Status:
ACTIVE_NOT_RECRUITING
Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1
Lead Sponsor:
Children's Hospital Medical Center, Cincinnati
Collaborating Sponsors:
United States Department of Defense
Conditions:
Neurofibromatosis 1
Eligibility:
All Genders
8-16 years
Phase:
PHASE2
Brief 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 ...
Detailed Description
This is a phase II clinical trial with the goal to explore safety, tolerability, and efficacy of NAC on motor behavior in children with NF1 aged 8 through 16 years old. The investigators hypothesize t...
Eligibility Criteria
Inclusion
- Inclusion Criteria ѱ:
- You can be in this study if you have any of the following:
- Males and females older than 8 years and younger than 16 years old
- Has a diagnosis of NF1 (neurofibromatosis type 1)
- Has an abnormal PANESS score
- Has an IQ (intelligence quotient) at or above 70
- 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:
- Younger than 8 years or older than 16 years ѱ
- Do not have a diagnosis of NF1 ѱ
- IQ below 70 ѱ
- Had a dose change of any stimulant or psychotropic medication in the last month (30 days) ѱ
- Are being treated with chemotherapy or had chemotherapy in the last 6 months
- Have epilepsy ѱ
- High risk of upper gastrointestinal (GI, the stomach and the small and large intestine) hemorrhage (bleeding). Examples: presence of esophageal varices or peptic ulcers
- Active intracranial lesions (abnormality found on brain imaging such as an MRI) (stable low-grade glioma is acceptable) or epilepsy diagnosis ѱ
- Have Major Depression, Bipolar Disorder, Conduct Disorder, Adjustment Disorder, other major Anxiety Disorders, or other developmental psychiatric diagnoses, based on history. ADHD is OK
- For females, pregnancy
- Is currently using antidepressants, dopamine blocking agents, or mood stabilizers
- Have any of the following medical devices: implanted brain stimulator, vagal nerve stimulator, VP (ventriculoperitoneal) shunt, cardiac pacemaker, or implanted medication port ѱ
- Asthma (bronchospasm has been reported as occurring infrequently and unpredictably when NAC is used as a mucolytic agent)
- 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).
Exclusion
Key Trial Info
Start Date :
December 15 2020
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2025
Estimated Enrollment :
58 Patients enrolled
Trial Details
Trial ID
NCT04481048
Start Date
December 15 2020
End Date
December 1 2025
Last Update
October 9 2024
Active Locations (1)
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1
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229