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Trial Title:
PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
NCT ID:
NCT06607627
Condition:
Generalized Myasthenia Gravis
gMG
Conditions: Official terms:
Myasthenia Gravis
Muscle Weakness
Conditions: Keywords:
Acetylcholine Receptor antibodies
AChR+
Generalized Myasthenia Gravis
gMG
ALXN1720
anti-acetylcholine receptor antibody-positive
acetylcholine receptor
AChR
myasthenia gravis
MG
complement component 5
C5
VHH antibody
pediatric
Study type:
Interventional
Study phase:
Phase 3
Overall status:
Not yet recruiting
Study design:
Allocation:
N/A
Intervention model:
Single Group Assignment
Primary purpose:
Treatment
Masking:
None (Open Label)
Intervention:
Intervention type:
Combination Product
Intervention name:
Gefurulimab
Description:
Combination product consisting of syringe prefilled with gefurulimab will be administered
weekly via subcutaneous (SC) injection.
Arm group label:
Gefurulimab
Summary:
The primary objective of this study is to assess the pharmacokinetics and
pharmacodynamics of gefurulimab in pediatric participants with AChR+ gMG for the duration
of the study.
Criteria for eligibility:
Criteria:
Inclusion Criteria:
- Participant must be 6 to < 18 years of age at the time of signing the informed
consent/assent.
- Diagnosis of MG with generalized muscle weakness meeting the clinical criteria
defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV
- Positive serological test for autoantibodies against AChR
- All participants must be vaccinated against meningococcal infection from serogroups
A, C, W, Y (and B where available) within 3 years prior to study intervention on Day
1. If vaccination occurs < 2 weeks prior to Day 1, the participants will receive
prophylactic antibiotics for at least 2 weeks after initial meningococcal
vaccination for serogroups A,C,W,Y (and B, where available)
Exclusion Criteria:
- History of thymectomy, or any other thymic surgery within 12 months prior to
Screening
- Untreated thymic malignancy, carcinoma, or thymoma
- History of Neisseria meningitidis infection
- Pregnancy, breastfeeding, or intention to conceive during the course of the study
Gender:
All
Minimum age:
6 Years
Maximum age:
17 Years
Healthy volunteers:
No
Locations:
Facility:
Name:
Research Site
Address:
City:
Washington
Zip:
20010
Country:
United States
Facility:
Name:
Research Site
Address:
City:
Norfolk
Zip:
23507
Country:
United States
Facility:
Name:
Research Site
Address:
City:
Joinville
Zip:
89202-451
Country:
Brazil
Facility:
Name:
Research Site
Address:
City:
Salvador
Zip:
41253-190
Country:
Brazil
Facility:
Name:
Research Site
Address:
City:
Sao Jose Do Rio Preto
Zip:
15090-000
Country:
Brazil
Facility:
Name:
Research Site
Address:
City:
Sao Paulo
Zip:
05403-000
Country:
Brazil
Facility:
Name:
Research Site
Address:
City:
São Paulo
Zip:
0438-002
Country:
Brazil
Facility:
Name:
Research Site
Address:
City:
Bydgoszcz
Zip:
85-086
Country:
Poland
Facility:
Name:
Research Site
Address:
City:
Warszawa
Zip:
02-097
Country:
Poland
Facility:
Name:
Research Site
Address:
City:
Łódź
Zip:
93-338
Country:
Poland
Facility:
Name:
Research Site
Address:
City:
Taipei
Zip:
11101
Country:
Taiwan
Facility:
Name:
Research Site
Address:
City:
Taipei
Zip:
23561
Country:
Taiwan
Start date:
March 4, 2025
Completion date:
October 1, 2029
Lead sponsor:
Agency:
Alexion Pharmaceuticals, Inc.
Agency class:
Industry
Source:
Alexion Pharmaceuticals, Inc.
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT06607627
