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Tests treatment safety and results for Lafora Disease
Official title A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease
ClinicalTrials.gov ID: NCT06609889
What this study is testing
What is ION283?
ION283 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for lafora disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 10 to 18
You may be able to join if
- General Must give written informed consent (and assent if indicated by patient's age and in accordance with local requirements) and be willing/able...
- Aged 10-18 (inclusive) years old at the time of informed consent.
- Non-pregnant and non-lactating females
- All male participants and women of childbearing potential must refrain from sperm/egg donation from the time of signing the informed consent/assent...
- For participants engaged in sexual relations of childbearing potential, highly effective contraception must be used from the time of signing the...
You likely can't join if
- Clinically significant abnormalities in medical history (e.g., previous stroke within 6 months of Screening, major surgery within 3 months of...
- Platelet count \< 80,000/mm3 or any other clinically significant laboratory abnormalities that would render a patient unsuitable for inclusion.
- History of bleeding diathesis or coagulopathy
- Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to Study Day 1
- Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the...
- Contraindication or unwillingness to undergo lumbar puncture
See the full eligibility criteria
- General Must give written informed consent (and assent if indicated by patient's age and in accordance with local requirements) and be willing/able to comply with all study requirements.
- Aged 10-18 (inclusive) years old at the time of informed consent.
- Non-pregnant and non-lactating females
- All male participants and women of childbearing potential must refrain from sperm/egg donation from the time of signing the informed consent/assent form until at least 12 weeks (approximately 5 half-lives of ION283)...
- For participants engaged in sexual relations of childbearing potential, highly effective contraception must be used from the time of signing the informed consent/assent form until at least 12 weeks (approximately 5...
- Must have LDPS score ≥ 9 and LDPS motor subscore of ≥ 2 (independent ambulation- walking 10 steps independently)
- Clinically significant abnormalities in medical history (e.g., previous stroke within 6 months of Screening, major surgery within 3 months of Screening) or physical examination
- Platelet count \< 80,000/mm3 or any other clinically significant laboratory abnormalities that would render a patient unsuitable for inclusion.
- History of bleeding diathesis or coagulopathy
- Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to Study Day 1
- Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the Investigator
- Contraindication or unwillingness to undergo lumbar puncture
- Known history of, or positive test for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B
- Moderate-to-severe hepatic impairment or renal impairment.
- Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix that has been successfully treated or benign pediatric tumors. Patients with a history of other...
- Uncontrolled hypertension defined as: for patients \< 13 years old, BP ≥ 95th percentile + 12 mmHg, or ≥ 140/90 mmHg, whichever is lower for patients ≥ 13 years old, BP ≥ 140/90 mmHg
- Previous treatment with an oligonucleotide (including small interfering ribonucleic acid [siRNA]) within 4 months of Screening if single dose received, or within 12 months of Screening if multiple doses received; or...
- History of alcohol or drug abuse within 12 months of Screening, or current drug or alcohol abuse
- Has enrolled in any clinical trial or used any investigational agent or device, or has participated in any investigational procedure, within the 30 days, or within 5 half-lives of investigational agent, whichever is...
- Use of antiplatelet or anticoagulant therapy within the 14 days prior to Screening (with the exception of aspirin ≤ mg/day) or anticipated use during the study, including but not limited to clopidogrel, dipyridamole...
The study team makes the final eligibility decision.
Where it's taking place
- Dallas, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 10 years to 18 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Dallas, Texas, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.