A Phase 1b Study to Assess the Safety, Tolerability, and Pharmacokinetics of ION269 in Adults With Down Syndrome (Hero Study)

Status: Recruiting
Location: See all (3) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
SUMMARY

The primary purpose of this study is to evaluate the safety and tolerability of ION269 in adults with Down syndrome with evidence of brain amyloid positivity.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 35
Maximum Age: 55
Healthy Volunteers: f
View:

• Has a reliable study partner, that is, a parent, sibling, or caregiver ≥ 21 years of age, who has known the participant for \> 6 months (i.e., a reliable and competent individual with a close relationship with the participant) and is capable of providing accurate information about the participant's history, can attend all scheduled study visits and provide feedback regarding the participant's symptoms and performance as described in the protocol, and can comply with all study requirements and activities.

• Has a diagnosis of Down syndrome and has an intelligence quotient (IQ) ≥ 45.

• Has evidence of amyloid pathology on amyloid-positron emission tomography (PET) scan.

• Is assessed as being cognitively stable.

• Is in good health as evidenced by medical history, physical, and neurological examination, and with no diagnosis of dementia or mild cognitive impairment.

Locations
United States
Kansas
Ionis Investigative Site
RECRUITING
Kansas City
Missouri
Ionis Investigative Site
RECRUITING
Saint Louis
Other Locations
Spain
Ionis Investigative Site
RECRUITING
Barcelona
Contact Information
Primary
Ionis Pharmaceuticals
IonisHEROStudy@clinicaltrialmedia.com
(844) 599-7690
Time Frame
Start Date: 2025-01
Estimated Completion Date: 2027-03
Participants
Target number of participants: 30
Treatments
Experimental: Cohort 1
Participants will receive a single dose of ION269.
Experimental: Cohort 2
Participants will receive a single dose of ION269.
Experimental: Cohort 3
Participants will receive a single dose of ION269.
Sponsors
Leads: Ionis Pharmaceuticals, Inc.

This content was sourced from clinicaltrials.gov