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Enzyme Replacement Therapy
AMT-191 for Fabry Disease
Phase 1 & 2
Recruiting
Research Sponsored by UniQure Biopharma B.V.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Confirmed clinical diagnosis of classic Fabry disease (FD) with specific criteria
Suboptimal response after at least 12 months of enzyme replacement therapy (ERT) treatment with specific criteria
Must not have
Uncontrolled allergic conditions or allergy/hypersensitivity to AMT-191 excipients
Malignancy within 5 years of Screening except for specific types
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 26 months
Awards & highlights
Summary
"This trial is studying the safety and effectiveness of a single dose of AMT-191 given intravenously. They will test two different doses on groups of 3-6 participants each and monitor them
Who is the study for?
This trial is for men aged 18-50 with classic Fabry Disease, showing minimal GLA enzyme activity or a specific genetic variant. Participants must have moderate to severe gastrointestinal symptoms and persistent neuropathic pain. They should have an eGFR indicating moderate kidney function and weigh ≤80 kg. Candidates must agree to vaccination requirements and use condoms for 18 months post-dosing.
What is being tested?
The study tests AMT-191's safety, how it affects the body (PK/PD), and potential benefits in treating Fabry Disease over two years. It involves two groups receiving different doses of AMT-191 intravenously while continuing their regular enzyme replacement therapy.
What are the potential side effects?
Specific side effects are not listed but will be monitored throughout the study following administration of AMT-191. These may include reactions related to infusion, changes due to pharmacokinetics/pharmacodynamics assessments, or other unforeseen responses.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been diagnosed with classic Fabry disease.
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I have been on enzyme replacement therapy for over a year with limited improvement.
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I weigh 80 kg or less.
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I am a man between 18 and 49 years old.
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I have received or agree to receive a meningococcal vaccine 6 weeks before starting AMT-191.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I am allergic to some ingredients in AMT-191.
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I haven't had any cancer except for certain types in the last 5 years.
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I have had gene therapy before.
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I am currently taking miglastat (Galafold®).
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I do not have liver disease affecting gene therapy.
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I have had a kidney transplant or am currently on dialysis.
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I cannot take systemic steroids or immunosuppressive drugs.
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My kidney and liver tests are not normal.
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My kidney ultrasound shows major abnormalities.
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I have a serious heart condition.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 26 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~26 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Duration of Vector deoxyribonucleic acid (DNA) shedding presented in blood, saliva, feces, semen, and urine
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Trial Design
2Treatment groups
Experimental Treatment
Group I: Expansion Dose CohortsExperimental Treatment1 Intervention
Dose level 1: 6.0 x 10e13 gc/kg - Additional 3 if needed to assess dose-limiting toxicity
Dose level 2: 3.0 x 10e14 gc/kg - Additional 3 if needed to assess dose-limiting toxicity
Group II: Dose Escalating Low and High CohortsExperimental Treatment1 Intervention
Dose level 1: 6.0 x 10e13 gc/kg - 3 Participants (plus additional 3 if needed to assess dose-limiting toxicity)
Dose level 2: 3.0 x 10e14 gc/kg - 3 Participants (plus additional 3 if needed to assess dose-limiting toxicity)
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Who is running the clinical trial?
UniQure Biopharma B.V.Lead Sponsor
10 Previous Clinical Trials
240 Total Patients Enrolled
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