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NCT04481048 · ClinicalTrials.gov registry record · Phase 2
Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1
A Phase 2 study of Neurofibromatosis 1, sponsored by Children's Hospital Medical Center, Cincinnati.
- Active
- Registry status
- Phase 2
- Development phase
- 58
- Enrollment target
- 1
- Study location
NCT04481048: Active Phase 2 study of Neurofibromatosis 1, sponsored by Children's Hospital Medical Center, Cincinnati.
NCT04481048 is a Phase 2 study of Neurofibromatosis 1 that is active but no longer recruiting, run by Children's Hospital Medical Center, Cincinnati. The registered enrollment target is 58 participants, below the 1,033-participant average among 24 other Neurofibromatosis 1 trials with a reported enrollment target (94% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04481048, a Phase 2 study of Neurofibromatosis 1, is active but no longer recruiting, sponsored by Children's Hospital Medical Center, Cincinnati.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 58 participants
- Enrollment target
- 1
- Study location
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. In this project, the investigators will assess the safety and clinical benefit of N-acetylcysteine (NAC) as a pharmacological intervention in children with NF1. This drug choice is based on the recent findings from mouse models to study the central nervous system manifestations of NF1 at Cincinnati Children's Hospital Medical Center (CCHMC). These findings revealed a role for myelin-forming oligodendrocytes in the control of nitric oxide synthases (NOS) and their product, nitric oxide (NO), in maintenance of brain structure and function, including regulation of behavior and motor control. Treating these mice with NAC corrected cellular and behavioral abnormalities. This data from animal models of NF1 along with uncontrolled clinical observations in children with NF1 suggest that the antioxidant compound, NAC, may reduce these impairments. Therefore, the investigators propose performing a single center double-blind placebo controlled, prospective, Phase II study to explore safety, tolerability, and efficacy of NAC on motor behavior and/or learning in children with NF1 aged 8 through 16 years old. Participants will be carefully monitored for side effects. Primary and secondary outcome measures will be administered at baseline, follow-up, and post-treatment.
Primary Outcome
Characterize effects of NAC treatment on motor function in kids with NF1 using the Physical and Neurological Examination for Soft 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 i
Conditions Studied
Interventions
- OTHER Placebo
- DRUG N-Acetyl cysteine
Study Locations (1)
Ohio
- Cincinnati Children's Hospital Medical Center - Cincinnati
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 58 participants |
| Start Date | 2020-12-15 |
| Est. Completion | 2025-12 |
| Phase | Phase 2 |
What the registry record for NCT04481048 still lists
NCT04481048 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 58 participants, a relatively small participant target, below the 1,033-participant average among 24 other Neurofibromatosis 1 trials with a reported enrollment target (94% lower).
The record links to 1 condition, with Neurofibromatosis 1 appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT04481048 reports a single indexed study location in Ohio.
Frequently Asked Questions
What is clinical trial NCT04481048 about?
NCT04481048 is a clinical study titled "Antioxidant Therapy With N-acetylcysteine for 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. In this project, the investigators will assess the safety and clinical benefit of N-acetylcysteine (NAC...
What is the current status of trial NCT04481048?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 58 participants. The study started on 2020-12-15. Estimated completion is 2025-12.
What conditions does trial NCT04481048 study?
This clinical trial studies the following conditions: Neurofibromatosis 1.
What interventions are being tested in trial NCT04481048?
The interventions under investigation include: Placebo (OTHER), N-Acetyl cysteine (DRUG).
Who is sponsoring clinical trial NCT04481048?
This trial is sponsored by Children's Hospital Medical Center, Cincinnati, which has 571 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04481048 being conducted?
This trial has 1 study location across Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04481048's enrollment target sits among peer trials
58 15th of 24 higher than 9 of 24 other Neurofibromatosis 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Neurofibromatosis 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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