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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 18 January 2021
Main ID:  NCT02965573
Date of registration: 20/10/2016
Prospective Registration: Yes
Primary sponsor: argenx
Public title: A Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness
Scientific title: A Randomized, Double-blind, Placebo-Controlled Phase II Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness
Date of first enrolment: December 30, 2016
Target sample size: 24
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT02965573
Study type:  Interventional
Study design:  Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).  
Phase:  Phase 2
Countries of recruitment
Belgium Canada Italy Netherlands Poland Spain Sweden United States
Contacts
Name:     Antonio Guglietta, MD
Address: 
Telephone:
Email:
Affiliation:  argenx
Key inclusion & exclusion criteria

Inclusion Criteria:

1. Have the ability to understand the requirements of the study, provide written informed
consent (including consent for the use and disclosure of research-related health
information), and comply with the study protocol procedures (including required study
visits).

2. Male or female patients aged =18 years.

3. Diagnosis of autoimmune MG with generalized muscle weakness meeting the clinical
criteria for diagnosis of MG as defined by the Myasthenia Gravis Foundation of America
(MGFA) Clinical Classification Class II, III, or IVa, and likely not in need of a
respirator for the duration of the study as judged by the Investigator.

The confirmation of the diagnosis should be documented and supported by:

- Positive serologic test for anti-AChR antibodies before Screening and

- at least 1 of the following 3 tests: (i) History of abnormal neuromuscular
transmission test demonstrated by single-fiber electromyography or repetitive
nerve stimulation or (ii) History of positive edrophonium chloride test, or (iii)
Patient has demonstrated improvement in MG signs on oral cholinesterase
inhibitors as assessed by the treating physician.

4. A total score of = 5 on the MG ADL at Screening and Baseline with more than 50% of
this score attributed to non ocular items.

5. Patients are required to be on a stable dose of their MG treatment prior to
randomization. For patients receiving AZA, other NSIDs, steroids, and/or
cholinesterase inhibitors as concomitant medications the following conditions will
apply:

- AZA: treatment initiated at least 12 months ago and no dose changes in the last 6
months before screening.

- Other NSIDs (e.g., methotrexate, cyclosporine, tacrolimus, mycophenolate mofetil,
and cyclophosphamide) treatment initiated at least 6 months ago and no dose
changes in the last 3 months before Screening.

- Steroids treatment initiated at least 3 months prior to and no dose changes in
the last month before Screening.

- Cholinesterase inhibitors: to be on a stable dose for >2 weeks before Screening.

Note: cholinesterase inhibitors must be held for at least 12 hours consistent with the
revised manual for the QMG test as recommended by the Myasthenia Gravis Foundation of
America Inc (MGFA), before the MGQoL15r, MG-ADL, QMG, and MGC assessments.

6. Females of childbearing potential must have a negative serum pregnancy test at
Screening and a negative urine pregnancy test at Visit 1 prior to administration of
IMP. Female of childbearing potential are defined as all female participants unless
they are postmenopausal (defined by continuous amenorrhea) for at least 2 years with a
Follicle stimulating hormone (FSH) > 40 IU/L or are surgically sterile (i.e., who had
a hysterectomy, bilateral oophorectomy, or have current documented tubal ligation or
any other permanent female sterilization procedure). Determination of FSH levels can
be used to confirm postmenopausal status in amenorrheic patients not on hormonal
replacement therapy if the test result is within the postmenopausal range per the
central laboratory.

7. Female participants of childbearing potential must agree to use a highly effective
method of contraception (i.e., pregnancy rate of less than 1% per year) during the
study and for 90 days after the discontinuation of the IMP. Adequate contraceptive
methods include combined hormonal contraception associated with inhibition of
ovulation (oral, intravaginal, transdermal), progestogen-only hormonal contraception
associated with inhibition of ovulation (oral, injectable, implantable), intrauterine
devices (IUDs), intrauterine hormone-releasing system (IUS), true sexual abstinence
(when this is in line with the preferred and usual lifestyle of the participant),
bilateral tubal occlusion, or a female participant who is not of childbearing
potential. Female participants and female partners of male study participants using a
hormonal contraceptive must also use a barrier method (i.e., condom or occlusive cap
[diaphragm or cervical/vault caps]) and should have been stable on their hormonal
contraceptive treatment for at least 4 weeks before Screening.

8. Sterilized male patients who have had vasectomy with documented aspermia post
procedure can be included. In addition, male patients must be advised not to donate
sperm during this period from signing of Informed Consent Form (ICF), throughout the
duration of the study, and for 90 days after the last administration of IMP.
Non-sterilized male patients who are sexually active with a female partner of
childbearing potential must use effective method of double barrier contraception
(e.g., condom with spermicidal cream or jelly, 1 hormonal plus 1 barrier method or 2
simultaneous barrier methods). Male patients practicing true sexual abstinence (when
this is in line with the preferred and usual lifestyle of the participant) can be
included.

Exclusion Criteria:

1. Females who are pregnant or lactating.

2. MGFA Class I, IVb, and V.

3. Have an active infection, a recent serious infection (i.e., requiring injectable
antimicrobial therapy or hospitalization) within the 8 weeks prior to Screening; or
history of or known infection with human immunodeficiency virus (HIV), hepatitis B
virus (HBV), hepatitis C virus (HCV), or Mycobacterium tuberculosis. Patients must
have negative test results for HBV surface antigen, HBV core antibody, HCV antibody,
HIV 1 and 2 antibodies, and a negative QuantiFERON®-TB Gold test at Screening.
Patients with an indeterminate QuantiFERON®-TB Gold test result will be allowed one
retest; if not negative on retesting, the patient will be excluded.

4. At Screening, have clinically significant laboratory abnormalities or as below:

- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) > 2 x upper
limit of normal (ULN).

- Total serum bilirubin of > 1.5 x ULN (except for Grade 1 hyperbilirubinemia
solely due to a medical diagnosis of Gilbert's syndrome).

- Serum creatinine > 1.5 mg/dL and creatinine clearance < 50 ml/min (using the
Chronic Kidney Disease Epidemiology [CKD-EPI]-Creatinine formula).

- Clinically Significant proteinuria (i.e., > 3 x ULN).

- Hemoglobin = 9 g/L.

- Thyroid stimulating hormone or thyroglobulin outside of the central laboratory
normal range.

- International n



Age minimum: 18 Years
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
Myasthenia Gravis
Intervention(s)
Biological: ARGX-113
Drug: Placebo
Primary Outcome(s)
Mean Change From Baseline in Vital Signs: Blood Pressure [Time Frame: Baseline and Days 8,15, 22, 29, 36, 43, 50, 64 and 78]
Mean Change From Baseline in Vital Signs: Heart Rate [Time Frame: Baseline and Days 8,15, 22, 29, 36, 43, 50, 64 and 78]
Mean Change From Baseline in Vital Signs: Temperature [Time Frame: Baseline and Days 8,15, 22, 29, 36, 43, 50, 64 and 78]
Mean Change From Baseline in Vital Signs: Weight [Time Frame: Baseline and Days 8,15, 22, 29, 36, 43, 50, 64 and 78]
Number of Patients With Abnormal Clinical Laboratory Findings Reported as TEAEs [Time Frame: Day 1 to Day 78]
Number of Patients With Abnormal Clinically Relevant Findings in Electrocardiogram (ECG) Parameters [Time Frame: Day 1 to Day 78]
Number of Patients With Treatment Emergent Adverse Events (TEAES) and Treatment Emergent Serious Adverse Events (SAEs) [Time Frame: Day 1 to Day 78]
Secondary Outcome(s)
Maximum Reduction From Baseline in MG-ADL Score [Time Frame: Day 1 to Day 78]
Maximum Reduction From Baseline in MGC Score [Time Frame: Day 1 to Day 78]
Maximum Reduction From Baseline in MGQoL15r Score [Time Frame: Day 1 to Day 78]
Maximum Reduction From Baseline in QMG Score [Time Frame: Day 1 to Day 78]
Mean Change From Baseline in MGC Score [Time Frame: Baseline and Days 8,15, 22, 29, 36, 43, 50, 64 and 78]
Mean Change From Baseline in MGQoL15r Score [Time Frame: Baseline and Days 8,15, 22, 29, 36, 43, 50, 64 and 78]
Mean Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score [Time Frame: Baseline and Days 8,15, 22, 29, 36, 43, 50, 64 and 78]
Mean Change From Baseline in QMG Score [Time Frame: Baseline and Days 8,15, 22, 29, 36, 43, 50, 64 and 78]
Mean Percent Change From Baseline in Anti-Acetylcholine Receptor (AChR) Antibodies [Time Frame: Baseline, Days 22 and 78]
Mean Percent Change From Baseline in Immunoglobulins (IgGs) [Time Frame: Baseline, Days 22 and 78]
Number of Patients With an Anti-drug Antibodies (ADA) Response [Time Frame: Baseline up to Day 78]
Pharmacokinetic (PK) Parameters - Plasma Concentrations of ARGX-113 [Time Frame: Days 1, 8, 15 and 22]
PK Parameters - Accumulation Ratio (Rac) of ARGX-113 [Time Frame: Days 1 and 22.]
PK Parameters - Apparent Terminal Half-life (t1/2 Lambda z) of ARGX-113 [Time Frame: Day 22]
PK Parameters - Median Time of Occurrence of Cmax (Tmax) of ARGX-113 [Time Frame: Days 1, 8 15 and 22.]
Secondary ID(s)
2016-002938-73
ARGX-113-1602
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Quintiles, Inc.
Ethics review
Results
Results available: Yes
Date Posted: 08/01/2021
Date Completed:
URL: https://clinicaltrials.gov/ct2/show/results/NCT02965573
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