ION440 for MECP2 Syndrome
Trial Summary
Will I have to stop taking my current medications?
The trial does not specify that you must stop taking your current medications. However, you need to be on stable doses of your current medications for at least 3 months before starting the trial, unless the investigator decides otherwise.
What is the purpose of this trial?
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
Eligibility Criteria
This trial is for children and adults with MECP2 Duplication Syndrome (MDS). Children must be aged 2-7, have a caregiver to consent and attend visits, while adults are eligible from ages 8-65. Participants need genetic confirmation of MDS and stable medication doses for at least 3 months.Inclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Multiple Ascending Dose (MAD)
Participants are randomized to receive ION440 or sham in a double-blind manner
Open-label Long-term Extension (LTE)
Participants receive ION440 for long-term evaluation
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- ION440
Find a Clinic Near You
Who Is Running the Clinical Trial?
Ionis Pharmaceuticals, Inc.
Lead Sponsor
Dr. Brett P. Monia
Ionis Pharmaceuticals, Inc.
Chief Executive Officer since 2020
PhD in Pharmacology from the University of Pennsylvania, BSc in Molecular Biology and Analytical Chemistry from Stockton State College
Dr. Eric Bastings
Ionis Pharmaceuticals, Inc.
Chief Medical Officer
MD