Study to Re-assess and Re-confirm Data Previously Recorded About the Incidence and Severity of Acute Abdominal "Pancreatitis" Episodes in Lipoprotein Lipase Deficient (LPLD) Subjects Previously Enrolled on AMT Clinical Studies

NCT01448577 | Unknown

• 1 other identifier: CT-AMT-011-03
• Study Type: observational
• Target: 22
• Locations: 1 country
• Sites: 2

Brief Summary

Lipoprotein lipase deficiency (LPLD) is an autosomal recessive inherited condition caused by homozygosity or compound heterozygosity for mutations within the LPL gene. LPLD results in subjects presenting with fasting plasma triglyceride (TG) levels of \> 10 mmol/l. LPLD typically presents in infancy or childhood with usual complaints of severe abdominal pain, repetitive colicky pains and repeated episodes of acute pancreatitis The most severe clinical complication associated with LPLD is acute pancreatitis. Pancreatitis in an LPLD subject often leads to prolonged hospital admissions (sometimes up to weeks). Subjects who survive repeated episodes of acute pancreatitis may develop chronic pancreatitis, ultimately resulting in endocrine and exocrine pancreatic insufficiency. The clinical manifestations of acute pancreatitis episodes related to LPLD are largely indistinguishable from acute pancreatitis due to other causes. However, collection of data relating to hospital admissions, laboratory test results, scan images and adverse events occurring concomitantly to the acute pancreatic episode should allow elimination of other causes of pancreatitis (e.g gallstones etc) and ultimately allow confirmation of LPLD-related acute pancreatitis. Characterization of the presentation of symptoms which occur around the time of known episodes of LPLD-related acute pancreatitis should also permit identification of episodes of acute pancreatitis which have previously been considered as unrelated or even unrecognized. The objective of the study is to re-assess and re-confirm data previously recorded about the incidence and severity of acute abdominal "pancreatitis" episodes in LPLD subjects previously enrolled on AMT clinical studies. To assess and document the presentation of acute abdominal episodes that occur around known episodes of pancreatitis and to permit the identification of possible new previously unrecorded episodes of pancreatitis based upon predefined diagnostic criteria. The objective is to recruit the 27 subjects previously enrolled in the above mentioned clinical studies.

Conditions

Lipoprotein Lipase Deficiency

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of acute abdominal "pancreatitis" episodes in LPLD subjects

  To re-assess and re-confirm data previously recorded about the incidence and severity of acute abdominal "pancreatitis" episodes in LPLD subjects previously enrolled on clinical studies PREPARATION-02, CT-AMT-011-02 and CT-AMT-011-02. Acute abdominal episodes will be reviewed and adjudicated using the Atlanta diagnostic criteria for acute pancreatitis

  Retrospective

Secondary Outcomes (5)

• Acute abdominal episodes that occur around known episodes of LPLD pancreatitis

  Retrospective

• Previously unrecorded episodes of pancreatitis

  Retrospective

• Initial onset, duration, and frequency of pancreatitis episodes

  Up to 5 years

• Initial onset and presence of chronic pancreatitis

  Up to 5 years

• initial onset and presence of the late complications of chronic pancreatitis

  Up to 5 years

Study Arms (2)

Dose 3 x 1011 gc/kg

Subjects received AMT-011 at dose 3 x 1011 gc/kg

Dose 1 x 1012 gc/kg

Subjects received AMT-011 at dose 1 x 1012 gc/kg

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Subjects who previously enrolled in studies PREPARATION-02 (observational), CT-AMT-011-01 and CT-AMT-011-02

You may qualify if:

• Subjects must have participated in clinical studies study PREPARATION-02, CT-AMT-011-01 or CT-AMT-011-02,

Sponsors & Collaborators

• Amsterdam Molecular Therapeutics: lead
• The Clinical Trial Company: collaborator

Study Sites (2)

ECOGENE-21 Clinical Trial Center

Chicoutimi, Quebec, G7H 7P2, Canada

Location

La Clinique de Maladies Lipidiques de Quebec Inc. (CMLQ, Inc.)

Québec, Quebec, G1V 4M6, Canada

Location

Related Publications (4)

• Black DM, Sprecher DL. Dietary treatment and growth of hyperchylomicronemic children severely restricted in dietary fat. Am J Dis Child. 1993 Jan;147(1):60-2. doi: 10.1001/archpedi.1993.02160250062018.

  PMID: 8418601 BACKGROUND

• Fortson MR, Freedman SN, Webster PD 3rd. Clinical assessment of hyperlipidemic pancreatitis. Am J Gastroenterol. 1995 Dec;90(12):2134-9.

  PMID: 8540502 BACKGROUND

• Santamarina-Fojo S. The familial chylomicronemia syndrome. Endocrinol Metab Clin North Am. 1998 Sep;27(3):551-67, viii. doi: 10.1016/s0889-8529(05)70025-6.

  PMID: 9785052 BACKGROUND

• Brunzell JD, Deeb SS. (2001). Familial lipoprotein lipase deficiency, Apo C-ІІ deficiency and hepatic lipase deficiency. In: Scriver CR, Beaudet AL, Sly WS, Valle D, eds. The Metabolic Baisis of Inherited Disease. 8th ED, New York, NY: McGraw-Hill: 2789-2816.

  BACKGROUND

Related Links

• Sponsor Homepage

MeSH Terms

Conditions

Hyperlipoproteinemia Type I

Condition Hierarchy (Ancestors)

Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hyperlipoproteinemias; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Daniel Gaudet, MD, PhD

  Ecogene-21 Clinical Trial Center Chicoutimi

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 3, 2011
• First Posted: October 7, 2011
• Study Start: November 1, 2010
• Primary Completion: March 1, 2016
• Study Completion: March 1, 2016
• Last Updated: October 7, 2011

Record last verified: 2011-10

Locations

CA (2)