Overview

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

Status:
Completed
Trial end date:
2017-11-08
Target enrollment:
0
Participant gender:
All
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.
Phase:
Phase 1/Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Ionis Pharmaceuticals, Inc.
Criteria
Key Inclusion Criteria:

- Diagnosed with early manifest Huntington's disease

- Male or female, aged 25 to 65 years, inclusive, at the time of informed consent

- Able and willing to meet all study requirements, including travel to Study Center and
participation in all procedures and measurements at study visits

- Have a trial partner who is reliable, competent and at least 18 years of age, is
willing to accompany the participant to select trial visits and to be available to the
Study Center by phone if needed

- Able to tolerate MRI scans, blood draws and lumbar punctures

- Reside within 4 hours travel of the Study Center

Key Exclusion Criteria:

- Clinically significant medical condition, such as severe chorea, active suicidal
ideation or any other conditions which would make the participant unsuitable for
inclusion or could interfere with the participant participating in or completing the
study

- Recent treatment with another investigational drug, biological agent, or device

- Prior treatment with an antisense oligonucleotide [including small interfering
ribonucleic acid (siRNA)]

- Any history of gene therapy or cell transplantation or any other experimental brain
surgery

- Presence of an implanted shunt for the drainage of cerebrospinal fluid (CSF) or an
implanted central nervous system (CNS) catheter

- History of post-lumbar-puncture headache of moderate or severe intensity and/or blood
patch

- Malignancy within 5 years of Screening, except for basal or squamous cell carcinoma of
the skin or carcinoma in situ of the cervix that has been successfully treated

- Hospitalization for any major medical or surgical procedure involving general
anesthesia within 12 weeks of Screening or planned during the study