Generalized Myasthenia Gravis
Conditions
Keywords
generalized Myasthenia Gravis, gMG, rozanolixizumab
Brief summary
The purpose of this study is to evaluate the ability of study participants with generalized Myasthenia Gravis (gMG) to successfully self-administer rozanolixizumab after training in the self-administration technique using the syringe driver and manual push methods.
Interventions
DRUGRozanolixizumab
Rozanolixizumab self-administration via Syringe Driver or Manual Push.
Sponsors
UCB Biopharma SRL
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Study participant must have a documented diagnosis of generalized Myasthenia Gravis (gMG) * Study participant is willing to perform and capable of performing home self-administration * Study participant is considered by the investigator for additional rozanolixizumab treatment with the posology proposed in this study. * Body weight ≥35 kg * Study participants may be male or female
Exclusion criteria
* Study participant has a known hypersensitivity to other anti-Fc receptor (FcRn) medications, to any components of the study medication, to any of the excipients (including polysorbate 80), or has a known history of hyperprolinemia, since both polysorbate 80 and L-proline are constituents of the rozanolixizumab formulation * Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current or history of nontuberculous mycobacterial infection (NTMBI) * Study participant has a clinically relevant active infection or a history of serious infection (resulting in hospitalization or requiring IV antibiotic treatment) within 6 weeks before the Baseline Visit * The study participant previously participated in any rozanolixizumab MG study and met any mandatory withdrawal criteria (unless the reason is directly related to MG0020 participation) or mandatory study drug discontinuation criteria. * Study participant has received a live vaccination within 4 weeks before starting treatment, or a Bacillus Calmette-Guérin (BCG) vaccine within 1 year before starting treatment; or intends to have a live vaccination during the course of the study or within 8 weeks following the last dose of rozanolixizumab * Study participant with severe (defined as Grade 3 on the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Successful Self-administration of Rozanolixizumab (With Correct Use of Syringe Driver and Manual Push, Respectively) During the Self-administration Period at Visit 13 (Week 12) | Week 12 (last dose of Self-administration Period 1) | Successful self-administration was defined by the participant (i) choosing the correct infusion site, (ii) administering SC, and (iii) delivering the intended dose. |
| Percentage of Participants With Successful Self-administration of Rozanolixizumab (With Correct Use of Syringe Driver and Manual Push, Respectively) During the Self-administration Period at Visit 19 (Week 18) | Week 18 (last dose of Self-administration Period 2) | Successful Self-administration was defined by the participant (i) choosing the correct infusion site, (ii) administering SC, and (iii) delivering the intended dose. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) After Syringe Driver or Manual Push Self-administration From Visit 2 (Week 1) up to the End of Study Visit (Visit 21 [Week 26]) | From Week 1 up to the End of Study Visit (Week 26) | An Adverse Event (AE) was any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product, which did not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. A TEAE was defined as an AE starting on or after the date of first administration of rozanolixizumab in the study, up to and including 8 weeks (56 days) after the final dose. |
| Percentage of Participants With Local Site Reactions up to 24 Hours After Each Administration During the Training Period and Self-administration Periods | Up to 24 hours after each administration during the Training Period (Baseline to Week 6) and Self-administration Periods (Week 7 to Week 18) | The local site reactions up to 24 hours after each administration were defined as AEs reported as local site reactions as per case report form within one day after RLZ administration. |
| Percentage of Participants With Medication Errors Associated With Adverse Reactions During the 2 Self-administration Periods of the Study | During the Self-administration Periods (Week 7 to Week 18) | Medication errors were defined as an unintended failure in the drug treatment process that leads to, or has the potential to lead to, harm to the study participant. Medication Errors associated with adverse reactions during the 2 Self-administration Periods were measured. |
Countries
Canada, Georgia, Germany, Italy, Japan, Poland, Serbia, Spain, United Kingdom, United States
Participant flow
Recruitment details
The study started to enroll participants in April 2023 and concluded in April 2024.
Pre-assignment details
Participant Flow refers to Safety Set (SS) for Training Period and Randomized Safety Set (RSS) for Self-administration Periods 1 and 2.
Participants by arm
| Arm | Count |
|---|---|
| All Participants: Training Period All participants received weekly doses of subcutaneous rozanolixizumab (RLZ) as per their body weight. During the Training Period, the study participants were trained by healthcare professionals on the subcutaneous self-administration of RLZ using both the Syringe Driver (SRD) and Manual Push (MP) methods for 6 weeks before randomization. | 62 |
| Total | 62 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Self-administration Period 1 (6 Weeks) | Adverse Event | 0 | 1 | 0 |
| Self-administration Period 1 (6 Weeks) | Consent withdrawn by participant (not due to AE) | 0 | 0 | 1 |
| Self-administration Period 1 (6 Weeks) | Diagnosis change to amyotrophic lateral sclerosis | 0 | 1 | 0 |
| Self-administration Period 1 (6 Weeks) | Missed infusion at Visit 13, SA Period1 incomplete | 0 | 2 | 0 |
| Self-administration Period 1 (6 Weeks) | Participant became ineligible for SA | 0 | 1 | 0 |
| Training Period (6-weeks) | Adverse Event | 3 | 0 | 0 |
| Training Period (6-weeks) | Consent withdrawn by participant (not due to AE) | 1 | 0 | 0 |
| Training Period (6-weeks) | Not Eligible for SA but continued in study | 3 | 0 | 0 |
Baseline characteristics
| Characteristic | All Participants: Training Period |
|---|---|
| Age, Continuous | 53.3 years STANDARD_DEVIATION 15.7 |
| Age, Customized 18 - <65 years | 45 Participants |
| Age, Customized 65 - <85 years | 17 Participants |
| Age, Customized >=85 years | 0 Participants |
| Race/Ethnicity, Customized Asian | 5 Participants |
| Race/Ethnicity, Customized Black or African American | 1 Participants |
| Race/Ethnicity, Customized Hispanic or Latino | 5 Participants |
| Race/Ethnicity, Customized Not Hispanic or Latino | 57 Participants |
| Race/Ethnicity, Customized Other/Mixed | 1 Participants |
| Race/Ethnicity, Customized White | 55 Participants |
| Sex: Female, Male Female | 35 Participants |
| Sex: Female, Male Male | 27 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 62 | 0 / 54 | 0 / 53 | 0 / 62 |
| other Total, other adverse events | 19 / 62 | 6 / 54 | 6 / 53 | 24 / 62 |
| serious Total, serious adverse events | 1 / 62 | 3 / 54 | 1 / 53 | 7 / 62 |
Outcome results
Primary
Percentage of Participants With Successful Self-administration of Rozanolixizumab (With Correct Use of Syringe Driver and Manual Push, Respectively) During the Self-administration Period at Visit 13 (Week 12)
Successful self-administration was defined by the participant (i) choosing the correct infusion site, (ii) administering SC, and (iii) delivering the intended dose.
Time frame: Week 12 (last dose of Self-administration Period 1)
Population: The Full Analysis Set (FAS) consisted of all participants who were included in SS, were randomized, and completed both self-administration periods in accordance with the randomization scheme.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Period 1: RLZ SRD | Percentage of Participants With Successful Self-administration of Rozanolixizumab (With Correct Use of Syringe Driver and Manual Push, Respectively) During the Self-administration Period at Visit 13 (Week 12) | 100 percentage of participants |
| Period 1: RLZ MP | Percentage of Participants With Successful Self-administration of Rozanolixizumab (With Correct Use of Syringe Driver and Manual Push, Respectively) During the Self-administration Period at Visit 13 (Week 12) | 100 percentage of participants |
Primary
Percentage of Participants With Successful Self-administration of Rozanolixizumab (With Correct Use of Syringe Driver and Manual Push, Respectively) During the Self-administration Period at Visit 19 (Week 18)
Successful Self-administration was defined by the participant (i) choosing the correct infusion site, (ii) administering SC, and (iii) delivering the intended dose.
Time frame: Week 18 (last dose of Self-administration Period 2)
Population: FAS consisted of all participants who were included in SS, were randomized, and completed both self-administration periods in accordance with the randomization scheme.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Period 1: RLZ SRD | Percentage of Participants With Successful Self-administration of Rozanolixizumab (With Correct Use of Syringe Driver and Manual Push, Respectively) During the Self-administration Period at Visit 19 (Week 18) | 100 percentage of participants |
| Period 1: RLZ MP | Percentage of Participants With Successful Self-administration of Rozanolixizumab (With Correct Use of Syringe Driver and Manual Push, Respectively) During the Self-administration Period at Visit 19 (Week 18) | 100 percentage of participants |
Secondary
Percentage of Participants With Local Site Reactions up to 24 Hours After Each Administration During the Training Period and Self-administration Periods
The local site reactions up to 24 hours after each administration were defined as AEs reported as local site reactions as per case report form within one day after RLZ administration.
Time frame: Up to 24 hours after each administration during the Training Period (Baseline to Week 6) and Self-administration Periods (Week 7 to Week 18)
Population: SS included all study participants who received at least 1 dose of investigational medicinal product (partial or full). Here, number of participants analyzed included those participants who were evaluable for the outcome measure.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Period 1: RLZ SRD | Percentage of Participants With Local Site Reactions up to 24 Hours After Each Administration During the Training Period and Self-administration Periods | 0 percentage of participants |
| Period 1: RLZ MP | Percentage of Participants With Local Site Reactions up to 24 Hours After Each Administration During the Training Period and Self-administration Periods | 0 percentage of participants |
| Period 2: RLZ SRD | Percentage of Participants With Local Site Reactions up to 24 Hours After Each Administration During the Training Period and Self-administration Periods | 0 percentage of participants |
| Period 2: RLZ MP | Percentage of Participants With Local Site Reactions up to 24 Hours After Each Administration During the Training Period and Self-administration Periods | 0 percentage of participants |
| RLZ Total | Percentage of Participants With Local Site Reactions up to 24 Hours After Each Administration During the Training Period and Self-administration Periods | 0 percentage of participants |
Secondary
Percentage of Participants With Medication Errors Associated With Adverse Reactions During the 2 Self-administration Periods of the Study
Medication errors were defined as an unintended failure in the drug treatment process that leads to, or has the potential to lead to, harm to the study participant. Medication Errors associated with adverse reactions during the 2 Self-administration Periods were measured.
Time frame: During the Self-administration Periods (Week 7 to Week 18)
Population: RSS consisted of all participants who were included in SS and were randomized. Here, number of participants analyzed included those participants who were evaluable for the outcome measure.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Period 1: RLZ SRD | Percentage of Participants With Medication Errors Associated With Adverse Reactions During the 2 Self-administration Periods of the Study | 0 percentage of participants |
| Period 1: RLZ MP | Percentage of Participants With Medication Errors Associated With Adverse Reactions During the 2 Self-administration Periods of the Study | 0 percentage of participants |
| Period 2: RLZ SRD | Percentage of Participants With Medication Errors Associated With Adverse Reactions During the 2 Self-administration Periods of the Study | 0 percentage of participants |
| Period 2: RLZ MP | Percentage of Participants With Medication Errors Associated With Adverse Reactions During the 2 Self-administration Periods of the Study | 0 percentage of participants |
Secondary
Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) After Syringe Driver or Manual Push Self-administration From Visit 2 (Week 1) up to the End of Study Visit (Visit 21 [Week 26])
An Adverse Event (AE) was any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product, which did not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. A TEAE was defined as an AE starting on or after the date of first administration of rozanolixizumab in the study, up to and including 8 weeks (56 days) after the final dose.
Time frame: From Week 1 up to the End of Study Visit (Week 26)
Population: SS included all study participants who received at least 1 dose of investigational medicinal product (partial or full). The Randomized Safety Set (RSS) consisted of all participants who were included in SS and were randomized. Here, number of participants analyzed included those participants who were evaluable for the outcome measure.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Period 1: RLZ SRD | Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) After Syringe Driver or Manual Push Self-administration From Visit 2 (Week 1) up to the End of Study Visit (Visit 21 [Week 26]) | 35.7 percentage of participants |
| Period 1: RLZ MP | Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) After Syringe Driver or Manual Push Self-administration From Visit 2 (Week 1) up to the End of Study Visit (Visit 21 [Week 26]) | 29.6 percentage of participants |
| Period 2: RLZ SRD | Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) After Syringe Driver or Manual Push Self-administration From Visit 2 (Week 1) up to the End of Study Visit (Visit 21 [Week 26]) | 26.9 percentage of participants |
| Period 2: RLZ MP | Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) After Syringe Driver or Manual Push Self-administration From Visit 2 (Week 1) up to the End of Study Visit (Visit 21 [Week 26]) | 38.5 percentage of participants |
| RLZ Total | Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) After Syringe Driver or Manual Push Self-administration From Visit 2 (Week 1) up to the End of Study Visit (Visit 21 [Week 26]) | 75.8 percentage of participants |