9 recruiting, 7 completed, 4 terminated, 1 active not recruiting. 9 Phase 2, 5 Phase 1, 2 Not applicable, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 9 | Completed 3, Recruiting 3, Terminated 2, Active not recruiting 1 |
| Phase 1 | 5 | Recruiting 2, Terminated 2, Completed 1 |
| Not applicable | 2 | Completed 1, Terminated 1 |
| Phase 3 | 1 | Completed 1 |
Follow Dyskeratosis Congenita: new trials by email Literature Review of Dyskeratosis Congenita trials Research Report on Dyskeratosis Congenita
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Quercetin Dyskeratosis Congenita (DC)/Telomere Biology Disorders (TBD) Quercetin - Dyskeratosis Congenita, Telomere Disease | Phase 1 | Recruiting | Children's Hospital Medical Center, Cincinnati | 12 | 2026-05-29 | 2026-09-10 |
| Nucleoside Therapy in Patients With Telomere Biology Disorders deoxycytidine, deoxythymidine - Telomere Biology Disorders, Dyskeratosis Congenita | Phase 1 | Recruiting | Suneet Agarwal | 36 | 2025-09-29 | 2026-08-04 |
| Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data Inherited Bone Marrow Failure Syndrome, Fanconi Anemia | Recruiting | Istituto Giannina Gaslini | 250 | 2025-05-29 | 2026-10-16 | |
| Needs Assessment for Individuals and Families Affected by Dyskeratosis Congenita (DC) and Related Telomere Biology Disorders (TBD) Dyskeratosis Congenita, Telomere Biology Disorders | Completed | National Cancer Institute (NCI) | 53 | 2021-09-09 | 2024-12-11 | |
| The TELO-SCOPE Study: Attenuating Telomere Attrition With Danazol. Is There Scope to Dramatically Improve Health Outcomes for Adults and Children With Pulmonary Fibrosis Danazol, Placebo - Pulmonary Fibrosis, Telomere Shortening | Phase 2 | Terminated | The University of Queensland | 29 | 2021-09-07 | 2025-08-05 |
| Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures Alemtuzumab, Fludarabine, Melphalan - Primary Immune Deficiency Disorder, Immune Deficiency Disease | Phase 2 | Recruiting | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | 27 | 2020-02-12 | 2025-11-28 |
| Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1), Fludarabine (FLU) - Fanconi Anemia, Severe Aplastic Anemia | Phase 2 | Recruiting | Masonic Cancer Center, University of Minnesota | 48 | 2018-11-13 | 2026-01-30 |
| Familial Investigations of Childhood Cancer Predisposition Acute Leukemia, Adenomatous Polyposis | Recruiting | St. Jude Children's Research Hospital | 1500 | 2017-04-06 | 2026-06-17 | |
| Investigation of the Genetics of Hematologic Diseases Bone Marrow Failure Syndromes, Erythrocyte Disorder | Recruiting | St. Jude Children's Research Hospital | 1716 | 2016-06-17 | 2026-06-09 | |
| Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases BPX-501 and Rimiducid - Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis | Phase 1 | Terminated | Bellicum Pharmaceuticals | 1 | 2015-02-01 | 2024-03-25 |
| Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia Alemtuzumab, Fludarabine, Cyclophosphamide - Dyskeratosis Congenita, Aplastic Anemia | Not applicable | Completed | Masonic Cancer Center, University of Minnesota | 61 | 2015-01-10 | 2025-05-08 |
| Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT Hydroxyurea, Alemtuzumab, Fludarabine - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Phase 2 | Recruiting | Paul Szabolcs | 100 | 2014-02-04 | 2025-12-15 |
| Bone Marrow Transplant With Abatacept for Non-Malignant Diseases Abatacept - Hurler Syndrome, Fanconi Anemia | Phase 1 | Completed | Emory University | 10 | 2014-01-01 | 2019-12-26 |
| Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita alemtuzumab, Fludarabine, Cyclosporins - Dyskeratosis Congenita, Hoyeraal Hreidarsson Syndrome | Phase 2 | Active not recruiting | Boston Children's Hospital | 40 | 2012-07-01 | 2026-03-27 |
| Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation Alefacept - Thalassemia, Sickle Cell Disease | Not applicable | Terminated | Emory University | 3 | 2010-09-01 | 2017-07-27 |
| Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita danazol - Fanconi Anemia, Dyskeratosis Congenita | Phase 1 / Phase 2 | Terminated | Boston Children's Hospital | 5 | 2009-11-01 | 2019-02-19 |
| Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine - Non-Neoplastic Hematologic and Lymphocytic Disorder | Phase 2 | Completed | Fred Hutchinson Cancer Center | 98 | 2009-07-31 | 2021-08-13 |
| Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA Campath 1H, Cyclophosphamide, Fludarabine - Dyskeratosis Congenita, Aplastic Anemia | Phase 2 / Phase 3 | Completed | Masonic Cancer Center, University of Minnesota | 36 | 2007-08-01 | 2017-12-05 |
| Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment polymerase chain reaction, flow cytometry, immunologic technique - Dyskeratosis Congenita, Fanconi Anemia | Completed | University Hospital Freiburg | 119 | 2007-01-01 | 2015-01-16 | |
| Cancer in Inherited Bone Marrow Failure Syndromes Diamond Blackfan Anemia, Dyskeratosis Congenita | Recruiting | National Cancer Institute (NCI) | 4000 | 2001-11-28 | 2026-10-16 |
Showing 20 of 22. Sign in to see all of them.
Fanconi Anemia (13) · Anemia, Aplastic (9) · Anemia, Diamond-Blackfan (8) · Anemia, Sickle Cell (7) · Bone Marrow Failure Disorders (7) · Shwachman-Diamond Syndrome (7) · Congenital Bone Marrow Failure Syndromes (4) · Granulomatous Disease, Chronic (4) · Lymphohistiocytosis, Hemophagocytic (4) · Neutropenia, Severe Congenital, Autosomal Recessive 3 (4) · Primary Immunodeficiency Diseases (4) · Wiskott-Aldrich Syndrome (4) · Chediak-Higashi Syndrome (3) · Hoyeraal Hreidarsson syndrome (3)
Bone Marrow Diseases (22) · Congenital Abnormalities (22) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (22) · Genetic Diseases, Inborn (22) · Genetic Diseases, X-Linked (22) · Hemic and Lymphatic Diseases (22) · Skin Abnormalities (22) · Skin Diseases, Genetic (22) · Skin and Connective Tissue Diseases (22) · Hematologic Diseases (21) · Skin Diseases (21) · Anemia (18) · Congenital Bone Marrow Failure Syndromes (18) · Anemia, Hypoplastic, Congenital (15) · Bone Marrow Failure Disorders (15)
Alemtuzumab (6) · fludarabine (6) · Whole-Body Irradiation (5) · fludarabine phosphate (5) · Cyclophosphamide (4) · Antilymphocyte Serum (3) · Melphalan (3) · Mycophenolic Acid (3) · Tacrolimus (3) · thymoglobulin (3) · Cyclosporine (2) · Cyclosporins (2) · Danazol (2) · Methylprednisolone (2) · Stem Cell Transplantation (2)
Masonic Cancer Center, University of Minnesota (3) · Boston Children's Hospital (2) · Emory University (2) · National Cancer Institute (NCI) (2) · St. Jude Children's Research Hospital (2) · Bellicum Pharmaceuticals (1) · Children's Hospital Medical Center, Cincinnati (1) · Fred Hutchinson Cancer Center (1) · Istituto Giannina Gaslini (1) · National Center for Research Resources (NCRR) (1) · Paul Szabolcs (1) · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (1) · Suneet Agarwal (1) · The University of Queensland (1) · University Hospital Freiburg (1)
United States (17) · Italy (2) · Australia (1) · Austria (1) · Belgium (1) · Czechia (1) · Denmark (1) · Germany (1) · Ireland (1) · Netherlands (1) · Norway (1) · Spain (1) · Sweden (1) · Switzerland (1)
Colin A Sieff, MB.BCh (1) · David A. Williams (1) · Heather J Symons, MD, MHS (1) · Helen Reed, MD, MPH (1) · Jakub Tolar, M.D., Ph.D. (1) · John Horan, MD (1) · John T Horan, MD (1) · Jordan Milner, MD (1) · Kim E. Nichols, MD (1) · Lauri M. Burroughs (1) · Lisa J McReynolds, M.D. (1) · Marcin Wlodarski, MD, PhD (1) · Margaret MacMillan, MD, Msc, FRCPC (1)
PMID 20383216, PMID 24786393, PMID 25304614, PMID 25405500, PMID 25425683, PMID 25963299, PMID 26095669, PMID 28280134, PMID 31836590, PMID 32634238, PMID 32750127, PMID 32790018, PMID 33159275, PMID 33810313, PMID 34086408, PMID 34162668, PMID 34852175, PMID 34857525, PMID 35322185, PMID 35360980, PMID 37354000, PMID 39279436, PMID 39680438, PMID 40762130
P30CA015704 (1)