ION373 for Alexander Disease

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
IONIS Investigative Site, Boston, MA
Alexander Disease
ION373 - Drug
Eligibility
Any Age
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a drug may help improve motor function in children with cerebral palsy.

See full description

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Other trials for Alexander Disease

Study Objectives

This trial is evaluating whether ION373 will improve 1 primary outcome and 16 secondary outcomes in patients with Alexander Disease. Measurement will happen over the course of Baseline and Week 61.

Baseline and Week 61
Change From Baseline in Alexander Disease Patient Domain Impression of Change (AxD-PDIC) Score
Change From Baseline in Alexander Disease Patient Domain Impression of Severity (AxD-PDIS) Score
Change From Baseline in Body Weight Percentile (for participants < 18 years old at screening) or body weight (for participants ≥ 18 years old at screening)
Change From Baseline in Cerebrospinal Fluid (CSF) Glial Fibrillary Acid Protein (GFAP) Levels
Change From Baseline in Clinical Global Impression of Change (CGIC) Score
Change From Baseline in Clinical Global Impression of Severity (CGIS) Score
Change From Baseline in Composite Autonomic Symptom Score 31 (COMPASS-31) Score
Change From Baseline in Gross Motor Function Measure-88, Dimensions C, D and E (GMFM-88, Dimensions C-E) Score
Change From Baseline in Most Bothersome Symptom (MBS)
Change From Baseline in Patient Global Impression of Change (PGIC) Score
Change From Baseline in Patient Global Impression of Severity (PGIS) Score
Change From Baseline in Pediatrics Quality of Life Inventory (PedsQL) Generic Core Scales Score
Percent change from Baseline in the 10-Meter Walk Test (10MWT)
Baseline to Week 61
Change From Baseline in 9-Hole Peg Test (9HPT) Score
Change From Baseline in Pediatrics Quality of Life Inventory Gastrointestinal Symptoms Scale (PedsQL GI) Score
Change From Baseline in Vineland Adaptive Behavior Composite, Third Edition (Vineland-3 ABC) Score
Change From Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Motor Skills Domain Score

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Other trials for Alexander Disease

Trial Design

2 Treatment Groups

ION373
1 of 2
Placebo
1 of 2
Experimental Treatment
Non-Treatment Group

This trial requires 58 total participants across 2 different treatment groups

This trial involves 2 different treatments. ION373 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.

ION373
Drug
ION373 will be administered by intrathecal bolus (ITB) injection once every 12 weeks through Week 109.
Placebo
Drug
Matching placebo will be administered by ITB injection once every 12 weeks through Week 109.

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline to week 61
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly baseline to week 61 for reporting.

Closest Location

IONIS Investigative Site - Boston, MA

Eligibility Criteria

This trial is for patients born any sex of any age. There are 6 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
to accompany them to all study visits Patients who are less than 18 years old at screening must have a trial partner to accompany them to all study visits. show original
The patient has a neurological disorder that is characterized by a clinical phenotype and brain imaging consistent with a diagnosis of Alexander disease. show original
is associated with Alexander disease A mutation in the GFAP gene is associated with Alexander disease, which is a documented genetic condition. show original
People who are at least 2 years old and no older than 65 can give consent. show original
Able and willing to meet all study requirements, including travel to Study Center, procedures, measurements and visits
Key

Patient Q&A Section

What is alexander disease?

"The name Alexander disease (AD) is used mostly for infants and young children presenting a progressive dystonia with bulbar function deficits. AD is not the common, long-cited variant of AD. The disease course is typically slowly progressive with poor response to therapy often requiring an early colectomy. A major question is whether the use of corticosteroids influences the short-term clinical evolution. Alexander disease can be diagnosed with magnetic resonance imaging but the only reliable way to confirm it is through genetic testing." - Anonymous Online Contributor

Unverified Answer

What are common treatments for alexander disease?

"Some treatments may include corticosteroids, baclofen, fludoclopirim and phenytoin. Other medications can be used for various complications. There is no cure for alexander disease. Treatment is also based on the specific patient and the severity of the disease." - Anonymous Online Contributor

Unverified Answer

What are the signs of alexander disease?

"Alexander disease results in loss of coordination or muscle control. Patients also exhibit an unsteady gait, incoordination and an awkward gait.\nPatients may also develop difficulties in swallowing and speak in a hoarse whisper. Alexander disease patients may also develop problems with vision, hearing and face deformity.\n" - Anonymous Online Contributor

Unverified Answer

What causes alexander disease?

"The presence of alexander disease on clinical grounds is highly suggestive of dengue infection. The diagnosis can only be confirmed by obtaining a sample for dengue serology and other laboratory tests." - Anonymous Online Contributor

Unverified Answer

How many people get alexander disease a year in the United States?

"About 36,000 people are diagnosed annually with alexander disease in the United States. Most cases arise at an early age and are clinically benign, whereas a small percentage progress to prostate cancer. The incidence of prostate cancer increases significantly from age 70 years and is a significant contributor to mortality." - Anonymous Online Contributor

Unverified Answer

Can alexander disease be cured?

"No cure can be found for alexander disease as it is caused by human herpes simplex virus 2. However, there are various treatments that are effective in controlling the disease and preventing recurrences. Nevertheless, it is still a progressive disease and will continue to keep deteriorating as more knowledge about the disease is gained." - Anonymous Online Contributor

Unverified Answer

What is ion373?

"Ion373, a lysosomal protein, is overexpressed in a subset of pediatric ependymomas, particularly those with an infiltrative phenotype. The function of ion373 and possible therapeutic implications will be the subject of further investigation." - Anonymous Online Contributor

Unverified Answer

What does ion373 usually treat?

"Many of the ion373 side effects are commonly experienced by the elderly. Furthermore, ion373 prescribing patterns are highly variable among patients. The most prevalent side effects are typically mild and can be managed by regular medications used by the elderly to treat their long-term illnesses. The ion373 prescribing patterns are usually consistent among patients, and ion373 is a relatively safe prescription from a hospital for patients, as well as those with various types of ailments. [Power(http://http://www." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating alexander disease?

"We have seen the most recent advances in disease prevention and treatment of alexander disease, but we need to find a cure for the disease. In addition, more research is necessary to find a cure for alexander disease. For now, we have seen the most recent advances in disease prevention and treatment of alexander disease, but we need to find a cure for the disease. For now, we have seen the most recent discoveries in patient treatment and disease prevention." - Anonymous Online Contributor

Unverified Answer

What is the latest research for alexander disease?

"The recent article by Ojada et al, (1997) illustrates that the presence of the same mutation in different individuals with SSc is common. It is thought to be a consequence of the complex genetic basis. When considering inheritance of SSc, it is vital to ascertain the specific underlying mutation and to obtain appropriate family members. The more we can identify the risk factors, the better we can tailor appropriate and effective treatment for our patients." - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving ion373?

"No randomized controlled trials of Ion373 were ever conducted. Data from a recent study presented in this article are a combination of published reports, observational studies, and unpublished abstracts of research conducted at IonAxon, which have been analysed by a qualified medical peer peer review group. These data do not include data from the IonAxon Clinical Trial Registry." - Anonymous Online Contributor

Unverified Answer

Who should consider clinical trials for alexander disease?

"Clinical trials for alexander disease are being considered but many patients do not consider clinical trials. Patient involvement in these trials is still a barrier despite the advent of clinical trials evaluating other diseases. Clinical trials are necessary and should be encouraged. There is no way to predict who will get alexander disease." - Anonymous Online Contributor

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