Symptoms and General Pathology

All Conditions

Neoplasms

Blood and Lymph Conditions

Diseases and Abnormalities at or Before Birth

Immune System Diseases

Nutritional and Metabolic Diseases

Rare Diseases

Syndrome

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Preleukemia

Myelodysplastic Syndromes

Anemia, Sickle Cell

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Thalassemia

beta-Thalassemia

Hematologic Neoplasms

Immunologic Deficiency Syndromes

Metabolism, Inborn Errors

Hematologic Diseases

Severe Combined Immunodeficiency

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Sickle Cell Anemia

Acute Lymphoblastic Leukemia

Lymphoblastic Lymphoma

Beta-thalassemia

Acute Graft Versus Host Disease

all subjects will be treated with UCB and HPDSC

single dose of UCB followed by one unit of HPDSC

UCB and HPDSC

Lolie Yu, MD

To investigate the safety of partially matched related human placental-derived stem cells (HPDSC) administered in conjunction with umbilical cord blood (UCB) stem cells from the same donor in subjects with various malignant or nonmalignant disorders potentially curable with stem cell transplantation and to assess potential restoration of normal hematopoiesis and immune function in subjects with these disorders

A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders

A Single-Arm Study to Assess the Safety of Transplantation With Umbilical Cord Blood Augmented With Human Placental-Derived Stem Cells From Partially Matched Related Donors in Subjects With Certain Malignant Hematologic Diseases and Non-Malignant Disorders

Celgene Corporation

Nervous System Diseases

Skin and Connective Tissue Diseases

Anemia

Anemia, Aplastic

Mucopolysaccharidosis II

Primary Immunodeficiency Diseases

Genetic Diseases, Inborn

Metabolic Diseases

Bone Marrow Failure Disorders

Pancytopenia

Bone Marrow Diseases

Aplastic Anemia

Hurler Syndrome

Mucopolysaccharidosis Type II

20% of UCB is ALDHbr sorted and primed and give on transplant day after conventional graft

20% of UCB is ALDHbr freshly sorted and give on transplant day 4-8 hrs after conventional graft

patient receives 1 conventional UCB unit and 1 unit that has been ALDHbr sorted and primed

Patient receives 1 UCB unit and a second UCB unit that has been freshly ALDHbr sorted

ALDHbr Umbilical Cord Blood Cells

Joanne Kurtzberg, MD

The main purpose of this investigational (not approved by the FDA) Phase I research is to test whether transplantation of umbilical cord blood cells can be safely supplemented with a transfusion of a portion of these cells that have been sorted (collected from a special machine called a cell sorter) and then either infused a few hours after the standard transplant or for some patients grown in a special system in the laboratory prior to the transplant, designed to increase the number of stem cells transplanted. This system is currently in the early phases of testing.

The main purpose of this study is to test whether transplantation of umbilical cord blood cells can be safely supplemented with transfusion of a portion of these cells that have been grown in a special system (designed to increase the number of cells transplanted) in the laboratory prior to the transplant. This system is currently in the early phases of testing in a FDA-IND-sponsored clinical trial. If the patient consents to participate in this study, approximately 1/5th (20%) of the cord blood unit selected for the transplant will be treated per protocol. The first 3 patients will receive ALDHbr sorted cells but not primed in culture. This is to test the safety of the ALDHbr cells. The treated cells will be given to the patient on the day of transplant approximately 4 hours after the standard or conventional transplant which will be given from the 80% fraction of the cord blood unit. A total of 26 evaluable patients are to be enrolled as outlined below (protocol has been amended to allow this enrollment):

* 10 evaluable patients who received ALDHbr freshly sorted cells (20% portion)
* 10 evaluable patients who received ALDHbr sorted and cytokine primed cells (20% portion)
* 3 evaluable patients who received a conventional cord blood unit and a cord blood unit that has been ALDHbr sorted (sort of the UCB unit will be done on Day -1 due to the time it takes to actually perform the sort)
* 3 evaluable patients who received a conventional cord blood unit and a cord blood unit that has been ALDHbr sorted and cytokine primed (sort and priming will be done on day -5 as described later in the protocol)

Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells

A Pilot Trial of Unrelated Umbilical Cord Blood Transplantation Augmented With Ex Vivo CytokinePrimed ALDHbr Umbilical Cord Blood Cells

Aldagen

Professor of Pediatrics

Respiratory Tract (Lung and Bronchial) Diseases

Asthma

Hemolysis

Anemia, Hemolytic

Anemia, Hemolytic, Congenital

Hemoglobinopathies

Participants will be receiving their standard medical care.

Participants will be receiving the intervention on top of their standard medical care.

A sphygmanometer, a digital thermometer, a pulse oximeter and a peak flow meter will be given to participants in the intervention arm to use during their telemedicine consultations with health care providers.

A sphygmanometer, a digital thermometer, a pulse oximeter and a peak flow meter will be given to participants to use during their telemedicine consultations with health care providers.

Maria Ntessalen

Peter Murchie

This project assesses feasibility of providing medically vulnerable rural patients with Medical-Self-Assessment-Boxes containing equipment to use at home during telephone and video consultations (telemedicine) with GPs and other healthcare professionals. COVID-19 has caused an upsurge in primary care telemedicine which the investigators believe can be sustained and optimized to make things better for medically vulnerable rural patients beyond the pandemic. The investigators will achieve this by equipping the participants to self-measure and report key clinical measurements (e.g. blood pressure, temperature, oxygen levels) during telemedicine consultations. Before conducting a major evaluation of the Medical-Self-Assessment-Box for medically vulnerable rural patients the investigators must establish three things: First, to show the investigators can issue a Medical-Self-Assessment-Box to medically vulnerable rural patients and enable them to use it properly. Second, to determine that patients can use the Medical-Self-Assessment-Box effectively during telemedicine consultations. Third, to show that it is possible to measure how well the Medical-Self-Assessment-Box is working by counting how often the boxes are being used and whether use is appropriate and helpful. The knowledge gained will provide the investigators with the information needed to develop a funding proposal to evaluate Medical-Self-Assessment-Boxes for medically vulnerable rural patients in the whole of the UK.

ORCHARD- Optimising Home Assessment of Rural Patients

Optimising Remote Consulting and Home Assessment of Medically Vulnerable Rural Patients During Unscheduled and Planned Primary Care- Assessing Feasibility of the ORCHARD Intervention

The questionnaire will assess use of equipment, perception of how helpful the equipment was for the consultation and the reasons why it was helpful.

Qualitative data collected at the end of the study according to a topic guide.

Number of Scottish ambulance service call-outs

Number of face-to-face paramedic assessment

University of Aberdeen

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders	NCT00596999	Hematologic Mal...	UCB and HPDSC	- 55 Years	Celgene
Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells	NCT00692926	MDS Anemia, Aplasti... Inborn Errors o... Congenital Marr... Congenital Immu...	ALDHbr Umbilica...	- 55 Years	Duke University
ORCHARD- Optimising Home Assessment of Rural Patients	NCT05732922	Organ Transplan... Cancer Inborn Errors o... Immunosuppressi... COPD Asthma Sickle Cell Dis...	A sphygmanomete...	18 Years - 100 Years	University of Aberdeen
A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders	NCT00596999	Hematologic Mal...	UCB and HPDSC	- 55 Years	Celgene
ORCHARD- Optimising Home Assessment of Rural Patients	NCT05732922	Organ Transplan... Cancer Inborn Errors o... Immunosuppressi... COPD Asthma Sickle Cell Dis...	A sphygmanomete...	18 Years - 100 Years	University of Aberdeen
ORCHARD- Optimising Home Assessment of Rural Patients	NCT05732922	Organ Transplan... Cancer Inborn Errors o... Immunosuppressi... COPD Asthma Sickle Cell Dis...	A sphygmanomete...	18 Years - 100 Years	University of Aberdeen

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