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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02519036
• Date of registration: 01/08/2015
• Prospective Registration: Yes
• Primary sponsor: Ionis Pharmaceuticals, Inc.
• Public title: Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants With Early Manifest Huntington's Disease
• Scientific title: 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
• Date of first enrolment: August 6, 2015
• Target sample size: 46
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02519036
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

Canada, Germany, United Kingdom

Key inclusion & exclusion 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



Age minimum: 25 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Huntington's Disease

Intervention(s)

Drug: ISIS 443139 90 mg

Drug: ISIS 443139 10 mg

Drug: ISIS 443139 30 mg

Other: Placebo

Drug: ISIS 443139 60 mg

Drug: ISIS 443139 120 mg

Primary Outcome(s)

Number of Participants With Treatment-related Adverse Events (TEAEs) [Time Frame: Up to approximately 28 weeks]

Secondary Outcome(s)

Observed Cerebrospinal Fluid (CSF) Concentration for ISIS 443139 [Time Frame: Days 1, 29, 57, 85, and 113 or 141]

Secondary ID(s)

2015-000381-66

ISIS 443139-CS1

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 31/05/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02519036