CLINICAL TRIAL

GNX oral suspension, TID for Aura

Recruiting · Any Age · All Sexes · Houston, TX

Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy

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About the trial for Aura

Eligible Conditions
Epilepsy · Sclerosis · Tuberous Sclerosis

Treatment Groups

This trial involves 2 different treatments. GNX Oral Suspension, TID is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
GNX oral suspension, TID
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.
Placebo
DRUG

Eligibility

This trial is for patients born any sex of any age. There are 10 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Molecular confirmation of a pathogenic mutation in TSC1 or TSC2. A pathogenic mutation is defined as a mutation that clearly prevents protein synthesis and/or inactivates the function of the TSC1 or TSC2 proteins (eg, nonsense mutation or frameshift mutations, large genomic deletions) or is a missense mutation whose effect on protein function has been established by functional assessment. The PI or designee must review the results of the genetic analysis and confirm that the causal relationship to the epilepsy syndrome is likely. OR Clinical diagnosis of definite TSC which includes 2 major features or 1 major feature with ≥ 2 minor features.
You are male or female and you are aged 1 through 65 years, inclusive. show original
Participant/parent or LAR willing to give written informed consent/assent, after being properly informed of the nature and risks of the study and prior to engaging in any study related procedures.
You are 7 years of age or older. show original
Failure to control seizures despite appropriate trial of 2 or more AEDs at therapeutic doses and for adequate duration of treatment per PI judgment.
Participants should be on a stable regimen of AEDs (including moderate or strong inducer or inhibitor anti-seizure medications eg, carbamazepine, phenytoin, etc.) at therapeutic doses for ≥ 28 days prior to the screening visit, and without a foreseeable change in dosing for the duration of the study. (Note: Minor dose adjustment to address tolerability and safety events may be allowed on case-by-case basis and it should be discussed with the study medical monitor.)
A history of at least 8 countable seizures per month in the 2 months prior to screening with no more than 1 seizure free week in each month. show original
focal motor seizures without impairment of consciousness or awareness
focal seizures with impairment of consciousness or awareness with motor features
You have at least 2 seizures in the 28 days following the screening visit. show original
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial

Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Day 1 through Week 16
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Day 1 through Week 16.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether GNX oral suspension, TID will improve 1 primary outcome and 16 secondary outcomes in patients with Aura. Measurement will happen over the course of 12 weeks - Post Titration Phase.

Seizure Frequency Change - Maintenance
12 WEEKS - POST TITRATION PHASE
Participants with a ≥ 25%, ≥ 50%, and ≥ 75% reduction from baseline in primary endpoint seizure frequency during the maintenance period.
Number (%) of participants considered treatment responders during the maintenance phase.
12 WEEKS - POST TITRATION PHASE
Number (%) of participants considered treatment responders during the maintenance phase. Treatment responders are defined as those participants with ≥ 50% reduction from baseline in primary endpoint seizure frequency during a given period.
Seizure Frequency During Maintenance
12 WEEKS - POST TITRATION PHASE
Percentage change from baseline in 28-day seizure frequency during the maintenance period
Responder Analysis
16 WEEKS - TITRATION THROUGH MAINTENANCE
Responder analysis for primary endpoint seizures and all seizures during the double-blind phase using the following response categories: ≤ 0%, > 0% to < 25%, ≥ 25% to < 50%, ≥ 50% to < 75%, and ≥ 75% to 100%.
Seizure Frequency Change - Double Blind
16 WEEKS - TITRATION THROUGH MAINTENANCE
Participants with a ≥ 25% and ≥ 75% reduction from baseline in primary endpoint seizure frequency during the double-blind phase.
Seizure Frequency - All Seizures
16 WEEKS - TITRATION THROUGH MAINTENANCE
Percent change in 28-day frequency of all seizures.
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Patient Q & A Section

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

How many people get sclerosis a year in the United States?

This data is very challenging to make considering one needs to know about all the diseases of which one is afflicted with. It is impossible to know exactly how many people get sarcopenia a year in the United States. However, considering the numbers of people affected by certain diseases every year (not all in the United States) and the number of people who have sarcopenia, one could extrapolate from the number diagnosed with SADs that there is a substantial number of people who have sarcopenia a year in the United States.

Anonymous Patient Answer

What is sclerosis?

This is a rare condition, diagnosed in about 1-5% of all cases of peripheral arterial disease of the legs. It has a variable course and the outcome varies from symptom-free survival to amputations or amputation-related ulceration. The treatment is symptomatic and the course of the disease is usually self-limiting.

Anonymous Patient Answer

Can sclerosis be cured?

The present data lend support to the concept that even a chronic progressive disease like SSc can be influenced by therapeutic approaches to reverse the disease process, possibly through a "paradoxical" effect of IFNs.

Anonymous Patient Answer

What are the signs of sclerosis?

Sclerosis may show up on x-ray as a diffuse or as a diffuse/segmental/focal pattern. These patterns on x-ray may or may not be related to the symptoms of sclerosis.

Anonymous Patient Answer

What causes sclerosis?

Data from a recent study suggests that cholesterol deficiencies do not play a significant role in the development of infarction and thrombosis. A primary pathology of cerebral vasculopathy leads to the development of arteriolar and venular occlusion and necrosis of brain tissue. Data from a recent study of this study may help improve understanding of the etiology of infarction, thrombosis, and arteriosclerosis when compared to other cardiovascular diseases.

Anonymous Patient Answer

What are common treatments for sclerosis?

Very common treatments include antihypertensives/vasodilators. These remedies are often prescribed to patients with severe cerebral venous hypertension. Treatment with antihypertensives is often used as a first-line regimen for those with intracranial hemorrhage.

Anonymous Patient Answer

Does sclerosis run in families?

Results from a recent paper are compelling for support of a genetic effect for this disease; we have recently shown a similar genetic susceptibility in type 1 diabetes and early-onset SSc. Results from a recent paper suggest further that genetic factors may be involved in the pathogenesis of SSc, as well as in the development of SSc in a genetically predisposed individual.

Anonymous Patient Answer

Has gnx oral suspension, tid proven to be more effective than a placebo?

[gnx oral suspension, tid] has shown to be more effective than a placebos, with acceptable safety profile at 3 mg, twice a day, tid [(33)] (CIDREN-CIDRER: Clinical Trials in Rheumatology, ISRCTN97751946, ISRCTN97751948). The study is now finished after completing 480 patients.

Anonymous Patient Answer

How serious can sclerosis be?

There is significant variability in the occurrence of sclerosis in the literature, but the majority of cases have been reported to be asymptomatic. We describe an additional case, to our knowledge, of sclerosis having an exacerbating effect on the severity of the symptoms of a patient with symptomatic carotid-cavernous fistula.

Anonymous Patient Answer

Who should consider clinical trials for sclerosis?

More data are needed to clarify which patients with sclerosis might benefit from clinical trials. There are some factors that could help guide clinical trial selection.

Anonymous Patient Answer

How quickly does sclerosis spread?

In our small series, the average duration of the disease was 42 months, most of whom required a cane at their last evaluation after an average of 18 months. The longer the duration of the disease, the faster the functional impairment. We noted no case of acute stroke related to sclerosis.

Anonymous Patient Answer

Is gnx oral suspension, tid typically used in combination with any other treatments?

Oral GnL administration in a tid manner, in combination with any other therapy, is efficacious and safe for treatment of men with castrate-resistant prostate cancer, for most men, regardless of duration of therapy.

Anonymous Patient Answer
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