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Tests treatment safety and results for Multiple System Atrophy
Official title Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)
ClinicalTrials.gov ID: NCT04165486
What this study is testing
What is ION464?
ION464 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for multiple system atrophy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The primary objectives are to evaluate the safety and tolerability of multiple doses of ION464 administered via intrathecal (IT) injection (Part 1) and to evaluate the long-term safety and tolerability of ION464 (Part 2) in participants with multiple system atrophy (MSA). The secondary objectives are to evaluate the pharmacodynamic (PD) effect of ION464 on the level of a potential biomarker of target engagement (Parts 1 and 2) and to evaluate the pharmacokinetic (PK) profile of ION464 in serum (Part 1).
- Phase 1: an early, usually small safety study
- Time commitment: about 12 weeks
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 40 to 70
You may be able to join if
- Screening single-photon emission computed tomography (SPECT) with DaTscan™ (ioflupane I123 injection) results demonstrating loss (whether symmetric...
- 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
You likely can't join if
- 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...
The study team makes the final eligibility decision.
Where it's taking place
- Innsbruck, Austria
- Lyon, France
- Paris, France
- Toulouse, France
- Ulm, Baden-Wurttemberg, Germany
- Düsseldorf, Germany
- Göttingen, Germany
- Hanover, Germany
- Marburg, Germany
- München, Germany
- Loures, Portugal
- London, England, United Kingdom
- Oxford, England, United Kingdom
- Salford, United Kingdom
- Southampton, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 12 weeks per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 40 years to 70 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Innsbruck, Austria; Lyon, France; Paris, France; Toulouse, France; Ulm, Baden-Wurttemberg, Germany; Düsseldorf, Germany and 9 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.