Status:

COMPLETED

Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1

Lead Sponsor:

Children's Hospital Medical Center, Cincinnati

Conditions:

Neurofibromatosis 1

Eligibility:

All Genders

8-16 years

Phase:

PHASE2

Brief Summary

Children with neurofibromatosis type 1 (NF1) commonly suffer from the effects of cognitive, behavioral, and motor impairments. At present there is no specific treatment for this NF1 complication. Howe...

Detailed Description

The aims of this application are to gain information in children with NF1 about possible clinical benefit of anti-oxidant treatment and to develop and evaluate quantitative brain-based and blood bioma...

Eligibility Criteria

Inclusion

  • Males and females aged 8 - 16 years at time of enrollment whom meet NIH diagnostic criteria for NF1.
  • Participants must have a full-scale intelligence quotient (IQ) of 70 or above, as determined by neurocognitive testing within the last 3 years or during the enrollment process.
  • Participants on stimulant or any other psychotropic medication should stay on a stable dose for at least 30 days before entering the study.

Exclusion

  • Participants should not be receiving chemotherapy currently, or have received chemotherapy in the 6 months prior to entering the study.
  • No active intracranial lesions (stable low grade glioma are acceptable) or epilepsy diagnosis.
  • Major Depression, Bipolar Disorder, Conduct Disorder, Adjustment Disorder, other major Anxiety Disorders, or other developmental psychiatric diagnoses, based on the child's history or on parent and child responses from the Kiddie Schedule for Affective Disorders and Schizophrenia (KSADS). Note that while this is an exclusion for participation in the study if there is a prior evaluation available, this becomes a criterion, after inclusion, for the investigator to withdraw the child from the study prior to completion if identified on the first study day.
  • For females, pregnancy.
  • Current use of antidepressants, non-stimulant ADHD medications, dopamine blocking agents, mood stabilizers.
  • Implanted brain stimulator, vagal nerve stimulator, ventriculoperitoneal (VP) shunt, cardiac pacemaker, or implanted medication port.
  • Asthma (bronchospasm has been reported as occurring infrequently and unpredictable when acetylcysteine is used as a mucolytica agent).
  • High risk of upper gastrointestinal (GI) hemorrhage. Examples: presence of esophageal varices or peptic ulcers

Key Trial Info

Start Date :

January 15 2019

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

December 16 2021

Estimated Enrollment :

5 Patients enrolled

Trial Details

Trial ID

NCT04481035

Start Date

January 15 2019

End Date

December 16 2021

Last Update

July 31 2025

Active Locations (1)

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Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States, 45229