This study has been transitioned to CTIS with ID 2023-507052-69-00 check the CTIS register for the current data. To evaluate the long-term safety and tolerability of ARGX-117 in adult participants with MMN
ID
Source
Brief title
Condition
- Other condition
- Autoimmune disorders
Synonym
Health condition
Multifocal Motor Neuropathy
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Safety outcomes based on AE monitoring and other safety assessments (clinical
laboratory tests)
Secondary outcome
Modified Medical Research Council (mMRC)
-Value and change from baseline in the mMRC10 sum score
-Proportion of participants showing a deterioration of at least 2 points in
mMRC10 sum score
-Value and change from baseline in the mMRC14 sum score
-Proportion of participants showing a deterioration of at least 2 points in the
mMRC-14 sum score
-Proportion of participants showing a deterioration of 1 or more points in at
least 2 muscle groups as assessed by the mMRC-14 sum score
-Proportion of participants with no deterioration in 2 or more muscle groups as
assessed by mMRC14 sum score
-Value and change from baseline in the average score of the 2 most important
muscle groups as assessed by the mMRC-14 sum score
Grip strength (GS)
-Values, change, and percent change from baseline in GS
-Proportion of participants with a decline of >30% in GS
-Proportion of participants with a GS decrease of 8*kilopascals (kPa) or more
Values and change from baseline in the Rasch-built overall disability scale for
MMN (MMN*RODS)
Values and change from baseline in the average time for the upper extremity
(arm and hand) function (9Hole Peg Test [9-HPT], or timed pegboard test)
Proportion of participants by level of severity on each dimension of EQ-5D-5L
Value and change from baseline in EQ-5D-5L visual analog scale (VAS)
Values and change from baseline in the Chronic Acquired Polyneuropathy
Patient-Reported Index (CAP-PRI)
Values and change from baseline in the 9-item Fatigue Severity Scale (FSS)
Values of the Patient Global Impression of Change (PGIC) scale
Proportion of participants by level of severity of MMN as assessed by the
Patient Global Impression of Severity (PGIS)
Values for work-related and household chore activities of the Health-Related
Productivity Questionnaire (HRPQ) at each visit:
-Hours lost because of absenteeism
-Hours lost because of presenteeism
-Total hours lost
-Percent of scheduled hours lost because of absenteeism
-Percent of scheduled hours lost because of presenteeism
-Percent of scheduled hours lost in total
Serum concentrations and PK parameters for ARGX117
Values and change from baseline in free C2, total C2, and functional complement
activity (CH50) over time
Incidence and prevalence of antidrug antibodies (ADA) against ARGX117
Background summary
This long-term extension trial serves to evaluate the longterm safety and
efficacy of ARGX-117 in adults with multifocal motor neuropathy (MMN).
MMN is a rare neuropathy characterized by progressive asymmetric weakness and
atrophy without sensory abnormalities.
Patients with MMN initially respond to the standard of care (SoC), intravenous
immunoglobulin (IVIg); however, the disease will continue to progress despite
treatment. There is an unmet medical need for an efficacious treatment option
with a more favorable safety and tolerability profile and a shorter duration of
administration than the current SoC.
ARGX-117, a therapeutic complement-inhibiting antibody that targets complement
factor (C2), is being developed to reduce tissue inflammation and attenuate the
adaptive immune response by blocking both the lectin and classical complement
pathways.
Participants in this long-term extension trial will roll over from the
antecedent trial, ARGX1172002, which evaluated the safety and tolerability,
efficacy, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of 3
dose regimens of ARGX-117 administered intravenously (IV) in adult participants
with MMN previously stabilized with IVIg.
Study objective
This study has been transitioned to CTIS with ID 2023-507052-69-00 check the CTIS register for the current data.
To evaluate the long-term safety and tolerability of ARGX-117 in adult
participants with MMN
Study design
This trial is an extension of the antecedent trial ARGX-117-2002. It is a
multicenter trial that has been designed to evaluate the long-term safety and
tolerability, efficacy, immunogenicity, PK, and PD of ARGX-117 IV in adults
with MMN. The trial will include a double-blinded rollover treatment period
(DTP), an open-label treatment period (OTP), and a safety follow-up period.
The investigator, participants, and sponsor trial team (except the sponsor*s
clinical trial supplies team) are blinded to the investigational medicinal
product (IMP) during the DTP and unblinded during the OTP.
Intervention
IMP administration via infusion, see also ARDA study.
Study burden and risks
1 SAE, 'abces', considered not related to IMP
NO SAE's considered related to blinded treatment
No death or life-threatening events reported
See also safety profile in IB
Industriepark Zwijnaarde 7 -
Zwijnaarde (Ghent) B-9052
BE
Industriepark Zwijnaarde 7 -
Zwijnaarde (Ghent) B-9052
BE
Listed location countries
Inclusion criteria
Participants are eligible to be included in the trial only if all of the
following criteria apply:
1. Capable of providing signed informed consent, and complying with protocol
requirements. Participants must be able to read and write.
2. Must have completed the double-blinded treatment period of the ARGX-117-2002
trial and considered to be eligible for treatment with ARGX-117
3. Agrees to use contraceptive measures consistent with local regulations and
the following:
a. Male participants: must use an acceptable contraceptive method that should
be maintained at minimum until 15 months after last dose of Investigational
Medicinal Product (IMP).
b. Female participants (women) of childbearing potential must have a negative
urine pregnancy test at baseline before IMP can be administered.
Exclusion criteria
Participants will be excluded from the trial if any of the following criteria
apply:
1. Clinically significant uncontrolled active or chronic bacterial, viral, or
fungal infection
2. 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.
3. Currently participating in another interventional clinical study
4. Pregnant or lactating or intend to become pregnant during the trial or
within 15 months after last dose of the IMP.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EU-CTR | CTIS2023-507052-69-00 |
| EudraCT | EUCTR2021-004998-32-NL |
| CCMO | NL82342.028.22 |