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NCT06609889 · ClinicalTrials.gov registry record · Phase 1
A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease
A Phase 1 study of Lafora Disease, sponsored by Berge Minassian.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 10
- Enrollment target
- 1
- Study location
NCT06609889: Recruiting Phase 1 study of Lafora Disease, sponsored by Berge Minassian.
NCT06609889 is a Phase 1 study of Lafora Disease that is actively recruiting participants, run by Berge Minassian. The registered enrollment target is 10 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06609889, a Phase 1 study of Lafora Disease, is actively recruiting participants, sponsored by Berge Minassian.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 10 participants
- Enrollment target
- 1
- Study location
Study Summary
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.
Primary Outcome
Safety of ION283 will be assessed by the number of participants with Treatment related Adverse Events (AEs) which will be listed according to the severity (mild, moderate, severe) as assessed by the Investigator. A dose limiting toxicity (DLT) is defined as any ≥ Grade 3 (severe, life-threatening, disabling, or fatal) AND related to the Study Drug. The occurrence of (i) DLTs in 2 patients following administration or (ii) a single serious adverse event (SAE) that is life threatening and related
Conditions Studied
Interventions
- GENETIC ION283
Study Locations (1)
Texas
- Childrens Health - Dallas
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2024-12-03 |
| Est. Completion | 2028-10 |
| Phase | Phase 1 |
What NCT06609889 shows while recruiting
NCT06609889 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower).
The record links to 1 condition, with Lafora Disease appearing as the primary indexed condition, and to 1 intervention - of which ION283 is the first listed.
NCT06609889 reports a single indexed study location in Texas.
Frequently Asked Questions
What is clinical trial NCT06609889 about?
NCT06609889 is a clinical study titled "A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease". 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.
What is the current status of trial NCT06609889?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2024-12-03. Estimated completion is 2028-10.
What conditions does trial NCT06609889 study?
This clinical trial studies the following conditions: Lafora Disease.
What interventions are being tested in trial NCT06609889?
The interventions under investigation include: ION283 (GENETIC).
Who is sponsoring clinical trial NCT06609889?
This trial is sponsored by Berge Minassian, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06609889 being conducted?
This trial has 1 study location across Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT06609889's enrollment target sits among peer trials
10 1913th of 2000 higher than 46 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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