Actively Recruiting
A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease
Led by Berge Minassian · Updated on 2025-12-18
10
Participants Needed
1
Research Sites
199 weeks
Total Duration
On this page
Sponsors
B
Berge Minassian
Lead Sponsor
E
Elpida Therapeutics SPC
Collaborating Sponsor
AI-Summary
What this Trial Is About
This study will test the safety and efficacy of multiple doses of ION283 administered as intrathecal (IT) injections by lumbar puncture (LP). All subjects will receive ION283. The dose level of 15 mg will be studied in all subjects.
CONDITIONS
Official Title
A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Must give written informed consent (and assent if required) and be willing to follow all study requirements
- Age between 10 and 18 years old at consent
- Female participants must not be pregnant or breastfeeding
- All males and females of childbearing potential must avoid sperm or egg donation from consent until 12 weeks after dosing
- Participants with childbearing potential must use effective contraception from consent until 12 weeks after dosing
- Genetically confirmed diagnosis of Lafora disease with documented mutations in EPM2A or EPM2B genes
- LDPS score of at least 9 and LDPS motor subscore of at least 2, indicating ability to walk 10 steps independently
You will not qualify if you...
- Significant medical history issues like stroke within 6 months or major surgery within 3 months
- Platelet count below 80,000/mm3 or other serious lab abnormalities
- History of bleeding disorders or coagulopathy
- Active infection needing antiviral or antibiotic treatment not completed before study start
- Unwillingness to follow study procedures or cooperate with investigators
- Contraindication to or refusal of lumbar puncture
- Known HIV, hepatitis C, or chronic hepatitis B infection
- Moderate to severe liver or kidney impairment
- Cancer within past 5 years except certain treated skin or cervical cancers or approved benign tumors
- Uncontrolled high blood pressure as defined by age-specific criteria
- Previous treatment with oligonucleotide therapies within defined time frames or allergy to ION283 or similar drugs
- History of alcohol or drug abuse in past 12 months
- Participation in other clinical trials or investigational treatments within 30 days or longer depending on drug half-life
- Use of anticoagulant or antiplatelet drugs (except low-dose aspirin) within 14 days before screening or anticipated during study
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Childrens Health
Dallas, Texas, United States, 75235
Actively Recruiting
Research Team
K
Kristy Riddle, RN, BSN
CONTACT
B
Ben Eckert, BA
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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