TerminatedPhase 1/2

A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

Stopped: Terminated by Sponsor for Business Reasons

United States9 participantsStarted 2016-03

Plain-language summary

This first-in-human study is intended to evaluate the safety and preliminary effectiveness of AAV (Adeno-associated virus)-based liver-directed gene therapy in the treatment of adults with Homozygous Familial Hypercholesterolemia (HoFH).

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Exclusion criteria

✕. niacin \> 250 mg/day: within 6 weeks of baseline
✕. fibrates: within 4 weeks of baseline
✕. lomitapide: within 8 weeks of baseline
✕. mipomersen: within 24 weeks of baseline

What they're measuring

Number of Participants With IP (Investigational Product) Related Adverse Events

Timeframe: Up to 24 weeks

Trial details

NCT IDNCT02651675

SponsorREGENXBIO Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-11-27

Results submitted2023-03-28

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