{"id":32747,"date":"2025-02-18T10:35:16","date_gmt":"2025-02-18T09:35:16","guid":{"rendered":"https:\/\/dupmecp2.eu\/debut-du-recruitment-des-patients-pour-attune\/"},"modified":"2025-07-23T10:27:23","modified_gmt":"2025-07-23T08:27:23","slug":"attune-recruitment","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/en\/attune-recruitment\/","title":{"rendered":"Start of recruitment for the ATTUNE clinical trial"},"content":{"rendered":"

The ATTUNE trial is a Phase 1\/2 clinical study sponsored by Ionis. It aims to evaluate ION440, an investigational Antisense Oligonucleotide (ASO) therapy designed to address the root cause of MECP2 Duplication Syndrome (MDS). <\/strong> <\/p>\n\n\n\n

Patient recruitment for the groundbreaking ATTUNE clinical trial has officially begun! The first site opened in November 2024 at Baylor College of Medicine in Houston, Texas (US), marking the start of this Phase 1\/2 study. At the end of January 2025, a second site opened in Minnesota (US). Additional sites in the United States and globally are expected to open soon. <\/p>\n\n\n\n

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Trial Overview<\/strong> <\/p>\n\n\n\n

The ATTUNE trial represents the first time ION440 has been administered to humans. The investigational ASO therapy aims to reduce the levels of MECP2 mRNA in individuals with MDS, addressing the root cause of symptoms caused by MECP2 overexpression. <\/p>\n\n\n\n

This potential treatment approach has shown impressive results during preclinical research experiments. Studies conducted in 2021 in mouse models of MDS demonstrated that ASO therapy effectively reduced MECP2 levels, and reduced disease symptoms without significant adverse effects. Almost 10 years ago, these findings generated significant excitement and hope within the MDS community. <\/p>\n\n\n\n

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To the publication<\/a><\/div>\n<\/div>\n\n\n\n
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The trial enrolled male participants aged 2 to 65 years with a confirmed diagnosis of MECP2 Duplication Syndrome. The study will be divided into 2 parts. Part 1 (Multiple ascending doses) will last 36 weeks for each participant. During that phase, participants will be randomized to receive either ION440 or a sham procedure. Upon completion of part 1, a participant may move to part 2 (Long-term extension) and receive ION440 for up to 156 weeks. <\/p>\n\n\n\n

Each participant will follow a detailed schedule, including an initial screening period, administration of ION440, and a follow-up phase to evaluate the safety, tolerability, and preliminary efficacy outcomes of the potential drug treatment. Participants will be enrolled sequentially in different cohorts, with each cohort receiving a different dose level of ION440. <\/p>\n\n\n\n

For more details about the study protocol and overall study design, we invite you to explore the following resources: <\/p>\n\n\n\n