Phenylketonuria Clinical Trials

A listing of Phenylketonuria medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

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Found 23 clinical trials
Liver Cell Transplant for Phenylketonuria

Human phenylketonuria (PKU) results from phenylalanine hydroxylase (PAH) deficiency, and represents one of the most common and extensively studied single-gene Mendelian disorders in humans

sapropterin
deficiency
psychological assessment
phenylalanine
kuvan
  • 51 views
  • 22 Mar, 2022
  • 1 location
Gene Therapy Clinical Study in Adult PKU (pheNIX)

This is a Phase 1/2, open-label, randomized, concurrently-controlled, dose escalation study to evaluate the safety and efficacy of HMI-102 in adult PKU subjects with PAH deficiency. Participants

deficiency
pah gene
gene therapy
  • 178 views
  • 09 Feb, 2022
  • 12 locations
Safety and Efficacy of HMI-103, a Gene Editing Development Candidate in Adults With Classical PKU Due to PAH Deficiency

development candidate, in adult participants aged 18 to 55 years, inclusive, with classical PKU due to PAH deficiency who have uncontrolled disease despite Phe restricted dietary management.

deficiency
pah gene
  • 0 views
  • 04 Oct, 2022
  • 3 locations
A Phase 3 Study of PTC923 in Subjects With Phenylketonuria

The main purpose of this trial is to evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in participants with phenylketonuria as measured by mean change in blood Phe

hyperphenylalaninemia
phenylalanine
  • 5 views
  • 25 Oct, 2022
  • 3 locations
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe

total protein
phenylalanine
  • 0 views
  • 07 Oct, 2022
  • 2 locations
Short-term Effects of Good Metabolic Control on Cognitive Function, Wellbeing, and Metabolic Parameters in Adult Patients With Phenylketonuria

The goal of this study is to evaluate if in adult patients with phenylketonuria (PKU) without strict metabolic control during the last 12 months, strict metabolic control for 8 weeks results in

low phenylalanine diet
phenylalanine
  • 0 views
  • 27 Apr, 2022
  • 1 location
Can Care of Adult PKU Be Improved With Additional Dietary Large Neutral Amino Acids: An N-of-1 Study

This research investigates the effects of combining a phenylalanine restricted diet (usual care) with LNAA supplementation (adjuvant LNAA) in well-controlled adults with classical PKU. The

  • 0 views
  • 23 Oct, 2022
  • 1 location
An Exploratory Study to Evaluate the Acceptability of PKU Explore, a Food for Special Medical Purposes, for Use in the Dietary Management of Phenylketonuria in Infants From 6 Months to 3 Years of Age With Regard to Product-acceptability, Tolerance, Phe Levels and Growth

PKU explore France is an exploratory study to evaluate the acceptability of PKU explore, a food for special medical purposes, for use in the dietary management of phenylketonuria in infants from

total protein
amino acid protein
  • 0 views
  • 14 Oct, 2022
  • 1 location
The Effect of Nursing Empowerment Program Applied to Families of PKU Children, Coping Attitudes of Families and Phenylalanine Levels of Children

The study has shown that the strengthening program applied to the families of children diagnosed with phenylketonuria (PKU) and the education given; It will be conducted in order to examine the

Accepts healthy volunteers
  • 0 views
  • 15 Oct, 2022
  • 1 location
A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12-17) With Phenylketonuria Featuring an Open-Label Randomized Two-Arm (Active vs Diet-Only Control) Design (PEGASUS)

This is a Phase 3 open-label randomized controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.

sapropterin
sapropterin dihydrochloride
depression
total protein
pegvaliase
  • 0 views
  • 24 Oct, 2022
  • 8 locations