A single arm study to investigate OCS tapering in adult participants with generalized Myasthenia Gravis treated with ravulizumab

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Alexion Pharmaceuticals Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Decision date (initial)

2026-06-08

Transition trial

No

Low-intervention

Yes

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Alexion Pharmaceuticals, Inc.

External identifiers

EU CT number

2025-522555-26-00

ClinicalTrials.gov

NCT07221838

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To assess the effectiveness of a predefined Oral corticosteroids (OCS) tapering schedule in adult participants with Generalized myasthenia gravis (gMG) treated with ravulizumab and OCS

Secondary objectives 4

1. To assess OCS discontinuation using a predefined OCS tapering schedule in adult participants with gMG treated with ravulizumab and OCS
2. To assess the change from baseline in OCS daily dose in adult participants with gMG treated with ravulizumab and a predefined OCS tapering schedule
3. To assess the impact on daily functions and quality of life maintained by predefined OCS tapering schedule in adult participants with gMG treated with ravulizumab and OCS
4. To evaluate corticosteroid toxicity after OCS reduction in adult participants with gMG treated with ravulizumab and OCS

Conditions and MedDRA coding

Generalized Myasthenia Gravis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. Aged ≥ 18 years at the time of enrollment.
2. Clinical diagnosis of gMG.
3. Receiving ravulizumab treatment prior to enrollment, in accordance with the locally approved label
4. Receipt of OCS therapy equivalent to a daily dose ≥ 7.5 mg of prednisone/prednisolone for ≥ 4 continuous weeks directly preceding enrollment. - Alternative dosing of OCS (such as every other day) is allowed at enrollment, provided the average daily dose per week is equivalent to ≥ 7.5 mg of prednisone/prednisolone and the participant is willing to switch to a daily intake of prednisone/prednisolone at Baseline/Visit 1. - The use of other OCS (e.g., methylprednisolone) is allowed prior to enrolment provided the participant is willing to switch to a daily intake of prednisone/prednisolone at Baseline/Visit 1
5. Male or female assigned at birth
6. Agrees to follow protocol-specified contraception guidance as outlined in Section 10.5
7. 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, see Section 8.3.3 Pregnancy Testing. • Additional requirements for pregnancy testing at Visit 3 are located in Section 8.3.3 Pregnancy Testing
8. Willing to provide informed consent as described in Section 10.1.3.

Exclusion criteria 5

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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Proportion of adult participants with gMG who either: • Discontinue OCS (0 mg/day), or • Reduce their daily OCS dosage ≤ 5 mg/day, if the reason for no further OCS reduction is suspected adrenal insufficiency and maintain this status for ≥ 4 weeks without clinical deterioration of gMG

Secondary endpoints 7

1. Proportion of adult participants with gMG who discontinue OCS (0 mg/day), sustained ≥ 4 weeks without clinical deterioration of gMG
2. Percentage change from daily OCS dose (mg/day) at baseline to the daily OCS dose (mg/day) at 4 weeks after OCS tapering is complete
3. Change from Baseline to 4 weeks after OCS tapering is complete in the MG-QoL-15r among adult participants with gMG • Change from Baseline to 4 weeks after OCS tapering is complete in MG-ADL total score among adult participants with gMG
4. Summary of GTI-MD including Cumulative Worsening Score and Aggregate Improvement Score at 4 weeks after OCS tapering is complete as assessed by BMI, glucose intolerance, blood pressure, and low-density lipoprotein collected at Baseline and at 4 weeks after OCS tapering is complete among adult participants with gMG
5. Proportion of adult participants with gMG with MG clinical deterioration events (including crises, exacerbations, MG-related hospitalization) at any time over the follow-up period • Proportion of adult participants with gMG with suspected adrenal insufficiency, based on symptoms as assessed at any time during the follow-up period
6. Proportion of adult participants with gMG with adrenal insufficiency confirmed by morning cortisol • Incidence of adverse events and serious adverse events among adult participants with gMG after initiation of reduction in OCS dose • Change from Baseline to 4 weeks after OCS tapering completion in laboratory parameters including HbA1c and LDL-C in adult participants with gMG
7. Summary of the Social Impact and Sleep questionnaire at 4 weeks after OCS tapering is complete among adult participants with gMG.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 7

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Alexion Pharmaceuticals Inc.

Sponsor organisation

Alexion Pharmaceuticals Inc.

Address

121 Seaport Boulevard

City

Boston

Postcode

02210-2050

Country

United States

Scientific contact point

Organisation

Alexion Pharmaceuticals Inc.

Contact name

European Clinical Trial Information

Public contact point

Organisation

Alexion Pharmaceuticals Inc.

Contact name

European Clinical Trial Information

Third parties 9

Locations

2 EU/EEA countries · 3 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 36 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications