A Phase II Trial to Assess the Efficacy, Safety and Feasibility of 20[percnt] Subcutaneous Immunoglobulin in Patients with Myasthenia Gravis Exacerbation - Trial Design (P3.169)
Bibliographic record
Abstract
OBJECTIVE: To present the clinical trial design of a phase II investigator initiated multi-center open label study to assess feasibility, safety and efficacy of 20[percnt] SCIg (Hizentra) in patients with moderately severe myasthenia gravis (MG). BACKGROUND: Intravenous immunoglobulin (IVIg) is an effective therapy for patients with worsening MG. Recently, the subcutaneous form of immunoglobulin (20[percnt] SCIG; Hizentra) has become available and has been used in neuromuscular conditions amenable to IVIg therapy. SCIg appears to be associated with fewer adverse effects, comparable efficacy and has the added advantage of flexibility due to self-infusion by the patients. The use of SCIg has not been systematically studied in patients with MG. DESIGN/METHODS: This is a two-year multicenter open label, single phase, investigator-initiated phase II clinical trial in 30 patients with moderately severe MG (MGFA Grade II and III). Enrolled subjects are trained to self-infuse 20[percnt] SCIg at 2gm/kg over 3 to 4 weeks in a dose escalating manner. Automated pumps allow accurate and convenient administration of the study drug concurrently at four infusion sites. The dosing regimen is flexible allowing two to four infusions per week. The primary efficacy outcome measure is the change in the QMG Score for Disease Severity from baseline to day 42 (6 weeks). In addition, MG Composite, MG-ADL, SF36, infusion related adverse effects, laboratory monitoring for safety and patient satisfaction with SCIg treatment will be assessed. The trial was approved by Health Canada and the local institution ethics board. RESULTS: The trial started in January 2015 and data collection is complete for eight of thirty expected patients. Two more Canadian sites are being initiated. CONCLUSIONS: We anticipate providing an update on the overall progress of this ongoing clinical trial.
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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 | 0.004 | 0.002 |
| Meta-epidemiology (narrow) | 0.002 | 0.000 |
| Meta-epidemiology (broad) | 0.002 | 0.001 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.001 | 0.001 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.001 | 0.000 |
| Research integrity | 0.002 | 0.003 |
| Insufficient payload (model declined to judge) | 0.008 | 0.001 |
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".