NCT04165486 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)
| NCT ID | NCT04165486 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | Ionis Pharmaceuticals, Inc. |
| Condition | Multiple System Atrophy |
| Study Type | INTERVENTIONAL |
| Enrollment | 40 participants |
| Start Date | 2022-07-21 |
| Primary Completion | 2027-09 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 40 participants in total. It began in 2022-07-21 with a primary completion date of 2027-09.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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).
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.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT04165486 clinical trial?
This trial is open to participants of all sexes, aged 40 Years or older, up to 70 Years, studying Multiple System Atrophy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT04165486 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT04165486 currently recruiting?
Yes, NCT04165486 is actively recruiting participants. Contact the research team at IonisHORIZONStudy@clinicaltrialmedia.com for enrollment information.
Where is the NCT04165486 trial being conducted?
This trial is being conducted at Innsbruck, Austria, Lyon, France, Paris, France, Toulouse, France and 11 additional locations.
Who is sponsoring the NCT04165486 clinical trial?
NCT04165486 is sponsored by Ionis Pharmaceuticals, Inc.. The trial plans to enroll 40 participants.