Clinical Trials List
Protocol NumberALXN1720-MG-302
Active
2024-11-01 - 2029-08-31
Phase III
Not yet recruiting1
Recruiting1
ICD-10G70.00
Myasthenia gravis without (acute) exacerbation
ICD-10G70.01
Myasthenia gravis with (acute) exacerbation
ICD-9358.0
Myasthenia gravis
An Open-Label, Single-arm Study to Evaluate the Pharmacokinetics (PK), Pharmacodynamics (PD), Safety, and Efficacy of Gefurulimab in Pediatric Patients (6 to < 18 years of age) with Generalized Myasthenia Gravis (gMG) Who Express Acetylcholine Receptor Antibodies (AChR+)
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Sponsor
-
Trial scale
Multi-Regional Multi-Center
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Update
2026/02/01
Investigators and Locations
Co-Principal Investigator
- 林冠佑 無
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Principal Investigator
Hou-Chang Chiu
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Generalized Myasthenia Gravis (gMG)
Objectives
To characterize the PK and PD of treatment with
gefurulimab in pediatric participants with AChR+
gMG
Test Drug
injective
Active Ingredient
ALXN1720
Dosage Form
230
Dosage
MG
Endpoints
Primary PK Estimand:
• Treatment: Gefurulimab
• Population: Pediatric participants (6 to
< 18 years) with AChR+ gMG who are
included in the PK Analysis Set
• Variables: Serum gefurulimab
concentrations from Day 1 predose
through Week 18 predose (Cmax, Ctrough)
− IE1: Study intervention
discontinuation prior to the end of
the Primary Evaluation Period
(Week 18)
− IE2: Initiation of disallowed
medication or therapy prior to the
end of the Primary Evaluation
Period (Week 18)
• For participants who experience IE1
and/or IE2, PK data collected after IE1
and/or IE2 will not be included for the
primary PK analysis
• Summary measures: Summary
statistics of gefurulimab concentrations
at Day 1 predose through Week 18
predose
• Treatment: Gefurulimab
• Population: Pediatric participants (6 to
< 18 years) with AChR+ gMG who are
included in the PK Analysis Set
• Variables: Serum gefurulimab
concentrations from Day 1 predose
through Week 18 predose (Cmax, Ctrough)
− IE1: Study intervention
discontinuation prior to the end of
the Primary Evaluation Period
(Week 18)
− IE2: Initiation of disallowed
medication or therapy prior to the
end of the Primary Evaluation
Period (Week 18)
• For participants who experience IE1
and/or IE2, PK data collected after IE1
and/or IE2 will not be included for the
primary PK analysis
• Summary measures: Summary
statistics of gefurulimab concentrations
at Day 1 predose through Week 18
predose
Inclution Criteria
Age
1. Participant must be 6 to < 18 years of age at the time of signing the informed
consent/assent.
Type of Participant and Disease Characteristics
2. Participants who have a documented diagnosis of gMG ≥ 3 months (90 days) prior to
Screening based on clinical disease features and at least 1 of the following confirmatory
tests. Test results may be collected from participant records or obtained during Screening:
• Positive response in an acetylcholinesterase inhibitor test, for example, the
edrophonium chloride test
• Abnormal neuromuscular transmission demonstrated by repetitive nerve stimulation
or single fiber electromyography
• Previous improvement of symptoms or signs related to gMG during treatment with on
oral acetylcholinesterase inhibitor, as confirmed by the treating physician
3. Positive serological test for AChR autoantibodies (previous test results, if applicable,
must be confirmed at Screening by central laboratory).
4. MGFA Class II to IV at Screening and on Day 1.
5. Must have QMG total score as outlined below:
• Participants 12 to < 18 years of age must have QMG total score ≥ 12 at Screening and
on Day 1
• Participants 6 to < 12 years of age will have no minimum QMG total score required
for inclusion; however, participants must have documented limb weakness in at least
1 limb
1. Participant must be 6 to < 18 years of age at the time of signing the informed
consent/assent.
Type of Participant and Disease Characteristics
2. Participants who have a documented diagnosis of gMG ≥ 3 months (90 days) prior to
Screening based on clinical disease features and at least 1 of the following confirmatory
tests. Test results may be collected from participant records or obtained during Screening:
• Positive response in an acetylcholinesterase inhibitor test, for example, the
edrophonium chloride test
• Abnormal neuromuscular transmission demonstrated by repetitive nerve stimulation
or single fiber electromyography
• Previous improvement of symptoms or signs related to gMG during treatment with on
oral acetylcholinesterase inhibitor, as confirmed by the treating physician
3. Positive serological test for AChR autoantibodies (previous test results, if applicable,
must be confirmed at Screening by central laboratory).
4. MGFA Class II to IV at Screening and on Day 1.
5. Must have QMG total score as outlined below:
• Participants 12 to < 18 years of age must have QMG total score ≥ 12 at Screening and
on Day 1
• Participants 6 to < 12 years of age will have no minimum QMG total score required
for inclusion; however, participants must have documented limb weakness in at least
1 limb
Exclusion Criteria
Participants are excluded from the study if any of the following criteria apply:
1. Clinical features that, in the opinion of the Investigator, are consistent with a Clinical
Deterioration, as defined in Section 4.1.6.1 including myasthenic crisis, ≤ 28 days prior to
Screening or during the Screening Period.
NOTE: Participants may be eligible for rescreening once the Clinical Deterioration has resolved,
and standard of care treatment has been resumed and stable for the periods of time described in
inclusion criteria (Table 11).
2. Any medical condition (eg, cardiac, pulmonary, renal, oncologic, neurological or
psychiatric disorder) or risk factor that, in the opinion of the Investigator or the Medical
Monitor, might interfere with participation in the study, pose any added risk to the
participant, or confound the assessment of safety or efficacy of the study intervention.
3. History of thymectomy or any other thymic surgery within 6 months prior to screening.
4. Any untreated thymic malignancy, carcinoma, or thymoma.
NOTE: Participants with a history of treated malignant thymoma or carcinoma are eligible if
they meet all of the following conditions:
a. Treatment completed > 5 years prior to the Screening Visit
b. No recurrence within the 5 years prior to the Screening Visit
c. No radiological indication of recurrence in a CT with contrast or MRI scan, including
administration of IV contrast, performed within 6 months of Day 1
1. Clinical features that, in the opinion of the Investigator, are consistent with a Clinical
Deterioration, as defined in Section 4.1.6.1 including myasthenic crisis, ≤ 28 days prior to
Screening or during the Screening Period.
NOTE: Participants may be eligible for rescreening once the Clinical Deterioration has resolved,
and standard of care treatment has been resumed and stable for the periods of time described in
inclusion criteria (Table 11).
2. Any medical condition (eg, cardiac, pulmonary, renal, oncologic, neurological or
psychiatric disorder) or risk factor that, in the opinion of the Investigator or the Medical
Monitor, might interfere with participation in the study, pose any added risk to the
participant, or confound the assessment of safety or efficacy of the study intervention.
3. History of thymectomy or any other thymic surgery within 6 months prior to screening.
4. Any untreated thymic malignancy, carcinoma, or thymoma.
NOTE: Participants with a history of treated malignant thymoma or carcinoma are eligible if
they meet all of the following conditions:
a. Treatment completed > 5 years prior to the Screening Visit
b. No recurrence within the 5 years prior to the Screening Visit
c. No radiological indication of recurrence in a CT with contrast or MRI scan, including
administration of IV contrast, performed within 6 months of Day 1
The Estimated Number of Participants
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Taiwan
3 participants
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Global
12 participants
