma.

status: Unknown status

ReferenceCitation: Zsiros J, Brugieres L, Brock P, Roebuck D, Maibach R, Zimmermann A, Childs M, Pariente D, Laithier V, Otte JB, Branchereau S, Aronson D, Rangaswami A, Ronghe M, Casanova M, Sullivan M, Morland B, Czauderna P, Perilongo G; International Childhood Liver Tumours Strategy Group (SIOPEL). Dose-dense cisplatin-based chemotherapy and surgery for children with high-risk hepatoblastoma (SIOPEL-4): a prospective, single-arm, feasibility study. Lancet Oncol. 2013 Aug;14(9):834-42. doi: 10.1016/S1470-2045(13)70272-9. Epub 2013 Jul 4.

ReferenceCitation: Weeda VB, Murawski M, McCabe AJ, Maibach R, Brugières L, Roebuck D, Fabre M, Zimmermann A, Otte JB, Sullivan M, Perilongo G, Childs M, Brock P, Zsíros J, Plaschkes J, Czauderna P, Aronson DC. Fibrolamellar variant of hepatocellular carcinoma does not have a better survival than conventional hepatocellular carcinoma--results and treatment recommendations from the Childhood Liver Tumour Strategy Group (SIOPEL) experience. Eur J Cancer. 2013 Aug;49(12):2698-704. doi: 10.1016/j.ejca.2013.04.012. Epub 2013 May 15.

lead_sponsor: University of Leicester

location_country:
Netherlands
France
United Kingdom
Ireland

location_facility: Our Lady's Hospital for Sick Children Crumlin

location_facility: Great Ormond Street Hospital for Children

location_facility: Royal Liverpool Children's Hospital, Alder Hey

location_facility: Institute of Child Health at University of Bristol

location_facility: Leeds Cancer Centre at St. James's University Hospital

location_facility: Royal Belfast Hospital for Sick Children

location_facility: Sir James Spence Institute of Child Health at Royal Victoria Infirmary


title: A Randomized, Double-blind, Multicenter Study to Demonstrate Equivalent Efficacy and to Compare Safety and Immunogenicity of a Biosimilar Adalimumab (GP2017) and Humira® in Patients With Moderate to Severe Chronic Plaque-type Psoriasis

acronym: ADACCESS

summary: The aim of the study is to demonstrate equivalent efficacy and similarity in the safety profile of GP2017 and Humira® in patients with moderate to severe chronic plaque-type psoriasis.

ReferenceCitation: Blauvelt A, Leonardi CL, Gaylis N, Jauch-Lembach J, Balfour A, Lemke L, Hachaichi S, Brueckmann I, Festini T, Wiland P. Treatment with SDZ-ADL, an Adalimumab Biosimilar, in Patients with Rheumatoid Arthritis, Psoriasis, or Psoriatic Arthritis: Results of Patient-Reported Outcome Measures from Two Phase III Studies (ADMYRA and ADACCESS). BioDrugs. 2021 Mar;35(2):229-238. doi: 10.1007/s40259-021-00470-1. Epub 2021 Mar 2.

ipd_sharing: Undecided

lead_sponsor: Sandoz

location_country:
Bulgaria
United States
Slovakia
France

location_facility: Dawes Fretzin Clinical Research Group, LLC

location_facility: Southern California Permanente Medical Group

location_facility: California Dermatology & Clinical Research Institute

location_facility: Kozne oddelenie,Nemocnica Kosice-Saca a.s.,1. sukromna nemoc

location_facility: SANARE s.r.o. Dermatovenerologická ambulancia

location_facility: Psoriasis Treatment Center of South Florida

location_facility: Bakersfield Dermatology and Skin Cancer Medical Group

location_facility: Diagnostic-consulting center 3-Varna EOOD

location_facility: University of Pittsburgh Medical Center Health System

location_facility: Ohio State University Clinical Trials Management Office

location_facility: Center for Skin and Venereal Diseases EOOD Sofia

location_facility: Pedi-Derma s.r.o., Dermatovenerologicka ambulancia


title: A MULTICENTER, INTERNATIONAL, PHASE 3, DOUBLE-BLIND, PLACEBO-CONTROLLED, RANDOMIZED STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF DAILY ORAL DOSING OF TAFAMIDIS MEGLUMINE (PF-06291826) 20 MG OR 80 MG IN COMPARISON TO PLACEBO IN SUBJECTS DIAGNOSED WITH TRANSTHYRETIN CARDIOMYOPATHY (TTR-CM)

acronym: ATTR-ACT

summary: This Phase 3 study will investigate the efficacy, safety and tolerability of an oral daily dose of 20 mg or 80 mg tafamidis meglumine capsules compared to placebo in subjects with either transthyretin genetic variants or wild-type transthyretin resulting in amyloid cardiomyopathy.

ReferenceCitation: Tess DA, Maurer TS, Li Z, Bulawa C, Fleming J, Moody AT. Relationship of binding-site occupancy, transthyretin stabilisation and disease modification in patients with tafamidis-treated transthyretin amyloid cardiomyopathy. Amyloid. 2022 Nov 18:1-12. doi: 10.1080/13506129.2022.2145876. [Epub ahead of print]

ReferenceCitation: Rozenbaum MH, Tran D, Bhambri R, Nativi-Nicolau J. Annual Cardiovascular-Related Hospitalization Days Avoided with Tafamidis in Patients with Transthyretin Amyloid Cardiomyopathy. Am J Cardiovasc Drugs. 2022 Jul;22(4):445-450. doi: 10.1007/s40256-022-00526-9. Epub 2022 Mar 30.

ReferenceCitation: Nativi-Nicolau J, Judge DP, Hoffman JE, Gundapaneni B, Keohane D, Sultan MB, Grogan M. How did transthyretin amyloid cardiomyopathy progress in patients who took placebo in the study ATTR-ACT? A plain language summary. Future Cardiol. 2022 Mar 17. doi: 10.2217/fca-2021-0150. Online ahead of print.

ReferenceCitation: Elliott P, Drachman BM, Gottlieb SS, Hoffman JE, Hummel SL, Lenihan DJ, Ebede B, Gundapaneni B, Li B, Sultan MB, Shah SJ. Long-Term Survival With Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy. Circ Heart Fail. 2022 Jan;15(1):e008193. doi: 10.1161/CIRCHEARTFAILURE.120.008193. Epub 2021 Dec 20.

ReferenceCitation: Hanna M, Damy T, Grogan M, Stewart M, Gundapaneni B, Sultan MB, Maurer MS. Tafamidis and quality of life in people with transthyretin amyloid cardiomyopathy in the study ATTR-ACT: A plain language summary. Future Cardiol. 2022 Mar;18(3):165-172. doi: 10.2217/fca-2021-0095. Epub 2021 Nov 15.

ReferenceCitation: Rozenbaum MH, Garcia A, Grima D, Tran D, Bhambri R, Stewart M, Li B, Heeg B, Postma M, Masri A. Health impact of tafamidis in transthyretin amyloid cardiomyopathy patients: an analysis from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) and the open-label long-term extension studies. Eur Heart J Qual Care Clin Outcomes. 2022 Aug 17;8(5):529-538. doi: 10.1093/ehjqcco/qcab031.

ReferenceCitation: Vong C, Boucher M, Riley S, Harnisch LO. Modeling of Survival and Frequency of Cardiovascular-Related Hospitalization in Patients with Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis. Am J Cardiovasc Drugs. 2021 Sep;21(5):535-543. doi: 10.1007/s40256-021-00464-y. Epub 2021 Mar 26.

ReferenceCitation: Miller AB, Januzzi JL, O'Neill BJ, Gundapaneni B, Patterson TA, Sultan MB, López-Sendón J. Causes of Cardiovascular Hospitalization and Death in Patients With Transthyretin Amyloid Cardiomyopathy (from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial [ATTR-ACT]). Am J Cardiol. 2021 Jun 1;148:146-150. doi: 10.1016/j.amjcard.2021.02.035. Epub 2021 Mar 3.

ReferenceCitation: Rapezzi C, Elliott P, Damy T, Nativi-Nicolau J, Berk JL, Velazquez EJ, Boman K, Gundapaneni B, Patterson TA, Schwartz JH, Sultan MB, Maurer MS. Efficacy of Tafamidis in Patients With Hereditary and Wild-Type Transthyretin Amyloid Cardiomyopathy: Further Analyses From ATTR-ACT. JACC Heart Fail. 2021 Feb;9(2):115-123. doi: 10.1016/j.jchf.2020.09.011. Epub 2020 Dec 9.

ReferenceCitation: Hanna M, Damy T, Grogan M, Stewart M, Gundapaneni B, Patterson TA, Schwartz JH, Sultan MB, Maurer MS. Impact of Tafamidis on Health-Related Quality of Life in Patients With Transthyretin Amyloid Cardiomyopathy (from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial). Am J Cardiol. 2021 Feb 15;141:98-105. doi: 10.1016/j.amjcard.2020.10.066. Epub 2020 Nov 19.

ReferenceCitation: Damy T, Garcia-Pavia P, Hanna M, Judge DP, Merlini G, Gundapaneni B, Patterson TA, Riley S, Schwartz JH, Sultan MB, Witteles R. Efficacy and safety of tafamidis doses in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) and long-term extension study. Eur J Heart Fail. 2021 Feb;23(2):277-285. doi: 10.1002/ejhf.2027. Epub 2020 Nov 12.

ReferenceCitation: Li B, Alvir J, Stewart M. Extrapolation of Survival Benefits in Patients with Transthyretin Amyloid Cardiomyopathy Receiving Tafamidis: Analysis of the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial. Cardiol Ther. 2020 Dec;9(2):535-540. doi: 10.1007/s40119-020-00179-2. Epub 2020 Jun 10.

ReferenceCitation: Maurer MS, Schwartz JH, Gundapaneni B, Elliott PM, Merlini G, Waddington-Cruz M, Kristen AV, Grogan M, Witteles R, Damy T, Drachman BM, Shah SJ, Hanna M, Judge DP, Barsdorf AI, Huber P, Patterson TA, Riley S, Schumacher J, Stewart M, Sultan MB, Rapezzi C; ATTR-ACT Study Investigators. Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy. N Engl J Med. 2018 Sep 13;379(11):1007-1016. doi: 10.1056/NEJMoa1805689. Epub 2018 Aug 27.

ReferenceCitation: Maurer MS, Elliott P, Merlini G, Shah SJ, Cruz MW, Flynn A, Gundapaneni B, Hahn C, Riley S, Schwartz J, Sultan MB, Rapezzi C; ATTR-ACT Study Investigators. Design and Rationale of the Phase 3 ATTR-ACT Clinical Trial (Tafamidis in Transthyretin Cardiomyopathy Clinical Trial). Circ Heart Fail. 2017 Jun;10(6). pii: e003815. doi: 10.1161/CIRCHEARTFAILURE.116.003815. Review.

location_country:
Brazil
Spain
Italy
United Kingdom
Netherlands
Canada
Sweden
United States
France
Belgium
Japan
Germany
Czechia

location_facility: The Mount Sinai Hospital Department of Pharmacy

location_facility: University of Maryland, School of Medicine

location_facility: Unidade de Pesquisa Clinica / Hospital Universitario Clementino Fraga Filho / UFRJ

location_facility: Hospital Universitario Puerta De Hierro - Majadahonda

location_facility: Icahn School of Medicine at Mount Sinai and Mount Sinai Hospital

location_facility: Boston Medical Center Investigational Pharmacy Services

location_facility: Columbia University Medical Center Research Pharmacy

location_facility: Boston Medical Center - Cardiovascular Center, Outpatient Clinic

location_facility: Hospital Clinico Universitario Virgen de la Arrixaca

location_facility: St Bartholomew's Hospital-Barts Health NHS Trust

location_facility: UCSD Clinical and Translational Research Institute, Investigational Drug Service

location_facility: Mayo Clinic Arizona - Mayo Clinic Building

location_facility: Centro per lo Studio e la Cura Delle Amiloidosi Sistemiche - Fondazione IRCCS Policlinico S. Matteo

location_facility: Cedars-Sinai Medical Center, Investigational Drug Pharmacy Services

location_facility: UCSF Cardiovascular Care and Prevention Center

location_facility: University of Utah,Health Sciences Center

location_facility: University of Calgary/Foothills Medical Centre

location_facility: Altman Clinical Translational Research institute

location_facility: University Hospital, University of Alabama at Birmingham

location_facility: Columbia University Medical Center/Clinical Cardiovascular Research Laboratory for the Elderly

location_facility: Altman Clinical Translational Research Institute

location_facility: Stanford University Hospital and Clinics

location_facility: Oregon Health and Science University (OHSU)

location_facility: Mayo Clinic Arizona - Mayo Clinic Speciality Building

location_facility: St. George's Hospital (St George's Healthcare NHS Trust)

location_facility: S.O.D. Cardiologia Generale 1 - D.A.I. del Cuore e dei Vasi

location_facility: Vseobecna fakultni nemocnice v Praze, Nemocnicni lekarna

location_facility: Michigan Medicine,University of Michigan

location_facility: Fakultni nemocnice u sv. Anny, Nemocnicni lekarna

location_facility: Boston University Medical Center General Clinical Research Unit

location_facility: VCU Medical Center - Ambulatory Care Clinic


title: Cryoablation of Intercostal Nerves for Better Control of Postoperative Pain After Thoracic and Thoracoabdominal Open Surgery - A Physician-initiated, International, Multicentre, Prospective, Observational Registry of Patients Undergoing Open Thoracic (TAA) and Thoracoabdominal Aortic Aneurysms Repair (TAAA) Using Cryoablation of Intercostal Nerves to Improve Postoperative Pain Control. (Cryo Registry)

summary:
The purpose of the registry is to evaluate the peri-operative and short- term outcomes of intercostal nerves cryoablation for post-operative pain control after open thoracic and thoracoabdominal aortic aneurysms repair.

The cryoablation procedure is one of the opportunities for pain control after thoracotomy. So, it is routinely performed in major several centers in aortic surgery around the world as a part of the TAA and TAAA procedure.

The Registry will include approximately 200 patients treated from January 2022 to January 2024 for thoracic or thoracoabdominal aortic aneurysm by means of open surgery and in whom intraoperative intercostal nerves cryoablation will be performed; to reach 200 patients, the enrolments can be extended until January 2027.

study_start_date_type: Anticipated

status: Not yet recruiting

ReferenceCitation: Carlsson CA, Persson K, Pelletieri L. Painful scars after thoracic and abdominal surgery. Acta Chir Scand. 1985;151(4):309-11.

ReferenceCitation: Soto RG, Fu ES. Acute pain management for patients undergoing thoracotomy. Ann Thorac Surg. 2003 Apr;75(4):1349-57. Review.

ReferenceCitation: Gottschalk A, Cohen SP, Yang S, Ochroch EA. Preventing and treating pain after thoracic surgery. Anesthesiology. 2006 Mar;104(3):594-600. Review.

ReferenceCitation: Clemence J Jr, Malik A, Farhat L, Wu X, Kim KM, Patel H, Yang B. Cryoablation of Intercostal Nerves Decreased Narcotic Usage After Thoracic or Thoracoabdominal Aortic Aneurysm Repair. Semin Thorac Cardiovasc Surg. 2020 Autumn;32(3):404-412. doi: 10.1053/j.semtcvs.2020.01.008. Epub 2020 Jan 20.

ReferenceCitation: Johnston KW, Rutherford RB, Tilson MD, Shah DM, Hollier L, Stanley JC. Suggested standards for reporting on arterial aneurysms. Subcommittee on Reporting Standards for Arterial Aneurysms, Ad Hoc Committee on Reporting Standards, Society for Vascular Surgery and North American Chapter, International Society for Cardiovascular Surgery. J Vasc Surg. 1991 Mar;13(3):452-8.

ReferenceCitation: Chaikof EL, Fillinger MF, Matsumura JS, Rutherford RB, White GH, Blankensteijn JD, Bernhard VM, Harris PL, Kent KC, May J, Veith FJ, Zarins CK. Identifying and grading factors that modify the outcome of endovascular aortic aneurysm repair. J Vasc Surg. 2002 May;35(5):1061-6. Review.

ReferenceCitation: Achouh PE, Madsen K, Miller CC 3rd, Estrera AL, Azizzadeh A, Dhareshwar J, Porat E, Safi HJ. Gastrointestinal complications after descending thoracic and thoracoabdominal aortic repairs: a 14-year experience. J Vasc Surg. 2006 Sep;44(3):442-6.

ReferenceCitation: Riambau V, Böckler D, Brunkwall J, Cao P, Chiesa R, Coppi G, Czerny M, Fraedrich G, Haulon S, Jacobs MJ, Lachat ML, Moll FL, Setacci C, Taylor PR, Thompson M, Trimarchi S, Verhagen HJ, Verhoeven EL, Esvs Guidelines Committee, Kolh P, de Borst GJ, Chakfé N, Debus ES, Hinchliffe RJ, Kakkos S, Koncar I, Lindholt JS, Vega de Ceniga M, Vermassen F, Verzini F, Document Reviewers, Kolh P, Black JH 3rd, Busund R, Björck M, Dake M, Dick F, Eggebrecht H, Evangelista A, Grabenwöger M, Milner R, Naylor AR, Ricco JB, Rousseau H, Schmidli J. Editor's Choice - Management of Descending Thoracic Aorta Diseases: Clinical Practice Guidelines of the European Society for Vascular Surgery (ESVS). Eur J Vasc Endovasc Surg. 2017 Jan;53(1):4-52. doi: 10.1016/j.ejvs.2016.06.005.

ReferenceCitation: Katz J, Jackson M, Kavanagh BP, Sandler AN. Acute pain after thoracic surgery predicts long-term post-thoracotomy pain. Clin J Pain. 1996 Mar;12(1):50-5.

ReferenceCitation: Richardson J, Sabanathan S, Shah R. Post-thoracotomy spirometric lung function: the effect of analgesia. A review. J Cardiovasc Surg (Torino). 1999 Jun;40(3):445-56. Review.

lead_sponsor: Germano Melissano

location_country:
Portugal
United Kingdom
Italy
Austria
Netherlands
Switzerland
France
Germany

location_facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS

location_facility: University Medical Center Hamburg-Eppendorf

location_facility: Centro Hospitalar de Entre o Douro e Vouga


title: Patisiran-LNP Pregnancy Surveillance Program

summary: The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal/neonatal/infant outcomes in women exposed to patisiran-LNP.

status: Recruiting

lead_sponsor: Alnylam Pharmaceuticals

location_country:
Portugal
Spain
Italy
Netherlands
United States
France
Germany


title: A Pan-European Prospective Interventional Cohort Study in Patients Hospitalized With Acute COPD Exacerbations With Longitudinal Follow-up

summary: The CATALINA study is a prospective interventional cohort study embedded within CICERO (Collaboration In COPD ExaceRbatiOns, a European Respiratory Society supported Clinical Research Collaboration), designed to collect standardised, longitudinal clinical data and biological samples in 20 pan-European centres.

lead_sponsor: Wim Janssens

location_country:
Spain
Italy
United Kingdom
Netherlands
Sweden
France
Belgium
Germany

location_facility: University Medical Centre of Gießen & Marburg


title: A Randomised, Double-Blind Study to Assess the Efficacy of Selumetinib (AZD6244: ARRY-142886) (Hyd-Sulfate) in Combination With Dacarbazine Compared With Placebo in Combination With Dacarbazine as First Systemic Therapy in Patients With Metastatic Uveal Melanoma (SUMIT)

acronym: SUMIT

summary: Selumetinib therapy in patients with metastatic uveal melanoma.

ReferenceCitation: Carvajal RD, Piperno-Neumann S, Kapiteijn E, Chapman PB, Frank S, Joshua AM, Piulats JM, Wolter P, Cocquyt V, Chmielowski B, Evans TRJ, Gastaud L, Linette G, Berking C, Schachter J, Rodrigues MJ, Shoushtari AN, Clemett D, Ghiorghiu D, Mariani G, Spratt S, Lovick S, Barker P, Kilgour E, Lai Z, Schwartz GK, Nathan P. Selumetinib in Combination With Dacarbazine in Patients With Metastatic Uveal Melanoma: A Phase III, Multicenter, Randomized Trial (SUMIT). J Clin Oncol. 2018 Apr 20;36(12):1232-1239. doi: 10.1200/JCO.2017.74.1090. Epub 2018 Mar 12. Erratum in: J Clin Oncol. 2018 Dec 10;36(35):3528.

ReferenceCitation: Carvajal RD, Schwartz GK, Mann H, Smith I, Nathan PD. Study design and rationale for a randomised, placebo-controlled, double-blind study to assess the efficacy of selumetinib (AZD6244; ARRY-142886) in combination with dacarbazine in patients with metastatic uveal melanoma (SUMIT). BMC Cancer. 2015 Jun 10;15:467. doi: 10.1186/s12885-015-1470-z.

lead_sponsor: AstraZeneca

location_country:
Spain
United Kingdom
Netherlands
Finland
Czech Republic
Canada
United States
France
Israel
Belgium
Germany


type: Registry Identifier

title: A Prospective Observational Study to Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

summary:
Primary Objective:

To describe the effect of routine practice with alglucosidase alfa in patients with IOPD ≤6 months of age, on invasive ventilation-free survival after 52 weeks of treatment.

Secondary Objectives:

To describe the effect of routine practice with alglucosidase alfa on invasive ventilation-free survival and survival at 12 and 18 months of age, as well as on change in left ventricular mass (LVM) Z score, Alberta Infant Motor Scale (AIMS) score, body weight, body length, and head circumference Z scores, and urinary glucose tetrasaccharide (Hex4), at Week 52 of treatment.
To describe the safety, tolerability, and immunogenicity of alglucosidase alfa in the routine practice of IOPD treatment.

lead_sponsor: Sanofi

location_country:
Taiwan
United Kingdom
Italy
Netherlands
France
Belgium
Germany

location_facility:
Investigational Site Number :1580001
Investigational Site Number :2500001
Investigational Site Number :0560001
Investigational Site Number :5280001
Investigational Site Number :3800002
Investigational Site Number :3800001
Investigational Site Number :8260002
Investigational Site Number :8260001
Investigational Site Number :2760001


title: European Geriatric Emergency Departments Registry Study

acronym: EGERS

summary: Geriatric presentations to emergency services comprise a significant percentage of all emergency service presentations in Europe and it has been reported that 3-23% of all emergency service presentations from various regions of the countries. There are specific management practices for patients who are 65 years and older at emergency services. On the other hand several risk-scoring systems have been developed to define the severity class of the patient during their initial evaluation at emergency services.Only a few studies in the literature have evaluated risk-scoring systems for the geriatric patient group.Several studies have reported that risk-scoring systems, such as Identification of Seniors at Risk and Triage Risk Screening Tool, which are specifically developed for geriatric patients over 65 years who present to emergency services, are not sufficiently effective for evaluating patients in more severe conditions. Previously the TEDGeS (Turkish Emergency Departments Geriatric Scoring Study) pilot study was carried out and published by some of the investigators of this project.This pilot study enrolled all geriatric patients (age ≥ 65 years) and carried out in 13 centers from different cities of Turkey. This pilot study showed that geriatric patients not only constitute significant proportion of emergency department presentations but also these patients need more hospitalization. The predictive powers of the Modified Early Warning Score, Rapid Emergency Medicine Score and The Vital PAC Early Warning Score for hospitalization and mortality in geriatric patients those presented to emergency department are significantly high and might be concerned in the emergency department triage of these patients. Within the light of these pilot study results, the investigators have decided to execute this prospective, multinational, multicentric study with the main objective to determine the epidemiological and age related characteristics of geriatric patients presenting to the emergency department across Europe and evaluate early warning scoring systems systems regarding hospitalization, ICU admission and in-hospital mortality for geriatric patients.

ReferenceCitation: Dundar ZD, Ergin M, Karamercan MA, Ayranci K, Colak T, Tuncar A, Cander B, Gul M. Modified Early Warning Score and VitalPac Early Warning Score in geriatric patients admitted to emergency department. Eur J Emerg Med. 2016 Dec;23(6):406-412.

ReferenceCitation: Cei M, Bartolomei C, Mumoli N. In-hospital mortality and morbidity of elderly medical patients can be predicted at admission by the Modified Early Warning Score: a prospective study. Int J Clin Pract. 2009 Apr;63(4):591-5. doi: 10.1111/j.1742-1241.2008.01986.x. Epub 2009 Feb 11.

ReferenceCitation: Dundar ZD, Ayranci MK. Presenting symptoms of older emergency department patients: a single-center experience of 10,692 patients in Turkey. Acta Clin Belg. 2020 Dec;75(6):405-410. doi: 10.1080/17843286.2019.1655215. Epub 2019 Aug 12.

ReferenceCitation: Lamantia MA, Stewart PW, Platts-Mills TF, Biese KJ, Forbach C, Zamora E, McCall BK, Shofer FS, Cairns CB, Busby-Whitehead J, Kizer JS. Predictive value of initial triage vital signs for critically ill older adults. West J Emerg Med. 2013 Sep;14(5):453-60. doi: 10.5811/westjem.2013.5.13411.

ReferenceCitation: Buurman BM, van den Berg W, Korevaar JC, Milisen K, de Haan RJ, de Rooij SE. Risk for poor outcomes in older patients discharged from an emergency department: feasibility of four screening instruments. Eur J Emerg Med. 2011 Aug;18(4):215-20. doi: 10.1097/MEJ.0b013e328344597e.

ReferenceCitation: Pines JM, Mullins PM, Cooper JK, Feng LB, Roth KE. National trends in emergency department use, care patterns, and quality of care of older adults in the United States. J Am Geriatr Soc. 2013 Jan;61(1):12-7. doi: 10.1111/jgs.12072.

ReferenceCitation: Platts-Mills TF, Travers D, Biese K, McCall B, Kizer S, LaMantia M, Busby-Whitehead J, Cairns CB. Accuracy of the Emergency Severity Index triage instrument for identifying elder emergency department patients receiving an immediate life-saving intervention. Acad Emerg Med. 2010 Mar;17(3):238-43. doi: 10.1111/j.1553-2712.2010.00670.x.

ReferenceCitation: Subbe CP, Kruger M, Rutherford P, Gemmel L. Validation of a modified Early Warning Score in medical admissions. QJM. 2001 Oct;94(10):521-6.

ReferenceCitation: Wheeler I, Price C, Sitch A, Banda P, Kellett J, Nyirenda M, Rylance J. Early warning scores generated in developed healthcare settings are not sufficient at predicting early mortality in Blantyre, Malawi: a prospective cohort study. PLoS One. 2013;8(3):e59830. doi: 10.1371/journal.pone.0059830. Epub 2013 Mar 29. Erratum in: PLoS One. 2014;9(2):e91623.

lead_sponsor: Gazi University

location_country: Croatia Ireland Greece France Turkey Germany

location_facility: Necmettin Erbakan University Meram Medical Faculty


title: Brain INvolvement in Dystrophinopathies (BIND): Deep Functional Phenotyping of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy Patients (WP5 and WP6) Part 2: a Neurobehavioural and MRI Study

summary: The objective of this study is to understand the relationship between DMD and BMD brain comorbidities, and the location of the gene mutation which causes the disease.

time_perspective: Cross-Sectional

lead_sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust

location_country:
Denmark
Spain
Italy
United Kingdom
France
Netherlands

location_facility: Imagine Institut Des Maladies Genetiques Necker Enfants Malades Fondation

location_facility: STICHTING KEMPENHAEGHE/Leiden University Medical Center


title: Brain INvolvement in Dystrophinopathies (BIND): Deep Functional Phenotyping of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy Patients (WP5) Part 1: a Multicentre Online Phenotyping and Neurobehavioural Data Collection Study

summary: The objective of this study is to collect data from a large cohort of individuals with DMD and BMD focusing on the neurobehavioural aspects of these conditions and their correlation to the location of the DMD gene mutation.

ipd_sharing_description: Pseudonymised data will be shared amongst collaborating partners in the project, but details on data sharing are still under discussion.


title: Comprehensive Long-term Follow up of Adults With Arterial Switch Operation - European Collaboration for Prospective Outcome Research in Congenital Heart Disease

acronym: EPOCH-ASO

summary:
Background: Long-term outcomes in adults with prior arterial switch operation (ASO) have not yet been well defined. The aim of this study is to elucidate incidence and predictors of adverse cardiac outcomes in a prospectively followed cohort of adults after their ASO.

Methods: The comprehensive long-term follow up of adults with ASO is a project within the European collaboration for prospective outcome research in congenital heart disease (EPOCH). It is designed as a prospective, international multicenter cohort study. Consecutive patients (aged 16 years or more) with prior ASO will be included at 11 European tertiary care centers. Participants will be followed according to a standardized protocol following international recommendations, including standardized protocols for imaging and for exercise testing. Main outcome measures are all-cause and cardiac-related mortality, rate of cardiac re-intervention, neo-aortic dissection, myocardial infarction, stroke, infective endocarditis, sustained atrial and ventricular arrhythmias, new-onset or worsening pulmonary hypertension and new-onset heart failure. Secondary endpoints are frequency and progression of right ventricular outflow tract stenosis, neo-aortic root dilatation, neo-aortic valve regurgitation and ventricular dysfunction. The impact of demographic, anatomic (e.g. coronary artery anatomy) and functional variables on the above-mentioned outcomes, as well as quality of life and incidence of pregnancy related complications will also be assessed.

Aim: The prospective, international, multicenter EPOCH-ASO study will provide a better understanding of adverse outcomes and their predictors in adults after ASO. The results of the EPOCH-ASO study may help to optimize future care of this novel patient cohort in adult cardiology.

lead_sponsor: University of Zurich

location_country: Spain Austria Switzerland France Netherlands

location_facility: Hospital Universitario y Politécnico La Fe Valencia

location_facility: CHU Paris IdF Ouest - HEGP Hôpital Européen Georges Pompidou


title: HER-Seq: A Blood-based Screening Study to Identify Patients With HER2 Mutations for Enrollment Into Clinical Research Studies of Neratinib

summary: This is a multi-center, observational genomic screening protocol to identify participants whose tumors harbor somatic mutations in the ERBB2 (HER2) gene, as measured in circulating tumor DNA (ctDNA) . Participants with histologically confirmed, hormone receptor positive, HER-2 negative, metastatic breast cancer (MBC) or metastatic cervical cancer (MCC) are eligible for screening at 6 months intervals, or if disease progression is suspected/confirmed. Blood samples will be collected from eligible participants and ctDNA will be extracted and sequenced at a central laboratory, using a HER2-targeted next generation sequencing (NGS) test. A certified molecular test report will be issued from the central laboratory and provided to the investigators and the study sponsor. Participants who are identified with HER2 mutations by this screening protocol will subsequently have access to an appropriate neratinib treatment protocol, pending medical eligibility.

ipd_sharing_description:
Puma Biotechnology is committed to sharing clinical trial data and information to help physicians and patients make informed treatment decisions, and to help qualified researchers advance scientific knowledge.

In accordance with legal and regulatory requirements, Puma publishes study protocol information and clinical study results on clinical trial registries, including ClinicalTrials.gov and EU Clinical Trials Register. Puma also publishes information about clinical studies in peer-reviewed scientific journals and shares data in scientific meetings.

Puma commits to safeguarding confidentiality and patient privacy throughout the clinical trial data and information sharing process. Any patient-level data will be anonymized to protect personally identifiable information.

Qualified researchers and study participants may submit requests for other study documentation and clinical trial data to clinicaltrials@pumabiotechnology.com for consideration.

lead_sponsor: Puma Biotechnology, Inc.

location_country:
Serbia
Spain
Ireland
Italy
United States
Israel
France

location_facility: Fundación Instituto Valenciano de Oncológia

location_facility: Rabin Medical Center, Davidoff Cancer Center

location_facility: Instituto Oncológico Dr. Rosell, Hospital Universitario Quirón Dexeus

location_facility: University Hospital Waterford, Department of Medical Oncology

location_facility: Beaumont Hospital, Cancer Clinical Trials & Research Unit

location_facility: Hospital Universitario Quirónsalud Madrid

location_facility: Institute for Oncology and Radiology of Serbia

location_facility: Hospital Universitario Madrid Sanchinarro

location_facility: Istituto Nazionale Tumori IRCCS "Fondazione G. Pascale"

location_facility: START Madrid, Hospital Universitario Fundacion Jimenez Diaz

location_facility: Mater Misericordiae University Hospital, Institute for Cancer Research

location_facility: Hospital Clínico Universitario de Valencia

intervention_type: Diagnostic Test


title: A Dose-Blinded, 2-Dose Level, Parallel-Group, Multicenter, Long-Term Extension Study to Evaluate the Long-Term Safety, Efficacy, and Immunogenicity of BIIB023 in Subjects With Lupus Nephritis

summary: The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB023 in participants with lupus nephritis (LN).

lead_sponsor: Biogen

location_country:
Poland
Brazil
Hong Kong
Spain
Colombia
Italy
Australia
Mexico
Argentina
Russian Federation
Thailand
Hungary
Korea, Republic of
United States
France
Peru
Belgium
Philippines
Malaysia


title: Prognostic Imaging Biomarkers for Diabetic Kidney Disease

acronym: iBEAt

summary:
Diabetic kidney disease (DKD) is a common complication of diabetes, and is now the most common form of chronic kidney disease. DKD is the leading cause of kidney disease requiring dialysis or kidney transplantation, and its global incidence and prevalence have reached epidemic levels. While the risk of developing DKD can be ameliorated by tight blood glucose and blood pressure control, it is not fully preventable and once established DKD cannot be cured. Therefore many patients are left with poor and worsening health and with increased mortality risk. Developing new ways to treat DKD requires healthcare professionals to be able to identify those patients most in need of treatment.

One promising approach for identifying patients that are at risk is the use of imaging measurements (called "biomarkers") derived from Magnetic Resonance Imaging (MRI) and Ultrasound (US) of the kidneys. Evidence from early studies shows that such imaging biomarkers can identify underlying problems in DKD such as blood supply, oxygen supply, kidney scarring and kidney function, in ways that are better than those currently available.

The investigators think that imaging biomarkers will improve the identification of patients who are likely to decline from DKD in the short term. The changes found by imaging may even happen before effects on the blood and urine.

The investigators plan to test this hypothesis by performing a study observing 500 patients with early stage DKD, recruited in 5 sites across Europe. All patients will have detailed assessment at the start of their involvement, including clinical assessment, blood and urine samples, and MRI and US scans. The investigators will look at whether imaging biomarkers are associated with other measures that predict progression in DKD, and follow patients every year for 3 years (4 years total study participation) to see if the imaging biomarkers predict worsening DKD.

ReferenceCitation: Grenier N, Merville P, Combe C. Radiologic imaging of the renal parenchyma structure and function. Nat Rev Nephrol. 2016 Jun;12(6):348-59. doi: 10.1038/nrneph.2016.44. Epub 2016 Apr 12. Review.

ReferenceCitation: Gooding KM, Lienczewski C, Papale M, Koivuviita N, Maziarz M, Dutius Andersson AM, Sharma K, Pontrelli P, Garcia Hernandez A, Bailey J, Tobin K, Saunavaara V, Zetterqvist A, Shelley D, Teh I, Ball C, Puppala S, Ibberson M, Karihaloo A, Metsärinne K, Banks RE, Gilmour PS, Mansfield M, Gilchrist M, de Zeeuw D, Heerspink HJL, Nuutila P, Kretzler M, Welberry Smith M, Gesualdo L, Andress D, Grenier N, Shore AC, Gomez MF, Sourbron S; BEAt-DKD consortium. Prognostic imaging biomarkers for diabetic kidney disease (iBEAt): study protocol. BMC Nephrol. 2020 Jun 29;21(1):242. doi: 10.1186/s12882-020-01901-x.

lead_sponsor: University of Leeds

location_country:
United Kingdom
Italy
Finland
Switzerland
France
Sweden

location_facility: Centre Hospitalier Universitaire de Bordeaux

location_facility: Università degli Studi di Bari Aldo Moro


title: A Phase Ib, Open-label Study of Oral BGJ398 in Combination With Oral BYL719 in Adult Patients With Select Advanced Solid Tumors

summary: To study the safety and efficacy of the combination of BGJ398 with BYL719 in patients whose tumors express mutations to PIK3CA with or without alterations to FGFR 1-3.

lead_sponsor: Novartis Pharmaceuticals

location_country:
Spain
Italy
Australia
Netherlands
Canada
Switzerland
Korea, Republic of
United States
France
Singapore
Belgium
Germany

location_facility: Cancer Therapy & Research Center / UT Health Science Center SC

location_facility: H. Lee Moffitt Cancer Center & Research Institute Moffitt 4

location_facility: Memorial Sloan Kettering Cancer Center Onc Dept

location_facility: University of Michigan Comprehensive Cancer Center SC

location_facility: Vanderbilt University Medical Center Dept of Onc

location_facility: Washington University School of Medicine Onc Dept


title: HEPANOVA: A Phase II Trial of Tumor Treating Fields (TTFields, 150kHz) Concomitant With Sorafenib For Advanced Hepatocellular Carcinoma (HCC)

summary: The study is a prospective, phase II trial single arm, historical control aimed to test the efficacy and safety of TTFields, using the NovoTTF-100L(P) System, in combination with sorafenib in patient with advanced HCC. The device is an experimental, portable, battery operated device for chronic administration of alternating electric fields (termed TTFields or TTF) to the region of the malignant tumor, by means of surface, insulated electrode arrays.

ReferenceCitation: Kirson ED, Gurvich Z, Schneiderman R, Dekel E, Itzhaki A, Wasserman Y, Schatzberger R, Palti Y. Disruption of cancer cell replication by alternating electric fields. Cancer Res. 2004 May 1;64(9):3288-95.

ReferenceCitation: Kirson ED, Dbalý V, Tovarys F, Vymazal J, Soustiel JF, Itzhaki A, Mordechovich D, Steinberg-Shapira S, Gurvich Z, Schneiderman R, Wasserman Y, Salzberg M, Ryffel B, Goldsher D, Dekel E, Palti Y. Alternating electric fields arrest cell proliferation in animal tumor models and human brain tumors. Proc Natl Acad Sci U S A. 2007 Jun 12;104(24):10152-7. Epub 2007 Jun 5.

ReferenceCitation: Stupp R, Wong ET, Kanner AA, Steinberg D, Engelhard H, Heidecke V, Kirson ED, Taillibert S, Liebermann F, Dbalý V, Ram Z, Villano JL, Rainov N, Weinberg U, Schiff D, Kunschner L, Raizer J, Honnorat J, Sloan A, Malkin M, Landolfi JC, Payer F, Mehdorn M, Weil RJ, Pannullo SC, Westphal M, Smrcka M, Chin L, Kostron H, Hofer S, Bruce J, Cosgrove R, Paleologous N, Palti Y, Gutin PH. NovoTTF-100A versus physician's choice chemotherapy in recurrent glioblastoma: a randomised phase III trial of a novel treatment modality. Eur J Cancer. 2012 Sep;48(14):2192-202. doi: 10.1016/j.ejca.2012.04.011. Epub 2012 May 18.

ReferenceCitation: Kirson ED, Giladi M, Gurvich Z, Itzhaki A, Mordechovich D, Schneiderman RS, Wasserman Y, Ryffel B, Goldsher D, Palti Y. Alternating electric fields (TTFields) inhibit metastatic spread of solid tumors to the lungs. Clin Exp Metastasis. 2009;26(7):633-40. doi: 10.1007/s10585-009-9262-y. Epub 2009 Apr 23.

ReferenceCitation: Giladi M, Schneiderman RS, Voloshin T, Porat Y, Munster M, Blat R, Sherbo S, Bomzon Z, Urman N, Itzhaki A, Cahal S, Shteingauz A, Chaudhry A, Kirson ED, Weinberg U, Palti Y. Mitotic Spindle Disruption by Alternating Electric Fields Leads to Improper Chromosome Segregation and Mitotic Catastrophe in Cancer Cells. Sci Rep. 2015 Dec 11;5:18046. doi: 10.1038/srep18046.

ReferenceCitation: Stupp R, Taillibert S, Kanner AA, Kesari S, Steinberg DM, Toms SA, Taylor LP, Lieberman F, Silvani A, Fink KL, Barnett GH, Zhu JJ, Henson JW, Engelhard HH, Chen TC, Tran DD, Sroubek J, Tran ND, Hottinger AF, Landolfi J, Desai R, Caroli M, Kew Y, Honnorat J, Idbaih A, Kirson ED, Weinberg U, Palti Y, Hegi ME, Ram Z. Maintenance Therapy With Tumor-Treating Fields Plus Temozolomide vs Temozolomide Alone for Glioblastoma: A Randomized Clinical Trial. JAMA. 2015 Dec 15;314(23):2535-43. doi: 10.1001/jama.2015.16669.

ReferenceCitation: Stupp R, Taillibert S, Kanner A, Read W, Steinberg D, Lhermitte B, Toms S, Idbaih A, Ahluwalia MS, Fink K, Di Meco F, Lieberman F, Zhu JJ, Stragliotto G, Tran D, Brem S, Hottinger A, Kirson ED, Lavy-Shahaf G, Weinberg U, Kim CY, Paek SH, Nicholas G, Bruna J, Hirte H, Weller M, Palti Y, Hegi ME, Ram Z. Effect of Tumor-Treating Fields Plus Maintenance Temozolomide vs Maintenance Temozolomide Alone on Survival in Patients With Glioblastoma: A Randomized Clinical Trial. JAMA. 2017 Dec 19;318(23):2306-2316. doi: 10.1001/jama.2017.18718. Erratum in: JAMA. 2018 May 1;319(17):1824.

lead_sponsor: NovoCure GmbH

location_country: Poland Spain Italy France Germany Czechia

location_facility: Uniwersytet Medyczny im. Karola Marcinkowskiego w Poznaniu

location_facility: Vall d'Hebron Institute of Oncology (VHIO)

location_facility: University Hospital Fakultni Nemocnice Olomouc, Oncology Clinic

location_facility: University Medical Center - University of Freiburg

location_facility: HM Hospitales - Centro Integral Oncológico Clara Campal


title: REspectful CAring for the AGitated Elderly. How to Best Meet the Needs of People With Dementia With Severe Behavioural Disturbances. Toward a Respectful and Cost-effective Model

acronym: RECAGE

summary: The treatment of Behavioural and Psychological Symptoms of Dementia (BPSD) continue to raise difficult problems. Both the pharmacologic and non-pharmacologic therapies often fail to achieve the expected results. The aim of this study is to assess the short- and long-term efficacy of an intervention, the Special Care Unit for patients with BPSD (SCU-B), already implemented in some countries but not validated so far, as well as its cost-effectiveness

ReferenceCitation: Koskas P, Belqadi S, Mazouzi S, Daraux J, Drunat O. [Behavioral and psychological symptoms of dementia in a pilot psychogeriatric unit: management and outcomes]. Rev Neurol (Paris). 2011 Mar;167(3):254-9. doi: 10.1016/j.neurol.2010.07.030. Epub 2010 Oct 14. French.

ReferenceCitation: Delphin-Combe F, Roubaud C, Martin-Gaujard G, Fortin ME, Rouch I, Krolak-Salmon P. [Effectiveness of a cognitive-behavioral unit on behavioral and psychological symptoms of dementia]. Rev Neurol (Paris). 2013 Jun-Jul;169(6-7):490-4. doi: 10.1016/j.neurol.2012.10.017. Epub 2013 Mar 21. French.

ReferenceCitation: Bianchetti A, Benvenuti P, Ghisla KM, Frisoni GB, Trabucchi M. An Italian model of dementia special care unit: results of a pilot study. Alzheimer Dis Assoc Disord. 1997 Mar;11(1):53-6.

ipd_sharing_description: De-identified participant data both for primary and secondary outcome measures will be made available

lead_sponsor: Fondazione Europea di Ricerca Biomedica Ferb Onlus

location_country: Italy Greece Switzerland France Belgium Norway Germany

location_facility: Azienda Unità Sanitaria Locale di Modena

location_facility: Aristotelio Panepistimio Thessalonikis (Greece)

location_facility: Zentralinstitut für Seelische Gesundheit

location_facility: Centre Hospitalier Universitaire St Pierre

location_facility: Azienda Socio Sanitaria Territoriale di Mantova


acronym: IMPALA-X

title: An Open-label, Non-controlled, Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients

lead_sponsor: Savara Inc.

location_country:
Denmark
United Kingdom
Greece
Italy
Netherlands
Russian Federation
France
Israel
Turkey
Germany

location_facility: Asklepios Fachkliniken München - Gauting Klinik für Pneumologie

location_facility: S.C. Pneumologia Fondazione IRCCS Policlinico San Matteo

location_facility: Universitätsklinikum Schleswig-Holstein Zentralklinikum Lübeck Medizinische Klinik III - Pneumologie

location_facility: Pavlov first Saint Petersburg State Medical Univerisity

location_facility: Dept. Of Intensive Care Unit Royal Brompton Hospital London

location_facility: CHU Rennes Hospital Pontchaillou, Service de Pneumologie

location_facility: Ruhrlandklinik Essen Westdeutsches Lungenzentrum am Universitätsklinikum Essen GmbH

location_facility: Thoraxklinik am Universitätsklinikum Heidelberg Abteilung für Pneumologie und Beatmungsmedizin

location_facility: Rabin Medical Center Institute of Pulomonary Medicine

location_facility: Yedikule Pulmonary Diseases and Pulmonary Surgery Training and Research Hospital

location_facility: Attikon University Hospital 2nd Pulmonary Department Athens Medical School National and Kapodistrian University of Athens

summary:
Clinical trial for subjects with autoimmune pulmonary alveolar proteinosis (aPAP) who have completed the IMPALA trial (NCT02702180).

At the Baseline visit, eligible subjects may continue or re-start treatment with 300 µg inhaled molgramostim (recombinant human Granulocyte-Macrophage Colony Stimulating Factor; GM-CSF) administered intermittently in cycles of seven days molgramostim, administered once daily, and seven days off treatment.

Subject will be treated with inhaled molgramostim for up to 36 months.

During the trial, whole lung lavage will be applied as rescue therapy.


title: A Randomized Phase II/III Trial of Doxycycline vs. Standard Supportive Therapy in Newly-diagnosed Cardiac AL Amyloidosis Patients Undergoing Bortezomib-based Therapy

summary:
Systemic amyloidoses are rare diseases affecting approximately 1 in 100,000 persons each year.

In systemic amyloidoses abnormal proteins deposit in bodily organs and severely impair their function, causing death if not treated effectively. Light chain (AL) amyloidosis is caused by a usually small population of plasma cells (the cells that produce antibodies). These cells produce part of antibodies, the light chains (LC) that form amyloid deposits. Almost every organ, with the exception of the brain, can be affected by AL amyloidosis. The heart is involved in three fourths of patients and is responsible for almost all the deaths occurring in the first 6 months after diagnosis. Current therapy of AL amyloidosis is based on drugs targeting the plasma cells producing the amyloid-forming LC. At present, most patients receive a powerful anti-plasma cell drug, bortezomib, as part of their initial treatment. However, bortezomib-based therapy, can improve heart involvement only in less than one third of patients with AL amyloidosis, and many patients (approximately one third) still die within 12 months from diagnosis. Early cardiac deaths remain an acute unmet need and the major determinant of overall outcome in this disease. Thus, there is the need of alternative means to treat heart involvement in AL amyloidosis. Doxycycline is a widely used, well tolerated, antibiotic that has been marketed for decades and used to treat a number of different infectious diseases caused by bacteria. This molecule has been extensively studied in the laboratory, in animal models and, more recently, in small studies involving patients, for its potential of improving cardiac damage in amyloidosis. These studies showed that doxycycline disrupts amyloid deposits, reduces the amyloid load in a mouse model, and counteracts the toxicity exerted by amyloid-forming LCs on C. elegans, a worm whose pharynx is used as a model resembling human heart. In a small clinical study, doxycycline was given to patients with cardiac AL amyloidosis during treatment for their underlying plasma cell disease. This resulted in a remarkable improvement of survival compared to "matched historical controls" (i.e. similar patients who had received only anti-plasma cell therapy without doxycycline in the past). Based on these promising preliminary results, we designed the present clinical trial to assess whether the addition of doxycycline to anti-plasma cell therapy can improve survival in patients with cardiac AL amyloidosis who were not previously treated. The rate of survival at 12 months will be compared in patients receiving doxycycline and in controls receiving standard antibiotic therapy, together with anti-plasma cell therapy. Patients will be assessed for parameters of plasma cell disease, heart involvement and possible involvement of other organs, as well as for quality of life. To make sure that patients who will receive doxycycline and those who will not have comparable severity of cardiac disease, patients will be stratified according to the stage of cardiac involvement. Patients with very advanced heart dysfunction will not be enrolled in the trial, because preliminary data indicate that doxycycline is of little or no benefit in these subjects. Patients will be randomized to receive doxycycline or standard antibiotics in combination with anti-plasma cell therapy. Bortezomib-based treatment directed against plasma cells will be delivered according to each participating institutions' guidelines. Doxycycline will be administered at a dosage of 100 mg two times a day, which is usual in the treatment of bacterial diseases. Standard antibiotics will be delivered according to each participating institutions' guidelines (provided that drugs of the same class as doxycycline are not administered) in the control arm. Patients will be provided a diary to record possible adverse events and will be instructed accordingly. Patients will be evaluated at trial centers every 2 months for treatment efficacy and toxicity. In case of unsatisfactory response second-line therapy will be initiated. In the absence of unacceptable toxicity, doxycycline administration will be continued for the entire duration of follow-up (12 months).

lead_sponsor: IRCCS Policlinico S. Matteo

location_country:
Spain
Italy
Greece
United Kingdom
Canada
France
Turkey
Germany

location_facility: University College London Medical School and Royal Free Hospital

location_facility: Cross Cancer Insititue, University of Alberta

location_facility: Istanbul University Cerrahpasa Faculty of Medicine


title: A Placebo-Controlled, Single-Blind, Single-Center Phase 1 Study in Normal Healthy Volunteers and Open-Label Multi-Center Study in Patients With Primary Hyperoxaluria to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of DCR-PHXC Solution for Injection (Subcutaneous Use)

summary: This is a double-blind, placebo-controlled, dose escalation trial of DCR-PHXC in Healthy Volunteers (HVs) and patients with Primary Hyperoxaluria (PH). Once safety has been established in HV, PH patients with a confirmed diagnosis of PH1 and PH2 will be enrolled across multiple dosing cohorts. The study design will allow enrollment of PH patient cohorts at a given dose level once safety has been demonstrated in HV at that dose level. The study will be conducted in two parts: Part A: Single ascending dose (SAD) in HV; Part B: SAD in patients with PH1 and PH2 (lagging Part A by 1 dose level cohort).

lead_sponsor: Dicerna Pharmaceuticals, Inc.

location_country:
United Kingdom
Netherlands
United States
France
Germany

location_facility: Centre d'Investigation Clinique - CIC 1407 - Hospices Civils de Lyon

location_facility: Universitätsklinikum Bonn-Institut für Klinische Chemie und Klinische Pharmakologie

location_facility: Birmingham Children's Hospital NHS Trust


title: FIRE AND ICE Re-Ablations: Retrospective Data Collection on Re-ablations Performed Within the FIRE AND ICE Trial

acronym: Re-Do

summary: Retrospective data collection on re-ablations performed within the FIRE AND ICE Trial.

time_perspective: Retrospective

ReferenceCitation: Kuck KH, Albenque JP, Chun KJ, Fürnkranz A, Busch M, Elvan A, Schlüter M, Braegelmann KM, Kueffer FJ, Hemingway L, Arentz T, Tondo C, Brugada J; FIRE AND ICE Investigators. Repeat Ablation for Atrial Fibrillation Recurrence Post Cryoballoon or Radiofrequency Ablation in the FIRE AND ICE Trial. Circ Arrhythm Electrophysiol. 2019 May 22;12(6):e007247. doi: 10.1161/CIRCEP.119.007247.

lead_sponsor: Medtronic Cardiac Rhythm and Heart Failure

location_country: Spain Netherlands Switzerland France Germany Czechia

location_facility: Cardioangiologisches Centrum Bethanien CCB

location_facility: Hospital Clinico Universitario Virgen de la Victoria


title: Phase II of Immunotherapy Plus Local Tumor Ablation (RFA or Stereotactic Radiotherapy) in Patients With Colorectal Cancer Liver Metastases

acronym: ILOC

summary:
This is a single-arm, open-label, multi-center early phase II study. This proof of concept study will investigate whether the combined use of local tumor ablation/radiation plus immunomodulating drugs may induce a significant immune response in patient with incurable liver metastases from colorectal cancer (CRC) (+/- limited extrahepatic disease) being stable or in partial remission after completion of 4-6 months first line systemic therapy.

The primary objective of the study is to show an overall response rate of lesions not treated by ablation/radiotherapy including the extrahepatic lesions (according to iRECIST criteria) higher than 10%. With the continuation of first line systemic treatment, no further responses are expected.

Secondary objectives are:

To establish the feasibility and safety of the combined treatment modalities;
To study the impact of the local technique (RFA/Radiotherapy) on the results;
To investigate biomarkers to predict response to the combined treatment

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

location_country: Austria Netherlands Switzerland Sweden France Germany

location_facility: Medical University Vienna - General Hospital AKH

location_facility: Radboud University Medical Center Nijmegen

location_facility: Hôpitaux universitaires de Genève - HUG - site de Cluse-Roseraie

location_facility: The Netherlands Cancer Institute-Antoni Van Leeuwenhoekziekenhuis

location_facility: Karolinska University Hospital - Karolinska Institutet - Danderyds Hospital

intervention_type: Radiation


title: An Open, Qualitative, Prospective, Multicenter Trial of a Novel Transanal Irrigation System in Spinal Cord Injured Patients.

summary: This study is designed as an open, prospective, non-controlled, qualitative, multicentre study of a novel transanal irrigation system performed in a population of 150 subjects suffering from spinal cord injury and confirmed neurological bowel dysfunction. The study is expected to last for a total of 1 year (treatment period) with a planned 12- month recruitment period and three scheduled site visits.

ReferenceCitation: Emmanuel A, Kurze I, Krogh K, Ferreiro Velasco ME, Christensen P, Del Popolo G, Bazzocchi G, Hultling C, Perrouin Verbe B, Bothig R, Glott T, Gonzalez Viejo MA. An open prospective study on the efficacy of Navina Smart, an electronic system for transanal irrigation, in neurogenic bowel dysfunction. PLoS One. 2021 Jan 29;16(1):e0245453. doi: 10.1371/journal.pone.0245453. eCollection 2021.

lead_sponsor: Dentsply International

location_country:
Denmark
Spain
Italy
United Kingdom
Sweden
France
Norway
Germany

location_facility: Unidad de Lesionados Medulares Hospital Traumatología

location_facility: Montecatone Rehabilitation Institute, Università di Bologna

location_facility: Neurologiska kliniken, Avd R18 Karolinska Universitetssjukhuset, Solna

location_facility: Complejo Hospitalario Universitario A Coruña

location_facility: Gastrointestinal Physiology Unit, University College London Hospital

location_facility: Azienda Ospedaliera- Universitaria Careggi


title: Diagnostic Accuracy of On-line Quantitative Flow Ratio. Functional Assessment by Virtual Online Reconstruction (The FAVOR II Europe-Japan Study)

acronym: FAVOR II EJ

summary: Quantitative Flow Ratio (QFR) is a novel method for evaluating the functional significance of coronary stenosis. QFR is assessed by calculation of the pressure in the vessel based on two angiographic projections. The purpose of the FAVOR II study is to evaluate the diagnostic accuracy of on-line QFR compared to 2D Quantitative Coronary Angiography (QCA) with FFR as gold standard.

ReferenceCitation: Tu S, Westra J, Yang J, von Birgelen C, Ferrara A, Pellicano M, Nef H, Tebaldi M, Murasato Y, Lansky A, Barbato E, van der Heijden LC, Reiber JHC, Holm NR, Wijns W; FAVOR Pilot Trial Study Group. Diagnostic Accuracy of Fast Computational Approaches to Derive Fractional Flow Reserve From Diagnostic Coronary Angiography: The International Multicenter FAVOR Pilot Study. JACC Cardiovasc Interv. 2016 Oct 10;9(19):2024-2035. doi: 10.1016/j.jcin.2016.07.013.

ReferenceCitation: Westra J, Andersen BK, Campo G, Matsuo H, Koltowski L, Eftekhari A, Liu T, Di Serafino L, Di Girolamo D, Escaned J, Nef H, Naber C, Barbierato M, Tu S, Neghabat O, Madsen M, Tebaldi M, Tanigaki T, Kochman J, Somi S, Esposito G, Mercone G, Mejia-Renteria H, Ronco F, Bøtker HE, Wijns W, Christiansen EH, Holm NR. Diagnostic Performance of In-Procedure Angiography-Derived Quantitative Flow Reserve Compared to Pressure-Derived Fractional Flow Reserve: The FAVOR II Europe-Japan Study. J Am Heart Assoc. 2018 Jul 6;7(14). pii: e009603. doi: 10.1161/JAHA.118.009603.

lead_sponsor: Aarhus University Hospital Skejby

location_country:
Denmark
United Kingdom
Italy
Netherlands
France
Japan
Germany

location_facility: Azienda ospedaliera Sant'Anna e S. sebastiano di Caserta

location_facility: Institut Cardiovasculaire Paris Sud Massy

location_facility: Azienda Ospedaliero-Universitaria di Ferrara, University of Ferrara


title: A Phase 3 Open-label Safety Study of Elvitegravir/Cobicistat/Emtricitabine/Tenofovir Alafenamide Single-Tablet Regimen in HIV-1 Positive Patients With Mild to Moderate Renal Impairment

summary: The primary objective of this study is to evaluate the effect of elvitegravir/cobicistat/emtricitabine/tenofovir alafenamide (E/C/F/TAF) fixed-dose combination (FDC) tablet on renal parameters at Week 24 in treatment-naive and treatment-experienced HIV-positive, adults with mild to moderate renal impairment.

ReferenceCitation: Pozniak A, Arribas JR, Gathe J, Gupta SK, Post FA, Bloch M, Avihingsanon A, Crofoot G, Benson P, Lichtenstein K, Ramgopal M, Chetchotisakd P, Custodio JM, Abram ME, Wei X, Cheng A, McCallister S, SenGupta D, Fordyce MW; GS-US-292-0112 Study Team. Switching to Tenofovir Alafenamide, Coformulated With Elvitegravir, Cobicistat, and Emtricitabine, in HIV-Infected Patients With Renal Impairment: 48-Week Results From a Single-Arm, Multicenter, Open-Label Phase 3 Study. J Acquir Immune Defic Syndr. 2016 Apr 15;71(5):530-7. doi: 10.1097/QAI.0000000000000908.

ReferenceCitation: Post FA, Tebas P, Clarke A, Cotte L, Short WR, Abram ME, Jiang S, Cheng A, Das M, Fordyce MW. Brief Report: Switching to Tenofovir Alafenamide, Coformulated With Elvitegravir, Cobicistat, and Emtricitabine, in HIV-Infected Adults With Renal Impairment: 96-Week Results From a Single-Arm, Multicenter, Open-Label Phase 3 Study. J Acquir Immune Defic Syndr. 2017 Feb 1;74(2):180-184. doi: 10.1097/QAI.0000000000001186.

ipd_sharing_description: Qualified external researchers may request IPD for this study after study completion. For more information, please visit our website at https://www.gilead.com/science-and-medicine/research/clinical-trials-transparency-and-data-sharing-policy.

lead_sponsor: Gilead Sciences

location_country:
Spain
Dominican Republic
United Kingdom
Australia
Thailand
United States
France
Mexico
Netherlands

location_facility: Instituto Dominicano de Estudios Virologicos (IDEV)

location_facility: Community Research Initiative of New England

location_facility: Infectious Disease Specialists of Atlanta

location_facility: University of PA HIV Clinical Trials Unit

location_facility: GHPS Service des maladies infectieuses et tropicales pavillon Laveran unité de recherche clinique

location_facility: Chelsea and Westminster NHS Foundation Trust Hospital

location_facility: Germans Trias i Pujol University Hospital

location_facility: HIV-NAT, Thai Red Cross AIDS Research Centre

location_facility: Clinical Research Infectious Diseases Department- Alfred Hospital

location_facility: Srinagarind Hospital, Khon Kaen University

location_facility: North Shore University Hospital/Division of Infectious Diseases

location_facility: Department of Preventive and Social Medicine, Faculty of Medicine, Siriraj Hospital

location_facility: Maricopa Integrated Health System - McDowell Clinic

location_facility: Central Manchester University Hospitals NHS foundation Trust

location_facility: Faculty of Medicine Ramathibodi Hospital, Mahidol University

location_facility: LA Gay & Lesbian Center - Jeffrey Goodman Special Care Clinic

location_facility: Hospital Civil de Guadalajara Dr. Juan I. Menchaca

location_facility: Brighton & Sussex University Hospitals NHS Trust

location_facility: The Kansas City Care Clinic (KC Free Health Clinic)

location_facility: North Texas Infectious Diseases Consultants, PA

location_facility: Long Beach Education and Research Consultants

location_facility: Desert Medical Group Inc. dba Desert Oasis Healthcare Medical Group


title: Evaluation of Virtual Touch Tissue Imaging Quantification (VTIQ - 2D-SWE) in the Assessment of BI-RADS® 3 and 4 Lesions: Can Patient Selection for Biopsy be Improved? - A Confirmatory Multi-Center-Study

summary: The primary aim of this study is to evaluate if VTIQ in addition to BI-RADS® categorization can improve the diagnostic accuracy with respect to detection of malignancies, in particular for BI-RADS® categories 3 and 4a. The idea of the study is to restage all patients in categories 3 and 4a according to a predefined VTIQ cut-off value of ≥ 3.5 m/s (37 kPa).

ReferenceCitation: Barr RG, Zhang Z. Shear-wave elastography of the breast: value of a quality measure and comparison with strain elastography. Radiology. 2015 Apr;275(1):45-53. doi: 10.1148/radiol.14132404. Epub 2014 Nov 24.

ReferenceCitation: Barr RG, Zhang Z. Effects of precompression on elasticity imaging of the breast: development of a clinically useful semiquantitative method of precompression assessment. J Ultrasound Med. 2012 Jun;31(6):895-902.

ReferenceCitation: Golatta M, Schweitzer-Martin M, Harcos A, Schott S, Junkermann H, Rauch G, Sohn C, Heil J. Normal breast tissue stiffness measured by a new ultrasound technique: virtual touch tissue imaging quantification (VTIQ). Eur J Radiol. 2013 Nov;82(11):e676-9. doi: 10.1016/j.ejrad.2013.06.029. Epub 2013 Aug 8.

ReferenceCitation: Golatta M, Schweitzer-Martin M, Harcos A, Schott S, Gomez C, Stieber A, Rauch G, Domschke C, Rom J, Schütz F, Sohn C, Heil J. Evaluation of virtual touch tissue imaging quantification, a new shear wave velocity imaging method, for breast lesion assessment by ultrasound. Biomed Res Int. 2014;2014:960262. doi: 10.1155/2014/960262. Epub 2014 Mar 31.

ReferenceCitation: Tozaki M, Saito M, Benson J, Fan L, Isobe S. Shear wave velocity measurements for differential diagnosis of solid breast masses: a comparison between virtual touch quantification and virtual touch IQ. Ultrasound Med Biol. 2013 Dec;39(12):2233-45. doi: 10.1016/j.ultrasmedbio.2013.07.012. Epub 2013 Sep 21.

ReferenceCitation: Barr RG, Nakashima K, Amy D, Cosgrove D, Farrokh A, Schafer F, Bamber JC, Castera L, Choi BI, Chou YH, Dietrich CF, Ding H, Ferraioli G, Filice C, Friedrich-Rust M, Hall TJ, Nightingale KR, Palmeri ML, Shiina T, Suzuki S, Sporea I, Wilson S, Kudo M. WFUMB guidelines and recommendations for clinical use of ultrasound elastography: Part 2: breast. Ultrasound Med Biol. 2015 May;41(5):1148-60. doi: 10.1016/j.ultrasmedbio.2015.03.008. Epub 2015 Mar 18. Review.

ReferenceCitation: Cosgrove DO, Berg WA, Doré CJ, Skyba DM, Henry JP, Gay J, Cohen-Bacrie C; BE1 Study Group. Shear wave elastography for breast masses is highly reproducible. Eur Radiol. 2012 May;22(5):1023-32. doi: 10.1007/s00330-011-2340-y. Epub 2011 Dec 31.

ReferenceCitation: Balleyguier C, Canale S, Ben Hassen W, Vielh P, Bayou EH, Mathieu MC, Uzan C, Bourgier C, Dromain C. Breast elasticity: principles, technique, results: an update and overview of commercially available software. Eur J Radiol. 2013 Mar;82(3):427-34. doi: 10.1016/j.ejrad.2012.03.001. Epub 2012 Mar 24. Review.

ReferenceCitation: Barr RG, Destounis S, Lackey LB 2nd, Svensson WE, Balleyguier C, Smith C. Evaluation of breast lesions using sonographic elasticity imaging: a multicenter trial. J Ultrasound Med. 2012 Feb;31(2):281-7.

ReferenceCitation: Barr RG. Sonographic breast elastography: a primer. J Ultrasound Med. 2012 May;31(5):773-83. Review.

ReferenceCitation: Evans A, Whelehan P, Thomson K, Brauer K, Jordan L, Purdie C, McLean D, Baker L, Vinnicombe S, Thompson A. Differentiating benign from malignant solid breast masses: value of shear wave elastography according to lesion stiffness combined with greyscale ultrasound according to BI-RADS classification. Br J Cancer. 2012 Jul 10;107(2):224-9. doi: 10.1038/bjc.2012.253. Epub 2012 Jun 12.

ReferenceCitation: Golatta M, Pfob A, Büsch C, Bruckner T, Alwafai Z, Balleyguier C, Clevert DA, Duda V, Goncalo M, Gruber I, Hahn M, Kapetas P, Ohlinger R, Rutten M, Togawa R, Tozaki M, Wojcinski S, Rauch G, Heil J, Barr RG. The potential of combined shear wave and strain elastography to reduce unnecessary biopsies in breast cancer diagnostics - An international, multicentre trial. Eur J Cancer. 2022 Jan;161:1-9. doi: 10.1016/j.ejca.2021.11.005. Epub 2021 Dec 5.

lead_sponsor: Heidelberg University

location_country:
Portugal
Netherlands
United States
France
Japan
Germany

location_facility: Universitätsklinikum Marburg, Klinik für Gynäkologie, gyn. Endokrinologie und Onkologie Senologische Diagnostik & Gynäkologischer Ultraschall

location_facility: Universitätsmedizin Greifswald, Klinik für Frauenheilkunde und Geburtshilfe

location_facility: Centro Hospitalar e Universitário de Coimbra, Departamento de Radiologia

location_facility: Institut Gustave Roussy, Service de Radiologie, Villejuif Cedex


title: A Double-blind, Randomized, Placebo and Ezetimibe-controlled, Multicenter Study to Evaluate Safety and Efficacy of Lipid Lowering Monotherapy With AMG 145 in Subjects With a 10-Year Framingham Risk Score of 10% or Less

summary: The primary objective was to evaluate the effect of 12 weeks of evolocumab subcutaneous (SC) monotherapy every 2 weeks (Q2W) and monthly (QM), compared with placebo and ezetimibe, on percent change from baseline in low-density lipoprotein cholesterol (LDL-C) in adults with a 10-year Framingham risk score of 10% or less.

ReferenceCitation: Koren MJ, Lundqvist P, Bolognese M, Neutel JM, Monsalvo ML, Yang J, Kim JB, Scott R, Wasserman SM, Bays H; MENDEL-2 Investigators. Anti-PCSK9 monotherapy for hypercholesterolemia: the MENDEL-2 randomized, controlled phase III clinical trial of evolocumab. J Am Coll Cardiol. 2014 Jun 17;63(23):2531-2540. doi: 10.1016/j.jacc.2014.03.018. Epub 2014 Mar 29.

ReferenceCitation: Daviglus ML, Ferdinand KC, López JAG, Wu Y, Monsalvo ML, Rodriguez CJ. Effects of Evolocumab on Low-Density Lipoprotein Cholesterol, Non-High Density Lipoprotein Cholesterol, Apolipoprotein B, and Lipoprotein(a) by Race and Ethnicity: A Meta-Analysis of Individual Participant Data From Double-Blind and Open-Label Extension Studies. J Am Heart Assoc. 2021 Jan 5;10(1):e016839. doi: 10.1161/JAHA.120.016839. Epub 2020 Dec 16.

ReferenceCitation: Koren MJ, Jones PH, Robinson JG, Sullivan D, Cho L, Hucko T, Lopez JAG, Fleishman AN, Somaratne R, Stroes E. A Comparison of Ezetimibe and Evolocumab for Atherogenic Lipid Reduction in Four Patient Populations: A Pooled Efficacy and Safety Analysis of Three Phase 3 Studies. Cardiol Ther. 2020 Dec;9(2):447-465. doi: 10.1007/s40119-020-00181-8. Epub 2020 Jun 20.

ReferenceCitation: Kuchimanchi M, Grover A, Emery MG, Somaratne R, Wasserman SM, Gibbs JP, Doshi S. Population pharmacokinetics and exposure-response modeling and simulation for evolocumab in healthy volunteers and patients with hypercholesterolemia. J Pharmacokinet Pharmacodyn. 2018 Jun;45(3):505-522. doi: 10.1007/s10928-018-9592-y. Epub 2018 May 7.

ReferenceCitation: Kasichayanula S, Grover A, Emery MG, Gibbs MA, Somaratne R, Wasserman SM, Gibbs JP. Clinical Pharmacokinetics and Pharmacodynamics of Evolocumab, a PCSK9 Inhibitor. Clin Pharmacokinet. 2018 Jul;57(7):769-779. doi: 10.1007/s40262-017-0620-7. Review.

ReferenceCitation: Shapiro MD, Minnier J, Tavori H, Kassahun H, Flower A, Somaratne R, Fazio S. Relationship Between Low-Density Lipoprotein Cholesterol and Lipoprotein(a) Lowering in Response to PCSK9 Inhibition With Evolocumab. J Am Heart Assoc. 2019 Feb 19;8(4):e010932. doi: 10.1161/JAHA.118.010932.

ReferenceCitation: Stroes E, Robinson JG, Raal FJ, Dufour R, Sullivan D, Kassahun H, Ma Y, Wasserman SM, Koren MJ. Consistent LDL-C response with evolocumab among patient subgroups in PROFICIO: A pooled analysis of 3146 patients from phase 3 studies. Clin Cardiol. 2018 Oct;41(10):1328-1335. doi: 10.1002/clc.23049. Epub 2018 Oct 21.

ReferenceCitation: Toth PP, Descamps O, Genest J, Sattar N, Preiss D, Dent R, Djedjos C, Wu Y, Geller M, Uhart M, Somaratne R, Wasserman SM; PROFICIO Investigators. Pooled Safety Analysis of Evolocumab in Over 6000 Patients From Double-Blind and Open-Label Extension Studies. Circulation. 2017 May 9;135(19):1819-1831. doi: 10.1161/CIRCULATIONAHA.116.025233. Epub 2017 Mar 1.

ReferenceCitation: Toth PP, Jones SR, Monsalvo ML, Elliott-Davey M, López JAG, Banach M. Effect of Evolocumab on Non-High-Density Lipoprotein Cholesterol, Apolipoprotein B, and Lipoprotein(a): A Pooled Analysis of Phase 2 and Phase 3 Studies. J Am Heart Assoc. 2020 Mar 3;9(5):e014129. doi: 10.1161/JAHA.119.014129. Epub 2020 Mar 2.

ReferenceCitation: Wasserman SM, Sabatine MS, Koren MJ, Giugliano RP, Legg JC, Emery MG, Doshi S, Liu T, Somaratne R, Gibbs JP. Comparison of LDL-C Reduction Using Different Evolocumab Doses and Intervals: Biological Insights and Treatment Implications. J Cardiovasc Pharmacol Ther. 2018 Sep;23(5):423-432. doi: 10.1177/1074248418774043. Epub 2018 May 16.

ReferenceCitation: May HT, Muhlestein JB, Ma Y, López JAG, Coll B, Nelson J. Effects of Evolocumab on the ApoA1 Remnant Ratio: A Pooled Analysis of Phase 3 Studies. Cardiol Ther. 2019 Jun;8(1):91-102. doi: 10.1007/s40119-019-0133-6. Epub 2019 Mar 9.

lead_sponsor: Amgen

location_country:
Denmark
Taiwan
Australia
Canada
Korea, Republic of
United States
France
South Africa
Turkey
Belgium


acronym: ARISE

title: A Multicenter, Randomized, Placebo-controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency

lead_sponsor: Alexion Pharmaceuticals

ReferenceCitation: Wilson DP, Friedman M, Marulkar S, Hamby T, Bruckert E. Sebelipase alfa improves atherogenic biomarkers in adults and children with lysosomal acid lipase deficiency. J Clin Lipidol. 2018 May - Jun;12(3):604-614. doi: 10.1016/j.jacl.2018.02.020. Epub 2018 Mar 9.

ReferenceCitation: Burton BK, Balwani M, Feillet F, Barić I, Burrow TA, Camarena Grande C, Coker M, Consuelo-Sánchez A, Deegan P, Di Rocco M, Enns GM, Erbe R, Ezgu F, Ficicioglu C, Furuya KN, Kane J, Laukaitis C, Mengel E, Neilan EG, Nightingale S, Peters H, Scarpa M, Schwab KO, Smolka V, Valayannopoulos V, Wood M, Goodman Z, Yang Y, Eckert S, Rojas-Caro S, Quinn AG. A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency. N Engl J Med. 2015 Sep 10;373(11):1010-20. doi: 10.1056/NEJMoa1501365.

ReferenceCitation: Burton BK, Feillet F, Furuya KN, Marulkar S, Balwani M. Sebelipase alfa in children and adults with lysosomal acid lipase deficiency: Final results of the ARISE study. J Hepatol. 2022 Mar;76(3):577-587. doi: 10.1016/j.jhep.2021.10.026. Epub 2021 Nov 10.

ReferenceCitation: Valayannopoulos V, Malinova V, Honzík T, Balwani M, Breen C, Deegan PB, Enns GM, Jones SA, Kane JP, Stock EO, Tripuraneni R, Eckert S, Schneider E, Hamilton G, Middleton MS, Sirlin C, Kessler B, Bourdon C, Boyadjiev SA, Sharma R, Twelves C, Whitley CB, Quinn AG. Sebelipase alfa over 52 weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency. J Hepatol. 2014 Nov;61(5):1135-42. doi: 10.1016/j.jhep.2014.06.022. Epub 2014 Jun 30.

location_country:
Croatia
Poland
Spain
Italy
Russian Federation
Australia
United Kingdom
Argentina
United States
France
Turkey
Mexico
Japan
Germany
Czechia

summary:
This Phase 3 study evaluated the efficacy and safety of 1 milligram/kilogram (mg/kg) intravenous (IV) infusions of SBC-102 (sebelipase alfa) administered every other week (qow) in participants with late onset lysosomal acid lipase deficiency (LAL-D) (cholesteryl ester storage disease [CESD]).

Late-onset LAL-D is an underappreciated cause of cirrhosis, liver failure and dyslipidemia. There is currently no standard treatment for LAL-D other than supportive care. Enzyme replacement therapy may be a potential new treatment option for LAL-D participants.


acronym: Not applicable

title: Pharmacoepidemiology Study to Define the Long-term Safety Profile of Tenofovir Disoproxil Fumarate (Tenofovir DF, Viread®) and Describe the Management of Tenofovir DF-associated Renal and Bone Toxicity in Chronic Hepatitis B (CHB)-Infected Adolescents Aged 12 to <18 Years in Europe

location_country:
Belgium
Spain
Italy
Greece
United Kingdom
France
Romania

location_facility: Spitatul Clinic de Boli Infectioase Constanta

location_facility: Grigore Alexandrescu Childrens Emergency Clinical Hospital

location_facility: Cliniques Universitaries Saint- Luc, Departem Pediatrie

location_facility: Institutul de Gastrenterologie si Hepatologie Iasi

location_facility: Hospital Universitari Germans Trias i Pujol

location_facility: Azienda Ospedaliero-Universitaria di Bologna

location_facility: Birmingham Children's Hospital NHS Trust

summary: The primary objective of this study is to characterize the long term (ie, 96 weeks of follow up) bone safety profile of open-label tenofovir disoproxil fumarate (tenofovir DF) treatment in CHB-infected adolescents. This includes prospectively evaluating and comparing the bone mineral density (BMD) change between CHB-infected adolescents 12 to < 18 years of age treated with tenofovir DF in European treatment centers who are assigned to one of two schedules for renal and bone laboratory monitoring and BMD measurement.