This study aims to evaluate the effects of Tetrahydrobiopterin (BH4) on the treatment and dietary regimen of children with phenylketonuria (PKU).
In this study, 20 Children ranging from 1 to 10 years old with controlled PKU are enrolled. Patients are prescribed normal dietary regimen for three days. Afterward, phenylalanine (Phe) level will be measured and BH4 is administered orally with a daily dose of 20 mg/kg. After 24 and 48 hours, Phe level will be re-measured. Then patients with Phe level less than 600 µmol/L or more than 1200 µmol/L and those with intolerance to treatment will be eliminated and the rest of the patients continue treatment in the second phase of the study. In addition to BH4, these children are prescribed 50 mg of phenylalanine powder daily (in addition to the current Phe in dietary regimen). If by the end of the week, the level of Phe is normal, another 50 mg of Phe is added for next week and this process goes on weekly up to 3 months until Phe dose is twice as the initial dose and if tolerated, patients return to normal diet.
General information
Acronym
IRCT registration information
IRCT registration number:IRCT2016100230082N1
Registration date:2017-06-01, 1396/03/11
Registration timing:retrospective
Last update:
Update count:0
Registration date
2017-06-01, 1396/03/11
Registrant information
Name
Peyman Eshraghi
Name of organization / entity
Imam Reza hospital, Mashhad University of Medicine
Country
Iran (Islamic Republic of)
Phone
+98 38593045
Email address
eshraghip2@mums.ac.ir
Recruitment status
Recruitment complete
Funding source
Mashhad University of Medical Sciences
Expected recruitment start date
2015-01-01, 1393/10/11
Expected recruitment end date
2016-03-09, 1394/12/19
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Effectiveness of Tetrahydrobiopterin for improvement of dietary regimen of children with Phenylketonuria
Public title
Effect of kuvan on relaxation of diet in phenylketonuria
Purpose
Supportive
Inclusion/Exclusion criteria
Inclusion criteria: patients with controlled PKU; age range of 1-10 years; lack of underlying disorders (e.g. malnutrition, hepatic and renal diseases).
Exclusion criteria: BH4 deficiency; Phe levels less than 600 micromoles/liter or more than 1200 micromoles/liter; presence of fever or acute illness; intolerance to BH4 therapy.
Age
From 1 year old to 10 years old
Gender
Both
Phase
N/A
Groups that have been masked
No information
Sample size
Target sample size:
20
Randomization (investigator's opinion)
N/A
Randomization description
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Single
Other design features
Secondary Ids
empty
Ethics committees
1
Ethics committee
Name of ethics committee
Ethics Committee of Mashhad University of Medical Sciences
Street address
Central Building of Mashhad University of Medical Sciences (Ghorshi),Daneshgah 16, Daneshgah Street
City
Mashhad
Postal code
Approval date
2013-11-20, 1392/08/29
Ethics committee reference number
920734
Health conditions studied
1
Description of health condition studied
mild phenylketonuria
ICD-10 code
E70.1
ICD-10 code description
Other hyperphenylalaninaemias
Primary outcomes
1
Description
Serum levels of phenylalanine
Timepoint
In first phases, after three days of free diet, then 24 and 48 houres after prescription of BH4. In second phase, measured weekly
Method of measurement
HPLC
Secondary outcomes
empty
Intervention groups
1
Description
Kuvan,100 mg oral tablet, daily, 20 mg/kg.
Phenylalanine, orally powder, daily, 50 mg (additive phenylalanine of dietary regime), If level of Phe is normal, another 50 mg of Phe is added for next week and this process goes on until Phe dose is twice as the initial dose and if tolerated, they return to normal diet
Category
Treatment - Drugs
Recruitment centers
1
Recruitment center
Name of recruitment center
Imam Reza Hospital
Full name of responsible person
Dr Vajihe Chalak
Street address
Department Of Pediatric Endocrinology, Imam Reza Hospital, Imam Reza Square
City
Mashhad
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Mashhad University of Medical Sciences, Vice Chancellor for Research
Full name of responsible person
Dr Mohsen Tafaghodi
Street address
Central Building of Mashhad University of Medical Sciences (Ghorshi),Daneshgah 16, Daneshgah Street
City
Mashhad
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Mashhad University of Medical Sciences, Vice Chancellor for Research
Proportion provided by this source
100
Public or private sector
empty
Domestic or foreign origin
empty
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
empty
Person responsible for general inquiries
Contact
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Dr Vajihe Chalak
Position
Pediatric Assistent
Other areas of specialty/work
Street address
Department Of Pediatric Endocrinology, Imam Reza Hospital, Imam Reza Square
City
Mashhad
Postal code
Phone
+98 51 3859 3045
Fax
Email
drvch1380@yahoo.com
Web page address
Person responsible for scientific inquiries
Contact
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Dr Peyman Eshraghi
Position
Associate Proffesor of Pediatic Endocrinology
Other areas of specialty/work
Street address
Department Of Pediatric Endocrinology, Imam Reza Hospital, Imam Reza Square
City
Mashhad
Postal code
Phone
+98 51 3859 3045
Fax
Email
eshraghip2@mums.ac.ir
Web page address
Person responsible for updating data
Contact
Name of organization / entity
Mashhad Univvercity of Medical Sciences
Full name of responsible person
Dr Peyman Eshraghi
Position
Associate Proffesor of Pediatic Endocrinology
Other areas of specialty/work
Street address
Department Of Pediatric Endocrinology, Imam Reza Hospital, Imam Reza Square
City
Mashhad
Postal code
Phone
+98 51 3859 3045
Fax
Email
eshraghip2@mums.ac.ir
Web page address
Sharing plan
Deidentified Individual Participant Data Set (IPD)