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Mucolytic Agent

N-acetylcysteine for Gaucher Disease

Phase 2

Recruiting

Led By Reena V. Kartha, PharmD

Research Sponsored by University of Minnesota

Eligibility Criteria Checklist

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

Must have

GD1 patients must be on a stable, specific ERT and/or SRT therapy at a specific dose for at least 2 years.

All participants must be 18 years or older.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, 45 days, 90 days, 120 days, 150 days, 180 days, and 270 days

Awards & highlights

Study Summary

This triallooks at how a disease (GD1) affects levels of blood & brain chemicals related to oxidative stress & inflammation in healthy & affected people.

Who is the study for?

This study is for adults over 18 with stable Type 1 Gaucher disease (GD1) who have been on consistent enzyme replacement or substrate reduction therapy for at least two years. Healthy volunteers matched by age can also join. Participants must not be pregnant, lactating, using certain antioxidants, or have conditions like asthma being treated currently.Check my eligibility

What is being tested?

The trial is investigating the effect of N-acetylcysteine on oxidative stress and inflammation in individuals with GD1 compared to healthy volunteers. It measures blood and brain chemicals to see if levels are different due to GD1.See study design

What are the potential side effects?

Potential side effects from N-acetylcysteine may include allergic reactions for those sensitive to it; however, specific side effects related to this trial are not detailed.

Eligibility Criteria

Inclusion Criteria

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

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I have been on the same enzyme or substrate replacement therapy for at least 2 years.

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I am 18 years old or older.

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I haven't taken coenzyme Q-10, vitamin C, or E for the last 3 weeks.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, 45 days, 90 days, 120 days, 150 days, 180 days, and 270 days

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, 45 days, 90 days, 120 days, 150 days, 180 days, and 270 days

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, 45 days, 90 days, 120 days, 150 days, 180 days, and 270 days 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 in subjects with Gaucher disease type 1, in concentration of glutathione in brain (μmol/g)

Secondary outcome measures

Change in healthy volunteers, in concentration of TNF-alpha in plasma (pg/mL)

Change in healthy volunteers, in concentration of glutathione in blood (μmol/g)

Change in subjects with Gaucher disease type 1, in concentration of N-acetylcysteine (NAC) in blood (µg/ml)

+5 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: N-acetylcysteineExperimental Treatment1 Intervention

The first 10 GD1 subjects will take 1800mg NAC twice daily (3600mg/day) orally for approximately 90 days. An interim analysis will be performed to determine if this dose produces changes in systemic redox status and brain glutathione (GSH) levels. If no signal of a significant change is observed, the remaining 20 subjects will receive up to 3600 mg NAC orally twice a day (7200 mg/day).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

N-acetylcysteine

2008

Completed Phase 4

~1290

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

National Center for Advancing Translational Science (NCATS)NIH

99 Previous Clinical Trials

36,259 Total Patients Enrolled

1 Trials studying Gaucher Disease

9 Patients Enrolled for Gaucher Disease

University of MinnesotaLead Sponsor

1,378 Previous Clinical Trials

1,588,459 Total Patients Enrolled

5 Trials studying Gaucher Disease

135 Patients Enrolled for Gaucher Disease

Rare Diseases Clinical Research NetworkNETWORK

67 Previous Clinical Trials

19,055 Total Patients Enrolled

1 Trials studying Gaucher Disease

9 Patients Enrolled for Gaucher Disease

Media Library

N-acetylcysteine (Mucolytic Agent) Clinical Trial Eligibility Overview. Trial Name: NCT02583672 — Phase 2

Gaucher Disease Research Study Groups: N-acetylcysteine

Gaucher Disease Clinical Trial 2023: N-acetylcysteine Highlights & Side Effects. Trial Name: NCT02583672 — Phase 2

N-acetylcysteine (Mucolytic Agent) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02583672 — Phase 2

Frequently Asked Questions

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

What diseases has N-acetylcysteine been demonstrated to ameliorate?

"N-acetylcysteine has been indicated to treat corneal ulceration, acute rhinitis, and acetaminophen."

Answered by AI

Is there any risk associated with supplementing N-acetylcysteine?

"Although no efficacy data has yet been collected on N-acetylcysteine, the safety information available warrants a rating of 2."

Answered by AI

Have any other research endeavors focused on N-acetylcysteine?

"N-acetylcysteine has been researched since 2002 by the Department of Anaesthesiology. Since then, a total of 162 clinical trials have concluded, with 38 more ongoing today - many out of Minneapolis in Minnesota."

Answered by AI

Is there currently an open enrollment period for this experiment?

"Affirmative. Clinicaltrials.gov's database confirms this clinical trial is actively recruiting participants, which began on September 1st 2015 and was recently updated on December 15th 2022. The research team requires 50 individuals to be enrolled between 2 seperate locations."

Answered by AI

How many participants are enrolled in this clinical experiment?

"Affirmative. The information found on clinicaltrials.gov displays that this medical trial is actively searching for subjects, having first been posted to the website on September 1st 2015 and most recently updated on December 15th 2022. A total of 50 participants are being sought from 2 distinct research centres."

Answered by AI

Recent research and studies

The Journal of Clinical PharmacologyJournal

Repeated-dose Oral N-acetylcysteine in Parkinson's Disease: Pharmacokinetics and Effect on Brain Glutathione and Oxidative Stress

Coles, Lisa D., Paul J. Tuite, Gülin Öz, Usha R. Mishra, Reena V. Kartha, Kathleen M. Sullivan, James C. Cloyd, and Melissa Terpstra. 2017. “Repeated-dose Oral N-acetylcysteine in Parkinson's Disease: Pharmacokinetics and Effect on Brain Glutathione and Oxidative Stress”. The Journal of Clinical Pharmacology. Wiley. doi:10.1002/jcph.1008.

Clinical NeuropharmacologyJournal

N-acetylcysteine Boosts Brain and Blood Glutathione in Gaucher and Parkinson Diseases

Holmay, Mary J., Melissa Terpstra, Lisa D. Coles, Usha Mishra, Matthew Ahlskog, Gülin Öz, James C. Cloyd, and Paul J. Tuite. 2013. “N-acetylcysteine Boosts Brain and Blood Glutathione in Gaucher and Parkinson Diseases”. Clinical Neuropharmacology. Ovid Technologies (Wolters Kluwer Health). doi:10.1097/wnf.0b013e31829ae713.

clinicaltrials.govStudy

Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)

2015. "Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02583672.

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