2 recruiting, 1 active not recruiting. 2 Phase 2, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Active not recruiting 1, Recruiting 1 |
| Phase 1 | 1 | Recruiting 1 |
Follow Hoyeraal Hreidarsson syndrome: new trials by email Literature Review of Hoyeraal Hreidarsson syndrome trials Research Report on Hoyeraal Hreidarsson syndrome
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
Bone Marrow Failure Disorders (3) · Dyskeratosis Congenita (3) · Revesz Debuse syndrome (2) · Anemia, Aplastic (1) · Fanconi Anemia (1) · Immune Deficiency Disease (1) · Primary Immunodeficiency Diseases (1) · Pulmonary Fibrosis (1)
Bone Marrow Diseases (3) · Congenital Abnormalities (3) · Congenital Bone Marrow Failure Syndromes (3) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Genetic Diseases, Inborn (3) · Genetic Diseases, X-Linked (3) · Hematologic Diseases (3) · Hemic and Lymphatic Diseases (3) · Skin Abnormalities (3) · Skin Diseases (3) · Skin Diseases, Genetic (3) · Skin and Connective Tissue Diseases (3) · Anemia (2) · Anemia, Aplastic (1) · Anemia, Hypoplastic, Congenital (1)
Alemtuzumab (2) · Mycophenolic Acid (2) · Tacrolimus (2) · fludarabine (2) · Cyclophosphamide (1) · Cyclosporine (1) · Cyclosporins (1) · Deoxycytidine (1) · Melphalan (1) · Thymidine (1) · Whole-Body Irradiation (1) · fludarabine phosphate (1)
Boston Children's Hospital (1) · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (1) · Suneet Agarwal (1)
United States (3) · Norway (1) · Sweden (1)
Heather J Symons, MD, MHS (1) · Helen Reed, MD, MPH (1) · Suneet Agarwal, MD, PHD (1)