A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501

NCT04080050 | Unknown FDA Drug

• 1 other identifier: RGX-501-102
• Study Type: observational
• Target: 8
• Locations: 3 countries
• Sites: 6

Brief Summary

This long-term observational study is designed to follow subjects who, during another Clinical Study, received gene therapy treatment used to treat their Homozygous Familial Hypercholesterolemia (HoFH) disease. This study is intended to follow those subjects for up to 5 years since they received treatment to look for any long-term safety concerns. There is no investigational drug or therapy provided as part of this study.

Conditions

Homozygous Familial Hypercholesterolemia (HoFH)

Keywords

LDL Receptors; Gene Therapy; Metabolic Diseases; Rare Diseases; Genetic Diseases; Atherosclerosis; Cardiovascular Disease

Outcome Measures

Primary Outcomes (1)

• Number of incidents of new and unexpected adverse events and serious adverse events.

  The number of times a new and unexpected adverse event and/or serious adverse event is reported.

  Up to 5 years after receiving treatment with RGX-501

Secondary Outcomes (3)

• The absolute LDL-C level in mg/dL by beta quantification

  Year 3 after receiving treatment with RGX-501

• Absolute total cholesterol, LDL-C, very low density lipoprotein cholesterol (VLDL-C), high density lipoprotein cholesterol (HDL-C), calculated non-HDL-C, triglycerides (TG), and lipoprotein a (Lp(a)) over the study duration

  Up to 5 years after receiving treatment with RGX-501

• Usage of lipid-lowering therapies over time

  Up to 5 years after receiving treatment with RGX-501

Study Arms (1)

RGX-501

Study participants who have received RGX-501 gene therapy in a separate parent trial

Drug: Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy

Interventions

Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy DRUG

No investigational product will be administered in this study. All participants have previously received a one-time intravenous infusion of RGX-501 in a separate clinical trial

Also known as: RGX-501

RGX-501

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Participants who have previously received RGX-501 in a separate parent trial

You may qualify if:

• To be eligible to participate in this study, a participant must have previously received RGX-501 in a separate parent trial, and the participant or participant's legal guardian(s) is/(are) willing and able to provide written, signed informed consent after the nature of the study has been explained, prior to any research-related procedures.

You may not qualify if:

• None

Sponsors & Collaborators

• REGENXBIO Inc.: lead

Study Sites (6)

Columbus Location

Columbus, Ohio, 43210, United States

Location

Portland location

Portland, Oregon, 97239, United States

Location

Philadelphia location

Philadelphia, Pennsylvania, 19104, United States

Location

Nashville location

Nashville, Tennessee, 37232, United States

Location

Montreal location

Montreal, Quebec, H1T1C8, Canada

Location

Rotterdam location

Rotterdam, 3015 CE, Netherlands

Location

MeSH Terms

Conditions

Homozygous Familial Hypercholesterolemia; Hyperlipoproteinemia Type II; Metabolic Diseases; Rare Diseases; Genetic Diseases, Inborn; Atherosclerosis; Cardiovascular Diseases

Interventions

Genetic Therapy

Condition Hierarchy (Ancestors)

Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hyperlipoproteinemias; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Nutritional and Metabolic Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Arteriosclerosis; Arterial Occlusive Diseases; Vascular Diseases

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics; Genetic Engineering; Genetic Techniques; Investigative Techniques

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 3, 2019
• First Posted: September 6, 2019
• Study Start: September 30, 2019
• Primary Completion: September 29, 2024
• Study Completion: September 29, 2025
• Last Updated: March 9, 2023

Record last verified: 2023-03

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1); US (4); NL (1)