Could a common supplement help kids with NF1 learn and move better?
NCT ID NCT04481035
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested whether N-acetylcysteine (NAC), an over-the-counter antioxidant, could improve learning and motor skills in children with neurofibromatosis type 1 (NF1). Five children aged 8 to 16 took NAC or a placebo to see if it helped with movement and attention problems. The goal was to explore safety and early signs of benefit for these common NF1-related challenges.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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5 people
The number who actually took part.
- Started
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Jan 2019
- Finished
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Dec 2021
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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8 to 16 years
- Sex
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Anyone
- Healthy volunteers
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Accepted
You do not need to have the condition being studied to take part.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Males and females aged 8 - 16 years at time of enrollment whom meet NIH diagnostic criteria for NF1. 2. 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. 3. Participants on stimulant or any other psychotropic medication should stay on a stable dose for at least 30 days before entering the study. Exclusion Criteria: 1. Participants should not be receiving chemotherapy currently, or have received chemotherapy in the 6 months prior to entering the study. 2. No active intracranial lesions (stable low grade glioma are acceptable) or epilepsy diagnosis. 3. 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. 4. For females, pregnancy. 5. Current use of antidepressants, non-stimulant ADHD medications, dopamine blocking agents, mood stabilizers. 6. Implanted brain stimulator, vagal nerve stimulator, ventriculoperitoneal (VP) shunt, cardiac pacemaker, or implanted medication port. 7. Asthma (bronchospasm has been reported as occurring infrequently and unpredictable when acetylcysteine is used as a mucolytica agent). 8. High risk of upper gastrointestinal (GI) hemorrhage. Examples: presence of esophageal varices or peptic ulcers
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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