Open Label Extension of Efgartigimod in Adults With Post-COVID-19 POTS (POTS)

• ClinicalTrials.gov Identifier: NCT05918978
• Recruitment Status: Active, not recruiting
• First Posted: June 26, 2023
• Last Update Posted: May 16, 2024
• Sponsor: argenx
• Collaborator: Iqvia Pty Ltd
• Information provided by (Responsible Party): argenx

Study Description

Brief Summary:

The OLE study aims to investigate the safety, efficacy, pharmacodynamics (PD), pharmacokinetics (PK), and immunogenicity of efgartigimod in participants with post-COVID-19 postural orthostatic.

Condition or disease	Intervention/treatment	Phase
Post-COVID Postural Orthostatic Tachycardia Syndrome Postural Orthostatic Tachycardia Syndrome	Drug: Efgartigimod	Phase 2

Detailed Description:

Study ARGX-113-2105 is a long-term, single-arm, open-label, multicenter extension of the ARGX-113-2104 study, designed to evaluate the long-term safety of efgartigimod IV in adult patients with PC-POTS. Participants will be enrolled from both active and placebo arms of the ARGX-113-2104 study and will receive efgartigimod IV 10 mg/kg in the extension study without knowledge of their prior treatment arm. To be eligible to enroll in this study, participants must have completed the 24-week treatment period of the ARGX-113-2104 study and must not have permanently discontinued the IMP in that study.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 33 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of Efgartigimod in Adult Patients With Post-COVID-19 Postural Orthostatic Tachycardia Syndrome (PC-POTS) Who Completed Study ARGX-113-2104
• Actual Study Start Date: June 20, 2023
• Estimated Primary Completion Date: May 7, 2025
• Estimated Study Completion Date: May 7, 2025

Arms and Interventions

Arm	Intervention/treatment
Experimental: Efgartigimod; Receive efgartigimod IV 10mg/kg infusions during a treatment period of 48 weeks	Drug: Efgartigimod; Participants will receive efgartigimod IV 10 mg/kg open label, respectively.

Outcome Measures

Primary Outcome Measures :

1. Incidence and severity of AESIs. [ Time Frame: Up to 55 weeks ]
   adverse events of special interest

Secondary Outcome Measures :

1. Change from baseline to week 24 and week 48 in COMPASS 31 (modified) [ Time Frame: Up to 48 weeks ]
   COMPASS 31 is a self-rated questionnaire with 31 questions in 6 domains
2. Change from baseline to week 24 and week 48 in MaPS [ Time Frame: Up to 48 weeks ]
   The MaPS questionnaire was developed specifically for patients with POTS by investigators at the Skåne University Hospital, Lund University in Malmö, Sweden. The 12-item evaluation score is being evaluated in a case-control study in patients with POTS compared to healthy controls.
3. Change from baseline to week 24 and week 48 in PGI-S [ Time Frame: Up to 48 weeks ]
4. PGI-C at week 24 and week 48 [ Time Frame: Up to 48 weeks ]
5. Change from baseline to week 24 and 48 in the PROMIS Fatigue Short Form 8a [ Time Frame: Up to 48 weeks ]
6. Change from baseline to week 24 and 48 in the PROMIS Cognitive Function Short Form 6a [ Time Frame: Up to 48 weeks ]
7. Percent reduction from baseline in total IgG levels over the 48-week treatment period [ Time Frame: Up to 48 weeks ]
8. Efgartigimod serum trough concentrations over the 48-week treatment period [ Time Frame: Up to 48 weeks ]
9. Incidence of ADA against efgartigimod over the 48-week treatment period [ Time Frame: Up to 48 weeks ]

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

1. The participant has completed the ARGX-113-2104 study without permanent discontinuation of IMP and agrees to directly roll over into the extension study without discontinuation of IMP.
2. The participant signs the informed consent form, and can comply with OLE study (ARGX-113-2105) protocol requirements.
3. The participant agrees to use contraceptives consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Contraceptive requirements are provided.
4. Female participants of childbearing potential must have a negative urine pregnancy test at baseline before receiving IMP.

Exclusion Criteria:

1. The participant has a clinically significant condition, based on the judgement of the Study Investigator, eg, laboratory abnormalities, 12-lead ECG readings, concomitant medical disease(s), etc., which may place them at undue risk or confound interpretation of study data.
2. The participant intends to become pregnant or start breastfeeding during the study.

Contacts and Locations

Locations

United States, California

UC Sand Diego Sulpizio Cardiovascular Center

La Jolla, California, United States, 92037

Standford Movement Disorder Center

Palo Alto, California, United States, 94304

United States, Illinois

North Shore University HealthSystem

Glenview, Illinois, United States, 60026

United States, Maryland

Johns Hopkins University

Baltimore, Maryland, United States, 21205

United States, Massachusetts

Brigham and Women's Hospital

Boston, Massachusetts, United States, 02115

United States, Ohio

University Hospitals, Neurology Clinical Trials

Cleveland, Ohio, United States, 44106

United States, Texas

Apex Trials Group

Fort Worth, Texas, United States, 76104

Pioneer Clinical Research

Rosharon, Texas, United States, 77583

United States, Utah

Metrodora Institute

West Valley City, Utah, United States, 84119

Sponsors and Collaborators

argenx

Iqvia Pty Ltd

More Information

• Responsible Party: argenx
• ClinicalTrials.gov Identifier: NCT05918978
• Other Study ID Numbers: ARGX-113-2105
• First Posted: June 26, 2023
• Last Update Posted: May 16, 2024
• Last Verified: May 2024

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

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

Keywords provided by argenx:

Tachycardia Post-COVID; Postural Orthostatic Tachycardia Syndrome efgartigimod

Additional relevant MeSH terms:

Postural Orthostatic Tachycardia Syndrome; Tachycardia; Syndrome; Disease; Pathologic Processes; Arrhythmias, Cardiac; Heart Diseases; Cardiovascular Diseases; Cardiac Conduction System Disease; Orthostatic Intolerance; Primary Dysautonomias; Autonomic Nervous System Diseases; Nervous System Diseases