A Phase 2, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group, Multicenter Trial to Evaluate the Safety and Tolerability, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of 2 Dose Regimens of ARGX-117 in Adults With Multifocal Motor Neuropathy

Phase 2

Recruiting

Conditions: Disease of the neuro muscular junction
serious muscle weakness
10003816
10029317

Registration Number: NL-OMON54143

Lead Sponsor: argenx BV

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 4

Inclusion Criteria

Participants are eligible to be included in the trial only if all of the
following criteria apply:
1. Capable of giving signed informed consent as described in Appendix 1,
Section 10.1.3 which includes compliance with the requirements and restrictions
listed in the informed consent form (ICF) and in this protocol (including
consent for the use and disclosure of research-related health information).
Participants must be able to read and write and be willing and able to comply
with the trial protocol procedures (including attending the required trial
visits).
2. Male/female at least 18 years of age at the time the ICF is signed
3. Probable or definite MMN according to the EFNS/PNS 2010 guidelines (Appendix
8: Section 10.8) at screening confirmed by the MCC
4. Receiving a stable IVIg regimen for at least 3 months before screening or
recently initiated IVIg treatment (refer to inclusion criterion 5.1a) and both
of the following:
a. IVIg treatment interval of 2 to 5 weeks
b. IVIg dose of 0.4 to 2.0 grams per kg body weight and infusion
5.1 IVIg treatment dependency confirmation by the MCC at screening or after
IVDP when applicable, based on 1 of the following:
a. Recently initiated IVIg treatment (less than 3 months):
- Clinical improvement following IVIg initiation documented in the
participant*s medical record
b. Maintenance therapy with IVIg (longer than 3 months), based on 1 of the
following:
- Clinical deterioration following IVIg withdrawal, IVIg dose reduction,
or IVIg delayed administration within 12 months prior to screening (documented
in the participant*s medical record)
- Clinical deterioration following IVIg delayed administration during the
IVDP
6. Immunization with the first meningococcal vaccine and pneumococcal vaccine,
and the single Haemophilus influenza type B vaccine must be performed at least
14 days before IMP administration at V1 according to local country-specific
immunization schedules. A documented history of vaccination against Neisseria
meningitides, Haemophilus influenza type B, and streptococcus pneumonia will be
permitted.
7. Contraceptive use by men and women should be consistent with local
regulations regarding the methods of contraception for those participating in
clinical studies
a. Male participants must agree to not donate sperm from the time the ICF is
signed until 15 months after the last IMP administration
b. Women of childbearing potential (WOCBP) (defined in Appendix 4, Section
10.4.1.1) must have a negative serum pregnancy test at screening and a negative
urine pregnancy test at baseline before IMP can be administered
The contraceptive requirements for WOCBP are described in Appendix 4, Section
10.4.2).

Exclusion Criteria

Participants are excluded from the trial if any of the following criteria apply:
1. Any coexisting condition which may interfere with the outcome assessments
(eg, diabetic neuropathy, CIDP, inflammatory arthritis, or osteoarthritis
affecting the hand)
2. Clinical signs or symptoms suggestive for neuropathies other than MMN such
as motor neuron disease (eg, bulbar signs or brisk reflexes) or other
inflammatory neuropathies (eg, sensory neuropathy)
3. Severe psychiatric disorder (such as severe depression, psychosis, bipolar
disorder), history of suicide attempt, or current suicidal ideation that in the
opinion of the investigator could create undue risk to the participant or could
affect adherence with the trial protocol. See Section 8.2.6.
Note: At screening, suicidality will be assessed using the Columbia-suicide
severity rating scale (C SSRS) (see Section 8.2.6.1); participants with a high
suicide risk will be excluded from the trial (ie, participants will be excluded
with a positive answer to questions #4 and/or #5 of the suicidal ideation
subscale [over the past 3 months]; and/or any positive answer to the suicidal
behavior subscale [over the past year]). Any positive answer to the these
questions under *Lifetime/time he/she felt most suicidal* should be carefully
evaluated for any current risk of suicide by the investigator prior to trial
entry.
4. Clinically significant uncontrolled active or chronic bacterial, viral, or
fungal infection during the screening and/or IVMP.
5. Any other known autoimmune disease that, in the opinion of the investigator,
would interfere with an accurate assessment of clinical symptoms of MMN or put
the participant at undue risk (eg, SLE).
6. History of malignancy unless resolved by adequate treatment with no evidence
of recurrence for >=3 years before the first administration of the IMP.
Participants with the following carcinomas will be eligible:
a. Adequately treated basal cell or squamous cell skin cancer
b. Carcinoma in situ of the cervix
c. Carcinoma in situ of the breast or
d. Incidental histological finding of prostate cancer (TNM stage T1a or T1b)
7. Clinical evidence of other significant serious diseases, (including
splenectomy at any time), have had a recent major surgery, or who have any
other condition in the opinion of the investigator, that could confound the
results of the trial or put the participant at undue risk
8. Prior/concomitant therapy
a. Cyclophosphamide and/or rituximab and/or eculizumab and/or mycophenolate
mofetil within 3 months prior to screening
b. Use of an investigational product within 3 months or 5 half-lives (whichever
is longer) before the first dose of the IMP.
9. Positive serum test at screening for an active viral infection with any of
the following conditions:
a. Hepatitis B virus (HBV) that is indicative of an acute or chronic infection
(https://www.cdc.gov/hepatitis/HBV/PDFs/SerologicChartv8.pdf)
b. Hepatitis C virus (HCV) based on HCV antibody assay
c. HIV based on test results that are associated with an AIDS-defining
condition or a CD4 count <200 cells/mm3
10. Current or history of (ie, within 12 months of screening) alcohol, drug, or
medication abuse
11. Known hypersensitivity reaction to 1 of the components of the IMP or any of
its excipients
12. Female participants wit

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Primary Objectives:<br /><br>To evaluate the safety and tolerability of ARGX-117 compared to placebo in<br /><br>adult participants previously stabilized with IVIg •<br /><br><br /><br>Primary Endpoints:<br /><br>Safety outcomes based on adverse event (AE) monitoring and other safety<br /><br>assessments </p><br>

Secondary Outcome Measures