Phase II Trial of Methotrexate in Myathenia Gravis (S16.002)
Bibliographic record
Abstract
OBJECTIVE: To determine if oral methotrexate is an effective therapy for prednisone dependent Myasthenia Gravis (MG) patients. BACKGROUND: Current effective medications for MG based on randomized controlled trials are azathioprine, cyclosporine and IVIG. Oral methotrexate is an inexpensive immunosuppressive agent that deserves to be studied in MG. DESIGN/METHODS: Randomized, double-blind, placebo-controlled multi-center trial of methotrexate versus placebo in MG patients requiring at least 10mg/day of prednisone or more prior to enrollment. Prednisone dose was adjusted during the study based on clinical response. Treatment allocation was to methotrexate 20 mg/week versus placebo in a 1:1 ratio. Clinical and laboratory evaluations were performed monthly for 12 months. The primary efficacy measure is 9 month prednisone area under the curve. Secondary outcome measures include MGADL, MGComposite and QMG. Twenty patients in each study arm provides .8 power of detecting .784 effect size (mean change/SD), which is equivalent to a 31.4% reduction in total prednisone doses in 9 months for the methotrexate versus placebo. RESULTS: Fourteen US sites and 1 Canadian site participated in this study. Fifty-eight patients were screened, 51 enrolled, 7 screen-failed and 4 active cases are completing by 12/2013. Male-to-female ratio was 2:1 with mean age 67±13 years. Seven patients had Myasthenia Gravis Foundation Association class of 3 at baseline, 44 class 2. Overall QMG score at baseline is 10.2+4.1. Total adverse events (AE) was 326 in 44 patients: unrelated 213, possibly/probably/definitely related 90, unknown 23. Serious AE’s were 19, all unrelated to study medication. Majority of AE’s were mild, CTCAE version 3.0 grade 1(187 events). Seven patients (17 events) had elevated LFT’s. Presently the study is still blinded. CONCLUSIONS: The study medication has been safe. The study will be completed by 12/2013. Unblinding and analysis will be performed at the end of the study and results will be presented at the meeting. Study Supported by: CTSA Grant No: UL1TR000001Funded/FDA-OPD: RO1FD003538
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.002 | 0.001 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.001 | 0.000 |
| Open science | 0.001 | 0.000 |
| Research integrity | 0.001 | 0.002 |
| Insufficient payload (model declined to judge) | 0.010 | 0.002 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".