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Found 27 clinical trials
Phenylalanine and Its Impact on Cognition (PICO)

The PICO-Study is a randomized, placebo-controlled, crossover, non-inferiority trial conducted to add evidence to the current European treatment guidelines for adult patients with phenylketonuria.

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

phenylalanine
total protein
  • 0 views
  • 29 Jun, 2022
  • 2 locations
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
psychological assessment
sapropterin
kuvan
  • 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

phenylalanine
hyperphenylalaninemia
  • 0 views
  • 14 May, 2022
  • 3 locations
Evaluation of PKU Express Plus

: adherence to prescribed dietary intakes, palatability, usability, gastrointestinal tolerance, neophobia score, blood phenylalanine and tyrosine levels, timeframe to transition and contribution of PKU

tyrosine
phenylalanine
total protein
  • 0 views
  • 07 Oct, 2021
  • 3 locations
Leucine Requirement in Adult Men and Women

The purpose of the study is to determine the body's nutritional need for the essential amino acid, leucine, in adult men and women 18 to 39 years of age.

Accepts healthy volunteers
  • 0 views
  • 05 Jun, 2022
  • 1 location
mTOR as Mediator of Insulin Sensitivity Study

The study investigates the role of mTOR in mediating enhancement of muscle insulin sensitivity following a single bout of exercise. This will be investigated in young healthy male subjects by administering the pharmacological mTOR inhibitor Rapamycin in a crossover blinded experimental setup known to enhance muscle insulin sensitivity following one-legged …

Accepts healthy volunteers
  • 0 views
  • 01 Jul, 2022
  • 1 location
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 amino acid substitutes and dietary supplements free of phenylalanine.One of the major difficulties, which is the source of many treatment failures, …

  • 3 views
  • 11 Mar, 2022
  • 1 location
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
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 this study may be conducted either at the clinical site or by a home healthcare professional at an alternative location (e.g., patient's …

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