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Glucosylceramide Synthase Inhibitor

Miglustat for Batten Disease

Phase 1 & 2
Waitlist Available
Research Sponsored by Beyond Batten Disease Foundation
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Male and female participants must use a highly effective method of contraception and must continue for the duration of the trial (and for 30 days after the end of treatment).
Have genetically confirmed diagnosis of syndromic CLN3 disease with specified mutation criteria
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 104 weeks
Awards & highlights

Study Summary

This trial will study the safety and effectiveness of a new drug for a rare disease called CLN3 disease. The trial will last 104 weeks, and will involve 6 subjects at 2 different centers.

Who is the study for?
This trial is for individuals aged 17 or older with genetically confirmed CLN3 disease, a form of Batten Disease. Participants must be able to perform study tasks and visit the clinic as needed. They should agree to use effective contraception during the trial and for a month after it ends.Check my eligibility
What is being tested?
The study tests Miglustat's safety, how it's processed in the body (pharmacokinetics), and effectiveness in treating CLN3 disease at doses from 100 mg once daily up to 200 mg three times daily over two years across two centers.See study design
What are the potential side effects?
Potential side effects of Miglustat may include stomach discomfort, diarrhea, weight loss, tremors, and numbness. These are based on known side effects but could vary among participants.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am using or willing to use effective birth control during and for 30 days after the trial.
Select...
My CLN3 disease is confirmed through genetic testing.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~104 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 104 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0
Secondary outcome measures
Clinical efficacy based on UBDRS score
Clinical efficacy based on Vineland score
Clinical efficacy with MRI
+3 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: Oral miglustatExperimental Treatment1 Intervention
The proposed dosing regimen is daily oral miglustat (MTD, up to 200 mg TID)

Find a Location

Who is running the clinical trial?

TheranexusIndustry Sponsor
4 Previous Clinical Trials
300 Total Patients Enrolled
Beyond Batten Disease FoundationLead Sponsor
1 Previous Clinical Trials
20,000 Total Patients Enrolled

Media Library

Miglustat (Glucosylceramide Synthase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05174039 — Phase 1 & 2
Batten Disease Research Study Groups: Oral miglustat
Batten Disease Clinical Trial 2023: Miglustat Highlights & Side Effects. Trial Name: NCT05174039 — Phase 1 & 2
Miglustat (Glucosylceramide Synthase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05174039 — Phase 1 & 2

Frequently Asked Questions

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

Have there been any other investigations involving Miglustat 100Mg Oral Capsule?

"Currently, there are 7 ongoing investigations into the efficacy of Miglustat 100Mg Oral Capsule. Of these studies, 5 are in a Phase 3 state. Primarily centred around Houston, Texas - 99 other medical centres across America also provide this medication to patients through clinical trials."

Answered by AI

Is this a pioneering attempt to investigate new treatments?

"Since its initial trial in 2016, sponsored by Amicus Therapeutics and involving 32 patients, Miglustat 100Mg Oral Capsule has been lab tested for approval. It is now accessible across 24 countries within 39 cities; with there being 7 active studies associated with the drug today."

Answered by AI

Is this medical experiment recruiting new participants?

"This medical examination has ceased recruiting patients. Initially posted on February 2nd 2022, and most recently revised on September 15th of the same year, this trial is no longer available to join. However, there are 6 other trials seeking participants with juvenile neuronal ceroid lipofuscinosis and 7 studies actively enrolling people for Miglustat 100Mg Oral Capsule treatment."

Answered by AI

How many volunteers are currently participating in this clinical experiment?

"Unfortunately, this medical trial is no longer accepting new patients. It was initially published on February 2nd 2022 and last amended on September 15th of the same year. However, there are 6 studies related to juvenile neuronal ceroid lipofuscinosis which are currently recruiting as well as 7 trials involving Miglustat 100Mg Oral Capsules in need of participants."

Answered by AI

Who else is applying?

What state do they live in?
Ohio
What site did they apply to?
Texas Children Hospital
What portion of applicants met pre-screening criteria?
Met criteria
How many prior treatments have patients received?
0

Why did patients apply to this trial?

Recent research and studies
clinicaltrials.govStudy
An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of CLN3 Disease
2022. "An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of CLN3 Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05174039.
~1 spots leftby Sep 2024
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