Clinical trials: Lymphatic Insufficiency
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Anaemia
Anemia Studies in CKD: Erythropoiesis Via a Novel PHI Daprodustat - Pediatric (ASCEND-P)
This is an international, multicenter trial, evaluating pharmacokinetics (PK) (4 weeks), safety (52 weeks), and hemoglobin (Hgb) response (52 weeks) to daprodustat in children and adolescent participants with anemia associated…
- Trial name
- ASCEND-P (NCT05682326)
- Testing
- Daprodustat
- Where
- 4 sites in Japan, South Korea
- Volunteers
- 4 people
- Sponsor
- GlaxoSmithKline
Thalassemia, Sickle Cell Disease
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01319851
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
Osteosarcoma, Leukemia
Open-label Leucovorin Pharmacokinetic Study in Patients Receiving High Dose Methotrexate With or Without Voraxaze
The purpose of this study is to investigate whether the administration of Voraxaze reduces exposure to leucovorin and its active metabolite to below the level achieved in patients who have not received Voraxaze.
- Conditions
- Osteosarcoma, Leukemia, Lymphoma
- Trial name
- LVPK (NCT00634504)
- Testing
- glucarpidase, high-dose methotrexate, leucovorin, high-dose methotrexate, leucovorin
- Where
- 25 sites in United States
- Volunteers
- 20 people
- Sponsor
- BTG International Inc.
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This phase II trial studies how well giving fludarabine phosphate, melphalan, and low-dose total-body irradiation (TBI) followed by donor peripheral blood stem cell transplant (PBSCT) works in treating patients with hematologic…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01529827
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 94 people
- Sponsor
- Roswell Park Cancer Institute
Adrenoleukodystrophy, Metachromatic Leukodystrophy
Stem Cell Transplant for Inborn Errors of Metabolism
The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for an inherited metabolic…
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease
- Trial name
- NCT00176904
- Testing
- Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
- Where
- United States
- Volunteers
- 135 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Anemia of Chronic Kidney Disease
Vitamin D as a Modifier of Serum Hepcidin in Children With Chronic Kidney Disease
This research is being done to study the effectiveness of vitamin D (cholecalciferol) to modify hepcidin levels in children with chronic kidney disease (CKD). Anemia is a common problem in children with CKD.
- Trial name
- D-fense (NCT01532349)
- Testing
- Cholecalciferol
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Johns Hopkins University
Anemia, Kidney Diseases
DELFT (Dynepo Evaluation of Long-Term Follow-Up Treatment)
This is a post-authorisation safety study to assess the incidence and severity of all pre-defined cardiovascular events in patients treated with DYNEPO, as well as to detect \& describe less common adverse drug reactions, and to…
- Conditions
- Anemia, Kidney Diseases, Renal Failure, Chronic, Kidney Failure, Chronic
- Trial name
- DELFT (NCT00664066)
- Testing
- DYNEPO (epoetin delta)
- Where
- 2 sites in Germany
- Volunteers
- 3 people
- Sponsor
- Shire
Diamond-Blackfan Anemia, Kostmann's Neutropenia
Stem Cell Transplant for Bone Marrow Failure Syndromes
The researchers hypothesize that it will be possible to perform unrelated bone marrow or cord blood transplants in a safer manner by using less intensive therapy yet still achieve an acceptable level of donor cell engraftment for…
- Conditions
- Diamond-Blackfan Anemia, Kostmann's Neutropenia, Shwachman-Diamond Syndrome
- Trial name
- NCT00176878
- Testing
- Stem cell transplant, Fludarabine monophosphate, Total lymphoid irradiation
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Iron Deficiency Anemia
A Trial of Ferumoxytol for the Episodic Treatment of Iron Deficiency Anemia in Pediatric Participants With Chronic Kidney Disease
Study AMAG-FER-CKD-253 is an extension study of the combined AMAG-FER-CKD-251 (NCT01155375) and AMAG-FER-CKD-252 (NCT01155388) studies to evaluate the efficacy and safety of episodic treatment of iron deficiency anemia (IDA) with…
- Trial name
- NCT01264679
- Testing
- Ferumoxytol
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- AMAG Pharmaceuticals, Inc.
Anemia, Chronic Kidney Disease
Study to Assess Darbepoetin Alfa Dosing for the Correction of Anemia in Pediatric Patients With Chronic Kidney Disease
The primary objectives of this study are the following: 1. To test if the proportion of participants achieving a hemoglobin value greater than or equal to 10.0 g/dL at any time point after the first dose during the study is…
- Conditions
- Anemia, Chronic Kidney Disease, Kidney Disease
- Trial name
- NCT00436748
- Testing
- Darbepoetin Alfa
- Where
- 67 sites in United States, Belgium and 8 more
- Volunteers
- 116 people
- Sponsor
- Amgen
Non-Neoplastic Hematologic and Lymphocytic Disorder
Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
This phase II clinical trial studies how well treosulfan and fludarabine phosphate with or without low dose radiation before donor stem cell transplantation works in treating patients with nonmalignant (noncancerous) diseases.
- Trial name
- NCT00919503
- Testing
- Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine
- Where
- 6 sites in United States
- Volunteers
- 98 people
- Sponsor
- Fred Hutchinson Cancer Center
Anemia, Renal Insufficiency, Chronic
Ascertain the Optimal Starting Dose of Mircera Given Subcutaneously for Maintenance Treatment of Anemia in Pediatric Patients With Chronic Kidney Disease on Dialysis or Not Yet on Dialysis.
Ascertain the starting dose of Mircera given subcutaneously for the maintenance treatment of anemia in pediatric participants with chronic kidney disease (CKD) on dialysis or not yet on dialysis when switching from stable…
- Trial name
- NCT03552393
- Testing
- Mircera
- Where
- 22 sites in United States, France and 5 more
- Volunteers
- 40 people
- Sponsor
- Hoffmann-La Roche
Sickle Cell Disease, Thalassemia
Stem Cell Transplant for Hemoglobinopathy
This study tests the clinical outcomes of one of two preparative regimens (determined by available donor source) in patients with non-malignant hemoglobinopathies.
- Conditions
- Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia
- Trial name
- NCT00176852
- Testing
- Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Anemia
VIT45 Versus Oral Iron in the Treatment of Anemia in Non-Dialysis Dependent Chronic Kidney Disease
This study compares the efficacy and safety of intravenous (IV) iron (VIT45) versus oral iron (ferrous sulfate) administered to subjects who suffer from anemia and are diagnosed with non-dialysis dependent chronic kidney disease…
- Trial name
- NCT00317239
- Testing
- Ferrous Sulfate tablets, Ferric Carboxymaltose (FCM)
- Where
- United States
- Volunteers
- 255 people
- Sponsor
- American Regent, Inc.
Anemia, Chronic Kidney Disease
Safety and Efficacy of Iron Sucrose in Children
Comparison of three potential iron sucrose maintenance regimens in pediatric chronic kidney disease (CKD) patients
- Trial name
- NCT00239642
- Testing
- Venofer (iron sucrose injection)
- Where
- United States
- Volunteers
- 141 people
- Sponsor
- American Regent, Inc.
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT03333486
- Testing
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Roswell Park Cancer Institute
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Anemia
Long Term Safety Study of (VIT45) Extension Study: Treatment of Anemia in Non-Dialysis Dependent Chronic Kidney Disease
The primary objective of this study is to evaluate the long term safety and tolerability of an iron maintenance dosing strategy utilizing VIT45 in the treatment of anemia of non-dialysis dependent chronic kidney disease…
- Trial name
- NCT00317226
- Testing
- Ferric Carboxymaltose (FCM)
- Where
- United States
- Volunteers
- 145 people
- Sponsor
- American Regent, Inc.
Sickle Cell Disease, Chronic Kidney Diseases
Peer i-Coaching for Activated Self-Management Optimization in Adolescents and Young Adults With Chronic Conditions
The purpose of this study is to test the efficacy of a peer support coaching intervention to improve activated chronic illness self-management versus an attention control group in 225 adolescents and young adults with childhood…
- Conditions
- Sickle Cell Disease, Chronic Kidney Diseases, Systemic Lupus Erythematosus, Childhood Cancer
- Trial name
- PiCASO (NCT03938324)
- Testing
- PiCASO Intervention Group
- Where
- United States
- Volunteers
- 222 people
- Sponsor
- Duke University