title: Innovative Liver Elasticity, Attenuation, and Dispersion Ultrasound Study for Patients With Nonalcoholic Steatohepatitis

acronym: iLEAD

summary: The objective of this study is: (1) to investigate the correlation of ultrasound parameters (SW speed, Dispersion slope, Attenuation value, Normalized Local Variance, Liver / Kidney Intensity Ratio) with the pathological parameters (fibrosis, intralobular inflammation, ballooning degeneration and steatosis); (2) to evaluate the diagnostic performance of SW speed for liver fibrosis, Dispersion slope for intralobular inflammation and Attenuation value for steatosis by comparison with the tissue diagnosis by liver biopsy.

time_perspective: Cross-Sectional

lead_sponsor: Tokyo Medical University

location_country:
United Kingdom
Italy
Korea, Republic of
United States
France
China
Japan
Germany

location_facility: Charing Cross Hospital / Imperial College London

location_facility: Policlinico Umberto I, Univ. La Sapienza

intervention_type: Diagnostic Test


title: AURORAX-0087A: Glycosaminoglycan Scores for Surveillance of Recurrence in Leibovich Points ≥5 Non-metastatic Clear Cell Renal Cell Carcinoma

summary: AUR87A is an observational prospective multicenter diagnostics test cohort study for detection of renal cell carcinoma recurrence as determined by the reference standard, which is imaging using computed tomography (CT) of the chest and abdomen at defined intervals after primary surgery.

study_start_date_type: Actual

status: Recruiting

study_completion_date_type: Anticipated

study_type: Observational

time_perspective: Prospective

enrollment_type: Anticipated

ipd_sharing: Undecided

ipd_sharing_description: Unidentified clinical and biochemical data will become available to the research community along with the publication of a scientific article related to the present investigation.

lead_sponsor: Elypta

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

location_facility:
Prostate Cancer Centre
Odense University Hospital
Hospital Universitario Cabueñes
Salford Royal NHS Foundation Trust
Frimley Park Hospital
AOU San Luigi Gonzaga
Istituto Nazionale Tumori Regina Elena
Hospital da Luz Coimbra
AOU San Orsola Malpighi
Careggi University Hospital
Emory University School of Medicine
Western General Hospital
Norfolk & Norwich University Hospital
Helsinki University Central Hospital
San Raffaele Hospital
Zealand University Hospital
MD Anderson Cancer Center
Guys & St Thomas Hospital
Aarhus University Hospital
Royal Berkshire Hospital
AOU Integrata Verona
Sahlgrenska University Hospital
Memorial Sloan Kettering Cancer Center
Royal Free Hospital
Addenbrooke's Hospital
Hôpital Henri Mondor

lead_sponsor_type: industriel

status_simplified: Ongoing


title: A Phase 2, 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)

acronym: 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.

status: Completed

study_completion_date_type: Actual

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Actual

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: Cheriyan J, Webb AJ, Sarov-Blat L, Elkhawad M, Wallace SM, Mäki-Petäjä KM, Collier DJ, Morgan J, Fang Z, Willette RN, Lepore JJ, Cockcroft JR, Sprecher DL, Wilkinson IB. Inhibition of p38 mitogen-activated protein kinase improves nitric oxide-mediated vasodilatation and reduces inflammation in hypercholesterolemia. Circulation. 2011 Feb 8;123(5):515-23. doi: 10.1161/CIRCULATIONAHA.110.971986. Epub 2011 Jan 24.

ReferenceCitation: de Greef JC, Lemmers RJ, van Engelen BG, Sacconi S, Venance SL, Frants RR, Tawil R, van der Maarel SM. Common epigenetic changes of D4Z4 in contraction-dependent and contraction-independent FSHD. Hum Mutat. 2009 Oct;30(10):1449-59. doi: 10.1002/humu.21091.

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: Jagannathan S, Shadle SC, Resnick R, Snider L, Tawil RN, van der Maarel SM, Bradley RK, Tapscott SJ. Model systems of DUX4 expression recapitulate the transcriptional profile of FSHD cells. Hum Mol Genet. 2016 Oct 15;25(20):4419-4431. doi: 10.1093/hmg/ddw271.

ReferenceCitation: Statland JM, Tawil R. Risk of functional impairment in Facioscapulohumeral muscular dystrophy. Muscle Nerve. 2014 Apr;49(4):520-7. doi: 10.1002/mus.23949. Epub 2014 Feb 10.

ReferenceCitation: Wang LH, Friedman SD, Shaw D, Snider L, Wong CJ, Budech CB, Poliachik SL, Gove NE, Lewis LM, Campbell AE, Lemmers RJFL, Maarel SM, Tapscott SJ, Tawil RN. MRI-informed muscle biopsies correlate MRI with pathology and DUX4 target gene expression in FSHD. Hum Mol Genet. 2019 Feb 1;28(3):476-486. doi: 10.1093/hmg/ddy364.

ReferenceType: derived

ReferenceCitation: Jagannathan S, de Greef JC, Hayward LJ, Yokomori K, Gabellini D, Mul K, Sacconi S, Arjomand J, Kinoshita J, Harper SQ. Meeting report: the 2021 FSHD International Research Congress. Skelet Muscle. 2022 Jan 17;12(1):1. doi: 10.1186/s13395-022-00287-8. Review.

ipd_sharing: No

lead_sponsor: Fulcrum Therapeutics

location_country:
United States
Spain
Canada
France

location_facility: University of Massachusetts Memorial Medical Center

location_facility: University of California Los Angeles (UCLA)

location_facility: Washington University School of Medicine

location_facility: Montreal Neurological Institute and Hospital

intervention_type: Drug

status_simplified: Completed


type: EudraCT Number

type: Other Identifier

title: An Open-label, First-in-human, Single Agent, Dose-escalation and Expansion Study for the Evaluation of Safety, Pharmacokinetics, Pharmacodynamics and Anti-tumor Activity of SAR442085 in Patients With Relapsed or Refractory Multiple Myeloma (RRMM)

summary:
Primary Objectives:

Dose Escalation Part A: To determine the maximum tolerated dose (MTD) of SAR442085 administered as a single agent in patients with relapsed or refractory multiple myeloma (RRMM), and determine the recommended Phase 2 dose (RP2D) for the subsequent Expansion Part B
Dose Expansion Part B: To assess the antitumor activity of single agent of SAR442085 at the RP2D in patients with RRMM

Secondary Objectives:

To characterize the safety profile of SAR442085
To characterize the pharmacokinetics (PK) profile of SAR442085 when administered as a single agent
To evaluate the potential immunogenicity of SAR442085
To assess preliminary evidence of antitumor activity in the Dose Escalation Part A

status: Active, not recruiting

design_allocation: Non-Randomized

ipd_sharing: Yes

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:
Taiwan
Spain
Greece
United States
France
Czechia

location_facility:
Investigational Site Number :3000001
Investigational Site Number :7240002
Investigational Site Number :8400005
Investigational Site Number :1580002
Investigational Site Number :2030003
Investigational Site Number :1580001
Investigational Site Number :8400003
Investigational Site Number :2500001
Investigational Site Number :2030002
Investigational Site Number :8400006
Investigational Site Number :8400002
Investigational Site Number :2030001
Investigational Site Number :8400004
Investigational Site Number :7240001


title: Randomized Controlled Clinical Study to Assess the Benefit of Non-invasive PGT-A, by the Analysis of Spent Blastocyst Media, as a Tool for Embryo Prioritization in Infertile Patients Undergoing Assisted Reproduction.

summary:
Chromosomal aneuploidies are linked with spontaneous miscarriages and abnormal offspring in human pregnancies. In addition, some types of aneuploidy are reported to prevent implantation. Thus, there is a need to identify the embryos with highest implantation potential on in vitro fertilization (IVF) programs.

Since embryo morphology and kinetics have a weak association with embryo ploidy, trophectoderm biopsy plus Next-Generation Sequencing (NGS) is becoming a very popular approach to determine the embryo chromosomal status. This technique is called Preimplantation Genetic Testing for Aneuploidy (PGT-A). Although shown to be efficient, it is invasive for the embryo, requires specific technical skills and it remains expensive. Therefore, the development of a non-invasive, rapid and cheaper method for assessing embryo ploidy status would represent a progress in the field of IVF.

The non-invasive approach has been explored by some groups that analyzed the Spent Blastocyst Medium (SBM) where the embryo was incubated up to the time of transfer or freezing. In daily routine, this media is discarded after finishing the culture of the embryo. Importantly, though, this media reportedly contains traces of embryonic cell-free DNA (cfDNA) that can represent the genetic load of the embryo.

On the basis of that, the hypothesis of this study is that embryo prioritization according to the analysis of the embryonic cfDNA in the SBM could improve ongoing pregnancy rate in 10 percentual points compared to standard blastocyst transfer based on morphology.

primary_purpose: Diagnostic

lead_sponsor: Igenomix

location_country:
Brazil
Spain
Italy
Argentina
United States
France
Turkey

location_facility: Società Italiana Studi di Medicina della Riproduzione (S.I.S.M.e.R.)

location_facility: South Florida Institute for Reproductive Medicine (IVFMD)

location_facility: Fertility Associates of California (FSAC)

location_facility: Crecer: Centro de Reproducción y Genética Humana

location_facility: Nilo Frantz - Centro de Reprodução Humana

intervention_type: Other


title: Long-term Extension Safety and Efficacy Study of SAR442168 in Participants With Relapsing Multiple Sclerosis

summary:
Primary Objective:

To determine the long-term safety and tolerability of SAR442168 in RMS participants

Secondary Objective:

To evaluate efficacy of SAR442168 on disease activity, assessed by clinical and imaging methods

location_country:
Spain
Russian Federation
Estonia
Canada
Ukraine
United States
France
Netherlands
Czechia

location_facility:
Investigational Site Number 5280001
Investigational Site Number 8400003
Investigational Site Number 2030001
Investigational Site Number 8400008
Investigational Site Number 8400009
Investigational Site Number 6430005
Investigational Site Number 6430006
Investigational Site Number 1240003
Investigational Site Number 8400007
Investigational Site Number 2030006
Investigational Site Number 2030005
Investigational Site Number 6430007
Investigational Site Number 8040009
Investigational Site Number 8400005
Investigational Site Number 8040001
Investigational Site Number 8040005
Investigational Site Number 1240001
Investigational Site Number 6430003
Investigational Site Number 7240003
Investigational Site Number 8400001
Investigational Site Number 7240001
Investigational Site Number 8400002
Investigational Site Number 8400006
Investigational Site Number 8040006
Investigational Site Number 8040003
Investigational Site Number 2500004
Investigational Site Number 6430001
Investigational Site Number 2030003
Investigational Site Number 2030002
Investigational Site Number 8040007
Investigational Site Number 2030004
Investigational Site Number 7240002
Investigational Site Number 2330001
Investigational Site Number 2030007
Investigational Site Number 7240004
Investigational Site Number 8040002
Investigational Site Number 7240005


title: Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

summary: Building on previous work of the Myotonic Dystrophy Clinical Research Network (DMCRN), the present study seeks to overcome insufficient data on natural history; lack of reliable biomarkers; and incomplete characterization and limited biological understanding of the phenotypic heterogeneity of Myotonic Dystrophy 1 by examining strategies to improve the reliability by making further refinements in our sample collection and analysis procedures by developing strategies for managing patient heterogeneity going forward.

time_perspective: Other

ipd_sharing_description: Aggregated and deidentified data will be shared with qualified investigators upon majority approval of the DMCRN investigators.

lead_sponsor: Virginia Commonwealth University

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

location_facility: Neuromuscular Reference Center Institute of Myology

lead_sponsor_type: academique


title: GBM AGILE: Global Adaptive Trial Master Protocol: An International, Seamless Phase II/III Response Adaptive Randomization Platform Trial Designed To Evaluate Multiple Regimens In Newly Diagnosed and Recurrent GBM

acronym: GBM AGILE

summary: Glioblastoma (GBM) adaptive, global, innovative learning environment (GBM AGILE) is an international, seamless Phase II/III response adaptive randomization platform trial designed to evaluate multiple therapies in newly diagnosed (ND) and recurrent GBM.

ipd_sharing_description: Global Coalition for Adaptive Research (GCAR) is in the planning stages at this time.

lead_sponsor: Global Coalition for Adaptive Research

location_country:
United States
Switzerland
Canada
France

location_facility: Perlmutter Cancer Center, NYU Langone Health

location_facility: University of Pittsburgh Medical Center - Hillman Cancer Center

location_facility: University of Texas - MD Anderson Cancer Center

location_facility: Medical University of South Carolina - Hollings Cancer Center

location_facility: LSU Health Sciences Center - New Orleans

location_facility: Comprehensive Cancer Center of Wake Forest

location_facility: Froedtert Hospital/Medical College of Wisconsin

location_facility: Centre Hospitalier Lyon Sud / Hôpital Neurologique P. Wertheimer

location_facility: University Hospitals Cleveland Medical Center

location_facility: University of Utah - Huntsman Cancer Institute

location_facility: Cedars Sinai - Samuel Oschin Comprehensive Cancer Institute

location_facility: Washington University School of Medicine - Siteman Cancer Center

location_facility: Yale Cancer Center / Smilow Cancer Hospital

location_facility: University of Mississippi Medical Center

location_facility: Montreal Neurological Institute and Hospital, McGill University

location_facility: University of Texas Southwestern Medical Center

location_facility: University of Pennsylvania - Perelman Center for Advanced Medicine

location_facility: Winship Cancer Institute of Emory University

intervention_type: Radiation


title: A Phase 1, Open-label, Dose-escalation and Expansion Study of MEDI1191 Administered Intratumorally as Monotherapy and in Combination With Durvalumab in Subjects With Advanced Solid Tumors

summary: To evaluate MEDI1191 administered intratumorally in sequential and concurrent combination with intravenous durvalumab in patients with solid tumors.

ipd_sharing_description:
Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment:

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

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

lead_sponsor: MedImmune LLC

location_country:
Netherlands
United States
Spain
France

intervention_type: Biological


primary_purpose: Supportive Care

title: A Randomized, Double-blind, Placebo-controlled Phase II Multi-center Study of Intravenous MBG453 Added to Hypomethylating Agents in Adult Subjects With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R Criteria

lead_sponsor: Novartis Pharmaceuticals

source: euctr

location_country:
Taiwan
Hong Kong
Spain
Greece
Austria
Italy
United Kingdom
Canada
Hungary
Korea, Republic of
United States
France
Turkey
Belgium
Japan
Germany
Czechia

location_facility: Dana Farber Cancer Institute Centerfor Sarcoma&BoneOncology

location_facility: Ohio State Comprehensive Cancer Center/James Cancer Hospital SC BCL201X2102C

location_facility: The Cancer Institute of New Jersey The Cancer Institute of NJ

location_facility: City of Hope National Medical Center Medical Oncology & Therapeutic

summary: This Phase II is a multicenter, randomized, two-arm parallel-group, double-blind, placebo-controlled study of MBG453 or placebo added to hypomethylating agents (azacitidine or decitabine) in adult subjects with IPSS-R intermediate, high or very high risk myelodysplastic syndrome (MDS) not eligible for Hematopoietic Stem Cell Transplant (HSCT) or intensive chemotherapy.


primary_purpose: Prevention

title: A Phase 2b Open-label, Single-arm Study to Evaluate Pharmacokinetics, Efficacy, Safety and Tolerability of Letermovir in Pediatric Participants From Birth to Less Than 18 Years of Age at Risk of Developing CMV Infection and/or Disease Following Allogeneic Haematopoietic Stem Cell Transplantation (HSCT)

lead_sponsor: Merck Sharp & Dohme LLC

type: Registry Identifier

location_country:
Poland
Spain
Australia
Mexico
United States
France
Israel
Turkey
Colombia
Japan
Germany

location_facility: Royal Childrens Hospital Melbourne ( Site 0181)

location_facility: Centro Medico Imbanaco de Cali S.A ( Site 0211)

location_facility: Ege Univ.Tip Fakultesi Cocuk Has ( Site 0163)

location_facility: Saitama Children's Medical Center ( Site 0202)

location_facility: Hospital Infantil de Mexico ( Site 0221)

location_facility: Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckieg-Klinika Transplantacji Szpiku, Onkolog

location_facility: University Of Chicago School Of Medicine ( Site 0253)

location_facility: Akdeniz University Faculty of Medicine ( Site 0161)

location_facility: National Center for Child Health and Development ( Site 0201)

location_facility: Hospital Universitari Vall d Hebron ( Site 0154)

location_facility: Nuevo Hospital Civil Dr Juan I Menchaca ( Site 0223)

location_facility: Schneider Children's Medical Center ( Site 0122)

location_facility: Cincinnati Children's Hospital Medical Center ( Site 0244)

location_facility: The Children s Hospital at Westmead ( Site 0185)

location_facility: Charite Universitaetsmedizin Berlin - Campus-Virchow-Klinikum ( Site 0113)

location_facility: Instituto Nacional de Pediatria ( Site 0224)

location_facility: Hôpital Universitaire Necker Enfants Malades-, Unite d'Immunologie-Hematologie et Rhumatologie Pedi

location_facility: Hospital Universitario "Dr. Jose Eleuterio Gonzalez" ( Site 0222)

location_facility: Szpital Uniwersytecki nr 1 im. Dr. Antoniego Jurasza w Bydgoszczy ( Site 0141)

location_facility: Children's Hospital of Orange County ( Site 0241)

location_facility: Memorial Sloan Kettering Cancer Center ( Site 0254)

location_facility: Pediatric Hemato Oncology Safra Children's Hospital, Sheba Medical Center ( Site 0123)

location_facility: H. de la Santa Creu I Sant Pau ( Site 0155)

location_facility: Duke University Health System ( Site 0252)

location_facility: Children's Hospital of Pittsburgh of UPMC ( Site 0258)

location_facility: Universitatsklinikum Hamburg-Eppendorf ( Site 0111)

location_facility: Hospital Universitario La Paz ( Site 0153)

location_facility: Lady Cilento Children s Hospital ( Site 0182)

location_facility: Instituto De Cancerologia S.A. ( Site 0213)

location_facility: Universitaetsklinikum Muenster ( Site 0114)

location_facility: Hospital Infantil Universitario Nino Jesus ( Site 0151)

location_facility: Universitaetsklinikum Frankfurt ( Site 0112)

location_facility: UCSF Benioff Children's Hospital San Francisco ( Site 0245)

location_facility: City of Hope Comprehensive Cancer Center ( Site 0251)

summary: The primary objective of this study is to evaluate the pharmacokinetics (PK) of letermovir (LET) in pediatric participants. Participants will be enrolled in the following 3 age groups: Age Group 1: From 12 to <18 years of age (adolescents); Age Group 2: From 2 to <12 years of age (children); and Age Group 3: From birth to <2 years of age (neonates, infants and toddlers). All participants will receive open label LET for 14 weeks (~100 days) post-transplant, with doses based on body weight and age.


title: Evaluation of an eXtended and proacTive Dosing regimEn in Treatment-Naïve Patients With Wet Age-related Macular Degeneration (wAMD)

acronym: XTEND

summary: In this observational study researchers want to learn more about changes in visual acuity (clarity of vision) with proactive flexible treatments over time in patients suffering from wet age-related macular degeneration (wAMD) after decision to treat with Aflibercept (Eylea) was made. Wet AMD is an eye disease that progressively destroys the macula, the central portion of the retina, impairing central vision.

ipd_sharing_description: Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.clinicalstudydatarequest.com to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the Study sponsors section of the portal.

lead_sponsor: Bayer

location_country:
Denmark
Spain
Colombia
Ireland
Australia
Italy
Argentina
Canada
Switzerland
Thailand
United Kingdom
Korea, Republic of
France
China
Belgium
Norway
Sweden


title: An Open-Label Study in Adolescent and Adult Severe (Coagulation Factor Activity <1%) Hemophilia A Participants With or Without Inhibitors or Moderately Severe to Severe Hemophilia B Participants (Coagulation Factor Activity ≤2%) With or Without Inhibitors Comparing Standard Treatment to PF-06741086 Prophylaxis

lead_sponsor: Pfizer

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

location_country:
Croatia
Taiwan
Hong Kong
Serbia
Saudi Arabia
Italy
Oman
Russian Federation
Spain
Canada
Korea, Republic of
United States
France
China
India
Mexico
Japan
South Africa
Turkey
Bulgaria

location_facility: McMaster University Medical Centre - Hamilton Health Sciences

location_facility: Gazi University Health Research and Practice Center Gazi Hospital

location_facility: Gaziantep University Sahinbey Research and Training Hospital

location_facility: Dr. Behcet Uz Child Diseases Surgery Education and Research Hospital

location_facility: Severance Hospital, Yonsei University Health System

location_facility: King Faisal Specialist Hospital & Research Center

location_facility: FGBOU VO "Samara State Medical University" of MoH of Russia

location_facility: Kyung Hee University Hospital At Gangdong

location_facility: USF Health Morsani Center For Advanced Healthcare

location_facility: Institute of Hematology, Chinese Academy of Medical Sciences

location_facility: National Specialized Hospital for the Active Treatment of Hematological Diseases - EAD, Sofia

location_facility: Università degli Studi di Perugia, Azienda Ospedaliera di Perugia, Ospedale Santa Maria della

location_facility: Sahyadri Clinical Research and Development Center

location_facility: Nagoya University Hospital - Transfusion Medicine

location_facility: Tokyo Metropolitan Children's Medical Center

location_facility: Hospital Universitario "Dr Jose Eleuterio Gonzalez"

location_facility: Beijing Children's Hospital, Capital Medical University

location_facility: Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

location_facility: Ondokuz Mayıs University Medical Faculty

location_facility: Nanfang Hospital, Southern Medical University

location_facility: Università degli Studi di Roma "Sapienza"-Policlinico Umberto I

location_facility: Institute for Mother and Child healthcare "Dr Vukan Cupic"

location_facility: Charlotte Maxeke Johannesburg Academic Hospital

location_facility: Karadeniz Technical University Medical Faculty

location_facility: Centro Multidisciplinario para el Desarrollo Especializado de la Inv. Clínica en Yucatán, S.C.P.

location_facility: The Affiliated Hospital of Guizhou Medical University

summary: Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently than factor replacement products and will work in the presence of inhibitors. The potential for once weekly (QW) subcutaneous (SC) administration provides for treatment options in the absence of reliable vascular access, increased convenience and may enable better compliance. Combined, these qualities should result in a reduction of bleeding episodes.


type: Other Grant/Funding Number

title: An Innovative Approach Towards Understanding and Arresting Type 1 Diabetes (INNODIA)

acronym: INNODIA

summary:
INNODIA is a global consortium linking 26 academic institutions, 4 industrial partners, a small to medium enterprise (SME), and 2 patient organisations, bringing their knowledge and experience together with one common goal: "To fight type 1 diabetes". (www.innodia.eu).

The project, approved in November 2015 and launched in January 2016, runs under the framework of the Innovative Medicines Initiative - Joint Undertaking (https://www.imi.europa.eu/projects-results/project-factsheets/innodia) with a dedicated governance structure ensuring close interaction, communication and adherence to the objectives and deliverables of the consortium.

The overall aim of INNODIA is to advance in a decisive way how to predict, stage, evaluate and prevent the onset and progression of type 1 diabetes (T1D). For this, INNODIA has established a comprehensive and interdisciplinary network of clinical and basic scientists, who are leading experts in the field of T1D research in Europe, with complementary expertise from the areas of immunology, Beta-cell biology, biomarker research and T1D therapy, joining forces in a coordinated fashion with industry partners and two foundations, as well as with all major stakeholders in the process, including regulatory bodies and patients with T1D and their families.

One of the objectives of INNODIA is to develop a new European clinical research network with standardized protocol based on repeated measures of C-peptide (including home measurements) and comprehensive collection of appropriate biological samples for 'omics', immune, viral and microbiome studies in new onset T1D patients and high-risk auto-antibody positive subjects. A protocol for the harmonization of sample collections in newly diagnosed type 1 diabetic patients and first degree relatives of patients with type 1 diabetes was developed following extensive preliminary work involving partners from across all specialities. Core laboratories with experience in their respective field were set up for analysis of auto-antibodies, fresh immune cells, handling of frozen immune cells, C-peptide measures. A series of standard operating procedures for sample collections and analysis were agreed. Sample tracking between clinical centres and central laboratories was included into a purposely designed electronic case report form (eCRF) into which all clinical and laboratory data collected are captured.

ReferenceCitation: Wilhelm-Benartzi CS, Miller SE, Bruggraber S, Picton D, Wilson M, Gatley K, Chhabra A, Marcovecchio ML, Hendriks AEJ, Morobé H, Chmura PJ, Bond S, Aschemeier-Fuchs B, Knip M, Tree T, Overbergh L, Pall J, Arnaud O, Haller MJ, Nitsche A, Schulte AM, Mathieu C, Mander A, Dunger D. Study protocol: Minimum effective low dose: anti-human thymocyte globulin (MELD-ATG): phase II, dose ranging, efficacy study of antithymocyte globulin (ATG) within 6 weeks of diagnosis of type 1 diabetes. BMJ Open. 2021 Dec 7;11(12):e053669. doi: 10.1136/bmjopen-2021-053669.

lead_sponsor: University of Cambridge

location_country:
Denmark
Poland
Luxembourg
Slovenia
Italy
Austria
United Kingdom
Finland
Sweden
France
Belgium
Norway
Germany

location_facility: Institut National de la Sante et de la Recherche Medicale (INSERM)

location_facility: Children's Hospital Auf der Bult, Hannover Medical School


title: Observational Study Evaluating Effectiveness and Safety of Real-World Treatment With Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A

acronym: HEM-POWR

summary: The aim of the HEM-POWR study is to understand better how Damoctocog alfa pegol (Jivi) is used to treat people with Hemophilia A in day-to-day life, how well the treatment is tolerated and how satisfied patients and physicians are with the treatment.

ReferenceCitation: Sanabria M, Álvarez Román MT, Castaman G, Janbain M, Matsushita T, Meijer K, Oldenburg J, Friedl S, Reding MT. Design of the HEM-POWR study: a prospective, observational study of real-world treatment with damoctocog alfa pegol in patients with haemophilia A. BMJ Open. 2021 Sep 2;11(9):e044997. doi: 10.1136/bmjopen-2020-044997.

ipd_sharing_description:
Availability of this study's data will be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access.

As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.

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

location_country:
Luxembourg
Saudi Arabia
Italy
Spain
United States
Belgium
Japan
Portugal
Slovenia
Austria
Russian Federation
Mexico
Norway
Sweden
Kuwait
Brazil
Canada
Switzerland
China
Germany
Denmark
Taiwan
Greece
Finland
France
Colombia
Netherlands
United Arab Emirates

location_facility: Orthopaedic Hospital DBA Orthopaedic Institute for Children

location_facility: University of Colorado Hemophilia and Thrombosis Center

location_facility: The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders

location_facility: South Alabama Medical Science Foundation

location_facility: East Carolina University - Brody School of Medicine


title: Feasibility of the Multiple Sclerosis Performance Test for Assessment of Functional Performance Measures in Patients With Multiple Sclerosis

summary: The primary objective is to evaluate the feasibility of the Multiple Sclerosis Performance Test (MSPT) in a clinical care setting when used by participants with Multiple Sclerosis (MS).

ipd_sharing_description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/

lead_sponsor: Biogen

location_country:
United States
France
Switzerland
Czechia


title: A Phase 4 Double-blinded, Randomized, Active Comparator-controlled Clinical Trial to Study the Efficacy, Safety, and Pharmacokinetics of Sugammadex (MK-8616) for Reversal of Neuromuscular Blockade in Pediatric Participants Aged Birth to <2 Years

location_country:
Denmark
Brazil
Russian Federation
Australia
Mexico
Finland
Netherlands
Guatemala
Hungary
United States
France
Peru
Belgium
Malaysia

location_facility: University of Vermont Medical Center ( Site 3013)

location_facility: The Children s Hospital at Westmead ( Site 3805)

location_facility: Instituto da Crianca do Hospital das Clinicas-FMUSP ( Site 3825)

location_facility: Unidad de Cirugía Cardiovascular ( Site 4126)

location_facility: Variety Children's Hospital D.B.A. Nicklaus Children's Hospital ( Site 3019)

location_facility: University Medical Center Groningen ( Site 4227)

location_facility: St.Petersburg State Pediatric Medical University ( Site 4281)

location_facility: Wilhelmina Kinderziekenhuis ( Site 4228)

location_facility: Centenario Hospital Miguel Hidalgo-Pediatrics Department ( Site 4152)

location_facility: Women and Children Hospital Kuala Lumpur (Hospital Tunku Azizah) ( Site 3875)

location_facility: Children City Clinical Hospital #9 n.a. G.N.Speransky ( Site 4290)

location_facility: Radboud University Medical Center ( Site 4226)

location_facility: UZ Leuven - Campus Gasthuisberg ( Site 3202)

location_facility: Lucille Packard Children's Hospital ( Site 3008)

location_facility: Pediatric Hematology Oncology and Immunology Centre n.a. D.Rogachev. ( Site 4275)

location_facility: Children City Clinical Hospital 13 n.a N.F.Filatov ( Site 4285)

location_facility: Debreceni Egyetem Klinikai Kozpont ( Site 4200)

location_facility: Hospital General de Zona No. 1 ( Site 4153)

location_facility: Queensland Children s Hospital ( Site 3806)

location_facility: McGovern Medical School at UT Health/ Memorial Hermann ( Site 3014)

location_facility: Scientific-Research Clinical Pediatric Institution n.a. Veltischev ( Site 4276)

location_facility: Erasmus University Medical Center ( Site 4225)

location_facility: Universitaire Ziekenhuis Antwerpen - UZA ( Site 3200)

location_facility: Scientific Research Institute Complex Problems Cardiovascular Disease ( Site 4288)

location_facility: The Children's Hospital of Philadelphia ( Site 3021)

location_facility: C.H.R.U. de Lille. Hopital Jeanne de Flandres ( Site 3304)

location_facility: NMRC Obstetrics Gynecology and Perinatology n.a. V.I. Kulakov ( Site 4287)

location_facility: Children's Hospital of Pittsburgh UPMC ( Site 3017)

location_facility: University Malaya Medical Centre. ( Site 3876)

location_facility: Royal Childrens Hospital Melbourne ( Site 3801)

location_facility: Instituto Nacional Cardiovascular Incor ( Site 3928)

summary: This study will evaluate the efficacy, safety, and pharmacokinetics (PK) of sugammadex (MK-8616) for reversal of both moderate and deep neuromuscular blockade (NMB) in pediatric participants aged birth to <2 years. The primary hypothesis of this study is that sugammadex is superior to neostigmine in reversing moderate NMB as measured by time to neuromuscular recovery.


title: A Phase 1/2 Study of the Oral RET Inhibitor LOXO 292 in Pediatric Patients With Advanced RET-Altered Solid or Primary Central Nervous System Tumors

lead_sponsor: Loxo Oncology, Inc.

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

location_facility: Seattle Children's Hospital Research Foundation

location_facility: The Children's Hospital for Cancer and Blood Disorders

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: University of Texas Southwestern Medical Center at Dallas

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori

summary: This is an open-label, multi-center Phase 1/2 study of oral LOXO-292 in pediatric participants with an activating rearranged during transfection (RET) alteration and an advanced solid or primary CNS tumor.


title: A Post-Market Study Evaluating the Safety of Infinity DBS System With MR Conditional Labeling

summary: This is a study of subjects with the St. Jude Medical Infinity deep brain stimulation (DBS) system who undergo an MRI imaging procedure. Enrollment may occur before DBS implant, or when an MRI scan is planned in a subject with an existing implant. There will be a follow-up visit one month after the MRI procedure to document any adverse events and verify device functionality.

lead_sponsor: Abbott Medical Devices

location_country:
Spain
Sweden
United States
France
Germany

location_facility: Wright State University & Premier Health

location_facility: UNIVERSITATSMEDIZIN der Johannes Gutenberg-Universität Mainz

location_facility: Banner University Medical Center Tucson Campus

location_facility: Medizinische Einrichtungen der Universität Düsseldorf

intervention_type: Device


acronym: CASSIOPEIA

title: A Phase II Trial of Tisagenlecleucel in First-line High-risk (HR) Pediatric and Young Adult Patients With B-cell Acute Lymphoblastic Leukemia (B-ALL) Who Are Minimal Residual Disease (MRD) Positive at the End of Consolidation (EOC) Therapy

ReferenceCitation: Seftel MD. Hyper-CVAD: a regimen for all seasons. Lancet Haematol. 2020 Jul;7(7):e501-e502. doi: 10.1016/S2352-3026(20)30179-4.

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

This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

location_country:
Denmark
Spain
Italy
United Kingdom
Netherlands
Finland
Canada
Sweden
United States
France
Belgium
Norway
Germany

location_facility: Cohen Children's Medical Center of New York

location_facility: University of Utah Clinical Trials Office

location_facility: Medical University of South Carolina Medical Univ of SC

location_facility: Columbia University Medical Center Oncology

location_facility: The Childrens Hospital of Philadelphia Div Gastroint., Hepat. & Nutr.

location_facility: Ann and Robert H Lurie Childrens Hospital of Chicago

location_facility: University of Wisconsin Hospital and Clinics Pharmacy/Drug Shipping Address

location_facility: Children's Hospital of Richmond at VCU Pediatric Hematology Oncology

location_facility: Univ Hospital - Cleveland/Rainbow Babies and Children's Hosp Pediatric Nephrology

location_facility: Texas Children's Cancer and Hematology Center

location_facility: Dana Farber Cancer Institute Dept.of DFCI

location_facility: University of Mississippi Medical Center Childrens Hospital

location_facility: Children's Healthcare of Atlanta Emory University IRB

location_facility: Prinses Maxima Centrum voor Kinderoncologie

location_facility: Childrens Hospital Los Angeles SC CTL019

location_facility: Johns Hopkins Oncology Center Cancer Research Building

location_facility: St. Louis University/Cardinal Glennon Children's Hospital

location_facility: Monroe Carell Jr Childrens Hospital at Vanderbilt

location_facility: University of Texas Southwestern Medical Center

location_facility: James Whitcomb Riley Hospital for Children

location_facility: Cincinnati Children s Hospital Medical Center

location_facility: Children's Hospital of Orange County CHOC Children's

summary: This is a single arm, open-label, multi-center, phase II study to determine the efficacy and safety of tisagenlecleucel in de novo HR pediatric and young adult B-ALL patients who received first-line treatment and are EOC MRD positive. The study will have the following sequential phases: screening, pre-treatment, treatment & follow-up, and survival. After tisagenlecleucel infusion, patient will have assessments performed more frequently in the first month and then at Day 29, then every 3 months for the first year, every 6 months for the second year, then yearly until the end of the study. Efficacy and safety will be assessed at study visits and as clinically indicated throughout the study. The study is expected to end in approximately 8 years after first patient first treatment (FPFT). A post-study long term follow-up for lentiviral vector safety will continue under a separate protocol per health authority guidelines.


title: ACHieve: A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia

summary: This is a long-term, multi-center, longitudinal, observational study in children with achondroplasia (ACH). The aim is to study height velocity and comorbidities in children with ACH. This is a natural history study and no study medication will be administered.

lead_sponsor: Ascendis Pharma A/S

location_country:
Denmark
Portugal
Spain
Ireland
Austria
Australia
Italy
Canada
New Zealand
Switzerland
United Kingdom
United States
France
China
Germany


title: A Phase 1b, Multicenter Study to Determine the Dose, Safety, Efficacy and Pharmacokinetics of TRK-950 When Used in Combinations With Selected Anti-Cancer Treatment Regimens in Patients With Selected Advanced Solid Tumors

summary: The main purpose of this study is to establish the safety and the recommended dose of TRK-950 in combination with FOLFIRI, Gemcitabine / Cisplatin, Gemcitabine / Carboplatin, Ramucirumab / Paclitaxel, PD1 inhibitors (Nivolumab or Pembrolizumab), and Imiquimod Cream, Bevacizumab, Gemcitabine / Carboplatin / Bevacizumab, Topotecan, and Pegylated liposomal doxorubicin (PLD) for selected advanced solid tumors.

lead_sponsor: Toray Industries, Inc

location_country:
United States
France

location_facility: Oncology Associates of Oregon, P.C.(Willamette Valley Cancer Institute and Research Center)

location_facility: Texas Oncology, P.A. Baylor Charles A. Sammons Cancer Center

location_facility: Texas Oncology - Downtown Fort Worth Cancer Center


title: EUS-guided Biliary Drainage of First Intent With the Lumen Apposing Metal Stent vs. ERCP in the Management of Malignant Distal Biliary Obstruction: a Randomized Controlled Trial

summary: Endoscopic ultrasound guided biliary drainage (EUS-BD) is an evolving field that has grown in popularity in the management of malignant biliary obstruction. Although Endoscopic retrograde cholangio-pancreatography (ERCP) with stent insertion has been the mainstay therapy throughout several decades, the transpapillary approach through tumor tissue is associated with significant risk for adverse events such as post-ERCP pancreatitis and stent dysfunction from tumor tissue overgrowth and ingrowth. EUS-BD, through the creation of a choledochoduodenostomy with a stent, has the potential advantage of avoiding the papilla and its associated complications while potentially improving stent patency with lower risks for tumor tissue ingrowth and/or overgrowth.

ReferenceCitation: Boulay BR, Lo SK. Endoscopic Ultrasound-Guided Biliary Drainage. Gastrointest Endosc Clin N Am. 2018 Apr;28(2):171-185. doi: 10.1016/j.giec.2017.11.005. Epub 2018 Feb 3. Review.

ReferenceCitation: Khashab MA, Messallam AA, Penas I, Nakai Y, Modayil RJ, De la Serna C, Hara K, El Zein M, Stavropoulos SN, Perez-Miranda M, Kumbhari V, Ngamruengphong S, Dhir VK, Park DH. International multicenter comparative trial of transluminal EUS-guided biliary drainage via hepatogastrostomy vs. choledochoduodenostomy approaches. Endosc Int Open. 2016 Feb;4(2):E175-81. doi: 10.1055/s-0041-109083. Epub 2016 Jan 15.

ReferenceCitation: Khashab MA, Van der Merwe S, Kunda R, El Zein MH, Teoh AY, Marson FP, Fabbri C, Tarantino I, Varadarajulu S, Modayil RJ, Stavropoulos SN, Peñas I, Ngamruengphong S, Kumbhari V, Romagnuolo J, Shah R, Kalloo AN, Perez-Miranda M, Artifon EL. Prospective international multicenter study on endoscopic ultrasound-guided biliary drainage for patients with malignant distal biliary obstruction after failed endoscopic retrograde cholangiopancreatography. Endosc Int Open. 2016 Apr;4(4):E487-96. doi: 10.1055/s-0042-102648. Epub 2016 Mar 30.

ReferenceCitation: Almadi MA, Barkun A, Martel M. Plastic vs. Self-Expandable Metal Stents for Palliation in Malignant Biliary Obstruction: A Series of Meta-Analyses. Am J Gastroenterol. 2017 Feb;112(2):260-273. doi: 10.1038/ajg.2016.512. Epub 2016 Nov 15. Review.

ReferenceCitation: Almadi MA, Barkun JS, Barkun AN. Stenting in Malignant Biliary Obstruction. Gastrointest Endosc Clin N Am. 2015 Oct;25(4):691-711. doi: 10.1016/j.giec.2015.06.002. Epub 2015 Aug 28. Review.

ReferenceCitation: American Society for Gastrointestinal Endoscopy (ASGE) Standards of Practice Committee, Anderson MA, Appalaneni V, Ben-Menachem T, Decker GA, Early DS, Evans JA, Fanelli RD, Fisher DA, Fisher LR, Fukami N, Hwang JH, Ikenberry SO, Jain R, Jue TL, Khan K, Krinsky ML, Malpas PM, Maple JT, Sharaf RN, Shergill AK, Dominitz JA, Cash BD. The role of endoscopy in the evaluation and treatment of patients with biliary neoplasia. Gastrointest Endosc. 2013 Feb;77(2):167-74. doi: 10.1016/j.gie.2012.09.029. Epub 2012 Dec 7.

ReferenceCitation: Bang JY, Navaneethan U, Hasan M, Hawes R, Varadarajulu S. Stent placement by EUS or ERCP for primary biliary decompression in pancreatic cancer: a randomized trial (with videos). Gastrointest Endosc. 2018 Jul;88(1):9-17. doi: 10.1016/j.gie.2018.03.012. Epub 2018 Mar 21.

ReferenceCitation: Lee TH, Choi JH, Park do H, Song TJ, Kim DU, Paik WH, Hwangbo Y, Lee SS, Seo DW, Lee SK, Kim MH. Similar Efficacies of Endoscopic Ultrasound-guided Transmural and Percutaneous Drainage for Malignant Distal Biliary Obstruction. Clin Gastroenterol Hepatol. 2016 Jul;14(7):1011-1019.e3. doi: 10.1016/j.cgh.2015.12.032. Epub 2015 Dec 31.

ReferenceCitation: Paik WH, Lee TH, Park DH, Choi JH, Kim SO, Jang S, Kim DU, Shim JH, Song TJ, Lee SS, Seo DW, Lee SK, Kim MH. EUS-Guided Biliary Drainage Versus ERCP for the Primary Palliation of Malignant Biliary Obstruction: A Multicenter Randomized Clinical Trial. Am J Gastroenterol. 2018 Jul;113(7):987-997. doi: 10.1038/s41395-018-0122-8. Epub 2018 Jul 2. Erratum in: Am J Gastroenterol. 2018 Oct;113(10):1566.

ReferenceCitation: Park JK, Woo YS, Noh DH, Yang JI, Bae SY, Yun HS, Lee JK, Lee KT, Lee KH. Efficacy of EUS-guided and ERCP-guided biliary drainage for malignant biliary obstruction: prospective randomized controlled study. Gastrointest Endosc. 2018 Aug;88(2):277-282. doi: 10.1016/j.gie.2018.03.015. Epub 2018 Mar 30.

ReferenceCitation: Kunda R, Pérez-Miranda M, Will U, Ullrich S, Brenke D, Dollhopf M, Meier M, Larghi A. EUS-guided choledochoduodenostomy for malignant distal biliary obstruction using a lumen-apposing fully covered metal stent after failed ERCP. Surg Endosc. 2016 Nov;30(11):5002-5008. Epub 2016 Mar 11.

ReferenceCitation: Anderloni A, Fugazza A, Troncone E, Auriemma F, Carrara S, Semeraro R, Maselli R, Di Leo M, D'Amico F, Sethi A, Repici A. Single-stage EUS-guided choledochoduodenostomy using a lumen-apposing metal stent for malignant distal biliary obstruction. Gastrointest Endosc. 2019 Jan;89(1):69-76. doi: 10.1016/j.gie.2018.08.047. Epub 2018 Sep 4.

ReferenceCitation: Cotton PB, Eisen GM, Aabakken L, Baron TH, Hutter MM, Jacobson BC, Mergener K, Nemcek A Jr, Petersen BT, Petrini JL, Pike IM, Rabeneck L, Romagnuolo J, Vargo JJ. A lexicon for endoscopic adverse events: report of an ASGE workshop. Gastrointest Endosc. 2010 Mar;71(3):446-54. doi: 10.1016/j.gie.2009.10.027.

ReferenceCitation: Siegel RL, Miller KD, Jemal A. Cancer statistics, 2018. CA Cancer J Clin. 2018 Jan;68(1):7-30. doi: 10.3322/caac.21442. Epub 2018 Jan 4.

ReferenceCitation: Liao WC, Chien KL, Lin YL, Wu MS, Lin JT, Wang HP, Tu YK. Adjuvant treatments for resected pancreatic adenocarcinoma: a systematic review and network meta-analysis. Lancet Oncol. 2013 Oct;14(11):1095-1103. doi: 10.1016/S1470-2045(13)70388-7. Epub 2013 Sep 12. Review.

ReferenceCitation: Freeman ML, Nelson DB, Sherman S, Haber GB, Herman ME, Dorsher PJ, Moore JP, Fennerty MB, Ryan ME, Shaw MJ, Lande JD, Pheley AM. Complications of endoscopic biliary sphincterotomy. N Engl J Med. 1996 Sep 26;335(13):909-18.

ReferenceCitation: Kochar B, Akshintala VS, Afghani E, Elmunzer BJ, Kim KJ, Lennon AM, Khashab MA, Kalloo AN, Singh VK. Incidence, severity, and mortality of post-ERCP pancreatitis: a systematic review by using randomized, controlled trials. Gastrointest Endosc. 2015 Jan;81(1):143-149.e9. doi: 10.1016/j.gie.2014.06.045. Epub 2014 Aug 1. Review.

ReferenceCitation: Almadi MA, Barkun A, Martel M. Self-expandable metal stents versus plastic stents for malignant biliary obstruction. Gastrointest Endosc. 2016 Apr;83(4):852-3. doi: 10.1016/j.gie.2015.10.035.

ReferenceCitation: Gardner TB, Spangler CC, Byanova KL, Ripple GH, Rockacy MJ, Levenick JM, Smith KD, Colacchio TA, Barth RJ, Zaki BI, Tsapakos MJ, Gordon SR. Cost-effectiveness and clinical efficacy of biliary stents in patients undergoing neoadjuvant therapy for pancreatic adenocarcinoma in a randomized controlled trial. Gastrointest Endosc. 2016 Sep;84(3):460-6. doi: 10.1016/j.gie.2016.02.047. Epub 2016 Mar 10.

ReferenceCitation: Barkun AN, Adam V, Martel M, AlNaamani K, Moses PL. Partially covered self-expandable metal stents versus polyethylene stents for malignant biliary obstruction: a cost-effectiveness analysis. Can J Gastroenterol Hepatol. 2015 Oct;29(7):377-83. Epub 2015 Jun 30.

ReferenceCitation: Yachimski PS, Ross A. The Future of Endoscopic Retrograde Cholangiopancreatography. Gastroenterology. 2017 Aug;153(2):338-344. doi: 10.1053/j.gastro.2017.06.015. Epub 2017 Jun 21.

ReferenceCitation: Giovannini M, Moutardier V, Pesenti C, Bories E, Lelong B, Delpero JR. Endoscopic ultrasound-guided bilioduodenal anastomosis: a new technique for biliary drainage. Endoscopy. 2001 Oct;33(10):898-900.

ReferenceCitation: Sharaiha RZ, Khan MA, Kamal F, Tyberg A, Tombazzi CR, Ali B, Tombazzi C, Kahaleh M. Efficacy and safety of EUS-guided biliary drainage in comparison with percutaneous biliary drainage when ERCP fails: a systematic review and meta-analysis. Gastrointest Endosc. 2017 May;85(5):904-914. doi: 10.1016/j.gie.2016.12.023. Epub 2017 Jan 4. Review.

ReferenceCitation: Binmoeller KF, Shah JN. Endoscopic ultrasound-guided gastroenterostomy using novel tools designed for transluminal therapy: a porcine study. Endoscopy. 2012 May;44(5):499-503. doi: 10.1055/s-0032-1309382. Epub 2012 Apr 24.

ReferenceCitation: Chen YI, Kunda R, Storm AC, Aridi HD, Thompson CC, Nieto J, James T, Irani S, Bukhari M, Gutierrez OB, Agarwal A, Fayad L, Moran R, Alammar N, Sanaei O, Canto MI, Singh VK, Baron TH, Khashab MA. EUS-guided gastroenterostomy: a multicenter study comparing the direct and balloon-assisted techniques. Gastrointest Endosc. 2018 May;87(5):1215-1221. doi: 10.1016/j.gie.2017.07.030. Epub 2017 Jul 24.

ReferenceCitation: Kamarajah SK, Burns WR, Frankel TL, Cho CS, Nathan H. Validation of the American Joint Commission on Cancer (AJCC) 8th Edition Staging System for Patients with Pancreatic Adenocarcinoma: A Surveillance, Epidemiology and End Results (SEER) Analysis. Ann Surg Oncol. 2017 Jul;24(7):2023-2030. doi: 10.1245/s10434-017-5810-x. Epub 2017 Feb 17.

ReferenceCitation: Chen YI, Callichurn K, Chatterjee A, Desilets E, Fergal D, Forbes N, Gan I, Kenshil S, Khashab MA, Kunda R, Lam E, May G, Mohamed R, Mosko J, Paquin SC, Sahai A, Sandha G, Teshima C, Barkun A, Barkun J, Bessissow A, Candido K, Martel M, Miller C, Waschke K, Zogopoulos G, Wong C; ELEMENT trial and for the Canadian Endoscopic Research Collaborative (CERC). ELEMENT TRIAL: study protocol for a randomized controlled trial on endoscopic ultrasound-guided biliary drainage of first intent with a lumen-apposing metal stent vs. endoscopic retrograde cholangio-pancreatography in the management of malignant distal biliary obstruction. Trials. 2019 Dec 9;20(1):696. doi: 10.1186/s13063-019-3918-y.

lead_sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre

location_country: Canada France

location_facility: Centre Hospitalier Universite de Montreal

intervention_type: Procedure


title: OCS Lung INSPIRE Long-Term Continuation Post-Approval Study - INSPIRE Continuation PAS

summary: To evaluate long-term BOS-free survival outcomes of the OCS™ Lung INSPIRE Trial patients.

lead_sponsor: TransMedics

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

location_facility: St. Joseph's Hospital and Medical Center

location_facility: University of California at San Francisco


title: Rostock International Parkinson's Disease Study: an International, Multicenter, Epidemiological Observational Study

acronym: ROPAD

summary: Rostock International Parkinson's Disease Study - An International, multicentre, epidemiological observational study aiming at identification of 1,500 LRRK2-positive patients, the identification of 1,500 non-LRRK2 PD patients and the establishment of a candidate biomarker in the LRRK2-positive cohort.

ReferenceType: result

ReferenceCitation: Baumann CR. Epidemiology, diagnosis and differential diagnosis in Parkinson's disease tremor. Parkinsonism Relat Disord. 2012 Jan;18 Suppl 1:S90-2. doi: 10.1016/S1353-8020(11)70029-3. Review.

ipd_sharing_description: The plan will be defined at a later stages

lead_sponsor: CENTOGENE GmbH Rostock

location_country:
Brazil
Albania
Portugal
Greece
Italy
Spain
United Kingdom
Canada
United States
France
Israel
Turkey
Belgium
Norway
Germany

location_facility: Royal Preston Hospital, Department of Neurology

location_facility: Faculdade de Medicina de Ribeirão Preto - USP

location_facility: University Hospital Marqués de Valdecilla

location_facility: Hospital Clinico Universitario Santiago de Compostela

location_facility: Hospital General Unversitario Gregorio Marañón

location_facility: Stony Brook University School of Medicine

location_facility: Toronto Western Hospital, University Health Network, University of Toronto

location_facility: Centro Hospitalar Universitário de São João

location_facility: Parkinson's Disease and Movement Disorder Center of Baco Raton

location_facility: Vero Beach Neurology & Research Institute

location_facility: Universitätsklinikum Carl Gustav Carus die Dresdner

location_facility: Universidade Federal de São Paulo, UNIFESP

location_facility: Michigan Institute for Neurological Disorders

location_facility: Indiana University, Department of Neurology

location_facility: Newcastle University, Clinical Ageing Research Unit

location_facility: Hospital das Clínicas da Universidade Federal de Minas Gerais

location_facility: Biocruces Bizkaia Health Research Institute

location_facility: Duke University, Department of Neurology

location_facility: HM CINAC | Integrated Neuroscience Center

location_facility: University of Oxford, Nuffield Department of Clinical Neurosciences

location_facility: Instituto Israelita de Ensino e Pesquisa Albert Einstein

location_facility: Hospital Universitário Walter Cândido/ Universidade Federal do Ceará

location_facility: Universitätsklinikum Schleswig-Holstein, Klinik für Neurologie

location_facility: Università di Pavia, Dipartimento di Medicina Molecolare

location_facility: Universitätsklinikum Marburg Klinik und Poliklinik für Neurologie Parkinson Netzwerk Allianz Marburg (PANAMA)

location_facility: Hospital Universitario Pedro Ernesto (HUPE)

location_facility: AOUP Secondo Policlinico Federico II Napoli

location_facility: Neurologisches Fachkrankenhaus für Bewegungsstörungen / Parkinson

location_facility: Tel Aviv Sourasky Medical Center - TASMC

location_facility: Samson Assuta Ashdod University Hospital

location_facility: Department of Neurology, Centre Hospitalier Universitaire (CHU) de Leige

location_facility: Hospital Universitario Fundación Alcorcón

location_facility: Asklepios Fachklinikum Stadtroda Klinik für Neurologie, Schmerztherapie und Schlafmedizin


title: REtroperitoneal SArcoma Registry (RESAR): Prospective Collection of Primary Retroperitoneal Sarcoma Patient's Data, Radiological and Pathological Material for the TransAtlantic Retroperitoneal Sarcoma Working Group

acronym: RESAR

summary:
Surgery is currently the only potentially curative treatment modality for localized retroperitoneal sarcoma (RPS). Available studies regarding oncologic outcomes are mainly retrospective in nature, and RPS are recognized as a rare disease. Therefore, prospective analysis of high quality data is a top priority.

Primary Objectives of this study are:

to prospectively collect standardized clinical data and radiological and pathological material from primary RPS patients treated with surgery at reference centers.
patient outcome will be evaluated in terms of overall survival (OS), disease-free survival (DFS), crude cumulative incidence (CCI) of local recurrence (LR) and distant metastasis (DM).

Secondary Objectives:

to estimate the efficacy and safety of surgical treatment, including extended surgical approach to primary RPS;
to prospectively evaluate the impact of multimodality therapy, including radiation therapy and chemotherapy;
to identify clinical, radiological and pathological characteristics that may influence the oncological outcome or may be used as predictors of LR/DM/OS. These may be important biomarkers of disease;
to utilize collected pathological material for research collaborations.

ReferenceCitation: Gronchi A, Strauss DC, Miceli R, Bonvalot S, Swallow CJ, Hohenberger P, Van Coevorden F, Rutkowski P, Callegaro D, Hayes AJ, Honoré C, Fairweather M, Cannell A, Jakob J, Haas RL, Szacht M, Fiore M, Casali PG, Pollock RE, Raut CP. Variability in Patterns of Recurrence After Resection of Primary Retroperitoneal Sarcoma (RPS): A Report on 1007 Patients From the Multi-institutional Collaborative RPS Working Group. Ann Surg. 2016 May;263(5):1002-9. doi: 10.1097/SLA.0000000000001447.

ReferenceCitation: Trans-Atlantic RPS Working Group. Management of primary retroperitoneal sarcoma (RPS) in the adult: a consensus approach from the Trans-Atlantic RPS Working Group. Ann Surg Oncol. 2015 Jan;22(1):256-63. doi: 10.1245/s10434-014-3965-2. Epub 2014 Oct 15.

ReferenceCitation: MacNeill AJ, Gronchi A, Miceli R, Bonvalot S, Swallow CJ, Hohenberger P, Van Coevorden F, Rutkowski P, Callegaro D, Hayes AJ, Honoré C, Fairweather M, Cannell A, Jakob J, Haas RL, Szacht M, Fiore M, Casali PG, Pollock RE, Barretta F, Raut CP, Strauss DC. Postoperative Morbidity After Radical Resection of Primary Retroperitoneal Sarcoma: A Report From the Transatlantic RPS Working Group. Ann Surg. 2018 May;267(5):959-964. doi: 10.1097/SLA.0000000000002250.

lead_sponsor: Fondazione IRCCS Istituto Nazionale dei Tumori, Milano

location_country:
Poland
Slovenia
Italy
Spain
Australia
United Kingdom
Canada
Korea, Republic of
United States
France
Germany

location_facility: Hospital Clinico Universitario "Virgen de la Arrixaca"

location_facility: University Medical Center and Medical Faculty Mannheim, University of Heidelberg

location_facility: University Hospital Birmingham (Queen Elizabeth)

location_facility: Maria Sklodowska-Curie Memorial Cancer Center and Institute of Oncology

location_facility: Brigham and Women's Hospital/Dana-Farber Cancer Institute

location_facility: Maisonneuve-Rosemont Hospital / University of Montreal


status: Terminated

acronym: ADmIRe

title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Evaluate the Efficacy and Safety of LY3375880 in Adult Subjects With Moderate-to-Severe Atopic Dermatitis: The ADmIRe Study

lead_sponsor: Eli Lilly and Company

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.

location_country:
Spain
Italy
Austria
Argentina
Canada
Hungary
Puerto Rico
United States
France
Mexico
Japan
Germany
Czechia

location_facility: The Guenther Dermatology Research Centre

location_facility: Instituto de Neumonología y Dermatología

location_facility: Dokkyo Medical University Saitama Medical Center

location_facility: Kyoto Prefectural University of Medicine

location_facility: Tien Q. Nguyen, MD inc. DBA First OC Dermatology

location_facility: Centro de Investigaciones Metabólicas (CINME)

location_facility: Piedmont Plastic Surgery and Dermatology

summary: The reason for this study is to see if the study drug LY3375880 is safe and effective in adults with moderate-to-severe atopic dermatitis (AD).

status_simplified: Unknown


title: A Phase 3, 22-week, Multi-center, Randomized Withdrawal Study of TD-9855 in Treating Symptomatic Neurogenic Orthostatic Hypotension in Subjects With Primary Autonomic Failure

acronym: REDWOOD

summary: A Phase 3, 22-week, Multi-center, Randomized Withdrawal Study of ampreloxetine in Treating Symptomatic Neurogenic Orthostatic Hypotension in Subjects with Primary Autonomic Failure

ipd_sharing_description: Theravance Biopharma, Inc. will not be sharing individual de-identified participant data or other relevant study documents.

lead_sponsor: Theravance Biopharma

location_country:
Denmark
Poland
Portugal
Spain
Italy
Austria
Australia
Estonia
Argentina
Canada
New Zealand
Russian Federation
Ukraine
Hungary
United States
France
Israel
United Kingdom
Germany
Bulgaria

location_facility: AOU San Giovanni di Dio e Ruggi d'Aragona

location_facility: Specjalistyczna Praktyka Lekarska, Prof. Grzegorz Opala

location_facility: Parkinson's Disease and Movement Disorders Center

location_facility: University of Cincinnati Medical Center (UCGNI)

location_facility: Charite Universitaetsmedizin Berlin - Campus Benjamin Franklin

location_facility: Communal Noncommercial Enterprise of Lviv Regional Council Lviv Regional Clinical Hospital

location_facility: Hospital da Senhora da Oliveira Guimarães

location_facility: Clinical Research Centre, William Harvey Heart Centre

location_facility: FSBI of Science Institute of Human Brain named after N .P. Bekhtereva of Russian Academy of Sciences

location_facility: City Neurological Center Sibneiromed, LLC

location_facility: Medizinische Universitat Innsbruck, Abteilung fur Neurologie

location_facility: MHATNP Sv. Naum EAD Clinic of Neurological Diseases for Locomotor Disorders

location_facility: Universitatsklinikum Tulln Abteilung fur Neurologie

location_facility: Communal Noncommercial Enterprise City Policlinic #9 of Kharkiv City Council

location_facility: Ohio State University - Wexner Medical Center

location_facility: Montreal Neurological Institute & Hospital

location_facility: Krakowska Akademia Neurologii Sp. Zo.o. Centrum Neurologii Klinicznej

location_facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS / Istituto di Neurologia - Ambulatorio Disturbi del Movimento

location_facility: FSBI National Medical Research Centre of psychiatry and neurology named after V.M. Bekhterev of the MOH of the Russian Federation

location_facility: Complejo Médico de la Policía Federal Argentina Churruca Visca

location_facility: Colorado Springs Neurological Associates, PC

location_facility: Fondazione PTV - Policlinico Tor Vergata I U.0.C. Neurologia

location_facility: Azienda Ospedaliero-Universitaria Pisana- Ospedale S. Chiara, U.O. di Neurologia - Neurofisiopatologia

location_facility: Perron Institute for Neurological and Translational Science

location_facility: The National Hospital for Neurology & Neurosurgery

location_facility: State Budgetary Institution of Healthcare of Novosibirsk region City Clinical Hospital #34

location_facility: Concord Hospital, Neurosciences Department

location_facility: University of Calgary Teaching Research and Wellness Building

location_facility: Ineba - Instituto de Neurociencias de Buenos Aires

location_facility: Fondazione IRCCS CA Granda Ospedale Maggiore Policlinico

location_facility: Wake Forest University Baptist Health Sciences

location_facility: Universita di Bologna-Clinica Neurologica - Dipt di Scienze Neurologiche Ospedale Bellaria

location_facility: Royal Devon and Exeter Hospital NHS Trust

location_facility: Azienda Ospedaliera Universitaria Policlinico - Vittorio Emanuele (Presidio Gaspare Rodolico)

location_facility: Communal Institution Acad. O.I. Yuschenko VRPsH Vinnytsia M.I. Pyrogov NMU Ch of ND with the Course of Neurosurgery

location_facility: Clinical Trials Centre, Level 3 Monash Health Translational Precinct Building Monash Medical Centre

location_facility: The Royal Melbourne Hospital Neurology Department

location_facility: Charite - Campus Virchow-Klinikum, Klinik fur Neurologie

location_facility: University of Texas Southwestern Medical Center

location_facility: FSBI Federal Sibirian Scientific and Clinical Center of Federal Medico-Biological Agency

location_facility: University of Kansas Medical Center Research Institute, Inc.

location_facility: Saint Petersburg State Budgetary Institution of Healthcare City Hospital #40 of Kurortnyi Region

location_facility: Universita degli studi Gabriele D' Annunzio Chieti


title: A Randomized, Double-Blind, Active-Controlled Phase 3 Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

acronym: DAHLIA

summary: This is a randomized, double-blind, active-controlled phase 3 study of ABP 959 in participants with paroxysmal nocturnal hemoglobinuria.

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:
Portugal
Slovenia
Ireland
Italy
Spain
United Kingdom
Finland
Sweden
United States
France
Turkey
Norway
Netherlands
Czechia

location_facility: Ege Universitesi Hastanesi - Sağlık Uygulama ve Araştırma Merkezi

location_facility: Azienda Ospedaliera San Gerardo di Monza

location_facility: Fondazione Policlinico Universitario Agostino Gemelli

location_facility: Instituto Português de Oncologia do Porto Francisco Gentil

location_facility: Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori

location_facility: Oslo University Hospital - Rikshospitalet

location_facility: King's College Hospital NHS Foundation Trust

location_facility: Children's Healthcare of Atlanta at Egleston

location_facility: Karolinska Universitetssjukhuset - Huddinge

location_facility: Azienda Ospedaliera S. Croce e Carle Cuneo


title: French African Pediatric Oncology Registry

acronym: RFAOP

summary:
The ultimate aim of this registry is to collect precise information concerning the children coming to oncology units working with the French African Oncology Group. This data will help to plan and provide correct pediatric oncology treatment and care for this population.

Collecting the data will give much needed information on numbers, stage, treatment and outcome. The register will give data for local and national health authorities in planning pediatric cancer programs.

ipd_sharing_description: Units have acess to their own data and can use it for reporting.

lead_sponsor: French Africa Pediatric Oncology Group

location_country:
Cameroon
Mauritania
Congo, The Democratic Republic of the
Niger
Madagascar
Côte D'Ivoire
Burkina Faso
France
Gabon
Mali
Senegal
Togo

location_facility: Hôpital Aristide Le Dantec, Avenue Pasteur, BP 3001

location_facility: HJRA, Hôpital universitaire Joseph Ravoahangy Andrianavalona

location_facility: Centre Mère et Enfant CME-FCB (Fondation Chantal Biya)

location_facility: Centre Hospitalier Universitaire Pédiatrique Charles De Gaulle

location_facility: CNLC, Centre National de Lutte contre le Cancer

location_facility: l'Institut de Cancérologie de Libreville

location_facility: Cliniques Universitaires de Lubumbashi (CUL)


title: A Phase 1 Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Efficacy of Prostate Specific Membrane Antigen Half-life Extended Bispecific T-cell Engager Acapatamab in Subjects With Metastatic Castration-resistant Prostate Cancer

summary: A phase 1 study evaluating the safety, tolerability, pharmacokinetics, and efficacy of prostate specific membrane antigen half-life extended bispecific T-cell engager acapatamab in subjects with metastatic castration-resistant prostate cancer, and to determine the maximum tolerated dose (MTD) or recommended phase 2 dose (RP2D).

location_country:
Taiwan
Austria
Australia
Canada
United States
France
Singapore
Belgium
Japan
Netherlands

location_facility: University of Iowa Hospitals and Clinics

location_facility: Universite Catholique de Louvain Cliniques Universitaires Saint Luc

location_facility: Krankenhaus der Barmherzigen Brueder Wien

location_facility: Universitaetsklinikum Allgemeines Krankenhaus Wien

location_facility: University of Texas Southwestern Medical Center

location_facility: Linkou Chang Gung Memorial Hospital of Chang Gung Medical Foundation


title: A Multicenter Study to Evaluate Long-term Safety and Tolerability of Open Label Sacubitril/Valsartan in Pediatric Patients With Heart Failure Due to Systemic Left Ventricle Systolic Dysfunction Who Have Completed Study CLCZ696B2319

summary: The purpose of this study is to evaluate long-term safety and tolerability data in eligible CLCZ696B2319 (PANORAMA-HF) patients receiving open-label sacubitril/valsartan.

lead_sponsor: Novartis Pharmaceuticals

location_country:
Croatia
Spain
Italy
United States
South Africa
Japan
Czechia
Poland
Portugal
Austria
Russian Federation
Argentina
India
Israel
Turkey
Singapore
Lebanon
Canada
Switzerland
Hungary
Germany
Bulgaria
Taiwan
Finland
Thailand
Korea, Republic of
France


title: FlowTriever All-Comer Registry for Patient Safety and Hemodynamics (FLASH)

acronym: FLASH

summary:
To evaluate the safety and effectiveness of the FlowTriever System for use in the removal of emboli from the pulmonary arteries in the treatment of acute pulmonary embolism (PE). The use of the device will be assessed in a real-world population, with eligibility criteria that closely approximate its use in clinical practice.

Up to 300 additional patients with anticoagulation treatment as the initial planned primary treatment strategy for intermediate risk PE will also be evaluated (US only).

status: Enrolling by invitation

lead_sponsor: Inari Medical

location_country:
Switzerland
United States
France
Belgium
Germany

location_facility: University of Arizona College of Medicine

location_facility: Providence Regional Medical Center Everett

location_facility: University Hospitals Cleveland Medical Center

location_facility: Metropolitan Heart and Vascular Institute


title: A Phase 2 Multicenter Study Evaluating the Efficacy and Safety of Axicabtagene Ciloleucel as First-Line Therapy in Subjects With High-Risk Large B-Cell Lymphoma (ZUMA-12)

lead_sponsor: Kite, A Gilead Company

ReferenceCitation: Neelapu SS, Dickinson M, Munoz J, Ulrickson ML, Thieblemont C, Oluwole OO, et al. 739 Primary Analysis of ZUMA-12: A Phase 2 Study of Axicabtagene Ciloleucel (Axi-Cel) As First-Line Therapy in Patients with High-Risk Large B-Cell Lymphoma (LBCL) [Abstract]. 63rd American Society of Hematology (ASH) Annual Meeting and Exposition; 2021 11-14 December.

ReferenceCitation: Neelapu SS, Dickinson M, Ulrickson ML, Oluwole OO, Herrera AF, Thieblemont C, et al. 405 Interim Analysis of ZUMA-12: A Phase 2 Study of Axicabtagene Ciloleucel (Axi-Cel) as First-Line Therapy in Patients (Pts) With High-Risk Large B-Cell Lymphoma (LBCL) [Abstract]. 62nd American Society of Hematology (ASH) Annual Meeting and Exposition Virtual; 2020 05-08 December.

ReferenceCitation: Neelapu SS, Chavez JC, Lin Y, Munoz J, Ujjani CS, Riedell P, et al. ZUMA-12: A Phase 2 Multicenter Study of Axicabtagene Ciloleucel (Axi-Cel) as a First-Line Therapy in Patients (Pts) with High-Risk Large B-Cell Lymphoma (LBCL) [Abstract]. J Clin Oncol 2019;37 (15).

ReferenceCitation: Neelapu SS, Dickinson M, Munoz J, Ulrickson ML, Thieblemont C, Oluwole OO, Herrera AF, Ujjani CS, Lin Y, Riedell PA, Kekre N, de Vos S, Lui C, Milletti F, Dong J, Xu H, Chavez JC. Axicabtagene ciloleucel as first-line therapy in high-risk large B-cell lymphoma: the phase 2 ZUMA-12 trial. Nat Med. 2022 Apr;28(4):735-742. doi: 10.1038/s41591-022-01731-4. Epub 2022 Mar 21.

location_country:
Australia
United States
France

location_facility: The University of Texas MD Anderson Cancer Center

location_facility: H. Lee Moffitt Cancer Center and Research Institute

summary:
The primary objective of this study is to estimate the efficacy of axicabtagene ciloleucel in participants with high-risk large B-cell lymphoma.

After the end of KTE-C19-112 (ZUMA-12), participants who received an infusion of axicabtagene ciloleucel will complete the remainder of the 15-year follow-up assessments in a separate long-term follow-up study, KT-US-982-5968.


summary: The purpose of the Micra Atrial TRacking Using A Ventricular AccELerometer 2 (MARVEL 2) study is to demonstrate safe and effective operation of the MARVEL 2 features for providing AV synchronous pacing in patients with normal sinus node function and AV block

ReferenceCitation: Garweg C, Khelae SK, Chan JYS, Chinitz L, Ritter P, Johansen JB, Sagi V, Epstein LM, Piccini JP, Pascual M, Mont L, Willems R, Splett V, Stromberg K, Sheldon T, Kristiansen N, Steinwender C. Behavior of AV synchrony pacing mode in a leadless pacemaker during variable AV conduction and arrhythmias. J Cardiovasc Electrophysiol. 2021 Jul;32(7):1947-1957. doi: 10.1111/jce.15061. Epub 2021 May 20.

ReferenceCitation: Garweg C, Khelae SK, Steinwender C, Chan JYS, Ritter P, Johansen JB, Sagi V, Epstein LM, Piccini JP, Pascual M, Mont L, Willems R, Sheldon T, Splett V, Stromberg K, Wood N, Chinitz L. Predictors of atrial mechanical sensing and atrioventricular synchrony with a leadless ventricular pacemaker: Results from the MARVEL 2 Study. Heart Rhythm. 2020 Dec;17(12):2037-2045. doi: 10.1016/j.hrthm.2020.07.024. Epub 2020 Jul 24.

ReferenceCitation: Steinwender C, Khelae SK, Garweg C, Chan JYS, Ritter P, Johansen JB, Sagi V, Epstein LM, Piccini JP, Pascual M, Mont L, Sheldon T, Splett V, Stromberg K, Wood N, Chinitz L. Atrioventricular Synchronous Pacing Using a Leadless Ventricular Pacemaker: Results From the MARVEL 2 Study. JACC Clin Electrophysiol. 2020 Jan;6(1):94-106. doi: 10.1016/j.jacep.2019.10.017. Epub 2019 Nov 11.

lead_sponsor: Medtronic Cardiac Rhythm and Heart Failure

location_country:
Denmark
Hong Kong
Spain
Austria
United States
France
Belgium
Malaysia

location_facility: Kepler Universitätsklinikum Med Campus III

location_facility: North Shore University Hospital- Northwell

location_facility: Hospital Universitari Clínic de Barcelona


title: A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

summary: This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

ReferenceCitation: Brusson M, Miccio A. Genome editing approaches to β-hemoglobinopathies. Prog Mol Biol Transl Sci. 2021;182:153-183. doi: 10.1016/bs.pmbts.2021.01.025. Epub 2021 Mar 1.

ReferenceCitation: Frangoul H, Altshuler D, Cappellini MD, Chen YS, Domm J, Eustace BK, Foell J, de la Fuente J, Grupp S, Handgretinger R, Ho TW, Kattamis A, Kernytsky A, Lekstrom-Himes J, Li AM, Locatelli F, Mapara MY, de Montalembert M, Rondelli D, Sharma A, Sheth S, Soni S, Steinberg MH, Wall D, Yen A, Corbacioglu S. CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. N Engl J Med. 2021 Jan 21;384(3):252-260. doi: 10.1056/NEJMoa2031054. Epub 2020 Dec 5.

ReferenceCitation: Modarai SR, Kanda S, Bloh K, Opdenaker LM, Kmiec EB. Precise and error-prone CRISPR-directed gene editing activity in human CD34+ cells varies widely among patient samples. Gene Ther. 2021 Feb;28(1-2):105-113. doi: 10.1038/s41434-020-00192-z. Epub 2020 Sep 1.

lead_sponsor: Vertex Pharmaceuticals Incorporated

location_country:
United Kingdom
Italy
Canada
United States
France
Belgium
Germany

location_facility: Lucille Packard Children's Hospital of Stanford University

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

location_facility: University of Illinois at Chicago Hospitals and Health Systems

location_facility: Methodist Children's Hospital/Texas Transplant Institute

location_facility: Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine, Paediatric Haemotology, Oncology and Stem Cell Transplantation

location_facility: Columbia University Medical Center (≤21 years)

location_facility: Royal London and St Bartholomew's Hospital, Pathology and Pharmacy Building

location_facility: Imperial College Healthcare NHS Trust, Hammersmith Hospital

location_facility: Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Ospedale Pediatrico Bambino Gesu - IRCCS

location_facility: The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers

location_facility: Columbia University Medical Center (21+ years)

location_facility: Hopital Universitaire des Enfants Reine Fabiola (HUDERF)


title: A Phase 1/2 Study of Oral LOXO-305 in Patients With Previously Treated Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL) or Non-Hodgkin Lymphoma (NHL)

lead_sponsor: Loxo Oncology, Inc.

ReferenceCitation: Cohen JB, Shah NN, Alencar AJ, Gerson JN, Patel MR, Fakhri B, Jurczak W, Tan XN, Lewis KL, Fenske T, Coombs CC, Flinn IW, Lewis DJ, Gouill SL, Palomba ML, Woyach JA, Pagel JM, Lamanna N, Barve MA, Ghia P, Eyre TA, Zinzani PL, Ujjani CS, Koh Y, Izutsu K, Lech-Maranda E, Tam CS, Sundaram S, Yin M, Nair B, Tsai DE, Balbas M, Mato AR, Cheah CY, Wang ML. MCL-133 Pirtobrutinib, a Highly Selective, Non-Covalent (Reversible) BTK Inhibitor in Previously Treated Mantle Cell Lymphoma: Updated Results From the Phase 1/2 BRUIN Study. Clin Lymphoma Myeloma Leuk. 2022 Oct;22 Suppl 2:S394-S395. doi: 10.1016/S2152-2650(22)01569-5.

ReferenceCitation: Aslan B, Kismali G, Iles LR, Manyam GC, Ayres ML, Chen LS, Gagea M, Bertilaccio MTS, Wierda WG, Gandhi V. Pirtobrutinib inhibits wild-type and mutant Bruton's tyrosine kinase-mediated signaling in chronic lymphocytic leukemia. Blood Cancer J. 2022 May 20;12(5):80. doi: 10.1038/s41408-022-00675-9.

ReferenceCitation: Kipps TJ. Mining the Microenvironment for Therapeutic Targets in Chronic Lymphocytic Leukemia. Cancer J. 2021 Jul-Aug 01;27(4):306-313. doi: 10.1097/PPO.0000000000000536. Review.

ReferenceCitation: Mato AR, Shah NN, Jurczak W, Cheah CY, Pagel JM, Woyach JA, Fakhri B, Eyre TA, Lamanna N, Patel MR, Alencar A, Lech-Maranda E, Wierda WG, Coombs CC, Gerson JN, Ghia P, Le Gouill S, Lewis DJ, Sundaram S, Cohen JB, Flinn IW, Tam CS, Barve MA, Kuss B, Taylor J, Abdel-Wahab O, Schuster SJ, Palomba ML, Lewis KL, Roeker LE, Davids MS, Tan XN, Fenske TS, Wallin J, Tsai DE, Ku NC, Zhu E, Chen J, Yin M, Nair B, Ebata K, Marella N, Brown JR, Wang M. Pirtobrutinib in relapsed or refractory B-cell malignancies (BRUIN): a phase 1/2 study. Lancet. 2021 Mar 6;397(10277):892-901. doi: 10.1016/S0140-6736(21)00224-5.

location_country:
Poland
United Kingdom
Italy
Australia
Switzerland
Korea, Republic of
United States
France
Japan
Sweden

location_facility: University of North Carolina at Chapel Hill

location_facility: Policlinico S. Orsola Malpighi - Universita di Bologna

location_facility: Kyoto Furitsu Medical University Hospital

location_facility: University of Texas MD Anderson Cancer Center

location_facility: University of California San Francisco, Medical Center at Paranassus

location_facility: Smilow Cancer Hospital at Yale-New Haven

location_facility: Ospedale Regionale di Bellinzona e Valli - Bellinzona

location_facility: Centre Hospitalier Universitaire de Nantes - L' Hopital l'hôtel-Dieu

summary: This is an open-label, multi-center Phase 1/2 study of oral LOXO-305 (pirtobrutinib) in patients with CLL/SLL and NHL who have failed or are intolerant to standard of care.


title: Peanut Oral Immunotherapy Study of Early Intervention for Desensitization (POSEIDON)

acronym: POSEIDON

summary: The purpose of this study is to determine the efficacy and safety of AR101 in peanut-allergic children aged 1 to < 4 years.

lead_sponsor: Aimmune Therapeutics, Inc.

location_country:
Germany
United States
United Kingdom
France

location_facility: University of Michigan Division of Allergy and Clinical Immunology

location_facility: Children's Center for Advanced Pediatrics Clinical Research Lab

location_facility: Royal Manchester Children's Hospital Central Manchester University Hospitals

location_facility: University Hospital Southampton Foundation NHS Trust Southampton General Hospital

location_facility: Sean N. Parker Center for Allergy & Asthma Reseach, LPCH at El Camino Hospital

location_facility: Allergy & Asthma Medical Group and Research Center

location_facility: Snowy Owl, First Floor, Evelina Childrens Hospital

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

location_facility: UNC-CH School of Medicine, Pediatric Allergy, Immunology & Rheumatology, Food Allergy


title: A Phase 2, Open-Label, Multi-center Study of AL101 in Patients With Adenoid Cystic Carcinoma (ACC) Bearing Activating Notch Mutations

acronym: ACCURACY

summary: This is a Phase 2, non comparative, open label, multicenter study of AL101 in patients with recurrent or metastatic ACC who harbor NOTCH 1,2,3,4 activating mutations.

lead_sponsor: Ayala Pharmaceuticals, Inc,

location_country:
United Kingdom
Canada
United States
Israel
France
Netherlands

location_facility: Hamilton Health Sciences Juravinski Cancer Centre

location_facility: University of Maryland School of Medicine

location_facility: H. Lee Moffitt Cancer Center & Research Institute


title: Multi-center, Double-blind, Parallel-group, Randomized, Placebo-controlled, Three Doses, 40-week Extension to Studies ID-078A301 and ID-078A302 to Assess the Long Term Safety and Tolerability of ACT-541468 in Adult and Elderly Subjects With Insomnia Disorder

lead_sponsor: Idorsia Pharmaceuticals Ltd.

location_country:
Denmark
Poland
Spain
Canada
Finland
Switzerland
Sweden
Hungary
Korea, Republic of
United States
France
Belgium
Germany
Bulgaria

location_facility: Zentrum für Integrative Psychiatrie (ZiP) Universität zu Lübeck

location_facility: ZMS Zentrum für medizinische Studien GmbH

location_facility: CRIUSMQ- CIUSSSCN, CETS (clinique du sommeil)

location_facility: Fleming Island Center for Clinical Research

location_facility: Klinikum Rechts der lsar TU München Dept. of Psychiatry and Psychotherapy

location_facility: Sömnutredningsmottagningen, smärtcentrum Akademiska sjukhuset

location_facility: Hospital MAZ - Neurophisiology and Sleep Department

location_facility: St Hedwig-Krankenhaus, Klinik für Schlaf- und Chronomedizin

location_facility: Universitetssjukhuset Örebro Neurokliniken, Sömnenheten

location_facility: 2F Psychiatry Outpatient, 93, Jungbu-daero, Paldal-gu

location_facility: SOMNIBENE Institut für Medizinische Forschung und Schlafmedizin Schwerin GmbH

location_facility: St. Francis Sleep Allergy and Lung Institute

location_facility: 4F Neuroimaging analysis laboratory, 56 Dalseong-ro, Jung-gu

location_facility: Hospital Universitario Araba - Unidad Funcional de Trastornos del Sueño

location_facility: EMC Instytut Medyczny SA, Przychodnia przy Łowieckiej

location_facility: Queensway Sleep Lab Sleep Clinic (MedSleep)

location_facility: Artemis Institute For Clinical Research - Riverside

location_facility: Acibadem City Clinic Tokuda Hospital EAD

location_facility: Klinik imd Poliklinik fur Neurochirurgie

location_facility: Sleep Disorders Centers of the Mid-Atlantic

location_facility: Zentrum für Schlafmedizin Zürcher Oberland, Zürcher RehaZentrum Wald

location_facility: Ośrodek Medycyny Study Nurseu Instytutu Psychiatrii i Neurologii (Sleep Disorders Center)

location_facility: Centro Médico Teknon - Medicina del Sueño

location_facility: Sleep Disorders Center of the Mid-Atlantic

location_facility: Charité - Universitätsmedizin Berlin - Campus Benjamin Franklin Kompetenzzentrum Schlafmedizin

location_facility: Jacksonville Center for Clinical Research

location_facility: Göteborgs Universitet, Centrum för sömn och vakenhetsstörningar

location_facility: Pulmonary Associates of the Southeast/WCR

location_facility: Klinik und Poliklinik für Psychiatrie, Psychosomatik und Psychotherapie der Universität am Bezirksklinikum Regensburg

location_facility: Clinical Trial Center North GmbH & Co. KG

location_facility: Hospital Universitari Vall d'Hebron - Neurophisiology Deparment - Sleep Unit

location_facility: Clinical Research Group of St. Petersburgh

location_facility: Oregon Center for Clinical Investigations,Inc

location_facility: Magyar Honvédség Egészségügyi Központ, Neurológiai Osztály

location_facility: Hospital UZ Leuven_ Pneumology Department

location_facility: Central Insitute of Mental Health Sleep laboratory Medical Faculty Mannheim/Heidelberg University

summary: Study to assess the long term safety and tolerability of daridorexant in adult and elderly subjects suffering from difficulties to sleep


title: A Phase III Clinical Trial to Demonstrate Efficacy / Safety of Liposomal Cyclosporine A + Standard of Care (SoC) vs SoC Alone in Treating Chronic Lung Allograft Dysfunction / Bronchiolitis Obliterans in Patients Post Single Lung Transplant

lead_sponsor: Zambon SpA

location_country:
Spain
United Kingdom
United States
France
Israel
Belgium
Germany

location_facility: Hospital Universitario y Politécnico La Fe

location_facility: Indiana University Health Methodist Hospital

location_facility: Hannover Medical School - MHH Klinik für Pneumologie

location_facility: Royal Papworth Hospital NHS Foundation Trust

location_facility: University of Kentucky Albert B. Chandler Hospital

location_facility: Wythenshawe Hospital - Cardiothoracic Transplant Unit

summary: The objective of the trial is to assess efficacy and safety of add-on aerosolized liposomal cyclosporine A (L-CsA) to Standard of Care (SoC) therapy as compared to SoC therapy alone in the treatment of Bronchiolitis obliterans syndrome (BOS) in single lung transplant recipients.


title: A Placebo-controlled, Patient and Investigator Blinded, Randomized Parallel Cohort Study to Assess Pharmacodynamics, Pharmacokinetics, Safety, Tolerability and Preliminary Clinical Efficacy of VAY736 and CFZ533 in Patients With Systemic Lupus Erythematosus (SLE)

summary: This study is designed to evaluate the safety, tolerability, pharmacokinetics and therapeutic efficacy of treatment with either VAY736 or CFZ533 in patients with SLE to enable further development of these compounds as treatment in this disease population

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

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

location_country:
Poland
Taiwan
Spain
Russian Federation
Australia
Argentina
Thailand
Hungary
Korea, Republic of
France
Israel
China
Japan
Germany
Czechia


acronym: Study of LN-144 and LN-145 in the Treatment of Patients with Solid Tumours

title: A Phase 2, Multicenter Study of Autologous Tumor Infiltrating Lymphocytes (LN 144/LN-145/LN-145-S1) in Patients With Solid Tumors

lead_sponsor: Iovance Biotherapeutics, Inc.

ReferenceCitation: Hensel J, Metts J, Gupta A, Ladle BH, Pilon-Thomas S, Mullinax J. Adoptive Cellular Therapy for Pediatric Solid Tumors: Beyond Chimeric Antigen Receptor-T Cell Therapy. Cancer J. 2022 Jul-Aug 01;28(4):322-327. doi: 10.1097/PPO.0000000000000603. Review.

location_country:
Spain
Greece
United Kingdom
Canada
Switzerland
United States
France
Germany

location_facility: Hospital General Universitario Gregorio Marañón

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: Universitätsklinikum Schleswig-Holstein - Campus Lübeck

location_facility: Hospital Regional Universitario de Malaga - Hospital General

location_facility: Hospital Universitario Fundacion Jimenez Diaz

location_facility: Klinikum rechts der Isar der Technischen Universität München

location_facility: Hospital Universitario Marques de Valdecilla

location_facility: ICO l'Hospitalet - Hospital Duran i Reynals

summary: A prospective, open-label, multi-cohort, non-randomized, multicenter Phase 2 study evaluating adoptive cell therapy (ACT) with TIL LN-144 (Lifileucel)/LN-145 in combination with checkpoint inhibitors or TIL LN-144 (Lifileucel)/LN-145/LN-145-S1 as a single agent therapy.


title: An Open-label Extension Study of Levoketoconazole (2S,4R-ketoconazole) in the Treatment of Endogenous Cushing's Syndrome

acronym: OPTICS

summary: This is a long-term, open-label extension study of levoketoconazole in subjects with endogenous Cushing's Syndrome.

lead_sponsor: Cortendo AB

location_country:
Poland
Spain
Greece
Italy
Hungary
United States
France
Israel
Romania
Netherlands
Bulgaria

location_facility: Washington University School of Medicine

location_facility: Hippokration General Hospital Endocrinology and Diabetes Department

location_facility: AOU Policlinico G. Martino Sezione di Endocrinologia

location_facility: General Hospital of Athens Evangelismos Department of Endocrinology and Diabete

location_facility: Semmelweis Egyetem II. Belgyógyászati Klinika

location_facility: Clinica Endocrinologia malattie del Metabolismo

location_facility: Tel Aviv-Sourasky Medical Center Institute Endocrinology Metabolism & Hypertension

location_facility: University Specialized Hospital for Active Treatment in Endocrinology

location_facility: Institutul National de Endocrinologie C.I. Parhon

location_facility: Spitalul Clinic Judetean de Urgenta Cluj-Napoca

location_facility: University of New Mexico HSC - HSC Sponsored Projects Office

location_facility: Via Sergio Pansini 5 Uni Naples FedericoII Dept of Molecular&Clinical Endocrinology&Oncology

location_facility: Sveti Georgy University Hospital Clinic of Endocrinology and Metabolic Diseases

location_facility: The Center for Diabetes and Endocrine Care

location_facility: Bnai Zion Medical Center Endocrinology Institute


title: Evaluation of The Minimally Invasive VenTouch™ System in The Treatment of Functional Mitral Valve Regurgitation (FMR): OUS Feasibility Study

summary: This is a prospective, multi-center, single-arm study to evaluate safety and efficacy of the VenTouch System for treatment of subjects with functional MR.

lead_sponsor: Mardil Medical

location_country: Poland Netherlands Canada Hungary France Panama Germany

location_facility: University of British Columbia, St. Paul's Hospital

location_facility: Institut Universitaire de Cardiologie et de Pneumologie De Québec (IUCPQ)

location_facility: Libin Cardiovascular Institute, University of Calgary

location_facility: London Health Sciences Centre, University Hospital

location_facility: Semmelweis University Heart and Vascular Center

location_facility: Centralny Szpital Kliniczny MSWiA w Warszawia


title: Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)

summary: The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.

lead_sponsor: Ultragenyx Pharmaceutical Inc

location_country:
Brazil
Portugal
Spain
Netherlands
Argentina
United States
France
Turkey
Germany

location_facility: Erasmus University Medical Center Rotterdam

location_facility: Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.

location_facility: Universitätsmedizin der Johannes Gutenberg-Universität Mainz

location_facility: Hospital Universitario Virgen del Rocío Pabellón Infantil

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

location_facility: Centre Hospitalier Universitaire La Timone

location_facility: New York University Langone Medical Center


title: Open-label, Single-Arm, Phase 2 Study of Oral HDAC-inhibitor Abexinostat in Patients With Relapsed or Refractory Follicular Lymphoma (FORERUNNER)

acronym: FORERUNNER

summary: This study in patients with relapsed/refractory follicular lymphoma who have undergone at least 3 lines of therapy. Patients will receive abexinostat 80 mg (4 × 20 mg tablets) twice a day (BID) in a "one week on, one week off" schedule.

lead_sponsor: Xynomic Pharmaceuticals, Inc.

location_country:
United States
Spain
France

location_facility: Central Texas Veterans Health Care System - NAVREF

location_facility: Bone Marrow Transplant Hematology Oncology Associates

location_facility: Advocate Medical Group - Park Ridge, Luther Lane - Oncology

location_facility: Norton Cancer Institute - St. Matthews Campus


title: International Study for Treatment of High Risk Childhood Relapsed ALL 2010 A Randomized Phase II Study Conducted by the Resistant Disease Committee of the International Berlin, Frankfurt, Münster (BFM) Study Group

summary: The main goal of this study is to improve the outcome of children and adolescents with acute lymphoblastic leukemia with high risk first relapse by optimization of treatment strategies within a large international trial and the integration of new agents.

lead_sponsor: Charite University, Berlin, Germany

location_country:
Denmark
Poland
Portugal
United Kingdom
Italy
Austria
Australia
Finland
Sweden
France
Israel
Belgium
Norway
Netherlands
Czechia

location_facility: Dpt. SCT and Hematology/Oncology University Wroclaw

location_facility: Copenhagen University Hospital (Rigshospitalet)

location_facility: Instituto Português de Oncologia de Lisboa

location_facility: Australian & New Zealand Childhood Hematology & Oncology Group

location_facility: Hòpital Universitaire des Enfants Reine Fabiola


title: A Post-approval Study of the Medtronic Resolute Onyx™ Zotarolimus-Eluting Coronary Stent System

summary:
To observe the continued performance of the Medtronic Resolute Onyx™ Zotarolimus-Eluting Coronary Stent System in a real-world more-comer population.

To collect data on the safety and efficacy of the Medtronic Resolute Onyx™ Zotarolimus-Eluting Coronary Stent System in bifurcated lesions.

primary_purpose: Other

lead_sponsor: Medtronic Vascular

location_country:
United States
France
Slovakia
Belgium

location_facility: Stredoslovensky Ustav Srdcovych a Cievnych Chorob a.s

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


acronym: SOLSTICE

intervention_type: Genetic

title: A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects With Choroideremia Previously Treated With Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects With X-Linked Retinitis Pigmentosa Previously Treated With Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study

lead_sponsor: NightstaRx Ltd, a Biogen Company

location_country:
Denmark
United Kingdom
Netherlands
Finland
Canada
United States
France
Germany

summary: The objective of the study is to evaluate the long-term safety and efficacy of a sub-retinal injection of BIIB111 in participants with Choroideremia (CHM) who have been previously treated with BIIB111 and who have exited an antecedent study; these treated participants will be compared with untreated control participants who have exited the STAR (NCT03496012) study and BIIB112 in participants with X-linked retinitis pigmentosa (XLRP) who have been previously treated with BIIB112 and who have exited an antecedent study.


title: A Phase 2, Multi-center, Open-label, Randomized Study of Oral Asciminib Added to Imatinib Versus Continued Imatinib Versus Switch to Nilotinib in Patients With CML-CP Who Have Been Previously Treated With Imatinib and Have Not Achieved Deep Molecular Response

summary: To evaluate efficacy, safety and pharmacokinetic profile of asciminib 40mg+imatinib or asciminib 60mg+imatinib versus continued imatinib and versus nilotinib versus asciminib 80mg in pre-treated patients with Chronic Myeloid Leukemia in chronic phase (CML-CP)

lead_sponsor: Novartis Pharmaceuticals

location_country:
Denmark
Poland
Taiwan
Hong Kong
Portugal
Spain
Italy
Austria
Australia
Russian Federation
Canada
United Kingdom
Korea, Republic of
United States
Chile
France
Japan
Germany
Czechia

location_facility: University of Texas MD Anderson Cancer Center

location_facility: SUNY Stony Brook Medical Oncology Hematology/Oncology

location_facility: Sidney Kimmel Comprehensive Cancer Center