Clinical trials: Anemia, Neonatal
Sickle Cell Anemia
Haploidentical PBMC Transplant for Severe Congenital Anemias
Background: Bone marrow transplantation (BMT), which involves transplanting a donor's marrow stem cells, is capable of curing some congenital anemias.
- Trial name
- NCT00977691
- Testing
- PBSC Transplant, Alemtuzumab, Sirolimus
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Thalassemia, Sickle Cell Disease
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot…
- Conditions
- Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
- Trial name
- NCT00235391
- Testing
- Deferasirox
- Where
- 141 sites in United States, Belgium and 10 more
- Volunteers
- 1,683 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Comparing Individualized vs. Weight Based Protocols to Treat Vaso-Occlusive Episodes in Sickle Cell Disease
The purpose of this research study is to compare two different ways to give opioid pain medicine to treat sickle cell disease pain that is bad enough to go to the emergency department for treatment.
- Trial name
- COMPARE-VOE (NCT03933397)
- Testing
- Patient-Specific Protocol, Weight-based Protocol, Morphine
- Where
- 6 sites in United States
- Volunteers
- 328 people
- Sponsor
- Duke University
Dyskeratosis Congenita, Aplastic Anemia
Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Fludarabine-based preparative regimen followed by an allogeneic hematopoietic stem cell transplant using related or unrelated donor in persons 0-70 years of age diagnosed with dyskeratosis congenita or severe aplastic anemia who…
- Trial name
- NCT02162420
- Testing
- Alemtuzumab, Fludarabine, Cyclophosphamide
- Where
- United States
- Volunteers
- 61 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Sickle Cell Disease
Dose Escalation Study to Evaluate the Safety, Tolerability, PK and PD of Voxelotor in Patients With SCD
Study participants will undergo up to four periods of voxelotor administered orally at progressively higher dose levels from 1500 mg until either a maximum tolerated dose (MTD) or 3000 mg/day dose is reached, whichever occurs…
- Trial name
- NCT04247594
- Testing
- Voxelotor
- Where
- 6 sites in United Kingdom
- Volunteers
- 6 people
- Sponsor
- Pfizer
Sickle Cell Disease
Development and Adaptation of I-STRONG for SCD
This study develops and tests the feasibility and acceptability of an adapted intervention, Integrative Strong Body and Mind Training (I-STRONG), in adolescents with pain from sickle cell disease.
- Trial name
- NCT06110754
- Testing
- I-STRONG for SCD
- Where
- 2 sites in United States
- Volunteers
- 45 people
- Sponsor
- Emory University
Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Deferasirox in Treating Iron Overload Caused By Blood Transfusions in Patients With Hematologic Malignancies
RATIONALE: Deferasirox may remove excess iron from the body caused by blood transfusions. PURPOSE: This clinical trial studies deferasirox in treating iron overload caused by blood transfusions in patients with hematologic…
- Conditions
- Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01273766
- Testing
- deferasirox, laboratory biomarker analysis, enzyme-linked immunosorbent assay
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Wake Forest University Health Sciences
Sickle Cell Disease
Low-Dose Ketamine Infusion for Children With Sickle Cell Disease-Related Pain
Acute pain episodes associated with sickle cell disease (SCD) are very difficult to manage effectively. Opioid tolerance and side effects have been major roadblocks in our ability to provide these patients with adequate pain…
- Trial name
- NCT00595530
- Testing
- ketamine
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- Connecticut Children's Medical Center
Leukemia, Myelodysplastic Syndromes
Busulfan, Etoposide, and Total-Body Irradiation in Treating Patients Undergoing Donor Stem Cell or Bone Marrow Transplant for Advanced Hematologic Cancer
RATIONALE: Giving chemotherapy and total-body irradiation before a donor stem cell transplant or a donor bone marrow transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from…
- Trial name
- NCT00534430
- Testing
- busulfan, cyclosporine, etoposide
- Volunteers
- 30 people
- Sponsor
- City of Hope Medical Center
Sickle Cell Anemia
Sparing Conversion to Abnormal TCD (Transcranial Doppler) Elevation (SCATE)
The primary goal of the Phase III SCATE trial is to compare 30 months of alternative therapy (hydroxyurea) to standard care (observation) in children with sickle cell anemia and conditional (170 - 199cm/sec) Transcranial Doppler…
- Trial name
- SCATE (NCT01531387)
- Testing
- Hydroxyurea
- Where
- 3 sites in United States, Brazil and 1 more
- Volunteers
- 38 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Advanced Cancers
A Study of LY2606368 (Prexasertib) in Patients With Solid Tumors With Replicative Stress or Homologous Repair Deficiency
This research study is studying a checkpoint kinase 1 (CHK1) inhibitor as a possible treatment for advanced solid tumors that harbor genetic alterations in the homologous repair (HR) pathway, genetic alterations that indicate…
- Trial name
- NCT02873975
- Testing
- LY2606368
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Dana-Farber Cancer Institute
Sickle Cell Disease (SCD)
Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of ALXN1820 in Adult Participants With Sickle Cell Disease
The primary objective of this study is to assess the safety and tolerability of ALXN1820 SC (subcutaneous) in participants with SCD (Sickle Cell Disease).
- Trial name
- PHOENIX (NCT05565092)
- Testing
- ALXN1820
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Sickle Cell Disease
Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO).
The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670 is studied in sickle cell disease patients with transfusional hemosiderosis.
- Trial name
- NCT01090323
- Testing
- ICL670
- Where
- 39 sites in United States, Canada and 3 more
- Volunteers
- 185 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease, Sickle Cell Anemia
Decision Aid for Therapeutic Options In Sickle Cell Disease
Sickle cell disease (SCD) is an inherited disorder with chronic multi-system manifestations affecting 100,000 individuals in the US, largely of minority origin and associated with substantial morbidity, premature mortality…
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Hemoglobin SS, Hemoglobin SC
- Trial name
- NCT02326597
- Testing
- Decision Aid Tool, Standard Practice
- Where
- United States
- Volunteers
- 134 people, healthy volunteers welcome
- Sponsor
- Emory University
Sickle Cell Disease, Renal Function Disorder
Evaluation of the Impact of Renal Function on the Pharmacokinetics of SIKLOS ® (DARH)
The use of hydroxyurea in sickle cell disease patients with glomerular hyperfiltration and renal failure requires a specific monitoring and dose adjustment in order to remain within the therapeutic interval while limiting the…
- Trial name
- NCT02522104
- Testing
- Siklos
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Theravia
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders
RATIONALE: Giving chemotherapy and total-body irradiation before a donor peripheral stem cell transplant helps stop the growth of cancer or abnormal cells.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00544115
- Testing
- busulfan, cyclophosphamide, cyclosporine
- Volunteers
- 260 people
- Sponsor
- City of Hope Medical Center
Anemia, Sickle Cell
Dexamethasone to Treat Acute Chest Syndrome in People With Sickle Cell Disease
People with sickle cell disease (SCD) may develop acute chest syndrome (ACS), which is a common and serious lung condition that usually requires hospitalization.
- Trial name
- NCT00530270
- Testing
- Dexamethasone
- Where
- 6 sites in United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Stem Cell Transplantation, Bone Marrow Transplantation
Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells
Many genetic diseases of lymphohematopoietic cells (such as sickle cell anemia, thalassemia, Diamond-Blackfan anemia, Combined Immune Deficiency (CID), Wiskott-Aldrich syndrome, chronic granulomatous disease, X-linked…
- Conditions
- Stem Cell Transplantation, Bone Marrow Transplantation, Peripheral Blood Stem Cell Transplantation, Allogeneic Transplantation
- Trial name
- NCT02512679
- Testing
- Cyclophosphamide Dose Level 1, Cyclophosphamide Dose Level 2, Cyclophosphamide Dose Level 3
- Volunteers
- 20 people
- Sponsor
- Children's Hospital Los Angeles
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
Sickle Cell Disease
Imatinib for Pain in Sickle Cell Anemia
In this protocol, the investigators propose to evaluate the biochemical effects of imatinib on sickle red blood cells (RBCs). Patients will be administered imatinib mesylate orally following the guidelines previously established…
- Trial name
- IMPACT (NCT03997903)
- Testing
- Imatinib Mesylate
- Where
- 2 sites in United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Indiana University