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Homozygous Familial Hypercholesterolemia (HoFH) - Healing Genes
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Homozygous Familial Hypercholesterolemia (HoFH)

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A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

A Phase 1/2a, Single Ascending IV Dose Clinical Trial Investigating Human Low Density Lipoprotein Receptor (LDLR) Gene Therapy in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)


Phase 1 / 2

DESCRIPTION:

Researchers at 5 research centers in FL, KS, OR, PA, and TN as well as abroad are recruiting patients with Homozygous Familial Hypercholesterolemia (HoFH) for a trial of a new liver-directed gene therapy to lower lipids. This therapy is a virus engineered to transfer a functional hLDLR gene to the patient, so that the liver will recognize and process LDL particles.

Participants will undergo an optional assessment of their body’s ability to process LDLs, before admission to the hospital and a single infusion of the gene therapy. Follow up blood tests will be drawn daily for 3 days up to 72 hours and then weekly up to 12 weeks. Twelve weeks after the test therapy administration, subjects that participated in the first optional kinetics assessment will be re-admitted for a repeat LDL kinetics study. After this visit subjects will be permitted to resume LDL apheresis and/or plasma exchange, and other lipid lowering treatments at the discretion of their caring physician. Follow up lab tests every 12 weeks up to 52 weeks will be required with bi-annual telephone calls and annual lipid tests thereafter up to 260 weeks.


PATIENT MUST:

  • Be 18 years of age or older
  • Have a diagnosis of HoFH with biallelic LDLR mutations
  • Be willing to use birth control, if sexually active, and not be pregnant

THE STUDY INVOLVES:

  1. Screening to confirm eligibility, including imaging procedures, heart and lung tests, and lab tests.
  2. Participants will undergo an optional assessment of their body’s ability to process LDLs (kinetics assessment). This could take 2 days,
  3. Admission to the hospital and a single infusion of the gene therapy.
  4. Follow up blood tests will be drawn daily for 3 days and then weekly up to 12 weeks.
  5. Twelve weeks after the investigatory therapy administration, subjects that participated in the first optional kinetics assessment will be re-admitted for a repeat LDL kinetics study. After this visit subjects will be permitted to resume LDL apheresis and/or plasma exchange, and other lipid lowering treatments at the discretion of their caring physician.
  6. Follow up lab tests every 12 weeks up to 52 weeks will be required with bi-annual telephone calls and annual lipid tests thereafter up to 260 weeks.

LOCATIONS AND CONTACTS:

The study sites include
Excel Medical Clinical Trials, LLC in Boca Raton, FL. Map.
University of Kansas Medical Center in Kansas City, KS. Map.
Oregon Health and Science University in Portland, OR. Map.
University of Pennsylvania in Philadelphia, PA. Map.
Vanderbilt University Medical Center in Nashville, TN. Map.
 
Contacts:
Robert Fiorentino, MD  |  240-552-8134  |  [email protected]
 
SPONSOR INFORMATION:
Regenxbio Inc.
 
Or go online:
https://clinicaltrials.gov/ct2/show/NCT02651675

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