Safety and Tolerability of Repatha® (Evolocumab) in Indian Participants With Homozygous Familial Hypercholesterolemia

NCT03403374 | Completed Phase 4 Results

• 2 other identifiers: 201701992020-005111-51
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 12

Brief 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.

Conditions

Homozygous Familial Hypercholesterolemia HoFH

Keywords

Evolocumab; Hypercholesterolemia

Outcome Measures

Primary Outcomes (1)

• 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.

  12 weeks

Secondary Outcomes (3)

• Percent Change From Baseline to Week 12 in Low-Density Lipoprotein Cholesterol (LDL-C)

  baseline, week 12

• Percent Change From Baseline to Week 12 in Apolipoprotein B (ApoB)

  baseline, week 12

• Percent Change From Baseline to Week 12 in Lipoprotein(a) (Lp[a])

  baseline, week 12

Study Arms (1)

Evolocumab

EXPERIMENTAL

Evolocumab 420 mg subcutaneous (SC) once monthly (QM) or every 2 weeks (Q2W; for participants on apheresis).

Drug: evolocumab

Interventions

evolocumab DRUG

Administered by SC injection via autoinjector (AI)/pen

Also known as: Repatha®, EvoMab

Evolocumab

Eligibility Criteria

• Age: 12 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Amgen: lead

Study Sites (12)

Research Site

Ahmedabad, Gujarat, 380 054, India

Location

Research Site

Bangalore, Karnataka, 560 017, India

Location

Research Site

Belagavi, Karnataka, 590010, India

Location

Research Site

Kochi, Kerala, 682 027, India

Location

Research Site

Mumbai, Maharashtra, 400 007, India

Location

Research Site

Pune, Maharashtra, 411 005, India

Location

Research Site

Pune, Maharashtra, 411 006, India

Location

Research Site

New Delhi, National Capital Territory of Delhi, 110 002, India

Location

Research Site

New Delhi, National Capital Territory of Delhi, 110 029, India

Location

Research Site

New Delhi, National Capital Territory of Delhi, 110 060, India

Location

Research Site

New Delhi, National Capital Territory of Delhi, 110 062, India

Location

Research Site

Lucknow, Uttar Pradesh, 226 003, India

Location

Related Publications (2)

• 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.

  PMID: 38545781 BACKGROUND

• Bansal 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.

  PMID: 34750081 DERIVED

Related Links

• AmgenTrials clinical trials website

MeSH Terms

Conditions

Hypercholesterolemia

Interventions

evolocumab

Condition Hierarchy (Ancestors)

Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Study Director
• Organization: Amgen Inc.

medinfo@amgen.com

866-572-6436

Study Officials

• MD

  Amgen

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 3, 2018
• First Posted: January 18, 2018
• Study Start: August 4, 2018
• Primary Completion: November 27, 2019
• Study Completion: November 27, 2019
• Last Updated: May 29, 2024
• Results First Posted: November 3, 2020

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication (or other new use) have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.
• Access Criteria: Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors, and if not approved, may be further arbitrated by a Data Sharing Independent Review Panel. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the link below.

Locations

IN (12)