A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02963311
• Date of registration: 10/11/2016
• Prospective Registration: Yes
• Primary sponsor: The Medicines Company
• Public title: A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH) ORION-2
• Scientific title: An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)
• Date of first enrolment: December 13, 2016
• Target sample size: 9
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02963311
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Netherlands, South Africa, United States

Contacts

• Name: Kees Hovingh, MD, PhD
• Affiliation: Department of Vascular Medicine, Academic Medical Center

Key inclusion & exclusion criteria

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.



Age minimum: 12 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Homozygous Familial Hypercholesterolemia

Intervention(s)

Drug: ALN-PCSSC

Drug: Standard of Care

Primary Outcome(s)

Percentage Change From Day 1 to Day 90 in LDL-C [Time Frame: Day 1, Day 90]

Percentage Change From Day 1 to Day 180 (or Final Visit) in LDL-C [Time Frame: Day 1, Day 180 (or Final Visit)]

Secondary Outcome(s)

Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Lipoprotein-a [Time Frame: Day 1, Day 90, Day 180 (or Final Visit)]

Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in VLDL-C [Time Frame: Day 1, Day 90, Day 180 (or Final Visit)]

Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Lipoprotein-a [Time Frame: Day 1, Day 90, Day 180 (or Final Visit)]

Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in HDL-C [Time Frame: Day 1, Day 90, Day 180 (or Final Visit)]

Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Apolipoprotein A1 [Time Frame: Day 1, Day 90, Day 180 (or Final Visit)]

Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in LDL-C [Time Frame: Day 1, Day 90, Day 180 (or Final Visit)]

Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Apolipoprotein A1 [Time Frame: Day 1, Day 90, Day 180 (or Final Visit)]

Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Apolipoprotein B [Time Frame: Day 1, Day 90, Day 180 (or Final Visit)]

Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in VLDL-C [Time Frame: Day 1, Day 90, Day 180 (or Final Visit)]

Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Triglycerides [Time Frame: Day 1, Day 90, Day 180 (or Final Visit)]

Percentage Change From Day 1 to Day 60 and to Day 90 in PCSK9 [Time Frame: Day 1, Day 60, Day 90]

Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in HDL-C [Time Frame: Day 1, Day 90, Day 180 (or Final Visit)]

Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Non-HDL-C [Time Frame: Day 1, Day 90, Day 180 (or Final Visit)]

Absolute Change From Day 1 to Day 60 and to Day 90 in PCSK9 [Time Frame: Day 1, Day 60, Day 90]

Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Non-HDL-C [Time Frame: Day 1, Day 90, Day 180 (or Final Visit)]

Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Total Cholesterol [Time Frame: Day 1, Day 90, Day 180 (or Final Visit)]

Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Triglycerides [Time Frame: Day 1, Day 90, Day 180 (or Final Visit)]

Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Total Cholesterol [Time Frame: Day 1, Day 90, Day 180 (or Final Visit)]

Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Apolipoprotein B [Time Frame: Day 1, Day 90, Day 180 (or Final Visit)]

Secondary ID(s)

MDCO-PCS-16-02

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 18/05/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02963311