title: A Phase II, Single Arm, Multicenter Trial to Determine the Efficacy and Safety of CTL019 in Adult Patients With Relapsed or Refractory Diffuse Large B-cell Lymphoma (DLBCL)

acronym: JULIET

summary: This is a multi-center, phase II study to determine the efficacy and safety of CTL019 in adult patients with relapsed or refractory DLBCL.

ReferenceCitation: Schuster SJ, Tam CS, Borchmann P, Worel N, McGuirk JP, Holte H, Waller EK, Jaglowski S, Bishop MR, Damon LE, Foley SR, Westin JR, Fleury I, Ho PJ, Mielke S, Teshima T, Janakiram M, Hsu JM, Izutsu K, Kersten MJ, Ghosh M, Wagner-Johnston N, Kato K, Corradini P, Martinez-Prieto M, Han X, Tiwari R, Salles G, Maziarz RT. Long-term clinical outcomes of tisagenlecleucel in patients with relapsed or refractory aggressive B-cell lymphomas (JULIET): a multicentre, open-label, single-arm, phase 2 study. Lancet Oncol. 2021 Oct;22(10):1403-1415. doi: 10.1016/S1470-2045(21)00375-2. Epub 2021 Sep 10.

family: Schuster

full_name: Stephen J Schuster

given: Stephen J

family: Tam

full_name: Constantine S Tam

given: Constantine S

family: Borchmann

full_name: Peter Borchmann

given: Peter

family: Worel

full_name: Nina Worel

given: Nina

family: McGuirk

full_name: Joseph P McGuirk

given: Joseph P

family: Holte

full_name: Harald Holte

given: Harald

family: Waller

full_name: Edmund K Waller

given: Edmund K

family: Jaglowski

full_name: Samantha Jaglowski

given: Samantha

family: Bishop

full_name: Michael R Bishop

given: Michael R

family: Damon

full_name: Lloyd E Damon

given: Lloyd E

family: Foley

full_name: Stephen Ronan Foley

given: Stephen Ronan

family: Westin

full_name: Jason R Westin

given: Jason R

family: Fleury

full_name: Isabelle Fleury

given: Isabelle

family: Ho

full_name: P Joy Ho

given: P Joy

family: Mielke

full_name: Stephan Mielke

given: Stephan

family: Teshima

full_name: Takanori Teshima

given: Takanori

family: Janakiram

full_name: Murali Janakiram

given: Murali

family: Hsu

full_name: Jing-Mei Hsu

given: Jing-Mei

family: Izutsu

full_name: Koji Izutsu

given: Koji

family: Kersten

full_name: Marie José Kersten

given: Marie José

family: Ghosh

full_name: Monalisa Ghosh

given: Monalisa

family: Wagner-Johnston

full_name: Nina Wagner-Johnston

family: Kato

full_name: Koji Kato

ReferenceCitation: Ernst M, Oeser A, Besiroglu B, Caro-Valenzuela J, Abd El Aziz M, Monsef I, Borchmann P, Estcourt LJ, Skoetz N, Goldkuhle M. Chimeric antigen receptor (CAR) T-cell therapy for people with relapsed or refractory diffuse large B-cell lymphoma. Cochrane Database Syst Rev. 2021 Sep 13;9:CD013365. doi: 10.1002/14651858.CD013365.pub2. Review.

name: Cochrane Haematology, Department I of Internal Medicine, Center for Integrated Oncology Aachen Bonn Cologne Duesseldorf; Faculty of Medicine and University Hospital Cologne, University of Cologne; Cologne Germany

name: Department of Surgery; Mayo Clinic; Rochester Minnesota USA

name: Department I of Internal Medicine; University Hospital of Cologne; Cologne Germany

name: Haematology/Transfusion Medicine; NHS Blood and Transplant; Oxford UK

name: Cochrane Cancer, Department I of Internal Medicine, Center for Integrated Oncology Aachen Bonn Cologne Duesseldorf; Faculty of Medicine and University Hospital Cologne, University of Cologne; Cologne Germany

ReferenceCitation: Westin JR, Kersten MJ, Salles G, Abramson JS, Schuster SJ, Locke FL, Andreadis C. Efficacy and safety of CD19-directed CAR-T cell therapies in patients with relapsed/refractory aggressive B-cell lymphomas: Observations from the JULIET, ZUMA-1, and TRANSCEND trials. Am J Hematol. 2021 Oct 1;96(10):1295-1312. doi: 10.1002/ajh.26301. Epub 2021 Aug 13. Review.

name: Department of Lymphoma and Myeloma The University of Texas MD Anderson Cancer Center  Houston Texas USA

name: Department of Hematology Amsterdam UMC, University of Amsterdam, LYMMCARE (Lymphoma and Myeloma Center Amsterdam)  Amsterdam The Netherlands

name: Memorial Sloan Kettering Cancer Center  New York New York USA

name: Center for Lymphoma Massachusetts General Hospital Cancer Center  Boston Massachusetts USA

name: Lymphoma Program Abramson Cancer Center, University of Pennsylvania  Philadelphia Pennsylvania USA

name: Department of Blood and Marrow Transplant and Cellular Immunotherapy Moffitt Cancer Center  Tampa Florida USA

name: Helen Diller Family Comprehensive Cancer Center University of California San Francisco  San Francisco California USA

title: Efficacy and safety of CD19 ‐directed CAR‐T cell therapies in patients with relapsed/refractory aggressive B‐cell lymphomas: Observations from the JULIET , ZUMA ‐1, and TRANSCEND trials

ReferenceCitation: Schuster SJ, Maziarz RT, Rusch ES, Li J, Signorovitch JE, Romanov VV, Locke FL, Maloney DG. Grading and management of cytokine release syndrome in patients treated with tisagenlecleucel in the JULIET trial. Blood Adv. 2020 Apr 14;4(7):1432-1439. doi: 10.1182/bloodadvances.2019001304.

name: Lymphoma Program, Abramson Cancer Center, University of Pennsylvania, Philadelphia, PA;

name: Center for Hematologic Malignancies, Oregon Health & Science Knight Cancer Institute, Portland, OR;

name: Novartis Pharmaceuticals Corporation, East Hanover, NJ;

name: H. Lee Moffitt Cancer Center, Tampa, FL; and

name: Fred Hutchinson Cancer Research Center, Seattle, WA

ReferenceCitation: Maziarz RT, Schuster SJ, Romanov VV, Rusch ES, Li J, Signorovitch JE, Maloney DG, Locke FL. Grading of neurological toxicity in patients treated with tisagenlecleucel in the JULIET trial. Blood Adv. 2020 Apr 14;4(7):1440-1447. doi: 10.1182/bloodadvances.2019001305.

name: Health Economics and Outcomes Research, Analysis Group, Inc., Boston, MA;

name: Fred Hutchinson Cancer Research Center, Seattle, WA; and

name: Department of Blood and Marrow Transplant and Cellular Immunotherapy, H. Lee Moffitt Cancer Center, Tampa, FL

ReferenceCitation: Maziarz RT, Waller EK, Jaeger U, Fleury I, McGuirk J, Holte H, Jaglowski S, Schuster SJ, Bishop MR, Westin JR, Mielke S, Teshima T, Bachanova V, Foley SR, Borchmann P, Salles GA, Zhang J, Tiwari R, Pacaud LB, Ma Q, Tam CS. Patient-reported long-term quality of life after tisagenlecleucel in relapsed/refractory diffuse large B-cell lymphoma. Blood Adv. 2020 Feb 25;4(4):629-637. doi: 10.1182/bloodadvances.2019001026.

name: Center for Hematologic Malignancies, Oregon Health and Science University (OHSU) Knight Cancer Institute, Portland, OR;

name: Bone Marrow and Stem Cell Transplant Center, Winship Cancer Institute of Emory University, Atlanta, GA;

name: Division of Hematology and Hemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria;

name: Maisonneuve-Rosemont Hospital, University of Montreal, Montreal, QC, Canada;

name: Department of Blood and Bone Marrow Transplant, The University of Kansas Cancer Center, Kansas City, KS;

name: Department of Oncology, Oslo University Hospital, Oslo, Norway;

name: K.G. Jebsen Centre for B cell Malignancies, Oslo, Norway;

name: Abramson Cancer Center, University of Pennsylvania, Philadelphia, PA;

name: Section of Hematology/Oncology, Department of Medicine and Comprehensive Cancer Center, University of Chicago, Chicago, IL;

name: Department of Lymphoma and Myeloma, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX;

name: Department of Cellular Therapy and Allogeneic Stem Cell Transplantation (CAST), Karolinska Institutet, Stockholm, Sweden;

name: Department of Laboratory Medicine, Karolinska University Hospital, Stockholm, Sweden;

name: Center for Allogeneic Stem Cell Transplantation, Department of Internal Medicine II, Würzburg University Medical Center, Würzburg, Germany;

name: Department of Hematology, Hokkaido University Hospital, Sapporo, Japan;

name: Division of Hematology, Oncology, and Transplantation, Department of Medicine, University of Minnesota, Minneapolis, MN;

name: Juravinski Hospital and Cancer Centre, McMaster University, Hamilton, ON, Canada;

name: Department of Haematology and Oncology, University Hospital of Cologne, Cologne, Germany;

name: Department of Hematology, Centre Hospitalier Lyon-Sud Hospital, Hospices Civils de Lyon, University of Lyon, Lyon, France;

name: Novartis Healthcare Pvt. Ltd, Hyderabad, India; and

name: Peter MacCallum Cancer Centre, St Vincent’s Hospital and University of Melbourne, Melbourne, VIC, Australia

repository_institution: University of Oslo - Duo Research Archive

repositories_institution: University of Oslo - Duo Research Archive

ReferenceCitation: Awasthi R, Pacaud L, Waldron E, Tam CS, Jäger U, Borchmann P, Jaglowski S, Foley SR, van Besien K, Wagner-Johnston ND, Kersten MJ, Schuster SJ, Salles G, Maziarz RT, Anak Ö, Del Corral C, Chu J, Gershgorin I, Pruteanu-Malinici I, Chakraborty A, Mueller KT, Waller EK. Tisagenlecleucel cellular kinetics, dose, and immunogenicity in relation to clinical factors in relapsed/refractory DLBCL. Blood Adv. 2020 Feb 11;4(3):560-572. doi: 10.1182/bloodadvances.2019000525.

name: Novartis Institutes for BioMedical Research, East Hanover, NJ;

name: Hematology Department, Peter MacCallum Cancer Center, University of Melbourne, Melbourne, Australia;

name: Department of Hematology and Oncology, University Hospital of Cologne, Cologne, Germany;

name: The James Cancer Hospital and Solove Research Institute, The Ohio State University Comprehensive Cancer Center, Columbus, OH;

name: Juravinski Hospital and Cancer Center, McMaster University, Hamilton, ON, Canada;

name: Division of Hematology and Oncology, Weill Cornell Medicine and New York-Presbyterian Hospital, New York, NY;

name: The Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, MD;

name: Department of Hematology, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands;

name: Department of Hematology, CHU Lyon-Sud Hospital, Hospices Civils de Lyon, Lyon, France;

name: Center for Hematologic Malignancies, Oregon Health & Science University Knight Cancer Institute, Portland, OR; and

ReferenceCitation: Bishop MR, Maziarz RT, Waller EK, Jäger U, Westin JR, McGuirk JP, Fleury I, Holte H, Borchmann P, Del Corral C, Tiwari R, Anak Ö, Awasthi R, Pacaud L, Romanov VV, Schuster SJ. Tisagenlecleucel in relapsed/refractory diffuse large B-cell lymphoma patients without measurable disease at infusion. Blood Adv. 2019 Jul 23;3(14):2230-2236. doi: 10.1182/bloodadvances.2019000151.

name: Hematopoietic Cellular Therapy Program, The University of Chicago Medicine, Chicago, IL;

name: Center for Hematologic Malignancies, Knight Cancer Institute, Oregon Health & Science University, Portland, OR;

name: Division of Hematology and Hemostaseology, Department of Internal Medicine I, Medical University of Vienna, Vienna, Austria;

name: Division of Cancer Medicine, Department of Lymphoma and Myeloma, University of Texas MD Anderson Cancer Center, Houston, TX;

name: Department of Blood and Bone Marrow Transplant, The University of Kansas Medical Center, Kansas City, KS;

name: Maisonneuve-Rosemont Hospital, Montreal, QC, Canada;

name: Hematology and Oncology, Department of Medicine, University of Montreal, Montreal, QC, Canada;

name: Novartis Healthcare Private Limited, Hyderabad, India;

name: Novartis Institutes for BioMedical Research, East Hanover, NJ; and

ReferenceCitation: Schuster SJ, Bishop MR, Tam CS, Waller EK, Borchmann P, McGuirk JP, Jäger U, Jaglowski S, Andreadis C, Westin JR, Fleury I, Bachanova V, Foley SR, Ho PJ, Mielke S, Magenau JM, Holte H, Pantano S, Pacaud LB, Awasthi R, Chu J, Anak Ö, Salles G, Maziarz RT; JULIET Investigators. Tisagenlecleucel in Adult Relapsed or Refractory Diffuse Large B-Cell Lymphoma. N Engl J Med. 2019 Jan 3;380(1):45-56. doi: 10.1056/NEJMoa1804980. Epub 2018 Dec 1.

name: From the Lymphoma Program, Abramson Cancer Center, University of Pennsylvania, Philadelphia (S.J.S.); the Hematopoietic Cellular Therapy Program, University of Chicago Medicine, Chicago (M.R.B.); Peter MacCallum Cancer Centre, St. Vincent’s Hospital and University of Melbourne, Melbourne, VIC (C.S.T.), and the Royal Prince Alfred Hospital and Department of Medicine, University of Sydney, Sydney (P.J.H.) — both in Australia; Winship Cancer Institute of Emory University, Bone Marrow and Stem Cell...

title: Tisagenlecleucel in Adult Relapsed or Refractory Diffuse Large B-Cell Lymphoma

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

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

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

location_facility: University of California San Francisco SC-4

location_facility: University of Chicago Medical Center, Hematology & Oncology

location_facility: The Ohio State University James Cancer Hospital &

location_facility: The Sidney Kimmel Cancer Center at Johns Hopkins Hospital SC-2

location_facility: Mayo Clinic - Arizona Mayo Clinic Building

location_facility: Emory University School of Medicine/Winship Cancer Institute


title: Global Clinical Study of Renal Denervation With the Symplicity Spyral™ Multi-electrode Renal Denervation System in Patients With Uncontrolled Hypertension on Standard Medical Therapy (SPYRAL HTN-ON MED)

summary: The purpose of this study is to test the hypothesis that renal denervation decreases blood pressure and is safe when studied in the presence of up to three standard antihypertensive medications.

study_start_date_type: Actual

status: Recruiting

study_completion_date_type: Anticipated

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Anticipated

ReferenceType: derived

ReferenceCitation: Kandzari DE, Hickey GL, Pocock SJ, Weber MA, Böhm M, Cohen SA, Fahy M, Lamberti G, Mahfoud F. Prioritised endpoints for device-based hypertension trials: the win ratio methodology. EuroIntervention. 2021 Apr 2;16(18):e1496-e1502. doi: 10.4244/EIJ-D-20-01090.

family: Kandzari

full_name: David E. Kandzari

given: David E.

sequence: first

family: Hickey

full_name: Graeme L. Hickey

given: Graeme L.

sequence: additional

family: Pocock

full_name: Stuart J. Pocock

given: Stuart J.

family: Weber

full_name: Michael A. Weber

given: Michael A.

family: Böhm

full_name: Michael Böhm

given: Michael

family: Cohen

full_name: Sidney A. Cohen

given: Sidney A.

family: Fahy

full_name: Martin Fahy

given: Martin

family: Lamberti

full_name: Giuseppina Lamberti

given: Giuseppina

family: Mahfoud

full_name: Felix Mahfoud

given: Felix

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: EuroIntervention

publisher: Europa Digital & Publishing

publisher_dissemination: Europa Digital & Publishing

publisher_group: Europa Digital & Publishing

publisher_normalized: Europa Digital & Publishing

ReferenceCitation: Kario K, Weber MA, Böhm M, Townsend RR, Mahfoud F, Schmieder RE, Tsioufis K, Cohen SA, Fahy M, Kandzari DE. Effect of renal denervation in attenuating the stress of morning surge in blood pressure: post-hoc analysis from the SPYRAL HTN-ON MED trial. Clin Res Cardiol. 2021 May;110(5):725-731. doi: 10.1007/s00392-020-01718-6. Epub 2020 Aug 1.

family: Kario

full_name: Kazuomi Kario

given: Kazuomi

family: Townsend

full_name: Raymond R. Townsend

given: Raymond R.

family: Schmieder

full_name: Roland E. Schmieder

given: Roland E.

family: Tsioufis

full_name: Konstantinos Tsioufis

given: Konstantinos

journal_name: Clinical Research in Cardiology

publisher: Springer Science and Business Media LLC

ReferenceCitation: Böhm M, Townsend RR, Kario K, Kandzari D, Mahfoud F, Weber MA, Schmieder RE, Tsioufis K, Hickey GL, Fahy M, DeBruin V, Brar S, Pocock S. Rationale and design of two randomized sham-controlled trials of catheter-based renal denervation in subjects with uncontrolled hypertension in the absence (SPYRAL HTN-OFF MED Pivotal) and presence (SPYRAL HTN-ON MED Expansion) of antihypertensive medications: a novel approach using Bayesian design. Clin Res Cardiol. 2020 Mar;109(3):289-302. doi: 10.1007/s00392-020-01595-z. Epub 2020 Feb 7. Erratum in: Clin Res Cardiol. 2020 May;109(5):653.

repository_institution: London School of Hygiene and Tropical Medicine - LSHTM Research Online

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

repository_institution: Saarland University and State Library - SciDok

ReferenceCitation: Kandzari DE, Böhm M, Mahfoud F, Townsend RR, Weber MA, Pocock S, Tsioufis K, Tousoulis D, Choi JW, East C, Brar S, Cohen SA, Fahy M, Pilcher G, Kario K; SPYRAL HTN-ON MED Trial Investigators. Effect of renal denervation on blood pressure in the presence of antihypertensive drugs: 6-month efficacy and safety results from the SPYRAL HTN-ON MED proof-of-concept randomised trial. Lancet. 2018 Jun 9;391(10137):2346-2355. doi: 10.1016/S0140-6736(18)30951-6. Epub 2018 May 23.

ReferenceCitation: Kandzari DE, Kario K, Mahfoud F, Cohen SA, Pilcher G, Pocock S, Townsend R, Weber MA, Böhm M. The SPYRAL HTN Global Clinical Trial Program: Rationale and design for studies of renal denervation in the absence (SPYRAL HTN OFF-MED) and presence (SPYRAL HTN ON-MED) of antihypertensive medications. Am Heart J. 2016 Jan;171(1):82-91. doi: 10.1016/j.ahj.2015.08.021. Epub 2015 Sep 11. Review.

location_country:
Austria
United States
Germany
France
Japan
Greece
Ireland
Australia
Canada
United Kingdom

location_facility: Hospital of the University of Pennsylvania

location_facility: University General Hospital of Thessaloniki (AHEPA)

location_facility: Universitäts-Herzzentrum Freiburg - Bad Krozingen GmbH

location_facility: Oregon Health & Science University Hospital

location_facility: Royal Devon & Exeter NHS Foundation Trust

location_facility: University Hospitals Cleveland Medical Center

location_facility: Cardiff and Vale University Health Board - University Hospital of Wales

location_facility: Weill Cornell Medical College/The New York Presbyterian Hospital


title: A Phase 3, Randomized, Double-Blind, Ivacaftor-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation and a Second CFTR Allele With a Gating Defect That Is Clinically Demonstrated to be Ivacaftor Responsive

summary: This is a Phase 3, randomized, double-blind, ivacaftor-controlled, parallel-group, multicenter study of tezacaftor in combination with ivacaftor in subjects aged 12 years and older with CF who are heterozygous for the F508del-CFTR mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive.

status: Completed

study_completion_date_type: Actual

enrollment_type: Actual

ReferenceCitation: McKone EF, DiMango EA, Sutharsan S, Barto TL, Campbell D, Ahluwalia N, Higgins M, Owen CA, Tullis E. A phase 3, randomized, double-blind, parallel-group study to evaluate tezacaftor/ivacaftor in people with cystic fibrosis heterozygous for F508del-CFTR and a gating mutation. J Cyst Fibros. 2021 Mar;20(2):234-242. doi: 10.1016/j.jcf.2020.11.003. Epub 2020 Dec 16.

family: McKone

full_name: Edward F. McKone

given: Edward F.

family: DiMango

full_name: Emily A. DiMango

given: Emily A.

family: Sutharsan

full_name: Sivagurunathan Sutharsan

given: Sivagurunathan

family: Barto

full_name: Tara Lynn Barto

given: Tara Lynn

family: Campbell

full_name: Daniel Campbell

given: Daniel

family: Ahluwalia

full_name: Neil Ahluwalia

given: Neil

family: Higgins

full_name: Mark Higgins

given: Mark

family: Owen

full_name: Caroline A. Owen

given: Caroline A.

family: Tullis

full_name: Elizabeth Tullis

given: Elizabeth

journal_name: Journal of Cystic Fibrosis

oa_host_type: publisher

evidence: open (via crossref license)

host_type: publisher

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

version: publishedVersion

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

lead_sponsor: Vertex Pharmaceuticals Incorporated

location_country:
Italy
Austria
United States
Germany
France
Ireland
Australia
Belgium
Canada
United Kingdom

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Completed


type: EudraCT Number

title: A Phase Ib, Open-Label, Dose-Escalation Study of the Safety and Pharmacokinetics of MOXR0916 and Atezolizumab in Patients With Locally Advanced or Metastatic Solid Tumors

summary: This Phase Ib, open-label, dose-escalation study will evaluate the safety, tolerability, and pharmacokinetics of the combination of MOXR0916 and atezolizumab in participants with locally advanced, recurrent, or metastatic incurable solid malignancy that has progressed after available standard therapy; or for which standard therapy has proven to be ineffective or intolerable or is considered inappropriate; or for which a clinical trial of an investigational agent is a recognized standard of care. Participants will be enrolled in two stages: a dose-escalation stage and an expansion stage.

design_allocation: Non-Randomized

lead_sponsor: Genentech, Inc.

location_country:
United States
France
Belgium
Australia
Spain
Canada
Korea, Republic of

location_facility: Georgetown University Medical Center Lombardi Cancer Center

location_facility: HonorHealth Research Institute - Bisgrove

location_facility: University Of Chicago Medical Center; Section Of Hematology/Oncology

location_facility: Yonsei University Health System/Severance Hospital

location_facility: Hosp de Madrid Norte Sanchinarro; Centro Integral; Onco Clara Campal

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

location_facility: British Columbia Cancer Agency (Bcca) - Vancouver Cancer Centre

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

location_facility: Hospital Clinico Universitario de Valencia


lead_sponsor: Novartis Pharmaceuticals

location_country:
Greece
Belgium
Australia
Russian Federation
France
Japan
Thailand
Slovakia
Korea, Republic of
South Africa
Chile
Sweden
Bulgaria
Hungary
China
Portugal
Germany
Italy
Lebanon
Mexico
Israel
Spain
Turkey

source: euctr

title: Open Label, Multi-center, Phase IV Study of Ruxolitinib or Ruxolitinib and Panobinostat Combination, for Patients Who Have Completed Prior Global Novartis or Incyte Sponsored Studies

summary:
This roll-over protocol allows patients who are still receiving clinical benefit to continue to be treated from multiple protocols in one program spanning multiple indications during the completion of the parent study/(ies). The population for the roll-over study should be consistent with the population defined in the program parent study/(ies). The primary eligibility criteria for a patient to enter the roll-over protocol is the participation and completion of a Novartis GDD&GMA/Incyte study with ruxolitinib monotherapy or combination of ruxolitinib and panobinostat. Efficacy parameters would not be measured; however safety data and an evaluation of clinical benefit will be collected.

Patients who have completed a prior study with ruxolitinib monotherapy or combination of ruxolitinib and panobinostat and who are assessed by the Investigator to continue to benefit from ongoing treatment will be eligible.

status_simplified: Ongoing


title: Age-adjusted D-dimer Cutoff Levels to Rule Out Deep Vein Thrombosis: a Prospective Outcome Study

acronym: ADJUST-DVT

summary: Prospective validation of an age-adjusted D-dimer cut-off to rule out deep vein thrombosis (DVT)

status: Unknown status

primary_purpose: Diagnostic

lead_sponsor: University Hospital, Geneva

location_country: Canada France Switzerland

location_facility: Thunder Bay regional Health Sciences center

intervention_type: Other

lead_sponsor_type: academique

status_simplified: Unknown


title: A Phase 3b, Randomized, Double-Blind Switch Study to Evaluate the Safety and Efficacy of Emtricitabine/Rilpivirine/Tenofovir Alafenamide (FTC/RPV/TAF) Fixed Dose Combination (FDC) in HIV-1 Positive Subjects Who Are Virologically Suppressed on Emtricitabine/Rilpivirine/Tenofovir Disoproxil Fumarate (FTC/RPV/TDF)

summary: The primary objective of this study is to evaluate the noninferiority of switching to emtricitabine/rilpivirine /tenofovir alafenamide (FTC/RPV/TAF) fixed-dose combination (FDC) as compared to continuing FTC/RPV/tenofovir disoproxil fumarate (TDF) FDC (FTC/RPV/TDF) in virologically suppressed HIV-1 infected participants.

ReferenceType: result

ReferenceCitation: Hagins D, Orkin C, Daar ES, Mills A, Brinson C, DeJesus E, Post FA, Morales-Ramirez J, Thompson M, Osiyemi O, Rashbaum B, Stellbrink HJ, Martorell C, Liu H, Liu YP, Porter D, Collins SE, SenGupta D, Das M. Switching to coformulated rilpivirine (RPV), emtricitabine (FTC) and tenofovir alafenamide from either RPV, FTC and tenofovir disoproxil fumarate (TDF) or efavirenz, FTC and TDF: 96-week results from two randomized clinical trials. HIV Med. 2018 Nov;19(10):724-733. doi: 10.1111/hiv.12664. Epub 2018 Aug 12.

name: Georgia Department of Public Health; Chatham Care Center; Savannah GA USA

family: Hagins

full_name: D Hagins

given: D

name: Grahame Hayton Unit; Royal London Hospital and Queen Mary University; London UK

family: Orkin

full_name: C Orkin

given: C

name: Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center; Torrance CA USA

family: Daar

full_name: ES Daar

given: ES

name: Mills Clinical Research; Los Angeles CA USA

family: Mills

full_name: A Mills

given: A

name: Central Texas Clinical Research; Austin TX USA

family: Brinson

full_name: C Brinson

name: Orlando Immunology Center; Orlando FL USA

family: DeJesus

full_name: E DeJesus

given: E

family: Post

full_name: FA Post

given: FA

name: Clinical Research Puerto Rico Inc.; San Juan Puerto Rico

family: Morales-Ramirez

full_name: J Morales-Ramirez

given: J

name: AIDS Research Consortium of Atlanta; Atlanta GA USA

family: Thompson

full_name: M Thompson

given: M

name: Triple O Research Institute PA; West Palm Beach FL USA

family: Osiyemi

full_name: O Osiyemi

given: O

name: Capital Medical Associates; Washington DC USA

family: Rashbaum

full_name: B Rashbaum

given: B

family: Stellbrink

full_name: H-J Stellbrink

given: H-J

name: The Research Institute; Springfield MA USA

family: Martorell

full_name: C Martorell

family: Liu

full_name: H Liu

given: H

full_name: Y-P Liu

given: Y-P

family: Porter

full_name: D Porter

family: Collins

full_name: SE Collins

given: SE

family: SenGupta

full_name: D SenGupta

family: Das

full_name: M Das

journal_name: HIV Medicine

oa_colors: green hybrid

oa_host_type: publisher;repository

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

host_type: repository

ReferenceCitation: Mills A, Brinson C, Martorell C, Crofoot G, Daar E, Osiyemi O, et al. Switching to RPV/FTC/TAF from RPV/FTC/TDF or EFV/FTC/TDF: Week 96 Results. Conference on Retroviruses and Opportunistic Infections,Boston. March 4-7, 2018, Abstract 504.

ReferenceCitation: Arribas JR, Rockstroh J, Post, Yazdanpanah Y, Cavassini, DeJesus E, et al. Bone and renal safety of switching to rilpivirine/emtricitabine/tenofovir alafenamide (RPV/FTC/TAF) from single-tablet regimens (STRs) containing efavirenz/emtricitabine/tenofovir disoproxil fumarate (EFV/FTC/TDF) or rilpivirine/emtricitabine/tenofovir disoproxil fumarate (RPV/FTC/TDF): Week48 subgroup analysis in patients at risk of or with comorbidities. Abstract accepted for presentation atthe 16th European AIDS Conference, 2017 25-27 October Milan, Italy.

ReferenceCitation: Porter DP, Kulkarni R, Cao H, SenGupta D, White KL. No Emergent Resistance in HIV-1 Virologically-Suppressed Subjects Who Switched to RPV/FTC/TAF [Poster1381]. ID Week™ (Infectious Diseases Society of America) 2017 4-8 October; San Diego, CA.

ReferenceCitation: Wohl D, Kulkarni R, Garner W, White KL, Porter DL. Viral Blips Were Infrequent in HIV1-Infected Virologically-Suppressed Adults Treated with Tenofovir Alafenamide or Tenofovir DF Rilpivirine-Containing Regimens [Poster1384]. ID Week™ (Infectious Diseases Society of America) 2017 4-8 October; San Diego, CA.

ReferenceCitation: DeJesus E, Ramgopal M, Crofoot G, Ruane P, LaMarca A, Molina J-M, et al. Efficacy and Safety of Switching to RPV/FTC/TAF in Older Adults. 8th International Workshopon HIV and Aging 2017 2-3 October, New York, New York.

ReferenceCitation: Molina JM, DeJesus E, Rijnders B, Post FAV, B., Stoeckle M, Thalme A, et al. Efficacy and Odefsey® StudyGS-US-366-1216Final Synoptic Clinical Study Report Final CONFIDENTIAL Page4 30July2019 Safety of Switching From RPV/FTC/TDF or EFV/FTC/TDF to RPV/FTC/TAF in Black Adults [Presentation MOPEB0291]. 9th IAS Conference on HIV Science 2017 23-26 July Paris, France.

ReferenceCitation: Rockstroh J, Orkin C, Yazdanpanah Y, Di Perri GDS, P. E., Arribas JR, Brinkman K, et al. Switching From TDF to TAF Improves Bone and Renal Safety Independent of Age, Sex, Race, or 3rd Agent: Results From Pooled Analysis (N=3816) of Virologically Suppressed HIV-1 Infected Adults [Presentation MOPEB0289]. 9th IAS Conference on HIV Science;2017 23-26July Paris, France.

ReferenceCitation: Orkin C, DeJesus E, Ramgopal M, Crofoot G, Ruane P, LaMarca A, Mills A, Vandercam B, de Wet J, Rockstroh J, Lazzarin A, Rijnders B, Podzamczer D, Thalme A, Stoeckle M, Porter D, Liu HC, Cheng A, Quirk E, SenGupta D, Cao H. Switching from tenofovir disoproxil fumarate to tenofovir alafenamide coformulated with rilpivirine and emtricitabine in virally suppressed adults with HIV-1 infection: a randomised, double-blind, multicentre, phase 3b, non-inferiority study. Lancet HIV. 2017 May;4(5):e195-e204. doi: 10.1016/S2352-3018(17)30031-0. Epub 2017 Mar 2.

ReferenceCitation: Majeed SR, Shao Y, Garner W, Scott J, Pérez-Ruixo C, SenGupta D, et al. Evaluation of RPV/FTC/TAF Exposure-Efficacy and Exposure-Safety Relationships [Poster427]. Conference on Retroviruses and Opportunistic Infections (CROI) 2017 13-16 February; Seattle, WA.

ReferenceCitation: Hagins D, Mills A, Martorell C, Walmsley S, Gallant J, Tebas P, et al. Efficacy and Safety of Switching toRPV/FTC/TAF in Women [Abstract12]. 7th International Workshop on HIV & Women; 2017 11-12 February; Seattle, Washington.

ReferenceCitation: Orkin C, DeJesus E, Ramgopal M, Crofoot G, Ruane P, LaMarca A, et al. 48Week Results from two studies: Switching to RPV/FTC/TAF from EFV/FTC/TDF (Study1160) or RPV/FTC/TDF (Study1216) [Presentation]. HIV Glasgow; 2016 23-26 October; Glasgow, United Kingdom.

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

location_country:
Italy
United States
Sweden
Switzerland
Netherlands
Germany
France
Belgium
Puerto Rico
Spain
Canada
United Kingdom

location_facility: Los Angeles BioMedical Institute at Harbor-UCLA Medical Center

location_facility: Infectious Diseases Associates of NW Florida, P.A.

location_facility: Baystate Infectious Diseases Clinical Research

location_facility: Center for HIV and Hepatogastroenterology

location_facility: North Texas Infectious Diseases Consultants

location_facility: Midway Immunology & Research Center, LLC

location_facility: Hôpital Gui de Chauliac - Service Maladies Infectieuses et Tropicales

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

location_facility: Long Beach Education and Research Consultants

location_facility: Zentrum für Infektiologie Berlin Prenzlauer Berg GmbH (zibp)

location_facility: Carolinas Medical Center--Myers Park Infectious Disease Clinic

location_facility: AIDS Research & Treatment Center of the Treasure Coast

location_facility: University of North Carolina at Chapel Hill

location_facility: AIDS Arms, Inc./Trinity Health & Wellness Center

location_facility: La Playa Medical Group and Clinical Research

location_facility: Hospital Clínico Universitario San Carlos

location_facility: Hospital General Universitario de Alicante

location_facility: Clinical Alliance for Research & Education - Infectious Diseases, LLC (CARE-ID)

location_facility: World Health Clinicians' CIRCLE CARE Center

location_facility: The University of Alabama at Birmingham (UAB)

location_facility: Fondazione IRCCS San Raffaele del Monte Tabor

location_facility: The Hathersage Integrated Contraception, Sexual Health and HIV Service

location_facility: Trinity Health and Wellness Center/AIDS Arms, Inc.

location_facility: Infectious Disease Research Institute Inc.

location_facility: Infectious Disease Specialists of Atlanta

location_facility: St. Joseph's Comprehensive Research Institute


ReferenceType: background

ReferenceCitation: Warady BA, Ng E, Bloss L, Mo M, Schaefer F, Bacchetta J. Cinacalcet studies in pediatric subjects with secondary hyperparathyroidism receiving dialysis. Pediatr Nephrol. 2020 Sep;35(9):1679-1697. doi: 10.1007/s00467-020-04516-4. Epub 2020 May 4. Review.

lead_sponsor: Amgen

location_country:
Italy
United States
France
Czechia
Greece
Poland
Hungary
Belgium
Russian Federation
Germany
Ukraine

title: A Multicenter Single-arm Extension Study to Characterize the Long-term Safety of Cinacalcet Hydrochloride in the Treatment of Secondary Hyperparathyroidism in Pediatric Subjects With Chronic Kidney Disease on Dialysis

summary: The primary objective of this study was to characterize the long-term safety and tolerability of cinacalcet in pediatric patients with chronic kidney disease (CKD) receiving dialysis.


title: A Multi-Center, Randomized, Double-Blind, Placebo- Controlled Study to Investigate the Efficacy and Safety of 52 Weeks Treatment With QGE031 Subcutaneous (s.c). in Asthma Patients Not Adequately Controlled by Medium- or High-dose Inhaled Corticosteroid (ICS) Plus Long Acting β2-agonist (LABA) With or Without Oral Corticosteroid (OCS)

summary: This study planned to assess the effect on the reduction in rate of severe asthma exacerbations of different dose levels of QGE031 in asthma patients that are inadequately controlled with inhaled steroid plus beta-2 agonist medication with or without oral steroid. However, this study was terminated due to the efficacy results from an interim analysis (at the end of treatment epoch) of the Phase II study CQGE031B2201 (NCT01716754). Planned data analyses were not performed for this study due to the early termination and the very limited dataset (only 10 participants received study medication of the 440 participants planned).

status: Terminated

location_country:
Italy
United States
Netherlands
Germany
France
Japan
Slovakia
Hungary
Belgium
Canada


title: European Registry on the Management of Helicobacter Pylori Infection

summary:
The great diversity of regimens and treatment lines, the different efficacy of these, mostly due to the increase in bacterial antibiotic resistance and regional differences, requires a continuous critical analysis of clinical practice, evaluating systematically the efficacy and safety of the different regimens and the cost-effectiveness of the different diagnostic-therapeutic strategies. This will help in the design of an efficient and optimized treatment that will reduce number of re-treatments, diagnostic tests and the appearance of associated pathologies such as peptic ulcers, gastrointestinal bleeding and, probably, gastric cancers. Therefore, the evaluation of real clinical practice using non-interventionist registries will help to improve the design and organization of European Consensus on the management of H. pylori infection, which is the best way to establish healthcare efficiency.

Primary aim

To obtain a database registering systematically over a year a large and representative sample of routine clinical practice of European gastroenterologists in order to produce descriptive studies of the management of H. pylori infection.

Secondary aims

To evaluate H. pylori infection consensus and clinical guidelines implementation in different countries.
To perform studies focused on epidemiology, efficacy and safety of the commonly used treatments to eradicate H. pylori.
To evaluate accessibility to healthcare technologies and drugs used in the management of H. pylori infection.
To allow the development of partial and specific analysis by the participating researchers after approval by the Registry's Scientific Committee

Methodology Non-interventionist prospective multicentre international registry promoted by the European Helicobacter Study Group. A renowned gastroenterologist from each country was selected as Local Coordinator (30 countries). They will in turn select up to ten gastroenterologists per country that will register the routine clinical practice consultations they receive over 10 years in an electronic Case Report Form (e-CRF). Variables retrieved will include clinical, diagnostic, treatment, eradication confirmation and outcome data. The database will allow researchers to perform specific subanalysis after approval by the Scientific Committee of the study.

study_type: Observational

time_perspective: Prospective

ReferenceCitation: Arkkila PE, Seppälä K, Kosunen TU, Sipponen P, Mäkinen J, Rautelin H, Färkkilä M. Helicobacter pylori eradication as the sole treatment for gastric and duodenal ulcers. Eur J Gastroenterol Hepatol. 2005 Jan;17(1):93-101.

ReferenceCitation: Azevedo NF, Huntington J, Goodman KJ. The epidemiology of Helicobacter pylori and public health implications. Helicobacter. 2009 Sep;14 Suppl 1:1-7. doi: 10.1111/j.1523-5378.2009.00703.x. Review. Erratum in: Helicobacter. 2010 Feb;15(1):78.

ReferenceCitation: Broutet N, Tchamgoué S, Pereira E, Lamouliatte H, Salamon R, Mégraud F. Risk factors for failure of Helicobacter pylori therapy--results of an individual data analysis of 2751 patients. Aliment Pharmacol Ther. 2003 Jan;17(1):99-109.

ReferenceCitation: de Vries AC, van Grieken NC, Looman CW, Casparie MK, de Vries E, Meijer GA, Kuipers EJ. Gastric cancer risk in patients with premalignant gastric lesions: a nationwide cohort study in the Netherlands. Gastroenterology. 2008 Apr;134(4):945-52. doi: 10.1053/j.gastro.2008.01.071. Epub 2008 Jan 30.

ReferenceCitation: Ford A, Moayyedi P. How can the current strategies for Helicobacter pylori eradication therapy be improved? Can J Gastroenterol. 2003 Jun;17 Suppl B:36B-40B.

ReferenceCitation: Ford AC, Axon AT. Epidemiology of Helicobacter pylori infection and public health implications. Helicobacter. 2010 Sep;15 Suppl 1:1-6. doi: 10.1111/j.1523-5378.2010.00779.x. Review.

ReferenceCitation: Gisbert JP, Calvet X. Review article: the effectiveness of standard triple therapy for Helicobacter pylori has not changed over the last decade, but it is not good enough. Aliment Pharmacol Ther. 2011 Dec;34(11-12):1255-68. doi: 10.1111/j.1365-2036.2011.04887.x. Epub 2011 Oct 21. Review.

ReferenceCitation: Gisbert JP, Calvet X, O'Connor A, Mégraud F, O'Morain CA. Sequential therapy for Helicobacter pylori eradication: a critical review. J Clin Gastroenterol. 2010 May-Jun;44(5):313-25. doi: 10.1097/MCG.0b013e3181c8a1a3. Review.

ReferenceCitation: Gisbert JP, Calvet X. Review article: non-bismuth quadruple (concomitant) therapy for eradication of Helicobater pylori. Aliment Pharmacol Ther. 2011 Sep;34(6):604-17. doi: 10.1111/j.1365-2036.2011.04770.x. Epub 2011 Jul 11. Review.

ReferenceCitation: Gisbert JP, Khorrami S, Carballo F, Calvet X, Gené E, Dominguez-Muñoz JE. H. pylori eradication therapy vs. antisecretory non-eradication therapy (with or without long-term maintenance antisecretory therapy) for the prevention of recurrent bleeding from peptic ulcer. Cochrane Database Syst Rev. 2004;(2):CD004062. Review.

ReferenceCitation: Graham DY, Rimbara E. Understanding and appreciating sequential therapy for Helicobacter pylori eradication. J Clin Gastroenterol. 2011 Apr;45(4):309-13. doi: 10.1097/MCG.0b013e31820ac05e.

ReferenceCitation: Graham DY, Fischbach L. Helicobacter pylori treatment in the era of increasing antibiotic resistance. Gut. 2010 Aug;59(8):1143-53. doi: 10.1136/gut.2009.192757. Epub 2010 Jun 4. Review.

ReferenceCitation: Greenberg ER, Anderson GL, Morgan DR, Torres J, Chey WD, Bravo LE, Dominguez RL, Ferreccio C, Herrero R, Lazcano-Ponce EC, Meza-Montenegro MM, Peña R, Peña EM, Salazar-Martínez E, Correa P, Martínez ME, Valdivieso M, Goodman GE, Crowley JJ, Baker LH. 14-day triple, 5-day concomitant, and 10-day sequential therapies for Helicobacter pylori infection in seven Latin American sites: a randomised trial. Lancet. 2011 Aug 6;378(9790):507-14. doi: 10.1016/S0140-6736(11)60825-8. Epub 2011 Jul 21.

ReferenceCitation: Hsu PI, Wu DC, Wu JY, Graham DY. Is there a benefit to extending the duration of Helicobacter pylori sequential therapy to 14 days? Helicobacter. 2011 Apr;16(2):146-52. doi: 10.1111/j.1523-5378.2011.00829.x.

ReferenceCitation: Ikenberry SO, Harrison ME, Lichtenstein D, Dominitz JA, Anderson MA, Jagannath SB, Banerjee S, Cash BD, Fanelli RD, Gan SI, Shen B, Van Guilder T, Lee KK, Baron TH; ASGE STANDARDS OF PRACTICE COMMITTEE. The role of endoscopy in dyspepsia. Gastrointest Endosc. 2007 Dec;66(6):1071-5. Epub 2007 Oct 29.

ReferenceCitation: Maconi G, Sainaghi M, Molteni M, Bosani M, Gallus S, Ricci G, Alvisi V, Porro GB. Predictors of long-term outcome of functional dyspepsia and duodenal ulcer after successful Helicobacter pylori eradication--a 7-year follow-up study. Eur J Gastroenterol Hepatol. 2009 Apr;21(4):387-93. doi: 10.1097/MEG.0b013e3283069db0.

ReferenceCitation: Malfertheiner P, Megraud F, O'Morain CA, Atherton J, Axon AT, Bazzoli F, Gensini GF, Gisbert JP, Graham DY, Rokkas T, El-Omar EM, Kuipers EJ; European Helicobacter Study Group. Management of Helicobacter pylori infection--the Maastricht IV/ Florence Consensus Report. Gut. 2012 May;61(5):646-64. doi: 10.1136/gutjnl-2012-302084.

ReferenceCitation: McColl KE. Clinical practice. Helicobacter pylori infection. N Engl J Med. 2010 Apr 29;362(17):1597-604. doi: 10.1056/NEJMcp1001110. Review.

ReferenceCitation: McNicholl AG, Linares PM, Nyssen OP, Calvet X, Gisbert JP. Meta-analysis: esomeprazole or rabeprazole vs. first-generation pump inhibitors in the treatment of Helicobacter pylori infection. Aliment Pharmacol Ther. 2012 Sep;36(5):414-25. doi: 10.1111/j.1365-2036.2012.05211.x. Epub 2012 Jul 15.

ReferenceCitation: Moayyedi P. The health economics of Helicobacter pylori infection. Best Pract Res Clin Gastroenterol. 2007;21(2):347-61. Review.

ReferenceCitation: Moayyedi P, Deeks J, Talley NJ, Delaney B, Forman D. An update of the Cochrane systematic review of Helicobacter pylori eradication therapy in nonulcer dyspepsia: resolving the discrepancy between systematic reviews. Am J Gastroenterol. 2003 Dec;98(12):2621-6. Review.

ReferenceCitation: Niv Y, Hazazi R. Helicobacter pylori recurrence in developed and developing countries: meta-analysis of 13C-urea breath test follow-up after eradication. Helicobacter. 2008 Feb;13(1):56-61. doi: 10.1111/j.1523-5378.2008.00571.x.

ReferenceCitation: Oderda G, Shcherbakov P, Bontems P, Urruzuno P, Romano C, Gottrand F, Gómez MJ, Ravelli A, Gandullia P, Roma E, Cadranel S, De Giacomo C, Canani RB, Rutigliano V, Pehlivanoglu E, Kalach N, Roggero P, Celinska-Cedro D, Drumm B, Casswall T, Ashorn M, Arvanitakis SN; European Pediatric Task Force on Helicobacter pylori. Results from the pediatric European register for treatment of Helicobacter pylori (PERTH). Helicobacter. 2007 Apr;12(2):150-6.

ReferenceCitation: Rokkas T, Sechopoulos P, Robotis I, Margantinis G, Pistiolas D. Cumulative H. pylori eradication rates in clinical practice by adopting first and second-line regimens proposed by the Maastricht III consensus and a third-line empirical regimen. Am J Gastroenterol. 2009 Jan;104(1):21-5. doi: 10.1038/ajg.2008.87.

ReferenceCitation: Nyssen OP, Perez-Aisa A, Tepes B, Castro-Fernandez M, Kupcinskas J, Jonaitis L, Bujanda L, Lucendo A, Jurecic NB, Perez-Lasala J, Shvets O, Fadeenko G, Huguet JM, Kikec Z, Bordin D, Voynovan I, Leja M, Machado JC, Areia M, Fernandez-Salazar L, Rodrigo L, Alekseenko S, Barrio J, Ortuño J, Perona M, Vologzhanina L, Romero PM, Zaytsev O, Rokkas T, Georgopoulos S, Pellicano R, Buzas GM, Modolell I, Gomez Rodriguez BJ, Simsek I, Simsek C, Lafuente MR, Ilchishina T, Camarero JG, Dominguez-Cajal M, Ntouli V, Dekhnich NN, Phull P, Nuñez O, Lerang F, Venerito M, Heluwaert F, Tonkic A, Caldas M, Puig I, Megraud F, O'Morain C, Gisbert JP; Hp-EuReg Investigators. Adverse Event Profile During the Treatment of Helicobacter pylori: A Real-World Experience of 22,000 Patients From the European Registry on H. pylori Management (Hp-EuReg). Am J Gastroenterol. 2021 Jun 1;116(6):1220-1229. doi: 10.14309/ajg.0000000000001246.

family: Nyssen

full_name: Olga P. Nyssen

given: Olga P.

family: Perez-Aisa

full_name: Angeles Perez-Aisa

given: Angeles

family: Tepes

full_name: Bojan Tepes

given: Bojan

family: Castro-Fernandez

full_name: Manuel Castro-Fernandez

given: Manuel

family: Kupcinskas

full_name: Juozas Kupcinskas

given: Juozas

family: Jonaitis

full_name: Laimas Jonaitis

given: Laimas

family: Bujanda

full_name: Luis Bujanda

given: Luis

family: Lucendo

full_name: Alfredo Lucendo

given: Alfredo

family: Jurecic

full_name: Natasa Brglez Jurecic

given: Natasa Brglez

family: Perez-Lasala

full_name: Jorge Perez-Lasala

given: Jorge

family: Shvets

full_name: Oleg Shvets

given: Oleg

family: Fadeenko

full_name: Galina Fadeenko

given: Galina

family: Huguet

full_name: Jose M. Huguet

given: Jose M.

family: Kikec

full_name: Zdenki Kikec

given: Zdenki

family: Bordin

full_name: Dmitry Bordin

given: Dmitry

family: Voynovan

full_name: Irina Voynovan

given: Irina

family: Leja

full_name: Marcis Leja

given: Marcis

family: Machado

full_name: Jose Carlos Machado

publisher: Ovid Technologies (Wolters Kluwer Health)

ReferenceCitation: Nyssen OP, Perez-Aisa A, Castro-Fernandez M, Pellicano R, Huguet JM, Rodrigo L, Ortuñ J, O, Gomez-Rodriguez BJ, Pinto RM, Areia M, Perona M, Nuñez O, Romano M, Gravina AG, Pozzati L, Fernandez-Bermejo M, Venerito M, Malfertheiner P, Fernanadez-Salazar L, Gasbarrini A, Vaira D, Puig I, Megraud F, O'Morain C, Gisbert JP; Hp-EuReg investigators. European Registry on Helicobacter pylori management: Single-capsule bismuth quadruple therapy is effective in real-world clinical practice. United European Gastroenterol J. 2021 Feb;9(1):38-46. doi: 10.1177/2050640620972615. Epub 2021 Feb 11.

name: Department of Gastroenterology Hospital Universitario de La Princesa Universidad Autónoma de Madrid (UAM) and Centro de Investigación Biomódica en Red de Enfermedades Hepáticas y Digestivas (CIBEREHD)  Madrid Spain

name: Department of Gastroenterology Agencia Sanitaria Costa del Sol Red de Investigación en Servicios de Salud en Enfermedades Crónicas (REDISSEC)  Málaga Spain

name: Department of Gastroenterology Hospital de Valme  Sevilla Spain

name: Department of Gastroenterology Molinette Hospital  Turin Italy

name: Department of Gastroenterology Hospital General Universitario de Valencia  Valencia Spain

name: Department of Gastroenterology Hospital Universitario Central de Asturias  Oviedo Spain

name: Department of Gastroenterology Hospital Universitari i Politecnic La Fe  Valencia Spain

name: Department of Gastroenterology Hospital Quiron Sagrado Corazón  Sevilla Spain

name: Department of Gastroenterology Centro Hospitalar do Porto Institute of Biomedical Sciences Abel Salazar  Porto Portugal

name: Department of Gastroenterology Center for Health Technology and Services Research (CINTESIS) University of Porto  Porto Portugal

name: Department of Gastroenterology Portuguese Oncology Institute of Coimbra  Coimbra Portugal

name: Department of Gastroenterology Hospital Quiron  Marbella Spain

name: Department of Gastroenterology Hospital Universitario Sanitas La Moraleja  Madrid Spain

name: Department of Gastroenterology Università degli Studi della Campania “Luigi Vanvitelli”  Napoli Italy

name: Department of Gastroenterology Hospital Mérida  Badajoz Spain

name: Department of Gastroenterology Clinica San Francisco, Cáceres  Spain

name: Department of Gastroenterology Otto‐von‐Guericke University Hospital  Magdeburg Germany

name: Department of Gastroenterology Hospital Clinico Universitario de Valladolid  Valladolid Spain

name: Fondazione Policlinico Universitario Agostino Gemelli  Rome Italy

name: Department of Surgical and Medical Sciences University of Bologna  Bologna Italy

name: Althaia Xarxa Assistencial Universitària de Manresa and Universitat de Vic‐Universitat Central de Catalunya (UVicUCC)  Manresa Spain

name: Laboratoire de Bacteriologie Hôpital Pellegrin  Bordeaux Cedex France

name: Department of Gastroenterology Faculty of Health Sciences Trinity College Dublin  Dublin Ireland

journal_name: United European Gastroenterology Journal

title: European Registry on Helicobacter pylori management: Single‐capsule bismuth quadruple therapy is effective in real‐world clinical practice

ReferenceCitation: McNicholl AG, Bordin DS, Lucendo A, Fadeenko G, Fernandez MC, Voynovan I, Zakharova NV, Sarsenbaeva AS, Bujanda L, Perez-Aisa Á, Vologzhanina L, Zaytsev O, Ilchishina T, Coba C, Lasala JP, Alekseenko S, Modolell I, Molina-Infante J, Ruiz-Zorrilla Lopez R, Alonso-Galan H, Moreno NF, Hinojosa J, Santaella I, Varela P, Gonzalez-Cordero PL, Barrio J, Dominguez-Jimenez JL, Nuñez O, Alcedo J, Nyssen OP, Caldas M, Donday MG, Shvetz O, Megraud F, O'Morain C, Gisbert JP. Combination of Bismuth and Standard Triple Therapy Eradicates Helicobacter pylori Infection in More than 90% of Patients. Clin Gastroenterol Hepatol. 2020 Jan;18(1):89-98. doi: 10.1016/j.cgh.2019.03.048. Epub 2019 Apr 10.

journal_name: Clinical Gastroenterology and Hepatology

location_country:
Finland
Greece
Romania
Belgium
Russian Federation
Denmark
Ukraine
Croatia
France
Lithuania
Poland
Norway
Slovenia
Turkey
Sweden
Switzerland
Netherlands
Bulgaria
Hungary
Ireland
Portugal
Germany
United Kingdom
Italy
Austria
Serbia
Czechia
Spain
Estonia
Latvia

location_facility: Central Scientific Research Institute of Gastroenterology

location_facility: Dokuz Eylul University School of Medicine

location_facility: Ferencváros Health Centre, Gaastroenterology

location_facility: Gastroenterology Clinic, Henry Dunant Hospital

location_facility: Ikazia Ziekenhuis/ Erasmus Medisch Centrum

location_facility: Departament of Gastroenterology, Medical Centre for Postgraduate Education

location_facility: Lithuanian university of Health Sciences Hospital


type: Other Identifier

title: An Open-Label, Multicenter, Dose-Escalation Phase I Study to Evaluate the Safety, Pharmacokinetics, and Therapeutic Activity of RO6958688, A Novel T-cell Bispecific Antibody That Targets the Human Carcinoembryonic Antigen (CEA) on Tumor Cells and CD3 on T Cells, Administered Intravenously in Patients With Locally Advanced and/or Metastatic CEA(+) Solid Tumors

summary: Study BP29541 is a first-in-human, open-label, multi-center, dose-escalation Phase I clinical study of single-agent RO6958688 in participants with locally advanced and/or metastatic carcinoembryonic antigen (CEA) positive solid tumors who have progressed on standard treatment, are intolerant to standard of care (SOC), and/or are non-amenable to SOC. The study will be conducted in two parts. Part I of the study will investigate the safety and pharmacokinetics of a single dose of RO6958688 in single participant cohorts with dosing starting from a minimal anticipated biological effect level dose of 0.05 milligrams (mg) and up to a maximum dose of 2.5 mg. Part II will establish the appropriate therapeutic dose based on safety, pharmacokinetics, and the maximum tolerated dose (MTD) of RO6958688 for the once per week (QW) regimen, every three weeks (Q3W) regimen, and for the step up dosing regimen.

lead_sponsor: Hoffmann-La Roche

location_country:
Italy
Netherlands
France
Spain
Denmark
United States
Canada

location_facility: Centre Leon Berard; Departement Oncologie Medicale

location_facility: Cedars Sinai Medical Center; Samuel-Oschin Comprehensive Cancer Institute

location_facility: Clinica Universitaria de Navarra; Servicio de Oncologia

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

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

location_facility: Hospital Universitario 12 de Octubre; Servicio de Oncologia

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

location_facility: START Madrid-FJD, Hospital Fundacion Jimenez Diaz

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


title: Clinical Study of the BreathID® System to Train the Algorithm for the ¹³C-Octanoate Breath Test With or Without the ¹³C-Methacetin Breath Test (OBT and MBT Respectively) for Correlation With Histological Findings Associated With Non-Alcoholic Steatohepatitis (NASH) and Other Liver Diseases

summary: This study's aim is to develop an algorithm for the ¹³C-Octanoate Breath Test with or without the ¹³C-Methacetin Breath Test (OBT and MBT respectively) for correlation with histological findings associated with of Non-Alcoholic Steatohepatitis (NASH) and other liver diseases using the BreathID® System

lead_sponsor: Exalenz Bioscience LTD.

location_country:
United States
United Kingdom
Belgium
France

location_facility: Northwestern University, Feinberg School of Medicine

intervention_type: Device


title: RIFTOS MKI - Radioactive Iodine reFractory Asymptomatic Patients in Differentiated Thyroid Cancer - an Observational Study to Assess the Use of Multikinase Inhibitors

acronym: RIFTOS MKI

summary: The purpose of the study was to assess the use of Multikinase Inhibitors (MKIs) in the treatment of patients with a progressive differentiated thyroid carcinoma (DTC) refractory to radioactive iodine (RAI) who do not have any symptoms.

ReferenceCitation: Porcelli T, Luongo C, Sessa F, Klain M, Masone S, Troncone G, Bellevicine C, Schlumberger M, Salvatore D. Long-term management of lenvatinib-treated thyroid cancer patients: a real-life experience at a single institution. Endocrine. 2021 Aug;73(2):358-366. doi: 10.1007/s12020-021-02634-z. Epub 2021 Feb 3.

family: Porcelli

full_name: Tommaso Porcelli

given: Tommaso

family: Luongo

full_name: Cristina Luongo

given: Cristina

family: Sessa

full_name: Francesca Sessa

given: Francesca

family: Klain

full_name: Michele Klain

given: Michele

family: Masone

full_name: Stefania Masone

given: Stefania

family: Troncone

full_name: Giancarlo Troncone

given: Giancarlo

family: Bellevicine

full_name: Claudio Bellevicine

given: Claudio

family: Schlumberger

full_name: Martin Schlumberger

given: Martin

family: Salvatore

full_name: Domenico Salvatore

given: Domenico

journal_name: Endocrine

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

ReferenceCitation: Brose MS, Smit J, Lin CC, Pitoia F, Fellous M, DeSanctis Y, Schlumberger M, Tori M, Sugitani I. Timing of multikinase inhibitor initiation in differentiated thyroid cancer. Endocr Relat Cancer. 2017 May;24(5):237-242. doi: 10.1530/ERC-17-0016. Epub 2017 Mar 7.

family: Brose

full_name: Marcia S Brose

given: Marcia S

family: Smit

full_name: Johannes Smit

given: Johannes

family: Lin

full_name: Chia-Chi Lin

given: Chia-Chi

family: Pitoia

full_name: Fabian Pitoia

given: Fabian

family: Fellous

full_name: Marc Fellous

given: Marc

family: DeSanctis

full_name: Yoriko DeSanctis

given: Yoriko

family: Tori

full_name: Masayuki Tori

given: Masayuki

family: Sugitani

full_name: Iwao Sugitani

given: Iwao

journal_name: Endocrine-Related Cancer

evidence: open (via page says license)

location_country:
Brazil
India
Saudi Arabia
Lebanon
Mexico
Germany
Netherlands
Philippines
Spain
France
Japan
Greece
Argentina
Algeria
Egypt
Russian Federation
Turkey
United States
Taiwan


title: LUMINIST: LUng Cancer Molecular Insights Non Interventional Study

acronym: LUMINIST

summary: The recent development of therapies targeting specific biomarkers mutations is changing the standards of care and prognosis of patients with advanced NSCLC, but very few data are currently available on those emerging biomarkers. In addition, the correlation of biomarkers with patients' clinical outcomes in a standard of care setting is poorly understood. This study aims to address that need.

lead_sponsor: AstraZeneca

location_country:
Peru
Romania
Australia
Belgium
Russian Federation
Ukraine
France
Poland
Canada
Chile
Sweden
Netherlands
Bulgaria
Hungary
Portugal
Germany
United Kingdom
Italy
Austria
Mexico
Israel
Spain
Turkey


ReferenceCitation: Darras BT, Farrar MA, Mercuri E, Finkel RS, Foster R, Hughes SG, Bhan I, Farwell W, Gheuens S. An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials. CNS Drugs. 2019 Sep;33(9):919-932. doi: 10.1007/s40263-019-00656-w.

family: Darras

full_name: Basil T. Darras

given: Basil T.

family: Farrar

full_name: Michelle A. Farrar

given: Michelle A.

family: Mercuri

full_name: Eugenio Mercuri

given: Eugenio

family: Finkel

full_name: Richard S. Finkel

given: Richard S.

family: Foster

full_name: Richard Foster

given: Richard

family: Hughes

full_name: Steven G. Hughes

given: Steven G.

family: Bhan

full_name: Ishir Bhan

given: Ishir

family: Farwell

full_name: Wildon Farwell

given: Wildon

family: Gheuens

full_name: Sarah Gheuens

given: Sarah

journal_name: CNS Drugs

license: cc-by-nc

license_normalized: cc-by-nc

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

evidence: oa repository (via pmcid lookup)

license_normalized: no license

ReferenceCitation: Mercuri E, Darras BT, Chiriboga CA, Day JW, Campbell C, Connolly AM, Iannaccone ST, Kirschner J, Kuntz NL, Saito K, Shieh PB, Tulinius M, Mazzone ES, Montes J, Bishop KM, Yang Q, Foster R, Gheuens S, Bennett CF, Farwell W, Schneider E, De Vivo DC, Finkel RS; CHERISH Study Group. Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy. N Engl J Med. 2018 Feb 15;378(7):625-635. doi: 10.1056/NEJMoa1710504.

name: From the Department of Pediatric Neurology, Catholic University, Rome (E.M., E.S.M.); the Department of Neurology, Boston Children’s Hospital, Boston (B.T.D.), and Biogen, Cambridge (R.F., S.G., W.F.) — both in Massachusetts; the Departments of Neurology (C.A.C., J.M., D.C.D.), Pediatrics (C.A.C., D.C.D.), and Rehabilitation and Regenerative Medicine (J.M.), Columbia University Medical Center, New York; the Department of Neurology, Stanford School of Medicine, Stanford (J.W.D.), David Geffen School of...

family: Chiriboga

full_name: Claudia A. Chiriboga

given: Claudia A.

family: Day

full_name: John W. Day

given: John W.

family: Campbell

full_name: Craig Campbell

given: Craig

family: Connolly

full_name: Anne M. Connolly

given: Anne M.

family: Iannaccone

full_name: Susan T. Iannaccone

given: Susan T.

family: Kirschner

full_name: Janbernd Kirschner

given: Janbernd

family: Kuntz

full_name: Nancy L. Kuntz

repository_institution: University of Oxford - Oxford University Research Archive (ORA)

location_country:
Italy
United States
Sweden
Hong Kong
France
Japan
Korea, Republic of
Spain
Germany
Canada

location_facility: Children's Hospital - London Health Sciences Centre

location_facility: The University of Hong Kong, Queen Mary Hospital, Department of Paediatrics and Adolescent Medicine

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

location_facility: Fondazione Policlinico Universitario Agostino Gemelli-Universita Cattolica de Sacro Cuore-UOC Neuropsichiatre Infantile

location_facility: UCLA Clinical and Translational Research Center

location_facility: Washington University School of Medicine

location_facility: Armand Trousseau Hospital, I-Motion, Clinical Trials Platform

location_facility: University of Gothenburg, The Queen Silvia Children's Hospital

location_facility: Children's Hospital of Philadelphia - Neurology

location_facility: Seoul National University Children's Hospital

location_facility: AOU Policlinico G. Martino Dipartimento di Neuroscienze e Centro Clinico Nemo Sud

location_facility: Lucile Packard Children's Hospital at Stanford

location_facility: McGill University Health Centre-Glen Site-CIM

location_facility: University Hospital Freiberg, Center for Paediatrics and Adolescent Medicine, Department of Neuropaediatrics and Muscular Disease

title: A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Later-onset Spinal Muscular Atrophy

summary: The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset Spinal Muscular Atrophy (SMA). The secondary objective is to examine the safety and tolerability of nusinersen administered intrathecally to participants with later-onset SMA.


title: Study Title: A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Effect of GS-6615 on Exercise Capacity in Subjects With Symptomatic Hypertrophic Cardiomyopathy

acronym: LIBERTY-HCM

summary: The primary objective of this study was to evaluate the effect of eleclazine (GS-6615) on exercise capacity as measured by Peak oxygen uptake (VO2) achieved during cardiopulmonary exercise testing (CPET), in participants with symptomatic hypertrophic cardiomyopathy (HCM).

ReferenceCitation: Olivotto I, Hellawell JL, Farzaneh-Far R, Blair C, Coppini R, Myers J, Belardinelli L, Maron MS. Novel Approach Targeting the Complex Pathophysiology of Hypertrophic Cardiomyopathy: The Impact of Late Sodium Current Inhibition on Exercise Capacity in Subjects with Symptomatic Hypertrophic Cardiomyopathy (LIBERTY-HCM) Trial. Circ Heart Fail. 2016 Mar;9(3):e002764. doi: 10.1161/CIRCHEARTFAILURE.115.002764.

lead_sponsor: Gilead Sciences

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

location_facility: Brigham & Women's Hospital and Harvard Medical School

location_facility: Columbia University Medical Center/ New York Presbyterian

location_facility: Azienda Ospedaliero Universitaria di Parma

location_facility: Azienda Ospedaliera San Camillo Forlanini

location_facility: Azienda Ospedaliera Universitaria Careggi

location_facility: Azienda Ospedaliero Universitaria Di Bologna

location_facility: University of Iowa Hospitals and Clinics


title: A Phase IIIb, Multinational, Multicenter, Open-Label Extension Study Assessing the Long-Term Safety of PRN Intravitreal Injections of DE-109 in Subjects With Non-Infectious Uveitis of the Posterior Segment of the Eye Who Have Participated in the SAKURA Development Program

acronym: SPRING

summary: The objective of this extension study is to evaluate the long-term safety of treatment with DE-109 (440 μg) in subjects with non-infectious uveitis of the posterior segment of the eye who have participated in the SAKURA development program.

lead_sponsor: Santen Inc.

location_country:
Italy
Austria
India
France
Turkey
United States


title: A Natural History Study of Acute Hepatic Porphyria (AHP) Patients With Recurrent Attacks

summary: The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.

ipd_sharing: No

lead_sponsor: Alnylam Pharmaceuticals

location_country:
Italy
South Africa
Switzerland
Mexico
Netherlands
Germany
Finland
France
Czechia
Bulgaria
Israel
Poland
Norway
Belgium
Australia
Spain
United States
United Kingdom
Korea, Republic of
Taiwan


title: A Randomized, Double-blind, Placebo-controlled Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P; CBD) as Adjunctive Treatment for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults.

summary: The primary objective of this study was to evaluate the efficacy of GWP42003-P as adjunctive treatment in reducing the number of drop seizures when compared with placebo in participants with Lennox-Gastaut syndrome (LGS).

ReferenceCitation: Devinsky O, Patel AD, Cross JH, Villanueva V, Wirrell EC, Privitera M, Greenwood SM, Roberts C, Checketts D, VanLandingham KE, Zuberi SM; GWPCARE3 Study Group. Effect of Cannabidiol on Drop Seizures in the Lennox-Gastaut Syndrome. N Engl J Med. 2018 May 17;378(20):1888-1897. doi: 10.1056/NEJMoa1714631.

name: From New York University Langone Comprehensive Epilepsy Center, New York (O.D.); Nationwide Children’s Hospital and the Ohio State University College of Medicine, Columbus (A.D.P.), and the University of Cincinnati Medical Center, Department of Neurology, Cincinnati (M.P.) — all in Ohio; UCL Great Ormond Street Institute of Child Health, London (J.H.C.), GW Research Ltd., Cambridge (S.M.G., C.R., D.C.), and the Royal Hospital for Children and School of Medicine, University of Glasgow, Glasgow (S.M.Z.) —...

family: Devinsky

full_name: Orrin Devinsky

given: Orrin

family: Patel

full_name: Anup D. Patel

given: Anup D.

family: Cross

full_name: J. Helen Cross

given: J. Helen

family: Villanueva

full_name: Vicente Villanueva

given: Vicente

family: Wirrell

full_name: Elaine C. Wirrell

given: Elaine C.

family: Privitera

full_name: Michael Privitera

given: Michael

family: Greenwood

full_name: Sam M. Greenwood

given: Sam M.

family: Roberts

full_name: Claire Roberts

given: Claire

family: Checketts

full_name: Daniel Checketts

given: Daniel

family: VanLandingham

full_name: Kevan E. VanLandingham

given: Kevan E.

family: Zuberi

full_name: Sameer M. Zuberi

given: Sameer M.

journal_name: New England Journal of Medicine

oa_colors: green other

evidence: open (via free article)

repository_institution: University of Edinburgh - Edinburgh Research Explorer

repository_institution: University College London - UCL Discovery

repository_normalized: UCL Discovery

publisher: Massachusetts Medical Society

publisher_dissemination: Massachusetts Medical Society

publisher_group: Massachusetts Medical Society

publisher_normalized: Massachusetts Medical Society

title: Effect of Cannabidiol on Drop Seizures in the Lennox–Gastaut Syndrome

ReferenceCitation: Brigo F, Jones K, Eltze C, Matricardi S. Anti-seizure medications for Lennox-Gastaut syndrome. Cochrane Database Syst Rev. 2021 Apr 7;4:CD003277. doi: 10.1002/14651858.CD003277.pub4.

name: Department of Neurology; Hospital of Merano (SABES-ASDAA); Merano-Meran Italy

family: Brigo

full_name: Francesco Brigo

given: Francesco

name: Cochrane Neuromuscular; Queen Square Centre for Neuromuscular Diseases; London UK

name: Cochrane Pain, Palliative and Supportive Care; Oxford UK

family: Jones

full_name: Katherine Jones

given: Katherine

name: Great Ormond Street Hospital for Children NHS Foundation Trust; London UK

family: Eltze

full_name: Christin Eltze

given: Christin

name: Department of Child Neuropsychiatry; Children’s Hospital “G. Salesi”, Ospedali Riuniti Ancona; Ancona Italy

family: Matricardi

full_name: Sara Matricardi

given: Sara

ReferenceCitation: Privitera M, Bhathal H, Wong M, Cross JH, Wirrell E, Marsh ED, Mazurkiewicz-Beldzinska M, Villanueva V, Checketts D, Knappertz V, VanLandingham K. Time to onset of cannabidiol (CBD) treatment effect in Lennox-Gastaut syndrome: Analysis from two randomized controlled trials. Epilepsia. 2021 May;62(5):1130-1140. doi: 10.1111/epi.16878. Epub 2021 Apr 2.

name: University of Cincinnati Medical Center Cincinnati OH USA

name: Neurocenter Barcelona Teknon Medical Center Barcelona Spain

name: Wake Forest Baptist Medical Center Winston‐Salem NC USA

name: University College London National Institute for Health Research Biomedical Research Center Great Ormond Street Institute of Child Health London UK

name: Children's Hospital of Philadelphia Philadelphia PA USA

name: Medical University of Gdańsk Gdańsk Poland

name: University and Polytechnic Hospital La Fe Valencia Spain

name: Greenwich Biosciences, Inc. Carlsbad CA USA

title: Time to onset of cannabidiol (CBD) treatment effect in Lennox–Gastaut syndrome: Analysis from two randomized controlled trials

ReferenceCitation: Perry MS. Don't Fear the Reefer-Evidence Mounts for Plant-Based Cannabidiol as Treatment for Epilepsy. Epilepsy Curr. 2019 Mar-Apr;19(2):93-95. doi: 10.1177/1535759719835671.

title: Don’t Fear the Reefer—Evidence Mounts for Plant-Based Cannabidiol as Treatment for Epilepsy

location_country:
United States
United Kingdom
France
Spain


ReferenceCitation: Plazzi G, Ruoff C, Lecendreux M, Dauvilliers Y, Rosen CL, Black J, Parvataneni R, Guinta D, Wang YG, Mignot E. Treatment of paediatric narcolepsy with sodium oxybate: a double-blind, placebo-controlled, randomised-withdrawal multicentre study and open-label investigation. Lancet Child Adolesc Health. 2018 Jul;2(7):483-494. doi: 10.1016/S2352-4642(18)30133-0. Epub 2018 May 21.

family: Plazzi

full_name: Giuseppe Plazzi

given: Giuseppe

family: Ruoff

full_name: Chad Ruoff

given: Chad

family: Lecendreux

full_name: Michel Lecendreux

given: Michel

family: Dauvilliers

full_name: Yves Dauvilliers

given: Yves

family: Rosen

full_name: Carol L Rosen

given: Carol L

family: Black

full_name: Jed Black

given: Jed

family: Parvataneni

full_name: Rupa Parvataneni

given: Rupa

family: Guinta

full_name: Diane Guinta

given: Diane

family: Wang

full_name: Youyu Grace Wang

given: Youyu Grace

family: Mignot

full_name: Emmanuel Mignot

given: Emmanuel

journal_name: The Lancet Child & Adolescent Health

lead_sponsor: Jazz Pharmaceuticals

location_country:
Italy
United States
France
Netherlands

location_facility: Cincinnati Children's Hospital Medical Center

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

location_facility: University Hospitals Cleveland Medical Center

location_facility: Dipartimento di Scienze Biomediche e Biomotorie

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

title:
A Double-Blind, Placebo-Controlled, Randomized-Withdrawal,
 Multicenter Study of the Efficacy and Safety of Xyrem with an Open- Label Pharmacokinetic Evaluation and Safety Extension in Pediatric Subjects with Narcolepsy with Cataplexy

summary: The purpose of this trial is to assess the efficacy and safety of Xyrem in pediatrics subjects with narcolepsy that includes cataplexy.


title: A Post-market Clinical Follow-up Study of the TITAN™ Reverse Shoulder System Used in Primary or Revision Total Shoulder Arthroplasty

summary: The Integra® TITAN™ Reverse Shoulder System (TRS) is a semi-constrained total shoulder construct. When used under the conditions and purposes intended the TRS will relieve pain and restore some functional joint motion to the affected shoulder. The purpose of this study is to assess the short, mid and long term outcomes of the TITAN™ Reverse Shoulder System.

status: Active, not recruiting

lead_sponsor: Smith & Nephew, Inc.

location_facility: Hospital of the University of Pennsylvania


ReferenceCitation: Brandão LR, Albisetti M, Halton J, Bomgaars L, Chalmers E, Mitchell LG, Nurmeev I, Svirin P, Kuhn T, Zapletal O, Tartakovsky I, Simetzberger M, Huang F, Sun Z, Kreuzer J, Gropper S, Brueckmann M, Luciani M; DIVERSITY Study Investigators. Safety of dabigatran etexilate for the secondary prevention of venous thromboembolism in children. Blood. 2020 Feb 13;135(7):491-504. doi: 10.1182/blood.2019000998. Erratum in: Blood. 2020 May 7;135(19):1720.

name: The Hospital for Sick Children, Toronto, ON, Canada;

family: Brandão

full_name: Leonardo R. Brandão

given: Leonardo R.

name: Hematology Department, University Children’s Hospital, Zürich, Switzerland;

family: Albisetti

full_name: Manuela Albisetti

given: Manuela

name: Children’s Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada;

family: Halton

full_name: Jacqueline Halton

given: Jacqueline

name: Department of Pediatrics, Texas Children’s Cancer and Hematology Centers, Baylor College of Medicine, Houston, TX;

family: Bomgaars

full_name: Lisa Bomgaars

given: Lisa

name: Royal Hospital for Children, Glasgow, Scotland, United Kingdom;

family: Chalmers

full_name: Elizabeth Chalmers

given: Elizabeth

name: Department of Pediatrics, University of Alberta, Edmonton, AB, Canada;

family: Mitchell

full_name: Lesley G. Mitchell

given: Lesley G.

name: Pediatric Hospital, Republic of Tatarstan, Kazan Medical University, Kazan, Russian Federation;

family: Nurmeev

full_name: Ildar Nurmeev

given: Ildar

name: Pediatric Hematology Department, Municipal Children’s Hospital “Morozovskaya,” Moscow, Russian Federation;

family: Svirin

full_name: Pavel Svirin

given: Pavel

name: Pediatric Hematology/Oncology Department, University Hospital Ostrava, Ostrava, Czech Republic;

name: Faculty of Medicine, University of Ostrava, Ostrava, Czech Republic;

family: Kuhn

full_name: Tomas Kuhn

given: Tomas

name: Pediatric Hematology Department, University Hospital Brno, Brno, Czech Republic;

name: Masaryk University, Brno, Czech Republic;

family: Zapletal

full_name: Ondrej Zapletal

given: Ondrej

name: Therapeutic Area Cardiometabolic Medicine, Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany;

family: Tartakovsky

full_name: Igor Tartakovsky

given: Igor

name: Department of Clinical Development, Boehringer Ingelheim RCV GmbH & Co KG, Vienna, Austria;

family: Simetzberger

full_name: Monika Simetzberger

given: Monika

name: Translational Medicine and Clinical Pharmacology and

family: Huang

full_name: Fenglei Huang

given: Fenglei

name: Biostatistics and Data Sciences, Boehringer Ingelheim Pharmaceuticals, Inc, Ridgefield, CT;

family: Sun

full_name: Zhichao Sun

given: Zhichao

name: Boehringer Ingelheim Singapore Pte Ltd, Singapore;

family: Kreuzer

full_name: Jörg Kreuzer

given: Jörg

family: Gropper

full_name: Savion Gropper

given: Savion

name: Faculty of Medicine Mannheim of the University of Heidelberg, Mannheim, Germany; and

family: Brueckmann

full_name: Martina Brueckmann

given: Martina

name: Pediatric Hematology/Oncology Department, Pediatric Hospital Bambino Gesù, Rome, Italy

family: Luciani

full_name: Matteo Luciani

given: Matteo

journal_name: Blood

repository_institution: University of Zurich - Zurich Open Repository and Archive

version: acceptedVersion

publisher: American Society of Hematology

location_country:
Belgium
Russian Federation
Denmark
Taiwan
Ukraine
France
Thailand
Lithuania
Norway
Canada
Sweden
Switzerland
Hungary
United States
Germany
Italy
Austria
Brazil
Mexico
Czechia
Israel
Turkey

location_facility: St.Petersburg State Pediatric Univ.Ministry of Healthcare RF

location_facility: Providence Sacred Heart Medical Center and Children's Hospital

location_facility: Cukurova Universitesi Tip Fakultesi Cocuk Sagligi

location_facility: Ege Universitesi Tip Fakultesi Cocuk Hematolojisi Bilim Dali

location_facility: University Hospital Plzen, Plzen-Lochotin

location_facility: Instituto de Oncologia Pediatrica - IOP / GRAAC - UNIFESP

location_facility: Oslo Universitetssykehus HF, Rikshospitalet

location_facility: Morozovskaya Children Clin.Hosp.,Haematological Dept, Moscow

location_facility: Child.CityClin.Hos.na.ZA Bashlyaeva MoscowHealth Dep,Cardiol

location_facility: Children Rep.Clin.Hosp of MoH,Cardio Vas.surgery Dept, Kazan

location_facility: Reg Clin.Hosp.#1,Healthcare Tyumen Region,Cardiovas.Surgery

location_facility: Necmettin Erbakan Universitesi Meram Tip Fakultesi

location_facility: Western Ukrainian Spec.Children Med.Center,Lviv

location_facility: HMCP - Hospital e Maternidade Celso Pierro - PUC-Campinas

location_facility: Children Intensive Care Hosp,Anaesthesiology Dept,Vilnius

location_facility: State Budget Healthcare Institution "Republican children's clinical hospital"

location_facility: Shaare Zedek Medical Center, Jerusalem 91031

location_facility: Faculdade de Ciencias Medicas da UNICAMP

location_facility: Science Res.Instit.CV Diseases,Scientific Res.Dept,Kemerovo

location_facility: Rigshospitalet, København, Børneonkologisk Afsnit 5002

location_facility: Istanbul Universitesi Cerrahpasa Tip Fakultesi

location_facility: Istanbul Saglik Bilimleri Uni. Kanuni Sultan Suleyman EAH

title: Open Label, Single Arm Safety Prospective Cohort Study of Dabigatran Etexilate for Secondary Prevention of Venous Thromboembolism in Children From 0 to Less Than 18 Years

summary: This open-label, single arm prospective cohort study will assess the safety of dabigatran etexilate in secondary prevention of venous thromboembolism in paediatric patients. Children from 0 to less than 18 years of age will be eligible to participate.


title: A Phase 3, Randomized, Double-Blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy

acronym: ENDEAR

summary: The primary objective of the study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally (IT) to participants with infantile-onset with infantile-onset spinal muscular atrophy (SMA). The secondary objective of the study is to examine the safety and tolerability of nusinersen administered intrathecally to participants with infantile-onset SMA.

family: Darras

full_name: Basil T. Darras

given: Basil T.

family: Farrar

full_name: Michelle A. Farrar

given: Michelle A.

family: Mercuri

full_name: Eugenio Mercuri

given: Eugenio

family: Finkel

full_name: Richard S. Finkel

given: Richard S.

family: Foster

full_name: Richard Foster

given: Richard

family: Hughes

full_name: Steven G. Hughes

given: Steven G.

family: Bhan

full_name: Ishir Bhan

given: Ishir

family: Farwell

full_name: Wildon Farwell

given: Wildon

family: Gheuens

full_name: Sarah Gheuens

given: Sarah

title: An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials

ReferenceCitation: Dabbous O, Maru B, Jansen JP, Lorenzi M, Cloutier M, Guérin A, Pivneva I, Wu EQ, Arjunji R, Feltner D, Sproule DM. Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1. Adv Ther. 2019 May;36(5):1164-1176. doi: 10.1007/s12325-019-00923-8. Epub 2019 Mar 16.

family: Dabbous

full_name: Omar Dabbous

given: Omar

family: Maru

full_name: Benit Maru

given: Benit

family: Jansen

full_name: Jeroen P. Jansen

given: Jeroen P.

family: Lorenzi

full_name: Maria Lorenzi

given: Maria

family: Cloutier

full_name: Martin Cloutier

given: Martin

family: Guérin

full_name: Annie Guérin

given: Annie

family: Pivneva

ReferenceCitation: Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, Chiriboga CA, Saito K, Servais L, Tizzano E, Topaloglu H, Tulinius M, Montes J, Glanzman AM, Bishop K, Zhong ZJ, Gheuens S, Bennett CF, Schneider E, Farwell W, De Vivo DC; ENDEAR Study Group. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. N Engl J Med. 2017 Nov 2;377(18):1723-1732. doi: 10.1056/NEJMoa1702752.

repositories_institution: University College London - UCL Discovery

repositories_institution: University of Oxford - Oxford University Research Archive (ORA)

ReferenceCitation: Finkel RS, Chiriboga CA, Vajsar J, Day JW, Montes J, De Vivo DC, Yamashita M, Rigo F, Hung G, Schneider E, Norris DA, Xia S, Bennett CF, Bishop KM. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet. 2016 Dec 17;388(10063):3017-3026. doi: 10.1016/S0140-6736(16)31408-8. Epub 2016 Dec 7.

location_country:
Italy
United States
Sweden
Germany
France
Japan
Belgium
Australia
Spain
Turkey
Canada
United Kingdom
Korea, Republic of

location_facility: Hospital Universitario La Paz, Pediatric Neurology Department

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

location_facility: Istituto Giannina Gaslini, Centro Traslazionale di Miologia e Patologie Neurodegenerative

location_facility: British Columbia Children's Hospital/UBC

location_facility: Washington University School of Medicine

location_facility: University of Gothenburg, The Queen Silvia Children's Hospital

location_facility: UCL Institute of Child Health/Great Ormond Street

location_facility: Royal Children's Hospital, Children's Neuroscience Centre

location_facility: Children's Hospital of Philadelphia - Neurology

location_facility: Universtatsklinikum Freiburg, Zentrum fur Kinder-und Jugendmedizin , Abteilung Neuropadiatrie und Muskelerkrankungen

location_facility: UT Southwestern Medical Center/Children's Medical Center Dallas

location_facility: Pediatric Neurology Unit, Catholic University

location_facility: MRC Centre for Neuromuscular Diseases at Newcastle, Institute of Genetic Medicine Newcastle University

location_facility: Hôpital Universitaire des Enfants Reine FABIOLA (HUDERF)

location_facility: Primary Children's Medical Center (University of Utah)


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

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

lead_sponsor: Shire

location_country:
Italy
Austria
Germany
France
Czechia
Australia
United States

location_facility: Ann and Robert H Lurie Childrens Hospital of Chicago

location_facility: Istituto Dermopatico Dell'immacolata IRCCS

location_facility: Cincinnati Childrens Hospital Medical Center

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


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

acronym: HIBISCUS II

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

ReferenceCitation: Rubin DT, Dotan I, DuVall A, Bouhnik Y, Radford-Smith G, Higgins PDR, Mishkin DS, Arrisi P, Scalori A, Oh YS, Tole S, Chai A, Chamberlain-James K, Lacey S, McBride J, Panés J; HIBISCUS Study Group. Etrolizumab versus adalimumab or placebo as induction therapy for moderately to severely active ulcerative colitis (HIBISCUS): two phase 3 randomised, controlled trials. Lancet Gastroenterol Hepatol. 2021 Nov 16. pii: S2468-1253(21)00338-1. doi: 10.1016/S2468-1253(21)00338-1. [Epub ahead of print]

family: Rubin

full_name: David T Rubin

given: David T

family: Dotan

full_name: Iris Dotan

given: Iris

family: DuVall

full_name: Aaron DuVall

given: Aaron

family: Bouhnik

full_name: Yoram Bouhnik

given: Yoram

family: Radford-Smith

full_name: Graham Radford-Smith

given: Graham

family: Higgins

full_name: Peter D R Higgins

given: Peter D R

family: Mishkin

full_name: Daniel S Mishkin

given: Daniel S

family: Arrisi

full_name: Pablo Arrisi

given: Pablo

family: Scalori

full_name: Astrid Scalori

given: Astrid

family: Oh

full_name: Young S Oh

given: Young S

family: Tole

full_name: Swati Tole

given: Swati

family: Chai

full_name: Akiko Chai

given: Akiko

family: Chamberlain-James

full_name: Kirsten Chamberlain-James

given: Kirsten

family: Lacey

full_name: Stuart Lacey

given: Stuart

family: McBride

full_name: Jacqueline McBride

given: Jacqueline

family: Panés

full_name: Julian Panés

given: Julian

family: Abdulkhakov

full_name: Rustem Abdulkhakov

given: Rustem

family: Abu Bakar

full_name: Norasiah Abu Bakar

given: Norasiah

family: Aguilar

full_name: Humberto Aguilar

given: Humberto

family: Aizenberg

full_name: Diego Aizenberg

given: Diego

family: Akpinar

full_name: Hale Akpinar

given: Hale

family: Akriviadis

full_name: Evangelos Akriviadis

given: Evangelos

family: Alexeeva

full_name: Olga Alexeeva

full_name: Carlos Fernando de Magalhães Francesconi

journal_name: The Lancet Gastroenterology & Hepatology

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

name: The Etrolizumab Global Steering Committee

title: Etrolizumab for the Treatment of Ulcerative Colitis and Crohn’s Disease: An Overview of the Phase 3 Clinical Program

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

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

location_facility: Haydarpasa Numune Training and Research Hospital; Gastroenterology

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

location_facility: Private Small Enterprise Medical Center Pulse

location_facility: Great Lakes Gastroenterology Research, LLC

location_facility: Irkutsk State Medical Academy of Continuing Education

location_facility: Hospital Raja Perempuan Zainab II; Department of Medicine

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

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

location_facility: Centro de Investigaciones Medicas Mar Del Plata

location_facility: Klaipeda Seamen's Hospital, Public Institution

location_facility: Pauls Stradins Clinical University Hospital

location_facility: Pavlov First Saint Petersburg State Medical University

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

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

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

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

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

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

location_facility: A.Novak Transcarpathian Regional Clinical Hospital

location_facility: CI Kherson Afanasii and Olha Tropiny City Clinical Hospital

location_facility: BHI of Omsk region Clinical Oncology Dispensary

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

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

location_facility: Hospital Universitario Walter Cantidio - UFC

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

location_facility: Niepubliczny Zaklad Opieki Zdrowotnej SONOMED

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

location_facility: Medical Center "Nov Rehabilitatsionen Tsentar", EOOD

location_facility: Pusat Perubatan Universiti Kebangsaan Malaysia

location_facility: Center of Emergency and Radiation Medicine; Pulmonology

location_facility: CI of Kyiv RC Kyiv Regional Clinical Hospital

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

location_facility: University General Hospital of Heraklion

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


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

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

location_facility: Hospital Ramon y Cajal ; Servicio de Reumatologia

location_facility: Hospital Universitario la Fe: Servicio de Reumatologia Pediatrica

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

location_facility: University of Utah; Immunology/Rheumatology/Allergy

location_facility: Alder Hey Children's NHS Foundation Trust

location_facility: Hospital Universitario Dr. Jose Eleuterio Gonzalez; Pediatria

location_facility: Bristol Royal Hospital for Children; Rheumatology Department

location_facility: Hospital de La Paz; Unidad de Reumatologia Pediatrica

location_facility: Hackensack University Medical Center; Pediatric Rheumatology

location_facility: SI Sceintific children health center RAMS

location_facility: Royal Children'S Hospital; Paediatric Rheumatology

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

location_facility: Universidade Federal de Sao Paulo - UNIFES

location_facility: Asklepios Klinik; Zentrum für Allgemeine Pädiatrie und Neonatologie

location_facility: Hospital de Ninos de la Santisima Trinidad; Hematología

location_facility: Arkansas Children's Hospital Research Institute

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

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

location_facility: Westmead Hospital; Paediatric Rheumatology

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

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


title: A Phase Ib, Open-Label, Multicenter Study to Investigate the Pharmacokinetics, Pharmacodynamics, and Safety of Tocilizumab Following Subcutaneous Administration to Patients With Systemic Juvenile Idiopathic Arthritis

summary: This open-label, multicenter study will evaluate the pharmacokinetics, pharmacodynamics, and safety of subcutaneously administered tocilizumab in participants with Systemic Juvenile Idiopathic Arthritis (sJIA). Participants with body weight less than (<) 30 kilograms (kg) will receive subcutaneous (SC) tocilizumab dose every 2 weeks (Q2W) and participants with body weight greater than or equal to (>=) 30 kg will receive weekly (QW), for 52 weeks. Tocilizumab was administered every 10 days until pre-planned interim analysis was performed and changed to Q2W in participants with body weight <30 kg.

ReferenceCitation: Ruperto N, Brunner HI, Ramanan AV, Horneff G, Cuttica R, Henrickson M, Anton J, Boteanu AL, Penades IC, Minden K, Schmeling H, Hufnagel M, Weiss JE, Pardeo M, Nanda K, Roth J, Rubio-Pérez N, Hsu JC, Wimalasundera S, Wells C, Bharucha K, Douglass W, Bao M, Mallalieu NL, Martini A, Lovell D, Benedetti F; Paediatric Rheumatology INternational Trials Organisation (PRINTO) and the Paediatric Rheumatology Collaborative Study Group (PRCSG). Subcutaneous dosing regimens of tocilizumab in children with systemic or polyarticular juvenile idiopathic arthritis. Rheumatology (Oxford). 2021 Oct 2;60(10):4568-4580. doi: 10.1093/rheumatology/keab047.

name: IRCCS Istituto Giannina Gaslini, Clinica Pediatrica e Reumatologia—PRINTO, Genoa, Italy

family: Ruperto

full_name: Nicolino Ruperto

given: Nicolino

name: Pediatric Rheumatology Collaborative Study Group (PRCSG), Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, USA

family: Brunner

full_name: Hermine I Brunner

given: Hermine I

name: University Hospitals Bristol NHS Foundation Trust & Bristol Medical School, University of Bristol, Bristol, UK

family: Ramanan

full_name: Athimalaipet V Ramanan

given: Athimalaipet V

name: Department of General Pediatrics, Asklepios Clinic Sankt Augustin, Sankt Augustin, Germany

name: Department of Pediatric and Adolescents Medicine, University Hospital of Cologne, Cologne, Germany

family: Horneff

full_name: Gerd Horneff

given: Gerd

name: Rheumatology Section, Hospital Pedro de Elizalde, Buenos Aires, Argentina

family: Cuttica

full_name: Rubén Cuttica

given: Rubén

family: Henrickson

full_name: Michael Henrickson

given: Michael

name: Hospital Sant Joan de Déu, Universitat de Barcelona, Unidad de Reumatología Pediátrica, Esplugues de Llobregat (Barcelona), Spain

family: Anton

full_name: Jordi Anton

given: Jordi

name: Pediatric Rheumatology Unit, University Hospital Ramón y Cajal, Madrid, Spain

family: Boteanu

full_name: Alina Lucica Boteanu

given: Alina Lucica

name: Pediatric Rheumatology Unit, Hospital Universitario y Politécnico La Fe, Valencia, Spain

family: Penades

full_name: Inmaculada Calvo Penades

given: Inmaculada Calvo

name: German Rheumatism Research Centre Berlin, Berlin, Germany

name: Department of Rheumatology and Clinical Immunology, Charité University Medicine, Berlin, Germany

family: Minden

full_name: Kirsten Minden

name: Department of Pediatrics, Alberta Children’s Hospital and Cumming School of Medicine/University of Calgary, Alberta, Canada

family: Schmeling

full_name: Heinrike Schmeling

given: Heinrike

name: University Medical Center Freiburg, Department of Pediatrics and Adolescent Medicine, Division of Pediatric Infectious Diseases and Rheumatology, Medical Faculty, University of Freiburg, Freiburg, Germany

family: Hufnagel

full_name: Markus Hufnagel

given: Markus

name: Hackensack University Medical Center, Pediatric Rheumatology, Hackensack, NJ, USA

family: Weiss

full_name: Jennifer E Weiss

given: Jennifer E

name: Division of Rheumatology, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy

family: Pardeo

full_name: Manuela Pardeo

given: Manuela

name: Seattle Children’s Hospital, Seattle, WA, USA

family: Nanda

full_name: Kabita Nanda

given: Kabita

name: University of Ottawa and Division of Pediatric Dermatology & Rheumatology, Children’s Hospital of Eastern Ontario, Ottawa, ON, Canada

family: Roth

full_name: Johannes Roth

given: Johannes

name: Universidad Autónoma de Nuevo León, Facultad de Medicina, Departamento de Pediatria, Hospital Universitario ‘Dr. J. E. González’, Monterrey, NL, Mexico

family: Rubio-Pérez

full_name: Nadina Rubio-Pérez

given: Nadina

name: Roche Innovation Center, New York, NY, USA

family: Hsu

full_name: Joy C Hsu

given: Joy C

name: Roche Products Ltd, Welwyn Garden City, UK

family: Wimalasundera

full_name: Sunethra Wimalasundera

given: Sunethra

family: Wells

full_name: Chris Wells

given: Chris

family: Bharucha

full_name: Kamal Bharucha

given: Kamal

family: Douglass

full_name: Wendy Douglass

given: Wendy

family: Bao

full_name: Min Bao

given: Min

name: Università degli Studi di Genova, Dipartimento di Neuroscienze, Riabilitazione, Oftalmologia, Genetica e Scienze Materno-Infantili, Genoa, Italy

name: for the Pediatric Rheumatology INternational Trials Organisation (PRINTO) and the Pediatric Rheumatology Collaborative Study Group (PRCSG)

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

location_facility: Hospital Ramon y Cajal ; Servicio de Reumatologia

location_facility: Hospital Universitario la Fe: Servicio de Reumatologia Pediatrica

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

location_facility: Hospital das Clinicas - FMUSP Ribeirao Preto; Pediatria - Imunologia e Reumatologia

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

location_facility: Levine Children's Hospital; Divison of Pediatric Rheumatology; Department of Pediatrics

location_facility: Nuovo Ospedale Pediatrico Meyer; Reumatologia - Clinica Pediatrica 1°

location_facility: FSBI "Scientific Research Institute of Rheumatology" of russian Academy of Medical Sciences

location_facility: Universidade Federal de Sao Paulo - UNIFESP

location_facility: Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS

location_facility: Asklepios Klinik; Zentrum fuer Allgemeine Paediatrie und Neonatologie

location_facility: Hospital Infantil Universitario Niño Jesus, Servicio Reumatologia

location_facility: Hospital das Clinicas - FMUSP; Instituto da Crianca - Reumatologia

location_facility: Hospital de Ninos de la Santisima Trinidad; Reumatologia Infantil

location_facility: The University of Chicago;Department of Pediatrics

location_facility: Great Ormond Street Hospital; Somers Clinical Research Facility

location_facility: Connecticut Children's Medical Center; 5E Clinical Trials Unit