Antioxidant drug shows promise for kids with NF1
NCT ID NCT04481048
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested whether N-acetylcysteine (NAC), an antioxidant drug, can improve motor skills and learning in children with neurofibromatosis type 1 (NF1). Twenty-five children aged 8 to 16 took NAC or a placebo for 8 weeks. The goal was to see if NAC is safe and helps with movement and attention problems common in NF1.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- N-acetylcysteine (NAC)
- What this could lead to
- If it works, this could point toward a treatment for motor and learning problems in children with NF1.
- What could go wrong
- This is a small, early-phase trial with only 25 participants. Results may not apply to all children with NF1, and the treatment may not show benefit.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
25 people
The number who actually took part.
- Started
-
Feb 2021
- Finished
-
Jun 2024
- Lead sponsor
-
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
-
8 to 16 years
- Sex
-
Anyone
- Healthy volunteers
-
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 ѱ: You can be in this study if you have any of the following: 1. Males and females older than 8 years and younger than 16 years old 2. Has a diagnosis of NF1 (neurofibromatosis type 1) 3. Has an abnormal PANESS score 4. Has an intelligence quotient (IQ) at or above 70 5. Participants on stimulant or any other psychotropic medication should stay on a stable dose (no change in dose) for at least 30 days before entering the study and maintain that dose while in the study Exclusion Criteria: You cannot be in this study if you have any of the following: 1. Younger than 8 years or older than 16 years ѱ 2. Do not have a diagnosis of NF1 ѱ 3. IQ below 70 ѱ 4. Had a dose change of any stimulant or psychotropic medication in the last month (30 days) ѱ 5. Are being treated with chemotherapy or had chemotherapy in the last 6 months 6. Have epilepsy ѱ 7. High risk of upper gastrointestinal (GI, the stomach and the small and large intestine) hemorrhage (bleeding). Examples: presence of esophageal varices or peptic ulcers 8. Active intracranial lesions (abnormality found on brain imaging such as an MRI) (stable low-grade glioma is acceptable) or epilepsy diagnosis ѱ 9. Have Major Depression, Bipolar Disorder, Conduct Disorder, Adjustment Disorder, other major Anxiety Disorders, or other developmental psychiatric diagnoses, based on history. ADHD is allowed. 10. For females, pregnancy 11. Is currently using antidepressants, dopamine blocking agents, or mood stabilizers 12. Have any of the following medical devices: implanted brain stimulator, vagal nerve stimulator, ventriculoperitoneal (VP) shunt, cardiac pacemaker, or implanted medication port ѱ 13. Asthma (bronchospasm has been reported as occurring infrequently and unpredictably when NAC is used as a mucolytic agent) 14. Current use of MEKINIST (MEK-inhibitor) or use within 30 days ѱ Indicates Inclusion/Exclusion Criteria for the treatment- and non-treatment cohorts (no mark indicates exclusion requirements for the 12-week treatment-cohort only).
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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
-
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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