Ignite Creation Date:
2024-06-16 @ 11:49 AM
Last Modification Date:
2024-10-26 @ 3:30 PM
Study NCT ID:
NCT06430385
Status:
RECRUITING
Last Update Posted:
2024-05-28
First Post:
2024-05-21
Brief 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
Sponsor:
Ionis Pharmaceuticals Inc
Organization:
Ionis Pharmaceuticals Inc
Study Overview
Official Title:
A Phase 1-2 Double-Blind Sham-Controlled Multiple Ascending Dose Study to Evaluate Safety Tolerability Pharmacokinetics and Pharmacodynamics of Intrathecally-Administered ION440 in Patients With MECP2 Duplication Syndrome
Status:
RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The primary purpose of this study is to evaluate the safety and tolerability of ION440
Detailed Description:
This is a phase 1-2 randomized double-blind sham-controlled multiple-ascending dose MAD study to evaluate ION440 in pediatric and adult participants with MECP2 Duplication Syndrome MDS and will be conducted in two parts During Part 1 MAD 36 weeks participants will be randomized in a 31 ratio to receive ION440 or sham Individuals who complete Part 1 may enter Part 2 an open label long-term extension study LTE where they will receive ION440 for up to approximately 156 weeks Multiple dose cohorts Dose A Dose B and Dose C will be evaluated in the study
All dosing cohorts will be further subdivided by age Sub cohort A will include participants 8 years of age and older and sub cohort B will include participants 2 through 7 years of age Dosing cohorts will be enrolled sequentially with sub cohort A initiating prior to sub cohort B
All dosing cohorts will be further subdivided by age Sub cohort A will include participants 8 years of age and older and sub cohort B will include participants 2 through 7 years of age Dosing cohorts will be enrolled sequentially with sub cohort A initiating prior to sub cohort B
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2023-507192-22 | EUDRACT_NUMBER | None | None |