Main objective:- To evaluate the long-term safety and tolerability of ARGX-113 in acetylcholine receptor antibody (AChR-Ab) seropositive patients.Secondary objective:- To evaluate the long-term safety and tolerability of ARGX-113 in the overall…
ID
Source
Brief title
Condition
- Autoimmune disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Incidence and severity of AEs, serious adverse events (SAEs), vital signs,
electrocardiogram (ECG) and laboratory assessments over 3 year in
AChR-Ab seropositive patients
Secondary outcome
Incidence and severity of AEs, SAEs, vital signs, ECG and laboratory
assessments over the duration of the trial in the overall population (AChR-Ab
seropositive patients and AChR-Ab seronegative patients).
Background summary
Generalized myasthenia gravis (gMG) is an autoimmune disorder. It is caused by
an error in the transmission of nerve impulses to muscles. It occurs when
normal communication between the nerve and muscle is interrupted at the
neuromuscular junction (the place where nerve cells connect with the muscles
they control). The treatment of gMG is based on a variety of medications and
medical procedures used either alone or in combination. Since the majority of
already existing treatment options can give sideeffects in patients while not
always giving the symptom control needed there is room for improvement.
ARGX-113 wants to be an alternative or addition to the existing therapies
offering more specific modulation of the immune system with less side-effects.
A previous study with ARGX-113 involving 24 patients diagnosed with gMG, has
shown that 75% of the patients treated with ARGX-113 experienced relief in
their symptoms for at least 6 weeks compared with only 25% of patients who
received a placebo.
Study objective
Main objective:
- To evaluate the long-term safety and tolerability of ARGX-113 in
acetylcholine receptor antibody (AChR-Ab) seropositive patients.
Secondary objective:
- To evaluate the long-term safety and tolerability of ARGX-113 in the overall
population (AChR-Ab seropositive and AChR-Ab seronegative
patients).
Study design
This is a 3-year, single-arm, open-label, multicenter, Phase 3 follow-on
extension trial of ARGX 113-1704 to evaluate the long-term safety and
tolerability of ARGX-113 in patients with gMG.
Intervention
A variable number of Treatment Periods consisting of 4 weekly infusions of
ARGX-113 (10 mg/kg of body weight) infused over a period of 3 weeks will be
administered to eligible patients on an *as needed basis* on top of their
standard of care (SoC) in Treatment Periods.
Study burden and risks
For full details see table 1 in the protocol (schedule of assessments) page
11-13
The patient participation in this study will last approximately 3 year. During
this time the patient will visit the hospital approximately 12 times (on a
annual base). The screening visit and the treatment visits will take about 3-4
hours. The other hospital visits will take about 2 hours.
During these visits the following tests and procedures will take place:
- physical examinations will be done and questions will be asked about medical
history.
- ECGs will be done
- weight, height, blood pressure, temperature, heartbeat will be measured
- blood and urine sampling will be taken.
- The research physician will also test female pa rticipants of childbearing
potential for pregnancy.
- Subjects need to complete several questionnaires that will evaluate the
Efficacy and quality of life and for an Suicidality
assessment
Possible side effects that are already known are described in the IB and
patient information letter.
Industriepark Zwijnaarde 7
Zwijnaarde B 9052
BE
Industriepark Zwijnaarde 7
Zwijnaarde B 9052
BE
Listed location countries
Age
Inclusion criteria
Patients will roll over in this trial only if they meet all of the following
criteria:
1. Patients with the ability to understand the requirements of the trial,
provide written informed consent, and can comply with the trial protocol
procedures.
2. Patients who participated in trial ARGX-113-1704 and are eligible for roll
over, as specified in the protocol. , Other more specific inclusion criteria
are further defined in the protocol.
Exclusion criteria
Patients will not roll over in this trial if they meet any of the following
criteria:
1. Patients who discontinued early from trial ARGX-113-1704 or patients who
discontinued early from randomized treatment for pregnancy or rescue reasons or
an (S)AE that might jeopardize the safety of the patient in that trial.
2. Pregnant and lactating women, and those intending to become pregnant during
the trial or within 90 days after the last dosing. Women of childbearing
potential should have a negative urine pregnancy test at SEB.
3. Male patients who are sexually active and do not intend to use effective
methods of contraception during the trial or within 90 days after the last
dosing or male patients who plan to donate sperm during the trial or within 90
days after the last dosing.
4. Patients with known hepatitis B virus (HBV), hepatitis C virus (HCV) or
human immunodeficiency virus (HIV) seropositivity., Other more specific
exclusion criteria are further defined in the protocol.
Design
Recruitment
Medical products/devices used
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Followed up by the following (possibly more current) registration
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Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2018-002133-37-NL |
| CCMO | NL67218.058.18 |
| Other | not available yet |