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Phenylketonuria clinical trials

View clinical trials related to Phenylketonuria.

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NCT ID: NCT05099640 Completed - Phenylketonuria Clinical Trials

A Study of PTC923 in Participants With Phenylketonuria

Start date: September 30, 2021
Phase: Phase 3
Study type: Interventional

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 levels from baseline to Weeks 5 and 6 (that is, the average of each respective treatment dose 2-week period of double-blind treatment).

NCT ID: NCT04879277 Completed - Phenylketonuria Clinical Trials

Study of Low-grade Systemic Inflammation in Adult Patients With Phenylketonuria

INGRAPH
Start date: May 26, 2021
Phase: N/A
Study type: Interventional

Patient suffering from phenylketonuria have chronic hyperphenylalaninemia. Hyperphenylalaninemia is known to be toxic to central nervous system and cardiovascular system in particular through oxydative stress. In this context, research of low grade systemic inflammation through cytokine assay appears legitimate. The primary outcome of this study is to describe inflammation profile of patients with phenylketonuria.

NCT ID: NCT04534842 Completed - Phenylketonuria Clinical Trials

Efficacy and Safety of SYNB1618 and SYNB1934 in Adult Patients With Phenylketonuria

SynPheny-1
Start date: August 25, 2020
Phase: Phase 2
Study type: Interventional

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 home, hotel).

NCT ID: NCT04085666 Completed - Phenylketonuria Clinical Trials

Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of CDX-6114 in Patients With Phenylketonuria (PKU)

Start date: June 1, 2019
Phase: Phase 1
Study type: Interventional

This study is an international, multi-center, randomized, double-blind, placebo-controlled, two-treatment, two-period cross-over study to evaluate the pharmacodynamics, safety, tolerability and pharmacokinetics of a single oral dose of CDX-6114 in patients with phenylketonuria (PKU).

NCT ID: NCT03858101 Completed - Phenylketonuria Clinical Trials

SNAP: Study Nutrients in Adult PKU

SNAP
Start date: April 15, 2019
Phase:
Study type: Observational

Phenylketonuria (PKU) is a rare inherited metabolic disorder, where subjects are born with a genetic deficiency in the phenylalanine hydroxylase enzyme (PAH), which leaves them unable to convert Phenylalanine (Phe) into Tyrosine (Tyr). PKU patients have specific dietary needs and must follow a restrictive diet in the aim of preventing toxic levels of the amino acid phenylalanine (Phe) accumulation.

NCT ID: NCT03788343 Completed - Phenylketonuria Clinical Trials

Phenylalanine and Its Impact on Cognition

PICO
Start date: August 19, 2019
Phase: Phase 4
Study type: Interventional

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.

NCT ID: NCT03516487 Completed - Healthy Clinical Trials

Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria

PKU
Start date: April 17, 2018
Phase: Phase 1/Phase 2
Study type: Interventional

This Phase 1/2a, first-in-human, oral single and multiple dose-escalation, randomized, double-blinded, placebo-controlled study is evaluating SYNB1618 in healthy volunteers (HV) and subjects diagnosed with phenylketonuria (PKU), a rare inherited metabolic disorder that occurs in people who are missing an enzyme that the body needs to use phenylalanine (Phe). Eligible subjects receive investigational product (IP) in the clinic and undergo safety monitoring, evaluations, and subsequent follow-up after IP administration.

NCT ID: NCT03168399 Completed - Phenylketonuria Clinical Trials

Evaluation of PKU Explore

PKU Explore
Start date: June 8, 2017
Phase: N/A
Study type: Interventional

To evaluate the acceptability, tolerance and effect on metabolic control of PKU Explore, a renovated Phe free protein substitute for the dietary management of PKU in children from 6 months to 5 years.

NCT ID: NCT03097250 Completed - Phenylketonuria Clinical Trials

MRI Spectroscopy and Neuropsychological Functioning in Phenylketonuria

Start date: October 25, 2017
Phase:
Study type: Observational

This is a study about the relationship of brain biomarkers with neuropsychological functioning in PKU. All participants will undergo MRI spectroscopy, will provide a blood specimen and will receive neuropsychological testing.

NCT ID: NCT03058848 Completed - Phenylketonuria Clinical Trials

Evaluation of PKU Start

PKU Start
Start date: March 6, 2017
Phase: N/A
Study type: Interventional

To evaluate the acceptability, tolerance and effect on metabolic control of PKU Start, a new Phe free protein substitute for the dietary management of PKU in infants from birth.