- Conditions
- Telomere Biology Disorders, Dyskeratosis Congenita, Revesz Syndrome, Hoyeraal Hreidarsson Syndrome, Telomere Biology Disorders With Bone Marrow Failure, Interstitial Lung Disease Due to Systemic Disease (Telomere Biology Disorder), Pulmonary Fibrosis, Familial (Telomere Biology Disorder)
- Interventions
- deoxycytidine, deoxythymidine
- Drug
- Lead sponsor
- Suneet Agarwal
- Other
- Eligibility
- 1 Year to 70 Years
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2029
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
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- Conditions
- Primary Immune Deficiency Disorder, Immune Deficiency Disease, Bone Marrow Failure, Short Telomere Length, Fanconi Anemia, Non Fanconi DNA-DSB Repair Disorder, Hoyeraal-Hreidarsson Syndrome, Dyskeratosis Congenita, Telomere Biology Disorder, Short Telomere Syndrome
- Interventions
- Alemtuzumab, Fludarabine, Melphalan, Low Dose Total Body Irradiation, Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil
- Drug · Radiation
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
- Other
- Eligibility
- 4 Months to 50 Years
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2028
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Dyskeratosis Congenita, Hoyeraal Hreidarsson Syndrome, Revesz Syndrome, Aplastic Anemia
- Interventions
- alemtuzumab, Fludarabine, Cyclosporins, Mycophenolate mofetil, Tacrolimus
- Biological · Drug
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 30 Days to 65 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2034
- U.S. locations
- 11
- States / cities
- Chicago, Illinois • Boston, Massachusetts • Kansas City, Missouri + 6 more