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NCT02297347 · ClinicalTrials.gov registry record

The Brain and Neuropsychological Functioning in Adults With Sapropterin Dihydrochloride Treated Phenylketonuria

A clinical trial, sponsored by Boston Children's Hospital.

Completed
Registry status
10
Enrollment target

NCT02297347: Completed study, sponsored by Boston Children's Hospital.

NCT02297347 is a clinical trial that has completed, run by Boston Children's Hospital. The registered enrollment target is 10 participants. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT02297347 has completed, sponsored by Boston Children's Hospital.

COMPLETED
Registry status
10 participants
Enrollment target

Study Summary

Newborn screening and early treatment prevent the most severe manifestations of phenylketonuria (PKU). However, executive functioning deficits, attention deficit disorder, slow processing speed, and visual-motor problems commonly occur. Many adults with this disorder also suffer depression and anxiety. In this study the investigators will examine adults with PKU on sapropterin dihydrochloride (Kuvan) treatment for PKU and compare their results to those of subjects with PKU not on Kuvan. Using magnetic resonance imaging (MRI) techniques, including novel MR spectroscopy (MRS) the investigators hope to discover why this distinct constellation of deficits occurs in PKU. Adult subjects with PKU will undergo a comprehensive MRI evaluations, including a novel method of MR spectroscopy to determine brain phenylalanine levels. In addition, participants will receive neurological and neuropsychological examinations and dietary evaluation.

Primary Outcome

Brain phe described as umol/L through MR Spectroscopy in both the , posterior cingulate gyrus (PCG) and parietal white matter (PWM)

Trial Details

FieldValue
Enrollment Target 10 participants
Start Date 2015-04
Est. Completion 2017-04
Boston Children's Hospital

462 total trials

What the finished NCT02297347 record still lists

NCT02297347 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 10 participants, a relatively small participant target.

The record links to 0 conditions, and to 0 interventions.

NCT02297347 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT02297347 about?

NCT02297347 is a clinical study titled "The Brain and Neuropsychological Functioning in Adults With Sapropterin Dihydrochloride Treated Phenylketonuria". Newborn screening and early treatment prevent the most severe manifestations of phenylketonuria (PKU). However, executive functioning deficits, attention deficit disorder, slow processing speed, and visual-motor problems commonly occur. Many adults with this disorder also suffer depression and anxie...

What is the current status of trial NCT02297347?

This trial is currently completed. The enrollment target is 10 participants. The study started on 2015-04. Estimated completion is 2017-04.

Who is sponsoring clinical trial NCT02297347?

This trial is sponsored by Boston Children's Hospital, which has 462 total clinical trials registered on ClinicalTrials.gov.

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Source: ClinicalTrials.gov NCT02297347, the US trial registry maintained by the National Library of Medicine. NCT02297347 (small enrollment · none site footprint · completed) retrieved and formatted by PlainTrial, see methodology.