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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/NCT05070858
Registration number
NCT05070858
Ethics application status
Date submitted
27/09/2021
Date registered
7/10/2021
Date last updated
3/06/2024
Titles & IDs
Public title
A Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Symptomatic Generalized Myasthenia Gravis
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Scientific title
Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Symptomatic Generalized Myasthenia Gravis
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Secondary ID [1]
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2020-003272-41
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Secondary ID [2]
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R3918-MG-2018
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Universal Trial Number (UTN)
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Trial acronym
NIMBLE
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Generalized Myasthenia Gravis
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Condition category
Condition code
Inflammatory and Immune System
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Autoimmune diseases
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Neurological
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0
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Other neurological disorders
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Musculoskeletal
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Other muscular and skeletal disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Pozelimab + Cemdisiran
Treatment: Drugs - Cemdisiran
Other interventions - Placebo
Treatment: Drugs - Pozelimab
Experimental: Group 1 - Placebo in DBTP; Re-randomized to Combination or Cemdisiran in ETP and OLTP
Experimental: Group 2 - Combination regimen throughout the study
Experimental: Group 3 - Cemdisiran throughout the study
Experimental: Group 4 - Pozelimab monotherapy in DBTP followed by combination in ETP and OLTP
Treatment: Drugs: Pozelimab + Cemdisiran
Subcutaneous administration as described in the protocol
Treatment: Drugs: Cemdisiran
SC administration as described in the protocol
Other interventions: Placebo
SC administration as described in the protocol
Treatment: Drugs: Pozelimab
SC administration as described in the protocol
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Intervention code [1]
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Treatment: Drugs
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Intervention code [2]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score
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Assessment method [1]
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The total MG-ADL score ranges from 0 to 24 points, with higher scores indicating greater functional impairment and disability
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Timepoint [1]
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From baseline to week 24
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Secondary outcome [1]
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Change from baseline in Quantitative Myasthenia Gravis (QMG) score
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Assessment method [1]
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QMG total scores range from 0 to 39, with higher scores representing greater impairment
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Timepoint [1]
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Week 24
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Secondary outcome [2]
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Proportion of patients responding on the MG-ADL
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Assessment method [2]
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=3-point improvement
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Timepoint [2]
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From baseline to week 24
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Secondary outcome [3]
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Proportion of patients responding on the QMG
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Assessment method [3]
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=5-point improvement
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Timepoint [3]
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From baseline to week 24
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Secondary outcome [4]
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Proportion of patients with consistent response on the MG-ADL
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Assessment method [4]
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At least a 2-point MG-ADL improvement on 2 or more consecutive assessments spanning 4 or more weeks during the DBTP
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Timepoint [4]
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From baseline to week 24
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Secondary outcome [5]
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Proportion of patients with minimal symptom expression (MSE)
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Assessment method [5]
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Score of 0 to 1 on the MG-ADL
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Timepoint [5]
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Week 24
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Secondary outcome [6]
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Change from baseline in the Myasthenia Gravis Composite (MGC) total score
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Assessment method [6]
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MGC score ranges from 0 to 50, with higher score indicating higher impairment
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Timepoint [6]
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Week 24
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Secondary outcome [7]
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Change from baseline in Myasthenia Gravis Quality of Life (MG QOL15r) total score
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Assessment method [7]
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Total score ranges from 0 to 30 points; a higher score represents greater impairment
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Timepoint [7]
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Week 24
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Secondary outcome [8]
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Proportion of patients with improvement point thresholds on MG-ADL
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Assessment method [8]
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=2, 4, 5, 6, 7, 8, 9, or 10
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Timepoint [8]
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From baseline to week 24
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Secondary outcome [9]
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Proportion of patients with improvement point thresholds on QMG
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Assessment method [9]
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=3, 4, 6, 7, 8, 9, or 10
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Timepoint [9]
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From baseline to week 24
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Secondary outcome [10]
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Incidence and severity of treatment-related adverse events (TEAEs) in patients treated with pozelimab + cemdisiran or placebo
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Assessment method [10]
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Timepoint [10]
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Through week 24
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Secondary outcome [11]
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Incidence and severity of serious adverse events (SAEs) in patients treated with pozelimab + cemdisiran or placebo
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Assessment method [11]
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Timepoint [11]
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Through week 24
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Secondary outcome [12]
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Incidence and severity of adverse events of special interest (AESIs) in patients treated with pozelimab + cemdisiran or placebo
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Assessment method [12]
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Timepoint [12]
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Through week 24
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Secondary outcome [13]
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Concentrations of total pozelimab in serum
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Assessment method [13]
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Timepoint [13]
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Through study duration, approximate 172 weeks
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Secondary outcome [14]
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Concentrations of cemdisiran and its metabolites in plasma
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Assessment method [14]
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Timepoint [14]
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Through study duration, approximate 172 weeks
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Secondary outcome [15]
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Incidence of treatment-emergent anti-drug antibodies (ADAs) to pozelimab over time
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Assessment method [15]
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Timepoint [15]
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Through study duration, approximately 172 weeks
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Secondary outcome [16]
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Incidence of treatment-emergent ADAs to cemdisiran over time
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Assessment method [16]
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Timepoint [16]
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Through study duration, approximate 172 weeks
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Secondary outcome [17]
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Change in CH50 over time
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Assessment method [17]
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Timepoint [17]
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Through study duration, approximately 172 weeks
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Secondary outcome [18]
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Percent change in CH50 over time
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Assessment method [18]
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Timepoint [18]
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Through study duration, approximately 172 weeks
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Eligibility
Key inclusion criteria
Key
1. Male or female patients =18 years of age at screening (or = legal age of adulthood
based on local regulations, whichever is older)
2. Patient with documented diagnosis of myasthenia gravis (MG) based on medical history
and supported by previous evaluations as described in the protocol
3. Documented prior history of positive serologic test or a positive result during
screening of anti-acetylcholine receptor (AChR) antibodies or anti-LRP4 antibodies.
4. Myasthenia Gravis Foundation of America (MGFA) Clinical Classification Class II to IVa
at screening
5. Myasthenia Gravis-Activities of Daily Living (MG-ADL) score =6 at screening. Ocular
items should not contribute more than 50% of MG-ADL total score
6. Currently receiving an acetylcholinesterase inhibitor or documented reason for not
using acetylcholinesterase inhibitor therapy per investigator 7. Currently receiving
an immunosuppressive therapy (IST) for MG, or documented reason why the patient is not
taking an IST per investigator
8. If currently receiving an IST, not anticipated to have IST dosage changed before
randomization or during double-blind treatment period (DBTP).
Key
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. Patients with antibody profile that is only positive for muscle specific tyrosine
kinase (MuSK) (MuSK positivity is based on a documented prior history of positive
serologic test for antibodies to MuSK or a positive result during screening
2. History of thymectomy within 12 months prior to screening or planned during the study
3. History of malignant thymoma (patients with stage 1 may be enrolled), or history of
cancer within the past 5 years, except for adequately treated basal cell skin cancer,
squamous cell skin cancer, or in situ cervical cancer
4. Myasthenic crisis or Myasthenia Gravis Foundation of America (MGFA) Class V within 1
month of screening
5. No documented meningococcal vaccination within 5 years prior to screening visit unless
vaccination will be administered during the screening period and prior to initiation
of study treatment
6. Known contraindication to meningococcal vaccines (group ACWY conjugate and group B
vaccines) as described in the protocol
7. Patients who require antibiotics for meningococcal prophylaxis and have a
contraindication, warning, or precaution precluding the use of penicillin class and
penicillin-alternative antibiotics planned to be used for prophylaxis, or a history of
intolerance leading to the discontinuation of these antibiotics
8. Positive hepatitis B surface antigen or hepatitis C virus ribonucleic acid (RNA)
during screening. NOTE: Cases with unclear interpretation should be discussed with the
medical monitor
9. History of HIV infection or a positive test at screening per local requirements
NOTE: Other protocol-defined Inclusion/ Exclusion Criteria apply
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
14/12/2021
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
23/03/2028
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Actual
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Sample size
Target
235
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,SA,VIC,WA
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Recruitment hospital [1]
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Southern Neurology - Sydney
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Recruitment hospital [2]
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Lyell McEwin Hospital - Elizabeth Vale
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Recruitment hospital [3]
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St. Vincent's Hospital Melbourne - Fitzroy
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Recruitment hospital [4]
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Perron Institute for Neurological and Translational Science - Nedlands
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Recruitment postcode(s) [1]
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2217 - Sydney
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Recruitment postcode(s) [2]
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5112 - Elizabeth Vale
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Recruitment postcode(s) [3]
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3065 - Fitzroy
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Recruitment postcode(s) [4]
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6009 - Nedlands
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Recruitment outside Australia
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United States of America
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Arizona
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United States of America
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California
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Colorado
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Florida
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United States of America
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Illinois
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Michigan
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New York
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North Carolina
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Ohio
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Antwerp
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Belgium
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Hainaut
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Belgium
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Oost-Vlaanderen
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Bruxelles
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Alberta
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Ontario
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Odense
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Hiroshima
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Nis
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Valencia
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Taichung
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Van
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Trabzon
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United Kingdom
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South Yorkshire
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United Kingdom
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Birmingham
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Regeneron Pharmaceuticals
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The primary objective is:
To evaluate the effect of pozelimab + cemdisiran on daily functioning that is impacted by
signs and symptoms in patients with symptomatic generalized myasthenia gravis (gMG)
The secondary objectives of the study are:
- To evaluate the effect of pozelimab + cemdisiran (ie, combination) and cemdisiran
monotherapy on:
- Clinician-assessed signs of myasthenia gravis (MG) and muscle strength
- Daily functioning that is impacted by signs and symptoms in patients with
symptomatic gMG (cemdisiran monotherapy only).
- Proportion of patients with improvements in daily function that is impacted by
signs and symptoms of MG
- Proportion of patients that have improvements in clinician-assessed signs of MG and
muscle strength
- Health related quality of life
- Proportion of patients with minimal MG symptoms
- Patient- and clinician-reported signs and symptoms of MG
- To evaluate the safety and tolerability of pozelimab + cemdisiran and cemdisiran
monotherapy
- To assess the concentration of total pozelimab in serum
- To assess the concentrations of cemdisiran and its metabolites in plasma
- To assess the immunogenicity of pozelimab
- To assess the concentration of total C5 in plasma
- To assess the immunogenicity of cemdisiran
- To study the effect of pozelimab + cemdisiran and cemdisiran monotherapy on complement
activation
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05070858
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Clinical Trial Management
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Regeneron Pharmaceuticals
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Clinical Trials Administrator
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844-734-6643
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[email protected]
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Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05070858
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