Study of ION373 Given by Spinal Injection for Patients with Alexander Disease

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What is this study about?

This study focuses on patients with Alexander Disease, a rare genetic condition that affects the nervous system. The research evaluates a new medication called ION373, which is given through an injection into the spinal fluid. The medication is designed to target a specific protein that is involved in the disease process.

The study tests how well ION373 works in improving or stabilizing movement abilities in people with Alexander Disease. Some participants will receive the actual medication, while others will receive a placebo. The medication or placebo will be given through injections into the space around the spinal cord, known as intrathecal administration.

During the study, participants will receive the treatment for 61 weeks. The researchers will monitor how well participants can walk and move, along with other symptoms related to Alexander Disease. They will also check the safety of the treatment and measure certain substances in the spinal fluid to understand how the medication works in the body.

1 Initial treatment period

You will receive either ION373 or a placebo through intrathecal injection (delivered directly into the spinal fluid)

This phase will last for 61 weeks (approximately 14 months)

Regular visits will be scheduled to monitor your progress and conduct assessments

2 Medical assessments

If you are 5 years or older, you will complete a 10-meter walking test to assess your movement ability

If you are under 5 years old, your gross motor function will be evaluated using specific movement tests

If you are 8 years or older, you will complete a 9-hole peg test to assess hand function

Your doctor will assess your overall condition and any changes in symptoms throughout the study

3 Additional evaluations

Regular assessment of your most bothersome symptoms

Completion of quality of life questionnaires

Monitoring of body weight

Collection of spinal fluid samples to measure GFAP levels (a protein associated with Alexander Disease)

4 Safety monitoring

Regular medical check-ups throughout the study period

If you are of reproductive age, pregnancy tests will be conducted every 12 weeks

Monitoring for any side effects or changes in your condition

5 Study completion

Final evaluations will be conducted at week 61

Assessment of changes in your condition compared to the start of the study

Review of all collected data and measurements

Who Can Join the Study?

Patient must provide written informed consent and be able to follow all study requirements
Age requirements:
– For main study: between 2 and 65 years old
– For open-label sub-study: less than 2 years old
Must have confirmed genetic mutation in the GFAP gene
Must have brain imaging results consistent with Alexander Disease
Physical ability requirements:
– Ages 2-5 years: must be able to sit with minimal support for at least 10 seconds or walk (with or without assistance) completing a 10-meter walk test within 5 minutes
– Ages 5 and above: must be able to walk (with or without assistance)
Must be on stable medications, nutrition plan, and therapy programs (physical, occupational, speech, respiratory) for at least 3 months before screening
For patients under 18: must have a trial partner (parent or caregiver) who is:
– At least 18 years old
– Available to accompany patient to visits
– Knowledgeable about patient’s condition
For females of reproductive age:
– Must not be pregnant or breastfeeding
– Must use effective contraception or be surgically sterile
– Regular pregnancy testing will be required
For males who can father children:
– Must use effective contraception or be surgically sterile
– Must continue contraception for 40 weeks after last dose

Who Cannot Join the Study?

Patients who do not have a confirmed genetic diagnosis of Alexander Disease
Individuals who have participated in another clinical trial within the past 30 days
Patients with severe allergic reactions to similar medications
Those with significant liver or kidney problems that could affect drug metabolism
Pregnant women or those planning to become pregnant during the study period
Patients who are currently receiving other experimental treatments
Individuals who have undergone major surgery in the past 3 months
Patients with uncontrolled medical conditions that could interfere with the study
Those unable to comply with study procedures or follow-up visits
Patients taking medications that could interact with the study drug ION373
Individuals with severe psychiatric conditions that could impact their participation
Those who cannot provide informed consent or have legal guardians who cannot provide consent

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country
ASST Fatebenefratelli Sacco	Milan	Italy
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Amxfwjkgn Uup	Amsterdam	The Netherlands

Trial status

Country	Status	Recruitment Start
Italy	Not recruiting	17.11.2022
The Netherlands	Not recruiting	17.11.2022

Trial locations

Investigated drugs:

2'-O-(2-Methoxyethyl)-D-Ribose Antisense Oligonucleotide Targeting Glial Fibrillary Acidic Protein Messenger Ribonucleic Acid

ION373 is an experimental medication that is administered through an intrathecal injection (directly into the spinal fluid). This medication is being studied for the treatment of Alexander disease, a rare genetic condition that affects the nervous system. The treatment is designed to target the underlying cause of Alexander disease with the goal of improving or stabilizing patients’ ability to move and control their muscles.

Investigated diseases:

Alexander disease

Alexander Disease – A rare genetic disorder that primarily affects the brain and spinal cord due to mutations in the GFAP gene. The disease causes the destruction of white matter in the brain, which affects transmission of nerve signals. It leads to the abnormal formation of protein deposits called Rosenthal fibers in specialized brain cells known as astrocytes. The condition typically causes progressive problems with movement, speech, and swallowing. Alexander Disease can develop at any age, with symptoms varying depending on when the disease first appears. The disease affects various functions of the nervous system, including coordination, mental development, and autonomic functions.

Trial ID:

2024-510603-11-00

Protocol code:

ION373-CS1

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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Study of ION373 Given by Spinal Injection for Patients with Alexander Disease

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?