Ocular Myasthenia Gravis
Conditions
Keywords
Phase 3, oMG, rozanolixizumab
Brief summary
The purpose of the study is to assess the long-term safety and efficacy of rozanolixizumab in adult study participants with ocular myasthenia gravis.
Interventions
DRUGRozanolixizumab
Rozanolixizumab will be administered by subcutaneous infusion.
Sponsors
UCB Biopharma SRL
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participant must be a minimum of 18 years of age inclusive at the time of signing the informed consent form (ICF) * Participant must have received at least 1 dose of investigational medicinal product (IMP) (rozanolixizumab or placebo) in MG0038 * Participant for whom the investigator considers a favorable benefit/risk for participation * Participant who, alone or with assistance of the caregiver, is considered reliable and capable of adhering to the protocol visit schedule or medication intake according to the judgement of the investigator * Male or female * A female participant is eligible to participate if she is not, not breastfeeding (including pumping breastmilk to feed a child), and at least 1 of the following conditions applies: * Not a woman of childbearing potential (WOCBP) OR * A WOCBP who agrees to follow the contraceptive guidance for the entire study period * Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in the protocol
Exclusion criteria
* Participant meets any of the withdrawal criteria defined in MG0038 * Participant has a clinically relevant active infection (resulting in hospitalization or requiring intravenous (IV) antibiotic treatment) within 6 weeks before study entry. * Participant intends to have a live vaccination during the study or within 8 weeks following the final dose of rozanolixizumab * Participant has been administered with the prohibited immunosuppressive medications, biologics, or other therapies * Participant has planned use of prohibited medications
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of treatment-emergent adverse events (TEAEs) | Up to 2 years | Treatment Emergent Adverse Events (TEAEs) are any untoward medical incidence in a participant after the administration of study treatment, whether or not these events are related to study treatment. |
| Incidence of treatment-emergent serious adverse events (TESAEs) | Up to 2 years | An SAE is defined as any untoward medical occurrence that, at any dose, meets 1 or more of the criteria listed: * Results in death * Is life-threatening * Requires inpatient hospitalization or prolongation of existing hospitalization * Results in persistent or significant disability/incapacity * Is a congenital anomaly/birth defect * Other important medical events which based on medical or scientific judgement may jeopardize the patients or may require medical or surgical intervention to prevent any of the above. |
| Incidence of TEAEs leading to permanent withdrawal of study treatment | Up to 2 years | Treatment Emergent Adverse Events (TEAEs) are any untoward medical incidence in a participant after the administration of study treatment, whether or not these events are related to study treatment. This measure considers any TEAE leading to permanent withdrawal from study. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline at Day 43 in (Myasthenia Gravis Impairment Index) MGII ocular score (Patient-Reported Outcome part) | At Day 43 (of the first 3 Symptom-driven Cycles) | MGII is a measure of disease severity based on the signs and symptoms of MG patients. The MGII has 22 patient-reported items and 6 examination items and scores are presented as a sum of all items for a total score but also as an ocular and generalized sub-score. The scoring range is 0 to 84, 0-23 for the ocular score, and 0-61 for the generalized score, where higher scores are indicative of more severe symptoms. The recall period is "the past week". |
| Change from Baseline at Day 43 in Myasthenia Gravis Activities of Daily Living (MG-ADL) ocular score | At Day 43 (of the first 3 Symptom-driven Cycles) | The MG-ADL score is an 8-item patient-reported outcome (PRO) instrument. The MG-ADL targets symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The item responses are scored from 0 to 3, and the total score of MG-ADL is the sum of the 8 items and ranges from 0 to 24, with a higher score indicating more disability. The two ocular items will be summed for the ocular score, ranging from 0 to 6. |
| Change from Baseline at Day 43 in Myasthenia Gravis Symptoms Patient-Reported Outcome (MGSPRO) ocular muscle weakness scale score | At Day 43 (of the first 3 Symptom-driven Cycles) | The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (items 1-5); bulbar muscle weakness (items 6-15); respiratory muscle weakness (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42). Only the ocular muscle weakness scale will be used in this study. Ocular symptom severity is assessed over the past 7 days using a 4-point verbal rating scale (none, mild, moderate, severe) |
| Change from Baseline at Day 43 in Myasthenia Gravis Quality of Life 15-item Scale (revised version) (MG-QoL15r) total score | At Day 43 (of the first 3 Symptom-driven Cycles) | The MG-QoL15r is a 15-item PRO measure that is designed to assess aspects of health-related quality of life related to MG. The recall period that will be used is "past 7 days". Each item has 3 response options (0-2) and the total score ranges from 0 to 30, with higher scores indicating more severe impact on health-related QoL (HRQOL). |
| Study participant who generalizes to Myasthenia Gravis Foundation of America (MGFA) class ≥2 over time | Up to 2 years | MGFA Clinical Classification is a 5-stage classification (I to V), with a higher class indicating more severe disease. |
| Change from Baseline in MGII total and generalized score over time | Up to 2 years | MGII is a measure of disease severity based on the signs and symptoms of MG patients. The MGII has 22 patient-reported items and 6 examination items and scores are presented as a sum of all items for a total score but also as an ocular and generalized sub-score. The scoring range is 0 to 84, 0-23 for the ocular score, and 0-61 for the generalized score, where higher scores are indicative of more severe symptoms. The recall period is "the past week". |
| Change from Baseline in MG-ADL total score over time | Up to 2 years | The MG-ADL score is an 8-item patient-reported outcome (PRO) instrument. The MG-ADL targets symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The item responses are scored from 0 to 3, and the total score of MG-ADL is the sum of the 8 items and ranges from 0 to 24, with a higher score indicating more disability. The two ocular items will be summed for the ocular score, ranging from 0 to 6. |
Contacts
CONTACTUCB Cares
Outcome results
None listed