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 18 clinical trials
featured
  • 380 views
  • 08 Nov, 2020
  • 1 location
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

phenylalanine
deficiency
sapropterin
psychological assessment
kuvan
  • 34 views
  • 16 Apr, 2021
  • 1 location
Evaluation of PKU Sphere in Italy

20 participants with PKU will build-up their dietary intake of PKU sphere over 2-16 weeks, depending on their level of metabolic control whilst doing so. Participants will complete a

  • 0 views
  • 26 Jan, 2021
  • 4 locations
Gene Therapy Clinical Study in Adult PKU

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

pah gene
gene therapy
deficiency
  • 122 views
  • 01 Apr, 2021
  • 8 locations
AAV Gene Therapy Study for Subjects With PKU

This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single

total protein
gene therapy
deficiency
  • 30 views
  • 03 Apr, 2021
  • 4 locations
Efficacy and Safety of SYNB1618 in Adult Patients With Phenylketonuria

This Phase 2 study in patients with phenylketonuria (PKU) will be an open-label, single-arm study of a SYNB1618 dose-ramp regimen. All evaluations and assessments throughout this study may be

gonadotropin
azoospermia
hysterectomy
cervical cap
tubal ligation
  • 17 views
  • 21 May, 2021
  • 8 locations
The Effects of CGMP in Children and Adults With PKU

Randomised controlled trial with a crossover design. For early and continuously treated patients with phenylketonuria (PKU) that are adherent. Two 12-week periods where patients consume either

amino acid protein
phenylalanine
omega-3
glycomacropeptide
total protein
  • 7 views
  • 26 Jan, 2021
  • 3 locations
Glytactin EfficiEncy in Non or Insufficiently Treated Adult PHENylketonuria Patients

Phenylketonuria is the most common inherited metabolic disease in France and is screened for neonatal exposure. Management consists of a strict and restrictive hypoproteic diet and the intake of

  • 3 views
  • 01 Aug, 2021
  • 14 locations
Phenylalanine and Its Impact on Cognition

phenylketonuria.

phenylalanine
neonatal screening
  • 8 views
  • 20 Jul, 2021
  • 1 location
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

phenylalanine
low phenylalanine diet
  • 0 views
  • 24 Feb, 2021
  • 1 location