IVIG (Gamunex-C) Treatment Study for POTS Subjects

• Status: Enrolling by invitation
• Trial end date: 2022-03-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this trial is to evaluate the symptomatic benefits of immunomodulatory treatment with IVIG for POTS (postural tachycardia syndrome) patients with evidence of autoimmunity.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Texas Southwestern Medical Center
• Collaborators: Dysautonomia International; Grifols Biologicals, LLC
• Treatments: gamma-Globulins; Immunoglobulins; Immunoglobulins, Intravenous; Rho(D) Immune Globulin

Criteria

Inclusion Criteria:

• 18 years of age or older, and able to provide informed consent

• Diagnosis of POTS (see Table 1)

• COMPASS-31 symptom score showing moderate to severe autonomic symptoms

• At least 3 of the following clinical or laboratory features of autoimmunity

• One or more serum autoantibodies (ANA ≥ 1:160, gAChR antibody > 0.2 nmol/L, positive ENA, aPL, TTG, gliadin) or inflammatory markers (ESR > 30, CRP > 2, low C3 complement or low immunoglobulin IgG level)

• Confirmed personal history or family history of defined autoimmune disease including Hashimoto's thyroiditis, celiac disease, antiphospholipid syndrome, rheumatoid arthritis, SLE, or Sjogren's syndrome

• Clear history of acute or subacute onset following infection, immunization, injury/concussion, surgery or pregnancy.

• Evidence of esophageal, gastric or intestinal dysmotility (with weight loss)

• Evidence of small fiber neuropathy (abnormal QSART or IENFD)

• Stable oral medical therapy for past 3 months

• Ambulatory at time of screening

Exclusion Criteria:

• Current or previous immunosuppression therapy or IVIG treatment

• Contraindication to intravenous immunoglobulin or intravenous albumin

• Known allergic reactions to blood products including intravenous immunoglobulin (IVIG) and/or subcutaneous immunoglobulin (SCIG), such as history of clinically relevant hemolysis after IVIG infusion, aseptic meningitis, recurrent severe headache, hypersensitivity, severe generalized or severe local skin reaction.

• Inadequate peripheral venous access

• Evidence of renal insufficiency (Cr > 1.5 x elevated) or liver disease (transaminases > 2.5x upper limit) at screening

• History of thrombotic episode within 3 years of enrollment

• Other major medical issue which, in investigators opinion, increases risk for adverse event over the next 12 months or may require separate management.

• Female patients who are premenopausal and are (a) pregnant based on serum pregnancy test, or (b) breast-feeding.