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Sepiapterin

Clinical trials are investigating Sepiapterin in children with phenylketonuria (PKU). These studies aim to evaluate long-term benefit, especially on brain and thinking skills, as well as overall treatment effect. The main trial listed here is a Phase 3 study in children and young people with PKU.

Table of Contents

Trial overview

The available trial is a study of Sepiapterin in participants with phenylketonuria (PKU), a genetic condition that affects how the body processes phenylalanine.[1] The study is called EPIPHENY and is designed to evaluate long-term efficacy, with a focus on preserving neurocognitive function when treatment begins in early childhood.[1]

Who is being studied

This trial is for children and young people with PKU.[1] The age range in the main outcome measure shows two groups: participants from 30 months to under 6 years of age, and participants from 6 years to 16 years of age.[1] This means the study is focused on early treatment in childhood and follows children as they grow.

Treatment and study design

The study is an interventional trial, which means researchers assign a treatment and then measure the results.[1] It is a Phase 3 study, so it is a later-stage trial meant to test how well the treatment works in a larger group of participants.[1]

The interventions listed are Sepiapterin given as Sephience oral powder in sachets, with two strengths named in the trial record: 250 mg and 1,000 mg.[1] The trial record also states a dose of 60 mg/kg by mouth for both listed forms.[1]

What is being measured

The main outcome is the mean change in full-scale intelligence quotient (FSIQ) over 2 years.[1] FSIQ is a score that reflects overall thinking ability, including learning and problem-solving skills.

The study uses age-appropriate tests to measure this outcome: the WPPSI-IV for participants aged 30 months to under 6 years, and the WISC-V for participants aged 6 years to 16 years.[1] These tests help researchers compare thinking skills over time in children of different ages.

The brief study summary says the goal is to evaluate long-term efficacy of Sepiapterin on preserving neurocognitive functioning in children with PKU when treatment starts early.[1] Neurocognitive functioning means brain-related skills such as attention, memory, learning, and reasoning.

Study status and size

The trial status is listed as Authorised, which means it has been approved to proceed.[1] The planned enrollment is 115 participants.[1] This size suggests a focused but meaningful study in a specific PKU population.

Trial ID Phase Condition studied Status Enrollment
2024-514435-20-00 Phase 3 Phenylketonuria (PKU) Authorised 115

FAQ

  • What is being studied in Sepiapterin clinical trials? The trial is studying Sepiapterin in children with phenylketonuria (PKU). It is looking at whether early treatment can help preserve thinking and learning skills over time.
  • Who can take part in the main trial? The main trial is for participants with PKU who are children and young people. The outcome measure shows age groups from 30 months to 16 years.
  • What phase is the Sepiapterin trial? The listed study is a Phase 3 trial. Phase 3 trials usually test how well a treatment works in a larger group and continue to gather safety information.
  • What result is the trial measuring? The main result is change in full-scale intelligence quotient, or FSIQ. This is a score that helps measure overall thinking ability.
  • What is the goal of the study? The study aims to see if Sepiapterin can help protect neurocognitive function. Neurocognitive function means brain-related skills such as thinking, learning, and memory.

Ongoing Clinical Trials on Sepiapterin

  • Study of Long-Term Neurocognitive Outcomes in Children with Phenylketonuria Treated with Sepiapterin

    Recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    France Ireland Poland Sweden

Glossary

  • Phenylketonuria (PKU): A genetic condition in which the body cannot properly process phenylalanine, an amino acid found in many foods.
  • Phase 3: A late-stage clinical trial that checks how well a treatment works in a larger group of people and continues safety monitoring.
  • Interventional study: A study where researchers give a treatment and then measure its effects.
  • Enrollment: The number of people planned to join a study.
  • Full-scale intelligence quotient (FSIQ): A score that estimates overall thinking and problem-solving ability.
  • Neurocognitive function: Brain-based skills such as attention, learning, memory, and thinking.
  • WPPSI-IV: A test used to measure thinking skills in younger children.
  • WISC-V: A test used to measure thinking skills in school-age children and teens.
  • Primary outcome: The main result the researchers plan to measure to see if the study treatment helps.
  • Authorised: The study has been approved to move forward.

References