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Tests treatment safety and results for Fabry Disease
Official title Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
ClinicalTrials.gov ID: NCT06270316
What this study is testing
What is AMT-191?
AMT-191 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for fabry disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 50, men only
You may be able to join if
- Male of age ≥ 18 years and ≤50 years
- Confirmed clinical diagnosis of classic Fabry disease (FD) defined as:
- Absent or minimal αGAL A enzyme activity \< 1% of mean normal measured in plasma regardless of variant status; OR
- α-galactosidase A (GLA) pathogenic or likely pathogenic variant associated with classic FD phenotype identified on molecular genetic testing with...
- eGFR ≥ 40 mL/min/1.73 m2
You likely can't join if
- 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...
- 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...
- Active or ongoing infection or any other significant concurrent, uncontrolled medical condition including, but not limited to, renal, hepatic...
See the full eligibility criteria
- Male of age ≥ 18 years and ≤50 years
- Confirmed clinical diagnosis of classic Fabry disease (FD) defined as:
- Absent or minimal αGAL A enzyme activity \< 1% of mean normal measured in plasma regardless of variant status; OR
- α-galactosidase A (GLA) pathogenic or likely pathogenic variant associated with classic FD phenotype identified on molecular genetic testing with plasma αGLA A enzyme activity below lower bound of the reference range...
- 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...
- 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...
- Weight ≤ 120 kilograms (kg) Key
- 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...
- 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...
- 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...
- 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...
- 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...
- 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:
- Alanine transaminase (ALT) \> 2 x upper limit of normal for the testing laboratory (ULN)
- Aspartate aminotransferase (AST) \> 2 x ULN
- Total Bilirubin \> 2 x ULN (except if this is caused by Gilbert disease)
- Alkaline phosphatase (ALP) \> 2 x ULN
- Creatinine \> 2 x ULN
- Screening laboratory values for hematologic and coagulation function that meet any of the following:
- Hemoglobin \< lower limit of normal (LLN) (as per reference laboratory ranges)
- Platelet count \< 150 x1000/μl
- International normalized ratio (INR) \>1.1
- Soluble terminal complement complex (sC5b-9)\>ULN
- 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...
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- Atlanta, Georgia, United States
- Chicago, Illinois, United States
- Minneapolis, Minnesota, United States
- New York, New York, United States
- Pittsburgh, Pennsylvania, United States
- Salt Lake City, Utah, United States
- Fairfax, Virginia, United States
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 years to 50 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; Atlanta, Georgia, United States; Chicago, Illinois, United States; Minneapolis, Minnesota, United States; New York, New York, United States; Pittsburgh, Pennsylvania, United States and 2 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.