| 1 |
NCT03161899 |
Not yet recruiting |
Evaluation of Nutritional Status in Thalassemia Major Patients in Assiut Children Hospital |
|
|
Observational
|
|
|
Other |
- Observational Model: Case-Only
- Time Perspective: Cross-Sectional
|
- percentage of malnutrition
- aetiology poor growth
- decrease morbidity
|
246 |
All |
6 Years to 14 Years (Child) |
NCT03161899 |
ENST |
|
October 2017 |
May 31, 2018 |
December 1, 2018 |
May 22, 2017 |
July 19, 2017 |
|
|
|
| 2 |
NCT01549080 |
Completed |
Study of Effects of YisuiShengxueGranules on Thalassemia |
|
- Drug: Yisuishengxue Granule
|
Observational
|
|
- Guang'anmen Hospital of China Academy of Chinese Medical Sciences
|
Other |
- Observational Model: Case Control
- Time Perspective: Prospective
|
- Clinical effects on heamoglobin changes
- Chinese Medicine syndrome improvment
|
2 |
All |
3 Years to 40 Years (Child, Adult) |
NCT01549080 |
2011001P02/June 3rd, 2011
2010CB530406 |
thalessemia |
July 2011 |
October 2011 |
January 2012 |
March 8, 2012 |
March 8, 2012 |
|
|
|
| 3 |
NCT03351829 |
Not yet recruiting |
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector |
|
- Genetic: Gene-modified autologous stem cells
|
Interventional
|
Not Applicable |
- Shenzhen Geno-Immune Medical Institute
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety in patients using CTCAE version 4.0 standard to evaluate the level of adverse events
- Treatment responses
- Quality of life
|
20 |
All |
4 Years to 70 Years (Child, Adult, Older Adult) |
NCT03351829 |
GIMI-IRB-17008 |
|
December 1, 2017 |
January 1, 2019 |
December 31, 2020 |
November 24, 2017 |
November 30, 2017 |
|
- Shenzhen Geno-immune Medical Institute
Shenzhen, Guangdong, China
|
|
| 4 |
NCT03342404 |
Recruiting |
A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia |
|
- Drug: Luspatercept
- Other: Placebo
- Other: Best Supportive Care (BSC)
|
Interventional
|
Phase 2 |
- Celgene
- Acceleron Pharma, Inc.
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Proportion of subjects who have an increase from baseline ≥1.0 g/dl in mean of hemoglobin values over a continuous 12-week interval Week 13 to Week 24 in the absence of transfusions
- Mean change from baseline in non-transfusion dependent β- thalassemia-patient reported outcome (NTDT-PRO) Tiredness and Weakness (TW) domain score over a continuous 12-week interval from Week 13 to Week 24
- Mean change from baseline in hemoglobin values over a continuous 12-week interval from Week 13 to Week 24
- (and 21 more...)
|
150 |
All |
18 Years and older (Adult, Older Adult) |
NCT03342404 |
ACE-536-B-THAL-002
U1111-1202-7068
2015-003225-33 |
BEYOND |
February 1, 2018 |
October 15, 2019 |
March 31, 2020 |
November 17, 2017 |
May 31, 2018 |
|
- Children's Hospital of Los Angeles
Los Angeles, California, United States - Children's Hospital and Research Center at Oakland
Oakland, California, United States - Ann & Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, United States - (and 11 more...)
|
|
| 5 |
NCT00872339 |
Completed
Has Results |
Assessment of Pain in People With Thalassemia |
|
|
Observational
|
|
- New England Research Institutes
- National Heart, Lung, and Blood Institute (NHLBI)
|
Other / NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Prevalence of Pain
- Common Sites of Pain
- Pain Occurrence by Age
- Impact of Pain on Functioning and Well-being
|
252 |
All |
12 Years to 90 Years (Child, Adult, Older Adult) |
NCT00872339 |
639
U01HL065238 |
Pain |
March 2009 |
June 2010 |
December 2010 |
March 31, 2009 |
June 5, 2014 |
June 5, 2014 |
- Children's Hospital and Research Institute at Oakland
Oakland, California, United States - Children's Healthcare of Atlanta
Atlanta, Georgia, United States - Children's Memorial Hospital of Chicago
Chicago, Illinois, United States - (and 5 more...)
|
|
| 6 |
NCT01772680 |
Completed |
Zinc and Diabetes in Patients With Thalassemia: a Pilot Study |
|
- Dietary Supplement: Zinc Supplementation
|
Interventional
|
Phase 1 |
- Children's Hospital & Research Center Oakland
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
|
- Oral glucose Tolerance Test
- Fructosamine
|
40 |
All |
12 Years and older (Child, Adult, Older Adult) |
NCT01772680 |
2012-071 |
|
November 2012 |
January 2015 |
May 2015 |
January 21, 2013 |
November 30, 2015 |
|
- Children's Hospital & Research Center Oakland
Oakland, California, United States
|
|
| 7 |
NCT01597765 |
Completed |
Effect of Antioxidant Cocktail in Beta-thalassemia/Hb E Patients |
|
- Drug: curcuminoids, N-acetylcysteine, deferiprone and vitamin E
- Drug: Curcuminoids and alpha-tocopherol
|
Interventional
|
Not Applicable |
- Mahidol University
- ChaingMai University
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double (Care Provider, Investigator)
- Primary Purpose: Treatment
|
|
60 |
All |
18 Years to 50 Years (Adult) |
NCT01597765 |
Si 063/2009 |
|
June 2009 |
June 2011 |
June 2011 |
May 14, 2012 |
May 15, 2012 |
|
- Department of Biochemistry , Faculty of Medicine Siriraj Hospital, Mahidol university
Bangkoknoi, Bangkok, Thailand
|
|
| 8 |
NCT00459732 |
Completed
Has Results |
Zinc & Bone Health in Thalassemia: The Think Zinc Study |
|
- Dietary Supplement: Zinc
- Dietary Supplement: Placebo
|
Interventional
|
Phase 2 |
- Children's Hospital & Research Center Oakland
- National Heart, Lung, and Blood Institute (NHLBI)
- Children's Hospital of Philadelphia
- University of California, San Francisco
|
Other / NIH |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double (Participant, Investigator)
- Primary Purpose: Treatment
|
- Change in Lumbar Spine Bone Mineral Density (BMD) by DXA (Baseline to 18 Months)
- Change in Whole Body Bone Mineral Content (BMC) by DXA (Baseline to 18 Months)
- Osteocalcin, a Marker of Bone Formation
|
45 |
All |
6 Years to 30 Years (Child, Adult) |
NCT00459732 |
2004-106
K23HL076468 |
ThinkZn |
April 2006 |
October 2009 |
February 2011 |
April 12, 2007 |
October 7, 2011 |
September 30, 2011 |
- Children's Hospital & Research Center, Oakland
Oakland, California, United States - University of California, San Francisco
San Francisco, California, United States - Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
|
|
| 9 |
NCT02453477 |
Active, not recruiting |
Gene Therapy for Transfusion Dependent Beta-thalassemia |
|
- Genetic: Autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene
|
Interventional
|
Phase 1
Phase 2 |
- IRCCS San Raffaele
- Fondazione Telethon
- GlaxoSmithKline
|
Other / Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Overall survival
- Achievement of hematological engraftment
- Safety of the administration of autologous haematopoietic stem cells transduced with LV-GLOBE
- (and 9 more...)
|
10 |
All |
3 Years to 64 Years (Child, Adult) |
NCT02453477 |
2014-004860-39 (EudraCT) |
TIGET-BTHAL |
May 2015 |
August 2019 |
August 2019 |
May 25, 2015 |
May 4, 2018 |
|
- Ospedale San Raffaele
Milano, Italy
|
|
| 10 |
NCT00661726 |
Completed
Has Results |
Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia |
|
- Drug: Decitabine (USAN, INN)
|
Interventional
|
Phase 2 |
- New England Research Institutes
- National Heart, Lung, and Blood Institute (NHLBI)
|
Other / NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of Evaluable Patients With an Increase From Baseline in Hemoglobin (Hb) of ≥1.5 g/dL
- Change in Total Hemoglobin (Hb) From Baseline to Peak (the Follow-up Time Point With the Highest Value)
- Change in Absolute Fetal Hemoglobin (HbF) From Baseline to Peak (the Follow-up Time Point With the Highest Value)
- (and 9 more...)
|
6 |
All |
18 Years and older (Adult, Older Adult) |
NCT00661726 |
569
U01HL065238
68,995 |
|
January 2008 |
May 2010 |
September 2010 |
April 18, 2008 |
April 25, 2014 |
April 8, 2014 |
- Children's Hospital and Research Center at Oakland
Oakland, California, United States - Children's Hospital Philadelphia
Philadelphia, Pennsylvania, United States - University Health Network
Toronto, Canada
|
|
| 11 |
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...)
|
|
| 12 |
NCT00789516 |
Completed |
The Change of Coagulation Markers in Children With β-thalassemia Disease After Stem Cell Transplantation |
|
|
Observational
|
|
|
Other |
- Observational Model: Case Control
- Time Perspective: Cross-Sectional
|
- Level of protein C,S and AT, TAT, P1+2 and D-dimer
|
60 |
All |
1 Year to 18 Years (Child, Adult) |
NCT00789516 |
ID11-48-16 |
|
June 2006 |
March 2009 |
December 2009 |
November 13, 2008 |
March 7, 2013 |
|
- Department of Pediatrics, Ramathibodi hospital
Bangkok, Thailand
|
|
| 13 |
NCT03170245 |
Not yet recruiting |
Adiponectin, IL-6 and hsC-RP in Relation to Carotid Intima-media Thickness in B-thalassemia Patients |
|
- Diagnostic Test: Interleukin-6
- Diagnostic Test: HsC-RP
- Diagnostic Test: Adiponectin level
- Diagnostic Test: Carotid intima media thickness
|
Observational
|
|
|
Other |
- Observational Model: Case-Control
- Time Perspective: Prospective
|
- Adiponectin
- HsC-reactive protein
- Interleukin-6
- carotid intima media thickness
|
80 |
All |
16 Years and older (Child, Adult, Older Adult) |
NCT03170245 |
ADICIBT |
|
January 1, 2018 |
January 1, 2020 |
September 2020 |
May 31, 2017 |
December 27, 2017 |
|
- Assiut University Hospital
Assiut, Egypt
|
|
| 14 |
NCT02308904 |
Recruiting |
Reproductive Capacity and Iron Burden in Thalassemia |
|
- Other: Blood Draw/Semen Exam
- Other: Retrospective data/Chart Review/Relevant Clinical Results
- Other: Pituitary MRI
|
Interventional
|
Not Applicable |
- Children's Hospital & Research Center Oakland
- Novartis Pharmaceuticals
|
Other / Industry |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Diagnostic
|
- Laboratory studies for pituitary and gonadal function and for iron level measures
- Retrospective analysis of annual LIC and of type and dose of iron chelation agent in the parallel time period
|
30 |
All |
12 Years and older (Child, Adult, Older Adult) |
NCT02308904 |
CICL670AUS46T |
Fertility thal |
June 2013 |
June 2018 |
June 2018 |
December 4, 2014 |
July 24, 2017 |
|
- UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
|
|
| 15 |
NCT03101423 |
Recruiting |
Monitoring of Chimerism After Transplantation in Patients With β Thalassemia Major and the Treatment Strategies for the Reduction of Chimerism |
|
- Drug: Interleukin-2
- Drug: Donor Regulatory T-Lymphocytes
|
Interventional
|
Not Applicable |
- First Affiliated Hospital of Guangxi Medical University
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Crossover Assignment
- Masking: Single (Participant)
- Primary Purpose: Treatment
|
- Chimerism after transplantation were monitored using fluorescence labeled multiplex PCR amplification of short tandem repeats (STR-PCR)
|
10 |
All |
Child, Adult, Older Adult |
NCT03101423 |
LBo |
|
August 1, 2016 |
December 31, 2017 |
December 31, 2017 |
April 5, 2017 |
May 9, 2017 |
|
- the First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, China
|
|
| 16 |
NCT00872833 |
Completed
Has Results |
Assessment of Pain in People With Thalassemia Who Are Treated With Regular Blood Transfusions |
|
|
Observational
|
|
- New England Research Institutes
- National Heart, Lung, and Blood Institute (NHLBI)
|
Other / NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Report of Pain by Age Group
- Report of Pain by Length of the Transfusion Cycle
|
32 |
All |
18 Years and older (Adult, Older Adult) |
NCT00872833 |
640
U01HL065238 |
|
July 2009 |
October 2010 |
October 2010 |
March 31, 2009 |
October 7, 2015 |
August 27, 2014 |
- Children's Hospital of Los Angeles
Los Angeles, California, United States - Children's Hospital Research Center Oakland
Oakland, California, United States - Stanford University - School of Medicine
Stanford, California, United States - (and 6 more...)
|
|
| 17 |
NCT02049450 |
Completed
Has Results |
Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia. |
|
|
Interventional
|
Phase 2 |
- Novartis Pharmaceuticals
- Novartis
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change of Hematocrit Adjusted Volume of Red Blood Cells (RBC)
- Percentage Change in Spleen Volume (cm3)
- Percentage Change in Mean Pre-transfusion Hemoglobin by 6 Week Time Intervals
- (and 3 more...)
|
30 |
All |
18 Years and older (Adult, Older Adult) |
NCT02049450 |
CINC424X2201
2013-002812-28 |
|
May 28, 2014 |
April 12, 2016 |
April 12, 2016 |
January 30, 2014 |
July 17, 2017 |
June 15, 2017 |
- Novartis Investigative Site
Athens, GR, Greece - Novartis Investigative Site
Milano, MI, Italy - Novartis Investigative Site
Palermo, PA, Italy - (and 4 more...)
|
|
| 18 |
NCT03275051 |
Recruiting |
Long-term Safety and Efficacy Follow-up of Subjects Treated With GSK2696277 for Transfusion Dependent Beta-thalassemia in San Raffaele Telethon Institute of Gene Therapy-Beta Thalassemia (TIGET-BTHAL) Study |
|
- Other: Safety and Efficacy assessments
|
Interventional
|
Phase 2 |
- GlaxoSmithKline
- San Raffaele Telethon Institute of Gene Therapy (SR-TIGET)
|
Industry / Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of subjects with absence of abnormal clonal proliferation (ACP)
- Number of subjects with Polyclonal engraftment
- Number of subjects with reduction in red blood cells (RBC) transfusion volume
- (and 19 more...)
|
10 |
All |
3 Years and older (Child, Adult, Older Adult) |
NCT03275051 |
207757 |
|
October 4, 2017 |
May 1, 2026 |
May 1, 2026 |
September 7, 2017 |
October 17, 2017 |
|
- GSK Investigational Site
Milano, Lombardia, Italy
|
|
| 19 |
NCT03462784 |
Not yet recruiting |
Complications in Children With B- Thalassemia Major |
|
- Other: Demographic, physical examination, data will becollected .
|
Observational
|
|
|
Other |
- Observational Model: Other
- Time Perspective: Cross-Sectional
|
- Prevelance of Comlications of Beta -Thalassemia major in children
|
201 |
All |
1 Year to 18 Years (Child, Adult) |
NCT03462784 |
Prev TM |
|
May 1, 2018 |
March 1, 2019 |
October 1, 2019 |
March 13, 2018 |
March 13, 2018 |
|
|
|
| 20 |
NCT03276455 |
Not yet recruiting |
Gene Therapy for Beta-Thalassemia Major Using Autologous Hematopoietic Stem Cell Genetically Modified |
|
- Genetic: Autologous CD34+ cells genetically modified
|
Interventional
|
Phase 1
Phase 2 |
- Nanfang Hospital of Southern Medical University
- Guangdong Yike Gene Science and Technology CO.,Ltd
|
Other / Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- incidence of adverse events
- hemoglobin conten
- Hematopoietic stem cell engraftment
- (and 3 more...)
|
10 |
All |
8 Years and older (Child, Adult, Older Adult) |
NCT03276455 |
NanFangHospital |
|
September 15, 2017 |
September 15, 2020 |
September 15, 2021 |
September 8, 2017 |
September 8, 2017 |
|
- Nanfang Hospial
Guangzhou, Guangdong, China
|
|
| 21 |
NCT01571635 |
Active, not recruiting |
Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia. |
- Beta Thalassemia Major
- Beta Thalassemia Intermedia
|
- Drug: Sotatercept 0.1mg/kg
- Drug: Sotatercept 0.3mg/kg
- Drug: Sotatercept 0.5mg/kg
- (and 3 more...)
|
Interventional
|
Phase 2 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Potential Recommended Dose (PRD)
- Actual Recommended Dose (RD)
- Reduction of Transfusion Burden
- (and 6 more...)
|
46 |
All |
18 Years and older (Adult, Older Adult) |
NCT01571635 |
ACE-011-B-THAL-001
2011-005659-15 |
|
October 10, 2012 |
July 2, 2015 |
February 28, 2020 |
April 5, 2012 |
May 16, 2018 |
|
- Hopital Henri Mondor
Créteil, France - Hospital of Necker
Paris, France - Laiko General Hospital
Ampelokipi - Athens, Greece - (and 4 more...)
|
|
| 22 |
NCT02069886 |
Withdrawn |
Effect of Deferasirox on Endocrine Complications in Subjects With Transfusion Dependent Thalassemia |
- Thalassemia (Transfusion Delendent)
|
|
Interventional
|
Phase 4 |
- Novartis Pharmaceuticals
- Novartis
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Supportive Care
|
- Change from baseline of glucose blood level measured after 2 h after receiving a glucose-equivalent oral challenge
- Glucose of OGTT ( AUC)
- change on insulin secretion and sensitivity
- (and 6 more...)
|
0 |
All |
2 Years to 18 Years (Child, Adult) |
NCT02069886 |
CICL670AIT12 |
CENTAurus |
December 2014 |
December 2018 |
December 2018 |
February 24, 2014 |
April 20, 2017 |
|
|
|
| 23 |
NCT02626689 |
Completed |
To Document the Burden of Illness on the Quality of Life and the Impact on Healthcare Utilization in (Beta) β-thalassemia Subjects Who Are Transfusion Dependent (TD) and Non-transfusion Dependent (NTD) Receiving Standard of Care |
|
- Other: Quality of Life (QOL) questionnaires
- Other: Healthcare Resource Utilization
|
Observational
|
|
|
Industry |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Change in the Physical component score (PSC) over the study period versus the country specific population norms using the 36-item Short Form (SF-36) Quality of Life instrument
- Change in the Mental component score (MCS) over the study period versus the country specific population norms using the 36-item Short Form (SF-36) Quality of Life instrument
- Change in the total scores over the study period using the The Functional Assessment of Cancer Therapy-Anemia (FACT-An) questionnaire
- (and 10 more...)
|
100 |
All |
18 Years and older (Adult, Older Adult) |
NCT02626689 |
ACE-536-B-THAL-003 |
|
March 1, 2016 |
January 3, 2017 |
January 3, 2017 |
December 10, 2015 |
April 7, 2017 |
|
- Aghia Sofia Children's Hosptial
Athens, Goudi, Greece - Fondazione IRCCS Ca Granda Ospedale Maggiore
Milano, Italy - American University of Beirut Medical Center
Beirut, Lebanon - (and 2 more...)
|
|
| 24 |
NCT03358498 |
Not yet recruiting |
Quality of Life and Treatment Satisfaction in β-Thalassemia Patients Receiving Deferasirox |
|
- Other: The Medical Outcomes Short-Form 36-Item Health Survey (SF-36v2) It
|
Observational
|
|
|
Other |
- Observational Model: Cohort
- Time Perspective: Cross-Sectional
|
- The Medical Outcomes Short-Form 36-Item Health Survey (SF-36v2)
|
75 |
All |
16 Years to 65 Years (Child, Adult, Older Adult) |
NCT03358498 |
QOLATSIBTPRD |
|
December 1, 2017 |
December 1, 2018 |
March 1, 2020 |
November 30, 2017 |
November 30, 2017 |
|
- Assiut University Hospital
Assiut, Egypt
|
|
| 25 |
NCT03171831 |
Recruiting |
Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major |
|
- Drug: Busulfan
- Drug: Cyclophosphamide
- Drug: Fludarabine
- (and 5 more...)
|
Interventional
|
Phase 4 |
- First Affiliated Hospital of Guangxi Medical University
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Overall Survival
- Disease Free Survival
- Engraftment
- (and 4 more...)
|
30 |
All |
2 Years to 18 Years (Child, Adult) |
NCT03171831 |
Guangxi-Haplo-HSCT-2016 |
|
April 1, 2017 |
December 31, 2021 |
April 1, 2022 |
May 31, 2017 |
May 31, 2017 |
|
- First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, China
|
|
| 26 |
NCT01709838 |
Active, not recruiting |
Efficacy and Safety Study of Deferasirox in Patients With Non-transfusion Dependent Thalassemia |
- Non-transfusion Dependent Thalassemia
|
|
Interventional
|
Phase 4 |
- Novartis Pharmaceuticals
- Novartis
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change in liver iron concentration
- Medical Outcomes Study Short Form-36
- Pediatric Quality of Life Questionnaires
- (and 5 more...)
|
134 |
All |
10 Years and older (Child, Adult, Older Adult) |
NCT01709838 |
CICL670E2419
2012-000650-64 |
THETIS |
December 6, 2012 |
January 3, 2015 |
January 17, 2019 |
October 18, 2012 |
May 10, 2018 |
|
- Novartis Investigative Site
Nanning, Guangxi, China - Novartis Investigative Site
Athens, GR, Greece - Novartis Investigative Site
Cagliari, CA, Italy - (and 8 more...)
|
|
| 27 |
NCT02342145 |
Recruiting |
Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major |
|
- Drug: Basiliximab,
- Drug: cyclosporine A
- Drug: Methotrexate
- Drug: Mycophenolate mofetil
|
Interventional
|
Phase 4 |
- Affiliated hospital of guangxi medical university,china
- Union hospital of Fujian Medical University
- Zhongshan Hospital Xiamen University
- (and 3 more...)
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Prevention
|
- acute graft-versus-host disease incidence
- Implantation rate
- Transplanted-related mortality
- (and 4 more...)
|
40 |
All |
2 Years to 18 Years (Child, Adult) |
NCT02342145 |
gxmuh-2014-14 |
|
June 2014 |
June 2016 |
December 2018 |
January 19, 2015 |
January 19, 2015 |
|
- Union hospital of fujian medical university
Fuzhou, Fujian, China - the zhongshan hospital of Xiamen University
Xia'men, Fujian, China - The affiliated hospital of guangxi medical university
Nanning, Guangxi, China - (and 2 more...)
|
|
| 28 |
NCT02950857 |
Completed |
A Study of EPEG in Beta Thalassemia Patients |
|
|
Interventional
|
Phase 1 |
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants in each treatment arm with abnormal laboratory values and/or adverse events that are related to treatment.
- Change in the Hemoglobin from Baseline to the Final Visit
- A mean increase in Hematocrit (Hct) from Baseline to the Final Visit
- (and 2 more...)
|
6 |
All |
18 Years to 65 Years (Adult, Older Adult) |
NCT02950857 |
PETH-001 |
|
June 28, 2017 |
September 27, 2017 |
September 27, 2017 |
November 1, 2016 |
May 25, 2018 |
|
- Faculty of Medicine, Chiang Mai University
Chiang Mai, Thailand
|
|
| 29 |
NCT01129076 |
Completed |
Perceptions of Thalassemia Major in Singapore: An Exploratory Study of Stigma |
|
|
Observational
|
|
- National Human Genome Research Institute (NHGRI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Time Perspective: Prospective
|
|
30 |
All |
14 Years and older (Child, Adult, Older Adult) |
NCT01129076 |
999910104
10-HG-N104 |
|
April 23, 2010 |
|
January 7, 2016 |
May 24, 2010 |
June 14, 2018 |
|
- Singapore General Hospital Outram Rd.
Singapore, Singapore - KK Women's & Children's Hospital
Singapore, Singapore
|
|
| 30 |
NCT01642758 |
Completed |
Trial of HQK-1001 in Beta Thalassemia Intermedia in Lebanon |
- Beta Thalassemia Intermedia
|
- Drug: Sodium 2,2 dimethylbutyrate
|
Interventional
|
Phase 2 |
- Boston University
- HemaQuest Pharmaceuticals Inc.
|
Other / Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- To measure changes from baseline in total hemoglobin when HQK-1001 is administered orally for 26 weeks in subjects with beta thalassemia intermedia.
- To measure the number of adverse events which occur with HQK-1001 treatment when given over 26 weeks in beta thalassemia intermedia.
- To measure changes from baseline in HbF during treatment with HQK-1001 for 26 weeks in beta thalassemia intermedia.
|
10 |
All |
16 Years to 50 Years (Child, Adult) |
NCT01642758 |
HQK-P2-THAL |
LB-04-THAL |
May 2012 |
November 2012 |
January 2013 |
July 17, 2012 |
March 14, 2013 |
|
- Chronic Care Center
Beirut, Lebanon
|
|
| 31 |
NCT03271541 |
Recruiting |
A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent (NTD) Beta-Thalassemia |
|
|
Interventional
|
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety Outcome: Percentage of Participants with Adverse Events (AEs) - Part 1 only
- Efficacy Outcome: Change in Total Hemoglobin (Hb) Level from Baseline to End of 16-Week Treatment Period in Part 1
- Long-term Safety Outcome : Percentage of Participants with Adverse Events (AEs) - Part 2 only
- (and 12 more...)
|
24 |
All |
18 Years to 55 Years (Adult) |
NCT03271541 |
BP39642
2016-004799-23 |
|
October 26, 2017 |
August 31, 2019 |
October 2, 2019 |
September 5, 2017 |
June 11, 2018 |
|
- Centro della Microcitemia e delle Anemie Congenite - Ospedale Galliera; Oncologia /Cardiologia
Genova, Liguria, Italy - Ospedale Maggiore di Milano; Cardio-Metabolic Diseases
Milano, Lombardia, Italy - Chronic Care Center
Baabda, Lebanon - Siriraj Hospital; Division of Haematology-Oncology
Bangkok Noi, Thailand
|
|
| 32 |
NCT01609595 |
Completed |
Study of SDMB (2,2 Dimethylbutyrate, Sodium Salt) in Beta Thalassemia Intermedia in Thailand |
- Beta Thalassemia Intermedia
|
- Drug: sodium 2,2 dimethylbutyrate
|
Interventional
|
Phase 2 |
- Boston University
- Mahidol University
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- The number of participants in which an increase in total hemoglobin of at least 1.5 g/dl above average baseline occurs with study drug treatment.
- The proportion of participants in which an increase in fetal hemoglobin occurs above the subjects' averaged baseline levels.
|
10 |
All |
18 Years to 55 Years (Adult) |
NCT01609595 |
ST20-P2T
HQK-1001-Thal P2 |
ST20-P2T |
March 2012 |
November 2012 |
December 2012 |
June 1, 2012 |
March 13, 2013 |
|
- Mahidol University Thalassemia Research Centre
Nakhonpathom, Thailand
|
|
| 33 |
NCT01206075 |
Completed |
Evaluating the Safety and Effectiveness of Mozobil Mobilization in Adults With Beta-Thalassemia Major |
|
|
Interventional
|
Not Applicable |
- University of Washington
- National Heart, Lung, and Blood Institute (NHLBI)
- George Papanicolaou Hospital
- Genzyme, a Sanofi Company
|
Other / NIH / Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety and effectiveness of Mozobil for mobilization of patients with beta thalassemia major
- Clonogenic capacity, transducibility, and engraftment potential (in a mouse model) of genetically modified cells
|
20 |
All |
18 Years to 50 Years (Adult) |
NCT01206075 |
36078-B
P01HL053750
2P01HL053750 |
|
October 2010 |
December 2012 |
December 2014 |
September 21, 2010 |
December 30, 2014 |
|
- George Papanicolaou Hospital
Thessaloniki, Greece
|
|
| 34 |
NCT00346242 |
Completed |
Evaluation of Efficacy of Zoledronic Acid in Patients With Haemoglobin Syndromes (Thalassemia and Sicle Cell Anaemia) and Risk of Skeletal Events |
|
|
Interventional
|
Phase 4 |
- Novartis Pharmaceuticals
- Novartis
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Single
- Primary Purpose: Prevention
|
- Bone density by means of bone densitometry with dual energy x-ray absorptiometry (DEXA) at three body sites (lumbar region, femoral head, wrist) prior to treatment initiation the start and at the end of treatment
- N-telopeptide of type I collagen (NTx) levels, TRACP-5b, with ELISA, prior to the start of treatment initiation and every 3 or 6 months
- Serum Bone alkaline phosphatase, Osteocalcin, OPG and S-RANKL with ELISA, prior to the start of treatment initiation, and every 3 or 6 months
- Biochemistry (Ca, P, urea/creatinine, SGOT/SGPT, ALT, γGT), endocrine and cardiac test prior to the start of treatment initiation and at 6 and 12 month.
|
60 |
All |
18 Years and older (Adult, Older Adult) |
NCT00346242 |
CZOL446EGR05 |
|
March 2004 |
March 2006 |
|
June 29, 2006 |
May 1, 2012 |
|
- Novartis Investigative Site
Athens, Greece
|
|
| 35 |
NCT02597595 |
Active, not recruiting |
Effects of Spirulina on Cardiac Functions in Children With Beta Thalassemia Major |
|
|
Interventional
|
Not Applicable |
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Single (Care Provider)
- Primary Purpose: Prevention
|
- cardiac functions measured by echocardiography
- cardiac functions measured by troponin-1 plasma level.
|
60 |
All |
4 Years to 18 Years (Child, Adult) |
NCT02597595 |
2771/09/14 |
|
November 2014 |
October 2017 |
October 2017 |
November 5, 2015 |
April 28, 2017 |
|
- Faculty of Medicine- Tanta University
Tanta, Gharbia, Egypt
|
|
| 36 |
NCT02307786 |
Unknown † |
Long Term Outcomes in β Thalassemia Major |
|
|
Observational
|
|
- Ann & Robert H Lurie Children's Hospital of Chicago
|
Other |
- Observational Model: Cohort
- Time Perspective: Retrospective
|
- Beta-Thalassemia patients and Quality of Life after HSCT versus supportive care therapy
|
150 |
All |
up to 30 Years (Child, Adult) |
NCT02307786 |
SCT 0414B |
|
June 2014 |
June 2016 |
June 2016 |
December 4, 2014 |
December 4, 2014 |
|
- Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
|
|
| 37 |
NCT01948817 |
Withdrawn |
Deferasirox BID (Twice a Day) in Transfusion Dependent Thalassemia Patients With Inadequate Response to High Doses |
- Focus on Transfusion Dependent Thalassemia Patients Who Are Inadequate Responders to Deferasirox > 35mg/kgQD
|
|
Interventional
|
Phase 2 |
- Novartis Pharmaceuticals
- Novartis
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Mean relative change in SF (Serum Ferritin)
- Mean Absolute/relative change in LIC
|
0 |
All |
2 Years and older (Child, Adult, Older Adult) |
NCT01948817 |
CICL670A2420
2013-002624-16 |
|
February 2014 |
February 2016 |
February 2016 |
September 24, 2013 |
April 20, 2017 |
|
|
|
| 38 |
NCT03183375 |
Recruiting |
The Efficacy and Safety of HYDROXYUREA in Management of Beta Thalassemia Patients in Karachi Pakistan |
|
|
Interventional
|
Phase 2 |
- Dr.Saqib Hussain Ansari
- Omair Sana Foundation
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- No. of participants who become responders or partial responders
- The compliance of Hydroxyurea in study participants.
- Number of participants with abnormal/deranged laboratory values
- No. of participants with severe adverse events related to the intervention
|
100 |
All |
6 Months and older (Child, Adult, Older Adult) |
NCT03183375 |
ERB0002 |
|
August 21, 2017 |
April 30, 2018 |
June 30, 2018 |
June 12, 2017 |
September 20, 2017 |
|
- Omair Sana Foundation
Karachi, Sindh, Pakistan
|
|
| 39 |
NCT00456690 |
Completed |
Endocrine and Nutritional Assessment in B Thalassemia Major |
|
- Other: Nutritional recommendations
|
Interventional
|
Not Applicable |
- HaEmek Medical Center, Israel
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Supportive Care
|
|
133 |
All |
5 Years to 40 Years (Child, Adult) |
NCT00456690 |
5190906/2.EMC |
|
May 2007 |
December 2014 |
December 2014 |
April 5, 2007 |
September 1, 2015 |
|
- Pediatric Hematology Unit - HaEmek Medical Center
Afula, Israel
|
|
| 40 |
NCT03207009 |
Recruiting |
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia, Who Have a β0/β0 Genotype |
|
- Genetic: LentiGlobin BB305 Drug Product
|
Interventional
|
Phase 3 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- The proportion of subjects who meet the definition of "transfusion reduction" (TR).
- The proportion of treated subjects who meet the definition of "transfusion independence" (TI). TI is defined as Hb ≥9g/dL without any RBC transfusions for a continuous period of ≥12 months at any time during the study after drug product infusion.
- Percentage of subjects with a reduction in the mL/kg RBC transfused from Month 12 through Month 24 after drug product infusion of at least 50% compared to the average annual RBC transfusion requirement during the 2 years prior to enrollment.
- (and 4 more...)
|
15 |
All |
up to 50 Years (Child, Adult) |
NCT03207009 |
HGB-212 |
|
June 8, 2017 |
April 2021 |
April 2021 |
July 2, 2017 |
February 7, 2018 |
|
- UCSF Benioff Children's Hospital Oakland
Oakland, California, United States - Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States - Hopital d'enfants de la Timone
Marseille, France - (and 4 more...)
|
|
| 41 |
NCT01323608 |
Unknown † |
The Effect of Vitamin D Supplementation on Calcium Excretion in Thalassemia: a Dose Response Study |
|
- Drug: Vitamin D3
- Drug: Placebo
|
Interventional
|
Phase 4 |
- Weill Medical College of Cornell University
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Diagnostic
|
- Vitamin D Dose Response Curve
|
40 |
All |
6 Years to 60 Years (Child, Adult) |
NCT01323608 |
1102011521 |
|
June 2011 |
June 2014 |
December 2014 |
March 25, 2011 |
April 19, 2011 |
|
- Weill Cornell Medical College
New York, New York, United States
|
|
| 42 |
NCT01005576 |
Completed
Has Results |
Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia |
|
- Drug: Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan
|
Interventional
|
Phase 2 |
- Washington University School of Medicine
- New England Research Institutes
- Pediatric Blood and Marrow Transplant Consortium
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Primary Objective: Event-free Survival at 1 Year.
- Development of Graft Versus Host Disease (GVHD)
- Median Time to ANC Engraftment
- (and 2 more...)
|
21 |
All |
1 Year to 16 Years (Child) |
NCT01005576 |
TCRN-NMD 0901 |
URTH |
January 2010 |
April 2014 |
July 2014 |
November 2, 2009 |
December 13, 2017 |
October 20, 2017 |
- Regents of University of California- UCLA
Los Angeles, California, United States - Children's Hospital and Research Center at Oakland
Oakland, California, United States - Children's National Medical Center
Washington, District of Columbia, United States - (and 15 more...)
|
|
| 43 |
NCT02268409 |
Active, not recruiting |
ACE-536 Extension Study - Beta Thalassemia |
|
|
Interventional
|
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Long-term safety and tolerability of ACE-536 in patients with β thalassemia who were previously enrolled in study A536-04
- Erythroid response (8-week) in non-transfusion dependent (NTD) patients
- Erythroid response (12-week) in non-transfusion dependent (NTD) patients
- (and 10 more...)
|
64 |
All |
18 Years and older (Adult, Older Adult) |
NCT02268409 |
A536-06 |
|
November 2014 |
February 2021 |
April 2021 |
October 20, 2014 |
January 9, 2018 |
|
- Acceleron Investigative Site
Athens, Greece - Acceleron Investigative Site
Brindisi, Italy - Acceleron Investigative Site
Catania, Italy - (and 4 more...)
|
|
| 44 |
NCT02906202 |
Recruiting |
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype |
|
- Genetic: LentiGlobin BB305 Drug Product
|
Interventional
|
Phase 3 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- The proportion of treated subjects who meet the definition of "transfusion independence" (TI).
- Engraftment defined as an absolute neutrophil count ≥500 cells/µL for 3 consecutive days
- Detection of vector-derived replication competent lentivirus (RCL) using a RCL screening assay
- (and 3 more...)
|
23 |
All |
up to 50 Years (Child, Adult) |
NCT02906202 |
HGB-207 |
|
July 2016 |
January 2020 |
January 2020 |
September 20, 2016 |
February 28, 2018 |
|
- Oakland, California, United States
- Chicago, Illinois, United States
- Philadelphia, Pennsylvania, United States
- (and 6 more...)
|
|
| 45 |
NCT03432364 |
Recruiting |
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT) |
- Transfusion Dependent Beta-thalassemia
|
- Genetic: ST-400 Investigational product
|
Interventional
|
Phase 1
Phase 2 |
- Sangamo Therapeutics
- Bioverativ Therapeutics Inc.
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Incidence of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) in subjects who receive ST-400 as assessed by Common Terminology Criteria for Adverse Events (CTCAE)
- Change from baseline clinical laboratory measurement of Hb fractions (A and F in g/dL)
- Change from baseline percent (%) HbF
- (and 2 more...)
|
6 |
All |
18 Years to 40 Years (Adult) |
NCT03432364 |
ST-400-01 |
|
May 2018 |
May 2020 |
May 2022 |
February 14, 2018 |
May 28, 2018 |
|
- UCSF Benioff Children's Hospital Oakland
Oakland, California, United States - University of Minnesota
Minneapolis, Minnesota, United States
|
|
| 46 |
NCT00578292 |
Terminated |
Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO |
|
- Drug: Busulfan
- Drug: Fludarabine
- Drug: Campath 1H
- (and 2 more...)
|
Interventional
|
Not Applicable |
- Baylor College of Medicine
- Texas Children's Hospital
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Evaluate engraftment.
- To evaluate the occurrence of transient and stable mixed hematopoietic chimerism (HC) after unrelated donor SCT, and its effect on the recurrence of clinically measurable thalassemia.
- To measure hematopoietic and immune reconstitution, and assess the effects on infectious complications.
- (and 4 more...)
|
10 |
All |
up to 64 Years (Child, Adult) |
NCT00578292 |
H-14539-THALLO
THALLO |
THALLO |
February 2004 |
May 2016 |
May 2016 |
December 21, 2007 |
July 22, 2016 |
|
- Texas Children's Hospital
Houston, Texas, United States
|
|
| 47 |
NCT01863173 |
Completed |
Effect of Metoprolol on Thalassemia Cardiomyopathy |
|
- Drug: metoprolol
- Drug: placebo
|
Interventional
|
Phase 2
Phase 3 |
- Shiraz University of Medical Sciences
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- ejection fraction
- 6 minutes walk test
- endsystolic volume
- patient compliance
|
45 |
All |
13 Years to 25 Years (Child, Adult) |
NCT01863173 |
91-3502 |
|
January 2012 |
May 2012 |
December 2012 |
May 27, 2013 |
May 27, 2013 |
|
- Shiraz University of Medical Sciences
Shiraz, Fars, Iran, Islamic Republic of
|
|
| 48 |
NCT01749540 |
Completed |
Study to Evaluate the Effects of ACE-536 in Patients With Beta-thalassemia |
|
|
Interventional
|
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Proportion of patients who have an erythroid response.
- Number of patients with adverse events.
- Change in hemoglobin level in non-transfusion dependent patients.
- (and 2 more...)
|
64 |
All |
18 Years and older (Adult, Older Adult) |
NCT01749540 |
A536-04
2012-002499-15 |
|
February 2013 |
November 2015 |
November 2015 |
December 13, 2012 |
December 14, 2016 |
|
- Laiko General Hospital, Ampelokipi
Athens, Greece - Ospedale "A. Perriino" U.O Ematologia
Brindisi, Italy - ARNAS Garibaldi - P.O. Garibaldi Centro
Catania, Italy - (and 5 more...)
|
|
| 49 |
NCT00661804 |
Completed |
Evaluating People With Thalassemia: The Thalassemia Longitudinal Cohort (TLC) Study |
|
|
Observational
|
|
- New England Research Institutes
- National Heart, Lung, and Blood Institute (NHLBI)
- Thalassemia Clinical Research Network
|
Other / NIH |
- Time Perspective: Prospective
|
- The prevalence and incidence of complications specific to thalassemia and its treatment among participants
- Fertility and pregnancy outcomes; causes of mortality and changes in mortality risk; genotypic and phenotypic variation; and body iron burden
- Relationships among adherence, quality of life, and complications of thalassemia
|
416 |
All |
5 Years and older (Child, Adult, Older Adult) |
NCT00661804 |
568
U01HL065238 |
|
May 2007 |
December 2010 |
June 2011 |
April 18, 2008 |
November 11, 2011 |
|
- Children's Hospital of Los Angeles
Los Angeles, California, United States - Children's Hospital of Oakland
Oakland, California, United States - Stanford Hospital
Stanford, California, United States - (and 11 more...)
|
|
| 50 |
NCT01745120 |
Completed |
A Study Evaluating the Safety and Efficacy of the LentiGlobin® BB305 Drug Product in Beta-Thalassemia Major Subjects |
|
- Genetic: LentiGlobin® BB305 Drug Product
|
Interventional
|
Phase 1
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Evaluate the efficacy of treatment with LentiGlobin® BB305 Drug Product in subjects with β-thalassemia major as measured by the production of hemoglobin containing the therapeutic globin protein [βA-T87Q-globin]
- Evaluate the safety of treatment with LentiGlobin® BB305 Drug Product in subjects with β-thalassemia major as measured by the incidence of adverse events
- Hematopoietic stem cell engraftment
- Assess transgene marking as determined by measurement of the average vector copy number in peripheral blood and bone marrow
|
18 |
All |
12 Years to 35 Years (Child, Adult) |
NCT01745120 |
HGB-204 |
|
August 2013 |
February 8, 2018 |
February 21, 2018 |
December 7, 2012 |
March 30, 2018 |
|
- Oakland, California, United States
- Chicago, Illinois, United States
- Philadelphia, Pennsylvania, United States
- (and 2 more...)
|
|
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