[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"inborn-errors-of-metabolism-disorders\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:inborn-errors-of-metabolism-disorders":22},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,58],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":17,"enrollmentInfo":4,"targetDuration":4,"studyType":18,"phases":4,"briefSummary":19,"conditions":20,"keywords":24,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":50,"lastUpdatePostDateStruct":51,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":54,"locationsCount":57},"100200115","expanded-access-to-t-cell-depleted-haplo-identical-stem-cells-for-patients-receiving-haplo-identical-and-unrelated-cord-blood-transplants-100200115",false,"NCT01881334","Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants","A Compassionate Release Protocol: Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Allogeneic Transplantation Using a Related Haplo-Identical Donor and Unrelated, Umbilical Cord Blood Donor(s) for the Treatment of High Risk Malignancies or Non-Malignant Disorders Requiring Allogeneic Transplantation","Inclusion Criteria:\n\n* Have a consenting related haplo-identical (3\u002F6, 4\u002F6, or 5\u002F6 if DRB1 mismatch) stem cell donor.\n* Have one or two available 4, 5, or 6\u002F6 antigen matching unrelated UCB unit(s) that will deliver a total cell dose \\>3.0 x 10e7 cells\u002Fkg. Patients who do not have a single UCB unit that will deliver the minimum required cell dose, two partially HLA-matched UCB units which together meet the minimum cell dose requirement, can be used for 1 transplant. These units must be HLA-matched minimally at 4 of 6 HLA-A and B (at intermediate resolution by molecular typing) and DRB1 (at high resolution by molecular typing) loci with the patient, and HLA-matched at 3 of 6 HLA- A, B, DRB1 loci with each other (using same resolution of HLA typing as indicated above). There is no limitation on maximum cell dose.\n* Have a high risk or refractory malignancy, or non-malignant disorder amenable to stem cell transplantation therapy.\n* Meet eligibility requirements for allogeneic transplant per institutional standard practices.\n* Have given written informed consent according to FDA guidelines (or consent of parent\u002Flegal guardian as applicable).\n* Be \\\u003C65 years of age at the time of study enrollment.\n\nExclusion Criteria:\n\n* Have a consenting 8\u002F8 or 10\u002F10 allele matched, consenting, related or unrelated hematopoietic stem cell transplant (HSCT) donor.\n* Have a life expectancy of less than 3 months.\n* Have uncontrolled infections at time of cytoreduction.","ALL","65 Years","EXPANDED_ACCESS","The objective of this study is to make T-cell depleted stem cells from a family member who is a half match (haplo-identical) available on an expanded access basis to patients receiving one or two unrelated cord blood transplants who are at a higher risk of not engrafting in a safe amount of time. The purpose of the related stem cells is to give the bone marrow a \"jump start\" towards recovery. Ultimately, the cord blood cells will grow and permanently rescue the bone marrow.",[21,22,23],"Hematologic Malignancies","Inborn Errors of Metabolism Disorders","Immune Deficiencies",[25,26,27,28,29,30,31,32,33,34,16,35,36,37,38,39,40,41,42,43,44,45,46,47,48],"Haploidentical Donor","T-cell depleted Stem Cells","Allogeneic Transplant","Umbilical Cord Blood Donor","High Risk Malignancies","Metabolic Disorders","Immune Deficiency","Acute Lymphoblastic Leukemia","Acute Myelogenous Leukemia","Myelodysplastic Syndrome","AML","MDS","CGD","SCID","Adrenoleukodystrophy","Metachromaticleukodystrophy","Krabbe","PMD","Hunter's","Hurler's","Severe Aplastic Anemia","Lymphoma","Sickle Cell Disease","Thalassemia","AVAILABLE","2025-11-17",{"date":52,"type":53},"2025-11-20","ACTUAL",{"name":55,"class":56},"Joanne Kurtzberg, MD","OTHER",1,{"id":59,"slug":60,"hasResults":11,"nctId":61,"briefTitle":62,"officialTitle":63,"acronym":4,"eligibilityCriteria":64,"healthyVolunteers":11,"sex":16,"minAge":65,"maxAge":66,"enrollmentInfo":67,"targetDuration":4,"studyType":70,"phases":71,"briefSummary":73,"conditions":74,"keywords":78,"overallStatus":87,"whyStopped":4,"lastUpdateSubmitDate":88,"lastUpdatePostDateStruct":89,"startDateStruct":91,"completionDateStruct":93,"leadSponsor":95,"locationsCount":97},"100572152","education--care-in-rare-efficacy-of-targeted-psychoeducational-intervention-among-pediatric-rare-disease-patients-100572152","NCT06729554","Education & Care in RARE: Efficacy of Targeted Psychoeducational Intervention Among Pediatric Rare Disease Patients","Education & Care in RARE - Efficacy of Targeted Psychoeducational Intervention to Improve Knowledge About Rare Diseases and to Promote Mental Health Among Pediatric Rare Disease Patients","Inclusion Criteria:\n\n* Children and adolescents with a confirmed diagnosis of a rare disease with\n* Age 5-20 years, corresponding to a developmental age of 5-18 years\n* Existing medical care at a participating study center because of the rare disease\n* Voluntary participation and informed consent\n* Ability to complete the questionnaires\n* Ability to actively participate the intervention (psychoeducation)\n\nExclusion Criteria:\n\n* Moderate or severe cognitive impairment\n* Simultaneous admission of the child \u002F adolescent to a setting with high-frequency psychotherapeutic intervention (e.g. admission to psychosomatic medicine, child and adolescent psychiatry)\n* No informed consent\n* Language barrier of the child \u002F adolescent\n* Assumption that compliance is too low to attend all study appointments","5 Years","20 Years",{"count":68,"type":69},100,"ESTIMATED","INTERVENTIONAL",[72],"NA","\"Rare Diseases\" is an umbrella term including more than 8.000 different diseases which individually affect only a small percentage of people. Rare diseases predominantly affect children and adolescents and are associated with high medical and psychosocial burden of disease.\n\nThe investigators invented Education \\& Care in RARE - a short-term, structured, resource-oriented and child-friendly psychoeducation program for children and adolescents with rare diseases.\n\nThis study is a prospective, multicenter, randomized and controlled study with a waiting list. Aim of the study is to investigate the efficacy of Education \\& Care in RARE on knowledge about rare diseases and on mental health well-being in pediatric rare disease patients, compared to a control group.\n\nIn this study participants are randomized in an intervention group and a waiting list control group. Both study groups thus receive the psychoeducation with Education \\& Care in RARE and complete the identical questionnaires. Compared to the Intervention group, the waiting list control group receives the intervention with a time delay (8-12 weeks later) and has one additional appointment for questionnaire evaluation before start of the psychoeducation.",[75,76,77,22],"Orphan Diseases","Rare Disorders","Pediatric Diseases",[79,80,81,82,83,84,85,86],"Rare diseases","ultra-rare diseases","psychoeducation in pediatrics","psychoeducation for alle rare diseases","rare pediatric diseases","orphan diseases psychoeducation","rare disease burden","waiting list control study","RECRUITING","2025-05-12",{"date":90,"type":53},"2025-05-13",{"date":92,"type":53},"2024-12-15",{"date":94,"type":69},"2027-12-01",{"name":96,"class":56},"Medical University of Vienna",7]