ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders : ATHN Transcends: A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment in People With Non-Neoplastic Hematologic Disorders

This is a longitudinal, natural history observational cohort study being conducted at approximately 150 ATHN-affiliated sites. Participants will be followed for a minimum of 15 years. Harmonized data elements will be collected at the time of enrollment, quarterly, annually, and ad hoc. Base data will be collected for all participants. Specific data will be collected for participants enrolled in cohort-specific Arms and Modules.Each participant will be assigned to a single cohort: Hemophilia, Von Willebrand Disease, Congenital Platelet Disorders, Rare Disorders, Bleeding Not Otherwise Specified (NOS), Thrombosis/Thrombophilia, or Non-Neoplastic Hematologic Conditions.Study Arms and study Modules may be developed to provision disease and/or disease specific insights related to stakeholders, including but not limited to pharmaceutical companies, ATHN, and Hemophilia Treatment Centers (HTCs). Arms may branch off into product-specific data collection via Modules to be collected during the study, in conjunction with planned study assessments.ATHN Transcends Principal InvestigatorsTammuella Chrisentery-Singleton, MD Ochsner Clinic Foundation American Thrombosis and Hemostasis NetworkMichael Recht, MD, PhD, MBA Yale University School of Medicine National Bleeding Disorders FoundationPUPs Arm:Co-Principal Investigators:Shannon Carpenter, MD, MS University of Missouri Kansas City School of Medicine Children's Mercy HospitalJulie Jaffray, MD University of California San Diego Rady Children's Hospital San DiegoALTUVIIO Module:Co-Principal Investigators Shannon Carpenter, MD, MS University of Missouri Kansas City School of Medicine Children's Mercy HospitalCo-Principal Investigator Julie Jaffray, MD University of California San Diego Rady Children's Hospital San DiegoINHIBIT Module:Co-Principal Investigators:Nicoletta Machin DO, MS Assistant Professor, Division of Hematology/Oncology Hemophilia Center of Western Pennsylvania University of Pittsburgh Medical CenterHemophilia Natural History Arm:Co-Principal Investigators:Tyler Buckner, MD, MSc Hemophilia and Thrombosis Center University of Colorado Anschutz Medical CampusMichael Recht, MD, PhD, MBA Yale University School of Medicine National Bleeding Disorders FoundationRebinyn ModuleCo-Principal Investigators:Lauren Amos, MD Children's Mercy Hospital, Kansas CityGuy Young, MD University of Southern California Children's Hospital Los AngelesHemophilia Gene Therapy Outcomes Arm:Co-Principal Investigators:Janice M. Staber, MD Iowa Hemophilia and Thrombosis Center University of Iowa Stead Family Children's HospitalUlrike M. Reiss, MD Hemophilia Treatment Center St. Jude's Children's Research HospitalSevere VWD Natural History Arm:Co-Principal Investigators:Robert F. Sidonio, Jr., MD, MSc Aflac Cancer and Blood Disorders Center, Hemophilia of Georgia Center for Bleeding and Clotting DisordersAngela C. Weyand, MD C.S. Mott Children's Hospital, University of Michigan Medical School, Ann ArborCongenital Platelet Disorders Natural History Arm:Principal Investigator Sanjay Ahuja, MD Rainbow Babies & Children's Hospital, Case Western Reserve UniversityGlanzmann Thrombasthenia Module:Co-Principal Investigators:Divya Citla-Sridhar, MD University of Arkansas for Medical Sciences Arkansas Children's HospitalMeera Chitlur, MD Children's Hospital of MichiganHemophilia CohortThis cohort includes three Arms and five Modules:Previously Untreated Patients (PUPs) Arm This is a pediatric focused Arm of PUPs with hemophilia A or B.ALTUVIIIO® Module The purpose is to investigate the safety and effectiveness of ALTUVIIIO® in PUPs with hemophilia A.INHIBIT Module This is an observational study assessing inhibitor formation in children with severe hemophilia A.Hemophilia Natural History Arm This Arm is investigating the safety, effectiveness, and practice of treatment for people with hemophilia.Hemlibra® Module All participants treated with Hemlibra® are eligible to participate.Rebinyn® Module The Rebinyn® Module is a prospective study in hemophilia B participants without inhibitors.Hemophilia Gene Therapy Outcomes Arm This Arm is investigating the safety and effectiveness of gene therapy in people with hemophilia.HEMGENIX® Module This is an observational study to characterize the effectiveness and safety of HEMGENIX® in participants with hemophilia B.Congenital Platelet Disorders (CPD) Natural History Arm:The CPD Arm is investigating the safety and efficacy of hemostatic therapies in the prevention or treatment of bleeding events in adult and pediatric participants with inherited congenital platelet disorders.Glanzmann Thrombasthenia (GT) Module:This Module is a study of bleeding symptoms, treatments, and treatment outcomes in patients with Glanzmann thrombasthenia.Von Willebrand Disease Cohort No arms or modules open at this time.Rare Disorders Cohort No arms or modules open at this time.Bleeding NOS No arms or modules open at this time.Thrombosis/Thrombophilia No arms or modules open at this time.Non-Neoplastic Hematologic Conditions No arms or modules open at this time..

Medienart:	Klinische Studie

Erscheinungsjahr:	2024
Erschienen:	2024

Enthalten in:	ClinicalTrials.gov - (2024) vom: 18. Apr. Zur Gesamtaufnahme - year:2024

Sprache:	Englisch

Links:	Volltext [kostenfrei]

Themen:	610 Anemia, Sickle Cell Blood Coagulation Disorders Blood Platelet Disorders Connective Tissue Diseases Hematologic Diseases Hemophilia A Hemophilia B Hemorrhage Hemorrhagic Disorders Hemostatic Disorders Recruitment Status: Recruiting Study Type: Observational Thalassemia Thrombophilia Thrombosis Von Willebrand Diseases

Anmerkungen:	Source: Link to the current ClinicalTrials.gov record., First posted: May 21, 2020, Last downloaded: ClinicalTrials.gov processed this data on April 24, 2024, Last updated: April 24, 2024

Study ID:

NCT04398628 ATHN Transcends

Veröffentlichungen zur Studie:

Weijer C, Freedman B, Fuks A, Robbins J, Shapiro S, Skrutkowska M. What difference does it make to be treated in a clinical trial? A pilot study. Clin Invest Med. 1996 Jun;19(3):179-83. Braunholtz DA, Edwards SJ, Lilford RJ. Are randomized clinical trials good for us (in the short term)? Evidence for a "trial effect". J Clin Epidemiol. 2001 Mar;54(3):217-24. doi: 10.1016/s0895-4356(00)00305-x. West J, Wright J, Tuffnell D, Jankowicz D, West R. Do clinical trials improve quality of care? A comparison of clinical processes and outcomes in patients in a clinical trial and similar patients outside a trial where both groups are managed according to a strict protocol. Qual Saf Health Care. 2005 Jun;14(3):175-8. doi: 10.1136/qshc.2004.011478. Unger JM, Barlow WE, Martin DP, Ramsey SD, Leblanc M, Etzioni R, Hershman DL. Comparison of survival outcomes among cancer patients treated in and out of clinical trials. J Natl Cancer Inst. 2014 Mar;106(3):dju002. doi: 10.1093/jnci/dju002. Epub 2014 Mar 13. https://www.fda.gov/drugs/resources-information-approved-drugs/hematologyoncology-cancer-approvals-safety-notifications. Accessed 04 Jul 2019 https://www.clinicaltrials.gov/ct2/results?cond=Hematologic+Diseases&term=&cntry=&state=&city=&dist=. Accessed 04 Jul 2019 Konkle BA, Recht M; members of Working Group 2, the NHLBI State of the Science Workshop on factor VIII inhibitors: Generating a national blueprint for future research. The national blueprint for 21st century data and specimen collection and observational cohort studies: NHLBI State of the Science Workshop on factor VIII inhibitors. Haemophilia. 2019 Jul;25(4):590-594. doi: 10.1111/hae.13772. Iorio A, Keepanasseril A, Foster G, Navarro-Ruan T, McEneny-King A, Edginton AN, Thabane L; WAPPS-Hemo co-investigator network. Development of a Web-Accessible Population Pharmacokinetic Service-Hemophilia (WAPPS-Hemo): Study Protocol. JMIR Res Protoc. 2016 Dec 15;5(4):e239. doi: 10.2196/resprot.6558.

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PPN (Katalog-ID):	CTG003399680