| 1 |
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
|
| 2 |
NCT03481738 |
Recruiting
New |
Pyruvate Kinase Deficiency Global Longitudinal Registry |
- Pyruvate Kinase Deficiency
|
|
Observational |
|
- Agios Pharmaceuticals, Inc.
|
Industry |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Clinical Course of PK Deficiency
- Severity of Disease
- Disease Impact on Pregnancy
- (and 2 more...)
|
500 |
All |
Child, Adult, Senior |
NCT03481738 |
AG348-C-008 |
PEAK Registry |
March 2018 |
March 2026 |
March 2026 |
March 29, 2018 |
March 29, 2018 |
|
- Children's Hospital of Pennslyvania
Philadelphia, Pennsylvania, United States
|
| 3 |
NCT02476916 |
Active, not recruiting |
A Study of AG-348 in Adult Patients With Pyruvate Kinase Deficiency |
- Pyruvate Kinase Deficiency
|
|
Interventional |
Phase 2 |
- Agios Pharmaceuticals, Inc.
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety: incidence of adverse events
- Pharmacokinetic parameters of AG-348 and its metabolite area-under-the-curve (AUC) 0-12hr
- Pharmacokinetic parameters of AG-348 and its metabolite (Tmax)
- (and 5 more...)
|
52 |
All |
18 Years and older (Adult, Senior) |
NCT02476916 |
AG348-C-003 |
|
June 12, 2015 |
May 8, 2017 |
May 2019 |
June 22, 2015 |
March 6, 2018 |
|
- Stanford University
Palo Alto, California, United States - Boston Children's Hospital
Boston, Massachusetts, United States - Wayne State University School of Medicine - Children's Hospital of Michigan
Detroit, Michigan, United States - (and 11 more...)
|
| 4 |
NCT03006718 |
Recruiting |
SCD-PROMIS: A Software Platform to Enhance Self-efficacy and Patient-provider Engagement for Patients With Sickle Cell Pain |
- Sickle Cell Disease
- Anemia
- Anemia, Hemolytic
- (and 2 more...)
|
- Other: PROMIS for Pain Management App
|
Interventional |
Not Applicable |
- Children's Research Institute
- Arizona State University
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Other
|
- Readmission rate
- Readmission risk
- Patient Compliance
- Patient Usability
|
150 |
All |
8 Years to 21 Years (Child, Adult) |
NCT03006718 |
7618 |
|
November 2016 |
September 2018 |
September 2018 |
December 30, 2016 |
April 2, 2018 |
|
- Children's National Medical Center
Washington, District of Columbia, United States
|
| 5 |
NCT00000586 |
Completed |
Multicenter Study of Hydroxyurea in Patients With Sickle Cell Anemia (MSH) |
- Anemia, Sickle Cell
- Hematologic Diseases
- Hemoglobinopathies
|
|
Interventional |
Phase 3 |
- National Heart, Lung, and Blood Institute (NHLBI)
|
NIH |
- Allocation: Randomized
- Masking: Double
- Primary Purpose: Treatment
|
- Occurrence of vaso-occlusive (painful) crisis
|
|
All |
18 Years to 50 Years (Adult) |
NCT00000586 |
306 |
|
January 1992 |
June 1994 |
June 1994 |
October 28, 1999 |
April 14, 2016 |
|
|
| 6 |
NCT02053480 |
Active, not recruiting |
Pyruvate Kinase Deficiency Natural History Study |
- Pyruvate Kinase Deficiency
- Congenital Non-Spherocytic Hemolytic Anemia
|
|
Observational |
|
- Boston Children’s Hospital
- Agios Pharmaceuticals, Inc.
|
Other / Industry |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- transfusion burden in splenectomized and non-splenectomized participants
- patient-reported outcomes
- changes over time in hemoglobin and markers of hemolysis
- prevalence and severity of iron overload
|
250 |
All |
Child, Adult, Senior |
NCT02053480 |
P00010515 |
PKD NHS |
December 2013 |
December 2018 |
December 2018 |
February 3, 2014 |
June 15, 2017 |
|
- Phoenix Children's Hospital
Phoenix, Arizona, United States - Stanford University
Palo Alto, California, United States - Children's Hospital of Atlanta
Atlanta, Georgia, United States - (and 27 more...)
|
| 7 |
NCT00000602 |
Completed |
Pediatric Hydroxyurea in Sickle Cell Anemia (PED HUG) |
- Anemia, Sickle Cell
- Hematologic Diseases
- Hemoglobinopathies
|
|
Interventional |
Phase 2 |
- National Heart, Lung, and Blood Institute (NHLBI)
|
NIH |
- Primary Purpose: Treatment
|
|
|
All |
1 Year to 18 Years (Child, Adult) |
NCT00000602 |
315
P60HL015157
P60HL028391
P60HL020985 |
|
April 1994 |
|
March 1997 |
October 28, 1999 |
September 16, 2016 |
|
|
| 8 |
NCT01141621 |
Terminated |
The Dallas Hereditary Spherocytosis Cohort Study |
|
|
Observational |
|
- University of Texas Southwestern Medical Center
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Health related quality of life
- Primary indications for splenectomy
- Complications of HS
- (and 2 more...)
|
55 |
All |
up to 21 Years (Child, Adult) |
NCT01141621 |
IRB # 022010-024 |
|
May 2010 |
May 2013 |
May 2013 |
June 10, 2010 |
May 14, 2013 |
|
|
| 9 |
NCT02372877 |
Completed |
Evaluation of the AMICUS RBCx System in Sickle Cell Patients |
|
- Device: Amicus Red Cell Exchange in SCD patients
|
Interventional |
Not Applicable |
- Fenwal, Inc.
- Blood Center of Wisconsin
- Children's Hospital of Philadelphia
- (and 4 more...)
|
Industry / Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Evaluation of the accuracy of patient's actual fraction of original red blood cells remaining as measured by the patient's pre-procedure Hb S and post-procedure Hb S to the target Fraction of Cells Remaining (FCR).
- Evaluate the accuracy of patient's End Hematocrit as measured by the patient's post-procedure hematocrit.
- Subject Cell Loss Post-Procedure
- Serious Adverse Events
|
59 |
All |
6 Years and older (Child, Adult, Senior) |
NCT02372877 |
AMIC-003-CMD |
|
May 2015 |
December 7, 2017 |
January 19, 2018 |
February 26, 2015 |
February 21, 2018 |
|
- Phoenix Children's Hospital
Phoenix, Arizona, United States - Barbara Ann Karmanos Cancer Institute
Detroit, Michigan, United States - Washington University
Saint Louis, Missouri, United States - (and 3 more...)
|
| 10 |
NCT02964494 |
Recruiting |
The Congenital Dyserythropoietic Anemia Registry (CDAR) |
- Congenital Dyserythropoietic Anemia (CDA)
|
|
Observational |
|
- Children's Hospital Medical Center, Cincinnati
|
Other |
- Observational Model: Other
- Time Perspective: Other
|
- Age and symptoms at presentation and/or diagnosis
- Degree of anemia
- Clinical course during
- (and 8 more...)
|
1000 |
All |
Child, Adult, Senior |
NCT02964494 |
2016-2727_CDAR |
|
July 2016 |
July 2026 |
January 2031 |
November 16, 2016 |
November 27, 2017 |
|
- Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
|
| 11 |
NCT02635269 |
Recruiting |
Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy |
- Metabolism, Inborn Errors
- Lipid Metabolism, Inborn Errors
- Carbohydrate Metabolism, Inborn Errors
- (and 20 more...)
|
|
Interventional |
Not Applicable |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Basic Science
|
- Total fat oxidation (rate of disappearance) during prolonged moderate intensity exercise. Measured as micro mol per kg per minute.
- Maximal oxidative capacity (mL*kg-1*minutes-1)
- Endurance measured as minutes:seconds
- (and 5 more...)
|
128 |
All |
15 Years to 80 Years (Child, Adult, Senior) |
NCT02635269 |
Metabolic Myopathy
H-15015150 |
|
January 2016 |
August 2020 |
December 2020 |
December 18, 2015 |
September 1, 2017 |
|
- Neuromuscular Research Unit, 3342
Copenhagen, Denmark
|
| 12 |
NCT00029393 |
Completed |
Induction of Stable Chimerism for Sickle Cell Anemia |
- Blood Disease
- Hematopoietic Stem Cell Transplantation
- Anemia, Sickle Cell
|
- Procedure: Hematopoietic Stem Cell Transplantation
- Drug: Immunosuppression
|
Interventional |
Phase 2 |
- National Heart, Lung, and Blood Institute (NHLBI)
|
NIH |
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
|
|
All |
up to 100 Years (Child, Adult, Senior) |
NCT00029393 |
142
U01HL068091 |
|
August 2001 |
July 2007 |
July 2007 |
January 11, 2002 |
July 29, 2016 |
|
|
| 13 |
NCT00029380 |
Completed |
Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia |
- Hematologic Diseases
- Anemia, Sickle Cell
- Beta-Thalassemia
- Hematopoietic Stem Cell Transplantation
|
- Drug: Sangstat
- Drug: Cyclophosphamide
- Drug: Busulfan
- (and 3 more...)
|
Interventional |
Phase 2 |
- National Heart, Lung, and Blood Institute (NHLBI)
|
NIH |
- Primary Purpose: Treatment
|
- Hematologic parameters
- GVHD
|
30 |
All |
3 Years to 14 Years (Child) |
NCT00029380 |
141
U01HL061877 |
|
January 1999 |
August 2006 |
August 2006 |
January 11, 2002 |
July 29, 2016 |
|
- Children's Hospital Oakland
Oakland, California, United States - Children's Hospital, Oakland
Oakland, California, United States - Children's National Medical Center
Washington, District of Columbia, United States - (and 12 more...)
|
| 14 |
NCT00006400 |
Completed |
Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia |
- Hematologic Diseases
- Anemia, Sickle Cell
|
- Drug: Hydroxyurea
- Drug: Placebo
|
Interventional |
Phase 3 |
- National Heart, Lung, and Blood Institute (NHLBI)
|
NIH |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double (Participant, Investigator)
- Primary Purpose: Prevention
|
- 50% reduction in rates of damage to the major organs with surrogate markers of organ function
|
191 |
All |
9 Months to 18 Months (Child) |
NCT00006400 |
89
N01 HB07150
N01 HB07151
N01 HB07152
N01 HB07153
N01 HB07154
N01 HB07155
N01 HB07156
N01 HB07157
N01 HB07158
N01 HB07159
N01 HB07160 |
|
August 2000 |
September 2009 |
September 2009 |
October 13, 2000 |
April 27, 2011 |
|
- University of Alabama at Birmingham
Birmingham, Alabama, United States - Children's National Medical Center
Washington, District of Columbia, United States - Howard University
Washington, District of Columbia, United States - (and 11 more...)
|
| 15 |
NCT03249831 |
Recruiting |
Non-Myeloablative Conditioning Regimen With Haploidentical T-Cell-Depleted Peripheral Blood Transplant for Patients With Severe Sickle Cell Disease |
- Sickle Cell Disease
- Sickle Cell Disorder
- Hemoglobinopathies
- (and 2 more...)
|
- Drug: Cyclophosphamide
- Drug: Pentostatin
- Drug: Rabbit anti-thymocyte globulin
- (and 3 more...)
|
Interventional |
Phase 1 |
- City of Hope Medical Center
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Toxicity per NCI-Common Terminology Criteria for Adverse Events version 4.0
- Unacceptable Toxicity at least possibly related to COH-MC-17
- Mixed Chimerism defined as 30-90% donor cells
- (and 21 more...)
|
6 |
All |
18 Years to 45 Years (Adult) |
NCT03249831 |
16453 |
|
June 2018 |
November 2021 |
November 2021 |
August 15, 2017 |
February 14, 2018 |
|
- City of Hope Medical Center
Duarte, California, United States
|
| 16 |
NCT02981329 |
Recruiting |
Fetal Hemoglobin Induction Treatment Metformin |
- Sickle Cell Anemia
- Sickle Cell Disease
- Hemoglobin Disorder
- Hemoglobin Disease; Sickle-Cell, Thalassemia
|
|
Interventional |
Early Phase 1 |
- Baylor College of Medicine
- Pfizer
|
Other / Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change in Fetal Hemoglobin (HbF) Percentage (SCA) or Change in Total Hemoglobin (Hb) (NTDT)
- Change in Laboratory Values
- Impact on Quality of Life
- Variability of Hemoglobin Response
|
64 |
All |
12 Years to 40 Years (Child, Adult) |
NCT02981329 |
H-38457 Metformin |
FITMet |
March 2, 2017 |
November 2019 |
November 2022 |
December 5, 2016 |
December 15, 2017 |
|
- Texas Children's Hospital
Houston, Texas, United States
|
| 17 |
NCT02633943 |
Enrolling by invitation |
Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy |
- Beta-Thalassemia
- Sickle Cell Disease
|
- Other: Safety and efficacy assessments
|
Observational |
|
|
Industry |
- Observational Model: Case-Only
- Time Perspective: Prospective
|
- Overall survival of subjects with hemoglobinopathies treated with gene therapy drug product in a bluebird bio-sponsored clinical study
- All adverse events (AEs) related to drug product
- All serious adverse events (SAEs)
- (and 5 more...)
|
86 |
All |
5 Years to 50 Years (Child, Adult) |
NCT02633943 |
LTF-303 |
|
September 2013 |
March 2031 |
March 2031 |
December 17, 2015 |
January 25, 2018 |
|
- Oakland, California, United States
- Chicago, Illinois, United States
- Philadelphia, Pennsylvania, United States
- (and 4 more...)
|
| 18 |
NCT02504619 |
Recruiting |
Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies |
- Sickle Cell Disease
- Thalassemia
|
|
Interventional |
Phase 1
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- The acute toxicities associated with the infusion of CordIn, within 24 hours post-infusion.
- The proportion of patients with donor-derived engraftment at 42 days following transplantation
- Overall survival at 365 days after transplantation
|
15 |
All |
2 Years to 25 Years (Child, Adult) |
NCT02504619 |
GC P#01.01.030 |
|
May 2015 |
December 31, 2018 |
December 2019 |
July 22, 2015 |
January 5, 2018 |
|
- UCSF Benioff Children's Hospital
Oakland, California, United States - Children's National
Washington, District of Columbia, United States - Hôpital Robert Debré
Paris, France
|
| 19 |
NCT02435901 |
Recruiting |
HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity |
- Sickle Cell Disease
- Beta Thalassemia-Major
|
- Drug: alemtuzumab (Campath IH)
- Drug: Fludarabine
- Drug: Melphalan
- (and 4 more...)
|
Interventional |
Phase 1
Phase 2 |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Assessment of Stem Cell Engraftment
- Assessment of treatment related mortality and morbidity
- Event Free Survival
- Immunological Reconstitution
|
29 |
All |
1 Year to 21 Years (Child, Adult) |
NCT02435901 |
08057 |
|
December 2008 |
January 2018 |
January 2019 |
May 6, 2015 |
May 16, 2017 |
|
- Cohen Children's Medical Center of New York
New Hyde Park, New York, United States
|
| 20 |
NCT02306993 |
Recruiting |
Sickle Cell Hemoglobinopathies and Bone Health |
- Sickle Cell Disease
- Sickle Cell Trait
|
- Other: Healthy volunteers without SCT
- Other: Healthy volunteers with SCT
- Other: Volunteers with SCD
|
Observational |
|
|
Other |
- Observational Model: Case-Control
- Time Perspective: Prospective
|
- Hemoglobin genotype with serum bone turnover markers and bone density
|
45 |
Female |
18 Years to 45 Years (Adult) |
NCT02306993 |
14-136-6 |
|
May 2014 |
December 2018 |
December 2019 |
December 3, 2014 |
April 13, 2018 |
|
- UConn Health
Farmington, Connecticut, United States
|
| 21 |
NCT03442114 |
Not yet recruiting |
Shared-Decision Making for Hydroxyurea |
- Sickle Cell Anemia
- Children, Only
|
- Behavioral: Hydroxyurea SDM Toolkit
- Behavioral: Clinician Pocket Guide
|
Interventional |
Phase 3 |
- Children's Hospital Medical Center, Cincinnati
|
Other |
- Allocation: Randomized
- Intervention Model: Sequential Assignment
- Masking: None (Open Label)
- Primary Purpose: Health Services Research
|
- Decisional conflict
- Shared decision making
- Hydroxyurea offered
|
260 |
All |
1 Month to 5 Years (Child) |
NCT03442114 |
CDR_1609_36055 |
ENGAGE-HU |
March 31, 2018 |
August 31, 2020 |
November 30, 2020 |
February 22, 2018 |
March 12, 2018 |
|
|
| 22 |
NCT03374111 |
Not yet recruiting |
Colla Corii Asini Treating Anemia in Pregnant Women With Thalassemia(Presenting the Syndrome of Blood Deficiency) |
|
- Drug: Colla corii asini
- Drug: a Simulate Agent of Colla corii asini granule
|
Interventional |
Phase 4 |
- The First Affiliated Hospital, Guangzhou University of Traditional Chinese Medicine
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Hemoglobin(Hb)
- reticulocyte (RET)
- immature reticulocyte fraction（IRF）
- (and 6 more...)
|
480 |
Female |
20 Years to 50 Years (Adult) |
NCT03374111 |
TH-2 |
|
March 1, 2018 |
October 1, 2019 |
December 30, 2019 |
December 15, 2017 |
March 2, 2018 |
|
- First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, Guangdong
Guangzhou, Guangdong, China - The Third Affiliated Hospital of Guangzhou Medical University
Guangzhou, Guangdong, China - Intranet of Guangzhou Women and Children＇s Medical Center
Guangzhou, Guangdong, China - (and 9 more...)
|
| 23 |
NCT03368924 |
Completed |
Implication of the Oxydative Stress in the Pathophysiology of Sickle Cell Anemia: |
|
- Other: SCA patients (SS genotype)
|
Interventional |
Not Applicable |
- Centre Hospitalier Universitaire de Pointe-a-Pitre
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Basic Science
|
- To compare the level of anti band 3 antibodies in steady state and during vaso-occlusive crises in SCA patients.
- To assess the relationship between level of biomarkers of oxidation of SS RBCs, altered hemorheological parameters, biomarkers of cellular activation (microparticles) and anti band 3 antibodies rate, taking into account the alpha-globin genes status
- To study the relationship between level of anti band 3 antibodies and severity of these VOC using an index of clinical severity (IS2) calculated at the end of SCA patients hospitalization for VOC.
- To study early clinical (including the activity of the autonomic nervous system activity) and biological items to evaluate the relationship between these items and severity of VOC.
|
38 |
All |
18 Years and older (Adult, Senior) |
NCT03368924 |
RBM-PAP-2012/47 |
STRESS |
April 9, 2013 |
September 19, 2015 |
September 19, 2015 |
December 11, 2017 |
December 11, 2017 |
|
- Hospital University Center of Pointe-à-Pitre
Pointe-à-Pitre, Guadeloupe
|
| 24 |
NCT01850108 |
Recruiting |
Non-Myeloablative Conditioning and Bone Marrow Transplantation |
- Sickle Cell Disease
- Hemoglobinopathies
|
- Drug: Thymoglobulin
- Drug: Fludarabine
- Drug: Cyclophosphamide (CTX)
- (and 5 more...)
|
Interventional |
Not Applicable |
- Vanderbilt-Ingram Cancer Center
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Transplant-related mortality (TRM)
- Progression-free survival
- Characterize donor hematopoietic chimerism in peripheral blood after mini-haploBMT
- Characterize hematologic and non-hematologic toxicities of minihaploBMT
|
25 |
All |
1 Year to 70 Years (Child, Adult, Senior) |
NCT01850108 |
VICCNC BMT 12108 |
|
May 2013 |
May 2018 |
November 2018 |
May 9, 2013 |
January 24, 2018 |
|
- Vanderbilt-Ingram Cancer Center
Nashville, Tennessee, United States
|
| 25 |
NCT01795794 |
Unknown † |
Evaluation of the Efficacy in Decreasing Iron Absorption in Patients With Congenital Dyserythropoietic Anemia Type I by Treatment With LOSEC |
|
|
Interventional |
Phase 4 |
- Soroka University Medical Center
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- evaluate number of adverse effects per patient treated with LOSEC + levels of iron, ferritin,complete blood count and chemistry panel.
|
12 |
All |
12 Years and older (Child, Adult, Senior) |
NCT01795794 |
sor034612ctil |
|
April 2013 |
December 2013 |
December 2013 |
February 21, 2013 |
February 27, 2013 |
|
|
| 26 |
NCT03062501 |
Recruiting |
Hydroxyurea in the Emergency Room to Lessen Pain in Sickle Cell Crisis |
- Anemia, Sickle Cell
- Anemia; Sickle-Cell, With Crisis
|
|
Interventional |
Phase 2 |
- Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants with treatment-related adverse events
- Number of participants with altered laboratory values related to treatment
- Time until hospital discharge
- (and 2 more...)
|
30 |
All |
18 Years to 60 Years (Adult) |
NCT03062501 |
Hemorio 397/16 |
HELPS |
November 2016 |
April 2017 |
July 2017 |
February 23, 2017 |
February 23, 2017 |
|
- Hemorio
Rio de Janeiro, Brazil
|
| 27 |
NCT02961218 |
Recruiting |
Study of Efficacy, Safety and Tolerability of ACZ885 (Canakinumab) in Pediatric and Young Adult Patients With Sickle Cell Anemia |
|
- Drug: Ilaris, Canakinumab
- Drug: Placebo
|
Interventional |
Phase 2 |
- Novartis Pharmaceuticals
- Novartis
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Reduction of average daily pain
- Reduction of average daily pain over 24 weeks
- The effect of canakinumab versus placebo on sickle cell anemia related days missed from school or work
- (and 11 more...)
|
60 |
All |
12 Years to 20 Years (Child, Adult) |
NCT02961218 |
CACZ885X2206 |
|
April 5, 2017 |
July 24, 2018 |
March 12, 2020 |
November 10, 2016 |
March 14, 2018 |
|
- Novartis Investigative Site
Hamburg, Germany - Novartis Investigative Site
Afula, Israel - Novartis Investigative Site
Adana, Turkey - (and 3 more...)
|
| 28 |
NCT02769845 |
Recruiting |
SACRED A Prospective Research Study to Reduce Stroke in Children With Sickle Cell Anemia |
|
- Drug: Hydroxyurea
- Procedure: TCD examination
|
Interventional |
Not Applicable |
- Children's Hospital Medical Center, Cincinnati
- Hospital Infantil Dr. Robert Reid Cabral
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Supportive Care
|
- Transcranial Doppler Ultrasound examinations
- Hydroxyurea toxicities
|
500 |
All |
3 Years to 15 Years (Child) |
NCT02769845 |
2015-8767 SACRED |
SACRED |
May 2016 |
November 2019 |
October 2020 |
May 12, 2016 |
August 30, 2017 |
|
- Encargada del Servicio de Hematología-Oncología Hospital Infantil Dr. Robert Reid Cabral
Santo Domingo, Dominican Republic
|
| 29 |
NCT02565849 |
Completed |
Study of Ventilatory Mechanics in Patients With Sickle Cell Anemia |
|
|
Observational |
|
- Rio de Janeiro State University
|
Other |
- Observational Model: Case Control
- Time Perspective: Cross-Sectional
|
- spirometrics parameters of pulmonary function tests assessed by forced expiratory volume in one second (FEV1)
- spirometrics parameters of pulmonary function tests assessed by forced vital capacity (FVC)
- spirometrics parameters of pulmonary function tests assessed by ratio forced expiratory volume in one second and forced vital capacity (FEV1 / FVC)
- (and 15 more...)
|
80 |
All |
18 Years and older (Adult, Senior) |
NCT02565849 |
EAF-8421 |
SCA |
February 2013 |
July 2015 |
August 2015 |
October 1, 2015 |
October 1, 2015 |
|
|
| 30 |
NCT02467257 |
Completed |
Gum Arabic as Fetal Hemoglobin Agent in Sickle Cell Anemia |
|
- Dietary Supplement: Gum Arabic
|
Interventional |
Phase 1
Phase 2 |
- Al-Neelain University
- Military hospital
- Ministry of Higher Education and Scientific Research, Republic of Sudan
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- The primary endpoint of the study will be the level of fetal hemoglobin after three months
|
47 |
All |
5 Years to 50 Years (Child, Adult) |
NCT02467257 |
AlNeelainU |
|
April 2014 |
January 2015 |
January 2015 |
June 10, 2015 |
June 10, 2015 |
|
- Military Hospital
Omdurman, Khartoum, Sudan
|
| 31 |
NCT02286154 |
Recruiting |
Therapeutic Response Evaluation and Adherence Trial (TREAT) |
|
|
Interventional |
Not Applicable |
- Children's Hospital Medical Center, Cincinnati
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Time to Reach Maximum Tolerated Dose (months)
- Hydroxyurea adherence
- Neurological function
- (and 6 more...)
|
150 |
All |
6 Months to 21 Years (Child, Adult) |
NCT02286154 |
CIN_TREAT_001 |
TREAT |
October 2014 |
October 2024 |
October 2024 |
November 7, 2014 |
January 18, 2018 |
|
- Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
|
| 32 |
NCT02162225 |
Suspended |
Study of Beet Juice for Patients With Sickle Cell Anemia |
|
- Drug: beet juice (Unbeetable)
|
Interventional |
Phase 2 |
- Wake Forest University Health Sciences
- Wake Forest University
- National Heart, Lung, and Blood Institute (NHLBI)
|
Other / NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Total Number of Participants with Adverse Events as a Measure of Safety and Tolerability as a function of time
- Changes in Red blood cell properties as a function of time
- Changes in Platelet function as a function of time
|
24 |
All |
19 Years to 65 Years (Adult) |
NCT02162225 |
Wake-58091
R37HL058091 |
|
September 2014 |
February 2019 |
April 2019 |
June 12, 2014 |
November 8, 2017 |
|
- Wake Forest University School of Medicine
Winston-Salem, North Carolina, United States
|
| 33 |
NCT02090296 |
Withdrawn |
Risk-based Therapy for Sickle Cell Anemia: A Feasibility Study |
|
- Drug: Hydroxyurea
- Other: Placebo
|
Interventional |
Phase 2 |
- Children's Research Institute
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Prevention
|
- Sickle Cell Clinical Change from Baseline to Study Completion ( 18 months)
|
0 |
All |
6 Months to 12 Months (Child) |
NCT02090296 |
RBTSCA |
|
February 2014 |
June 2015 |
June 2015 |
March 18, 2014 |
October 18, 2017 |
|
- Children's National Medical Center
Washington, D.C., District of Columbia, United States
|
| 34 |
NCT00971984 |
Completed |
Demographic, Clinical and Laboratory Characteristics of Children With Alpha Thalassemia in Northern Israel |
- Thalassemia Alpha
- Hemolytic Anemia
|
- Other: Medical records summary
|
Observational |
|
- HaEmek Medical Center, Israel
|
Other |
- Observational Model: Case-Only
- Time Perspective: Retrospective
|
- Analysis of the Demographic, Clinical and Laboratory Characteristics of Children With Alpha Thalassemia
|
50 |
All |
up to 20 Years (Child, Adult) |
NCT00971984 |
0074-09-EMC |
|
October 2009 |
April 2012 |
April 2012 |
September 4, 2009 |
September 1, 2015 |
|
- Pediatric Hematology Unit and Pediatric Dpt B - HaEmek Medical Center
Afula, Israel
|
| 35 |
NCT01960413 |
Completed |
Phase 2 Study of Montelukast for the Treatment of Sickle Cell Anemia |
- Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
|
- Drug: Montelukast added to Hydroxyurea
- Drug: Placebo added to Hydroxyurea
|
Interventional |
Phase 2 |
- Vanderbilt University Medical Center
- Medical College of Wisconsin
- Blood Center of Wisconsin
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Triple (Participant, Care Provider, Investigator)
- Primary Purpose: Treatment
|
- Improvement in soluble vascular cell adhesion molecule-1 (sVCAM)
|
46 |
All |
16 Years to 70 Years (Child, Adult, Senior) |
NCT01960413 |
R01FD004117 |
|
November 2013 |
March 2018 |
March 2018 |
October 10, 2013 |
April 3, 2018 |
|
- Vanderbilt University Medical Center
Nashville, Tennessee, United States - Medical College of Wisconsin
Milwaukee, Wisconsin, United States
|
| 36 |
NCT01925001 |
Withdrawn |
Phase 2 Study of MP4CO to Treat Vaso-occlusive Sickle Crisis |
- Anemia, Sickle Cell
- Sickle Cell Anemia
- Sickle Cell Disease
- (and 3 more...)
|
- Drug: MP4CO
- Drug: Sodium chloride solution
|
Interventional |
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Triple (Participant, Care Provider, Investigator)
- Primary Purpose: Treatment
|
- Duration of hospitalization for treatment of painful vaso-occlusive crisis (VOC)
- Pain levels
- Readmission to emergency room (ER)
- (and 2 more...)
|
0 |
All |
16 Years and older (Child, Adult, Senior) |
NCT01925001 |
SCD-206 |
|
October 2013 |
June 2015 |
October 2015 |
August 19, 2013 |
October 28, 2013 |
|
- Salmaniya Medical Complex
Manama, Bahrain - University Hospital Brugmann
Brussels, Belgium - Rio de Janerio Instituto Estadual de Hematologie
Rio de Janerio, Brazil - (and 11 more...)
|
| 37 |
NCT01905787 |
Recruiting |
Sickle Cell Anemia - A Comparative Study Between Three Ethnical Communities, a Multicenter Study |
- Sickle Cell Anemia
- Sickle Cell β+ or β0 Thalassemia
|
|
Observational |
|
- HaEmek Medical Center, Israel
|
Other |
- Observational Model: Case-Only
- Time Perspective: Retrospective
|
- Clinical comparative study between four different Sickle Cell Populations
- Clinical severity and laboratory results related to the Sickle Cell disease
|
300 |
All |
1 Year to 40 Years (Child, Adult) |
NCT01905787 |
0128-11-EMC |
|
January 2012 |
December 31, 2017 |
December 31, 2018 |
July 23, 2013 |
August 9, 2017 |
|
- Children's Hospital of Michigan, Detroit Medical Center - Wayne State University
Detroit, Michigan, United States - Pediatric Hematology Unit HaEmek Medical Center
Afula, Israel - Pediatric Hematology Unit - Schneider Children's Hospital - Beilinson Medical Center
Petah Tikva, Israel - Pediatric Hematology Unit - Dana Children's Hospital - Ichilov Medical Center
Tel Aviv, Israel
|
| 38 |
NCT00840567 |
Terminated |
Skin and Blood Research Samples From Healthy Volunteers and Patients With Hematologic Diseases |
|
- Procedure: Skin biopsy
- Procedure: Blood draw & sample
|
Interventional |
Not Applicable |
- Washington University School of Medicine
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Basic Science
|
- Obtain skin biopsy samples from normal healthy volunteers and patients with a benign, inherited hematologic disease to created induced pluripotent stem cells
- Define the efficiency of homologous recombination in induced pluripotent stem cells derived from skin biopsy samples.
- Define the efficiency of homologous recombination in human embryonic stem cells using NIH-approved cell lines
- (and 2 more...)
|
7 |
All |
18 Years and older (Adult, Senior) |
NCT00840567 |
08-1409 |
|
February 2009 |
October 2010 |
October 2010 |
February 10, 2009 |
July 11, 2013 |
|
- Washington University School of Medicine
St. Louis, Missouri, United States
|
| 39 |
NCT01788631 |
Completed
Has Results |
A Phase II Trial of Regadenoson in Sickle Cell Anemia |
|
- Drug: Regadenoson
- Drug: Placebo
|
Interventional |
Phase 2 |
- Dana-Farber Cancer Institute
- Brigham and Women's Hospital
- Boston Children’s Hospital
- (and 11 more...)
|
Other / NIH |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Triple (Participant, Care Provider, Investigator)
- Primary Purpose: Treatment
|
- Number of Participants With a Reduction in Invariant Natural-Killer T-Cell (iNKT Cell) Activation by 70% or More
- Length of Hospital Stay
- Number of Participants With an Improvement in Respiratory Symptoms
- (and 4 more...)
|
100 |
All |
10 Years to 70 Years (Child, Adult, Senior) |
NCT01788631 |
13-005
1P50HL110790-01 |
|
July 2013 |
November 10, 2016 |
December 12, 2016 |
February 11, 2013 |
February 7, 2018 |
February 7, 2018 |
- Children's Hospital and Research Center at Oakland
Oakland, California, United States - University of Illinois at Chicago
Chicago, Illinois, United States - Johns Hopkins University
Baltimore, Maryland, United States - (and 9 more...)
|
| 40 |
NCT00777231 |
Completed |
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies |
|
- Biological: Enriched Hematopoetic Stem Cell Infusion
|
Interventional |
Phase 1
Phase 2 |
- University of Louisville
- Duke University
- St. Christopher's Hospital for Children
- (and 4 more...)
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Level of Donor Chimerism from Enriched Hematopoietic Stem Cell Engraftment
|
9 |
All |
Child, Adult, Senior |
NCT00777231 |
ICT-7621-042298
549.02
430.98
445.98
417.98
Pro00009281 |
|
January 2005 |
August 2010 |
August 2013 |
October 22, 2008 |
September 19, 2017 |
|
- University of Louisville
Louisville, Kentucky, United States - Duke University Medical Center
Durham, North Carolina, United States - St. Christopher's Hospital for Children
Pittsburgh, Pennsylvania, United States
|
| 41 |
NCT00723567 |
Completed |
A Novel Mutation of the Spectrin Gene |
- Hereditary Elliptocytosis (HE)
- Hereditary Pyropoikilocytosis (HPP)
|
|
Observational |
|
- University of Utah
- University of Witwatersrand, South Africa
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Identify a gene or its mutation (an altered gene) that puts individuals at risk for developing HE or HPP
|
12 |
All |
7 Years and older (Child, Adult, Senior) |
NCT00723567 |
27669 |
|
February 2008 |
December 2008 |
December 2008 |
July 28, 2008 |
February 19, 2009 |
|
- University of Utah
Salt Lake City, Utah, United States
|
| 42 |
NCT01703104 |
Completed |
The Efficacy of Jobelyn (Sorghum Bicolor Extract)in the Treatment of Sickle Cell Anemia |
|
- Drug: Paludrine + Folic Acid
- Dietary Supplement: Paludrine + Folic Acid + Jobelyn
|
Interventional |
Phase 1
Phase 2 |
|
Other |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Single (Care Provider)
- Primary Purpose: Treatment
|
- Number of Participants with Adverse Events
- number of blood transfusions during the 12-week trial period
|
150 |
All |
14 Years to 45 Years (Child, Adult) |
NCT01703104 |
LASUTH/SCD02/2012 |
|
January 2012 |
March 2012 |
August 2012 |
October 10, 2012 |
October 10, 2012 |
|
- Lagos State University Teaching Hospital
Ikeja, Lagos, Nigeria
|
| 43 |
NCT00695123 |
Recruiting |
Screening for Subjects to Participate in Studies of Blood Disorders |
- Bone Marrow Transplant
- Sickle Cell Disease
- G-CSF
|
|
Observational |
|
- National Heart, Lung, and Blood Institute (NHLBI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Observational Model: Other
- Time Perspective: Prospective
|
- Evaluate consented subjects
|
99999999 |
All |
Child, Adult, Senior |
NCT00695123 |
080156
08-H-0156 |
|
June 3, 2008 |
|
|
June 11, 2008 |
April 4, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
| 44 |
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
|
| 45 |
NCT01566890 |
Active, not recruiting |
Microvascular Blood Flow in Sickle Cell Anemia |
- Sickle Cell Disease
- Sickle Cell Anemia
|
- Drug: regadenoson infusion with contrast-enhanced ultrasound
- Procedure: contrast-enhanced ultrasound
|
Interventional |
Not Applicable |
- Blood Center of Wisconsin
- Medical College of Wisconsin
- La Jolla Institute for Allergy & Immunology
- (and 3 more...)
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Factorial Assignment
- Masking: None (Open Label)
- Primary Purpose: Diagnostic
|
- Microvascular Blood Flow in Sickle Cell Anemia with Regadenoson Use
- Changes in Microvascular Blood Flow in Subjects with Sickle Cell Anemia During a Pain Crisis
- Microvascular Blood Flow in Sickle Cell Anemia Subjects Versus Control Subjects
|
200 |
All |
18 Years to 70 Years (Adult, Senior) |
NCT01566890 |
DD2011101 |
|
July 2012 |
December 30, 2017 |
December 31, 2019 |
March 29, 2012 |
November 13, 2017 |
|
- The University of Illinois
Chicago, Illinois, United States - Medical College of Wisconsin
Milwaukee, Wisconsin, United States
|
| 46 |
NCT01558076 |
Unknown † |
Longitudinal Changes in Exercise Capacity in Children and Young Adults With Sickle Cell Anemia |
|
|
Observational |
|
- Ann & Robert H Lurie Children's Hospital of Chicago
- National Heart, Lung, and Blood Institute (NHLBI)
|
Other / NIH |
- Observational Model: Cohort
- Time Perspective: Cross-Sectional
|
- VO2 max on cardiopulmonary exercise test
- Quality of life questionnaires
- Echocardiogram
- (and 2 more...)
|
90 |
All |
8 Years to 21 Years (Child, Adult) |
NCT01558076 |
2011-14565
1K23HL094376 |
|
January 2012 |
December 2016 |
December 2016 |
March 20, 2012 |
September 23, 2015 |
|
- Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
|
| 47 |
NCT01547793 |
Completed |
Carbon Monoxide Levels and Sickle Cell Disease Severity |
- Sickle Cell Disease
- Sickle Cell Anemia
- Anemia, Sickle Cell
|
|
Observational |
|
- National Heart, Lung, and Blood Institute (NHLBI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Time Perspective: Prospective
|
|
106 |
All |
18 Years and older (Adult, Senior) |
NCT01547793 |
120069
12-H-0069 |
|
January 26, 2012 |
|
November 4, 2015 |
March 8, 2012 |
July 2, 2017 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
| 48 |
NCT00578344 |
Terminated |
Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP |
- Sickle Cell Disease
- Hemoglobin SC
|
- Drug: Busulfan
- Biological: Campath 1H
- Drug: Cyclophosphamide and MESNA
|
Interventional |
Not Applicable |
- Baylor College of Medicine
- Texas Children's Hospital
- The Methodist Hospital System
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Evaluate recovery of organ function in patients with sickle cell disease (SCD) or sickle hemoglobin variants after undergoing allogeneic SCT/BMT from HLA genotype identical donors and if they can be improved or reversed.
- Evaluate the use of PET scan examination in assessing metabolic function of organs in patients with SCD, hemoglobin SC, or hemoglobin Sb0/+ after undergoing allogeneic SCT/BMT from HLA genotype identical donors.
- Evaluate response to immunization after BMT in patients with SCD, hemoglobin SC, or hemoglobin Sb0/+.
|
8 |
All |
up to 40 Years (Child, Adult) |
NCT00578344 |
H-16447-SCALLOP
SCALLOP |
SCALLOP |
July 2005 |
July 2012 |
July 2012 |
December 21, 2007 |
April 23, 2014 |
|
- Methodist Hospital
Houston, Texas, United States - Texas Children's Hospital
Houston, Texas, United States
|
| 49 |
NCT01531387 |
Terminated
Has Results |
Sparing Conversion to Abnormal TCD (Transcranial Doppler) Elevation (SCATE) |
|
|
Interventional |
Phase 3 |
- Children's Hospital Medical Center, Cincinnati
- National Heart, Lung, and Blood Institute (NHLBI)
- St. Jude Children's Research Hospital
- (and 2 more...)
|
Other / NIH |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Prevention
|
- Conversion to Abnormal Maximum TAMV
- Serial TCD Velocities
- Cumulative Incidence of Neurological Events
- (and 2 more...)
|
38 |
All |
2 Years to 10 Years (Child) |
NCT01531387 |
H-29205 SCATE
R01HL098239 |
SCATE |
May 2012 |
January 2014 |
January 2014 |
February 13, 2012 |
February 8, 2016 |
December 9, 2015 |
- St. Jude Children's Research Hospital
Memphis, Tennessee, United States - Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti (HEMORIO)
Centro, Rio de Janeiro, Brazil - Tropical Medicine Research Institute, University of the West Indies (UWI)
Mona, Kingston, Jamaica
|
| 50 |
NCT01527799 |
Completed |
Exercise Capacity in Pediatric Sickle Cell Anemia |
|
|
Observational |
|
- Ann & Robert H Lurie Children's Hospital of Chicago
- National Heart, Lung, and Blood Institute (NHLBI)
|
Other / NIH |
- Observational Model: Cohort
- Time Perspective: Cross-Sectional
|
- VO2 max on cardiopulmonary exercise test
- Skin fold measurements to detemine percent body fat
- All patient reported pain episodes
- (and 2 more...)
|
60 |
All |
8 Years to 21 Years (Child, Adult) |
NCT01527799 |
2009-13659
1K23HL094376 |
|
June 2009 |
July 2015 |
July 2015 |
February 7, 2012 |
February 4, 2016 |
|
- Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
|
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