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7 recruiting studies matching your filters

Phase 3 Single-Arm Open-Label Study to Evaluate the Efficacy and Safety of Zodasiran in Adolescent Participants (Age 12 to <18 Years) With Homozygous Familial Hypercholesterolemia (SPRUCE)

This study will evaluate the efficacy and safety of zodasiran subcutaneous (sc) injection in participants 12 to \<18 years of age with genetically or clinicall…

Homozygous Familial Hypercholesterolemia

Estimated

~$10 – $8,750

Arrowhead PharmaceuticalsNCT07473843

Phase 3

An Open-label, Single Arm, Multicenter Extension Study to Evaluate Long-term Safety and Tolerability of Inclisiran in Participants With Heterozygous or Homozygous Familial Hypercholesterolemia Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20 or ORION-19 Studies (VICTORION-PEDS-OLE)

The purpose of this open-label, single arm, multicenter extension study is to evaluate the long-term safety and tolerability of inclisiran in participants with…

Heterozygous or Homozygous Familial Hypercholesterolemia

Estimated

~$10 – $8,750

Novartis PharmaceuticalsNCT05682378

Phase 1

A Phase 1 Open-label, Multicenter, First-in-human, Ascending Dose Trial Evaluating the Safety and Tolerability of a Lipid Nanoparticle Formulation of CRISPR-Guide RNA-Cas9 Nuclease (CTX310) for In Vivo Editing of the Angiopoietin-like 3 (ANGPTL3) Gene in Participants With Refractory Dyslipidemias

This is a single-arm, open-label, multicenter, ascending dose Phase 1 trial that will enroll participants 18 to 75 years of age with dyslipidemias that are ref…

CardiovascularMetabolic DiseaseDyslipidemias+7 more

Estimated

~$10 – $8,750

CRISPR Therapeutics AGNCT07491172

HoFH, the International Clinical Collaborators - A Global HoFH Data-sharing Platform

Homozygous familial hypercholesterolemia (HoFH), a rare inherited disorder caused by bi-allelic mutations in the LDL Receptor pathway, is characterized by extr…

Homozygous Familial Hypercholesterolemia

Estimated

~$10 – $750

University of PennsylvaniaNCT04815005

LOWER: Lomitapide Observational Worldwide Evaluation Registry

This global product exposure registry is a multicentre, long-term, prospective, observational cohort study (exposure registry), designed to evaluate the long t…

Homozygous Familial Hypercholesterolemia

Estimated

~$10 – $1,050

Amryt PharmaNCT02135705

Observational

Opportunity to Reach Individuals With Genetic Dyslipidemia During Infancy and the Newborn Period to Find Familial Hypercholesterolemia

The goal of this clinical trial is to identify different types of Familial Hypercholesterolemia (FH) in infants and newborns. Participants will: * undergo a c…

Heterozygous Familial Hypercholesterolemia (HeFH)Homozygous Familial Hypercholesterolemia (HoFH)Familial Hypercholesterolemia

Estimated

~$10 – $6,250

University of Wisconsin, MadisonNCT07470723

The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository

This repository will establish for the first time a system to carefully assess and monitor over time the general health and the amount of cholesterol in the ar…

Homozygous Familial Hypercholesterolemia

Estimated

~$10 – $1,125

The Rogosin InstituteNCT01109368