AIM01 – Ublituximab in Autoantibody Positive Immune Mediated Necrotizing Myopathy
NCT Number: NCT07109746
Conditions: Autoimmune Disorders
Purpose of Study:
This is a multi-center, Phase 2 trial of ublituximab as first-line combination therapy in early, active autoantibody positive immune-mediated necrotizing myopathy.
The primary objective is to estimate the effect of ublituximab as first add-on combination therapy at 24 weeks compared to placebo in treating early, active autoantibody positive immune-mediated necrotizing myopathy using the validated 2016 American College of Rheumatology/ European League Against Rheumatism (ACR/EULAR) Myositis Response Criteria, as measured by the Total Improvement Score (TIS)
Interventions / DRUG: Ublituximab – Initial dose includes 150 mg dose followed by 450 mg two weeks later; maintenance dose is 450 mg ; Placebo for Ublituximab – 0.9% sodium chloride injection
Trial Information: Enrollment: 30 participants; Study Start Date: 2026-01; Primary Completion Date: 2029-06; Study Completion Date: 2030-06
Eligibility Criteria: Age Range: 18 years to N/A; Sex: ALL
Study Status: Recruiting
Phase: PHASE2
Sponsor & Collaborators: Lead Sponsor: National Institute of Allergy and Infectious Diseases (NIAID); Collaborators: Autoimmunity Centers of Excellence, TG Therapeutics, Rho Federal Systems Division, Inc.
Johns Hopkins Locations: Johns Hopkins Hospital: Division of Rheumatology
Coordinator/Contact: Zuzana Bologna / E-mail: jh.myositis.research@jh.edu
Principal Investigator: Julie Paik, MD, MPH
JASMINE – A Multicenter, Parallel-group, Double-blind, 2-Arm, Phase III Study to Investigate the Efficacy and Safety of Anifrolumab Administered as Subcutaneous Injection and Added to Standard of Care Compared with Placebo Added to Standard of Care in Adult Participants with Idiopathic Inflammatory Myopathies (Polymyositis and Dermatomyositis)
Study Description:The purpose of this multicenter, randomized, placebo-controlled and double-blind study is to evaluate the efficacy and safety of subcutaneous anifrolumab compared with placebo on the overall disease activity in participants with moderate to severe Idiopathic Inflammatory Myopathies (IIM) [polymyositis (PM) or dermatomyositis (DM)] while receiving standard of care (SoC) treatment.
NCT Number: NCT06455449
Conditions: Autoimmune Disorders
Interventions / DRUG: Placebo or Anifrolumab (1:1 randomization) – weekly subcutaneous injection
Trial Information: Enrollment: 240 participants; Study Start Date: 2024-06-20; Primary Completion Date: 2027-05-14; Study Completion Date: 2028-08-04
Eligibility Criteria: Age Range: 18 years to 75 years; Sex: ALL
Study Status: Recruiting
Phase: PHASE3
Sponsor & Collaborators: AstraZeneca
Johns Hopkins Locations: Johns Hopkins Hospital: Division of Rheumatology
Coordinator/Contact: Zuzana Bologna / E-mail: jh.myositis.research@jh.edu
Principal Investigator: Julie Paik, MD, MPH
RAINBOW – Phase 1b Trial of RAY121 in Immunological Diseases
Study Description: This Phase 1b basket trial will investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity and preliminary efficacy of RAY121, a inhibitor of classical complement pathway, after multiple dose administration in patients with immunological diseases such as antiphospholipid syndrome (APS), bullous pemphigoid (BP), Behçet’s Syndrome (BS), dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM) and immune thrombocytopenia (ITP).
NCT Number: NCT06371417
Conditions: Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP), Behçet’s Syndrome (BS), Dermatomyositis (DM), Immune-mediated Necrotizing Myopathy (IMNM), Immune Thrombocytopenia (ITP)
Interventions / DRUG: RAY121 – Injection
Trial Information: Enrollment: 144 participants; Study Start Date: 2024-08-19; Primary Completion Date: 2026-06-30; Study Completion Date: 2026-06-30
Eligibility Criteria: Age Range: 18 years to 85 years
Study Status: Recruiting
Phase: PHASE1
Sponsor & Collaborators: Chugai Pharmaceutical
Johns Hopkins Locations: Johns Hopkins University
Coordinator/Contact: Grazyna Purwin / E-mail: jh.myositis.research@jh.edu
Principal Investigator: Lisa Christopher-Stine, MD, MPH
VALOR – A Study to Investigate the Efficacy and Safety of Brepocitinib in Adults with Dermatomyositis
NCT Number: NCT05437263
Conditions: Autoimmune Disorders
Purpose of Study: This is a phase 3, multicenter, randomized, placebo-controlled, double-blind study of treatment with brepocitinib (TYK2/JAK1 inhibitor) in adults with dermatomyositis (DM). The primary objective of this study is to assess the efficacy of two dose levels of brepocitinib in comparison to placebo, as measured by differences in the Total Improvement Score (TIS). After 52 weeks of double-blind treatment, participants have the option to continue therapy in a 52 week open-label extension phase where all participants will receive brepocitinib.
Interventions / DRUG: Placebo or Brepocitinib (1:1 randomization) – oral, daily
Trial information: Enrollment: 241 participants; Study Start Date: 2022-10-31; Primary Completion Date: 2025-07-17; Study Completion Date: 2026-07
Eligibility Criteria: Age Range: 18 years to 75 years; Sex: ALL
Study Status: Active – Not Recruiting
Phase: PHASE3
Sponsor& Collaborators: Priovant Therapeutics, Inc.
Johns Hopkins Locations: Johns Hopkins Hospital: Division of Rheumatology
Coordinator / Contact: Zuzana Bologna / E-mail: jh.myositis.research@jh.edu
Principal Investigator: Julie Paik, MD, MPH
Our Oncology partners are recruiting for the following study
Nivolumab in Treating Patients with Autoimmune Disorders and Advanced, Metastatic, or Unresectable Cancer
NCT number: NCT03816345
Conditions: Autoimmune Disease, Crohn Disease, Dermatomyositis, Hematopoietic and Lymphoid Cell Neoplasm, Inflammatory Bowel Disease, Malignant Solid Neoplasm, Multiple Sclerosis, Psoriasis, Psoriatic Arthritis, Rheumatoid Arthritis, Sjogren Syndrome, Systemic Lupus Erythematosus, Systemic Scleroderma, Ulcerative Colitis
Purpose of Study: This phase Ib trial studies the side effects of nivolumab and to see how well it works in treating patients with autoimmune disorders and cancer that has spread to other places in the body or cannot removed by surgery. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body’s immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.
Interventions / DRUG: PROCEDURE: Biospecimen Collection – Undergo collection of blood, CSF, tissue, stool and urine samples; BIOLOGICAL: Nivolumab – Given IV
Trial Information: Enrollment: 300 participants; Study Start Date: 2019-07-16; Primary Completion Date: 2028-03-30; Study Completion Date: 2028-03-30
Eligibility Criteria: Age Range: 18 years to N/A; Sex: ALL
Study Status: Recruiting
Phase: PHASE1
Sponsor & Collaborators: National Cancer Institute (NCI)
Johns Hopkins Locations: Johns Hopkins University/Sidney Kimmel Cancer Center; Johns Hopkins Hospital: Division of Rheumatology
Coordinator / Contact:
Principal Investigator:
Prior studies the Johns Hopkins Myositis Center participated in
A Study to Evaluate the Efficacy and Safety of ABC008 for Inclusion Body Myositis
NCT number: NCT05721573
Conditions: Inclusion Body Myositis
Purpose of Study: A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of ABC008 in the Treatment of Subjects with Inclusion Body Myositis
Interventions / DRUG: ABC008 – Given by subcutaneous injection
Trial Information: Enrollment: 272 participants; Study Start Date: 2023-02-28; Primary Completion Date: 2025-11-06; Study Completion Date: 2025-11-27
Eligibility Criteria: Age Range: 40 years to N/A; Sex: ALL
Study Status: Completed (Now in longterm extension phase)
Phase: PHASE2, PHASE3
Sponsor & Collaborators: Lead Sponsor: Abcuro, Inc.; Collaborators: Syneos Health
Johns Hopkins Locations: Johns Hopkins Bayview Medical Center; Johns Hopkins Bayview Medical Center
Coordinator / Contact:
Principal Investigator:
Phase III Trial of Sirolimus in IBM
NCT number: NCT04789070
Conditions: Inclusion Body Myositis
Purpose of Study: The hypothesis is that Sirolimus, (Rapamycin (R)) which is currently used in organ transplantation and works by blocking the activity of T effector cells but preserving T regulatory cells, as well as by inducing autophagy (protein degradation), will be effective in IBM to slow or stabilize disease progression, helping to maintain patient function and independence. This phase III trial will confirm pilot data showing statistically significant clinical outcomes.
Interventions / DRUG: Sirolimus – Sirolimus is a currently licensed drug primarily used for immunosuppression post-kidney transplantation to prevent organ rejection. Sirolimus was initially considered as a treatment in IBM for its imm; Placebo
Trial Information: Enrollment: 140 Participants; Study Start Date: 2022-07-01; Primary Completion Date: 2027-12; Study Completion Date: 2027-12
Eligibility Criteria: Age Range: 45 years to N/A; Sex: ALL
Study Status: Active – Not Recruiting
Phase: PHASE3
Sponsor & Collaborators: Lead Sponsor: University of Kansas Medical Center; Collaborators: The Perron Institute
Johns Hopkins Locations:
Coordinator / Contact:
Principal Investigator:
A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants with Active Idiopathic Inflammatory Myopathy.
NCT number: NCT05523167
Conditions: Active Idiopathic Inflammatory Myopathy, Myositis, Dermatomyositis, Polymyositis, Immune-Mediated Necrotizing Myopathy, Antisynthetase Syndrome
Purpose of Study: This study’s purpose is to measure the treatment response from efgartigimod PH20 SC compared with placebo in participants with Idiopathic Inflammatory Myopathy (IIM). Participants with the IIM subtypes of dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM), or certain other subtypes of polymyositis (PM; including antisynthetase syndrome [ASyS]) will be included in the study. Treatment response will be measured by Total improvement score (TIS). Additional information can be found on https://myositis-study.com/.
Interventions / DRUG: BIOLOGICAL: EFG PH20 SC – Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer; OTHER: PBO – Subcutaneous injection of placebo coformulated with rHuPH20, a permeation enhancer
Trial Information: Enrollment: 265 participants; Study Start Date: 2022-10-12; Primary Completion Date: 2026-12-01; Study Completion Date: 2027-02-01
Eligibility Criteria: Age Range: 18 years to N/A; Sex: ALL
Study Status: Active- Not Recruiting
Phase: PHASE2, PHASE3
Sponsor & Collaborators: Argenx
Johns Hopkins Locations: Johns Hopkins Medicine – Johns Hopkins Myositis Center
Coordinator / Contact:
Principal Investigator:
A Study of Nipocalimab in Participants with Active Idiopathic Inflammatory Myopathies
NCT number: NCT05379634
Conditions: Myositis
Purpose of Study: The purpose of this study is to evaluate the efficacy and safety of Nipocalimab versus placebo in participants with active idiopathic inflammatory myopathies (IIM).
Interventions / DRUG: DRUG: Nipocalimab – Nipocalimab will be administered intravenously in double-blind period and LTE period; DRUG: Glucocorticoids – Prednisone or equivalent will be administered orally as Glucocorticoid; OTHER: Placebo – Nipocalimab matching placebo will be administered intravenously in double-blind period.
Trial Information: Enrollment: 36 participants; Study Start Date: 2022-07-05; Primary Completion Date: 2025-09-04; Study Completion Date: 2026-09-16
Eligibility Criteria: Age Range: 18 years to N/A; Sex: ALL
Study Status: Active – Not Recruiting
Phase: PHASE2
Sponsor & Collaborators: Janssen Research & Development, LLC
Johns Hopkins Locations:
Coordinator / Contact:
Principal Investigator:
