Homozygous Familial HypercholesterolemiaPhase 3ODYSSEY HoFHNCT03156621
Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
A Phase 3 study testing Alirocumab in people with Homozygous Familial Hypercholesterolemia, run by Regeneron Pharmaceuticals at 28 sites in 13 countries.
Trial summary
The primary objective of the study is to demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) with alirocumab subcutaneous (SC) every 2 weeks (Q2W) in comparison to placebo after 12 weeks of treatment. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Homozygous Familial Hypercholesterolemia
- What is being tested?
- Alirocumab
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 69 participants
- Where
- 28 sitesin 13 countries
- Study dates
- 3 Oct 2017to 13 Feb 2020
- Phase
- Phase 3
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of HoFH by at least 1 of the following genotype or clinical criteria (all patients on LDL apheresis must be diagnosed based on genotype):
- Documented homozygous or compound heterozygous mutations in both low-density lipoprotein receptor (LDLR) alleles
- Presence of homozygous or compound heterozygous mutations in Apo B, PCSK9 or LDL receptor adaptor protein 1 (LDLRAP1)
- Presence of double heterozygous mutations, i.e, mutations on different genes in the LDLR, Apo B or PCSK9 alleles
- Untreated TC >500 mg/dL (12.93 mmol/L) and TG <300 mg/dL (3.39 mmol/L) AND Both parents with history of TC >250 mg/dL (6.46 mmol/L) OR cutaneous or tendinous xanthoma before age 10
- Receiving a stable dose of a statin at the screening visit (documentation if statin ineffective or patient unable to tolerate statin)
- If undergoing LDL apheresis, must have initiated LDL apheresis at least 3 months prior to screening and must have been on a stable weekly (every 7 days) or every other week (every 14 days) schedule or stable settings for at least 8 weeks
Who cannot join
- Documented evidence of a null mutation in both LDLR alleles
- Use of a PCSK9 inhibitor within 10 weeks from screening visit
- Background medical lipid modifying therapy (LMT) that has not been stable for at least 4 weeks (6 weeks for fibrates, 24 weeks for mipomersen, 12 weeks for maximum tolerated dose of lomitapide) before the screening visit.
- LDL apheresis schedule/apheresis settings that have not been stable for at least 8 weeks before the screening visit or an apheresis schedule/settings that is not anticipated to be stable over the next 24 weeks.
- Use of nutraceuticals or over-the-counter (OTC) therapies known to affect lipids, at a dose/amount that has not been stable for at least 4 weeks prior to the screening visit or between the screening and randomization visits.
- Chronic use of systemic corticosteroids, unless on a stable regimen of 10 mg daily prednisone equivalent or less for at least 6 weeks prior to randomization. Note: topical, intra-articular, nasal, inhaled and ophthalmic steroid therapies are not considered as 'systemic' and are allowed
Show all 9Show fewer
- Systolic blood pressure >160 mmHg or diastolic blood pressure >100 mmHg at the screening visit (1 repeat measurement is allowed).
- LDL-C level <70 mg/dL (1.81 mmol/L) at the screening visit
- History of a myocardial infarction (MI), unstable angina leading to hospitalization, coronary artery bypass graft surgery, percutaneous coronary intervention , uncontrolled cardiac arrhythmia, carotid surgery or stenting, stroke, transient ischemic attack, valve replacement surgery, carotid revascularization, endovascular procedure or surgical intervention for peripheral vascular disease within 3 months prior to the screening visit.
Trial locations and contacts
28 sites in 13 countries.
28 sites in 13 countries
Ukraine5 sites
Regeneron Research Site
Ivano-Frankivsk, 76005
Regeneron Research Site
Kharkiv, 61039
Regeneron Research Site
Kharkiv, 61176
Regeneron Research Site
Kyiv, 02166
Regeneron Research Site
Kyiv, 03680
United States4 sites
Regeneron Research Site
Boca Raton, Florida, 33434
Regeneron Research Site
Cincinnati, Ohio, 45227
Regeneron Study Site
Dallas, Texas, 78226
Regeneron Research Site
New York, New York, 10029
Japan3 sites
Regeneron Research Site
Kanazawa, Ishikawa-ken, 920-8641
Regeneron Research Site
Nishinomiya, Hyōgo, 662-0918
Regeneron Research Site
Suita, Osaka, 565-8565
Canada2 sites
Regeneron Research Site
Chicoutimi, Quebec, G7H 7K9
Regeneron Research Site
Québec, Quebec, GIV 4W2
France2 sites
Regeneron Research Site
Marseille, 13285
Regeneron Research Site
Paris, 75651
Greece2 sites
Regeneron Research Site
Athens, 17674
Regeneron Research Site
Ioannina, 45500
Italy2 sites
Regeneron Research Site
Naples, 80131
Regeneron Research Site
Roma, 00161
South Africa2 sites
Regeneron Research Site
Cape Town, Western Cape, 7925
Regeneron Research Site
Parktown, Johannesburg, 2000
Turkey (Türkiye)2 sites
Regeneron Research Site
Beşevler, Ankara, 06500
Regeneron Research Site
Izmir, Bornova, 35040
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Alirocumab in Patients With Homozygous Familial Hypercholesterolemia
The primary objective of the study is to demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) with alirocumab subcutaneous (SC) every 2 weeks (Q2W) in comparison to placebo after 12 weeks of treatment.
The secondary objectives of the study are:
- To evaluate the effect of alirocumab Q2W on other lipid parameters (ie, apolipoprotein [Apo] A-1 and B, non-high-density lipoprotein cholesterol [non-HDL-C], total-cholesterol [TC], proportion of participants with 15%, 30%, and 50% LDL-C reductions, Lp(a), HDL-C, triglycerides [TG]) in participants with HoFH
- To evaluate the safety and tolerability of alirocumab SC Q2W in participants with HoFH
- To assess the pharmacokinetics of alirocumab SC Q2W in participants with HoFH
- To assess the potential development of anti-drug (alirocumab) antibodies
- Conditions
- Homozygous Familial Hypercholesterolemia
- Being tested
- Alirocumab, Placebo
- Medical subject headings
- Homozygous Familial Hypercholesterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AlirocumabDrug
Also called PRALUENT®, REGN727, SAR236553
Alirocumab SC Q2W
PlaceboDrug
Matching placebo SC Q2W
| Study group | Role | What they receive |
|---|---|---|
| Alirocumab SC Q2W | Experimental | Alirocumab SC every 2 weeks (Q2W) from baseline (day 1) through week 10 during the double-blind treatment period
Starting at week 12, and continuing through week 22, participants will receive open-label alirocumab SC Q2W Receives: Drug: Alirocumab |
| Placebo SC Q2W | Experimental | Matching placebo SC Q2W from baseline through week 10 during the double-blind treatment period
Starting at week 12, and continuing through week 22, participants will receive open-label alirocumab SC Q2W Receives: Drug: Alirocumab; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 12 (Intent-to-Treat [ITT] Estimand)
The percent change in LDL-C from baseline to week 12 is defined as: 100x (LDL-C value at week 12 - LDL-C value at baseline) / LDL-C value at baseline.
Measured: Baseline to Week 12
Secondary outcomes (22)
Percent Change in Apolipoprotein (Apo) B From Baseline to Week 12 (ITT Estimand)
ITT estimand; The percent change in Apo B from baseline to week 12 is defined as: 100x (Apo B value at week 12 - Apo B value at baseline) / Apo B value at baseline.
Measured: Baseline to Week 12
Percent Change in Non-high-density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 12
ITT estimand; The percent change in non-HDL-C from baseline to week 12 is defined as: 100x (non-HDL-C value at week 12 - non-HDL-C value at baseline) / non-HDL-C value at baseline.
Measured: Baseline to Week 12
Percent Change in Total Cholesterol (TC) From Baseline to Week 12
ITT estimand; The percent change in TC from baseline to week 12 is defined as: 100x (TC value at week 12 - TC value at baseline) / TC value at baseline.
Measured: Baseline to Week 12
Percentage of Participants With ≥15% Reduction in LDL-C at Week 12
ITT estimand
Measured: At Week 12
Percentage of Participants With ≥30% Reduction in LDL-C at Week 12
ITT estimand
Measured: At Week 12
Percent Change in Lipoprotein(a) [Lp(a)] From Baseline to Week 12
ITT estimand; The percent change in Lp(a) from baseline to week 12 is defined as: 100x (Lp(a) value at week 12 - Lp(a) value at baseline) / Lp(a) value at baseline.
Measured: Baseline to Week 12
Percentage of Participants With ≥50% Reduction in LDL-C at Week 12
ITT estimand
Measured: At Week 12
Percent Change in HDL-C From Baseline to Week 12 - ITT Analysis
ITT estimand; The percent change in HDL-C from baseline to week 12 is defined as: 100x (HDL-C value at week 12 - HDL-C value at baseline) / HDL-C value at baseline.
Measured: Baseline to Week 12
Percent Change in Fasting Triglycerides (TG) From Baseline to Week 12
ITT estimand; The percent change in TG from baseline to week 12 is defined as: 100x (TG value at week 12 - TG value at baseline) / TG value at baseline.
Measured: Baseline to Week 12
Percent Change in Apo A-1 From Baseline to Week 12 -- ITT Analysis
ITT estimand; The percent change in Apo A-1 from baseline to week 12 is defined as: 100x (Apo A-1 value at week 12 - Apo A-1 value at baseline) / Apo A-1 value at baseline.
Measured: Baseline to Week 12
Percent Change in LDL-C From Baseline to Week 12 (On-treatment Estimand)
Percent change for LDL-C from baseline to Week 12 during the efficacy treatment period, which is defined as the time from the first double-blind investigational study drug injection up to 21 days after the last double-blind investigational study drug injection, or the first dose of the open-label investigational study drug, whichever is earlier.
Measured: Baseline to Week 12
Percent Change in Apo B From Baseline to Week 12 (On-treatment Estimand)
Percent change for Apo B from baseline to Week 12 during the efficacy treatment period, which is defined as the time from the first double-blind investigational study drug injection up to 21 days after the last double-blind investigational study drug injection, or the first dose of the open-label nvestigational study drug, whichever is earlier.
Measured: Baseline to Week 12
Percent Change in Non-HDL-C From Baseline to Week 12 (On-treatment Estimand)
Percent change for non-HDL-C from baseline to Week 12 during the efficacy treatment period, which is defined as the time from the first double-blind investigational study drug injection up to 21 days after the last double-blind investigational study drug injection, or the first dose of the open-label investigational study drug, whichever is earlier.
Measured: Baseline to Week 12
Percent Change in TC From Baseline to Week 12 (On-treatment Estimand)
Percent change for TC from baseline to Week 12 during the efficacy treatment period, which is defined as the time from the first double-blind investigational study drug injection up to 21 days after the last double-blind investigational study drug injection, or the first dose of the open-label investigational study drug, whichever is earlier.
Measured: Baseline to Week 12
Percent Change in Lp(a) From Baseline to Week 12 (On-treatment Estimand)
Percent change for LP(a) from baseline to Week 12 during the efficacy treatment period, which is defined as the time from the first double-blind investigational study drug injection up to 21 days after the last double-blind investigational study drug injection, or the first dose of the open-label investigational study drug, whichever is earlier.
Measured: Baseline to Week 12
Percent Change in HDL-C From Baseline to Week 12 (On-treatment Estimand)
Percent change for HDL-C from baseline to Week 12 during the efficacy treatment period, which is defined as the time from the first double-blind investigational study drug injection up to 21 days after the last double-blind investigational study drug injection, or the first dose of the open-label investigational study drug, whichever is earlier.
Measured: Baseline to Week 12
Percent Change in Fasting TG From Baseline to Week 12 (On-treatment Estimand)
Percent change for fasting TG from baseline to Week 12 during the efficacy treatment period, which is defined as the time from the first double-blind investigational study drug injection up to 21 days after the last double-blind investigational study drug injection, or the first dose of the open-label investigational study drug, whichever is earlier.
Measured: Baseline to Week 12
Percent Change in Apo A-1 From Baseline to Week 12 (On-treatment Estimand)
Percent change for Apo A-1 from baseline to Week 12 during the efficacy treatment period, which is defined as the time from the first double-blind investigational study drug injection up to 21 days after the last double-blind investigational study drug injection, or the first dose of the open-label investigational study drug, whichever is earlier.
Measured: Baseline to Week 12
Percentage of Participants With ≥15% Reduction, ≥30% Reduction, and ≥50% Reduction in LDL-C at Week 12 (On-treatment Estimand)
Measured: At Week 12
Absolute Change in the Ratio of Apo B/Apo A-1 From Baseline to Week 12 (ITT Estimand)
Ratio of Apo B/Apo A1 at week 12 minus ratio of Apo B/Apo A1 at baseline
Measured: Baseline to Week 12
Number of Participants With Anti-Drug Antibodies (ADA) to REGN727 Over Time
Measured: 26 weeks
Number of Participants With Adverse Events (AEs)
All AEs will be recorded from time of informed consent to end of study. Only treatment-emergent adverse events (TEAE) will be reported. Double-blind TEAE observation period is defined as time from first dose of double-blind study drug to last dose of double-blind study drug +70 days, or up to day before first dose of open-label study drug administration, whichever is earlier. Open-label TEAE observation period is defined as time from first open-label study treatment administration to last open-label study treatment administration +70 days.
Measured: Baseline to week 32 (End of Study)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 May 2017
- Study start3 Oct 2017
- Primary completion27 Sep 2019
- Study completion13 Feb 2020
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 Homozygous Familial HypercholesterolemiaStudies of the same condition, open and under way.
- More trials run by Regeneron PharmaceuticalsThe sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 69 participants (actual)
Sponsor and investigators
- Lead sponsor
- Regeneron Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Sanofi
- Responsible party
- Sponsor
- Investigators
- Clinical Trial Management, Study director, Regeneron Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03156621
- Sponsor's protocol ID
- R727-CL-1628
- Other IDs
- 2017-000351-95, 2017
- Acronym
- ODYSSEY HoFH
- Status last verified
- Jun 2021
- First submitted
- 15 May 2017
- Last updated
- 29 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Blom DJ, Harada-Shiba M, Rubba P, Gaudet D, Kastelein JJP, Charng MJ, Pordy R, Donahue S, Ali S, Dong Y, Khilla N, Banerjee P, Baccara-Dinet M, Rosenson RS. Efficacy and Safety of Alirocumab in Adults With Homozygous Familial Hypercholesterolemia: The ODYSSEY HoFH Trial. J Am Coll Cardiol. 2020 Jul 14;76(2):131-142. doi: 10.1016/j.jacc.2020.05.027.
PubMed 32646561
Study at a glance
From the registry record, last updated by the sponsor on 29 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Alirocumab in Patients With Homozygous Familial Hypercholesterolemia
- ClinicalTrials.gov ID
- NCT03156621
- Acronym / sponsor ID
- ODYSSEY HoFH / R727-CL-1628
- Conditions
- Homozygous Familial Hypercholesterolemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 69 participants (actual)
- Lead sponsor
- Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- Sanofi
- Study start
- 3 Oct 2017 (actual)
- Primary completion
- 27 Sep 2019 (actual)
- Study completion
- 13 Feb 2020 (actual)
- First posted
- 17 May 2017 (actual)
- Results
- Posted 29 Jun 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of the study is to demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) with alirocumab subcutaneous (SC) every 2 weeks (Q2W) in comparison to placebo after 12 weeks of treatment.
The secondary objectives of the study are:
- To evaluate the effect of alirocumab Q2W on other lipid parameters (ie, apolipoprotein [Apo] A-1 and B, non-high-density lipoprotein cholesterol [non-HDL-C], total-cholesterol [TC], proportion of participants with 15%, 30%, and 50% LDL-C reductions, Lp(a), HDL-C, triglycerides [TG]) in participants with HoFH
- To evaluate the safety and tolerability of alirocumab SC Q2W in participants with HoFH
- To assess the pharmacokinetics of alirocumab SC Q2W in participants with HoFH
- To assess the potential development of anti-drug (alirocumab) antibodies
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Alirocumab SC Q2W | Experimental | Drug: AlirocumabAlirocumab SC every 2 weeks (Q2W) from baseline (day 1) through week 10 during the double-blind treatment period Starting at week 12, and continuing through week 22, participants will receive open-label alirocumab SC Q2W |
| Placebo SC Q2W | Experimental | Drug: Alirocumab, Drug: PlaceboMatching placebo SC Q2W from baseline through week 10 during the double-blind treatment period Starting at week 12, and continuing through week 22, participants will receive open-label alirocumab SC Q2W |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Diagnosis of HoFH by at least 1 of the following genotype or clinical criteria (all patients on LDL apheresis must be diagnosed based on genotype):
- Receiving a stable dose of a statin at the screening visit (documentation if statin ineffective or patient unable to tolerate statin)
- If undergoing LDL apheresis, must have initiated LDL apheresis at least 3 months prior to screening and must have been on a stable weekly (every 7 days) or every other week (every 14 days) schedule or stable settings for at least 8 weeks
Key exclusion criteria (9 listed)
- Documented evidence of a null mutation in both LDLR alleles
- Use of a PCSK9 inhibitor within 10 weeks from screening visit
- Background medical lipid modifying therapy (LMT) that has not been stable for at least 4 weeks (6 weeks for fibrates, 24 weeks for mipomersen, 12 weeks for maximum tolerated dose of lomitapide) before the screening visit.
- LDL apheresis schedule/apheresis settings that have not been stable for at least 8 weeks before the screening visit or an apheresis schedule/settings that is not anticipated to be stable over the next 24 weeks.
- Use of nutraceuticals or over-the-counter (OTC) therapies known to affect lipids, at a dose/amount that has not been stable for at least 4 weeks prior to the screening visit or between the screening and randomization visits.
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 |
|---|---|---|
| AlirocumabAlso: PRALUENT®, REGN727, SAR236553Alirocumab SC Q2W | Drug | Alirocumab SC Q2W, Placebo SC Q2W |
| PlaceboMatching placebo SC Q2W | Drug | Placebo SC Q2W |
Primary outcome
Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 12 (Intent-to-Treat [ITT] Estimand)
Time frame: Baseline to Week 12
The percent change in LDL-C from baseline to week 12 is defined as: 100x (LDL-C value at week 12 - LDL-C value at baseline) / LDL-C value at baseline.
22 secondary outcomes
Percent Change in Apolipoprotein (Apo) B From Baseline to Week 12 (ITT Estimand)
Time frame: Baseline to Week 12
Percent Change in Non-high-density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 12
Time frame: Baseline to Week 12
Percent Change in Total Cholesterol (TC) From Baseline to Week 12
Time frame: Baseline to Week 12
Percentage of Participants With ≥15% Reduction in LDL-C at Week 12
Time frame: At Week 12
Percentage of Participants With ≥30% Reduction in LDL-C at Week 12
Time frame: At Week 12
Percent Change in Lipoprotein(a) [Lp(a)] From Baseline to Week 12
Time frame: Baseline to Week 12
Percentage of Participants With ≥50% Reduction in LDL-C at Week 12
Time frame: At Week 12
Percent Change in HDL-C From Baseline to Week 12 - ITT Analysis
Time frame: Baseline to Week 12
Percent Change in Fasting Triglycerides (TG) From Baseline to Week 12
Time frame: Baseline to Week 12
Percent Change in Apo A-1 From Baseline to Week 12 -- ITT Analysis
Time frame: Baseline to Week 12
Percent Change in LDL-C From Baseline to Week 12 (On-treatment Estimand)
Time frame: Baseline to Week 12
Percent Change in Apo B From Baseline to Week 12 (On-treatment Estimand)
Time frame: Baseline to Week 12
Percent Change in Non-HDL-C From Baseline to Week 12 (On-treatment Estimand)
Time frame: Baseline to Week 12
Percent Change in TC From Baseline to Week 12 (On-treatment Estimand)
Time frame: Baseline to Week 12
Percent Change in Lp(a) From Baseline to Week 12 (On-treatment Estimand)
Time frame: Baseline to Week 12
Percent Change in HDL-C From Baseline to Week 12 (On-treatment Estimand)
Time frame: Baseline to Week 12
Percent Change in Fasting TG From Baseline to Week 12 (On-treatment Estimand)
Time frame: Baseline to Week 12
Percent Change in Apo A-1 From Baseline to Week 12 (On-treatment Estimand)
Time frame: Baseline to Week 12
Percentage of Participants With ≥15% Reduction, ≥30% Reduction, and ≥50% Reduction in LDL-C at Week 12 (On-treatment Estimand)
Time frame: At Week 12
Absolute Change in the Ratio of Apo B/Apo A-1 From Baseline to Week 12 (ITT Estimand)
Time frame: Baseline to Week 12
Number of Participants With Anti-Drug Antibodies (ADA) to REGN727 Over Time
Time frame: 26 weeks
Number of Participants With Adverse Events (AEs)
Time frame: Baseline to week 32 (End of Study)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 32646561Blom DJ, Harada-Shiba M, Rubba P, Gaudet D, Kastelein JJP, Charng MJ, Pordy R, Donahue S, Ali S, Dong Y, Khilla N, Banerjee P, Baccara-Dinet M, Rosenson RS. Efficacy and Safety of Alirocumab in Adults With Homozygous Familial Hypercholesterolemia: The ODYSSEY HoFH Trial. J Am Coll Cardiol. 2020 Jul 14;76(2):131-142. doi: 10.1016/j.jacc.2020.05.027.
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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.
Clinical Trial Management study director
Regeneron Pharmaceuticals
28 sites in 13 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Alirocumab in people with Homozygous Familial Hypercholesterolemia, run by Regeneron Pharmaceuticals at 28 sites in 13 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 7 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 29 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in Ukraine, United States, Japan, Canada, France and 8 more countries. 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. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 29 Jun 2021). 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 in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 12 (Intent-to-Treat [ITT] Estimand) (time frame: Baseline to Week 12). 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 NCT03156621, 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 29 Jun 2021.