Homozygous Familial Hypercholesterolemia HoFHPhase 4RAMANNCT03403374
Safety and Tolerability of Repatha® (Evolocumab) in Indian Participants With Homozygous Familial Hypercholesterolemia
A Phase 4 study testing evolocumab in people with Homozygous Familial Hypercholesterolemia HoFH, run by Amgen at 12 sites in India.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 12 to 80 years
- Conditions the study is for
- Homozygous Familial Hypercholesterolemia HoFH
- What is being tested?
- evolocumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 12 sitesIndia
- Study dates
- 4 Aug 2018to 27 Nov 2019
- Phase
- Phase 4
- 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 12 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female ≥ 12 to ≤ 80 years of age at the time of signing the informed consent
- Diagnosis of HoFH based on low-density lipoprotein cholesterol (LDL-C), familial history and xanthoma
- On a low-fat diet and receiving background lipid-lowering therapy stable for 4 weeks prior to screening and during the time frame of the trial
- Fasting LDL-C at screening > 130 mg/dL (3.4 mmol/L)
- Fasting triglycerides at screening ≤ 400 mg/dL (4.5 mmol/L)
Who cannot join
- Use of mipomersen or lomitapide within 6 months of screening.
- Known active infection or major hematologic, renal, metabolic, gastrointestinal, hepatic, or endocrine dysfunction
- Currently receiving treatment in another investigational device or drug study, or less than 30 days since ending treatment on another investigational device or drug study(ies)
- Female subject is pregnant or breastfeeding or planning to become pregnant or breastfeed
- Female subjects of childbearing potential unwilling to use an acceptable method of effective contraception
- Subject has known sensitivity to any of the products to be administered during dosing
- History or evidence of any other clinically significant disorder, condition or disease
- Subject has previously received evolocumab or any other proprotein convertase subtilisin/kexin type 9 (PKSK-9)-inhibiting therapy
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
India12 sites
Research Site
Ahmedabad, Gujarat, 380 054
Research Site
Bangalore, Karnataka, 560 017
Research Site
Belagavi, Karnataka, 590010
Research Site
Kochi, Kerala, 682 027
Research Site
Lucknow, Uttar Pradesh, 226 003
Research Site
Mumbai, Maharashtra, 400 007
Research Site
New Delhi, National Capital Territory of Delhi, 110 002
Research Site
New Delhi, National Capital Territory of Delhi, 110 029
Research Site
New Delhi, National Capital Territory of Delhi, 110 060
Research Site
New Delhi, National Capital Territory of Delhi, 110 062
Research Site
Pune, Maharashtra, 411 005
Research Site
Pune, Maharashtra, 411 006
About this study
Official title: A Multicenter, Open-label, Single-arm, Study to Evaluate Safety and Tolerability of Repatha in Patients With Homozygous Familial Hypercholesterolemia (HoFH) in India
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.
Read the detailed description
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.
- Being tested
- evolocumab
- Keywords
- Evolocumab, Hypercholesterolemia
- Medical subject headings
- Hypercholesterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
evolocumabDrug
Also called Repatha®, EvoMab
Administered by SC injection via autoinjector (AI)/pen
| Study group | Role | What they receive |
|---|---|---|
| Evolocumab | Experimental | Evolocumab 420 mg subcutaneous (SC) once monthly (QM) or every 2 weeks (Q2W; for participants on apheresis). Receives: Drug: evolocumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 12 weeks
Secondary outcomes (3)
Percent Change From Baseline to Week 12 in Low-Density Lipoprotein Cholesterol (LDL-C)
Measured: baseline, week 12
Percent Change From Baseline to Week 12 in Apolipoprotein B (ApoB)
Measured: baseline, week 12
Percent Change From Baseline to Week 12 in Lipoprotein(a) (Lp[a])
Measured: baseline, week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jan 2018
- Study start4 Aug 2018
- Primary completion27 Nov 2019
- Study completion27 Nov 2019
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 Homozygous Familial Hypercholesterolemia HoFHStudies of the same condition, open and under way.
- More trials run by AmgenThe 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amgen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- MD, Study director, Amgen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03403374
- Sponsor's protocol ID
- 20170199
- Other IDs
- 2020-005111-51
- Acronym
- RAMAN
- Status last verified
- May 2024
- First submitted
- 3 Jan 2018
- Last updated
- 29 May 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
Publications
Raal FJ, Hegele RA, Ruzza A, Lopez JAG, Bhatia AK, Wu J, Wang H, Gaudet D, Wiegman A, Wang J, Santos RD. Evolocumab Treatment in Pediatric Patients With Homozygous Familial Hypercholesterolemia: Pooled Data From Three Open-Label Studies. Arterioscler Thromb Vasc Biol. 2024 May;44(5):1156-1164. doi: 10.1161/ATVBAHA.123.320268. Epub 2024 Mar 28.
PubMed 38545781Bansal S, Ruzza A, Sawhney J, Kulkarni G, Iyengar S, Mehta V, Hamer A, Wu Y, Raal FJ. Evolocumab in patients with homozygous familial hypercholesterolemia in India. J Clin Lipidol. 2021 Nov-Dec;15(6):814-821. doi: 10.1016/j.jacl.2021.10.003. Epub 2021 Oct 20.
PubMed 34750081
Study at a glance
From the registry record, last updated by the sponsor on 29 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-label, Single-arm, Study to Evaluate Safety and Tolerability of Repatha in Patients With Homozygous Familial Hypercholesterolemia (HoFH) in India
- ClinicalTrials.gov ID
- NCT03403374
- Acronym / sponsor ID
- RAMAN / 20170199
- Keywords
- Evolocumab, Hypercholesterolemia
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- AmgenIndustry (pharma, biotech, devices)
- Study start
- 4 Aug 2018 (actual)
- Primary completion
- 27 Nov 2019 (actual)
- Study completion
- 27 Nov 2019 (actual)
- First posted
- 18 Jan 2018 (actual)
- Results
- Posted 3 Nov 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Evolocumab | Experimental | Drug: evolocumabEvolocumab 420 mg subcutaneous (SC) once monthly (QM) or every 2 weeks (Q2W; for participants on apheresis). |
Who may qualify
- Ages
- 12 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Male or female ≥ 12 to ≤ 80 years of age at the time of signing the informed consent
- Diagnosis of HoFH based on low-density lipoprotein cholesterol (LDL-C), familial history and xanthoma
- On a low-fat diet and receiving background lipid-lowering therapy stable for 4 weeks prior to screening and during the time frame of the trial
- Fasting LDL-C at screening > 130 mg/dL (3.4 mmol/L)
- Fasting triglycerides at screening ≤ 400 mg/dL (4.5 mmol/L)
Key exclusion criteria (8 listed)
- Use of mipomersen or lomitapide within 6 months of screening.
- Known active infection or major hematologic, renal, metabolic, gastrointestinal, hepatic, or endocrine dysfunction
- Currently receiving treatment in another investigational device or drug study, or less than 30 days since ending treatment on another investigational device or drug study(ies)
- Female subject is pregnant or breastfeeding or planning to become pregnant or breastfeed
- Female subjects of childbearing potential unwilling to use an acceptable method of effective contraception
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 |
|---|---|---|
| evolocumabAlso: Repatha®, EvoMabAdministered by SC injection via autoinjector (AI)/pen | Drug | Evolocumab |
Primary outcome
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame: 12 weeks
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.
3 secondary outcomes
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 May 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 38545781Raal FJ, Hegele RA, Ruzza A, Lopez JAG, Bhatia AK, Wu J, Wang H, Gaudet D, Wiegman A, Wang J, Santos RD. Evolocumab Treatment in Pediatric Patients With Homozygous Familial Hypercholesterolemia: Pooled Data From Three Open-Label Studies. Arterioscler Thromb Vasc Biol. 2024 May;44(5):1156-1164. doi: 10.1161/ATVBAHA.123.320268. Epub 2024 Mar 28.
- Derived from this study: PubMed 34750081Bansal S, Ruzza A, Sawhney J, Kulkarni G, Iyengar S, Mehta V, Hamer A, Wu Y, Raal FJ. Evolocumab in patients with homozygous familial hypercholesterolemia in India. J Clin Lipidol. 2021 Nov-Dec;15(6):814-821. doi: 10.1016/j.jacl.2021.10.003. Epub 2021 Oct 20.
- AmgenTrials clinical trials websiteLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
Related trials for Homozygous Familial Hypercholesterolemia HoFH
Active, not recruitingObservational study
Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia (HoFH), Major Adverse Cardiovascular Events (MACE)
RecruitingPhase 3
Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia
Recruiting
The ORIGIN-FH Study
Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH)
RecruitingPhase 3
Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies
Heterozygous or Homozygous Familial Hypercholesterolemia
RecruitingPhase 1
A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias
Cardiovascular, Metabolic Disease
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.
MD study director
Amgen
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing evolocumab in people with Homozygous Familial Hypercholesterolemia HoFH, run by Amgen at 12 sites in India.
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 8 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 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in India. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 3 Nov 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?
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) (time frame: 12 weeks). 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 NCT03403374, 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 29 May 2024.