Register a trial

Please note the ANZCTR will be unattended from Friday 20 December 2024 for the holidays. The Registry will re-open on Tuesday 7 January 2025. Submissions and updates will not be processed during that time.

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

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




Registration number
NCT05556096
Ethics application status
Date submitted
23/09/2022
Date registered
27/09/2022

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
Other interventions - ALXN1720
Other interventions - 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.


Other interventions: ALXN1720
Combination product consisting of syringe prefilled with ALXN1720.

Other interventions: Placebo
Combination product consisting of syringe prefilled with placebo.
Intervention code [1] 0 0
Other interventions
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
Secondary outcome [5] 0 0
Change from Baseline in the QMG total score at Week 4
Timepoint [5] 0 0
Baseline, Week 4

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
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
District of Columbia
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
State/province [6] 0 0
Georgia
Country [7] 0 0
United States of America
State/province [7] 0 0
Illinois
Country [8] 0 0
United States of America
State/province [8] 0 0
Kansas
Country [9] 0 0
United States of America
State/province [9] 0 0
Kentucky
Country [10] 0 0
United States of America
State/province [10] 0 0
Massachusetts
Country [11] 0 0
United States of America
State/province [11] 0 0
Michigan
Country [12] 0 0
United States of America
State/province [12] 0 0
Nevada
Country [13] 0 0
United States of America
State/province [13] 0 0
New York
Country [14] 0 0
United States of America
State/province [14] 0 0
North Carolina
Country [15] 0 0
United States of America
State/province [15] 0 0
Oregon
Country [16] 0 0
United States of America
State/province [16] 0 0
South Carolina
Country [17] 0 0
United States of America
State/province [17] 0 0
Texas
Country [18] 0 0
United States of America
State/province [18] 0 0
Virginia
Country [19] 0 0
United States of America
State/province [19] 0 0
Washington
Country [20] 0 0
United States of America
State/province [20] 0 0
West Virginia
Country [21] 0 0
United States of America
State/province [21] 0 0
Wisconsin
Country [22] 0 0
Argentina
State/province [22] 0 0
Buenos Aires
Country [23] 0 0
Argentina
State/province [23] 0 0
Caba
Country [24] 0 0
Argentina
State/province [24] 0 0
Capital Federal
Country [25] 0 0
Argentina
State/province [25] 0 0
Rosario
Country [26] 0 0
Austria
State/province [26] 0 0
Innsbruck
Country [27] 0 0
Austria
State/province [27] 0 0
Vienna
Country [28] 0 0
Austria
State/province [28] 0 0
Wels-Grieskirchen
Country [29] 0 0
Brazil
State/province [29] 0 0
Barretos
Country [30] 0 0
Brazil
State/province [30] 0 0
Joinville
Country [31] 0 0
Brazil
State/province [31] 0 0
Porto Alegre
Country [32] 0 0
Brazil
State/province [32] 0 0
Ribeirão Preto
Country [33] 0 0
Brazil
State/province [33] 0 0
Salvador
Country [34] 0 0
Brazil
State/province [34] 0 0
Sao Jose Do Rio Preto
Country [35] 0 0
Brazil
State/province [35] 0 0
Sao Paulo
Country [36] 0 0
Brazil
State/province [36] 0 0
São Paulo
Country [37] 0 0
Canada
State/province [37] 0 0
Alberta
Country [38] 0 0
Canada
State/province [38] 0 0
Ontario
Country [39] 0 0
Canada
State/province [39] 0 0
Quebec
Country [40] 0 0
China
State/province [40] 0 0
Beijing
Country [41] 0 0
China
State/province [41] 0 0
Changchun
Country [42] 0 0
China
State/province [42] 0 0
Changsha
Country [43] 0 0
China
State/province [43] 0 0
Chengdu
Country [44] 0 0
China
State/province [44] 0 0
Chongqing
Country [45] 0 0
China
State/province [45] 0 0
Fuzhou
Country [46] 0 0
China
State/province [46] 0 0
Hangzhou
Country [47] 0 0
China
State/province [47] 0 0
Jinan
Country [48] 0 0
China
State/province [48] 0 0
Qingdao
Country [49] 0 0
China
State/province [49] 0 0
Shanghai
Country [50] 0 0
China
State/province [50] 0 0
Suzhou
Country [51] 0 0
China
State/province [51] 0 0
Wuhan
Country [52] 0 0
China
State/province [52] 0 0
Xi'an
Country [53] 0 0
Denmark
State/province [53] 0 0
Aalborg
Country [54] 0 0
Denmark
State/province [54] 0 0
København Ø
Country [55] 0 0
France
State/province [55] 0 0
Angers
Country [56] 0 0
France
State/province [56] 0 0
Garches
Country [57] 0 0
France
State/province [57] 0 0
Lille
Country [58] 0 0
France
State/province [58] 0 0
Marseille
Country [59] 0 0
France
State/province [59] 0 0
Montpellier Cedex 5
Country [60] 0 0
France
State/province [60] 0 0
Nice
Country [61] 0 0
France
State/province [61] 0 0
Paris Cedex 13
Country [62] 0 0
France
State/province [62] 0 0
Strasbourg Cedex
Country [63] 0 0
Germany
State/province [63] 0 0
Berlin
Country [64] 0 0
Germany
State/province [64] 0 0
Düsseldorf
Country [65] 0 0
Germany
State/province [65] 0 0
Giessen
Country [66] 0 0
Germany
State/province [66] 0 0
München
Country [67] 0 0
Germany
State/province [67] 0 0
Tübingen
Country [68] 0 0
Germany
State/province [68] 0 0
Wuerzburg
Country [69] 0 0
Israel
State/province [69] 0 0
Hadera
Country [70] 0 0
Israel
State/province [70] 0 0
Haifa
Country [71] 0 0
Israel
State/province [71] 0 0
Petah Tikva
Country [72] 0 0
Israel
State/province [72] 0 0
Ramat Gan
Country [73] 0 0
Italy
State/province [73] 0 0
Bergamo
Country [74] 0 0
Italy
State/province [74] 0 0
Brescia
Country [75] 0 0
Italy
State/province [75] 0 0
Firenze
Country [76] 0 0
Italy
State/province [76] 0 0
Milano
Country [77] 0 0
Italy
State/province [77] 0 0
Napoli
Country [78] 0 0
Italy
State/province [78] 0 0
Palermo
Country [79] 0 0
Italy
State/province [79] 0 0
Pisa
Country [80] 0 0
Italy
State/province [80] 0 0
Roma
Country [81] 0 0
Italy
State/province [81] 0 0
Rome
Country [82] 0 0
Italy
State/province [82] 0 0
Salerno
Country [83] 0 0
Italy
State/province [83] 0 0
Treviso
Country [84] 0 0
Italy
State/province [84] 0 0
Udine
Country [85] 0 0
Japan
State/province [85] 0 0
Chiba-shi
Country [86] 0 0
Japan
State/province [86] 0 0
Fukushima-shi
Country [87] 0 0
Japan
State/province [87] 0 0
Higashimatsuyama
Country [88] 0 0
Japan
State/province [88] 0 0
Hiroshima-shi
Country [89] 0 0
Japan
State/province [89] 0 0
Isehara-shi
Country [90] 0 0
Japan
State/province [90] 0 0
Itabashi-ku
Country [91] 0 0
Japan
State/province [91] 0 0
Kawasaki-shi
Country [92] 0 0
Japan
State/province [92] 0 0
Morioka
Country [93] 0 0
Japan
State/province [93] 0 0
Nagasaki
Country [94] 0 0
Japan
State/province [94] 0 0
Nagoya-shi
Country [95] 0 0
Japan
State/province [95] 0 0
Nagoya
Country [96] 0 0
Japan
State/province [96] 0 0
Ota-ku
Country [97] 0 0
Japan
State/province [97] 0 0
Shinjuku-ku
Country [98] 0 0
Japan
State/province [98] 0 0
Suita-shi
Country [99] 0 0
Korea, Republic of
State/province [99] 0 0
Busan
Country [100] 0 0
Korea, Republic of
State/province [100] 0 0
Daegu
Country [101] 0 0
Korea, Republic of
State/province [101] 0 0
Gwangju
Country [102] 0 0
Korea, Republic of
State/province [102] 0 0
Seoul
Country [103] 0 0
Netherlands
State/province [103] 0 0
Amsterdam
Country [104] 0 0
Poland
State/province [104] 0 0
Bydgoszcz
Country [105] 0 0
Poland
State/province [105] 0 0
Katowice
Country [106] 0 0
Poland
State/province [106] 0 0
Kraków
Country [107] 0 0
Poland
State/province [107] 0 0
Lublin
Country [108] 0 0
Poland
State/province [108] 0 0
Poznan
Country [109] 0 0
Portugal
State/province [109] 0 0
Almada
Country [110] 0 0
Portugal
State/province [110] 0 0
Porto
Country [111] 0 0
Serbia
State/province [111] 0 0
Belgrade
Country [112] 0 0
Serbia
State/province [112] 0 0
Nis
Country [113] 0 0
Spain
State/province [113] 0 0
Badalona(Barcelona)
Country [114] 0 0
Spain
State/province [114] 0 0
Córdoba
Country [115] 0 0
Spain
State/province [115] 0 0
Hospitalet de Llobregat
Country [116] 0 0
Spain
State/province [116] 0 0
Madrid
Country [117] 0 0
Spain
State/province [117] 0 0
Málaga
Country [118] 0 0
Spain
State/province [118] 0 0
Pamplona
Country [119] 0 0
Spain
State/province [119] 0 0
Sevilla
Country [120] 0 0
Switzerland
State/province [120] 0 0
Lugano
Country [121] 0 0
Switzerland
State/province [121] 0 0
St. Gallen
Country [122] 0 0
Switzerland
State/province [122] 0 0
Zürich
Country [123] 0 0
Taiwan
State/province [123] 0 0
Kaohsiung City
Country [124] 0 0
Taiwan
State/province [124] 0 0
Tainan City
Country [125] 0 0
Taiwan
State/province [125] 0 0
Taipei
Country [126] 0 0
Turkey
State/province [126] 0 0
Atakum
Country [127] 0 0
Turkey
State/province [127] 0 0
Bursa
Country [128] 0 0
Turkey
State/province [128] 0 0
Istanbul
Country [129] 0 0
Turkey
State/province [129] 0 0
Kocaeli
Country [130] 0 0
Turkey
State/province [130] 0 0
Kütahya
Country [131] 0 0
United Kingdom
State/province [131] 0 0
Liverpool
Country [132] 0 0
United Kingdom
State/province [132] 0 0
London
Country [133] 0 0
United Kingdom
State/province [133] 0 0
Oxford
Country [134] 0 0
United Kingdom
State/province [134] 0 0
Sheffield

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

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
Alexion Pharmaceuticals, Inc. (Sponsor)
Address 0 0
Country 0 0
Phone 0 0
1-855-752-2356
Fax 0 0
Email 0 0
clinicaltrials@alexion.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05556096