A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves) (ADHERE+)

• ClinicalTrials.gov Identifier: NCT04280718
• Recruitment Status: Active, not recruiting
• First Posted: February 21, 2020
• Last Update Posted: August 4, 2023
• Sponsor: argenx
• Information provided by (Responsible Party): argenx

Tracking Information

• First Submitted Date: February 20, 2020
• First Posted Date: February 21, 2020
• Last Update Posted Date: August 4, 2023
• Actual Study Start Date: September 18, 2020
• Estimated Primary Completion Date: March 1, 2027 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: December 7, 2022): Incidence of treatment-emergent adverse events and serious adverse events [ Time Frame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study ]
• Original Primary Outcome Measures (submitted: February 20, 2020): Incidence of treatment-emergent adverse events and serious adverse events [ Time Frame: Up to 51 weeks ]

Current Secondary Outcome Measures (submitted: December 7, 2022)

• Change from baseline over time of the adjusted INCAT score [ Time Frame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study ]
• Change from baseline over time of the MRC Sum score [ Time Frame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study ]
• Change from baseline over time of I-RODS disability scores [ Time Frame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study ]
• Change from baseline over time of mean grip strength [ Time Frame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study ]
• Change from baseline over time of TUG score [ Time Frame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study ]
• Percentage of patients without clinical deterioration over time, defined by adjusted INCAT deterioration ≥1 point compared to baseline. [ Time Frame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study ]
• Percentage of patients with titers of binding antibodies towards efgartigimod and the presence of neutralizing antibodies against efgartigimod. [ Time Frame: Up to 51 weeks ]
• Efgartigimod serum concentrations [ Time Frame: Up to 51 weeks ]
• Changes from baseline over time of serum IgG levels (total) [ Time Frame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study ]
• Change from baseline over time in EQ-5D-5L [ Time Frame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study ]
• Change from baseline over time in BPI SF [ Time Frame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study ]
• Change from baseline over time in TSQM-9 [ Time Frame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study ]
• Change from baseline over time in RT-FSS [ Time Frame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study ]
• Change from baseline over time in HADS [ Time Frame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study ]
• Percentage of patients performing self-administration over time [ Time Frame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study ]
• Percentage of patients with treatment administered by caregiver over time. [ Time Frame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study ]

Original Secondary Outcome Measures (submitted: February 20, 2020)

• Change from baseline over time of the adjusted INCAT score [ Time Frame: Up to 48 weeks ]
• Change from baseline over time of the MRC Sum score [ Time Frame: Up to 48 weeks ]
• Change from baseline over time of I-RODS disability scores [ Time Frame: Up to 48 weeks ]
• Change from baseline over time of mean grip strength [ Time Frame: Up to 48 weeks ]
• Change from baseline over time of TUG score [ Time Frame: Up to 48 weeks ]
• Percentage of patients without clinical deterioration over time, defined by adjusted INCAT deterioration ≥1 point compared to baseline. [ Time Frame: Up to 51 weeks ]
• Percentage of patients with and titers of binding antibodies towards efgartigimod and/or rHuPH20 and the presence of neutralizing antibodies against efgartigimod and titers of NAb against rHuPH20. [ Time Frame: Up to 51 weeks ]
• Efgartigimod serum concentrations over time during the trial [ Time Frame: Up to 51 weeks ]
• Changes from baseline over time of serum IgG levels [ Time Frame: Up to 51 weeks ]
• Change from baseline over time in EQ-5D-5L [ Time Frame: Up to 48 weeks ]
• Change from baseline over time in BPI SF [ Time Frame: Up to 48 weeks ]
• Change from baseline over time in TSQM-9 [ Time Frame: Up to 48 weeks ]
• Change from baseline over time in RT-FSS [ Time Frame: Up to 48 weeks ]
• Change from baseline over time in HADS [ Time Frame: Up to 48 weeks ]
• Percentage of patients performing self-administration over time [ Time Frame: Up to 48 weeks ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves)
• Official Title: Open-label Extension of the ARGX-113-1802 Trial to Investigate the Long-term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Patients With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Brief Summary

This is the open-label extension study of phase II ARGX-113-1802 to evaluate the long-term safety and efficacy of the subcutaneous formulation of efgartigimod in adults with CIDP.

Patients already stabilized on efgartigimod PH20 SC will also have the opportunity to participate in a sub study to explore less frequent dosing of efgartigimod PH20 SC.

• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Intervention

Biological: Efgartigimod PH20 SC

Subcutaneous administration of efgartigimod

Other Name: ARGX-113

Study Arms

Experimental: efgartigimod PH20 SC

Patients treated with efgartigimod PH20 SC

Intervention: Biological: Efgartigimod PH20 SC

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Actual Enrollment (submitted: August 3, 2023): 226
• Original Estimated Enrollment (submitted: February 20, 2020): 360
• Estimated Study Completion Date: March 1, 2027
• Estimated Primary Completion Date: March 1, 2027 (Final data collection date for primary outcome measure)

Eligibility Criteria

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 an acceptable method of contraception from signing the ICF until the date of the last administration of IMP.

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.
3. Patients with clinical evidence of other significant serious disease or patients who underwent a recent or have a planned major surgery, or patients who (intend to) use prohibited medications (see protocol) and therapies during the trial, or any other reason which could confound the results of the trial or put the patient at undue risk.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Austria, Belgium, Bulgaria, China, Czechia, Denmark, France, Georgia, Germany, Israel, Italy, Japan, Latvia, Netherlands, Poland, Romania, Russian Federation, Serbia, Spain, Taiwan, Turkey, Ukraine, United Kingdom, United States

Administrative Information

• NCT Number: NCT04280718
• Other Study ID Numbers: ARGX-113-1902; 2019-003107-35 ( EudraCT Number )
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Undecided

• Current Responsible Party: argenx
• Original Responsible Party: Same as current
• Current Study Sponsor: argenx
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: argenx
• Verification Date: August 2023