Actively Recruiting
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
Led by UniQure Biopharma B.V. · Updated on 2025-10-23
12
Participants Needed
8
Research Sites
360 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The main goals of this clinical study are to characterize safety and PK/PD of AMT-191 i.e. if drug doses used in the study are safe and tolerable and to understand how it acts in the body of people with Fabry disease.
CONDITIONS
Official Title
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male aged 18 to 50 years
- Confirmed diagnosis of classic Fabry disease defined by either absent or minimal alpha-galactosidase A enzyme activity or a genetic variant associated with classic Fabry disease and low enzyme activity
- Estimated glomerular filtration rate (eGFR) of 40 mL/min/1.73 m2 or higher
- Suboptimal response after at least 12 months of enzyme replacement therapy, including plasma lyso-Gb3 level of 2.3 ng/mL or higher and either persistent moderate or severe neuropathic pain for at least 3 months or moderate to severe gastrointestinal symptoms occurring two or more times in the prior 12 weeks
- Weight of 120 kilograms or less
You will not qualify if you...
- Allergic reaction to enzyme replacement therapy or infusion reaction within 12 months prior to consent that was severe and required emergency treatment
- Proteinuria with urine protein/creatinine ratio of 1 mg/mg or higher at screening
- Current use of chaperone therapy such as migalastat
- History of cancer within 5 years except basal or squamous cell skin cancer
- Active or latent hepatitis B or C, HIV, or tuberculosis infection
- Significant uncontrolled medical conditions including renal, liver, heart, blood, gastrointestinal, endocrine, lung, neurological, psychiatric diseases, alcoholism, drug dependency, or psychological disorders impacting safety or study adherence
- Liver disease such as hepatitis, fibrosis, cirrhosis, or tumor
- History of kidney transplant or current dialysis treatment
- Uncontrolled hypertension confirmed by repeated measurements
- Glycated hemoglobin (HbA1c) of 7% or higher at screening
- Contraindications to systemic corticosteroid or immunosuppressive therapy
- Chronic steroid use of 3 months or more within 12 months prior to screening
- Abnormal liver or kidney function laboratory values exceeding twice the upper limit of normal
- Low hemoglobin, low platelet count, elevated INR, or elevated soluble terminal complement complex
- Significant kidney abnormalities on ultrasound such as single kidney, large size difference, or cysts
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 8 locations
1
The Kirklin Clinic Of university of Alabama Birmingham Hospital
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
Emory University School of Medicine
Atlanta, Georgia, United States, 30322
Actively Recruiting
3
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
4
MHealth Fairview University of Minnesota Medical Center East Bank
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
5
NYC Health + Hospitals/Metropolitan
New York, New York, United States, 10029
Actively Recruiting
6
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
7
University of Utah, Clinical and Translational Sciences Institute
Salt Lake City, Utah, United States, 84108
Actively Recruiting
8
Lysosomal & Rare Disorders Research and Treatment Center, Inc
Fairfax, Virginia, United States, 22030
Actively Recruiting
Research Team
U
uniQure
CONTACT
C
Christy Quintana
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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