title: Randomized Trial of Fetoscopic Endoluminal Tracheal Occlusion (FETO) Versus Expectant Management During Pregnancy in Fetuses With Left Sided and Isolated Congenital Diaphragmatic Hernia and Moderate Pulmonary Hypoplasia.

acronym: TOTAL moderate

summary:
Isolated Congenital Diaphragmatic Hernia (CDH) can be diagnosed in the prenatal period, and remains associated with a 30 % chance of perinatal death and morbidity mainly because of pulmonary hypoplasia and pulmonary hypertension. In addition, in the survivors there is a high rate of morbidity with evidence of bronchopulmonary dysplasia in more than 70% of cases. The risk for these can be predicted prenatally by the ultrasonographic measurement of the observed/expected lung area to head circumference ratio (O/E LHR) which is a measure of pulmonary hypoplasia. Also position of the liver is predictive of outcome.

The proposing consortium has developed a prenatal therapeutic approach, which consists of percutaneous fetoscopic endoluminal tracheal occlusion (FETO) with subsequent removal of the balloon. Both procedures are performed percutaneously, there is now experience with more than 150 cases and it has been shown to be safe for the mother. We have witnessed an improvement of survival in fetuses with a predicted chance of survival of less than 30% (referred to as fetuses with severe pulmonary hypoplasia; O/E LHR <25% and liver herniation) to 55% on average. Also there is an apparent reduction in morbidity with the rate of bronchopulmonary dysplasia decreasing from the estimated rate of more than 70% to less than 40% in the same severity group.

Further we have shown that results of FETO are predicted by LHR measurement prior to the procedure, so that better results can be expected in fetuses with larger lung size. Therefore we now aim to offer FETO to fetuses with moderate CDH (=O/E LHR 25-34.9%, irrespective of the liver position as well as O/E LHR 35-44.9% with intrathoracic herniation of the liver). When managed expectantly the estimated rate of postnatal survival is 55%.

This trial will test whether temporary fetoscopic tracheal occlusion rather than expectant management during pregnancy, both followed by standardized postnatal management increases survival or decrease oxygen dependency at 6 months of age. The balloon will be placed between 30 and 31+6 weeks, and will be removed between 34 and 34+6 weeks.

ReferenceCitation: Jani J, Nicolaides KH, Keller RL, Benachi A, Peralta CF, Favre R, Moreno O, Tibboel D, Lipitz S, Eggink A, Vaast P, Allegaert K, Harrison M, Deprest J; Antenatal-CDH-Registry Group. Observed to expected lung area to head circumference ratio in the prediction of survival in fetuses with isolated diaphragmatic hernia. Ultrasound Obstet Gynecol. 2007 Jul;30(1):67-71.

ReferenceCitation: Doné E, Gucciardo L, Van Mieghem T, Jani J, Cannie M, Van Schoubroeck D, Devlieger R, Catte LD, Klaritsch P, Mayer S, Beck V, Debeer A, Gratacos E, Nicolaides K, Deprest J. Prenatal diagnosis, prediction of outcome and in utero therapy of isolated congenital diaphragmatic hernia. Prenat Diagn. 2008 Jul;28(7):581-91. doi: 10.1002/pd.2033. Review.

ReferenceCitation: Deprest J, Jani J, Gratacos E, Vandecruys H, Naulaers G, Delgado J, Greenough A, Nicolaides K; FETO Task Group. Fetal intervention for congenital diaphragmatic hernia: the European experience. Semin Perinatol. 2005 Apr;29(2):94-103. Review.

ReferenceCitation: Deprest J, Gratacos E, Nicolaides KH; FETO Task Group. Fetoscopic tracheal occlusion (FETO) for severe congenital diaphragmatic hernia: evolution of a technique and preliminary results. Ultrasound Obstet Gynecol. 2004 Aug;24(2):121-6. Erratum in: Ultrasound Obstet Gynecol. 2004 Oct;24(5):594.

ReferenceCitation: Jani JC, Nicolaides KH, Gratacós E, Valencia CM, Doné E, Martinez JM, Gucciardo L, Cruz R, Deprest JA. Severe diaphragmatic hernia treated by fetal endoscopic tracheal occlusion. Ultrasound Obstet Gynecol. 2009 Sep;34(3):304-10. doi: 10.1002/uog.6450.

ReferenceCitation: Deprest JA, Gratacos E, Nicolaides K, Done E, Van Mieghem T, Gucciardo L, Claus F, Debeer A, Allegaert K, Reiss I, Tibboel D. Changing perspectives on the perinatal management of isolated congenital diaphragmatic hernia in Europe. Clin Perinatol. 2009 Jun;36(2):329-47, ix. doi: 10.1016/j.clp.2009.03.004. Review.

ReferenceCitation: Deprest JA, Hyett JA, Flake AW, Nicolaides K, Gratacos E. Current controversies in prenatal diagnosis 4: Should fetal surgery be done in all cases of severe diaphragmatic hernia? Prenat Diagn. 2009 Jan;29(1):15-9. doi: 10.1002/pd.2108.

ReferenceCitation: Gregoir C, Engels AC, Gomez O, DeKoninck P, Lewi L, Gratacos E, Deprest JA. Fertility, pregnancy and gynecological outcomes after fetoscopic surgery for congenital diaphragmatic hernia. Hum Reprod. 2016 Sep;31(9):2024-30. doi: 10.1093/humrep/dew160. Epub 2016 Jul 4.

ReferenceCitation: Snoek KG, Reiss IK, Greenough A, Capolupo I, Urlesberger B, Wessel L, Storme L, Deprest J, Schaible T, van Heijst A, Tibboel D; CDH EURO Consortium. Standardized Postnatal Management of Infants with Congenital Diaphragmatic Hernia in Europe: The CDH EURO Consortium Consensus - 2015 Update. Neonatology. 2016;110(1):66-74. doi: 10.1159/000444210. Epub 2016 Apr 15.

ReferenceCitation: Nawapun K, Eastwood MP, Diaz-Cobos D, Jimenez J, Aertsen M, Gomez O, Claus F, Gratacós E, Deprest J. In vivo evidence by magnetic resonance volumetry of a gestational age dependent response to tracheal occlusion for congenital diaphragmatic hernia. Prenat Diagn. 2015 Nov;35(11):1048-56. doi: 10.1002/pd.4642. Epub 2015 Sep 29.

ReferenceCitation: Deprest J, Brady P, Nicolaides K, Benachi A, Berg C, Vermeesch J, Gardener G, Gratacos E. Prenatal management of the fetus with isolated congenital diaphragmatic hernia in the era of the TOTAL trial. Semin Fetal Neonatal Med. 2014 Dec;19(6):338-48. doi: 10.1016/j.siny.2014.09.006. Epub 2014 Nov 11. Review.

ReferenceCitation: Done E, Debeer A, Gucciardo L, Van Mieghem T, Lewi P, Devlieger R, De Catte L, Lewi L, Allegaert K, Deprest J. Prediction of neonatal respiratory function and pulmonary hypertension in fetuses with isolated congenital diaphragmatic hernia in the fetal endoscopic tracleal occlusion era: a single-center study. Fetal Diagn Ther. 2015;37(1):24-32. doi: 10.1159/000364805. Epub 2014 Nov 8.

ReferenceCitation: DeKoninck P, Gomez O, Sandaite I, Richter J, Nawapun K, Eerdekens A, Ramirez JC, Claus F, Gratacos E, Deprest J. Right-sided congenital diaphragmatic hernia in a decade of fetal surgery. BJOG. 2015 Jun;122(7):940-6. doi: 10.1111/1471-0528.13065. Epub 2014 Sep 17.

ReferenceCitation: Windrim R, Ryan G, Lebouthillier F, Campisi P, Kelly EN, Baud D, Yoo SJ, Deprest J. Development and use of a high-fidelity simulator for fetal endotracheal balloon occlusion (FETO) insertion and removal. Prenat Diagn. 2014 Feb;34(2):180-4. doi: 10.1002/pd.4284. Epub 2013 Dec 27.

ReferenceCitation: Engels AC, DeKoninck P, van der Merwe JL, Van Mieghem T, Stevens P, Power B, Nicolaides KH, Gratacos E, Deprest JA. Does website-based information add any value in counseling mothers expecting a baby with severe congenital diaphragmatic hernia? Prenat Diagn. 2013 Nov;33(11):1027-32. doi: 10.1002/pd.4190. Epub 2013 Aug 29.

ReferenceCitation: Dekoninck P, Gratacos E, Van Mieghem T, Richter J, Lewi P, Ancel AM, Allegaert K, Nicolaides K, Deprest J. Results of fetal endoscopic tracheal occlusion for congenital diaphragmatic hernia and the set up of the randomized controlled TOTAL trial. Early Hum Dev. 2011 Sep;87(9):619-24. doi: 10.1016/j.earlhumdev.2011.08.001.

ReferenceCitation: Deprest J, Flake A. How should fetal surgery for congenital diaphragmatic hernia be implemented in the post-TOTAL trial era: A discussion. Prenat Diagn. 2022 Mar;42(3):301-309. doi: 10.1002/pd.6091. Epub 2022 Jan 22. Review.

ReferenceCitation: Van Calster B, Benachi A, Nicolaides KH, Gratacos E, Berg C, Persico N, Gardener GJ, Belfort M, Ville Y, Ryan G, Johnson A, Sago H, Kosinski P, Bagolan P, Van Mieghem T, DeKoninck PLJ, Russo FM, Hooper SB, Deprest JA. The randomized Tracheal Occlusion To Accelerate Lung growth (TOTAL)-trials on fetal surgery for congenital diaphragmatic hernia: reanalysis using pooled data. Am J Obstet Gynecol. 2022 Apr;226(4):560.e1-560.e24. doi: 10.1016/j.ajog.2021.11.1351. Epub 2021 Nov 19.

ReferenceCitation: Deprest JA, Benachi A, Gratacos E, Nicolaides KH, Berg C, Persico N, Belfort M, Gardener GJ, Ville Y, Johnson A, Morini F, Wielgoś M, Van Calster B, DeKoninck PLJ; TOTAL Trial for Moderate Hypoplasia Investigators. Randomized Trial of Fetal Surgery for Moderate Left Diaphragmatic Hernia. N Engl J Med. 2021 Jul 8;385(2):119-129. doi: 10.1056/NEJMoa2026983. Epub 2021 Jun 8.

lead_sponsor: University Hospital, Gasthuisberg

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

location_facility: University of Texas Health Science Center

location_facility: 1st Department of Obstetrics and Gynecology, Medical University of Warsaw

location_facility: Baylor College of Medicine/Texas Children's Hospital


status: Completed

primary_purpose: Treatment

intervention_type: Drug

title: A Randomized, Double-Blind Evaluation of the Antiviral Efficacy, Safety, and Tolerability of Tenofovir Disoproxil Fumarate Versus Placebo in Adolescents With Chronic Hepatitis B Infection

study_completion_date_type: Actual

lead_sponsor: Gilead Sciences

source: euctr

design_allocation: Randomized

study_type: Interventional

ReferenceType: result

ReferenceCitation: Murray KF, Szenborn L, Wysocki J, Rossi S, Corsa AC, Dinh P, McHutchison J, Pang PS, Luminos LM, Pawlowska M, Mizerski J. Randomized, placebo-controlled trial of tenofovir disoproxil fumarate in adolescents with chronic hepatitis B. Hepatology. 2012 Dec;56(6):2018-26. doi: 10.1002/hep.25818. Epub 2012 Aug 27.

enrollment_type: Actual

location_country:
Poland
Spain
United States
France
Turkey
Romania
Bulgaria

location_facility: Children's Hospital & Regional Medical Center, d/b/a Seattle Children's Research Institute

location_facility: Ege Universitesi Tip Fakultesi Hastanesi

location_facility: Samodzielny Publiczny Dzieciecy Szpital Kliniczny Akademii Medycznej w Bialymstoku

location_facility: Samodzielny Publiczny Szpital Kliniczny Nr 1

location_facility: Wojewodzki Specjalistyczny Szpital im Bieganskiego

location_facility: Krakowski Szpital Specjalistyczny im. Jana Pawla II

location_facility: Clinic of Gastroenterology, Specialized Hospital for Active Treatment of Pediatric Diseases, Sofia

location_facility: Multiprofile Hospital for Active Treatment Sveti Georgi

location_facility: Children's Hospital & Research Center at Oakland

location_facility: Hosp Univ y Politecnico La Fe de Valencia

location_facility: Samodzielny Publiczny Szpital Kliniczny im. Karola Johschera

location_facility: Wojewodzki Szpital Obserwacyjno-Zakazny im. T. Browicza

location_facility: Specjalistyczny Zespol Opieki Zdrowotnej nad Matka i Dzieckiem

summary:
The primary purpose of the study is to evaluate the effectiveness, safety, and tolerability of tenofovir disoproxil fumarate (TDF) in adolescents (aged 12-17 years) with chronic hepatitis B virus (HBV) infection.

The optimal treatment for adolescents with chronic HBV infection is currently unknown. Treatment with interferon alfa, lamivudine, and adefovir dipivoxil in pediatric populations has been shown to be less than optimal. Further, the safety and efficacy of entecavir and telbivudine have not been established in patients < 16 years of age. A study evaluating TDF in adolescents (ages 12-17) was needed to assess the safety and efficacy of this agent in the treatment of chronic hepatitis B in this patient population. In addition, the study will help to further elucidate the pharmacokinetic (PK) and resistance profiles of TDF. Through their participation, study participants will help generate critical new information to help guide the most optimal treatment of chronic HBV infection in adolescents.

This is a randomized, double-blind study to evaluate the antiviral efficacy, safety, and tolerability of TDF versus placebo in adolescents with chronic HBV infection. TDF treatment-naive participants were randomized in a 1:1 ratio to TDF or placebo. After 72 weeks of blinded treatment, participants were to switch to open-label TDF for an additional 2.5 years of treatment, provided that no safety concerns are identified by the Independent Data Monitoring Committee monitoring the study.

lead_sponsor_type: industriel

status_simplified: Completed


title: A Multi-center, Open-label, Phase I Study of Single Agent R7112 Administered Orally in Patients With Advanced Malignancies, Except All Forms of Leukaemia

summary: This study will determine the maximum tolerated dose and the optimal associated 4 weekly dosing schedule of RO5045337, administered as monotherapy in patients with advanced solid tumors. A first cohort of patients will receive the starting dose of 20mg/m2/day, once daily for 10 days in each 28 day cycle. Subsequent cohorts of patients will receive dose escalations, and possible changes in dosing schedule, based on tolerability and pharmacokinetic knowledge gained from prior treatment cohorts. The anticipated time on study treatment is until disease progression or intolerable toxicity.

design_allocation: Non-Randomized

lead_sponsor: Hoffmann-La Roche

location_country:
United States
France


title: Phase II Study of Dasatinib (BMS-354825) for Advanced 'Triple-negative' Breast Cancer

summary: This study will determine whether the investigational drug dasatinib is effective in treatment of women with progressive advanced triple-negative breast cancer.

lead_sponsor: Bristol-Myers Squibb

location_country:
United States
Italy
Spain
France

location_facility: University Of Texas Md Anderson Cancer Ctr


title: Phase II Study Of SKI-606 In Subjects With Advanced Or Metastatic Breast Cancer

summary: The purpose of this study is to determine if SKI-606 (Bosutinib) is effective in the treatment of advanced or metastatic breast cancer. Patients must have current Stage IIIB, IIIC or IV breast cancer and have progressed after 1 to 3 prior chemotherapy regimens.

lead_sponsor: Pfizer

location_country:
Poland
Hong Kong
Russian Federation
Australia
Ukraine
United States
Malta
France


title: Follow-up of Thyroid Cancer Patients From Study THYR-008-00 Who Received Thyroid Remnant Ablation Using Either the Hypothyroid or the Thyrogen Method.

summary:
Patients diagnosed with thyroid cancer are commonly treated with surgery to remove their thyroid gland followed by radioiodine ablation to destroy any remaining parts of the thyroid gland that may have been missed during surgery. It is thought that ablation with radioiodine destroys normal remaining thyroid tissue as well as cancerous cells either in the thyroid area or at other sites.

Following successful treatment, patients are then monitored by their physicians at regular intervals with testing to detect any recurrence of thyroid cancer throughout the body. If thyroid cells are detected by these follow up tests, the physician will decide the best method to re-treat the patient.

In 2001-2003 Genzyme conducted a clinical study to test if Thyrogen® can be used to accomplish radioiodine ablation treatment. This study aimed to determine that the success rates of radioiodine ablation were comparable when patients were prepared for ablation with Thyrogen® while being maintained on their normal thyroid hormone therapy, or, alternatively, by thyroid hormone withdrawal. Thyroid hormone withdrawal commonly causes uncomfortable side effects for patients, and these might be avoided by the use of Thyrogen.

Eight months after the initial Thyrogen plus radioiodine treatment to achieve ablation, all patients in both groups were given Thyrogen® to test for any remaining thyroid tissue. The results of this testing showed that all patients (in both groups) had successfully achieved remnant ablation and had no detectable thyroid tissue remaining.

In order to confirm these remnant ablation results we will conduct follow up testing in this study for all patients that were enrolled in the previous study and we also will determine if their thyroid cancer has recurred. Only patients who completed this previous Thyrogen ablation study are eligible for entry into this study.

ReferenceType: derived

ReferenceCitation: Elisei R, Schlumberger M, Driedger A, Reiners C, Kloos RT, Sherman SI, Haugen B, Corone C, Molinaro E, Grasso L, Leboulleux S, Rachinsky I, Luster M, Lassmann M, Busaidy NL, Wahl RL, Pacini F, Cho SY, Magner J, Pinchera A, Ladenson PW. Follow-up of low-risk differentiated thyroid cancer patients who underwent radioiodine ablation of postsurgical thyroid remnants after either recombinant human thyrotropin or thyroid hormone withdrawal. J Clin Endocrinol Metab. 2009 Nov;94(11):4171-9. doi: 10.1210/jc.2009-0869. Epub 2009 Oct 22.

lead_sponsor: Genzyme, a Sanofi Company

location_country:
Italy
Canada
United States
France
Germany

location_facility: University of Colorado Health Sciences Centre


title: An Open Label Extension, Investigator Initiated Trial to Examine Radiographic Progression , Efficacy and Safety of Long-Term Treatment With Infliximab in Patients With Ankylosing Spondylitis. EASIC (European Ankylosing Spondylitis Infliximab Cohort)

summary: Ankylosing spondylitis (AS) is a chronic inflammatory disease that involves the sacroiliac joints, axial skeleton, entheses and peripheral joints. Current therapy for AS is mainly NSAIDs and physiotherapy which are oft insufficient. Treatment with the TNF-alpha blocking agent infliximab was shown to have definite clinical efficacy in patients with active AS on a short- and a long-term-basis over 2 years. We want to show that treatment with infliximab on a long-term basis over 4 years is safe and efficient and can prevent radiographic progression over a long period of time. Further we want to learn about the outcome after discontinuation of anti-TNF-alpha therapy.

status: Unknown status

study_completion_date_type: Anticipated

enrollment_type: Anticipated

ReferenceCitation: Webers C, Essers I, van Tubergen A, Braun J, Heldmann F, Baraliakos X, Boonen A. Valuing Treatment With Infliximab for Ankylosing Spondylitis Using a Willingness-to-Pay Approach. Arthritis Care Res (Hoboken). 2018 Apr;70(4):608-616. doi: 10.1002/acr.23299. Epub 2018 Feb 18.

lead_sponsor: Rheumazentrum Ruhrgebiet

location_country:
United Kingdom
Netherlands
Finland
France
Belgium
Germany

location_facility: Universitair Ziekenhuis, Afdeling Rheumatologie

location_facility: Universitat R. Descartes, Hopital Cochin

location_facility: University Central Hospital, Division of Rheumatology

status_simplified: Unknown


title: A Pharmacokinetic Study to Assess Nevirapine Levels in HIV-infected Patients With Impaired Hepatic Function

summary: The objective of this study was to evaluate the steady-state clearance of nevirapine among HIV-1 positive patients with hepatic fibrosis, and to examine whether the degree of hepatic impairment influences clearance.

lead_sponsor: Boehringer Ingelheim

location_country:
United States
Spain
France

location_facility: Hospital Clinico y Provincial de Barcelona - HIV

location_facility: Boehringer Ingelheim Investigational Site


title: Dose Finding and Phase II Study of STI 571 in Advanced Soft Tissue Sarcoma

summary:
RATIONALE: STI571 may interfere with the growth of cancer cells and may be an effective treatment for soft tissue sarcoma.

PURPOSE: Phase I/II trial to study the effectiveness of STI571 in treating patients who have recurrent or refractory soft tissue sarcoma.

ReferenceCitation: van Erp N, Gelderblom H, van Glabbeke M, Van Oosterom A, Verweij J, Guchelaar HJ, Debiec-Rychter M, Peng B, Blay JY, Judson I. Effect of cigarette smoking on imatinib in patients in the soft tissue and bone sarcoma group of the EORTC. Clin Cancer Res. 2008 Dec 15;14(24):8308-13. doi: 10.1158/1078-0432.CCR-08-1303.

ReferenceCitation: Judson I, Ma P, Peng B, Verweij J, Racine A, di Paola ED, van Glabbeke M, Dimitrijevic S, Scurr M, Dumez H, van Oosterom A. Imatinib pharmacokinetics in patients with gastrointestinal stromal tumour: a retrospective population pharmacokinetic study over time. EORTC Soft Tissue and Bone Sarcoma Group. Cancer Chemother Pharmacol. 2005 Apr;55(4):379-386. doi: 10.1007/s00280-004-0876-0. Epub 2004 Dec 9.

ReferenceCitation: Debiec-Rychter M, Dumez H, Judson I, Wasag B, Verweij J, Brown M, Dimitrijevic S, Sciot R, Stul M, Vranck H, Scurr M, Hagemeijer A, van Glabbeke M, van Oosterom AT; EORTC Soft Tissue and Bone Sarcoma Group. Use of c-KIT/PDGFRA mutational analysis to predict the clinical response to imatinib in patients with advanced gastrointestinal stromal tumours entered on phase I and II studies of the EORTC Soft Tissue and Bone Sarcoma Group. Eur J Cancer. 2004 Mar;40(5):689-95.

ReferenceCitation: Verweij J, van Oosterom A, Blay JY, Judson I, Rodenhuis S, van der Graaf W, Radford J, Le Cesne A, Hogendoorn PC, di Paola ED, Brown M, Nielsen OS. Imatinib mesylate (STI-571 Glivec, Gleevec) is an active agent for gastrointestinal stromal tumours, but does not yield responses in other soft-tissue sarcomas that are unselected for a molecular target. Results from an EORTC Soft Tissue and Bone Sarcoma Group phase II study. Eur J Cancer. 2003 Sep;39(14):2006-11.

ReferenceCitation: van Oosterom AT, Judson IR, Verweij J, Stroobants S, Dumez H, Donato di Paola E, Sciot R, Van Glabbeke M, Dimitrijevic S, Nielsen OS; European Organisation for Research and Treatment of Cancer Soft Tissue and Bone Sarcoma Group. Update of phase I study of imatinib (STI571) in advanced soft tissue sarcomas and gastrointestinal stromal tumors: a report of the EORTC Soft Tissue and Bone Sarcoma Group. Eur J Cancer. 2002 Sep;38 Suppl 5:S83-7.

ReferenceCitation: van Oosterom AT, Judson I, Verweij J, Stroobants S, Donato di Paola E, Dimitrijevic S, Martens M, Webb A, Sciot R, Van Glabbeke M, Silberman S, Nielsen OS; European Organisation for Research and Treatment of Cancer Soft Tissue and Bone Sarcoma Group. Safety and efficacy of imatinib (STI571) in metastatic gastrointestinal stromal tumours: a phase I study. Lancet. 2001 Oct 27;358(9291):1421-3.

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

location_country:
Denmark
United Kingdom
France
Belgium
Netherlands

location_facility: Herlev Hospital - University Hospital of Copenhagen


type: Other Identifier

type: EudraCT Number

title: Efficacy and Safety of NNC6019-0001 at Two Dose Levels in Participants With Transthyretin Amyloid Cardiomyopathy (ATTR CM)

summary: This study is testing a potential new medicine, NNC6019-0001, for people who have a heart disease due to TTR amyloidosis.The study will look at if this medicine can reduce the symptoms of a heart disease due to TTR amyloidosis, such as heart failure. Participants will either get NNC6019-0001 (apotential new medicine) or placebo (a medicine which has no effect on the body). Which treatment participants get is decided by chance. The chance of getting NNC6019-0001 is two times higher than getting placebo. NNC6019-0001 is not yet approved in any country or region in the world. It is a new medicine that doctors cannot prescribe yet. Participants will get an infusion of the study medicine 13 times, once every 4 weeks. The study will last for about 64 weeks after the first dose of medicine. Participants cannot participate in this study if they have a heart disease other than a heart disease due to TTR amyloidosis.

study_start_date_type: Actual

status: Recruiting

ipd_sharing: Yes

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

lead_sponsor: Novo Nordisk A/S

location_country:
Portugal
Spain
Italy
Netherlands
Canada
United States
France
Japan
Germany
Czechia

status_simplified: Ongoing


title: A Randomized, Open-Label, Phase 2 Study Evaluating Abemaciclib in Combination With Irinotecan and Temozolomide in Participants With Relapsed or Refractory Ewing's Sarcoma

summary: The purpose of this study is to measure the benefit of adding abemaciclib to chemotherapy for Ewing's sarcoma that has come back or did not respond to treatment. This trial is part of the CAMPFIRE master protocol, which is a platform to speed development of new treatments for children and young adults with cancer. Your participation in this trial could last 11 months or longer, depending on how you and your tumor respond.

ipd_sharing_description: Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.

lead_sponsor: Eli Lilly and Company

location_country:
Spain
Italy
Australia
United States
France
Japan
Germany

location_facility: Hyogo Prefectural Kobe Children's Hospital

location_facility: Fondazione Policlinico Universitario Agostino Gemelli

location_facility: The Regents of the University of California - Los Angeles (UCLA Pediatrics)

location_facility: Hospital Universitari i Politecnic La Fe

location_facility: Hospital Infantil Universitario Niño Jesús

location_facility: Hospital General Universitario Gregorio Marañon

location_facility: Institut Bergonié - Centre Régional de Lutte Contre Le Cancer de Bordeaux et Sud Ouest

location_facility: Riley Hospital for Children at Indiana University Health

location_facility: Hospital Universitario Fundación Jiménez Díaz


title: A Multicentre Trial Evaluating the Efficacy and Safety of Oral Decitabine Tetrahydrouridine (NDec) in Patients With Sickle Cell Disease

summary:
This study will look at how well a potentially new medicine called NDec works and is tolerated in people with sickle cell disease. NDec is a combination of two medicines (decitabine-tetrahydrouridine). Both medicines are new for the treatment of sickle cell disease,

Participants will either get NDec, placebo or continue on Hydroxyurea (HU) - which treatment participants get is decided by chance. If participants get NDec or the placebo, they will get capsules to take twice weekly.

The study will last for about a year.

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

location_country:
Spain
Greece
Italy
United Kingdom
Canada
Switzerland
India
United States
France
Turkey
South Africa


title: A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)

summary: This is a study to evaluate the safety and efficacy of losmapimod in treating patients with Facioscapulohumeral Muscular Dystrophy (FSHD) over 48 weeks.

ReferenceType: background

ReferenceCitation: Barbour AM, Sarov-Blat L, Cai G, Fossler MJ, Sprecher DL, Graggaber J, McGeoch AT, Maison J, Cheriyan J. Safety, tolerability, pharmacokinetics and pharmacodynamics of losmapimod following a single intravenous or oral dose in healthy volunteers. Br J Clin Pharmacol. 2013 Jul;76(1):99-106. doi: 10.1111/bcp.12063.

ReferenceCitation: Han JJ, Kurillo G, Abresch RT, de Bie E, Nicorici A, Bajcsy R. Reachable workspace in facioscapulohumeral muscular dystrophy (FSHD) by Kinect. Muscle Nerve. 2015 Feb;51(2):168-75. doi: 10.1002/mus.24287. Epub 2014 Nov 19.

ReferenceCitation: Mellion ML, Ronco L, Berends CL, Pagan L, Brooks S, van Esdonk MJ, van Brummelen EMJ, Odueyungbo A, Thompson LA, Hage M, Badrising UA, Raines S, Tracewell WG, van Engelen B, Cadavid D, Groeneveld GJ. Phase 1 clinical trial of losmapimod in facioscapulohumeral dystrophy: Safety, tolerability, pharmacokinetics, and target engagement. Br J Clin Pharmacol. 2021 Dec;87(12):4658-4669. doi: 10.1111/bcp.14884. Epub 2021 May 14.

ipd_sharing: No

lead_sponsor: Fulcrum Therapeutics

location_country:
Denmark
Spain
Italy
United Kingdom
Netherlands
Canada
United States
France
Germany

location_facility: University of Colorado Anschutz Medical Campus

location_facility: Washington University School of Medicine

location_facility: Aarhus Universitetshospital, Department of Neurology

location_facility: Newcastle upon Tyne NHS Foundation Trust

location_facility: University of California Los Angeles (UCLA)

location_facility: Montreal Neurological Institute and Hospital

location_facility: Department of Neurosciences, Reproductive Sciences, and Odontostomatology, University of Naples "Federico II

location_facility: University of Texas Southwestern Medical Center

location_facility: University of Massachusetts Memorial Medical Center

location_facility: LMU Klinikum Ludwig-Maximilians-Universität München

location_facility: Institute de Myologie, Groupe Hospitalier Pitié-Salpêtrière

location_facility: Fondazione IRCCS Istituto Neurologico Carlo Besta


title: Characteristics and Management of Acute Kidney Injury in Hospitalized Patients With Cirrhosis: a Multicenter Intercontinental Observational Prospective Study: The International Club of Ascites GLOBAL AKI Project

acronym: GLOBAL-AKI

summary:
The aims of this study will be to identify the clinical characteristics, the management and the outcomes of acute kidney injury in patients with cirrhosis worldwide.

Specific aims:

To establish the severity of AKI across different regions
To identify precipitants of AKI across different centers
To identify the phenotypes of AKI across different centers
To evaluate differences in the management of AKI across different centers and their impact on clinical outcomes
To assess outcomes of acute kidney injury (resolution of AKI, in-hospital mortality, 28-day mortality, 90-day mortality)

study_type: Observational

time_perspective: Prospective

ReferenceCitation: Angeli P, Ginès P, Wong F, Bernardi M, Boyer TD, Gerbes A, Moreau R, Jalan R, Sarin SK, Piano S, Moore K, Lee SS, Durand F, Salerno F, Caraceni P, Kim WR, Arroyo V, Garcia-Tsao G. Diagnosis and management of acute kidney injury in patients with cirrhosis: revised consensus recommendations of the International Club of Ascites. J Hepatol. 2015 Apr;62(4):968-74. doi: 10.1016/j.jhep.2014.12.029. Epub 2015 Jan 28. Erratum in: J Hepatol. 2015 Jul;63(1):290.

ipd_sharing: Undecided

lead_sponsor: Azienda Ospedaliera di Padova

location_country:
Spain
Italy
United States
Peru
Poland
Russian Federation
Argentina
Ethiopia
India
Mexico
Egypt
Brazil
Hungary
China
Germany
Denmark
Korea, Republic of
France
Chile
Netherlands
Paraguay

location_facility: Hospital de Clínicas Facultad de Ciencias Médicas U.N.A.

location_facility: Hospital Nacional Prof. Alejandro Posadas

location_facility: Hospital de Gastroenterología "Dr. Carlos Bonorino Udaondo"

location_facility: IRCCS Azienda Ospedaliera-Universitaria di Bologna

location_facility: Shanghai Jiao Tong University School of Medicine

location_facility: Instituto Nacional de Ciencias Médicas y Nutrición "Salvador Zubirán"

location_facility: Azienda Ospedaliero Universitaria Città della Salute e della Scienza di Torino

intervention_type: Combination Product


title: A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Nipocalimab in Participants With Active Idiopathic Inflammatory Myopathies

acronym: SPIREA

summary: The purpose of this study is to evaluate the efficacy and safety of Nipocalimab versus placebo in participants with active idiopathic inflammatory myopathies (IIM).

ipd_sharing_description: The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

lead_sponsor: Janssen Research & Development, LLC

location_country:
Poland
Spain
Italy
United Kingdom
Netherlands
Canada
Hungary
Korea, Republic of
United States
France
Mexico
Japan
Germany
Czechia

location_facility: A.O. Universitaria Ospedali Riuniti di Ancona

location_facility: Wexner Medical Center at the Ohio State University

location_facility: Fondazione Policlinico Universitario A. Gemelli IRCCS

location_facility: Hôpitaux Universitaires de Strasbourg - Hôpital de Hautepierre

location_facility: Centro Integral en Reumatología S.A de C.V.

location_facility: Centro de Estudios de Investigacion Basica y Clinica, S.C.

location_facility: Országos Mozgásszervi Intézet ORFI telephely

location_facility: University of California Irvine Medical Center

location_facility: National Hospital Organization Kyushu Medical Center

location_facility: Academisch Medisch Centrum Universiteit van Amsterdam

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

location_facility: Instituto Nacional de Ciencias Medicas y Nutrición Salvador Zubirán

location_facility: Attune Health Autoimmune and Inflamation Care and Research

location_facility: ACME Research Arthritis and Osteoporosis Center

location_facility: National Hospital Organization Osaka Minami Medical Center

location_facility: University College London Hospitals NHSFT

location_facility: Azienda Ospedaliera Universitaria di Padova

location_facility: Narodowy Instytut Geriatrii, Reumatologii i Rehabilitacji im. prof. dr hab. med. Eleonory Reicher

location_facility: University of Pennsylvania - Perelman School of Medicine

location_facility: Lawson Health Research / London Health Sciences Center Research

intervention_type: Other


title: A Phase 3, Long-Term Extension Study to Evaluate the Safety and Efficacy of Imsidolimab (ANB019) in the Treatment of Adult Subjects With Generalized Pustular Psoriasis

summary: This is a Phase 3, long term extension study to evaluate the safety and efficacy of imsidolimab compared with placebo in adult subjects with generalized pustular psoriasis (GPP).

lead_sponsor: AnaptysBio, Inc.

location_country:
Poland
Taiwan
Spain
Georgia
Australia
Thailand
Tunisia
Korea, Republic of
United States
France
Malaysia
Turkey
Romania
Germany
Morocco

intervention_type: Biological


title: A Phase 1b Study Evaluating the Safety and Efficacy of First-Line Tarlatamab in Combination With Carboplatin, Etoposide, and PD-L1 Inhibitor in Subjects With Extensive Stage Small Cell Lung Cancer

summary: This is a phase 1b study to assess the safety and tolerability of tarlatamab in combination with programmed death ligand (PD-L1) inhibition with and without chemotherapy.

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:
Taiwan
Spain
Australia
Canada
Switzerland
Korea, Republic of
United States
France
Belgium
Japan
Germany

location_facility: The University of North Carolina at Chapel Hill

location_facility: New York University Langone Medical Center

location_facility: West Virginia University Health Sciences Center

location_facility: University of Southern California, Norris Comprehensive Cancer Center

location_facility: Centre Hospitalier Universitaire de Nantes, Hôpital Nord Laënnec

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


title: A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients With Late-onset OTC Deficiency

summary: The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels with removal of dietary protein restriction and alternative pathway medication.

study_start_date_type: Anticipated

lead_sponsor: Ultragenyx Pharmaceutical Inc

location_country:
Brazil
Portugal
Spain
Italy
United Kingdom
Australia
Netherlands
Argentina
Canada
United States
France
Japan
Germany

location_facility: Fundacio Hospital Universitari Vall D'Hebron-Institute de Recerca

location_facility: University Hospitals Cleveland Medical Center

location_facility: University of Arkansas for Medical Sciences

location_facility: Centro Hospitalar Universitario de Sao Joao

location_facility: Erasmus Universitair Medisch Centrum Rotterrdam

location_facility: Hospital Clinico Universitario de Santiago

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


title: A Phase 2/3 Adaptive, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of VX-147 in Subjects Aged 18 Years and Older With APOL1-mediated Proteinuric Kidney Disease

summary: The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics (PK) of VX-147 in participants aged 18 years and older with apolipoprotein L1 (APOL1)-mediated proteinuric kidney disease.

ipd_sharing_description: Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

lead_sponsor: Vertex Pharmaceuticals Incorporated

location_country:
Spain
United Kingdom
Canada
Puerto Rico
United States
France

location_facility: Northwest Louisiana Nephrology, LLC - Shreveport

location_facility: Nevada Kidney Disease and Hypertension Centers

location_facility: Vanderbilt University Medical Center, Nephrology Clinical Trials Center

location_facility: AKDHC Medical Research Service LLC - Banner Desert Office

location_facility: Eastern Nephrology Associates - New Bern Office

location_facility: Children's Hospital of Pittsburgh of UPMC

location_facility: Nephrology and Hypertension Associates, LTD

location_facility: Clinical Research Development Associates LLC

location_facility: Eastern Nephrology Associates - Wilmington Office

location_facility: Eastern Nephrology Associates - Jacksonville Office

location_facility: Eastern Nephrology Associates - Kinston Office

location_facility: Centre Hospitalier Universitaire (CHU) - Grenoble Alpes

location_facility: Eastern Nephrology Associates - Greenville Office

location_facility: University Hospitals of Leicester NHS Trust - Leicester General Hospital

location_facility: Mid Florida Kidney and Hypertension Care, PL

location_facility: Nephrology & Hypertension Associates, PC

location_facility: South Florida Nephrology Group PA, Research Div.

location_facility: Children's Healthcare of Atlanta - Center for Advanced Pediatrics

location_facility: Renal and Transplant Associates of New England, PC

location_facility: King's College Hospital NHS Foundation Trust - Guthrie Clinic

location_facility: SUNY Downstate Health Sciences University

location_facility: Barts Health NHS Trust, The Royal London Hospital

location_facility: UNC Clinical and Translational Research Center

location_facility: Albert Einstein Hospital - Montefiore Medical Center


type: Registry Identifier

title: A Phase III, Randomized, Open-Label Study Evaluating the Efficacy and Safety of Giredestrant in Combination With Phesgo Versus Phesgo After Induction Therapy With Phesgo + Taxane in Patients With Previously Untreated HER2-Positive, Estrogen Receptor-Positive Locally-Advanced or Metastatic Breast Cancer

summary: This Phase III, randomized, two-arm, open-label, multicenter study will evaluate the efficacy and safety of giredestrant plus Phesgo compared with Phesgo after induction therapy with Phesgo plus taxane in participants with human epidermal growth factor receptor 2 (HER2)-positive, estrogen receptor (ER)-positive advanced breast cancer (metastatic or locally advanced disease not amenable to curative treatment) who have not previously received a systemic non-hormonal anti-cancer therapy in the advanced setting.

ipd_sharing_description:
Qualified researchers may request access to individual patient level data through the 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 Sharing of Clinical Study 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).

location_country:
Poland
Taiwan
Portugal
Spain
Italy
United Kingdom
Mexico
Argentina
Thailand
Hungary
Korea, Republic of
France
United States
China
Turkey
Belgium
Germany

location_facility: CH de la Côte Basque - Hôpital de Bayonne

location_facility: Maryland Oncology Hematology; Maryland Oncology Hematology, PA

location_facility: Prof. Dr. Cemil Tascioglu City Hospital; Med Onc

location_facility: Hospital Universitario La Paz; Servicio de Oncologia

location_facility: Ataturk University Medical Faculty Yakutiye Research Hospital Medical Oncology Department

location_facility: Universitätsklinikum des Saarlandes; Klinik f. Frauenheilkunden und Geburtshilfe

location_facility: Studienzentrum Onkologie Ravensburg GbR; Onkologie Ravensburg

location_facility: Policlinico Universitario Agostino Gemelli

location_facility: ASST DEGLI SPEDALI CIVILI DI BRESCIA; Oncologia Medica

location_facility: Hospital Universitario Miguel Servet; Servicio Oncologia

location_facility: Azienda Unità Sanitaria Locale di Reggio Emilia/IRCCS

location_facility: Changhua Christian Hospital; Dept of Surgery

location_facility: Gazi Uni Medical Faculty Hospital; Oncology Dept

location_facility: Hospital Civil de Guadalajara Fray Antonio Alcalde

location_facility: Ospedale Civile; Unita Operativa Di Oncologia Medica

location_facility: the First Affiliated Hospital of Bengbu Medical College

location_facility: Mersin City Education and Research Hospital

location_facility: Gesundheitszentrum Wetterau, Hochwaldkrankenhaus Bad Nauheim; Gynäkologie/ onkolog. Tagesklinik

location_facility: Ramathibodi Hospital; Dept of Med.-Div. of Med. Onc

location_facility: Istituto Nazionale Tumori Irccs Fondazione g. PASCALE;U.O.C. Oncologia Medica Senologica

location_facility: Ospedale Infermi AUSL della Romagna; U.O Operativa di Oncologia

location_facility: Trakya Universitesi Tip Fakultesi, Medikal Onkoloji Bilim Dali, Balkan Yerleskesi

location_facility: Kocaeli University Faculty of Medicine; Medical oncology

location_facility: Royal Cornwall Hospital; Dept of Clinical Oncology

location_facility: IOV - Istituto Oncologico Veneto - IRCCS; Oncologia Medica II

location_facility: Hospital Arnau de Vilanova (Valencia) Servicio de Oncologia

location_facility: Los Angeles Hematology Oncology Medical Group

location_facility: Hospital Clinico Universitario Virgen de la Victoria; Servicio de Oncologia

location_facility: Medipol University MF; Oncology Department

location_facility: Hospital Universitario de Canarias;servicio de Oncologia

location_facility: Kyungpook National University Chilgok Hospital

location_facility: Katip Celebi University Ataturk Training and Research Hospital; Oncology

location_facility: Zhejiang Cancer Hospital; Zhejiang Cancer Hospital cancer department

location_facility: Severance Hospital, Yonsei University Health System

location_facility: National Taiwan University Hospital ; Dept of Surgery & Hepatitis Research Center

location_facility: Fundación CENIT para la Investigación en Neurociencias

location_facility: Hospital Beatriz Angelo; Departamento de Oncologia

location_facility: Humanitas Centro Catanese Di Oncologia; Oncologia Medica

location_facility: Ärztehaus am Bahnhofsplatz; Praxis Uleer/Pourfard

location_facility: National Cheng Kung University Hospital; Oncology

location_facility: Taipei Veterans General Hospital Office of General Surgery

location_facility: Affiliated Hospital of Hebei University; Department of medical oncology

location_facility: Jasz-Nagykun-Szolnok Megyei Hetenyi Geza Korhaz-Rendelointezet; Megyei Onkologiai Kozpont

location_facility: China Medical University Hospital; Surgery

location_facility: Magyar Honvédség Egészségügyi Központ; Onkológiai Osztály

location_facility: Namik Kemal Universitesi Tip Fakultesi, Medikal Onkoloji Bilim Dali, 100

location_facility: Florida Cancer Specialists - EAST - SCRI - PPDS

location_facility: Kaposi Mor County Hospital; Dept. of Oncology

location_facility: Texas Oncology-Baylor Sammons Cancer Center

location_facility: Istituto Clinico Humanitas;U.O. Oncologia Medica Ed Ematologia

location_facility: Maharaj Nakorn Chiang Mai Hospital; Department of Surgery/Head Neck and Breast Unit; Clinical Trial

location_facility: Adana Baskent University Hospital; Medical Oncology

location_facility: Complejo Hospitalario Universitario de Santiago (CHUS) ; Servicio de Oncologia

location_facility: Hospital Clínic i Provincial; Servicio de Oncología

location_facility: Hacettepe Uni Medical Faculty Hospital; Oncology Dept


title: Randomised, Open-label and Parallel Group Trial to Investigate the Effects of Oral BI 685509 Alone or in Combination With Empagliflozin on Portal Hypertension After 8 Weeks Treatment in Patients With Clinically Significant Portal Hypertension (CSPH) in Compensated Cirrhosis

summary:
This study is open to adults with liver cirrhosis caused by hepatitis B, hepatitis C or nonalcoholic steatohepatitis (NASH). People can join this study if they have high blood pressure in the portal vein (main vessel going to the liver).

The purpose of this study is to find out whether a medicine called BI 685509 taken alone or in combination with a medicine called empagliflozin helps people with this condition.

Participants take BI 685509 as tablets twice a day for 8 weeks. Half of the participants with NASH who also have type 2 diabetes take empagliflozin as tablets once a day in addition to BI 685509.

Participants are in the study for about 3 months. During this time, they visit the study site about 10 times. At 2 of the visits, the doctors check the pressure in a liver vein to see whether the treatment works. This is done with a catheter (a long thin tube) and gives information about the pressure in the portal vein. The doctors also regularly check participants' 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.

location_country:
Denmark
Spain
Italy
Austria
Canada
United States
France
China
Belgium
Japan
Netherlands

location_facility: The Affiliated Hospital of Hangzhou Normal University

location_facility: ASST Grande Ospedale Metropolitano Niguarda

location_facility: National Hospital Organization Yokohama Medical Center


title: A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid

summary:
ARGX-113-2009 is an operationally seamless 2-part, phase 2/3, prospective, global, multicenter, randomized, double-blinded, placebo-controlled study to investigate the efficacy, safety, tolerability, immunogenicity, participant-reported outcome measures (including those assessing participant QoL), PK, and PD of efgartigimod PH20 SC administered via subcutaneous (SC) injection in adult participants with moderate to severe BP. This study intends to demonstrate that efgartigimod is an effective and safe treatment for BP, providing participants with control of disease activity (CDA) and eventually remission while reducing their cumulative exposure to OCS.

study will consist of 2 parts:

Part A of the study is a phase 2 evaluation that intends to provide proof of concept for the therapeutic activity of efgartigimod PH20 SC in participants with BP.
Part B of the study is a phase 3 evaluation that intends to confirm the results obtained from part A in a separate, larger group of participants with BP.

An interim analysis will be performed during part A (on data obtained through week 26 for all Part A participants) to assess the primary endpoint and several secondary endpoints, confirm the appropriate sample size for part B of the study, and determine whether the efficacy results observed through week 26 of part A warrant continued study of efgartigimod PH20 SC for the treatment of participants with BP (futility analysis).

Other than differences in main goals, endpoints, and statistical analyses, parts A and B are identical in schedule, structure, assessments, and conduct.

lead_sponsor: argenx

location_country:
Croatia
Poland
Spain
Italy
United Kingdom
Australia
Hungary
United States
France
Israel
Germany
Bulgaria

location_facility:
Investigator site 14 - BG3590010
Investigator site 43 - IT0390060
Investigator site 30 - IT0390040
Investigator site 11 - HU0360003
Investigator site 49 - ES0340058
Investigator site 28 - BG3590018
Investigator site 32 - ES0340050
Investigator site 8 - ES0340053
Investigator site 33 - UK0440022
Investigator site 27 - AU0610006
Investigator site 45 - DE0490001
Investigator site 37 - HR3850001
Investigator site 2 - US0010087
Investigator site 18 - PL0480048
Investigator site 4 - US0010137
Investigator 24 - ES0340051
Investigator site 31 - ES0340057
Investigator site 46 - DE0490039
Investigator site 41 - IL9720001
Investigator site 17 - PL0480047
Investigator site 42 - IT0390005
Investigator site 3 - US0010151
Investigator 20 - ES0340029
Investigator site 44 - UK0440037
Investigator site 50 - US0010149
Investigator site 5 - US0010088
Investigator site 29 - FR0330029
Investigator site 36 - AU0610013
Investigator site 19 - PL0480046
Investigator site 40 - IL9720002
Investigator site 26 - HU0360023
Investigator site 15 - BG3590020
Investigator site 48 - ES0340059
Investigator site 6 - US0010138
Investigator site 12 - ES0340025
Investigator site 34 - US0010182
Investigator site 1 - US0010017
Investigator site 13 - US0010155
Investigator site 23 - IT0390006
Investigator site 25 - US0010158
Investigator site 35 - US0010156
Investigator site 38 - FR0330040
Investigator site 10 - US0010153
Investigator site 21 - US0010155
Investigator site 7 - HU0360008
Investigator site 47 - IT0390031
Investigator site 9 - ES0340052
Investigator site 16 - HR3850002
Investigator site 22 - HR3850003
Investigator site 39 - IL9720003


title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study With an Open-Label Extension Assessing the Efficacy, Safety, and Pharmacokinetics/Pharmacodynamics of Tirzepatide in Pediatric and Adolescent Participants With Type 2 Diabetes Mellitus Inadequately Controlled With Metformin, or Basal Insulin, or Both

acronym: SURPASS-PEDS

summary:
The purpose of this study is to learn more about the safety and efficacy of tirzepatide compared to placebo in children or teenagers with type 2 diabetes taking metformin, or basal insulin, or both.

The overall study will last about 60 weeks with up to 14 clinic visits and 6 phone visits. Clinic visits will include blood sample collection, physical exam and questionnaire.

location_country:
Brazil
United Kingdom
Italy
Australia
India
United States
France
Israel
Mexico

location_facility: Indiana University Health University Hospital

location_facility: Universita degli Studi della Campania Luigi Vanvitelli

location_facility: CEPIC - Centro Paulista de Investigação Clínica

location_facility: Postgraduate Institute of Medical Education & Research

location_facility: Centre Hospitalier Universitaire d'Angers

location_facility: Nemours Childrens Clinic - Delaware Valley of The Nemours Foundation

location_facility: Consultorio Médico de Endocrinología y Pediatría

location_facility: Bhakti Vedanta Hospital and Research Institute

location_facility: Center Of Excellence in Diabetes and Endocrinology

location_facility: Kovai Diabetes Speciality Center and Hospital

location_facility: Instituto da Crianca do Hospital das Clinicas da FMUSP

location_facility: Assistance Publique - Hopitaux de Paris (AP-HP) - Hopital Robert Debre - Centre Hospitalo Universitaire (CHU)

location_facility: Azienda Ospedaliera Universitaria Integrata Verona - Ospedale Borgo Trento

location_facility: SUNY Downstate Health Sciences University

location_facility: Children's Hospital of Philadelphia (CHOP)

location_facility: Instituto de Pesquisa clinica de Campinas

location_facility: M S Ramaiah Medical College and Hospitals


title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3, Three-way Crossover Trial to Evaluate the Efficacy and Safety of Two Dose Levels of KVD900, an Oral Plasma Kallikrein Inhibitor, for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema Type I or II

summary: This study is a randomized, double-blind, placebo-controlled, phase III, three-way crossover clinical trial evaluating the efficacy and safety of KVD900, in the treatment of hereditary angioedema attacks in adolescent and adult Patients

lead_sponsor: KalVista Pharmaceuticals, Ltd.

location_country:
Poland
Spain
Greece
United Kingdom
Australia
New Zealand
Canada
Hungary
North Macedonia
Puerto Rico
United States
France
Israel
Netherlands
Bulgaria

location_facility:
KalVista Investigative Site
Kalvista Investigative Site


title: Aveir Dual-Chamber Leadless i2i IDE Study

summary: Prospective, non-randomized, multi-center, international study designed to evaluate the safety and effectiveness of the Aveir™ Dual-Chamber (DR) Leadless Pacemaker system.

lead_sponsor: Abbott Medical Devices

location_country:
Hong Kong
Spain
Italy
Austria
Netherlands
United Kingdom
Canada
United States
France
Belgium
Japan
Germany
Czechia

location_facility: Azienda Ospedaliero Universitaria Pisana

location_facility: Kansas City Cardiac Arrhythmia Research Foundation

location_facility: New York-Presbyterian/Columbia University Medical Center

location_facility: Prairie Education & Research Cooperative

location_facility: Kaiser Permanente Los Angeles Medical Center

location_facility: Donald Guthrie Foundation for Education & Research

location_facility: Institut de Cardiologie de Montreal (Montreal Heart Inst.)

location_facility: Wake Forest University Medical Center Clinical Sciences

location_facility: New York Presbyterian Hospital/Cornell University

location_facility: University of California at San Diego (UCSD) Medical Center

intervention_type: Device


title: A Phase 1b, Single Ascending Dose, Randomized, Double-blind Study to Evaluate the Safety, Tolerability, and Activity of SAR439459 in Adults With Osteogenesis Imperfecta

summary:
SAR439459 is a human anti-TGFβ monoclonal antibody. This phase 1 clinical study investigates the safety, tolerability, and activity of a single dose of SAR439459 in adult participants with OI.

Participants will receive a single IV dose of SAR439459 with safety, pharmacokinetic (PK), and pharmacodynamic (PD) assessments over 24 weeks.

There will be up to 3 dose cohorts. In addition to safety, tolerability, and PK assessments, bone mineral density (BMD) will be evaluated by dual-energy Xray absorptimetry (DXA) scan and a series of blood biomarkers will be monitored to document pharmacodynamic effects of the single dose of SAR439459.

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:
Australia
United States
Canada
France

location_facility: New Mexico Clinical Research & Osteoporosis Center-Site Number:8400001

location_facility: Department of Medicine/ School of Clinical Sciences at Monash Health Monash University_246 Clayton Road_Site Number :0360002

location_facility: Bone Research and Education Centre_Site Number :1240003

location_facility: Hopital Edouard Herriot _Site Number :2500002

location_facility: Hopital Lariboisiere_Site Number :2500001

location_facility: Baylor College of Medicine - Site Number:8400003


title: A Phase I/II Open-label, Multi-center Study to Assess Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of AZD7789, an Anti-PD-1 and Anti-TIM-3 Bispecific Antibody, in Patients With Relapsed or Refractory Classical Hodgkin Lymphoma

summary: The study is intended to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of AZD7789 in patients with relapsed/refractory classical Hodgkin Lymphoma (r/r cHL).

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 requests will be evaluated as per the AZ disclosure commitment:

https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

lead_sponsor: AstraZeneca

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


title: An Open Label, Single Arm, Extension Trial to Examine Long-term Safety of BI 425809 Once Daily in Patients With Schizophrenia Who Have Completed Previous BI 425809 Phase III Trials (CONNEX-X)

summary:
This study is open to adults with schizophrenia who took part in a previous CONNEX study (study 1346-0011, 1346-0012, or 1346-0013). The purpose of this study is to find out how well people with schizophrenia can tolerate a medicine called BI 425809 in the long term.

Participants take BI 425809 as tablets once a day for 1 year. In addition, all participants take their normal medication for schizophrenia.

Participants are in the study for a little more than 1 year. During this time, they visit the study site about 13 times and get about 9 phone calls from the study team.

The doctors collect information on any health problems of the participants. Doctors also regularly check the participants' symptoms of schizophrenia.

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.

location_country:
Denmark
Poland
Taiwan
Brazil
Spain
Greece
Italy
Mexico
Argentina
Sweden
Korea, Republic of
United States
Chile
China
France
Belgium
Japan
Germany
Norway
Czechia

location_facility: Universitair Psychiatrisch Centrum Duffel (UPC Duffel)

location_facility: Instituto Médico de la Fundación Estudios Clínicos

location_facility: Zentrum für klinische Forschung Dr. Schöll, Dr. Steidl & Kollegen

location_facility: National Hospital Organization Hizen Psychiatric Medical Center

location_facility: Hospital Provincial. Complejo Asistencial de Zamora

location_facility: The Affiliated Brain Hospital of Guangzhou Medical University

location_facility: The Second Affiliated Hospital of Xinxiang Medical Univ.

location_facility: The First Hospital of Hebei Medical University

location_facility: Klinikum der Universität München - Campus Innenstadt

location_facility: National Center of Neurology and Psychiatry

location_facility: Podlassian Center of Psychogeriatry, Bialystok

location_facility: "Attikon" University General Hospital of Attica

location_facility: Seoul National University Bundang Hospital

location_facility: Artemis Institute for Clinical Research, LLC

location_facility: Resolution Psychopharmacology Research Institute

location_facility: The Second Xiangya Hospital Of Central South University

location_facility: Clínica Viver - Centro de Desospitalização Humana

location_facility: Individual Specialist Medical Practice Filip Rybakowski

location_facility: Pusan National University Yangsan Hospital

location_facility: Azienda Sanitaria Ospedale S. Luigi Gonzaga

location_facility: CEN (Centro Especializado Neurociencias)

location_facility: The Affiliated Hospital of Guizhou Medical University

location_facility: First Affiliated Hospital of Xi'an JiaoTong University

location_facility: Institute of Psychiatry and Neurology in Warsaw

location_facility: The sixth People's Hospital of Hebei Province


title: A Modular Phase I/II, Open-label, Dose Escalation and Expansion, Multicentre Study to Assess the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of AZD0466 as Monotherapy or in Combination With Anticancer Agents in Patients With Advanced Non-Hodgkin Lymphoma.

summary: This study evaluates the safety, tolerability, PK, and preliminary efficacy of AZD0466 as monotherapy or in combination with other anticancer agents in patients with advanced NHL

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 requests 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.

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


title: A Phase 3 Open-Label Extension Study of PTC923 in Phenylketonuria

summary: The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein consumption.

lead_sponsor: PTC Therapeutics

location_country:
Denmark
Brazil
Portugal
Spain
Italy
Georgia
Australia
Netherlands
Canada
United Kingdom
United States
France
Turkey
Mexico
Germany

location_facility: Stanford University Center for Academic Medicine

location_facility: Birmingham Children's Hospital NHS Foundation Trust

location_facility: University Children's Hospital Hamburg Eppendorf (Kinder-UKE) Klinik für Kinder- und Jugendmedizin (Kinder-UKE)

location_facility: Metabolics and Genetics in Calgary (MAGIC) Clinic, Ltd.

location_facility: Cukurova Üniversity Balcali Hospital Health Application and Research Center

location_facility: Centro Hospitalar Universitário Do Porto, Epe

location_facility: Icahn School of Medicine at Mount Sinai (ISMMS)

location_facility: The Hospital for Sick Children University of Toronto Adult Clinic: The Fred A Litwin Family Centre in Genetic Medicine University Health Network & Mt. Sinai Hospital

location_facility: Ege University Faculty of Medicine Children Hospital

location_facility: University of Utah, Division of Medical Genetics (pediatric and adult clinic)

location_facility: CHRU de Tours- Hôpital Pédiatrique de Clocheville

location_facility: İstanbul Üniversitesi Cerrahpaşa Tıp Fakültesi

location_facility: University of Texas Health Science Center of Texas

location_facility: Universitätsklinikum Heidelberg / Zentrum für Kinder- und Jugendmedizin / Sektion für Neuropädiatrie & Stoffwechselmedizin

location_facility: University of Colorado and the Children's Hospital CO

location_facility: UF College of Medicine, Department of Pediatrics Division of Genetics and Metabolism

location_facility: Copenhagen University Hospital, Rigshospitalet

location_facility: CENTRO HOSPITALAR UNIVERSITÁRIO LISBOA NORTE Hospital de Santa Maria,

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

location_facility: Division of Inherited Metabolic Diseases, Azienda Ospedaliera-Università Padova


title: A Phase 2 Open-label Clinical Study to Evaluate the Efficacy and Safety of Zilovertamab Vedotin (MK-2140) in Participants With Relapsed or Refractory Diffuse Large B-Cell Lymphoma (waveLINE-004)

summary: The purpose of this study is to evaluate zilovertamab vedotin with respect to objective response rate and duration of response per Lugano Response Criteria as assessed by blinded independent central review (BICR). Safety and tolerability will also be evaluated in this Phase 2, single arm, interventional study.

lead_sponsor: Merck Sharp & Dohme LLC

location_country:
Poland
Spain
Italy
Estonia
Thailand
Korea, Republic of
United States
Chile
China
France
Israel
Norway
Sweden
Turkey
Czechia

location_facility: Szpital Specjalistyczny im. Ludwika Rydygiera w Krakowie-Oncology Department ( Site 1707)

location_facility: Hospital Universitario Fundación Jiménez Díaz-Oncology & Hematology ( Site 2000)

location_facility: Georgetown University Medical Center ( Site 0204)

location_facility: University Hospitals Cleveland Medical Center ( Site 0222)

location_facility: Fakultní nemocnice Brno Bohunice-Interni hematologicka a onkologicka klinika ( Site 0800)

location_facility: Fudan University Shanghai Cancer Center ( Site 2908)

location_facility: Massachusetts General Hospital-Cancer Center Protocol Office ( Site 0203)

location_facility: St. Joseph Hospital-The Center for Cancer Prevention and Treatment ( Site 0229)

location_facility: James Lind Centro de Investigación del Cáncer ( Site 2705)

location_facility: Karolinska Universitetssjukhuset Solna ( Site 2102)

location_facility: Faculty of Medicine Siriraj Hospital ( Site 0701)

location_facility: Avera Cancer Institute- Research ( Site 0233)

location_facility: Hospital Universitario de Salamanca - Complejo Asistencial Universitario de Salamanca ( Site 2003)

location_facility: Pitie Salpetriere University Hospital-Clinical haematology ( Site 1000)

location_facility: The James Cancer Hospital and Solove Research Institute at The Ohio State University Comprehensive C

location_facility: Vseobecna fakultni nemocnice v Praze-I. Interní klinika - klinika hematologie ( Site 0801)

location_facility: Soroka Medical Center-Hematology Department ( Site 1403)

location_facility: Severance Hospital, Yonsei University Health System-Medical oncology ( Site 0601)

location_facility: SUN YAT-SEN UNIVERSITY CANCER CENTRE-Internal Medicine ( Site 2907)

location_facility: North Estonia Medical Centre Foundation ( Site 0900)

location_facility: University of Maryland-Greenebaum Comprehensive Cancer Center ( Site 0211)

location_facility: Centre Hospitalier de la Côte Basque ( Site 1002)

location_facility: Ankara University Hospital Cebeci-hematology ( Site 2300)

location_facility: Oslo universitetssykehus, Radiumhospitalet ( Site 1600)

location_facility: University of Cincinnati Medical Center-University of Cincinnati Cancer Center ( Site 0217)

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori-Struttura Complessa di Ematologia ( Site 1501)

location_facility: Dokuz Eylül Üniversitesi-Hematology ( Site 2304)

location_facility: Franciscan St. Francis Health ( Site 0225)

location_facility: Karadeniz Teknik Universitesi Tip Fakultesi-Hematology ( Site 2307)

location_facility: Instituto Catalan de Oncologia - Hospital Duran i Reynals-Haematology Department ( Site 2002)

location_facility: University of Chicago Medical Center ( Site 0207)

location_facility: Saint Louis University Cancer Center ( Site 0209)

location_facility: Shaare Zedek Medical Center ( Site 1404)

location_facility: Clínica Alemana de Santiago ( Site 2704)

location_facility: Hospital Universitari Vall d'Hebron ( Site 2005)

location_facility: Skånes Universitetssjukhus Lund ( Site 2100)

location_facility: Hacettepe Universitesi-Department of Hematology ( Site 2302)

location_facility: Narodowy Instytut Onkologii im. Marii Sklodowskiej-Curie - P-Klinika Nowotworów Układu Chłonnego ( S

location_facility: Szpitale Pomorskie Sp. z o. o.-Hematology and Bone Marrow Transplantation Department ( Site 1702)

location_facility: Istituto Nazionale Tumori IRCCS Fondazione Pascale ( Site 1502)

location_facility: Innovative Clinical Research Institute ( Site 0202)

location_facility: Humanitas-U.O di Oncologia medica ed Ematologia ( Site 1503)

location_facility: Tianjin Medical University Cancer Institute and Hospital-lymphoma ( Site 2901)

location_facility: IRCCS - AOU di Bologna-Istituto di Ematologia "L. e A. Seragnoli" ( Site 1500)


title: A Phase 2/3 Multicenter, Open-label, Randomized, Active-Control Study of Zilovertamab Vedotin (MK-2140) in Combination With Standard of Care in Participants With Relapsed or Refractory Diffuse Large B-Cell Lymphoma (waveLINE-003)

summary: The purpose of this Phase 2/3, randomized, multisite, open-label, dose confirmation, and expansion study is to evaluate the safety, and efficacy of zilovertamab vedotin (ZV) in combination with standard of care options for the treatment of rrDLBCL. This study will be divided into 2 parts: Dose Confirmation (Part 1) and Efficacy Expansion (Part 2) and will enroll participants who are at least 18 years of age with rrDLBCL. The hypotheses are: ZV in combination with rituximab, gemcitabine, and oxaliplatin (R-GemOx) is superior to R-GemOx with respect to progression-free survival (PFS) per Lugano response criteria by blinded independent review committee (BICR); and that ZV in combination with bendamustine rituximab (BR) is superior to BR with respect to PFS per Lugano response criteria by BICR.

location_country:
Poland
Italy
United States
Israel
France
Turkey

location_facility: Norton Women's and Children's Hospital-Norton Cancer Institute - St. Matthews ( Site 0133)

location_facility: centre hospitalier lyon sud-Service Hématologie ( Site 0702)

location_facility: Humanitas-U.O di Oncologia medica ed Ematologia ( Site 1203)

location_facility: Pitie Salpetriere University Hospital-Clinical haematology ( Site 0700)

location_facility: Istituto Nazionale Tumori IRCCS Fondazione Pascale ( Site 1202)

location_facility: Uniwersytecki Szpital Kliniczny-Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku ( Site

location_facility: Innovative Clinical Research Institute ( Site 0122)

location_facility: Louisiana State University Health Sciences Center New Orleans ( Site 0134)

location_facility: Dokuz Eylül Üniversitesi-Hematology ( Site 1905)

location_facility: Centrum Onkologii Ziemi Lubelskiej-Oddzial Hematologiczny ( Site 1304)

location_facility: University of Massachusetts Medical School ( Site 0119)

location_facility: St. Vincent Frontier Cancer Center-Research ( Site 0108)

location_facility: IRCCS - AOU di Bologna-Istituto di Ematologia "L. e A. Seragnoli" ( Site 1200)

location_facility: Ege University Medicine of Faculty ( Site 1902)

location_facility: Ankara University Hospital Cebeci-hematology ( Site 1901)

location_facility: Szpitale Pomorskie Sp. z o. o.-Hematology and Bone Marrow Transplantation Department ( Site 1302)


title: Open-label Study Investigating Efficacy, Safety and Pharmacokinetics of Concizumab Prophylaxis in Children Below 12 Years With Haemophilia A or B With or Without Inhibitors

summary:
This study will test how well a new medicine called concizumab works for participants who have haemophilia A or B with or without inhibitors. The purpose is to show that concizumab can prevent bleeds and is safe to use.

Participants will have to inject the study medicine every day under the skin with a pen-injector.

The study will last for about 2 years.

location_country:
Spain
Italy
United Kingdom
Lithuania
United States
Romania
Japan
Poland
Russian Federation
India
Turkey
Norway
Sweden
Bosnia and Herzegovina
Canada
North Macedonia
Bulgaria
Greece
Estonia
Thailand
France


title: An Open-Label Extension Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)

summary: The purpose of this study is to evaluate the effect of olezarsen (formerly known as AKCEA-APOCIII-LRx) on the percent change in fasting triglycerides (TG) from baseline.

lead_sponsor: Ionis Pharmaceuticals, Inc.

location_country:
Spain
Italy
Canada
United States
France
Netherlands
Slovakia

location_facility: University of Kansas Medical Center (KUMC)

location_facility: Clinique des Maladies Lipidiques de Quebec Inc.

location_facility: Academisch Medisch Centrum Goederenontvangst Apotheek t.a.v. Kenniscentrum

location_facility: Institute de Recherches Cliniques de Montreal

location_facility: Groupement Hospitalier Est- Pharmacie Secteur Essais Clinique

location_facility: Diabetes/Lipid Management & Research Center


title: A Phase 3 Study of PTC923 in Subjects With Phenylketonuria

summary: The main purpose of this trial is to evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in participants with phenylketonuria as measured by mean change in blood Phe levels from baseline to Weeks 5 and 6 (that is, the average of each respective treatment dose 2-week period of double-blind treatment).

location_facility: Stanford University Center for Academic Medicine

location_facility: Birmingham Children's Hospital NHS Foundation Trust

location_facility: University Children's Hospital Hamburg Eppendorf (Kinder-UKE) Klinik für Kinder- und Jugendmedizin (Kinder-UKE)

location_facility: Metabolics and Genetics in Calgary (MAGIC) Clinic, Ltd.

location_facility: Cukurova Üniversity Balcali Hospital Health Application and Research Center

location_facility: Centro Hospitalar Universitário Do Porto, Epe

location_facility: Icahn School of Medicine at Mount Sinai (ISMMS)

location_facility: Ege University Faculty of Medicine Children Hospital

location_facility: University of Utah, Division of Medical Genetics (pediatric and adult clinic)

location_facility: CHRU de Tours- Hôpital Pédiatrique de Clocheville

location_facility: İstanbul Üniversitesi Cerrahpaşa Tıp Fakültesi

location_facility: University of Texas Health Science Center of Texas

location_facility: Universitätsklinikum Heidelberg / Zentrum für Kinder- und Jugendmedizin / Sektion für Neuropädiatrie & Stoffwechselmedizin

location_facility: The Hospital for Sick Children University of Toronto, Adult Clinic: The Fred A Litwin Family Centre in Genetic Medicine University Health Network & Mt. Sinai Hospital

location_facility: University of Colorado and the Children's Hospital CO

location_facility: UF College of Medicine, Department of Pediatrics Division of Genetics and Metabolism

location_facility: Copenhagen University Hospital, Rigshospitalet

location_facility: CENTRO HOSPITALAR UNIVERSITÁRIO LISBOA NORTE Hospital de Santa Maria,

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

location_facility: Division of Inherited Metabolic Diseases, Azienda Ospedaliera-Università Padova


title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva

acronym: Progress

summary: This Phase 2, Randomized, Double-Blind, Placebo-Controlled Study is intended to evaluate the Efficacy, Safety, and Tolerability and PK of INCB000928 administered to participants with a clinical diagnosis of fibrodysplasia ossificans progressiva (FOP).

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

lead_sponsor: Incyte Corporation

location_country:
Brazil
Spain
Italy
United Kingdom
Australia
Netherlands
Canada
Korea, Republic of
United States
Chile
China
France
Mexico
South Africa
Germany
Turkey

location_facility:
Investigative Site AU003
Investigative Site CL001
Investigative Site FR001
Investigative Site KR001
Investigative Site US003
Investigative Site TR001
Investigative Site FR002
Investigative Site MX001
Investigative Site CA001
Investigative Site GB001
Investigative Site AU001
Investigative Site ES001
Investigative Site BR001
Investigative Site DE001
Investigative Site CN001
Investigative Site ZA001
Investigative Site US002
Investigative Site IT002
Investigative Site NL001
Investigative Site US004


title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of ISIS 678354 Administered Subcutaneously to Patients With Severe Hypertriglyceridemia

summary: The purpose of the study is to evaluate the efficacy of olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.

location_country:
Poland
Portugal
Spain
Italy
United Kingdom
Australia
New Zealand
Canada
Sweden
Hungary
United States
France
South Africa
Netherlands
Bulgaria
Czechia

location_facility: Institut de recherches cliniques de Montréal (IRCM)

location_facility: TREAD Research - Department of Cardiology

location_facility: Washington University School of Medicine

location_facility: Hospital Virgen del Rocio Edificio Centro de Diagnostico y Tratamiento (CDT)

location_facility: Novant Health Cancer Institute Elizabeth Investigational Drug Services

location_facility: Instytut Centrum Zdrowia Matki Polki Klinika

location_facility: West Virginia University Heart and Vascular Institute

location_facility: A.O. Policlinico Universitario P. Giaccone

location_facility: Institut d'Investigació i Innovació Parc Taulí (I3PT) Departament Medicina Universitat

location_facility: Hospital Egas Moniz - Centro Hospitalar de Lisboa Ocidental

location_facility: UMHAT Prof. Dr. Stoyan Kirkovich AD, Clinic of Cardiology

location_facility: Debreceni Egyetem Klinikai Kozpont Belgyogyaszat A epulet

location_facility: Clinique des Maladies Lipidiques de Quebec Inc.

location_facility: Medical Center Smolyan Clinical Research OOD

location_facility: Dr. Heart Pty Ltd at Cholesterol Care Australia

location_facility: University of Michigan Hospital and Health Systems/ Dept. of Pharmacy-Research Pharmacy

location_facility: Diabetes/Lipid Management & Research Center


title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Magrolimab Versus Placebo in Combination With Venetoclax and Azacitidine in Newly Diagnosed, Previously Untreated Patients With Acute Myeloid Leukemia Who Are Ineligible for Intensive Chemotherapy

summary: The primary objectives of this study are to compare the efficacy of magrolimab + venetoclax + azacitidine versus placebo + venetoclax + azacitidine in participants with previously untreated acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy.

lead_sponsor: Gilead Sciences

location_country:
Poland
Taiwan
Hong Kong
Spain
United Kingdom
Austria
Australia
Hungary
Korea, Republic of
United States
France
Belgium

location_facility: Oxford University Hospital NHS Foundation Trust, Churchill Hospital

location_facility: Hospital Regional Universitario de Malaga

location_facility: Semmelweis Egyetem Belgyogyaszati es Hematologiai Klinika

location_facility: St George Hospital, Clinical Research Unit Haemotology, Division of Cancer Services

location_facility: Hospital Universitario Quironsalud Madrid

location_facility: Ziekenhuis Netwerk Antwerp (ZNA) - Stuivenberg

location_facility: The Catholic University of Korea Seoul Saint Mary's Hospital

location_facility: University of Arkansas for Medical Sciences

location_facility: Centrum Onkologii Ziemi Lubelskiej im. Sw. Jana z Dukli, Oddzial Hematologiczny

location_facility: Hospital General Universitario Gregorio Maranon

location_facility: Complejo Hospitalario Universitario de Santiago de Compostela

location_facility: University of Maryland, Greenebaum Comprehensive Cancer Center

location_facility: Szpital Wojewodozki w Opolu Sp. z 0.0. Oddzial Klinixzny Hematologii, Onkologii Hematologicznej i Chorob Wemnetrznych

location_facility: Monash University, Eastern Health-Box Hill Hospital

location_facility: Institut Universitaire du Cancer de Toulouse (IUCT)-Oncopole

location_facility: MidAmerica Division, Inc., c/o Research Medical Center


title: Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial to Evaluate the Efficacy and Safety of 3 Dose Levels of KVD824, an Oral Plasma Kallikrein Inhibitor, for Long-Term Prophylactic Treatment of Hereditary Angioedema Type I or II

summary: A study to assess whether different doses of KVD824 are effective in preventing attacks of Hereditary Angiodedema Type I or Type II.

status: Terminated

primary_purpose: Prevention

location_country:
United Kingdom
Italy
Australia
New Zealand
Canada
Hungary
North Macedonia
Puerto Rico
United States
France
Germany
Bulgaria
Czechia


title: A Phase 2, Single Arm Study of Mirvetuximab Soravtansine in Recurrent Platinum-Sensitive, High-Grade Epithelial Ovarian, Primary Peritoneal, or Fallopian Tube Cancers With High Folate Receptor-Alpha Expression (PICCOLO)

summary: PICCOLO (IMGN853-0419) is a Phase 2 multicenter, open label study designed to evaluate the safety and efficacy of Mirvetuximab Soravtansine in participants with platinum-sensitive ovarian, primary peritoneal or fallopian tube cancers with high folate receptor-alpha (FRα) expression.

lead_sponsor: ImmunoGen, Inc.

location_country:
Spain
Ireland
Italy
Australia
Canada
United States
France
Belgium

location_facility: Zangmeister Cancer Center / Sarah Cannon Research Institute

location_facility: Washington University School of Medicine

location_facility: Women & Infants Hospital of Rhode Island Oncology Research

location_facility: Fondazione Policlinico Universitario A. Gemelli IRCCS

location_facility: Princess Margaret Cancer Centre - University Health Network

location_facility: Complejo Hospitalario Provincial de Castellón

location_facility: UCLA-JCCC Dept of OBGYN - Women's Health Clinical Research Unit

location_facility: Burnside War Memorial Hospital - The Brian Fricker Oncology Centre

location_facility: Alaska Women's Cancer Care/Providence Alaska Medical Center

location_facility: Florida Cancer Specialist North Division

location_facility: Tennessee Oncology / Sarah Cannon Research Institute

location_facility: Institut Català d'Oncologia - Hospital Universitari Germans Trias i Pujol, Unidad de Investigación Clínica, Servicio de Oncología, Institut Josep Carreres (IJC, 1ª planta)

location_facility: University of Colorado School of Medicine

location_facility: Azienda Unita Sanitaria Locale di Ravenna

location_facility: Institut de Cancerologie Strabsourg Europe Unité de recherche clinique

location_facility: Hospital MD Anderson Cancer Center Madrid

location_facility: Cleveland Clinic Fairview Hospital-Moll Cancer Center

location_facility: Willamette Valley Cancer Institute and Research Center

location_facility: The University of Alabama at Birmingham - Division of Gynecology Oncology O'Neal Comprehensive Cancer Center


title: A Phase 2, Two-part, Placebo-controlled, Parallel-group, Double-blind Study to Assess the Efficacy and Safety of 2 Dosage Regimens of Oral IPN60130 for the Treatment of Fibrodysplasia Ossificans Progressiva in Male and Female Participants 5 Years of Age and Older.

acronym: FALKON

summary:
Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by the presence of bone in soft tissue where bone normally does not exist, known as Heterotopic Ossification (HO). It is often associated with painful, recurrent episodes of soft tissue swelling (flare-ups) that lead to abnormal stiffening and immobility (ankyloses) of major joints with cumulative and irreversible loss of movement and disability.

This study will evaluate the efficacy of 2 dosing regimens of IPN60130 in inhibiting new HO volume compared with placebo (a dummy treatment) in adult and paediatric participants with FOP. It will be assessed by a scan (provides internal images of the body) called low dose Whole Body Computed Tomography (WBCT), excluding head.

Adults and participants 15 years of age or older are also eligible for a sub study to evaluate HO lesions assessed by another type of scan, Fluorine-18-labelled natrium fluoride Positron Emission Tomography-Computed Tomography ([18F]NaF PET-CT ).

ipd_sharing_description: Where patient data can be anonymised, Ipsen will share all individual participant data that underlie the results reported in the published journal article with qualified researchers who provide a valid research question. Study documents, such as the study protocol and clinical study report, are not always available.

lead_sponsor: Clementia Pharmaceuticals Inc.

location_country:
Brazil
Spain
Italy
United Kingdom
Australia
Argentina
Canada
Korea, Republic of
United States
France
China
Mexico
Japan
Sweden

location_facility: University of Alberta, Alberta Health Services (AHS)

location_facility: University of California San Francisco (UCSF)

location_facility: University Health Network (UHN), Toronto General Hospital (TGH)

location_facility: Groupe Hospitalier Necker Enfants Malades

location_facility: The Perelman School of Medicine - The University of Pennsylvania

location_facility: Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Istituto Ortopedico Rizzoli (Rizzoli Orthopedic Institute)

location_facility: Hospital Universitario Y Politecnico La Femerge

location_facility: Children's Hospital Capital Institute of Pediatrics (CIP)

location_facility: Hospital Regional de Alta Especialidad del Bajio


title: An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT-EXT)

acronym: ASSERT-EXT

summary: An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT)

status: Active, not recruiting

lead_sponsor: Albireo

location_country:
Poland
United Kingdom
Italy
Netherlands
United States
France
Malaysia
Turkey
Belgium
Germany

location_facility: The Childrens Hospital at Montefiore Albert Einstein School of Medicine

location_facility: Istanbul University Istanbul Medical Faculty Hospital

location_facility: Antenne pediatrique du CIC-Hopital Jeanne De Flandre

location_facility: Cliniques Universitaires Saint-Luc Bruxelles

location_facility: Universitatsklinik fur Kinder-und Jugendmedizin Tubingen


title: A Phase 3b, Randomized, Double-blind, Placebo-controlled, 24-week Study to Assess the Efficacy and Safety of Fezolinetant in Menopausal Women Suffering From Moderate to Severe Vasomotor Symptoms (Hot Flashes) and Considered Unsuitable for Hormone Replacement Therapy

acronym: Daylight

summary:
This study is for women in menopause who have moderate to severe hot flashes. It is for women who are unable to use hormone replacement therapy (HRT). Menopause, a normal part of life, is the time after a woman's last period. Hot flashes often occur during menopause. They can disrupt a woman's daily life.

The study medicines (also called investigational products, or IP) are tablets of fezolinetant or placebo. An investigational product means that the product is not yet licensed. In this study, a placebo is a dummy treatment that looks like fezolinetant but does not have any medicine in it. The study will compare fezolinetant with the placebo to learn if fezolinetant reduces the number and severity of hot flashes.

Women that want to take part in the study will be given an electronic handheld device with an app to track their hot flashes. Some women may be able to use the app on their own smartphone. In the last 10 days before their next clinic visit, the women will record information about their hot flashes. They can take part in the study if they have an average of 7 or more moderate to severe hot flashes each day. Women will be picked for 1 of 2 treatments (fezolinetant or placebo) by chance alone.

Women who take part in the study will take 2 tablets every day for 24 weeks. Treatment will be double-blinded. That means that the women in the study and the study doctors will not know who takes which of the study medicines (fezolinetant or placebo). The women will continue recording information about their hot flashes on the electronic device or their phone. They will also use another device to answer questions about how hot flashes affect their daily life.

During the study, the women will visit their study clinic several times for a check-up. This will happen during Weeks 2, 4, 8, 12, 16, 20, 24, and 27. Some women may be able to have home visits instead, from Week 2 to Week 20. At the check-up, they will be asked if they have any medical problems. Other checks will include vital signs (heart rate, temperature and blood pressure) and some blood samples taken for laboratory tests. At some check-ups, the women will have a physical exam. In Week 2 and Week 24, the women will have an ECG to check their heart rhythm. Women who have a uterus will also have a test called a transvaginal ultrasound. A probe is gently placed inside the vagina. Sound waves will create a picture of the organs in the pelvis. This will allow the study doctor to look more closely at the uterus and surrounding organs.

The last check-up (at Week 27) will be 3 weeks after they take their last tablets of study medicine (fezolinetant or placebo).

ipd_sharing_description: Access to anonymized individual participant level data collected during the study, in addition to study-related supporting documentation, is planned for studies conducted with approved product indications and formulations, as well as compounds terminated during development. Studies conducted with product indications or formulations that remain active in development are assessed after study completion to determine if Individual Participant Data can be shared. Conditions and exceptions are described under the Sponsor Specific Details for Astellas on www.clinicalstudydatarequest.com.

lead_sponsor: Astellas Pharma Global Development, Inc.

location_country:
Denmark
Poland
Spain
Italy
United Kingdom
Netherlands
Finland
Canada
Sweden
Hungary
France
Turkey
Belgium
Norway
Germany
Czechia

location_facility:
Site PL48004
Site DE49007
Site CA15008
Site DE49004
Site TR90001
Site NL31001
Site DK45005
Site FR33003
Site PL48010
Site CZ42004
Site PL48005
Site CZ42006
Site PL48017
Site ES34003
Site TR90003
Site DK45003
Site PL48007
Site ES34002
Site CZ42011
Site DE49002
Site CA15012
Site CZ42007
Site FI35801
Site PL48006
Site GB44004
Site IT39002
Site DE49006
Site NO47001
Site DE49005
Site CA15006
Site PL48011
Site CZ42008
Site HU36002
Site CZ42010
Site CA15002
Site GB44002
Site GB44007
Site PL48012
Site DE49008
Site SE46003
Site DK45002
Site CZ42002
Site CZ42003
Site TR90002
Site IT39006
Site CA15001
Site FI35803
Site PL48009
Site DK45004
Site CA15007
Site CA15010
Site PL48003
Site ES34001
Site SE46004
Site FR33001
Site CA15009
Site BE32005
Site TR90007
Site HU36004
Site CA15003
Site SE46002
Site TR90008
Site PL48013
Site HU36001
Site PL48001
Site GB44006
Site NL31004
Site GB44003
Site CA15011
Site CZ42009
Site CZ42005
Site BE32002
Site ES34005
Site CA15014
Site CA15005


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

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

lead_sponsor: Wave Life Sciences Ltd.

location_country:
Denmark
Poland
Spain
Italy
United Kingdom
Australia
Canada
France
Germany

location_facility: Institut du Cerveau et de la Moelle Epiniere

location_facility: Centre Hospitalier de l-Universite de Montreal

location_facility: Cardiff University, Schools of Medicine and Biosciences

location_facility: Royal Hospital for Children, Pharmacy Aseptic Unit

location_facility: Royal Devon and Exeter Hospital NHS Trust


title: A Phase 1b Study to Evaluate the Efficacy and Safety of JNJ-64251330, a Janus Kinase (JAK) Inhibitor, in Participants With Familial Adenomatous Polyposis

summary: The purpose of this study is to determine the effect of JNJ-64251330 in participants with Familial Adenomatous Polyposis (FAP) on colorectal polyp burden (sum of the polyp diameters).

lead_sponsor: Janssen Research & Development, LLC

location_country:
Spain
Netherlands
Korea, Republic of
United States
France
Puerto Rico
Germany

location_facility: University of Utah Huntsman Cancer Institute

location_facility: Wexner Medical Center at the Ohio State University

location_facility: Pan American Center for Oncology Trials LLC

location_facility: Severance Hospital, Yonsei University Health System

location_facility: Academisch Medisch Centrum Universiteit van Amsterdam

location_facility: University of Texas MD Anderson Cancer Center

location_facility: University of Pennsylvania - Perelman School of Medicine


title: Phase I/II, Open-label, Dose Escalation and Dose Expansion Study to Evaluate Safety, Pharmacokinetics, Pharmacodynamics and Efficacy of AZD2936 Anti-TIGIT/Anti-PD-1 Antibody in Participants With Advanced or Metastatic Non-small Cell Lung Cancer

summary: This is a Phase I/II study designed to evaluate if experimental anti-TIGIT/anti-PD-1 bispecific antibody, AZD2936 is safe, tolerable and efficacious in participants with Advanced or Metastatic Non-small Cell Lung Cancer.

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.

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


title: A Phase II Study Evaluating the Safety and Efficacy of Glofitamab in Combination With Rituximab (R) Plus Cyclophosphamide, Doxorubicin, Vincristine, and Prednisone (CHOP) in Circulating Tumor (ct)DNA High-Risk Patients With Untreated Diffuse Large B-Cell Lymphoma

summary: This Phase II, open-label, multicenter study will evaluate the safety, efficacy, and pharmacokinetics of glofitamab in combination with Rituximab, Cyclophosphamide, Doxorubicin, Vincristine, and Prednisone (R-CHOP) in individuals with circulating tumor DNA (ctDNA) high-risk diffuse large B-cell lymphoma (DLBCL), as the first line of treatment.

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:
Denmark
Poland
Spain
United States
France
Netherlands

location_facility: Aarhus Universitetshospital Skejby; Blodsygdomme

location_facility: Hopital Henri Mondor; Hematologie Clinique

location_facility: Hospital Clinic i Provincial de Barcelona; Hematology

location_facility: Centrum Onkologii Ziemi Lubelskiej im. św. Jana z Dukli

location_facility: Oddzial Kliniczny Hematologii SPZOZ MSWiA z Warminsko-Mazurskim Centrum Onkologii w Olsztynie

location_facility: Szpital Kliniczny im. Heliodora Święcickiego; Oddzial Hematologii i Transplantacji Szpiku

location_facility: Hospital General Universitario Gregorio Marañon; Servicio de Hematología

location_facility: Hospital Univ. 12 de Octubre; Servicio de Hematologia

location_facility: Uniwersyteckie Centrum Kliniczne, Klinika Hematologii i Transplantologii

location_facility: Uniwersytecki Szpital Kliniczny; Klinika Hematologii, Nowotworów Krwi i Transplantacji Szpiku

location_facility: City of Hope Comprehensive Cancer Center

location_facility: Washington University; Wash Uni. Sch. Of Med

location_facility: Hospital Clinico Universitario de Salamanca;Servicio de Hematologia


title: A Phase 1, Multicenter, Open-Label, Dose Escalation and Expansion Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Clinical Activity of Intravenously Administered FHD-609 in Subjects With Advanced Synovial Sarcoma or Advanced SMARCB1-Loss Tumors

summary: This Phase 1, multicenter, open-label, dose escalation and expansion study is designed to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of FHD-609 given intravenously in subjects with advanced synovial sarcoma or advanced SMARCB1-loss tumors.

lead_sponsor: Foghorn Therapeutics Inc.

location_country:
United States
Italy
Spain
France

location_facility:
Sarah Cannon Research Institute
Hospital Universitari Vall d'Hebron
City of Hope
Institut Gustave Roussy
Memorial Sloan Kettering Cancer Center
Istituto Nazionale dei Tumori
Centre Leon Berard
Fred Hutchinson Cancer Research Center
MD Anderson Cancer Center
Dana-Farber Cancer Institute
University of Miami Health System
Massachusetts General Hospital