Research

Dr. Piquet’s research program seeks to advance our understanding of rare autoimmune neurologic disorders through observational studies, translational studies focused on identifying potential biomarkers associated with neurological outcomes and treatment response, and innovative clinical trials.

Highlighted Clinical Trials in our Autoimmune Neurology Program

ExTINGUISH: A Study of Inebilizumab in NMDAR Encephalitis

The ExTINGUISH trial evaluates inebilizumab, a monoclonal antibody that targets CD19-positive B cells, in individuals with anti-NMDAR encephalitis. The study is investigating whether targeting B cells can improve neurological outcomes and reduce the ongoing autoimmune activity associated with this condition.

Participation dates: 09/2024 – Ongoing

NCT: 04372615

ClinicalTrials.gov: NCT04372615

KYSA-8: A Phase 2 Open-Label, Single-Arm, Multicenter Study of KYV-101, an Autologous Fully Human Anti-CD19 Chimeric Antigen Receptor T-Cell (CD19 CAR T) Therapy, in Subjects with Treatment-Refractory Stiff Person Syndrome

This Phase 2 study evaluates KYV-101, an autologous CD19-directed CAR T-cell therapy, in individuals with treatment-refractory stiff person syndrome (SPS). The study is designed to evaluate the safety, tolerability, and potential clinical benefit of CD19 CAR T-cell therapy in people with severe SPS who have not responded adequately to standard treatments.

Participation dates: 09/2024 – 08/2026

NCT: 06588491

ClinicalTrials.gov: NCT06588491

Cielo: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Satralizumab in Participants With Anti-N-Methyl-D-Aspartate Receptor (NMDAR) or Anti-Leucine-Rich Glioma-Inactivated 1 (LGI1) Encephalitis

This study evaluates satralizumab, an interleukin-6 (IL-6) receptor inhibitor, in people with autoimmune encephalitis associated with antibodies against the NMDAR or LGI1 proteins. The study is designed to assess the safety and potential efficacy of satralizumab, as well as how the medication behaves in the body and affects immune activity.

Participation dates: 01/2023 – 09/2026

NCT: 05503264

ClinicalTrials.gov: NCT05503264

Meteoroid: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Satralizumab in Participants With Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (MOGAD)

This international Phase III study evaluated satralizumab (Enspryng), an interleukin-6 (IL-6) receptor inhibitor, in adults and adolescents aged 12 years and older with relapsing MOGAD. Results presented at the 2026 American Academy of Neurology (AAN) meeting showed that satralizumab reduced the risk of a new relapse by 68% compared with placebo, with 87% of participants receiving satralizumab remaining relapse-free at 48 weeks compared with 67% receiving placebo.

Participation dates: 09/2023 – 05/2026

NCT: 05271409

ClinicalTrials.gov: NCT05271409

cosMOG: A Study to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Myelin Oligodendrocyte Glycoprotein (MOG) Antibody-Associated Disease (MOGAD)

Sponsored by UCB Biopharma, this Phase III study evaluates rozanolixizumab, a neonatal Fc receptor (FcRn) inhibitor, in adults with MOGAD. By increasing the breakdown of circulating antibodies, including disease-associated MOG antibodies, the treatment is being studied for its potential to reduce the risk of relapse and its safety in people with MOGAD.

Participation dates: 03/2022 – 02/2026

NCT: 05063162

ClinicalTrials.gov: NCT05063162

PIONEER-1: Privosegtor Investigation in Optic Neuropathies Efficacy Evaluation Research

This clinical trial is to evaluate the safety and efficacy of privosegtor, a neuroprotective candidate, in patients diagnosed with optic neuritis (ON). Researchers will compare privosegtor and the standard of care (methylprednisolone) to a placebo and standard of care (methylprednisolone).

Participation dates: 10/2026 – Ongoing

NCT: 07623668

ClinicalTrials.gov: NCT07623668

TIMELY-PLEX: A Randomized Controlled, Open-Label, Rater-Blinded Pragmatic Trial, "Treatment of Inflammatory Myelitis and Optic Neuritis with Early Versus Rescue Plasma Exchange"

This trial will evaluate if early vs rescue Therapeutic Plasma Exchange (PLEX) treatment algorithm leads to better visual outcomes in severe Optic Neuritis and leads to better neurological disability outcomes in severe Transverse Myelitis.

Participation dates: 01/2026 – Ongoing

NCT: 07100990

ClinicalTrials.gov: NCT07100990

SPHERES: Synergy of Prospective Health & Experimental Research for Emerging Solutions Registry for Neuromyelitis Optica Spectrum Disorder (NMOSD)

This is a registry study to create a cohort of patients with NMOSD. Data collected will be used to prospectively study the natural history of NMOSD, real-word effectiveness and safety of medications used to treat NMOSD, and drug utilization treatment patterns.

Participation dates: 10/2021 – Ongoing

NCT: 04886492

ClinicalTrials.gov: NCT04886492

Recent Publications