Trial of Orencia in Patients With Myasthenia Gravis

NCT ID: NCT03059888

Last Updated: 2019-11-22

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-04-12
• Study Completion Date: 2019-07-15

Brief Summary

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.

Conditions

Myasthenia Gravis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Abatacept

125mg abatacept in 1ml solution administered once per week by subcutaneous injection

Group Type: EXPERIMENTAL

Abatacept Injection

Intervention Type: DRUG

Subcutaneous administration of abatacept

Interventions

Abatacept Injection

Subcutaneous administration of abatacept

Intervention Type: DRUG

Other Intervention Names

Orencia

Eligibility Criteria

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

• Minimum Eligible Age: 16 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Bristol-Myers Squibb

INDUSTRY

Sponsor Role: collaborator

Johns Hopkins University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Daniel Drachman, MD

Role: PRINCIPAL_INVESTIGATOR

Johns Hopkins University

Locations

Johns Hopkins University

Baltimore, Maryland, United States

Countries

United States

Other Identifiers

IRB00100059

Identifier Type: -

Identifier Source: org_study_id