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Tests treatment safety and results for Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Official title ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)
ClinicalTrials.gov ID: NCT06430385
What this study is testing
What is ION440?
ION440 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for methyl cpg binding protein 2 (mecp2) duplication syndrome.
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
- The primary purpose of this study is to evaluate the safety and tolerability of ION440.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 65, men only
You may be able to join if
- Key for Part 1:
- Males aged ≥ 2 to ≤ 65 years, depending on specific cohort and group, at the time of informed consent.
- Group A: ≥ 8 to ≤ 65 years old
- Group B: 2 to 7 years old, inclusive
- Participant has at least one parent or caregiver ≥ 18 years old capable of providing informed consent and able to comply with all study requirements...
You likely can't join if
- for Part 1:
- Documented diagnosis of severe MECP2 duplications including terminal duplication and/or translocation or MECP2 triplication OR clinical features...
- Clinically significant vital sign or ECG abnormality at Screening
- Known brain or spinal disease that would interfere with the LP procedure, or CSF circulation or presence of other factors would affect the safety of...
- Has any concomitant disease or condition or circumstance, or any finding at Screening that, in the opinion of the Investigator, makes the participant...
- Treatment with an investigational drug, biological agent, or device within 30 days of Screening, or 5 half-lives of investigational agent, whichever...
See the full eligibility criteria
- Key for Part 1:
- Males aged ≥ 2 to ≤ 65 years, depending on specific cohort and group, at the time of informed consent.
- Group A: ≥ 8 to ≤ 65 years old
- Group B: 2 to 7 years old, inclusive
- Participant has at least one parent or caregiver ≥ 18 years old capable of providing informed consent and able to comply with all study requirements and activities.
- Participant has a documented diagnosis of MDS with genetic confirmation of MECP2 duplication.
- Is currently receiving stable doses of concomitant medications for at least 1 month prior to screening.
- Able to complete all study procedures, measurements and visits to support primary and secondary endpoints, in the opinion of the Investigator. Key
- for Part 1:
- Documented diagnosis of severe MECP2 duplications including terminal duplication and/or translocation or MECP2 triplication OR clinical features associated with severe variant structure including (a) onset of seizures...
- Clinically significant vital sign or ECG abnormality at Screening
- Known brain or spinal disease that would interfere with the LP procedure, or CSF circulation or presence of other factors would affect the safety of the LP procedure.
- Has any concomitant disease or condition or circumstance, or any finding at Screening that, in the opinion of the Investigator, makes the participant unsuitable for enrollment or that could interfere with the conduct of...
- Treatment with an investigational drug, biological agent, or device within 30 days of Screening, or 5 half-lives of investigational agent, whichever is longer.
- Previous treatment with an oligonucleotide (including siRNA) within 4 months of Screening if single dose received, or within 12 months of Screening if multiple doses received (this exclusion does not apply to vaccines -...
- Currently enrolled in a clinical trial of an investigational agent or device or has used any investigational agent or device within 5 half-lives of investigational agent, whichever is longer.
- Has a history of gene therapy or cell transplantation or any other experimental brain surgery.
- Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to Baseline (Day 1).
- Has experienced Status Epilepticus in the past 6 months. Key Inclusion criteria for Part 2:
- Participants in ION440-CS1, Part 1/MAD who received at least one dose of Study Drug /Sham in Part 1/MAD, missed no more than 1 study visit, and attended the Follow Up visit (Visit 6).
- All inclusion criteria in Part 1/MAD apply (participants will not be required to undergo new Screening bloodwork). Key Exclusion criteria for Part 2: 1\. Has developed any concomitant disease (e.g., gastrointestinal...
The study team makes the final eligibility decision.
Where it's taking place
- San Diego, California, United States
- Aurora, Colorado, United States
- Baltimore, Maryland, United States
- Boston, Massachusetts, United States
- Saint Paul, Minnesota, United States
- Philadelphia, Pennsylvania, United States
- Nashville, Tennessee, United States
- Houston, Texas, United States
- Linz, Austria
- Dijon, France
- Barcelona, Spain
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 male, 2 years to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include San Diego, California, United States; Aurora, Colorado, United States; Baltimore, Maryland, United States; Boston, Massachusetts, United States; Saint Paul, Minnesota, United States; Philadelphia, Pennsylvania, United States and 5 more location(s). 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.