The Struggle That Is Phenylketonuria : What Do The Patients and Caregivers Suffer From

February 19, 2024 updated by: Dilara Demirel, Hacettepe University
Objective: To assess the stress levels and life hardships of phenylketonuria patients and their parents.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Study Design: Between January 2020 - June 2020, a total of 156 phenylketonuria patients and their parents who arrived for their regular examinations were included. Parents were asked to fill the parenting stress index, Zarit burden scale and the strengths and difficulties questionnaire and children over the age of eleven were asked to fill the Rosenberg self-esteem scale, the stait-trait anxiety inventory and strengths and difficulties questionnaire.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
156

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Turkey
    • Sihhiye
      • Ankara, Sihhiye, Turkey, 06100
        • Hacettepe University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

N/A

Sampling Method

Probability Sample

Study Population

This study was carried out at Hacettepe University, İhsan Doğramacı Children's Hospital, Department of Metabolism, between January 2020 and June 2020. It was planned to include patients who were followed up with the diagnosis of phenylketonuria with diet or Sapropterin hydrochloride therapy, who didn't have BH4 metabolism disorder or any other health problem. Pregnant patients were also excluded

Description

Inclusion Criteria:

  • Phenylketonuria patients

Exclusion Criteria:

  • BH4 metabolism disorde
  • Other chronic illness
  • Pregnancy

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Rosenberg Self-Esteem Scale
Time Frame: 12 minutes
Rosenberg Self-Esteem Scale was developed by Morris Rosenberg in 1960. It evaluates self-esteem and consists of 10 items in total. In the Likert-type scale, which includes positively and negatively charged items, 0-1 points are considered as high self-esteem, 2-4 points as medium self-esteem, and 5-6 points as low self-esteem.
12 minutes
Stait trait anxiety inventory
Time Frame: 45 minutes
State-Trait Anxiety Inventory is an inventory that measures state and trait anxiety levels of children. It has been developed by Spielberges et. al in 1983. It consists of two questionnaires with a total of 40 questions, 20 that assesses the anxiety level stately, that is, how the child feels at that moment, and 20 that assesses how he feels constantly, that is, in general. The scale is in 3-point Likert type and is evaluated with 1-3 points according to symptom severityThe lowest score that can be obtained from the scales is 20, and the highest score is 60, higher scores indicate higher anxiety.
45 minutes
Strenghts and difficulties questionnaire
Time Frame: 30 minutes
Strengths and Difficulties Questionnaire was developed by Goodman in 1997 to assess children's social, emotional and behavioral difficulties. There is a parent form for ages 4-17 and a child form for ages 11-17. It consists of 25 questions in total
30 minutes
Zarit burden scale
Time Frame: 25 minutes
Zarit Burden Scale was developed by Zarit et al. in 1980. It is a 5-point Likert-type scale consisting of 22 questions that aims to measure the distress experienced by caregivers. Answers are scored between 0-4. A minimum of 0 and a maximum of 88 points can be obtained from the scale. The high score indicates the height of the distress experienced.
25 minutes
Parenting stress index
Time Frame: 20 minutes
Parenting Stress Index aims to measure the stress experienced by parents in their relationships with their children. It is a 4-point Likert-type scale consisting of 16 questions in total. A minimum of 16 and a maximum of 64 points can be obtained from the scale. A high score from the scale indicates that parental stress is high.
20 minutes

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Hacettepe University

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Serap Sivri, Prof, Hacettepe University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 27, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 1, 2020

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 1, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 10, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 19, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 28, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 28, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 19, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • GO20/296

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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