Clinical trial NCT04541186: Study to Explore the Efficacy and Safety of BIO89-100 (Pegozafermin) in Participants With Severe Hypertriglyceridemia
Official title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Explore the Efficacy and Safety of BIO89-100 in Subjects With Severe Hypertriglyceridemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Severe Hypertriglyceridemia
Keywords: Hypertriglyceridemia
Dyslipidemia
Lipid disorder
FGF21
Lead sponsor: 89bio, Inc.
Sponsor class: INDUSTRY
First posted: 2020-09-09
Enrollment: 86
Sex: ALL
Minimum age (years): 21
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study is designed to assess the efficacy, safety, and tolerability of different doses and dose regimens (once weekly \[QW\] or every 2 weeks \[Q2W\]), subcutaneous (SC) dosing of BIO89-100 (pegozafermin) compared to placebo in participants with severe hypertriglyceridemia (SHTG).


Eligibility criteria: Inclusion Criteria:

1. Male or female age ≥21 to ≤75 years.
2. Screening fasting triglyceride ≥500 milligrams (mg)/deciliters (dL) and ≤2000 mg/dL.
3. Willing to maintain current eating and exercise habits from time of signing the informed consent and for the duration of the study.
4. Participants could be taking statins and/or prescription fish oil as background therapy or not be taking any background therapy.
5. Magnetic resonance imaging - whole liver proton density fat fraction (MRI-PDFF) of ≥6% for participants screened for the Fibrate Expansion cohort.

Exclusion Criteria:

1. Uncontrolled or newly diagnosed hypertension.
2. Body mass index \>45 kilograms (kg)/meters squared (m\^2).
3. Receiving niacin, proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors, or supplements that could lower lipid levels.
4. Type 1 diabetes mellitus.
5. Diagnosis of Type 2 diabetes mellitus \<6 months prior to screening.
6. History of malignancy within 5 years prior to screening.
7. Participants with known lipoprotein lipase impairment or deficiency (Fredrickson Type 1), apolipoprotein C-II deficiency, or familial dysbetalipoproteinemia (Fredrickson Type 3).
8. Clinically or otherwise documented cardiovascular or cerebrovascular disease.
9. Weight change ≥5% in 3 months prior to first screening visit or weight change ≥5% during screening or planning to try to lose weight during conduct of study.

Primary outcomes:
measure: Percent Change From Baseline to Week 8 in Serum Triglyceride (TG)
timeFrame: Baseline, Week 8
Secondary outcomes:
measure: Number of Participants Who Achieved TG <500 mg/dL at Week 8
timeFrame: Week 8
measure: Percent Change From Baseline to Week 8 in Non-high-density Lipoprotein Cholesterol (Non-HDL-C), Apolipoprotein B100 (ApoB), Low-density Lipoprotein Cholesterol (LDL-C), and High-density Lipoprotein Cholesterol (HDL-C)
timeFrame: Baseline, Week 8
measure: Percent Change From Baseline to Week 8 in Very Low-density Lipoprotein Cholesterol (VLDL-C) and VLDL-TG
timeFrame: Baseline, Week 8
measure: Percent Change in Baseline to Week 8 in Fasting Plasma Glucose, Adiponectin, and Body Weight
timeFrame: Baseline, Week 8
measure: Percent Change From Baseline to Week 8 in Liver Fat as Assessed by Magnetic Resonance Imaging - Whole Liver Proton Density Fat Fraction (MRI-PDFF)
timeFrame: Baseline, Week 8
measure: Percent Change From Baseline to Week 8 in High-sensitivity C-reactive Protein (hsCRP)
timeFrame: Baseline, Week 8
measure: Percent Change From Baseline to Week 8 in Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST)
timeFrame: Baseline, Week 8
Other outcomes:
Locations:
city: La Mesa
country: United States
facility: 89Bio Clinical Study Site
state: California
city: San Francisco
country: United States
facility: 89Bio Clinical Study Site
state: California
city: Clearwater
country: United States
facility: 89Bio Clinical Study Site
state: Florida
city: Greenacres City
country: United States
facility: 89Bio Clinical Study Site
state: Florida
city: Jacksonville
country: United States
facility: 89Bio Clinical Study Site
state: Florida
city: Miami Lakes
country: United States
facility: 89Bio Clinical Study Site
state: Florida
city: Miami Lakes
country: United States
facility: 89Bio Clinical Study Site
state: Florida
city: Miami
country: United States
facility: 89Bio Clinical Study Site
state: Florida
city: Miami
country: United States
facility: 89Bio Clinical Study Site
state: Florida
city: North Miami Beach
country: United States
facility: 89Bio Clinical Study Site
state: Florida
city: Orlando
country: United States
facility: 89Bio Clinical Study Site
state: Florida
city: Tampa
country: United States
facility: 89Bio Clinical Study Site
state: Florida
city: Lawrenceville
country: United States
facility: 89Bio Clinical Study Site
state: Georgia
city: Quincy
country: United States
facility: 89Bio Clinical Study Site
state: Illinois
city: Wauconda
country: United States
facility: 89Bio Clinical Study Site
state: Illinois
city: West Des Moines
country: United States
facility: 89Bio Clinical Study Site
state: Iowa
city: Louisville
country: United States
facility: 89Bio Clinical Study Site
state: Kentucky
city: Olive Branch
country: United States
facility: 89Bio Clinical Study Site
state: Mississippi
city: Albany
country: United States
facility: 89Bio Clinical Study Site
state: New York
city: Greensboro
country: United States
facility: 89Bio Clinical Study Site
state: North Carolina
city: Morganton
country: United States
facility: 89Bio Clinical Study Site
state: North Carolina
city: Marion
country: United States
facility: 89Bio Clinical Study Site
state: Ohio
city: Summerville
country: United States
facility: 89Bio Clinical Study Site
state: South Carolina
city: Chattanooga
country: United States
facility: 89Bio Clinical Study Site
state: Tennessee
city: Kingsport
country: United States
facility: 89Bio Clinical Study Site
state: Tennessee
city: Austin
country: United States
facility: 89Bio Clinical Study Site
state: Texas
city: Dallas
country: United States
facility: 89Bio Clinical Study Site
state: Texas
city: Dallas
country: United States
facility: 89Bio Clinical Study Site
state: Texas
city: Houston
country: United States
facility: 89Bio Clinical Study Site
state: Texas
city: Lampasas
country: United States
facility: 89Bio Clinical Study Site
state: Texas
city: Magnolia
country: United States
facility: 89Bio Clinical Study Site
state: Texas
city: Manassas
country: United States
facility: 89Bio Clinical Study Site
state: Virginia
city: Pardubice
country: Czechia
facility: 89Bio Clinical Study Site
city: Prague
country: Czechia
facility: 89Bio Clinical Study Site
city: Prague
country: Czechia
facility: 89Bio Clinical Study Site
city: Prague
country: Czechia
facility: 89Bio Clinical Study Site
city: Praha 4
country: Czechia
facility: 89Bio Clinical Study Site
city: Miskolc
country: Hungary
facility: 89Bio Clinical Study Site
state: Borsod-Abauj-Zemplen
city: Baja
country: Hungary
facility: 89Bio Clinical Study Site
city: Budapest
country: Hungary
facility: 89Bio Clinical Study Site
city: Békéscsaba
country: Hungary
facility: 89Bio Clinical Study Site
city: Debrecen
country: Hungary
facility: 89Bio Clinical Study Site
city: Białystok
country: Poland
facility: 89Bio Clinical Study Site
city: Białystok
country: Poland
facility: 89Bio Clinical Study Site
city: Lodz
country: Poland
facility: 89Bio Clinical Study Site
city: Oswiecim
country: Poland
facility: 89Bio Clinical Study Site
city: Rzeszów
country: Poland
facility: 89Bio Clinical Study Site
city: Toruń
country: Poland
facility: 89Bio Clinical Study Site
Study officials:
References:
citation: Bhatt DL, Bays HE, Miller M, Cain JE 3rd, Wasilewska K, Andrawis NS, Parli T, Feng S, Sterling L, Tseng L, Hartsfield CL, Agollah GD, Mansbach H, Kastelein JJP; ENTRIGUE Principal Investigators. The FGF21 analog pegozafermin in severe hypertriglyceridemia: a randomized phase 2 trial. Nat Med. 2023 Jul;29(7):1782-1792. doi: 10.1038/s41591-023-02427-z. Epub 2023 Jun 24.