DYSKERATOSIS CONGENITA
Clinical trials for DYSKERATOSIS CONGENITA explained in plain language.
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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…
Matched conditions: DYSKERATOSIS CONGENITA
Phase: PHASE1 • Sponsor: Suneet Agarwal • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC
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New stem cell filter could make transplants safer for vulnerable patients
Disease control AVAILABLEThis 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…
Matched conditions: DYSKERATOSIS CONGENITA
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…
Matched conditions: DYSKERATOSIS CONGENITA
Phase: PHASE2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Jun 27, 2026 09:00 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…
Matched conditions: DYSKERATOSIS CONGENITA
Phase: PHASE2 • 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…
Matched conditions: DYSKERATOSIS CONGENITA
Phase: PHASE1 • Sponsor: Children's Hospital Medical Center, Cincinnati • Aim: Symptom relief
Last updated Jun 27, 2026 13:03 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…
Matched conditions: DYSKERATOSIS CONGENITA
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 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…
Matched conditions: DYSKERATOSIS CONGENITA
Sponsor: National Cancer Institute (NCI) • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:06 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…
Matched conditions: DYSKERATOSIS CONGENITA
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:04 UTC