Oral steroid tapering for adults with generalized myasthenia gravis on ravulizumab.

OCTAGON: A Phase 4, Single Arm Study to Investigate Oral Corticosteroid Tapering in Adult Patients With Generalized Myasthenia Gravis Treated With Ravulizumab

This study will try a predefined prednisone taper to see if adults with generalized myasthenia gravis on ravulizumab can safely reduce their daily steroid dose.

Quick facts

• Phase: PHASE4
• Study type: Interventional
• Enrollment: 75 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Alexion Pharmaceuticals, Inc. (industry)
• Drugs / interventions: ravulizumab, rozanolixizumab, inebilizumab, rituximab, prednisone
• Locations: 12 sites (Chicago, Illinois and 11 other locations)
• Trial ID: NCT07221838 on ClinicalTrials.gov

What this trial studies

This is a prospective, multicenter, single-arm Phase 4 study of adults with acetylcholine receptor–positive generalized myasthenia gravis who are already receiving ravulizumab. Eligible participants must have been taking at least 7.5 mg/day prednisone (or equivalent) for at least four continuous weeks prior to enrollment and will follow a predefined oral corticosteroid taper while continuing ravulizumab. The study will track steroid dose reduction, disease control, and adverse events during and after the taper. Data collected at U.S. research sites will inform whether this tapering approach can be implemented safely in routine care.

Who should consider this trial

Good fit: Adults (≥18 years) with AChR-positive generalized myasthenia gravis who are already receiving ravulizumab and have been on at least 7.5 mg/day prednisone (or equivalent) for four continuous weeks are the intended participants.

Not a fit: Patients using oral steroids for other medical conditions, those with chronic hypoadrenalism, pregnant or breastfeeding individuals, or those who recently received other biologic gMG therapies are unlikely to benefit or may be ineligible.

Why it matters

Potential benefit: If successful, the taper could let patients lower their steroid doses and reduce long-term steroid-related side effects while maintaining disease control.

How similar studies have performed: Complement C5 inhibitors like eculizumab and ravulizumab have improved gMG symptoms and have been associated with steroid-sparing in some reports, but a prospective predefined OCS taper specifically with ravulizumab is relatively novel.

Eligibility criteria

Inclusion Criteria:

* Aged greater than 18 years and male or female
* Clinical diagnosis of gMG
* Receiving ravulizumab treatment prior to enrollment
* Receipt of OCS therapy equivalent to a daily dose ≥ 7.5 mg of prednisone/prednisolone for ≥ 4 continuous weeks directly preceding enrollment
* A participant of childbearing potential must have a negative highly sensitive pregnancy test (serum or urine, as required by local regulations) taken at Screening before OCS tapering begins.
* Willing to sign informed consent

Exclusion Criteria:

* Concurrent participation in an interventional clinical trial.
* History of chronic hypoadrenalism (ie, Addison's disease).
* Use of concomitant OCS for comorbid conditions other than gMG
* Receipt of a biologic for gMG (e.g., efgartigimod, rozanolixizumab, inebilizumab, rituximab, intravenous immunoglobulin) within 5 half-lives of enrollment
* Pregnant, breastfeeding, or intending to conceive during the course of the study

Where this trial is running

Chicago, Illinois and 11 other locations

• Research Site — Chicago, Illinois, United States (NOT_YET_RECRUITING)
• Research Site — Schaumburg, Illinois, United States (NOT_YET_RECRUITING)
• Research Site — Neptune City, New Jersey, United States (NOT_YET_RECRUITING)
• Research Site — Raleigh, North Carolina, United States (RECRUITING)
• Research Site — Knoxville, Tennessee, United States (NOT_YET_RECRUITING)
• Research Site — Bochum, Germany (NOT_YET_RECRUITING)
• Research Site — Milan, Italy (NOT_YET_RECRUITING)
• Research Site — Naples, Italy (NOT_YET_RECRUITING)
• Research Site — Rome, Italy (NOT_YET_RECRUITING)
• Research Site — Ibaraki, Japan (NOT_YET_RECRUITING)
• Research Site — Kitakyushu-shi, Japan (NOT_YET_RECRUITING)
• Research Site — Kumamoto, Japan (NOT_YET_RECRUITING)

Study contacts

• Study coordinator: Alexion Pharmaceuticals, Inc. (Sponsor)

Conditions: Generalized Myasthenia Gravis, gMG, Generalized myasthenia gravis, Glucocorticoid toxicity, Complement C5 inhibitor, Prednisone reduction, Cortisol monitoring, ravulizumab