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 (NCT04046224) tested a gene therapy called ST-920 in people with Fabry disease, a rare inherited condition. The trial enrolled 33 participants in total, spread across nine groups that received either different doses of ST-920 or the same high dose but were selected based on specific characteristics (such as having heart or kidney involvement, being female, or having particular antibody levels). The trial was measuring how often unwanted health events occurred after receiving ST-920, and how levels of a specific enzyme — called alpha-galactosidase A (alpha Gal-A), which is deficient in Fabry disease — changed in the blood over 52 weeks. The reported data shows that across the four initial dose-testing groups (2 people each in the lowest two doses, 3 in the next, and 2 in the highest), every participant who started the trial completed it, and no unwanted health events related to ST-920 were recorded in those groups. In the later expansion groups, the reported data shows that all five participants in the cardiac group, four of five in the female group, two of two in the renal group, all seven in the antibody-negative group, and all five in the antibody-positive group experienced at least one health event considered related to ST-920. Serious health events were recorded in one participant each in the renal, antibody-negative, and antibody-positive groups. The reported data shows no participant in any group left the study early because of a health event. Regarding the enzyme level outcome, the reported data shows increases in alpha Gal-A enzyme activity in the blood from baseline at the 24-week and 52-week time points across the groups where figures were provided — for example, increases ranging from around 16 to 76 units (nmol/h/mL) at 24 weeks, and figures of approximately 50, 31, and 163 units reported at 52 weeks for the first three dose groups (data for the remaining groups at 52 weeks was not reported in the submitted results). These numbers represent changes from each participant's starting level, not absolute values. 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.
Phase 2 Rare Disease Trial, Completed
voxsanity.com.au · Eligibility summary from public government registries · 18 August 2026 · not medical advice
Who may and may not 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 may be able to join
- You are 18 years of age or older
- You have been officially diagnosed with Fabry disease
- You have one or more of the following symptoms: cloudy patches on the surface of the eye (cornea verticillata), burning or tingling pain in the hands and feet (acroparesthesia), inability to sweat normally (anhidrosis), or small dark red skin spots (angiokeratoma)
- You have been fully vaccinated against COVID-19 (following CDC guidelines in the US, or local guidelines in other countries) at least one month before receiving the study treatment
If joining the kidney (renal) group, you also need:
- A kidney function test result (eGFR) between 40 and 90 mL/min/1.73 m² at the screening visit
- Evidence that your kidney function has been declining at a certain rate, based on at least 3 blood test results taken within the past 18 months (confirm with trial site)
If joining the heart (cardiac) group, you also need:
- Evidence of thickening of the heart muscle (left ventricular hypertrophy) shown on an ultrasound or MRI scan, with walls measuring 12mm or more and no other explanation for this, OR signs that your heart condition is getting worse as shown by specific heart scan measurements (confirm with trial site)
Who may not be able to join
Each point below is a reason the trial team may not be able to accept someone. It is not a list of requirements to meet.
- You have antibodies in your blood that could block a specific part of the study treatment (neutralizing antibodies to AAV6)
- Your kidney function test result (eGFR) is below 40 mL/min/1.73 m²
- Your heart failure is classified as moderate-to-severe or worse (New York Heart Association Class III or higher)
- You currently have an active infection with Hepatitis A, B, or C, HIV, or tuberculosis (TB)
- You have a history of significant liver disease such as serious fat build-up in the liver, liver scarring, a specific type of liver inflammation (NASH), cirrhosis, or bile duct problems within the past 6 months (a condition called Gilbert's syndrome is allowed)
- You have raised levels of a specific protein in your blood called alpha fetoprotein (AFP)
- You have had a serious allergic reaction to enzyme replacement therapy (ERT) within the past 6 months
- You are currently using, or have used in the past 6 months, certain medicines that affect the immune system taken by mouth or injection (topical creams or inhaled treatments are allowed)
- You cannot take corticosteroid medications (a type of anti-inflammatory drug) for any reason
- You have a history of cancer, except for certain skin cancers or prostate cancer that was fully treated
- You have a recent history of alcohol or substance misuse
- You have taken part in another clinical drug or medical device trial in the past 3 months, or plan to do so during this study
- You have previously received any gene therapy treatment
- You have a known allergy to any ingredient in the study treatment (ST-920)
- The trial doctor believes there is any other reason you would not be suitable for the study, including risk of COVID-19 infection
If joining the kidney (renal) group, you also cannot join if:
- You have ever been on kidney dialysis or had a kidney transplant
- You had a sudden serious loss of kidney function in the 6 months before screening
- You recently started or changed the dose of certain blood pressure or kidney-protecting medicines (ACE inhibitors or ARBs) within 4 weeks before screening
- You have a certain level of protein in your urine (UPCR above 0.5 g/g) and are not taking ACE inhibitor or ARB medicines (confirm with trial site)
If joining the heart (cardiac) group, you also cannot join if:
- You have significant scarring of the heart muscle shown on a cardiac MRI scan
- You have any reason you cannot have a cardiac MRI scan, based on your local hospital's guidelines
- You recently started or changed the dose of certain blood pressure or heart-protecting medicines (ACE inhibitors or ARBs) within 4 weeks before screening
- Your heart failure is classified as the most severe level (New York Heart Association Class IV)
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.
Trial contact details on record
Principal Investigator: Medical Monitor, Sangamo Therapeutics, Inc.
Australian sites
This trial is not accepting new participants. These are the contact details ClinicalTrials.gov holds for it, kept here for reference. They are not an invitation to enrol, and the sites listed may no longer be running this trial.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial was run
These are the locations recorded on the registry, including 1 in Australia. They are a historical record: this trial is not enrolling, so they are not places you can join it.
Primary endpoints
Incidence of Treatment-emergent Adverse Events (TEAEs) - All; Incidence of Treatment-emergent Adverse Events (TEAEs) - Related to ST-920; Incidence of Treatment-emergent Adverse Events (TEAEs) - Serious; Incidence of Treatment-emergent Adverse Events (TEAEs) - Any TEAEs Leading to Study Discontinuation or Withdrawal
Other options
Data last synced from ClinicalTrials.gov: 14 August 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.