IVIG (Gamunex-C) Treatment Study for POTS Subjects (iSTAND)
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|ClinicalTrials.gov Identifier: NCT03919773|
Recruitment Status : Enrolling by invitation
First Posted : April 18, 2019
Last Update Posted : May 13, 2019
|Condition or disease||Intervention/treatment||Phase|
|Postural Tachycardia Syndrome||Drug: IVIG Drug: Albumin||Phase 1 Phase 2|
Gammunex-C, a form of intravenous immunoglobulin (IVIG), is approved for the treatment of chronic inflammatory demyelinating neuropathy (CIDP) or idiopathic thrombocytopenic purpura (ITP). IVIG has been in use for many decades in the treatment of these disorders and many other inflammatory/autoimmune diseases. It is generally very safe and well tolerated. More recently, IVIG has been proposed as an effective treatment for presumed inflammatory neurological disorders which do not meet the criteria for CIDP. Specifically, case reports and cases series have indicated therapeutic responses to IVIG in autonomic neuropathies.
Intravenous Albumin is approved for the treatment of hypovolemia (see attached package insert). The use of albumin to increase plasma volume in patients with POTS has been suggested. In this study, albumin will be used as an active control treatment to provide the same volume and protein load as IVIG but without the immunomodulatory effects.
There have been few well designed clinical therapy trials aimed at POTS patients and even fewer that are aimed at a particular pathophysiological subtype of POTS. Evidence suggests that POTS is a heterogeneous disorder with differing underlying mechanisms. Several uncontrolled case series have suggested a benefit of IVIG for POTS, but the volume expansion associated with infusion of IVIG make it difficult to assess the immunomodulatory effects of this treatment. We propose to evaluate the efficacy of IVIG using a double-blind randomized cross over design that will determine efficacy while reducing effects of inter-subject variability and placebo effect which are common problems in POTS therapy research. Even with the statistical advantages of a crossover design, the treatment cohort will be small, and this study is designed to be a pilot (phase II) study to evaluate the feasibility, tolerability and potential benefits of treatment. The results of this pilot study will provide the impetus and rationale for a larger multicenter clinical trial to definitively evaluate immunomodulatory treatment in POTS.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Crossover Assignment|
|Intervention Model Description:||double-blind placebo controlled crossover pilot study|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Masking Description:||double-blind placebo controlled|
|Official Title:||IVIG (Gamunex-C) Study of Treatment for Autoimmune Neuropathic Dysautonomia/Postural Tachycardia (POTS)|
|Actual Study Start Date :||October 29, 2018|
|Estimated Primary Completion Date :||July 2020|
|Estimated Study Completion Date :||December 2020|
Active Comparator: Treatment Arm
IVIG (Gammunex-C) infusion (0.4 gm/kg) every week for 4 weeks, then every 2 weeks for 8 weeks (12 weeks total).
If you participate in this study there will be 18 scheduled treatment infusions during the 30 week study period. All the study visits and treatment visits will be outpatient visits.
Once you qualify to participate in the study and begin treatment, there will be two 12 week treatment periods separated by a 6 week washout period. The infusion visits will take approximately 3-4 hours each.
Other Name: intravenous immunoglobulin
Placebo Comparator: Treatment Placebo Arm
albumin infusion (0.4 gm/kg) every week for 4 weeks then every 2 weeks for 8 weeks (12 weeks total) during
This will be the matching placebo used in the study.
- Improvement in symptoms measured by change in COMPASS-31 score. [ Time Frame: 12 weeks ]Primary outcome with POTS symptoms
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03919773
|United States, Texas|
|UT Southwestern Medical Center|
|Dallas, Texas, United States, 75208|
|Principal Investigator:||Steven Vernino, MD, PhD||UT Southwestern Medical Center|