Ignite Creation Date:
2024-05-05 @ 11:53 PM
Last Modification Date:
2024-10-26 @ 10:41 AM
Study NCT ID:
NCT01448577
Status:
UNKNOWN
Last Update Posted:
2011-10-07
First Post:
2011-10-03
Brief Title:
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
Sponsor:
Amsterdam Molecular Therapeutics
Organization:
Amsterdam Molecular Therapeutics
Study Overview
Official Title:
A Clinical Records Review Study of the Frequency and Severity of Acute Abdominal Pancreatitis Episodes Reported From LPLD Subjects Previously Recruited to Clinical Studies PREPARATION-02 CT-AMT-011-01 and CT-AMT-011-02
Status:
UNKNOWN
Status Verified Date:
2011-10
Last Known Status:
ENROLLING_BY_INVITATION
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 mmoll 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 eg 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
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 eg 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
Detailed Description:
Lipoprotein lipase deficiency LPLD is an autosomal recessive inherited condition caused by homozygosity or compound heterozygosity for mutations within the LPL gene
The most severe clinical complication associated with LPLD is acute pancreatitis Pancreatitis in an LPLD subject often leads to prolonged hospital admissions 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 eg gallstones etc and ultimately allow confirmation of LPLD-related acute pancreatitis Characterisation 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
Alipogene tiparvovec Glybera is in development for the therapy of LPLD In summary alipogene tiparvovec contains the human lipoprotein LPL gene variant LPLS447X in a non-replicating vector in solution administered in a one-time series of intramuscular injections in the armslegs
Studies conducted to date with Glybera have evaluated total triglyceride levels as a surrogate marker for efficacy and have not evaluated a clinical endpoint such as acute pancreatitis episodes as a primary endpoint Post-hoc analysis has suggested that there may be a reduction in the frequency of acute abdominal pancreatitis episodes reported following treatment compared to the frequency reported pre-treatment from past medical history records The recorded episodes used in this post-hoc analysis were collected from medical history and adverse event data but no uniform criteria were used to classify these as episodes of acute pancreatitis Review of the post hoc analysis has raised questions that the recorded past medical history of pancreatitis episodes may be inaccurate with respect to diagnosis and number of episodes
In this case record review study data will be collected on pancreatitis episodes from subjects who previously enrolled in studies PREPARATION-02 observational CT-AMT-011-01 and CT-AMT-011-02 In studies CT-AMT-011-01 and CT-AMT-011-02 subjects received AMT-011 at either dose 3 x 1011 gckg or 1 x 1012 gckg Data obtained from medical records hospital admissiondischarge charts laboratory results and imaging scans will be evaluated for evidence of LPLD-related episodes of pancreatitis by an expert review panel The evaluation will consider data collected from three time periods
subjects entire past medical history
the period after enrolment into study but prior to AMT-011 therapy
the period post-administration of AMT-011
Data from the subjects who did not progress to receive AMT-011 will be evaluated as a control group using data collected from past medical history and from the period after enrolment in the PREPARATION-02
The most severe clinical complication associated with LPLD is acute pancreatitis Pancreatitis in an LPLD subject often leads to prolonged hospital admissions 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 eg gallstones etc and ultimately allow confirmation of LPLD-related acute pancreatitis Characterisation 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
Alipogene tiparvovec Glybera is in development for the therapy of LPLD In summary alipogene tiparvovec contains the human lipoprotein LPL gene variant LPLS447X in a non-replicating vector in solution administered in a one-time series of intramuscular injections in the armslegs
Studies conducted to date with Glybera have evaluated total triglyceride levels as a surrogate marker for efficacy and have not evaluated a clinical endpoint such as acute pancreatitis episodes as a primary endpoint Post-hoc analysis has suggested that there may be a reduction in the frequency of acute abdominal pancreatitis episodes reported following treatment compared to the frequency reported pre-treatment from past medical history records The recorded episodes used in this post-hoc analysis were collected from medical history and adverse event data but no uniform criteria were used to classify these as episodes of acute pancreatitis Review of the post hoc analysis has raised questions that the recorded past medical history of pancreatitis episodes may be inaccurate with respect to diagnosis and number of episodes
In this case record review study data will be collected on pancreatitis episodes from subjects who previously enrolled in studies PREPARATION-02 observational CT-AMT-011-01 and CT-AMT-011-02 In studies CT-AMT-011-01 and CT-AMT-011-02 subjects received AMT-011 at either dose 3 x 1011 gckg or 1 x 1012 gckg Data obtained from medical records hospital admissiondischarge charts laboratory results and imaging scans will be evaluated for evidence of LPLD-related episodes of pancreatitis by an expert review panel The evaluation will consider data collected from three time periods
subjects entire past medical history
the period after enrolment into study but prior to AMT-011 therapy
the period post-administration of AMT-011
Data from the subjects who did not progress to receive AMT-011 will be evaluated as a control group using data collected from past medical history and from the period after enrolment in the PREPARATION-02
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None