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

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ClinicalTrials.gov Identifier: NCT04080050
Recruitment Status : Not yet recruiting
First Posted : September 6, 2019
Last Update Posted : September 23, 2019
Sponsor:
Information provided by (Responsible Party):
Regenxbio Inc.

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.

Condition or disease Intervention/treatment
Homozygous Familial Hypercholesterolemia (HoFH) Drug: Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy

Detailed Description:

Homozygous Familial Hypercholesterolemia (HoFH) is a rare genetic metabolic disorder characterized by absent or severely reduced capacity to catabolize circulating LDL particles by the hepatic LDL receptor. As a consequence, HoFH subjects present abnormal total plasma cholesterol (LDL-C) levels, resulting in severe atherosclerosis often leading to early onset of cardiovascular disease. Early initiation of aggressive treatment for these patients is therefore essential. Unfortunately, despite existing therapies, treated LDL-C levels could remain well above acceptable levels. Thus, the functional replacement of the defective LDLR via AAV-based liver-directed gene therapy, RGX-501, may be a viable approach to treat this disease and improve response to current lipid-lowering treatments.

This is a prospective, observational study to evaluate the long-term safety and efficacy after a single administration of RGX-501. Eligible participants are those who previously have enrolled in a clinical study and received a single intravenous infusion of RGX-501.


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Study Type : Observational
Estimated Enrollment : 12 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501
Estimated Study Start Date : September 30, 2019
Estimated Primary Completion Date : September 29, 2024
Estimated Study Completion Date : September 29, 2025


Group/Cohort Intervention/treatment
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
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
Other Name: RGX-501




Primary Outcome Measures :
  1. Number of incidents of new and unexpected adverse events and serious adverse events. [ Time Frame: Up to 5 years after receiving treatment with RGX-501 ]
    The number of times a new and unexpected adverse event and/or serious adverse event is reported.


Secondary Outcome Measures :
  1. The absolute LDL-C level in mg/dL by beta quantification [ Time Frame: Year 3 after receiving treatment with RGX-501 ]
    Absolute LDL-C level by beta quantification at Year 3 after receiving treatment with RGX-501

  2. 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 [ Time Frame: Up to 5 years 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

  3. Usage of lipid-lowering therapies over time [ Time Frame: Up to 5 years after receiving treatment with RGX-501 ]
    Usage of lipid-lowering therapies over time



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Participants who have previously received RGX-501 in a separate parent trial
Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • None

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04080050


Contacts
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Contact: Patient Advocacy 800-446-3401 HoFH@regenxbio.com

Locations
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United States, Florida
Boca Raton Location Not yet recruiting
Boca Raton, Florida, United States, 33434
United States, Kansas
Kansas City Location Not yet recruiting
Kansas City, Kansas, United States, 66160
United States, Oregon
Portland location Not yet recruiting
Portland, Oregon, United States, 97239
United States, Pennsylvania
Philadelphia location Not yet recruiting
Philadelphia, Pennsylvania, United States, 19104
United States, Tennessee
Nashville location Not yet recruiting
Nashville, Tennessee, United States, 37232
Canada, Quebec
Montreal location Not yet recruiting
Montreal, Quebec, Canada, H1T1C8
Montreal Location Not yet recruiting
Montreal, Quebec, Canada, H3A1A1
Italy
Palermo location Not yet recruiting
Palermo, PA, Italy, 90127
Rome location Not yet recruiting
Roma, RM, Italy, 00161
Netherlands
Rotterdam location Not yet recruiting
Rotterdam, Netherlands, 3015 CE
Sponsors and Collaborators
Regenxbio Inc.

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Responsible Party: Regenxbio Inc.
ClinicalTrials.gov Identifier: NCT04080050     History of Changes
Other Study ID Numbers: RGX-501-102
First Posted: September 6, 2019    Key Record Dates
Last Update Posted: September 23, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Regenxbio Inc.:
LDL Receptors
Gene Therapy
Metabolic Diseases
Rare Diseases
Genetic Diseases
Atherosclerosis
Cardiovascular Disease
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type II
Hypercholesterolemia
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias