E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated | Chronic Inflammatory Demyelinating Polyneuropathy | |
E.1.1.1 | Medical condition in easily understood language | Inflammation of peripheral nervous system | |
E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 | E.1.2 | Level | LLT | E.1.2 | Classification code | 10077384 | E.1.2 | Term | Chronic inflammatory demyelinating polyneuropathy | E.1.2 | System Organ Class | 100000004852 | |
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial | To assess the long-term safety and tolerability of efgartigimod PH20 SC (efgartigimod co-formulated with recombinant human hyaluronidase PH20 [rHuPH20] for subcutaneous [SC] administration) | |
E.2.2 | Secondary objectives of the trial | - To determine the long-term efficacy - To evaluate the immunogenicity (anti-drug antibodies [ADA]) of efgartigimod and rHuPH20 - To evaluate the pharmacokinetics (PK) of efgartigimod PH20 SC - To evaluate the pharmacodynamic (PD) effect of efgartigimod PH20 SC (ie, immunoglobulin G [IgG] levels) - To evaluate additional patient-reported outcomes (PROs) (including patient-reported quality of life and satisfaction with treatment) - To explore self-administration of the treatment | |
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion 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 method of contraception (failure rate of less than 1% per year) from baseline to 90 days after the last administration of IMP 5. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use a condom and his partner must use a highly effective method of contraception (failure rate of less than 1% per year) from baseline to 90 days after the last administration of IMP. Male patients practicing true sexual abstinence (when this is in line with the preferred and usual life style of the participant) can be included. Sterilized male patients who have had vasectomy with documented aspermia post-procedure can be included. In addition, male patients are not allowed to donate sperm from baseline to 90 days after the last administration of IMP. | |
E.4 | Principal exclusion 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. | |
E.5 End points |
E.5.1 | Primary end point(s) | - Incidence of TEAEs and SAEs by system organ class (SOC) and preferred term (PT). - Incidence of clinically significant laboratory abnormalities. | |
E.5.1.1 | Timepoint(s) of evaluation of this end point | TEAEs and SAEs are monitored throughout the entire study duration Blood sampling for laboratory analysis is taken at every study visit | |
E.5.2 | Secondary end point(s) | Efficacy; Change from baseline over time in the following scores and measurements: − Adjusted INCAT score; − MRC Sum score; − 24-item I-RODS disability scores; − Mean grip strength assessed by Martin vigorimeter; − TUG score. Percentage of patients without clinical deterioration over time, defined by adjusted INCAT increase ≥1 point compared to baseline. Immunogenicity; - Percentage of patients with and titers of binding antibodies (BAb) towards efgartigimod and/or rHuPH20; and the presence of neutralizing antibodies (NAb) against efgartigimod and titers of NAb against rHuPH20. Pharmacokinetics; - Efgartigimod serum concentrations over time during the trial. Pharmacodynamics; - Changes from baseline over time of serum IgG levels (total and IgG subtypes) Additional Patient-reported Outcome; Change from baseline over time in: − 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). | |
E.5.2.1 | Timepoint(s) of evaluation of this end point | Efficacy, immunogenicity, pharmacokinetic and pharmacodynamic endpoints are assessed at every study visit Patient report outcomes are assessed at every study visit with the exception of visit 2 (week 4) | |
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description | |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 | The trial involves single site in the Member State concerned | No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 61 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned | Canada | Georgia | Israel | Japan | Russian Federation | Serbia | Ukraine | United States | Belgium | Bulgaria | Czechia | Denmark | France | Germany | Hungary | Italy | Latvia | Netherlands | Poland | Romania | Spain | United Kingdom | |
E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 | Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial | |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |