Neuromuscular Disorders Research

A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects with INMN (Immune-Mediated Necrotizing Myopathy)

Investigator: Suur Biliciler, MD

Lead CRC: Estela Acosta

Purpose: To Evaluate the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects
with Immune-Mediated Necrotizing Myopathy

Key Inclusion Criteria: 18-75 yr old with Dx of IMNM, positive for anti-HMGCR or anti-SRP autoantibodies. Must be vaccinated against meningococcal infections within 3 yrs or prior to initiation study drug. (History of meningococcal disease, the use of Rituximab w/in 90 days plasma exchange w/in 4 weeks and active malignancy are excluded)

Enrollment Status: Actively enrolling

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A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis

Investigator: Thy Nguyen, MD

Lead CRC: Estela Acosta

Purpose: To Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis.

Key Inclusion Criteria: 18 yrs and older with at least 6 o DX of MG, MGFA Class II to IV. (patients with history N meningiditis, HIV, thymectomy, thymomectomy, or any thymic surgery within the 12 months are excluded). (IVIg within 4 weeks prior to randomization and the use of rituximab w/in 6 months is excluded.

Enrollment Status: Actively enrolling

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A Phase 1b Study to Evaluate the Safety, Tolerability and Drug-Drug Interactions of ANX005 in Combination with Intravenous Immunoglobulin (IVIg) in Subjects with Guillain-Barré Syndrome

Investigator: Kazim Sheikh, MD

Lead CRC: Carla Wilkerson

Key Inclusion Criteria: 18 yrs and older dx of GBS, onset of GBS-related weakness ≤ 14 days prior to infustion, GBS-DS score of 3-5. (Subjects with body weight > 150 kg, variants of GBS, hyperviscosity, IgA deficiency and treatment with any monoclonal antibody w/in 6 monthmeningiditiss are excluded).

Enrollment Status: actively enrolling

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A Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase (HYQVIA/HyQvia) and Immune Globulin Infusion (Human), 10% (GAMMAGARD LIQUID/KIOVIG) for the Treatment of Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

Investigator: Kazim Sheikh, MD

Lead CRC: Carla Wilkerson

Key Inclusion Criteria: 18 yrs and older with dx of definite/probable CIDP confirmed by EFNS/PNS 2010 criteria; subject has responded to IgG tx in the past; INCAT score between 0-7. (focal atypical CIDP, pure sensory atypical CIDP, known type I or II hyperprolinemia, Thrombophilic abnormalities including blood hyperviscosity, protein C or protein S deficiency, anti-thrombin-III deficiency, plasminogen deficiency, antiphospholipid antibodies, Factor V Leiden mutation, dysfibrinogenemia, or prothrombin G20210A mutation are excluded)

Enrollment Status: Actively enrolling

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