Trial · NCT04481035
Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1
NCT04481035, Phase 2, Completed, Children's Hospital Medical Center, Cincinnati; results first posted 2022-03-15
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1. Trials, record NCT04481035. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04481035
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1
- NCT number
- NCT04481035
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 5 participants, actual
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Conditions
- Neurofibromatosis 1
- Interventions
- N-acetylcysteine (NAC), Placebo
- First posted
- 2020-07-22
- Last updated
- 2026-04-21
- Results first posted
- 2022-03-15
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Motor Function Measured by Physical and Neurological Examination for Subtle Signs (PANESS) | At baseline and end of 8 weeks treatment with either NAC or placebo (weeks 0 and 8 for treatment phase one of this cross-over double blind study and at weeks 10 and 18 for treatment phase two). | Characterize effects of NAC treatment on motor function in kids with NF1 using the Physical and Neurological Examination for Subtle Signs (PANESS). This is a validated scale that consistently demonstrates significant impairments in children with ADHD, and which preliminary data suggest may demonstrate more extreme problems in children with NF1 than age-matched healthy controls (unpublished data from CCHMC). The investigators hypothesize that motor function scores rated with the PANESS scale will improve after treatment with NAC. The range of this scale is 0-119, higher scores correlate with symptom severity (worse outcome). |
| Primary | Change From Baseline in ADHD Symptoms as Reported Via Parent/Teacher Surveys | At baseline and end of 8 weeks treatment with either NAC or placebo (weeks 0 and 8 for treatment phase one of this cross-over double blind study and at weeks 10 and 18 for treatment phase two). | Characterize effects of NAC treatment on ADHD symptoms in children with NF1. The investigators hypothesize that ADHD attention and hyperactive/impulsive symptoms, rated with the DuPaul DSM-5 based clinical rating scales, will improve after treatment with NAC. The range of this scale is 0-56, higher scores correlate with symptom severity (worse outcome). |
| Secondary | Transcranial Magnetic Stimulation (TMS) - Cortical Silent Period | At baseline and end of 8 weeks treatment with either NAC or placebo (weeks 0 and 8 for treatment phase one of this cross-over double blind study and at weeks 10 and 18 for treatment phase two). | Transcranial Magnetic Stimulation (TMS) - Cortical Silent Period (CSP). This measure describes the function and physiology of the motor system using Transcranial Magnetic Stimulation (TMS) over the brain to evoke a muscle twitch in the hand. These evoked potentials provide information about the instantaneous balance of excitation and inhibition in the brain, which in turn relate in part to neurotransmitter levels that can be altered by diseases and by treatments. This measure reflects an inhibitory neurotransmitter called GABA-B and its action at a particular receptor - the "GABA B" receptor. A lengthening of the duration of CSP indicates more inhibition, which is good (within a healthy range of approximately 50 to 150 ms, because outside of that range is abnormal). Here we report the average difference before and after treatment. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Motor Function Measured by Physical and Neurological Examination for Subtle Signs (PANESS) | At baseline and end of 8 weeks treatment with either NAC or placebo (weeks 0 and 8 for treatment phase one of this cross-over double blind study and at weeks 10 and 18 for treatment phase two). | Descriptive only | score on a scale | MEDIAN |
| PRIMARY | Change From Baseline in ADHD Symptoms as Reported Via Parent/Teacher Surveys | At baseline and end of 8 weeks treatment with either NAC or placebo (weeks 0 and 8 for treatment phase one of this cross-over double blind study and at weeks 10 and 18 for treatment phase two). | Modified ITT - all participants who received at least one dose of study drug | units on a scale | MEAN |
| SECONDARY | Transcranial Magnetic Stimulation (TMS) - Cortical Silent Period | At baseline and end of 8 weeks treatment with either NAC or placebo (weeks 0 and 8 for treatment phase one of this cross-over double blind study and at weeks 10 and 18 for treatment phase two). | modified ITT - at least one dose given | ms | MEAN |
| SECONDARY | Metabolic Testing Biomarkers | At baseline and end of 8 weeks treatment with either NAC or placebo (weeks 0 and 8 for treatment phase one of this cross-over double blind study and at weeks 10 and 18 for treatment phase two). | |||
| OTHER_PRE_SPECIFIED | Evaluation of Change From Baseline in Metabolomics Profiles as a Possible Disease Biomarker Metabolic Testing Biomarkers - Samples in Storage for Follow up Study | At baseline and end of 8 weeks treatment with either NAC or placebo (weeks 0 and 8 for treatment phase one of this cross-over double blind study and at weeks 10 and 18 for treatment phase two). |
Source ClinicalTrials.gov
Results by group6
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Motor Function Measured by Physical and Neurological Examination for Subtle Signs (PANESS) | N-Acetylcysteine | 0 | Full Range -8 to 2 | score on a scale | |
| Change From Baseline in Motor Function Measured by Physical and Neurological Examination for Subtle Signs (PANESS) | Placebo | 3 | Full Range -4 to 25 | score on a scale | |
| Change From Baseline in ADHD Symptoms as Reported Via Parent/Teacher Surveys | N-Acetylcysteine | -12.5 | Standard Deviation 13.1 | units on a scale | |
| Change From Baseline in ADHD Symptoms as Reported Via Parent/Teacher Surveys | Placebo | -3.75 | Standard Deviation 12.0 | units on a scale | |
| Transcranial Magnetic Stimulation (TMS) - Cortical Silent Period | N-Acetylcysteine | 6 | Standard Deviation 19 | ms | |
| Transcranial Magnetic Stimulation (TMS) - Cortical Silent Period | Placebo | -4 | Standard Deviation 18 | ms |
Source ClinicalTrials.gov
Participant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Period 1 | STARTED | N-Acetylcysteine Followed by Placebo | 3 | |
| Period 1 | STARTED | Placebo Followed by N-Acetylcysteine | 2 | |
| Period 1 | COMPLETED | N-Acetylcysteine Followed by Placebo | 3 | |
| Period 1 | COMPLETED | Placebo Followed by N-Acetylcysteine | 2 | |
| Period 1 | NOT COMPLETED | N-Acetylcysteine Followed by Placebo | 0 | |
| Period 1 | NOT COMPLETED | Placebo Followed by N-Acetylcysteine | 0 | |
| Period 2 | STARTED | N-Acetylcysteine Followed by Placebo | 3 | |
| Period 2 | STARTED | Placebo Followed by N-Acetylcysteine | 2 | |
| Period 2 | COMPLETED | N-Acetylcysteine Followed by Placebo | 3 | |
| Period 2 | COMPLETED | Placebo Followed by N-Acetylcysteine | 2 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | N-Acetylcysteine Treatment | 0 | 5 |
| Total, all-cause mortality | Placebo | 0 | 5 |
| Total, other adverse events | N-Acetylcysteine Treatment | 0 | 5 |
| Total, other adverse events | Placebo | 0 | 5 |
| Total, serious adverse events | N-Acetylcysteine Treatment | 0 | 5 |
| Total, serious adverse events | Placebo | 0 | 5 |
Source ClinicalTrials.gov
Papers27
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| N-Acetylcysteine Treatment vs Placebo: deaths from any cause | Trial comparisons |
| N-Acetylcysteine Treatment vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04481035
GET https://conductscience.org/api/trials/v1/NCT04481035/papers?page=2