Safety and Tolerability of Repatha in Indian Subjects With Homozygous Familial Hypercholesterolemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03403374
Recruitment Status : Recruiting
First Posted : January 18, 2018
Last Update Posted : October 12, 2018
Information provided by (Responsible Party):

Brief Summary:
To describe the safety and tolerability of Repatha in subjects with homozygous familial hypercholesterolemia (HoFH) in India. All subjects will receive Repatha over an 8 week period.

Condition or disease Intervention/treatment Phase
Homozygous Familial Hypercholesterolemia HoFH Drug: Repatha® (evolocumab) Phase 4

Detailed Description:
An open-label, multicentre, phase 4 study to describe the safety and tolerability of Repatha in 30 Indian subjects 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. Subjects will receive Repatha 420 mg QM SC and study visits will occur approximately every 4 weeks. Apheresis subjects will receive Repatha 420 mg SC every 2 weeks to correspond with their apheresis schedule. Final administration of Repatha (for all subjects) will occur at week 8. The end of study (EOS) visit will occur at week 12 for all subjects.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : August 4, 2018
Estimated Primary Completion Date : October 25, 2020
Estimated Study Completion Date : October 25, 2020

Resource links provided by the National Library of Medicine

Drug Information available for: Evolocumab

Arm Intervention/treatment
Experimental: Repatha® (evolocumab)
Single arm, all subjects receive Repatha administered by subcutaneous injection via autoinjector(AI)/pen
Drug: Repatha® (evolocumab)
Administered of Repatha by subcutaneous injection via autoinjector(AI)/pen

Primary Outcome Measures :
  1. Number of participants with treatment related Adverse Events [ Time Frame: 12 weeks ]
    Characterize the safety and tolerability of Repatha based on an analysis of the subject incidence of treatment-emergent adverse events

Secondary Outcome Measures :
  1. Change in LDC-C [ Time Frame: Week 12 ]
    change from baseline to week 12 in low-density lipoprotein cholesterol (LDL-C)

  2. Change in ApoB [ Time Frame: Week 12 ]
    change from baseline to week 12 apolipoprotein B (ApoB)

  3. Change in Lp(a) [ Time Frame: week 12 ]
    change from baseline to week 12 in lipoprotein(a) [Lp(a)] in HoFH patients in India

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years to 80 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Male or female ≥ 12 to ≤ 80 years of age at the time of signing the informed consent.
  • Diagnosis of HoFH based on 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)

Exclusion Criteria:

  • 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 PCSK9-inhibiting therapy.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03403374

Contact: Amgen Call Center 866-572-6436

Research Site Recruiting
New Delhi, Delhi, India, 110 002
Research Site Recruiting
New Delhi, Delhi, India, 110 029
Research Site Recruiting
Ahmedabad, Gujarat, India, 380 054
Research Site Recruiting
Bangalore, Karnataka, India, 560 017
Research Site Recruiting
Belgaum, Karnataka, India, 590 010
Research Site Recruiting
Mumbai, Maharashtra, India, 400 007
Research Site Recruiting
Pune, Maharashtra, India, 411 005
Research Site Recruiting
Pune, Maharashtra, India, 411 006
Sponsors and Collaborators
Study Director: MD Amgen

Additional Information:
Responsible Party: Amgen Identifier: NCT03403374     History of Changes
Other Study ID Numbers: 20170199
First Posted: January 18, 2018    Key Record Dates
Last Update Posted: October 12, 2018
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (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.

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Amgen:

Additional relevant MeSH terms:
Hyperlipoproteinemia Type II
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Antibodies, Monoclonal
Immunologic Factors
Physiological Effects of Drugs