Registry for patients with Lysosomal Acid Lipase Deficiency

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational, multi-center, international registry aims to collect longitudinal data on patients with Lysosomal Acid Lipase (LAL) Deficiency, a rare lysosomal storage disorder. The registry will gather information to enhance understanding and improve the care and treatment of individuals affected by this condition. Participation is voluntary for both patients and physicians, and the data collected will contribute to a knowledge base that addresses the challenges associated with LAL Deficiency.

Why it matters

Who can join: the full rules

Patients must have a confirmed diagnosis of LAL Deficiency. An Informed Consent and Authorization must be obtained prior to patient enrollment where required under applicable laws and regulations, or a waiver must be obtained by the Institutional Review Board/Independent Ethics Committee.

Patients cannot be currently participating in an Alexion-sponsored clinical trial. Patients who have concluded participation in an Alexion-sponsored sebelipase alfa clinical trial are eligible to enroll in this Registry, and enrollment in the Registry will not exclude a patient from enrolling in a future clinical trial.

Where it's running

• Clinical Trial Site
  Phoenix, Arizona, United States Recruiting
• Clinical Trial Site
  Stanford, California, United States Recruiting
• Clinical Trial Site
  Miramar, Florida, United States Recruiting
• Clinical Trial Site
  Orlando, Florida, United States Recruiting
• Clinical Trial Site
  Atlanta, Georgia, United States Recruiting
• Clinical Trial Site
  Chicago, Illinois, United States Recruiting

+54 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Alexion Pharmaceuticals, Inc. (Sponsor)
• Email: clinicaltrials@alexion.com
• Phone: 1-855-752-2356

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Registry for patients with lysosomal storage diseases

  This study is collecting information from patients with lysosomal storage diseases to see how these conditions progress and how treatments can improve care for them and their families.

  Recruiting University of California, San Francisco · San Francisco, California · NCT05619900

• In utero enzyme replacement therapy for lysosomal storage disorders

  This study is testing whether giving enzyme replacement therapy to unborn babies with lysosomal storage disorders can improve their health and development before they are born.

  Recruiting Phase 1 University of California, San Francisco · San Francisco, California · NCT04532047

• Ravulizumab for Generalized Myasthenia Gravis in Chinese Adults

  This trial tests ravulizumab in Chinese adults with generalized myasthenia gravis to see if it helps symptoms while checking safety and how the drug behaves in the body.

  Recruiting at 8 sites Phase 3 Alexion Pharmaceuticals, Inc. · Beijing and 9 other locations · NCT07596784

• Ravulizumab in Chinese adults with NMOSD

  This study tests ravulizumab in Chinese adults with NMOSD who are AQP4 antibody positive to see if it helps reduce relapses and is safe.

  Recruiting Phase 3 Alexion Pharmaceuticals, Inc. · Beijing and 9 other locations · NCT07557420

• Safety and how the body handles ALXN2230 in healthy adults

  This test gives single under-the-skin doses of ALXN2230 to healthy adults to see if the medicine is safe and how the body processes and responds to it.

  Recruiting Phase 1 Alexion Pharmaceuticals, Inc. · London · NCT07352423

• Ravulizumab for people with clinical aHUS

  This study will test whether ravulizumab raises platelet counts in people diagnosed with clinical atypical hemolytic uremic syndrome (aHUS), including those with certain infections (excluding pneumococcal and STEC), pregnancy/postpartum, recent kidney transplant, malignant hypertension, or autoimmune conditions.

  Recruiting at 3 sites Phase 4 Alexion Pharmaceuticals, Inc. · Bunkyō City and 11 other locations · NCT07308574

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.