RecruitingPhase 1/2

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)

United States, Austria, France48 participantsStarted 2024-10-21

Plain-language summary

The primary purpose of this study is to evaluate the safety and tolerability of ION440.

Who can participate

Age range

2 Years – 65 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. 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.

Exclusion criteria

. 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 prior to age 5 (for those aged 5 and above at signing of ICF), (b) oxygen dependence, (c) microcephaly, IF MECP2 genetic structure information is unavailable.
. Clinically significant vital sign or ECG abnormality at Screening

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

Timeframe: Up to approximately 36 weeks

Part 1: Number of Participants With Clinically Significant Change From Baseline in Vital Signs

Timeframe: Baseline up to approximately 36 weeks

Part 1: Number of Participants With Clinically Significant Change From Baseline in Physical and Neurological Examination Findings

Timeframe: Baseline up to approximately 36 weeks

Part 1: Number of Participants With Clinically Significant Change from Baseline in Laboratory Assessments

Timeframe: Baseline up to approximately 36 weeks

Part 1: Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG)

Timeframe: Baseline up to approximately 36 weeks

Part 2: Number of Participants With TEAEs

Timeframe: Up to approximately 192 weeks

Trial details

NCT IDNCT06430385

SponsorIonis Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-09

Contact for this trial

Ionis Pharmaceuticals

(844) 779-1497 IonisMECP2study@clinicaltrialmedia.com

View on ClinicalTrials.gov More Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome trials

Who can participate

Age range

2 Years – 65 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. 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.

Exclusion criteria

. 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 prior to age 5 (for those aged 5 and above at signing of ICF), (b) oxygen dependence, (c) microcephaly, IF MECP2 genetic structure information is unavailable.
. Clinically significant vital sign or ECG abnormality at Screening

What they're measuring

Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

Timeframe: Up to approximately 36 weeks

Part 1: Number of Participants With Clinically Significant Change From Baseline in Vital Signs

Timeframe: Baseline up to approximately 36 weeks

Part 1: Number of Participants With Clinically Significant Change From Baseline in Physical and Neurological Examination Findings

Timeframe: Baseline up to approximately 36 weeks

Part 1: Number of Participants With Clinically Significant Change from Baseline in Laboratory Assessments

Timeframe: Baseline up to approximately 36 weeks

Part 1: Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG)

Timeframe: Baseline up to approximately 36 weeks

Part 2: Number of Participants With TEAEs

Timeframe: Up to approximately 192 weeks