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New Treatment for Lipoprotein Lipase Deficiency (AGL12 Trial)

N/A

Recruiting

Led By André Carpentier

Research Sponsored by Université de Sherbrooke

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age 18 to 75 years old

8 healthy LPL-deficient individuals (LPLD subjects) with history of fasting TG > 5 mmol/l and homozygote or compound heterozygote for a LPL-gene mutation

Must not have

Age less than 18 years old

Presence of liver or renal disease; uncontrolled thyroid disorder

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 6 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new treatment for lipoprotein lipase deficiency, which is a condition that causes high triglyceride levels in the blood. The new treatment is designed to help the body clear triglycerides from the blood.

Who is the study for?

This trial is for adults aged 18-75 with Lipoprotein Lipase Deficiency (LPLD), evidenced by high fasting triglyceride levels and specific gene mutations. Healthy individuals with normal blood sugar and triglyceride levels can also join as controls. Participants must not have cardiovascular disease, a history of certain blood disorders or be on medications affecting lipid metabolism.

What is being tested?

The study investigates how LPLD affects the body's handling of dietary fats after eating, specifically looking at fat storage in tissues and its use as fuel for heart function. It involves administering heparin and a liquid meal to understand these metabolic processes in both LPLD patients and healthy controls.

What are the potential side effects?

Potential side effects may include discomfort at the injection site from heparin, increased risk of bleeding due to heparin's blood-thinning properties, and possible gastrointestinal symptoms following the liquid meal.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 18 and 75 years old.

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I have LPL deficiency with high fasting triglycerides and a confirmed LPL gene mutation.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am under 18 years old.

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I do not have liver, kidney diseases, or uncontrolled thyroid issues.

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I am taking medication that thins my blood or prevents blood clots.

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I have heart disease confirmed by my doctor and tests.

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I am not pregnant, breastfeeding, or planning to become pregnant.

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I have had a major bleeding event in the past.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Myocardial DFA uptake

Organ-specific Dietary Fatty Acid (DFA) partitioning

Secondary study objectives

Dietary fatty acid oxidation rate

Insulin sensitivity

Left ventricular function by Positron Emitting Positron (PET) ventriculography

+7 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

4Treatment groups

Experimental Treatment

Group I: LPLD group-A1Experimental Treatment2 Interventions

LPLD group: LPL deficient subjects with history of fasting TG \> 5 mmol/l and homozygote or compound heterozygote for a LPL-gene mutation; A1: with an intravenous (i.v.) heparin bolus (50 IU/kg i.v.) followed by 250 IU/h i.v. during 6 hours starting 15 minutes before ingestion of liquid meal.

Group II: LPLD group-A0Experimental Treatment1 Intervention

LPLD group: LPL deficient subjects with history of fasting TG \> 5 mmol/l and homozygote or compound heterozygote for a LPL-gene mutation; A0: without heparin administered

Group III: Control group-A1Experimental Treatment2 Interventions

Control group: Healthy subjects with fasting glucose \< 5.6, 2-hour post 75g OGTT glucose \< 7.8 mmol/l and HbA1c \< 5.8%; fasting TG \< 1.5 mmol/l); A1: with an intravenous (i.v.) heparin bolus (50 IU/kg i.v.) followed by 250 IU/h i.v. during 6 hours starting 15 minutes before ingestion of liquid meal.

Group IV: Control group- A0Experimental Treatment1 Intervention

Control group: Healthy subjects with fasting glucose \< 5.6, 2-hour post 75g Oral Glucose Tolerance Test (OGTT) glucose \< 7.8 mmol/l and HbA1c \< 5.8%; fasting TG \< 1.5 mmol/l); A0: without heparin administered

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Heparin

2003

Completed Phase 4

~22600

liquid meal

2015

N/A

~70