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Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients 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.
 
ClinicalTrials.gov Identifier: NCT04233918
Recruitment Status : Not yet recruiting
First Posted : January 18, 2020
Last Update Posted : January 18, 2020
Sponsor:
Information provided by (Responsible Party):
Regeneron Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE January 6, 2020
First Posted Date  ICMJE January 18, 2020
Last Update Posted Date January 18, 2020
Estimated Study Start Date  ICMJE June 5, 2020
Estimated Primary Completion Date December 16, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 16, 2020)
  • PK parameter: Maximum serum concentration observed (Cmax) [ Time Frame: Up to week 24 ]
    Part A
  • PK parameter: Area under the concentration-time curve (AUC) [ Time Frame: Up to week 24 ]
    Part A
  • PK parameter: Observed terminal half-life linear (t1/2) [ Time Frame: Up to week 24 ]
    Part A
  • Percent change in calculated low-density lipoprotein cholesterol (LDL-C) from baseline to week 24 [ Time Frame: Week 24 ]
    Part B
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: January 16, 2020)
  • Incidence of treatment-emergent adverse events (TEAE) and other safety variables over time [ Time Frame: Up to week 68 ]
    Parts A & B; safety variables include laboratory data, vital signs, Tanner stages, and electrocardiograms (ECG).
  • Percent change in Apoliprotein (Apo) B from baseline to week 24 [ Time Frame: Week 24 ]
    Part B
  • Percent change in non-High-density lipoprotein cholesterol (HDL-C) from baseline to week 24 [ Time Frame: Week 24 ]
    Part B
  • Percent change in total cholesterol (TC) from baseline to week 24 [ Time Frame: Week 24 ]
    Part B
  • Proportion of patients with ≥50% reduction in calculated LDL-C at week 24 [ Time Frame: Week 24 ]
    Part B
  • Percent change in calculated LDL-C from baseline to week 24 in patients who have negative/negative and null/null mutations [ Time Frame: Week 24 ]
    Part B
  • Percent change in lipoprotein a [Lp(a)] from baseline to week 24 [ Time Frame: Week 24 ]
    Part B
  • Concentrations of total evinacumab over time [ Time Frame: Up to week 68 ]
    Part B
  • PK parameter: Cmax steady state(Cmax.ss) [ Time Frame: Up to week 68 ]
    Part B
  • PK parameter: Concentration of drug over time (the area under the concentration verses time curve over the dosing interval [AUCtau.ss[) [ Time Frame: Up to week 68 ]
    Part B
  • PK parameter: Ctrough.ss [ Time Frame: Up to week 68 ]
    Part B
  • Incidence and titer of treatment-emergent anti-drug antibodies (ADA) over time [ Time Frame: Up to week 68 ]
    Part B
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia
Official Title  ICMJE A Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia
Brief Summary

The primary objective for Part A of the study is to assess the pharmacokinetics (PK) of evinacumab in pediatric patients with homozygous familial hypercholesterolemia (HoFH).

The primary objective for Part B of the study is to demonstrate a reduction of low-density lipoprotein cholesterol (LDL-C) by evinacumab in pediatric (5 to 11 years of age) patients with HoFH.

The secondary objective for Part A of the study is to evaluate the safety and tolerability of evinacumab administered intravenous (IV) in pediatric patients with HoFH.

The secondary objectives for Part B of the study are:

  • To evaluate the effect of evinacumab on other lipid parameters (ie, apolipoprotein B (Apo B), non-high-density lipoprotein cholesterol (non-HDL-C), total cholesterol (TC), lipoprotein a [Lp(a)]) in pediatric patients with HoFH
  • To evaluate the safety and tolerability of evinacumab administered IV in pediatric patients with HoFH
  • To assess the PK of evinacumab in pediatric patients with HoFH
  • To assess the immunogenicity of evinacumab in pediatric patients with HoFH over time
  • To evaluate patient efficacy by mutation status
Detailed Description Part A is Phase 1b Part B is Phase 3
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Homozygous Familial Hypercholesterolemia
Intervention  ICMJE Drug: Evinacumab
Part A: Single IV dose Part B & C: IV dose Q4W
Other Name: REGN1500
Study Arms  ICMJE Experimental: Evinacumab
Part A: Single intravenous (IV) dose Part B: IV dose every 4 weeks (Q4W) until week 44 Part C: IV dose Q4W in the long-term extension
Intervention: Drug: Evinacumab
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: January 16, 2020)
24
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE January 27, 2028
Estimated Primary Completion Date December 16, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  1. Diagnosis of functional HoFH by either genetic or clinical criteria as defined in the protocol
  2. LDL-C >130 mg/dL at the screening visit
  3. Body weight ≥15 kg
  4. Receiving stable maximally tolerated therapy*at the screening visit *Maximally tolerated therapy could include a daily statin.
  5. Willing and able to comply with clinic visits and study-related procedures
  6. Provide signed informed consent or assent

Key Exclusion Criteria:

  1. Background pharmacologic LMT, nutraceuticals or over-the-counter (OTC) therapies known to affect lipids, at a dose/regimen that has not been stable for at least 4 weeks (8 weeks for PCSK9 inhibitors) before the screening visit and patient is unwilling to enter the run-in period
  2. For patients entering Part A, unable to temporarily discontinue apheresis from the baseline visit through the week 4 visit
  3. Receiving lipid apheresis, a setting (if applicable) and schedule that has not been stable for approximately 8 weeks before the screening visit or an apheresis schedule that is not anticipated to be stable over the duration of the treatment period (48 weeks).
  4. Plasmapheresis within 8 weeks of the screening visit, or plans to undergo plasmapheresis during the 48-week open-label treatment period
  5. Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins
  6. Newly diagnosed (within 3 months prior to randomization visit) diabetes mellitus or poorly controlled diabetes as defined in the protocol

Note: Other protocol-defined criteria apply

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years to 11 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Clinical Trials Administrator 844-734-6643 clinicaltrials@regeneron.com
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04233918
Other Study ID Numbers  ICMJE R1500-CL-17100
2019-001931-30 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
Supporting Materials: Study Protocol
Supporting Materials: Statistical Analysis Plan (SAP)
Supporting Materials: Informed Consent Form (ICF)
Supporting Materials: Clinical Study Report (CSR)
Supporting Materials: Analytic Code
Time Frame: Individual anonymized participant data will be considered for sharing once the indication has been approved by a regulatory body, if there is legal authority to share the data and there is not a reasonable likelihood of participant re-identification.
Access Criteria: Qualified researchers may request access to anonymized patient level data or aggregate study data when Regeneron has received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency [EMA], Pharmaceuticals and Medical Devices Agency [PMDA], etc) for the product and indication, has the legal authority to share the data, and has made the study results publicly available (eg, scientific publication, scientific conference, clinical trial registry).
URL: https://errs.regeneron.com/external
Responsible Party Regeneron Pharmaceuticals
Study Sponsor  ICMJE Regeneron Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Clinical Trial Management Regeneron Pharmaceuticals
PRS Account Regeneron Pharmaceuticals
Verification Date January 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP