Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH)

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

18 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-04-22

Study Completion Date

2025-11-30

Brief Summary

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Participants with documented homozygous familial hypercholesterolemia (HoFH) who have provided informed consent will receive 2 open-label doses of ARO-ANG3 and be evaluated for safety and efficacy parameters through 36 weeks. Participants who complete the first 36 week treatment period may opt to continue in an additional 24-month extension period during which they will receive up to 8 doses open-label doses of ARO-ANG3.

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Detailed Description

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Conditions

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Homozygous Familial Hypercholesterolemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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ARO-ANG3 Dose 1

ARO-ANG3 Dose Level 1 subcutaneous (SC)

Group Type EXPERIMENTAL

ARO-ANG 3 Injection

Intervention Type DRUG

Participants will be randomized to receive ARO-ANG3 SC on Day 1 and Day 84 during the initial 36 Weeks of the study and on Day1 and Months 3, 6, 9, 12, 15, 18, and 21 of the extension period

ARO-ANG3 Dose 2

ARO-ANG3 Dose Level 2 SC

Group Type EXPERIMENTAL

ARO-ANG 3 Injection

Intervention Type DRUG

Participants will be randomized to receive ARO-ANG3 SC on Day 1 and Day 84 during the initial 36 Weeks of the study and on Day1 and Months 3, 6, 9, 12, 15, 18, and 21 of the extension period

Interventions

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ARO-ANG 3 Injection

Participants will be randomized to receive ARO-ANG3 SC on Day 1 and Day 84 during the initial 36 Weeks of the study and on Day1 and Months 3, 6, 9, 12, 15, 18, and 21 of the extension period

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Fasting LDL-C \>100 mg/dL at Screening
* Weight of ≥ 40 kg and body mass index ≥ 18.5 and ≤ 40 kg/m2
* Diagnosis of HoFH based on a supportive genetic test or clinical diagnosis
* On stable maximally tolerated lipid lowering therapy
* Willing to abide by stable low-fat, low-cholesterol, heart-healthy diet for at least 4 weeks prior to Day 1
* Participants of childbearing potential (males \& females) must agree to use highly-effective contraception during the study and for at least 24 weeks from the last dose of study medication.
* Women of childbearing potential must have a negative pregnancy test and cannot be breastfeeding
* Women of childbearing potential on hormonal contraceptives must be stable on the medications for \> 2 menstrual cycles prior to Day 1
* Willing to provide written informed consent and to comply with study requirements

Exclusion Criteria

* Current use or use within 365 days from Day 1 of any hepatocyte targeted small interfering RNA oligonucleotides (siRNA) or antisense oligonucleoside molecule
* Use of evinacumab (some exceptions apply)
* Fasting TG \> 300 mg/dL at Screening
* Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins
* Newly diagnosed (within 3 months prior to informed consent) or poorly controlled diabetes (Hemoglobin A1c \> 9%)
* Use of systemic corticosteroids (some exceptions apply)
* Symptoms of myocardial ischemia or severe left ventricular dysfunction
* History of metastatic malignancy within 3 years of Day 1 (some exceptions apply)
* Planned cardiac procedure/surgery such as coronary artery bypass graft (CABG) surgery, percutaneous coronary intervention (PCI), carotid surgery or stenting, or carotid revascularization

Minimum Eligible Age

16 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No