PhenylketonuriasNCT05497050
The Effect of Nursing Empowerment Program
A study testing Family education in people with Phenylketonurias, run by Cukurova University at 1 site in Turkey (Türkiye).
Trial summary
The study has shown that the strengthening program applied to the families of children diagnosed with phenylketonuria (PKU) and the education given; It will be conducted in order to examine the effect of the family's coping attitudes, knowledge scores about the disease and its management. Read more about this study
- Who is it for?
- Ages 1 day to 3 yearsHealthy volunteers can take part
- Conditions the study is for
- Phenylketonurias
- What is being tested?
- Family education
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 24 Nov 2021to 22 Jun 2023
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 day to 3 years
- All sexes
- Healthy volunteers accepted
- Child
Who may be able to join
- Residing in Van and/or being followed up in the pediatric metabolism polyclinic,
- Agreeing to participate in the research,
- literate,
- Speaking and understanding Turkish,
- 18 years old and over
- Fully oriented and cooperative and open to communication,
Show all 9Show fewer
- a parent who is the primary caregiver of the child,
- The child is in the 0-3 age range (≤3 years old),
- Families of children with definitive diagnosis of PKU will be included.
Who cannot join
- Not residing in Van and/or not being followed up in the pediatric metabolism polyclinic,
- Those who do not accept to participate in the research, who are illiterate,
- Does not speak or understand Turkish,
- Parent/family member who is aged 17 or younger, who is not fully oriented and cooperative and not open to communication, and does not provide primary care for the child,
- The child is over 3 years old (3 years <),
- Dropping out of the training to be applied,
- Families of children with PKU who are not definitively diagnosed will not be included in the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: The Effect of Nursing Empowerment Program Applied to Families of PKU Children, Coping Attitudes of Families and Phenylalanine Levels of Children
The study has shown that the strengthening program applied to the families of children diagnosed with phenylketonuria (PKU) and the education given; It will be conducted in order to examine the effect of the family's coping attitudes, knowledge scores about the disease and its management. The research is of the randomized controlled experimental type. Children from PKU constitute the universe in the city center of Van. The families of children between the ages of 0-3 who were diagnosed with PKU will form the sample of the study. A nursing strengthening program will be applied to families in the experimental group for 10 weeks. The scope of this program; It consists of face-to-face training on disease management, giving the educational guide (booklet) for the families of babies diagnosed with PKU, telephone calls during the education process and counseling services. No application will be made to the families in the control group. After the application of the last tests to the control group; Face-to-face training and a booklet will be given to the experimental group. Data; It will be collected with 'Child and Family Introductory Information Form', 'Parents' Information Level Assessment Form about Phenylketonuria' and 'Coping Attitudes Assessment Scale'.
In the literature, there is no study showing the effect of the education given to the families of babies diagnosed with PKU on their coping attitudes. Based on the need to fill this knowledge gap in the literature, it is thought that the study will contribute to the field of child health and disease nursing.
Read the detailed description
Phenylketonuria is a disorder of phenylalanine metabolism and causes intellectual destruction if left untreated. It is an autosomal recessive metabolic disease that develops as a result of the absence of the enzyme phenylalanine hydroxylase (FAH), which provides the necessary amino acid metabolism for protein production in the body, which causes high phenylalanine in the blood. In our country, it is one of the places where PKU is commonly encountered (Çavuşoğlu, 2015; Hafid et al, 2015; Törner and Büyükgönenç, 2013). While the incidence of this disease in European countries is 1/10000-30000, it is 1/3000-4500 in our country (Alparslan, 2021). This situation is directly related to consanguineous marriages. It is stated that one out of every five marriages in our country is consanguineous marriage. If left untreated, it can cause extreme mental retardation, convulsions and death (Alparslan, 2021; Çavuşoğlu, 2015; Hafid et al, 2015; Törner and Büyükgönenç, 2013).
The care of the child with phenylketonuria is carried out by the parents. Extra care (diet therapy, control of blood phenylalanine level, etc.) for children in this diagnosis group is provided by their parents. The treatment and care process of children with PKU affects family routines, relationships and parenting styles. Parents of children experience emotional and economic burden. Therefore, it is stated that the care burden of parents in this diagnosis group is higher than that of healthy parents, and their quality of life is lower (Hatzmann et al, 2009). In addition, studies have shown that families of children with PKU have high levels of anxiety, depression (Mahmoudi-Gharaei et al. 2011) and stress (Irannejad et al. 2018). In another study; It is reported that the family's routines have changed (not eating in front of the child, not eating in restaurants, etc.) and that they experience anxiety about the effect of diet incompatibility on blood phenylalanine levels, feeling overwhelmed and guilt in the care process (Carpenter et al 2018). Therefore, having a child with PKU may affect coping attitudes.
Child health and diseases nurse should support children with PKU and their families on issues such as informing, training and counseling about the disease and its management.
- Conditions
- Phenylketonurias
- Being tested
- Family education
- Keywords
- family, Coping, Education, Phenylketonuria(PKU), Empowerment, Nursing.
- Medical subject headings
- Phenylketonurias, Empowerment
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Family educationBehavioural
By calling the families in the experimental group by phone; Nursing interventions for family empowerment will last for 10 weeks, they will be invited to the Family Health Center (FHC) or child metabolism polyclinic twice in this process, they will be called once a week (with 2 phone calls) for two weeks after the face-to-face training initiative in the first stage, In the second stage, at the end of the face-to-face training, they will be informed that they will be called once a week for two weeks (4 phone calls in total). The families in the experimental group will be informed that 4 weeks after the completion of the training and phone calls (at the 10th week), they will call the FHC or the pediatric metabolism polyclinic again for the application of the post-tests and that the nursing training for family empowerment will be completed.
| Study group | Role | What they receive |
|---|---|---|
| Experimental group | Experimental | Family education Receives: Behavioral: Family education |
| Control group | No intervention | No training will be given. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from Baseline in Evaluation Of Coping Attitudes (COPE) at week 10
*COPE is a validated. The scale is a 4-point Likert type scale. While scoring the items, 1 point is given for "I Never Do This", 2 points for "I rarely do this", 3 points for "I do this way", and 4 points for "I Do This Mostly". There is no negative item in the scale. The highest score that can be obtained from the scale is 128, and the lowest score is 32. A high score from the scale means a high level of coping attitude, and a low score means a low level of coping attitude.
change = (Week 10 Score - Baseline Score)
Measured: 10 week
Secondary outcomes (1)
Change from Baseline in Evaluation of the level of knowledge about PKU at week 1
Form for Evaluation of Families' Knowledge Levels on Phenylketonuria:
*Question form prepared by the researcher in line with the literature; The level of knowledge of families about the disease and disease management of their child with PKU will be measured. The form consists of 20 questions.The increase in the mean of correct answers obtained in the form indicates that the level of knowledge about the disease is good.
change = (Week 10 Score - Baseline Score)
Measured: 10 week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Aug 2022
- Study start24 Nov 2021
- Primary completion28 Feb 2023
- Study completion22 Jun 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for PhenylketonuriasStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- The study was completed with the families of 36 children with PKU, 19 in the intervention group and 17 in the control group, who met the inclusion criteria.
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cukurova University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Fahri AŞKAN, Cukurova University
- Investigators
- Şenay ÇETİNKAYA, Doç. Dr., Study director, Cukurova University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05497050
- Sponsor's protocol ID
- FAHRİ AŞKAN PHD THESIS
- Status last verified
- Oct 2023
- First submitted
- 8 Aug 2022
- Last updated
- 26 Oct 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
MacDonald A, Depondt E, Evans S, Daly A, Hendriksz C, Chakrapani A A, Saudubray JM. Breast feeding in IMD. J Inherit Metab Dis. 2006 Apr-Jun;29(2-3):299-303. doi: 10.1007/s10545-006-0332-x.
PubMed 16763891Pinto A, Adams S, Ahring K, Allen H, Almeida MF, Garcia-Arenas D, Arslan N, Assoun M, Atik Altinok Y, Barrio-Carreras D, Belanger Quintana A, Bernabei SM, Bontemps C, Boyle F, Bruni G, Bueno-Delgado M, Caine G, Carvalho R, Chrobot A, Chyz K, Cochrane B, Correia C, Corthouts K, Daly A, De Leo S, Desloovere A, De Meyer A, De Theux A, Didycz B, Dijsselhof ME, Dokoupil K, Drabik J, Dunlop C, Eberle-Pelloth W, Eftring K, Ekengren J, Errekalde I, Evans S, Foucart A, Fokkema L, Francois L, French M, Forssell E, Gingell C, Goncalves C, Gokmen Ozel H, Grimsley A, Gugelmo G, Gyure E, Heller C, Hensler R, Jardim I, Joost C, Jorg-Streller M, Jouault C, Jung A, Kanthe M, Koc N, Kok IL, Kozanoglu T, Kumru B, Lang F, Lang K, Liegeois I, Liguori A, Lilje R, Lubina O, Manta-Vogli P, Mayr D, Meneses C, Newby C, Meyer U, Mexia S, Nicol C, Och U, Olivas SM, Pedron-Giner C, Pereira R, Plutowska-Hoffmann K, Purves J, Re Dionigi A, Reinson K, Robert M, Robertson L, Rocha JC, Rohde C, Rosenbaum-Fabian S, Rossi A, Ruiz M, Saligova J, Gutierrez-Sanchez A, Schlune A, Schulpis K, Serrano-Nieto J, Skarpalezou A, Skeath R, Slabbert A, Straczek K, Gizewska M, Terry A, Thom R, Tooke A, Tuokkola J, van Dam E, van den Hurk TAM, van der Ploeg EMC, Vande Kerckhove K, Van Driessche M, van Wegberg AMJ, van Wyk K, Vasconcelos C, Velez Garcia V, Wildgoose J, Winkler T, Zolkowska J, Zuvadelli J, MacDonald A. Weaning practices in phenylketonuria vary between health professionals in Europe. Mol Genet Metab Rep. 2018 Nov 25;18:39-44. doi: 10.1016/j.ymgmr.2018.11.003. eCollection 2019 Mar.
PubMed 30705824van Spronsen FJ, Blau N, Harding C, Burlina A, Longo N, Bosch AM. Phenylketonuria. Nat Rev Dis Primers. 2021 May 20;7(1):36. doi: 10.1038/s41572-021-00267-0.
PubMed 34017006
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of Nursing Empowerment Program Applied to Families of PKU Children, Coping Attitudes of Families and Phenylalanine Levels of Children
- ClinicalTrials.gov ID
- NCT05497050
- Acronym / sponsor ID
- FAHRİ AŞKAN PHD THESIS
- Conditions
- Phenylketonurias
- Keywords
- family, Coping, Education, Phenylketonuria(PKU), Empowerment, Nursing.
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Cukurova UniversityUniversity, hospital or other
- Study start
- 24 Nov 2021 (actual)
- Primary completion
- 28 Feb 2023 (actual)
- Study completion
- 22 Jun 2023 (actual)
- First posted
- 11 Aug 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study has shown that the strengthening program applied to the families of children diagnosed with phenylketonuria (PKU) and the education given; It will be conducted in order to examine the effect of the family's coping attitudes, knowledge scores about the disease and its management. The research is of the randomized controlled experimental type. Children from PKU constitute the universe in the city center of Van. The families of children between the ages of 0-3 who were diagnosed with PKU will form the sample of the study. A nursing strengthening program will be applied to families in the experimental group for 10 weeks. The scope of this program; It consists of face-to-face training on disease management, giving the educational guide (booklet) for the families of babies diagnosed with PKU, telephone calls during the education process and counseling services. No application will be made to the families in the control group. After the application of the last tests to the control group; Face-to-face training and a booklet will be given to the experimental group. Data; It will be collected with 'Child and Family Introductory Information Form', 'Parents' Information Level Assessment Form about Phenylketonuria' and 'Coping Attitudes Assessment Scale'.
In the literature, there is no study showing the effect of the education given to the families of babies diagnosed with PKU on their coping attitudes. Based on the need to fill this knowledge gap in the literature, it is thought that the study will contribute to the field of child health and disease nursing.
Detailed description
Phenylketonuria is a disorder of phenylalanine metabolism and causes intellectual destruction if left untreated. It is an autosomal recessive metabolic disease that develops as a result of the absence of the enzyme phenylalanine hydroxylase (FAH), which provides the necessary amino acid metabolism for protein production in the body, which causes high phenylalanine in the blood. In our country, it is one of the places where PKU is commonly encountered (Çavuşoğlu, 2015; Hafid et al, 2015; Törner and Büyükgönenç, 2013). While the incidence of this disease in European countries is 1/10000-30000, it is 1/3000-4500 in our country (Alparslan, 2021). This situation is directly related to consanguineous marriages. It is stated that one out of every five marriages in our country is consanguineous marriage. If left untreated, it can cause extreme mental retardation, convulsions and death (Alparslan, 2021; Çavuşoğlu, 2015; Hafid et al, 2015; Törner and Büyükgönenç, 2013).
The care of the child with phenylketonuria is carried out by the parents. Extra care (diet therapy, control of blood phenylalanine level, etc.) for children in this diagnosis group is provided by their parents. The treatment and care process of children with PKU affects family routines, relationships and parenting styles. Parents of children experience emotional and economic burden. Therefore, it is stated that the care burden of parents in this diagnosis group is higher than that of healthy parents, and their quality of life is lower (Hatzmann et al, 2009). In addition, studies have shown that families of children with PKU have high levels of anxiety, depression (Mahmoudi-Gharaei et al. 2011) and stress (Irannejad et al. 2018). In another study; It is reported that the family's routines have changed (not eating in front of the child, not eating in restaurants, etc.) and that they experience anxiety about the effect of diet incompatibility on blood phenylalanine levels, feeling overwhelmed and guilt in the care process (Carpenter et al 2018). Therefore, having a child with PKU may affect coping attitudes.
Child health and diseases nurse should support children with PKU and their families on issues such as informing, training and counseling about the disease and its management.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- The study was completed with the families of 36 children with PKU, 19 in the intervention group and 17 in the control group, who met the inclusion criteria.
| Arm | Type | Receives |
|---|---|---|
| Experimental group | Experimental | Behavioral: Family educationFamily education |
| Control group | No intervention | –No training will be given. |
Who may qualify
- Ages
- 1 day to 3 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Residing in Van and/or being followed up in the pediatric metabolism polyclinic,
- Agreeing to participate in the research,
- literate,
- Speaking and understanding Turkish,
- 18 years old and over
Key exclusion criteria (7 listed)
- Not residing in Van and/or not being followed up in the pediatric metabolism polyclinic,
- Those who do not accept to participate in the research, who are illiterate,
- Does not speak or understand Turkish,
- Parent/family member who is aged 17 or younger, who is not fully oriented and cooperative and not open to communication, and does not provide primary care for the child,
- The child is over 3 years old (3 years <),
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Family educationBy calling the families in the experimental group by phone; Nursing interventions for family empowerment will last for 10 weeks, they will be invited to the Family Health Center (FHC) or child metabolism polyclinic twice in this process, they will be called once a week (with 2 phone calls) for two weeks after the face-to-face training initiative in the first stage, In the second stage, at the end of the face-to-face training, they will be informed that they will be called once a week for two weeks (4 phone calls in total). The families in the experimental group will be informed that 4 weeks after the completion of the training and phone calls (at the 10th week), they will call the FHC or the pediatric metabolism polyclinic again for the application of the post-tests and that the nursing training for family empowerment will be completed. | Behavioural | Experimental group |
Primary outcome
Change from Baseline in Evaluation Of Coping Attitudes (COPE) at week 10
Time frame: 10 week
*COPE is a validated. The scale is a 4-point Likert type scale. While scoring the items, 1 point is given for "I Never Do This", 2 points for "I rarely do this", 3 points for "I do this way", and 4 points for "I Do This Mostly". There is no negative item in the scale. The highest score that can be obtained from the scale is 128, and the lowest score is 32. A high score from the scale means a high level of coping attitude, and a low score means a low level of coping attitude. change = (Week 10 Score - Baseline Score)
1 secondary outcome
Change from Baseline in Evaluation of the level of knowledge about PKU at week 1
Time frame: 10 week
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Oct 2023.
- Background: PubMed 16763891MacDonald A, Depondt E, Evans S, Daly A, Hendriksz C, Chakrapani A A, Saudubray JM. Breast feeding in IMD. J Inherit Metab Dis. 2006 Apr-Jun;29(2-3):299-303. doi: 10.1007/s10545-006-0332-x.
- Background: PubMed 30705824Pinto A, Adams S, Ahring K, Allen H, Almeida MF, Garcia-Arenas D, Arslan N, Assoun M, Atik Altinok Y, Barrio-Carreras D, Belanger Quintana A, Bernabei SM, Bontemps C, Boyle F, Bruni G, Bueno-Delgado M, Caine G, Carvalho R, Chrobot A, Chyz K, Cochrane B, Correia C, Corthouts K, Daly A, De Leo S, Desloovere A, De Meyer A, De Theux A, Didycz B, Dijsselhof ME, Dokoupil K, Drabik J, Dunlop C, Eberle-Pelloth W, Eftring K, Ekengren J, Errekalde I, Evans S, Foucart A, Fokkema L, Francois L, French M, Forssell E, Gingell C, Goncalves C, Gokmen Ozel H, Grimsley A, Gugelmo G, Gyure E, Heller C, Hensler R, Jardim I, Joost C, Jorg-Streller M, Jouault C, Jung A, Kanthe M, Koc N, Kok IL, Kozanoglu T, Kumru B, Lang F, Lang K, Liegeois I, Liguori A, Lilje R, Lubina O, Manta-Vogli P, Mayr D, Meneses C, Newby C, Meyer U, Mexia S, Nicol C, Och U, Olivas SM, Pedron-Giner C, Pereira R, Plutowska-Hoffmann K, Purves J, Re Dionigi A, Reinson K, Robert M, Robertson L, Rocha JC, Rohde C, Rosenbaum-Fabian S, Rossi A, Ruiz M, Saligova J, Gutierrez-Sanchez A, Schlune A, Schulpis K, Serrano-Nieto J, Skarpalezou A, Skeath R, Slabbert A, Straczek K, Gizewska M, Terry A, Thom R, Tooke A, Tuokkola J, van Dam E, van den Hurk TAM, van der Ploeg EMC, Vande Kerckhove K, Van Driessche M, van Wegberg AMJ, van Wyk K, Vasconcelos C, Velez Garcia V, Wildgoose J, Winkler T, Zolkowska J, Zuvadelli J, MacDonald A. Weaning practices in phenylketonuria vary between health professionals in Europe. Mol Genet Metab Rep. 2018 Nov 25;18:39-44. doi: 10.1016/j.ymgmr.2018.11.003. eCollection 2019 Mar.
- Background: PubMed 34017006van Spronsen FJ, Blau N, Harding C, Burlina A, Longo N, Bosch AM. Phenylketonuria. Nat Rev Dis Primers. 2021 May 20;7(1):36. doi: 10.1038/s41572-021-00267-0.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Şenay ÇETİNKAYA, Doç. Dr. study director
Cukurova University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Family education in people with Phenylketonurias, run by Cukurova University at 1 site in Turkey (Türkiye).
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Turkey (Türkiye). Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from Baseline in Evaluation Of Coping Attitudes (COPE) at week 10 (time frame: 10 week). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05497050, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Oct 2023.