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A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease

Study acronym: Lafora
StatusRecruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗

Record History

  1. Minor Re-verified, no change to tracked fieldsv4
  2. Minor Start date changed from estimate of Dec 1, 2024 to Dec 3, 2024v3
  3. Notable Start date: Oct 1, 2024 to Dec 1, 2024v2
  4. Minor Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.
Trial Details
NCT Number NCT06609889
Lead Sponsor Berge Minassian
Collaborators: Elpida Therapeutics SPC
Conditions
(from sponsor)
Lafora Disease
Related terms Lafora Disease
Enrollment 10 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov