Trial of Orencia in Patients With Myasthenia Gravis

Johns Hopkins University

Status and phase

Terminated

Early Phase 1

Conditions

Myasthenia Gravis

Treatments

Drug: Abatacept Injection

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT03059888

IRB00100059

Details and patient eligibility

About

This pilot research study is being done to see if the drug abatacept (Orencia ®) will be helpful in treating patients with myasthenia gravis (MG) who do not respond satisfactorily to other drugs that are used to suppress the immune system. Abatacept has been successful in treating experimental MG in laboratory animals, and this study is to determine its effectiveness in patients with MG.

Enrollment

6 patients

Sex

All

Ages

16 to 85 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Able to comprehend and willing to sign an Informed Consent Form (ICF)
Male or female 16 to 85 years of age
Diagnosis of DEFINITE Myasthenia Gravis
History of inadequate response to conventional MG treatment
Clinical laboratory findings within the normal range or, if outside the normal range, deemed not clinically significant by the Investigator
Female patients of childbearing potential who are not be breastfeeding, have a negative pregnancy test, have no intention to become pregnant during the course of the study, and are using contraceptive drugs or devices to prevent pregnancy during their participation in the study
Willing to cooperate with study requirements, including visits to the study site every 2 months.

Exclusion criteria

Subject's MG is responding adequately to conventional immunosuppressive treatment
Subject has had no previous trial of other immunosuppressive agents
Subject has a history of Chronic Obstructive Pulmonary Disease (COPD)
Subject is impaired, incapacitated or incapable of completing study-related assessments
Subject has the presence at screening of any severe, progressive or uncontrolled renal, hepatic, hematologic, gastrointestinal, pulmonary, cardiac, neurologic or cerebral disease, whether or not related to MG that, in the opinion of the Investigator, might place the subject at unacceptable risk for participation in the study.
Female subject has a history of breast cancer screening that is suspicious for malignancy and in whom the possibility of malignancy cannot be reasonably excluded by additional clinical, laboratory or other diagnostic evaluations
Subject has a history of cancer in the last 5 years, other than non-melanoma skin cell cancers cured by local resection or carcinoma in situ
Subject currently abuses drugs or alcohol
Subject has evidence of active or latent bacterial or viral infections, including positive infectious disease laboratory test result (Hepatitis B, Hepatitis C or HIV)
Subject has history of herpes zoster or cytomegalovirus (CMV) infection that resolved less than 2 months prior to screening visit
Subject has received any live vaccine within 3 months of screening
Subject has a history of serious bacterial infection within in the last 3 months, unless treated and resolved with antibiotics; or, any chronic bacterial infection
Subject is at risk for tuberculosis
Subject has any of the following laboratory values: Hemoglobin < 8.5 g/dL; White Blood Cells (WBC) < 3000 mm3; Platelets < 100,000 mm3; Serum creatinine > 2x upper limit of normal (ULN); Serum Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST) > 2x ULN
Subject is unable or unwilling to maintain weekly injection schedule
Subject has undergone a change in immunosuppressive medications within the last three months prior to enrollment.
Subject has neurological impairment due to a condition other than MG, including history of transient ischemic attack within the past year
Subject has taken any investigational study drug within 28 days or five half-lives of the prior agent, whichever is greater, prior to dosing
Subject has had previous exposure to Orencia (abatacept)
Subject is a prisoner or is involuntarily incarcerated
Subject is compulsorily detained for treatment of either psychiatric or physical illness
Subject is judged to be actively suicidal or a suicide risk by the Investigator