Ended Phase I and Phase II (Integrated)- First administration to humans Fabry Disease (X-linked lysosomal storage disease).

A Phase I/II Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects with Fabry Disease.

EU CTIS ID: 2024-512695-34-00

What this study is testing

To assess the safety and tolerability of ST-920.

  • Phase I and Phase II (Integrated)- First administration to humans

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

  • 1. ≥ 18 years of age.
  • 5. Subjects who are on ERT or are ERT-naïve or are ERT-pseudo-naïve. For subjects receiving ERT, ERT must have been administered at a stable dose and regimen for at least 6 months (defined as not having missed more than 3 doses of ERT during the 6 months prior to consent).
  • 6. Male subjects must agree to use an effective form of contraception and refrain from sperm donation from the time of ST-920 administration until a minimum of 3 consecutive semen samples are negative for AAV2/6 after administration of ST-920 and a minimum of 90 days after ST-920 administration. Sexual abstinence is acceptable only as true abstinence and when in line with the preferred and usual lifestyle of the subject. Periodic abstinence and withdrawal are not acceptable methods of contraception.
  • 7. Female subjects must refrain from egg donation from the time of ST-920 administration until all the samples are negative for AAV2/6 after administration of ST-920
  • 8. Female subjects from menarche until becoming post-menopausal or permanently sterile must have a negative serum pregnancy test at screening and must agree to use a highly effective contraception method from screening through the end of the study.
  • 9. Subject must be fully vaccinated (as per the Centers for Disease Control and Prevention (CDC) definition in the US and as per local guidelines in other countries) for COVID-19 at least one month prior to dosing.

You likely can't join if

  • 1. Current treatment with migalastat (Galafold®).
  • 18. Any other reason that, in the opinion of the Site Investigator or Medical Monitor, would render the subject unsuitable for participation in the study, including but not limited to risk of COVID-19 infection.
  • 19. Additional exclusion criteria for Cohorts 1- 4, anti α-Gal A Ab positive and negative, female and renal cohort: Subjects who meet New York Heart Association (NYHA) Class III and IV.
  • 2. Positive neutralizing antibodies to AAV6.
  • 20. Additional exclusion criteria for Renal cohort: History of renal dialysis or transplantation.
  • 21. Additional exclusion criteria for Renal cohort: History of acute kidney insufficiency in the 6 months prior to screening.
See the full eligibility criteria
Who can join
  • 1. ≥ 18 years of age.
  • 5. Subjects who are on ERT or are ERT-naïve or are ERT-pseudo-naïve. For subjects receiving ERT, ERT must have been administered at a stable dose and regimen for at least 6 months (defined as not having missed more than 3 doses of ERT during the 6 months prior to consent).
  • 6. Male subjects must agree to use an effective form of contraception and refrain from sperm donation from the time of ST-920 administration until a minimum of 3 consecutive semen samples are negative for AAV2/6 after administration of ST-920 and a minimum of 90 days after ST-920 administration. Sexual abstinence is acceptable only as true abstinence and when in line with the preferred and usual lifestyle of the subject. Periodic abstinence and withdrawal are not acceptable methods of contraception.
  • 7. Female subjects must refrain from egg donation from the time of ST-920 administration until all the samples are negative for AAV2/6 after administration of ST-920
  • 8. Female subjects from menarche until becoming post-menopausal or permanently sterile must have a negative serum pregnancy test at screening and must agree to use a highly effective contraception method from screening through the end of the study.
  • 9. Subject must be fully vaccinated (as per the Centers for Disease Control and Prevention (CDC) definition in the US and as per local guidelines in other countries) for COVID-19 at least one month prior to dosing.
  • 10. Additional inclusion criteria for Cohorts 1-4b, anti α-Gal A Ab positive and negative: Male subjects with classical Fabry disease as defined by <5% α-Gal A activity in either plasma or leukocytes.
  • 11. Additional inclusion criteria for Female cohort: Female subjects with a documented mutation that is indicative of classical Fabry (i.e., listed in a database, such as http://dbfgp.org) and treatment (ERT) is clinically indicated.
  • 12. Additional inclusion criteria for Renal and Cardiac cohorts: Symptomatic Fabry disease defined for male subjects by <30% α-Gal A activity in either plasma or leukocytes and for female subjects based on genetic test results consistent with Fabry pathogenic mutation, or in the case of novel mutations a firstdegree male family member with Fabry disease with the same mutation.
  • 13. Additional inclusion criteria for Renal cohort: Screening eGFR value between 40-90 mL/min/1.73 m².
  • 14. Additional inclusion criteria for Renal cohort: Linear negative eGFR slope.
  • 2. Signed, written informed consent.
  • 3. Diagnosis of Fabry disease.
  • 4. One or more of the following symptoms: i) cornea verticillata, ii) acroparesthesia, iii) anhidrosis, iv) angiokeratoma.
What rules you out
  • 1. Current treatment with migalastat (Galafold®).
  • 18. Any other reason that, in the opinion of the Site Investigator or Medical Monitor, would render the subject unsuitable for participation in the study, including but not limited to risk of COVID-19 infection.
  • 19. Additional exclusion criteria for Cohorts 1- 4, anti α-Gal A Ab positive and negative, female and renal cohort: Subjects who meet New York Heart Association (NYHA) Class III and IV.
  • 2. Positive neutralizing antibodies to AAV6.
  • 20. Additional exclusion criteria for Renal cohort: History of renal dialysis or transplantation.
  • 21. Additional exclusion criteria for Renal cohort: History of acute kidney insufficiency in the 6 months prior to screening.
  • 22. Additional exclusion criteria for Renal cohort: Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated within 4 weeks prior to screening or changed ACE inhibitor or ARB dose in the 4 weeks prior to screening.
  • 23. Additional exclusion criteria for Renal cohort: Urine protein to creatinine ratio (UPCR) > 0.5 g/g who are not being treated with an ACE inhibitor or ARB.
  • 24. Additional exclusion criteria for Cardiac cohort: Significant cardiac fibrosis.
  • 25. Additional exclusion criteria for Cardiac cohort: Any contraindications to Cardiovascular magnetic resonance (CMR) as per local hospital/institution guidelines.
  • 26. Additional exclusion criteria for Cardiac cohort: Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated within 4 weeks prior to screening or changed ACE inhibitor or ARB dose in the 4 weeks prior to screening.
  • 10. One or more of the following: a. Albumin ≤ 3.5 g/dL b. Total bilirubin > upper limit of normal (ULN) and direct bilirubin ≥ 0.5 mg/dL c. Alkaline phosphatase (ALP) > 2.0 x ULN d. Alanine aminotransferase (ALT) > 1.5 x ULN
  • 3. Intercurrent illness expected to impair evaluation of safety or efficacy during the observation period of the study.
  • 4. Estimated glomerular filtration rate <40 mL/min/1.73m2.
  • 5. Active infection with hepatitis A virus (HAV ribonucleic acid [RNA] positive), active or occult hepatitis B virus infection (positive HBV-DNA or anti-HBc positive), active infection with hepatitis C virus (HCV RNA positive), infection with the human immunodeficiency virus (HIV) as measured by quantitative polymerase chain reaction (qPCR), or active or latent infection with tuberculosis (TB) measured by QuantiFERON® test.
  • 6. Breastfeeding at screening or breastfeeding during required period of contraception.
  • 7. History of liver disease such as clinically significant steatosis, fibrosis, non-alcoholic steatohepatitis (NASH) and cirrhosis, biliary disease within 6 months of informed consent; except for Gilbert’s syndrome.
  • 8. Elevated circulating serum Alpha fetoprotein (AFP).
  • 9. For subjects receiving ERT, recent or recurrent hypersensitivity reaction manifested by significant infusion reaction to ERT treatment within 6 months prior to consent.
  • 27. Additional exclusion criteria for Cardiac cohort: New York Heart Association Class IV.
  • 11. Current or history of systemic IV or oral immunomodulatory agents, or biologics or steroid use in the past 6 months prior to consent (topical and inhaled treatment are allowed, [e.g., for asthma or eczema]). Occasional use of systemic steroid may be allowed based on discussion and agreement with the Medical Monitor.
  • 12. Contraindication to use of corticosteroids.
  • 13. History of malignancy, except for non-melanoma skin cancer and localized prostate cancer treated with curative intent.
  • 14. Recent history of alcohol or substance abuse. The use of marijuana may be considered on an individual basis with discussion and agreement from the Medical Monitor.
  • 15. Participation in investigational interventional drug or medical device study throughout the duration of this study and within the last 3 months prior to consent (with the exception of implantable loop recorders as in the RaILRoAD trial).
  • 16. Prior treatment with a gene therapy product.
  • 17. Known hypersensitivity to components of ST-920 formulation.

The study team makes the final eligibility decision.

Where it's taking place

  • United States
  • Canada
  • Taiwan
  • Australia
  • United Kingdom

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling male, 18-64 years. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include United States; Canada; Taiwan; Australia; United Kingdom. Enter your location above to see the nearest site and check your eligibility.

Explore other conditions

ObesityType 2 diabetesType 1 diabetesPrediabetesWeight lossMetabolic syndromeHigh cholesterolHigh triglyceridesHigh blood pressurePCOSHypothyroidismThyroid diseaseGoutFatty liver disease (NAFLD/NASH)Heart failureCoronary artery disease

BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.