Heterozygous Familial HypercholesterolemiaPhase 3ODYSSEY ESCAPENCT02326220
Study of Alirocumab (REGN727/SAR236553) in Patients With Heterozygous Familial Hypercholesterolemia (HeFH) Undergoing Low-density Lipoprotein (LDL) Apheresis Therapy
A Phase 3 study testing Alirocumab in people with Heterozygous Familial Hypercholesterolemia, run by Regeneron Pharmaceuticals at 13 sites in 2 countries.
Trial summary
The primary objective of the study is to evaluate the effect of alirocumab 150 mg every 2 weeks (Q2W) in comparison with placebo on the frequency of low-density lipoprotein (LDL) apheresis treatments in participants with heterozygous familial hypercholesterolemia (HeFH) undergoing weekly or bi-weekly LDL apheresis… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Heterozygous Familial Hypercholesterolemia
- What is being tested?
- Alirocumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 62 participants
- Where
- 13 sitesin 2 countries
- Study dates
- 31 Mar 2015to 30 Apr 2016
- 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
- Men and women ≥18 years of age at the time of the screening visit
- Diagnosis of HeFH (Heterozygous familial hypercholesterolemia)
- Currently undergoing LDL (low-density lipoprotein) apheresis therapy QW (weekly) or Q2W (every 2 weeks) or at least 8 weeks prior to the screening visit
Who cannot join
- Homozygous FH (familial hypercholesterolemia)
- Background medical LMT (lipid-modifying therapy) (if applicable) that has not been stable for at least 8 weeks prior to the screening visit
- LDL apheresis schedule/ apheresis settings that have not been stable for at least 8 weeks prior to the screening visit
- An LDL apheresis schedule other than QW to Q2W
- Initiation of a new exercise program or exercise that has not remained stable within 8 weeks prior to the screening visit (week -2)
- Initiation of a new diet or a diet that has not been stable within 8 weeks prior to the screening visit (week -2)
Show all 12Show fewer
- Use of nutraceuticals or over-the-counter therapies known to affect lipids, at a dose/amount that has not been stable for at least 8 weeks prior to the screening visit (week -2), or between the screening and randomization visit
- Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins
- Known history of a positive test for human immunodeficiency virus
- Use of any active investigational drugs within 1 month or 5 half-lives of screening, whichever is longer
- Patients who have been treated with at least 1 dose of alirocumab or any other anti-PCSK9 monoclonal antibody in any other clinical studies
- Pregnant or breastfeeding women
Trial locations and contacts
13 sites in 2 countries.
13 sites in 2 countries
United States7 sites
Study site
Aurora, Colorado
Study site
Hartford, Connecticut
Study site
Kansas City, Kansas
Study site
Philadelphia, Pennsylvania
Study site
Portland, Oregon
Study site
Rochester, Minnesota
Study site
Scarborough, Maine
Germany6 sites
Study site
Berlin
Study site
Dresden, Saxony
Study site
Göttingen
Study site
Muenchen (2 Locations)
Study site
Passau
Study site
Rostock
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 Heterozygous Familial Hypercholesterolemia Undergoing Lipid Apheresis Therapy
The primary objective of the study is to evaluate the effect of alirocumab 150 mg every 2 weeks (Q2W) in comparison with placebo on the frequency of low-density lipoprotein (LDL) apheresis treatments in participants with heterozygous familial hypercholesterolemia (HeFH) undergoing weekly or bi-weekly LDL apheresis therapy.
Read the detailed description
LDL apheresis removes low-density lipoproteins (LDL) that transport cholesterol in the plasma portion of the blood. This treatment is mainly used for familial hypercholesterolemia, but can be used in other rare diseases. Familial hypercholesterolemia (HeFH) is an inherited genetic condition that causes accumulation of cholesterol in the blood, which can lead to atherosclerosis and heart disease. This treatment is recommended for patients who do not respond to dietary and/or medication control of LDL cholesterol.
- Being tested
- Placebo, Alirocumab
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
AlirocumabDrug
Also called REGN727, SAR236553, Praluent®
| Study group | Role | What they receive |
|---|---|---|
| Placebo Q2W (Double Blind Period) | Experimental | Placebo (for alirocumab) subcutaneous (SC) injection Q2W up to Week 16. Receives: Drug: Placebo |
| Alirocumab 150 mg Q2W (Double Blind Period) | Experimental | Alirocumab 150 mg SC injection Q2W up to Week 16. Receives: Drug: Alirocumab |
| Alirocumab 150 Q2W (Open Label Treatment Period) | Experimental | Alirocumab 150 mg SC injection Q2W starting from Week 18 up to Week 76. Receives: Drug: Alirocumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Standardized Rate of Apheresis Treatments From Week 7 to Week 18
Rate of apheresis treatments were normalized by the number of planned apheresis treatments according to each participant's established schedule at screening, week -10 to week -2. The normalized rate of apheresis was defined for each participant as the number of actual apheresis treatments received from week 7 to week 18 divided by the number of planned apheresis treatments per randomization strata at baseline (6 for Q2W and 12 for QW).
Measured: Week 7 to Week 18 (before start of open-label treatment)
Secondary outcomes (22)
Percent Change From Baseline in Calculated LDL-C (Pre-apheresis) at Week 6
Adjusted Least-squares (LS) means and standard errors at Week 6 were obtained from a mixed-effect model with repeated measures (MMRM) to account for missing data. All available post-baseline data from Week 2 to Week 18 regardless of status on or off-treatment were used in the model (ITT analysis).
Measured: Baseline and at Week 6
Change in Standardized Rate of Apheresis Treatments From Week 15 to Week 18
Rate of apheresis treatments were normalized by the rate by the number of actual apheresis treatments according to received from week 15 to week 18 divided by the planned apheresis treatments per randomization strata at baseline (2 for Q2W and 4 for QW). Only legitimate apheresis treatment skipping per point-of-care LDL-C value is counted as "apheresis not occurred". Missing apheresis treatment information (any reason) from week 7 to week 18 is assigned an outcome of the apheresis treatment occurred at the visit (i.e. impute 1 apheresis treatment for that visit).
Measured: Week 15 up to Week 18 (before the start of open-label treatment dose)
Percent Change From Baseline in Apolipoprotein B (Apo B) (Pre-apheresis) to Week 6
Adjusted LS means and standard errors at Week 6 were obtained from MMRM to account for missing data. All available post-baseline data from Week 2 (pre-apheresis) to Week 18 (pre-apheresis) regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 6
Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) (Pre-apheresis) to Week 6
Adjusted LS means and standard errors at Week 6 were obtained from MMRM to account for missing data. All available post-baseline data from Week 2 (pre-apheresis) to Week 18 (pre-apheresis) regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 6
Percent Change From Baseline in Total Cholesterol (Pre-apheresis) to Week 6
Adjusted LS means and standard errors at Week 6 were obtained from MMRM to account for missing data. All available post-baseline data from Week 2 (pre-apheresis) to Week 18 (pre-apheresis) regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 6
Percent Change From Baseline in Apolipoprotein A (Apo A1) (Pre-apheresis) to Week 6
Adjusted LS means and standard errors at Week 6 were obtained from MMRM to account for missing data. All available post-baseline data from Week 2 (pre-apheresis) to Week 18 (pre-apheresis) regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 6
Percentage of Participants With At Least (>=) 30% Reduction in Calculated LDL-C (Pre-apheresis) at Week 6
Percentage of participants at Week 6 was obtained from a last observation carried forward (LOCF) model for handling of missing data. All available post-baseline data regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 6
Percentage of Participants With At Least (>=) 50% Reduction in Calculated LDL-C (Pre-apheresis) at Week 6
Percentage of participants at Week 6 was obtained from LOCF model for handling of missing data. All available post-baseline data regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 6
Percent Change From Baseline in Calculated LDL-C (Pre-Apheresis) to Week 18
Adjusted LS means and standard errors at Week 18 were obtained from MMRM to account for missing data. All available post-baseline data from Week 2 (pre-apheresis) to Week 18 (pre-apheresis) regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 18
Percent Change From Baseline in Apolipoprotein B (Apo B) (Pre-apheresis) to Week 18
Adjusted LS means and standard errors at Week 18 were obtained from MMRM to account for missing data. All available post-baseline data from Week 2 (pre-apheresis) to Week 18 (pre-apheresis) regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 18
Percent Change From Baseline in Non-HDL-C (Pre-apheresis) to Week 18
Adjusted LS means and standard errors at Week 18 were obtained from MMRM to account for missing data. All available post-baseline data from Week 2 (pre-apheresis) to Week 18 (pre-apheresis) regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 18
Percent Change From Baseline in Total Cholesterol (Pre-apheresis) to Week 18
Adjusted LS means and standard errors at Week 18 were obtained from MMRM to account for missing data. All available post-baseline data from Week 2 (pre-apheresis) to Week 18 (pre-apheresis) regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 18
Percent Change From Baseline in Apo A1 (Pre-apheresis) to Week 18
Adjusted LS means and standard errors at Week 18 were obtained from MMRM to account for missing data. All available post-baseline data from Week 2 (pre-apheresis) to Week 18 (pre-apheresis) regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 18
Percentage of Participants With At Least (>=) 30% Reduction in Calculated LDL-C (Pre-apheresis) at Week 18
Percentage of participants at Week 18 was obtained from LOCF model for handling of missing data. All available post-baseline data regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 18
Percentage of Participants With At Least (>=) 50% Reduction in Calculated LDL-C (Pre-apheresis) at Week 18
Percentage of participants at Week 18 was obtained from LOCF model for handling of missing data. All available post-baseline data regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 18
Change From Baseline in W-BQ22 (Well-being Questionnaire) Index Score at Week 18
The W-BQ22 (well-being) questionnaire was a standardized and generic instrument used for measuring the impact of hypercholesterolemia and treatment on well-being of participants. The general well-being score was calculated as the sum of 22 questions in the W-BQ22 questionnaire (each question scored from 0 to 3 [0 = not at all and 3 = all the time]). Total score for 22 questions range from 0 to 66 [0 = worst condition and 66 = best well-being condition).
Measured: From Baseline to Week 18
Percent Change From Baseline in Lipoprotein (a) (Lp [a]) (Pre-apheresis) to Week 6
Adjusted means and standard errors at Week 6 from a multiple imputation approach followed by robust regression model including all available post-baseline data from Week 2 (pre-apheresis) to Week 18 (pre-apheresis) regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 6
Percent Change From Baseline in High-Density Lipoprotein Cholesterol (HDL-C) (Pre-apheresis) to Week 6
Adjusted LS means and standard errors at Week 6 were obtained from MMRM to account for missing data. All available post-baseline data from Week 2 (pre-apheresis) to Week 18 (pre-apheresis) regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 6
Percent Change From Baseline in Triglyceride (TG) Levels (Pre-apheresis) to Week 6
Adjusted means and standard errors at Week 6 from a multiple imputation approach followed by robust regression model including all available post-baseline data from Week 2 (pre-apheresis) to Week 18 (pre-apheresis) regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 6
Percent Change From Baseline in Lp (a) (Pre-apheresis) to Week 18
Adjusted means and standard errors at Week 18 from a multiple imputation approach followed by robust regression model including all available post-baseline data from Week 2 (pre-apheresis) to Week 18 (pre-apheresis) regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 18
Percent Change From Baseline in HDL-C (Pre-apheresis) to Week 18
Adjusted LS means and standard errors at Week 18 were obtained from MMRM to account for missing data. All available post-baseline data from Week 2 (pre-apheresis) to Week 18 (pre-apheresis) regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 18
Percent Change From Baseline in TG Levels (Pre-apheresis) to Week 18
Adjusted means and standard errors at Week 18 from a multiple imputation approach followed by robust regression model including all available post-baseline data from Week 2 (pre-apheresis) to Week 18 (pre-apheresis) regardless of status on- or off-treatment were used in the model (ITT analysis).
Measured: From Baseline to Week 18
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Dec 2014 (estimated)
- Study start31 Mar 2015
- Primary completion31 Jan 2016
- Study completion30 Apr 2016
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 (2)Papers the sponsor has linked to this study.
- Other trials for Heterozygous 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 62 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
- NCT02326220
- Sponsor's protocol ID
- R727-CL-1216
- Acronym
- ODYSSEY ESCAPE
- Status last verified
- Apr 2020
- First submitted
- 22 Dec 2014
- Last updated
- 1 May 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Moriarty PM, Parhofer KG, Babirak SP, Cornier MA, Duell PB, Hohenstein B, Leebmann J, Ramlow W, Schettler V, Simha V, Steinhagen-Thiessen E, Thompson PD, Vogt A, von Stritzky B, Du Y, Manvelian G. Alirocumab in patients with heterozygous familial hypercholesterolaemia undergoing lipoprotein apheresis: the ODYSSEY ESCAPE trial. Eur Heart J. 2016 Dec 21;37(48):3588-3595. doi: 10.1093/eurheartj/ehw388. Epub 2016 Aug 29.
PubMed 27572070Moriarty PM, Parhofer KG, Babirak SP, deGoma E, Duell PB, Hohenstein B, Ramlow W, Simha V, Steinhagen-Thiessen E, Thompson PD, Vogt A, von Stritzky B, Du Y, Manvelian G. Alirocumab in patients with heterozygous familial hypercholesterolemia undergoing lipoprotein apheresis: Rationale and design of the ODYSSEY ESCAPE trial. J Clin Lipidol. 2016 May-Jun;10(3):627-34. doi: 10.1016/j.jacl.2016.02.003. Epub 2016 Feb 18.
PubMed 27206951
Study at a glance
From the registry record, last updated by the sponsor on 1 May 2020. 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 Heterozygous Familial Hypercholesterolemia Undergoing Lipid Apheresis Therapy
- ClinicalTrials.gov ID
- NCT02326220
- Acronym / sponsor ID
- ODYSSEY ESCAPE / R727-CL-1216
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 62 participants (actual)
- Lead sponsor
- Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- Sanofi
- Study start
- 31 Mar 2015 (actual)
- Primary completion
- 31 Jan 2016 (actual)
- Study completion
- 30 Apr 2016 (actual)
- First posted
- 29 Dec 2014 (estimated)
- Results
- Posted 1 May 2020
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 evaluate the effect of alirocumab 150 mg every 2 weeks (Q2W) in comparison with placebo on the frequency of low-density lipoprotein (LDL) apheresis treatments in participants with heterozygous familial hypercholesterolemia (HeFH) undergoing weekly or bi-weekly LDL apheresis therapy.
Detailed description
LDL apheresis removes low-density lipoproteins (LDL) that transport cholesterol in the plasma portion of the blood. This treatment is mainly used for familial hypercholesterolemia, but can be used in other rare diseases. Familial hypercholesterolemia (HeFH) is an inherited genetic condition that causes accumulation of cholesterol in the blood, which can lead to atherosclerosis and heart disease. This treatment is recommended for patients who do not respond to dietary and/or medication control of LDL cholesterol.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo Q2W (Double Blind Period) | Experimental | Drug: PlaceboPlacebo (for alirocumab) subcutaneous (SC) injection Q2W up to Week 16. |
| Alirocumab 150 mg Q2W (Double Blind Period) | Experimental | Drug: AlirocumabAlirocumab 150 mg SC injection Q2W up to Week 16. |
| Alirocumab 150 Q2W (Open Label Treatment Period) | Experimental | Drug: AlirocumabAlirocumab 150 mg SC injection Q2W starting from Week 18 up to Week 76. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Men and women ≥18 years of age at the time of the screening visit
- Diagnosis of HeFH (Heterozygous familial hypercholesterolemia)
- Currently undergoing LDL (low-density lipoprotein) apheresis therapy QW (weekly) or Q2W (every 2 weeks) or at least 8 weeks prior to the screening visit
Key exclusion criteria (12 listed)
- Homozygous FH (familial hypercholesterolemia)
- Background medical LMT (lipid-modifying therapy) (if applicable) that has not been stable for at least 8 weeks prior to the screening visit
- LDL apheresis schedule/ apheresis settings that have not been stable for at least 8 weeks prior to the screening visit
- An LDL apheresis schedule other than QW to Q2W
- Initiation of a new exercise program or exercise that has not remained stable within 8 weeks prior to the screening visit (week -2)
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 |
|---|---|---|
| Placebo | Drug | Placebo Q2W (Double Blind Period) |
| AlirocumabAlso: REGN727, SAR236553, Praluent® | Drug | Alirocumab 150 Q2W (Open Label Treatment Period), Alirocumab 150 mg Q2W (Double Blind Period) |
Primary outcome
Change in Standardized Rate of Apheresis Treatments From Week 7 to Week 18
Time frame: Week 7 to Week 18 (before start of open-label treatment)
Rate of apheresis treatments were normalized by the number of planned apheresis treatments according to each participant's established schedule at screening, week -10 to week -2. The normalized rate of apheresis was defined for each participant as the number of actual apheresis treatments received from week 7 to week 18 divided by the number of planned apheresis treatments per randomization strata at baseline (6 for Q2W and 12 for QW).
22 secondary outcomes
Percent Change From Baseline in Calculated LDL-C (Pre-apheresis) at Week 6
Time frame: Baseline and at Week 6
Change in Standardized Rate of Apheresis Treatments From Week 15 to Week 18
Time frame: Week 15 up to Week 18 (before the start of open-label treatment dose)
Percent Change From Baseline in Apolipoprotein B (Apo B) (Pre-apheresis) to Week 6
Time frame: From Baseline to Week 6
Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) (Pre-apheresis) to Week 6
Time frame: From Baseline to Week 6
Percent Change From Baseline in Total Cholesterol (Pre-apheresis) to Week 6
Time frame: From Baseline to Week 6
Percent Change From Baseline in Apolipoprotein A (Apo A1) (Pre-apheresis) to Week 6
Time frame: From Baseline to Week 6
Percentage of Participants With At Least (>=) 30% Reduction in Calculated LDL-C (Pre-apheresis) at Week 6
Time frame: From Baseline to Week 6
Percentage of Participants With At Least (>=) 50% Reduction in Calculated LDL-C (Pre-apheresis) at Week 6
Time frame: From Baseline to Week 6
Percent Change From Baseline in Calculated LDL-C (Pre-Apheresis) to Week 18
Time frame: From Baseline to Week 18
Percent Change From Baseline in Apolipoprotein B (Apo B) (Pre-apheresis) to Week 18
Time frame: From Baseline to Week 18
Percent Change From Baseline in Non-HDL-C (Pre-apheresis) to Week 18
Time frame: From Baseline to Week 18
Percent Change From Baseline in Total Cholesterol (Pre-apheresis) to Week 18
Time frame: From Baseline to Week 18
Percent Change From Baseline in Apo A1 (Pre-apheresis) to Week 18
Time frame: From Baseline to Week 18
Percentage of Participants With At Least (>=) 30% Reduction in Calculated LDL-C (Pre-apheresis) at Week 18
Time frame: From Baseline to Week 18
Percentage of Participants With At Least (>=) 50% Reduction in Calculated LDL-C (Pre-apheresis) at Week 18
Time frame: From Baseline to Week 18
Change From Baseline in W-BQ22 (Well-being Questionnaire) Index Score at Week 18
Time frame: From Baseline to Week 18
Percent Change From Baseline in Lipoprotein (a) (Lp [a]) (Pre-apheresis) to Week 6
Time frame: From Baseline to Week 6
Percent Change From Baseline in High-Density Lipoprotein Cholesterol (HDL-C) (Pre-apheresis) to Week 6
Time frame: From Baseline to Week 6
Percent Change From Baseline in Triglyceride (TG) Levels (Pre-apheresis) to Week 6
Time frame: From Baseline to Week 6
Percent Change From Baseline in Lp (a) (Pre-apheresis) to Week 18
Time frame: From Baseline to Week 18
Percent Change From Baseline in HDL-C (Pre-apheresis) to Week 18
Time frame: From Baseline to Week 18
Percent Change From Baseline in TG Levels (Pre-apheresis) to Week 18
Time frame: From Baseline to Week 18
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 May 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 27572070Moriarty PM, Parhofer KG, Babirak SP, Cornier MA, Duell PB, Hohenstein B, Leebmann J, Ramlow W, Schettler V, Simha V, Steinhagen-Thiessen E, Thompson PD, Vogt A, von Stritzky B, Du Y, Manvelian G. Alirocumab in patients with heterozygous familial hypercholesterolaemia undergoing lipoprotein apheresis: the ODYSSEY ESCAPE trial. Eur Heart J. 2016 Dec 21;37(48):3588-3595. doi: 10.1093/eurheartj/ehw388. Epub 2016 Aug 29.
- Derived from this study: PubMed 27206951Moriarty PM, Parhofer KG, Babirak SP, deGoma E, Duell PB, Hohenstein B, Ramlow W, Simha V, Steinhagen-Thiessen E, Thompson PD, Vogt A, von Stritzky B, Du Y, Manvelian G. Alirocumab in patients with heterozygous familial hypercholesterolemia undergoing lipoprotein apheresis: Rationale and design of the ODYSSEY ESCAPE trial. J Clin Lipidol. 2016 May-Jun;10(3):627-34. doi: 10.1016/j.jacl.2016.02.003. Epub 2016 Feb 18.
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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
13 sites in 2 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 Heterozygous Familial Hypercholesterolemia, run by Regeneron Pharmaceuticals at 13 sites in 2 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 3 inclusion and 12 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 1 May 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States, Germany. 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 1 May 2020). 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?
Change in Standardized Rate of Apheresis Treatments From Week 7 to Week 18 (time frame: Week 7 to Week 18 (before start of open-label treatment)). 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 NCT02326220, which also lists 2 linked publications.
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 1 May 2020.