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

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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.


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.
We found this trial at
2
sites
Los Angeles, California
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Los Angeles, CA
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Amsterdam,
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