Clinical Trials for Phenylketonuria

A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

NCT ID: NCT06302348

This clinical trial is studying a medication called sepiapterin to see how well it helps children with Phenylketonuria (PKU) maintain their brain function over the long term. PKU is a genetic condi...

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria

NCT ID: NCT05972629

This clinical trial is studying a new treatment called SAR444836 for adults with a condition known as phenylketonuria (PKU). PKU is a genetic disorder that affects the body’s ability to process a s...

A Study to Evaluate the Safety and Efficacy of JNT-517 in Participants With Phenylketonuria (PKU)

NCT ID: NCT06971731

This clinical trial is studying a new oral medication called JNT-517 to see how safe and effective it is for adults with Phenylketonuria (PKU), a genetic condition that affects how the body process...

A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

NCT ID: NCT05166161

This clinical trial is looking at the long-term safety of a medication called PTC923 for people with a condition known as phenylketonuria (PKU). PKU is a genetic disorder that affects how the body ...

GMP Powdered Substitutes in PKU and TYR

NCT ID: NCT06941532

This clinical trial is studying four new powdered protein substitutes designed to help people with phenylketonuria (PKU) or tyrosinemia (TYR) manage their diets. These substitutes are made from a s...

Educational, Social Support, and Nutritional Interventions and Their Cumulative Effect on Pregnancy Outcomes and Quality of Life in Teen and Adult Women With Phenylketonuria

NCT ID: NCT01659749

This clinical trial is exploring how helpful it can be for young women and teens with a condition called Phenylketonuria (PKU) to learn about their health and nutrition in a supportive camp setting...

Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq

NCT ID: NCT06780332

This clinical trial is studying a method called rapid drug desensitization (RDD) to help adults with a condition called phenylketonuria (PKU) who have experienced allergic reactions to a medication...

Announcement of Rare Metabolic Diseases in Systematic Newborn Screening: the Phenylketonuria Experience.

NCT ID: NCT06289348

This clinical trial is focused on studying the emotional and psychological effects of informing parents about a rare condition called Phenylketonuria (PKU), which is detected at birth through routi...

Evaluation of Capillary Blood Collection Devices (Mitra® and HemaPEN®)

NCT ID: NCT05894122

This clinical trial is studying two blood collection devices called Mitra® and HemaPEN®, which are typically used in other areas of medicine but are being tested for children with a condition calle...

Efficacy and Safety of Orally Administered Engineered Probiotics (CBT102-A) for the Treatment of Children With Phenylketonuria

NCT ID: NCT05948020

This clinical trial is studying a new treatment for children with Phenylketonuria (PKU), a condition where the body cannot properly break down a substance called phenylalanine found in many foods. ...

Long-Term Follow Up Study of Subjects Previously Administered HMI 102

NCT ID: NCT04348708