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)

Conditions

• Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

Eligibility Criteria

Sex

MALE

Age

2 Years - 65 Years

Healthy Volunteers

Not accepted

Interventions

• ION440 — DRUG
  ION440 will be administered by intrathecal bolus (ITB) injection.
• Sham procedure — PROCEDURE
  An LP will be performed with CSF collection but will not be followed by the administration of study treatment by ITB injection.

Study Details

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

Key Dates

Start date

Oct 21, 2024

Status verified

Mar 2026

Primary completion

Sep 30, 2027

Completion

Apr 30, 2030

Study Design

Enrollment

48 participants (estimated)

Allocation

RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Cohort 1: ION440 Dose A
  Participants will receive ION440 intrathecally at Dose A during Part 1/MAD, followed by ION440 Dose A during Part 2/LTE.
• Experimental: Cohort 2: ION440 Dose B
  Participants will receive ION440 intrathecally at Dose B during Part 1/MAD, followed by ION440 Dose B during Part 2/LTE.
• Experimental: Cohort 3: ION440 Dose C
  Participants will receive ION440 intrathecally at Dose C during Part 1/MAD, followed by ION440 Dose C during Part 2/LTE.
• Sham Comparator: Sham Procedure
  During the Part 1/MAD period, a lumbar procedure (LP) will be performed at the same frequency as ION440 administration. Participants will not receive ITB injections during this period. It will be followed by the open-label Part 2/LTE period, where participants will receive ION440 at the same dose as their enrolled cohort (e.g. Dose A, Dose B or Dose C).

Primary Outcome Measure

Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) [ Time Frame: Up to approximately 36 weeks ]

Central Contacts

• Ionis Pharmaceuticals
  (844) 779-1497
  [email protected]

Locations (8)

Find similar trials in San Diego, CA