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Antioxidant

Antioxidant Therapy for Neurofibromatosis Type 1 (DoDNAC Trial)

Phase 2

Recruiting

Led By Donald Gilbert, MD MS

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

Has a diagnosis of NF1 (neurofibromatosis type 1)

Has an IQ (intelligence quotient) at or above 70

Timeline

Screening 3 weeks

Treatment Varies

Follow Up through 12 weeks (at weeks 0, 8, and 12)

Awards & highlights

DoDNAC Trial Summary

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

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 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.Check my eligibility

What is being tested?

The study tests N-acetylcysteine (NAC), an antioxidant therapy against a placebo in children with NF1 to see if it improves cognitive and motor functions. This Phase II trial is double-blind and controlled; participants won't know which treatment they're getting to ensure unbiased results.See study design

What are the potential side effects?

While closely monitoring for side effects during the trial, potential risks may include allergic reactions due to NAC's mucolytic properties causing bronchospasm (tightening of airways), gastrointestinal bleeding in high-risk individuals, and other unspecified drug-related adverse events.

DoDNAC Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have been diagnosed with neurofibromatosis type 1.

Select...

My IQ is 70 or higher.

DoDNAC Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ through 12 weeks (at weeks 0, 8, and 12)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~through 12 weeks (at weeks 0, 8, and 12)

This trial's timeline: 3 weeks for screening, Varies for treatment, and through 12 weeks (at weeks 0, 8, and 12) 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 Motor Function Measured by Physical and Neurological Examination for Subtle Signs (PANESS)

Secondary outcome measures

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 and Physiology Measured by Transcranial Magnetic Stimulation (TMS)

Side effects data

From 2024 Phase 2 trial • 13 Patients • NCT03499249

62%

GGT 400 IU/L >pre-KP value

46%

AST 400 IU/L >pre-KP value

31%

Loss of an IV

23%

Emesis with feeds

15%

Conjugated bilirubin 3 mg/dL >pre-KP value

15%

Febrile illness

INR >2.0

Total bilirubin 5 mg/dL >pre-KP value

Tachycardia

ALT 700 IU/L >pre-KP value

100%

80%

60%

40%

20%

Study treatment Arm

N-Acetylcysteine Treatment

DoDNAC Trial Design

3Treatment groups

Experimental Treatment

Active Control

Placebo Group

Group I: N-Acetylcysteine (NAC)Experimental Treatment1 Intervention

Each subject will be dosed with approximately 70 mg/kg/day of NAC for 8 weeks. To facilitate drug compounding, three tiers of drug dose will be administered based on body weight as described in Table 3. Table 3: NAC Dosing Participant's weight (kg) Dose (BID) < 20 700 mg 21-39 1050 mg > 40 1350 mg *Max dose not to exceed 2700mg/day (1350mg BID)

Group II: Single Visit/Non-Treatment ArmActive Control1 Intervention

Based on preliminary data, an additional "Single visit, non-treatment" cohort will include 40 individuals with NF1 for a single "biomarker" study visit. These individuals will undergo motor function (PANESS) and brain-based measures (TMS, MRI-MRS, DTI) as biomarkers of impaired executive function (ADHD-RS; BRIEF-2; TOVA) but will not be assigned to receive NAC/Placebo.

Group III: PlaceboPlacebo Group1 Intervention

Each subject will be dosed with placebo for 8 weeks.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

N-Acetyl cysteine

2022

Completed Phase 4

~190

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Children's Hospital Medical Center, CincinnatiLead Sponsor

815 Previous Clinical Trials

6,531,533 Total Patients Enrolled

United States Department of DefenseFED

865 Previous Clinical Trials

327,641 Total Patients Enrolled

1 Trials studying Neurofibromatosis

19 Patients Enrolled for Neurofibromatosis

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

Media Library

N-Acetyl cysteine (Antioxidant) Clinical Trial Eligibility Overview. Trial Name: NCT04481048 — Phase 2

Neurofibromatosis Research Study Groups: Single Visit/Non-Treatment Arm, N-Acetylcysteine (NAC), Placebo

Neurofibromatosis Clinical Trial 2023: N-Acetyl cysteine Highlights & Side Effects. Trial Name: NCT04481048 — Phase 2

N-Acetyl cysteine (Antioxidant) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04481048 — Phase 2

Frequently Asked Questions

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

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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"

Answered by AI