ALGSA Clinical studies are the key to better understanding ALGS as well as determining the safety and effectiveness of new therapies that are discovered. A clinical study involves research using human volunteers (also called participants) that is intended to add to medical knowledge. There are two main types of clinical studies: clinical trials (also called interventional studies) and observational studies., a database of publicly and privately funded clinical studies conducted around the world, includes both interventional and observational studies. The website is an excellent source of information about clinical studies, what they are and why they matter, and how potential participants can get involved. Click HERE.

In 2018 there are 13 clinical studies in ALGS in various stages of completion being conducted around the world. These clinical studies are listed here and potential participants are encouraged to review them and, should they meet the eligibility criteria for studies that are recruiting or enrolling by invitation, contact the research study coordinator for more information. Each clinical study is very specific about who is eligible to participate and there are exclusion criteria that apply as well so potential participants should review this information carefully before contacting the research study coordinator.

Clinical studies are categorized based on where they are at in the completion process. Clinical studies in ALGS are listed here under their current status, which is subject to change as updates are made on, so it is a good idea to check back frequently to look for updates as well as new clinical studies that might be added in the future.

It is likely that there are other clinical studies on various aspects of ALGS that individuals with the disease may be eligible for even though the studies are not focused specifically on Alagille Syndrome. For instance, a recent search of on “Moya Moya Disease” returned 13 clinical studies of which 4 are actively recruiting around the world. Another recent search of “Tetralogy of Fallot” returned 54 studies of which 13 are actively recruiting participants. In the future some or all of these related clinical studies may be listed here, but in the meantime, potential participants are encouraged to conduct their own searches on and consider enrolling in ones for which they are eligible.

None at this time.
Characterization of Pulmonary Artery Stenoses in Alagille Syndrome – a Medical Record Review
Evaluating the Genetic Causes and Progression of Cholestatic Liver Diseases (LOGIC)
FibroScan™ in Pediatric Cholestatic Liver Disease Study Protocol
A Long-Term, Open-Label Study of LUM001 With a Double-Blind, Placebo Controlled, Randomized Drug Withdrawal Period to Evaluate Safety and Efficacy in Children With Alagille Syndrome
An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS)
An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome
None at this time.
None at this time.
An Open Label, Single-dose, Single Period ADME Study of A4250 in Healthy Subjects
A Safety and Pharmakokinetic Study of A4250 Alone or in Combination With A3384
Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome
Positional Cloning of the Gene(s) Responsible for Alagille Syndrome
Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome
Study of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver Disease
Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease
None at this time.
None at this time.


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