Evaluating ION464 for Multiple System Atrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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

Study contacts

• Study coordinator: Ionis Pharmaceuticals, Inc.
• Email: IonisHORIZONStudy@clinicaltrialmedia.com
• Phone: (844) 748-5844

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Tributyrin for PSP: a virtual, early-phase study

  This study tests whether tributyrin is safe and tolerable in people with Progressive Supranuclear Palsy who are 45 or older, using virtual visits.

  Recruiting Phase 1/2 University of Michigan · Ann Arbor, Michigan · NCT07863180

• Natural history of atypical parkinsonian syndromes: a multi-center observation

  This study follows people with APS like MSA, DLB, PSP, and CBD to see how their symptoms and brain images change over time.

  Recruiting at 6 sites Second Affiliated Hospital of Soochow University · Changshu, Jiangsu and 19 other locations · NCT07805967

• Longitudinal registry of people with multiple system atrophy in China

  This study follows Chinese patients with multiple system atrophy over time to describe how the disease starts and progresses and to compare with people with Parkinson's and healthy controls.

  Recruiting Peking University First Hospital · Beijing, Beijing Municipality · NCT07644013

• Personalized magnetic brain stimulation for parkinsonian disorders

  This study will try personalized repetitive transcranial magnetic stimulation (rTMS) to see if it eases motor and non-motor symptoms in people with Parkinson's disease, multiple system atrophy, or progressive supranuclear palsy.

  Recruiting Peking University First Hospital · Beijing, Beijing Municipality · NCT07570212

• Butylphthalide for Multiple System Atrophy: tests safety and symptoms

  This study tests whether 3-n-butylphthalide capsules can help reduce symptoms in people with multiple system atrophy compared with a placebo.

  Recruiting at 4 sites Phase 2/3 Second Affiliated Hospital, Zhejiang University, School of Medicine · Nanning, Guangxi and 6 other locations · NCT07518810

• ALT001 expansion study for people with multiple system atrophy

  ALT001 is being tested in an open-label, single-center study to see if it is safe, tolerable, and shows early signs of benefit for adults with multiple system atrophy.

  Recruiting Early Phase 1 Beijing Tiantan Hospital · Beijing, Beijing Municipality · NCT07476352

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.