Dyskeratosis Congenita
15
6
7
5
Key Insights
Highlights
Success Rate
71% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 59/100
13.3%
2 terminated out of 15 trials
71.4%
-15.1% vs benchmark
0%
0 trials in Phase 3/4
60%
3 of 5 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 5 completed trials
Clinical Trials (15)
Familial Investigations of Childhood Cancer Predisposition
Cancer in Inherited Bone Marrow Failure Syndromes
Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita
Nucleoside Therapy in Patients With Telomere Biology Disorders
Expanded Access to CD34+ Selection Utilizing Miltenyi CliniMACS Prodigy® for Patients Receiving Peripheral Blood Stem Cell Transplantations and Stem Cell Boosts
Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures
Investigation of the Genetics of Hematologic Diseases
The TELO-SCOPE Study: Attenuating Telomere Attrition With Danazol. Is There Scope to Dramatically Improve Health Outcomes for Adults and Children With Pulmonary Fibrosis
Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Needs Assessment for Individuals and Families Affected by Dyskeratosis Congenita (DC) and Related Telomere Biology Disorders (TBD)
Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita
Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment
Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes