title: Testing the Effect of Intrauterine Slow Release Insemination (SRI) on the Pregnancy Rate in Women Designated for Standard Intrauterine Insemination (IUI)

acronym: EVIE

summary:
Overall Study Design and Plan Description

The study will be conducted on women with fertility difficulties who are designated for intrauterine insemination treatment (IUI). The research sample size is 137 treatment cycles per group (total of 274 treatment cycles). The women will be randomly divided into two groups. Some will undergo the standard bolus IUI treatment and others will be treated with the EVIE Slow Release Insemination method (SRI). Women who fail to conceive in the first treatment will next receive the alternative treatment. This means that a woman treated with the IUI method who does not become pregnant will then be treated with SRI, and vice versa (crossover method). The study will include women treated with Clomiphene Citrate or with other Gonadotropin-based treatment. Every couple treated will undergo at least 2 insemination cycles (unless pregnancy has already been achieved).
Before beginning the hormone treatment, each couple will have the trial procedure explained to them. This explanation will include all the detailed information and instruction about the IUI and the Slow Release Insemination methods. A Patient Information Sheet will be available to them.
Preparation of the spermatozoa for intra uterine insemination will be identical to the method performed currently before IUI.

Approximately two weeks after the insemination treatment, a blood sample will be taken from the woman to check for Beta hCG levels to indicate pregnancy.

Hypothesis:

SRI leads to higher pregnancy rates compared to standard IUI

Primary endpoint:

• Pregnancy rate Effect of controlled Intra-uterine slow release insemination on the pregnancy rate of women designated for intra uterine insemination in comparison to the accepted IUI method.

ReferenceCitation: Muharib NS, Abdel Gadir A, Shaw RW. Slow release intrauterine insemination versus the bolus technique in the treatment of women with cervical mucus hostility. Hum Reprod. 1992 Feb;7(2):227-9.

lead_sponsor: Medical University of Vienna

location_country:
Austria
Germany
United Kingdom
France

location_facility: Dept. Obstetrics and Gynaekology, Medical Faculty of Aachen

location_facility: Klinikum der Johann Wolfgang-Goethe-Universität Klinik für Frauenheilkunde und Geburtshilfe

location_facility: Kinderwunschzentrum der Goldenes Kreuz Privatklinik

location_facility: LMU, Klinikum der Universität München Klinik und Poliklinik für Frauenheilkunde und Geburtshilfe

location_facility: Centre for Reproductive and Genetic Health - Eastman Dental Hospital

location_facility: Frauenärztliche Gemeinschaftspraxis Dr. Maarten R. van Santen; KriegsstraBe 216

location_facility: Dept. Obstetrics and Gynaecology, Medical University of Vienna


type: EudraCT Number

title: A Randomised Phase IIb Trial of Bevacizumab Added to Temozolomide ± Irinotecan for Children With Refractory/Relapsed Neuroblastoma - BEACON-Neuroblastoma Trial

acronym: BEACON

summary:
The purpose of this study is to investigate whether Bevacizumab (an anti-VEGF monoclonal antibody) added to a backbone chemotherapy regimen (Temozolomide, Irinotecan-Temozolomide or Topotecan-Temozolomide) demonstrates activity in children with relapsed or refractory neuroblastoma. Also, to investigate whether the addition of Irinotecan or Topotecan to Temozolomide increases the activity of chemotherapy.The primary objective of the study is the best response (Complete Response or Partial Response) while trial treatment, within 18 or 24 weeks depending on the arm of the trial the participant is randomised to. Secondary endpoints are assessing the side effects, the length of time before progression (Progression Free Survival) and overall survival (OS).

This trial will address two important questions:

does targeting blood vessel development using bevacizumab, (a monoclonal antibody against the Vascular Endothelial Growth Factor (VEGF)), add to the effect on a tumour when used with existing chemotherapy, compared to the effect of the existing chemotherapy alone (temozolomide)? NOTE- This question has been completed.
does the addition of a second chemotherapy drug (irinotecan or topotecan) increase the effect on a tumour compared to the effect of one alone (temozolomide) NOTE - This question has been completed.
does the addition of dinutuximab beta added to a backbone chemotherapy (temozolomide or temozolomide + topotecan) increase the effect of backbone alone.

Patients aged 1-21 years of age with relapsed or refractory high-risk neuroblastoma are randomised to one of two treatment arms: temozolomide-topotecan (TTo) or dinutuximab beta-temozolomide-topotecan (dBTTo). Temozolomide (T), irinotecan-temozolomide (IT), bevacizumab-T (BT), BIT (bevacizumab-IT), bevacizumab-temozolomide-topotecan (BTTo) and dinutuximab beta-temozolomide (dBT) are now closed to recruitment.

study_start_date_type: Actual

status: Active, not recruiting

study_completion_date_type: Anticipated

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Actual

ipd_sharing: No

lead_sponsor: University of Birmingham

location_country:
Italy
Austria
Switzerland
Netherlands
France
Ireland
Belgium
Spain
Denmark
United Kingdom

location_facility: St Anna Children's Hospital and CCRI/Studies and Statistics

location_facility: The Royal Marsden NHS Foundation Trust and Institute of Cancer Research

intervention_type: Drug

lead_sponsor_type: academique

status_simplified: Ongoing


status: Completed

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

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

lead_sponsor: Novartis Pharmaceuticals

location_country:
Spain
United States
France
Netherlands

location_facility: Memorial Sloan Kettering Oncology Department.

location_facility: USC Kenneth Norris Comprehensive Cancer Center Oncology Dept

ipd_sharing: Undecided

study_completion_date_type: Actual

source: euctr

title: A Randomized, Blinded, Placebo-controlled, Phase II Trial of LEE011 in Patients With Relapsed, Refractory, Incurable Teratoma With Recent Progression

summary:
This was a multi-center, randomized, double blind (investigator and subject), placebo controlled Phase II study to determine the efficacy and safety of treatment with ribociclib versus placebo in subjects with progressive relapsed, refractory incurable teratoma. Eligible subjects were randomized in a 2:1 ratio to ribociclib or placebo.

After discontinuation of study treatment, patients were followed up for safety, disease progression and overall survival.

lead_sponsor_type: industriel

status_simplified: Completed


title: Open-Label, Non Randomized Phase 2 Study With Safety Run-In Evaluating Efficacy and Safety of PQR309 in Patients With Relapsed or Refractory Lymphoma

summary: The main goal of this study is to determine the Maximum Tolerated Dose (MTD) and the Recommended Phase II Dose (RP2D) as well as preliminary antitumor activity of bimiralisib (PQR309) administered orally, as once daily capsules continuously and on intermittent schedule in patients with relapsed or refractory lymphomas.

lead_sponsor: PIQUR Therapeutics AG

location_country:
Bosnia and Herzegovina
Serbia
France
Israel
United States
United Kingdom

location_facility: Institute for Oncology and radiology of Serbia

location_facility: University Clinical Center Republic of Srpska


type: Other Identifier

title: A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Enzyme Replacement Therapy (ERT) Treatment-naïve Adult Male Patients Diagnosed With Fabry Disease

summary:
Primary Objective:

To assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy of GZ/SAR402671 in enzyme replacement therapy treatment-naïve adult male participants diagnosed with Fabry disease.

lead_sponsor: Genzyme, a Sanofi Company

location_country:
France
Poland
Russian Federation
United States
United Kingdom

location_facility:
Investigational Site Number 826002
Investigational Site Number 840001
Investigational Site Number 840003
Investigational Site Number 616001
Investigational Site Number 826003
Investigational Site Number 840002
Investigational Site Number 250001
Investigational Site Number 643002


lead_sponsor: Sanofi

location_country:
Italy
Brazil
India
Mexico
Morocco
Philippines
France
Thailand
Tunisia
Algeria
China
Turkey
Indonesia
Korea, Republic of

location_facility:
Investigational Site Number 764002
Investigational Site Number 792001
Investigational Site Number 356001
Investigational Site Number 504001
Investigational Site Number 380001
Investigational Site Number 608002
Investigational Site Number 504002
Investigational Site Number 250001
Investigational Site Number 788002
Investigational Site Number 012001
Investigational Site Number 410002
Investigational Site Number 410003
Investigational Site Number 792002
Investigational Site Number 076001
Investigational Site Number 356004
Investigational Site Number 410001
Investigational Site Number 156005
Investigational Site Number 076002
Investigational Site Number 356002
Investigational Site Number 792003
Investigational Site Number 764001
Investigational Site Number 360001
Investigational Site Number 504003
Investigational Site Number 788003
Investigational Site Number 356003
Investigational Site Number 484001

title: International Randomized Study to Evaluate the Addition of Docetaxel to the Combination of Cisplatin-5-fluorouracil (TCF) vs. Cisplatin-5-fluorouracil (CF) in the Induction Treatment of Nasopharyngeal Carcinoma (NPC) in Children and Adolescents

summary:
The primary objective is to estimate the Complete Response rate of docetaxel to the combination of cisplatin-5-fluorouracil (TCF) compared to cisplatin-5-fluorouracil (CF) in the Induction treatment of Nasopharyngeal Carcinoma (NPC).

The secondary objectives are to determine:

the safety of TCF in comparison to CF after induction treatment of NPC,
the pharmacokinetics of docetaxel when added to CF,
the Overall Response rate of TCF and CF on completion of induction and consolidation (chemo-radiotherapy) treatment of NPC, and to compare overall survival between TCF and CF.


location_country:
India
Serbia
Spain
Philippines
Germany
France
Bulgaria
Malaysia
Poland
Slovakia
Singapore
Russian Federation
Turkey
Portugal
Estonia
United States
Canada
Taiwan

acronym: ALFACHIN

location_facility:
Sanofi-Aventis Aministrative Office
Sanofi-Aventis Administrative Office

title: 12-week, Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel-group Study to Investigate the Efficacy, Pharmacodynamic and Safety of 2 Doses of Alfuzosin (0.1 mg/kg/Day, 0.2 mg/kg/Day) in the Treatment of Children and Adolescents 2-16 Years With Elevated Detrusor Leak Point Pressure of Neuropathic Etiology Followed by a 40-week Open-label Extension

summary:
The primary objective of the study was to evaluate the efficacy of Alfuzosin in comparison to Placebo on the detrusor Leak Point Pressure (LPP) in children and adolescents 2-16 years of age with elevated detrusor LPP of neuropathic etiology and detrusor LPP ≥ 40 cm H2O.

Secondary objectives were:

To investigate the safety and tolerability of two doses of Alfuzosin in comparison to Placebo in children and adolescents,

To evaluate the effects of the two doses of Alfuzosin in comparison to Placebo on:

Detrusor compliance,
Urinary tract infection,
To investigate the pharmacokinetics of Alfuzosin (population kinetics),
To evaluate the 12-month long-term safety of Alfuzosin 0.1 mg/kg/day and 0.2 mg/kg/day.

The study consisted of 2 periods:

a 12-week double blind treatment period where patients were to receive either Alfuzosin 0.1 mg/kg/day or Alfuzosin 0.2 mg/kg/day or placebo then,
a 40-week open label extension treatment period where patients were to receive either Alfuzosin 0.1 mg/kg/day or Alfuzosin 0.2 mg/kg/day.


title: Efficacy and Safety of 2 mg/Day M100907 on Sleep Maintenance Insomnia: a 6-week, Multicenter, Randomized, Double-blind, Placebo-controlled Polysomnographic Study

summary: The purpose of this study is to assess efficacy and safety of volinanserin in the population of patients complaining of sleep maintenance insomnia. The patients suffering from that condition frequently wake up during the night, their sleep is non restorative and they suffer from a significant distress or impairment in their daily activities consecutive to insomnia.

location_country:
Austria
United States
Germany
France
Australia
Russian Federation
Canada


title: Treatment Protocol for Relapsed Anaplastic Large Cell Lymphoma of Childhood and Adolescence

summary:
RATIONALE: Giving combination chemotherapy and total-body irradiation before a peripheral stem cell transplant that uses the patient's or a donor's stem cells, helps stop both the growth of cancer cells and the patient's immune system from rejecting the stem cells. When the stem cells are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Giving combination chemotherapy and total-body irradiation followed by a stem cell transplant may be an effective treatment for anaplastic large cell lymphoma.

PURPOSE: This clinical trial is studying how well combination chemotherapy followed by stem cell transplant works in treating young patients with progressive or relapsed anaplastic large cell lymphoma.

status: Unknown status

design_allocation: Non-Randomized

enrollment_type: Anticipated

ReferenceType: derived

ReferenceCitation: Knörr F, Brugières L, Pillon M, Zimmermann M, Ruf S, Attarbaschi A, Mellgren K, Burke GAA, Uyttebroeck A, Wróbel G, Beishuizen A, Aladjidi N, Reiter A, Woessmann W; European Inter-Group for Childhood Non-Hodgkin Lymphoma. Stem Cell Transplantation and Vinblastine Monotherapy for Relapsed Pediatric Anaplastic Large Cell Lymphoma: Results of the International, Prospective ALCL-Relapse Trial. J Clin Oncol. 2020 Dec 1;38(34):3999-4009. doi: 10.1200/JCO.20.00157. Epub 2020 Jul 30.

name: Pediatric Hematology and Oncology, University Hospital Hamburg-Eppendorf, Germany

family: Knörr

full_name: Fabian Knörr

given: Fabian

sequence: first

name: Department of Children and Adolescents Oncology, Gustave Roussy University Hospital, Paris-Saclay University, Villejuif, France

family: Brugières

full_name: Laurence Brugières

given: Laurence

sequence: additional

name: Pediatric Hematology and Oncology, University Hospital of Padova, Italy

family: Pillon

full_name: Marta Pillon

given: Marta

name: Department of Pediatric Hematology and Oncology, Hannover Medical School, Germany

family: Zimmermann

full_name: Martin Zimmermann

given: Martin

name: Department of Pediatric Hematology and Oncology, Justus-Liebig-University, Giessen, Germany

family: Ruf

full_name: Stephanie Ruf

given: Stephanie

name: Pediatric Hematology and Oncology, St Anna Children's Hospital, Medical University of Vienna, Austria

family: Attarbaschi

full_name: Andishe Attarbaschi

given: Andishe

name: Department of Pediatric Oncology and Hematology, The Queen Silvia Children's Hospital, Göteborg, Sweden

family: Mellgren

full_name: Karin Mellgren

given: Karin

name: Department of Pediatric Hematology, Oncology and Palliative Care, Cambridge University Hospital NHS Trust, Cambridge, United Kingdom.

family: Burke

full_name: G. Amos A. Burke

given: G. Amos A.

name: Department of Pediatric Oncology & Hematology, University Hospitals Leuven, Belgium

family: Uyttebroeck

full_name: Anne Uyttebroeck

given: Anne

name: Department of Bone Marrow Transplantation, Children's Oncology and Hematology, Wroclaw Medical University, Poland

family: Wróbel

full_name: Grażyna Wróbel

given: Grażyna

name: Princess Maxima Center for Pediatric Oncology, Utrecht, the Netherlands

family: Beishuizen

full_name: Auke Beishuizen

given: Auke

name: Department of Pediatric Hematology and Oncology, CIC1401, INSERM CICP, University Hospital of Bordeaux, France

family: Aladjidi

full_name: Nathalie Aladjidi

given: Nathalie

family: Reiter

full_name: Alfred Reiter

given: Alfred

family: Woessmann

full_name: Wilhelm Woessmann

given: Wilhelm

name: on behalf of the European Inter-Group for Childhood Non-Hodgkin Lymphoma

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Journal of Clinical Oncology

publisher: American Society of Clinical Oncology (ASCO)

lead_sponsor: European Inter-group Cooperation on Childhood and Adolescent Non Hodgkin Lymphoma

location_country:
Italy
Austria
Sweden
Switzerland
Netherlands
United Kingdom
France
Poland
Ireland
Belgium
Germany
Czech Republic

location_facility: University Hospital Schleswig-Holstein - Kiel Campus

location_facility: Krankenanstalt Mutterhaus der Borromaerinnen

location_facility: Helios Kliniken Wuppertal University Hospital

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

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

location_facility: University Medical Center Hamburg - Eppendorf

location_facility: Kinderklinik - Universitaetsklinikum Rostock

location_facility: Klinik und Poliklinik fuer Kinder und Jugendmedizin - Universitaetsklinikum Muenster

location_facility: Universitaetskinderklinik - Universitaetsklinikum Freiburg

location_facility: Kinderklink Siegen Deutsches Rotes Kreuz

location_facility: Institute of Child Health at University of Bristol

location_facility: Royal Belfast Hospital for Sick Children

location_facility: Great Ormond Street Hospital for Children

location_facility: Comprehensive Cancer Center Ulm at Universitaetsklinikum Ulm

location_facility: Dr. von Haunersches Kinderspital der Universitaet Muenchen

location_facility: Charite University Hospital - Campus Virchow Klinikum

location_facility: Kinderklinik - Universitaetsklinikum Aachen

location_facility: Royal Liverpool Children's Hospital, Alder Hey

location_facility: Kliniken der Stadt Koeln gGmbH - Kinderkrankenhaus Riehl

location_facility: Klinik und Poliklinik Fuer Kinder-und Jugendmedizin - Universitaetsklinikum Greifswald

location_facility: Our Lady's Hospital for Sick Children Crumlin

intervention_type: Radiation

status_simplified: Unknown


title: A Randomized, Double Blind, Two-arm Placebo Controlled, 12-Month Study of the Effects of Rimonabant 20mg Once Daily on the Amount and the Activity of Visceral Fat in Abdominally Obese Patients With Metabolic Syndrome.

acronym: VICTORIA

summary:
Primary objective:

To assess the effect of rimonabant on visceral fat area over a period of 12 months when prescribed with a mild hypocaloric diet in abdominally obese patients with metabolic syndrome

Secondary objectives:

To assess the effect of rimonabant over a period of 12 months on:

Liver fat content using CT scan (Computed Tomography scan)
Anthropometric measures (weight, waist circumference, body composition using Dual Energy X-ray Absorptiometry (DEXA))
Lipid, lipoprotein profile
Glycemia, insulinemia and HbA1c
Adipokines, inflammatory and hemostatic markers
To evaluate the percentage of patients with metabolic syndrome at 12 months
To evaluate the safety and tolerability of rimonabant in these patients

In four selected US sites the effect of rimonabant at 12 months will be also assessed on:

Basal lipolysis and insulin suppressed lipolysis (euglycemic hyperinsulinemic clamp).
Resting metabolic rate and substrate oxidation at rest using indirect calorimetry.
Adipose tissue histology and expression of genes involved in glucose and lipid metabolism (superficial adipose tissue biopsy).

ReferenceType: result

ReferenceCitation: Triay J, Mundi M, Klein S, Toledo FG, Smith SR, Abu-Lebdeh H, Jensen M. Does rimonabant independently affect free fatty acid and glucose metabolism? J Clin Endocrinol Metab. 2012 Mar;97(3):819-27. doi: 10.1210/jc.2011-2486. Epub 2011 Dec 14.

name: Mayo Clinic (J.T., M.M., H.A.-L., M.J.), Endocrinology, Rochester, Minnesota 55905

family: Triay

full_name: Jessica Triay

given: Jessica

family: Mundi

full_name: Manpreet Mundi

given: Manpreet

name: Washington University School of Medicine (S.K.), St. Louis, Missouri 63110

family: Klein

full_name: Samuel Klein

given: Samuel

name: University of Pittsburgh (F.G.T.), Winter Park, Florida 32789

family: Toledo

full_name: Frederico G. Toledo

given: Frederico G.

name: Pittsburgh, Pennsylvania 15213; and Florida Hospital (S.R.S.), Winter Park, Florida 32789

family: Smith

full_name: Steven R. Smith

given: Steven R.

family: Abu-Lebdeh

full_name: Haitham Abu-Lebdeh

given: Haitham

family: Jensen

full_name: Michael Jensen

given: Michael

journal_name: The Journal of Clinical Endocrinology & Metabolism

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via free pdf)

host_type: publisher

license_normalized: no license

version: publishedVersion

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

host_type: repository

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

evidence: oa repository (via pmcid lookup)

publisher: The Endocrine Society

publisher_dissemination: The Endocrine Society

publisher_group: The Endocrine Society

publisher_normalized: The Endocrine Society

title: Does Rimonabant Independently Affect Free Fatty Acid and Glucose Metabolism?

location_country:
Italy
Sweden
Finland
France
Spain
Denmark
United States
United Kingdom
Canada

intervention_type: Other


title: A Double Blind, Randomized, Phase II-III Maintenance Study of SR48692 Versus Placebo in Patients With Extensive Stage Small Cell Lung Cancer Following a First Line Chemotherapy With Cisplatin + Etoposide

acronym: SESAME

summary:
To demonstrate an increase in overall survival for patients with newly diagnosed extended stage small cell lung cancer when treated with SR48692 versus placebo, after an initial response (complete or partial response or stable) to first line cisplatin plus etoposide.

Primary objective: comparison of overall survival between patients in the control arm and the meclinertant arm.

Secondary objectives: comparison of the progression free survival, the time to progression, the clinical benefit, the quality of life, the toxicity and safety between patients in the control arm and the meclinertant arm.

location_country:
Italy
Brazil
Mexico
Netherlands
Spain
France
Poland
Hungary
Belgium
Argentina
Australia
Russian Federation
Germany
United Kingdom

location_facility:
Sanofi-aventis adminsitrative office
Sanofi-Aventis Administrative Office


summary: The purpose of this study was to determine the safety and effectiveness of different doses of botulinum toxin Type A in treating lower urinary tract symptoms due to benign prostatic hyperplasia.

ReferenceCitation: Marberger M, Chartier-Kastler E, Egerdie B, Lee KS, Grosse J, Bugarin D, Zhou J, Patel A, Haag-Molkenteller C. A randomized double-blind placebo-controlled phase 2 dose-ranging study of onabotulinumtoxinA in men with benign prostatic hyperplasia. Eur Urol. 2013 Mar;63(3):496-503. doi: 10.1016/j.eururo.2012.10.005. Epub 2012 Oct 12.

family: Marberger

full_name: Michael Marberger

family: Chartier-Kastler

full_name: Emmanuel Chartier-Kastler

given: Emmanuel

family: Egerdie

full_name: Blair Egerdie

given: Blair

family: Lee

full_name: Kyu-Sung Lee

given: Kyu-Sung

family: Grosse

full_name: Joachim Grosse

given: Joachim

family: Bugarin

full_name: Denise Bugarin

given: Denise

family: Zhou

full_name: Jihao Zhou

given: Jihao

family: Patel

full_name: Anand Patel

given: Anand

family: Haag-Molkenteller

full_name: Cornelia Haag-Molkenteller

given: Cornelia

journal_name: European Urology

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

title: A Randomized Double-blind Placebo-controlled Phase 2 Dose-ranging Study of OnabotulinumtoxinA in Men with Benign Prostatic Hyperplasia

lead_sponsor: Allergan

location_country:
Italy
Austria
United Kingdom
Germany
France
Slovakia
Australia
Canada
Czech Republic
Korea, Republic of
Taiwan


title: Cooperative Multicenter Study for Children and Adolescents With Low Grade Glioma

summary:
RATIONALE: Radiation therapy uses high-energy x-rays to kill tumor cells. Drugs used in chemotherapy, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.

PURPOSE: This clinical trial is studying giving radiation therapy or combination chemotherapy to see how well it works in treating patients with clinically or radiologically progressive low-grade gliomas.

ReferenceCitation: Gnekow AK, Walker DA, Kandels D, Picton S, Giorgio Perilongo, Grill J, Stokland T, Sandstrom PE, Warmuth-Metz M, Pietsch T, Giangaspero F, Schmidt R, Faldum A, Kilmartin D, De Paoli A, De Salvo GL; of the Low Grade Glioma Consortium and the participating centers. A European randomised controlled trial of the addition of etoposide to standard vincristine and carboplatin induction as part of an 18-month treatment programme for childhood (≤16 years) low grade glioma - A final report. Eur J Cancer. 2017 Aug;81:206-225. doi: 10.1016/j.ejca.2017.04.019. Epub 2017 Jun 22. Erratum in: Eur J Cancer. 2017 Dec 13;:.

family: Gnekow

full_name: Astrid K. Gnekow

given: Astrid K.

family: Walker

full_name: David A. Walker

given: David A.

family: Kandels

full_name: Daniela Kandels

given: Daniela

family: Picton

full_name: Susan Picton

given: Susan

family: Giorgio Perilongo

full_name: Giorgio Perilongo

family: Grill

full_name: Jacques Grill

given: Jacques

family: Stokland

full_name: Tore Stokland

given: Tore

family: Sandstrom

full_name: Per Eric Sandstrom

given: Per Eric

family: Warmuth-Metz

full_name: Monika Warmuth-Metz

given: Monika

family: Pietsch

full_name: Torsten Pietsch

given: Torsten

family: Giangaspero

full_name: Felice Giangaspero

given: Felice

family: Schmidt

full_name: René Schmidt

given: René

family: Faldum

full_name: Andreas Faldum

given: Andreas

family: Kilmartin

full_name: Denise Kilmartin

family: De Paoli

full_name: Angela De Paoli

given: Angela

family: De Salvo

full_name: Gian Luca De Salvo

given: Gian Luca

family: Slavc

full_name: Irene Slavc

given: Irene

family: Perilongo

given: Giorgio

full_name: Sue Picton

given: Sue

full_name: David Walker

given: David

full_name: Per Erik Sandstrom

given: Per Erik

repository_institution: University of Nottingham - Repository@Nottingham

repository_institution: Augsburg University - OPUS - Augsburg University Publication Server

repository_institution: University of Padova - Padua Research Archive

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

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

title: A European randomised controlled trial of the addition of etoposide to standard vincristine and carboplatin induction as part of an 18-month treatment programme for childhood (≤16 years) low grade glioma – A final report

ReferenceCitation: Kandels D, Pietsch T, Bison B, Warmuth-Metz M, Thomale UW, Kortmann RD, Timmermann B, Hernáiz Driever P, Witt O, Schmidt R, Gnekow AK. Loss of efficacy of subsequent nonsurgical therapy after primary treatment failure in pediatric low-grade glioma patients-Report from the German SIOP-LGG 2004 cohort. Int J Cancer. 2020 Dec 15;147(12):3471-3489. doi: 10.1002/ijc.33170. Epub 2020 Jul 11. Erratum in: Int J Cancer. 2021 Jul 15;149(2):E3-E4.

name: Swabian Children's Cancer Center Medical Faculty, University of Augsburg  Augsburg Germany

name: Institute of Neuropathology, DGNN Brain Tumor Reference Center University of Bonn  Bonn Germany

name: Institute of Diagnostic and Interventional Neuroradiology University Hospital Würzburg  Würzburg Germany

name: Pediatric Neurosurgery, Charité Universitaetsmedizin Berlin, Corporate Member of Freie Universitaet Berlin Humboldt‐Universitaet zu Berlin, and Berlin Institute of Health  Berlin Germany

name: Department of Radio‐Oncology University Hospital Leipzig  Leipzig Germany

name: Department of Particle Therapy University Hospital Essen, West German Proton Therapy Centre Essen (WPE), West German Cancer Center (WTZ), German Cancer Consortium (DKTK)  Essen Germany

name: Department of Pediatric Oncology/Hematology, Charité Universitaetsmedizin Berlin Corporate Member of Freie Universitaet Berlin, Humboldt‐Universitaet zu Berlin, and Berlin Institute of Health  Berlin Germany

name: Hopp Children's Cancer Center Heidelberg (KiTZ) German Cancer Research Center (DKFZ), and Heidelberg University Hospital  Heidelberg Germany

name: Institute of Biostatistics and Clinical Research University of Münster  Münster Germany

repository_institution: Würzburg University - Online Publication Service of Würzburg University

title: Loss of efficacy of subsequent nonsurgical therapy after primary treatment failure in pediatric low‐grade glioma patients—Report from the German SIOP‐LGG 2004 cohort

lead_sponsor: Societe Internationale d'Oncologie Pediatrique

location_country:
Italy
Austria
Switzerland
Croatia
France
Poland
Norway
Belgium
Portugal
Denmark
Germany
United Kingdom

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

location_facility: Allg. Krankenhaus der Stadt Wien Universitaets-Kinderklinik


title: An Analysis of Historical Data on Hematological Remission and Survival Among Adult Patients With Relapsed / Refractory B-Precursor Acute Lymphoblastic Leukemia

summary: A retrospective analysis of historical data looking at hematological remission and survival in adult relapsed / refractory B-precursor acute lymphoblastic leukemia patients.

study_type: Observational

time_perspective: Retrospective

ReferenceCitation: Barlev A, Lin VW, Katz A, Hu K, Cong Z, Barber B. Estimating Long-Term Survival of Adults with Philadelphia Chromosome-Negative Relapsed/Refractory B-Precursor Acute Lymphoblastic Leukemia Treated with Blinatumomab Using Historical Data. Adv Ther. 2017 Jan;34(1):148-155. doi: 10.1007/s12325-016-0447-x. Epub 2016 Nov 21.

family: Barlev

full_name: Arie Barlev

given: Arie

family: Lin

full_name: Vincent W. Lin

given: Vincent W.

family: Katz

full_name: Aaron Katz

given: Aaron

family: Hu

full_name: Kuolung Hu

given: Kuolung

family: Cong

full_name: Ze Cong

given: Ze

family: Barber

full_name: Beth Barber

given: Beth

journal_name: Advances in Therapy

evidence: open (via crossref license)

license: cc-by

license_normalized: cc-by

license: implied-oa

license_normalized: implied-oa

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

ReferenceCitation: Gökbuget N, Dombret H, Ribera JM, Fielding AK, Advani A, Bassan R, Chia V, Doubek M, Giebel S, Hoelzer D, Ifrah N, Katz A, Kelsh M, Martinelli G, Morgades M, O'Brien S, Rowe JM, Stieglmaier J, Wadleigh M, Kantarjian H. International reference analysis of outcomes in adults with B-precursor Ph-negative relapsed/refractory acute lymphoblastic leukemia. Haematologica. 2016 Dec;101(12):1524-1533. Epub 2016 Sep 1.

lead_sponsor: Amgen

location_country:
Italy
United States
United Kingdom
France
Poland
Spain
Germany
Czech Republic


type: U.S. FDA Grant/Contract

title: Extension Study of XLHED-Affected Male Subjects Treated With EDI200 in Protocol ECP-002

summary: The goal of the ECP-002e extension study is to continue the evaluation of all EDI200-treated ECP-002 subjects up to age 10 yrs. No additional study drug administration is planned. The efficacy evaluations will incorporate growth and development parameters, frequency of infections and hospitalizations, and age-appropriate assessments of ectoderm-derived organ function. The safety evaluations will include physical examinations, adverse events and concomitant medication documentation, and laboratory testing. Funding Source - FDA OOPD

time_perspective: Prospective

lead_sponsor: Edimer Pharmaceuticals

location_country:
Italy
United States
France
Germany
United Kingdom

location_facility: Azienda Ospedaliera-Polo Universitario "Luigi Sacco"

location_facility: Washington University School of Medicine


title: A Randomized, Phase II, Placebo-Controlled Study of Ipatasertib (GDC-0068), an Inhibitor to Akt, in Combination With Fluoropyrimidine Plus Oxaliplatin in Patients With Locally Advanced or Metastatic Gastric or Gastroesophageal Junction Adenocarcinoma

summary: This multicenter, randomized, double-blind, placebo-controlled study will evaluate the efficacy of ipatasertib in combination with oxaliplatin, 5-fluorouracil, and leucovorin (modified FOLFOX6 [mFOLFOX6]) chemotherapy in participants with advanced or metastatic gastric or gastroesophageal junction (GEJ) cancer. Participants will be randomized to receive either ipatasertib or placebo orally daily on Days 1 to 7 of each 14-day cycle in combination with mFOLFOX6 on Day 1 of each cycle. Anticipated time on study treatment is until disease progression or unacceptable toxicity occurs (up to approximately 2 years).

ReferenceCitation: Bang YJ, Kang YK, Ng M, Chung HC, Wainberg ZA, Gendreau S, Chan WY, Xu N, Maslyar D, Meng R, Chau I, Ajani JA. A phase II, randomised study of mFOLFOX6 with or without the Akt inhibitor ipatasertib in patients with locally advanced or metastatic gastric or gastroesophageal junction cancer. Eur J Cancer. 2019 Feb;108:17-24. doi: 10.1016/j.ejca.2018.11.017. Epub 2018 Dec 25.

family: Bang

full_name: Y.-J. Bang

family: Kang

full_name: Y.-K. Kang

family: Ng

full_name: M. Ng

given: M.

family: Chung

full_name: H.C. Chung

family: Wainberg

full_name: Z.A. Wainberg

family: Gendreau

full_name: S. Gendreau

given: S.

family: Chan

full_name: W.Y. Chan

family: Xu

full_name: N. Xu

given: N.

family: Maslyar

full_name: D. Maslyar

given: D.

family: Meng

full_name: R. Meng

given: R.

family: Chau

full_name: I. Chau

given: I.

family: Ajani

full_name: J.A. Ajani

journal_name: European Journal of Cancer

lead_sponsor: Genentech, Inc.

location_country:
Italy
United States
Hong Kong
France
Malaysia
Singapore
Spain
Germany
United Kingdom
Korea, Republic of
Taiwan

location_facility: Gachon Medical School Gil Medical Centre; Internal Medicine

location_facility: Univ of Calif, Los Angeles; Hematology/Oncology

location_facility: Charité-Universitätsm. Berlin; Med. Klinik mit Schwerpunkt Hämatologie, Onkologie und Tumorimmunolo.

location_facility: Seoul National University Hosp; Dept Internal Med Hem Onc

location_facility: HOSPITAL DE MADRID NORTE SANCHINARRO- CENTRO INTEGRAL ONCOLOGICO CLARA CAMPAL; Servicio de Oncologia

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

location_facility: IRCCS Istituto Oncologico Veneto (IOV); Oncologia Medica Seconda

location_facility: Chang Gung Medical Foundation - Linkou; Division of Hematology- Oncology

location_facility: Universitatsklinik Heidelberg; Universitätshautklinik und Nationales Centrum für Tumorerkrankungen

location_facility: Hospital Universiti Sains Malaysia [Neurology]

location_facility: Seoul National University Bundang Hospital

location_facility: Medizinische Hochschule; Zentrum Innere Medizin; Abt. Hämatologie u. Onkologie

location_facility: Azienda Ospedaliero Universitaria Pisana; Uff. Sperim. Clin. U.O. Farmaceutica

location_facility: University of Texas M.D. Anderson Cancer Center

location_facility: Queen Mary Hospital; Dept. of Clinical Oncology

location_facility: National Taiwan Uni Hospital; Dept of Oncology

location_facility: Institut Gustave Roussy; Departement Oncologie Medicale

location_facility: University Malaya Medical Centre; Clinical Oncology Unit,

location_facility: National Cancer Centre; Medical Oncology

location_facility: Yonsei Uni College of Medicine, Severance Hospital; Internal Medicine Dept.

location_facility: IRCCS Istituto Nazionale Per La Ricerca Sul Cancro (IST); Oncologia Medica A

location_facility: Mount Vernon Hospital; Centre For Cancer Treatment

location_facility: Hospital Clinico Universitario de Valencia


title: A Prospective Randomized Clinical Trial on 90Yttrium Trans-arterial Radio-Embolization (TheraSphere®) vs. Standard of Care (Sorafenib) for the Treatment of Advanced Hepatocellular Carcinoma (HCC) With Portal Vein Thrombosis (PVT)

acronym: YES-P

summary: This is a two-arm, open-label, prospective, multi-center, randomized, active-controlled clinical trial to assess efficacy and safety of TheraSphere in comparison to standard of care therapy (sorafenib) in the treatment of participants with inoperable liver cancer and blockage of the portal vein.

status: Terminated

lead_sponsor: Boston Scientific Corporation

location_country:
Italy
France
Belgium
Spain
United States
United Kingdom

location_facility: University Hospitals Birmingham NHS Foundation Trust

location_facility: Hospital Universitario Puerta de Hierro, Gastroenterology & Hepatology Dept

location_facility: UO Radiologia, Ospedali Riuniti di Bergamo

location_facility: Fondazione Istituto Nazionale Tumori di Milano

location_facility: Hospital Clínic Barcelona. IDIBAPS. CIBEREHD. Liver Unit

location_facility: S.C Radiologia Interventistica, A.O.Ospedale Niguarda Ca Granda

location_facility: Hospital Universitari i Politecnic la Fe

location_facility: Azienda Ospedaliera Ospedali Riuniti "Villa Sofia-Cervello"

location_facility: Azienda Ospedaliero-Universitaria di Bologna

location_facility: Northwestern Medical Faculty Foundation, Div of Hematology/Oncology

intervention_type: Device


location_country:
Italy
United States
Hong Kong
Netherlands
France
Germany
Korea, Republic of
Taiwan

location_facility: Washington University School of Medicine Dept of Oncology.

location_facility: Sarah Cannon Research Institute Dept of Onc

location_facility: Memorial Sloan Kettering Cancer Center Onc. Dept.

title: An Open-Label, Single-arm, Phase Ib/II Study of AEB071 (a Protein Kinase C Inhibitor) and Everolimus (mTOR Inhibitor) in Patients With CD79-mutant or ABC Subtype Diffuse Large B-Cell Lymphoma

summary:
Study of the safety and efficacy of AEB071 and EVEROLIMUS in patients with CD79-mutant or ABC subtype Diffuse Large B-Cell Lymphoma.

The trial did not progress into Phase II due to the suboptimal tolerability of the combination treatment of sotrastaurin and everolimus in the Phase Ib part of the study. There were no serious safety concerns associated with this combination.


location_facility: Azienda Ospedaliera-Polo Universitario "Luigi Sacco"

location_facility: Washington University School of Medicine

title: A Phase 2 Open-label, Dose-escalation Study to Evaluate the Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200, an EDA-A1 Replacement Protein, Administered to Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)

summary: This Phase 2 first-in-neonate EDI200 study will enroll treatment-naïve, XLHED-affected male newborns in the first two weeks of life. All subjects will meet entry criteria including documentation of an Ectodysplasin (EDA) mutation associated with XLHED. Following Baseline evaluations, EDI200 dosing will be initiated between day-of-life 2 and 14, with each study subject receiving 2 doses/week for a total of 5 doses. The study will enroll subjects in two cohorts with subjects in cohort 1 dosed at 3 mg/kg/dose, associated with partial efficacy, and cohort 2 dosed at 10 mg/kg/dose where enhanced efficacy was demonstrated in the most relevant preclinical model. Given the challenge of identifying families where the subject is yet to be born, it is expected that cohort size and time for recruitment will be variable.


type: Other Grant/Funding Number

title: RELIANCE 4-FRONT™ Active Fixation Defibrillation Lead Post Market Clinical Follow-Up (PMCF) Study

summary: The objective of this study is to gather data to establish the chronic safety, performance and effectiveness of the RELIANCE 4-FRONT™ Active Fixation Defibrillation Leads.

location_country:
Italy
Austria
Sweden
Spain
France
Israel
China
Denmark
Germany
United Kingdom
Portugal

location_facility: Hospital Universitario Virgen de la Victoria

location_facility: Azienda Ospedaliero Universitaria Pisana


title: A Phase Ib, Open-label, Multi-center, Two-arm, Dose-finding Study to Assess Safety and Efficacy of the Oral Combination or INC424 (INC424) and BKM120 in Patients With Primary Myelofibrosis (PMF), Postpolycythemia Vera-myelofibrosis (PPV-MF), or Post-essential Thrombocythemia-myelofibrosis (PET-MF)

summary: The purpose of this phase Ib clinical trial is to evaluate the safety of the combination of INC424 and BKM120 in the myelofibrosis population and to establish the maximum tolerated dose and or the Recommended Phase II dose of the combination guided by the Bayesian dose escalation model. INC424 has shown efficacy in myelofibrosis (MF) and is approved in the US and EU for the treatment of MF. BKM120 is a PI3K inhibitor. Preclinical and early clinical experience support inhibition of the PI3K/mTOR pathway in MF as aberrant activation of the pathway has been observed in MF models and may contribute to the pathogenesis of the disease.

location_country:
Italy
Austria
France
Israel
Singapore
Australia
Spain
Germany
United Kingdom


title: A Phase IV Open-label Study of Predictive Markers in Growth Hormone Deficient and Turner Syndrome Pre-pubertal Children Treated With SAIZEN®

summary: The study aims at identifying the predictive markers after one month of Saizen therapy in Growth Hormone Deficiency (GHD) and Turner Syndrome children.

ReferenceCitation: Stevens A, Clayton P, Tatò L, Yoo HW, Rodriguez-Arnao MD, Skorodok J, Ambler GR, Zignani M, Zieschang J, Della Corte G, Destenaves B, Champigneulle A, Raelson J, Chatelain P. Pharmacogenomics of insulin-like growth factor-I generation during GH treatment in children with GH deficiency or Turner syndrome. Pharmacogenomics J. 2014 Feb;14(1):54-62. doi: 10.1038/tpj.2013.14. Epub 2013 Apr 9.

family: Stevens

full_name: A Stevens

given: A

family: Clayton

full_name: P Clayton

given: P

family: Tatò

full_name: L Tatò

given: L

family: Yoo

full_name: H W Yoo

given: H W

family: Rodriguez-Arnao

full_name: M D Rodriguez-Arnao

given: M D

family: Skorodok

full_name: J Skorodok

given: J

family: Ambler

full_name: G R Ambler

given: G R

family: Zignani

full_name: M Zignani

given: M

family: Zieschang

full_name: J Zieschang

family: Della Corte

full_name: G Della Corte

given: G

family: Destenaves

full_name: B Destenaves

given: B

family: Champigneulle

full_name: A Champigneulle

family: Raelson

full_name: J Raelson

family: Chatelain

full_name: P Chatelain

journal_name: The Pharmacogenomics Journal

evidence: open (via page says license)

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

publisher_dissemination: Springer

publisher_group: Springer

ReferenceCitation: Stevens A, Murray P, De Leonibus C, Garner T, Koledova E, Ambler G, Kapelari K, Binder G, Maghnie M, Zucchini S, Bashnina E, Skorodok J, Yeste D, Belgorosky A, Siguero JL, Coutant R, Vangsøy-Hansen E, Hagenäs L, Dahlgren J, Deal C, Chatelain P, Clayton P. Gene expression signatures predict response to therapy with growth hormone. Pharmacogenomics J. 2021 Oct;21(5):594-607. doi: 10.1038/s41397-021-00237-5. Epub 2021 May 27.

full_name: Adam Stevens

given: Adam

family: Murray

full_name: Philip Murray

ReferenceCitation: Murray PG, Stevens A, De Leonibus C, Koledova E, Chatelain P, Clayton PE. Transcriptomics and machine learning predict diagnosis and severity of growth hormone deficiency. JCI Insight. 2018 Apr 5;3(7). pii: 93247. doi: 10.1172/jci.insight.93247. eCollection 2018 Apr 5.

repository_institution: The University of Manchester - Research Explorer

publisher: American Society for Clinical Investigation

ReferenceCitation: Valsesia A, Chatelain P, Stevens A, Peterkova VA, Belgorosky A, Maghnie M, Antoniazzi F, Koledova E, Wojcik J, Farmer P, Destenaves B, Clayton P; PREDICT Investigator group. GH deficiency status combined with GH receptor polymorphism affects response to GH in children. Eur J Endocrinol. 2015 Dec;173(6):777-89. doi: 10.1530/EJE-15-0474. Epub 2015 Sep 4.

location_country:
Italy
Austria
Sweden
Germany
France
Norway
Singapore
Argentina
Australia
Russian Federation
Spain
Canada
United Kingdom

location_facility:
Local Medical Information Office
Local Medical InformationOffice


title: An Eight-Week, Multicenter, Double-Blind, Placebo-Controlled Study Evaluating the Efficacy, Safety, and Tolerability of One Fixed 100 mg Dose of Saredutant in Patients With Major Depressive Disorder

summary:
The purpose of the study is to evaluate the efficacy and safety of saredutant (or SR48968C) in patients with depression.

The primary objective is to evaluate the efficacy of a 100 mg dose of saredutant compared to placebo in patients with depression.

The secondary objectives are to evaluate the safety of saredutant, to evaluate the efficacy of saredutant on disability and quality of life in patients with depression, and to evaluate blood levels of saredutant.

location_country:
Chile
Mexico
Germany
France
Croatia
Portugal
Canada
Czech Republic


title: An Open Label, Randomized, Parallel Design Study to Investigate the Efficacy and Safety of Sevelamer Hydrochloride (Renagel®) Compared With Calcium Acetate in Peritoneal Dialysis Patients

summary: The purpose of this study is to demonstrate that sevelamer hydrochloride is non-inferior to calcium acetate for the treatment of hyperphosphataemia in patients receiving peritoneal dialysis.

location_country:
Italy
Netherlands
France
Belgium
Spain
Denmark
United Kingdom

location_facility: University Hospital ,Queen Elizabeth Hospital

location_facility: The Churchill Hospital, Oxford Radcliffe Hospitals NHS Trust


title: A Double-blind, Randomized Study of Clopidogrel 75 mg/d vs Placebo, on a Background of ASA 75-100 mg/d,in Peripheral Arterial Disease (PAD) Patients Receiving a Unilateral Below Knee Bypass Graft.

summary:
Primary objective:

To evaluate whether clopidogrel 75 mg o.d. versus placebo (on a background of ASA 75-100 mg/d) will lead to an increased rate of primary patency, limb salvage and survival, in patients receiving a below knee bypass graft for the treatment of PAD.

Secondary objectives:

Comparison, between the two treatment groups, of :

Primary patency,
Assisted primary patency,
Cardiovascular death / myocardial infarction / stroke / any amputation above the ankle.
Ankle Brachial Pressure Index (ABPI) changes from baseline

ReferenceCitation: Belch JJ, Dormandy J; CASPAR Writing Committee, Biasi GM, Cairols M, Diehm C, Eikelboom B, Golledge J, Jawien A, Lepäntalo M, Norgren L, Hiatt WR, Becquemin JP, Bergqvist D, Clement D, Baumgartner I, Minar E, Stonebridge P, Vermassen F, Matyas L, Leizorovicz A. Results of the randomized, placebo-controlled clopidogrel and acetylsalicylic acid in bypass surgery for peripheral arterial disease (CASPAR) trial. J Vasc Surg. 2010 Oct;52(4):825-33, 833.e1-2. doi: 10.1016/j.jvs.2010.04.027. Epub 2010 Aug 1. Erratum in: J Vasc Surg. 2011 Feb;53(2):564. Biasi, B M [corrected to Biasi, G M].

family: Belch

full_name: Jill J.F. Belch

given: Jill J.F.

family: Dormandy

full_name: John Dormandy

given: John

journal_name: Journal of Vascular Surgery

oa_host_type: publisher

license: elsevier-specific: oa user license

license_normalized: elsevier-specific

location_country:
Italy
Austria
Sweden
Switzerland
Netherlands
Finland
France
Poland
Hungary
Australia
Belgium
Spain
Germany
United Kingdom


title: An Open-label, Single Arm, Phase II Trial to Investigate the Safety and Efficacy of Sym004 in Patients With Recurrent and/or Metastatic Squamous Cell Carcinoma of the Head and Neck (SCCHN) Who Have Failed Anti-EGFR Monoclonal Antibody-based Therapy

summary:
The trial is designed as a multi-center, open label Phase 2 trial that investigates the efficacy and safety of Sym004 in subjects with squamous cell cancer of the head and neck (SCCHN). Subjects included must have responded to previous anti-epidermal growth factor receptor (anti-EGFR) monoclonal antibody-based therapy and subsequently become resistant to that therapy. It is believed that Sym004 has the potential to induce tumor responses and provide a superior treatment option to subjects with advanced SCCHN.

Symphogen was the sponsor for planning/conducting and reporting results for this trial.

ReferenceCitation: Machiels JP, Specenier P, Krauß J, Dietz A, Kaminsky MC, Lalami Y, Henke M, Keilholz U, Knecht R, Skartved NJ, Horak ID, Pamperin P, Braun S, Gauler TC. A proof of concept trial of the anti-EGFR antibody mixture Sym004 in patients with squamous cell carcinoma of the head and neck. Cancer Chemother Pharmacol. 2015 Jul;76(1):13-20. doi: 10.1007/s00280-015-2761-4. Epub 2015 May 8.

family: Machiels

full_name: J.-P. Machiels

family: Specenier

full_name: P. Specenier

given: P.

family: Krauß

full_name: J. Krauß

given: J.

family: Dietz

full_name: A. Dietz

given: A.

family: Kaminsky

full_name: M.-C. Kaminsky

family: Lalami

full_name: Y. Lalami

given: Y.

family: Henke

full_name: M. Henke

given: M.

family: Keilholz

full_name: U. Keilholz

given: U.

family: Knecht

full_name: R. Knecht

family: Skartved

full_name: N. J. Skartved

given: N. J.

family: Horak

full_name: I. D. Horak

given: I. D.

family: Pamperin

full_name: P. Pamperin

family: Braun

full_name: S. Braun

given: S.

family: Gauler

full_name: T. C. Gauler

given: T. C.

journal_name: Cancer Chemotherapy and Pharmacology

lead_sponsor: Symphogen A/S

location_country: Germany Belgium France

location_facility: 3303; Centre Alexis Vautrin; Département d'Oncologie Médicale

location_facility: 4906; University Medical Center Hamburg Eppendorf; Department of Otorhinolaryngology and Head and Neck Surgery

location_facility: 4905; Charité Campus Benjamin Franklin; Hematology, Oncology and Transfusion Medicine

location_facility: 4907; University Hospital Heidelberg; Nationales Centrum für Tumorerkrankungen (NCT)

location_facility: 3203; Antwerp University Hospital; Department of Medical Oncology

location_facility: 3202; Jules Bordet Institute; Clinique d'Oncologie Médicale

location_facility: 3201; Cliniques Universitaires St-Luc; Centre du Cancer


summary: This study is conducted in 3 stages. Stage 1 is an open-label, dose-escalation assessment of the safety of AGN-150998 administered as a single intravitreal injection to patients with advanced exudative Age-related Macular Degeneration (AMD). Stage 2 and Stage 3 are randomized, double-masked, comparisons of the safety and treatment effects on retinal edema and best-corrected visual acuity (BCVA) of AGN-150998 and ranibizumab in treatment-naive patients with exudative AMD. Study medication is administered as needed in Stage 2 and with a fixed-dosing schedule in Stage 3. The study objectives are (1) to identify the highest tolerated dose of AGN-150998, (2) to assess the safety and duration of treatment effects on retinal edema and BCVA, and (3) to characterize the systemic pharmacokinetic profile of AGN-150998.

ReferenceType: background

ReferenceCitation: Callanan D, Kunimoto D, Maturi RK, Patel SS, Staurenghi G, Wolf S, Cheetham JK, Hohman TC, Kim K, López FJ, Schneider S. Double-Masked, Randomized, Phase 2 Evaluation of Abicipar Pegol (an Anti-VEGF DARPin Therapeutic) in Neovascular Age-Related Macular Degeneration. J Ocul Pharmacol Ther. 2018 Dec;34(10):700-709. doi: 10.1089/jop.2018.0062. Epub 2018 Nov 9.

location_country:
Italy
Austria
Switzerland
Germany
France
Israel
Australia
United States


title: A Re-Survey Study of Patients With MPS VI (Maroteaux-Lamy Syndrome) Who Previously Participated in ASB-00-02

summary: There is limited information on the long-term effects of treating patients with MPS VI with Naglazyme® and limited data on the natural history of treated and untreated MPS VI patients. The Re-survey Study ASB-00-03 will assist in understanding the effects of long-term Naglazyme treatment and the natural history of the MPS VI patient population.

ReferenceCitation: Giugliani R, Lampe C, Guffon N, Ketteridge D, Leão-Teles E, Wraith JE, Jones SA, Piscia-Nichols C, Lin P, Quartel A, Harmatz P. Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)--10-year follow-up of patients who previously participated in an MPS VI Survey Study. Am J Med Genet A. 2014 Aug;164A(8):1953-64. doi: 10.1002/ajmg.a.36584. Epub 2014 Apr 24.

name: Medical Genetics ServiceHCPADepartment of GeneticsUFRGSand INAGEMPPorto AlegreBrazil

family: Giugliani

full_name: Roberto Giugliani

given: Roberto

name: Department of Pediatric and Adolescent MedicineVilla MetabolicaUniversity Medical Center of the University of MainzMainzGermany

family: Lampe

full_name: Christina Lampe

given: Christina

family: Guffon

full_name: Nathalie Guffon

given: Nathalie

name: Women's and Children's HospitalNorth AdelaideAustralia

family: Ketteridge

full_name: David Ketteridge

given: David

name: Hospital Pediátrico IntegradoCentro Hospitalar de S. JoãoPortoPortugal

family: Leão‐Teles

full_name: Elisa Leão‐Teles

given: Elisa

name: Manchester Children's HospitalCentre for Genomic MedicineSt Mary's HospitalCMFTUniversity of ManchesterManchesterUnited Kingdom

family: Wraith

full_name: James E. Wraith

given: James E.

family: Jones

full_name: Simon A. Jones

given: Simon A.

family: Piscia‐Nichols

full_name: Cheri Piscia‐Nichols

given: Cheri

family: Lin

full_name: Ping Lin

given: Ping

family: Quartel

full_name: Adrian Quartel

given: Adrian

name: Children's Hospital & Research Center OaklandOaklandCalifornia

family: Harmatz

full_name: Paul Harmatz

given: Paul

journal_name: American Journal of Medical Genetics Part A

version: acceptedVersion

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

title: Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux–Lamy syndrome)—10‐year follow‐up of patients who previously participated in an MPS VI survey study

lead_sponsor: BioMarin Pharmaceutical

location_country:
Brazil
Germany
France
Australia
Portugal
United States
United Kingdom

location_facility: Manchester Academic Health Sciences Centre


title: An Open-label, Multicenter, Multinational Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid Alpha-glucosidase Treatment in Patients Less Than 6 Months Old With Infantile-onset Pompe Disease

summary: Pompe disease (also known as glycogen storage disease type II, "GSD-II") 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 Pompe disease, 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 Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are less than or equal to 6 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: Li

full_name: Jennifer S. Li

given: Jennifer S.

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

oa_host_type: repository

location_country:
France
Israel
United States
United Kingdom
Taiwan

location_facility: University of Florida College of Medicine

intervention_type: Biological


type: Registry Identifier

title: A Randomized Trial of Adjuvant Treatment With Radiation Plus Chemotherapy Versus Radiation Alone in High Risk Endometrial Carcinoma

summary:
RATIONALE: Radiation therapy uses high-energy x-rays to damage tumor cells. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. It is not yet known whether radiation therapy with chemotherapy is more effective than radiation therapy alone in treating high-risk endometrial cancer.

PURPOSE: This randomized phase III trial is studying radiation therapy and chemotherapy to see how well they work compared to radiation therapy alone in treating patients with high-risk endometrial cancer.

ReferenceCitation: Hogberg T, Signorelli M, de Oliveira CF, Fossati R, Lissoni AA, Sorbe B, Andersson H, Grenman S, Lundgren C, Rosenberg P, Boman K, Tholander B, Scambia G, Reed N, Cormio G, Tognon G, Clarke J, Sawicki T, Zola P, Kristensen G. Sequential adjuvant chemotherapy and radiotherapy in endometrial cancer--results from two randomised studies. Eur J Cancer. 2010 Sep;46(13):2422-31. doi: 10.1016/j.ejca.2010.06.002. Epub 2010 Jul 7.

ReferenceCitation: Hogberg T, Rosenberg P, Kristensen G, et al.: A randomized phase-III study on adjuvant treatment with radiation (RT) ± chemotherapy (CT) in early-stage high-risk endometrial cancer (NSGO-EC-9501/EORTC 55991). [Abstract] J Clin Oncol 25 (Suppl 18): A-5503, 274s, 2007.

lead_sponsor: Nordic Society of Gynaecological Oncology - Clinical Trials Unit

location_country:
Italy
South Africa
Netherlands
Spain
France
Poland
Norway
Ireland
Belgium
Portugal
United Kingdom

location_facility: Centre for Cancer Research and Cell Biology at Belfast City Hospital

location_facility: Fondazione I.R.C.C.S. Policlinico San Matteo

location_facility: Hospitais da Universidade de Coimbra (HUC)

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

location_facility: Cazk Groeninghe - Campus Maria's Voorzienigheid

location_facility: Hospital Universitario Central de Asturias

location_facility: Universitair Medisch Centrum St. Radboud - Nijmegen


title: A Randomised Trial in Relapsed Ovarian Cancer: Early Treatment Based on CA 125 Levels Alone Vs. Delayed Treatment Based On Conventional Clinical Indicators

summary:
RATIONALE: It is not yet known if treatment for recurrent ovarian epithelial, fallopian tube, or primary peritoneal cancer is more effective if it is begun when blood levels of CA 125 become elevated rather than waiting for other indicators of disease recurrence.

PURPOSE: This randomized phase III trial is studying early chemotherapy based on blood levels of CA 125 alone to see how well it works compared to chemotherapy based on conventional clinical indicators in patients with recurrent ovarian epithelial, fallopian tube, or primary peritoneal cancer.

ReferenceCitation: Markman M, Petersen J, Belland A, Burg K. CA-125 monitoring in ovarian cancer: patient survey responses to the results of the MRC/EORTC CA-125 Surveillance Trial. Oncology. 2010;78(1):1-2. doi: 10.1159/000292352. Epub 2010 Mar 6.

ReferenceCitation: Rustin GJ, van der Burg ME, Griffin CL, Guthrie D, Lamont A, Jayson GC, Kristensen G, Mediola C, Coens C, Qian W, Parmar MK, Swart AM; MRC OV05; EORTC 55955 investigators. Early versus delayed treatment of relapsed ovarian cancer (MRC OV05/EORTC 55955): a randomised trial. Lancet. 2010 Oct 2;376(9747):1155-63. doi: 10.1016/S0140-6736(10)61268-8.

family: Rustin

full_name: Gordon JS Rustin

given: Gordon JS

family: van der Burg

full_name: Maria EL van der Burg

given: Maria EL

family: Griffin

full_name: Clare L Griffin

given: Clare L

family: Guthrie

full_name: David Guthrie

given: David

family: Lamont

full_name: Alan Lamont

given: Alan

family: Jayson

full_name: Gordon C Jayson

given: Gordon C

family: Kristensen

full_name: Gunnar Kristensen

given: Gunnar

family: Mediola

full_name: César Mediola

given: César

family: Coens

full_name: Corneel Coens

given: Corneel

family: Qian

full_name: Wendi Qian

given: Wendi

family: Parmar

full_name: Mahesh KB Parmar

given: Mahesh KB

family: Swart

full_name: Ann Marie Swart

given: Ann Marie

journal_name: The Lancet

ReferenceCitation: Rustin GJ, van der Burg ME: A randomized trial in ovarian cancer (OC) of early treatment of relapse based on CA125 level alone versus delayed treatment based on conventional clinical indicators (MRC OV05/EORTC 55955 trials). [Abstract] J Clin Oncol 27 (Suppl 18): A-1, 2009.

lead_sponsor: Medical Research Council

location_country:
Italy
Austria
South Africa
Netherlands
Spain
France
Ireland
Belgium
Portugal
United Kingdom

location_facility: Mount Vernon Cancer Centre at Mount Vernon Hospital

location_facility: Academisch Medisch Centrum at University of Amsterdam

location_facility: Daniel Den Hoed Cancer Center at Erasmus Medical Center

location_facility: Nijmegen Cancer Center at Radboud University Medical Center

location_facility: Academisch Ziekenhuis der Vrije Universiteit Brussel


title: A Multicenter, Randomized, Double-blind, Placebo Controlled, Phase 1b/2a Study of WVE-003 Administered Intrathecally in Patients With Huntington's Disease

summary: This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of WVE-003 in adult patients with early-manifest HD who carry the targeted single nucleotide polymorphism (SNP) - SNP3.

status: Recruiting

lead_sponsor: Wave Life Sciences Ltd.

location_country:
Germany
France
Poland
Australia
Denmark
Canada
United Kingdom

location_facility: Centre Hospitalier de l-Universite de Montreal

location_facility: Institut du Cerveau et de la Moelle Epiniere

location_facility: Royal Devon and Exeter Hospital NHS Trust

location_facility: Cardiff University, Schools of Medicine and Biosciences


title: A Multicenter, Open-label, Phase 2 Basket Study of MK-7684A, a Co-formation of Vibostolimab (MK-7684) With Pembrolizumab (MK-3475), With or Without Other Anticancer Therapies in Participants With Selected Solid Tumors

summary: The purpose of this study is to determine the safety, tolerability, and preliminary efficacy of pembrolizumab/vibostolimab co-formulation (MK-7684A) with or without other anticancer therapies in participants with selected advanced solid tumors. The primary hypothesis is that pembrolizumab/vibostolimab co-formulation is superior to pembrolizumab alone in terms of objective response rate or progression-free survival in participants with cervical cancer.

ipd_sharing: Yes

lead_sponsor: Merck Sharp & Dohme Corp.

location_country:
Chile
Netherlands
France
Japan
Poland
Israel
Spain
United States
Korea, Republic of
Taiwan

location_facility: NATIONAL CHENG-KUNG UNI. HOSP.-clinical trial center ( Site 1302)

location_facility: Szpital Wojewódzki im. Mikoaja Kopernika w Koszalinie-Oddzial Dzienny Chemioterapii ( Site 1104)

location_facility: James Lind Centro de Investigación del Cáncer ( Site 1404)

location_facility: Sourasky Medical Center-Oncology ( Site 1143)

location_facility: Hadassah Medical Center-Oncology ( Site 1142)

location_facility: Sheba Medical Center-ONCOLOGY ( Site 1144)

location_facility: National Taiwan University Hospital-Oncology ( Site 1301)

location_facility: HOSPITAL UNIVERSITARIO QUIRONSALUD MADRID-ONCOLOGIA MEDICA ( Site 1117)

location_facility: National Cancer Center Hospital ( Site 1322)

location_facility: Hospital Universitario Ramón y Cajal-Medical Oncology ( Site 1111)

location_facility: Severance Hospital, Yonsei University Health System-Medical oncology ( Site 1311)

location_facility: HOSPITAL UNIVERSITARIO VIRGEN DEL ROCIO ( Site 1114)

location_facility: City of Hope Comprehensive Cancer Center ( Site 1001)

location_facility: Nederlands Kanker Instituut - Antoni van Leeuwenhoek (NKI-AVL) ( Site 1121)

location_facility: Narodowy Instytut Onkologii im. Marii Sklodowskiej-Curie - P-Oddzial Badan Wczesnych Faz ( Site 1101

location_facility: Instituto Catalan de Oncologia - Hospital Duran i Reynals-Medical Oncology ( Site 1113)

location_facility: Centre Georges François Leclerc ( Site 1155)

location_facility: National Cancer Center Hospital East ( Site 1321)

location_facility: Rambam Health Care Campus-Oncology ( Site 1141)

location_facility: Seoul National University Hospital-Internal Medicine ( Site 1312)


title: A Phase 3, Randomized Study to Evaluate the Efficacy and Safety of Pembrolizumab (MK-3475) + Lenvatinib (E7080/MK-7902) + Chemotherapy Compared With Standard of Care as First-line Intervention in Participants With Metastatic Esophageal Carcinoma

summary:
The purpose of this study is to assess the efficacy and safety of pembrolizumab plus lenvatinib plus chemotherapy compared with pembrolizumab plus chemotherapy as first-line intervention in participants with metastatic esophageal carcinoma

The primary hypotheses are that pembrolizumab plus lenvatinib plus chemotherapy is superior to pembrolizumab plus chemotherapy with respect to overall survival (OS) and progression-free survival (PFS) per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1) by blinded independent central review (BICR).

location_country:
Chile
Guatemala
France
Japan
Spain
Turkey
Denmark
Korea, Republic of
Taiwan

location_facility: Fundacion Arturo Lopez Perez FALP ( Site 0403)

location_facility: China Medical University Hospital ( Site 6003)

location_facility: National Cancer Center Hospital East ( Site 9002)

location_facility: Bradford Hill Centro de Investigaciones Clinicas ( Site 0404)

location_facility: Centro Regional de Sub Especialidades Medicas SA ( Site 0604)

location_facility: Korea University Guro Hospital ( Site 5001)

location_facility: Hospital General Universitari Vall d Hebron ( Site 1607)

location_facility: Taipei Veterans General Hospital ( Site 6006)

location_facility: National Cancer Center Hospital ( Site 9001)

location_facility: Hacettepe Universitesi Tip Fakultesi Hastanesi ( Site 1701)

location_facility: Centro de Investigacion y desarrollo Oncologico SpA - CIDO SpA ( Site 0401)

location_facility: National Cheng Kung University Hospital ( Site 6004)

location_facility: Institut du Cancer de Montpellier ( Site 1002)

location_facility: Hospital Universitario Marques de Valdecilla ( Site 1602)


title: A Multicenter Study to Estimate the Prevalence of HER2 Low and Describe the SoC, Treatment Patterns, and Outcome in Real-world Practice Among Unresectable and/or Metastatic Breast Cancer Patients With HER2-low Status - the RetroBC-HER2L Study

summary: This is a worldwide, multicenter, non-interventional, retrospective study of patient medical records from metastatic breast cancer (mBC) patients previously identified as human epidermal growth factor receptor 2 negative (HER2-neg), regardless of hormone status.

ipd_sharing_description:
Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal.

All request will be evaluated as per the AZ disclosure commitment:

https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

lead_sponsor: AstraZeneca

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


title: Psychosocial Care During the COVID-19 Pandemic in Acute Hospitals - an International Online Survey of Psychosomatic, Psychiatric and Psychological Consultation and Liaison (C&L) Services

summary: Heads of all psychosomatic, psychiatric and psychological consultation and liaison (C&L) services in acute care hospitals across all participating countries will be contacted and asked to participate in this international cross-sectional online survey. The survey consists of questions about care structures and their utilisation that psychosomatic, psychiatric and psychological C&L services have established in acute hospitals in the context of the COVID 19 pandemic.

time_perspective: Cross-Sectional

lead_sponsor: University Hospital, Basel, Switzerland

location_country:
Italy
Austria
Switzerland
Spain
Germany
Finland
France
Poland
Greece
Norway
Ireland
Belgium
Portugal
Canada
United Kingdom
Iran, Islamic Republic of

location_facility: University Hospital Cologne, Department of Psychosomatics and Psychotherapy

location_facility: Medical University of Graz, University Hospital Graz

location_facility: Nuremberg Hospital, Department of Psychosomatic Medicine and Psychotherapy

location_facility: University of Basel, Department of Psychiatry (UPK)

location_facility: Université de Paris, Service de Psychiatrie de l'adulte

location_facility: University of Medical Sciences, Department of Psychiatry

location_facility: University of Montreal, Department of Psychosomatic Medicine

location_facility: University Hospital Basel, Department of Psychosomatic Medicine

location_facility: Ghent University Hospital, Department of Psychiatry

location_facility: TorontoSinai Health, University of Toronto, Department of Psychiatry

location_facility: Oulu University Hospital and University of Oulu


title: A Phase 1, Open-Label, Multicenter Study of INCB106385 as Monotherapy or in Combination With Immunotherapy in Participants With Advanced Solid Tumors

summary: This is a multicenter, open-label, dose-escalation/dose-expansion Phase 1 clinical study to investigate the safety, tolerability, PK profile, pharmacodynamics, and preliminary clinical efficacy of INCB106385 when given as monotherapy or in combination with INCMGA00012 in participants with selected CD8 T-cell-positive advanced solid tumors including SCCHN, NSCLC, ovarian cancer, CRPC, TNBC, or bladder cancer

ipd_sharing_description:
Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

The trial data availability is according to the criteria and process described on https://www.incyte.com/our-company/compliance-and-transparency

lead_sponsor: Incyte Corporation

location_country:
United States
France
Spain

location_facility: Hospital General Universitario Vall D Hebron

location_facility: Institut Universitaire Du Cancer de Toulouse Oncopole

location_facility: University of Maryland - Greenebaum Cancer Center


ReferenceCitation: Atkinson MA, Eisenbarth GS, Michels AW. Type 1 diabetes. Lancet. 2014 Jan 4;383(9911):69-82. doi: 10.1016/S0140-6736(13)60591-7. Epub 2013 Jul 26. Review.

ReferenceCitation: Lachin JM, McGee P, Palmer JP; DCCT/EDIC Research Group. Impact of C-peptide preservation on metabolic and clinical outcomes in the Diabetes Control and Complications Trial. Diabetes. 2014 Feb;63(2):739-48. doi: 10.2337/db13-0881. Epub 2013 Oct 2.

ReferenceCitation: Sørensen JS, Johannesen J, Pociot F, Kristensen K, Thomsen J, Hertel NT, Kjaersgaard P, Brorsson C, Birkebaek NH; Danish Society for Diabetes in Childhood and Adolescence. Residual β-Cell function 3-6 years after onset of type 1 diabetes reduces risk of severe hypoglycemia in children and adolescents. Diabetes Care. 2013 Nov;36(11):3454-9. doi: 10.2337/dc13-0418. Epub 2013 Aug 29.

ReferenceCitation: Ovalle F, Grimes T, Xu G, Patel AJ, Grayson TB, Thielen LA, Li P, Shalev A. Verapamil and beta cell function in adults with recent-onset type 1 diabetes. Nat Med. 2018 Aug;24(8):1108-1112. doi: 10.1038/s41591-018-0089-4. Epub 2018 Jul 9.

ReferenceCitation: Yin T, Kuo SC, Chang YY, Chen YT, Wang KK. Verapamil Use Is Associated With Reduction of Newly Diagnosed Diabetes Mellitus. J Clin Endocrinol Metab. 2017 Jul 1;102(7):2604-2610. doi: 10.1210/jc.2016-3778.

ReferenceCitation: Cooper-Dehoff R, Cohen JD, Bakris GL, Messerli FH, Erdine S, Hewkin AC, Kupfer S, Pepine CJ; INVEST Investigators. Predictors of development of diabetes mellitus in patients with coronary artery disease taking antihypertensive medications (findings from the INternational VErapamil SR-Trandolapril STudy [INVEST]). Am J Cardiol. 2006 Oct 1;98(7):890-4. Epub 2006 Aug 7.

ReferenceCitation: Chen J, Saxena G, Mungrue IN, Lusis AJ, Shalev A. Thioredoxin-interacting protein: a critical link between glucose toxicity and beta-cell apoptosis. Diabetes. 2008 Apr;57(4):938-44. doi: 10.2337/db07-0715. Epub 2008 Jan 2.

ReferenceCitation: Xu G, Chen J, Jing G, Shalev A. Preventing β-cell loss and diabetes with calcium channel blockers. Diabetes. 2012 Apr;61(4):848-56. doi: 10.2337/db11-0955.

ReferenceCitation: Seaquist ER, Anderson J, Childs B, Cryer P, Dagogo-Jack S, Fish L, Heller SR, Rodriguez H, Rosenzweig J, Vigersky R. Hypoglycemia and diabetes: a report of a workgroup of the American Diabetes Association and the Endocrine Society. Diabetes Care. 2013 May;36(5):1384-95. doi: 10.2337/dc12-2480. Epub 2013 Apr 15.

ReferenceCitation: Workgroup on Hypoglycemia, American Diabetes Association. Defining and reporting hypoglycemia in diabetes: a report from the American Diabetes Association Workgroup on Hypoglycemia. Diabetes Care. 2005 May;28(5):1245-9. Review.

lead_sponsor: Medical University of Graz

location_country:
Italy
Austria
Sweden
France
Poland
Belgium
Germany
United Kingdom

location_facility: Slaski Uniwersytet Medyczny w Katowicach

location_facility: Institut National de la Sante er de la Recherche Medicale

location_facility: Medical University of Graz, Department of Internal Medicine Division of Endocrinology and Metabolism

title: A Randomised, Double-blind, Placebo Controlled, Parallel Group, Multi-centre Trial in Adult Subjects With Newly Diagnosed Type 1 Diabetes Mellitus Investigating the Effect of Verapamil SR on Preservation of Beta-cell Function (Ver-A-T1D)

summary:
This study has been set up within the framework of the INNODIA network. INNODIA is a global partnership between 31 academic institutions, 6 industrial partners, a small sized enterprise and 2 patient organizations, bringing their knowledge and experience together with one common goal: "To fight type 1 diabetes". (www.innodia.eu) The overall aim of INNODIA is to advance in a decisive way how to predict, stage, evaluate and prevent the onset and progression of type 1 diabetes (T1D).

For this, INNODIA has established a comprehensive and interdisciplinary network of clinical and basic scientists, who are leading experts in the field of T1D research in Europe and UK (United Kingdom), with complementary expertise from the areas of immunology, Beta-cell biology, biomarker research and T1D therapy, joining forces in a coordinated fashion with industry partners and two foundations, as well as with all major stakeholders in the process, including regulatory bodies and patients with T1D and their families.


title: A Natural History Study of Patients With Genetically Confirmed Primary Hyperoxaluria Type 3 and, as Applicable Per Age, a History of Stone Events

acronym: PHYOX-OBX

summary: This is a natural history study of adults, adolescents, and children (starting at birth) with genetically confirmed primary hyperoxaluria type 3 (PH3) who have a history of stone events during the last 3 years and/or the presence of pre existing stones detected by renal ultrasound at screening.

lead_sponsor: Dicerna Pharmaceuticals, Inc.

location_country:
United States
Lebanon
France
Poland
Germany
United Kingdom
Canada


title: Treatment of Colonic Obstruction With Cook Evolution® Colonic Stent System

summary: The objective of this registry is to compile clinical data on the use of the Evolution® Colonic Stent System for the palliative treatment of colonic obstruction or strictures caused by malignant neoplasms and to relieve acute large bowel obstruction prior to colectomy in patients with malignant strictures.

lead_sponsor: Cook Group Incorporated

location_country:
Italy
Netherlands
France
Spain
Denmark
Canada
United Kingdom

location_facility:
University College of London Hospital
Hospital Universitari de Girona
St. Michael's Hospital
Kingston General Hospital
Hospital Son Llatzer
Instituto Clinico Humanitas IRCCS
Deventer Ziekenhuis
Institut Paoli Calmettes
Odense Universitetshospital
Hospital de Basurto


title: Clinical Experience With the Hindfoot Arthrodesis Nail (HAN) for the Surgical Treatment of Ankle and Hindfoot Pathologies. A Retrospective Case Series.

summary: The aim of this observational study is to collect and evaluate the current clinical experience gained from the Hindfoot Arthrodesis Nail (HAN) for ankle and hindfoot fusions.

lead_sponsor: AO Clinical Investigation and Publishing Documentation

location_country:
Germany
United States
Sweden
France

location_facility: Centre Hospitalier Universitaire d'Angers


ReferenceCitation: Merlini G, Coelho T, Waddington Cruz M, Li H, Stewart M, Ebede B. Evaluation of Mortality During Long-Term Treatment with Tafamidis for Transthyretin Amyloidosis with Polyneuropathy: Clinical Trial Results up to 8.5 Years. Neurol Ther. 2020 Jun;9(1):105-115. doi: 10.1007/s40120-020-00180-w. Epub 2020 Feb 27.

family: Merlini

full_name: Giampaolo Merlini

given: Giampaolo

family: Coelho

full_name: Teresa Coelho

given: Teresa

family: Waddington Cruz

full_name: Márcia Waddington Cruz

given: Márcia

family: Li

full_name: Huihua Li

given: Huihua

family: Stewart

full_name: Michelle Stewart

given: Michelle

family: Ebede

full_name: Ben Ebede

given: Ben

journal_name: Neurology and Therapy

oa_colors: green gold

evidence: oa journal (via doaj)

license: cc-by-nc

license_normalized: cc-by-nc

ReferenceCitation: Huber P, Flynn A, Sultan MB, Li H, Rill D, Ebede B, Gundapaneni B, Schwartz JH. A comprehensive safety profile of tafamidis in patients with transthyretin amyloid polyneuropathy. Amyloid. 2019 Dec;26(4):203-209. doi: 10.1080/13506129.2019.1643714. Epub 2019 Jul 27.

family: Huber

full_name: Peter Huber

given: Peter

family: Flynn

full_name: Alison Flynn

given: Alison

family: Sultan

full_name: Marla B. Sultan

given: Marla B.

family: Rill

full_name: Denise Rill

given: Denise

family: Gundapaneni

full_name: Balarama Gundapaneni

given: Balarama

family: Schwartz

full_name: Jeffrey H. Schwartz

given: Jeffrey H.

journal_name: Amyloid

publisher: Informa UK Limited

publisher_dissemination: Informa

publisher_group: Informa

ReferenceCitation: Amass L, Li H, Gundapaneni BK, Schwartz JH, Keohane DJ. Influence of baseline neurologic severity on disease progression and the associated disease-modifying effects of tafamidis in patients with transthyretin amyloid polyneuropathy. Orphanet J Rare Dis. 2018 Dec 17;13(1):225. doi: 10.1186/s13023-018-0947-7.

ReferenceCitation: Barroso FA, Judge DP, Ebede B, Li H, Stewart M, Amass L, Sultan MB. Long-term safety and efficacy of tafamidis for the treatment of hereditary transthyretin amyloid polyneuropathy: results up to 6 years. Amyloid. 2017 Sep;24(3):194-204. doi: 10.1080/13506129.2017.1357545. Epub 2017 Jul 31.

name: Department of Neurology, Institute for Neurological Research Raúl Carrea, FLENI, Buenos Aires, Argentina;

name: Johns Hopkins University Center for Inherited Heart Disease, Baltimore, MD, USA;

ReferenceCitation: Waddington Cruz M, Amass L, Keohane D, Schwartz J, Li H, Gundapaneni B. Early intervention with tafamidis provides long-term (5.5-year) delay of neurologic progression in transthyretin hereditary amyloid polyneuropathy. Amyloid. 2016 Sep;23(3):178-183. Epub 2016 Aug 5.

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

location_country:
Italy
Brazil
Sweden
Germany
France
Argentina
Portugal
United States

location_facility: Centro per lo Studio e la Cura delle Amiloidosi Sistemiche IRCCS - Policlinico San Matteo

location_facility: Centro Hospitalar Lisboa Norte, EPE- Hospital de Santa Maria

location_facility: Unidade Clinica de Paramiloidose Centro Hospitalar do Porto, EPE - Hospital Geral de Santo António

location_facility: Hospital Universitário Clementino Fraga Filho -HUCFF Universidade Federal do Rio de Janeiro

title: OPEN-LABEL SAFETY AND EFFICACY EVALUATION OF FX-1006A IN SUBJECTS WITH TRANSTHYRETIN (TTR) AMYLOIDOSIS

summary: This is a Phase 3, open-label study designed to obtain additional long-term safety and efficacy data for oral tafamidis (20 mg soft gelatin capsule) administered once daily (QD). In addition, this study continued to provide tafamidis to Val30Met subjects who had completed Protocol Fx-006 (a 1-year, open-label extension study to Protocol Fx-005 which was a randomized, double-blind, placebo-controlled, 18-month study to evaluate the safety and efficacy of tafamidis) or non-Val30Met subjects who had completed Protocol Fx1A-201 (a Phase 2, open-label study to evaluate TTR stabilization, safety, and tolerability of tafamidis) for up to 10 years or until subjects had access to tafamidis for ATTR-PN via prescription. Upon regulatory approval for the treatment of ATTR-PN in their respective country and access to prescription tafamidis, subjects may have been withdrawn from the study. Such subjects were considered study completers.


title: Renal Denervation in Patients With Uncontrolled Hypertension (Symplicity HTN-2)

summary: An international, multi-center, prospective, randomized, controlled study of the safety and effectiveness of renal denervation in patients with uncontrolled hypertension.

ReferenceCitation: Symplicity HTN-2 Investigators, Esler MD, Krum H, Sobotka PA, Schlaich MP, Schmieder RE, Böhm M. Renal sympathetic denervation in patients with treatment-resistant hypertension (The Symplicity HTN-2 Trial): a randomised controlled trial. Lancet. 2010 Dec 4;376(9756):1903-9. doi: 10.1016/S0140-6736(10)62039-9. Epub 2010 Nov 17.

ReferenceCitation: Eikelis N, Hering D, Marusic P, Duval J, Hammond LJ, Walton AS, Lambert EA, Esler MD, Lambert GW, Schlaich MP. The Effect of Renal Denervation on Plasma Adipokine Profile in Patients with Treatment Resistant Hypertension. Front Physiol. 2017 May 30;8:369. doi: 10.3389/fphys.2017.00369. eCollection 2017.

family: Eikelis

full_name: Nina Eikelis

given: Nina

family: Hering

full_name: Dagmara Hering

given: Dagmara

family: Marusic

full_name: Petra Marusic

given: Petra

family: Duval

full_name: Jacqueline Duval

given: Jacqueline

family: Hammond

full_name: Louise J. Hammond

given: Louise J.

family: Walton

full_name: Antony S. Walton

given: Antony S.

family: Lambert

full_name: Elisabeth A. Lambert

given: Elisabeth A.

family: Esler

full_name: Murray D. Esler

given: Murray D.

full_name: Gavin W. Lambert

given: Gavin W.

family: Schlaich

full_name: Markus P. Schlaich

given: Markus P.

currency_apc_doaj: USD

journal_name: Frontiers in Physiology

publisher: Frontiers Media SA

publisher_dissemination: Frontiers

publisher_group: Frontiers

publisher_normalized: Frontiers

ReferenceCitation: Schmieder RE, Ott C, Schmid A, Friedrich S, Kistner I, Ditting T, Veelken R, Uder M, Toennes SW. Adherence to Antihypertensive Medication in Treatment-Resistant Hypertension Undergoing Renal Denervation. J Am Heart Assoc. 2016 Feb 12;5(2). pii: e002343. doi: 10.1161/JAHA.115.002343.

name: Department of Nephrology and Hypertension, University Hospital, Friedrich‐Alexander University Erlangen‐Nürnberg, Erlangen, Germany

family: Schmieder

full_name: Roland E. Schmieder

given: Roland E.

family: Ott

full_name: Christian Ott

given: Christian

name: Department of Diagnostic Radiology, University Hospital, Friedrich Alexander University Erlangen‐Nürnberg, Erlangen, Germany

family: Schmid

full_name: Axel Schmid

given: Axel

family: Friedrich

name: Department of Forensic Toxicology, Institute of Legal Medicine, Frankfurt, Germany

journal_name: Journal of the American Heart Association

publisher: Ovid Technologies (Wolters Kluwer Health)

title: Adherence to Antihypertensive Medication in Treatment‐Resistant Hypertension Undergoing Renal Denervation

ReferenceCitation: Esler MD, Böhm M, Sievert H, Rump CL, Schmieder RE, Krum H, Mahfoud F, Schlaich MP. Catheter-based renal denervation for treatment of patients with treatment-resistant hypertension: 36 month results from the SYMPLICITY HTN-2 randomized clinical trial. Eur Heart J. 2014 Jul;35(26):1752-9. doi: 10.1093/eurheartj/ehu209. Epub 2014 Jun 4.

name: Baker IDI Heart and Diabetes Institute, Monash University, 75 Commercial Road, Melbourne, Victoria 3004, Australia

name: CardioVaskuläres Centrum, Frankfurt, Germany

name: Department of Nephrology, Heinrich-Heine-University, Medical Faculty, Düsseldorf, Germany

name: Department of Nephrology, Hypertension of the University Hospital, Clinical Research Competence Center of Hypertension and Vascular Medicine, Erlangen, Germany

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

ReferenceCitation: Mahfoud F, Ukena C, Schmieder RE, Cremers B, Rump LC, Vonend O, Weil J, Schmidt M, Hoppe UC, Zeller T, Bauer A, Ott C, Blessing E, Sobotka PA, Krum H, Schlaich M, Esler M, Böhm M. Ambulatory blood pressure changes after renal sympathetic denervation in patients with resistant hypertension. Circulation. 2013 Jul 9;128(2):132-40. doi: 10.1161/CIRCULATIONAHA.112.000949. Epub 2013 Jun 18. Erratum in: Circulation. 2014 May 20;129(20):e502.

name: From the Klinik für Innere Medizin III, Kardiologie, Angiologie und Internistische Intensivmedizin, Universitätsklinikum des Saarlandes, Homburg/Saar, Germany (F.M., C.U., B.C., M.B.); Medizinische Klinik IV, Universitätsklinikum Erlangen, Germany (R.E.S., C.O.); Klinik für Nephrologie, Universitätsklinikum Düsseldorf, Germany (L.C.R., O.V.); Medizinische Klinik 2, Universitätsklinikum Schleswig-Holstein, Lübeck, Germany (J.W.); Klinik für Kardiologie, Klinikum München-Bogenhausen, Germany (M.S.);...

title: Ambulatory Blood Pressure Changes After Renal Sympathetic Denervation in Patients With Resistant Hypertension

ReferenceCitation: Esler MD, Krum H, Schlaich M, Schmieder RE, Böhm M, Sobotka PA; Symplicity HTN-2 Investigators. Renal sympathetic denervation for treatment of drug-resistant hypertension: one-year results from the Symplicity HTN-2 randomized, controlled trial. Circulation. 2012 Dec 18;126(25):2976-82. doi: 10.1161/CIRCULATIONAHA.112.130880.

name: From the Baker IDI Heart and Diabetes Institute, Melbourne, Australia (M.D.E., M.S.); Monash Centre of Cardiovascular Research and Education in Therapeutics, School of Public Health and Preventive Medicine, Monash University, Melbourne, Australia (H.K.); Clinical Research Competence Center of Hypertension and Vascular Medicine, Department of Nephrology and Hypertension of University Hospital, Erlangen, Germany (R.E.S.); Universitätsklinium des Saarlandes, Homburg/Saar, Germany (M.B.); and Ohio State...

title: Renal Sympathetic Denervation for Treatment of Drug-Resistant Hypertension

ReferenceCitation: Ukena C, Mahfoud F, Kindermann I, Barth C, Lenski M, Kindermann M, Brandt MC, Hoppe UC, Krum H, Esler M, Sobotka PA, Böhm M. Cardiorespiratory response to exercise after renal sympathetic denervation in patients with resistant hypertension. J Am Coll Cardiol. 2011 Sep 6;58(11):1176-82. doi: 10.1016/j.jacc.2011.05.036.

journal_name: Journal of the American College of Cardiology

title: Cardiorespiratory Response to Exercise After Renal Sympathetic Denervation in Patients With Resistant Hypertension

ReferenceCitation: Mahfoud F, Schlaich M, Kindermann I, Ukena C, Cremers B, Brandt MC, Hoppe UC, Vonend O, Rump LC, Sobotka PA, Krum H, Esler M, Böhm M. Effect of renal sympathetic denervation on glucose metabolism in patients with resistant hypertension: a pilot study. Circulation. 2011 May 10;123(18):1940-6. doi: 10.1161/CIRCULATIONAHA.110.991869. Epub 2011 Apr 25.

name: From the Klinik für Innere Medizin III, Universitätsklinikum des Saarlandes, Homburg/Saar, Germany (F.M., I.K., C.U., B.C., M.B.); Baker IDI Heart and Diabetes Institute, Melbourne, Victoria, Australia (M.S., M.E.); Herzzentrum der Universität zu Köln, Klinik III für Innere Medizin, Köln, Germany (M.C.B., U.C.H.); Klinik für Nephrologie, Universitätskliniken Düsseldorf, Germany (O.V., L.C.R.); Ohio State University, Columbus, and Ardian, Inc., Palo Alto, CA (P.A.S.); and Centre of Cardiovascular...

title: Effect of Renal Sympathetic Denervation on Glucose Metabolism in Patients With Resistant Hypertension

ReferenceCitation: Schlaich MP, Straznicky N, Grima M, Ika-Sari C, Dawood T, Mahfoud F, Lambert E, Chopra R, Socratous F, Hennebry S, Eikelis N, Böhm M, Krum H, Lambert G, Esler MD, Sobotka PA. Renal denervation: a potential new treatment modality for polycystic ovary syndrome? J Hypertens. 2011 May;29(5):991-6. doi: 10.1097/HJH.0b013e328344db3a.

location_country:
Austria
Switzerland
United Kingdom
France
Poland
Australia
Belgium
Spain
Germany
Latvia

location_facility: Universitatsklinikum Schleswig-Holstein, Luebeck Campus

location_facility: Pauls Stradins Clinical University Hospital

location_facility: Barts and the London School of Dentistry & Medicine


title: A Non-comparative, Multi-centre Study to Evaluate the Safety and Efficacy of the DePuy Silent™ Hip Prosthesis in Primary Total Hip Replacement

summary: The purpose of this study is to determine if the Silent™ hip is effective in the treatment of patients with hip joint disease requiring a total hip replacement. Patients who enter the study will be evaluated at regular intervals following hip surgery using patient, clinical and x-ray assessments. A subset of patients will undergo scans to allow the bone mineral density of the bone surrounding the implant to be monitored

lead_sponsor: DePuy International

location_country:
Italy
France
Australia
Germany
United Kingdom

location_facility:
Sydney Adventist Hospital
Ospedali Riuniti di Jesi
Hôpital Raymond Poincaré
Llandough Hospital
Orthopädishe Universitätsklinik
Royal Newcastle Hospital
Prince of Wales Private Hospital


title: International PMS Study - KOGENATE Bayer

summary:
To evaluate long-term safety (primarily by recording adverse events including inhibitors), efficacy and patient acceptance of KOGENATE Bayer in home treatment either on prophylaxis or on demand.

To evaluate both safety and efficacy with respect to lot variability, in particular regarding lot-groups formulated with or without fix between.

lead_sponsor: Bayer

location_country: Italy Austria Sweden Switzerland Netherlands France Greece Belgium Spain Denmark


lead_sponsor: Sanofi

location_country:
United States
Finland
France
Norway
Spain
Germany
Canada

location_facility:
Sanofi-aventis Administrative Office
Sanofi-Aventis Administrative Office

title: A 14-Week, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy, Safety, and Tolerability of Nerispirdine 50 mg, 100 mg, and 200 mg in Patients With Multiple Sclerosis.

summary:
The primary objective is to assess the activity of nerispirdine in improving the ability to walk, in patients with multiple sclerosis (MS).

Secondary objectives:

To assess other measures of walking ability, tiredness, and lower limb muscular strength, spasticity, clinical assessment by subject and clinical assessment of change by the Study Investigator
To assess the safety and tolerance of nerispirdine
To evaluate the pharmacokinetics (PK) parameters of nerispirdine


location_country:
Austria
Germany
France
Poland
Hungary
United States
Canada

acronym: ECLIPSE

title: Efficacy and Safety of Eplivanserin 5mg/Day in Insomnia Characterized by Sleep Maintenance Difficulties: a 6-week, Randomized, Double-blind, Placebo-controlled, Polysomnography Study.

summary:
Primary objective:

- To assess the efficacy of eplivanserin 5mg/day in comparison to placebo after 6 weeks of treatment on sleep maintenance of insomniac patients, as measured by Polysomnography Wake Time After Sleep Onset (PSG-WASO) and Polysomnography Number of Awakenings (PSG-NAW).

Secondary objectives:

To evaluate the effects of eplivanserin 5mg/day as compared to placebo after 6 weeks of treatment on other sleep parameters measured by PSG recordings (Total Sleep Time - PSG-TST, Sleep Efficiency - PSG-SE, Latency to Persistent Sleep - PSG-LPS) and reported by patients (Wake Time After Sleep Onset - pr-WASO, Number of Awakenings - pr-NAW, Total Sleep Time - pr-TST, Quality of Sleep - QoS and Refreshing Quality of Sleep - RqoS).
To evaluate the effects of eplivanserin 5mg/day on sleep architecture compared to placebo.
To evaluate the effect of eplivanserin 5mg/day on daytime functioning using the Sleep Impact Scale (SIS), as compared with placebo after 6 weeks of treatment.
To evaluate patient's impression of treatment effects using the Patient's Global Impression questionnaire.
To evaluate the potential for next-day residual effects (using patient's morning questionnaire and psychometric tests) with eplivanserin 5mg/day as compared to placebo.
To evaluate the potential for rebound insomnia following abrupt discontinuation of eplivanserin 5mg/day in comparison with placebo.
To evaluate the effect of eplivanserin, compared to placebo, on the quality of life of patients with primary insomnia using the SF-36 Health Survey.
To evaluate the clinical safety and tolerability of eplivanserin 5mg/day compared to placebo.