Studies: Dysautonomia disorders and related chronic illnesses

Open Recruitment Studies

Recruiting:  The investigators propose to conduct a safety, tolerability and early proof of concept efficacy study of phosphatidylserine in patients with FD.  The long-term goal is to find an effection nutritional therapy that will improve the quality of life for patients with FD and alter disease prognosis  Contact & participation information is available  - https://clinicaltrials.gov/ct2/show/NCT02276716?cond=Dysautonomia&rank=5

Recruiting:  PET Imaging Study of Neurochemical and Autonomic disorders in Multiple System Atrophy (MSA) from the University of Michigan.
This study aims to better understand the patterns and timings of nerve degeneration relatively early on and how it affects symptoms and progression. 
Eligibility:  30 - 80 years old
Gender:  AllSubject:  diagnosis of MSA or probable MSA of the Parkisonion subtype (MSA-P) or Cerebellar subtype (MSA-C), Subject must be willing and able to give informed consent. Normal cognition as assessed by MiniMental State Examination
Timeline:  Less than 4 years from the time of documented diagnosis
See study online for exclusions and further information. 
https://clinicaltrials.gov/ct2/show/study/NCT02035761?recrs=ab&cond=Dysautonomia+Orthostatic+Hypotension+Syndrome&rank=4 
Contacts:  Arijit K Bhaumik, B.A. CCRP, 734-936-8281 arijit@umich.edu
Edna Rose, Ph.D., RN, MSW, BC, 734-936-7359, ednarose@umich.edu
Estimated Primary Completion Date:  July 2018

Recruiting:  A Study of Pyridostigmine in Postural Tachycardia Syndrome.
This is a 3-day study being conducted by the Mayo Clinic comparing pyridostigmine vs placebo in the treatment of POTS.  Researchers expect pyridostigmine to improve tachycardia and stabilize blood pressure.
Eligibility:  Diagnosis of Postural Tachycardia Syndrome using the following criteria:

• Orthostatic heart rate increment greater than or equal to 30 beats per minute (BPM) within 5 minutes of head-up tilt.

• Symptoms of orthostatic intolerance. These include weakness, lightheadedness blurred vision, nausea, palpitations, and difficulty with concentration and thinking.

• Both criteria must be fulfilled

See study online for exclusions and further information
http://www.mayo.edu/research/clinical-trials/cls-20128843
Mayo Clinic location, Rochester MINN, Principal Investigator:  Dr. Philip Low
Contact:  Tonette Gehrking, CAP, 507-284-4462, gehrking.tonette@mayo.edu

Recruiting:  The Safety and Tolerability of Kinetin, in Patients with Familial Dysautonomia 
The overall objective of this study is to assess the safety and tolerability of administering kinetin in patients with FD. The specific aim of this proposal is to determine the safety of a once-daily dose of kinetin in patients with FD using a dose ascending titration and to determine the long-term safety and tolerability during 3-years of receiving a maximum tolerated steady state dose of kinetin. The investigators hope to also demonstrate early proof of concept that kinetin enhances the ability of the neuronal tissue to correctly splice IKAP mRNA.

Eligibility: Male or female 16 years of age and older, confirmed a diagnosis of familial dysautonomia by genetic testing, written informed consent to participate in the trial and understanding that they can withdraw consent at any time without affecting their future care. And the ability to comply with the requirements of the study procedures.

See study for more info and exclusions:  
https://clinicaltrials.gov/ct2/show/NCT02274051?cond=Dysautonomia&rank=3 
New York University School of Medicine 
Estimated Completion date:  September 2019
Contact:  Horacio Kaufmann MD, 212-263-7225,horacio.kaufmann@nyumc.org Contact:  Jose Martinez, MA, 212-263-7225, jose.martinez@nyumc.org

Recruiting:  Two new studies open for patients with MSA 
The NYU Dysautonomia Center has 2 new clinical trials to test new drugs that are being developed for the treatment of OH in patients with MSA.  Both compounds work by enhancing the body’s levels of norepinephrine. Both studies are also being carried out at the Autonomic Dysfunction Center at Vanderbilt University.  NYU Dysautonomia Center and Vanderbilt are longtime collaborative partners in rare autonomic disorders.

Study 1: A Phase 2 Study to Assess the Effect and Safety of TD-9855 in Subjects with Neurogenic Orthostatic Hypotension

This Study is in collaboration with Theravance Biopharma R & D  They are testing the acute efficacy and safety of a new compound (TD-9855) in improving blood pressure and reducing orthostatic symptoms in patients that have neurogenic orthostatic hypotension (nOH), which includes those with MSA.  This study is also open to patients with Pure Autonomic Failure (PAF) and Parkinson’s Disease (PD) with NOH.

Eligibility: 40 years and older, All Genders

• Diagnosed with symptomatic orthostatic hypotension due to Parkinson’s disease, MSA or PAF (ie.neurogenic orthostatic hypotension)

• At screening, a subject must meet the diagnostic criteria of neurogenic orthostatic hypotension, as demonstrated by a > 30 mm Hg drop in systolic blood pressure (SBP) within 5 minutes of standing.

• Impaired autonomic reflexes, as determined by the absence of BP overshoot during phase IV of the Valsalva maneuver, in subjects where Valsalva is performs as appropriate.

• For the optional open-label extension study subjects must have demonstrated a pressor effect and completed dosing in Cohort 1.

Estimated completion date:  June 2018 
See study for further info and exclusions: https://clinicaltrials.gov/ct2/show/NCT02705755 
NYU Information sheet:  https://dysautonomiacenter.com/2017/04/10/two-new-studies-open-for-patients-with-msa/

Study 2:  Norepinephrine Transporter Blockade, “Autonomic Failure”

This study is supported by The Food and Drug Administration’s Office of Orphan Product development. , We are testing whether we can repurpose an available drug (atomoxetine) as a treatment for neurogenic orthostatic hypotension (nOH). This study is also open to patients with MSA, pure autonomic failure (PAF), and Parkinson disease (PD) with OH.

Eligibility:  40 to 80 years old, any gender, with Neurogenic Orthostatic Hypotension (defined by a reduction of > 30 mmHg drop in SBP within 3 minutes of standing, associated with impaired autonomic reflexes as assessed by autonomic function tests.

Estimated completion date:  July 2020 
Contact:  Bonnie Black, RN, 615-*343-6862, bonnie.black@vanderbilt.edu
Contact: Jose Martinez, 212-263-7225, Jose.Martinez@nyumc.org

See study for further info and exclusions:  https://clinicaltrials.gov/ct2/show/NCT02784535

NYU Information sheet: https://dysautonomiacenter.com/2017/04/10/two-new-studies-open-for-patients-with-msa/

Recruiting:  Vagal Stimulation in POTS - The Autonomic Inflammatory Reflex (Pilot 3) 
The purpose of this study is to investigate how the electrical stimulation of a nerve in the skin of your earlobe (transcutaneous vagal nerve stimulation) affects the way your autonomic nervous system controls your heart rhythm.

Eligibility:  Participants must be female between the ages of 18-45 diagnosed with POTS.

See Study for further information and exclusions: https://www.rarediseasesnetwork.org/cms/autonomic/6111 
This study is a part of the Rare Disease Clinical Research Network and the Autonomic Disorders Consortium 
Contact:  Vanderbilt University Medical Center Nashville 
Misty Hale CCRP, 615-322-2931, misty.hale@Vanderbilt.edu

Recruiting:  The Big POTS Survey is still recruiting  
This study’s lead investigator is Dr. Satish Raj MD MSCI, Adjunct Professor of Medicine at Vanderbilt University’s Autonomic Dysfunction Center.  Dr. Rah says that the information collected as part of this survey “will help us learn more about the possible underlying causes and risk factors for developing POTS, treatments, and the economic, educational and social impact of POTS on patients and their families.”  Dr. Raj serves on the Medical Advisory Board for DINET and Dysautonomia International. DI is sponsoring this survey. 

https://redcap.vanderbilt.edu/surveys/?s=9rB9NkqMrC%20%20