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Antioxidant

Antioxidant Therapy with N-acetylcysteine for Neurofibromatosis Type 1 (NF1NAC Trial)

Phase 2

Waitlist Available

Led By Carlos E Prada, MD

Research Sponsored by Children's Hospital Medical Center, Cincinnati

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at baseline and end of 8 weeks treatment with either nac or placebo (weeks 0 and 8 for treatment phase one of this cross-over double blind study and at weeks 10 and 18 for treatment phase two).

Awards & highlights

NF1NAC Trial Summary

This trial will study the effects of N-Acetyl Cysteine on children with neurofibromatosis type 1 who commonly suffer from cognitive, behavioral, and motor impairments with the goal of reducing these impairments.

Who is the study for?

This trial is for children aged 8-16 with Neurofibromatosis type 1 (NF1) and an IQ of 70 or above. They must not be on chemotherapy, have active brain lesions, epilepsy, or use certain medications like antidepressants. Children with asthma or at high risk for GI bleeding are excluded.Check my eligibility

What is being tested?

The study tests if the antioxidant N-acetylcysteine (NAC) can improve learning and motor behavior in kids with NF1 compared to a placebo. It's a double-blind study, meaning neither participants nor researchers know who gets NAC or placebo.See study design

What are the potential side effects?

While generally considered safe as it's available over-the-counter, NAC may cause infrequent bronchospasm in people with asthma and has potential risks when used as a mucolytic agent.

NF1NAC Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at baseline and end of 8 weeks treatment with either nac or placebo (weeks 0 and 8 for treatment phase one of this cross-over double blind study and at weeks 10 and 18 for treatment phase two).

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at baseline and end of 8 weeks treatment with either nac or placebo (weeks 0 and 8 for treatment phase one of this cross-over double blind study and at weeks 10 and 18 for treatment phase two).

This trial's timeline: 3 weeks for screening, Varies for treatment, and at baseline and end of 8 weeks treatment with either nac or placebo (weeks 0 and 8 for treatment phase one of this cross-over double blind study and at weeks 10 and 18 for treatment phase two). for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change From Baseline in ADHD Symptoms as Reported Via Parent/Teacher Surveys

Change From Baseline in Motor Function Measured by Physical and Neurological Examination for Subtle Signs (PANESS)

Secondary outcome measures

Tumor Markers

Transcranial Magnetic Stimulation (TMS) - Cortical Silent Period

Other outcome measures

Evaluation of Change From Baseline in Metabolomics Profiles as a Possible Disease Biomarker Metabolic Testing Biomarkers - Samples in Storage for Follow up Study

Side effects data

From 2014 Phase 3 trial • 264 Patients • NCT00650091

17%

Cough

14%

Dyspnea

13%

Upper respiratory tract infection

13%

Nausea

12%

Idiopathic Pulmonary Fibrosis

10%

Fatigue

10%

Idiopathic pulmonary fibrosis

Diarrhoea

Bronchitis

Dizziness

Sinusitis

Nasopharyngitis

Pneumonia

Headache

Atrial Fibrillation

Constipation

Acute Myocardial infarction

Oedema peripheral

Arthralgia

Vomiting

Adverse Drug Reaction

pneumothorax

Epistaxis

Dyspnoea

Lung Squamous Cell Carcinoma

Death

prostatitis

Dehydration

Intersticial Lung Disease

Prostate Cancer

syncope

Drug Fever

Exacerbation of Idiopathic Pulmonary Fibrosis

Atelectasis

Respiratory Tract Infection

100%

80%

60%

40%

20%

Study treatment Arm

Initial Study: Pred/AZA/NAC

N-Acetylcysteine

Placebo

Initial Study: Placebo

NF1NAC Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: N-AcetylcysteineExperimental Treatment1 Intervention

Participants will be dosed with 70 mg/kg/dose (max dose 900 mg) three times per day of N-Acetylcysteine (NAC) for eight (8) weeks. This is a double-blind study, neither study participant nor study team members will know whether the participant is given study drug or placebo until after all data is collected.

Group II: PlaceboPlacebo Group1 Intervention

Participants will be dosed three times per day with a placebo for eight (8) weeks. This is a double-blind study, neither study participant nor study team members will know whether the participant is given study drug or placebo until after all data is collected.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

N-acetylcysteine (NAC)

1998

Completed Phase 3

~780

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Children's Hospital Medical Center, CincinnatiLead Sponsor

815 Previous Clinical Trials

6,531,591 Total Patients Enrolled

Carlos E Prada, MDPrincipal InvestigatorChildren's Hospital Medical Center, Cincinnati

2 Previous Clinical Trials

144 Total Patients Enrolled

Donald L Gilbert, MD, MSPrincipal InvestigatorChildren's Hospital Medical Center, Cincinnati

Media Library

N-acetylcysteine (NAC) (Antioxidant) Clinical Trial Eligibility Overview. Trial Name: NCT04481035 — Phase 2

Neurofibromatosis Clinical Trial 2023: N-acetylcysteine (NAC) Highlights & Side Effects. Trial Name: NCT04481035 — Phase 2

N-acetylcysteine (NAC) (Antioxidant) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04481035 — Phase 2

Neurofibromatosis Research Study Groups: N-Acetylcysteine, Placebo

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Do the participants of this research have to be a certain age?

"According to the eligibility requirements for this clinical trial, individuals between 8 and 16 years old may apply. Out of the total 150 clinical trials, 60 are for patients under 18 and 90 are for those over 65."

Answered by AI

How many people have been recruited for this experiment?

"Recruitment for this particular clinical trial wrapped up on July 18th, 2022. If you're interested in other research studies, there are currently 56 trials actively enrolling patients with watson syndrome and 83 N-acetylcysteine (NAC) studies seeking participants."

Answered by AI

Is this study still recruiting new participants?

"The listing for this trial on clinicaltrials.gov suggests that it is not presently looking for new subjects. Although the study's last update was on July 18th, 2022, recruitment may start again in the future. There are currently 139 other trials searching for participants."

Answered by AI

What are the goals that this research is hoping to achieve?

"The primary outcome of this trial, which will be evaluated over roughly through 18 weeks (at weeks 0, 8, 10, and 18) is to Change from Baseline in ADHD Symptoms as Reported via Parent/Teacher Surveys. Secondary outcomes include Transcranial magnetic stimulation (TMS) - Cortical Silent Period which is defined as Transcranial Magnetic Stimulation (TMS) - Cortical Silent Period (CSP). This measure describes the function and physiology of the motor system using Transcranial Magnetic Stimulation (TMS) over the brain to evoke a muscle twitch in the hand. These evoked"

Answered by AI

N-acetylcysteine -- is this drug sanctioned by the FDA?

"N-acetylcysteine (NAC) has been studied in Phase 2 trials, meaning that while there is evidence suggesting it is safe, none of the data collected thus far indicates that it is an effective medication."

Answered by AI

What is N-acetylcysteine (NAC)'s most popular application?

"N-acetylcysteine (NAC) has a variety of applications in treating everything from asthma to corneal ulceration."

Answered by AI

Are there any restrictions for volunteers in this clinical trial?

"Eligibility requirements for this clinical trial state that patients must have watson syndrome and be between 8 to 16 years old. They are looking to enroll around 5 young participants."

Answered by AI

N-acetylcysteine (NAC) has been studied before, correct?

"83 clinical studies are currently underway to research N-acetylcysteine (NAC), with 19 of them in the critical third phase. The majority of these trials taking place in Lopburi and Songkhla, but there are 316 total locations conducting N-acetylcysteine (NAC) studies."

Answered by AI

Recent research and studies

CellJournal

The NF1 Locus Encodes a Protein Functionally Related to Mammalian GAP and Yeast IRA Proteins

Ballester, Roymarie, Douglas Marchuk, Mark Boguski, Ann Saulino, Roxanne Letcher, Michael Wigler, and Francis Collins. 1990. “The NF1 Locus Encodes a Protein Functionally Related to Mammalian GAP and Yeast IRA Proteins”. Cell. Elsevier BV. doi:10.1016/0092-8674(90)90151-4.

Journal of Affective DisordersJournal

The Efficacy of N-acetylcysteine as an Adjunctive Treatment in Bipolar Depression: An Open Label Trial

Berk, Michael, Olivia Dean, Sue M. Cotton, Clarissa S. Gama, Flavio Kapczinski, Brisa S. Fernandes, Kristy Kohlmann, et al.. 2011. “The Efficacy of N-acetylcysteine as an Adjunctive Treatment in Bipolar Depression: An Open Label Trial”. Journal of Affective Disorders. Elsevier BV. doi:10.1016/j.jad.2011.06.005.

New England Journal of MedicineJournal

Fluvoxamine for the Treatment of Anxiety Disorders in Children and Adolescents

Walkup, John T., Michael J. Labellarte, Mark A. Riddle, Daniel S. Pine, Laurence Greenhill, Rachel Klein, Mark Davies, et al.. 2001. “Fluvoxamine for the Treatment of Anxiety Disorders in Children and Adolescents”. New England Journal of Medicine. Massachusetts Medical Society. doi:10.1056/nejm200104263441703.

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