Testing a new drug for patients with Postural Orthostatic Tachycardia Syndrome

A Phase 2 Double-Blind Placebo-Controlled Single-Dose Study of Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of REGN7544, an NPR1 Antagonist Monoclonal Antibody, in Patients With Postural Orthostatic Tachycardia Syndrome

This study is testing a new drug called REGN7544 to see if it can help adults with Postural Orthostatic Tachycardia Syndrome feel better and manage their symptoms.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 81 (estimated)
• Ages: 18 Years to 55 Years
• Sex: All
• Sponsor: Regeneron Pharmaceuticals (Industry-sponsored)
• Locations: 15 sites (Gilbert, Arizona and 14 other locations)
• Trial ID: NCT06593600 on ClinicalTrials.gov

What this trial studies

This study investigates the safety, tolerability, and effectiveness of an experimental drug called REGN7544 in adult patients diagnosed with Postural Orthostatic Tachycardia Syndrome (POTS). Participants will be monitored for changes in heart rate and blood pressure, potential side effects, and the drug's pharmacokinetics. The study also aims to assess whether the body produces antibodies against the drug, which could impact its effectiveness. The trial includes both the study drug and a placebo for comparison.

Who should consider this trial

Good fit: Ideal candidates are adults diagnosed with POTS who meet specific diagnostic criteria and have ongoing symptoms.

Not a fit: Patients with other conditions that explain their orthostatic tachycardia or those who do not meet the inclusion criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new treatment option for patients suffering from POTS, potentially improving their quality of life.

How similar studies have performed: While there have been studies on POTS treatments, the specific approach of using NPR1 antagonists is relatively novel and has not been extensively tested.

Eligibility criteria

Key Inclusion Criteria:

1. Is diagnosed with POTS and demonstrates consensus criteria (a), (b), (c) and (d) below during screening:

   1. Increase in HR ≥30 beats per minute (BPM) within 10 minutes of changing from supine to a standing position, as described in the protocol
   2. Absence of orthostatic hypotension, defined as a decrease in systolic blood pressure (SBP) \>20 mm Hg within 3 minutes of standing
   3. Absence of other conditions explaining orthostatic tachycardia in the judgment of the investigator, as defined in the protocol
   4. Ongoing episodic symptoms consistent with POTS (for example, lightheadedness, palpitations, tremulousness, generalized weakness, blurred vision, and fatigue) that are worse with standing and are relieved by lying down and which have been present for ≥3 months
2. During screening, a participant must score ≥3 on the Patient Global Impressions of Severity (PGIS)
3. Has a body mass index between 18 and 35 kg/m2, inclusive

Key Exclusion Criteria:

1. History of hypertension or a seated SBP during screening that is \>140 mm Hg
2. SBP during active stand (AS) test during screening, either supine or standing, that is \>140 mm Hg systolic on ≥2 measurements
3. Increase in HR \<20 BPM within 10 minutes of changing from supine to a standing position, as defined in protocol
4. Is judged by the investigator to have significant heart failure, cardiovascular disease, liver disease, or renal disease (ie, estimated glomerular filtration rate (eGFR) \<60 ml/min/1.73m2) based on medical history, physical exam, laboratory studies, and/or electrocardiogram (ECG) performed during screening period
5. Is confined to bed more than 50% of waking hours
6. Within 5 days of screening visit has used medications with direct effects on blood volume, BP, or HR (eg, midodrine, droxidopa, octreotide, clonidine, methyldopa, ivabradine, beta-blockers, calcium channel blockers, pyridostigmine, fludrocortisone, desmopressin, stimulants or intravenous (IV) saline)

NOTE: Other protocol-defined inclusion/exclusion criteria apply

Where this trial is running

Gilbert, Arizona and 14 other locations

• Mercy Gilbert Medical Center — Gilbert, Arizona, United States (Recruiting)
• North County Neurology Associates — Carlsbad, California, United States (Recruiting)
• Stanford University — Palo Alto, California, United States (Recruiting)
• Southern California Heart Specialists — Pasadena, California, United States (Recruiting)
• Yale University — New Haven, Connecticut, United States (Recruiting)
• Innovative Research of West Florida, Inc. — Clearwater, Florida, United States (Recruiting)
• Indiana University Health Neuroscience Center — Indianapolis, Indiana, United States (Recruiting)
• Saint Luke's MidAmerica Heart Institute — Kansas City, Missouri, United States (Recruiting)
• New York University Langone — New York, New York, United States (Recruiting)
• Columbia University — New York, New York, United States (Recruiting)
• University of Oklahoma Health Sciences Center — Oklahoma City, Oklahoma, United States (Recruiting)
• Vanderbilt University Medical Center — Nashville, Tennessee, United States (Recruiting)
• University of Texas Southwestern — Dallas, Texas, United States (Recruiting)
• University of Utah — Salt Lake City, Utah, United States (Recruiting)
• University of Calgary — Calgary, Alberta, Canada (Recruiting)

Study contacts

• Study coordinator: Clinical Trials Administrator

Conditions Postural Orthostatic Tachycardia Syndrome; Orthostatic intolerance