A clinical trial to investigate the safety and tolerability, efficacy pharmacokinetics, pharmacodynamics, and immunogenicity of 2 dose regimens of ARGX-117 in adults with multifocal motor neuropathy
- Conditions
- Multifocal Motor NeuropathyMedDRA version: 21.1Level: PTClassification code 10065579Term: Multifocal motor neuropathySystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2021-003302-50-IT
- Lead Sponsor
- ARGENX BV
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 48
1. Capable of giving signed informed consent 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
at screening confirmed by the MCC
3. Probable or definite MMN according to the EFNS/PNS 2010 guidelines
at screening confirmed by the MCC
4. Receiving a stable IVIg regimen before screening and both of the
following:
a) IVIg treatment interval of 2 to 5 weeks
b) IVIg dose of 0.4 to 2.0 g per kg body weight and infusion
5. IVIg treatment dependency confirmation by the MCC at screening or
at IMV1, 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 12 months after the last IMP administration
b) Women of childbearing potential (WOCBP) must have a negative
serum pregnancy test at screening and a negative urine pregnancy test
at baseline before IMP can be administered
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 36
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 12
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.
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, 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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the safety and tolerability of ARGX-117 compared to placebo<br>in adult participants previously stabilized with IVIg;Secondary Objective: - To evaluate the efficacy of ARGX-117 compared to placebo on muscle<br>strength and/or motor function in adult participants previously<br>stabilized with IVIg<br>- To evaluate the efficacy of ARGX-117 on functional ability, arm and<br>hand function, quality of life, and fatigue in adult participants with MMN<br>- To evaluate the effect of ARGX-117 on health-related productivity and<br>work productivity<br>- To evaluate medication treatment satisfaction<br>- To assess the PK, PD, and immunogenicity of ARGX-117;Primary end point(s): Safety outcomes based on adverse event (AE) monitoring and other<br>safety assessments;Timepoint(s) of evaluation of this end point: Days 1, 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 and 113 + continuous<br>monitoring (CM)
- Secondary Outcome Measures
Name Time Method