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Classical Phenylketonuria(PKU)NCT01650909

The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU

An observational study of Classical Phenylketonuria(PKU), run by Stanford University.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

This study is an independent sub-study of the protocol titled PKU-016: A double-blind, placebo-controlled, randomized study to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuro-psychiatric symptoms in subjects with phenylketonuria (PKU ASCEND). Read more about this study

Who is it for?
Ages 8 years and older
Conditions the study is for
Classical Phenylketonuria(PKU)
How many people
0 participants
Where
No sites listed yet
Study dates
Jul 2012to not given
Phase
Observational
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 8 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Study population: Children 8+ and adults, any gender or ethic background, diagnosed with classical phenylketonuria and participating in PKU-016.

Inclusion Criteria:Sub-study Inclusion criteria same as main study:

  • ≥ 8 years of age
  • Confirmed diagnosis of PKU
  • Willing to continue current diet (typical diet for the 3 months prior to study entry)unchanged while participating in the study
  • Willing and able to provide written, signed informed consent or in the case of subjects under the age of 18, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures
  • Sexually active subjects must be willing to use an acceptable method of contraception while participating in the study and for at least 30 days following the last dose of sapropterin dihydrochloride
  • Females of childbearing potential must have a negative pregnancy test at screening and be willing to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have been in menopause at least 2 years, or had tubal ligation at least 1 year prior to screening, or have had total hysterectomy
  • Willing and able to comply with all study procedures

Exclusion Criteria:All other sub-study Exclusion criteria same as main study:

  • Has known hypersensitivity to sapropterin dihydrochloride or its excipients
  • Subject breastfeeding at screening or planning to become pregnant (subject or partner) at any time during the study
  • Use of any investigational product or investigational medical device within 30 days prior to screening, or requirement for any investigational agent prior to the completion of all scheduled study assessments
  • Received sapropterin dihydrochloride within 16 weeks of randomization • Have initiated or adjusted medication for treatment of ADHD, depression, or anxiety ≤ 8 weeks prior to randomization
  • Taking medication known to inhibit folate synthesis (eg, methotrexate)
  • Any condition requiring treatment with levodopa or any PDE-5 inhibitor
  • Concurrent disease or condition that would interfere with study participation, compliance or safety as determined by the Investigator
  • Any condition that, in the view of the Investigator, places the subject at high risk of poor treatment compliance or not completing the study

About this study

Official title: The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical Phenylketonuria

This study is an independent sub-study of the protocol titled PKU-016: A double-blind, placebo-controlled, randomized study to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuro-psychiatric symptoms in subjects with phenylketonuria (PKU ASCEND).

The primary objective of this study is to determine oxidative stress in patients with classical phenylketonuria (PKU) enrolled in PKU-016, using a brain scan (called an HMPAO SPECT) at baseline and 26 weeks, and blood redox biomarkers.

Medical subject headings
Phenylketonurias

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Determine in vivo redox status in patients with classical phenylketonuria

    The primary objective of this study is to determine in vivo redox status in patients with classical phenylketonuria enrolled in PKU-016, compared to historical normal controls, using:

    • Serial Tc99m-HMPAO SPECT brain imaging (baseline and 26 weeks); and
    • Blood redox biomarkers, including oxidized and reduced glutathione, tetrahydrobiopterin, ascorbate, alpha-tocotrienol, selenium, etc.
Secondary outcomes (2)

  1. Compare redox status with neuropsychological and neuro-cognitive symptoms

    Compare redox status as determined by brain imaging and blood redox biomarkers to measures of neuropsychological and neuro-cognitive symptoms (ADHD, anxiety, depression and executive function) and global function using data collected as part of PKU-016

  2. Explore the utility of other blood redox biomarkers

    Explore the utility of other blood redox biomarkers (e.g. NAD+/NADH, NADP+/NADPH, protein carbonyls) in determining level of oxidative stress.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Jul 2012 (estimated)
  2. Study startJul 2012

Why it stopped: Withdrawn due to no funding

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Time perspective
Prospective
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01650909
Sponsor's protocol ID
Redox Sub-study of PKU-016
Status last verified
Apr 2014
First submitted
24 Jul 2012
Last updated
23 Apr 2014
Results posted
No

Publications

  • Atkuri KR, Cowan TM, Kwan T, Ng A, Herzenberg LA, Herzenberg LA, Enns GM. Inherited disorders affecting mitochondrial function are associated with glutathione deficiency and hypocitrullinemia. Proc Natl Acad Sci U S A. 2009 Mar 10;106(10):3941-5. doi: 10.1073/pnas.0813409106. Epub 2009 Feb 17.

    PubMed 19223582

  • Christ SE, Huijbregts SC, de Sonneville LM, White DA. Executive function in early-treated phenylketonuria: profile and underlying mechanisms. Mol Genet Metab. 2010;99 Suppl 1:S22-32. doi: 10.1016/j.ymgme.2009.10.007.

    PubMed 20123466

  • Fernandes CG, Leipnitz G, Seminotti B, Amaral AU, Zanatta A, Vargas CR, Dutra Filho CS, Wajner M. Experimental evidence that phenylalanine provokes oxidative stress in hippocampus and cerebral cortex of developing rats. Cell Mol Neurobiol. 2010 Mar;30(2):317-26. doi: 10.1007/s10571-009-9455-6. Epub 2009 Sep 23.

    PubMed 19774456

  • Jacquier-Sarlin MR, Polla BS, Slosman DO. Oxido-reductive state: the major determinant for cellular retention of technetium-99m-HMPAO. J Nucl Med. 1996 Aug;37(8):1413-6.

    PubMed 8708786

  • Sasaki T, Senda M. Technetium-99m-meso-HMPAO as a potential agent to image cerebral glutathione content. J Nucl Med. 1997 Jul;38(7):1125-9.

    PubMed 9225804

  • Sirtori LR, Dutra-Filho CS, Fitarelli D, Sitta A, Haeser A, Barschak AG, Wajner M, Coelho DM, Llesuy S, Bello-Klein A, Giugliani R, Deon M, Vargas CR. Oxidative stress in patients with phenylketonuria. Biochim Biophys Acta. 2005 Apr 15;1740(1):68-73. doi: 10.1016/j.bbadis.2005.02.005. Epub 2005 Feb 25.

    PubMed 15878743

  • Sitta A, Barschak AG, Deon M, Terroso T, Pires R, Giugliani R, Dutra-Filho CS, Wajner M, Vargas CR. Investigation of oxidative stress parameters in treated phenylketonuric patients. Metab Brain Dis. 2006 Dec;21(4):287-96. doi: 10.1007/s11011-006-9035-0. Epub 2006 Dec 5.

    PubMed 17146735

  • Sitta A, Manfredini V, Biasi L, Tremea R, Schwartz IV, Wajner M, Vargas CR. Evidence that DNA damage is associated to phenylalanine blood levels in leukocytes from phenylketonuric patients. Mutat Res. 2009 Sep-Oct;679(1-2):13-6. doi: 10.1016/j.mrgentox.2009.07.013. Epub 2009 Aug 7.

    PubMed 19665577

Questions about this trial

What is this clinical trial studying?

An observational study of Classical Phenylketonuria(PKU), run by Stanford University.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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: withdrawn, as of the sponsor's last update on 23 Apr 2014. Status can change between updates, so confirm with the study contact before making plans.

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 was withdrawn before enrolling participants, so there are no results.

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?

Determine in vivo redox status in patients with classical phenylketonuria. 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 NCT01650909, which also lists 8 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 23 Apr 2014.

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