LAL-CSS01: STUDY OF FREQUENCY OF LYSOSOMAL ACID LIPASE DEFICIENCY

• Research type

  Research Study

• Full title

  LAL-CSS01: A STUDY TO IDENTIFY THE FREQUENCY OF LYSOSOMAL ACID LIPASE DEFICIENCY IN AT-RISK PATIENT POPULATIONS

• IRAS ID

  177696

• Contact name

  Mark Bechter

• Contact email

  mark.bechter@synageva.com

• Sponsor organisation

  Synageva BioPharma Corp

• Clinicaltrials.gov Identifier

  NCT02345421

• Duration of Study in the UK

  0 years, 11 months, 19 days

• Research summary

  Lysosomal Acid Lipase Deficiency (LALD) is a disease that causes build-up of fat (lipids) in the liver and other parts of the body. This is due to lack of the enzyme called lysosomal acid lipase, a substance that normally breaks down fat. The build-up causes health problems, including swelling and damage (scarring) of the liver and high cholesterol in the blood. High cholesterol in the blood may increase the risk of heart disease. It can also cause problems due to blocking of arteries in the heart and elsewhere in the body. There are no approved treatments for LALD.

  The purpose of this research study sponsored by Synageva BioPharma Corp. is to see if certain factors can predict if a person is at risk to have LALD. This study will include participants that have one or some of these factors and test to see if they have LALD. These factors will then be used in other patients so that the disease can be recognised more easily.

  About 10,000 participants aged 2 years or over will take part in this study at hospitals and clinics all over the world.

  The planned duration of participation in the study will be for approximately 4 weeks in total. The study involves the following visits and procedures:

  Screening
  • Informed Consent
  • Demographic
  • Medical History
  • Blood Sample – may be required to check the levels of certain naturally occurring substances in the blood.
  • Height and weight

  Visit 1
  • Blood test to determine whether participants have LALD (test for LAL enzyme).
  • Review of side effects if they had a blood test at the screening visit.

  Follow-up phone call - 2-4 weeks after the screening/visit 1:
  • To inform the participant of the results of their LAL enzyme test
  • To review if any side effects from the blood test at visit 1.

• REC name

  London - Bromley Research Ethics Committee

• REC reference

  15/LO/0934

• Date of REC Opinion

  6 Jul 2015

• REC opinion

  Further Information Favourable Opinion