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Antioxidant drug shows promise for kids with NF1

NCT ID NCT04481048

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

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.