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Dyskeratosis congenita
MONDO:0015780Dyskeratosis congenita (DC) is a rare ectodermal dysplasia that often presents with the classic triad of nail dysplasia, skin pigmentary changes, and oral leukoplakia associated with a high risk of bone marrow failure (BMF) and cancer.
Also known as: DC, DKC, Zinsser-Engman-Cole syndrome, dyskeratosis congenita, Hoyeraal-Hreidarsson syndrome, Zinsser Cole Engman syndrome
43 clinical trials for this condition and its sub-types, 12 tagged with Dyskeratosis congenita itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Dyskeratosis congenita
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DKC1-related disorder 0 trials · 3 incl. sub-types
1 sub-type
- Dyskeratosis congenita, X-linked 0 trials · 3 incl. sub-types Sub-types →
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Revesz syndrome 2 trials
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2 sub-types
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Dyskeratosis congenita, digenic 0 trials
Most studied deeper sub-types
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Could a simple sugar pill fix faulty telomeres? early trial launches
Disease control Recruiting nowThis early-stage trial is testing whether a combination of two natural substances, deoxycytidine and deoxythymidine, is safe for people with telomere biology disorders. These rare genetic conditions cause premature aging, bone marrow failure, and lung scarring. Up to 36 participa…
Phase 1 • Sponsor: Suneet Agarwal • Aim: Disease control
Last updated Aug 06, 2026 00:00 UTC
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New stem cell filter could make transplants safer for vulnerable patients
Disease control Expanded accessThis expanded access program offers a special stem cell purification technique (CD34+ selection) for patients undergoing stem cell transplants. The method removes many T-cells from the donated stem cells, which lowers the risk of a serious complication called graft-versus-host di…
Sponsor: University of Florida • Aim: Disease control
Last updated Jun 27, 2026 09:06 UTC
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New stem cell transplant could reduce immune suppression in bone marrow failure patients
Disease control Recruiting nowThis phase 2 trial tests a special stem cell transplant for people with inherited bone marrow failure disorders like Fanconi anemia. The transplant uses a technique to remove certain immune cells, aiming to reduce the need for long-term immune-suppressing drugs and lower infectio…
Phase 2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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Milder transplant method aims to help young patients with rare blood diseases
Disease control Recruiting nowThis study is testing a less intense chemotherapy and radiation regimen before a stem cell transplant for children and young adults up to age 55 with non-cancerous blood disorders like immune deficiencies, anemias, and metabolic diseases. The goal is to see if this gentler prepar…
Phase 2 • Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Jun 27, 2026 08:04 UTC
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New transplant approach aims to fix broken immune systems
Disease control Recruiting nowThis phase 2 trial is testing a stem cell transplant using a milder chemotherapy regimen to treat people with severe immune deficiencies and inherited bone marrow failure. The goal is to see if donor cells can safely take over and rebuild a healthy immune system. Up to 27 partici…
Phase 2 • Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins • Aim: Disease control
Last updated Jun 27, 2026 08:00 UTC
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Can a common plant compound help rare telomere disease?
Symptom relief Recruiting nowThis pilot study tests whether quercetin, a natural antioxidant found in many plants, is safe for people with Dyskeratosis congenita or telomere biology disorders. Twelve participants will take quercetin for 24 weeks while researchers monitor side effects and how well people stic…
Phase 1 • Sponsor: Children's Hospital Medical Center, Cincinnati • Aim: Symptom relief
Last updated Sep 12, 2026 00:00 UTC
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Massive study aims to uncover cancer secrets in rare bone marrow diseases
Knowledge-focused Recruiting nowThis natural history study follows up to 4,000 people with inherited bone marrow failure syndromes (IBMFS) and their families to learn why they are prone to certain cancers. Researchers will track health over time, collect genetic samples, and look for clues that separate those w…
Sponsor: National Cancer Institute (NCI) • Aim: Knowledge-focused
Last updated Sep 18, 2026 00:00 UTC
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Hunt for hidden cancer genes: families needed to unlock hereditary secrets
Knowledge-focused Recruiting nowThis study aims to discover new genes that may cause certain cancers to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had cancer, especially childhood cancers. The goal is to build a regist…
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC
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Massive gene hunt aims to unlock secrets of blood disorders
Knowledge-focused Recruiting nowThis study collects blood, bone marrow, and other samples along with health information from up to 1,716 people with non-cancerous blood diseases and their family members. Researchers will analyze the participants' genes to find new genetic causes of these conditions and understa…
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:04 UTC