HELIOS-A: A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and
Safety of ALN-TTRSC02 in Patients with Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
PI: Michelle Mezei Co-investigator: Kristin Jack Study Coordinator: Deborah Kraus
Ionis CS13 Roll-Over Study – (Eplontersen open label study)
January 2023-present
Full Name of Study:
An Open-Label, Extension Study to Assess the Long-Term Safety and Efficacy of ION-
682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy (ION-682884-CS13)
A Phase 3, Multi-center, Randomized, Quadruple-blind, Placebo-controlled Study to
Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants
with Generalized Myasthenia Gravis (gMG) - (IMVT-1401-3101 Study)
An investigator lead project with the goal to: 1. Quantify balance deficits in SG 2. Examine relation of balance in SG with patient reported outcome and bedside balance testing 3. Understand the impact of balance impairment on quality of life
PI: Mark Carpenter; Mike Berger
Outcome Measures in Autoimmune Neuropathy
We are looking at how to incorporate patient reported outcome measures into routine clinical practice, utilizing a value based healthcare mode. This work is supported by grants and generous donations by patients and families to the hospital foundation.
PI: Kristine Chapman; Katie Beadon
Transition to Subcutaneous Immunoglobulin
Subcutaneous Immunoglobulin therapy is an effective treatment for autoimmune neuropathies, that can be self-administered by the patient at home. This Quality Improvement project looks at patient reported outcome to better understand the transition from intravenous to subcutaneous immunoglobulin.
A series of three Value Based Health Care workshops were held with the full team, to explore the full cycle of care and the attributes of a high functioning Integrated Practice Unit. An outcome measure form, including pain and functional scale, was developed. Evaluation of impact of the pathway in terms of time to effective treatment and improving patient experience is planned.
PI: Kristine Chapman; Sean Bristol
Neurofibromatosis
Currently, care for patients with Neurofibromatosis in BC is fragmented. We are currently conducting a QI project to create a clinical pathway with recommendations for providers. As part of the Quality Improvement initiative, we are holding a series of focus groups with patients and providers to understand the current gaps in care and needs, in conjunction with UBC NeuroEthics. A new multidisciplinary rounds on NF has been initiated, inconjunction with BCCA.
PI: Kristine Chapman; Judy Illes
Lead by neuromuscular fellow Dr. Laura Marulanda
IVIg Utilization
In conjunction with the Provincial Blood Coordinating Office and the IVIg Neuromuscular Panel, we are supporting optimal utilization for Immunoglobulin treatment in BC. Ongoing work is being done to identify and evaluate appropriate use in high and chronic user groups.
