| 1 |
NCT02105766 |
Recruiting |
Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure |
- Sickle Cell Disease
- Thalassemia
- Stem Cell Transplantation
- Graft vs Host Disease
|
- Drug: Alemtuzumab
- Drug: Sirolimus
- Drug: Cyclophosphamide
- (and 2 more...)
|
Interventional |
Phase 1
Phase 2 |
- National Heart, Lung, and Blood Institute (NHLBI)
- National Cancer Institute (NCI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Determine regimen failure rate, defined as graft rejection, severe GVHD (acute GVHD grade 3 or higher or extensive chronic GVHD), or prolonged donor red cell aplasia ( greater than 2 years post -HSCT)
- Examine the level of chimerism required to maintain both graft survival as well as hematologic normalcy using a regimen containing pentostatin, cyclophosphamide, alemtuzumab, and low total body irradiation.
|
162 |
All |
16 Years to 80 Years (Child, Adult, Senior) |
NCT02105766 |
140077
14-H-0077 |
|
April 1, 2014 |
August 31, 2021 |
August 31, 2021 |
April 7, 2014 |
April 4, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
| 2 |
NCT02692872 |
Recruiting |
Screening for Alpha Thalassemia in Healthy Volunteers |
|
|
Observational |
|
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Observational Model: Other
- Time Perspective: Other
|
- Identify Presence of Double Alpha Globin Deletions in Healthy Volunteers.
|
2000 |
All |
18 Years to 50 Years (Adult) |
NCT02692872 |
160065
16-I-0065 |
|
February 25, 2016 |
December 31, 2018 |
December 31, 2018 |
February 26, 2016 |
April 4, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
| 3 |
NCT00061568 |
Recruiting |
Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias |
- Congenital Hemolytic Anemia
- Diamond-Blackfan Anemia
|
- Procedure: Radiotherapy
- Drug: Alemtuzumab (Campath )
- Drug: Sirolimus (Rapamune )
|
Interventional |
Phase 2 |
- National Heart, Lung, and Blood Institute (NHLBI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Primary Purpose: Treatment
|
- Rate of engraftment
- The chimeric status of patients will be measured on days, +30, +60 and +100 by microsatellite analysis of the peripheral blood. More frequent monitoring may be required.
- Incidence of acute and chronic GVHD or relapse rate. GvHD or relapse together count together toward the combined endpoint for regimen failure
- (and 5 more...)
|
150 |
All |
2 Years to 80 Years (Child, Adult, Senior) |
NCT00061568 |
030170
03-H-0170 |
|
May 23, 2003 |
January 31, 2019 |
January 31, 2019 |
May 29, 2003 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
| 4 |
NCT03329404 |
Not yet recruiting |
Efficacy and Safety of RBCs Derived From Mirasol-treated Whole Blood in Patients Requiring Chronic Transfusion (PRAISE) |
- Transfusion Dependent Thalassemia
|
- Device: Mirasol Red Blood Cells (MIR RBCs)
- Device: Reference Red Blood Cells (REF RBCs)
|
Interventional |
Not Applicable |
- Terumo BCTbio
- United States Department of Defense
- Joint Warfighter Medical Research Program
- U.S. Army Medical Research and Materiel Command
|
Industry / U.S. Fed / Other |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Normalized hemoglobin (Hb AUC) calculated from normalized Hb between successive transfusions as a measure of percent surviving RBCs
- Hb increment
- Actual Hb level post-transfusion (15 min)
|
97 |
All |
12 Years and older (Child, Adult, Senior) |
NCT03329404 |
CTS-5056 |
PRAISE |
February 2018 |
June 2020 |
June 2020 |
November 6, 2017 |
February 14, 2018 |
|
- UCSF Benioff Children's Hospital Oakland
Oakland, California, United States - Boston Children's Hospital
Boston, Massachusetts, United States - Weill-Cornell Medical College
New York, New York, United States - The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
|
| 5 |
NCT00669305 |
Recruiting |
Bone Marrow for Hemoglobinopathy Research |
- Sickle Cell Anemia
- Thalassemia
|
|
Observational |
|
- St. Jude Children's Research Hospital
- National Heart, Lung, and Blood Institute (NHLBI)
- Assisi Foundation
- University of Tennessee
|
Other / NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Percentage of successful achievement of therapeutic level in mouse models resulting from retroviral vector mediated gene transfer, gene editing or drug treatment.
|
28 |
All |
Child, Adult, Senior |
NCT00669305 |
BMR
U54HL070590
P01HL053749
201003 |
|
May 15, 2008 |
July 2023 |
July 2024 |
April 30, 2008 |
February 13, 2018 |
|
- St. Jude Children's Research Hospital
Memphis, Tennessee, United States
|
| 6 |
NCT02373241 |
Recruiting |
Preventing Sickle Cell Kidney Disease |
- Anemia, Sickle Cell
- Sickle Cell Disease
- Kidney Disease
- (and 2 more...)
|
|
Interventional |
Phase 2 |
- University of Alabama at Birmingham
- National Heart, Lung, and Blood Institute (NHLBI)
|
Other / NIH |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Feasibility as measured by the number of patients that accept enrollment, remain adherent to losartan, and remain adherent to study procedures.
- Feasibility as measured by number of patients with adverse events to losartan.
- To prospectively evaluate the incidence of hypertension (clinic and ambulatory) in patients with SCD and number of patients with abnormal blood pressure biomarkers.
- Feasibility as measured by the number of patients with improvement in nocturnal blood pressure while receiving losartan.
|
40 |
All |
5 Years to 25 Years (Child, Adult) |
NCT02373241 |
F141107009
1K23HL127100-01 |
|
April 2015 |
April 2020 |
April 2020 |
February 26, 2015 |
May 1, 2017 |
|
- University of Alabama at Birmingham
Birmingham, Alabama, United States
|
| 7 |
NCT03020615 |
Recruiting |
Hydroxyurea Management in Kids: Intensive Versus Stable Dosage Strategies |
|
|
Interventional |
Phase 2 |
- St. Jude Children's Research Hospital
- National Heart, Lung, and Blood Institute (NHLBI)
|
Other / NIH |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Single (Investigator)
- Primary Purpose: Treatment
|
- Number of patients enrolled and randomized
- Number of randomized patients with ≥80% chronic medication compliance
- Number of patients who have the % fetal hemoglobin (%HbF) collected at baseline and at study exit
- (and 25 more...)
|
50 |
All |
9 Months to 36 Months (Child) |
NCT03020615 |
HUGKISS
R34HL127162 |
|
May 12, 2017 |
September 30, 2020 |
September 30, 2020 |
January 13, 2017 |
September 6, 2017 |
|
- Emory University/Children's Health Care of Atlanta
Atlanta, Georgia, United States - St. Jude Children's Research Hospital
Memphis, Tennessee, United States - University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, United States
|
| 8 |
NCT03333486 |
Recruiting |
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer |
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
- Acute Leukemia in Remission
- Acute Lymphoblastic Leukemia
- (and 34 more...)
|
- Drug: Cyclophosphamide
- Drug: Fludarabine Phosphate
- Other: Laboratory Biomarker Analysis
- (and 2 more...)
|
Interventional |
Phase 2 |
- Roswell Park Cancer Institute
- National Cancer Institute (NCI)
|
Other / NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Relapse rate
- Engraftment rate
- Incidence of acute graft versus host disease (GVHD)
- (and 4 more...)
|
58 |
All |
1 Year to 75 Years (Child, Adult, Senior) |
NCT03333486 |
I 40916
NCI-2017-01949
P30CA016056 |
|
December 7, 2017 |
September 6, 2021 |
September 6, 2022 |
November 7, 2017 |
December 11, 2017 |
|
- Roswell Park Cancer Institute
Buffalo, New York, United States
|
| 9 |
NCT02297126 |
Recruiting |
A Prospective Trial to Assess Cost and Clinical Outcomes of a Clinical Pharmacogenomic Program |
|
- Device: Pharmacogenetic Intervention Arm (pharmacogenetic testing)
|
Interventional |
Not Applicable |
- Indiana University
- National Human Genome Research Institute (NHGRI)
|
Other / NIH |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Other
|
- Financial impact on the total cost of patient care resulting from implementation of a Pharmacogenetics testing program within a safety-net and Academic healthcare system
- Impact of implementing a Pharmacogenetics program on Clinical Outcomes (incidence and severity of adverse events, frequency of healthcare visits, length of hospital stay, and readmissions) within a safety-net and Academic healthcare system
- Impact of implementing a Pharmacogenetics program on prescribing patterns within a safety-net healthcare system
|
6000 |
All |
18 Years and older (Adult, Senior) |
NCT02297126 |
1401206188
U01HG007762-01 |
INGenious |
January 2015 |
June 2018 |
June 2018 |
November 21, 2014 |
November 20, 2017 |
|
- Eskenazi Health System
Indianapolis, Indiana, United States
|
| 10 |
NCT00081523 |
Recruiting |
Natural History of Sickle Cell Disease |
|
|
Observational |
|
- National Heart, Lung, and Blood Institute (NHLBI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
|
|
99999999 |
All |
2 Years and older (Child, Adult, Senior) |
NCT00081523 |
040161
04-H-0161 |
|
April 14, 2004 |
|
|
April 15, 2004 |
March 29, 2018 |
|
- Childrens National Medical Center
Washington, District of Columbia, United States - National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
| 11 |
NCT02712346 |
Recruiting |
The Role of Endothelin-1 in Sickle Cell Disease |
|
- Drug: Ambrisentan
- Drug: Placebo
|
Interventional |
Phase 1 |
- Augusta University
- Gilead Sciences
- National Heart, Lung, and Blood Institute (NHLBI)
|
Other / Industry / NIH |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Triple (Participant, Care Provider, Investigator)
- Primary Purpose: Treatment
|
- Safety and Tolerability of ambrisentan in patients with sickle cell disease measured by physical exam, vital signs, blood and urine testing, ECG (specified visits), concomitant medication review, adverse events review
- Efficacy of ambrisentan in improving kidney function in patients with sickle cell disease measured by blood
- Efficacy of ambrisentan in decreasing TRJ velocity
- (and 5 more...)
|
30 |
All |
18 Years to 65 Years (Adult) |
NCT02712346 |
Endothelin
5U01HL117684 |
|
September 2015 |
May 2018 |
May 2018 |
March 18, 2016 |
June 29, 2017 |
|
- Augusta University
Augusta, Georgia, United States
|
| 12 |
NCT01174108 |
Recruiting |
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells |
- Severe Aplastic Anemia
- MDS (Myelodysplastic Syndrome)
|
- Device: Miltenyi CD34 Reagent System
- Other: Donor derived G-CSF mobilized PBC
|
Interventional |
Phase 2 |
- National Heart, Lung, and Blood Institute (NHLBI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Primary endpoint of this study is chronic GVHD by one year.
- Transplant related mortality, engraftment, degree of donor-host chimerism, incidence of acute and chronic graft versus host disease (GVHD), transplant related morbidity and overall survival.
|
117 |
All |
4 Years to 80 Years (Child, Adult, Senior) |
NCT01174108 |
100154
10-H-0154 |
|
July 30, 2010 |
June 30, 2019 |
June 30, 2020 |
August 3, 2010 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States - University of Maryland at Baltimore/MPRC
Catonsville, Maryland, United States
|
| 13 |
NCT00003838 |
Recruiting |
Specialized Blood Cell Transplants for Cancers of the Blood and Bone Marrow |
- Hematologic Disease
- Lymphoma
- Multiple Myeloma
- (and 2 more...)
|
- Procedure: Stem cell transplantation
|
Interventional |
Phase 2 |
- National Heart, Lung, and Blood Institute (NHLBI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Allocation: Non-Randomized
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- To evaluate engraftment by bone marrow chimerism analysis.
- To determine the incidence and severity of acute and chronic graft-versus-host disease (GVHD).
|
214 |
All |
2 Years to 80 Years (Child, Adult, Senior) |
NCT00003838 |
990050
99-H-0050 |
|
February 23, 1999 |
December 1, 2019 |
December 1, 2019 |
January 27, 2003 |
April 13, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
| 14 |
NCT03381989 |
Not yet recruiting |
Bioprosthetic or Native Aortic Scallop Intentional Laceration to Prevent Iatrogenic Coronary Artery Obstruction (BASILICA) Prospective Investigation |
|
- Device: ASHI_INTECC Astato XS 20 0.014 guidewire
|
Interventional |
Phase 1
Phase 2 |
- National Heart, Lung, and Blood Institute (NHLBI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- The primary efficacy endpoint is clinical success at exit from catheterization lab
- The primary safety endpoint is freedom from major adverse clinical events (MACE) at 30days.
- Clinical success irrespective of coronary artery stenting
- (and 16 more...)
|
60 |
All |
21 Years and older (Adult, Senior) |
NCT03381989 |
999918018
18-H-N018 |
|
December 25, 2017 |
November 30, 2018 |
September 30, 2019 |
December 22, 2017 |
December 22, 2017 |
|
- Emory University Hospital
Atlanta, Georgia, United States
|
| 15 |
NCT00919503 |
Recruiting |
Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders |
|
- Procedure: Allogeneic Bone Marrow Transplantation
- Biological: Anti-Thymocyte Globulin
- Drug: Cyclosporine
- (and 9 more...)
|
Interventional |
Phase 2 |
- Fred Hutchinson Cancer Research Center
- medac GmbH
- National Cancer Institute (NCI)
- National Heart, Lung, and Blood Institute (NHLBI)
|
Other / Industry / NIH |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Preliminary efficacy
- Disease response following hematopoietic cell transplantation
- Donor chimerism
- (and 6 more...)
|
120 |
All |
up to 49 Years (Child, Adult) |
NCT00919503 |
2256.00
NCI-2010-01277
P30CA015704 |
|
July 31, 2009 |
February 1, 2019 |
|
June 12, 2009 |
April 5, 2018 |
|
- Children's Hospital Colorado
Aurora, Colorado, United States - Oregon Health and Science University
Portland, Oregon, United States - Vanderbilt University/Ingram Cancer Center
Nashville, Tennessee, United States - (and 3 more...)
|
| 16 |
NCT03412760 |
Not yet recruiting |
Fetal Birth Defects: Toward a Precision-based Approach |
- Hydrops Fetalis
- Birth Defect
- Fetal Anomaly
|
- Diagnostic Test: Whole exome sequencing
|
Interventional |
Not Applicable |
- University of California, San Francisco
- National Institutes of Health (NIH)
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
|
Other / NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Diagnostic
|
- Genetic mutations detected on whole exome sequencing that explain the cause of non-immune hydrops fetalis (NIHF) and other birth defects.
|
500 |
All |
up to 55 Years (Child, Adult) |
NCT03412760 |
HydropsUCSF
5K12HD001262 |
|
April 2018 |
April 2025 |
December 2025 |
January 26, 2018 |
January 26, 2018 |
|
- University of California, San Francisco
San Francisco, California, United States
|
| 17 |
NCT03049475 |
Recruiting |
Pathophysiology of Acute Pain in Patients With Sickle Cell Disease |
|
|
Observational |
|
- National Heart, Lung, and Blood Institute (NHLBI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Observational Model: Case-Control
- Time Perspective: Prospective
|
- To measure and compare defined sets of markers of obstruction ofblood vessels (vaso-occlusion), red cell breakdown (hemolysis), and inflammation during acute painful crisis vs steady state in patients withsickle cell disease.
|
100 |
All |
18 Years to 80 Years (Adult, Senior) |
NCT03049475 |
170056
17-H-0056 |
|
March 13, 2017 |
October 31, 2021 |
October 31, 2021 |
February 10, 2017 |
April 4, 2018 |
|
- National Institutes of Health Clinical Center
Bethesda, Maryland, United States
|
| 18 |
NCT03178643 |
Recruiting |
Enhancing Preventive Therapy of Malaria In Children With Sickle Cell Anemia in East Africa (EPiTOMISE) |
|
- Drug: Proguanil Oral Tablet
- Drug: Sulfadoxine/Pyrimethanine-Amodiaquine (SP-AQ)
- Drug: Dihydroartemisinin-Piperaquine (DP)
|
Interventional |
Phase 4 |
- Duke University
- National Heart, Lung, and Blood Institute (NHLBI)
|
Other / NIH |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Prevention
|
- Incidence of malaria over 12 months.
- Incidence of SCA-related morbidity
- Severe malaria
- (and 14 more...)
|
246 |
All |
1 Year to 10 Years (Child) |
NCT03178643 |
Pro00077428
R01HL134211 |
EPiTOMISE |
January 23, 2018 |
October 2019 |
October 2019 |
June 7, 2017 |
January 31, 2018 |
|
- Homa Bay County Referral Hospital
Homa Bay Town, Homa Bay County, Kenya
|
| 19 |
NCT03380351 |
Not yet recruiting |
Specifying Interventions From the Sickle Cell Disease Implementation Consortium (SCDIC) |
|
- Behavioral: SCDIC interventions
- Behavioral: SCDIC control groups
|
Observational |
|
- Washington University School of Medicine
- National Heart, Lung, and Blood Institute (NHLBI)
|
Other / NIH |
- Observational Model: Case-Only
- Time Perspective: Prospective
|
- SCDIC Framework of Interventions
|
24 |
All |
18 Years and older (Adult, Senior) |
NCT03380351 |
3U01HL133994-02S1 |
|
April 2, 2018 |
September 1, 2019 |
June 30, 2020 |
December 21, 2017 |
March 20, 2018 |
|
- Washington University in St. Louis
Saint Louis, Missouri, United States
|
| 20 |
NCT00027274 |
Recruiting |
Cancer in Inherited Bone Marrow Failure Syndromes |
- Aplastic Anemia
- Head and Neck Neoplasms
- Bone Marrow Failure
|
|
Observational |
|
- National Cancer Institute (NCI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Observational Model: Family-Based
- Time Perspective: Other
|
- Cohort of Families with IBMFS
- Biology of Patients Compared with Healthy Controls
- Differences Between Patients and Healthy Controls
- Risk of Cancer with Specific Mutations
|
4000 |
All |
up to 120 Years (Child, Adult, Senior) |
NCT00027274 |
020052
02-C-0052 |
|
January 1, 2002 |
|
|
November 30, 2001 |
February 14, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
| 21 |
NCT02560935 |
Recruiting |
Primary Prevention of Stroke in Children With SCD in Sub-Saharan Africa II |
- Sickle Cell Disease
- Stroke
|
- Drug: Hydroxyurea (Moderate Dose)
- Drug: Hydroxyurea (Low Dose)
|
Interventional |
Phase 3 |
- Vanderbilt University Medical Center
- Aminu Kano Teaching Hospital
- Murtala Muhammed Specialist Hospital
- (and 2 more...)
|
Other / NIH |
- Allocation: Randomized
- Intervention Model: Single Group Assignment
- Masking: Double (Investigator, Outcomes Assessor)
- Primary Purpose: Prevention
|
- Incidence rate of clinical stroke or TIA
- Incidence of all cause hospitalizations
- Long-term safety of hydroxyurea therapy
|
440 |
All |
5 Years to 12 Years (Child) |
NCT02560935 |
121383
1R01NS094041-01 |
SPRING |
July 2016 |
December 2021 |
December 2021 |
September 25, 2015 |
March 29, 2018 |
|
- Barau Dikko Teaching Hospital/Kaduna State University
Kaduna, Nigeria - Aminu Kano Teaching Hospital
Kano, Nigeria - Murtala Muhammad Specialist Hospital
Kano, Nigeria
|
| 22 |
NCT02098863 |
Recruiting |
Sickle Cell Clinical Research and Intervention Program |
|
|
Observational |
|
- St. Jude Children's Research Hospital
- Methodist Healthcare
- University of Memphis School of Public Health
- (and 12 more...)
|
Other / NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Relationship between treatment plan and health outcomes in participants with sickle cell disease (SCD)
- Relationship between genetic properties of biological samples and health outcomes in participants with sickle cell disease
|
1550 |
All |
Child, Adult, Senior |
NCT02098863 |
SCCRIP
1U01HL133996
UTHSC-MRC Sub |
|
April 15, 2014 |
December 2044 |
December 2044 |
March 28, 2014 |
February 1, 2018 |
|
- Children's Hospital of Illinois at OSF-Saint Francis Medical Center
Peoria, Illinois, United States - Our Lady of the Lake Regional Medical Center
Baton Rouge, Louisiana, United States - Novant Health Hemby Children's Hospital
Charlotte, North Carolina, United States - (and 3 more...)
|
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