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

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

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

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

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

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

family: Postel-Vinay

full_name: Sophie Postel-Vinay

family: Herbschleb

full_name: Karin Herbschleb

given: Karin

family: Massard

full_name: Christophe Massard

given: Christophe

family: Woodcock

full_name: Victoria Woodcock

given: Victoria

family: Soria

full_name: Jean-Charles Soria

given: Jean-Charles

family: Walter

full_name: Annette O. Walter

given: Annette O.

family: Ewerton

full_name: Flavio Ewerton

given: Flavio

family: Poelman

full_name: Martine Poelman

given: Martine

family: Benson

full_name: Neil Benson

given: Neil

family: Ocker

full_name: Matthias Ocker

given: Matthias

family: Wilkinson

full_name: Gary Wilkinson

given: Gary

family: Middleton

full_name: Mark Middleton

given: Mark

bso_classification: Chemistry

journal_name: European Journal of Cancer

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

version: submittedVersion

lead_sponsor: Bayer

location_country:
United States
Switzerland
Finland
France
Germany
United Kingdom


type: EudraCT Number

title: A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate AFQ056 in Adult Patients With Fragile X Syndrome

summary: This Phase IIb study is designed to assess whether 3 doses of AFQ056 are safe and effective in treating the behavioral symptoms of Fragile X Syndrome.

status: Completed

study_completion_date_type: Actual

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Actual

ReferenceType: derived

ReferenceCitation: Berry-Kravis E, Des Portes V, Hagerman R, Jacquemont S, Charles P, Visootsak J, Brinkman M, Rerat K, Koumaras B, Zhu L, Barth GM, Jaecklin T, Apostol G, von Raison F. Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials. Sci Transl Med. 2016 Jan 13;8(321):321ra5. doi: 10.1126/scitranslmed.aab4109.

family: Berry-Kravis

full_name: Elizabeth Berry-Kravis

given: Elizabeth

sequence: first

family: Des Portes

full_name: Vincent Des Portes

given: Vincent

sequence: additional

family: Hagerman

full_name: Randi Hagerman

given: Randi

family: Jacquemont

full_name: Sébastien Jacquemont

given: Sébastien

family: Charles

full_name: Perrine Charles

given: Perrine

family: Visootsak

full_name: Jeannie Visootsak

given: Jeannie

family: Brinkman

full_name: Marc Brinkman

given: Marc

family: Rerat

full_name: Karin Rerat

family: Koumaras

full_name: Barbara Koumaras

given: Barbara

family: Zhu

full_name: Liansheng Zhu

given: Liansheng

family: Barth

full_name: Gottfried Maria Barth

given: Gottfried Maria

family: Jaecklin

full_name: Thomas Jaecklin

given: Thomas

family: Apostol

full_name: George Apostol

given: George

family: von Raison

full_name: Florian von Raison

given: Florian

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Science Translational Medicine

publisher: American Association for the Advancement of Science (AAAS)

lead_sponsor: Novartis Pharmaceuticals

location_country:
Italy
United States
Switzerland
Germany
France
Australia
Spain
Denmark
Canada
United Kingdom

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Completed


title: A Phase 3, Open-label Study of the Safety and Effectiveness of AA4500 Administered Twice Per Treatment Cycle for up to Four Treatment Cycles (2 x 4) in Men With Peyronie's Disease

summary:
This study is a Phase 3, open-label study of the safety and efficacy of AA4500 0.58 mg in subjects with Peyronie's disease. Subjects will be screened for study eligibility within 21 days before the initial injection of study drug in the first treatment cycle.

After up to four treatment cycles, each subject will be followed for additional safety and efficacy assessments on Days 168 (± 7 days) and 252 (± 7 days) (nominal weeks 24 and 36).

After the final injection of each treatment cycle, the investigator will model the plaque in an attempt to stretch or elongate the plaque. If the subject's penile curvature is reduced to <15° after the first, second, or third cycle of injections or if the investigator determines further treatment is not clinically indicated (eg, adverse events; allergic reaction), subsequent treatment cycles will not be administered.

Approximately 300 subjects will be enrolled.

ReferenceCitation: Levine LA, Cuzin B, Mark S, Gelbard MK, Jones NA, Liu G, Kaufman GJ, Tursi JP, Ralph DJ. Clinical safety and effectiveness of collagenase clostridium histolyticum injection in patients with Peyronie's disease: a phase 3 open-label study. J Sex Med. 2015 Jan;12(1):248-58. doi: 10.1111/jsm.12731. Epub 2014 Nov 12.

family: Levine

full_name: Laurence A. Levine

given: Laurence A.

family: Cuzin

full_name: Beatrice Cuzin

given: Beatrice

family: Mark

full_name: Stephen Mark

given: Stephen

family: Gelbard

full_name: Martin K. Gelbard

given: Martin K.

family: Jones

full_name: Nigel A. Jones

given: Nigel A.

family: Liu

full_name: Genzhou Liu

given: Genzhou

family: Kaufman

full_name: Gregory J. Kaufman

given: Gregory J.

family: Tursi

full_name: James P. Tursi

given: James P.

family: Ralph

full_name: David J. Ralph

given: David J.

journal_name: The Journal of Sexual Medicine

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

title: Clinical Safety and Effectiveness of Collagenase Clostridium Histolyticum Injection in Patients with Peyronie's Disease: A Phase 3 Open‐Label Study

lead_sponsor: Endo Pharmaceuticals

location_country:
Italy
Sweden
Germany
France
New Zealand
Spain
Denmark
United States
United Kingdom

location_facility: Aarhus Universitiy Hospital, Aalborg Sygehus

location_facility: Waitemata Urology Research North Shore Hospital

location_facility: Tristate Urologic Services PSC, Inc./ dba TUG Research

location_facility: Urology of Virginia-Sentara Medical Group

location_facility: Universitatsklinikum Freiburg, Abteilung Urologie

location_facility: Connecticut Clinical Research Center, LLC

location_facility: Maimonides Medical Center, Division of Urology

intervention_type: Biological


title: Safety and Efficacy Study Comparing a New hMG Formulation (hMG-IBSA) to a Reference Product (Menopur®) in Patients Undergoing Ovarian Stimulation for in Vitro Fertilisation (IVF)

summary: The purpose of the non-inferiority study is to evaluate the clinical efficacy and the safety of two different subcutaneous hMG preparations when administered to patients undergoing controlled ovarian stimulation for IVF.

lead_sponsor: IBSA Institut Biochimique SA

location_country:
Switzerland
France
Hungary
Denmark
United Kingdom

location_facility: Groupe Hospitalier Cochin - Saint Vincent de Paul

location_facility: First Dept. Obstetric and Gynaecology, Semmelweiss University


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

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

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

study_type: Observational

lead_sponsor: Integra LifeSciences Services

location_country:
Austria
Germany
France
Spain
Canada
Korea, Republic of

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


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

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

status: Terminated

lead_sponsor: Mundipharma Research Limited

location_country:
Italy
Austria
United Kingdom
France
Germany
Czech Republic

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

status_simplified: Unknown


title: A Phase 2, Multicenter, Double-Blinded, Placebo-Controlled, Multiple-Dose Study to Evaluate Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

summary: The purpose of this study is to evaluate of the safety, efficacy, pharmacokinetics (PK) and pharmacodynamic (PD) effects of lumacaftor (VX-809) alone and when coadministered with ivacaftor (VX-770) in participants with cystic fibrosis, homozygous or heterozygous for the F508del-CFTR mutation.

ReferenceCitation: Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12:CD010966. doi: 10.1002/14651858.CD010966.pub3.

name: Department of Women's and Children's Health; University of Liverpool; Liverpool UK

family: Southern

full_name: Kevin W Southern

given: Kevin W

family: Murphy

full_name: Jared Murphy

given: Jared

family: Sinha

full_name: Ian P Sinha

given: Ian P

name: Department of Biostatistics; University of Liverpool; Liverpool UK

family: Nevitt

full_name: Sarah J Nevitt

given: Sarah J

journal_name: Cochrane Database of Systematic Reviews

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

ReferenceCitation: Rowe SM, McColley SA, Rietschel E, Li X, Bell SC, Konstan MW, Marigowda G, Waltz D, Boyle MP; VX09-809-102 Study Group. Lumacaftor/Ivacaftor Treatment of Patients with Cystic Fibrosis Heterozygous for F508del-CFTR. Ann Am Thorac Soc. 2017 Feb;14(2):213-219. doi: 10.1513/AnnalsATS.201609-689OC.

ReferenceCitation: Boyle MP, Bell SC, Konstan MW, McColley SA, Rowe SM, Rietschel E, Huang X, Waltz D, Patel NR, Rodman D; VX09-809-102 study group. A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. Lancet Respir Med. 2014 Jul;2(7):527-38. doi: 10.1016/S2213-2600(14)70132-8. Epub 2014 Jun 24.

family: Boyle

full_name: Michael P Boyle

given: Michael P

family: Bell

full_name: Scott C Bell

given: Scott C

family: Konstan

full_name: Michael W Konstan

given: Michael W

family: McColley

full_name: Susanna A McColley

given: Susanna A

family: Rowe

full_name: Steven M Rowe

given: Steven M

family: Rietschel

full_name: Ernst Rietschel

given: Ernst

family: Huang

full_name: Xiaohong Huang

given: Xiaohong

family: Waltz

full_name: David Waltz

given: David

family: Patel

full_name: Naimish R Patel

given: Naimish R

family: Rodman

full_name: David Rodman

journal_name: The Lancet Respiratory Medicine

lead_sponsor: Vertex Pharmaceuticals Incorporated

location_country:
Germany
France
Belgium
Australia
New Zealand
United States
United Kingdom


study_start_date_type: Actual

lead_sponsor: Actelion

location_country:
Italy
South Africa
India
Serbia
Mexico
Netherlands
Germany
Spain
France
Czechia
Poland
Israel
Belarus
Australia
Hungary
Russian Federation
China
United States
Ukraine

acronym: FUTURE 3

location_facility: Instituto Nacional de Cardiologia (INC) Ignacio Chavez - Site 8401

location_facility: Columbia University Medical Center Children's Hospital of New York Presbyterian - Site 9101

location_facility: Instytut Pomnik - Centrum Zdrowia Dziecka Klinika Kardiologii Dziecięcej - Site 3601

location_facility: Fakultní nemocnice v Motole, dětské kardiocentrum - Site 3301

location_facility: Royal Children's Hospital Melbourne, Cardiology - Site 5001

location_facility: Cardiovascular Institute and Fuwai Hospital

location_facility: Children's National Medical Center - Site 9104

location_facility: Clinical Diagnostic Center - Pediatric Cardiovascular and ANES and Intensive Care Department - Site 4103

location_facility: Gover INS - Scientific Practical Cardiovascular Pediatric Center - MOH Ukraine - Site 4102

location_facility: Ospedale Pediatrico "Bambino Gesù" - Dipartimento Medico Chirurgico di Cardiologia Pediatrica - Site 1502

location_facility: West China 2nd university Hospital-Center of interventional diagnosis and therapy for Children's cardiovascular disease - Site 5104

location_facility: Hospital Universitatario Vall d'Hebron, Neumologia - Site 1907

location_facility: Scientific Center of Cardiovascular Surgery named after A.N.Bakulev of the RAMS - Site 3803

location_facility: Szegedi Tudományegyetem ÁOK Szent-Györgyi Albert Klinikai Központ, Gyermekgyógyászati Klinika és Gyermekegészségügyi Központ - Site 3402

location_facility: Moscow Scientific Research Institute for Pediatrics and Childrens Surgery of Rosmedtechnologies - Site 3804

location_facility: Institut za zdravstvenu zaštitu majke i deteta Srbije "Dr Vukan Čupić", Služba za ispitivanje i lečenje bolesti srca i krvnih sudova - Site 3902

location_facility: Shanghai Pulmonary Hospital, Department of Pulmonary Circulation - Site 5101

location_facility: Paediatric Cardiology Albert Luthuli Central Hospital - Site 6003

location_facility: Indraprashta Apollo Hospitals, Pediatric Cardiology - Site 5303

location_facility: Hospital Universitario La Paz - Paediatric Cardiology Department - Site 1906

location_facility: Gusak Ins Urgent and Recovery SUR AMS - Cardiovascular Rehabilitation Pediatric Department - Site 4101

location_facility: Università Degli Studi di Padova - Dipartimento di Pediatria - Servizio di Cardiologia Pediatrica - Site 1501

location_facility: Instytut Centrum Zdrowia Matki Polki Klinika Kardiologii ICMP w Lodz - Site 3602

location_facility: Department of Paediatric Cardiology University of the Free State - Site 6001

location_facility: Universitair Medish Centrum Groningen, Kindercardiologie - Site 1601

location_facility: Beijing Anzhen Hospital, Capital Medical University- Department of Pediatric Cardiology - Site 5103

location_facility: Univerzitetska dečja klinika, Služba za kardiologiju - Site 3901

location_facility: Unidad de Investigacion Clinica en Medicina, SC (UDICEM) - Site 8402

location_facility: Division of Paediatric Cardiology, Steve Biko Academic Hospital - Site 6002

location_facility: Wojewódzki Szpital Specjalistyczny we Wrocławiu Oddział Kardiologii Dziecięcej z pododdziałem Intensywnego Nadzoru Kardiologicznego - Site 3605

location_facility: The Republican Scientific-Practical Center "Cardiology" - Site 3001

location_facility: Universitätsklinikum Bonn Abteilung für Kinderkardiologie - Site 1404

location_facility: Shanghai Children's Medical Center - Site 5102

location_facility: Gottsegen György Országos Kardiológiai Intézet, Gyermekszív Központ, Gyermek Kardiológiai osztály - Site 3401

location_facility: Texas Children's Hospital - Department of Cardiology - Site 9107

location_facility: Uniwersyteckie Centrum Kliniczne Klinika Kardiologii Dziecięcej i Wad Wrodzonych Serca - Site 3604

location_facility: Hopital Necker-Enfants Malades, Service de Cardiologie Pédiatrique - Site 2201

location_facility: Deutsches Herzzentrum Kinderkardiologie - Site 1401

location_facility: RAMS Institution, Research Institute for complex issues of cardiovascular diseases, Siberian branch of the Russian Academy of Medical Sciences - Site 3805

location_facility: State Educational Institution of Higher Professional Education "Saint Petersburg State Pediatric Medical Academy of Roszdrav" - Site 3801

location_facility: Justus-Liebig-Universität Giessen, Kinderherzzentrum - Site 1403

location_facility: CARE Hospitals, Cardiology Dep. Hyderabad - Site 5302

location_facility: CHU de Toulouse - Hôpital des Enfants, Service de Cardiologie Pédiatrique - Site 2202

location_facility: Federal State Institution "Federal center of Heart, Blood and Endocrinology named after V.A.Almazov Rosmedtekhnologies" - Site 3802

location_facility: Schneider Children's Medical Center- Institute of pediatric cardiology - Site 7101

source: euctr

title: An Open-label, Prospective Multicenter Study to Assess the Pharmacokinetics, Tolerability, Safety and Efficacy of the Pediatric Formulation of Bosentan Two Versus Three Times a Day in Children With Pulmonary Arterial Hypertension

summary: The primary objective of AC-052-373 was to assess the pharmacokinetic (PK) profile of two dosing regimens of the pediatric formulation of bosentan in children with pulmonary arterial hypertension (PAH) <12 years of age.


title: STA®-Liatest®D-Di-Exclusion of Venous Thromboembolism

acronym: DiET

summary: The purpose of this study is to demonstrate the ability of STA® Liatest® D-Di combined with a clinical pretest probability (PTP) to safely exclude pulmonary embolism (PE) or Deep Venous Thrombosis (DVT) in a 3 month follow-up.

time_perspective: Prospective

ReferenceType: background

ReferenceCitation: Keeling DM, Mackie IJ, Moody A, Watson HG; Haemostasis and Thrombosis Task Force of the British Committee for Standards in Haematology. The diagnosis of deep vein thrombosis in symptomatic outpatients and the potential for clinical assessment and D-dimer assays to reduce the need for diagnostic imaging. Br J Haematol. 2004 Jan;124(1):15-25.

ReferenceCitation: Goekoop RJ, Steeghs N, Niessen RW, Jonkers GJ, Dik H, Castel A, Werker-van Gelder L, Vlasveld LT, van Klink RC, Planken EV, Huisman MV. Simple and safe exclusion of pulmonary embolism in outpatients using quantitative D-dimer and Wells' simplified decision rule. Thromb Haemost. 2007 Jan;97(1):146-50.

ReferenceCitation: Kelly J, Hunt BJ. A clinical probability assessment and D-dimer measurement should be the initial step in the investigation of suspected venous thromboembolism. Chest. 2003 Sep;124(3):1116-9. Review.

ReferenceCitation: Pasha SM, Klok FA, Snoep JD, Mos IC, Goekoop RJ, Rodger MA, Huisman MV. Safety of excluding acute pulmonary embolism based on an unlikely clinical probability by the Wells rule and normal D-dimer concentration: a meta-analysis. Thromb Res. 2010 Apr;125(4):e123-7. doi: 10.1016/j.thromres.2009.11.009. Epub 2009 Nov 26.

ReferenceCitation: Perrier A, Desmarais S, Goehring C, de Moerloose P, Morabia A, Unger PF, Slosman D, Junod A, Bounameaux H. D-dimer testing for suspected pulmonary embolism in outpatients. Am J Respir Crit Care Med. 1997 Aug;156(2 Pt 1):492-6.

ReferenceCitation: Wells PS, Anderson DR, Rodger M, Forgie M, Kearon C, Dreyer J, Kovacs G, Mitchell M, Lewandowski B, Kovacs MJ. Evaluation of D-dimer in the diagnosis of suspected deep-vein thrombosis. N Engl J Med. 2003 Sep 25;349(13):1227-35.

ReferenceCitation: Pernod G, Caterino J, Maignan M, Tissier C, Kassis J, Lazarchick J; DIET study group. D-Dimer Use and Pulmonary Embolism Diagnosis in Emergency Units: Why Is There Such a Difference in Pulmonary Embolism Prevalence between the United States of America and Countries Outside USA? PLoS One. 2017 Jan 13;12(1):e0169268. doi: 10.1371/journal.pone.0169268. eCollection 2017.

family: Pernod

full_name: Gilles Pernod

given: Gilles

family: Caterino

full_name: Jeffrey Caterino

given: Jeffrey

family: Maignan

full_name: Maxime Maignan

given: Maxime

family: Tissier

full_name: Cindy Tissier

given: Cindy

family: Kassis

full_name: Jeannine Kassis

given: Jeannine

family: Lazarchick

full_name: John Lazarchick

given: John

currency_apc_doaj: USD

journal_name: PLOS ONE

oa_colors: green gold

oa_host_type: publisher;repository

evidence: oa journal (via doaj)

host_type: publisher

license: cc-by

license_normalized: cc-by

version: publishedVersion

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

host_type: repository

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

evidence: oa repository (via pmcid lookup)

license_normalized: no license

evidence: oa repository (semantic scholar lookup)

publisher: Public Library of Science (PLoS)

publisher_dissemination: Public Library of Science

publisher_group: Public Library of Science

publisher_normalized: Public Library of Science

lead_sponsor: Diagnostica Stago

location_country:
Italy
France
Spain
United States
Canada

location_facility:
General Hospital
Brigham and Woman's Hosp
Moses Cone Memorial Hospital
Maisonneuve Rosemont Hospital
Oklahoma Health Sciences Center
Medical Center of South Carolina
Univeristy Medical Center
Texas Health Fort Worth Hospital
Northwestern Medical Center
University Hospital
University of Iowa
Lehigh Valley Hospital Health Network
Indiana University Health
Massachusetts General Hospital
IRCCS H San Raffaele


title: Testing the Usefulness of Lantus When Initiated Prematurely In Patients With Type 2 Diabetes

summary:
Primary objective:

The primary objective of this trial is:

To evaluate the efficacy of initiating Lantus in combination with oral antidiabetics drugs compared to oral antidiabetic treatment optimised by enhancing hygienic and dietary measures in type 2 diabetics whose blood glucose control is acceptable but not optimal on maximum oral treatment, based on the number of patients achieving a HbA1c value < 7% at the end of treatment.

Secondary objectives:

The secondary objectives of this trial are to compare between the two treatment groups:

The variation in HbA1c between baseline and end of trial.
The frequency of episodes of symptomatic hypoglycaemia (diurnal and nocturnal), severe hypoglycaemia (diurnal and nocturnal) and asymptomatic hypoglycaemia.
Mean blood glucose levels at different times of the day.
The variation in weight and lipid in each group between baseline and end of trial.
The incidence of adverse events.

lead_sponsor: Sanofi

location_country:
Croatia
France
Slovenia
Romania
Russian Federation
Czech Republic


title: A Multi-centre Study to Define Novel Individualised Dosing Regimens to Maximise Antibiotic Effectiveness for Treatment of Pneumonia in Intensive Care Units

acronym: PNEUDOS

summary:
Pneumonia is the most common infection in intensive care unit (ICU) patients and occurs in 10% of all ICU admissions. Unfortunately, ICU patient outcomes remain poor with a high mortality rate associated with pneumonia despite recent therapeutic advances.

Previous studies of antibiotics used in ICU patients, which includes ceftriaxone, meropenem and piperacillin/tazobactam, have quantified major differences in pharmacokinetics (PK) between ICU and non-ICU patients, with ICU patients displaying a unique spectrum of plasma concentration-time profiles. These PK differences can lead to suboptimal antibiotic concentrations in blood, which have been associated with a reduced likelihood of clinical cure for pneumonia. Furthermore, highlighting the importance of optimised dosing for pneumonia is that multi-drug resistant (MDR) pathogens emerge during antibiotic therapy in approximately half of the ICU patients, frequently emerging from the lung.

Previous work has highlighted how infection site concentrations determine patient outcome. For pneumonia, the infection site is best described as the epithelial lining fluid (ELF) in the lung.

Although optimal antibiotic therapy should be considered a priority for ICU patients with pneumonia to improve the persisting poor outcomes, the dosing regimens that can achieve therapeutic concentrations at the infection site (i.e., ELF) in ICU patients with pneumonia remain unknown.

The PNEUDOS study aims to address this significant knowledge gap by defining novel individualised dosing regimens that can maximise antibiotic efficacy by achieving therapeutic concentrations in the blood and ELF of ICU patients with pneumonia. These dosing regimens can then be validated in future clinical trials.

status: Recruiting

study_completion_date_type: Anticipated

enrollment_type: Anticipated

ReferenceCitation: Abdul-Aziz MH, Alffenaar JC, Bassetti M, Bracht H, Dimopoulos G, Marriott D, Neely MN, Paiva JA, Pea F, Sjovall F, Timsit JF, Udy AA, Wicha SG, Zeitlinger M, De Waele JJ, Roberts JA; Infection Section of European Society of Intensive Care Medicine (ESICM); Pharmacokinetic/pharmacodynamic and Critically Ill Patient Study Groups of European Society of Clinical Microbiology and Infectious Diseases (ESCMID); Infectious Diseases Group of International Association of Therapeutic Drug Monitoring and Clinical Toxicology (IATDMCT); Infections in the ICU and Sepsis Working Group of International Society of Antimicrobial Chemotherapy (ISAC). Antimicrobial therapeutic drug monitoring in critically ill adult patients: a Position Paper(.). Intensive Care Med. 2020 Jun;46(6):1127-1153. doi: 10.1007/s00134-020-06050-1. Epub 2020 May 7. Review.

ipd_sharing: Undecided

lead_sponsor: The University of Queensland

location_country:
Hong Kong
France
Malaysia
Belgium
Australia

location_facility: Centre Hospitalier Universitaire de Nimes

intervention_type: Other

lead_sponsor_type: academique

status_simplified: Ongoing


title: Phase 1/2a Open Label, Multicenter Clinical Trial of a Novel Small Molecule EBNA1 Inhibitor, VK 2019, in Patients With Epstein Barr Virus Positive Nasopharyngeal Cancer, With Pharmacokinetic and Pharmacodynamic Correlative Studies

summary: VK-2019-001 is a 1/2a trial of the oral EBNA-1 targeting agent VK-2019 in patients with EBV-positive recurrent or metastatic NPC to determine the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D), as well as to evaluate the PK profile of VK-2019.

design_allocation: Non-Randomized

ReferenceCitation: Young LS. Epstein-Barr virus at 50-future perspectives. Chin J Cancer. 2014 Nov;33(11):527-8. doi: 10.5732/cjc.014.10208.

ReferenceCitation: Young LS, Dawson CW. Epstein-Barr virus and nasopharyngeal carcinoma. Chin J Cancer. 2014 Dec;33(12):581-90. doi: 10.5732/cjc.014.10197. Epub 2014 Nov 21.

ReferenceCitation: Tsao SW, Tsang CM, Lo KW. Epstein-Barr virus infection and nasopharyngeal carcinoma. Philos Trans R Soc Lond B Biol Sci. 2017 Oct 19;372(1732). pii: 20160270. doi: 10.1098/rstb.2016.0270. Review.

ipd_sharing: No

lead_sponsor: Cullinan Oncology, LLC

location_country:
Hong Kong
France
Singapore
China
United States

location_facility: Hong Kong University - Queen Mary Hospital

location_facility: The University of Texas - MD Anderson Cancer Center

location_facility: Stanford University, School of Medicine, Stanford Cancer Institute


title: A Prospective Observational, Long Term Follow up Study of Patients Treated With Imlifidase Prior to Kidney Transplantation

summary:
The rationale for the current protocol is to collect data from extended follow up in subjects that have received a kidney transplant following imlifidase dosing to provide a better understanding regarding the long-term outcome for these subjects. Data of parameters such as patient and graft survival, comorbidity, treatment of graft rejection episodes and quality of life as well as anti-drug antibody levels will be collected.

This prospective, observational follow up study of subjects who have received imlifidase prior to kidney transplantation will provide important data to future prescribers and patients of the potential long-term benefits of imlifidase mediated transplantation.

status: Active, not recruiting

lead_sponsor: Hansa Biopharma AB

location_country:
United States
Sweden
France

location_facility:
Uppsala University Hospital
New York University School of Medicine
Necker Hospital
Karolinska University Hospital
Cedars-Sinai Medical Center
The Johns Hopkins Hospital


title: A Multicentre Open-label Phase IIa Study With Escalating Dose of MS1819- SD, to Investigate the Efficacy and Safety of a Yarrowia Lipolytica Lipase Preparation for the Compensation of Exocrine Pancreatic Insufficiency Caused by Chronic Pancreatitis and/or Distal Pancreatectomy

summary:
This is a Phase IIa study sponsored by AzurRx SAS and Syneos Health is a local representative sponsor and involves testing of a new medication for the compensation of exocrine pancreatic insufficiency (EPI) caused by chronic pacreatitis (CP) and/or distal pancreatectomy. The new medication is called MS1819 Spray Dried (MS1819-SD) which is a lipase produced by the LIP2 gene of Yarrowia lipolytica using recombinant DNA technology.

The primary purpose of this study is to investigate the safety of escalating doses of study drug MS1819-SD in people with chronic pancreatitis. This enzyme has demonstrated an appropriate profile to compensate the pancreatic lipase (enzyme) deficiency that is common with CP patients. The deficiency in this enzyme can be responsible of greasy diarrhea, fecal urge and weight loss.

The design of the study is open-label, meaning that all eligible participants will receive the study drug MS1819-SD. The MS1819-SD dose will increase throughout the study during dose escalation visits in each treatment period; study includes a total of four treatment periods.

The total duration of the MS1819-SD treatment phase is of 48-60 days, The total duration of patient participation in the study is of 74-93 days.

Approximately twelve patients will be enrolled in this study.

lead_sponsor: AzurRx SAS

location_country:
Australia
France
New Zealand

location_facility: Mackay Institute of Research and Innovation


title: Long Term Follow-up of Integra® Cadence™ Total Ankle System in Primary Ankle Joint Replacement

summary: This study will evaluate the long term performance and safety data for the Cadence™ Total Ankle System (CTAS) when used for primary arthroplasty in patients with primary arthritis (e.g. degenerative disease), secondary arthritis (e.g. post-traumatic, avascular necrosis, if minimally 2/3 of the talus is preserved), and systemic arthritis of the ankle (e.g. rheumatoid arthritis, hemochromatosis)

lead_sponsor: Smith & Nephew, Inc.

location_country:
France
Belgium
Spain
Canada
United Kingdom

location_facility:
Foot and Ankle Institute
Clinica Nostra Senhora del Remei
St. Michael's Hospital
Universitair Ziekenhuis Gent
North Cumbria University Hospitals
CHRU Tours

intervention_type: Device


ReferenceType: result

ReferenceCitation: Benjamin R, Graham C, Yallop D, Jozwik A, Mirci-Danicar OC, Lucchini G, Pinner D, Jain N, Kantarjian H, Boissel N, Maus MV, Frigault MJ, Baruchel A, Mohty M, Gianella-Borradori A, Binlich F, Balandraud S, Vitry F, Thomas E, Philippe A, Fouliard S, Dupouy S, Marchiq I, Almena-Carrasco M, Ferry N, Arnould S, Konto C, Veys P, Qasim W; UCART19 Group. Genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor T cells in paediatric and adult B-cell acute lymphoblastic leukaemia: results of two phase 1 studies. Lancet. 2020 Dec 12;396(10266):1885-1894. doi: 10.1016/S0140-6736(20)32334-5.

family: Benjamin

full_name: Reuben Benjamin

given: Reuben

family: Graham

full_name: Charlotte Graham

given: Charlotte

family: Yallop

full_name: Deborah Yallop

given: Deborah

family: Jozwik

full_name: Agnieszka Jozwik

given: Agnieszka

family: Mirci-Danicar

full_name: Oana C Mirci-Danicar

given: Oana C

family: Lucchini

full_name: Giovanna Lucchini

given: Giovanna

family: Pinner

full_name: Danielle Pinner

given: Danielle

family: Jain

full_name: Nitin Jain

given: Nitin

family: Kantarjian

full_name: Hagop Kantarjian

given: Hagop

family: Boissel

full_name: Nicolas Boissel

given: Nicolas

family: Maus

full_name: Marcela V Maus

given: Marcela V

family: Frigault

full_name: Matthew J Frigault

given: Matthew J

family: Baruchel

full_name: André Baruchel

given: André

family: Mohty

full_name: Mohamad Mohty

given: Mohamad

family: Gianella-Borradori

full_name: Athos Gianella-Borradori

given: Athos

family: Binlich

full_name: Florence Binlich

given: Florence

family: Balandraud

full_name: Svetlana Balandraud

given: Svetlana

family: Vitry

full_name: Fabien Vitry

given: Fabien

family: Thomas

full_name: Elisabeth Thomas

given: Elisabeth

family: Philippe

full_name: Anne Philippe

given: Anne

family: Fouliard

full_name: Sylvain Fouliard

given: Sylvain

family: Dupouy

full_name: Sandra Dupouy

given: Sandra

family: Marchiq

full_name: Ibtissam Marchiq

given: Ibtissam

family: Almena-Carrasco

full_name: Maria Almena-Carrasco

given: Maria

family: Ferry

full_name: Nicolas Ferry

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

Access can be requested for all interventional clinical studies:

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

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

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

lead_sponsor: Institut de Recherches Internationales Servier

location_country:
United States
United Kingdom
France
Spain

location_facility: University of Texas Southwestern Medical Center

title: A Phase 1, Open Label, Non-comparative Study to Evaluate the Safety and the Ability of UCART19 to Induce Molecular Remission in Paediatric Patients With Relapsed/Refractory B-cell Acute Lymphoblastic Leukaemia

summary: This study aims to evaluate the safety and feasibility of UCART19 to induce molecular remission in pediatric patients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia (B-ALL).


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

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

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

lead_sponsor: Abivax S.A.

location_country: Belgium France Spain

location_facility: Hospital Universitari Germans Trias i Pujo

location_facility: CHU de Montpellier - Hôpital Gui de Chauliac


title: A Phase 2a, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of AMG 714 in Adult Patients With Type II Refractory Celiac Disease, an In Situ Small Bowel T Cell Lymphoma

summary: Protocol CELIM-RCD-002 is designed to evaluate the efficacy and safety of AMG 714 for the treatment of adult patients with type II refractory celiac disease (RCD-II), an in-situ small bowel T-cell lymphoma.

ReferenceCitation: Cellier C, Bouma G, van Gils T, Khater S, Malamut G, Crespo L, Collin P, Green PHR, Crowe SE, Tsuji W, Butz E, Cerf-Bensussan N, Macintyre E, Parnes JR, Leon F, Hermine O, Mulder CJ; RCD-II Study Group Investigators. Safety and efficacy of AMG 714 in patients with type 2 refractory coeliac disease: a phase 2a, randomised, double-blind, placebo-controlled, parallel-group study. Lancet Gastroenterol Hepatol. 2019 Dec;4(12):960-970. doi: 10.1016/S2468-1253(19)30265-1. Epub 2019 Sep 4.

family: Cellier

full_name: Christophe Cellier

given: Christophe

family: Bouma

full_name: Gerd Bouma

given: Gerd

family: van Gils

full_name: Tom van Gils

given: Tom

family: Khater

full_name: Sherine Khater

given: Sherine

family: Malamut

full_name: Georgia Malamut

given: Georgia

family: Crespo

full_name: Laura Crespo

given: Laura

family: Collin

full_name: Pekka Collin

given: Pekka

family: Green

full_name: Peter H R Green

given: Peter H R

family: Crowe

full_name: Sheila E Crowe

given: Sheila E

family: Tsuji

full_name: Wayne Tsuji

given: Wayne

family: Butz

full_name: Eric Butz

given: Eric

family: Cerf-Bensussan

full_name: Nadine Cerf-Bensussan

given: Nadine

family: Macintyre

full_name: Elizabeth Macintyre

given: Elizabeth

family: Parnes

full_name: Jane R Parnes

given: Jane R

family: Leon

full_name: Francisco Leon

given: Francisco

family: Hermine

full_name: Olivier Hermine

given: Olivier

family: Mulder

full_name: Chris J Mulder

given: Chris J

family: Jabri

full_name: Bana Jabri

given: Bana

family: Murray

full_name: Joseph Murray

given: Joseph

family: DiMarino

full_name: Anthony DiMarino

given: Anthony

family: Kelly

full_name: Ciaran P Kelly

given: Ciaran P

family: Byrnes

full_name: Valerie Byrnes

given: Valerie

family: Sanders

full_name: David Sanders

given: David

journal_name: The Lancet Gastroenterology & Hepatology

location_country:
Netherlands
Finland
France
Spain
United States


title: Dasatinib (BMS-354825) Combined With SMO Inhibitor (BMS-833923; XL139) in CML With Resistance or Suboptimal Response to a Prior TKI

summary: The purpose of the study is to determine the safety and tolerability of the combination of BMS-833923 plus dasatinib in patients with chronic myeloid leukemia.

lead_sponsor: Bristol-Myers Squibb

location_country:
Italy
United States
Finland
France
Germany
United Kingdom
Canada

location_facility: Sidney Kimmel Cancer Center At Johns Hopkins


title: International Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of the Efficacy and Safety of KIACTA in Preventing Renal Function Decline in Patients With AA Amyloidosis

summary: The primary purpose of this study is to assess the efficacy and safety of treatment with Kiacta in adult patients with AA Amyloidosis.

primary_purpose: Prevention

lead_sponsor: C.T. Development America, Inc.

location_country:
Peru
Finland
Belgium
Russian Federation
Ukraine
India
France
Poland
Lithuania
Turkey
Sweden
Netherlands
Germany
United States
United Kingdom
Italy
Israel
Georgia
Tunisia
Egypt
Spain
Estonia
Latvia

location_facility: National Scientific Center "Institute of cardiology n.a. academician M.D Strazhesko"

location_facility: State Institution "Institute of Nephrology of AMS of Ukraine"

location_facility: Karolinska Universitetssjukhuset i Huddinge

location_facility: Sverdlovsk Regional Clinical Hospital #1

location_facility: Helsingin yliopistollinen keskussairaala / Meilahti

location_facility: Research Institute of Clinical and Experimental Lymphology

location_facility: Municipal Medical & Preventive Institution Donetsk Regional Clinical Territorial Medical Association

location_facility: Vilnius University Hospital Santariskiu Klinikos

location_facility: Pauls Stradins Clinical University Hospital

location_facility: Akademicki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu

location_facility: Kemerovo State Medical Academy of Roszdrav

location_facility: Eskisehir Osmangazi University Medical Faculty

location_facility: Cukurova University Medical Faculty Balcali Hospital

location_facility: Hospital of Lithuanian University of Health Sciences Kaunas Clinics


title: Post-Myocardial Infarction Remodeling Prevention Therapy

acronym: PRomPT

summary: The purpose of this study is to demonstrate the feasibility of pacing as a therapy to prevent adverse remodeling of the myocardium following an acute myocardial infarction (MI) in patients at highest risk for adverse myocardial remodeling.

ReferenceCitation: Stone GW, Chung ES, Stancak B, Svendsen JH, Fischer TM, Kueffer F, Ryan T, Bax J, Leon A; PRomPT Trial Investigators. Peri-infarct zone pacing to prevent adverse left ventricular remodelling in patients with large myocardial infarction. Eur Heart J. 2016 Feb 1;37(5):484-93. doi: 10.1093/eurheartj/ehv436. Epub 2015 Aug 30.

family: Stone

full_name: Gregg W. Stone

given: Gregg W.

family: Chung

full_name: Eugene S. Chung

given: Eugene S.

family: Stancak

full_name: Branislav Stancak

given: Branislav

family: Svendsen

full_name: Jesper H. Svendsen

given: Jesper H.

family: Fischer

full_name: Trent M. Fischer

given: Trent M.

family: Kueffer

full_name: Fred Kueffer

given: Fred

family: Ryan

full_name: Thomas Ryan

given: Thomas

family: Bax

full_name: Jeroen Bax

given: Jeroen

full_name: Angel Leon

given: Angel

journal_name: European Heart Journal

oa_host_type: publisher

evidence: open (via free pdf)

publisher: Oxford University Press (OUP)

publisher_dissemination: Oxford University Press

publisher_group: Oxford University Press

publisher_normalized: Oxford University Press

lead_sponsor: Medtronic Cardiac Rhythm and Heart Failure

location_country:
Saudi Arabia
Germany
France
Slovakia
Hungary
Denmark
United States

location_facility: Hospital of the University of Pennsylvania

location_facility: Vychodoslovensky ustav srdcovych a cievnych chorob, a.s.

location_facility: Baylor Jack and Jane Hamilton Heart and Vascular Hospital

location_facility: Centre Hospitalier Régional Universitaire de Lille


title: A Prospective, Randomized, Intra-patient, Comparative, Open, Multi-centre Study to Evaluate the Efficacy of a Single-Use NPWT System on the Prevention of Incision Healing Complications in Patients Undergoing Reduction Mammoplasty

summary: The purpose of this study is to see if the use of a single-use negative pressure wound therapy system (NPWT) will have an effect on the reduction of post-surgical incision healing complications following breast reduction surgery and to assess the medium-term aesthetic appearance and quality of the resultant scar, compared with standard of care dressings.

location_country:
South Africa
United States
France
Netherlands

location_facility: Northwestern University Feinberg School of Medicine


title: CardioRoot Post-Marketing Surveillance Registry

acronym: CardioRoot

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

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

family: Tasca

full_name: Giordano Tasca

given: Giordano

family: Lindner

full_name: Jaroslav Lindner

given: Jaroslav

family: Barandon

full_name: Laurent Barandon

given: Laurent

family: Santavy

full_name: Petr Santavy

given: Petr

family: Antona

full_name: Carlo Antona

given: Carlo

family: Burkert

full_name: Jan Burkert

given: Jan

family: Gamba

full_name: Amando Gamba

given: Amando

currency_apc_doaj: GBP

journal_name: Journal of Cardiothoracic Surgery

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

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

lead_sponsor: Maquet Cardiovascular

location_country: Italy France Czechia

location_facility: Centre Hospitalier Universitaire (CHU) de Bordeaux


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

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

lead_sponsor: Genzyme, a Sanofi Company

location_country:
United Kingdom
France
Denmark
Germany
Czech Republic


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

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

location_country:
Sweden
Mexico
Finland
France
Argentina
United States


title: A Randomized Phase III Multicenter Trial of Neoadjuvant Docetaxel (Taxotere®) Plus Cisplatin and 5-Fluorouracil (TPF) Versus Neoadjuvant Cisplatin Plus 5-Fluorouracil Followed by Concomitant Chemoradiotherapy to Improve the Overall Survival and Progression Free Survival in Patients With Locally Advanced Squamous Cell Carcinoma of the Head and Neck

summary:
1.To compare overall survival after treatment with the test tri-therapy (TPF: docetaxel plus cisplatin and 5FU) or the control treatment (PF: cisplatin plus 5-FU) followed by chemoradiotherapy in patients with locally advanced SCCHN.
2.The main secondary endpoint is progression free survival (PFS). The other secondary endpoints are to evaluate and compare improvement of local symptoms; time-to-treatment failure; quality of life; clinical complete response rate (CR and CR/PR); toxicity and to evaluate the relationship of tumor markers and response to therapy.

ReferenceCitation: Posner MR, Hershock DM, Blajman CR, Mickiewicz E, Winquist E, Gorbounova V, Tjulandin S, Shin DM, Cullen K, Ervin TJ, Murphy BA, Raez LE, Cohen RB, Spaulding M, Tishler RB, Roth B, Viroglio Rdel C, Venkatesan V, Romanov I, Agarwala S, Harter KW, Dugan M, Cmelak A, Markoe AM, Read PW, Steinbrenner L, Colevas AD, Norris CM Jr, Haddad RI; TAX 324 Study Group. Cisplatin and fluorouracil alone or with docetaxel in head and neck cancer. N Engl J Med. 2007 Oct 25;357(17):1705-15.

ReferenceCitation: Lorch JH, Goloubeva O, Haddad RI, Cullen K, Sarlis N, Tishler R, Tan M, Fasciano J, Sammartino DE, Posner MR; TAX 324 Study Group. Induction chemotherapy with cisplatin and fluorouracil alone or in combination with docetaxel in locally advanced squamous-cell cancer of the head and neck: long-term results of the TAX 324 randomised phase 3 trial. Lancet Oncol. 2011 Feb;12(2):153-9. doi: 10.1016/S1470-2045(10)70279-5. Epub 2011 Jan 11.

family: Lorch

full_name: Jochen H Lorch

given: Jochen H

family: Goloubeva

full_name: Olga Goloubeva

given: Olga

family: Haddad

full_name: Robert I Haddad

given: Robert I

family: Cullen

full_name: Kevin Cullen

given: Kevin

family: Sarlis

full_name: Nicholas Sarlis

given: Nicholas

family: Tishler

full_name: Roy Tishler

given: Roy

family: Tan

full_name: Ming Tan

given: Ming

family: Fasciano

full_name: John Fasciano

given: John

family: Sammartino

full_name: Daniel E Sammartino

given: Daniel E

family: Posner

full_name: Marshall R Posner

given: Marshall R

journal_name: The Lancet Oncology

oa_host_type: repository

version: acceptedVersion

location_country:
United States
France
Argentina
Russian Federation
Portugal
Canada


title: Dose-Ranging Pharmacodynamic Assessment of Platelet Aggregation Inhibition With Clopidogrel in Children of Blalock-Taussig Shunt Age Categories (Neonates and Infants/Toddlers)

summary: PICOLO is a double blind placebo controlled phase II dose ranging, dose escalating study in patients of Blalock-Taussig age categories (neonates and infants/toddlers), to determine the dose providing inhibition of platelet aggregation similar to adults.

ReferenceCitation: Li JS, Yow E, Berezny KY, Bokesch PM, Takahashi M, Graham TP Jr, Sanders SP, Sidi D, Bonnet D, Ewert P, Jennings LK, Michelson AD; PICOLO Investigators. Dosing of clopidogrel for platelet inhibition in infants and young children: primary results of the Platelet Inhibition in Children On cLOpidogrel (PICOLO) trial. Circulation. 2008 Jan 29;117(4):553-9. doi: 10.1161/CIRCULATIONAHA.107.715821. Epub 2008 Jan 14.

name: From the Division of Pediatric Cardiology (J.S.L., E.Y., K.Y.B.), Department of Pediatrics, Duke University Medical Center and Duke Clinical Research Institute, Durham, NC; Children’s Healthcare of Atlanta (P.M.B.), Cardiac Research Department, Atlanta, Ga; Children’s Hospital of Los Angeles (M.T.), Los Angeles, Calif; Division of Cardiology (T.P.G.), Vanderbilt Children’s Hospital, Nashville, Tenn; Ospedale Pediatrico Bambino Gesu (S.P.S.), Rome, Italy; Départment de Pédiatrie (D.S., D.B.),...

family: Li

full_name: Jennifer S. Li

given: Jennifer S.

family: Yow

full_name: Eric Yow

given: Eric

family: Berezny

full_name: Katherine Y. Berezny

given: Katherine Y.

family: Bokesch

full_name: Paula M. Bokesch

given: Paula M.

family: Takahashi

full_name: Matsato Takahashi

given: Matsato

family: Graham

full_name: Thomas P. Graham

given: Thomas P.

suffix: Jr

family: Sanders

full_name: Stephen P. Sanders

given: Stephen P.

family: Sidi

full_name: Daniel Sidi

given: Daniel

family: Bonnet

full_name: Damien Bonnet

given: Damien

family: Ewert

full_name: Peter Ewert

given: Peter

family: Jennings

full_name: Lisa K. Jennings

given: Lisa K.

family: Michelson

full_name: Alan D. Michelson

given: Alan D.

journal_name: Circulation

publisher: Ovid Technologies (Wolters Kluwer Health)

publisher_dissemination: Wolters Kluwer Health

publisher_group: Wolters Kluwer Health

publisher_normalized: Wolters Kluwer Health

title: Dosing of Clopidogrel for Platelet Inhibition in Infants and Young Children

ReferenceCitation: Pasquali SK, Yow E, Jennings LK, Li JS. Platelet activity associated with concomitant use of clopidogrel and proton pump inhibitors in children with cardiovascular disease. Congenit Heart Dis. 2010 Nov-Dec;5(6):552-5. doi: 10.1111/j.1747-0803.2010.00461.x.

family: Pasquali

full_name: Sara K. Pasquali

given: Sara K.

journal_name: Congenital Heart Disease

publisher: Computers, Materials and Continua (Tech Science Press)

title: Platelet Activity Associated with Concomitant Use of Clopidogrel and Proton Pump Inhibitors in Children with Cardiovascular Disease

location_country:
Italy
United States
Germany
France
Belgium
Canada


title: An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of rhGAA Treatment in Patients Greater Than 6 Months and Less Than or Equal to 36 Months Old With Infantile-Onset GSD-II

summary: Glycogen Storage Disease Type II ("GSD-II"; also known as Pompe disease) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with GSD-II, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. This study is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for GSD-II. Patients diagnosed with infantile-onset GSD-II who are greater than 6 months old, but less than or equal to 36 months old will be studied.

ReferenceCitation: Kishnani PS, Goldenberg PC, DeArmey SL, Heller J, Benjamin D, Young S, Bali D, Smith SA, Li JS, Mandel H, Koeberl D, Rosenberg A, Chen YT. Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants. Mol Genet Metab. 2010 Jan;99(1):26-33. doi: 10.1016/j.ymgme.2009.08.003.

family: Kishnani

full_name: Priya S. Kishnani

given: Priya S.

family: Goldenberg

full_name: Paula C. Goldenberg

given: Paula C.

family: DeArmey

full_name: Stephanie L. DeArmey

given: Stephanie L.

family: Heller

full_name: James Heller

given: James

family: Benjamin

full_name: Danny Benjamin

given: Danny

family: Young

full_name: Sarah Young

given: Sarah

family: Bali

full_name: Deeksha Bali

given: Deeksha

family: Smith

full_name: Sue Ann Smith

given: Sue Ann

family: Mandel

full_name: Hanna Mandel

given: Hanna

family: Koeberl

full_name: Dwight Koeberl

given: Dwight

family: Rosenberg

full_name: Amy Rosenberg

given: Amy

family: Chen

full_name: Y-T Chen

given: Y-T

journal_name: Molecular Genetics and Metabolism

location_country:
Israel
United States
United Kingdom
France

location_facility: University of Florida College of Medicine


title: A PHASE I TRIAL OF BUTYRATE AND GANCICLOVIR IN EBV-ASSOCIATED MALIGNANCIES

summary:
RATIONALE: The Epstein Barr virus can cause cancer and lymphoproliferative disorders. Ganciclovir is an antiviral drug that acts against the Epstein Barr virus. Arginine butyrate may make virus cells more sensitive to ganciclovir. Combining ganciclovir and arginine butyrate may kill more Epstein Barr virus cells and tumor cells.

PURPOSE: Phase I trial to study the effectiveness of arginine butyrate plus ganciclovir in treating patients who have cancer or lymphoproliferative disorders that are associated with the Epstein Barr virus.

ReferenceCitation: Faller DV, Mentzer SJ, Perrine SP. Induction of the Epstein-Barr virus thymidine kinase gene with concomitant nucleoside antivirals as a therapeutic strategy for Epstein-Barr virus-associated malignancies. Curr Opin Oncol. 2001 Sep;13(5):360-7. Review.

ReferenceCitation: Mentzer SJ, Perrine SP, Faller DV. Epstein--Barr virus post-transplant lymphoproliferative disease and virus-specific therapy: pharmacological re-activation of viral target genes with arginine butyrate. Transpl Infect Dis. 2001 Sep;3(3):177-85. Review.

ReferenceCitation: Perrine SP, Hermine O, Small T, Suarez F, O'Reilly R, Boulad F, Fingeroth J, Askin M, Levy A, Mentzer SJ, Di Nicola M, Gianni AM, Klein C, Horwitz S, Faller DV. A phase 1/2 trial of arginine butyrate and ganciclovir in patients with Epstein-Barr virus-associated lymphoid malignancies. Blood. 2007 Mar 15;109(6):2571-8. Epub 2006 Nov 21.

ReferenceCitation: Mentzer SJ, Fingeroth J, Reilly JJ, Perrine SP, Faller DV. Arginine butyrate-induced susceptibility to ganciclovir in an Epstein-Barr-virus-associated lymphoma. Blood Cells Mol Dis. 1998 Jun;24(2):114-23.

lead_sponsor: Boston Medical Center

location_country:
Italy
Germany
United States
France

location_facility: Cancer Research Center at Boston Medical Center

location_facility: Istituto Nazionale per lo Studio e la Cura dei Tumori

location_facility: Methodist Cancer Center at Methodist Hospital


title: An Open-label Extension Study Evaluating the Long-term Safety and Efficacy of Oral Inhalation of GB002 for the Treatment of WHO Group 1 Pulmonary Arterial Hypertension (PAH)

summary: This open-label extension study will evaluate the long-term effects of GB002 (seralutinib) in subjects who previously participated in a GB002 PAH study.

lead_sponsor: GB002, Inc., a wholly owned subsidiary of Gossamer Bio, Inc.

location_country:
United States
France
Spain

location_facility: Washington University School of Medicine

location_facility: Lundquist Institute for Biomedical Innovation at Harbor UCLA


title: Repetitive Transcranial Magnetic Stimulation of the Precentral-gyrus in the Relief of Fibromyalgia Pain: an International Multicenter Controlled Adaptative Trial

acronym: TMSFFI

summary: There are currently several points concerning rTMS for pain relief that have a huge impact in the practical application of rTMS in clinical practice, but which have not been approached by previous studies. Also, an even more important issue is the frequency of the sessions in each treatment protocol. The idea of performing stimulation in chronic pain patients on a consecutive daily basis for long-term treatments seem unrealistic and potentially costly. However, it has been reported in animals, healthy subjects and FM patients that the analgesic effects of rTMS can outlast the stimulation session for several minutes and even days. This has led to the proposal that pain treatment with rTMS could be performed in consecutive daily induction sessions, followed by spaced (weekly, fortnightly and even monthly) maintenance sessions. This approach is similar to what is performed routinely in patients with major depression treated by rTMS to the dorsolateral prefrontal cortex, and has successfully been tested in FM patients in a single- center study. Should this approach prove effective in larger prospective multicentric studies, rTMS could enter the armamentarium of non-invasive, non- pharmacological, low-adverse event therapeutic options to relieve fibromyalgia- related pain and associate symptoms. Hence, our aim is to conduct a multicentric international clinical trial on the influence of rTMS in the control of pain in a consecutive adaptive trial design where real rTMS of two different doses (1500 vs 3000 pulses per session) will be studied. Stimulations will be performed in a clinical practice-friendly approach, where daily maintenance sessions will be followed by weekly, and fortnightly stimulation sessions. There have been very scarce adaptive trials in the pain and non-invasive stimulation fields. This methodological approach allows for the use of lower number of patients in consecutive trials and, among other qualities, it allows for the imputation of data from the first trial into the second one, thus decreasing the duration of the studies and the number of participants.

lead_sponsor: University of Sao Paulo

location_country: Brazil France Japan

location_facility: Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo (HC/FMUSP)

location_facility: Department of Neuromodulation Osaka University

location_facility: Centre d'Evaluation et de Traitement de la Douleur Hôpital Ambroise Paré

location_facility: Ambulatório de Fisioterapia do Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo

intervention_type: Procedure


title: A Phase 1/2, Multi-Center, Open-Label, Dose Finding Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of the mTOR Kinase Inhibitor CC-223 Administered Orally to Subjects With Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Multiple Myeloma

summary: The main purpose of this first human study with CC-223 is to assess the safety and action of a new class of experimental drug (dual mTOR inhibitors) in patients with advanced tumors unresponsive to standard therapies and to determine the appropriate dose and tumor type for later-stage clinical trials.

lead_sponsor: Celgene

location_facility: NYU Cancer Institute - Bellevue Hospital

location_facility: Institut Gustave Roussy Faculte de Medecine Paris Sud Service de pneumologie

location_facility: Mayo Clinic Cancer Clinical Studies Unit

location_facility: Sarah Cannon Research Institute Drug Development Unit

location_facility: University of California, San Francisco Hellen Diller Family Comprehensive Cancer Center


ReferenceCitation: Padidela R, Yates R, Benscoter D, McPhail G, Chan E, Nichani J, Mughal MZ, Myer C 4th, Narayan O, Nissenbaum C, Wilkinson S, Zhou S, Saal HM. Characterization of tracheobronchomalacia in infants with hypophosphatasia. Orphanet J Rare Dis. 2020 Aug 6;15(1):204. doi: 10.1186/s13023-020-01483-9.

family: Padidela

full_name: Raja Padidela

given: Raja

family: Yates

full_name: Robert Yates

given: Robert

family: Benscoter

full_name: Dan Benscoter

given: Dan

family: McPhail

full_name: Gary McPhail

given: Gary

family: Chan

full_name: Elaine Chan

given: Elaine

family: Nichani

full_name: Jaya Nichani

given: Jaya

family: Mughal

full_name: M. Zulf Mughal

given: M. Zulf

family: Myer

full_name: Charles Myer

given: Charles

suffix: IV

family: Narayan

full_name: Omendra Narayan

given: Omendra

family: Nissenbaum

full_name: Claire Nissenbaum

given: Claire

family: Wilkinson

full_name: Stuart Wilkinson

given: Stuart

family: Zhou

full_name: Shanggen Zhou

given: Shanggen

family: Saal

full_name: Howard M. Saal

given: Howard M.

journal_name: Orphanet Journal of Rare Diseases

ReferenceCitation: Hofmann CE, Harmatz P, Vockley J, Högler W, Nakayama H, Bishop N, Martos-Moreno GÁ, Moseley S, Fujita KP, Liese J, Rockman-Greenberg C; ENB-010-10 Study Group. Efficacy and Safety of Asfotase Alfa in Infants and Young Children With Hypophosphatasia: A Phase 2 Open-Label Study. J Clin Endocrinol Metab. 2019 Jul 1;104(7):2735-2747. doi: 10.1210/jc.2018-02335.

name: University Children’s Hospital, University of Würzburg, Würzburg, Germany

family: Hofmann

full_name: Christine E Hofmann

given: Christine E

name: University of California San Francisco Benioff Children’s Hospital Oakland, Oakland, California

family: Harmatz

full_name: Paul Harmatz

given: Paul

name: University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania

name: University of Pittsburgh, Graduate School of Public Health, Pittsburgh, Pennsylvania

family: Vockley

full_name: Jerry Vockley

given: Jerry

name: Department of Paediatrics and Adolescent Medicine, Johannes Kepler University, Linz, Austria

name: Institute of Metabolism and Systems Research, University of Birmingham, Birmingham, United Kingdom

family: Högler

full_name: Wolfgang Högler

given: Wolfgang

name: Fukuoka Higashi Medical Center, Fukuoka, Japan

family: Nakayama

full_name: Hideki Nakayama

given: Hideki

name: Sheffield Children’s Hospital, Sheffield, United Kingdom

name: University of Sheffield, Sheffield, United Kingdom

family: Bishop

full_name: Nick Bishop

given: Nick

name: Hospital Infantil Universitario Niño Jesús, IIS La Princesa, Madrid, Spain

name: Universidad Autónoma de Madrid, Madrid, Spain

name: Centro de Investigación Biomédica en Red de la Fisiopatología de la Obesidad y Nutrición, Instituto de Salud Carlos III, Madrid, Spain

name: Alexion Pharmaceuticals, Inc., Boston, Massachusetts

name: Department of Pediatrics, University Hospital of Würzburg, Würzburg, Germany

name: Children’s Hospital Research Institute of Manitoba, Winnipeg, Manitoba, Canada

name: University of Manitoba, Winnipeg, Manitoba, Canada

journal_name: The Journal of Clinical Endocrinology & Metabolism

repository_institution: Red Federada de Repositorios Institucionales de Publicaciones Científicas - LA Referencia

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

repository_institution: University of Birmingham - University of Birmingham Research Portal

repositories:
PubMed Central
Pure (Denmark)
dnet
eprints.whiterose.ac.uk

ReferenceCitation: Whyte MP, Rockman-Greenberg C, Ozono K, Riese R, Moseley S, Melian A, Thompson DD, Bishop N, Hofmann C. Asfotase Alfa Treatment Improves Survival for Perinatal and Infantile Hypophosphatasia. J Clin Endocrinol Metab. 2016 Jan;101(1):334-42. doi: 10.1210/jc.2015-3462. Epub 2015 Nov 3.

location_country:
Italy
United States
Saudi Arabia
Germany
France
Japan
Australia
Russian Federation
Spain
Turkey
Canada
United Kingdom

location_facility: King Faisal Specialist Hospital and Research Center

location_facility: Hospital Infantil Universitario Nino Jesus

location_facility: Health Sciences Centre Winnipeg, University of Manitoba

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: Children's Hospital & Research Center Oakland

location_facility: Children's Hospital of Pittsburgh of UPMC

location_facility: St. Marianna University School of Medicine, Yokohayama City Seibu Hospital

title: An Open-Label, Multicenter, Multinational Study of the Safety, Efficacy and Pharmacokinetics of Asfotase Alfa (Human Recombinant Tissue-nonspecific Alkaline Phosphatase Fusion Protein) in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)

summary: This clinical trial was conducted to study hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone. The purpose of this study was to test the safety and efficacy of a study drug called asfotase alfa (human recombinant tissue non-specific alkaline phosphate fusion protein) to see what effects it has on patients 5 years of age or less with HPP.


ReferenceCitation: Kavanaugh A, Mease PJ, Gomez-Reino JJ, Adebajo AO, Wollenhaupt J, Gladman DD, Hochfeld M, Teng LL, Schett G, Lespessailles E, Hall S. Longterm (52-week) results of a phase III randomized, controlled trial of apremilast in patients with psoriatic arthritis. J Rheumatol. 2015 Mar;42(3):479-88. doi: 10.3899/jrheum.140647. Epub 2015 Jan 15.

family: Kavanaugh

full_name: Arthur Kavanaugh

given: Arthur

family: Mease

full_name: Philip J. Mease

given: Philip J.

family: Gomez-Reino

full_name: Juan J. Gomez-Reino

given: Juan J.

family: Adebajo

full_name: Adewale O. Adebajo

given: Adewale O.

family: Wollenhaupt

full_name: Jürgen Wollenhaupt

given: Jürgen

family: Gladman

full_name: Dafna D. Gladman

given: Dafna D.

family: Hochfeld

full_name: Marla Hochfeld

given: Marla

family: Teng

full_name: Lichen L. Teng

given: Lichen L.

family: Schett

full_name: Georg Schett

given: Georg

family: Lespessailles

full_name: Eric Lespessailles

given: Eric

family: Hall

full_name: Stephen Hall

given: Stephen

journal_name: The Journal of Rheumatology

publisher: The Journal of Rheumatology

publisher_dissemination: The Journal of Rheumatology

publisher_group: The Journal of Rheumatology

publisher_normalized: The Journal of Rheumatology

title: Longterm (52-week) Results of a Phase III Randomized, Controlled Trial of Apremilast in Patients with Psoriatic Arthritis

ReferenceCitation: Mease PJ, Gladman DD, Kavanaugh A, McGonagle D, Nash P, Guerette B, Nakasato P, Brunori M, Teng L, McInnes IB. Articular and Extra-Articular Benefits in ACR20 Non-responders at Week 104 Treated With Apremilast: Pooled Analysis of Three Randomized Controlled Trials. Rheumatol Ther. 2021 Dec;8(4):1677-1691. doi: 10.1007/s40744-021-00369-x. Epub 2021 Sep 18.

ReferenceCitation: Mease PJ, Gladman DD, Ogdie A, Coates LC, Behrens F, Kavanaugh A, McInnes I, Queiro R, Guerette B, Brunori M, Teng L, Smolen JS. Treatment-to-Target With Apremilast in Psoriatic Arthritis: The Probability of Achieving Targets and Comprehensive Control of Disease Manifestations. Arthritis Care Res (Hoboken). 2020 Jun;72(6):814-821. doi: 10.1002/acr.24134. Epub 2020 May 8.

name: Swedish Medical Center/Providence St. Joseph Health and University of Washington School of Medicine Seattle

name: Toronto Western Hospital Toronto Ontario Canada

family: Ogdie

full_name: Alexis Ogdie

given: Alexis

family: Coates

full_name: Laura C. Coates

given: Laura C.

family: Behrens

full_name: Frank Behrens

given: Frank

name: University of California San Diego La Jolla

family: McInnes

full_name: Iain McInnes

given: Iain

name: Hospital Universitario Central de Asturias Oviedo Spain

family: Queiro

full_name: Rubén Queiro

given: Rubén

family: Guerette

full_name: Benoit Guerette

given: Benoit

family: Brunori

full_name: Michele Brunori

given: Michele

name: Medical University of Vienna Vienna Austria

title: Treatment‐to‐Target With Apremilast in Psoriatic Arthritis: The Probability of Achieving Targets and Comprehensive Control of Disease Manifestations

ReferenceCitation: Kavanaugh A, Gladman DD, Edwards CJ, Schett G, Guerette B, Delev N, Teng L, Paris M, Mease PJ. Long-term experience with apremilast in patients with psoriatic arthritis: 5-year results from a PALACE 1-3 pooled analysis. Arthritis Res Ther. 2019 May 10;21(1):118. doi: 10.1186/s13075-019-1901-3.

repository_institution: Kooperativer Bibliotheksverbund Berlin-Brandenburg - OPUS Repository

title: Long-term experience with apremilast in patients with psoriatic arthritis: 5-year results from a PALACE 1–3 pooled analysis

ReferenceCitation: Gladman DD, Kavanaugh A, Gómez-Reino JJ, Wollenhaupt J, Cutolo M, Schett G, Lespessailles E, Guerette B, Delev N, Teng L, Edwards CJ, Birbara CA, Mease PJ. Therapeutic benefit of apremilast on enthesitis and dactylitis in patients with psoriatic arthritis: a pooled analysis of the PALACE 1-3 studies. RMD Open. 2018 Jun 27;4(1):e000669. doi: 10.1136/rmdopen-2018-000669. eCollection 2018.

repository_institution: American Medical Association - The Journal of the American Medical Association (JAMA) Network

title: Therapeutic benefit of apremilast on enthesitis and dactylitis in patients with psoriatic arthritis: a pooled analysis of the PALACE 1–3 studies

ReferenceCitation: Kavanaugh A, Mease PJ, Gomez-Reino JJ, Adebajo AO, Wollenhaupt J, Gladman DD, Lespessailles E, Hall S, Hochfeld M, Hu C, Hough D, Stevens RM, Schett G. Treatment of psoriatic arthritis in a phase 3 randomised, placebo-controlled trial with apremilast, an oral phosphodiesterase 4 inhibitor. Ann Rheum Dis. 2014 Jun;73(6):1020-6. doi: 10.1136/annrheumdis-2013-205056. Epub 2014 Mar 4.

repository_institution: Kooperativer Bibliotheksverbund Berlin-Brandenburg (KOBV), on behalf of the Universitätsbibliothek Erlangen-Nürnberg - OPUS FAU - Online-Publikationssystem der Friedrich-Alexander-Universität Erlangen-Nürnberg

repository_institution: University of Oxford - Oxford Academic Notes & Queries

ipd_sharing_description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request

location_country:
Austria
United States
South Africa
Germany
France
Poland
Hungary
Australia
Russian Federation
New Zealand
Spain
Canada
United Kingdom

location_facility: Piedmont Medical Research Associates Inc

location_facility: Hospital Universitario Marques de Valdecilla

location_facility: Voronezh Regional Clinical Hopsital #1, Voronezh State Medical Academy

location_facility: The Center for Rheumatology and Bone Research

location_facility: Veszprém Megyei Önkormányzat Csolnoky Ferenc Kórház-Rendelöintézet

location_facility: Szpital Uniwersytecki nr 2 im. Dr Jana Biziela w Bydgoszczy

location_facility: Hospital General Universitario Gregorio Maranon

location_facility: Queen Elizabeth Hospital for Rheumatic Disease

location_facility: Arthritis and Osteoporosis Associates LLP

location_facility: Hospital Clinico Universitario de Santiago

location_facility: Arthritis and Rheumatic Disease Specialties

location_facility: Clinical Research Center of Reading, LLP

location_facility: Ryazan I.P. Pavlov State Medical University

location_facility: Honvéd Kórház - Állami Egészségügyi Központ

location_facility: Departmental Hospital at Smolensk Station RZhD JSC

location_facility: The Arthritis Program Research Group Inc.

location_facility: St. Clare's Health Care Corporation of St. John's

location_facility: Centre Hospitalier Universitaire de Sherbrooke-Hospital Fleurimont

location_facility: Dermatology Treatment and Research Center

title: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group, Efficacy and Safety Study of Two Doses of Apremilast (CC-10004) in Subjects With Active Psoriatic Arthritis

summary: The purpose of this study is to determine whether apremilast is safe and effective in the treatment of patients with psoriatic arthritis, specifically in improving signs and symptoms of psoriatic arthritis (tender and swollen joints, pain, physical function) in treated patients.


type: Other Identifier

title: An Open-Label, Multicenter, Phase 1a/1b Study of Olaratumab (LY3012207) Plus Pembrolizumab (MK3475) in Patients With Unresectable Locally Advanced or Metastatic Soft Tissue Sarcoma (STS) Who Have Failed Standard Treatments

summary: The purpose of this study is to evaluate the safety of olaratumab plus pembrolizumab in participants with previously treated advanced or metastatic soft tissue sarcoma.

lead_sponsor: Eli Lilly and Company

location_country:
Denmark
United States
Belgium
France

location_facility: Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg


title: A Phase I, Multicenter, Open-Label, Dose-Escalation and Expansion, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-881 in Patients With Advanced Hematologic Malignancies With an IDH1 and/or IDH2 Mutation

summary: The purpose of this Phase I, multicenter study is to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-881 in advanced hematologic malignancies that harbor an IDH1 and/or IDH2 mutation

lead_sponsor: Agios Pharmaceuticals, Inc.

location_country:
United States
France


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

acronym: chemo-ameno

summary: Relation between chemotherapy and amennorhea

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

location_country: Belgium France

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


title: A Phase Ib Multiple Ascending Dose Study to Evaluate the Safety of Brivanib in Combination With 5-Fluorouracil/Leucovorin (5FU/LV) and Brivanib in Combination With 5-Fluorouracil/Leucovorin/Irinotecan (FOLFIRI) in Subjects With Advanced or Metastatic Gastrointestinal Malignancies

summary: The purpose of this study is to determine a safe and maximum tolerable dose of Brivanib when combined with standard dose 5FU/LV and FOLFIRI.

location_country:
United States
Canada
France

location_facility: Scott & White Memorial Hospital And Clinic


title: A Phase Ib Study Investigating the Combination of RAD001 With Cisplatin and Etoposide in Patients With Extensive-stage Small-cell Lung Cancer Not Previously Treated With Chemotherapy

summary: This study aims to establish a tolerable dose level and regimen of RAD001 in combination with cisplatin and etoposide (standard-of-care chemotherapy) in patients with extensive stage small-cell lung cancer (SCLC) who have not previously been treated with systemic chemotherapy.

lead_sponsor: Novartis Pharmaceuticals

location_facility: Cancer Therapy and Research Center at UTHSCSA

location_facility: University of Colorado Health Sciences Center


title: Open-label, Multicenter,PhaseI Trial in Order To Determine the Safety and Pharmacokinetics of BAY43-9006 in Combination With Docetaxel as First-line Treatment in Metastatic Hormone Refractory Prostate Cancer Patients

summary:
The purpose of the trial is to determine the most effective dose of BAy 46-9003 associated to taxotere for first-line treatment of patient with prostatic cancer.

BAY 43-9006 (SORAFENIB) is a novel dual-action Raf kinase and VEGFR inhibitor, which is orally available and has a favorable safety profile in patients with advanced solid tumors. This, together with the antitumor activity observed after treatment with BAY 43-9006 (SORAFENIB), provides a rationale for further evaluation in patients with advanced cancer. The recommended dose of BAY 43-9006 (SORAFENIB) for future studies is 400 mg bid as a continuous dosing schedule.

location_facility:
Cliniques Universitaires St Luc
Sainte Elisabeth
Notre Dame et Reine Fabiola
St Pierre
Hôpital Européen Georges Pompidou
Clinique Universiataire de Mont Godinne


location_country:
United States
Jordan
Netherlands
France
Germany
United Kingdom

location_facility:
Novartis Investigator Site
Novartis Investigative Site

title: A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel Group, Repeated-dose Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Three Different Dosing Regimens of Inhaled Indacaterol Maleate in Patients With Persistent Asthma

summary: This study assessed the bronchodilator efficacy of three different regimens of indacaterol in patients with asthma


study_start_date_type: Anticipated

lead_sponsor: BioCryst Pharmaceuticals

location_country:
France
Hungary
Spain
United Kingdom
Taiwan

title: A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy

summary: The purpose of this study is to determine the efficacy and safety of BCX9930 monotherapy for the treatment of PNH compared to continued C5 inhibitor therapy in adult PNH patients with residual anemia despite treatment with a C5 inhibitor.


location_country:
United Kingdom
Korea, Republic of
France
Spain

location_facility: University College London - Hospitals NHS Foundation Trust

location_facility: Seoul National University Hospital - Department of Hematology-Oncology

location_facility: C. Universidad de Navarra Servicio de Hematologia

location_facility: Samsung Medical Center - Division of Hematology-Oncology

location_facility: Hospital 12 de Octubre Servicio de Hematología

location_facility: H. Universitario La Fe Servicio de Hematologia

ipd_sharing: Yes

title: Phase I / II, Open Label, Dose Escalation Part (Phase I) Followed by Noncomparative Expansion Part (Phase II), Multi-centre Study, Evaluating Safety, Pharmacokinetics and Efficacy of S65487, a Bcl2 Inhibitor Combined With Azacitidine in Adult Patients With Previously Untreated Acute Myeloid Leukemia Not Eligible for Intensive Treatment

summary: The purpose of this study is to assess the safety, tolerability and clinical activity of the combination S65487 with azacitidine in patients with acute myeloid leukaemia.


title: Open-Label Evaluation of the Pharmacokinetic Profile and Safety of Tapentadol Oral Solution for the Treatment of Postsurgical Pain in Children and Adolescents Aged From 6 to Less Than 18 Years

summary: The purpose of this study is to evaluate the pharmacokinetic (PK) profile; and safety and tolerability of a single dose of tapentadol oral solution (OS) 1 mg/kg in children and adolescents aged from 6 to less than 18 years with acute, moderate to severe postsurgical pain.

lead_sponsor: Janssen Research & Development, LLC

location_country:
United States
Canada
France
Spain


title: A Phase Ib/II, Open Label Study of Sabatolimab as a Treatment for Patients With Acute Myeloid Leukemia and Presence of Measurable Residual Disease After Allogeneic Stem Cell Transplantation

summary: The primary purpose of this study is to test the hypothesis that preemptive treatment with sabatolimab, alone or in combination with azacitidine, when administered to participants with AML/secondary AML who are in complete remission with positive measurable residual disease post-allogeneic hematopoietic stem cell transplantation (MRD+ post-aHSCT), can enhance the graft versus leukemia (GvL) response and prevent or delay hematologic relapse without an unacceptable level of treatment-emergent toxicities, including clinically significant acute and/or chronic graft-versus-host disease (GvHD) and immune-related adverse events

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 expert 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 is currently available according to the process described on www.clinicalstudydatarequest.com.

location_country: Germany France Spain


location_country:
United States
France
Japan
Israel
Norway
Germany
Iceland

title: A Multicenter Extension Trial of Subcutaneously Administered AIN457 in Participants With Moderate to Severe Chronic Plaque-type Psoriasis

summary: The purpose of this study was to provide long term clinical data for the compound for the treatment of the indication of moderate to severe chronic plaque-type psoriasis.


title: A Study to Compare IPX066 and Carbidopa/Levodopa/Entacapone (CLE) Followed by an Open-Label Safety Study of IPX066 in Advanced Parkinson's Disease

summary: This is a study to compare the efficacy of IPX066 and CLE in subjects with advanced Parkinson's disease.

ReferenceCitation: Morgan JC, Dhall R, Rubens R, Khanna S, Gupta S. Dosing Patterns during Conversion to IPX066, Extended-Release Carbidopa-Levodopa (ER CD-LD), in Parkinson's Disease with Motor Fluctuations. Parkinsons Dis. 2018 Oct 22;2018:9763057. doi: 10.1155/2018/9763057. eCollection 2018.

name: Movement Disorders Program, Parkinson’s Foundation Center of Excellence, Department of Neurology, Medical College of Georgia, Augusta University, 1429 Harper Street, HF-1154, Augusta, GA 30912, USA

family: Morgan

full_name: John C. Morgan

given: John C.

name: University of Arkansas for Medical Science, 4301 W. Markham Street, Little Rock, AR 72205, USA

family: Dhall

full_name: Rohit Dhall

given: Rohit

name: Impax Laboratories, LLC, 31047 Genstar Road, Hayward, CA 94544, USA

family: Rubens

full_name: Robert Rubens

given: Robert

family: Khanna

full_name: Sarita Khanna

given: Sarita

family: Gupta

full_name: Suneel Gupta

given: Suneel

journal_name: Parkinson's Disease

repository_institution: Hindawi - Hindawi

repositories_institution:
Hindawi - Hindawi
PubMed Central - Europe PMC

publisher: Hindawi Limited

publisher_dissemination: Hindawi

publisher_group: Hindawi

publisher_normalized: Hindawi

title: Dosing Patterns during Conversion to IPX066, Extended-Release Carbidopa-Levodopa (ER CD-LD), in Parkinson’s Disease with Motor Fluctuations

lead_sponsor: Impax Laboratories, LLC

location_facility: Klinikum rechts der Isar der Technischen Universität München

location_facility: RKU, Neurologische Klinik der Universität Ulm

location_facility: Hôpital Gabriel Montpied-Service de Neurologie A-

location_facility: Praxis für Neurologie, Psychiatrie und Psychotherapie Achim

location_facility: Parkinson's Disease and Movement Disorders Center of Long Island

location_facility: San Raffaele Cassino, San Raffaele Cassino,

location_facility: USF Parkinson's and Movement Disorders Center

location_facility: Service de neurologie-Hôpital de la Timone-

location_facility: Dipartimento di Oncologia e Neuroscienze, Università G. D'Annunzio


location_country:
Italy
United States
France
Australia
Canada
United Kingdom

location_facility: Children's Healthcare of Atlanta Childern Hosp - ATL

location_facility: Seattle Children's Hospital CPKC412A2114

location_facility: Sidney Kimmel Comprehensive Cancer Center/Johns Hopkins Med. John Hopkins

title: A Phase I/II Study of LDE225 in Pediatric Patients With Recurrent or Refractory Medulloblastoma or Other Tumors Potentially Dependent on the Hedgehog-signaling Pathway and Adult Patients With Recurrent or Refractory Medulloblastoma

summary:
Phase I dose-escalation study to characterize the safety, tolerability, pharmacokinetics and pharmacodynamics of LDE225 given orally on a daily dosing schedule in children with recurrent or refractory medulloblastoma, or other tumors potentially dependent on Hedgehog signaling pathway.

Phase II study is to assess preliminary efficacy in both adult and pediatric patients with recurrent or refractory MB.


lead_sponsor: Cephalon

location_country:
Italy
Brazil
South Africa
Serbia
Germany
Croatia
Bulgaria
France
Poland
Slovakia
Hungary
Argentina
Australia
Spain
United States
Canada
Ukraine

location_facility:
Teva Investigational Site 635
Teva Investigational Site 184
Teva Investigational Site 404
Teva Investigational Site 689
Teva Investigational Site 128
Teva Investigational Site 248
Teva Investigational Site 103
Teva Investigational Site 205
Teva Investigational Site 156
Teva Investigational Site 624
Teva Investigational Site 336
Teva Investigational Site 688
Teva Investigational Site 633
Teva Investigational Site 190
Teva Investigational Site 256
Teva Investigational Site 106
Teva Investigational Site 257
Teva Investigational Site 708
Teva Investigational Site 236
Teva Investigational Site 111
Teva Investigational Site 661
Teva Investigational Site 406
Teva Investigational Site 136
Teva Investigational Site 433
Teva Investigational Site 135
Teva Investigational Site 131
Teva Investigational Site 853
Teva Investigational Site 837
Teva Investigational Site 608
Teva Investigational Site 259
Teva Investigational Site 102
Teva Investigational Site 664
Teva Investigational Site 255
Teva Investigational Site 201
Teva Investigational Site 651
Teva Investigational Site 834
Teva Investigational Site 662
Teva Investigational Site 138
Teva Investigational Site 223
Teva Investigational Site 666
Teva Investigational Site 301
Teva Investigational Site 712
Teva Investigational Site 258
Teva Investigational Site 606
Teva Investigational Site 198
Teva Investigational Site 296
Teva Investigational Site 132
Teva Investigational Site 280
Teva Investigational Site 711
Teva Investigational Site 370
Teva Investigational Site 400
Teva Investigational Site 127
Teva Investigational Site 196
Teva Investigational Site 217
Teva Investigational Site 237
Teva Investigational Site 235
Teva Investigational Site 872
Teva Investigational Site 609
Teva Investigational Site 207
Teva Investigational Site 303
Teva Investigational Site 286
Teva Investigational Site 182
Teva Investigational Site 181
Teva Investigational Site 176
Teva Investigational Site 104
Teva Investigational Site 616
Teva Investigational Site 153
Teva Investigational Site 613
Teva Investigational Site 212
Teva Investigational Site 141
Teva Investigational Site 852
Teva Investigational Site 861
Teva Investigational Site 148
Teva Investigational Site 430
Teva Investigational Site 706
Teva Investigational Site 855
Teva Investigational Site 833
Teva Investigational Site 299
Teva Investigational Site 282
Teva Investigational Site 121
Teva Investigational Site 213
Teva Investigational Site 147
Teva Investigational Site 634
Teva Investigational Site 698
Teva Investigational Site 180
Teva Investigational Site 195
Teva Investigational Site 300
Teva Investigational Site 247
Teva Investigational Site 192
Teva Investigational Site 134
Teva Investigational Site 610
Teva Investigational Site 881
Teva Investigational Site 245
Teva Investigational Site 295
Teva Investigational Site 177
Teva Investigational Site 175
Teva Investigational Site 249
Teva Investigational Site 183
Teva Investigational Site 224
Teva Investigational Site 240
Teva Investigational Site 450
Teva Investigational Site 225
Teva Investigational Site 204
Teva Investigational Site 434
Teva Investigational Site 238
Teva Investigational Site 129
Teva Investigational Site 832
Teva Investigational Site 117
Teva Investigational Site 409
Teva Investigational Site 133
Teva Investigational Site 710
Teva Investigational Site 118
Teva Investigational Site 229
Teva Investigational Site 107
Teva Investigational Site 655
Teva Investigational Site 709
Teva Investigational Site 155
Teva Investigational Site 113
Teva Investigational Site 193
Teva Investigational Site 600
Teva Investigational Site 411
Teva Investigational Site 100
Teva Investigational Site 696
Teva Investigational Site 110
Teva Investigational Site 871
Teva Investigational Site 401
Teva Investigational Site 605
Teva Investigational Site 122
Teva Investigational Site 873
Teva Investigational Site 228
Teva Investigational Site 884
Teva Investigational Site 105
Teva Investigational Site 403
Teva Investigational Site 371
Teva Investigational Site 200
Teva Investigational Site 290
Teva Investigational Site 854
Teva Investigational Site 612
Teva Investigational Site 462
Teva Investigational Site 219
Teva Investigational Site 157
Teva Investigational Site 281
Teva Investigational Site 145
Teva Investigational Site 202
Teva Investigational Site 699
Teva Investigational Site 119
Teva Investigational Site 831
Teva Investigational Site 408
Teva Investigational Site 292
Teva Investigational Site 146
Teva Investigational Site 687
Teva Investigational Site 697
Teva Investigational Site 115
Teva Investigational Site 116
Teva Investigational Site 692
Teva Investigational Site 875
Teva Investigational Site 851
Teva Investigational Site 603

title: A 6-Month, Open-Label, Flexible-Dosage (150 to 200 mg/Day) Extension Study of the Safety and Efficacy of Armodafinil Treatment as Adjunctive Therapy in Adults With Major Depression Associated With Bipolar I Disorder

summary: The primary objective of this study is to evaluate the safety and tolerability of long term (6 months) armodafinil treatment as adjunctive therapy to mood-stabilizing medications in adults with bipolar I disorder.


ReferenceCitation: Pose E, Napoleone L, Amin A, Campion D, Jimenez C, Piano S, Roux O, Uschner FE, de Wit K, Zaccherini G, Alessandria C, Angeli P, Bernardi M, Beuers U, Caraceni P, Durand F, Mookerjee RP, Trebicka J, Vargas V, Andrade RJ, Carol M, Pich J, Ferrero J, Domenech G, Llopis M, Torres F, Kamath PS, Abraldes JG, Solà E, Ginès P. Safety of two different doses of simvastatin plus rifaximin in decompensated cirrhosis (LIVERHOPE-SAFETY): a randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Gastroenterol Hepatol. 2020 Jan;5(1):31-41. doi: 10.1016/S2468-1253(19)30320-6. Epub 2019 Oct 10.

family: Pose

full_name: Elisa Pose

given: Elisa

family: Napoleone

full_name: Laura Napoleone

given: Laura

family: Amin

full_name: Ahmed Amin

given: Ahmed

family: Campion

full_name: Daniela Campion

given: Daniela

family: Jimenez

full_name: César Jimenez

given: César

family: Piano

full_name: Salvatore Piano

given: Salvatore

family: Roux

full_name: Olivier Roux

given: Olivier

family: Uschner

full_name: Frank Erhard Uschner

given: Frank Erhard

family: de Wit

full_name: Koos de Wit

given: Koos

family: Zaccherini

full_name: Giacomo Zaccherini

given: Giacomo

family: Alessandria

full_name: Carlo Alessandria

given: Carlo

family: Angeli

full_name: Paolo Angeli

given: Paolo

family: Bernardi

full_name: Mauro Bernardi

given: Mauro

family: Beuers

full_name: Ulrich Beuers

given: Ulrich

family: Caraceni

full_name: Paolo Caraceni

family: Durand

full_name: François Durand

given: François

family: Mookerjee

full_name: Rajeshwar P Mookerjee

given: Rajeshwar P

family: Trebicka

full_name: Jonel Trebicka

given: Jonel

family: Vargas

full_name: Victor Vargas

given: Victor

family: Andrade

full_name: Raúl J Andrade

given: Raúl J

family: Carol

full_name: Marta Carol

given: Marta

family: Pich

full_name: Judit Pich

given: Judit

family: Ferrero

full_name: Juan Ferrero

given: Juan

family: Domenech

full_name: Gema Domenech

given: Gema

family: Llopis

full_name: Marta Llopis

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

repository_institution: University College London - UCL Discovery

location_country:
Italy
Netherlands
France
Spain
Germany
United Kingdom

location_facility:
Academic Medical Centre
San Giovanni Battista Hospital
Beajuon Hospital
Hospital Universitari Vall d'Hebrón
Royal Free Hospital
Hospital Clínic de Barcelona
Bologna University Hospital
Padova University Hospital
Universitatsklinikum Bonn

title: Safety and Tolerability of the Combination of Simvastatin Plus Rifaximin in Patients With Decompensated Cirrhosis: a Multicenter, Double-blind, Placebo Controlled Randomized Clinical Trial.

summary:
The main purpose of this study is to investigate whether the combination of two different drugs, simvastatin and rifaximin, is safe in the treatment of patients with decompensated cirrhosis.

The secondary purpose is to see if this combination results in an improvement in inflammation markers in patients with cirrhosis and in an improvement in analytic parameters of progression of liver disease.


title: Current Treatment of HCV Infection After HSCT

summary:
The availability of novel therapies with DAAs might prompt clinicians caring for HSCT recipients with HCV infection to prescribe the treatment more frequently and possibly earlier after HSCT. Since numerous possible therapeutic combinations exist, the choice of the most appropriate one is not straightforward. It depends not only on its efficacy, toxicity and rate of pharmacological interactions, but also on availability (both through healthcare system and in expanded access programs), and cost.

This observational prospective study will focus on treatment strategies in HSCT recipients with HCV infection. The main focus will be the therapeutic approach, the combination of drugs chosen, the length of treatment and the outcome. The main reasons for treating or not treating HSCT recipients with HCV infection will be also explored. Compared to previous cohorts, this study might provide additional data in the area of non-invasive assessment of fibrosis, HCV-RNA levels and genotyping.

In order to get as much data as possible on HCV treatment, patients with chronic HCV infection transplanted during the last 10 years will be included.

Finally the prevalence of HCV-RNA positive patients among those transplanted in the year 2016 will be reported.

lead_sponsor: European Society for Blood and Marrow Transplantation

location_country: Italy France Spain

location_facility:
Ospedale San Raffaele s.r.l.
Centre Hospitalier de Meaux
Hospital de la Princesa
Policlinico G.B. Rossi
Hospital Santa Creu i Sant Pau
Ospedale Civile
Ospedale Santa Maria della Misericordia


title: A Phase II, Multicenter, Randomized, Controlled, Open-label Study of the Safety, Efficacy and Pharmacokinetics of ABT-263 in Combination With Dose-intensive Rituximab, or Dose-intensive Rituximab Alone, in Previously Untreated Patients With B-Cell, Chronic Lymphocytic Leukemia (CLL)

summary: This Phase II, randomized, open-label, international, multicenter trial is designed to evaluate the safety and efficacy of rituximab monotherapy when given according to a dose intense regimen and to assess the safety, efficacy, and pharmacokinetics of ABT-263 when combined with dose-intense rituximab in previously untreated patients with B-cell CLL.

lead_sponsor: Genentech, Inc.

location_country:
Italy
Brazil
United Kingdom
France
Poland
Israel
Australia
Puerto Rico
Russian Federation
United States
Czech Republic
Ukraine


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

location_country:
France
Russian Federation
United States
United Kingdom
Canada

location_facility:
Teva Investigational Site 1021
Teva Investigational Site 3510
Teva Investigational Site 5007
Teva Investigational Site 5005
Teva Investigational Site 1033
Teva Investigational Site 1024
Teva Investigational Site 3409
Teva Investigational Site 3411
Teva Investigational Site 1017
Teva Investigational Site 1031
Teva Investigational Site 1016
Teva Investigational Site 5002
Teva Investigational Site 3413
Teva Investigational Site 1026
Teva Investigational Site 1030
Teva Investigational Site 1020
Teva Investigational Site 5001
Teva Investigational Site 1025
Teva Investigational Site 1019
Teva Investigational Site 1022
Teva Investigational Site 1028
Teva Investigational Site 1032
Teva Investigational Site 1115
Teva Investigational Site 3509
Teva Investigational Site 5006
Teva Investigational Site 3412
Teva Investigational Site 3410
Teva Investigational Site 1018
Teva Investigational Site 1113
Teva Investigational Site 5008
Teva Investigational Site 5003

title: A Phase IIa, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Safety, Tolerability and Clinical Effect of Laquinimod in Active Lupus Nephritis Patients, in Combination With Standard of Care (Mycophenolate Mofetil and Steroids)

summary: The study aims to evaluate the safety and clinical effect of daily oral treatment with laquinimod capsules in active lupus nephritis patients. This study will assess Laquinimod doses of 0.5mg /day and 1 mg/day in combination with standard of care treatment (mycophenolate mofetil and corticosteroids). Laquinimod is a novel immunomodulating drug which is currently in advanced stages of development by Teva Pharmaceuticals Ltd. for Multiple Sclerosis.