Clinical trials: Familial Chylomicronemia
Hypertriglyceridemia, Familial Chylomicronemia Syndrome
Study of RN0361in Adult Healthy Subjects and Adult Hypertriglyceridemic Subjects
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and immunogenicity of single doses of RN0361 in Adult healthy subjects and Adult Hypertriglyceridemic Subjects.
- Trial name
- NCT06471543
- Testing
- RN0361
- Where
- 10 sites in United States, Australia
- Volunteers
- 108 people, healthy volunteers welcome
- Sponsor
- Ikaria Bioscience Pty Ltd
LPL Deficiency
Alipogene Tiparvovec for the Treatment of LPLD Patients
The aim of the study is to provide further confirmatory evidence of clinical benefit in LPLD patients treated with alipogene tiparvovec by assessing both the "clinical response" (as defined by a range of parameters), and "the…
- Trial name
- NCT02904772
- Testing
- alipogene tiparvovec, Prednisolone, Cyclosporins
- Where
- 2 sites in United States, Canada
- Sponsor
- UniQure Biopharma B.V.
Familial Chylomicronemia Syndrome
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
The purpose of the study was to evaluate the efficacy of olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.
- Trial name
- BALANCE (NCT04568434)
- Testing
- Olezarsen
- Where
- 47 sites in United States, Canada and 9 more
- Volunteers
- 66 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency
Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)
This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of triglyceride (TG) levels in participants with FCS.
- Conditions
- Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency, Hyperlipoproteinemia Type 1
- Trial name
- NCT03360747
- Testing
- AKCEA-ANGPTL3-LRx
- Where
- Canada
- Volunteers
- 3 people
- Sponsor
- Akcea Therapeutics
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Lipoprotein Lipase Deficiency
Postprandial Fatty Acid Metabolism in Subjects With Lipoprotein Lipase Deficiency
Lipoprotein lipase (LPL) is an enzyme that plays an important role in removing triglycerides (TG) (molecules that transport dietary fat) from the blood.
- Trial name
- AGL12 (NCT04227678)
- Testing
- Heparin, liquid meal
- Where
- Canada
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Université de Sherbrooke
Familial Chylomicronemia
Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
The purpose of this program is to provide expanded access to volanesorsen for up to 100 Patients with Familial Chylomicronemia Syndrome (FCS).
- Trial name
- NCT03544060
- Testing
- Volanesorsen
- Sponsor
- Akcea Therapeutics
Familial Chylomicronemia Syndrome, Multifactorial Chylomicronemia Syndrome
InFocus France Epidemiological Study of Health Burden in Major Hypertriglyceridemia
FCS and MCS patients recruited from 7 academic reference centers were invited to answer a paper or a web questionnaire. Questions encompassed demographics, physical, cognitive and mental symptoms, health care circuit, past and…
- Trial name
- InFocus (NCT04223908)
- Testing
- observational : questionnaries for assessment of health burden
- Where
- France
- Volunteers
- 52 people
- Sponsor
- New French Society of Atherosclerosis
Familial Chylomicronemia Syndrome
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetic (PK) and pharmacodynamic (PD) effects of olezarsen (formerly known as AKCEA -APOCIII-LRX) in participants with FCS previously treated with…
- Trial name
- NCT05185843
- Testing
- Olezarsen
- Where
- 11 sites in United States, Canada and 1 more
- Volunteers
- 24 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Familial Chylomicronemia Syndrome (FCS)
A Randomized, Double-blind, Placebo Controlled Study to Assess Efficacy, Safety and Tolerability of LCQ908 in Subjects With Familial Chylomicronemia Syndrome
The purpose of this study is to determine whether LCQ908 is effective and safe in lowering triglycerides in subjects with Familial Chylomicronemia Syndrome (FCS) (Hyperlipoproteinemia [HLP] type I).
- Trial name
- NCT01514461
- Testing
- LCQ908
- Where
- 14 sites in United States, Canada and 6 more
- Volunteers
- 45 people
- Sponsor
- Novartis Pharmaceuticals
Familial Chylomicronemia Syndrome (FCS) (HLP Type I)
Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome.
This study was to determine long-term safety and tolerability, and continued efficacy in lowering triglycerides of LCQ908 in subjects with Familial Chylomicronemia Syndrome (FCS) (HLP type I).
- Trial name
- NCT01589237
- Testing
- LCQ908
- Where
- 10 sites in United States, Canada and 5 more
- Volunteers
- 38 people
- Sponsor
- Novartis Pharmaceuticals
Lipoprotein Lipase Deficiency, Familial, Hyperlipoproteinemia Type I
Pilot Study To Assess CAT-2003 in Patients With Chylomicronemia
The purpose of this study is to assess safety and efficacy of CAT-2003 in patients with chylomicronemia. The study will evaluate the effects of CAT-2003 on fasting total and chylomicron triglyceride levels, as well as…
- Conditions
- Lipoprotein Lipase Deficiency, Familial, Hyperlipoproteinemia Type I, Chylomicronemia, Familial, Hypertriglyceridemia
- Trial name
- NCT02098278
- Testing
- CAT-2003
- Where
- 2 sites in Canada
- Volunteers
- 13 people
- Sponsor
- Catabasis Pharmaceuticals
Lipoprotein Lipase Deficiency
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
Lipoprotein lipase deficiency (LPLD) is an autosomal recessive inherited condition caused by homozygosity or compound heterozygosity for mutations within the LPL gene.
- Trial name
- NCT01448577
- Where
- 2 sites in Canada
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Amsterdam Molecular Therapeutics
Hyperlipoproteinemia
Safety, Tolerability,Pharmacokinetics(PK)and Pharmacodynamics(PD)Assessment of LCQ908 in Patients With Severe Hypertriglyceridemia
This study will assess safety, tolerability, and effect of LCQ908 on blood lipids in patients with severe hypertriglyceridemia.
- Trial name
- NCT01146522
- Testing
- LCQ908
- Where
- Canada
- Volunteers
- 8 people
- Sponsor
- Novartis Pharmaceuticals