Antioxidant Therapy for Neurofibromatosis Type 1
(DoDNAC Trial)
Trial Summary
What is the purpose of this trial?
This trial is testing a medication called NAC in children aged 8-16 with a condition called NF1. These children often have problems with movement and behavior, and there is no current treatment for these issues. NAC works by reducing harmful molecules in the brain, which may help improve these symptoms.
Will I have to stop taking my current medications?
If you are taking stimulant or psychotropic medications, you need to stay on a stable dose for at least 30 days before joining the study and keep that dose during the study. If you are using antidepressants, dopamine blocking agents, or mood stabilizers, you cannot participate in the trial.
What evidence supports the effectiveness of the drug N-Acetyl cysteine (NAC) for treating Neurofibromatosis Type 1?
Research suggests that oxidative stress plays a role in the development of tumors in Neurofibromatosis Type 1 (NF1), and NAC is known to help reduce oxidative stress. Although not directly tested for NF1, NAC's ability to balance oxidative stress may offer potential benefits for managing NF1-related conditions.12345
Is N-Acetyl cysteine (NAC) safe for use in humans?
How does the drug N-Acetyl cysteine (NAC) differ from other treatments for Neurofibromatosis Type 1?
N-Acetyl cysteine (NAC) is unique because it is an antioxidant that aims to reduce oxidative stress, which is a different approach compared to other treatments that may not target oxidative stress directly. This drug is also used in other conditions to protect cells from damage, which highlights its potential to address underlying cellular issues in Neurofibromatosis Type 1.6891011
Research Team
Donald Gilbert, MD MS
Principal Investigator
Children's Hospital Medical Center, Cincinnati
Eligibility Criteria
This trial is for children aged 8-16 with neurofibromatosis type 1 (NF1) and an IQ of 70 or above. They must have a specific abnormal score on a motor skills test and be on stable doses of any psychotropic medication. It excludes those without NF1, under chemotherapy, with epilepsy, certain medical devices, using specific drugs like MEKINIST within the last month, or with severe psychiatric disorders.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive N-acetylcysteine (NAC) or placebo for 8 weeks to assess safety, tolerability, and efficacy on motor behavior and neurocognitive symptoms
Washout
Participants undergo a 4-week washout period after treatment to assess the persistence of NAC effects
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- N-Acetyl cysteine
- Placebo
N-Acetyl cysteine is already approved in United States, European Union for the following indications:
- Antidote for acetaminophen overdose
- Mucolytic agent
- Antidote for paracetamol overdose
- Mucolytic agent
Find a Clinic Near You
Who Is Running the Clinical Trial?
Children's Hospital Medical Center, Cincinnati
Lead Sponsor
United States Department of Defense
Collaborator