A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
Status: | Completed |
---|---|
Conditions: | High Cholesterol |
Therapuetic Areas: | Cardiology / Vascular Diseases |
Healthy: | No |
Age Range: | 12 - Any |
Updated: | 12/23/2018 |
Start Date: | December 13, 2016 |
End Date: | October 8, 2018 |
An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)
The purpose of this study is to assess the safety, tolerability, and efficacy of ALN-PCSSC in
participants with homozygous familial hypercholesterolemia.
participants with homozygous familial hypercholesterolemia.
Inclusion Criteria:
- Males and females, ≥12 years of age with a diagnosis of homozygous familial
hypercholesterolemia by genetic confirmation or a clinical diagnosis based on a
history of an untreated low-density lipoprotein cholesterol (LDL-C) concentration >500
mg/deciliter (dL) [13 millimoles/liter (mmol/L)] together with either xanthoma before
10 years of age or evidence of heterozygous familial hypercholesterolemia in both
parents.
- Stable on a low-fat diet.
- Stable on pre-existing, lipid-lowering therapies (such as statins,
cholesterolabsorption inhibitors, bile-acid sequestrants, or combinations thereof) for
at least 4 weeks with no planned medication or dose change for the duration of study
participation.
- Fasting central lab LDL-C concentration >130 mg/dL (3.4 mmol/L) and triglyceride
concentration <400 mg/dL (4.5 mmol/L).
- Body weight of 40 kilograms (kg) or greater at screening.
Exclusion Criteria:
- LDL or plasma apheresis within 8 weeks prior to the screening visit, and no plan to
receive it during the study because of the attendant difficulty in maintaining stable
concentrations of LDL-C while receiving apheresis.
- Use of mipomersen or lomitapide therapy within 5 months of screening.
- Previous treatment with monoclonal antibodies directed towards PCSK9 within 8 weeks of
screening.
The above information is not intended to contain all considerations relevant to a
participant's potential participation in a clinical trial.
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