Evaluating ION464 for Multiple System Atrophy

A Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of ION464 Administered Intrathecally to Adults With Multiple System Atrophy

This study is testing a new drug called ION464 to see if it is safe and helps adults with multiple system atrophy feel better over a 12-week period and beyond.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: 40 Years to 70 Years
• Sex: All
• Sponsor: Ionis Pharmaceuticals, Inc. (industry)
• Locations: 15 sites (Innsbruck and 14 other locations)
• Trial ID: NCT04165486 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the safety, tolerability, and pharmacokinetics of ION464 administered through intrathecal injection in adults diagnosed with multiple system atrophy (MSA). The study is divided into two parts: the first part involves a randomized, blinded, placebo-controlled design to evaluate the drug's effects over a 12-week treatment period, while the second part focuses on long-term safety and tolerability over approximately 96 weeks. Participants will undergo screening and follow-up assessments to monitor their response to the treatment and any potential side effects.

Who should consider this trial

Good fit: Ideal candidates are adults diagnosed with probable or possible MSA who can walk unassisted for at least 10 meters.

Not a fit: Patients with cognitive dysfunction or a known genetic cause of ataxia or parkinsonism may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from multiple system atrophy.

How similar studies have performed: While this approach is novel in the context of MSA, similar studies targeting neurodegenerative conditions have shown promise.

Eligibility criteria

Key Inclusion Criteria:

* Screening single-photon emission computed tomography (SPECT) with DaTscan™ (ioflupane I123 injection) results demonstrating loss (whether symmetric or asymmetric) of dopamine nerve terminals in the striatum consistent with neurodegenerative parkinsonism, as assessed with qualitative, visual read.
* Diagnosed with probable or possible MSA, either parkinsonian-type (MSA-P) or cerebellar-type (MSA-C).
* Must be able to walk unassisted for at least 10 meters (approximately 30 feet)

Key Exclusion Criteria:

* Presence of cognitive dysfunction (defined as Montreal Cognitive Assessment (MoCA) score \<25)
* Family history of ataxia or parkinsonism and known genetic cause of ataxia or parkinsonism.

NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.

Where this trial is running

Innsbruck and 14 other locations

• Medizinische Universität Innsbruck — Innsbruck, Austria (RECRUITING)
• CHU de Lyon - Hospices Civils de Lyon-H6pital Pierre Wertheimer, Neurologique HCL — Lyon, France (RECRUITING)
• Groupe Hospitalier Pitie-Salpetriere — Paris, France (RECRUITING)
• Hopital Purpan — Toulouse, France (RECRUITING)
• Universitaetsklinikum Ulm — Ulm, Baden-Wurttemberg, Germany (RECRUITING)
• Universitaetsklinikum Duesseldorf AoeR — Düsseldorf, Germany (RECRUITING)
• University Medical Center Göttingen, Clinic for Neurology — Göttingen, Germany (RECRUITING)
• Medizinische Hochschule Hannover (MHH) — Hanover, Germany (COMPLETED)
• University Hospital Marburg — Marburg, Germany (RECRUITING)
• Klinikum der Universtiatet Muenchen -Campus Grosshadern — München, Germany (RECRUITING)
• Hospital Beatriz Ângelo — Loures, Portugal (RECRUITING)
• Institute of Neurology & The National Hospital for Neurology and Neurosurgery — London, England, United Kingdom (RECRUITING)
• The John Radcliffe Hospital — Oxford, England, United Kingdom (RECRUITING)
• Salford Royal Hospital — Salford, United Kingdom (RECRUITING)
• Southampton General Hospital — Southampton, United Kingdom (RECRUITING)

Study contacts

• Study coordinator: Ionis Pharmaceuticals, Inc.

Conditions: Multiple System Atrophy