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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

No Placebo-Only Group

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 below

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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:

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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

This trial's timeline: 3 weeks for screening, Varies for treatment, and 26 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Duration of Vector deoxyribonucleic acid (DNA) shedding presented in blood, saliva, feces, semen, and urine

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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)

0 / 15Eligibility criteria met