Studies led by investigators in the Division of Autonomic Disorders aim to advance our ability to effectively treat genetic disorders, synucleinopathies, and multiple system atrophy, among other conditions.

Genetic Disorders

Natural History of Familial Dysautonomia (FD)
Principal investigator: Horacio Kaufmann, MD

Natural History of Congenital Insensitivity to Pain with Anhidrosis (CIPA)
Principal investigator: Horacio Kaufmann, MD

Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
Principal investigator: Alejandra Gonzalez-Duarte Briseno, MD

Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
Principal investigator: Alejandra Gonzalez-Duarte Briseno, MD

Synucleinopathies

Natural History Study of Synucleinopathies
Principal investigator: Horacio Kaufmann, MD

Multiple System Atrophy

Clinical Trial Readiness for Multiple System Atrophy (MSA)
Principal investigator: Horacio Kaufmann, MD

The Global Multiple System Atrophy Registry (GLoMSAR)
Principal investigator: Horacio Kaufmann, MD

TRACK-MSA
Principal investigator: Horacio Kaufmann, MD

Phase 3 Efficacy and Durability of Ampreloxetine for the Treatment of Symptomatic nOH in Participants With Multiple System Atrophy (CYPRESS)
Principal investigator: Patricio A. Millar Vernetti, MD

AMULET—Delay Multiple System Atrophy
Principal investigator: Patricio A. Millar Vernetti, MD

UB-312 in Patients With Synucleinopathies
Principal investigator: Horacio Kaufmann

A Study of TAK-341 in Treatment of Multiple System Atrophy
Principal investigator: Patricio A. Millar Vernetti, MD

A Phase 2, double-blind, placebo-controlled, parallel-group study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and potential efficacy of multiple doses of ONO-2808 in patients with Multiple System Atrophy (MSA)
Principal investigator: Patricio A. Millar Vernetti, MD

A Study to Test if TEV-56286 is Effective in Relieving Multiple System Atrophy (MSA): A Safety and Efficacy Study (TOPAS-MSA)
Principal investigator: Patricio A. Millar Vernetti, MD

GDNF Gene Therapy for Multiple System Atrophy
Principal investigator:  Alejandra Gonzalez Duarte Briseno, MD

ATTR Amyloidosis

Autonomic dysfunction and peripheral neuropathy in patients with cardiac ATTR amyloidosis
Principal investigator: Alejandra Gonzalez-Duarte Briseno, MD

Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
Principal investigator: Alejandra Gonzalez-Duarte Briseno, MD

Postural Orthostatic Tachycardia Syndrome (POTS)

Study of Natriuretic Peptide Receptor 1 (NPR1) Antagonist in Adult Patients With Postural Orthostatic Tachycardia Syndrome (POTS)
Principal investigator: Alejandra Gonzalez-Duarte Briseno, MD

Contact Us

To learn more about our clinical trials or to participate, please contact our research coordinators, Britney Paredes Lopez or Grace Nkrumah, at 212-263-7225.