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Your next step helps guide the way forward

Enrollment in a clinical trial may give your journey more meaning. And while the path is personal, we know the process of choosing to participate in a clinical trial may be challenging. Learn more about trial opportunities and information to discuss with your care team – so you can explore what may be possible in clinical trials, together.

CURRENT AREAS OF RESEARCH

Insmed is committed to helping people with serious diseases. We’re currently conducting research in the following areas:

AT is a rare genetic disorder that affects the nervous system (helps all parts of the body to communicate with each other), immune system (helps the body to fight against infections and other diseases), and other body systems. It is caused by a change (mutation) in the gene that controls the speed at which cells grow and divide. Symptoms of the disease get worse over time and during the lifetime of the AT patient and may include lack of body balance, lack of coordination of body movements, difficulty speaking (slurred speech), difficulty swallowing, unintentional movement of body parts, difficulty coordinating eye movement, and a weak immune system.
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a neurodegenerative disorder that affects motor neurons. Motor neurons are the nerve cells in the brain and spinal cord that control muscle movement. Early symptoms of ALS include muscle cramping and weakness, slurred speech, and difficulty chewing or swallowing. As a progressive condition, these symptoms tend to worsen over time.
DMD is a genetic condition that causes progressive muscle weakness. It primarily affects young males and is caused by a mutation in the gene responsible for producing dystrophin, a protein that helps keep muscles strong.
HS is a long-term inflammatory condition that affects areas of the skin with hair follicles and sweat glands, such as the armpits, groin, under the breasts, and buttocks. It causes painful lumps or boils that can rupture and leak pus, often leading to scarring. In more severe cases, tunnels (sinus tracts) can form under the skin, connecting different lesions.
Pulmonary hypertension (PH) is a rare, progressive disease in which the arteries of the lungs become narrowed and stiff. This causes high blood pressure (hypertension) in these arteries.

Latest Clinical Trials

Learn more about our current clinical trials. Be sure to talk to your doctor and care team to see if one is right for you.

Pulmonary Arterial Hypertension (PAH)
Study to Evaluate the Efficacy and Safety of Once Daily Treprostinil Palmitil Inhalation Powder in Participants with Pulmonary Arterial Hypertension
Trial Status
Active, not yet recruiting
Phase 3
Multiple US locations available
Key Participation Criteria
Diagnosis of PAH confirmed by right heart catheterization
6-minute walking distance ≥150 and ≤450 meters
Must not have obstructive or restrictive lung disease
PALM-PAH
See full study details
Ataxia Telangiectasia (AT)
Longitudinal Study Comparing the Natural History of Ataxia Telangiectasia in Children to Healthy Comparators
Trial Status
Recruiting
Observational, Ongoing
Multiple US locations available
Key Participation Criteria
Aged from 1 year (12 months) and 10 years (<11 years) old, inclusive
Diagnosis of classic AT: Group 1
No diagnosis of any form of AT: Group 2
INSPIRE AT
See full study details
Amyotrophic Lateral Sclerosis (ALS)
A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)
Trial Status
Recruiting
Phase 1
Multiple US locations available
Key Participation Criteria
Participant with symptomatic ALS as diagnosed by Gold Coast diagnostic criteria
Baseline ALSFRS-R ≥ 24
ALS disease duration ≤ 42 months
ARMOR
See full study details
Duchenne Muscular Dystrophy (DMD)
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
Trial Status
Recruiting
Phase 1
Multiple US locations available
Key Participation Criteria
Male, 2-4 years old
Definitive diagnosis of DMD
Ability to walk >10 meters unassisted
ASCEND
See full study details
Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD)
Study to Evaluate the Efficacy and Safety of Treprostinil Palmitil Inhalation Powder in Participants with Pulmonary Hypertension Associated with Interstitial Lung Disease
Trial Status
Recruiting
Phase 3
Multiple US locations available
Key Participation Criteria
Diagnosis of Pulmonary Hypertension WHO Group 3 associated with Interstitial Lung Disease
Pulmonary Hypertension confirmed by right heart catheterization
Must not have primary diagnosis of chronic obstructive pulmonary disease (COPD)
PALM-ILD
See full study details
Hidradenitis Suppurativa (HS)
A Study of the Efficacy and Safety of Brensocatib in Adults With Moderate to Severe Hidradenitis Suppurativa (HS)
Trial Status
Active, no longer recruiting
Phase 2b
Multiple US locations available
Key Participation Criteria
Diagnosis of moderate to severe Hidradenitis Suppurativa (HS) for at least 6 months
Adult
HS lesions in at least 2 distinct anatomic areas
CEDAR
See full study details

As research opens new doors to finding ways to diagnose, prevent, treat, or cure disease and disability, clinical trial participation is essential to help us find the answers.

What’s in it for you?

Getting involved in clinical trials helps to advance our understanding of science and diseases. In addition, some patients who join may gain access to:

Better condition understanding
Access to diagnostic tests and education from research team
Enhanced care and monitoring
Frequent and comprehensive checkups with detailed assessments
Thorough screening process
In-depth evaluations that may affect your condition management
Psychological or emotional value
A proactive approach to address overall health
Personalized support
Gained resources including clinicians and care team
Reimbursement for costs
Participation often includes covering study-related expenses

All clinical trials are different and experiences may vary.

It’s also important to keep these factors in mind when deciding whether to apply to a clinical trial:

Convenience of the trial location
Participation could require significant travel to the nearest trial location
Medication may come with side effects
Early testing results may also uncover potential safety concerns
Effectiveness of the drug
Positive treatment results are not guaranteed for trial participants
Experimental treatment may not be given to participants
Those in the “control” group may be given a placebo instead of investigational drug
Significant time commitment
For trial participants as well as caregivers and loved ones that are involved
Impact on daily life
Participation may disrupt daily routines and activities