NCT02519036

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants With Early Manifest Huntington's Disease

A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ISIS 443139 in Patients With Early Manifest Huntington's Disease

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 46 (actual)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 25 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This study tested the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending doses of ISIS 443139 administered intrathecally to adult participants with early manifest Huntington's Disease.

Conditions

• Huntington's Disease

Interventions

Type	Name	Description
DRUG	ISIS 443139 10 mg	ISIS 443139, 10 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.
DRUG	ISIS 443139 30 mg	ISIS 443139, 30 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.
DRUG	ISIS 443139 60 mg	ISIS 443139, 60 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.
DRUG	ISIS 443139 90 mg	ISIS 443139, 90 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.
DRUG	ISIS 443139 120 mg	ISIS 443139, 120 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.
OTHER	Placebo	Placebo was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.

Timeline

• Start date: 2015-08-06
• Primary completion: 2017-11-08
• Completion: 2017-11-08
• First posted: 2015-08-10
• Last updated: 2019-05-31
• Results posted: 2019-05-31

Locations

9 sites across 3 countries: Canada, Germany, United Kingdom

Regulatory

• FDA-regulated drug study