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Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1

NCT04481048 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 58

Last updated 2024-10-09

No results posted yet for this study

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 this project, the investigators will assess the safety and clinical benefit of N-acetylcysteine (NAC) as a pharmacological intervention in children with NF1. This drug choice is based on the recent findings from mouse models to study the central nervous system manifestations of NF1 at Cincinnati Children's Hospital Medical Center (CCHMC). These findings revealed a role for myelin-forming oligodendrocytes in the control of nitric oxide synthases (NOS) and their product, nitric oxide (NO), in maintenance of brain structure and function, including regulation of behavior and motor control. Treating these mice with NAC corrected cellular and behavioral abnormalities. This data from animal models of NF1 along with uncontrolled clinical observations in children with NF1 suggest that the antioxidant compound, NAC, may reduce these impairments. Therefore, the investigators propose performing a single center double-blind placebo controlled, prospective, Phase II study to explore safety, tolerability, and efficacy of NAC on motor behavior and/or learning in children with NF1 aged 8 through 16 years old. Participants will be carefully monitored for side effects. Primary and secondary outcome measures will be administered at baseline, follow-up, and post-treatment.

Conditions

  • Neurofibromatosis 1

Interventions

DRUG

N-Acetyl cysteine

Eight (8) weeks of treatment with an FDA approved medication, N-acetylcysteine (NAC).

OTHER

Placebo

Eight (8) weeks of treatment with placebo.

Sponsors & Collaborators

  • United States Department of Defense

    collaborator FED

  • Children's Hospital Medical Center, Cincinnati

    lead OTHER

Principal Investigators

  • Donald Gilbert, MD MS · Children's Hospital Medical Center, Cincinnati

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
8 Years
Max Age
16 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2020-12-15
Primary Completion
2024-06-25
Completion
2025-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04481048 on ClinicalTrials.gov