Study of AMT-191 for Classic Fabry Disease
This study is investigating AMT-191, a gene therapy delivered through a single intravenous (IV) infusion, for men with classic Fabry disease. Fabry disease is caused by a missing or deficient enzyme called alpha-galactosidase A (αGAL A). AMT-191 is designed to help the body produce this enzyme. The main goals are to understand if different doses of AMT-191 are safe and tolerable, and how the treatment works in the body. You may be eligible if you are a male between 18 and 50 years old with a confirmed diagnosis of classic Fabry disease, meaning you have very low αGAL A enzyme activity or a specific genetic change. The study plans to enroll 12 participants.
- Study design
- This is a first-in-human study where all 12 eligible participants will receive AMT-191 at one of two or more dose levels; there is no placebo.
- What's involved
- Participants will be monitored through study site visits, blood tests, imaging, questionnaires, and other assessments.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be monitored for safety and tolerability for 60 months after receiving the treatment.
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NCT06270316
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
Recruiting
PHASE1Ages 18–50InterventionalTreatmentUniQure Biopharma B.V.
~12 participants
Updated 2025-10-23 on ClinicalTrials.gov
What's tested:AMT-191
At a glance
Recruiting sites
8 of 8 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Evaluate the safety and tolerability of different dose levels of intravenously-administered AMT-191 in Participants with FD
Measured over 60 Months
+1 more outcome measured
Conditions
Where it's being run
8 sites across 8 statesAlabama1
Georgia1
Illinois1
Minnesota1
New York1
Pennsylvania1
Utah1
Virginia1
Study leadership
- Arian Pano, MD, MPH · STUDY_DIRECTOR · Clinical Development and Progam Lead, uniQure Biopharma, B.V.
Who to contact
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Male of age ≥ 18 years and ≤50 years
Confirmed clinical diagnosis of classic Fabry disease (FD) defined as:
eGFR ≥ 40 mL/min/1.73 m2
Suboptimal response after at least 12 months of enzyme replacement therapy (ERT) treatment. Suboptimal response is defined as plasma lyso-Gb3 ≥ 2.3 nanograms per milliliter (ng/mL) at Screening and one or both of the following:
Persistent moderate or severe neuropathic pain (intermittent or continuous) over a period of at least 3 months prior to consent
Presence of gastrointestinal symptoms (abdominal cramping, constipation, or diarrhea), reported by the Participant as moderate or severe and that are either persistent or occurring two or more times over the 12 weeks prior to consent
Weight ≤ 120 kilograms (kg)
Exclusion
Any allergic hypersensitivity reaction to ERT or infusion reaction in the 12 months prior to consent that was of severity grade 3 or above based on Common Terminology Criteria for Adverse Events (CTCAE v5.0) and required emergency intervention for hypertension/hypotension to stabilize blood pressure or hypoxia OR any other life-threatening complication.
Proteinuria, with random urine protein/creatinine ratio (rUPCR) ≥1 mg/mg at Screening
Current use of chaperone therapy such as migalastat (Galafold®)
Malignancy within 5 years of Screening, except for basal or squamous cell carcinoma of the skin
Presence of chronic, active, or latent infection with hepatitis B or C, human immunodeficiency virus (HIV), or tuberculosis (TB) as assessed at the screening visit
Active or ongoing infection or any other significant concurrent, uncontrolled medical condition including, but not limited to, renal, hepatic, cardiovascular, hematological, GI, endocrine (such as diabetes mellitus with poor glycemic control), pulmonary, neurological, cerebral, or psychiatric disease, alcoholism, drug dependency, or any other psychological disorder that could, in the opinion of the Investigator, risk the safety of the Participant, or interfere with adherence to the protocol procedures or interpretation of results
Evidence of any liver disease, including hepatitis, fibrosis, cirrhosis of the liver, neoplastic lesion, or any known medical condition that could impact the intended transduction of the vector and/or expression and activity of the protein
History of kidney transplantation or currently on hemodialysis or peritoneal dialysis
Uncontrolled hypertension, defined as systolic blood pressure \>140 millimeters of mercury (mmHg) (inclusive) and/or diastolic blood pressure outside the range of 60 to 85 mmHg (inclusive) at Screening, confirmed on at least 2 repeated measurements
Patients taking blood pressure medication to control blood pressure or proteinuria (eg, angiotensin-converting enzyme \[ACE\] inhibitors and angiotensin II receptor blockers \[ARBs\]) and have been titrated to a stable dose for at least 3 months prior to Screening are allowed in the study.
Glycated hemoglobin (HbA1c) at Screening ≥7%
Contraindication to systemic corticosteroid therapy or immunosuppressive therapy
Chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the 12 months prior to Screening
Screening laboratory values for renal and liver function that meet or exceed any of the following:
Screening laboratory values for hematologic and coagulation function that meet any of the following:
Significant anatomical abnormalities on renal ultrasound such as the presence of only 1 kidney, significant differences in kidney sizes between the right and left kidneys \>1.5 centimeters (about 0.59 inch), or presence of kidney cysts
What this trial measures
- Evaluate the safety and tolerability of different dose levels of intravenously-administered AMT-191 in Participants with FD60 Months
- Incidence of Treatment-Emergent Adverse Events (TEAE)60 Months