title: A Multicenter, Open-Label, Randomized, Phase III Trial Comparing Immediate Adjuvant Hormonal Therapy (ELIGARD®- Leuprolide Acetate) in Combination With TAXOTERE® (Docetaxel) Administered Every Three Weeks Versus Hormonal Therapy Alone Versus Deferred Therapy Followed by the Same Therapeutic Options in Patients With Prostate Cancer at High Risk of Relapse After Radical Prostatectomy

summary:
This is a prospective, multicenter, open-label, randomized phase III study in participants at high risk of recurrent prostate cancer after radical prostatectomy. The study will investigate

Treatment with docetaxel (TAXOTERE®) every three weeks (q3w) plus leuprolide acetate (ELIGARD®) versus leuprolide acetate alone (ELIGARD®)
Immediate treatment following prostatectomy versus deferred treatment at the time of relapse

Using a 2x2 factorial design participants will therefore be randomized to

Immediate adjuvant treatment with docetaxel plus leuprolide acetate (chemotherapy and hormonal therapy)
Immediate adjuvant treatment with leuprolide acetate alone (hormonal therapy)
Deferred treatment with docetaxel plus leuprolide acetate (chemotherapy and hormonal therapy)
Deferred treatment with leuprolide acetate alone (hormonal therapy)

Primary Objective:

The primary objective of the study is to compare progression-free survival using a 2x2 factorial design

Secondary Objectives:

To compare the 5-year overall, cancer-specific and metastasis-free survival after systemic treatment between the groups
To compare the safety and tolerability between Docetaxel in combination with leuprolide acetate and leuprolide acetate alone.
To evaluate quality of life as measured by the FACT-P questionnaire.

Originally, 1696 participants were planned in the study (with 424 participants randomized to each arm). However, only a total of 211 participants completed the randomization procedure as of 26 September 2007. Thus, sanofi-aventis, in accordance with the Steering Committee, decided to stop the participant recruitment as of 26 September 2007. Participants who had already signed their Informed Consent (IC) before September 26, 2007 were allowed to enter the randomization if they met eligibility criteria. The final revised number of planned participants to be randomly assigned to the 4 treatment arms was 250, and 228 participants were actually randomized.

The final sample size did not allow all the statistical analyses to be conducted on efficacy data. Therefore, the protocol was amended to reflect the change in the plans for statistical analysis. The study was underpowered to serve as the basis for drawing conclusions regarding efficacy and quality of life (QoL) endpoints.

location_country:
Germany
Italy
Poland
India
Israel
United Kingdom
Australia
Brazil
Netherlands
Austria
Mexico
United States
South Africa
Turkey
Canada
Russian Federation
France


title: A Randomized, Double-blind, Placebo-controlled Phase II/III Trial to Evaluate the Efficacy and Safety of 2 Doses of Oral Liarozole (75 mg od and 150 mg od) Given During 12 Weeks in Lamellar Ichthyosis

summary: Lamellar ichthyosis is a congenital disease of the skin with a generalized scaling. The primary activity of liarozole is considered to be the inhibition of the degradation of a substance called retinoic acid, which is the principal endogenous regulator of growth and differentiation of epithelial tissues in mammals. The current study intends to evaluate the efficacy and safety in patients with lamellar ichthyosis.

status: Completed

study_completion_date_type: Actual

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Actual

ReferenceType: background

ReferenceCitation: Van Wauwe JP, Coene MC, Goossens J, Cools W, Monbaliu J. Effects of cytochrome P-450 inhibitors on the in vivo metabolism of all-trans-retinoic acid in rats. J Pharmacol Exp Ther. 1990 Jan;252(1):365-9.

ReferenceCitation: Van Wauwe J, Van Nyen G, Coene MC, Stoppie P, Cools W, Goossens J, Borghgraef P, Janssen PA. Liarozole, an inhibitor of retinoic acid metabolism, exerts retinoid-mimetic effects in vivo. J Pharmacol Exp Ther. 1992 May;261(2):773-9.

ReferenceCitation: Van Wauwe J, Coene MC, Cools W, Goossens J, Lauwers W, Le Jeune L, Van Hove C, Van Nyen G. Liarozole fumarate inhibits the metabolism of 4-keto-all-trans-retinoic acid. Biochem Pharmacol. 1994 Feb 11;47(4):737-41. doi: 10.1016/0006-2952(94)90137-6.

ReferenceCitation: Kang S, Duell EA, Kim KJ, Voorhees JJ. Liarozole inhibits human epidermal retinoic acid 4-hydroxylase activity and differentially augments human skin responses to retinoic acid and retinol in vivo. J Invest Dermatol. 1996 Aug;107(2):183-7. doi: 10.1111/1523-1747.ep12329579.

ReferenceCitation: Dockx P, Decree J, Degreef H. Inhibition of the metabolism of endogenous retinoic acid as treatment for severe psoriasis: an open study with oral liarozole. Br J Dermatol. 1995 Sep;133(3):426-32. doi: 10.1111/j.1365-2133.1995.tb02672.x.

ReferenceCitation: Berth-Jones J, Todd G, Hutchinson PE, Thestrup-Pedersen K, Vanhoutte FP. Treatment of psoriasis with oral liarozole: a dose-ranging study. Br J Dermatol. 2000 Dec;143(6):1170-6. doi: 10.1046/j.1365-2133.2000.03884.x.

ReferenceCitation: Bhushan M, Burden AD, McElhone K, James R, Vanhoutte FP, Griffiths CE. Oral liarozole in the treatment of palmoplantar pustular psoriasis: a randomized, double-blind, placebo-controlled study. Br J Dermatol. 2001 Oct;145(4):546-53. doi: 10.1046/j.1365-2133.2001.04411.x.

ReferenceCitation: Lucker GP, Heremans AM, Boegheim PJ, van de Kerkhof PC, Steijlen PM. Oral treatment of ichthyosis by the cytochrome P-450 inhibitor liarozole. Br J Dermatol. 1997 Jan;136(1):71-5.

ReferenceType: derived

ReferenceCitation: Vahlquist A, Blockhuys S, Steijlen P, van Rossem K, Didona B, Blanco D, Traupe H. Oral liarozole in the treatment of patients with moderate/severe lamellar ichthyosis: results of a randomized, double-blind, multinational, placebo-controlled phase II/III trial. Br J Dermatol. 2014 Jan;170(1):173-81. doi: 10.1111/bjd.12626.

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via crossref license)

host_type: publisher

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

version: publishedVersion

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

host_type: repository

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

evidence: oa repository (via pmcid lookup)

license_normalized: no license

repositories_concat: PubMed Central

unpaywall_oa_status: hybrid

publisher_dissemination: Oxford University Press

lead_sponsor: Stiefel, a GSK Company

location_country:
Germany
Italy
Norway
Sweden
Netherlands
Belgium
Dominican Republic
Canada
France

location_facility: Fondazione Policlinico Mangiagalli e Regina Elena

location_facility: Academisch Ziekenhuis Vrije Universiteit Brussel

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Completed


title: An Open-Label, Multi-Centre, Extension Study, Evaluating the Long-Term Safety and Tolerability of Different Three-Month Degarelix Dosing Regimens in Patients With Prostate Cancer

summary: The purpose of this extension study was to collect long-term safety and tolerability information to support a marketing authorisation application for a three-month dosage regimen of degarelix.

design_allocation: Non-Randomized

lead_sponsor: Ferring Pharmaceuticals

location_country:
Finland
Germany
Montenegro
United Kingdom
Romania
Netherlands
Belgium
Serbia
Russian Federation
France

location_facility: Clinical Center Novi Sad, Clinic of Urology

location_facility: Mount Vernon Cancer Centre, Marie Cuire Research Wing

location_facility: Clinical Hospital "Prof. Dr. Theodor Burghele", Urology Department

location_facility: "Centrul Medical Privat" Prof. Dr. Ioiart Ioan"

location_facility: Gemeinschaftspraxis Dres Effert und Benedic

location_facility: Clinical Center of Serbia, Institute of Urology and Nephrology

location_facility: Helsinki University Hospital, Maria Hospital, Building 11

location_facility: Fédération d'Urologie et Néphrologie, BP69 Hôpital Pasteur

location_facility: Pavlov State Medical University, Outpatient Diagnostic Center affiliated with the Urology Department

location_facility: Ward 13, NHS Forth Valley Acute Operating Division, Falkirk and District Royal Infirmary, Majors Loans

location_facility: Level 7, Urology Research Unit, Derriford Hospital

location_facility: Pavlov State Medical University, Urology Department


title: An Open-Label, Multicenter Study Evaluating the Long-Term Safety and Efficacy of SR121463B in Patients With Syndrome of Inappropriate Antidiuretic Hormone Secretion

acronym: SIADH Safety

summary:
To determine the long-term safety and tolerability of SR121463B in patients with syndrome of inappropriate antidiuretic secretion (SIADH). To assess the long-term efficacy of SR121463B in maintaining normal levels of serum sodium in patients with SIADH.

Long term safety is evaluated further in an open label extension study with flexible doses of satavaptan.

lead_sponsor: Sanofi

location_country:
Germany
Hungary
Hong Kong
Australia
Brazil
Switzerland
Belgium
United States
Canada
Russian Federation
France

location_facility:
Sanofi-Aventis Administrative Office
sanofi-aventis Canada


title: Evaluation of Two Doses of SR31747A (75 mg and 125 mg) in Non-Metastatic Androgen-Independent Prostate Cancer. Randomized, Double-Blind, Placebo Controlled Phase II Study

acronym: ODYSSEY

summary: To evaluate the efficacy of SR31747 given at 75 or 125mg per day versus placebo in androgen prostate cancer patient without distant metastases with Time to Clinical progression as main objective and PSA parameters, Tumor response, survival , safety,Tumor-related symptoms deterioration Quality of Life, PK analysis as secondary objectives

location_country:
France
Italy
Poland
Portugal
United Kingdom
Australia
Netherlands
Mexico
Belgium
Spain
Chile
Canada
Czech Republic

location_facility: sanofi-aventis Australia & New Zealand administrative office


title: Phase III Randomized Trial of Concomitant Radiation, Cisplatin, and Tirapazamine Versus Concomitant Radiation and Cisplatin in Patients With Advanced Head and Neck Cancer

acronym: TRACE

summary: The trial will compare the efficacy and safety of concomitant chemoradiation with tirapazamine, cisplatin and radiation versus cisplatin and radiation.

collaborators: Groupe Oncologie Radiotherapie Tete et Cou

location_country:
Germany
Italy
Poland
New Zealand
Hungary
Argentina
Hong Kong
Australia
Singapore
Switzerland
United States
Spain
South Africa
Chile
Taiwan
Canada
France

location_facility: sanofi-aventis New Zealand administrative office


type: EudraCT Number

title: Extension to a Multi-Center, Randomized, Crossover, Open Label, Dose Finding Study to Compare the Safety, Efficacy, and Pharmacokinetics/Pharmacodynamics (PK/PD) Relationship of Multiple Doses of Pasireotide (SOM230) (200, 400, and 600 μg Bid) and Doses of Open Label Sandostatin® (SMS) (100 μg Tid) in Acromegalic Patients

summary: Acromegaly is a rare, serious condition characterized by chronic hypersecretion of growth hormone (GH), generally caused by a GH-secreting pituitary adenoma. The study assessed the long-term safety and efficacy of pasireotide in participants with acromegaly.

study_start_date_type: Actual

ReferenceType: result

ReferenceCitation: Petersenn S, Farrall AJ, De Block C, Melmed S, Schopohl J, Caron P, Cuneo R, Kleinberg D, Colao A, Ruffin M, Hermosillo Resendiz K, Hughes G, Hu K, Barkan A. Long-term efficacy and safety of subcutaneous pasireotide in acromegaly: results from an open-ended, multicenter, Phase II extension study. Pituitary. 2014 Apr;17(2):132-40. doi: 10.1007/s11102-013-0478-0. Erratum In: Pituitary. 2014 Apr;17(2):141. Block, Christophe [corrected to De Block, Christophe].

evidence: open (via page says license)

license: cc-by

license_normalized: cc-by

repository_institution: University of Edinburgh - Edinburgh Research Explorer

repository_institution: University of Antwerp - Institutional Repository University of Antwerp

repository_normalized: irua

version: submittedVersion

repositories:
PubMed Central
irua
pure.ed.ac.uk

repositories_concat: PubMed Central;irua;pure.ed.ac.uk

publisher_dissemination: Springer

lead_sponsor: Novartis Pharmaceuticals

location_country:
Germany
Italy
Australia
Switzerland
Belgium
United States
France

location_facility: University of Michigan Health System StudyCoordinatorCSOM230B2201E1

location_facility: Cedars Sinai Medical Center Dept. of Pituitary Ctr.


summary: This study will evaluate the safety and efficacy of an intravitreal implant of dexamethasone for the treatment of macular edema associated with retinal vein occlusion.

ReferenceCitation: Haller JA, Bandello F, Belfort R Jr, Blumenkranz MS, Gillies M, Heier J, Loewenstein A, Yoon YH, Jacques ML, Jiao J, Li XY, Whitcup SM; OZURDEX GENEVA Study Group. Randomized, sham-controlled trial of dexamethasone intravitreal implant in patients with macular edema due to retinal vein occlusion. Ophthalmology. 2010 Jun;117(6):1134-1146.e3. doi: 10.1016/j.ophtha.2010.03.032. Epub 2010 Apr 24.

lead_sponsor: Allergan

location_country:
Germany
Portugal
Israel
Australia
Austria
Mexico
United States
South Africa
Taiwan
Czechia
Canada
Philippines
France


title: A Single Dose, Randomized, 2-Period Crossover Study in Patients With Hemophilia A to Evaluate the Pharmacokinetics of ReFacto (B-domain Deleted Recombinant Human Factor VIII (BDDrFVIII) and Advante (a Full-length Recombinant Factor VIII (FLrFVIII)

summary: The purpose of the study is to compare the pharmacokinetic parameters of ReFacto and Advate, using the chromogenetic substrate assay to measure plasma Factor VIII activity in plasma.

status: Withdrawn

lead_sponsor: Wyeth is now a wholly owned subsidiary of Pfizer

location_country:
Germany
Italy
New Zealand
United Kingdom
Netherlands
Belgium
United States
France

status_simplified: Unknown


title: Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients With Late-Onset Pompe Disease.

summary: Pompe disease (also known as glycogen storage disease Type 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. The overall objective is to evaluate the safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa treatment in patients with late-onset Pompe disease as compared to placebo.

ReferenceCitation: Forsha D, Li JS, Smith PB, van der Ploeg AT, Kishnani P, Pasquali SK; Late-Onset Treatment Study Investigators. Cardiovascular abnormalities in late-onset Pompe disease and response to enzyme replacement therapy. Genet Med. 2011 Jul;13(7):625-31. doi: 10.1097/GIM.0b013e3182142966.

ReferenceCitation: van der Ploeg AT, Clemens PR, Corzo D, Escolar DM, Florence J, Groeneveld GJ, Herson S, Kishnani PS, Laforet P, Lake SL, Lange DJ, Leshner RT, Mayhew JE, Morgan C, Nozaki K, Park DJ, Pestronk A, Rosenbloom B, Skrinar A, van Capelle CI, van der Beek NA, Wasserstein M, Zivkovic SA. A randomized study of alglucosidase alfa in late-onset Pompe's disease. N Engl J Med. 2010 Apr 15;362(15):1396-406. doi: 10.1056/NEJMoa0909859.

lead_sponsor: Genzyme, a Sanofi Company

location_country:
United States
Netherlands
France

location_facility: University of Pittsburgh, Dept. of Neurology


intervention_type: Procedure

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

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Giving chemotherapy drugs before surgery may shrink the tumor so that it can be removed during surgery. Radiation therapy uses high-energy x-rays to kill tumor cells. Combining radiation therapy with chemotherapy may kill more tumor cells. It is not yet known whether chemotherapy is more effective followed by surgery or combined with radiation therapy in treating cervical cancer.

PURPOSE: Randomized phase III trial to compare the effectiveness of chemotherapy followed by radical hysterectomy with that of chemotherapy plus radiation therapy in treating patients who have stage IB or stage II cervical cancer.

location_facility: Queen Elizabeth The Queen Mother Hospital

location_facility: Istituto Nazionale Per Lo Studio E La Cura Dei Tumori

location_facility: Hospitais da Universidade de Coimbra (HUC)

location_facility: Academisch Medisch Centrum at University of Amsterdam

location_facility: NHS Greater Glasgow and Clyde - Beatson West of Scotland Cancer Centre

location_facility: Universitair Medisch Centrum - Academisch Ziekenhuis

location_facility: Universitair Medisch Centrum St. Radboud - Nijmegen

location_facility: Erasmus MC - Daniel den Hoed Cancer Center

location_facility: Centre Hospitalier Regional de la Citadelle

status: Unknown status

title: Randomized Phase III Study Of Neoadjuvant Chemotherapy Followed By Surgery Vs. Concomitant Radiotherapy And Chemotherapy In FIGO Ib2, IIa>4 cm or IIb Cervical Cancer

source: euctr

enrollment_type: Anticipated

location_country:
Italy
Portugal
United Kingdom
Netherlands
Austria
Belgium
Spain
France


title: A Randomized Phase II Study Of CMF Alone And In Combination With Anti c-erbB2 Antibody (Herceptin) In Women With c-erbB2 Positive Metastatic Breast Cancer

summary:
RATIONALE: Drugs used in chemotherapy such as cyclophosphamide, methotrexate, and fluorouracil use different ways to stop tumor cells from dividing so they stop growing or die. Monoclonal antibodies, such as trastuzumab, can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells. Combining chemotherapy with trastuzumab may kill more tumor cells.

PURPOSE: Phase II trial to study the effectiveness of combining combination chemotherapy with trastuzumab in treating women who have metastatic breast cancer.

ReferenceCitation: Neskovic-Konstantinovic Z, Nooij M, Khaled H, et al.: Safety and efficacy of combined trastuzumab and CMF therapy in women with metastatic breast cancer: EORTC protocol 10995. [Abstract] J Clin Oncol 25 (Suppl 18): A-1040, 2007.

ReferenceCitation: Tryfonidis K, Marreaud S, Khaled H, De Valk B, Vermorken J, Welnicka-Jaskiewicz M, Aalders K, Bartlett JMS, Biganzoli L, Bogaerts J, Cameron D; EORTC- Breast Cancer Group. Cardiac safety, efficacy, and correlation of serial serum HER2-extracellular domain shed antigen measurement with the outcome of the combined trastuzumab plus CMF in women with HER2-positive metastatic breast cancer: results from the EORTC 10995 phase II study. Breast Cancer Res Treat. 2017 Jun;163(3):507-515. doi: 10.1007/s10549-017-4203-y. Epub 2017 Mar 21.

oa_host_type: repository

repositories_institution: University of Edinburgh - Edinburgh Research Explorer

unpaywall_oa_status: green

publisher_dissemination: Springer-Nature

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

location_country:
Poland
United Kingdom
Denmark
Netherlands
Belgium
South Africa
Serbia
Egypt
France

location_facility: Nijmegen Cancer Center at Radboud University Medical Center

location_facility: Centre de Lutte Contre le Cancer Georges-Francois Leclerc

location_facility: Herlev Hospital - University Hospital of Copenhagen

location_facility: Institute of Oncology and Radiology of Serbia


title: International Collaborative Treatment Protocol for Infants Under One Year With Acute Lymphoblastic Leukemia

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. It is not yet known which combination chemotherapy regimen is most effective for treating infants with acute lymphoblastic leukemia.

PURPOSE: Randomized phase III trial to compare the effectiveness of different combination chemotherapy regimens in treating infants who have newly diagnosed acute lymphoblastic leukemia.

ReferenceCitation: Hempel G, Relling MV, de Rossi G, Stary J, De Lorenzo P, Valsecchi MG, Barisone E, Boos J, Pieters R. Pharmacokinetics of daunorubicin and daunorubicinol in infants with leukemia treated in the interfant 99 protocol. Pediatr Blood Cancer. 2010 Mar;54(3):355-60. doi: 10.1002/pbc.22266.

ReferenceCitation: Mann G, Attarbaschi A, Schrappe M, De Lorenzo P, Peters C, Hann I, De Rossi G, Felice M, Lausen B, Leblanc T, Szczepanski T, Ferster A, Janka-Schaub G, Rubnitz J, Silverman LB, Stary J, Campbell M, Li CK, Suppiah R, Biondi A, Vora A, Valsecchi MG, Pieters R; Interfant-99 Study Group. Improved outcome with hematopoietic stem cell transplantation in a poor prognostic subgroup of infants with mixed-lineage-leukemia (MLL)-rearranged acute lymphoblastic leukemia: results from the Interfant-99 Study. Blood. 2010 Oct 14;116(15):2644-50. doi: 10.1182/blood-2010-03-273532. Epub 2010 Jun 30.

ReferenceCitation: Lonnerholm G, Valsecchi MG, De Lorenzo P, Schrappe M, Hovi L, Campbell M, Mann G, Janka-Schaub G, Li CK, Stary J, Hann I, Pieters R; Interfant-99 study group. Pharmacokinetics of high-dose methotrexate in infants treated for acute lymphoblastic leukemia. Pediatr Blood Cancer. 2009 May;52(5):596-601. doi: 10.1002/pbc.21925.

ReferenceCitation: van der Linden MH, Valsecchi MG, De Lorenzo P, Moricke A, Janka G, Leblanc TM, Felice M, Biondi A, Campbell M, Hann I, Rubnitz JE, Stary J, Szczepanski T, Vora A, Ferster A, Hovi L, Silverman LB, Pieters R. Outcome of congenital acute lymphoblastic leukemia treated on the Interfant-99 protocol. Blood. 2009 Oct 29;114(18):3764-8. doi: 10.1182/blood-2009-02-204214. Epub 2009 Aug 5.

ReferenceCitation: Van der Velden VH, Corral L, Valsecchi MG, Jansen MW, De Lorenzo P, Cazzaniga G, Panzer-Grumayer ER, Schrappe M, Schrauder A, Meyer C, Marschalek R, Nigro LL, Metzler M, Basso G, Mann G, Den Boer ML, Biondi A, Pieters R, Van Dongen JJ; Interfant-99 Study Group. Prognostic significance of minimal residual disease in infants with acute lymphoblastic leukemia treated within the Interfant-99 protocol. Leukemia. 2009 Jun;23(6):1073-9. doi: 10.1038/leu.2009.17. Epub 2009 Feb 12.

ReferenceCitation: Pieters R, Schrappe M, De Lorenzo P, Hann I, De Rossi G, Felice M, Hovi L, LeBlanc T, Szczepanski T, Ferster A, Janka G, Rubnitz J, Silverman L, Stary J, Campbell M, Li CK, Mann G, Suppiah R, Biondi A, Vora A, Valsecchi MG. A treatment protocol for infants younger than 1 year with acute lymphoblastic leukaemia (Interfant-99): an observational study and a multicentre randomised trial. Lancet. 2007 Jul 21;370(9583):240-250. doi: 10.1016/S0140-6736(07)61126-X.

ReferenceCitation: Pieters R, Schrappe M, de Lorenzo P, et al.: Outcome of infants less than one year of age with acute lymphoblastic leukemia treated with the Interfant-99 protocol. [Abstract] Blood 108 (11): A-145, 2006.

lead_sponsor: Dutch Childhood Oncology Group

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

location_facility: Royal Liverpool Children's Hospital, Alder Hey

location_facility: Sir James Spence Institute of Child Health

location_facility: Our Lady's Hospital for Sick Children Crumlin

location_facility: Nuovo Ospedale San Gerardo at University of Milano-Bicocca

location_facility: Royal Belfast Hospital for Sick Children

location_facility: Royal Marsden NHS Foundation Trust - Surrey

location_facility: Institute of Child Health at University of Bristol

location_facility: Southampton University Hospital NHS Trust

location_facility: Central Manchester and Manchester Children's University Hospitals NHS Trust

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

location_facility: Great Ormond Street Hospital for Children NHS Trust

location_facility: University Medical Center Hamburg - Eppendorf

location_facility: Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute

location_facility: Hopital Universitaire Des Enfants Reine Fabiola

location_facility: Addenbrooke's Hospital at Cambridge University Hospitals NHS Foundation Trust


title: Neuropsychological Evaluation of Psychiatric and Neurological Patients

summary:
This study will allow researchers to use various types of tests to evaluate cognitive and sensory functions. These tests, referred to as "batteries" will evaluate attention, executive functions, general intellectual functioning, language, memory, motor functions, orientation, personality, selected sensory and perceptual functions, vigilance (alertness), and visual-spatial functions. Children and adult patient will receive different test batteries.

The goals of this research study are to;

Create descriptions based on the performance of each patient on the test batteries. Then use this information to relate patient behavior to their neurophysiological, neuroradiological, and biochemical descriptions.
Define subgroups of patients based on their neurobehavior in order to decrease the variability of psychiatric diagnoses, treatments, and prognoses.

study_type: Observational

ReferenceCitation: Johnstone EC, Owens DG, Bydder GM, Colter N, Crow TJ, Frith CD. The spectrum of structural brain changes in schizophrenia: age of onset as a predictor of cognitive and clinical impairments and their cerebral correlates. Psychol Med. 1989 Feb;19(1):91-103. doi: 10.1017/s0033291700011053.

lead_sponsor: National Institute of Mental Health (NIMH)

location_country:
United States
Russian Federation
France

location_facility: National Institutes of Health Clinical Center, 9000 Rockville Pike


title: Evaluation of a Clinical Diagnostic Test for Calcium Release Deficiency Syndrome: The DIAGNOSE CRDS Study

acronym: DIAGNOSE CRDS

summary: Calcium Release Deficiency Syndrome (CRDS) is a novel inherited arrhythmia syndrome secondary to RyR2 loss-of-function that confers a risk of sudden cardiac death. Diagnosis of CRDS presently requires cellular-based in vitro confirmation that an RyR2 variant causes loss-of-function. We hypothesize that CRDS can be diagnosed clinically through evaluation of the repolarization response to brief tachycardia, mediated by cardiac pacing, and a subsequent pause.

status: Recruiting

study_completion_date_type: Anticipated

primary_purpose: Diagnostic

ReferenceCitation: Sun B, Yao J, Ni M, Wei J, Zhong X, Guo W, Zhang L, Wang R, Belke D, Chen YX, Lieve KVV, Broendberg AK, Roston TM, Blankoff I, Kammeraad JA, von Alvensleben JC, Lazarte J, Vallmitjana A, Bohne LJ, Rose RA, Benitez R, Hove-Madsen L, Napolitano C, Hegele RA, Fill M, Sanatani S, Wilde AAM, Roberts JD, Priori SG, Jensen HK, Chen SRW. Cardiac ryanodine receptor calcium release deficiency syndrome. Sci Transl Med. 2021 Feb 3;13(579):eaba7287. doi: 10.1126/scitranslmed.aba7287.

repository_institution: Erasmus University Rotterdam - EUR Research Repository

publisher_dissemination: American Association for the Advancement of Science

ReferenceCitation: Nof E, Belhassen B, Arad M, Bhuiyan ZA, Antzelevitch C, Rosso R, Fogelman R, Luria D, El-Ani D, Mannens MM, Viskin S, Eldar M, Wilde AA, Glikson M. Postpacing abnormal repolarization in catecholaminergic polymorphic ventricular tachycardia associated with a mutation in the cardiac ryanodine receptor gene. Heart Rhythm. 2011 Oct;8(10):1546-52. doi: 10.1016/j.hrthm.2011.05.016. Epub 2011 May 26.

ReferenceCitation: Ormerod JOM, Ormondroyd E, Li Y, Taylor J, Wei J, Guo W, Wang R, Sarton CNS, McGuire K, Dreau HMP, Taylor JC, Ginks MR, Rajappan K, Chen SRW, Watkins H. Provocation Testing and Therapeutic Response in a Newly Described Channelopathy: RyR2 Calcium Release Deficiency Syndrome. Circ Genom Precis Med. 2022 Feb;15(1):e003589. doi: 10.1161/CIRCGEN.121.003589. Epub 2021 Dec 24.

oa_host_type: publisher

license: cc-by-nc

license_normalized: cc-by-nc

repositories_concat: closed

publisher_dissemination: Wolters Kluwer Health

ReferenceCitation: Roston TM, Wei J, Guo W, Li Y, Zhong X, Wang R, Estillore JP, Peltenburg PJ, Noguer FRI, Till J, Eckhardt LL, Orland KM, Hamilton R, LaPage MJ, Krahn AD, Tadros R, Vinocur JM, Kallas D, Franciosi S, Roberts JD, Wilde AAM, Jensen HK, Sanatani S, Chen SRW. Clinical and Functional Characterization of Ryanodine Receptor 2 Variants Implicated in Calcium-Release Deficiency Syndrome. JAMA Cardiol. 2022 Jan 1;7(1):84-92. doi: 10.1001/jamacardio.2021.4458.

repository_institution: Elsevier - Scopus

repository_normalized: Pure (Denmark)

repositories:
PubMed Central
Pure (Denmark)

repositories_concat: PubMed Central;Pure (Denmark)

publisher_dissemination: American Medical Association

ipd_sharing: No

lead_sponsor: Population Health Research Institute

collaborators: Canadian Institutes of Health Research (CIHR)

location_country:
Israel
United Kingdom
Denmark
Belgium
United States
Canada
France

location_facility: London Health Sciences Centre - University Hospital

location_facility: Oxford Heart Centre, John Radcliffe Hospital

location_facility: Institut Universitaire de Cardiologie et de Pneumologie de Québec-Université Laval

intervention_type: Diagnostic Test

status_simplified: Ongoing


type: Other Identifier

title: A Master Rollover Study to Provide Continued Access to and Assess Long-Term Safety of the Study Drug(s)

summary: This open-label, multicenter, rollover study will provide continued treatment for participants deriving benefit from different therapies received in studies sponsored by Daiichi Sankyo, Inc. (DS) or DS/Astra Zeneca (AZ)-sponsored study (eg. DS8201-A-J101, DS8201-A-U201, DS8201-A-U204, DS8201-A-U207, DS8201-A-U303).

study_start_date_type: Anticipated

status: Not yet recruiting

ipd_sharing: Yes

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

lead_sponsor: Daiichi Sankyo, Inc.

sponsor_collaborators:
Daiichi Sankyo, Inc.
AstraZeneca

location_country:
Italy
United Kingdom
Australia
Japan
Belgium
United States
China
Korea, Republic of
Spain
Taiwan
France

location_facility: Complejo Hospitalario Universitario A Coruña

location_facility: Istituto Nazionale Tumori Fondazione G. Pascale

location_facility: Seoul National University Hospital - Site B

location_facility: ICO l'Hospitalet - Hospital Duran i Reynals

location_facility: Seoul National University Hospital - Site A

location_facility: Seoul National University Bundang Hospital

location_facility: Harbin Medical University Cancer Hospital

location_facility: Kyungpook National University Chilgok Hospital


title: Fractional Flow Reserve-guided Percutaneous Coronary Intervention Plus Optimal Medical Treatment Versus Optimal Medical Treatment Alone in Patients With Stable Coronary Artery Disease - FAME II, a 10-year Follow-up Study.

summary:
The FAME-II trial was a prospective, multicenter, multinational, multi-continental, randomized clinical trial with an 'all comers' design.

The overall purpose of the FAME-II trial was to compare the clinical outcomes, safety and cost-effectiveness of FFR-guided PCI plus optimal medical treatment (OMT) versus OMT alone in patients with stable coronary artery disease and in whom both PCI and medical treatment can be considered on the basis of the presently existing scientific evidence.

FAME-II was conducted from 2009 to 2012 and 1-year, 2-year and 5-year results have been published.

The purpose of this 10-Year Follow-up is to evaluate the 10-year major adverse cardiac event rate (MACE, defined as all-cause death, documented myocardial infarction, unplanned hospitalization leading to urgent revascularization).

Patients will have to sign a specific informed consent for the present 10-year follow-up.

This study will be conducted for about approximately 6 months.

time_perspective: Retrospective

lead_sponsor: CoreAalst BV

sponsor_collaborators:
CoreAalst BV
Abbott Medical Devices

location_country:
Germany
Italy
Hungary
United Kingdom
Denmark
Sweden
Netherlands
Belgium
United States
Czechia
Canada
Serbia
France

location_facility: Northern Light Eastern Maine Medical Center

location_facility: Southampton University Hospital - Wessex Cardiac Unit

location_facility: Azienda Ospedaliera Universitaria S. Anna (Ferrara)

location_facility: Masaryk University - Dept. of Internal Cardiology Medicine

location_facility: CHUM (Centre hospitalier de l'Université de Montréal)

location_facility: Gottsegen Hungarian Institute of Cardiology

location_facility: Hopital Cardiovasculaire et Pneumologique Louis Pradel


title: A Phase 1, Open-Label, Dose-Finding Study of BMS-986453, Dual Targeting BCMAxGPRC5D Chimeric Antigen Receptor T Cells, in Participants With Relapsed and/or Refractory Multiple Myeloma

summary: The purpose of this study is to assess BMS-986453 in participants with relapsed and/or refractory multiple myeloma (RRMM).

ipd_sharing_description: BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myers Squibb's data sharing policy and process can be found at: https://www.bms.com/researchers-and-partners/clinical-trials-andresearch/disclosure-commitment.html

lead_sponsor: Juno Therapeutics, Inc., a Bristol-Myers Squibb Company

location_country:
United States
Spain
Germany
France

location_facility:
Local Institution - 0004
Local Institution - 0005
Local Institution - 0017
Local Institution - 0014
Local Institution - 0012
Local Institution - 0016
Local Institution - 0003
Local Institution - 0010
Local Institution - 0018
Local Institution - 0015
Local Institution - 0011
Local Institution - 0019
Local Institution - 0001
Local Institution - 0008
Local Institution - 0006
Local Institution - 0013


title: An Observational Study in Patients With Unresectable Hepatocellular Carcinoma (uHCC) Following Treatment With Atezolizumab Plus Bevacizumab (AB) or With Another Approved Immuno-oncology Immune Checkpoint Inhibitor Combination in First-line

acronym: REFINE-IO

summary:
This is an observational study in which only data will be collected from adults with unresectable hepatocellular carcinoma. These adults should be prescribed a different treatment after treatment with atezolizumab and bevacizumab, or another similar combination of drugs, by their doctors.

Unresectable hepatocellular carcinoma (uHCC) is a type of liver cancer that cannot be treated with surgery.

In the past, sorafenib was the only approved first-line anti-cancer drug for people with uHCC. Regorafenib and other drugs were approved as second-line treatments for uHCC if a person could not take sorafenib or it stopped working for them.

Lately, another first-line (1L) treatment called immuno-oncology (IO) immune checkpoint inhibitor combination (1L-IO combo), like atezolizumab with bevacizumab (AB), has become the preferred choice of treatment. This is because of the meaningful impact on patient survival. 1L-IO combo are drugs that help the body's defense system recognize and kill cancer cells.

Since the other treatments were previously approved for use following sorafenib, the best order to take these treatments in following an 1L-IO combo is unknown.

To better understand and determine this order, more knowledge is needed about how well different treatments work in participants with uHCC who have been treated with AB or another 1L-IO combo.

The main purpose of this study is to learn more about how well different treatments work when given after first-line treatment with AB or another approved 1L-IO combo. To do this, researchers will collect data on how long the participants live (also called overall survival) from the start of any treatment given after the first-line treatment.

In addition, researchers will also collect the following information to learn more about the participants who will be given a different treatment after the 1L-IO combo:

characteristics including age, sex, and race, and signs and symptoms of the participants over the duration of their first-line treatment
the length of time from the first to the last dose (also called duration of therapy) of the treatments given after the 1L-IO combo
the length of time until a participant's cancer worsens, or they die (also called progression free survival) from the start of the treatments given after the 1L-IO combo
the number of participants whose tumor completely disappears or shrinks (also called overall tumor response) after taking the treatments given after the 1L-IO combo
the sequence of treatments given after the 1L-IO combo Data will be collected from September 2023 to December 2026 and cover a period of around 3 years. The data will be collected using medical records or by interviewing the participants during their routine visits to the doctor.

Researchers will observe participants from the start of the treatment given after the 1L-IO combo until the end of their participation in the study.

In this study, only data from routine care will be collected. No visits or tests are required as part of this study.

time_perspective: Prospective

ipd_sharing_description:
Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.

Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.

lead_sponsor: Bayer

location_country:
Italy
Colombia
Argentina
Brazil
Mexico
United States
China
Korea, Republic of
Saudi Arabia
Greece
Spain
Thailand
Taiwan
Turkey
France

location_facility: Roswell Park Comprehensive Cancer Center

location_facility: Corporal Michael J Crescenz Department Of Veterans Affairs Medical Center

location_facility: University Of Cincinnati Division Of Hematology Oncology - UC Health System

location_facility: Mercy Medical Center - Medline Healthcare Services LLC


title: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study of Tarlatamab Therapy in Subjects With Limited-Stage Small-Cell Lung Cancer (LS-SCLC) Who Have Not Progressed Following Concurrent Chemoradiation Therapy

summary: The primary objective of this study is to compare the efficacy of tarlatamab with placebo as assessed by progression free survival (PFS).

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

lead_sponsor: Amgen

location_country:
Germany
Poland
Argentina
Australia
Brazil
Japan
Turkey
France
Portugal
Hong Kong
Switzerland
United States
Romania
Belgium
Italy
United Kingdom
Singapore
Sweden
Austria
Mexico
Spain
Greece
Taiwan
Bulgaria
Israel

location_facility: Hospital General Universitario Gregorio Marañon

location_facility: Instituto Oncologico de Cordoba Ionc Fundacion Richardet Longo

location_facility: Hospital Universitario Puerta de Hierro Majadahonda

location_facility: Instituto do Cancer do Estado de Sao Paulo Octavio Frias de Oliveira Icesp

location_facility: University Multiprofile Hospital for Active Treatment Sofiamed OOD

location_facility: Narodowy Instytut Onkologii im Marii Sklodowskiej-Curie - Panstwowy Instytut Badawczy

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori

location_facility: Institutul Oncologic Prof Dr Ion Chiricuta Cluj-Napoca

location_facility: Kocaeli Universitesi Arastirma ve Uygulama Hastanesi

location_facility: Sanatorio 9 de julio - C.I.C.E. 9 de Julio

location_facility: University Multiprofile Hospital for Active Treatment Tsaritsa Yoanna - ISUL EAD

location_facility: Multiprofile Hospital for Active Treatment - Uni Hospital OOD

location_facility: Institutul Oncologic, Prof Dr Alexandru Trestioreanu

location_facility: Allina Health System dba Virginia Piper Cancer Institute

location_facility: National Hospital Organization Hokkaido Cancer Center

location_facility: Wojewodzki Szpital im Sw Ojca Pio w Przemyslu

location_facility: Centre Hospitalier Universitaire de Marseille - Hopital de la Timone

location_facility: Universitair Ziekenhuis Leuven - Campus Gasthuisberg

location_facility: Wielkopolskie Centrum Pulmonologii i Torakochirurgii imienia Eugenii i Janusza Zeylandow

location_facility: National Hospital Organization Nagoya Medical Center

location_facility: Universitaetsklinikum Allgemeines Krankenhaus Wien

location_facility: The Cancer Institute Hospital of Japanese Foundation for Cancer Research

location_facility: Multiprofile Hospital for Active Treatment Serdika EOOD

location_facility: Specjalistyczny Szpital Onkologiczny NU-MED Sp z o o

location_facility: Centro de pesquisa em oncologia Hospital Ana Nery

location_facility: Military Medical Academy Multiprofile Hospital for Active Treatment - Sofia

location_facility: Hospital Universitari Germans Trias i Pujol

location_facility: National Hospital Organization Shikoku Cancer Center

location_facility: Saitama Medical University International Medical Center

location_facility: Hospital Regional Universitario de Malaga

location_facility: Kaohsiung Medical University Chung-Ho Memorial Hospital

location_facility: Samodzielny Publiczny Szpital Kliniczny nr 4 w Lublinie

location_facility: Hospital Clinico Universitario de Santiago

location_facility: Icon Cancer Centre Singapore - Farrer Park

location_facility: Policlinico Universitario Agostino Gemelli

location_facility: Laura and Isaac Perlmutter Cancer Center at New York University Langone

location_facility: Fundacao Pio 12 Hospital de Amor de Barretos

location_facility: Montefiore Einstein Center for Cancer Care

location_facility: Kanagawa Prefectural Hospital Organization Kanagawa Cancer Center

location_facility: Centrum Pulmunologii i Torakochirurgii w Bystrej

location_facility: Uniwersytecki Szpital Kliniczny w Bialymstoku

location_facility: Hospital Universitario Insular de Gran Canaria

location_facility: Centro Hospitalar de Setubal EPE Hospital de Sao Bernardo

location_facility: Marmara Universitesi Pendik Egitim Arastirma Hastanesi

location_facility: Azienda Ospedaliero Universitaria Pisana, Presidio Cisanello

location_facility: Azienda Ospedaliera di Perugia Ospedale Santa Maria della Misericordia

location_facility: Cipo Centro Integrado de Pesquisa e Servicos Hospitalares em Oncologia Ltda


title: GUIding Multi-moDal thErapies Against MRD by Liquid Biopsies in Colorectal Cancer - GUIDE.MRD-01-CRC

summary:
Improving personalized cancer treatments and finding the best strategies to treat each patient relies on using new diagnostic technologies. Currently, for colorectal cancer, the methods used to decide who gets additional post-surgery treatment are suboptimal. Some patients get too much treatment, while others do not get enough.

There is a new way to explore if there is any cancer left in a patient's body using circulating tumor DNA (ctDNA) detected in blood samples. This can help decide who needs more treatment after surgery. Even though many tests have been developed, it has yet to be determined which test performs best at relevant time points.

The GUIDE.MRD consortium is a group of experts, including scientists, technology, and pharmaceutical companies. The consortium is working on creating a reliable standard for the ctDNA tests, validating their clinical utility, and collecting data to help decide on the best treatment for each patient.

GUIDE.MRD-01-CRC is a part of the GUIDE.MRD project.

lead_sponsor: Claus Lindbjerg Andersen

collaborators: University Medical Centre of Montpellier

location_country: Germany France Denmark Austria Sweden

location_facility: Department of General-, Visceral- and Thoracic Surgery, University Medical Center Hamburg-Eppendorf

location_facility: Abteilung für Onkologie, Medizinische Universität Graz

location_facility: LCCRH (Laboratoire Cellules Circulantes Rares Humaines) - CHU de Montpellier


title: A Randomized, Double-Blind Study Evaluating the Efficacy, Safety, and Immunogenicity of ABP 206 Compared With OPDIVO® (Nivolumab) in Subjects With Treatment-Naïve Unresectable or Metastatic Melanoma

summary: The purpose of this study is to assess the efficacy, safety, and immunogenicity of ABP 206 compared with Nivolumab in Subjects with Treatment-Naïve Unresectable or Metastatic Melanoma.

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

location_country:
Lithuania
United States
Bosnia and Herzegovina
Spain
Georgia
Canada
Estonia
France

location_facility: Hospital Regional Universitario de Malaga

location_facility: Consorcio Hospital General Universitario de Valencia

location_facility: Hospital Universitario Virgen De La Macarena

location_facility: University Clinical Center of Republic of Srpska


title: IDE196 (Darovasertib) in Combination With Crizotinib Versus Investigator's Choice of Treatment as First-line Therapy in HLA-A2 Negative Metastatic Uveal Melanoma (DAR-UM-2)

summary: This is a Phase 2/3, multi-arm, multi-stage, open-label study of human leukocyte antigen (HLA)-A*02:01 negative participants with metastatic uveal melanoma (MUM) who will be randomized to receive either IDE196 + crizotinib or investigator's choice of treatment (pembrolizumab, ipilimumab + nivolumab, or dacarbazine).

lead_sponsor: IDEAYA Biosciences

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

location_facility: The Angeles Clinic and Research Institute

location_facility: LUMC (Leids Universitair Medisch Centrum)

location_facility: Dermatologische Klinik, USZ Flughafen Geschoss 7 - Klinische Forschung

location_facility: University of Miami Sylvester Comprehensive Cancer Center

location_facility: Mount Vernon Cancer Centre East & North Herts NHS Trust

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori

location_facility: Istituto Nazionale dei Tumori Fondazione Pascale

location_facility: Narodowy Instytut Onkologii im. Marii Skłodowskiej-Curie Państwowy Instytut Badawczy

location_facility: The Beatson West of Scotland Cancer Centre

location_facility: Ośrodek Badań Klinicznych Wczesnych Faz, Uniwersyteckie Centrum Kliniczne w Gdańsku

location_facility: The Clatterbridge Cancer Centre NHS Foundation Trust

location_facility: Universitätsklinikum Carl Gustav Carus Dresden

location_facility: Hospital Clínico Universitario de Santiago de Compostela

location_facility: California Pacific Medical Center (CPMC)


title: AT-1501-K207: BESTOW: A Phase 2, Multicenter, Randomized, Open-Label Study to Evaluate the Safety and Efficacy of Tegoprubart in Patients Undergoing Kidney Transplantation

summary: This study will evaluate the safety and efficacy of AT-1501 compared with tacrolimus in patients undergoing kidney transplantation.

lead_sponsor: Eledon Pharmaceuticals

location_country:
United States
Spain
Australia
France

location_facility: University of California, Irvine Medical Center

location_facility: Columbia University Irving Medical Center / New York Presbyterian Hospital


title: Drug-coated Balloon in de Novo Chronic Total Occlusions (CTO-DENOVO Registry)

acronym: CTO-DENOVO

summary: The CTO-DENOVO study is a multicenter registry of consecutive patients with de novo CTO undergoing successful CTO recanalization with the use of DCB-only strategy. The primary endpoint is target lesion failure at 6 months. The secondary endpoints are: 1) late lumen loss on follow-up angiography, and 2) minimal lumen area on follow-up intravascular ultrasound.

lead_sponsor: National Institute of Cardiology, Warsaw, Poland

location_country: Germany Italy Poland Hungary Israel Romania Austria Spain Croatia Czechia France

location_facility: Dr Alfred Sokolowski Specialist Hospital

location_facility: Hospital Clínico Universitario de Valladolid

intervention_type: Device


title: CAMBRIA-2: A Phase III, Open-Label, Randomised Study to Assess the Efficacy and Safety of Camizestrant (AZD9833, a Next Generation, Oral Selective Estrogen Receptor Degrader) vs Standard Endocrine Therapy (Aromatase Inhibitor or Tamoxifen) as Adjuvant Treatment for Patients With ER+/HER2- Early Breast Cancer and an Intermediate-High or High Risk of Recurrence Who Have Completed Definitive Locoregional Treatment and Have No Evidence of Disease

summary: This is a Phase III open-label study to assess if camizestrant improves outcomes compared to standard adjuvant endocrine therapy for patients with ER+/HER2- early breast cancer with intermediate-high or high risk for disease recurrence who completed definitive locoregional therapy (with or without chemotherapy). The planned duration of treatment in either arm within the study will be 7 years.

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

collaborators: Austrian Breast and Colorectal Cancer Study Group (ABCSG)

location_country:
Germany
Ireland
Poland
New Zealand
Argentina
Australia
Brazil
Japan
Thailand
Turkey
Croatia
Chile
Czechia
Canada
Serbia
France
Colombia
Portugal
Hungary
Switzerland
United States
India
Romania
Belgium
China
Malaysia
Peru
Estonia
Philippines
Italy
United Kingdom
Austria
Mexico
Korea, Republic of
Spain
Greece
South Africa
Taiwan
Bulgaria
Georgia
Israel


type: Registry Identifier

title: A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Deucravacitinib in Adults With Active Sjögren's Syndrome (POETYK SjS-1)

acronym: POETYK SjS-1

summary: The purpose of this study is to assess the safety and efficacy of two doses of Deucravacitinib in adult participants with Active Sjögren's Syndrome.

ipd_sharing_description: BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myers Squibb's data sharing policy and process can be found at: https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosurecommitment.html

lead_sponsor: Bristol-Myers Squibb

location_country:
Poland
Colombia
United Kingdom
Argentina
Australia
Brazil
Japan
Austria
Belgium
Mexico
United States
China
Chile
Taiwan
Peru
Canada
Puerto Rico
France

location_facility: Centre De Recherche Musculo-Squelettique

location_facility: New Mexico Clinical Research Osteoporosis

location_facility: Institute for Rheumatic & Autoimmune Diseases, Overlook Medical Center

location_facility: Arthritis & Rheumatology Research Institute


title: A Randomized, Double-blind, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Efficacy and Safety of Izokibep in Subjects With Moderate to Severe Hidradenitis Suppurativa

summary: Izokibep is a small protein molecule that acts as a selective, potent inhibitor of interleukin 17A, to which it binds with high affinity. This study investigates izokibep in subjects with active Hidradenitis Suppurativa (HS), including tumor necrosis factor-alpha inhibitor (TNFi) naïve subjects, and those who had an inadequate response or intolerance to TNFi, or for whom TNFi is contraindicated.

ipd_sharing: Undecided

lead_sponsor: ACELYRIN Inc.

location_country:
Germany
Poland
Hungary
United States
Canada
France


title: A Phase 2 Study to Evaluate the Efficacy and Safety of Ianalumab (VAY736) in Patients With Primary Immune Thrombocytopenia (ITP) Previously Treated With at Least a Corticosteroid and a Thrombopoietin Receptor Agonist (TPO-RA)

summary: The purpose of this study is to evaluate the therapeutic efficacy, safety and tolerability of ianalumab in adult patients with primary ITP previously treated with at least one corticosteroid and one TPO-RA.

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:
Germany
Italy
United Kingdom
Australia
United States
China
Korea, Republic of
Spain
Malaysia
Turkey
Czechia
France

location_facility: Georgetown University Lombardi Cancer Center Dept. of Pharmacy Research (4)

location_facility: New York Oncology Hematology Saratoga NYOH

intervention_type: Biological


title: Open-label Safety Study in Adults and Adolescents With Haemophilia A With and Without FVIII Inhibitors Switching Directly From Emicizumab Prophylaxis to NNC0365-3769 (Mim8) Prophylaxis

acronym: FRONTIER 5

summary: This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.

ipd_sharing_description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

lead_sponsor: Novo Nordisk A/S

location_country:
Germany
Italy
United Kingdom
Japan
Austria
Belgium
United States
Korea, Republic of
Spain
South Africa
Canada
France


title: EvasayilTM : A Placebo-controlled Trial to Evaluate the Efficacy and Safety of Spesolimab in the Treatment of Patients With Netherton Syndrome

summary:
This study is open to people with a skin disease called Netherton syndrome (NS). People can join the study if they are 12 years and older. The purpose of this study is to find out whether a medicine called spesolimab helps people with NS.

Participants are divided into a spesolimab and a placebo group. Placebo injections look like spesolimab injections but do not contain any medicine. Every participant has a 2 in 3 chance of being in the spesolimab group. In the beginning, participants get the study medicine as an injection into a vein. Afterwards, they get it as an injection under the skin every month. After 4 months, participants in the placebo group switch to spesolimab treatment.

Participants are in the study for about 1 year. During this time, they visit the study site 16 times. Where possible, 4 of 16 visits can be done at the participant's home instead of the study site. The doctors regularly check participants' NS symptoms. The results are compared between the groups to see whether spesolimab works. The doctors also regularly check participants' general health and take note of any unwanted effects.

ipd_sharing_description:
Once the criteria in section "Time Frame" are fulfilled, researchers can use the following link https://www.mystudywindow.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed "Document Sharing Agreement".

Furthermore, researchers can request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined in the website.

lead_sponsor: Boehringer Ingelheim

location_country:
Germany
Italy
Portugal
Israel
United Kingdom
Australia
Japan
Switzerland
Austria
Belgium
United States
China
Malaysia
Bulgaria
France

location_facility: Xinhua Hospital Affiliated to Shanghai Jiaotong University

location_facility: Beijing Children's Hospital, Capital Medical University

location_facility: Medical University of Salzburg (SALK) Paracelsus Medical Privatuniversity Salzburg (PMU)

location_facility: Istituto Dermopatico Dell'Immacolata - IDI - IRCCS

location_facility: Dermatology Hospital, Chinese Academy of Medical Sciences

location_facility: Universitätsklinikum Schleswig-Holstein, Campus Kiel

location_facility: CHULC, EPE - Hospital Sto. António Capuchos

location_facility: The First Affiliated Hospital, Zhejiang University

location_facility: Southern Medical University Dermatology Hospital


title: A Randomized, Double-blind, Placebo-controlled, Proof-of-concept Study Assessing the Efficacy and Safety of an Anti-TNF-OX40L NANOBODY® Molecule, SAR442970, in Participants With Moderate to Severe Hidradenitis Suppurativa

acronym: HS OBTAIN

summary: This is a Phase 2 study in adult participants with moderate to severe hidradenitis suppurativa (HS). The purpose of the study is to evaluate the efficacy and safety of SAR442970 compared to placebo.

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

lead_sponsor: Sanofi

location_country:
Germany
Italy
Poland
Denmark
Australia
Sweden
Netherlands
Belgium
United States
Spain
Greece
Chile
Czechia
Canada
France

location_facility: Advanced Medical Research PC Site Number : 8400002

location_facility: Clinical Partners, LLC Site Number : 8400010

location_facility: Medical Dermatology Specialists Site Number : 8400007

location_facility: Renstar Medical Research Site Number : 8400011

location_facility: Center for Clinical Studies, LTD. LLP Site Number : 8400003

location_facility: ForCare Clinical Research Site Number : 8400006


title: A Phase 1/2a, First in Human, Open-Label, Multicenter, Dose Escalation Study With Expansion Cohorts to Evaluate the Safety, Tolerability, and Preliminary Activity of LB101 in Subjects With Advanced Solid Tumors

summary: The purpose of this study is to assess safety, tolerability, and preliminary activity of LB101 monotherapy in participants with advanced solid tumors.

lead_sponsor: Centessa Pharmaceuticals (UK) Limited

sponsor_collaborators:
Centessa Pharmaceuticals (UK) Limited
LockBody Therapeutics Ltd

location_country:
United States
France

location_facility: Sarah Cannon Research Institute at Florida Cancer Specialists

location_facility: Sarah Cannon Research Institute at Tennessee Oncology Nashville

location_facility: Sarah Cannon Research Institute at HealthONE.


title: Phase 1 First-In-Human Study to Explore the Safety, Tolerability, and Pharmacokinetics of AMG 305 in Subjects With Advanced Solid Tumors

summary:
The primary objective of this study is to:

Evaluate the safety and tolerability of AMG 305 in adult participants
Determine the optimal biologically active dose (OBD), at or below the maximum tolerated dose (MTD) with MTD 1 as the maximum tolerated starting dose and MTD 2 as the maximum tolerated target dose
Determine the recommended phase 2 dose (RP2D)

primary_purpose: Basic Science

location_country:
Germany
Australia
United States
Korea, Republic of
Spain
France

location_facility: Hospital Clinic i Provincial de Barcelona

location_facility: Institut Universitaire du Cancer Toulouse Oncopole

location_facility: Hospital Universitario Madrid Sanchinarro


title: A Phase III, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of RO7434656, an Antisense Inhibitor of Complement Factor B, in Patients With Primary IgA Nephropathy at High Risk of Progression

acronym: IMAGINATION

summary: The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of RO7434656, a novel Antisense Oligonucleotide (ASO) therapy in participants with primary IgA nephropathy (IgAN) who are at high risk of progressive kidney disease despite optimized supportive care.

ipd_sharing_description: Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).

lead_sponsor: Hoffmann-La Roche

location_country:
United States
Spain
Poland
France

location_facility: North America Research Institute-San Dimas

location_facility: SCM Clinic - Prywatny Szpital Specjalistyczny

location_facility: Nephrology Associates of Northern Virginia Inc

location_facility: Nephrology Associates of Northern Illinois

location_facility: LCC Medical Research - Miami - ClinEdge - PPDS

location_facility: SPZOZ Centralny Szpital Klin; UM w Lodzi

location_facility: Hospital Universitario Virgen del Rocio - PPDS

location_facility: Southern California Medical Research Center


title: An Open-label, Randomized, Active-Controlled, Phase 3 Study of Setrusumab Compared With Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta Types I, III or IV

acronym: Cosmic

summary: The primary objective of the study is to evaluate the effect of setrusumab vs intravenous bisphosphonates (IV-BP) on reduction in fracture rate, including morphometric vertebral fractures in pediatric participants.

lead_sponsor: Ultragenyx Pharmaceutical Inc

location_country:
Germany
Italy
Poland
Brazil
Netherlands
United States
Canada
France

location_facility: Childrens Hospital Of Eastern Ontario Research Institute, University Of Ottawa

location_facility: Uniwersytet Medyczny w Lodzi - Klinika Endokrynologii i Chorob Metabolicznych

location_facility: Nemours/ Alfred i. duPoint Hospital for Children

location_facility: Vanderbilt University Medical Center (VUMC)

location_facility: Children's Hospital at London Health Sciences Centre

location_facility: Universitätsklinikum Köln (University of Cologne) - Children's hospital University

location_facility: University of Missouri-Kansas City (UMKC) Medical School - Children's Mercy Hospitals & Clinics (CMHC)

location_facility: Washington University School of Medicine

location_facility: Hospital de Clinicas de Porto Alegre (HCPA)

location_facility: Azienda Ospedaliera Universitaria Policlinico Umberto I

location_facility: Universitair Medisch Centrum Utrecht (UMCU) - Wilhelmina Kinderziekenhuis


title: A Phase 3, Multicenter, 12-Week, Double Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Atogepant for the Preventive Treatment of Episodic Migraine in Pediatric Subjects 6-17 Years of Age.

summary:
A migraine is a moderate to severe headache on one side of the head. A migraine attack is a headache that may be accompanied by throbbing, nausea, vomiting, sensitivity to light and sound, or other symptoms. A number of treatments are available for adults with migraine but there are limited approved treatments available for pediatric participants. The main goal of the study is to evaluate the safety and efficacy (how well treatment works) of a low-dose and high-dose of atogepant in pediatric participants between the ages of 6 and 17.

Atogepant is a medicine currently approved to treat adults with episodic migraine (0 to 14 migraine days per month) and is being studied in pediatric participants between the ages of 6 and 17 with a history of episodic migraine. This is a Phase 3, randomized, double-blind study of atogepant in participants with a history of episodic migraine with an open-label pharmacokinetic substudy. Eligible participants will be randomized into 6 different groups. Participants between the ages of 12 and 17 will be randomized to receive placebo, low-dose atogepant, or high-dose atogepant for 12 weeks. Participants between the ages of 6 and 11 will also be randomized to receive placebo, low-dose atogepant, or high-dose atogepant for 12 weeks. The specific atogepant doses to be used in participants between the ages of 6 and 11 will be determined after the PK substudy is complete. Around 450 participants will be enrolled in approximately 100 sites.

Placebo, low-dose atogepant, and high-dose atogepant are given as a tablet to take by mouth once a day. At the end of Week 12, participants will either undergo a follow-up visit 4 weeks after last study treatment or join an extension study where they can continue to receive atogepant for another 52 weeks.

There may be a bigger responsibility for participants in this study. Participants will attend regular visits during the study at a hospital or clinic. The effects of treatment will be checked by medical assessments, blood tests, checking for side effects, and completing questionnaires.

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

lead_sponsor: AbbVie

location_country:
Poland
Hungary
Israel
Denmark
Japan
Sweden
Netherlands
United States
Spain
Canada
Puerto Rico
France

location_facility: Hospital Universitario Vall d'Hebron /ID# 247967

location_facility: Lynn Health Science Institute (LHSI) /ID# 247600

location_facility: Clinical Research Center Sp. z.o.o. Medic-R sp. k /ID# 247383

location_facility: Alliance for Research - Long Beach /ID# 248521

location_facility: Sendai Headache and Neurology Clinic Medical Corporation /ID# 254211

location_facility: Cognitive Clinical Trials (CCT) - Papillion /ID# 248536

location_facility: Access Clinical Trials, Inc. /ID# 248532

location_facility: Indywidualna Praktyka Lekarska dr hab. med. Anna Szczepanska-Szerej /ID# 247382

location_facility: Montreal Children's Hospital /ID# 247271

location_facility: Aalborg Universitetshospital /Id# 247428

location_facility: Northwest Florida Clinical Research Group, LLC /ID# 251382

location_facility: Advanced Neurosciences Research, LLC /ID# 247592

location_facility: Deaconess Clinic - Gateway Health Center /ID# 247589

location_facility: Hospital Universitario y Politecnico La Fe /ID# 247969

location_facility: Lumos Clinical Research Center /ID# 249731

location_facility: Puerto Rico Health Institute /ID# 249741

location_facility: Advanced Research Institute of Miami /ID# 248539

location_facility: Michigan Headache & Neurological Institute (MHNI) /ID# 247468

location_facility: Caribbean Medical Research Center /ID# 251634

location_facility: Patient Priority Clinical Sites, LLC /ID# 247535

location_facility: Dent Neurosciences Research Center, Inc. /ID# 248534

location_facility: College Park Family Care Center Overland Park /ID# 249734

location_facility: ZorgSaam Zorggroep Zeeuws-Vlaanderen /ID# 247301

location_facility: Rehabilitation & Neurological Services /ID# 248517

location_facility: MIGRE Polskie Centrum Leczenia Migreny Anna Gryglas-Dworak /ID# 247381

location_facility: Hospital Clinico Universitario San Carlos /ID# 249267

location_facility: Family Psychiatry of The Woodlands /ID# 249727

location_facility: Centre Hosp Intercommunal de Creteil /ID# 248832

location_facility: Coastal Georgia Child Neurology /ID# 249733

location_facility: Shamir Medical Center (Assaf Harofeh) /ID# 256857

location_facility: Vastra Gotealandsregionen Regionhalsan /ID# 248277

location_facility: Asclepes Research Centers - Spring Hill /ID# 248525

location_facility: Hospital Universitario Virgen del Rocio /ID# 248632

location_facility: Specjalistyczne Gabinety Sp. z o.o. /ID# 247384

location_facility: FutureSearch Trials of Neurology /ID# 247470


acronym: V-PEDS-OLE

summary: The purpose of this open-label, single arm, multicenter extension study is to evaluate the long-term safety and tolerability of inclisiran in participants with HeFH or HoFH who have completed the ORION-16 or ORION-13 studies.

title: An Open-label, Single Arm, Multicenter Extension Study to Evaluate Long-term Safety and Tolerability of Inclisiran in Participants With Heterozygous or Homozygous Familial Hypercholesterolemia Who Have Completed the Adolescent ORION-16 or ORION-13 Studies (VICTORION-PEDS-OLE)

location_country:
Germany
Brazil
Turkey
Canada
France
Slovenia
Hungary
Switzerland
United States
Lebanon
Italy
Norway
United Kingdom
Netherlands
Spain
South Africa
Taiwan
Russian Federation
Israel


title: An Open-Label Extension Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Patients With Severe Hypertriglyceridemia (SHTG)

summary: The purpose of this study is to evaluate the safety and tolerability of olezarsen in participants with SHTG.

lead_sponsor: Ionis Pharmaceuticals, Inc.

location_country:
New Zealand
Hungary
United States
Spain
South Africa
Czechia
Bulgaria
Canada
France

location_facility: DRC Gyógyszervizsgáló Központ Korlátolt felelősségű társaság Balatonfüred

location_facility: The Presbyterian Hospital DBA Novant Health Heart and Vascular Institute

location_facility: Louisville Metabolic and Atherosclerosis Research Center (L-MARC)

location_facility: University of Michigan- Endocrinology & Metabolism

location_facility: Corporacio Sanitaria Parc Tauli- Hospital de Sabadell

location_facility: Clinique des Maladies Lipidiques de Quebec Inc.

location_facility: University of Kansas Medical Center (KUMC)

location_facility: MU Dr. Nina Zemkova s.r.o. Interni Ambulance

location_facility: Medical Center Smolyan Clinical Research OOD


title: CALIBRATE: A Phase 3, Randomized, Open-Label Study Evaluating the Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With Autosomal Dominant Hypocalcemia Type 1 (ADH1)

acronym: CALIBRATE

summary: The primary purpose of the study is to understand the effectiveness, safety, and tolerability of encaleret when compared to standard of care (SoC) treatment in participants with ADH1.

lead_sponsor: Calcilytix Therapeutics, Inc., a BridgeBio company

location_country:
Denmark
Australia
United States
Czechia
Canada
France

location_facility: Indiana University Health University Hospital

location_facility: UCSF Benioff Children's Hospital, Oakland


title: International Study of Cerebral Oxygenation and Electrical Activity During Major Neonatal Surgery

summary:
The goal of this observational study is to determine the incidence of perioperative cerebral desaturation in neonates undergoing surgery for congenital malformations. The main questions it aims to answer are:

The perioperative factors associated with occurrence of cerebral desaturation
The association between perioperative cerebral desaturation, perioperative/hospital outcomes, and physiological conditions.

Participants will undergo Near-infrared spectroscopy (NIRS) and electroencephalogram (EEG) monitoring for one hour before surgery, during surgery, and up to 24 hours after surgery.

ReferenceCitation: CDC. https://www.cdc.gov/ncbddd/birthdefects/data.html. Accessed April 13, 2021.

ReferenceCitation: Bonasso PC, Dassinger MS, Ryan ML, Gowen MS, Burford JM, Smith SD. 24-hour and 30-day perioperative mortality in pediatric surgery. J Pediatr Surg. 2019 Apr;54(4):628-630. doi: 10.1016/j.jpedsurg.2018.06.026. Epub 2018 Jun 25.

ReferenceCitation: Laing S, Walker K, Ungerer J, Badawi N, Spence K. Early development of children with major birth defects requiring newborn surgery. J Paediatr Child Health. 2011 Mar;47(3):140-7. doi: 10.1111/j.1440-1754.2010.01902.x. Epub 2010 Nov 21.

ReferenceCitation: Stolwijk LJ, Lemmers PM, Harmsen M, Groenendaal F, de Vries LS, van der Zee DC, Benders MJ, van Herwaarden-Lindeboom MY. Neurodevelopmental Outcomes After Neonatal Surgery for Major Noncardiac Anomalies. Pediatrics. 2016 Feb;137(2):e20151728. doi: 10.1542/peds.2015-1728. Epub 2016 Jan 12.

ReferenceCitation: Tusor N, Benders MJ, Counsell SJ, Nongena P, Ederies MA, Falconer S, Chew A, Gonzalez-Cinca N, Hajnal JV, Gangadharan S, Chatzi V, Kersbergen KJ, Kennea N, Azzopardi DV, Edwards AD. Punctate White Matter Lesions Associated With Altered Brain Development And Adverse Motor Outcome In Preterm Infants. Sci Rep. 2017 Oct 16;7(1):13250. doi: 10.1038/s41598-017-13753-x.

ReferenceCitation: Stolwijk LJ, Keunen K, de Vries LS, Groenendaal F, van der Zee DC, van Herwaarden MYA, Lemmers PMA, Benders MJNL. Neonatal Surgery for Noncardiac Congenital Anomalies: Neonates at Risk of Brain Injury. J Pediatr. 2017 Mar;182:335-341.e1. doi: 10.1016/j.jpeds.2016.11.080. Epub 2016 Dec 30.

ReferenceCitation: Sood ED, Benzaquen JS, Davies RR, Woodford E, Pizarro C. Predictive value of perioperative near-infrared spectroscopy for neurodevelopmental outcomes after cardiac surgery in infancy. J Thorac Cardiovasc Surg. 2013 Feb;145(2):438-445.e1; discussion 444-5. doi: 10.1016/j.jtcvs.2012.10.033. Epub 2012 Dec 6.

ReferenceCitation: Hoffman GM, Brosig CL, Mussatto KA, Tweddell JS, Ghanayem NS. Perioperative cerebral oxygen saturation in neonates with hypoplastic left heart syndrome and childhood neurodevelopmental outcome. J Thorac Cardiovasc Surg. 2013 Nov;146(5):1153-64. doi: 10.1016/j.jtcvs.2012.12.060. Epub 2013 Jan 12.

ReferenceCitation: Vida VL, Tessari C, Cristante A, Nori R, Pittarello D, Ori C, Cogo PE, Perissinotto E, Stellin G. The Role of Regional Oxygen Saturation Using Near-Infrared Spectroscopy and Blood Lactate Levels as Early Predictors of Outcome After Pediatric Cardiac Surgery. Can J Cardiol. 2016 Aug;32(8):970-7. doi: 10.1016/j.cjca.2015.09.024. Epub 2015 Oct 22.

ReferenceCitation: Danzer E, Hoffman C, D'Agostino JA, Gerdes M, Bernbaum J, Antiel RM, Rintoul NE, Herkert LM, Flake AW, Adzick NS, Hedrick HL. Neurodevelopmental outcomes at 5years of age in congenital diaphragmatic hernia. J Pediatr Surg. 2017 Mar;52(3):437-443. doi: 10.1016/j.jpedsurg.2016.08.008. Epub 2016 Aug 30.

ReferenceCitation: Birthdefects C. https://www.cdc.gov/ncbddd/birthdefects/data.html. Published 2021. Accessed April 21, 2021.

ReferenceCitation: Escobar MA Jr, Caty MG. Complications in neonatal surgery. Semin Pediatr Surg. 2016 Dec;25(6):347-370. doi: 10.1053/j.sempedsurg.2016.10.005. Epub 2016 Nov 1.

ReferenceCitation: Andropoulos DB, Ahmad HB, Haq T, Brady K, Stayer SA, Meador MR, Hunter JV, Rivera C, Voigt RG, Turcich M, He CQ, Shekerdemian LS, Dickerson HA, Fraser CD, Dean McKenzie E, Heinle JS, Blaine Easley R. The association between brain injury, perioperative anesthetic exposure, and 12-month neurodevelopmental outcomes after neonatal cardiac surgery: a retrospective cohort study. Paediatr Anaesth. 2014 Mar;24(3):266-74. doi: 10.1111/pan.12350.

ReferenceCitation: Mahle WT, Tavani F, Zimmerman RA, Nicolson SC, Galli KK, Gaynor JW, Clancy RR, Montenegro LM, Spray TL, Chiavacci RM, Wernovsky G, Kurth CD. An MRI study of neurological injury before and after congenital heart surgery. Circulation. 2002 Sep 24;106(12 Suppl 1):I109-14.

ReferenceCitation: Olbrecht VA, Skowno J, Marchesini V, Ding L, Jiang Y, Ward CG, Yu G, Liu H, Schurink B, Vutskits L, de Graaff JC, McGowan FX Jr, von Ungern-Sternberg BS, Kurth CD, Davidson A. An International, Multicenter, Observational Study of Cerebral Oxygenation during Infant and Neonatal Anesthesia. Anesthesiology. 2018 Jan;128(1):85-96. doi: 10.1097/ALN.0000000000001920.

ReferenceCitation: Dent CL, Spaeth JP, Jones BV, Schwartz SM, Glauser TA, Hallinan B, Pearl JM, Khoury PR, Kurth CD. Brain magnetic resonance imaging abnormalities after the Norwood procedure using regional cerebral perfusion. J Thorac Cardiovasc Surg. 2006 Jan;131(1):190-7. doi: 10.1016/j.jtcvs.2005.10.003.

RetractionSource: J Thorac Cardiovasc Surg. 2006 Jun;131(6):1226

ReferenceCitation: Suemori T, Skowno J, Horton S, Bottrell S, Butt W, Davidson AJ. Cerebral oxygen saturation and tissue hemoglobin concentration as predictive markers of early postoperative outcomes after pediatric cardiac surgery. Paediatr Anaesth. 2016 Feb;26(2):182-9. doi: 10.1111/pan.12800. Epub 2015 Dec 1.

ReferenceCitation: Kurth CD, Levy WJ, McCann J. Near-infrared spectroscopy cerebral oxygen saturation thresholds for hypoxia-ischemia in piglets. J Cereb Blood Flow Metab. 2002 Mar;22(3):335-41. doi: 10.1097/00004647-200203000-00011.

ReferenceCitation: Yuan I, Xu T, Kurth CD. Using Electroencephalography (EEG) to Guide Propofol and Sevoflurane Dosing in Pediatric Anesthesia. Anesthesiol Clin. 2020 Sep;38(3):709-725. doi: 10.1016/j.anclin.2020.06.007.

ReferenceCitation: Yuan I, Olbrecht VA, Mensinger JL, Zhang B, Davidson AJ, von Ungern-Sternberg BS, Skowno J, Lian Q, Song X, Zhao P, Zhang J, Zhang M, Zuo Y, de Graaff JC, Vutskits L, Szmuk P, Kurth CD. Statistical Analysis Plan for "An international multicenter study of isoelectric electroencephalography events in infants and young children during anesthesia for surgery". Paediatr Anaesth. 2019 Mar;29(3):243-249. doi: 10.1111/pan.13589. Erratum In: Paediatr Anaesth. 2020 Jun;30(6):730.

lead_sponsor: Children's Hospital of Philadelphia

sponsor_collaborators:
Children's Hospital of Philadelphia
Masimo Corporation

location_country:
India
United Kingdom
Australia
Brazil
United States
China
France

location_facility: Centre Hospitalier Universitaire de Bordeaux, Hopital des enfants

location_facility: Great Ormond Street Hospital for Children

location_facility: Children's Hospital at Montefiore/Albert Einstein College of Medicine

location_facility: Post Graduate Institute of Medical Education and Research

location_facility: Guangzhou women and children's medical center

location_facility: Shengjing Hospital of China Medical University

location_facility: West China Hospital and West China Second University Hospital

location_facility: Hospital das Clínicas, Faculdade de Medicina, Universidade de São Paulo

lead_sponsor_type: academique


title: International, Multi-center, Open-label, Treatment Extension Study in Patients With Multiple Myeloma Who Are Still Benefitting From Isatuximab-based Therapy Following Completion of a Phase 1, 2, or 3 Parental Study

summary:
This is a multi-center, open-label, Phase 2 treatment extension study in patients with multiple myeloma who are still benefitting from isatuximab based therapy following completion of a Phase 1, 2, or 3 parental study.
This Treatment Extension study has the purpose to provide continued access to isatuximab. Adult participants with multiple myeloma who have enrolled on an isatuximab parental study for which study objectives are completed will be eligible to be enrolled in this Treatment Extension study.
The primary objective of the study is to assess long-term safety of isatuximab as study treatment.

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

location_country:
Finland
Australia
Brazil
Japan
Sweden
United States
China
Korea, Republic of
Spain
Greece
Chile
Taiwan
Czechia
Russian Federation
France

location_facility: Instituto COI de Educacao e Pesquisa Site Number : 0760001

location_facility: Washington University School of Medicine Site Number : 8400001


title: An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients With Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab

acronym: ONYX

summary: The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Type 2 and Type 3 SMA who have completed TOPAZ or SAPPHIRE.

status: Active, not recruiting

lead_sponsor: Scholar Rock, Inc.

location_country:
Germany
Italy
Poland
Netherlands
United States
Spain
France

location_facility: Carlo Besta Neurological Research Institute

location_facility: Hospital Universitari i Politecnic La Fe

location_facility: UCSD Altman Clinical and Translational Research

location_facility: Instytut Pomnik - Centrum Zdrowia Dziecka

location_facility: Gillette Children's Specialty Healthcare

location_facility: University of Texas Southwestern - Pediatric Neurology

location_facility: Children's Specialty Group PLLC (Children's Hospital of The King's Daughters)

location_facility: Fondazione Policlinico Universitario A. Gemelli

location_facility: Wake Forest University School of Medicine


title: An Open-Label, Multicenter Study of LOXO-435 (LY3866288) In Advanced Solid Tumor Malignancies With FGFR3 Alterations

summary: The main purpose of this study is to learn more about the safety, side effects, and effectiveness of LOXO-435. LOXO-435 may be used to treat cancer of the cells that line the urinary system and other solid tumor cancers that have a change in a particular gene (known as the FGFR3 gene). Participation could last up to 30 months (2.5 years) and possibly longer if the disease does not get worse.

lead_sponsor: Eli Lilly and Company

sponsor_collaborators:
Eli Lilly and Company
Loxo Oncology, Inc.
Merck Sharp & Dohme LLC

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

location_facility: Severance Hospital, Yonsei University Health System

location_facility: Washington University School of Medicine

location_facility: David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center

location_facility: University of North Carolina at Chapel Hill

location_facility: University of Oklahoma Health Sciences Center

location_facility: University of Texas Southwestern Medical Center


title: AN OPEN-LABEL STUDY IN PEDIATRIC (<18 YEARS OF AGE), SEVERE HEMOPHILIA A PARTICIPANTS (COAGULATION FACTOR ACTIVITY <1%) WITH OR WITHOUT INHIBITORS OR MODERATELY SEVERE TO SEVERE HEMOPHILIA B PARTICIPANTS (COAGULATION FACTOR ACTIVITY =2%) WITH OR WITHOUT INHIBITORS COMPARING 12 MONTHS OF HISTORICAL STANDARD TREATMENT TO MARSTACIMAB PROPHYLAXIS

acronym: BASIS KIDS

summary:
The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.

This study will enroll pediatric participants from ages 1 to 17 years in a sequential manner. The study will open enrollment to adolescent participants aged 12 to 17 years first. Then children aged 6 to 11 years will be permitted to enroll. Lastly, children aged 1 to 5 years will be permitted to enroll.

This study will enroll participants who:

have severe Hemophilia A or moderately severe to severe Hemophilia B (with or without inhibitors)
have accurate historical records documenting all factor VIII, factor IX, or bypass agent infusions and hemophilia bleed events for at least 1 year prior to entering the study
if a non-inhibitor patient, must be on a stable routine prophylaxis regimen with factor VIII or factor IX replacement products for at least 12 months prior to study entry
if an inhibitor patient, must be on an on-demand bypass treatment regimen during the 12 months prior to study entry

All participants in this study will receive marstacimab to use prophylactically. Marstacimab will be given once a week as a subcutaneous (under the skin) shot. The first dose of marstacimab will be given at the study site by the study site staff. During the 12-month treatment period, weekly doses of marstacimab can be given at home, or if preferred, the doses may be given by the study site staff.

To help us determine if the study medicine is safe and effective, we will compare participant experiences when they are taking the study medicine to a historical period when they were not. Researchers want to see if the study medicine works to prevent the bleeding episodes commonly experienced by patients with Hemophilia.

Participants will be in this study for about 14 months (approximately 1 month in a Screening period, 12 months receiving treatment, and 1 month in a follow-up period) during which they will visit the study site at least 10 times. If preferred, and if local regulations allow it, 2 of the study visits can be completed at the participant's home instead of at the study site. There will also be 6 scheduled telephone calls approximately every 2 months.

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.

lead_sponsor: Pfizer

location_country:
Germany
Argentina
Australia
Brazil
Japan
Turkey
Canada
France
Slovakia
Denmark
United States
India
China
Austria
Korea, Republic of
Saudi Arabia
Spain
South Africa
Taiwan
Israel

location_facility: The Affiliated Hospital of Guizhou Medical University

location_facility: Intermountain - Primary Children's Hospital

location_facility: Institute of hematology&blood disease hospital

location_facility: Hyogo prefectural Kobe Children's Hospital

location_facility: Hamilton Health Sciences - McMaster University Medical Centre

location_facility: Univerzitna nemocnica Bratislava, Nemocnica sv. Cyrila a Metoda

location_facility: Istanbul Universitesi Istanbul Tıp Fakultesi Hastanesi

location_facility: King Faisal Specialist Hospital & Research Center

location_facility: Gazi University Health Research and Application Center Gazi Hospital

location_facility: Tongji Hospital of Tongji Medical College of HUST/Pediatric Hematology Department Pharmacy

location_facility: Beijing Children's hospital, Capital Medical University

location_facility: Charlotte Maxeke Johannesburg Academic Hospital

location_facility: Hôpital Universitaire Necker Enfants Malades

location_facility: Saitama Prefectural Children's Medical Center

location_facility: Erciyes Universitesi Tıp Fakultesi Hastaneleri

location_facility: Nil Ratan Sircar Medical College and Hospital

location_facility: Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology

location_facility: Southern Medical University Nanfang Hospital

location_facility: Kyung Hee University Hospital at Gangdong


title: Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants

acronym: Uni-Rare

summary:
This is an international, multicenter study with two components:

Registry

A standardized genetic screening and a prospective, standardized, cross-sectional clinical data collection
Enrollment is open to all genes on the RD Rare Gene List

Natural History Study

A prospective, standardized, longitudinal Natural History Study
Enrollment opens gene-by-gene, based on funding and within-gene Registry enrollment The study objectives are as follows.

Registry Objectives

Genotype Characterization
Cross-Sectional Phenotype Characterization (within gene)
Establish a Link to My Retina Tracker Registry (MRTR)
Ancillary Exploratory Studies - Pooling of Genes

Natural History Study Objectives

Natural History (within gene)
Structure-Function Relationship (within gene)
Risk Factors for Progression (within gene)
Ancillary Exploratory Studies - Pooling of Genes

time_perspective: Cross-Sectional

ipd_sharing_description: A de-identified database is placed in the public domain on the FFB/Jaeb public website after the completion of each protocol and publication of the manuscript.

lead_sponsor: Jaeb Center for Health Research

sponsor_collaborators:
Jaeb Center for Health Research
Foundation Fighting Blindness

location_country:
Finland
Israel
United Kingdom
Australia
Brazil
Netherlands
Mexico
Switzerland
United States
Canada
France

location_facility: Oregon Health & Science Univ., Casey Eye Institute

location_facility: Center for Advanced Retinal and Ocular Therapeutics

location_facility: University of Florida Health Jacksonville

location_facility: University of Michigan, Kellogg Eye Center

location_facility: Baylor College of Medicine, Alkek Eye Center

location_facility: University of Arkansas, Jones Eye Institute

location_facility: Hadassah-Hebrew University Medical Center

location_facility: University of Toronto, Hospital for Sick Children

location_facility: University of Utah, John Moran Eye Center

location_facility: Univ. of California San Diego, Jacobs Retina Center

location_facility: University of Miami, Bascom Palmer Eye Institute

location_facility: Medical College of Wisconsin Eye Institute

location_facility: Johns Hopkins University, Wilmer Eye Institute

location_facility: Harvard Univ., Massachusetts Eye and Ear Infirmary

location_facility: University of Alberta and Alberta Health Services


title: A Randomized, Double-blind, Placebo Controlled, Dose-finding Study to Assess the Efficacy and Safety of SAR443122 in Adult Patients With Moderate to Severe Ulcerative Colitis

acronym: Resolute

summary:
This is a randomized, double-blind, placebo controlled, dose-ranging Phase 2 study. The primary objective is to evaluate the efficacy and safety of SAR443122 compared to placebo in participants with moderate to severe UC. Dose selection for further clinical development will be based on the multiple efficacy, safety and PK parameters.

The study consists of 4 parallel arms (3 dose groups of SAR443122 vs placebo) to assess the efficacy and safety of SAR443122 in participants with moderate to severe UC. All participants will receive a total of 52 weeks (a 12-week induction treatment phase and a 40-week maintenance phase) of study treatment, except if treatment should be discontinued per investigator's assessment.

At the end of the first 12 weeks of induction treatment, all participants in clinical response or remission will be offered study treatment up to 40 weeks and will continue with the same blinded treatment that was assigned. Participants who do not achieve clinical response or remission at the end of the initial 12 weeks induction treatment will roll over in an open-label treatment arm and will be treated with SAR443122 at the highest tested dose.

In addition, participants from the maintenance treatment that lose clinical efficacy at any time up to V10/Week 40 (Week 28 of maintenance) will be offered to roll over in the open-label treatment arm with SAR443122 at the highest dose.

location_country:
Germany
Italy
Poland
India
Hungary
Argentina
United Kingdom
Japan
Netherlands
Mexico
United States
China
Spain
Chile
Czechia
France

location_facility: Las Vegas Medical Research Site Number : 8400004

location_facility: United Medical Doctors Site Number : 8400005

location_facility: GI Alliance Research Site Number : 8400010

location_facility: Gastrointestinal Bioscience Site Number : 8400006

location_facility: Southern Star Research Site Number : 8400011


title: Avalus European Registry - an Observational Study to Evaluate Safety and Efficacy in a Real World Population

acronym: Avalus

summary: This is a prospective, observational, single-arm, multi-center registry of patients undergoing bioprosthetic aortic valve replacement with the Avalus valve. This prospective registry aims to examine the outcome and performance of surgical aortic valve replacement with the Avalus pericardial bioprosthesis.

lead_sponsor: Universitaire Ziekenhuizen KU Leuven

location_country: Finland Germany Italy Denmark Netherlands Belgium Spain France

location_facility:
Fondazione Poliambulanza
ZOL
Villa Maria Cecilia
UZ Leuven
European Hospital
Schüchtermann-Klinik
Osp. S.Orsola Malpighi
Kuopio University Hospital
UCL St Luc
UZ Gent
CHU Nantes
Rigshospitalet
UMC Maastricht
Policlinico San Donato S.P.A
A. Sanitaria O. Ordine Mauriziano
AZ Maria Middelares
Hospital Rambla
Universität Herzzentrum
Hospital Clinico De Valladolid


title: A Phase 1 Study of SGN-BB228 in Advanced Melanoma and Other Solid Tumors

summary:
This study will test the safety of a drug called SGN-BB228 in participants with melanoma and other solid tumors that are hard to treat or have spread through the body. It will also study the side effects of this drug. A side effect is anything a drug does to the body besides treating the disease.

This study will have 3 parts. Parts A and B of the study will find out how much SGN-BB228 should be given to participants. Part C will use the information from Parts A and B to see if SGN-BB228 is safe and if it works to treat solid tumor cancers.

lead_sponsor: Seagen Inc.

location_country:
United Kingdom
Switzerland
United States
Canada
France

location_facility: NHS Greater Glasgow and Clyde (NHSGGC) - The Beatson West of Scotland Cancer Centre

location_facility: Fred Hutchinson Cancer Center / Seattle Cancer Care Alliance / University of Washington

location_facility: University of California, San Francisco | HDFCCC - Hematopoietic Malignancies

location_facility: University Health Network, Princess Margaret Hospital

location_facility: Sarah Cannon Research Institute at HealthONE - Denver


title: An Open-Label Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder

summary: A Phase 2, open-label, multicenter study to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and safety of inebilizumab in eligible pediatric participants 2 to < 18 years of age with recently active neuromyelitis optica spectrum disorder (NMOSD) who are seropositive for autoantibodies against aquaporin-4 (AQP4-immunoglobulin [Ig]G).

lead_sponsor: Horizon Therapeutics Ireland DAC

location_country:
Poland
United Kingdom
Argentina
Brazil
Sweden
United States
Spain
Canada
Serbia
France

location_facility: Centre Hospitalier Universitaire de Bicêtre

location_facility: Great Ormond Street Hospital for Children

location_facility: Clinic for Neurology and Psychiatry for Children and Youth

location_facility: Loma Linda University Children's Hospital

location_facility: UC San Diego Altman Clinical and Translational Research Institute (ACTRI) Building

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

location_facility: University of Texas Southwestern Medical Center

location_facility: Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan

location_facility: Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)