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

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

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.

Study Overview

• Status: Recruiting
• Conditions: Post-COVID Postural Orthostatic Tachycardia Syndrome Postural Orthostatic Tachycardia Syndrome
• Intervention / Treatment: Drug: Efgartigimod

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 Type: Interventional
• Enrollment (Estimated): 38
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Sabine Coppieters, MD; Phone Number: 1-857-350-4834; Email: clinicaltrials@argenx.com

Study Locations

• United States
  • California
    • La Jolla, California, United States, 92037
      • Recruiting
      • UC Sand Diego Sulpizio Cardiovascular Center
      • Principal Investigator: Pam Taub, MD
      • Contact: Erika Padilla
    • Palo Alto, California, United States, 94304
      • Recruiting
      • Standford Movement Disorder Center
      • Contact: Brianna Heath
      • Principal Investigator: Mitchel Miglis, MD
  • Illinois
    • Glenview, Illinois, United States, 60026
      • Recruiting
      • North Shore University HealthSystem
      • Contact: Alexandru Barboi, MD
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Recruiting
      • Johns Hopkins University
      • Contact: Stephen Cappello
      • Principal Investigator: Tae Chung, MD
  • Utah
    • West Valley City, Utah, United States, 84119
      • Recruiting
      • Metrodora Institute
      • Contact: Vonda Page
      • Principal Investigator: Anne Maitland, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence and severity of AESIs.	adverse events of special interest	Up to 56 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline to week 24 and week 48 in COMPASS 31 (modified)	COMPASS 31 is a self-rated questionnaire with 31 questions in 6 domains	Up to 48 weeks
Change from baseline to week 24 and week 48 in MaPS	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.	Up to 48 weeks
Change from baseline to week 24 and week 48 in PGI-S		Up to 48 weeks
PGI-C at week 24 and week 48		Up to 48 weeks
Change from baseline to week 24 and 48 in the PROMIS Fatigue Short Form 8a		Up to 48 weeks
Change from baseline to week 24 and 48 in the PROMIS Cognitive Function Short Form 6a		Up to 48 weeks
Percent reduction from baseline in total IgG levels over the 48-week treatment period		Up to 48 weeks
Efgartigimod serum trough concentrations over the 48-week treatment period		Up to 48 weeks
Incidence of ADA against efgartigimod over the 48-week treatment period		Up to 48 weeks

Collaborators and Investigators

• Sponsor: argenx
• Collaborators: Iqvia Pty Ltd

Study record dates

Study Major Dates

• Study Start (Actual): June 16, 2023
• Primary Completion (Estimated): May 1, 2025
• Study Completion (Estimated): May 1, 2025

Study Registration Dates

• First Submitted: June 22, 2023
• First Submitted That Met QC Criteria: June 22, 2023
• First Posted (Actual): June 26, 2023

Study Record Updates

• Last Update Posted (Estimated): January 3, 2024
• Last Update Submitted That Met QC Criteria: January 2, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Keywords: Tachycardia Post-COVID; Postural Orthostatic Tachycardia Syndrome efgartigimod
• Additional Relevant MeSH Terms: Pathologic Processes; Heart Diseases; Cardiovascular Diseases; Nervous System Diseases; Disease; Arrhythmias, Cardiac; Cardiac Conduction System Disease; Autonomic Nervous System Diseases; Primary Dysautonomias; Orthostatic Intolerance; Syndrome; Tachycardia; Postural Orthostatic Tachycardia Syndrome
• Other Study ID Numbers: ARGX-113-2105

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No