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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05556096




Registration number
NCT05556096
Ethics application status
Date submitted
23/09/2022
Date registered
27/09/2022
Date last updated
1/11/2023

Titles & IDs
Public title
Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis
Scientific title
A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel, Multicenter Study to Evaluate the Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis
Secondary ID [1] 0 0
2022-000460-21
Secondary ID [2] 0 0
ALXN1720-MG-301
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Generalized Myasthenia Gravis 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Neurological 0 0 0 0
Other neurological disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Combination Product - ALXN1720
Combination Product - Placebo

Experimental: ALXN1720 - Participants will receive a weight-based initial (loading) dose of ALXN1720 on Day 1, followed by weight-based maintenance treatment with ALXN1720 on Day 8 and once every week (Q1W) thereafter for a total of 26 weeks. Following this randomized controlled treatment (RCT) period, all participants will receive ALXN1720 in an open-label extension (OLE) period of 105 weeks.

Placebo Comparator: Placebo - Participants will receive placebo during the 26-week RCT period, after which they will enter the OLE period of the study and receive ALXN1720.


Combination Product: ALXN1720
Combination product consisting of syringe prefilled with ALXN1720.

Combination Product: Placebo
Combination product consisting of syringe prefilled with placebo.
Intervention code [1] 0 0
Combination Product
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Week 26
Timepoint [1] 0 0
Baseline, Week 26
Secondary outcome [1] 0 0
Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Week 26
Timepoint [1] 0 0
Baseline, Week 26
Secondary outcome [2] 0 0
Percentage of Responders Based on Reduction of the MG-ADL Total Score at Week 26
Timepoint [2] 0 0
Baseline up to Week 26
Secondary outcome [3] 0 0
Percentage of Responders based on Reduction of the QMG Total Score at Week 26
Timepoint [3] 0 0
Baseline up to Week 26
Secondary outcome [4] 0 0
Change From Baseline in Myasthenia Gravis Composite (MGC) Total Score at Week 26
Timepoint [4] 0 0
Baseline, Week 26

Eligibility
Key inclusion criteria
- 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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
21/11/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
7/07/2027
Actual
Sample size
Target
254
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to evaluate the safety and efficacy of ALXN1720 for the
treatment of generalized MG (gMG) in adults with autoantibodies against acetylcholine
receptor (AChR).
Trial website
https://clinicaltrials.gov/ct2/show/NCT05556096
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
AstraZeneca Clinical Study Information Center
Address 0 0
Country 0 0
Phone 0 0
1-877-240-9479
Fax 0 0
Email 0 0
information.center@astrazeneca.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05556096