ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Iā¦ (NCT06430385) | Clinical Trial Compass
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 States48 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 range2 Years ā 65 Years
SexMALE
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
ā. Males aged ā„ 2 to ā¤ 65 years, depending on specific cohort and group, at the time of informed consent.
ā. Group A: ā„ 8 to ā¤ 65 years old
ā. Group B: 2 to 7 years old, inclusive
ā. Participant has at least one parent or caregiver ā„ 18 years old capable of providing informed consent and able to comply with all study requirements and activities.
ā. Participant has a documented diagnosis of MDS with genetic confirmation of MECP2 duplication.
ā. Is currently receiving stable doses of concomitant medications for at least 1 month prior to screening.
ā. Able to complete all study procedures, measurements and visits to support primary and secondary endpoints, in the opinion of the Investigator.
Exclusion criteria
ā. 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.
ā. Clinically significant vital sign or ECG abnormality at Screening
ā. 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.
What they're measuring
1
Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Timeframe: Up to approximately 36 weeks
2
Part 1: Number of Participants With Clinically Significant Change From Baseline in Vital Signs
Timeframe: Baseline up to approximately 36 weeks
3
Part 1: Number of Participants With Clinically Significant Change From Baseline in Physical and Neurological Examination Findings
Timeframe: Baseline up to approximately 36 weeks
4
Part 1: Number of Participants With Clinically Significant Change from Baseline in Laboratory Assessments
Timeframe: Baseline up to approximately 36 weeks
5
Part 1: Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG)
Timeframe: Baseline up to approximately 36 weeks
6
Part 2: Number of Participants With TEAEs
Timeframe: Up to approximately 192 weeks
7
Part 2: Number of Participants With Clinically Significant Change From Baseline in Vital Signs
. 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 the study or that would pose an unacceptable risk to the participant in this study.
ā. Treatment with an investigational drug, biological agent, or device within 30 days of Screening, or 5 half-lives of investigational agent, whichever is longer.
ā. 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 - both mRNA and viral vector vaccines are allowed including COVID-19). For centrally administered ASOs, a minimum of 12 months washout is required irrespective of the number of doses received.
ā. 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.
ā. Has a history of gene therapy or cell transplantation or any other experimental brain surgery.
8
Part 2: Number of Participants With Clinically Significant Change From Baseline in Physical and Neurological Examination Findings
Timeframe: Baseline up to approximately 192 weeks
9
Part 2: Number of Participants With Clinically Significant Change from Baseline in Laboratory Assessments
Timeframe: Baseline up to approximately 192 weeks
10
Part 2: Number of Participants With Clinically Significant Change From Baseline in ECG