Hemoglobinopathies
Information
- Disease name
- Hemoglobinopathies
- Disease ID
- Description
Disease area statistics
[No Data.]
Chromosome band
[No Data.]
Annotation
| Genes | Mutation | Description | Source | Links |
|---|
| NCT ID | Status | Phase | Summary | Start date | Completion date |
|---|---|---|---|---|---|
| NCT01050855 | Active, not recruiting | Phase 2 | Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders | January 2008 | December 2026 |
| NCT03745287 | Active, not recruiting | Phase 2/Phase 3 | A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease | November 27, 2018 | October 2024 |
| NCT03249831 | Active, not recruiting | Phase 1 | A Blood Stem Cell Transplant for Sickle Cell Disease | January 4, 2019 | November 17, 2024 |
| NCT01850108 | Active, not recruiting | N/A | Non-Myeloablative Conditioning and Bone Marrow Transplantation | May 2013 | December 31, 2024 |
| NCT01966367 | Active, not recruiting | Phase 1/Phase 2 | CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation | March 2013 | February 2030 |
| NCT03655678 | Active, not recruiting | Phase 2/Phase 3 | A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia | September 14, 2018 | August 2024 |
| NCT00427661 | Completed | N/A | A Pilot Study of HSCT for Patients With High-risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen | June 2002 | May 2014 |
| NCT00673608 | Completed | Phase 4 | Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload | November 2007 | September 2011 |
| NCT00744692 | Completed | Phase 1 | Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders | October 2008 | April 2014 |
| NCT00000585 | Completed | Phase 3 | Penicillin Prophylaxis in Sickle Cell Disease (PROPS) | August 1983 | October 1994 |
| NCT00000586 | Completed | Phase 3 | Multicenter Study of Hydroxyurea in Patients With Sickle Cell Anemia (MSH) | January 1992 | June 1994 |
| NCT01316549 | Completed | Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation | January 1, 2011 | April 30, 2016 | |
| NCT00000588 | Completed | Phase 2 | Chelation Therapy of Iron Overload With Pyridoxal Isonicotinoyl Hydrazone | June 5, 1989 | March 31, 1995 |
| NCT04445402 | Completed | Pediatrics HOT COVID-19 Database in NY Tristate | April 30, 2020 | September 12, 2022 | |
| NCT03609814 | Completed | Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT) | January 26, 2016 | June 30, 2020 | |
| NCT03609840 | Completed | Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients | May 24, 2017 | July 31, 2023 | |
| NCT00000595 | Completed | Phase 2 | Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosis | January 1978 | September 1994 |
| NCT00000602 | Completed | Phase 2 | Pediatric Hydroxyurea in Sickle Cell Anemia (PED HUG) | April 1994 | March 1997 |
| NCT05389891 | Completed | N/A | Hemoglobinopathy Nursing Program and Pediatric Nursing Students | February 27, 2017 | May 11, 2017 |
| NCT00153985 | Completed | Phase 2 | Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies | March 2004 | July 2009 |
| NCT04029142 | Completed | Screening for Hemoglobinopathies in Pregnant Women | July 3, 2019 | September 30, 2019 | |
| NCT03149289 | Completed | Hepatitis C Virus Infection in Patients With Hemoglobinopathies | March 1, 2015 | February 1, 2017 | |
| NCT03609827 | Completed | Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients | September 1, 2015 | May 31, 2021 | |
| NCT02341586 | Completed | N/A | Lucky Iron Fish Home Fortification of Iron | April 2015 | May 2016 |
| NCT00000592 | Completed | Phase 3 | Stroke Prevention in Sickle Cell Anemia (STOP 1) | July 1994 | August 2000 |
| NCT04208529 | Enrolling by invitation | Phase 3 | A Long-term Follow-up Study in Participants Who Received CTX001 | January 20, 2021 | September 2039 |
| NCT02986698 | Enrolling by invitation | Phase 1 | In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM) | October 5, 2017 | February 2026 |
| NCT06363760 | Not yet recruiting | A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301 | June 4, 2024 | August 2040 | |
| NCT05356195 | Recruiting | Phase 3 | Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) | May 3, 2022 | May 2026 |
| NCT00920972 | Recruiting | Phase 1/Phase 2 | Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases | December 2001 | December 2031 |
| NCT01666080 | Recruiting | N/A | Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC) | August 2012 | June 2024 |
| NCT03128996 | Recruiting | Phase 1/Phase 2 | Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders | March 20, 2017 | April 2026 |
| NCT04671212 | Recruiting | Discarded Bone Marrow for Hematology Research | July 26, 2022 | January 2035 | |
| NCT04853576 | Recruiting | Phase 1/Phase 2 | A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) | May 4, 2021 | August 2025 |
| NCT05236764 | Recruiting | N/A | Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion | December 6, 2023 | August 1, 2030 |
| NCT05329649 | Recruiting | Phase 3 | Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) | May 2, 2022 | May 2026 |
| NCT05444894 | Recruiting | Phase 1/Phase 2 | EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT) | April 29, 2022 | December 2025 |
| NCT05477563 | Recruiting | Phase 3 | Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease | August 2, 2022 | February 2025 |
| NCT05799118 | Recruiting | Study of the Role of Genetic Modifiers in Hemoglobinopathies | October 1, 2022 | September 30, 2027 | |
| NCT06107400 | Recruiting | Early Phase 1 | Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease | October 8, 2023 | October 31, 2026 |
| NCT04644016 | Recruiting | Phase 2 | Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders | November 20, 2020 | December 20, 2025 |
| NCT03733249 | Terminated | Phase 1/Phase 2 | Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study | January 2017 | April 14, 2023 |
| NCT02065869 | Terminated | Phase 1/Phase 2 | Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant | April 2014 | September 7, 2021 |
| NCT00102245 | Terminated | Clinical and Laboratory Investigation of Humans With Informative Iron or Erythroid Phenotypes | January 18, 2005 | August 4, 2017 | |
| NCT00034528 | Terminated | Phase 2 | Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia | September 2001 | November 2003 |
| NCT02231710 | Terminated | Phase 1 | Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases | February 2015 | January 2018 |
| NCT04798157 | Unknown status | N/A | Transcranial Doppler in Children With Hemoglobinopathies | April 2021 | October 2021 |
| NCT00887081 | Unknown status | Phase 4 | Interferon and Ribavirin Treatment in Patients With Hemoglobinopathies | January 2009 | December 2013 |
| NCT03141398 | Withdrawn | Comparing CGM and OGTT in Relation to Iron Overload Detected by Pancreas T2* MRI in High-Risk Hematology Group | August 2017 | December 2018 |
- MeSH unique ID (MeSH (Medical Subject Headings))
- D006453