A Phase 2 Trial of ARGX-113 PH20 SC in Adult Patients with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) (Open-label Extension)

JRCT ID: jRCT2051210001

Registered date:02/04/2021

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: CIDP
• Date of first enrollment: 02/04/2021
• Target sample size: 180
• Countries of recruitment: US,Japan,Canada,Japan,UK,Japan,France,Japan,Russia,Japan,Belgium,Japan,Bulgaria,Japan,Czech Republic,Japan,Denmark,Japan,Germany,Japan,Hungary,Japan,Italy,Japan,Netherlands,Japan,Poland,Japan,Romania,Japan,Spain,Japan,Georgia,Japan,Israel,Japan,Serbia,Japan,Ukraine,Japan,Latvia,Japan
• Study type: Interventional
• Intervention(s): Administration of Efgartigimod PH20 SC weekly

Outcome(s)

Primary Outcome

1.Safety and tolerability of the drug:
- Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) by system organ class (SOC) and preferred term (PT);
- Incidence of clinically significant laboratory abnormalities.

Secondary Outcome

1.Change from baseline over time in the following scores and measurements:
- Adjusted INCAT score;
- Medical Research Council (MRC) sum score;
- 24-item Inflammatory Rasch-built Overall Disability Scale (I-RODS) disability scores;
- Mean grip strength assessed by Martin vigorimeter;
- Timed up and go (TUG) score.
2.Percentage of patients without clinical deterioration over time, defined by adjusted INCAT deterioration >=1 point compared to OLE SD1.
3.Percentage of patients with and titers of binding antibodies (BAb) toward efgartigimod and/or rHuPH20; and the presence of neutralizing antibodies (NAb) against efgartigimod and titers of NAb against rHuPH20.
4.Efgartigimod serum concentrations over time during the trial.
5.Changes from baseline over time of serum IgG levels (total)
6.Change from baseline over time in:
- EuroQol 5 dimensions and 5 levels health-related quality-of-life questionnaire (EQ-5D-5L);
- Brief Pain Inventory - Short Form (BPI-SF);
- 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9);
- Rasch-transformed-Fatigue Severity Scale (RT-FSS);
- Hospital Anxiety and Depression Scale (HADS).
7.Percentage of patients performing self-administration over time.

Key inclusion & exclusion criteria

• Age minimum: >= 20age 00month 00weeks old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1.Ability to understand the requirements of the trial, provide written informed consent (including consent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including required trial visits) of this trial.
2.Male or female patient with one of the following options:
- Have completed the week-48 visit of Stage B of the ARGX-113-1802 trial and are considered to be eligible for treatment with efgartigimod PH20 SC; or
- Have deteriorated during Stage B of the ARGX-113-1802 trial and are considered to be eligible for treatment with efgartigimod PH20 SC, or
- Have been offered the participation in the OLE trial due to early termination of the ARGX-113-1802 trial (because sufficient events for the primary endpoint analysis of the that trial have been reached and it is stopped) and are considered to be eligible for treatment with efgartigimod PH20 SC treatment; or
- Have completed the week-48 visit of the previous cycle of the OLE trial and are considered to be eligible to continue with efgartigimod PH20 SC treatment.
3.Women of childbearing potential who have a negative urine pregnancy test at baseline before IMP administration.
4.Women of childbearing potential must use a highly effective or acceptable method of contraception from baseline to 90 days after the last administration of IMP.
5.Male patients agree not to donate sperm during the trial period and 90 days thereafter.

Exclude criteria

1.Week-48/ED visit in the ARGX-113-1802 trial or the week-48 visit of the previous OLE participation occurred more than 14 days prior to SD1 of the OLE trial or the start of a new treatment cycle in the OLE trial and more than 21 days since the last dose of IMP.
2.Pregnant and lactating women and those intending to become pregnant during the trial or within 90 days after last IMP administration.
3.Patients with clinical evidence of other significant serious disease or patients who underwent a recent or have a planned major surgery, or any other reason which could confound the results of the trial or put the patient at undue risk.

Related Information

• Primary Sponsor: Tsuruyama Eri
• Secondary ID(s): NCT04280718,2019-003107-35