This trial is evaluating whether Droxidopa will improve 1 primary outcome in patients with Multiple System Atrophy. Measurement will happen over the course of Up to 10 min of head up tilt.
This trial requires 34 total participants across 2 different treatment groups
This trial involves 2 different treatments. Droxidopa is the primary treatment being studied. Participants will be divided into 2 treatment groups. Some patients will receive a placebo treatment. The treatments being tested are in Phase 1 and are in the first stage of evaluation with people.
MSA is a genetic autoimmune disease whose pathogenesis is not fully understood. The two main hypotheses are:\n1. MSA is an autoimmune disease caused by an autoimmune attack against the nervous system. Some authors believe that the diagnosis is just the first step in the analysis of this pathophysiological mechanism. We would like to present a plausible alternative hypothesis: MSA is a degenerative process which is triggered by some external factor. The external factor could be either the autoimmune attack or the genetic characteristics of the patient. In our opinion, the first explanation is the most plausible.
We show that if a single therapy is appropriate for managing MSA, the same therapy can be successful for treating CFTD. We have found that a multidisciplinary assessment is effective in diagnosing CFTD and MSA. We think that a standardized definition for MSA and CFTD is now essential for guiding treatment plans.
MSA is usually treated with tricyclic antidepressants. Chlorpromazine reduces involuntary muscle movements so antipsychotics are often used to treat involuntary muscles movement. CBT is sometimes used along with medications to treat motor symptoms. In severe cases, cholinesterase inhibitors may be used to treat extrapyramidal symptoms such as dystonia and gait difficulties. In recent years, botulinum toxin A has been used to treat motor symptoms in MSA patients. It is possible to use this treatment in the treatment of MSA as it is beneficial in the alleviation of both motor and cognitive symptoms.
A new diagnosis of MSA is challenging because of the many manifestations of this disorder. The hallmark symptoms of MSA are progressive multiple system degeneration of the autonomic, neurological, and visual systems. Clinical presentation of MSA varies widely but always includes multiple neurologic, neuromotor, and psychiatric complaints. In addition to systemic and neurologic features, MSA presents with many neurocutaneous signs, such as vitiligo, alopecia, nail dystrophy, and palmar-plantar erythrodysesthesia. MSA has two clinically recognized subtypes: a parkinsonian type and a cerebellar/ataxic type.
About 45,000 people per year in the United States are diagnosed with MSA. This makes up 5.5% of all Americans at some point of their life.
There is a progressive loss of autonomic and sensory nervous system function. The autonomic dysfunction results in signs of dry mouth, salivation, urinary retention, anhidrosis (loss of sweating), slowed heart rate, orthostatic hypotension, and other autonomic symptoms. The gradual loss of sensory input to the limbs results in a loss of reflexes. The progressive degeneration of the spinal cord results in weakness, paresis and eventually atrophy of limbs. Some of the features of MSA can be confused with features of Parkinson's disease and essential tremor and can lead to erroneous diagnosis.
If you are wondering what the average age is, you're not sure if you get multiple system atrophy. You may be thinking of the average age of people in your community, or you may have heard that MSA is a rare disease, meaning that it probably is a rare disease. But there is no average age of diagnosis because there is not just one case in every 10,000 that people in your area are diagnosed with MSA.
Results from a recent clinical trial, we did not find significant difference between RCA’s and RRCA’s in terms of age at the time of diagnosis, duration from symptom onset to confirmation diagnosis, age at the time of assessment or treatment; therefore, we should consider clinical trial in patients with motor neuropathy or autonomic neuropathy in addition to the clinical criteria in patients with ataxia, including RCA and RRCA as well.
Our analysis of the cause of MT-CJD and MSA shows that, unlike other neurodegenerative diseases of the CNS the most frequently cited etiological agent, with a proportion of 8% in our population, can be categorized as an unspecified neurological condition. However, because a large proportion of patients with MT-CJD and MSA also have psychiatric disorders, the most frequently cited etiological agent appears to be psychiatric rather than neurological.
Findings from a recent study, we show that a novel and more efficient absorption system using a porous alveolar membrane for a prolonged formulation that is not dependent on the food or gastrointestinal transit time was developed.
There have been clinical trials where droxidopa (Droxilin SRL) has been used for treatment of MSA with clinical and research relevance. It has been used either alone, or in combination with another medication. In all clinical studies, the combination of droxidopa with some other medications has been better than droxidopa alone in slowing down the progression of the disease and improving the symptoms in the long term.(https://www.medscape.com/es/viewarticle/176819-droxidopa-combination-therapy-treats-multiple-systems-atrophy?).
The symptoms of multiple system atrophy often improve a lot while on droxidopa, compared to the placebo. Patients should take droxidopa in smaller doses when symptoms are first starting up or become worse; this will help slow disease progression at the earliest and most effective time. After the disease starts progressing or progressing more quickly than expected, patients who take droxidopa should continue to take the medication in larger doses or take the more effective medication diazepam to help with restlessness or difficulty moving around.