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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)

Description

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

Conditions

Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
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Related Conditions:

Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

Study Overview

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Study Details

Study overview

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

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

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)

Condition
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Intervention / Treatment

-

Contacts and Locations

Houston

Baylor College of Medicine, Houston, Texas, United States, 77030

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

    Ages Eligible for Study

    2 Years to 65 Years

    Sexes Eligible for Study

    MALE

    Accepts Healthy Volunteers

    No

    Collaborators and Investigators

    Ionis Pharmaceuticals, Inc.,

    Study Record Dates

    2030-04