CLINICAL TRIALS PROFILE FOR DROXIDOPA

All Clinical Trials for Droxidopa

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00004478 ↗	Droxidopa in Treating Patients With Neurogenic Hypotension	Completed	Icahn School of Medicine at Mount Sinai	N/A	1999-03-01	Please note that the continuation study can be found at http://clinicaltrials.gov/show/NCT00633880. RATIONALE: Neurogenic hypotension is a fall in blood pressure that occurs when one moves from a lying down to a standing position or after eating a meal. It causes one to feel dizzy, light headed, and weak. Neurogenic hypotension is caused by a problem in the part of the nervous system that controls such functions as heart rate and blood pressure. Droxidopa, a drug that may increase blood pressure, may be an effective treatment for neurogenic hypotension. PURPOSE: Clinical trial to study the effectiveness of droxidopa in treating patients who have neurogenic hypotension.
NCT00547911 ↗	Augmenting Effects of L-DOPS With Carbidopa and Entacapone	Terminated	National Institute of Neurological Disorders and Stroke (NINDS)	Phase 1/Phase 2	2007-10-01	An experimental drug called L-DOPS increases production in the body of a messenger chemical called norepinephrine. Cells in the brain that make norepinephrine are often gone in Parkinson disease. The exact consequences of this loss are unknown, but they may be related to symptoms such as fatigue, depression, or decreased attention that occur commonly in Parkinson disease. This study will explore effects of L-DOPS in conjunction with carbidopa and entacapone, which are drugs used to treat Parkinson disease. We wish to find out what the effects are of increasing norepinephrine production in the brain and whether carbidopa and entacapone augment those effects. Volunteers for this study must be at least 18 years of age and able to give consent to participate in the study. To participate in the study, volunteers must discontinue use of alcohol, tobacco, and certain herbal medicines or dietary supplements, and must also taper or discontinue certain kinds of medications that might interfere with the results of the study. Candidates will be screened with a medical history and physical exam. Participants will be admitted to the National Institutes of Health Clinical Center for two weeks of testing. The study will have three testing phases in a randomly chosen order for each participant: - Single dose of L-DOPS - Single dose of L-DOPS in conjunction with carbidopa - Single dose of L-DOPS in conjunction with entacapone Each phase will last two days, with a washout day between each phase in which no drugs will be given and no testing will be performed. In each phase, participants will undergo a series of tests and measurements, including blood pressure and electrocardiogram tests. Participants who are healthy volunteers will also have blood drawn and will undergo a lumbar puncture (also known as a spinal tap) to obtain spinal fluid for chemical tests.
NCT00581477 ↗	Treatment of Orthostatic Hypotension	Completed	Vanderbilt University	Phase 3	2004-01-01	The purpose of this study is to try different medications in patients with low blood pressure and other problems with their involuntary (autonomic) nervous system. The pharmacological trials in this study will perhaps lead to more effective treatment. This study consists of single dose trials, dose selection trials, 5-day trials and chronic (approximately 2 months) trials.
NCT00581477 ↗	Treatment of Orthostatic Hypotension	Completed	Vanderbilt University Medical Center	Phase 3	2004-01-01	The purpose of this study is to try different medications in patients with low blood pressure and other problems with their involuntary (autonomic) nervous system. The pharmacological trials in this study will perhaps lead to more effective treatment. This study consists of single dose trials, dose selection trials, 5-day trials and chronic (approximately 2 months) trials.
NCT00633880 ↗	Clinical Study of Droxidopa in Patients With Neurogenic Orthostatic Hypotension (NOH)	Completed	Chiltern International Inc.	Phase 3	2008-01-01	The purpose of this study is to see whether droxidopa is effective in treating symptoms of neurogenic orthostatic hypotension in patients with Primary Autonomic Failure (Pure Autonomic Failure, Multiple System Atrophy, Parkinson's Disease), Non-diabetic neuropathy, or Beta Hydroxylase deficiency.
NCT00633880 ↗	Clinical Study of Droxidopa in Patients With Neurogenic Orthostatic Hypotension (NOH)	Completed	Chelsea Therapeutics	Phase 3	2008-01-01	The purpose of this study is to see whether droxidopa is effective in treating symptoms of neurogenic orthostatic hypotension in patients with Primary Autonomic Failure (Pure Autonomic Failure, Multiple System Atrophy, Parkinson's Disease), Non-diabetic neuropathy, or Beta Hydroxylase deficiency.

Clinical Trial Conditions for Droxidopa

Condition Name

Condition Name for Droxidopa
Intervention	Trials
Orthostatic Hypotension	7
Parkinson Disease	6
Dopamine Beta Hydroxylase Deficiency	5
Multiple System Atrophy	5

Condition MeSH

Condition MeSH for Droxidopa
Intervention	Trials
Hypotension	21
Hypotension, Orthostatic	19
Parkinson Disease	11
Autonomic Nervous System Diseases	8

Clinical Trial Locations for Droxidopa

Trials by Country

Trials by Country for Droxidopa
Location	Trials
United States	141
Canada	12
Australia	6
New Zealand	4
Taiwan	1

Trials by US State

Trials by US State for Droxidopa
Location	Trials
New York	11
Tennessee	8
Texas	7
North Carolina	6
Michigan	6

Clinical Trial Progress for Droxidopa

Clinical Trial Phase

Clinical Trial Phase for Droxidopa
Clinical Trial Phase	Trials
Phase 4	5
Phase 3	8
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for Droxidopa
Clinical Trial Phase	Trials
Completed	17
Recruiting	5
Terminated	4

Clinical Trial Sponsors for Droxidopa

Sponsor Name

Sponsor Name for Droxidopa
Sponsor	Trials
Chelsea Therapeutics	15
Vanderbilt University Medical Center	4
H. Lundbeck A/S	4

Sponsor Type

Sponsor Type for Droxidopa
Sponsor	Trials
Industry	25
Other	21
U.S. Fed	3

Droxidopa: Clinical Trials, Market Analysis, and Projections

Introduction

Droxidopa, marketed under the brand name NORTHERA, is a medication designed to treat symptoms of neurogenic orthostatic hypotension (nOH), a condition characterized by a significant drop in blood pressure upon standing. This article delves into the current state of clinical trials, market analysis, and future projections for droxidopa.

Clinical Trials Overview

Phases of Clinical Trials

Clinical trials for droxidopa have progressed through several phases. Here’s a brief overview:

• Phase 1 and 2 Trials: These early phases focus on assessing the safety, side effects, optimal dosages, and risks/benefits of the drug. For droxidopa, these trials established its efficacy in increasing norepinephrine and dopamine levels in patients with nOH[1][4].
• Phase 3 Trials: These trials compare the new treatment with the current standard therapy to determine if it works better and is safer. Droxidopa’s Phase 3 trials, such as Studies 301, 302, and 303, evaluated its effectiveness in reducing symptoms of nOH. However, these studies failed to reach statistical significance in their primary endpoint analyses beyond two weeks of treatment[4].
• Phase 4 Trials: These trials examine the long-term safety and effectiveness of the drug after it has been approved. For droxidopa, ongoing monitoring is necessary to assess its continued benefit and manage potential side effects like supine hypertension[1][4].

Specific Trials and Findings

• Studies 301, 302, and 303: These Phase 3 trials included patients with primary autonomic failure, dopamine beta-hydroxylase deficiency, and non-diabetic autonomic neuropathy. While the trials showed some improvement in symptoms, the effectiveness beyond two weeks was uncertain[4].
• Comparison with Other Treatments: Ampreloxetine, another drug being tested for nOH, has shown promising results in Phase II trials but faces challenges in demonstrating long-term efficacy. Droxidopa’s approval was based on a 0.9-point improvement in the Orthostatic Hypotension Symptom Assessment (OHSA) scale, which, although clinically meaningful, highlights the need for ongoing evaluation[3].

Market Analysis

Market Size and Growth

The droxidopa market is expected to experience significant growth:

• By 2027, the market is forecast to reach $487.1 million, growing at a Compound Annual Growth Rate (CAGR) of 7.8% from 2022 to 2027[2].
• Another projection suggests the market could reach $1.0 billion by 2032, with a CAGR of 11.25%[5].

Market Drivers

Several factors are driving the growth of the droxidopa market:

• Increasing Prevalence of nOH: Conditions such as anemia, dehydration, and endocrine problems contribute to the rise in nOH cases, thereby increasing the demand for droxidopa[2].
• Growing Working Class and Stress Levels: In regions like the Asia Pacific, the increasing working class and stress levels are associated with a higher risk of nOH, boosting the demand for droxidopa[2].
• Research and Development: Ongoing research to treat symptomatic nOH caused by various deficiencies and conditions is driving the market forward[2].

Market Segmentation

The droxidopa market is segmented by:

• Form: Tablets and capsules are the primary forms, with capsules being widely used[2].
• Dosage: The 300 mg dosage segment accounted for approximately 38% of the market share in 2021 and is expected to grow significantly[2].
• Geography: The Asia Pacific region holds the largest share, driven by the growing working class and related symptomatic diseases in countries like China, India, and Japan[2].

Market Challenges

Adverse Effects

Droxidopa is associated with several adverse effects:

• Supine Hypertension: A significant concern, supine hypertension requires careful monitoring and management. Elevating the head of the bed and adjusting doses can help mitigate this risk[4].
• Long-term Efficacy: The effectiveness of droxidopa beyond two weeks is uncertain, necessitating periodic evaluations to ensure continued benefit[4].

Competitive Landscape

The market for nOH treatments is competitive, with other drugs like ampreloxetine being developed:

• Ampreloxetine: This once-daily drug inhibits norepinephrine reuptake and has shown promising results in Phase II trials. However, it faces challenges in demonstrating long-term efficacy and real-world relevance[3].

Projections and Future Outlook

Market Growth Projections

The droxidopa market is expected to continue growing due to several factors:

• Increasing Disease Prevalence: The rise in hypertension and related diseases globally, particularly in low- and middle-income countries, will drive the demand for droxidopa[2].
• Expanding Research: Continued research into treating nOH and related conditions will further boost the market[2].

Regional Growth

The Asia Pacific region is anticipated to remain a significant market:

• Growing Working Class: The increasing working class and associated health issues in countries like China, India, and Japan will continue to drive the demand for droxidopa[2].

Innovations and Developments

Future innovations and developments in the treatment of nOH will be crucial:

• New Dosage Forms: Potential new formulations, such as oral solutions or injectables, could expand the market and improve patient compliance[5].
• Combination Therapies: Studies exploring combination therapies, like the use of pyridostigmine with droxidopa, may offer enhanced treatment options for nOH patients[1].

Key Takeaways

• Clinical Trials: Droxidopa has shown efficacy in reducing symptoms of nOH, but long-term effectiveness remains uncertain.
• Market Growth: The droxidopa market is projected to grow significantly, driven by increasing disease prevalence and ongoing research.
• Adverse Effects: Careful management of supine hypertension and periodic evaluations are necessary for optimal treatment.
• Competitive Landscape: The market is competitive, with other drugs like ampreloxetine being developed.

FAQs

What is droxidopa used for?

Droxidopa is used to treat symptoms of neurogenic orthostatic hypotension (nOH), including dizziness, lightheadedness, and the feeling of fainting in adult patients.

What are the common dosages of droxidopa?

Common dosages include 100 mg, 200 mg, and 300 mg, with the 300 mg dosage being widely used for treating orthostatic dizziness and related symptoms[2].

What are the potential side effects of droxidopa?

Significant side effects include supine hypertension, which requires careful monitoring and management. Other potential side effects include increased heart rate and hypertension[4].

How does droxidopa work?

Droxidopa works by increasing the deficient supply of norepinephrine and dopamine in patients with nOH. It crosses the blood-brain barrier and is converted into norepinephrine via decarboxylation by L-aromatic-amino-acid decarboxylase[2].

What is the current market size and growth projection for droxidopa?

The droxidopa market is forecast to reach $487.1 million by 2027, growing at a CAGR of 7.8% from 2022 to 2027. Another projection suggests it could reach $1.0 billion by 2032 with a CAGR of 11.25%[2][5].

Are there any ongoing clinical trials for droxidopa?

While the primary clinical trials for droxidopa have been completed, ongoing monitoring and periodic evaluations are necessary to assess its continued benefit and manage potential side effects[4].

Sources

1. Mayo Clinic: Droxidopa / Pyridostigmine in Orthostatic Hypotension.
2. IndustryARC: Droxidopa Market Size Report, 2022-2027.
3. Clinical Trials Arena: Theravance's ampreloxetine study needs dramatic improvement in neurogenic orthostatic hypotension to spur market uptake.
4. NORTHERA: Clinical Trials Information.
5. Market Research Future: Droxidopa Market Size, Growth, Trends, Report 2032.