[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Joanne Kurtzberg, MD\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":120},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,34,77],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":16,"enrollmentInfo":4,"targetDuration":4,"studyType":17,"phases":4,"briefSummary":18,"conditions":19,"keywords":4,"overallStatus":25,"whyStopped":4,"lastUpdateSubmitDate":26,"lastUpdatePostDateStruct":27,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":30,"locationsCount":33},"100310906","expanded-access-protocol-umbilical-cord-blood-infusions-for-children-with-brain-injuries-100310906",false,"NCT03327467","Expanded Access Protocol: Umbilical Cord Blood Infusions for Children With Brain Injuries","Under this protocol, participants may be eligible for infusion of autologous, sibling, and\u002For unrelated donor CB cells (Duke only). Some eligibility criteria vary based on CB source. For all participants, eligibility is predicated on the availability of a qualifying CB unit To be eligible, patients must:\n\nInclusion Criteria:\n\n1. Age at Consent:\n\n   1. Autologous: 0-26 years\n   2. Sibling: 6 months - 26 years\n   3. Unrelated Donor (Duke only): 6 months - 26 years\n2. Diagnosis\n\n   1. Autologous and Sibling: Cerebral palsy, Hypoxic brain injury, Stroke, Hydrocephalus, Apraxia (without autism), Other brain injury\n   2. Unrelated Donor (Duke only): Motor impairment secondary to: Hypoxic brain injury, Periventricular leukomalacia, Stroke\u002Fbleed, Congenital hydrocephalus\n3. Functional Status:\n\n   1. Autologous and Sibling: See exclusion criteria\n   2. Unrelated Donor (Duke only): GMFCS level I-IV (age ≥2)\n4. No clinically significant abnormalities in blood counts and basic chemistries for age.\n5. Absolute Lymphocyte Count:\n\n   * 1200 for African American participants\n   * 1500 for all other participants\n6. Availability of a qualified autologous or Sibling CB Unit\n\n   1. Autologous or Sibling: available\n   2. Unrelated Donor (Duke only): N\u002FA\n7. Written informed consent obtained from the parent or legal guardian.\n\nExclusion Criteria:\n\n1. Documented HIV or Hepatitis or other disease transmittable through the blood.\n2. A cord blood unit that fails to meet specifications\n3. Refusal of consent\n4. Uncontrolled seizure disorder\n5. Uncontrolled infection\n6. Diagnosed with a genetic or metabolic disorder related to the neurologic condition\n7. History of an immune deficiency\n8. History of treatment with chemo or immunosuppressive therapy\n9. History of previous allogeneic cell therapy outside of participation in a Duke clinical trial\n10. Need for mechanical ventilation or chronic O2 support\n11. Unstable airway\n12. Eligible for an active clinical trial of cellular therapy at Duke. If previously enrolled on another Duke cell therapy protocol the participant may still be considered for enrollment on this study after the follow-up period for the clinical trial is completed.\n13. Cerebral palsy secondary to infection, congenital or otherwise, without another known cause\n14. Pregnant or breastfeeding\n15. Treated with autologous cellular therapy through a non-intravenous route (ie. intrathecally) within one year prior to enrollment.\n\nUmbilical Cord Blood Criteria\n\nParticipant enrollment is dependent on the availability of a banked unit of autologous or sibling CB that has been stored at a private or public bank, or, if enrolling at Duke, availability of a publicly banked unrelated donor unit. Units must meet the following criteria:\n\nPrecryopreservation:\n\n1. TNCC ≥2x107\u002Fkg\n2. Sterility cultures performed and negative\n3. Viability ≥70%\n4. At least haploidentical HLA match for sibling units and at least a 4\u002F6 HLA match for unrelated donor units\n5. Donor screening testing performed and negative\n\nCBU Test sample\n\n1. Segment or test vial available\n2. Identity confirmed via HLA typing of test sample and donor\n3. Viability testing recommended, but not required","ALL","26 Years","EXPANDED_ACCESS","This protocol is designed to enable access to intravenous infusions of banked umbilical cord blood (CB), that is thawed and not more than minimally manipulated, for children with various brain disorders. Children with cerebral palsy, congenital hydrocephalus, apraxia, stroke, hypoxic brain injury and related conditions will be eligible if they have normal immune function and do not qualify for, have previously participated in, or are unable to participate in an active cell therapy clinical trial at Duke Medicine. For the purpose of this protocol the term children refers to patients less than 26 years of age. Cord blood is administered as a cellular infusion without prior treatment with chemotherapy or immunosuppression. The mechanism of action is through paracrine signaling of cord blood monocytes inducing endogenous cells to repair existing damage.",[20,21,22,23,24],"Cerebral Palsy","Hydrocephalus","Apraxia of Speech","Hypoxia Ischemia, Cerebral","Drowning; Anoxia","AVAILABLE","2026-01-22",{"date":28,"type":29},"2026-01-23","ACTUAL",{"name":31,"class":32},"Joanne Kurtzberg, MD","OTHER",2,{"id":35,"slug":36,"hasResults":11,"nctId":37,"briefTitle":38,"officialTitle":39,"acronym":4,"eligibilityCriteria":40,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":41,"enrollmentInfo":4,"targetDuration":4,"studyType":17,"phases":4,"briefSummary":42,"conditions":43,"keywords":47,"overallStatus":25,"whyStopped":4,"lastUpdateSubmitDate":72,"lastUpdatePostDateStruct":73,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":75,"locationsCount":76},"100200115","expanded-access-to-t-cell-depleted-haplo-identical-stem-cells-for-patients-receiving-haplo-identical-and-unrelated-cord-blood-transplants-100200115","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.","65 Years","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.",[44,45,46],"Hematologic Malignancies","Inborn Errors of Metabolism Disorders","Immune Deficiencies",[48,49,50,51,52,53,54,55,56,57,15,58,59,60,61,62,63,64,65,66,67,68,69,70,71],"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","2025-11-17",{"date":74,"type":29},"2025-11-20",{"name":31,"class":32},1,{"id":78,"slug":79,"hasResults":11,"nctId":80,"briefTitle":81,"officialTitle":82,"acronym":83,"eligibilityCriteria":84,"healthyVolunteers":11,"sex":15,"minAge":85,"maxAge":86,"enrollmentInfo":87,"targetDuration":4,"studyType":90,"phases":91,"briefSummary":93,"conditions":94,"keywords":106,"overallStatus":111,"whyStopped":4,"lastUpdateSubmitDate":112,"lastUpdatePostDateStruct":113,"startDateStruct":115,"completionDateStruct":117,"leadSponsor":119,"locationsCount":76},"100228697","phase-1-ucb-transplant-of-inherited-metabolic-diseases-with-administration-of-intrathecal-ucb-derived-oligodendrocyte-like-cells-100228697","NCT02254863","UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells","Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like Cells","DUOC-01","Inclusion Criteria:\n\n1. Patients must be age ≥1 week to ≤21 years.\n2. Patients must have one of the following inherited metabolic diseases detected by enzyme or mutation analysis, and confirmed by repeat testing on a separately obtained sample:\n\n   Adrenoleukodystrophy (ALD) Batten Disease Hunter Syndrome (MPS II) Krabbe disease (Globoid Leukodystrophy) Metachromatic Leukodystrophy (MLD) Niemann Pick disease type A or B Pelizaeus-Merzbacher disease (PMD) Sandhoff disease Tay Sachs disease. Alpha Mannosidosis Sanfilippo (MPS III)\n3. Patients must have neurologic evidence of their disease, either clinically or via neuroimaging or neurophysiological testing. Examples of evidence of neurologic involvement include, but are not limited to the following:\n\n   * Abnormal EEG, Brainstem Auditory Evoked Response (BAER), and\u002For Visual Evoked Potentials (VEP).\n   * Abnormal brain MRI, ie. increased Loes score (measure of white matter damage, demyelination, and brain atrophy) and\u002For abnormal corticospinal tracts as assessed by MRI with diffusion tensor imaging (DTI).\n   * Three or more of the early clinical markers: problems sleeping, increased activity, behavior difficulties, seizure-like activity, chewing behavior, inappropriate bladder training, inappropriate bowel training.\n4. Patients must have adequate organ function as measured by:\n\n   * Renal: Serum creatinine ≤ 2.0 mg\u002Fdl\n   * Hepatic: Hepatic transaminases (ALT\u002FAST) ≤ 5 x normal, bilirubin ≤ 2.0 mg\u002Fdl (except in patients with Gilbert's disease or newborns with physiological or breast milk associated jaundice).\n   * Cardiac: Normal cardiac function by echocardiogram or radionuclide scan (shortening fraction or ejection fraction\n\n     * 80% of normal value for age). Patients with acquired or congenital cardiomyopathy may receive melphalan as a substitute for cyclophosphamide.\n   * Pulmonary: Pulmonary function tests demonstrating FVC, FEV1, and DLCO ≥ 60% of predicted in patients who can complete the testing. If patient cannot perform PFT's, an O2 sat must be \\>90% on room air.\n5. Patients must have an available, suitably matched, banked UCB unit for transplant.\n6. Patients must have a performance status as follows: Lansky ≥ 40%, or Karnofsky ≥ 40%\n7. Patients must have a life expectancy of ≥ 6 months.\n\nExclusion Criteria:\n\n1. Prior organ, tissue, or stem cell transplant within 3 years of study entry.\n2. Prior participation in any gene or regenerative cell therapy study.\n3. Inability to have an MRI scan or lumbar puncture.\n4. Intractable seizures.\n5. Chronic aspiration.\n6. Bleeding disorder.\n7. Evidence of HIV infection or HIV positive serology.\n8. Uncontrolled bacterial, viral, or fungal infection at the time of pre-UCBT cytoreduction.\n9. Inability to obtain patient's, parent's or legal guardian's consent.\n10. Requirement of ventilatory support.\n11. Pregnant or breastfeeding.\n12. Active concurrent malignancy, or receiving concurrent radiotherapy, immunosuppressive medications, or cytotoxic chemotherapy","1 Week","22 Years",{"count":88,"type":89},40,"ESTIMATED","INTERVENTIONAL",[92],"PHASE1","The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of metabolism who have evidence of early demyelinating disease in the central nervous system (CNS) who are undergoing standard treatment with unrelated umbilical cord blood transplantation (UCBT). The secondary objective of the study is to describe the efficacy of UCBT with intrathecal administration of DUOC-01 in these patients.",[62,95,96,97,98,99,100,101,102,103,104,105],"Batten Disease","Mucopolysaccharidosis II","Leukodystrophy, Globoid Cell","Leukodystrophy, Metachromatic","Neimann Pick Disease","Pelizaeus-Merzbacher Disease","Sandhoff Disease","Tay-Sachs Disease","Brain Diseases, Metabolic, Inborn","Alpha-Mannosidosis","Sanfilippo Mucopolysaccharidoses",[62,95,107,64,108,109,110,65],"Hunter Syndrome","Metachromatic Leukodystrophy","ALD","MLD","RECRUITING","2025-09-02",{"date":114,"type":29},"2025-09-08",{"date":116,"type":4},"2014-09",{"date":118,"type":89},"2026-10",{"name":31,"class":32},""]