Efficacy of Prednisone In the Treatment of Ocular Myasthenia

NCT ID: NCT00995722

Last Updated: 2017-06-02

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 11 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-12-31
• Study Completion Date: 2013-10-31

Brief Summary

The purpose of this study is to evaluate the efficacy and tolerability of prednisone in patients diagnosed with ocular myasthenia.

Funding Source - FDA OOPD

Detailed Description

The purpose of this study is to learn two things about prednisone in patients with ocular myasthenia. The first thing we aim to learn is whether or not prednisone is effective in improving the symptoms of double vision and drooping eyes that are experienced by patients with ocular myasthenia. The second thing we aim to learn is whether we can find a dose of prednisone that is well tolerated and safe. The overall goal is to find out whether a dose of prednisone that is safe and well tolerated is also effective in improving the symptoms of ocular myasthenia.

After completing screening assessments to confirm eligibility, all participants will receive treatment with pyridostigmine. If a participant's symptoms do not resolve within the first month while being treated with pyridostigmine, they will be randomized to receive prednisone or placebo. The amount of study medication a participant receives will depend on how their symptoms respond to the medication and if they experience any side effects.

After four months, participants that continue to have symptoms of ocular myasthenia and do not have side effects will receive open label high dose prednisone. Participants that no longer have symptoms will taper their dose of study drug in a double-blind fashion.

Conditions

Ocular Myasthenia Gravis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Prednisone + Pyridostigmine

Corticosteroid

Group Type: EXPERIMENTAL

Prednisone

Intervention Type: DRUG

Prednisone will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain 10mg of prednisone.

Placebo + Pyridostigmine

Matched, inactive substance

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo dosages will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain matching placebo.

Interventions

Prednisone

Prednisone will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain 10mg of prednisone.

Intervention Type: DRUG

Placebo

Placebo dosages will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain matching placebo.

Intervention Type: DRUG

Other Intervention Names

Steroid; Sugar pill

Eligibility Criteria

Inclusion Criteria

• Weakness confined to the extra-ocular muscles, eyelid levator and eye closure with an ocular-QMG1 score ≥ 1
• At least one of the following combinations of abnormal diagnostic testing: a) Elevated acetylcholine receptor antibody titers, (b) Abnormal repetitive nerve stimulation (> 10% decrement following slow repetitive nerve stimulation) of any nerve-muscle pair, (c) Abnormal jitter on single fiber or concentric needle electromyography in any muscle, (d) Positive ice test and brain MRI that demonstrates no central nervous system pathology that mimics ocular myasthenia, or (e) Positive Tensilon test and brain MRI that demonstrate no central nervous system pathology that mimics ocular myasthenia
• Either no prior treatment with pyridostigmine, or participant has persistent ocular symptoms that are functionally limiting or troublesome despite treatment with pyridostigmine.
• Age 18 years or older, male or female
• Capable of providing informed consent and complying with study procedures
• Identifiable primary care physician to assist with management of medical complications that may arise as a consequence of steroid therapy
• Willing to be randomized to a trial of prednisone or placebo if symptoms respond inadequately to pyridostigmine.

Exclusion Criteria

• Disease duration (time since symptom onset) > 5 years
• Treatment with prednisone or other corticosteroids within 90 days of randomization
• Treatment with azathioprine, cyclosporine, mycophenolate mofetil or other immune suppressive medication since onset of MG unless dosages of these medications and/or duration of therapy with these medications are clinically insignificant in the judgment of the PI
• Intravenous immunoglobulin or plasma exchange within 90 days of randomization
• Prior thymectomy or history of thymoma
• Contraindication to steroids (poorly controlled diabetes, glaucoma or hypertension, history of prior steroid intolerance, obesity [BMI > 39.9kg/m2] or a history of osteoporotic fracture)
• Pregnant or lactating
• Renal failure, active thyroid or hepatocellular disease, chronic infection, poorly controlled cardiac disease, unstable psychiatric illness, untreated major depression or any other illness that would, in the opinion of the treating neurologist, make it unsafe for the patient to participate in the trial
• Receipt of another investigational drug within 30 days of Screening

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Miami

OTHER

Sponsor Role: collaborator

University of Rochester

OTHER

Sponsor Role: collaborator

Michael Benatar

OTHER

Sponsor Role: lead

Responsible Party

Michael Benatar

MBChB, DPhil

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Michael Benatar, MBChB, DPhil

Role: PRINCIPAL_INVESTIGATOR

University of Miami

Gil Wolfe, MD

Role: STUDY_DIRECTOR

State University of New York at Buffalo

Donald Sanders, MD

Role: STUDY_DIRECTOR

Duke University

Locations

Yale University

New Haven, Connecticut, United States

University of Miami

Miami, Florida, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

University at Buffalo, Buffalo General Medical Center

Buffalo, New York, United States

Duke University Medical Center

Durham, North Carolina, United States

University of Texas Southwestern Medical Center

Dallas, Texas, United States

University of Vermont

Burlington, Vermont, United States

University of Virginia

Charlottesville, Virginia, United States

University of Alberta Hospital

Edmonton, Alberta, Canada

Toronto General Hospital

Toronto, Ontario, Canada

Countries

United States; Canada

Other Identifiers

FD-R-03710-01

Identifier Type: -

Identifier Source: org_study_id