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Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

StatusRecruiting
PhasePhase 1/2
Started2024-06-05
View on ClinicalTrials.gov ↗
Trial flagged as At Risk
Primary endpoint removed 2 → 1 primary endpoints
See other at-risk trials from UniQure Biopharma B.V.

Amendment history

2026-09-25
Primary endpoint(s) modified2 to 1 entries
WasEvaluate the safety and tolerability of different dose levels of intravenously-administered AMT-191 in Participants with FD, Incidence of Treatment-Emergent Adverse Events (TEAE)
NowEvaluate the safety and tolerability of different dose levels of intravenously-administered AMT-191 in Participants with FD
Enrollment increased: 12 → 20 participants
Completion pushed: 2031-04-30 → 2031-11-30
2025-10-21
Study Identification, Study Status, Arms and Interventions, Outcome Measures v9
Completion date2027-12-01→2031-04-30
Secondary endpointsdetails revised at 1 of 1 entry
Primary endpoints2 entries, revised
Evaluate the safety and tolerability of different dose levels of intravenously-administered AMT-191 inPparticipantsin Participants with FD
2025-07-24
Study Status, Study Description, Arms and Interventions, Outcome Measures, Eligibility, Contacts/Locations v8
Primary completion date2027-04-01→2027-12-01
Completion date2027-04-29→2027-12-01
Study arms2→3
Eligibility criteria-1,761 characters
Key Inclusion Criteria: Male of age ≥ 18 years and ≤50 years Con [...] bsent or minimal αGAL A enzyme activity < 1% of mean normal (Vardarli, 2020) measured in isolated peripheral leukocytesplasma regardless of variant status; OR Galactosidaseα-galactosidase A gene (GLA) pathogenic or likely pathogenic variant associated wi [...] classic FD phenotype identified on molecular genetic testing with plasma αGLA A enzyme activity below lower bound of the reference range (as measured at trough enzyme replacement therapy [ERT] levels). eGFR ≥ 40 mL/min/1.73 m2 Participant agrees to use a condom during sexual interc [...] [...]
Secondary endpoints+70 characters
Characterize the vector shedding of intravenously-administered AMT-191
Primary endpoints2 entries, revised
DurationEvaluate the safety and tolerability of Vectordifferent deoxyribonucleicdose acidlevels (DNA)of sheddingintravenously-administered presentedAMT-191 ininPparticipants blood,with saliva, feces, semen, and urineFD Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability](TEAE)
Study description+467 characters
In Fabry disease, the enzyme α-galactosidase A is deficient. AMT-191 is an investigational gene therapy that encodes a re [...] eno-associated viral vector (rAAV5). AMT-191 is designed to delivertarget athe liver-specific expressionfor production of the transgeneenzyme coding for human aα-galactosidase A gene(αGAL). AMT-191 (GLA)is to Fabry patientsdelivered via a single (one-time) intravenous (IV) infusion. DeliveryIn this first-in-human study of AMT-191, totwo theor systemicmore circulationdose islevels expectedwill tobe resulttested. All ineligible aparticipants therapeuticwill effectreceive byAMT [...] [...]
2025-04-10
Study Status, Oversight, Contacts/Locations, IPDSharing v7
Study sites5→8
2025-02-25
Study Status, Contacts/Locations v6
Study sites2→5
Show 5 earlier versions
2024-10-28
Study Status, Contacts/Locations v5
Study sites1→2
2024-08-22
Study Status, Study Description, Arms and Interventions, Eligibility v4
Eligibility criteria+614 characters
Inclusion Criteria: Male of age ≥ 18 years and < ≤50 years Confirmed clinical diagnosis of classic Fabry disease (FD) defined as: Absent or minimal GLAαGAL A enzyme activity < 1% of the testing laboratory'smean normal reference(Vardarli, level2020) measured in isolated peripheral leukocytes regardless of va [...] FD phenotype identified on molecular genetic testing eGFR ≥ 4540 mL/min/1.73 m2 and ≤ 75 mL/min/1.73m2 Participant agrees thatto foruse a condom during sexual intercourse until semen shedding samples have tested negative at 3 consecutive visits (expected not to exceed a period of at least 18 [...] [...]
Study description-323 characters
Fabry disease (FD)AMT-191 is a lysosomal storage disorder resulting from the absent or deficient activity of the lysosomal enzyme, α-galactosidase A (GLA). Despite availability of an enzymeinvestigational replacementgene therapy (ERT)that toencodes prevent the progression of the renal, cardiac, and cerebrovascular symptoms, ERT is not curative and has variable impact on the disease progression for patients. Aa recombinant serotype 5 based adeno-associated viral vector (rAAV5). AMT-191) foris one-timedesigned intravenousto (IV) administration will be investigated in this study. This recombinant [...] [...]
2024-07-17
Study Status, Contacts/Locations v3
Start dateestimated 2024-05-31→confirmed 2024-06-05
2024-05-16
Not Yet Recruiting→Recruiting Study Status, Contacts/Locations v2
Trial statusNot Yet Recruiting→Recruiting
Start date2024-05-27→2024-05-31
Study sites0→1
2024-05-09
Study Status v1
Start date2024-03-15→2024-05-27
2024-02-20
Original filing
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.
Trial Details
NCT Number NCT06270316
Lead Sponsor UniQure Biopharma B.V.
Conditions Fabry Disease
Enrollment 20 participants
Start Date 2024-06-05
Primary Completion 2027-12-01 (estimated)
Study Completion 2031-11-30 (estimated)
Updated on ClinicalTrials.gov 2026-09-24