CompletedPhase 3

Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)

Germany, Israel, Italy46 participantsStarted 2020-12-14

Plain-language summary

This is a single arm, open label, multi centre phase III study to evaluate the efficacy and long term safety of lomitapide in paediatric patients with HoFH receiving stable lipid lowering therapy (LLT) (including lipoprotein apheresis (LA), when applicable) comprising of the following phases: * Screening Period (starting at Week 12, i.e. ≤12 weeks prior to Baseline for up to 6 weeks) * Stratified Enrolment and Start of Run in Period (starting at minimum at Week 6, i.e., 6 weeks prior to Baseline for a minimum of 6 weeks): * Efficacy Phase (starting at Baseline, i.e. Day \[D\] 0 for 24 weeks±3 days * Safety Phase (starting at Week 24±3 days for 80±1 weeks)

Who can participate

Age range5 Years – 17 Years

SexALL

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Inclusion criteria

✓. Genetic confirmation of 2 mutant alleles at the LDL receptor (LDLR), apo B, Proprotein convertase subtilisin/kexin type 9 (PCSK9), or LDL receptor adapter protein 1 (LDLRAP1) gene locus OR
✓. An untreated LDL C \>500 mg/dL (13 mmol/L) or treated LDL C ≥300 mg/dL (8 mmol/L ) together with either Cutaneous or tendon xanthoma before age 10 years or Untreated LDL C levels consistent with heterozygous FH in both parents
✓. \>160 mg/dL (4.1 mmol/L, no documented cardiovascular disease \[CVD\]) or
✓. \>130 mg/dL (3.4 mmol/L, established CVD defined as aortic valve disease and/or coronary atherosclerosis)
✓. LLT (including LA, when applicable) must be stable for at least 6 weeks prior to Baseline (Run in Period) and remain stable through Week 24±3 days (end of Efficacy Phase)
✓. The patient must be compliant with both the low fat diet supplying \<20% of energy (calories) from fat or \<30 g fat, whichever is the lesser amount starting at the beginning of the Run in Period and the dietary supplement regimen starting at Week 2 of the Run in Period, both continuing until completion of the study

What they're measuring

Efficacy Endpoint: Percent Change in Low-density Lipoprotein Cholesterol (LDL C) at Week 24 Compared to Baseline

Timeframe: Baseline through Week 24

Trial details

NCT IDNCT04681170

SponsorAmryt Pharma

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-10-16

Results submitted2025-06-12

View on ClinicalTrials.gov More Homozygous Familial Hypercholesterolaemia (HoFH) trials