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NCT04481035 · ClinicalTrials.gov registry record · Phase 2
Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1
A Phase 2 study, sponsored by Children's Hospital Medical Center, Cincinnati.
- Completed
- Registry status
- Phase 2
- Development phase
- 5
- Enrollment target
NCT04481035 is a Phase 2 study that has completed, run by Children's Hospital Medical Center, Cincinnati. The registered enrollment target is 5 participants.
The verdict
NCT04481035, a Phase 2 study, has completed, sponsored by Children's Hospital Medical Center, Cincinnati.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 5 participants
- Enrollment target
Study 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. However, data from rodent models of NF1 along with uncontrolled clinical observations in children with NF1 suggest that the anti-oxidant, glutamate modulating compound N-Acetyl Cysteine (NAC) may reduce these impairments. Of particular interest is a murine study analyzing the central nervous system manifestations of NF1 at our institution. That study revealed a role for myelin-forming oligodendrocytes in the control of nitric oxide synthases (NOS) and their product, nitric oxide, in maintenance of brain structure and function, including regulation of behavior and motor control. Treating these mice with NAC corrected cellular and behavioral abnormalities. N-Acetyl Cysteine is available over the counter and has been used by thousands of individuals; moreover, it has shown some promise in clinical trials for psychiatric disorders. In order to better understand treatment mechanisms, and possibly predict long-term outcomes, the investigators propose concurrently to explore Specific Aim 1 (1.1, 1.2, and 1.3) exploratory potential disease biomarkers as outlined below. The primary outcome of this study is motor function rated with the Physical and Neurological Examination for Subtle Signs (PANESS), a validated scale that consistently demonstrates significant impairments in children with Attention Deficit Hyperactivity Disorder (ADHD), and which our preliminary data suggest may demonstrate more extreme problems in children with NF1. The first exploratory biomarker is motor system inhibitory physiology, measured using Transcranial Magnetic Stimulation (TMS). Preliminary measures in our NF1 population also show abnormalities similar to established findings in ADHD. The second exploratory biomarker is metabolomics profiling for the biomarker of oligodendrocyte dysfunction in NF1 parti
Interventions
- OTHER Placebo
- DRUG N-acetylcysteine (NAC)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 5 participants |
| Start Date | 2019-01-15 |
| Est. Completion | 2021-12-16 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04481035
The ClinicalTrials.gov registry entry for NCT04481035 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 5 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Children's Hospital Medical Center, Cincinnati, which has 571 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT04481035 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT04481035 about?
NCT04481035 is a clinical study titled "Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1". 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. However, data from rodent models of NF1 along with uncontrolled clinical observations in children with N...
What is the current status of trial NCT04481035?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 5 participants. The study started on 2019-01-15. Estimated completion is 2021-12-16.
What interventions are being tested in trial NCT04481035?
The interventions under investigation include: Placebo (OTHER), N-acetylcysteine (NAC) (DRUG).
Who is sponsoring clinical trial NCT04481035?
This trial is sponsored by Children's Hospital Medical Center, Cincinnati, which has 571 total clinical trials registered on ClinicalTrials.gov.
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