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Found 25 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

kuvan
sapropterin
deficiency
psychological assessment
phenylalanine
  • 40 views
  • 22 Mar, 2022
  • 1 location
A Study of PTC923 in Participants 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
  • 0 views
  • 14 May, 2022
  • 3 locations
Glytactin EfficiEncy in Non or Insufficiently Treated Adult PHENylketonuria Patients (GLEEPHEN)

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
  • 11 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
  • 176 views
  • 09 Feb, 2022
  • 12 locations
Phenylalanine and Its Impact on Cognition (PICO)

phenylketonuria.

phenylalanine
Accepts healthy volunteers
neonatal screening
  • 8 views
  • 12 Feb, 2022
  • 1 location
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

polyunsaturated fatty acids
total protein
glycomacropeptide
casein
omega-3
  • 14 views
  • 22 Sep, 2021
  • 3 locations
Evaluation of PKU Sphere Liquid

PKU Sphere Liquid is a prospective, open-label, acceptability study to evaluate PKU sphere liquid in up to 15 participants aged 3 and above for the dietary management of PKU over 31 days.

total protein
  • 0 views
  • 16 Dec, 2021
  • 1 location
Evaluation of PKU Express Plus

A prospective, open label, acceptability study to evaluate PKU express plus in the dietary management of 15 patients aged 3 years and above with PKU. The study sees patients transition from their current protein substitute to at least one sachet of PKU express plus and then enter a 28 day …

phenylalanine
total protein
tyrosine
  • 0 views
  • 07 Oct, 2021
  • 3 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

phenylalanine
low phenylalanine diet
  • 0 views
  • 27 Apr, 2022
  • 1 location
Efficacy and Safety of SYNB1618 and SYNB1934 in Adult Patients With Phenylketonuria (SynPheny-1)

This Phase 2 study in patients with phenylketonuria (PKU) will be an open-label, dual-arm study of either a SYNB1618 or SYNB1934 dose-ramp regimen. All evaluations and assessments throughout

gonadotropin
cervical cap
hysterectomy
tubal ligation
azoospermia
  • 31 views
  • 25 May, 2022
  • 11 locations