ATTUNE: A Study for MECP2 Duplication Syndrome
This study, called ATTUNE, is testing a new drug called ION440 for males with MECP2 Duplication Syndrome (MDS). Researchers want to see if ION440 is safe and how well people tolerate it. You might be able to join if you are a male between 2 and 65 years old with MDS. The study will compare ION440 to a sham procedure (a spinal tap without the drug) to understand its effects. If you complete the first part of the study, you may have the option to continue receiving ION440 for a longer period. The study aims to enroll 48 participants.
- Study design
- This is a Phase 1-2 randomized, double-blind, sham-controlled study with multiple ascending doses. It will enroll 48 participants and has two parts, with the second part being an open-label extension.
- What's involved
- In Part 1, you would receive either ION440 or a sham procedure for up to approximately 36 weeks. If you continue to Part 2, you would receive ION440 for up to approximately 156 weeks.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for safety and other changes from baseline for up to approximately 36 weeks in Part 1, and up to approximately 156 weeks in Part 2.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
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)
At a glance
Conditions
Where it's being run
11 sites across 11 statesWho to contact
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What this trial measures
- Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)Up to approximately 36 weeks
- Part 1: Number of Participants With Clinically Significant Change From Baseline in Vital SignsBaseline up to approximately 36 weeks
- Part 1: Number of Participants With Clinically Significant Change From Baseline in Physical and Neurological Examination FindingsBaseline up to approximately 36 weeks
- Part 1: Number of Participants With Clinically Significant Change from Baseline in Laboratory AssessmentsBaseline up to approximately 36 weeks
- Part 1: Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG)Baseline up to approximately 36 weeks
- Part 2: Number of Participants With TEAEsUp to approximately 192 weeks
- Part 2: Number of Participants With Clinically Significant Change From Baseline in Vital SignsBaseline up to approximately 192 weeks
- Part 2: Number of Participants With Clinically Significant Change From Baseline in Physical and Neurological Examination FindingsBaseline up to approximately 192 weeks
- Part 2: Number of Participants With Clinically Significant Change from Baseline in Laboratory AssessmentsBaseline up to approximately 192 weeks
- Part 2: Number of Participants With Clinically Significant Change From Baseline in ECGBaseline up to approximately 192 weeks