E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Homozygous Familial Hypercholesterolemia (HoFH) |
|
E.1.1.1 | Medical condition in easily understood language |
Hypercholesterolemia (high cholesterol), familial |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10057080 |
E.1.2 | Term | Homozygous familial hypercholesterolemia |
E.1.2 | System Organ Class | 100000004850 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To characterize the safety and tolerability in homozygous familial hypercholesterolemia HoFH patients in India exposed to 12 weeks of Repatha. |
|
E.2.2 | Secondary objectives of the trial |
To characterize the efficacy of 12 weeks of Repatha on low-density lipoprotein cholesterol (LDL-C), apolipoprotein B (ApoB) and lipoprotein(a) [Lp(a)] in HoFH patients in India |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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) |
|
E.4 | Principal 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. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
The subject incidence of treatment emergent adverse events |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Continually during treatment period and at EOS |
|
E.5.2 | Secondary end point(s) |
- Percent change from baseline in LDL-C at week 12 - Percent change from baseline in ApoB at week 12 - Percent change from baseline in Lp(a) at week 12 |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Continually during treatment period and at EOS |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
|
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial months | 17 |