N-Acetyl cysteine for Neurofibromatosis

Phase-Based Progress Estimates
Cincinnati Children's Hospital Medical Center, Cincinnati, OHNeurofibromatosisN-Acetyl cysteine - Drug
8 - 16
All Sexes
What conditions do you have?

Study Summary

This trial will assess the safety and clinical benefit of N-acetylcysteine (NAC) as a treatment for children with neurofibromatosis type 1 (NF1).

Eligible Conditions
  • Neurofibromatosis Type 1

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 3 Secondary · Reporting Duration: through 12 weeks (at weeks 0, 8, and 12)

Week 12
Change from Baseline in ADHD Symptoms as Reported via Parent/Teacher Surveys
Change from Baseline in Microstructural Properties of Brain Tissue Visualized by Magnetic Resonance Imaging (MRI)
Change from Baseline in Motor Function Measured by Physical and Neurological Examination for Subtle Signs (PANESS)
Change from Baseline in Motor Function and Physiology Measured by Transcranial Magnetic Stimulation (TMS)

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Trial Design

2 Treatment Groups

N-Acetylcysteine (NAC)
1 of 2
1 of 2

Experimental Treatment

Non-Treatment Group

58 Total Participants · 2 Treatment Groups

Primary Treatment: N-Acetyl cysteine · Has Placebo Group · Phase 2

N-Acetylcysteine (NAC)
Experimental Group · 1 Intervention: N-Acetyl cysteine · Intervention Types: Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Other
First Studied
Drug Approval Stage
How many patients have taken this drug
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: through 12 weeks (at weeks 0, 8, and 12)

Who is running the clinical trial?

Children's Hospital Medical Center, CincinnatiLead Sponsor
765 Previous Clinical Trials
5,422,902 Total Patients Enrolled
United States Department of DefenseFED
792 Previous Clinical Trials
213,235 Total Patients Enrolled
1 Trials studying Neurofibromatosis
19 Patients Enrolled for Neurofibromatosis
Carlos E Prada, MDPrincipal InvestigatorChildren's Hospital Medical Center, Cincinnati
2 Previous Clinical Trials
91 Total Patients Enrolled
Donald Gilbert, MD MSPrincipal InvestigatorChildren's Hospital Medical Center, Cincinnati

Eligibility Criteria

Age 8 - 16 · All Participants · 5 Total Inclusion Criteria

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Frequently Asked Questions

Are there any available vacancies for this treatment program?

"This clinical trial is actively searching for participants. The trial was first posted on December 15th 2020 and was last updated on February 24th 2022." - Anonymous Online Contributor

Unverified Answer

What other research studies have there been on N-Acetyl cysteine?

"As of right now, 83 different clinical trials are studying the effects of N-Acetyl cysteine. Out of those 83 live studies, 19 are in Phase 3 testing. The majority of these studies are based out Lopburi and Songkhla in Thailand; however, there are 316 total locations running similar tests." - Anonymous Online Contributor

Unverified Answer

Are the patients in this clinical trial restricted to those over 20 years old?

"In order to participate in this particular clinical trial, children aged 8 to 16 years old are eligible. Out of the total 150 trials, 60 are for patients below 18 years of age and 90 are for those over 65." - Anonymous Online Contributor

Unverified Answer

What medical conditions is N-Acetyl cysteine used to treat?

"The common cold, acetaminophen, and other conditions can often be treated effectively with N-Acetyl cysteine. This medication is most well-known, however, for its asthma relief properties." - Anonymous Online Contributor

Unverified Answer

For which patient population is this research project intended?

"This trial is aiming to enroll 58 individuals that have watson syndrome. These participants will be between 8 and 16 years old. To meet the qualifications for this study, applicants must also satisfy the following conditions: Be male or female, no more than 16 years and 6 months old, have an IQ of 70 or above (this must be from a neurocognitive test administered in last 3 years or during enrollment), meet NIH criteria for NF1 diagnosis, abnormal PANESS score (this must be at or above mean based on age and sex), currently taking stimulant medication or any other psychotropic drug but with a stable 30-day dose before" - Anonymous Online Contributor

Unverified Answer

Has N-Acetyl cysteine received authorization from the FDA?

"N-Acetyl cysteine has not been proven effective yet, however, it did receive a score of 2 due to data collected that supports its safety profile." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.