Phase 2 Rare Disease Trial, Terminated (NCT02651675)

Phase 2 Rare Disease Trial, Terminated NCT02651675 Sponsor: REGENXBIO Inc. Condition: Rare Disease

Printed from Voxsanity (voxsanity.com.au) — NCT02651675 — Data sourced from ClinicalTrials.gov. Verify directly with the trial site before attending. Not medical advice.

Back to Rare Disease

Trial results

AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

According to the results reported on ClinicalTrials.gov, this trial enrolled nine participants in total, split across three groups: Cohort 1 (3 people), Cohort 2 (3 people), and a Cohort 2 Expansion group (3 people). Eight of the nine participants completed the study — one person in the Cohort 2 Expansion group did not finish. The trial was measuring how the investigational product (a gene therapy delivered via a viral vector) behaved in the body, including tracking unwanted events (called adverse events) linked to the treatment, changes in blood fat levels such as LDL cholesterol, and how much of the viral carrier could be detected in participants' urine and blood. The reported data shows that, for the primary outcome — the number of participants who experienced an adverse event linked to the investigational product — 1 out of 3 participants in Cohort 1, all 3 out of 3 in Cohort 2, and 2 out of 3 in the Cohort 2 Expansion group had such an event recorded. For the secondary outcomes related to blood fat levels, the reported data shows percentage changes from each participant's starting (baseline) levels: LDL cholesterol (a type of fat in the blood often called "bad cholesterol") changed by approximately +7.2% in Cohort 1, +27.4% in Cohort 2, and +9.0% in the Cohort 2 Expansion group. Changes in other blood fat measurements (total cholesterol, HDL cholesterol, triglycerides, and others) were also reported across the three groups, with figures varying considerably between cohorts; for example, one measure showed changes ranging from +32.6% in Cohort 1 to +54.2% in Cohort 2 and −5.5% in the Expansion group. The reported data also shows that detectable amounts of the viral carrier (the vehicle used to deliver the gene therapy) were found in both urine and blood samples for participants in Cohort 1 and Cohort 2, while the reported figure for the Cohort 2 Expansion group was zero for both. Because this was a very small, early-phase trial with only three people per group, the numbers reflect a preliminary exploration rather than a broad population study, and not all detail for every sub-measure was fully reported in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

View full results on ClinicalTrials.gov ↗

These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

You are a man or woman aged 18 or older.
Your LDL cholesterol levels (the "bad" cholesterol) and medical history match the diagnosis of a severe inherited cholesterol condition called homozygous familial hypercholesterolemia (HoFH), whether or not you have already received treatment for it.
Genetic testing has confirmed that you have specific gene mutations affecting both copies of the LDL receptor gene (the gene that helps your body process cholesterol).
A blood test shows that your immune system has a very low level of antibodies against a particular virus used in gene therapy (called AAV8), meaning your body is unlikely to fight off the treatment before it works (confirm with trial site).

Who may not be able to join:

You are unwilling to stop taking certain cholesterol-lowering medicines before the study starts — specifically: niacin (high-dose) at least 6 weeks before, fibrates at least 4 weeks before, lomitapide at least 8 weeks before, or mipomersen at least 24 weeks before.
You have a history of severe liver scarring (cirrhosis) or long-term liver disease, confirmed by a biopsy, scan, or other tests.
Your liver function blood tests at your screening appointment show abnormal results, suggesting your liver may not be working properly (confirm with trial site).

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.

Last synced 27 July 2026

Phase 2: approximately ~30% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.

Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

GP referral letter

Print a one-page summary to share with your doctor.

Voxsanity

Clinical trials and treatments, explained plainly · voxsanity.com.au

Date:

To: My Doctor

Re: Clinical trial enquiry

I found the following past clinical trial relevant to my condition, and wanted to raise it with you. It is no longer recruiting, but I wanted to ask whether similar active trials exist, or whether its results are relevant to my care:

Trial: NCT02651675

Sponsor: REGENXBIO Inc.

Phase: Phase 2

Status: Terminated

Condition: Rare Disease

More information: https://clinicaltrials.gov/study/NCT02651675

Eligibility summary (AI generated)

Written by an AI model from the ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

You are a man or woman aged 18 or older.
Your LDL cholesterol levels (the "bad" cholesterol) and medical history match the diagnosis of a severe inherited cholesterol condition called homozygous familial hypercholesterolemia (HoFH), whether or not you have already received treatment for it.
Genetic testing has confirmed that you have specific gene mutations affecting both copies of the LDL receptor gene (the gene that helps your body process cholesterol).
A blood test shows that your immune system has a very low level of antibodies against a particular virus used in gene therapy (called AAV8), meaning your body is unlikely to fight off the treatment before it works (confirm with trial site).

Who may not be able to join:

You are unwilling to stop taking certain cholesterol-lowering medicines before the study starts — specifically: niacin (high-dose) at least 6 weeks before, fibrates at least 4 weeks before, lomitapide at least 8 weeks before, or mipomersen at least 24 weeks before.
You have a history of severe liver scarring (cirrhosis) or long-term liver disease, confirmed by a biopsy, scan, or other tests.
Your liver function blood tests at your screening appointment show abnormal results, suggesting your liver may not be working properly (confirm with trial site).

Important: Always verify eligibility with the trial site directly before applying.

Access in Australia: A treatment being studied in a clinical trial is generally not yet listed on the PBS. Where a medicine is not approved or not available through a trial, the TGA Special Access Scheme may allow a doctor to access unapproved therapeutic goods for individual patients. See voxsanity.com.au/sas-navigator/ for a plain English explainer.

This information was sourced from ClinicalTrials.gov via Voxsanity (data last synced 27 July 2026). It is not medical advice. Please verify the trial's current status directly with the trial site before acting.

Trial details

Status

Terminated

Phase

Phase 2

Sponsor

REGENXBIO Inc.

Registry

ClinicalTrials.gov

Start date

1 March 2016

Est. completion

27 November 2020

Where this trial is recruiting

🇨🇦 Canada 🇮🇹 Italy 🇳🇱 Netherlands 🇺🇸 United States

Primary endpoints

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

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 27 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov