[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT04872179":3,"trial-entities:NCT04872179":97,"trial-summary:NCT04872179":102},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":22,"study_type":23,"primary_purpose":24,"phases":25,"enrollment_info":26,"interventions":29,"primary_outcomes":30,"secondary_outcomes":39,"sex":54,"minimum_age":24,"maximum_age":24,"healthy_volunteers":15,"eligibility_criteria":55,"std_ages":61,"locations":65,"central_contacts":79,"overall_officials":85,"references":87,"see_also_links":96},"NCT04872179","16-21157-B","International Registry of Patients With Alpha Thalassemia","International Prospective Registry of Patients With Alpha Thalassemia","RECRUITING","2037-01","2025-04","2025-04-04","2017-01","University of California, San Francisco","OTHER",false,"This is an international prospective registry of patients with Alpha thalassemia to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with Alpha thalassemia.","The aim of this registry is to prospectively and retrospectively collect data on patients who are diagnosed with alpha thalassemia major and other alpha thalassemia mutations. Data collected will be used to:\n\n1. Identify patient outcomes of therapies.\n2. Improve clinical management of patients with ATM.\n3. Improve medical decision making.\n4. Improve quality of care.",[19,20,21],"Alpha-Thalassemia","Alpha Thalassemia Major","Alpha Thalassemia Minor",[],"OBSERVATIONAL",null,[],{"count":27,"type":28},500,"ESTIMATED",[],[31,35],{"measure":32,"description":33,"timeFrame":34},"Survival to birth","Number of fetuses diagnosed with alpha thalassemia who survive to birth, compared to number of fetuses diagnosed with alpha thalassemia who have fetal demise or are terminated in utero. This is measured in number of fetuses alive at birth divided by number of all fetuses.","6 months",{"measure":36,"description":37,"timeFrame":38},"Vineland-3 Adaptive Behavior Scale","Results of neurodevelopmental testing using the Vineland Adaptive Behavior Scale version 3. The Vineland-3 scoring system is based on scores for three specific adaptive behavior domains: Communication, Daily Living Skills, and Socialization. The domain scores are expressed as standard scores with a mean of 100 and standard deviation of 15.","10-15 years",[40,43,47,51],{"measure":41,"description":42,"timeFrame":34},"Gestational age at birth","Gestational age of the child at birth. This is measured in weeks.",{"measure":44,"description":45,"timeFrame":46},"Mechanical ventilation","Duration (if any) of requiring mechanical ventilation after birth. This is measured in days.","1 year",{"measure":48,"description":49,"timeFrame":50},"Length of hospitalization","Duration of the child's hospitalization after birth. This is measured in days.","6 months-1 year",{"measure":52,"description":53,"timeFrame":34},"Resolution of hydrops","Evaluate whether receiving fetal therapy leads hydrops fetalis to resolve. This is measured by ultrasound findings.","ALL",{"inclusion":56,"exclusion":59,"raw_text":60},[57,58],"diagnosis of alpha thalassemia (prenatal or postnatal) with genotype consistent with ATM or BHFS phenotype","referred to the University of California, San Francisco Fetal Treatment Center for fetal diagnosis, management and\u002For evaluation for the ongoing in utero stem cell transplantation clinical trial",[],"Inclusion Criteria:\n\n* diagnosis of alpha thalassemia (prenatal or postnatal) with genotype consistent with ATM or BHFS phenotype\n* referred to the University of California, San Francisco Fetal Treatment Center for fetal diagnosis, management and\u002For evaluation for the ongoing in utero stem cell transplantation clinical trial\n\nExclusion Criteria:\n\n\\- none",[62,63,64],"CHILD","ADULT","OLDER_ADULT",[66],{"facility":67,"status":8,"city":68,"state":69,"zip":70,"country":71,"contacts":72,"geoPoint":76},"University of California San Francisco","San Francisco","California","94143","United States",[73],{"name":74,"role":75},"Tippi C MacKenzie, MD","PRINCIPAL_INVESTIGATOR",{"lat":77,"lon":78},37.77493,-122.41942,[80],{"name":81,"role":82,"phone":83,"email":84},"Billie Lianoglou, LCGC","CONTACT","(415) 476-2461","billie.lianoglou@ucsf.edu",[86],{"name":74,"affiliation":13,"role":75},[88,92],{"pmid":89,"type":90,"citation":91},"27862048","BACKGROUND","Kreger EM, Singer ST, Witt RG, Sweeters N, Lianoglou B, Lal A, Mackenzie TC, Vichinsky E. Favorable outcomes after in utero transfusion in fetuses with alpha thalassemia major: a case series and review of the literature. Prenat Diagn. 2016 Dec;36(13):1242-1249. doi: 10.1002\u002Fpd.4966. Epub 2016 Dec 7.",{"pmid":93,"type":94,"citation":95},"36306387","DERIVED","Schwab ME, Lianoglou BR, Gano D, Gonzalez Velez J, Allen IE, Arvon R, Baschat A, Bianchi DW, Bitanga M, Bourguignon A, Brown RN, Chen B, Chien M, Davis-Nelson S, de Laat MWM, Ekwattanakit S, Gollin Y, Hirata G, Jelin A, Jolley J, Meyer P, Miller J, Norton ME, Ogasawara KK, Panchalee T, Schindewolf E, Shaw SW, Stumbaugh T, Thompson AA, Towner D, Tsai PS, Viprakasit V, Volanakis E, Zhang L, Vichinsky E, MacKenzie TC. The impact of in utero transfusions on perinatal outcomes in patients with alpha thalassemia major: the UCSF registry. Blood Adv. 2023 Jan 24;7(2):269-279. doi: 10.1182\u002Fbloodadvances.2022007823.",[],{"nct_id":4,"conditions":98,"biomarkers":100},[99],"Alpha Thalassemia",[101],"Hemoglobin Subunit Alpha",{"nct_id":4,"found":103,"summary":104,"prompt_version":114},true,{"design":105,"status":106,"heading":107,"summary":108,"follow_up":109,"word_count":110,"commitments":111,"compensation":112,"drugs_mentioned":113},"This is an observational study, meaning researchers will collect information without giving any specific intervention. It plans to include 500 participants.","completed","International Registry for Alpha Thalassemia","This study is an international registry for people with alpha thalassemia, including alpha thalassemia major and minor. It aims to gather information about how the disease progresses naturally and how different treatments given before birth (fetal therapies) affect patients. The goal is to improve how alpha thalassemia is managed before birth. You can join if you have an alpha thalassemia diagnosis (before or after birth) with a specific genetic makeup (genotype) and have been referred to the University of California, San Francisco Fetal Treatment Center for diagnosis, management, or evaluation for an ongoing stem cell transplant trial. The study will look at how long patients survive after birth (measured at 6 months) and their adaptive behaviors (measured at 10-15 years). The current recruitment status is unclear.","Participants will be followed to see survival at 6 months after birth and adaptive behavior between 10-15 years of age.",126,"Not specified in the trial record.","Not stated in the trial record.",[],"v2"]