title: A Phase II, Double-Blind, Randomised, Placebo-Controlled Study to Assess the Efficacy and Safety of Selumetinib (AZD6244; ARRY-142886) (Hyd-Sulfate) in Combination With Docetaxel, Compared With Placebo in Combination With Docetaxel, in Patients Receiving Second Line Treatment for Locally Advanced or Metastatic Non Small Cell Lung Cancer (Stage IIIB - IV)

summary: The purpose of this study is to treat patients with locally advanced or metastatic NSCLC with a combination therapy of selumetinib and two different doses of docetaxel 75mg/m2 or 60 mg/m2 vs placebo and compare how well each dose affects how their cancer responds. It will also help us to understand the tolerability profile of the different dosing regimens in these patients

ReferenceCitation: Soria JC, Fülöp A, Maciel C, Fischer JR, Girotto G, Lago S, Smit E, Ostoros G, Eberhardt WEE, Lishkovska P, Lovick S, Mariani G, McKeown A, Kilgour E, Smith P, Bowen K, Kohlmann A, Carlile DJ, Jänne PA. SELECT-2: a phase II, double-blind, randomized, placebo-controlled study to assess the efficacy of selumetinib plus docetaxel as a second-line treatment of patients with advanced or metastatic non-small-cell lung cancer. Ann Oncol. 2017 Dec 1;28(12):3028-3036. doi: 10.1093/annonc/mdx628.

ipd_sharing: Yes

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

location_country:
Poland
Brazil
Netherlands
Hungary
United States
France
Germany
Bulgaria


title: Prevalence of Fabry's Disease in a Population of Patients With Chronic Pain

acronym: DOUFABIS

summary: Fabry disease is a rare inherited metabolic disorder that predominantly affects heart, kidneys and nervous system. Fabry disease has been searched in series of patients presenting different isolated signs caused by the affection of one of these organs. Acroparesthesias and chronic crises of pain of different origins are reported in the large majority of patients during the progression of the disease. Moreover, this signs are frequently inaugurating the disease. The investigators have previously performed a preliminary single-center study which permitted to identify one female patient with Fabry disease in a series of 147 consecutive patients with chronic pain tested. The investigators now propose to confirm the results of our preliminary study. The investigators plan to evaluate the prevalence of Fabry disease in a series of 1000 patients suffering from chronic pains of undetermined aetiology and consecutively recruited.

study_start_date_type: Actual

status: Unknown status

study_completion_date_type: Anticipated

study_type: Interventional

primary_purpose: Diagnostic

enrollment_type: Anticipated

ipd_sharing: No

lead_sponsor: University Hospital, Bordeaux

location_facility: Cs douleur chronique GHSR, CHU Sud Réunion

location_facility: Centre Douleurs Chroniques, CHU de Bordeaux

location_facility: Centre d'évaluation et de traitement de la Douleur, Hôpital Pierre-Paul Ricquet

location_facility: CeRCa, Unité de Neuromyologie, CHU de Fort de France

location_facility: Service de médecine Polyvalent, CH de La Dracénie

location_facility: Centre Douleurs Chroniques, CH de la Côte Basque

location_facility: Service de Médecine de la Douleur et de Médecine Palliative, Hôpital Lariboisière-APHP

location_facility: Service de lutte contre la douleur et d'Anesthésie, CH Périgueux

location_facility: Pôle Anesthésie Consultation douleur, CHRU de Lille

location_facility: Centre de la Douleur, CHRU de Strasbourg

location_facility: Pole enfant - CETD Salengro, CHRU de Lille

location_facility: Service d'Explorations Fonctionnelles et Consultations Neurologiques, CH Lyon-Sud

location_facility: Unité Douleur et Soins Palliatifs, CH La Rochelle-Réaunis

location_facility: Centre d'Analgésie Pédiatrique, Equipe Régionale Ressource en Soins Palliatifs-Equipe " Enfant-Do ", CHU de Toulouse

location_facility: Centre de la douleur de l'Adulte et de l'Enfant, CHU de Grenoble

location_facility: Centre d'Evaluation et de Traitement de la Douleur, CHU de Nîmes

location_facility: Médecine - Consultations Douleur, CH de La Réole

intervention_type: Genetic

lead_sponsor_type: academique

status_simplified: Unknown


title: Long Term Prognostic of Neonatal Hypoxic Ischemic Encephalopathy in the Era of Neuroprotective Treatment With Hypothermia

acronym: LyTONEPAL

summary:
The primary objective is to evaluate neonatal characteristics, and biological and clinical investigations as predictive factors of death, or of severe and moderate neurodevelopmental disability at 3 years, in a large population-based cohort of full-term and late preterm neonates with moderate or severe HIE.

Contrary to most previous studies which have often analyzed the accuracy of one factor among all other clinical investigations, the investigators objective's is to seek a relevant combination of several factors among the following list:

Neonatal characteristics: gestational age and birthweight, maternal disease, acute intrapartum event, delivery mode, acidosis, neurological examination, place of birth and neonatal transfer
Laboratory investigations: pH, lactates and new biological markers as detailed below
Clinical investigations: aEEG, EEG, MRI, diffusion-weighted MRI

study_type: Observational

time_perspective: Prospective

ReferenceType: background

ReferenceCitation: Volpe JJ. Neonatal encephalopathy: an inadequate term for hypoxic-ischemic encephalopathy. Ann Neurol. 2012 Aug;72(2):156-66. doi: 10.1002/ana.23647.

ReferenceCitation: Use and abuse of the Apgar score. Committee on Fetus and Newborn, American Academy of Pediatrics, and Committee on Obstetric Practice, American College of Obstetricians and Gynecologists. Pediatrics. 1996 Jul;98(1):141-2.

ReferenceCitation: Fenichel GM. Hypoxic-ischemic encephalopathy in the newborn. Arch Neurol. 1983 May;40(5):261-6. Review.

ReferenceCitation: Levene ML, Kornberg J, Williams TH. The incidence and severity of post-asphyxial encephalopathy in full-term infants. Early Hum Dev. 1985 May;11(1):21-6.

ReferenceType: derived

ReferenceCitation: Debillon T, Sentilhes L, Kayem G, Chevallier M, Zeitlin J, Baud O, Vilotitch A, Pierrat V, Guellec I, Ancel PY, Bednarek N, Ego A. Risk factors for unfavorable outcome at discharge of newborns with hypoxic-ischemic encephalopathy in the era of hypothermia. Pediatr Res. 2022 Oct 22. doi: 10.1038/s41390-022-02352-w. [Epub ahead of print]

ReferenceCitation: Beck J, Debillon T, Guellec I, Vilotitch A, Loron G, Bednarek N, Ancel PY, Pierrat V, Ego A. Healthcare organizational factors associated with delayed therapeutic hypothermia in neonatal hypoxic-ischemic encephalopathy: the LyTONEPAL cohort. Eur J Pediatr. 2022 Oct 21. doi: 10.1007/s00431-022-04666-7. [Epub ahead of print]

ReferenceCitation: Beck J, Bednarek N, Pierrat V, Vilotitch A, Loron G, Alison M, Guellec I, Hertz-Pannier L, de Launay C, Ego A, Vo-Van P, Ancel PY, Debillon T. Cerebral injuries in neonatal encephalopathy treated with hypothermia: French LyTONEPAL cohort. Pediatr Res. 2022 Sep;92(3):880-887. doi: 10.1038/s41390-021-01846-3. Epub 2021 Nov 20.

ReferenceCitation: Debillon T, Bednarek N, Ego A; LyTONEPAL Writing Group. LyTONEPAL: long term outcome of neonatal hypoxic encephalopathy in the era of neuroprotective treatment with hypothermia: a French population-based cohort. BMC Pediatr. 2018 Aug 1;18(1):255. doi: 10.1186/s12887-018-1232-6.

lead_sponsor: University Hospital, Grenoble

location_facility:
Chu Dijon
Chu Marseille
CHU St Denis
Chu Amiens
Chu Grenoble
Chu Strasbourg
Chu Reims
CHU CAEN
CHU St Pierre
Chu Besancon
Chu Brest
Chru Lille
Chu Toulouse
Chi Creteil
Chu Limoges
Chu La Miletrie
CHU Clermond-Ferrand
CHU FORT de France
Chu Bordeaux
CHU Montpellier
Chu Rouen
Chu Tours


title: Comparison of Subclavian, Femoral and Internal Jugular Venous Catheterization in Term of Complications in the Intensive Care Unit: a Randomized Controlled Trial

summary:
Central venous catheters are needed in the critical care setting to administer drugs. Three sites are available to gain vascular access: subclavian, internal jugular and femoral. Each site has complications, but there is no randomized controlled study which compared the 3 sites.

The investigators hypothesis is that subclavian catheterization reduces the risk of major complications compared to internal jugular or femoral.

status: Completed

study_completion_date_type: Actual

design_allocation: Randomized

primary_purpose: Prevention

enrollment_type: Actual

ReferenceType: result

ReferenceCitation: Parienti JJ, Mongardon N, Mégarbane B, Mira JP, Kalfon P, Gros A, Marqué S, Thuong M, Pottier V, Ramakers M, Savary B, Seguin A, Valette X, Terzi N, Sauneuf B, Cattoir V, Mermel LA, du Cheyron D; 3SITES Study Group. Intravascular Complications of Central Venous Catheterization by Insertion Site. N Engl J Med. 2015 Sep 24;373(13):1220-9. doi: 10.1056/NEJMoa1500964.

ReferenceCitation: Pages J, Hazera P, Mégarbane B, du Cheyron D, Thuong M, Dutheil JJ, Valette X, Fournel F, Mermel LA, Mira JP, Daubin C, Parienti JJ; 3SITES Study Group. Comparison of alcoholic chlorhexidine and povidone-iodine cutaneous antiseptics for the prevention of central venous catheter-related infection: a cohort and quasi-experimental multicenter study. Intensive Care Med. 2016 Sep;42(9):1418-26. doi: 10.1007/s00134-016-4406-4. Epub 2016 Jun 16.

ReferenceCitation: Iachkine J, Buetti N, de Grooth HJ, Briant AR, Mimoz O, Mégarbane B, Mira JP, Ruckly S, Souweine B, du Cheyron D, Mermel LA, Timsit JF, Parienti JJ. Development and validation of a multivariable prediction model of central venous catheter-tip colonization in a cohort of five randomized trials. Crit Care. 2022 Jul 7;26(1):205. doi: 10.1186/s13054-022-04078-x.

lead_sponsor: University Hospital, Caen

location_facility: Polyvalent Intensive Care Unit, Mémorial France Etats-Unis

location_facility: Médical Intensive Care Unit, Lariboisière, AP-HP

location_facility: Medical Intensive Care Unit, Cochin, AP-HP

intervention_type: Procedure

status_simplified: Completed


type: Other Identifier

title: Identification of Genetic Factors Predisposing to Dysglobulinemia

acronym: PreFamDys

summary:
Multiple Myeloma (MM) is a malignant proliferation of monoclonal plasma cells. Myeloma accounts for almost 14% of all hematologic cancers and is essentially incurable. Myeloma commonly evolves from a precursor disease, Monoclonal gammopathy of undetermined significance (MGUS). Despite intensive study, the etiology of MGUS and myeloma are unknown and no lifestyle or environmental exposure factors have been identified that are consistently linked to increased risk of MM, MGUS or the transition between the two.

The overall goal is to identify risk genes for dysglobulinemia, and more specifically Multiple Myeloma. This will involve the conservation of cells in a bank and genetic sequencing on samples obtained from families with at least two cases of dysglobulinemia. Material used for sequencing is likely to include fresh peripheral blood cells or lymphoblastoid lines established from peripheral blood lymphocytes of patients.

status: Recruiting

primary_purpose: Screening

lead_sponsor: Hospices Civils de Lyon

location_facility: Service d'Onco-hématologie, Hôpital Saint-Vincent GH-ICL

location_facility: Jean Paul FermandService Immuno-Hématologie Hôpital Saint Louis

location_facility: Hôpital St Antoine Service des maladies du Sang

location_facility: Département d'Oncogénitique - Centre Jean Perrin

location_facility: Service d'Oncologie Médical, Centre Azuréen de Canrérologie-1

location_facility: CHR-Metz-Thionville, Hôpital de Mercy, Service d'Hématologie

location_facility: CH William Morey, Service d'Hémato-Oncologie

location_facility: Service d'Hématologie clinique et Thérapie Cellulaire, CHU de BORDEAUX

location_facility: Hématolgie- Hôpital privé Cesson-Sévigné

location_facility: Département d'Oncogénétique, Centre Paoli Calmettes

location_facility: Centre Jean Bernard Clinique Victor Hugo

location_facility: Service d'Oncologie-Radiothérapie - CHU Pointe-à-Pitre/Abymes Guadeloupe

location_facility: CHU Groupe Hospitalier Sud Hématologie Clinique

location_facility: Service d'Hématologie, CHU de Versailles

location_facility: Département d'Hématologie à l'Institut de Cancérologie de la Loire

location_facility: Service d'Hématologie Clinique, CHU de Dijon

location_facility: Hôpital Saint-Eloi, Département d'Hématologie clinique

location_facility: Hôpital Robert Debré Service du Professeur A. DELMER Hématologie Clinique

location_facility: Service de Médecine Interne-Hématologie, CHU de Fort de France Fort de France

status_simplified: Ongoing


title: Histoplasmosis in the Guiana Shield and the French West Indies: Evaluating Prevalence in HIV + Patients With a Rapid Diagnostic Test Using an ELISA Capture Method for the Detection of Histoplasma Antigens in Blood and Urine.

acronym: EDIRAPHIS

summary: Histoplasma capsulatum var. capsulatum histoplasmosis is the leading cause of acquired immunodeficiency syndrome (AIDS) and death in French Guiana and probably in the Amazon. The diagnosis of this disease requires invasives procedures, laboratory performance, and delays up to several weeks. The Mycotic Diseases Branch of the Centers for Disease Control and Prevention (CDC) has established a rapid, sensitive and specific ELISA test for blood and urine samples that looks interesting in endemic areas, particularly in developing countries. The study aims to measure the proportion of HIV-infected patients hospitalized or in outpatient awaiting hospitalization for a suspicion of infectious syndrome whose serum and/or urinary antigen detection tests are positive for Histoplasma capsulatum var. capsulatum.

time_perspective: Cross-Sectional

lead_sponsor: ANRS, Emerging Infectious Diseases

location_country: Suriname France

location_facility: Academisch Ziekenhuis Paramaribo Hospital


acronym: AVC-MODE

summary:
The oropharyngeal junction participates in 3 interpenetrating functions: Swallowing, Breathing and Speech. In the context of ischemic or hemorrhagic stroke, these are altered, isolated or associated manner, causing major disorders and representing a source of handicap for patients.

Understanding the swallowing mechanisms is therefore a major challenge to guide the rehabilitation and improve the initial and long-term prognosis of patients with stroke.

The main objective of this study is to describe and modeling, thanks to a clinical, physiological and radiological knowledge base, swallowing, speech and breathing, in patients with stroke and in healthy subjects.

location_facility:
Hospital
University hospital

intervention_type: Other


title: Immediate vs. Delayed Stenting After Primary Percutaneous Reperfusion in ST Elevation Myocardial Infarction

acronym: PRIMACY

summary: The purpose of this research project is to improve how we treat heart attacks. The study will assess whether it might not be better to wait a few hours before implanting the coronary stent into the coronary artery rather than implanting it immediately, in order to allow the drugs to completely dissolve the clot. This might help to reduce the risks of the clot migrating and damaging the heart. By delaying coronary stent implantation, we hope to be able to reduce the size of the infarction and reduce the risk of suffering from heart failure following a heart attack.

primary_purpose: Treatment

lead_sponsor: Montreal Heart Institute

location_country: Canada France

location_facility: Centre Hospitalier Régional de Lanaudière

location_facility: Ch de Montpellier-Hôpital A. de Villneuve

lead_sponsor_type: industriel


title: Contribution of Preoperative Biological Data in Risk Assessment in Cardiac Surgery in Addition to the EuroSCORE

acronym: EuroSCOREbio

summary:
In cardiac surgery, the assessment of operative risk and quality of care is a major challenge for both patient, and surgical team. It is also important for health care decisionmakers to have predictive tools to compare alternative technics such as conventional cardiac surgery and interventional cardiology. Since 1998, the European System for Cardiac Operative Risk Evaluation (EuroSCORE), updated in 2012 (EuroSCORE II) is the most universally used system in this purpose. Its success is the result of a good balance between predictive capability and simplicity. It consists almost exclusively of clinical variables.

However, the objectivity and the predictive ability of some of those clinical items remain controversial, particularly those addressing severity of illness for high-risk patients. For instance, the degree of priority is submitted to the subjective assessment by the surgical team at the time of surgery. Objective data describing the severity of patients arriving in the operating room are still missing.

Many biomarkers are relevant in qualifying severity of syndromes: shock (PH, lactates, LDH), heart disease (N-terminal pro b-type natriuretic peptide (NTproBNP), troponin T and I), respiratory disorder (blood gaz analysis), liver insufficiency (TP, factor V), renal impairment (serum creatinine, creatinine clearance), inflammatory condition (fibrinogen, CRP), or the underlying medical condition such as diabetes (HbA1c, microalbuminuria) and nutritional status (albumin).

In ICU, many scores use biological data to measure, on daily basis, the severity of the patient status. Their routine use is simplified by applications available on smartphones. They are drawn into hospital information systems.

In cardiac surgery, some studies seem to demonstrate the measurement of some preoperative biological variables (eg NT Pro-BNP ...) in risk prediction. In terms of diabetes, HbA1c is of particular interest because it detects underlying diabetes if unknown (emergent situation) or reflects its poor control before surgery. This criterion could be more reliable than the simple information of patient treated by insulin (EuroSCORE II criterion).

Finally, the reinforcement of existing scores with biological variables is recommended by the group of recommendations in prognostic research strategy (PROGRESS 2014), rather than creating new scores ex nihilo.

The hypothesis is that adding biological data collected at the time of arrival of the patient in the operating room would better qualify the patients' severity condition and therefore increase the risk prediction of early mortality and severe morbidity after cardiac surgery. The purpose of this study is to test this hypothesis and especially test whether the biological data would increase the EuroSCORE II performance, by improving the prediction for high-risk patients.

lead_sponsor: University Hospital Center of Martinique

location_facility:
Hôpital Pitié-Salpêtrière (AP-HP)
CHU d'Angers
Hôpital Bichat (AP-HP)
CHU de Clermont-Ferrand
CHU de Dijon
CHU de Toulouse
CHU de La Réunion
CHU de Bordeaux
CHU de Limoges
CHU de Martinique


title: A Phase II Study Evaluating FOLFOX + Panitumumab According to a "Stop and go" Strategy With a Reintroduction Loop After Progression on Fluoropyrimidine as Maintenance Treatment, as the First Line in Patients With Metastatic Colorectal Adenocarcinoma Without a RAS Mutation

acronym: OPTIPRIME

summary: Single-arm, multi-centre phase II study The primary objective is to evaluate the time to failure of the strategy.

ipd_sharing: Undecided

lead_sponsor: Federation Francophone de Cancerologie Digestive

location_country: France Martinique

location_facility:
Hopital Sud
Groupe Hospitalier Pitie-Salpetriere
Centre Paul Strauss
Centre Hospitalier
Chru Hopital Claude Huriez 4Eme Est
Hopital Layne
Polyclinique de L'Ormeau
Hopital Jacques Monod
Ch de Meaux
Ch de Beauvais
CH
Ch St Joseph-St Luc
Polyclinique de Gentilly
Chu Cochin
Hopital Les Bonnettes
Chi de Frejus Saint-Raphael
Hopital Morvan - Chu
Ch de Cholet
Centre Hospitalier Schaffner
Clinique Claude Bernard
Ch Cote Basque
Centre Marie Curie
Clinique Pasteur
Polyclinique Saint Privat
Ch de Niort - Sce D'Oncologie
Clinique de L'Europe
Polyclinique de Courlancy
Clinique Ste Anne
Ch Du Mans
Hopital Prive D'Antony
Hopital Prive Du Confluent Sas
Chd Vendee
Cmco Cote D'Opale
Service de Medecine
Centre Hospitalier General
Clinique Trenel
Ch de Beziers Cedex
Centre Francois Baclesse
Chu de Fort de France
Chu D'Angers
Hopital Duchenne
Hopitaux Civils de Colmar
Ch Cahors
Polyclinique de Blois - 3Eme Etage
Hopital Europeen Marseille

intervention_type: Combination Product


title: Evaluation of the DIABEO System in Poorly Controlled DM1 or DM2 Patients Treated With a Basal-bolus Insulin Regimen

acronym: TELESAGE

summary:
The aim of the TELESAGE study is to demonstrate

the superiority of follow up with the DIABEO system (software alone) versus usual follow up in terms of improvement in HbA1c after 1 year in DM1 or DM2 patients treated with a basal-bolus insulin regimen.
the superiority of follow up with the DIABEO system (software + telemonitoring) compared to usual follow up in terms of improvement in HbA1c after 1 year in DM1 or DM2 patients treated with a basal-bolus insulin regimen.

The DIABEO software alone is an electronic diary system incorporating an immediate automatic aid to calculating the insulin dose according to the prescription given by the doctor, and a data processing, automated analysis message and secure internet data transfer system.

The DIABEO software added by paramedical telemonitoring is the electronic diary combined with remote paramedical monitoring of data (telemeonitoring)

ReferenceCitation: Jeandidier N, Chaillous L, Franc S, Benhamou PY, Schaepelynck P, Hanaire H, Catargi B, Farret A, Fontaine P, Guerci B, Reznik Y, Penfornis A, Borot S, Serusclat P, Kherbachi Y, D'Orsay G, Detournay B, Simon P, Charpentier G. DIABEO App Software and Telemedicine Versus Usual Follow-Up in the Treatment of Diabetic Patients: Protocol for the TELESAGE Randomized Controlled Trial. JMIR Res Protoc. 2018 Apr 19;7(4):e66. doi: 10.2196/resprot.9154.

lead_sponsor: Centre d'Etudes et de Recherche pour l'Intensification du Traitement du Diabète

location_facility: Centre hospitalier de Bigorre site La Gespe

intervention_type: Device


title: ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension

summary: As safety information pertaining to the long-term use of HU remains incomplete in spite of the first safety study (ESCORT-HU), an extension of the latter is proposed. ESCORT-HU Extension study aims at evaluating the long-term safety of Siklos® focusing on some questions regarding its safety when used in current practice in adults and paediatric patients treated with Siklos® and followed for up to 5 years. The study will focus on the following concerns : occurrence and incidence of malignancies, leg ulcers, male fertility impairment and serious unexpected AEs causally related to Siklos®.

lead_sponsor: ADDMEDICA SASA

location_country:
Greece
Italy
Guadeloupe
Martinique
France
French Guiana
Germany

location_facility: Marne-La-Vallée Hospital Center Children

location_facility: Hematology and oncology institute Children

location_facility: Hôpital RICOU - CHU Pointe-à-Pitre Abymes Children

location_facility: Centre Hospitalier de l'Ouest Guyanais Franck Joly Children

location_facility: Intercommunal Hospital Center of Créteil Adults and Children

location_facility: Centre Hospitalier de l'Ouest Guyanais Franck Joly Adults

location_facility: Clocheville Regional University Hospital Center Children

location_facility: Georges Pompidou European Hospital Adults

location_facility: Tours Regional University Hospital Center Adults

location_facility: Toulouse University Institute of cancer Adults

intervention_type: Drug


title: Multicenter Large-scale Validation Study of the Interest of Broadband Spectroscopy Analysis (Femto/Attosecond by Infrared Laser) on Liquid Biopsies in Breast Cancer Screening

summary: The purpose of this study is to validate the interest of broadband spectroscopy analysis (femto/attosecond by infrared laser) on liquid biopsies in breast cancer screening.

design_allocation: Non-Randomized

primary_purpose: Other

lead_sponsor: Centre Jean Perrin

location_facility:
Pôle Santé République
Hôpital Tenon
La Chataigneraie (Selimed 63)
Centre Jean PERRIN
Hôpital Saint-Louis
Hôpital La Pitié Salpêtrière


title: Safety Evaluation of a Combination of Brain Radiation Therapy and Bevacizumab (Avastin®) for Treatment of Brain Metastasis

summary:
This phase I pilot study aims to define the safety of a combined treatment of bevacizumab and whole brain radiation therapy for the treatment of patients with brain metastasis of solid tumors. If this therapeutic scheme confirms it's safety profile, the investigators can expect:

first, to allow that all patients can receive bevacizumab for their advanced and/or metastatic cancer if necessary, even in case of brain metastasis.
Secondly, if this trial confirm a synergic effect of the combination of angiogenesis inhibitors and brain radiotherapy for local control of brain metastasis, an improvement of the therapeutic results for these patients which have a poor hope of survival and for which none innovative approach is currently suggested.

Moreover, the investigators hope that the analysis of the different data of MRI evaluation - morphological and functional - will allow better definition of radiological evaluation of the therapeutic effect of angiogenesis inhibitors on brain metastasis.

ReferenceCitation: Lévy C, Allouache D, Lacroix J, Dugué AE, Supiot S, Campone M, Mahe M, Kichou S, Leheurteur M, Hanzen C, Dieras V, Kirova Y, Campana F, Le Rhun E, Gras L, Bachelot T, Sunyach MP, Hrab I, Geffrelot J, Gunzer K, Constans JM, Grellard JM, Clarisse B, Paoletti X. REBECA: a phase I study of bevacizumab and whole-brain radiation therapy for the treatment of brain metastasis from solid tumours. Ann Oncol. 2014 Dec;25(12):2351-2356. doi: 10.1093/annonc/mdu465. Epub 2014 Oct 1. Erratum in: Ann Oncol. 2015 Nov;26(11):2359.

lead_sponsor: Centre Francois Baclesse

location_facility:
Centre René Gauducheau
Centre Léon Bérard
Centre Oscar Lambret
Centre Henri Becquerel
Institut Curie
Centre François Baclesse


title: Non Interventional Study Evaluating Efficacy and Safety in a Cohort of Elderly Patients of First Line Therapy With Avastin ® Regimen for Metastatic Colorectal Cancer.

summary: This multicenter, prospective, observational study will evaluate the efficacy and safety of Avastin (bevacizumab) in first-line therapy in elderly patients with metastatic colorectal cancer. Data will be collected from eligible patients for24 months.

ReferenceCitation: François E, Mineur L, Deplanque G, Laplaige P, Smith D, Gourgou S, Tanang A, Ionescu-Goga M, Veerabudun K, Lelarge Y, Kim S, Rollot F. Efficacy and Safety of Bevacizumab Combined With First-Line Chemotherapy in Elderly (≥75 Years) Patients With Metastatic Colorectal Cancer: A Real-World Study. Clin Colorectal Cancer. 2020 Sep;19(3):e100-e109. doi: 10.1016/j.clcc.2020.02.009. Epub 2020 Apr 13.

lead_sponsor: Hoffmann-La Roche

location_facility: Polyclinique de Blois; Chimiotherapie Ambulatoire

location_facility: Clinique sainte marie; chimiotherapie ambulatoire

location_facility: Centre Hospitalier Departemental Les Oudairies

location_facility: CH Pitie Salpetriere; Hepathologie Gastro Enterologie

location_facility: Clinique D Occitanie; Chimiotherapie Ambulatoire

location_facility: Hopital Saint Antoine; Oncologie Medicale

location_facility: Memorial France Etats Unis; Hopital De Jour

location_facility: Hopital De La Croix Rousse;Hepato Gastro Entero

location_facility: Hopital Prive Du Vert Galant; Cons Specialisees

location_facility: Hopital Bichat Claude Bernard; Hepatologie Gastro Enterologie

location_facility: Centre Hospitalier de La Cote Basque; Oncologie

location_facility: Centre Hospitalier Saint Louis; Oncologie Medicale

location_facility: Hopital Victor Jousselin; Gastro Enterologie

location_facility: Pole Sante Republique;Oncologie Hematologie

location_facility: Clinique Du Parc; Centre de Radiotherapie Gray

location_facility: Gh Mutualiste Les Portes Du Sud; Gastro-Enterologie

location_facility: Hopital Prive Paul d Egine; Chimiotherapie Ambulatoire

location_facility: Polyclinique Du Languedoc; Chimiotherapie

location_facility: Centre D'Oncologie de Gentilly; Oncology

location_facility: Centre Hospitalier Fleyriat; Oncologie/Hematologie

location_facility: GH Paris Saint Joseph; Hopital De Jour Oncologie

location_facility: Hopital De Falconaja Furiani; Hopital De Jour

location_facility: C.H. Du Pays D'aix En Provence Service du Dr Blanc

location_facility: Clinique Chir De L Orangerie; Chimiotherapie

location_facility: Ch De Saint Quentin; Gastro Enterologie Endoscopie

location_facility: Clinique Claude Bernard; Onco Hematologie

location_facility: Centre Hospitalier de L'Agglomeration Montargeoise; Medecine Polyvalente A Orientation Mi & Cancero

location_facility: CHU Henri Mondor; Service d'Oncologie Medicale

location_facility: Chi Alpes Du Sud Site De Gap; Med Interne Et Polyvalente

location_facility: Clinique Mutualiste Bonneveine; Oncologie Medicale

location_facility: Centre Hospitalier Dr Duchenne; Hepato Gastro Enterologie

location_facility: Hopital Augustin Morvan; Federation De Cancerologie

location_facility: Hopital Alpha Sante De Hayange; Medecine B

location_facility: Ghi Le Raincy Montfermeil; Radiotherapie Oncologie

location_facility: Centre Georges Francois Leclerc; Oncologie 3

location_facility: Hopital Du Haut-Leveque; Gastro-Enterologie

location_facility: Clinique Anne D Artois; Gastro Enterologie

location_facility: Ch Arrondissement De Montreuil; Medecine 1

location_facility: Centre Azureen De Cancerologie; Cons externes

location_facility: Ch Lyon Sud; Gastro Secteur Jules Courmont

location_facility: Polyclinique Saint Come; Hopital De Jour Oncologie

location_facility: Chi Eure Seine D Evreux; Oncologie Conventionnelle

location_facility: Chi De Frejus Saint Raphael; Hopital De Jour

location_facility: Clinique Des 4 Pavillons; Chimiotherapie

location_facility: Polyclinique Saint Roch; Hop Jour Chimio Radiotherapie

location_facility: Hopital Saint Jean; Hospitalisation Oncologie

location_facility: Clinique Sainte Catherine; Hopital De Semaine

location_facility: Hopital Prive Jean Mermoz; Gastro Enterologie Hepatologie

location_facility: Ch De Brive La Gaillarde; Radiotherapie Oncologie

location_facility: Medipole De Savoie; Departement Oncologie

location_facility: Clinique Champeau Mediterranee; Radiotherapie Oncologie

location_facility: Polyclinique De La Louviere; Chimiotherapie

location_facility: Ctre Hosp St Joseph Et St Luc; Gastro Enterologie Endoscopie

location_facility: Fondation Maison Sante Bagatelle; Gastro Enterologie

location_facility: Clinique Du Docteur Maymard; Hopital De Jour Chimiotherapie

location_facility: Ctre Hosp Des Vals D Ardeche; Medecine C

location_facility: Hopital Cote De Nacre; Gastro Enterologie

location_facility: Ch De Longjumeau; Hopital De Jour Et Semaine

location_facility: Polyclin Bordeaux Nord Aquitaine; Gastro Enterologie

location_facility: Hopital Joseph Ducuing; Medecine Interne

location_facility: Polyclinique Francheville; MED CHIMIOTHERAPIE RADIOTHERAPIE

location_facility: Polyclinique Gentilly; CHIMIOTHERAPIE AMBULATOIRE

location_facility: Poly Parc Rambot La Provencale; Chimiotherapie Ambulatoire

location_facility: Hopital Jacques Monod; Hopital De Semaine Unite 53

location_facility: Hopital Edouard Herriot; Pavillon E Oncologie Medicale

location_facility: Clin Mut De Lyon Eugene Andre; Hopital De Jour 1er B

location_facility: Hopital Prive Villeneuve d Ascq;Chimiotherapie Radiotherapie

location_facility: Clinique Pasteur; Oncologie Medicale, Hematologie

location_facility: Hopital Yves Le Foll; Hematologie Oncologie

location_facility: Hopital Clinique Claude Bernard; Oncologie Medicale

location_facility: Clinique De L Europe; Radiotherapie Chimiotherapie

location_facility: Hopital Timone Adultes; Oncologie Digestive

location_facility: Ch De Montelimar; Gastro Enterologie Oncologie

location_facility: Clinique Saint Francois; Oncologie Medicale

location_facility: Centre Antoine Lacassagne; Hopital De Jour A2

location_facility: Ch Lyon Sud; Chir Visc Sect Jules Courmont

location_facility: Hop Europeen Georges Pompidou; Gastro Enterologie

location_facility: Clinique Sainte Marguerite; Oncologie Medicale

location_facility: Hopitaux Du Leman Site Thonon; Gastro Enterologie

location_facility: Hôpital Saint Joseph; Oncologie Medicale

location_facility: Ghi Le Raincy Montfermeil; Gastro Medecine Interne

location_facility: Hopital Prive Armand Brillard; Groupe Sein Paris Est

location_facility: Clinique Pasteur Lanroze; Chimiotherapie Radiotherapie

location_facility: Infirmerie Protestante; Endos Digestive Coelioscopie

location_facility: CH Prive Clairval; Chimiotherapie Ambulatoire


title: Hemocontrol's Effectiveness in a RAndomized Controlled Trial on the Reduction of Cardiovascular Long-term EventS

acronym: HERACLES

summary: It is hypothesized that a consistent use of the Hemocontrol TM biofeedback function improves long-term cardiovascular outcome, mediated by reduced hypertension due to fluid overload and by reduced incidence of intradialytic hypotensive episodes

lead_sponsor: IRCCS Azienda Ospedaliero-Universitaria di Bologna

location_country: Canada Italy France


title: GENetic & Immunologic Abnomalies in Systemic Lupus Erythematosus

acronym: GENIAL

summary:
Systemic Lupus Erythematosus (SLE) is a chronic autoimmune disease for which the aetiology includes genet-ic and environmental factors. It is rare in children as compared to adults. The severity may be related to greater involvement of genetic factors in children. The impact of genetics in the development of SLE is important, and the risk of recurrence in siblings evaluated by lambda S ratio is 30 in SLE, while it is 15 for type-1 diabetes and 8 rheumatoid arthritis, thereby indicating high impact of genetics in SLE.

Recently, the group of Professor Yanick Crow in Manchester and other teams has identified new forms of lupus Mendelian genetics. The TREX1 and genes involved in the SAMHD1 frostbite lupus.

Nearly 2 % of all adult subjects with SLE have a heterozygous mutation in the TREX1 gene, which therefore represents the first genetic cause of SLE. The team of Professor Crow also identified the ACP5 gene that is responsible for SLE associated with Spondylo-epiphyseal enchondro-epiphyseal dysplasia (syndromic lupus). Other groups have identified mutations in two genes encoding a DNAse (DNAse1 and DNAse1L3) responsible for familial monogenic forms of SLE. These new genes SLE were identified through research of germ-line mutations in cases of lupus syndromic or family. In collaboration with Professor Crow, we are currently undergoing characterization of a novel gene of SLE in a family and we have identified a second locus identified in another family. The identification of these genes provides a better understanding of the mechanisms regulating immune tolerance in humans. The frequency of these genetic forms is not known. There is very little data on the immunological phenotype of these patients.

This is a clinical study to investigate the genetic and immunological abnormalities associated with pediatric SLE. The aim are to:

study the genetics of pediatric SLE (or syndromic or family) and to search for mutations in the known genetic lupus or new genes in collaboration with Professor Yanick Crow.
study the lymphocyte subpopulations and serum cytokines in pediatric patients with SLE (or syndromic or family) in the large Rhône- Alpes- Auvergne area.

ReferenceCitation: Weill O, Decramer S, Malcus C, Kassai B, Rouvet I, Ginhoux T, Crow YJ, Rieux-Laucat F, Soulas-Sprauel P, Pagnier A, Koné-Paut I, Piram M, Galeotti C, Samaille C, Reumaux H, Lanteri A, Dubois SM, Lefebvre H, Burtey S, Maurier F, Carbasse A, Lemelle I, Meinzer U, Despert V, Flodrops H, Fabien N, Ranchin B, Hachulla E, Bader-Meunier B, Belot A. Familial and syndromic lupus share the same phenotype as other early-onset forms of lupus. Joint Bone Spine. 2017 Oct;84(5):589-593. doi: 10.1016/j.jbspin.2016.12.008. Epub 2016 Dec 28.

location_facility: Médecine Interne Adulte - Centre Hospitalier Lyon Sud

location_facility: Service de médecine interne - Hôpitaux privés de Metz

location_facility: Service de médecine infantile- Hôpital Nord

location_facility: Service de néphrologie, endocrinologie, maladie métabolique et hématologie bénigne pédiatriques - Hôpital Jeanne de Flandre

location_facility: Service de néphrologie - médecine interne - Hypertension pédiatrique - Hôpital des enfants

location_facility: Service de médecine interne - Hôpital Saint Antoine

location_facility: Service de pédiatrie générale - Hôpital Robert-Debré

location_facility: Service de Néphrologie - Hôpital Edouard Herriot

location_facility: Service de médecine interne - Centre de référence des maladies rares

location_facility: Service d'hématologie / oncologie pédiatrique - CHU

location_facility: Centre de néphrologie et de transplantation rénale - Hôpital de la conception

location_facility: édiatrie générale, urgences et maladies infectieuses, Hôpital Salengro

location_facility: Service pédiatrie grands enfants-adolescents - CHU Hôpital Sud

location_facility: Service d'immunologie et rhumatologie pédiatrique - Centre de référence de maladies rhumatologiques et inflammatoires rares en pédiatrie-Hôpital Necker-Enfants malades

location_facility: Service de pédiatrie - CHU Fort de France

location_facility: Service de Rhumatologie - Centre Hospitalier Lyon Sud

location_facility: Service de néphrologie pédiatrique - CHU de Nantes

location_facility: ervice d'urgence et post-urgences pédiatriques - CHU Arnaud de Villeneuve

location_facility: Service de Néphrologie et Rhumatologie Pédiatrique

location_facility: Service de Rhumatologie Pédiatrique - Hôpital de Bicêtre

location_facility: Service de Pédiatrie Générale - CHU Réunion


title: Prospective Study of Antenatal Diagnostic Criteria for Digestive Complications of Gastroschisis

acronym: LaparoDAN

summary:
Gastroschisis is a rare congenital malformation consisting in a right para-umbilical defect of the abdominal wall leading to an evisceration of the abdominal content. About 25% of the cases are associated with intestinal malformation, leading to an increase in peri-natal morbidity and mortality. No prospective study has been able to definitely describe predictive factors for complicated gastroschisis on prenatal ultrasound examinations.

The aim of the current study is to prospectively look for US prenatal features predictive of complicated gastroschisis, on a national multicentric cohort.

Patients will be included during the 22 weeks of amenorrhea (WA) ultrasound examination, and pre- and postnatal clinical data will be recorded up to the 6th months of the child.

Data will then be analysed and compared in the groups of simple or complicated gastroschisis, in order to define pre-natal prognostic factors, to correlate them with specific prognosis, and to might better determine specific strategies for better care of these patients.

ReferenceCitation: Geslin D, Clermidi P, Gatibelza ME, Boussion F, Saliou AH, Le Manac'h Dove G, Margaryan M, De Vries P, Sentilhes L, Levard G, Lardy H, Arnaud A, Leclair MD, Podevin G, Schmitt F. What prenatal ultrasound features are predictable of complex or vanishing gastroschisis? A retrospective study. Prenat Diagn. 2017 Feb;37(2):168-175. doi: 10.1002/pd.4984. Epub 2017 Jan 25.

ReferenceCitation: Kassa AM, Lilja HE. Predictors of postnatal outcome in neonates with gastroschisis. J Pediatr Surg. 2011 Nov;46(11):2108-14. doi: 10.1016/j.jpedsurg.2011.07.012.

ReferenceCitation: Kuleva M, Khen-Dunlop N, Dumez Y, Ville Y, Salomon LJ. Is complex gastroschisis predictable by prenatal ultrasound? BJOG. 2012 Jan;119(1):102-9. doi: 10.1111/j.1471-0528.2011.03183.x. Epub 2011 Oct 21.

lead_sponsor: University Hospital, Angers

location_facility:
University Hospital of Nancy
University Hospital of Brest
University Hospital of Saint-Etienne
University Hospital of Angers
University Hospital of Toulouse
University Hospital of Besançon
University Hospital of Strasbourg
University Hospital of Lyon
University Hospital of Bordeaux
University Hospital of Dijon
University Hospital of René Dubos
University Hospital of Lens
University Hospital of Grenoble
University Hospital of Le Mans
University Hospital of Montpellier
University Hospital of Limoges
University Hospital of Nantes
University Hospital of Fort de France
University Hospital of Tours
University Hospital of Nîmes
University Hospital of Marseille
University Hospital of Poitiers
University Hospital of Le Havre
University Hospital of Bicêtre
University Hospital of Trousseau
University Hospital of Nice
University Hospital of Reims
University Hospital of Robert Debré
University Hospital of Lille
University Hospital of Clermont-Ferrand
University Hospital of Amiens
University Hospital of Rennes
University Hospital of Necker
University Hospital of Orléans
University Hospital of Caen
University Hospital of Rouen


title: A Phase III, Open-label, Randomized Study of Osimertinib With or Without Platinum Plus Pemetrexed Chemo, as First-line Treatment in Patients With Epidermal Growth Factor Receptor (EGFR) Mutation Positive, Locally Advanced or Metastatic Non-small Cell Lung Cancer (FLAURA2).

summary:
The reason for the study is to find out if an experimental combination of an oral medication called osimertinib (TAGRISSO®) when used in combination with chemotherapy is more effective than giving osimertinib alone for the treatment of locally advanced or metastatic non-small cell lung cancer. Some lung cancers are due to mutations in the Deoxyribonucleic acid (DNA) which, if known, can help physicians decide the best treatment for their patients. One type of mutation can occur in the gene that produces a protein on the surface of cells called the Epidermal Growth Factor Receptor (EGFR).

Osimertinib is an Epidermal Growth Factor Receptor (EGFR) tyrosine kinase inhibitor (TKI) that targets Epidermal Growth Factor Receptor (EGFR) mutations. Unfortunately, despite the benefit observed for patients treated with osimertinib, the vast majority of cancers are expected to develop resistance to the drug over time. The exact reasons why resistance develops are not fully understood but based upon clinical research it is hoped that combining osimertinib with another type of anti-cancer therapy known as chemotherapy will delay the onset of resistance and the worsening of a patient's cancer.

In total the study aims to enroll approximately 586 patients, consisting of approximately 30 patients who will participate in a safety run-in component of the trial, and approximately 556 who will receive osimertinib alone or osimertinib in combination with chemotherapy in the main trial. In the main part of the trial there is a one in two chance of receiving osimertinib alone, and the treatment is decided at random by a computer.

The study involves a Screening Period, Treatment Period, and Follow up Period. Whilst receiving study medication, it is expected patients will attend, on average, approximately 15 visits over the first 12 months and then approximately 4 visits per year afterwards. Each visit will last about 2 to 6 hours depending on the arrangement of medical assessments by the study centre.

status: Active, not recruiting

ReferenceCitation: Planchard D, Feng PH, Karaseva N, Kim SW, Kim TM, Lee CK, Poltoratskiy A, Yanagitani N, Marshall R, Huang X, Howarth P, Jänne PA, Kobayashi K. Osimertinib plus platinum-pemetrexed in newly diagnosed epidermal growth factor receptor mutation-positive advanced/metastatic non-small-cell lung cancer: safety run-in results from the FLAURA2 study. ESMO Open. 2021 Oct;6(5):100271. doi: 10.1016/j.esmoop.2021.100271. Epub 2021 Sep 17.

ReferenceCitation: White MN, Piotrowska Z, Stirling K, Liu SV, Banwait MK, Cunanan K, Sequist LV, Wakelee HA, Hausrath D, Neal JW. Combining Osimertinib With Chemotherapy in EGFR-Mutant NSCLC at Progression. Clin Lung Cancer. 2021 May;22(3):201-209. doi: 10.1016/j.cllc.2021.01.010. Epub 2021 Jan 27.

ReferenceCitation: Asahina H, Tanaka K, Morita S, Maemondo M, Seike M, Okamoto I, Oizumi S, Kagamu H, Takahashi K, Kikuchi T, Isobe T, Sugio K, Kobayashi K. A Phase II Study of Osimertinib Combined With Platinum Plus Pemetrexed in Patients With EGFR-Mutated Advanced Non-Small-cell Lung Cancer: The OPAL Study (NEJ032C/LOGIK1801). Clin Lung Cancer. 2021 Mar;22(2):147-151. doi: 10.1016/j.cllc.2020.09.023. Epub 2020 Oct 16.

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.

lead_sponsor: AstraZeneca

location_country:
United Kingdom
United States
Peru
South Africa
Philippines
Japan
Czechia
Russian Federation
Australia
Argentina
India
Slovakia
Brazil
Canada
China
Taiwan
Thailand
Vietnam
Korea, Republic of
France
Chile


title: Evaluation of the Diagnostic Value of Level of Bone Marrow Tryptase in Adult Systemic Mastocytosis

acronym: EvaTryMS

summary:
The hypothesis of the study is that Bone Marrow Tryptase (MT) level is a diagnostic marker of Systemic Mastocytosis (SM). Determination of the bone marrow tryptase in Bone Marrow Aspirate (BMA) could be a new diagnostic criteria for systemic mastocytosis with sensitivity close to 100% and a low false negative rate. This new test could be useful to improve the ability to diagnose accurately systemic mastocytosis (in particular the indolent forms). Because of its limited invasiveness compared to bone marrow biopsy, it could also be considered as a test performed before bone marrow biopsy. Only patients with high bone marrow tryptase would then undergo bone marrow biopsy.

In the future and if validated by this study, bone marrow tryptase could be a useful marker of mast cell load and help to monitor the efficacy of treatment in systemic mastocytosis.

ReferenceCitation: Valent P, Horny HP, Escribano L, Longley BJ, Li CY, Schwartz LB, Marone G, Nuñez R, Akin C, Sotlar K, Sperr WR, Wolff K, Brunning RD, Parwaresch RM, Austen KF, Lennert K, Metcalfe DD, Vardiman JW, Bennett JM. Diagnostic criteria and classification of mastocytosis: a consensus proposal. Leuk Res. 2001 Jul;25(7):603-25. Review.

ReferenceCitation: Valent P, Akin C, Escribano L, Födinger M, Hartmann K, Brockow K, Castells M, Sperr WR, Kluin-Nelemans HC, Hamdy NA, Lortholary O, Robyn J, van Doormaal J, Sotlar K, Hauswirth AW, Arock M, Hermine O, Hellmann A, Triggiani M, Niedoszytko M, Schwartz LB, Orfao A, Horny HP, Metcalfe DD. Standards and standardization in mastocytosis: consensus statements on diagnostics, treatment recommendations and response criteria. Eur J Clin Invest. 2007 Jun;37(6):435-53. Review.

ReferenceCitation: Akin C, Valent P, Metcalfe DD. Mast cell activation syndrome: Proposed diagnostic criteria. J Allergy Clin Immunol. 2010 Dec;126(6):1099-104.e4. doi: 10.1016/j.jaci.2010.08.035. Epub 2010 Oct 28.

ReferenceCitation: Alvarez-Twose I, González de Olano D, Sánchez-Muñoz L, Matito A, Esteban-López MI, Vega A, Mateo MB, Alonso Díaz de Durana MD, de la Hoz B, Del Pozo Gil MD, Caballero T, Rosado A, Sánchez Matas I, Teodósio C, Jara-Acevedo M, Mollejo M, García-Montero A, Orfao A, Escribano L. Clinical, biological, and molecular characteristics of clonal mast cell disorders presenting with systemic mast cell activation symptoms. J Allergy Clin Immunol. 2010 Jun;125(6):1269-1278.e2. doi: 10.1016/j.jaci.2010.02.019.

ReferenceCitation: Barete S, Assous N, de Gennes C, Grandpeix C, Feger F, Palmerini F, Dubreuil P, Arock M, Roux C, Launay JM, Fraitag S, Canioni D, Billemont B, Suarez F, Lanternier F, Lortholary O, Hermine O, Francès C. Systemic mastocytosis and bone involvement in a cohort of 75 patients. Ann Rheum Dis. 2010 Oct;69(10):1838-41. doi: 10.1136/ard.2009.124511. Epub 2010 Jun 22.

ReferenceCitation: Paul C, Sans B, Suarez F, Casassus P, Barete S, Lanternier F, Grandpeix-Guyodo C, Dubreuil P, Palmérini F, Mansfield CD, Gineste P, Moussy A, Hermine O, Lortholary O. Masitinib for the treatment of systemic and cutaneous mastocytosis with handicap: a phase 2a study. Am J Hematol. 2010 Dec;85(12):921-5. doi: 10.1002/ajh.21894.

lead_sponsor: University Hospital, Toulouse

location_facility: CHU Bordeaux Hôpital Haut-Lévêque, service de dermatologie

location_facility: CHU Fort de France, service de dermatologie

location_facility: CHU Lyon Sud, service de médecine interne

location_facility: CHU Toulouse, Hôpital Larrey, service de dermatologie


title: STrategic Reperfusion in Elderly Patients Early After Myocardial Infarction

summary: In patients ≥ 60yrs with acute ST-elevation myocardial infarction randomised within 3 hours of onset of symptoms the efficacy and safety of a strategy of early fibrinolytic treatment with half-dose tenecteplase and additional antiplatelet therapy with a loading dose of 300 mg clopidogrel, aspirin and coupled with antithrombin therapy followed by catheterisation within 6-24 hours or rescue coronary intervention as required, will be compared to a strategy of primary PCI with a P2Y12 antagonist and antithrombin treatment according to local standards.

ReferenceCitation: Armstrong PW, Gershlick AH, Goldstein P, Wilcox R, Danays T, Lambert Y, Sulimov V, Rosell Ortiz F, Ostojic M, Welsh RC, Carvalho AC, Nanas J, Arntz HR, Halvorsen S, Huber K, Grajek S, Fresco C, Bluhmki E, Regelin A, Vandenberghe K, Bogaerts K, Van de Werf F; STREAM Investigative Team. Fibrinolysis or primary PCI in ST-segment elevation myocardial infarction. N Engl J Med. 2013 Apr 11;368(15):1379-87. doi: 10.1056/NEJMoa1301092. Epub 2013 Mar 10.

ReferenceCitation: Sinnaeve PR, Armstrong PW, Gershlick AH, Goldstein P, Wilcox R, Lambert Y, Danays T, Soulat L, Halvorsen S, Ortiz FR, Vandenberghe K, Regelin A, Bluhmki E, Bogaerts K, Van de Werf F; STREAM investigators. ST-segment-elevation myocardial infarction patients randomized to a pharmaco-invasive strategy or primary percutaneous coronary intervention: Strategic Reperfusion Early After Myocardial Infarction (STREAM) 1-year mortality follow-up. Circulation. 2014 Sep 30;130(14):1139-45. doi: 10.1161/CIRCULATIONAHA.114.009570. Epub 2014 Aug 26.

ReferenceCitation: Dianati Maleki N, Van de Werf F, Goldstein P, Adgey JA, Lambert Y, Sulimov V, Rosell-Ortiz F, Gershlick AH, Zheng Y, Westerhout CM, Armstrong PW. Aborted myocardial infarction in ST-elevation myocardial infarction: insights from the STrategic Reperfusion Early After Myocardial infarction trial. Heart. 2014 Oct;100(19):1543-9. doi: 10.1136/heartjnl-2014-306023. Epub 2014 Jun 10.

ReferenceCitation: Sinnaeve PR, Danays T, Bogaerts K, Van de Werf F, Armstrong PW. Drug Treatment of STEMI in the Elderly: Focus on Fibrinolytic Therapy and Insights from the STREAM Trial. Drugs Aging. 2016 Feb;33(2):109-18. doi: 10.1007/s40266-016-0345-6. Review.

lead_sponsor: KU Leuven

location_country:
Montenegro
Brazil
Serbia
Spain
Russian Federation
Australia
Canada
France
Chile
Mexico

location_facility: General Hospital Pancevo/Department of internal medicine - cardiology section

location_facility: General Hospital Danilo the First Cetinje

location_facility: Military Medical Academy, Clinic for Emergency Internal Medicine

location_facility: CH Louis Pradel - Hospices civils de Lyon

location_facility: Tomsk Regional State Autonomous Healthcare Institution Emergency Care Station

location_facility: Federal State Budgetary Scientific Inst "Tomsk Nat Research Med.Center of Russian Academy Sciences"

location_facility: General Hospital Jagodina/Intenal Medicine department

location_facility: Groupe Hospitalier Sud Ile de France - CH de Melun - Service SAMU 77

location_facility: General Hospital "Dr. Laza K. Lazarevic" Sabac, Internal medicine department

location_facility: General Hospital Vrsac/Cardiology department with coronary unit

location_facility: Clinical Center Kragujevac, Cardiology Clinic

location_facility: Hospital de Antequera, Unidad de Cuidados Intensivos

location_facility: Instituto Nacional de Cardiologia Ignacio Chavez

location_facility: State Budgetary Healthcare Institution of Tverskoy Region "Region Clinical Hospital"

location_facility: Opsta bolnica Vrbas, Cardiology Department

location_facility: Liverpool Hospital - Cardiology Department

location_facility: Institute for cardiovascular diseases Vojvodina - Sremska Kamenica, Cardiology Clinic

location_facility: Centro de Pesquisa São Lucas - Hospital E Maternidade Celso Pierro

location_facility: General Hospital Cuprija, Cardiology Department

location_facility: Federal State Budgetary Inst "Research Inst. for Complex Issues of Card. Diseases"

location_facility: Clinical Center of Serbia, Cardiology Clinic

location_facility: State Budgetary Healthcare Inst. Kemerovo-Clinical Emergency Care Station

location_facility: Hospital Virgen de la Victoria, Unidad de Cuidados Intensivos

location_facility: Institute for cardiovascular diseases Dedinje, Cardiovascular research sector

location_facility: Hospital Comarcal Axarquia, Unidad de Cuidados Intensivos

location_facility: General Hospital "Sveti Luka" Smederevo, Dept of Internal Med - Cardiology Section

location_facility: Hospital Serrania Ronda, Unidad de Cuidados Intensivos

location_facility: Tver Region State Budgetary Healthcare Institution "Tver Emergency Station"


status: Terminated

title: A Phase 3, Randomized, Controlled Study Evaluating the Efficacy and Safety of Idelalisib (GS-1101) in Combination With Ofatumumab for Previously Treated Chronic Lymphocytic Leukemia

lead_sponsor: Gilead Sciences

type: EudraCT Number

source: euctr

ReferenceCitation: Jones JA, Robak T, Brown JR, Awan FT, Badoux X, Coutre S, Loscertales J, Taylor K, Vandenberghe E, Wach M, Wagner-Johnston N, Ysebaert L, Dreiling L, Dubowy R, Xing G, Flinn IW, Owen C. Efficacy and safety of idelalisib in combination with ofatumumab for previously treated chronic lymphocytic leukaemia: an open-label, randomised phase 3 trial. Lancet Haematol. 2017 Mar;4(3):e114-e126. doi: 10.1016/S2352-3026(17)30019-4.

ReferenceCitation: Jones J, Robak T, Wach M, Brown JR, Menter AR, Vandenberghe E, et al. Updated results of a phase 3 randomized, controlled study of idelalisib in combination with ofatumumab for previously treated chronic lymphocytic leukemia (CLL) [Poster 7515]. American Society of Clinical Oncology (ASCO) 52nd Annual Meeting; 2016 02 - 06 June; Chicago, IL.

ReferenceCitation: Jones JA, Wach M, Robak T, Brown JR, Menter AR, Vanderberghe E, et al. Results of a Phase 3 Randomized, Controlled Study Evaluating the Efficacy and Safety of Idelalisib (IDELA) in Combination with Ofatumumab (OFA) for Previously Treated Chronic Lymphocytic Leukemia (CLL) [Poster 7023]. American Society of Clinical Oncology (ASCO) 51st Annual Meeting; 2015 29 May - 02 June; Chicago, IL.

ReferenceCitation: Robak T, Jones J, Wach M, Brown JR, Menter AR, Vandenberghe E, et al. Updated results of a phase 3 randomized, controlled study of idelalisib in combination with ofatumumab for previously treated chronic lymphocytic leukemia (CLL) [Poster 213]. 21st Congress of the European Hematology Association (EHA); 2015 09-12 June; Copenhagen, Denmark.

ReferenceCitation: Robak T, Wach M, Jones J, Owen C, Brown J, Menter A, et al. Results Of A Phase 3 Randomized Controlled Study Evaluating The Efficacy And Safety Of Idelalisib (Idela) In Combination With Ofatumumab (Ofa) For Previously Treated Chronic Lymphocytic Leukemia (CLL) [Poster LB598]. 20th Congress of the European Hematology Association (EHA); 2015 11-14 June; Vienna, Austria.

ReferenceCitation: Flinn I, Kimby E, Cotter FE, Giles FJ, Janssens A, Pulczynski EJ, et al. A Phase 3, Randomized, Controlled Study Evaluating the Efficacy and Safety of Idelalisib (GS-1101) in Combination with Ofatumumab for Previously Treated Chronic Lymphocytic Leukemia (CLL) [Poster TPS7131]. American Society of Clinical Oncology (ASCO); 2013 May 31-June 4; Chicago, IL.

ReferenceCitation: Gordon MJ, Huang J, Chan RJ, Bhargava P, Danilov AV. Medical comorbidities in patients with chronic lymphocytic leukaemia treated with idelalisib: analysis of two large randomised clinical trials. Br J Haematol. 2021 Feb;192(4):720-728. doi: 10.1111/bjh.16879. Epub 2020 Jun 29.

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

location_country:
Denmark
Poland
Spain
Ireland
United Kingdom
Australia
Canada
United States
France
Belgium
Sweden

location_facility: Samodzielny Publiczny Szpital Kliniczny N1 Klinika

location_facility: Department of Haematology, Cancer and Haematology Centre, Churchill Hospital (Oxford University Hospitals)

location_facility: Wojewódzki Szpital Specjalistyczny im. Mikolaja Kopernika w Lodzi

location_facility: Centre Hospitalier Universitaire de Montréal

location_facility: Kaiser Permanente Vallejo Medical Center

location_facility: California Cancer Associates for Research and Excellence (CCARE)

location_facility: Centre Hospitalier Universitaire Hôpital Avicenne

location_facility: Center Hospitalier Universitaire de Bordeaux

location_facility: Center for Cancer and Blood Disorders, PC

location_facility: Hopital Regional De Sudbury Regional Hospital (HRSRH) - Regional Cancer Program (RCP)

location_facility: Haematology and Oncology Clinics of Australia at Mater

location_facility: Collegium Medicum Uniwersytetu Jagiellonskiego w K

location_facility: The Ohio State University Medical Center

location_facility: Wojewódzki Szpital Specjalistyczny im. Janusza Kor

summary: The primary objective of this study is to evaluate the effect of the addition of idelalisib to ofatumumab on progression-free survival (PFS) in participants with previously treated chronic lymphocytic leukemia (CLL).


title: Prevalence of Pressure Ulcers Among Critically Ill Patients and Factors Associated With Their Occurrence in the ICU

acronym: PRESSURE

summary: The prevalence of and risk factors for pressure ulcers (PUs) in critically ill patients have been poorly studied. The main objective of this study was to describe the prevalence of PUs in critically ill patients. Secondary objectives were to describe PUs, means of prevention of PUs, and factors associated with occurrence of PUs in the ICU.

time_perspective: Retrospective

lead_sponsor: French Society for Intensive Care

location_facility:
CHU Grenoble Alpes
Hopital Delafontaine
CHU Kremlin-Bicetre
CHU Robert Debré
HIA Percy
CHU Strasbourg
CHU Dijon Bourgogne
HIA Desgenettes Lyon
Hopital l'Archet
CHU Henri Mondor
Hopital Privé du Val d'Yerres
Hopital Jean Bernard
Hopital Européen Marseille
Cochin
CHU Réunion Saint Pierre
Hopital Ambroise Pare
Hopital Antoine Beclere
Hopital Notre-Dame de Bon Secours
GHIRM
Hopital St-Andre
Hopital Laennec
Institut Mutualiste Montsouris
GH Diaconnesses Croix Saint Simon
HIA Clermont Tonnerre
Hopital Saint Clair
Clinique de l'Union
Clinique des Cèdres
Groupe Hospitalier Sud Ile de France
Hopital Avicenne
HIA Béjin
CHU Nancy
Hopital Nord Franche Comté
CHU Marseille Hopital Nord
Groupe Hospitalier Edouard Herriot
Institut Gustave Roussy
Hopital Francois Quesnay
HEGP
CHU Lapeyronie
Hopital Privé du Vert Galan
CHU Pellegrin
Hopital St Louis
CHU Poitiers
Hopital de la Timone
CHU Necker Enfants Malades
Hopital Privé Claude Galien
Hopital Privé d'Antony
Hopital Mercy
Hopital Tenon
Hopital Raymond Poincare
CHU Nimes
Hôpital Saint-Esprit
CHU Nantes
HIA Robert Pique
Hopital Louis Mourier
Hopital Roger Salengro
CHU d'Angers
Grand Hopital de l'Est Francilien
Hopital Simone Veil
CHU Toulouse Rangueil
La Pitié Salpetriere


title: End Of Life in the Critically Ill patiEnt

acronym: EOLE

summary: The purpose of this multicentric study is to evaluate the perception of the quality of the end of life in intensive care units seen from the side of the caregivers, trough the CAESAR scale.

ReferenceCitation: SRLF Trial Group. End of life in the critically ill patient: evaluation of experience of end of life by caregivers (EOLE study). Ann Intensive Care. 2021 Nov 26;11(1):162. doi: 10.1186/s13613-021-00944-z. Erratum in: Ann Intensive Care. 2021 Dec 29;11(1):186.

location_facility: Centre Hospitalier Lucie et Raymond Aubrac

location_facility: Centre Hospitalier Communal des Alpes du Sud


title: Safety of Exposure to Natalizumab During Pregnancy

summary:
Multiple sclerosis (MS) preferentially affects young adults with a female predominance. MS is not associated with an increased risk of complications or abnormal pregnancy outcomes. Nevertheless, disease-modifying therapies can have a teratogenic effect. Discussions about discontinuation should be made with a view to or upon discovery of pregnancy, taking into account the risk of untreated relapses and the risk of toxicity to the fetus.

Natalizumab (NTZ) is a humanized anti-alpha4-integrin monoclonal antibody used as a treatment for highly active relapsing-remitting MS (RRMS). When it is stopped, there is frequent reactivation of the disease with possible relapses and a rebound effect could occur. At present, depending on the center, attitudes of neurologist may vary and 3 main scenarios can be observed: Pregnancy and postpartum under NTZ (group1), Pregnancy partially under NTZ (with or without immunomodulator (IM) supplementation, group 2), or NTZ stopped before pregnancy (with or without IM supplementation, group3).

The first part of the BABYZUMAB study, a retrospective study of Natalizumab exposure during pregnancy, analysed the comparison the clinical activity of the disease (annualized relapse rate) according to these 3 scenarios of NTZ treatment The investigators analyzed the annual relapse rate (ARR) during a two-year period (9 months before and 15 months after the beginning of the pregnancy) in 117 patients identified in the OFSEP database. The investigators showed that the risk of relapses was four times higher in Group 2 versus Group 1 (p=0,014) and six times higher in Group 3 versus Group 1 (p=0,001).

In the literature, there are few studies of newborns from NTZ-exposed pregnancies. No specific pattern of birth defects has been found, but mild to moderate transient thrombocytopenia and anemia have been reported in infants born to NTZ-exposed mothers in the third trimester of pregnancy.

lead_sponsor: University Hospital, Bordeaux

location_facility: CHU de Fort de France - service de neurologie

location_facility: CH de Saint-Denis - service de neurologie

location_facility: AP-HP - Hôpital Saint-Antoine - service de neurologie

location_facility: CHRU de Besançon - service de neurologie

location_facility: CH Sud Francilien - service de neurologie

location_facility: CHRU de Strasbourg - service de neurologie

location_facility: Fondation Rothschild - service de neurologie

location_facility: AP-HP - Hôpital de Créteil - service de neurologie

location_facility: CHU de Montpellier - service de neurologie

location_facility: AP-HP - Hôpital La Pitié Salpétrière - service de neurologie

intervention_type: Behavioral


title: Transthyretin Amyloidosis Outcomes Survey (THAOS): A Global, Multi-Center, Longitudinal, Observational Survey of Patients With Documented Transthyretin Gene Mutations or Wild-Type Transthyretin Amyloidosis.

acronym: THAOS

summary: THAOS is a global, multi-center, longitudinal observational survey open to all patients with transthyretin amyloidosis (ATTR), including ATTR-PN (polyneuropathy), ATTR-CM (cardiomyopathy) and wild-type ATTR-CM. It is open-ended with a minimum duration of 10 years. Patients will be followed as long as they are able to participate. The principal aims of this outcome survey are to better understand and characterize the natural history of the disease by studying a large and heterogenous patient population. Survey data may be used to develop new treatment guidelines and recommendations, and to inform and educate clinicians about the management of this disease.

ReferenceCitation: Coelho T, Conceição I, Waddington-Cruz M, Keohane D, Sultan MB, Chapman D, Amass L; THAOS investigators. A natural history analysis of asymptomatic TTR gene carriers as they develop symptomatic transthyretin amyloidosis in the Transthyretin Amyloidosis Outcomes Survey (THAOS). Amyloid. 2022 Jun 22:1-9. doi: 10.1080/13506129.2022.2070470. [Epub ahead of print]

ReferenceCitation: Dispenzieri A, Coelho T, Conceição I, Waddington-Cruz M, Wixner J, Kristen AV, Rapezzi C, Planté-Bordeneuve V, Gonzalez-Moreno J, Maurer MS, Grogan M, Chapman D, Amass L; THAOS investigators. Clinical and genetic profile of patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS): 14-year update. Orphanet J Rare Dis. 2022 Jun 18;17(1):236. doi: 10.1186/s13023-022-02359-w.

ReferenceCitation: Campbell CM, LoRusso S, Dispenzieri A, Kristen AV, Maurer MS, Rapezzi C, Lairez O, Drachman B, Garcia-Pavia P, Grogan M, Chapman D, Amass L; THAOS investigators. Sex Differences in Wild-Type Transthyretin Amyloidosis: An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Cardiol Ther. 2022 Sep;11(3):393-405. doi: 10.1007/s40119-022-00265-7. Epub 2022 May 18.

ReferenceCitation: Barroso FA, Coelho T, Dispenzieri A, Conceição I, Waddington-Cruz M, Wixner J, Maurer MS, Rapezzi C, Planté-Bordeneuve V, Kristen AV, González-Duarte A, Chapman D, Stewart M, Amass L; THAOS investigators. Characteristics of patients with autonomic dysfunction in the Transthyretin Amyloidosis Outcomes Survey (THAOS). Amyloid. 2022 Sep;29(3):175-183. doi: 10.1080/13506129.2022.2043270. Epub 2022 Apr 22.

ReferenceCitation: Nativi-Nicolau J, Siu A, Dispenzieri A, Maurer MS, Rapezzi C, Kristen AV, Garcia-Pavia P, LoRusso S, Waddington-Cruz M, Lairez O, Witteles R, Chapman D, Amass L, Grogan M; THAOS Investigators. Temporal Trends of Wild-Type Transthyretin Amyloid Cardiomyopathy in the Transthyretin Amyloidosis Outcomes Survey. JACC CardioOncol. 2021 Oct 19;3(4):537-546. doi: 10.1016/j.jaccao.2021.08.009. eCollection 2021 Oct.

ReferenceCitation: González-Moreno J, Losada-López I, Cisneros-Barroso E, Garcia-Pavia P, González-Costello J, Muñoz-Beamud F, Campistol JM, Fernandez-Torron R, Chapman D, Amass L. A Descriptive Analysis of ATTR Amyloidosis in Spain from the Transthyretin Amyloidosis Outcomes Survey. Neurol Ther. 2021 Dec;10(2):833-845. doi: 10.1007/s40120-021-00267-y. Epub 2021 Jul 30.

ReferenceCitation: Gentile L, Tournev I, Amass L, Chapman D, Mazzeo A; THAOS investigators. Phenotypic Differences of Glu89Gln Genotype in ATTR Amyloidosis From Endemic Loci: Update From THAOS. Cardiol Ther. 2021 Dec;10(2):481-490. doi: 10.1007/s40119-021-00226-6. Epub 2021 Jun 19.

ReferenceCitation: Waddington-Cruz M, Wixner J, Amass L, Kiszko J, Chapman D, Ando Y; THAOS investigators. Characteristics of Patients with Late- vs. Early-Onset Val30Met Transthyretin Amyloidosis from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Neurol Ther. 2021 Dec;10(2):753-766. doi: 10.1007/s40120-021-00258-z. Epub 2021 May 22.

ReferenceCitation: González-Duarte A, Barroso F, Mundayat R, Shapiro B. Blood pressure and orthostatic hypotension as measures of autonomic dysfunction in patients from the transthyretin amyloidosis outcomes survey (THAOS). Auton Neurosci. 2019 Dec;222:102590. doi: 10.1016/j.autneu.2019.102590. Epub 2019 Oct 23.

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

ReferenceCitation: Pinto MV, Pinto LF, Dias M, Rosa RS, Mundayat R, Pedrosa RC, Waddington-Cruz M. Late-onset hereditary ATTR V30M amyloidosis with polyneuropathy: Characterization of Brazilian subjects from the THAOS registry. J Neurol Sci. 2019 Aug 15;403:1-6. doi: 10.1016/j.jns.2019.05.030. Epub 2019 May 28.

ReferenceCitation: Damy T, Kristen AV, Suhr OB, Maurer MS, Planté-Bordeneuve V, Yu CR, Ong ML, Coelho T, Rapezzi C; THAOS Investigators. Transthyretin cardiac amyloidosis in continental Western Europe: an insight through the Transthyretin Amyloidosis Outcomes Survey (THAOS). Eur Heart J. 2019 Apr 1. pii: ehz173. doi: 10.1093/eurheartj/ehz173. [Epub ahead of print]

ReferenceCitation: Cruz MW, Pinto MV, Pinto LF, Gervais R, Dias M, Perez C, Mundayat R, Ong ML, Pedrosa RC, Foguel D. Baseline disease characteristics in Brazilian patients enrolled in Transthyretin Amyloidosis Outcome Survey (THAOS). Arq Neuropsiquiatr. 2019 Feb;77(2):96-100. doi: 10.1590/0004-282X20180156.

ReferenceCitation: Rubin J, Steidley DE, Carlsson M, Ong ML, Maurer MS. Myocardial Contraction Fraction by M-Mode Echocardiography Is Superior to Ejection Fraction in Predicting Mortality in Transthyretin Amyloidosis. J Card Fail. 2018 Aug;24(8):504-511. doi: 10.1016/j.cardfail.2018.07.001. Epub 2018 Aug 17.

ReferenceCitation: Mundayat R, Stewart M, Alvir J, Short S, Ong ML, Keohane D, Rill D, Sultan MB. Positive Effectiveness of Tafamidis in Delaying Disease Progression in Transthyretin Familial Amyloid Polyneuropathy up to 2 Years: An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Neurol Ther. 2018 Jun;7(1):87-101. doi: 10.1007/s40120-018-0097-9. Epub 2018 Apr 9.

ReferenceCitation: Kristen AV, Maurer MS, Rapezzi C, Mundayat R, Suhr OB, Damy T; THAOS investigators. Impact of genotype and phenotype on cardiac biomarkers in patients with transthyretin amyloidosis - Report from the Transthyretin Amyloidosis Outcome Survey (THAOS). PLoS One. 2017 Apr 6;12(4):e0173086. doi: 10.1371/journal.pone.0173086. eCollection 2017.

ReferenceCitation: Maurer MS, Hanna M, Grogan M, Dispenzieri A, Witteles R, Drachman B, Judge DP, Lenihan DJ, Gottlieb SS, Shah SJ, Steidley DE, Ventura H, Murali S, Silver MA, Jacoby D, Fedson S, Hummel SL, Kristen AV, Damy T, Planté-Bordeneuve V, Coelho T, Mundayat R, Suhr OB, Waddington Cruz M, Rapezzi C; THAOS Investigators. Genotype and Phenotype of Transthyretin Cardiac Amyloidosis: THAOS (Transthyretin Amyloid Outcome Survey). J Am Coll Cardiol. 2016 Jul 12;68(2):161-72. doi: 10.1016/j.jacc.2016.03.596.

ReferenceCitation: Damy T, Maurer MS, Rapezzi C, Planté-Bordeneuve V, Karayal ON, Mundayat R, Suhr OB, Kristen AV. Clinical, ECG and echocardiographic clues to the diagnosis of TTR-related cardiomyopathy. Open Heart. 2016 Feb 8;3(1):e000289. doi: 10.1136/openhrt-2015-000289. eCollection 2016.

ReferenceCitation: Coelho T, Maurer MS, Suhr OB. THAOS - The Transthyretin Amyloidosis Outcomes Survey: initial report on clinical manifestations in patients with hereditary and wild-type transthyretin amyloidosis. Curr Med Res Opin. 2013 Jan;29(1):63-76. doi: 10.1185/03007995.2012.754348. Epub 2012 Dec 13.

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

lead_sponsor: Pfizer

location_country:
Spain
Italy
United States
Romania
Belgium
Japan
Portugal
Argentina
Israel
Turkey
Mexico
Sweden
Malaysia
Cyprus
Brazil
Canada
Germany
Bulgaria
Denmark
Taiwan
Korea, Republic of
France
Netherlands

location_facility: Washington University School of Medicine

location_facility: Peters Township Health and Wellness Pavillion

location_facility: AOU Policlinico G. Martino - Messina - Dr. Vita

location_facility: University of California - San Francisco, UCSF Department of Neurology

location_facility: Smilow Cancer Hospital at Yale-New Haven

location_facility: Cardiology Clinic at Montefiore Hutchinson Campus

location_facility: Instituto Dante Pazzanese De Cardiologia

location_facility: The University of Utah Health Sciences Center

location_facility: Alexandrovska University Hospital Clinic of Neurology

location_facility: AOU Policlinico G. Martino - Messina - Comitato Etico Scientifico

location_facility: Centro per lo Studio e la Cura delle Amiloidosi Sistemiche - Pavia - Prof. Merlini

location_facility: Azienda Ospedaliero-Universitaria di Careggi

location_facility: Institutul De Cardiologie Prof. Dr. C. C. Iliescu Spitalului Fundeni

location_facility: Instituto Nacional de Ciencia Medicas y Nutricion Salvador Zubiran

location_facility: Universitätsmedizin der Johannes Gutenberg Universität Mainz KöR

location_facility: Montefiore Medical Center-Jack D. Weiler Hospital

location_facility: University Medical Center, Johannes Gutenberg-University Mainz

location_facility: Centro Hospitalar Lisboa Norte (CHLN) EPE - Hospital de Santa Maria

location_facility: University of Alberta Foothills Medical Centre

location_facility: VA Greater Los Angeles Healthcare System

location_facility: University of Pittsburgh Medical Center (UPMC)

location_facility: Vanderbilt University School of Medicine

location_facility: Comitato Etico Indipendente dell Azienda

location_facility: University of Maryland School of Medicine

location_facility: Karolinska University Hospital, Huddinge

location_facility: Hospital Italiano de Buenos Aires (HIBA)

location_facility: University of Chicago Medical Center IRB

location_facility: Universitatsklinikum Muenster - Transplant Hepatology

location_facility: Fondazione Policlinico Gemelli - Universita Cattolica del Sacro Cuore

location_facility: Samsung Medical Center, Sungkyunkwan University School of Medicine

location_facility: Comitato di Bioetica della Fondazione IRCCS Policlinico S. Matteo

location_facility: Penn Presbyterian Medical Center-University of Pennsylvania

location_facility: Cyprus Institute of Neurology and Genetics

location_facility: Toronto General Hospital-University Health Network

location_facility: Hospital de Rehabilitacion y Traumatologia Virgen de las Nieves

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

location_facility: Fondazione Toscana Gabriele Monasterio Per La Ricerca Medica E Di Sanita Pubblica (Ftgm)

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

location_facility: Istanbul University,Istanbul Faculty of Medicine,Department of Neurology

location_facility: Instituto De Investigaciones Medicas Dr Alfredo Lanari

location_facility: University of Pennsylvania - Perelman Center for Advanced Medicine

location_facility: Institut Clinic de Nefrologia i Urologia - ICNU, Hospital Clinic i Provincial de Barcelona


type: Registry Identifier

title: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of MK-4482 for the Prevention of COVID-19 (Laboratory-confirmed SARS-CoV-2 Infection With Symptoms) in Adults Residing With a Person With COVID-19

acronym: MOVe-AHEAD

summary: The purpose of the study is to assess if the study medication (molnupiravir, MK-4482) will prevent symptomatic coronavirus disease 2019 (COVID-19) in adults who live with someone with confirmed COVID-19 infection. This is a phase 3, multicenter, randomized, double-blind, placebo-controlled study; half of the study participants will receive molnupiravir twice daily by mouth and the other half will receive a placebo. The primary objectives of the study are to determine if molnupiravir prevents symptomatic COVID-19 disease and to evaluate its safety and tolerability. All participants who develop COVID-19 during the study are still eligible for any COVID-19 treatment recommended by their doctor.

lead_sponsor: Merck Sharp & Dohme LLC

location_country:
Spain
Ukraine
United States
Peru
Romania
Philippines
South Africa
Japan
Russian Federation
Argentina
Turkey
Mexico
Malaysia
Egypt
Kenya
Brazil
Guatemala
Hungary
Bulgaria
Dominican Republic
Thailand
France
Colombia

location_facility: Debreceni Egyetem Klinikai Kozpont- Infektologiai Klinika ( Site 0824)

location_facility: Social Medical Corporation Yuuaikai Yuuai Medical Center ( Site 1108)

location_facility: Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran ( Site 1308)

location_facility: Health Index Multi-Specialty and Lying -in Clinic ( Site 1504)

location_facility: Clinical Site Partners, LLC d/b/a CSP Miami ( Site 2425)

location_facility: LLC -Clinic of modern medicine of Doctor Bogorodskaya ( Site 1828)

location_facility: Mautalen Salud e Investigacion ( Site 0103)

location_facility: Fundaçao de Medicina Tropical Doutor Heitor Vieira Dourado ( Site 0303)

location_facility: Partners in Health Research and Development PHRD ( Site 1928)

location_facility: Asian Hospital and Medical Center ( Site 1503)

location_facility: LLC -Medical Center . Capital-Polis ( Site 1825)

location_facility: NPME -Central Clinical Hospital of Ivano-Frankivsk City Council ( Site 2204)

location_facility: National Hepatology and Tropical Medicine Research Institute ( Site 2930)

location_facility: HIV-NAT AIDS Research Centre ( Site 2804)

location_facility: Global Clinical Trial Centre ( Site 1904)

location_facility: Velocity Clinical Research, Hallandale Beach ( Site 2485)

location_facility: Hacettepe Universitesi Tip Fakultesi ( Site 2102)

location_facility: Hôpital Pitié - Salpêtrière ( Site 0501)

location_facility: Right To Care Research - Esizayo ( Site 1907)

location_facility: University of Nebraska Medical Center ( Site 2430)

location_facility: Indago Research and Health Center Inc ( Site 2412)

location_facility: The Lindner Center for Research and Education at The Christ Hospital ( Site 2517)

location_facility: Preferred Clinical Research ( Site 2470)

location_facility: The Golden Jubilee Medical Center ( Site 2800)

location_facility: Clinical Site Partners, LLC d/b/a CSP Miami ( Site 2508)

location_facility: PCNE Kharkiv City polyclinic 9 of the Kharkiv City Council ( Site 2201)

location_facility: Hospital Italiano de Buenos Aires ( Site 0102)

location_facility: Sancaktepe Prof Dr Ilhan Varank EAH-Prof Dr Feriha Oz Hastanesi ( Site 2113)

location_facility: Centro de Referencia Professor Helio Fraga - FIOCRUZ/RJ ( Site 0314)

location_facility: Maison de santé du Pays Neufchatelois ( Site 0507)

location_facility: Rush University Medical Center ( Site 2510)

location_facility: Oaxaca Site Management Organization S.C. ( Site 1304)

location_facility: Sakarya Universitesi Egitim ve Arastirma Hastanesi ( Site 2107)

location_facility: Allina Health Infectious Diseases Research Clinic ( Site 2507)

location_facility: Maison de Sante Universitaire La Providence ( Site 0506)

location_facility: Clínica Médica Especializada en Pediatría e Infectología Pediátrica - Dr. Mario Melgar ( Site 0702)

location_facility: Instituto Jalisciense de Metabolismo SC ( Site 1314)

location_facility: SPb SBHI City outpatient clinic 112 ( Site 1829)

location_facility: Hospital Universitario La Paz ( Site 2024)

location_facility: Chicago Clinical Research Institute Inc ( Site 2454)

location_facility: Unidad de Aislamiento del Centro de Obstetricia y Ginecologia ( Site 2650)

location_facility: SPb SBHI. City outpatient clinic No.44 ( Site 1830)

location_facility: Oncosite-Centro de Pesquisa Clinica em Oncologia ( Site 0300)

location_facility: Centro de Investigacion Medica Aguascalientes ( Site 1305)

location_facility: Enhancing Care Foundation-DICRS ( Site 1922)

location_facility: Hospital Universitari Germans Trias i Pujol ( Site 2010)

location_facility: Voronezh Regional Clinical Hospital #1 ( Site 1823)

location_facility: Faculdade de Medicina da Universidade Federal de Mato Grosso do Sul - UFMS ( Site 0305)

location_facility: Fundacion Centro de Investigacion Clinica CIC ( Site 0402)

location_facility: National Hospital Organization Okinawa National Hospital ( Site 1109)

location_facility: Instituto Neumologico del Oriente S.A. ( Site 0408)

location_facility: CHU de la Reunion - Groupe Hospitalier Sud ( Site 0508)

location_facility: Smorodintsev Research Institute of Influenza ( Site 1833)

location_facility: Policlínico Especializado en Neurología - Unid. Inv. Neurológica ( Site 1403)

location_facility: Centro de Investigacion y Avances Medicos Especializados -CIAME ( Site 1301)

location_facility: Municipal Noncommercial Enterprise Lviv 4th City Clinical Hospital ( Site 2200)

location_facility: Cherokee Nation WW Hastings Indian Hospital/Cherokee Nation Health Services ( Site 2459)

location_facility: SC Policlinica CCBR SRL. Bucuresti ( Site 1704)

location_facility: Medical Research Institute LLC. ( Site 1814)

location_facility: Hospital Universitario Infanta Leonor ( Site 2002)

location_facility: Jadestone Clinical Research, LLC ( Site 2530)

location_facility: Unidad de Diagnostico Cardiologico ( Site 0701)

location_facility: Non profit municipal enterprise City hospital student of Kharkiv city council ( Site 2229)

location_facility: Fundação Faculdade Regional de Medicina de São José do Rio Preto ( Site 0312)

location_facility: Centrul Medical Unirea SRL Constanta ( Site 1703)

location_facility: The Institute for Liver Health DBA Arizona Clinical Trials ( Site 2448)

location_facility: Köhler & Milstein Research S.A. de C.V. ( Site 1300)

location_facility: Quirino Memorial Medical Center ( Site 1501)

location_facility: DTHF Masiphumelele Clinical Research Site ( Site 1919)

location_facility: KEMRI-CMR-RCTP- Kenya Medical Research Institute Kisumu ( Site 1925)

location_facility: Instituto de Investigaciones Clínicas Mar del Plata ( Site 0105)

location_facility: Wits Health Consortium. Clinical HIV Research Unit ( Site 1923)

location_facility: Healthy Medical Center S.A.S ( Site 0413)

location_facility: MNPE Consultative-Diagnostic Centre of Desnyanskyi District of Kyiv ( Site 2205)

location_facility: Centro de Investigaciones Pediatricas ( Site 0703)

location_facility: Center Hospital of the National Center for Global Health and Medicine ( Site 1101)

location_facility: Obudai Egeszsegugyi Centrum - Zalaegerszeg ( Site 0819)

location_facility: ICARO Investigaciones en Medicina ( Site 1313)

location_facility: Mzansi Ethical Research Centre ( Site 1918)

location_facility: Ege Universitesi Tip Fakultesi Hastanesi ( Site 2105)

location_facility: National Center for allergies and chest ( Site 2940)

location_facility: Ankara Universitesi Ibni Sina Hastanesi ( Site 2101)

location_facility: Centrul Medical Unirea SRL IASI ( Site 1701)

location_facility: Asociacion Civil IMPACTA Salud y Educacion - Sede San Miguel ( Site 1410)

location_facility: Masri - CRC - Ain Shams University ( Site 2900)

location_facility: Limpopo Clinical Research Initiative ( Site 1901)

location_facility: Advanced Research For Health Improvement LLC ( Site 2436)

location_facility: Hospital Nossa Senhora das Gracas ( Site 0310)

location_facility: CDC Research Institute, LLC ( Site 2540)

location_facility: University of Missouri Hospital ( Site 2486)

location_facility: Clinical Research Trials of Florida ( Site 2402)

location_facility: Institute of Human Virology ( Site 2504)

location_facility: Obudai Egeszsegugyi Centrum - OEC ( Site 0821)

location_facility: Hospital Universitario Clinica San Rafael ( Site 0410)

location_facility: Medical Clinical Investigational Center of Medical Center Health Clinic ( Site 2210)

location_facility: Center for Clinical Research (CCR) KEMRI ( Site 1929)

location_facility: Istanbul Universitesi Istanbul Tip Fakultesi ( Site 2110)

location_facility: Medical Center Hera EOOD - Montana branch ( Site 3002)

location_facility: Balanced Life Healthcare Solutions/SKYCRNG ( Site 2539)

location_facility: Southern Clinical Research Associates ( Site 2542)

location_facility: Infectious Clinical Hospital # 1 ( Site 1834)

location_facility: Republican Clinical Infectious Hospital n.a. A.F. Agafonov ( Site 1802)

location_facility: Gazi Universitesi Tip Fakultesi Hastanesi ( Site 2106)

location_facility: Asociacion Civil por la Salud ( Site 1411)

location_facility: Klinik Kesihatan Masjid Tanah ( Site 2850)

location_facility: Caja de Compensacion Familiar CAFAM ( Site 0403)

location_facility: Crossroads Clinical Research LLC ( Site 2451)

location_facility: Hospital General de Acapulco ( Site 1319)

location_facility: Medical Center Hera Kyustendi EOOD ( Site 3003)

location_facility: Mercury Street Medical Group PLLC ( Site 2476)

location_facility: Chiba Aoba Municipal Hospital ( Site 1100)

location_facility: Centro Cientifico Asistencial Jose Luis Accini ( Site 0412)

location_facility: Smolensk State Medical Academy ( Site 1803)

location_facility: FSBI Central Hospital with Policlinics ( Site 1807)

location_facility: Fundacion Centro de Investigaciones Clinicas CARDIOMET ( Site 0409)

location_facility: Tribe Clinical Research, LLC ( Site 2409)

location_facility: The Institute for Liver Health DBA Arizona Clinical Trials ( Site 2484)

location_facility: Michigan Center of Medical Research ( Site 2445)

location_facility: Grupo Oroño Centro medico San Nicolas ( Site 0109)

location_facility: Michigan Center of Medical Research ( Site 2525)

location_facility: Excel Clinical Research, LLC ( Site 2404)

location_facility: University of California Davis ( Site 2497)

location_facility: Private Practice - Dr. Neyaseelan Gounden ( Site 1911)

location_facility: Basaksehir Cam ve Sakura City Hospital ( Site 2112)

location_facility: Municipal Enterprise Poltava Regional Clinical Infectious Hospital ( Site 2227)

location_facility: Instituto Medico de la Fundacion Estudios Clinicos ( Site 0106)

location_facility: Kyiv railway clinical hospital 2 of Branch Health center ( Site 2207)

location_facility: Regional Phthisiopulmonological Center ( Site 2203)

location_facility: Morales Vargas Centro de Investigacion, S.C. ( Site 1318)

location_facility: Prime Health and Wellness/SKYCRNG ( Site 2538)

location_facility: Limited Liability Company Medical center Healthy Happy ( Site 2225)

location_facility: Masri - CRC - Ain Shams University ( Site 2910)

location_facility: Ivano-Frankivsk Regional Clinical Infectious Diseases Hospital ( Site 2232)

location_facility: Instituto de Investigacion Nutricional - Anexo Huascar ( Site 1404)

location_facility: Limited liability company "Scientific research center Eco-safety" ( Site 1809)

location_facility: Private Clinic - Dr. Hugo Pezzarossi ( Site 0704)

location_facility: National Research Institute ( Site 2452)

location_facility: C.S. Vallcarca-Sant Gervasi ( Site 2000)

location_facility: Sanatorio de la Trinidad Mitre ( Site 0108)

location_facility: Amici Clinical Research LLC ( Site 2426)

location_facility: SPb SBHI City outpatient clinic 4 ( Site 1831)

location_facility: Hospital Raja Perempuan Zainab II ( Site 2851)

location_facility: CHRISTUS - LATAM HUB CENTER OF EXCELLENCE AND INNOVATION S.C. ( Site 1315)

location_facility: Velocity Clinical Research-Providence ( Site 2432)

location_facility: Accel Research Sites-DeLand Clinical Research Unit ( Site 2535)

location_facility: Valley Clinical Trials Inc. ( Site 2406)

location_facility: Future Innovative Treatments, LLC ( Site 2471)

location_facility: Centro Médico Universitario ( Site 1316)

location_facility: St.Petersburg Outpatient Clinic No. 109 ( Site 1818)

location_facility: DRC Gyogyszervizsgalo Kozpont Kft. ( Site 0801)

location_facility: Covid-19 Clinical Research Center (CCRC) ( Site 2421)

location_facility: Air Force Specialized Hospital Clinical Research Center ( Site 2920)

location_facility: Investigaciones Medicas en Salud - INMENSA ( Site 1401)

location_facility: Medipol Universite Hastanesi ( Site 2108)

location_facility: Odesa Regional Center of Socially Significant Diseases ( Site 2230)

location_facility: MNCE -Odesa regional clinical hospital of Odesa regional council ( Site 2226)

location_facility: Centrul Medical Unirea SRL Bucuresti ( Site 1706)

location_facility: Emerson Clinical Research Institute ( Site 2457)

location_facility: Centro de Referência e Treinamento DST/AIDS ( Site 0301)


title: One-year Follow-up of Iron in Basal Ganglia - R2*: a Biomarker of Parkinson's Disease Progression?

summary: The study of non-invasive and reliable biomarkers to track progression of Parkinson's disease (PD) is essential while disease-modifying treatments are being developed. Many clinical biological or imaging biomarkers have been tested but no "gold standard" has been found as of yet. Among these, Magnetic Resonance Imaging (MRI) relaxometry using R2* measurement (R2* = 1/T2*), which is a validated marker for estimating brain iron concentration, appears to be an attractive technique because its safety, rapidly measured in clinical conditions and its ease to ensure individual longitudinal follow-up. Current data of cross sectional studies of R2*, which have shown an iron increase in Substantia Nigra (SN), led to suppose that it could be a biomarker of disease vulnerability. Recently, the investigators have conducted the first longitudinal follow-up of R2* (1.5 T MRI), which showed a rapid R2* increase in both parts of the SN and in the caudal putamen. We propose, here, a multicenter prospective study of one-year cohort follow-up of R2* variations (ΔR2*) in three regions of interest (ROIs) (the SN, the Ventral Tegmental Area (VTA) and the Putamen) of 160 patients with PD, using a 3 Tesla MRI, to evaluate the potential interest of R2* as a biomarker of disease progression. The variation of R2* (ΔR2*) will be correlated with clinical markers of disease progress, non-motor symptoms. 80 healthy controls subjects will also be included to assess the effect of aging on cerebral physiological iron levels.

lead_sponsor: University Hospital, Clermont-Ferrand

location_facility:
Chu Dupuytren
Chu Grenoble
CHU Clermont-Ferrand
CHU Pitié Salpétrière
Chu Nancy
Chu Poitiers
Chu Reims
Chu Pellegrin
Chu Montpellier
Hôpital neurologique Pierre Wertheimer
Chu Toulouse
Hôpital Henri Mondor
Chu Lille