Why we urgently need the help of patients with MSA to develop a novel treatment for low blood pressure

Low blood pressure in the upright position (orthostatic hypotension, OH) is common among patients with multiple system atrophy (MSA). OH can cause lightheadedness, weakness, troublesome fatigue, or feeling faint when standing, and can result in injuries and falls. At present, treatment options for OH are limited, often not sufficiently effective or associated with problematic side effects. New approaches to treat OH are therefore much needed for better management.


Clinical trials are the key step in advancing potential new therapies from the research setting to the clinic. The most critical element for any trial to succeed is the willingness of the patients to participate. Theravance BioPharma has taken on the challenge to find a new treatment for OH, which based on the science, may be a particularly promising new medication for patients with MSA.  So much so, that the trial was designed with a proportion of enrollment slots reserved for patients with MSA to participate.

A number of medical centers with expertise in MSA, including NYU’s Dysautonomia Center and the Mayo Clinic, are very supportive of this undertaking, and are enrolling patients into this study. We have an ideal window of opportunity to find candidates with MSA, seeking care for their OH, who can help us test a potential new treatment, explained Dr. Wolfgang Singer – a world expert in MSA at the Mayo Clinic.

images-1We encourage you to consider participating in this study if you have been diagnosed with MSA and have symptoms of OH. – Wolfgang Singer, MD, MSA Expert

Q1: How do I know if I qualify for the study?

The study is looking to enroll patients with MSA who have symptoms of low blood pressure when they are upright. You cannot be enrolled if you are currently enrolled in another treatment trial, if you have poorly controlled diabetes, take insulin, or take medications to treat high blood pressure. Several other criteria must be fulfilled in order to qualify, and those will be screened for by the study physicians when you decide you may want to participate.

Q2: Where can I participate?

You will have to be evaluated at one of the study sites to confirm your diagnosis, and to find out if you qualify for the study. The study sites include NYU and Mayo Clinic Rochester. There are also over one hundred other treatment sites that might be geographically closer to you (click here, and scroll down to locations).

Q3: If I have MSA and symptoms of low blood pressure, am I guaranteed to be enrolled if I go to one of the study sites?

No. There are a number of criteria to be fulfilled before you can be enrolled, and it may turn out that you do not qualify after undergoing screening. One quick easy way to determine if you might have OH is to measure your blood pressure lying and standing and see if it falls by 20/10 mmHg within 3 minutes. You can also call our study coordinators at each of the sites, who can provide more guidance.

Q4: What does the study involve?

The study involves a screening visit, 5 visits while on treatment (each about 1 week apart), and a follow-up visit after treatment. If you are taking midodrine or droxidopa (Northera), you will have to come off those medications for the duration of the study. During the treatment phase, you will be asked to take the study medication by mouth once a day. You may receive the actual study medication or a placebo (50/50 chance). You would be in the study up to approximately 10 weeks. Besides taking study medication, procedures at the different study visits include a medical history and examination, standing and tilt table testing, EKG, questionnaires, and blood work.

Screen Shot 2020-03-10 at 2.27.26 PMClinical trials in rare diseases benefit from flexibility, offering willing participants options when it comes to how they are carefully followed in a trial  – Lucy Norcliffe-Kaufmann, PhD

Recognizing that travel to and from a site to attend visits in-person might be a barrier for some patients to participate, under certain circumstances, it may be possible to complete some of the study visits remotely. This is something that you can discuss with the study team. If you complete the study, you may be eligible to enroll in an open-label study of this medication, which does not include placebo and you would for sure receive the study drug for 16 weeks.


Not wanting to travel? You can discuss with us the possibility of remote virtual visits.
Not wanting to travel? Talk with our study teams about possibly doing some of your visits virtually.

Q5: Who do I contact if I am interested?

The study coordinators at NYU and Mayo will be happy to talk to you and discuss potential participation. They are knowledgeable staff, who work closely with our physicians to support patients through the logistics of a study. Feel free to call or email to learn more.

NYU: Jose Martinez E-mail: Jose.Martinez4@nyulangone.org, phone 212-263-7225

Mayo Clinic: Kristine Severson E-mail: severson.kristine@mayo.edu, phone: 507-422-5314