Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Dyskeratosis Congenita
Open-data reference.
8 US clinical trials · 5 currently recruiting
Active & Recent Trials
Cancer in Inherited Bone Marrow Failure Syndromes
National Cancer Institute (NCI)
NCT00027274
Investigation of the Genetics of Hematologic Diseases
St. Jude Children's Research Hospital
NCT02720679
Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
Masonic Cancer Center, University of Minnesota
NCT03579875
Nucleoside Therapy in Patients With Telomere Biology Disorders
Suneet Agarwal
NCT06817590
Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
NCT04232085
Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita
Boston Children's Hospital
NCT01659606
Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Masonic Cancer Center, University of Minnesota
NCT02162420
Needs Assessment for Individuals and Families Affected by Dyskeratosis Congenita (DC) and Related Telomere Biology Disorders (TBD)
National Cancer Institute (NCI)
NCT04959188
Phase Distribution
| Phase | Trial count |
|---|---|
| Early Phase 1 | 1 |
| Phase 2 | 3 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Dyskeratosis Congenita Trial Landscape
ClinicalTrials.gov lists 8 US studies indexed under Dyskeratosis Congenita, and 5 of those are currently open to recruitment — roughly 63% of the total volume on the registry. That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.
The phase distribution for Dyskeratosis Congenita shows 0 late-stage studies (Phase 3 and Phase 4 combined) alongside 4 earlier-phase entries (Phase 1 through Phase 2). Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.
Top sponsor activity for Dyskeratosis Congenita is led by Masonic Cancer Center, University of Minnesota with 2 indexed trials, alongside 5 other organizations in the top contributor list. The list on this page surfaces up to 8 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation — eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.
Frequently Asked Questions
How many clinical trials are there for Dyskeratosis Congenita?
PlainTrial tracks 8 US clinical trials for Dyskeratosis Congenita, of which 5 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Dyskeratosis Congenita?
Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.
Is this data current?
Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.
Related
Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.
Read our methodology — how this data is sourced, computed, and verified.
Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2024 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.