Abstract Details

Title Response Rates in Placebo arm of MG Clinical Trials

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P5 - Poster Session 5 (5:30 PM-6:30 PM)

Poster/Presentation
Number 12-025

Objective To analyze the extent of improvement in reported outcomes measures in patients in the placebo groups in Myasthenia Gravis (MG) clinical trials

Background Various immunosuppressive agents used in MG clinical practice have failed to meet primary end points in randomized double blind placebo controlled clinical trials. Patients in the placebo group of MG clinical trials are treated with prednisone and other standard of care therapies. Clinical improvement from these therapies within the placebo group could have led to the failure of research trials to meet primary end points. Many MG outcomes measures are used in multiple randomized clinical trials allowing for comparison of clinical improvement across studies.

Design/Methods

We reviewed published manuscripts of all randomized double blind clinical trials in myasthenia gravis that used the quantitative MG (QMG) and MG-Activities of daily living (MG-ADL) scales. We analyzed the improvements in clinical outcome measures and evaluated clinical trial phase, mode of drug/placebo administration and duration of the clinical trails. We also reviewed the inclusion criteria and whether MG patients were refractory at the time of enrollment.

Results We analyzed 8 recent MG clinical trails evaluating 5 different therapies. Clinical trial duration ranged from 3 to 12 months. Three trials required refractory MG status before recruitment. QMG was used as a primary or secondary end point in 8 trials and MG-ADL was used as a primary or secondary end point in 7 clinical trials. In the placebo groups, the mean QMG score improvement was 2.24 (-0.3-3.67) and mean MG-ADL score improvement was 2.03 (0.6-2.8).

Conclusions

Average improvement in QMG and MG-ADL scores in the placebo arm of MG trials meets criteria for clinically significant change. Future clinical trials in MG should consider this observation and ascertain goals for primary and secondary end points for clinical trials accordingly.

Authors/Disclosures
Srikanth Muppidi, MD, FAAN PRESENTER	Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for argenx. Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB/Ra Pharma. Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Horizont Pharma. Dr. Muppidi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for J & J pharma. Dr. Muppidi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dianthus Pharma. Dr. Muppidi has received publishing royalties from a publication relating to health care.
Verena C. Samara, MD (Carson Tahoe Medical Group)	An immediate family member of Dr. Samara has received personal compensation for serving as an employee of Supernus. The institution of Dr. Samara has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. The institution of Dr. Samara has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Argenx. The institution of Dr. Samara has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for UCB. The institution of Dr. Samara has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Johnson and Johnson. An immediate family member of Dr. Samara has stock in Supernus.
Kathie Lin, MD (Stanford Neuromuscular Department)	Dr. Lin has nothing to disclose.
Neelam Goyal, MD, FAAN (Stanford University)	Dr. Goyal has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx. Dr. Goyal has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Goyal has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Janssen. Dr. Goyal has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. The institution of Dr. Goyal has received research support from Argenx.

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