This study is a multi-center, open-label study of intravenous (IV) ANX005 in participants with, or at risk for, manifest Huntington's Disease (HD).
The objective of this study is to evaluate the effects of IV ANX005 administered for up to 22 weeks in participants with, or at risk for, manifest HD. Participants will receive induction dosing of ANX005 administered by IV infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks through Week 22, with follow up visits on Weeks 24, 28, and 36. All participants will be contacted (in clinic visit or phone call) 6 months after study completion.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
28
Intravenous Infusion
Annexon Investigational Site 02
Birmingham, Alabama, United States
Annexon Investigational Site 03
Englewood, Colorado, United States
Annexon Investigational Site 04
Washington D.C., District of Columbia, United States
Annexon Investigational Site 07
Durham, North Carolina, United States
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
An adverse event (AE) was defined as any untoward medical occurrence that developed or worsened in severity during the conduct of a clinical study and does not necessarily have a causal relationship to study drug. Serious AEs (SAEs) included death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or, a congenital anomaly or birth defect, or an important medical event that jeopardized the participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was any AE with an onset on or after the day of infusion through Week 36 (end of study). AEs were graded per National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE): Grade: 1=Mild, 2=Moderate, 3=Severe or medically significant but not immediately life-threatening. A summary of serious and non-serious AEs regardless of causality is located in 'Reported Adverse Events module'.
Time frame: From first dose of study drug up to end of study (Week 36)
Actual Dose of ANX005 Administered on Day 1
Time frame: Day 1
Actual Dose of ANX005 Administered on Week 22
Time frame: Week 22
Area Under the Concentration Versus Time Curve From Time 0 to t (AUC0-t) of ANX005 at Day 1
Time frame: Pre-dose up to 4 hours post-dose on Day 1
CSF Free Complement Component 1q (C1q) at Day 1
Time frame: Predose at Baseline (Day 1)
Blood Free C1q at Day 1
Time frame: Predose at Baseline (Day 1)
Change From Baseline in Complement C4a in CSF at Week 24
Time frame: Baseline, Week 24
Change From Baseline in Complement C4a in CSF at Week 36
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Annexon Investigational Site 06
Cincinnati, Ohio, United States
Annexon Investigational Site 08
Kirkland, Washington, United States
Time frame: Baseline, Week 36
Change From Baseline in CSF NfL Level at Week 24
Time frame: Baseline, Week 24
Change From Baseline in CSF NfL Level at Week 36
Time frame: Baseline, Week 36
Change From Baseline in Blood NfL Level at Week 24
Time frame: Baseline, Week 24
Change From Baseline in Blood NfL Level at Week 36
Time frame: Baseline, Week 36