Clinical Trials for Genetic Diseases, Inborn

Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria

NCT ID: NCT06337864

This clinical trial is exploring a new treatment option for adults with Classical Phenylketonuria (PKU), a condition that affects how the body processes certain proteins. The study is looking at th...

A Study to Learn About the Safety and Efficacy of the Drug Oleogel-S10 in Japanese Patients With Epidermolysis Bullosa

NCT ID: NCT06917690

This clinical trial is exploring the safety and effectiveness of a new gel called Oleogel-S10 for treating skin wounds in people with a rare condition called epidermolysis bullosa (EB). Specificall...

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

NCT ID: NCT05477563

This clinical trial is studying a new treatment called CTX001 for people with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The treatment involves modifying a patie...

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

NCT ID: NCT05356195

This clinical trial is studying a new treatment called CTX001 for children with a serious blood disorder known as transfusion-dependent beta-thalassemia (TDT). TDT is a condition where the body doe...

A Long-term Follow-up Study in Participants Who Received CTX001

NCT ID: NCT04208529