A Phase 4 interventional study of evolocumab in Homozygous Familial Hypercholesterolemia HoFH, sponsored by Amgen. Completed at 12 sites in India. Open to participants aged 12 Years to 80 Years. Per ClinicalTrials.gov, last updated 2024-05-29.
Sponsored by Amgen · Phase 4, Interventional, and Treatment
To describe the safety and tolerability of evolocumab in participants with homozygous familial hypercholesterolemia (HoFH) in India. All participants will receive evolocumab over an 8-week period.
An open-label, multicentre, phase 4 study to describe the safety and tolerability of evolocumab in 30 Indian participants with HoFH. Subjects who meet the inclusion/exclusion criteria and laboratory assessments at screening will be enrolled and will be required to maintain their current lipid-lowering drug therapy throughout the duration of the trial. Participants will receive evolocumab 420 mg subcutaneous (SC) once monthly (QM) and study visits will occur approximately every 4 weeks. Apheresis participants will receive evolocumab 420 mg SC every 2 weeks to correspond with their apheresis schedule. Final administration of evolocumab (for all participants) will occur at week 8. The end of study (EOS) visit will occur at week 12 for all participants.
Exclusion Criteria:
Evolocumab 420 mg subcutaneous (SC) once monthly (QM) or every 2 weeks (Q2W; for participants on apheresis).
Drug: evolocumab
Administered by SC injection via autoinjector (AI)/pen
Also known as: Repatha®, EvoMab
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Includes both serious and non-serious adverse events (AEs). AE: any untoward medical occurrence in a participant. SAE: an AE that meets 1 on the following serious criteria: fatal; life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity; congenital anomaly/birth defect; other medically important serious event. TEAE: any AE starting on or after the first dose of study drug and up to and including 30 days after the end of study drug or the end of study date, whichever is earlier.
Time frame: 12 weeks
Percent Change From Baseline to Week 12 in Low-Density Lipoprotein Cholesterol (LDL-C)
Time frame: baseline, week 12
Percent Change From Baseline to Week 12 in Apolipoprotein B (ApoB)
Time frame: baseline, week 12
Percent Change From Baseline to Week 12 in Lipoprotein(a) (Lp[a])
Time frame: baseline, week 12
Participants were enrolled at 10 study centers in India. The first participant was enrolled on 04 August 2018 and the last participant was enrolled on 29 August 2019.
| Milestone | Evolocumab |
|---|---|
| Started | 30 |
| Completed | 29 |
| Not completed | 1 |
| Withdrew: Lost to follow-up | 1 |
Includes both serious and non-serious adverse events (AEs). AE: any untoward medical occurrence in a participant. SAE: an AE that meets 1 on the following serious criteria: fatal; life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity; congenital anomaly/birth defect; other medically important serious event. TEAE: any AE starting on or after the first dose of study drug and up to and including 30 days after the end of study drug or the end of study date, whichever is earlier.
| Participants | Evolocumab |
|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | 10 |
| percent change | Evolocumab |
|---|---|
| Percent Change From Baseline to Week 12 in Low-Density Lipoprotein Cholesterol (LDL-C) | -6.4 ± 22.7 |
| percent change | Evolocumab |
|---|---|
| Percent Change From Baseline to Week 12 in Apolipoprotein B (ApoB) | -6.0 ± 19.8 |
| percent change | Evolocumab |
|---|---|
| Percent Change From Baseline to Week 12 in Lipoprotein(a) (Lp[a]) | -0.2 ± 26.2 |
Collected over All-cause mortality: from enrollment (up to 4 weeks prior to treatment) through end of study (week 12). Serious and other adverse events: from the first dose of study treatment through end of treatment (at week 8) plus 4 weeks (week 12).. Non-serious events are listed at a 2% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Evolocumab | 0/30 (0%) | 2/30 (6.7%) | 8/30 (26.7%) |
| Event | Evolocumab |
|---|---|
| Coronary artery diseaseCardiac disorders | 1/30 |
| SyncopeNervous system disorders | 1/30 |
| Event | Evolocumab |
|---|---|
| Angina pectorisCardiac disorders | 1/30 |
| Injection site painGeneral disorders | 1/30 |
| Injection site swellingGeneral disorders | 1/30 |
| Peripheral swellingGeneral disorders | 1/30 |
| PyodermaInfections and infestations | 1/30 |
| VaricellaInfections and infestations | 1/30 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 1/30 |
| ArthritisMusculoskeletal and connective tissue disorders | 1/30 |
| CoughRespiratory, thoracic and mediastinal disorders | 1/30 |
| Diabetic woundSkin and subcutaneous tissue disorders | 1/30 |
| Age, Continuous(years) | Evolocumab |
|---|---|
| Mean | 23.2 ± 13.1 |
| Age, Customized(Participants) | Evolocumab |
|---|---|
| Adolescents (12 - 17 years old) | 13 |
| Adults (18 - 64 years old) | 16 |
| Adults (65 - 84 years old) | 1 |
| Sex: Female, Male(Participants) | Evolocumab |
|---|---|
| Female | 13 |
| Male | 17 |
| Ethnicity (NIH/OMB)(Participants) | Evolocumab |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 30 |
| Unknown or Not Reported | 0 |
| Race/Ethnicity, Customized(Participants) | Evolocumab |
|---|---|
| Asian | 30 |
| Low-Density Lipoprotein Cholesterol (LDL-C)(mg/dL) | Evolocumab |
|---|---|
| Mean | 473.5 ± 135.2 |
| Apolipoprotein B (ApoB)(mg/dL) | Evolocumab |
|---|---|
| Mean | 275.3 ± 69.1 |
| Lipoprotein(a) (Lp[a])(nmol/L) | Evolocumab |
|---|---|
| Mean | 201.3 ± 177.6 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: Yes — De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
Supporting information: Study protocol, Sap, Icf, Csr
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