Severe HypertriglyceridemiaPhase 2NCT04541186

Study to Explore the Efficacy and Safety of BIO89-100 (Pegozafermin) in Participants With Severe Hypertriglyceridemia

A Phase 2 study testing Pegozafermin in people with Severe Hypertriglyceridemia, run by 89bio, Inc. at 48 sites in 4 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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). Read more about this study

Who is it for?: Ages 21 to 75 years
Conditions the study is for: Severe Hypertriglyceridemia
What is being tested?: Pegozafermin
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 86 participants
Where: 48 sitesin 4 countries
Study dates: 1 Sep 2020to 31 May 2022
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 21 to 75 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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

Who cannot join

Uncontrolled or newly diagnosed hypertension.
Body mass index >45 kilograms (kg)/meters squared (m^2).
Receiving niacin, proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors, or supplements that could lower lipid levels.
Type 1 diabetes mellitus.
Diagnosis of Type 2 diabetes mellitus <6 months prior to screening.
History of malignancy within 5 years prior to screening.

Show all 9Show fewer

Participants with known lipoprotein lipase impairment or deficiency (Fredrickson Type 1), apolipoprotein C-II deficiency, or familial dysbetalipoproteinemia (Fredrickson Type 3).
Clinically or otherwise documented cardiovascular or cerebrovascular disease.
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.

Trial locations and contacts

48 sites in 4 countries.

Country

City, postcode or hospital

48 sites in 4 countries

United States32 sites

89Bio Clinical Study Site
Albany, New York, 12203
89Bio Clinical Study Site
Austin, Texas, 78705
89Bio Clinical Study Site
Chattanooga, Tennessee, 37421
89Bio Clinical Study Site
Clearwater, Florida, 33756
89Bio Clinical Study Site
Dallas, Texas, 75204
89Bio Clinical Study Site
Dallas, Texas, 77365
89Bio Clinical Study Site
Greenacres City, Florida, 33467
89Bio Clinical Study Site
Greensboro, North Carolina, 27408
89Bio Clinical Study Site
Houston, Texas, 77089
89Bio Clinical Study Site
Jacksonville, Florida, 32205
89Bio Clinical Study Site
Kingsport, Tennessee, 37660
89Bio Clinical Study Site
La Mesa, California, 91942
89Bio Clinical Study Site
Lampasas, Texas, 76550
89Bio Clinical Study Site
Lawrenceville, Georgia, 30044
89Bio Clinical Study Site
Louisville, Kentucky, 40213
89Bio Clinical Study Site
Magnolia, Texas, 77355
89Bio Clinical Study Site
Manassas, Virginia, 20110
89Bio Clinical Study Site
Marion, Ohio, 43303
89Bio Clinical Study Site
Miami, Florida, 33125
89Bio Clinical Study Site
Miami, Florida, 33135
89Bio Clinical Study Site
Miami Lakes, Florida, 33014
89Bio Clinical Study Site
Miami Lakes, Florida, 33016
89Bio Clinical Study Site
Morganton, North Carolina, 28655
89Bio Clinical Study Site
North Miami Beach, Florida, 33162
89Bio Clinical Study Site
Olive Branch, Mississippi, 38654
89Bio Clinical Study Site
Orlando, Florida, 32825
89Bio Clinical Study Site
Quincy, Illinois, 62301
89Bio Clinical Study Site
San Francisco, California, 94158
89Bio Clinical Study Site
Summerville, South Carolina, 29435
89Bio Clinical Study Site
Tampa, Florida, 33606
89Bio Clinical Study Site
Wauconda, Illinois, 60084
89Bio Clinical Study Site
West Des Moines, Iowa, 50266

Other trials in United States

Poland6 sites

89Bio Clinical Study Site
Bialystok, 15-351
89Bio Clinical Study Site
Bialystok, 15-879
89Bio Clinical Study Site
Lodz, 93-338
89Bio Clinical Study Site
Oświęcim, 32-600
89Bio Clinical Study Site
Rzeszów, 35-055
89Bio Clinical Study Site
Torun, 87-100

Other trials in Poland

Czechia5 sites

89Bio Clinical Study Site
Pardubice, 530 02
89Bio Clinical Study Site
Prague, 100 00
89Bio Clinical Study Site
Prague, 14021
89Bio Clinical Study Site
Prague, 150 00
89Bio Clinical Study Site
Prague, 158 00

Other trials in Czechia

Hungary5 sites

89Bio Clinical Study Site
Baja
89Bio Clinical Study Site
Békéscsaba, 5600
89Bio Clinical Study Site
Budapest, 1032
89Bio Clinical Study Site
Debrecen, 4032
89Bio Clinical Study Site
Miskolc, Borsod-Abauj Zemplen county, 3529

Other trials in Hungary

About this study

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

Conditions: Severe Hypertriglyceridemia
Being tested: Pegozafermin, Placebo
Keywords: Hypertriglyceridemia, Dyslipidemia, Lipid disorder, FGF21
Medical subject headings: Hypertriglyceridemia, Dyslipidemias, Lipid Metabolism Disorders

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

PegozaferminDrug
Also called BIO89-100
Subcutaneous injection
PlaceboDrug
Matching placebo

Study groups in NCT04541186
Study group	Role	What they receive
Pegozafermin 9 mg QW	Experimental	Participants received pegozafermin 9 mg QW as an SC injection for 8 weeks in the Main Study cohort. Receives: Drug: Pegozafermin
Pegozafermin 18 mg QW	Experimental	Participants received pegozafermin 18 mg QW as an SC injection for 8 weeks in the Main Study cohort. Receives: Drug: Pegozafermin
Pegozafermin 27 mg QW	Experimental	Participants received pegozafermin 27 mg QW as an SC injection for 8 weeks in the Main Study cohort or Fibrate Expansion cohort. Main Study and Fibrate Expansion pegozafermin 27 mg QW groups were pooled together due to low sample size in the expansion cohort. Receives: Drug: Pegozafermin
Pegozafermin 36 mg Q2W	Experimental	Participants received pegozafermin 36 mg Q2W as an SC injection for 8 weeks in the Main Study cohort. Receives: Drug: Pegozafermin
Placebo	Placebo comparator	Matching placebo was injected at matching frequency per assigned cohort for 8 weeks in the Main Study cohort or Fibrate Expansion cohort. Main Study and Fibrate Expansion placebo groups were pooled together due to low sample size in the expansion cohort. Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Percent Change From Baseline to Week 8 in Serum Triglyceride (TG)
Measured: Baseline, Week 8

Secondary outcomes (7)

Number of Participants Who Achieved TG <500 mg/dL at Week 8
Measured: Week 8
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)
Least Squares Means were calculated using mixed-model repeated measures (MMRM).
Measured: Baseline, Week 8
Percent Change From Baseline to Week 8 in Very Low-density Lipoprotein Cholesterol (VLDL-C) and VLDL-TG
Measured: Baseline, Week 8
Percent Change in Baseline to Week 8 in Fasting Plasma Glucose, Adiponectin, and Body Weight
Least Squares Mean was calculated using MMRM.
Measured: Baseline, Week 8
Percent Change From Baseline to Week 8 in Liver Fat as Assessed by Magnetic Resonance Imaging - Whole Liver Proton Density Fat Fraction (MRI-PDFF)
Least Squares Mean was calculated using analysis of covariance (ANCOVA).
Measured: Baseline, Week 8
Percent Change From Baseline to Week 8 in High-sensitivity C-reactive Protein (hsCRP)
Measured: Baseline, Week 8
Percent Change From Baseline to Week 8 in Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST)
Least Squares Mean was calculated using MMRM.
Measured: Baseline, Week 8

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1 Sep 202031 May 2022

First posted9 Sep 2020
Study start1 Sep 2020
Primary completion19 May 2022
Study completion31 May 2022

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Severe HypertriglyceridemiaStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 86 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04541186
Sponsor's protocol ID: BIO89-100-221
Status last verified: Jun 2024
First submitted: 14 Aug 2020
Last updated: 25 Jul 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

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.
PubMed 37355760

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 2
A Study of Solbinsiran (LY3561774) in Participants With Severe Hypertriglyceridemia
Severe Hypertriglyceridemia
RecruitingPhase 2
A Study to Assess the Safety, Tolerability and Efficacy of ION775
Hypertriglyceridemia, Severe Hypertriglyceridemia
Not yet recruitingPhase 2
A Phase II Study of FXR0906 in Patients With Severe Hypertriglyceridemia or Hypertriglyceridemia With ASCVD.
sHTG, HTG With ASCVD (Diagnosed or at Increased Risk)
RecruitingPhase 3
Study of Plozasiran in Adults With Severe Hypertriglyceridemia at Risk of Acute Pancreatitis
Severe Hypertriglyceridemia
RecruitingPhase 1
A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias
Cardiovascular, Metabolic Disease

All Severe Hypertriglyceridemia trials

Study at a glance

From the registry record, last updated by the sponsor on 25 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT04541186
Acronym / sponsor ID: BIO89-100-221
Conditions: Severe Hypertriglyceridemia
Keywords: Hypertriglyceridemia, Dyslipidemia, Lipid disorder, FGF21
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 86 participants (actual)
Lead sponsor: 89bio, Inc.Industry (pharma, biotech, devices)
Study start: 1 Sep 2020 (actual)
Primary completion: 19 May 2022 (actual)
Study completion: 31 May 2022 (actual)
First posted: 9 Sep 2020 (actual)
Results: Posted 25 Jul 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 5

Arm	Type	Receives
Pegozafermin 9 mg QW	Experimental	Drug: PegozaferminParticipants received pegozafermin 9 mg QW as an SC injection for 8 weeks in the Main Study cohort.
Pegozafermin 18 mg QW	Experimental	Drug: PegozaferminParticipants received pegozafermin 18 mg QW as an SC injection for 8 weeks in the Main Study cohort.
Pegozafermin 27 mg QW	Experimental	Drug: PegozaferminParticipants received pegozafermin 27 mg QW as an SC injection for 8 weeks in the Main Study cohort or Fibrate Expansion cohort. Main Study and Fibrate Expansion pegozafermin 27 mg QW groups were pooled together due to low sample size in the expansion cohort.
Pegozafermin 36 mg Q2W	Experimental	Drug: PegozaferminParticipants received pegozafermin 36 mg Q2W as an SC injection for 8 weeks in the Main Study cohort.
Placebo	Placebo comparator	Drug: PlaceboMatching placebo was injected at matching frequency per assigned cohort for 8 weeks in the Main Study cohort or Fibrate Expansion cohort. Main Study and Fibrate Expansion placebo groups were pooled together due to low sample size in the expansion cohort.

Who may qualify

Ages: 21 to 75 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (5 listed)

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

Key exclusion criteria (9 listed)

Uncontrolled or newly diagnosed hypertension.
Body mass index >45 kilograms (kg)/meters squared (m^2).
Receiving niacin, proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors, or supplements that could lower lipid levels.
Type 1 diabetes mellitus.
Diagnosis of Type 2 diabetes mellitus <6 months prior to screening.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
PegozaferminAlso: BIO89-100Subcutaneous injection	Drug	Pegozafermin 18 mg QW, Pegozafermin 27 mg QW, Pegozafermin 36 mg Q2W, Pegozafermin 9 mg QW
PlaceboMatching placebo	Drug	Placebo

Primary outcome

Percent Change From Baseline to Week 8 in Serum Triglyceride (TG)
Time frame: Baseline, Week 8

7 secondary outcomes

Number of Participants Who Achieved TG <500 mg/dL at Week 8
Time frame: Week 8
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)
Time frame: Baseline, Week 8
Percent Change From Baseline to Week 8 in Very Low-density Lipoprotein Cholesterol (VLDL-C) and VLDL-TG
Time frame: Baseline, Week 8
Percent Change in Baseline to Week 8 in Fasting Plasma Glucose, Adiponectin, and Body Weight
Time frame: Baseline, Week 8
Percent Change From Baseline to Week 8 in Liver Fat as Assessed by Magnetic Resonance Imaging - Whole Liver Proton Density Fat Fraction (MRI-PDFF)
Time frame: Baseline, Week 8
Percent Change From Baseline to Week 8 in High-sensitivity C-reactive Protein (hsCRP)
Time frame: Baseline, Week 8
Percent Change From Baseline to Week 8 in Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST)
Time frame: Baseline, Week 8

Results and safety

Results posted to the registry on 25 Jul 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Pegozafermin 9 mg QW	16	15
Pegozafermin 18 mg QW	17	16
Pegozafermin 27 mg QW	16	10
Pegozafermin 36 mg Q2W	16	15
Placebo	15	14

Primary outcome: Percent Change From Baseline to Week 8 in Serum Triglyceride (TG)

Time frame: Baseline, Week 8. Median (Inter-Quartile Range). Unit: percent change from Baseline

Measure	Pegozafermin n = 64	Placebo n = 17
Value	-57.33 (-69.07 to -32.31)	-11.85 (-23.22 to 7.88)

Pegozafermin vs Placebo: Median Percent Change Difference: -43.73 (95% CI -57.08 to -30.31), p <0.001, van Elteren test

Population analysed: FAS: Participants who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline TG measurement not including EOS visit. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. As pre-specified in the SAP, the sample size in each arm (by dose group) was insufficient (under powered) to effectively evaluate differences in efficacy by dose, therefore, efficacy analysis was performed using total pegozafermin and placebo groups.

7 secondary outcomes are reported in the full results.

Adverse events

Time frame: Day 1 (after dosing) up to 12 weeks. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Pegozafermin 9 mg QW	0 of 12 (0%)	7 of 12 (58.3%)	0 of 12
Pegozafermin 18 mg QW	0 of 21 (0%)	13 of 21 (61.9%)	0 of 21
Pegozafermin 27 mg QW	1 of 18 (5.6%)	13 of 18 (72.2%)	0 of 18
Pegozafermin 36 mg Q2W	0 of 16 (0%)	7 of 16 (43.8%)	0 of 16
Placebo	0 of 18 (0%)	9 of 18 (50%)	0 of 18

Limitations and caveats (from the sponsor): The study did not separately evaluate the response to pegozafermin in participants who were on and who were not on concurrent fibrate therapy due to small sample size in the Fibrate Expansion cohort.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 37355760Bhatt 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.
Individual participant dataSharing planned: No

Related trials for Severe Hypertriglyceridemia

RecruitingPhase 2
A Study of Solbinsiran (LY3561774) in Participants With Severe Hypertriglyceridemia
Severe Hypertriglyceridemia
RecruitingPhase 2
A Study to Assess the Safety, Tolerability and Efficacy of ION775
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Not yet recruitingPhase 2
A Phase II Study of FXR0906 in Patients With Severe Hypertriglyceridemia or Hypertriglyceridemia With ASCVD.
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RecruitingPhase 3
Study of Plozasiran in Adults With Severe Hypertriglyceridemia at Risk of Acute Pancreatitis
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RecruitingPhase 1
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

48 sites in 4 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Pegozafermin in people with Severe Hypertriglyceridemia, run by 89bio, Inc. at 48 sites in 4 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 25 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 48 sites in United States, Poland, Czechia, Hungary. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 25 Jul 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percent Change From Baseline to Week 8 in Serum Triglyceride (TG) (time frame: Baseline, Week 8). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04541186, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Jul 2024.